transformational therapies to treat lung infections ... presentation.pdf · we have never generated...
TRANSCRIPT
March 2019
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
Forward Looking Statement
This presentation is for confidential and informational purposes only This presentation shall not constitute an offer tosell or the solicitation of an offer to sell or the solicitation of an offer to buy anysecurities of AIT Therapeutics Inc (the ldquoCompanyrdquo) nor shall there be any sale of securities in any jurisdiction in which such offer solicitation or sale would be unlawful prior to the registration orqualification under the securities laws of any such jurisdiction
This presentation may not be reproduced photocopied redistributed published or used for any purpose other than for the recipientrsquos information Each recipient of this presentation agrees that allinformation contained herein is of a confidential material non-public nature that it will treat such information in a confidential manner and that it will not directly or indirectly use or disclose orpermit its agents oraffiliates to use or disclose anysuch information without our priorwrittenconsent
The Company files annual quarterly and other reports with the Securities and Exchange Commission (the ldquoSECrdquo) including its Annual Report on Form 10-K for the year ended December 31 2016 (theldquoForm 10-Krdquo) which was filed on March 31 2017 You may get these documents for free by visiting EDGAR on the SECrsquos website at wwwsecgov For a more complete discussion of the risk factorsaffecting our business please refer to the Form 10-K
This presentation includes statements that are or may be deemed lsquolsquoforward-looking statementsrsquorsquo In some cases these forward-looking statements can be identified by the use of forward-lookingterminology including the terms ldquobelievesrdquo ldquoestimatesrdquo ldquoanticipatesrdquo ldquotargetsrdquo ldquoexpectsrdquo ldquoplansrdquo ldquoprojectsrdquo ldquointendsrdquo ldquopredictsrdquo ldquomayrdquo ldquocouldrdquo ldquomightrdquo ldquowillrdquo ldquoshouldrdquo ldquoapproximatelyrdquopotentialrdquo or in each case their negative orothervariations thereon orcomparable terminology although not all forward-looking statements contain these words
These statements appear in a number of places throughout this presentation and include statements regarding our intentions beliefs projections outlook analyses or current expectationsconcerning among other things the patient market size and market adoption of our products by physicians and patients the timing and cost of clinical trials for our products or whether such trialswill be conducted at all completion and receiving favorable results of clinical trials for our products the development andapproval of the use of nitric oxide for additional indications the use of theproceeds from this offering FDA approval of or other regulatory action with respect to the timing cost or other aspects of the commercial launch of our products and the commercial launch andfuture sales of ourproducts or any other future products or product candidates
By their nature forward-looking statements involve risks and uncertainties because they relate to events competitive dynamics and healthcare regulatory and scientific developments and dependon the economic circumstances that may or may not occur in the future ormay occur on longer orshorter timelines than anticipated orat all Although we believe that we have a reasonable basis foreach forward-looking statement contained in this presentation we caution you that forward-looking statements are not guarantees of future performance and that our actual results of operationsfinancial condition and liquidity and the development of the industry in which we operate may differ materially fromthe forward looking statements contained in this presentation
We have never generated any revenue from product sales and may never be profitable
We wi ll need to raise substantial additional funding before we can expect to become profitable from sales of our products
We are heavily dependent on the success of our product candidates which are in various stages of clinical development We ca nnot give any assurance that any of our
product candidates will receive regulatory approval which is necessary before they can be commercialized
Cl inical drug and medical device development involves a lengthy and expensive process with an uncertain outcome and results of earlier studies may not be predictive of
future s tudy results
Our del ivery system may be classified as a Class I II medical device by the FDA and require premarket approval (PMA) by the FD A which is a rigorous time-consuming and
expensive process
We rely on third parties to conduct our preclinical and clinical studies and perform other tasks for us If these third parties do not successfully carry out their contractual
duties meet expected deadlines or comply with regulatory requirements we may not be able to obtain regulatory approval for or commercialize our product candidates
and our business could be substantially harmed
The commercial success of any current or future product candidate will depend upon the degree of market acceptance by physicians patients third-party payers and
others in the medical community
If we are unable to obtain and maintain effective patent rights for our product candidates or any future product candidates we may not be able to compete effectively in
our markets
We manage our business through a small number of employees and key consultants We depend on them even more than similarly-situated companies
International expansion of our business exposes us to business regulatory political operational financial and economic ri sks associated with doing business outside of the
United States or Israel
Our main subsidiary with significant operations are located in Israel and therefore our results may be adversely affected b y political economic and military instability in
Is rael
Risk Factors
Risks associated with our business include but are not limited to the following
4
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
5
Nitric Oxide (NO) is Naturally Occurring in the Human Body
CELL PROLIFERATIONANGIOGENESIS
CARDIOVASCULAR HEMOSTASISIMMUNE RESPONSE
APOPTOSIS
NEUROTRANSMISSION
ANTIBACTERIAL
Bian K amp Murad F Nitric Oxide (2014) | Bodgan C Trends in Immunol (2015)
Nitric Oxide
6
First Indication Pulmonary Hypertension (PH) Overview
NO is an established therapeutic option for patients suffering from Pulmonary Hypertension worldwide
Life-threatening condition from increasedpulmonary vascular resistance resulting indecreased pulmonary blood flow
Generally not diagnosed until multi-organ system function is affected
NO is the de facto standard of care for PH in the hospital setting
(1) ldquoPediatric Pulmonary Hypertensionrdquo ndash Guidelines from the American heart Association and American Thoracic Society(2) Pulmonary Hypertension News ndash ldquoPulmonary Hypertension and Nitric Oxiderdquo(3) Pers istent Pulmonary Hypertension of the Newborn
NO has been used as a long-term therapeutic option for patients with pulmonary hypertension
Approved in the US by the FDA in 1999 forPPHN(3)
Approved in the EU in 2001 for PPHN(3) and cardiac surgery
Inhaled NO causes increase in the concentration level of intracellular Cyclic Guanosine Monophosphate (cGMP) and an activation of the soluble guanylate cyclase
Causes smooth muscle relaxation which increases blood flow to the lungs and decreases the workload on the right ventricle
Pulmonary Hypertension Overview Effects of Pulmonary Hypertension(1)
Benefits of NO in Treatment of PH(2)
Narrowing of the Pulmonary Arteries
Failure of Right Ventricle
7
Nitric Oxide US market
(1) MNK Company Reports(2) American Academy of Pediatrics American Hospital Association
Approved indication persistent pulmonary hypertension of the newborn (PPHN)
FY 2018 Mallinckrodt reported INOmax sales gt$500m1
Praxair expected to enter the market in 2019
o Praxair system is cylinder based like INOmax
o Anticipate rational price decline
AIT will expand the market
o ~800 hospitals have NO today1
ndash AITrsquos AirNOvent will allow NO use by hospitals unable to use a cylinder system
o gt 1000 NICUs in the US today2
o Increase use with a lower cost and ease of use vs cylinder systems
o Volume expansion with AirNOvent expected to offset price decline
The Bottom Line is that all the problems associated with NO cylinders disappear
TYPICAL NO CYLINDER PROFILE IN THE US
Height 45rdquo Diameter 75rdquo Weight ~45 lbs (Weightfor 2 cylinders on cart wdelivery system is ~175 lbs)
GENERATOR PROFILE
Height 14rdquo Width 17rdquo Depth 13rdquo Weight ~16 lbs(Weight on cart with back-up system is ~45 lbs)
8
For illustration purposes onlyFor investigational use only
AirNOvent Next generation NO care for patients worldwide
The next generation phasic flow nitricoxide delivery system The cylinder freesystem will be used for the treatment of pulmonary hypertension in the hospital setting
For investigational use only
Detachable unit
Backup switch
User interface
Ventilator Connectors
NO2 Filter
A i r N O v e n t tradeCylinder Free Nitric Oxide Therapeutic Platform
Ai rNOvent may not be the final commercial product name
Width ~24 inches
Depth ~ 28 inches
Height ~5 feet
Weight ~45 lbs
9
For illustration purposes onlyFor investigational use only
AirNOvent Next generation NO care for patients worldwide
A i r N O v e n t tradeCylinder Free Nitric Oxide Therapeutic Platform
Detachable Unit
User Interface
Ai rNOvent may not be the final commercial product name
10
Improved operating economics for the hospital
No significant capital investment required for hospitals new to NO
No burdensome inventory and storage requirements
NO supplied as a non-hypoxic gas mixture
No purging procedures or additional safety measures due to NO2 buildup
NO now available to hospitals unable to use NO cylinder systems today
Reduced training burden
Pregnant staff members not impacted
AIT does not have any expenses associated with a manufacturing facility for nitric oxide
AIT does not have any expenses associated with logistics related to nitric oxide cylinders
Hospitals will have significant cost amp logistics Advantages Our device will have significant cost Advantages
Losing the High-Pressure Cylinder is a Significant Gain
11
Circassia world-class specialty biopharma company backed by Astra Zeneca
SnapshotCircassia Pharmaceuticals plcStatus Public company traded on AIM CIR | Stock Price (02282019) GBP 35About Specialty pharmaceutical company founded in 2006 Focused on respiratory diseases based out of the UKIPO date Mar 2014Market Cap (02282019) GBP 130 M | Sales (1H 2018) GBP 284 MLoss (1H 2018) GBP 235 M | Cash in Hand (1H 2018) GBP 508 MCommercial Team US = ~200 | Total Employees ~400Major Shareholders Invesco Asset mgmt Woodford Investment mgmt AstraZeneca own about 67 combinedDirect Sales Force in United States China and certain European Countries
Area of Expertise Strategic Fit with AirNOvent
Respiratory NIOXo Used for asthma management
Tudorzao indicated for the long-term maintenance treatment of bronchospasm associated with COPD including
chronic bronchitis and emphysema
Duaklir (March 31 PDUFA)o Aclidinium bromide amp formoterol fumarate for treating COPD
Nitric Oxide NIOX is a nitric oxide measurement system for monitoring airway inflammation
AirNOvent will be a meaningful product
2018 Circassia company revenues expected to be $62m - $67m Two currently marketed products with a third expected later in 2019 Currently a small hospital presence Just rolling out commercial infrastructure in China
Exposure to US hospitals NIOX is detailed in the hospital and there is overlap in the US with top hospitals that use NO today
12
$3255 million in Total Milestones and 15-20 Royaltyo $105 million received to date
Royalties to AIT on Gross Profito5 on the first $50 million in the US (one time)o5 on the first $20 million in China (one time)o15 up to $100m annually (US amp China combined)o20 above $100m annually (US and China combined)oGross profit defined as net sales less only the cost of AirNOvent and
NO2 filters
PMA filing with FDA is anticipated in the Second Quarter 2019
US commercial launch planned First Half 2020
A Transforming Partnership ndash Transaction Details
Key Terms
High Concentration NO Delivery Opportunitiesbull Bronchiolitisbull Nontuberculous Mycobacteria (NTM)
14
Date Study Indication Primary Results
2011 Phase 1 Safety (n=10) All comers Safety bull No SAEs
2013 ndash 2014Phase 2 double blind randomized (n=43)
Bronchiolitis (all causes)
Safety amp Efficacy
bull No SAEsbull 24 hour reduction in hospital length of stay
2013 - 2014Phase 2 open label
(n=9)Cystic Fibrosis (CF)
Safety amp Efficacy
bull No SAEsbull Lowered bacterial load
2016Compassionate use Israel
(n=2)NTM in
CF patientsEfficacy
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2017Compassionate use National
Institute of Health (n=1)NTM in
CF patientEfficacy
bull No SAEsbull Improvements in clinical endpoints
2017 Pilot open label (N=9)Refractory NTM
abscessusSafety
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2018Pilot study double blind
randomized (n=67)Bronchiolitis (all causes)
Efficacybull No SAEsbull 23 hour reduction in hospital length of stay
Safety First ndash AITrsquos High Concentration NO Delivery for Lung Infections
Our Nitric Oxide Delivery System Has a Demonstrated Safety Record at a concentration of 160 ppm NO
Treatments administered patients Different clinicalsettings
Serious Adverse Events (SAEs)
related to NO
2100+ 85+ 7 0
15
Second Indication Bronchiolitis (BRO) Overview
Bronchiolitis is the leading cause of hospitalization for infants worldwide (1)
~150000 infant hospitalizations per year in the US(2)
Significant impact on the elderly from equivalent viral infections with 177000 hospitalizations per year in the US(3)
No drugs approved for the treatment of BRO patients(4)
Standard of care in the hospital is oxygen and hydration
(1) Scand J Trauma Resusc Emerg Med 2014 22 23 WHO(2) Pel letier et a l Direct medical costs of hospitalizations in the United States Pediatrics 2006(3) CDC (due to RSV only)(4) American Academy of Pediatrics
Bronchiolitis Overview amp Market Dynamics Market Size
AIT estimates US market size to be gt$2 Band projects global market to be similar size to the US market with no competition
AITrsquos goal would be to reduce length of hospitalization in infants
Elderly population trials to follow infants
16
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in length of hospital stay (LOS)
(1) httpsonlinelibrarywileycomdoiepdf101002ppul23905
2014 Trial Design and Highlights
Randomized Prospective Double-blind 43 patients (age 2-12 months) with acute
bronchiolitis (mostly due to RSV) and at least 36 weeks of gestation
N=22 Supportive Care (O₂ amp hydration) N=21 Supportive Care + 160 ppm NO for 30
minutes 5xday up to 5 days Follow up visits 2 3 amp 4 weeks post discharge Single center at Soroka University Medical
Center in Israel Data presented at ATS 2015 in an oral session Reduced length of hospital stay by ~24hrs in
patients who stayed in the hospital for at least 24 hours
No treatment related SAEs Improvements in composite endpoint
(modified Tal score) and O2 consistent with improvement in LOS
Published in the December 2017 Pediatric Pulmonology Journal(1)
17
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Design and Baseline Characteristics
Randomized 67 subjects at 6 sites in Israel with a 11 randomization between 160 ppm NO + supportive care (O2 + hydration) and supportive care alone
Subjects were 0-12 months old with acute bronchiolitis requiring hospitalization with at least 28 weeks of gestation
PE (primary endpoint) the difference in hospital length of stay (LOS)
SE (secondary endpoint) time to clinical improvement using the Modified Tal score (score ge7 and lt10 to enroll le 5 is goal)
SE the difference in time to SpO2 of gt92 SE Safety (specifically NO2 levels and
methemoglobinemia) and Tolerability Treatment was five 30 minute sessions per
day not to exceed 25 treatments All inhalations delivered by airoxygen
blender +NO via a simple mask with a minimum FiO2 of 21
DATA PRESENTED AT THE SEPTEMBER 2018 EUROPEAN RESPIRATORY SOCIETY (ERS)
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
Forward Looking Statement
This presentation is for confidential and informational purposes only This presentation shall not constitute an offer tosell or the solicitation of an offer to sell or the solicitation of an offer to buy anysecurities of AIT Therapeutics Inc (the ldquoCompanyrdquo) nor shall there be any sale of securities in any jurisdiction in which such offer solicitation or sale would be unlawful prior to the registration orqualification under the securities laws of any such jurisdiction
This presentation may not be reproduced photocopied redistributed published or used for any purpose other than for the recipientrsquos information Each recipient of this presentation agrees that allinformation contained herein is of a confidential material non-public nature that it will treat such information in a confidential manner and that it will not directly or indirectly use or disclose orpermit its agents oraffiliates to use or disclose anysuch information without our priorwrittenconsent
The Company files annual quarterly and other reports with the Securities and Exchange Commission (the ldquoSECrdquo) including its Annual Report on Form 10-K for the year ended December 31 2016 (theldquoForm 10-Krdquo) which was filed on March 31 2017 You may get these documents for free by visiting EDGAR on the SECrsquos website at wwwsecgov For a more complete discussion of the risk factorsaffecting our business please refer to the Form 10-K
This presentation includes statements that are or may be deemed lsquolsquoforward-looking statementsrsquorsquo In some cases these forward-looking statements can be identified by the use of forward-lookingterminology including the terms ldquobelievesrdquo ldquoestimatesrdquo ldquoanticipatesrdquo ldquotargetsrdquo ldquoexpectsrdquo ldquoplansrdquo ldquoprojectsrdquo ldquointendsrdquo ldquopredictsrdquo ldquomayrdquo ldquocouldrdquo ldquomightrdquo ldquowillrdquo ldquoshouldrdquo ldquoapproximatelyrdquopotentialrdquo or in each case their negative orothervariations thereon orcomparable terminology although not all forward-looking statements contain these words
These statements appear in a number of places throughout this presentation and include statements regarding our intentions beliefs projections outlook analyses or current expectationsconcerning among other things the patient market size and market adoption of our products by physicians and patients the timing and cost of clinical trials for our products or whether such trialswill be conducted at all completion and receiving favorable results of clinical trials for our products the development andapproval of the use of nitric oxide for additional indications the use of theproceeds from this offering FDA approval of or other regulatory action with respect to the timing cost or other aspects of the commercial launch of our products and the commercial launch andfuture sales of ourproducts or any other future products or product candidates
By their nature forward-looking statements involve risks and uncertainties because they relate to events competitive dynamics and healthcare regulatory and scientific developments and dependon the economic circumstances that may or may not occur in the future ormay occur on longer orshorter timelines than anticipated orat all Although we believe that we have a reasonable basis foreach forward-looking statement contained in this presentation we caution you that forward-looking statements are not guarantees of future performance and that our actual results of operationsfinancial condition and liquidity and the development of the industry in which we operate may differ materially fromthe forward looking statements contained in this presentation
We have never generated any revenue from product sales and may never be profitable
We wi ll need to raise substantial additional funding before we can expect to become profitable from sales of our products
We are heavily dependent on the success of our product candidates which are in various stages of clinical development We ca nnot give any assurance that any of our
product candidates will receive regulatory approval which is necessary before they can be commercialized
Cl inical drug and medical device development involves a lengthy and expensive process with an uncertain outcome and results of earlier studies may not be predictive of
future s tudy results
Our del ivery system may be classified as a Class I II medical device by the FDA and require premarket approval (PMA) by the FD A which is a rigorous time-consuming and
expensive process
We rely on third parties to conduct our preclinical and clinical studies and perform other tasks for us If these third parties do not successfully carry out their contractual
duties meet expected deadlines or comply with regulatory requirements we may not be able to obtain regulatory approval for or commercialize our product candidates
and our business could be substantially harmed
The commercial success of any current or future product candidate will depend upon the degree of market acceptance by physicians patients third-party payers and
others in the medical community
If we are unable to obtain and maintain effective patent rights for our product candidates or any future product candidates we may not be able to compete effectively in
our markets
We manage our business through a small number of employees and key consultants We depend on them even more than similarly-situated companies
International expansion of our business exposes us to business regulatory political operational financial and economic ri sks associated with doing business outside of the
United States or Israel
Our main subsidiary with significant operations are located in Israel and therefore our results may be adversely affected b y political economic and military instability in
Is rael
Risk Factors
Risks associated with our business include but are not limited to the following
4
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
5
Nitric Oxide (NO) is Naturally Occurring in the Human Body
CELL PROLIFERATIONANGIOGENESIS
CARDIOVASCULAR HEMOSTASISIMMUNE RESPONSE
APOPTOSIS
NEUROTRANSMISSION
ANTIBACTERIAL
Bian K amp Murad F Nitric Oxide (2014) | Bodgan C Trends in Immunol (2015)
Nitric Oxide
6
First Indication Pulmonary Hypertension (PH) Overview
NO is an established therapeutic option for patients suffering from Pulmonary Hypertension worldwide
Life-threatening condition from increasedpulmonary vascular resistance resulting indecreased pulmonary blood flow
Generally not diagnosed until multi-organ system function is affected
NO is the de facto standard of care for PH in the hospital setting
(1) ldquoPediatric Pulmonary Hypertensionrdquo ndash Guidelines from the American heart Association and American Thoracic Society(2) Pulmonary Hypertension News ndash ldquoPulmonary Hypertension and Nitric Oxiderdquo(3) Pers istent Pulmonary Hypertension of the Newborn
NO has been used as a long-term therapeutic option for patients with pulmonary hypertension
Approved in the US by the FDA in 1999 forPPHN(3)
Approved in the EU in 2001 for PPHN(3) and cardiac surgery
Inhaled NO causes increase in the concentration level of intracellular Cyclic Guanosine Monophosphate (cGMP) and an activation of the soluble guanylate cyclase
Causes smooth muscle relaxation which increases blood flow to the lungs and decreases the workload on the right ventricle
Pulmonary Hypertension Overview Effects of Pulmonary Hypertension(1)
Benefits of NO in Treatment of PH(2)
Narrowing of the Pulmonary Arteries
Failure of Right Ventricle
7
Nitric Oxide US market
(1) MNK Company Reports(2) American Academy of Pediatrics American Hospital Association
Approved indication persistent pulmonary hypertension of the newborn (PPHN)
FY 2018 Mallinckrodt reported INOmax sales gt$500m1
Praxair expected to enter the market in 2019
o Praxair system is cylinder based like INOmax
o Anticipate rational price decline
AIT will expand the market
o ~800 hospitals have NO today1
ndash AITrsquos AirNOvent will allow NO use by hospitals unable to use a cylinder system
o gt 1000 NICUs in the US today2
o Increase use with a lower cost and ease of use vs cylinder systems
o Volume expansion with AirNOvent expected to offset price decline
The Bottom Line is that all the problems associated with NO cylinders disappear
TYPICAL NO CYLINDER PROFILE IN THE US
Height 45rdquo Diameter 75rdquo Weight ~45 lbs (Weightfor 2 cylinders on cart wdelivery system is ~175 lbs)
GENERATOR PROFILE
Height 14rdquo Width 17rdquo Depth 13rdquo Weight ~16 lbs(Weight on cart with back-up system is ~45 lbs)
8
For illustration purposes onlyFor investigational use only
AirNOvent Next generation NO care for patients worldwide
The next generation phasic flow nitricoxide delivery system The cylinder freesystem will be used for the treatment of pulmonary hypertension in the hospital setting
For investigational use only
Detachable unit
Backup switch
User interface
Ventilator Connectors
NO2 Filter
A i r N O v e n t tradeCylinder Free Nitric Oxide Therapeutic Platform
Ai rNOvent may not be the final commercial product name
Width ~24 inches
Depth ~ 28 inches
Height ~5 feet
Weight ~45 lbs
9
For illustration purposes onlyFor investigational use only
AirNOvent Next generation NO care for patients worldwide
A i r N O v e n t tradeCylinder Free Nitric Oxide Therapeutic Platform
Detachable Unit
User Interface
Ai rNOvent may not be the final commercial product name
10
Improved operating economics for the hospital
No significant capital investment required for hospitals new to NO
No burdensome inventory and storage requirements
NO supplied as a non-hypoxic gas mixture
No purging procedures or additional safety measures due to NO2 buildup
NO now available to hospitals unable to use NO cylinder systems today
Reduced training burden
Pregnant staff members not impacted
AIT does not have any expenses associated with a manufacturing facility for nitric oxide
AIT does not have any expenses associated with logistics related to nitric oxide cylinders
Hospitals will have significant cost amp logistics Advantages Our device will have significant cost Advantages
Losing the High-Pressure Cylinder is a Significant Gain
11
Circassia world-class specialty biopharma company backed by Astra Zeneca
SnapshotCircassia Pharmaceuticals plcStatus Public company traded on AIM CIR | Stock Price (02282019) GBP 35About Specialty pharmaceutical company founded in 2006 Focused on respiratory diseases based out of the UKIPO date Mar 2014Market Cap (02282019) GBP 130 M | Sales (1H 2018) GBP 284 MLoss (1H 2018) GBP 235 M | Cash in Hand (1H 2018) GBP 508 MCommercial Team US = ~200 | Total Employees ~400Major Shareholders Invesco Asset mgmt Woodford Investment mgmt AstraZeneca own about 67 combinedDirect Sales Force in United States China and certain European Countries
Area of Expertise Strategic Fit with AirNOvent
Respiratory NIOXo Used for asthma management
Tudorzao indicated for the long-term maintenance treatment of bronchospasm associated with COPD including
chronic bronchitis and emphysema
Duaklir (March 31 PDUFA)o Aclidinium bromide amp formoterol fumarate for treating COPD
Nitric Oxide NIOX is a nitric oxide measurement system for monitoring airway inflammation
AirNOvent will be a meaningful product
2018 Circassia company revenues expected to be $62m - $67m Two currently marketed products with a third expected later in 2019 Currently a small hospital presence Just rolling out commercial infrastructure in China
Exposure to US hospitals NIOX is detailed in the hospital and there is overlap in the US with top hospitals that use NO today
12
$3255 million in Total Milestones and 15-20 Royaltyo $105 million received to date
Royalties to AIT on Gross Profito5 on the first $50 million in the US (one time)o5 on the first $20 million in China (one time)o15 up to $100m annually (US amp China combined)o20 above $100m annually (US and China combined)oGross profit defined as net sales less only the cost of AirNOvent and
NO2 filters
PMA filing with FDA is anticipated in the Second Quarter 2019
US commercial launch planned First Half 2020
A Transforming Partnership ndash Transaction Details
Key Terms
High Concentration NO Delivery Opportunitiesbull Bronchiolitisbull Nontuberculous Mycobacteria (NTM)
14
Date Study Indication Primary Results
2011 Phase 1 Safety (n=10) All comers Safety bull No SAEs
2013 ndash 2014Phase 2 double blind randomized (n=43)
Bronchiolitis (all causes)
Safety amp Efficacy
bull No SAEsbull 24 hour reduction in hospital length of stay
2013 - 2014Phase 2 open label
(n=9)Cystic Fibrosis (CF)
Safety amp Efficacy
bull No SAEsbull Lowered bacterial load
2016Compassionate use Israel
(n=2)NTM in
CF patientsEfficacy
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2017Compassionate use National
Institute of Health (n=1)NTM in
CF patientEfficacy
bull No SAEsbull Improvements in clinical endpoints
2017 Pilot open label (N=9)Refractory NTM
abscessusSafety
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2018Pilot study double blind
randomized (n=67)Bronchiolitis (all causes)
Efficacybull No SAEsbull 23 hour reduction in hospital length of stay
Safety First ndash AITrsquos High Concentration NO Delivery for Lung Infections
Our Nitric Oxide Delivery System Has a Demonstrated Safety Record at a concentration of 160 ppm NO
Treatments administered patients Different clinicalsettings
Serious Adverse Events (SAEs)
related to NO
2100+ 85+ 7 0
15
Second Indication Bronchiolitis (BRO) Overview
Bronchiolitis is the leading cause of hospitalization for infants worldwide (1)
~150000 infant hospitalizations per year in the US(2)
Significant impact on the elderly from equivalent viral infections with 177000 hospitalizations per year in the US(3)
No drugs approved for the treatment of BRO patients(4)
Standard of care in the hospital is oxygen and hydration
(1) Scand J Trauma Resusc Emerg Med 2014 22 23 WHO(2) Pel letier et a l Direct medical costs of hospitalizations in the United States Pediatrics 2006(3) CDC (due to RSV only)(4) American Academy of Pediatrics
Bronchiolitis Overview amp Market Dynamics Market Size
AIT estimates US market size to be gt$2 Band projects global market to be similar size to the US market with no competition
AITrsquos goal would be to reduce length of hospitalization in infants
Elderly population trials to follow infants
16
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in length of hospital stay (LOS)
(1) httpsonlinelibrarywileycomdoiepdf101002ppul23905
2014 Trial Design and Highlights
Randomized Prospective Double-blind 43 patients (age 2-12 months) with acute
bronchiolitis (mostly due to RSV) and at least 36 weeks of gestation
N=22 Supportive Care (O₂ amp hydration) N=21 Supportive Care + 160 ppm NO for 30
minutes 5xday up to 5 days Follow up visits 2 3 amp 4 weeks post discharge Single center at Soroka University Medical
Center in Israel Data presented at ATS 2015 in an oral session Reduced length of hospital stay by ~24hrs in
patients who stayed in the hospital for at least 24 hours
No treatment related SAEs Improvements in composite endpoint
(modified Tal score) and O2 consistent with improvement in LOS
Published in the December 2017 Pediatric Pulmonology Journal(1)
17
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Design and Baseline Characteristics
Randomized 67 subjects at 6 sites in Israel with a 11 randomization between 160 ppm NO + supportive care (O2 + hydration) and supportive care alone
Subjects were 0-12 months old with acute bronchiolitis requiring hospitalization with at least 28 weeks of gestation
PE (primary endpoint) the difference in hospital length of stay (LOS)
SE (secondary endpoint) time to clinical improvement using the Modified Tal score (score ge7 and lt10 to enroll le 5 is goal)
SE the difference in time to SpO2 of gt92 SE Safety (specifically NO2 levels and
methemoglobinemia) and Tolerability Treatment was five 30 minute sessions per
day not to exceed 25 treatments All inhalations delivered by airoxygen
blender +NO via a simple mask with a minimum FiO2 of 21
DATA PRESENTED AT THE SEPTEMBER 2018 EUROPEAN RESPIRATORY SOCIETY (ERS)
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
We have never generated any revenue from product sales and may never be profitable
We wi ll need to raise substantial additional funding before we can expect to become profitable from sales of our products
We are heavily dependent on the success of our product candidates which are in various stages of clinical development We ca nnot give any assurance that any of our
product candidates will receive regulatory approval which is necessary before they can be commercialized
Cl inical drug and medical device development involves a lengthy and expensive process with an uncertain outcome and results of earlier studies may not be predictive of
future s tudy results
Our del ivery system may be classified as a Class I II medical device by the FDA and require premarket approval (PMA) by the FD A which is a rigorous time-consuming and
expensive process
We rely on third parties to conduct our preclinical and clinical studies and perform other tasks for us If these third parties do not successfully carry out their contractual
duties meet expected deadlines or comply with regulatory requirements we may not be able to obtain regulatory approval for or commercialize our product candidates
and our business could be substantially harmed
The commercial success of any current or future product candidate will depend upon the degree of market acceptance by physicians patients third-party payers and
others in the medical community
If we are unable to obtain and maintain effective patent rights for our product candidates or any future product candidates we may not be able to compete effectively in
our markets
We manage our business through a small number of employees and key consultants We depend on them even more than similarly-situated companies
International expansion of our business exposes us to business regulatory political operational financial and economic ri sks associated with doing business outside of the
United States or Israel
Our main subsidiary with significant operations are located in Israel and therefore our results may be adversely affected b y political economic and military instability in
Is rael
Risk Factors
Risks associated with our business include but are not limited to the following
4
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
5
Nitric Oxide (NO) is Naturally Occurring in the Human Body
CELL PROLIFERATIONANGIOGENESIS
CARDIOVASCULAR HEMOSTASISIMMUNE RESPONSE
APOPTOSIS
NEUROTRANSMISSION
ANTIBACTERIAL
Bian K amp Murad F Nitric Oxide (2014) | Bodgan C Trends in Immunol (2015)
Nitric Oxide
6
First Indication Pulmonary Hypertension (PH) Overview
NO is an established therapeutic option for patients suffering from Pulmonary Hypertension worldwide
Life-threatening condition from increasedpulmonary vascular resistance resulting indecreased pulmonary blood flow
Generally not diagnosed until multi-organ system function is affected
NO is the de facto standard of care for PH in the hospital setting
(1) ldquoPediatric Pulmonary Hypertensionrdquo ndash Guidelines from the American heart Association and American Thoracic Society(2) Pulmonary Hypertension News ndash ldquoPulmonary Hypertension and Nitric Oxiderdquo(3) Pers istent Pulmonary Hypertension of the Newborn
NO has been used as a long-term therapeutic option for patients with pulmonary hypertension
Approved in the US by the FDA in 1999 forPPHN(3)
Approved in the EU in 2001 for PPHN(3) and cardiac surgery
Inhaled NO causes increase in the concentration level of intracellular Cyclic Guanosine Monophosphate (cGMP) and an activation of the soluble guanylate cyclase
Causes smooth muscle relaxation which increases blood flow to the lungs and decreases the workload on the right ventricle
Pulmonary Hypertension Overview Effects of Pulmonary Hypertension(1)
Benefits of NO in Treatment of PH(2)
Narrowing of the Pulmonary Arteries
Failure of Right Ventricle
7
Nitric Oxide US market
(1) MNK Company Reports(2) American Academy of Pediatrics American Hospital Association
Approved indication persistent pulmonary hypertension of the newborn (PPHN)
FY 2018 Mallinckrodt reported INOmax sales gt$500m1
Praxair expected to enter the market in 2019
o Praxair system is cylinder based like INOmax
o Anticipate rational price decline
AIT will expand the market
o ~800 hospitals have NO today1
ndash AITrsquos AirNOvent will allow NO use by hospitals unable to use a cylinder system
o gt 1000 NICUs in the US today2
o Increase use with a lower cost and ease of use vs cylinder systems
o Volume expansion with AirNOvent expected to offset price decline
The Bottom Line is that all the problems associated with NO cylinders disappear
TYPICAL NO CYLINDER PROFILE IN THE US
Height 45rdquo Diameter 75rdquo Weight ~45 lbs (Weightfor 2 cylinders on cart wdelivery system is ~175 lbs)
GENERATOR PROFILE
Height 14rdquo Width 17rdquo Depth 13rdquo Weight ~16 lbs(Weight on cart with back-up system is ~45 lbs)
8
For illustration purposes onlyFor investigational use only
AirNOvent Next generation NO care for patients worldwide
The next generation phasic flow nitricoxide delivery system The cylinder freesystem will be used for the treatment of pulmonary hypertension in the hospital setting
For investigational use only
Detachable unit
Backup switch
User interface
Ventilator Connectors
NO2 Filter
A i r N O v e n t tradeCylinder Free Nitric Oxide Therapeutic Platform
Ai rNOvent may not be the final commercial product name
Width ~24 inches
Depth ~ 28 inches
Height ~5 feet
Weight ~45 lbs
9
For illustration purposes onlyFor investigational use only
AirNOvent Next generation NO care for patients worldwide
A i r N O v e n t tradeCylinder Free Nitric Oxide Therapeutic Platform
Detachable Unit
User Interface
Ai rNOvent may not be the final commercial product name
10
Improved operating economics for the hospital
No significant capital investment required for hospitals new to NO
No burdensome inventory and storage requirements
NO supplied as a non-hypoxic gas mixture
No purging procedures or additional safety measures due to NO2 buildup
NO now available to hospitals unable to use NO cylinder systems today
Reduced training burden
Pregnant staff members not impacted
AIT does not have any expenses associated with a manufacturing facility for nitric oxide
AIT does not have any expenses associated with logistics related to nitric oxide cylinders
Hospitals will have significant cost amp logistics Advantages Our device will have significant cost Advantages
Losing the High-Pressure Cylinder is a Significant Gain
11
Circassia world-class specialty biopharma company backed by Astra Zeneca
SnapshotCircassia Pharmaceuticals plcStatus Public company traded on AIM CIR | Stock Price (02282019) GBP 35About Specialty pharmaceutical company founded in 2006 Focused on respiratory diseases based out of the UKIPO date Mar 2014Market Cap (02282019) GBP 130 M | Sales (1H 2018) GBP 284 MLoss (1H 2018) GBP 235 M | Cash in Hand (1H 2018) GBP 508 MCommercial Team US = ~200 | Total Employees ~400Major Shareholders Invesco Asset mgmt Woodford Investment mgmt AstraZeneca own about 67 combinedDirect Sales Force in United States China and certain European Countries
Area of Expertise Strategic Fit with AirNOvent
Respiratory NIOXo Used for asthma management
Tudorzao indicated for the long-term maintenance treatment of bronchospasm associated with COPD including
chronic bronchitis and emphysema
Duaklir (March 31 PDUFA)o Aclidinium bromide amp formoterol fumarate for treating COPD
Nitric Oxide NIOX is a nitric oxide measurement system for monitoring airway inflammation
AirNOvent will be a meaningful product
2018 Circassia company revenues expected to be $62m - $67m Two currently marketed products with a third expected later in 2019 Currently a small hospital presence Just rolling out commercial infrastructure in China
Exposure to US hospitals NIOX is detailed in the hospital and there is overlap in the US with top hospitals that use NO today
12
$3255 million in Total Milestones and 15-20 Royaltyo $105 million received to date
Royalties to AIT on Gross Profito5 on the first $50 million in the US (one time)o5 on the first $20 million in China (one time)o15 up to $100m annually (US amp China combined)o20 above $100m annually (US and China combined)oGross profit defined as net sales less only the cost of AirNOvent and
NO2 filters
PMA filing with FDA is anticipated in the Second Quarter 2019
US commercial launch planned First Half 2020
A Transforming Partnership ndash Transaction Details
Key Terms
High Concentration NO Delivery Opportunitiesbull Bronchiolitisbull Nontuberculous Mycobacteria (NTM)
14
Date Study Indication Primary Results
2011 Phase 1 Safety (n=10) All comers Safety bull No SAEs
2013 ndash 2014Phase 2 double blind randomized (n=43)
Bronchiolitis (all causes)
Safety amp Efficacy
bull No SAEsbull 24 hour reduction in hospital length of stay
2013 - 2014Phase 2 open label
(n=9)Cystic Fibrosis (CF)
Safety amp Efficacy
bull No SAEsbull Lowered bacterial load
2016Compassionate use Israel
(n=2)NTM in
CF patientsEfficacy
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2017Compassionate use National
Institute of Health (n=1)NTM in
CF patientEfficacy
bull No SAEsbull Improvements in clinical endpoints
2017 Pilot open label (N=9)Refractory NTM
abscessusSafety
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2018Pilot study double blind
randomized (n=67)Bronchiolitis (all causes)
Efficacybull No SAEsbull 23 hour reduction in hospital length of stay
Safety First ndash AITrsquos High Concentration NO Delivery for Lung Infections
Our Nitric Oxide Delivery System Has a Demonstrated Safety Record at a concentration of 160 ppm NO
Treatments administered patients Different clinicalsettings
Serious Adverse Events (SAEs)
related to NO
2100+ 85+ 7 0
15
Second Indication Bronchiolitis (BRO) Overview
Bronchiolitis is the leading cause of hospitalization for infants worldwide (1)
~150000 infant hospitalizations per year in the US(2)
Significant impact on the elderly from equivalent viral infections with 177000 hospitalizations per year in the US(3)
No drugs approved for the treatment of BRO patients(4)
Standard of care in the hospital is oxygen and hydration
(1) Scand J Trauma Resusc Emerg Med 2014 22 23 WHO(2) Pel letier et a l Direct medical costs of hospitalizations in the United States Pediatrics 2006(3) CDC (due to RSV only)(4) American Academy of Pediatrics
Bronchiolitis Overview amp Market Dynamics Market Size
AIT estimates US market size to be gt$2 Band projects global market to be similar size to the US market with no competition
AITrsquos goal would be to reduce length of hospitalization in infants
Elderly population trials to follow infants
16
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in length of hospital stay (LOS)
(1) httpsonlinelibrarywileycomdoiepdf101002ppul23905
2014 Trial Design and Highlights
Randomized Prospective Double-blind 43 patients (age 2-12 months) with acute
bronchiolitis (mostly due to RSV) and at least 36 weeks of gestation
N=22 Supportive Care (O₂ amp hydration) N=21 Supportive Care + 160 ppm NO for 30
minutes 5xday up to 5 days Follow up visits 2 3 amp 4 weeks post discharge Single center at Soroka University Medical
Center in Israel Data presented at ATS 2015 in an oral session Reduced length of hospital stay by ~24hrs in
patients who stayed in the hospital for at least 24 hours
No treatment related SAEs Improvements in composite endpoint
(modified Tal score) and O2 consistent with improvement in LOS
Published in the December 2017 Pediatric Pulmonology Journal(1)
17
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Design and Baseline Characteristics
Randomized 67 subjects at 6 sites in Israel with a 11 randomization between 160 ppm NO + supportive care (O2 + hydration) and supportive care alone
Subjects were 0-12 months old with acute bronchiolitis requiring hospitalization with at least 28 weeks of gestation
PE (primary endpoint) the difference in hospital length of stay (LOS)
SE (secondary endpoint) time to clinical improvement using the Modified Tal score (score ge7 and lt10 to enroll le 5 is goal)
SE the difference in time to SpO2 of gt92 SE Safety (specifically NO2 levels and
methemoglobinemia) and Tolerability Treatment was five 30 minute sessions per
day not to exceed 25 treatments All inhalations delivered by airoxygen
blender +NO via a simple mask with a minimum FiO2 of 21
DATA PRESENTED AT THE SEPTEMBER 2018 EUROPEAN RESPIRATORY SOCIETY (ERS)
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
4
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
5
Nitric Oxide (NO) is Naturally Occurring in the Human Body
CELL PROLIFERATIONANGIOGENESIS
CARDIOVASCULAR HEMOSTASISIMMUNE RESPONSE
APOPTOSIS
NEUROTRANSMISSION
ANTIBACTERIAL
Bian K amp Murad F Nitric Oxide (2014) | Bodgan C Trends in Immunol (2015)
Nitric Oxide
6
First Indication Pulmonary Hypertension (PH) Overview
NO is an established therapeutic option for patients suffering from Pulmonary Hypertension worldwide
Life-threatening condition from increasedpulmonary vascular resistance resulting indecreased pulmonary blood flow
Generally not diagnosed until multi-organ system function is affected
NO is the de facto standard of care for PH in the hospital setting
(1) ldquoPediatric Pulmonary Hypertensionrdquo ndash Guidelines from the American heart Association and American Thoracic Society(2) Pulmonary Hypertension News ndash ldquoPulmonary Hypertension and Nitric Oxiderdquo(3) Pers istent Pulmonary Hypertension of the Newborn
NO has been used as a long-term therapeutic option for patients with pulmonary hypertension
Approved in the US by the FDA in 1999 forPPHN(3)
Approved in the EU in 2001 for PPHN(3) and cardiac surgery
Inhaled NO causes increase in the concentration level of intracellular Cyclic Guanosine Monophosphate (cGMP) and an activation of the soluble guanylate cyclase
Causes smooth muscle relaxation which increases blood flow to the lungs and decreases the workload on the right ventricle
Pulmonary Hypertension Overview Effects of Pulmonary Hypertension(1)
Benefits of NO in Treatment of PH(2)
Narrowing of the Pulmonary Arteries
Failure of Right Ventricle
7
Nitric Oxide US market
(1) MNK Company Reports(2) American Academy of Pediatrics American Hospital Association
Approved indication persistent pulmonary hypertension of the newborn (PPHN)
FY 2018 Mallinckrodt reported INOmax sales gt$500m1
Praxair expected to enter the market in 2019
o Praxair system is cylinder based like INOmax
o Anticipate rational price decline
AIT will expand the market
o ~800 hospitals have NO today1
ndash AITrsquos AirNOvent will allow NO use by hospitals unable to use a cylinder system
o gt 1000 NICUs in the US today2
o Increase use with a lower cost and ease of use vs cylinder systems
o Volume expansion with AirNOvent expected to offset price decline
The Bottom Line is that all the problems associated with NO cylinders disappear
TYPICAL NO CYLINDER PROFILE IN THE US
Height 45rdquo Diameter 75rdquo Weight ~45 lbs (Weightfor 2 cylinders on cart wdelivery system is ~175 lbs)
GENERATOR PROFILE
Height 14rdquo Width 17rdquo Depth 13rdquo Weight ~16 lbs(Weight on cart with back-up system is ~45 lbs)
8
For illustration purposes onlyFor investigational use only
AirNOvent Next generation NO care for patients worldwide
The next generation phasic flow nitricoxide delivery system The cylinder freesystem will be used for the treatment of pulmonary hypertension in the hospital setting
For investigational use only
Detachable unit
Backup switch
User interface
Ventilator Connectors
NO2 Filter
A i r N O v e n t tradeCylinder Free Nitric Oxide Therapeutic Platform
Ai rNOvent may not be the final commercial product name
Width ~24 inches
Depth ~ 28 inches
Height ~5 feet
Weight ~45 lbs
9
For illustration purposes onlyFor investigational use only
AirNOvent Next generation NO care for patients worldwide
A i r N O v e n t tradeCylinder Free Nitric Oxide Therapeutic Platform
Detachable Unit
User Interface
Ai rNOvent may not be the final commercial product name
10
Improved operating economics for the hospital
No significant capital investment required for hospitals new to NO
No burdensome inventory and storage requirements
NO supplied as a non-hypoxic gas mixture
No purging procedures or additional safety measures due to NO2 buildup
NO now available to hospitals unable to use NO cylinder systems today
Reduced training burden
Pregnant staff members not impacted
AIT does not have any expenses associated with a manufacturing facility for nitric oxide
AIT does not have any expenses associated with logistics related to nitric oxide cylinders
Hospitals will have significant cost amp logistics Advantages Our device will have significant cost Advantages
Losing the High-Pressure Cylinder is a Significant Gain
11
Circassia world-class specialty biopharma company backed by Astra Zeneca
SnapshotCircassia Pharmaceuticals plcStatus Public company traded on AIM CIR | Stock Price (02282019) GBP 35About Specialty pharmaceutical company founded in 2006 Focused on respiratory diseases based out of the UKIPO date Mar 2014Market Cap (02282019) GBP 130 M | Sales (1H 2018) GBP 284 MLoss (1H 2018) GBP 235 M | Cash in Hand (1H 2018) GBP 508 MCommercial Team US = ~200 | Total Employees ~400Major Shareholders Invesco Asset mgmt Woodford Investment mgmt AstraZeneca own about 67 combinedDirect Sales Force in United States China and certain European Countries
Area of Expertise Strategic Fit with AirNOvent
Respiratory NIOXo Used for asthma management
Tudorzao indicated for the long-term maintenance treatment of bronchospasm associated with COPD including
chronic bronchitis and emphysema
Duaklir (March 31 PDUFA)o Aclidinium bromide amp formoterol fumarate for treating COPD
Nitric Oxide NIOX is a nitric oxide measurement system for monitoring airway inflammation
AirNOvent will be a meaningful product
2018 Circassia company revenues expected to be $62m - $67m Two currently marketed products with a third expected later in 2019 Currently a small hospital presence Just rolling out commercial infrastructure in China
Exposure to US hospitals NIOX is detailed in the hospital and there is overlap in the US with top hospitals that use NO today
12
$3255 million in Total Milestones and 15-20 Royaltyo $105 million received to date
Royalties to AIT on Gross Profito5 on the first $50 million in the US (one time)o5 on the first $20 million in China (one time)o15 up to $100m annually (US amp China combined)o20 above $100m annually (US and China combined)oGross profit defined as net sales less only the cost of AirNOvent and
NO2 filters
PMA filing with FDA is anticipated in the Second Quarter 2019
US commercial launch planned First Half 2020
A Transforming Partnership ndash Transaction Details
Key Terms
High Concentration NO Delivery Opportunitiesbull Bronchiolitisbull Nontuberculous Mycobacteria (NTM)
14
Date Study Indication Primary Results
2011 Phase 1 Safety (n=10) All comers Safety bull No SAEs
2013 ndash 2014Phase 2 double blind randomized (n=43)
Bronchiolitis (all causes)
Safety amp Efficacy
bull No SAEsbull 24 hour reduction in hospital length of stay
2013 - 2014Phase 2 open label
(n=9)Cystic Fibrosis (CF)
Safety amp Efficacy
bull No SAEsbull Lowered bacterial load
2016Compassionate use Israel
(n=2)NTM in
CF patientsEfficacy
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2017Compassionate use National
Institute of Health (n=1)NTM in
CF patientEfficacy
bull No SAEsbull Improvements in clinical endpoints
2017 Pilot open label (N=9)Refractory NTM
abscessusSafety
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2018Pilot study double blind
randomized (n=67)Bronchiolitis (all causes)
Efficacybull No SAEsbull 23 hour reduction in hospital length of stay
Safety First ndash AITrsquos High Concentration NO Delivery for Lung Infections
Our Nitric Oxide Delivery System Has a Demonstrated Safety Record at a concentration of 160 ppm NO
Treatments administered patients Different clinicalsettings
Serious Adverse Events (SAEs)
related to NO
2100+ 85+ 7 0
15
Second Indication Bronchiolitis (BRO) Overview
Bronchiolitis is the leading cause of hospitalization for infants worldwide (1)
~150000 infant hospitalizations per year in the US(2)
Significant impact on the elderly from equivalent viral infections with 177000 hospitalizations per year in the US(3)
No drugs approved for the treatment of BRO patients(4)
Standard of care in the hospital is oxygen and hydration
(1) Scand J Trauma Resusc Emerg Med 2014 22 23 WHO(2) Pel letier et a l Direct medical costs of hospitalizations in the United States Pediatrics 2006(3) CDC (due to RSV only)(4) American Academy of Pediatrics
Bronchiolitis Overview amp Market Dynamics Market Size
AIT estimates US market size to be gt$2 Band projects global market to be similar size to the US market with no competition
AITrsquos goal would be to reduce length of hospitalization in infants
Elderly population trials to follow infants
16
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in length of hospital stay (LOS)
(1) httpsonlinelibrarywileycomdoiepdf101002ppul23905
2014 Trial Design and Highlights
Randomized Prospective Double-blind 43 patients (age 2-12 months) with acute
bronchiolitis (mostly due to RSV) and at least 36 weeks of gestation
N=22 Supportive Care (O₂ amp hydration) N=21 Supportive Care + 160 ppm NO for 30
minutes 5xday up to 5 days Follow up visits 2 3 amp 4 weeks post discharge Single center at Soroka University Medical
Center in Israel Data presented at ATS 2015 in an oral session Reduced length of hospital stay by ~24hrs in
patients who stayed in the hospital for at least 24 hours
No treatment related SAEs Improvements in composite endpoint
(modified Tal score) and O2 consistent with improvement in LOS
Published in the December 2017 Pediatric Pulmonology Journal(1)
17
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Design and Baseline Characteristics
Randomized 67 subjects at 6 sites in Israel with a 11 randomization between 160 ppm NO + supportive care (O2 + hydration) and supportive care alone
Subjects were 0-12 months old with acute bronchiolitis requiring hospitalization with at least 28 weeks of gestation
PE (primary endpoint) the difference in hospital length of stay (LOS)
SE (secondary endpoint) time to clinical improvement using the Modified Tal score (score ge7 and lt10 to enroll le 5 is goal)
SE the difference in time to SpO2 of gt92 SE Safety (specifically NO2 levels and
methemoglobinemia) and Tolerability Treatment was five 30 minute sessions per
day not to exceed 25 treatments All inhalations delivered by airoxygen
blender +NO via a simple mask with a minimum FiO2 of 21
DATA PRESENTED AT THE SEPTEMBER 2018 EUROPEAN RESPIRATORY SOCIETY (ERS)
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
5
Nitric Oxide (NO) is Naturally Occurring in the Human Body
CELL PROLIFERATIONANGIOGENESIS
CARDIOVASCULAR HEMOSTASISIMMUNE RESPONSE
APOPTOSIS
NEUROTRANSMISSION
ANTIBACTERIAL
Bian K amp Murad F Nitric Oxide (2014) | Bodgan C Trends in Immunol (2015)
Nitric Oxide
6
First Indication Pulmonary Hypertension (PH) Overview
NO is an established therapeutic option for patients suffering from Pulmonary Hypertension worldwide
Life-threatening condition from increasedpulmonary vascular resistance resulting indecreased pulmonary blood flow
Generally not diagnosed until multi-organ system function is affected
NO is the de facto standard of care for PH in the hospital setting
(1) ldquoPediatric Pulmonary Hypertensionrdquo ndash Guidelines from the American heart Association and American Thoracic Society(2) Pulmonary Hypertension News ndash ldquoPulmonary Hypertension and Nitric Oxiderdquo(3) Pers istent Pulmonary Hypertension of the Newborn
NO has been used as a long-term therapeutic option for patients with pulmonary hypertension
Approved in the US by the FDA in 1999 forPPHN(3)
Approved in the EU in 2001 for PPHN(3) and cardiac surgery
Inhaled NO causes increase in the concentration level of intracellular Cyclic Guanosine Monophosphate (cGMP) and an activation of the soluble guanylate cyclase
Causes smooth muscle relaxation which increases blood flow to the lungs and decreases the workload on the right ventricle
Pulmonary Hypertension Overview Effects of Pulmonary Hypertension(1)
Benefits of NO in Treatment of PH(2)
Narrowing of the Pulmonary Arteries
Failure of Right Ventricle
7
Nitric Oxide US market
(1) MNK Company Reports(2) American Academy of Pediatrics American Hospital Association
Approved indication persistent pulmonary hypertension of the newborn (PPHN)
FY 2018 Mallinckrodt reported INOmax sales gt$500m1
Praxair expected to enter the market in 2019
o Praxair system is cylinder based like INOmax
o Anticipate rational price decline
AIT will expand the market
o ~800 hospitals have NO today1
ndash AITrsquos AirNOvent will allow NO use by hospitals unable to use a cylinder system
o gt 1000 NICUs in the US today2
o Increase use with a lower cost and ease of use vs cylinder systems
o Volume expansion with AirNOvent expected to offset price decline
The Bottom Line is that all the problems associated with NO cylinders disappear
TYPICAL NO CYLINDER PROFILE IN THE US
Height 45rdquo Diameter 75rdquo Weight ~45 lbs (Weightfor 2 cylinders on cart wdelivery system is ~175 lbs)
GENERATOR PROFILE
Height 14rdquo Width 17rdquo Depth 13rdquo Weight ~16 lbs(Weight on cart with back-up system is ~45 lbs)
8
For illustration purposes onlyFor investigational use only
AirNOvent Next generation NO care for patients worldwide
The next generation phasic flow nitricoxide delivery system The cylinder freesystem will be used for the treatment of pulmonary hypertension in the hospital setting
For investigational use only
Detachable unit
Backup switch
User interface
Ventilator Connectors
NO2 Filter
A i r N O v e n t tradeCylinder Free Nitric Oxide Therapeutic Platform
Ai rNOvent may not be the final commercial product name
Width ~24 inches
Depth ~ 28 inches
Height ~5 feet
Weight ~45 lbs
9
For illustration purposes onlyFor investigational use only
AirNOvent Next generation NO care for patients worldwide
A i r N O v e n t tradeCylinder Free Nitric Oxide Therapeutic Platform
Detachable Unit
User Interface
Ai rNOvent may not be the final commercial product name
10
Improved operating economics for the hospital
No significant capital investment required for hospitals new to NO
No burdensome inventory and storage requirements
NO supplied as a non-hypoxic gas mixture
No purging procedures or additional safety measures due to NO2 buildup
NO now available to hospitals unable to use NO cylinder systems today
Reduced training burden
Pregnant staff members not impacted
AIT does not have any expenses associated with a manufacturing facility for nitric oxide
AIT does not have any expenses associated with logistics related to nitric oxide cylinders
Hospitals will have significant cost amp logistics Advantages Our device will have significant cost Advantages
Losing the High-Pressure Cylinder is a Significant Gain
11
Circassia world-class specialty biopharma company backed by Astra Zeneca
SnapshotCircassia Pharmaceuticals plcStatus Public company traded on AIM CIR | Stock Price (02282019) GBP 35About Specialty pharmaceutical company founded in 2006 Focused on respiratory diseases based out of the UKIPO date Mar 2014Market Cap (02282019) GBP 130 M | Sales (1H 2018) GBP 284 MLoss (1H 2018) GBP 235 M | Cash in Hand (1H 2018) GBP 508 MCommercial Team US = ~200 | Total Employees ~400Major Shareholders Invesco Asset mgmt Woodford Investment mgmt AstraZeneca own about 67 combinedDirect Sales Force in United States China and certain European Countries
Area of Expertise Strategic Fit with AirNOvent
Respiratory NIOXo Used for asthma management
Tudorzao indicated for the long-term maintenance treatment of bronchospasm associated with COPD including
chronic bronchitis and emphysema
Duaklir (March 31 PDUFA)o Aclidinium bromide amp formoterol fumarate for treating COPD
Nitric Oxide NIOX is a nitric oxide measurement system for monitoring airway inflammation
AirNOvent will be a meaningful product
2018 Circassia company revenues expected to be $62m - $67m Two currently marketed products with a third expected later in 2019 Currently a small hospital presence Just rolling out commercial infrastructure in China
Exposure to US hospitals NIOX is detailed in the hospital and there is overlap in the US with top hospitals that use NO today
12
$3255 million in Total Milestones and 15-20 Royaltyo $105 million received to date
Royalties to AIT on Gross Profito5 on the first $50 million in the US (one time)o5 on the first $20 million in China (one time)o15 up to $100m annually (US amp China combined)o20 above $100m annually (US and China combined)oGross profit defined as net sales less only the cost of AirNOvent and
NO2 filters
PMA filing with FDA is anticipated in the Second Quarter 2019
US commercial launch planned First Half 2020
A Transforming Partnership ndash Transaction Details
Key Terms
High Concentration NO Delivery Opportunitiesbull Bronchiolitisbull Nontuberculous Mycobacteria (NTM)
14
Date Study Indication Primary Results
2011 Phase 1 Safety (n=10) All comers Safety bull No SAEs
2013 ndash 2014Phase 2 double blind randomized (n=43)
Bronchiolitis (all causes)
Safety amp Efficacy
bull No SAEsbull 24 hour reduction in hospital length of stay
2013 - 2014Phase 2 open label
(n=9)Cystic Fibrosis (CF)
Safety amp Efficacy
bull No SAEsbull Lowered bacterial load
2016Compassionate use Israel
(n=2)NTM in
CF patientsEfficacy
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2017Compassionate use National
Institute of Health (n=1)NTM in
CF patientEfficacy
bull No SAEsbull Improvements in clinical endpoints
2017 Pilot open label (N=9)Refractory NTM
abscessusSafety
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2018Pilot study double blind
randomized (n=67)Bronchiolitis (all causes)
Efficacybull No SAEsbull 23 hour reduction in hospital length of stay
Safety First ndash AITrsquos High Concentration NO Delivery for Lung Infections
Our Nitric Oxide Delivery System Has a Demonstrated Safety Record at a concentration of 160 ppm NO
Treatments administered patients Different clinicalsettings
Serious Adverse Events (SAEs)
related to NO
2100+ 85+ 7 0
15
Second Indication Bronchiolitis (BRO) Overview
Bronchiolitis is the leading cause of hospitalization for infants worldwide (1)
~150000 infant hospitalizations per year in the US(2)
Significant impact on the elderly from equivalent viral infections with 177000 hospitalizations per year in the US(3)
No drugs approved for the treatment of BRO patients(4)
Standard of care in the hospital is oxygen and hydration
(1) Scand J Trauma Resusc Emerg Med 2014 22 23 WHO(2) Pel letier et a l Direct medical costs of hospitalizations in the United States Pediatrics 2006(3) CDC (due to RSV only)(4) American Academy of Pediatrics
Bronchiolitis Overview amp Market Dynamics Market Size
AIT estimates US market size to be gt$2 Band projects global market to be similar size to the US market with no competition
AITrsquos goal would be to reduce length of hospitalization in infants
Elderly population trials to follow infants
16
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in length of hospital stay (LOS)
(1) httpsonlinelibrarywileycomdoiepdf101002ppul23905
2014 Trial Design and Highlights
Randomized Prospective Double-blind 43 patients (age 2-12 months) with acute
bronchiolitis (mostly due to RSV) and at least 36 weeks of gestation
N=22 Supportive Care (O₂ amp hydration) N=21 Supportive Care + 160 ppm NO for 30
minutes 5xday up to 5 days Follow up visits 2 3 amp 4 weeks post discharge Single center at Soroka University Medical
Center in Israel Data presented at ATS 2015 in an oral session Reduced length of hospital stay by ~24hrs in
patients who stayed in the hospital for at least 24 hours
No treatment related SAEs Improvements in composite endpoint
(modified Tal score) and O2 consistent with improvement in LOS
Published in the December 2017 Pediatric Pulmonology Journal(1)
17
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Design and Baseline Characteristics
Randomized 67 subjects at 6 sites in Israel with a 11 randomization between 160 ppm NO + supportive care (O2 + hydration) and supportive care alone
Subjects were 0-12 months old with acute bronchiolitis requiring hospitalization with at least 28 weeks of gestation
PE (primary endpoint) the difference in hospital length of stay (LOS)
SE (secondary endpoint) time to clinical improvement using the Modified Tal score (score ge7 and lt10 to enroll le 5 is goal)
SE the difference in time to SpO2 of gt92 SE Safety (specifically NO2 levels and
methemoglobinemia) and Tolerability Treatment was five 30 minute sessions per
day not to exceed 25 treatments All inhalations delivered by airoxygen
blender +NO via a simple mask with a minimum FiO2 of 21
DATA PRESENTED AT THE SEPTEMBER 2018 EUROPEAN RESPIRATORY SOCIETY (ERS)
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
6
First Indication Pulmonary Hypertension (PH) Overview
NO is an established therapeutic option for patients suffering from Pulmonary Hypertension worldwide
Life-threatening condition from increasedpulmonary vascular resistance resulting indecreased pulmonary blood flow
Generally not diagnosed until multi-organ system function is affected
NO is the de facto standard of care for PH in the hospital setting
(1) ldquoPediatric Pulmonary Hypertensionrdquo ndash Guidelines from the American heart Association and American Thoracic Society(2) Pulmonary Hypertension News ndash ldquoPulmonary Hypertension and Nitric Oxiderdquo(3) Pers istent Pulmonary Hypertension of the Newborn
NO has been used as a long-term therapeutic option for patients with pulmonary hypertension
Approved in the US by the FDA in 1999 forPPHN(3)
Approved in the EU in 2001 for PPHN(3) and cardiac surgery
Inhaled NO causes increase in the concentration level of intracellular Cyclic Guanosine Monophosphate (cGMP) and an activation of the soluble guanylate cyclase
Causes smooth muscle relaxation which increases blood flow to the lungs and decreases the workload on the right ventricle
Pulmonary Hypertension Overview Effects of Pulmonary Hypertension(1)
Benefits of NO in Treatment of PH(2)
Narrowing of the Pulmonary Arteries
Failure of Right Ventricle
7
Nitric Oxide US market
(1) MNK Company Reports(2) American Academy of Pediatrics American Hospital Association
Approved indication persistent pulmonary hypertension of the newborn (PPHN)
FY 2018 Mallinckrodt reported INOmax sales gt$500m1
Praxair expected to enter the market in 2019
o Praxair system is cylinder based like INOmax
o Anticipate rational price decline
AIT will expand the market
o ~800 hospitals have NO today1
ndash AITrsquos AirNOvent will allow NO use by hospitals unable to use a cylinder system
o gt 1000 NICUs in the US today2
o Increase use with a lower cost and ease of use vs cylinder systems
o Volume expansion with AirNOvent expected to offset price decline
The Bottom Line is that all the problems associated with NO cylinders disappear
TYPICAL NO CYLINDER PROFILE IN THE US
Height 45rdquo Diameter 75rdquo Weight ~45 lbs (Weightfor 2 cylinders on cart wdelivery system is ~175 lbs)
GENERATOR PROFILE
Height 14rdquo Width 17rdquo Depth 13rdquo Weight ~16 lbs(Weight on cart with back-up system is ~45 lbs)
8
For illustration purposes onlyFor investigational use only
AirNOvent Next generation NO care for patients worldwide
The next generation phasic flow nitricoxide delivery system The cylinder freesystem will be used for the treatment of pulmonary hypertension in the hospital setting
For investigational use only
Detachable unit
Backup switch
User interface
Ventilator Connectors
NO2 Filter
A i r N O v e n t tradeCylinder Free Nitric Oxide Therapeutic Platform
Ai rNOvent may not be the final commercial product name
Width ~24 inches
Depth ~ 28 inches
Height ~5 feet
Weight ~45 lbs
9
For illustration purposes onlyFor investigational use only
AirNOvent Next generation NO care for patients worldwide
A i r N O v e n t tradeCylinder Free Nitric Oxide Therapeutic Platform
Detachable Unit
User Interface
Ai rNOvent may not be the final commercial product name
10
Improved operating economics for the hospital
No significant capital investment required for hospitals new to NO
No burdensome inventory and storage requirements
NO supplied as a non-hypoxic gas mixture
No purging procedures or additional safety measures due to NO2 buildup
NO now available to hospitals unable to use NO cylinder systems today
Reduced training burden
Pregnant staff members not impacted
AIT does not have any expenses associated with a manufacturing facility for nitric oxide
AIT does not have any expenses associated with logistics related to nitric oxide cylinders
Hospitals will have significant cost amp logistics Advantages Our device will have significant cost Advantages
Losing the High-Pressure Cylinder is a Significant Gain
11
Circassia world-class specialty biopharma company backed by Astra Zeneca
SnapshotCircassia Pharmaceuticals plcStatus Public company traded on AIM CIR | Stock Price (02282019) GBP 35About Specialty pharmaceutical company founded in 2006 Focused on respiratory diseases based out of the UKIPO date Mar 2014Market Cap (02282019) GBP 130 M | Sales (1H 2018) GBP 284 MLoss (1H 2018) GBP 235 M | Cash in Hand (1H 2018) GBP 508 MCommercial Team US = ~200 | Total Employees ~400Major Shareholders Invesco Asset mgmt Woodford Investment mgmt AstraZeneca own about 67 combinedDirect Sales Force in United States China and certain European Countries
Area of Expertise Strategic Fit with AirNOvent
Respiratory NIOXo Used for asthma management
Tudorzao indicated for the long-term maintenance treatment of bronchospasm associated with COPD including
chronic bronchitis and emphysema
Duaklir (March 31 PDUFA)o Aclidinium bromide amp formoterol fumarate for treating COPD
Nitric Oxide NIOX is a nitric oxide measurement system for monitoring airway inflammation
AirNOvent will be a meaningful product
2018 Circassia company revenues expected to be $62m - $67m Two currently marketed products with a third expected later in 2019 Currently a small hospital presence Just rolling out commercial infrastructure in China
Exposure to US hospitals NIOX is detailed in the hospital and there is overlap in the US with top hospitals that use NO today
12
$3255 million in Total Milestones and 15-20 Royaltyo $105 million received to date
Royalties to AIT on Gross Profito5 on the first $50 million in the US (one time)o5 on the first $20 million in China (one time)o15 up to $100m annually (US amp China combined)o20 above $100m annually (US and China combined)oGross profit defined as net sales less only the cost of AirNOvent and
NO2 filters
PMA filing with FDA is anticipated in the Second Quarter 2019
US commercial launch planned First Half 2020
A Transforming Partnership ndash Transaction Details
Key Terms
High Concentration NO Delivery Opportunitiesbull Bronchiolitisbull Nontuberculous Mycobacteria (NTM)
14
Date Study Indication Primary Results
2011 Phase 1 Safety (n=10) All comers Safety bull No SAEs
2013 ndash 2014Phase 2 double blind randomized (n=43)
Bronchiolitis (all causes)
Safety amp Efficacy
bull No SAEsbull 24 hour reduction in hospital length of stay
2013 - 2014Phase 2 open label
(n=9)Cystic Fibrosis (CF)
Safety amp Efficacy
bull No SAEsbull Lowered bacterial load
2016Compassionate use Israel
(n=2)NTM in
CF patientsEfficacy
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2017Compassionate use National
Institute of Health (n=1)NTM in
CF patientEfficacy
bull No SAEsbull Improvements in clinical endpoints
2017 Pilot open label (N=9)Refractory NTM
abscessusSafety
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2018Pilot study double blind
randomized (n=67)Bronchiolitis (all causes)
Efficacybull No SAEsbull 23 hour reduction in hospital length of stay
Safety First ndash AITrsquos High Concentration NO Delivery for Lung Infections
Our Nitric Oxide Delivery System Has a Demonstrated Safety Record at a concentration of 160 ppm NO
Treatments administered patients Different clinicalsettings
Serious Adverse Events (SAEs)
related to NO
2100+ 85+ 7 0
15
Second Indication Bronchiolitis (BRO) Overview
Bronchiolitis is the leading cause of hospitalization for infants worldwide (1)
~150000 infant hospitalizations per year in the US(2)
Significant impact on the elderly from equivalent viral infections with 177000 hospitalizations per year in the US(3)
No drugs approved for the treatment of BRO patients(4)
Standard of care in the hospital is oxygen and hydration
(1) Scand J Trauma Resusc Emerg Med 2014 22 23 WHO(2) Pel letier et a l Direct medical costs of hospitalizations in the United States Pediatrics 2006(3) CDC (due to RSV only)(4) American Academy of Pediatrics
Bronchiolitis Overview amp Market Dynamics Market Size
AIT estimates US market size to be gt$2 Band projects global market to be similar size to the US market with no competition
AITrsquos goal would be to reduce length of hospitalization in infants
Elderly population trials to follow infants
16
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in length of hospital stay (LOS)
(1) httpsonlinelibrarywileycomdoiepdf101002ppul23905
2014 Trial Design and Highlights
Randomized Prospective Double-blind 43 patients (age 2-12 months) with acute
bronchiolitis (mostly due to RSV) and at least 36 weeks of gestation
N=22 Supportive Care (O₂ amp hydration) N=21 Supportive Care + 160 ppm NO for 30
minutes 5xday up to 5 days Follow up visits 2 3 amp 4 weeks post discharge Single center at Soroka University Medical
Center in Israel Data presented at ATS 2015 in an oral session Reduced length of hospital stay by ~24hrs in
patients who stayed in the hospital for at least 24 hours
No treatment related SAEs Improvements in composite endpoint
(modified Tal score) and O2 consistent with improvement in LOS
Published in the December 2017 Pediatric Pulmonology Journal(1)
17
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Design and Baseline Characteristics
Randomized 67 subjects at 6 sites in Israel with a 11 randomization between 160 ppm NO + supportive care (O2 + hydration) and supportive care alone
Subjects were 0-12 months old with acute bronchiolitis requiring hospitalization with at least 28 weeks of gestation
PE (primary endpoint) the difference in hospital length of stay (LOS)
SE (secondary endpoint) time to clinical improvement using the Modified Tal score (score ge7 and lt10 to enroll le 5 is goal)
SE the difference in time to SpO2 of gt92 SE Safety (specifically NO2 levels and
methemoglobinemia) and Tolerability Treatment was five 30 minute sessions per
day not to exceed 25 treatments All inhalations delivered by airoxygen
blender +NO via a simple mask with a minimum FiO2 of 21
DATA PRESENTED AT THE SEPTEMBER 2018 EUROPEAN RESPIRATORY SOCIETY (ERS)
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
7
Nitric Oxide US market
(1) MNK Company Reports(2) American Academy of Pediatrics American Hospital Association
Approved indication persistent pulmonary hypertension of the newborn (PPHN)
FY 2018 Mallinckrodt reported INOmax sales gt$500m1
Praxair expected to enter the market in 2019
o Praxair system is cylinder based like INOmax
o Anticipate rational price decline
AIT will expand the market
o ~800 hospitals have NO today1
ndash AITrsquos AirNOvent will allow NO use by hospitals unable to use a cylinder system
o gt 1000 NICUs in the US today2
o Increase use with a lower cost and ease of use vs cylinder systems
o Volume expansion with AirNOvent expected to offset price decline
The Bottom Line is that all the problems associated with NO cylinders disappear
TYPICAL NO CYLINDER PROFILE IN THE US
Height 45rdquo Diameter 75rdquo Weight ~45 lbs (Weightfor 2 cylinders on cart wdelivery system is ~175 lbs)
GENERATOR PROFILE
Height 14rdquo Width 17rdquo Depth 13rdquo Weight ~16 lbs(Weight on cart with back-up system is ~45 lbs)
8
For illustration purposes onlyFor investigational use only
AirNOvent Next generation NO care for patients worldwide
The next generation phasic flow nitricoxide delivery system The cylinder freesystem will be used for the treatment of pulmonary hypertension in the hospital setting
For investigational use only
Detachable unit
Backup switch
User interface
Ventilator Connectors
NO2 Filter
A i r N O v e n t tradeCylinder Free Nitric Oxide Therapeutic Platform
Ai rNOvent may not be the final commercial product name
Width ~24 inches
Depth ~ 28 inches
Height ~5 feet
Weight ~45 lbs
9
For illustration purposes onlyFor investigational use only
AirNOvent Next generation NO care for patients worldwide
A i r N O v e n t tradeCylinder Free Nitric Oxide Therapeutic Platform
Detachable Unit
User Interface
Ai rNOvent may not be the final commercial product name
10
Improved operating economics for the hospital
No significant capital investment required for hospitals new to NO
No burdensome inventory and storage requirements
NO supplied as a non-hypoxic gas mixture
No purging procedures or additional safety measures due to NO2 buildup
NO now available to hospitals unable to use NO cylinder systems today
Reduced training burden
Pregnant staff members not impacted
AIT does not have any expenses associated with a manufacturing facility for nitric oxide
AIT does not have any expenses associated with logistics related to nitric oxide cylinders
Hospitals will have significant cost amp logistics Advantages Our device will have significant cost Advantages
Losing the High-Pressure Cylinder is a Significant Gain
11
Circassia world-class specialty biopharma company backed by Astra Zeneca
SnapshotCircassia Pharmaceuticals plcStatus Public company traded on AIM CIR | Stock Price (02282019) GBP 35About Specialty pharmaceutical company founded in 2006 Focused on respiratory diseases based out of the UKIPO date Mar 2014Market Cap (02282019) GBP 130 M | Sales (1H 2018) GBP 284 MLoss (1H 2018) GBP 235 M | Cash in Hand (1H 2018) GBP 508 MCommercial Team US = ~200 | Total Employees ~400Major Shareholders Invesco Asset mgmt Woodford Investment mgmt AstraZeneca own about 67 combinedDirect Sales Force in United States China and certain European Countries
Area of Expertise Strategic Fit with AirNOvent
Respiratory NIOXo Used for asthma management
Tudorzao indicated for the long-term maintenance treatment of bronchospasm associated with COPD including
chronic bronchitis and emphysema
Duaklir (March 31 PDUFA)o Aclidinium bromide amp formoterol fumarate for treating COPD
Nitric Oxide NIOX is a nitric oxide measurement system for monitoring airway inflammation
AirNOvent will be a meaningful product
2018 Circassia company revenues expected to be $62m - $67m Two currently marketed products with a third expected later in 2019 Currently a small hospital presence Just rolling out commercial infrastructure in China
Exposure to US hospitals NIOX is detailed in the hospital and there is overlap in the US with top hospitals that use NO today
12
$3255 million in Total Milestones and 15-20 Royaltyo $105 million received to date
Royalties to AIT on Gross Profito5 on the first $50 million in the US (one time)o5 on the first $20 million in China (one time)o15 up to $100m annually (US amp China combined)o20 above $100m annually (US and China combined)oGross profit defined as net sales less only the cost of AirNOvent and
NO2 filters
PMA filing with FDA is anticipated in the Second Quarter 2019
US commercial launch planned First Half 2020
A Transforming Partnership ndash Transaction Details
Key Terms
High Concentration NO Delivery Opportunitiesbull Bronchiolitisbull Nontuberculous Mycobacteria (NTM)
14
Date Study Indication Primary Results
2011 Phase 1 Safety (n=10) All comers Safety bull No SAEs
2013 ndash 2014Phase 2 double blind randomized (n=43)
Bronchiolitis (all causes)
Safety amp Efficacy
bull No SAEsbull 24 hour reduction in hospital length of stay
2013 - 2014Phase 2 open label
(n=9)Cystic Fibrosis (CF)
Safety amp Efficacy
bull No SAEsbull Lowered bacterial load
2016Compassionate use Israel
(n=2)NTM in
CF patientsEfficacy
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2017Compassionate use National
Institute of Health (n=1)NTM in
CF patientEfficacy
bull No SAEsbull Improvements in clinical endpoints
2017 Pilot open label (N=9)Refractory NTM
abscessusSafety
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2018Pilot study double blind
randomized (n=67)Bronchiolitis (all causes)
Efficacybull No SAEsbull 23 hour reduction in hospital length of stay
Safety First ndash AITrsquos High Concentration NO Delivery for Lung Infections
Our Nitric Oxide Delivery System Has a Demonstrated Safety Record at a concentration of 160 ppm NO
Treatments administered patients Different clinicalsettings
Serious Adverse Events (SAEs)
related to NO
2100+ 85+ 7 0
15
Second Indication Bronchiolitis (BRO) Overview
Bronchiolitis is the leading cause of hospitalization for infants worldwide (1)
~150000 infant hospitalizations per year in the US(2)
Significant impact on the elderly from equivalent viral infections with 177000 hospitalizations per year in the US(3)
No drugs approved for the treatment of BRO patients(4)
Standard of care in the hospital is oxygen and hydration
(1) Scand J Trauma Resusc Emerg Med 2014 22 23 WHO(2) Pel letier et a l Direct medical costs of hospitalizations in the United States Pediatrics 2006(3) CDC (due to RSV only)(4) American Academy of Pediatrics
Bronchiolitis Overview amp Market Dynamics Market Size
AIT estimates US market size to be gt$2 Band projects global market to be similar size to the US market with no competition
AITrsquos goal would be to reduce length of hospitalization in infants
Elderly population trials to follow infants
16
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in length of hospital stay (LOS)
(1) httpsonlinelibrarywileycomdoiepdf101002ppul23905
2014 Trial Design and Highlights
Randomized Prospective Double-blind 43 patients (age 2-12 months) with acute
bronchiolitis (mostly due to RSV) and at least 36 weeks of gestation
N=22 Supportive Care (O₂ amp hydration) N=21 Supportive Care + 160 ppm NO for 30
minutes 5xday up to 5 days Follow up visits 2 3 amp 4 weeks post discharge Single center at Soroka University Medical
Center in Israel Data presented at ATS 2015 in an oral session Reduced length of hospital stay by ~24hrs in
patients who stayed in the hospital for at least 24 hours
No treatment related SAEs Improvements in composite endpoint
(modified Tal score) and O2 consistent with improvement in LOS
Published in the December 2017 Pediatric Pulmonology Journal(1)
17
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Design and Baseline Characteristics
Randomized 67 subjects at 6 sites in Israel with a 11 randomization between 160 ppm NO + supportive care (O2 + hydration) and supportive care alone
Subjects were 0-12 months old with acute bronchiolitis requiring hospitalization with at least 28 weeks of gestation
PE (primary endpoint) the difference in hospital length of stay (LOS)
SE (secondary endpoint) time to clinical improvement using the Modified Tal score (score ge7 and lt10 to enroll le 5 is goal)
SE the difference in time to SpO2 of gt92 SE Safety (specifically NO2 levels and
methemoglobinemia) and Tolerability Treatment was five 30 minute sessions per
day not to exceed 25 treatments All inhalations delivered by airoxygen
blender +NO via a simple mask with a minimum FiO2 of 21
DATA PRESENTED AT THE SEPTEMBER 2018 EUROPEAN RESPIRATORY SOCIETY (ERS)
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
8
For illustration purposes onlyFor investigational use only
AirNOvent Next generation NO care for patients worldwide
The next generation phasic flow nitricoxide delivery system The cylinder freesystem will be used for the treatment of pulmonary hypertension in the hospital setting
For investigational use only
Detachable unit
Backup switch
User interface
Ventilator Connectors
NO2 Filter
A i r N O v e n t tradeCylinder Free Nitric Oxide Therapeutic Platform
Ai rNOvent may not be the final commercial product name
Width ~24 inches
Depth ~ 28 inches
Height ~5 feet
Weight ~45 lbs
9
For illustration purposes onlyFor investigational use only
AirNOvent Next generation NO care for patients worldwide
A i r N O v e n t tradeCylinder Free Nitric Oxide Therapeutic Platform
Detachable Unit
User Interface
Ai rNOvent may not be the final commercial product name
10
Improved operating economics for the hospital
No significant capital investment required for hospitals new to NO
No burdensome inventory and storage requirements
NO supplied as a non-hypoxic gas mixture
No purging procedures or additional safety measures due to NO2 buildup
NO now available to hospitals unable to use NO cylinder systems today
Reduced training burden
Pregnant staff members not impacted
AIT does not have any expenses associated with a manufacturing facility for nitric oxide
AIT does not have any expenses associated with logistics related to nitric oxide cylinders
Hospitals will have significant cost amp logistics Advantages Our device will have significant cost Advantages
Losing the High-Pressure Cylinder is a Significant Gain
11
Circassia world-class specialty biopharma company backed by Astra Zeneca
SnapshotCircassia Pharmaceuticals plcStatus Public company traded on AIM CIR | Stock Price (02282019) GBP 35About Specialty pharmaceutical company founded in 2006 Focused on respiratory diseases based out of the UKIPO date Mar 2014Market Cap (02282019) GBP 130 M | Sales (1H 2018) GBP 284 MLoss (1H 2018) GBP 235 M | Cash in Hand (1H 2018) GBP 508 MCommercial Team US = ~200 | Total Employees ~400Major Shareholders Invesco Asset mgmt Woodford Investment mgmt AstraZeneca own about 67 combinedDirect Sales Force in United States China and certain European Countries
Area of Expertise Strategic Fit with AirNOvent
Respiratory NIOXo Used for asthma management
Tudorzao indicated for the long-term maintenance treatment of bronchospasm associated with COPD including
chronic bronchitis and emphysema
Duaklir (March 31 PDUFA)o Aclidinium bromide amp formoterol fumarate for treating COPD
Nitric Oxide NIOX is a nitric oxide measurement system for monitoring airway inflammation
AirNOvent will be a meaningful product
2018 Circassia company revenues expected to be $62m - $67m Two currently marketed products with a third expected later in 2019 Currently a small hospital presence Just rolling out commercial infrastructure in China
Exposure to US hospitals NIOX is detailed in the hospital and there is overlap in the US with top hospitals that use NO today
12
$3255 million in Total Milestones and 15-20 Royaltyo $105 million received to date
Royalties to AIT on Gross Profito5 on the first $50 million in the US (one time)o5 on the first $20 million in China (one time)o15 up to $100m annually (US amp China combined)o20 above $100m annually (US and China combined)oGross profit defined as net sales less only the cost of AirNOvent and
NO2 filters
PMA filing with FDA is anticipated in the Second Quarter 2019
US commercial launch planned First Half 2020
A Transforming Partnership ndash Transaction Details
Key Terms
High Concentration NO Delivery Opportunitiesbull Bronchiolitisbull Nontuberculous Mycobacteria (NTM)
14
Date Study Indication Primary Results
2011 Phase 1 Safety (n=10) All comers Safety bull No SAEs
2013 ndash 2014Phase 2 double blind randomized (n=43)
Bronchiolitis (all causes)
Safety amp Efficacy
bull No SAEsbull 24 hour reduction in hospital length of stay
2013 - 2014Phase 2 open label
(n=9)Cystic Fibrosis (CF)
Safety amp Efficacy
bull No SAEsbull Lowered bacterial load
2016Compassionate use Israel
(n=2)NTM in
CF patientsEfficacy
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2017Compassionate use National
Institute of Health (n=1)NTM in
CF patientEfficacy
bull No SAEsbull Improvements in clinical endpoints
2017 Pilot open label (N=9)Refractory NTM
abscessusSafety
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2018Pilot study double blind
randomized (n=67)Bronchiolitis (all causes)
Efficacybull No SAEsbull 23 hour reduction in hospital length of stay
Safety First ndash AITrsquos High Concentration NO Delivery for Lung Infections
Our Nitric Oxide Delivery System Has a Demonstrated Safety Record at a concentration of 160 ppm NO
Treatments administered patients Different clinicalsettings
Serious Adverse Events (SAEs)
related to NO
2100+ 85+ 7 0
15
Second Indication Bronchiolitis (BRO) Overview
Bronchiolitis is the leading cause of hospitalization for infants worldwide (1)
~150000 infant hospitalizations per year in the US(2)
Significant impact on the elderly from equivalent viral infections with 177000 hospitalizations per year in the US(3)
No drugs approved for the treatment of BRO patients(4)
Standard of care in the hospital is oxygen and hydration
(1) Scand J Trauma Resusc Emerg Med 2014 22 23 WHO(2) Pel letier et a l Direct medical costs of hospitalizations in the United States Pediatrics 2006(3) CDC (due to RSV only)(4) American Academy of Pediatrics
Bronchiolitis Overview amp Market Dynamics Market Size
AIT estimates US market size to be gt$2 Band projects global market to be similar size to the US market with no competition
AITrsquos goal would be to reduce length of hospitalization in infants
Elderly population trials to follow infants
16
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in length of hospital stay (LOS)
(1) httpsonlinelibrarywileycomdoiepdf101002ppul23905
2014 Trial Design and Highlights
Randomized Prospective Double-blind 43 patients (age 2-12 months) with acute
bronchiolitis (mostly due to RSV) and at least 36 weeks of gestation
N=22 Supportive Care (O₂ amp hydration) N=21 Supportive Care + 160 ppm NO for 30
minutes 5xday up to 5 days Follow up visits 2 3 amp 4 weeks post discharge Single center at Soroka University Medical
Center in Israel Data presented at ATS 2015 in an oral session Reduced length of hospital stay by ~24hrs in
patients who stayed in the hospital for at least 24 hours
No treatment related SAEs Improvements in composite endpoint
(modified Tal score) and O2 consistent with improvement in LOS
Published in the December 2017 Pediatric Pulmonology Journal(1)
17
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Design and Baseline Characteristics
Randomized 67 subjects at 6 sites in Israel with a 11 randomization between 160 ppm NO + supportive care (O2 + hydration) and supportive care alone
Subjects were 0-12 months old with acute bronchiolitis requiring hospitalization with at least 28 weeks of gestation
PE (primary endpoint) the difference in hospital length of stay (LOS)
SE (secondary endpoint) time to clinical improvement using the Modified Tal score (score ge7 and lt10 to enroll le 5 is goal)
SE the difference in time to SpO2 of gt92 SE Safety (specifically NO2 levels and
methemoglobinemia) and Tolerability Treatment was five 30 minute sessions per
day not to exceed 25 treatments All inhalations delivered by airoxygen
blender +NO via a simple mask with a minimum FiO2 of 21
DATA PRESENTED AT THE SEPTEMBER 2018 EUROPEAN RESPIRATORY SOCIETY (ERS)
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
9
For illustration purposes onlyFor investigational use only
AirNOvent Next generation NO care for patients worldwide
A i r N O v e n t tradeCylinder Free Nitric Oxide Therapeutic Platform
Detachable Unit
User Interface
Ai rNOvent may not be the final commercial product name
10
Improved operating economics for the hospital
No significant capital investment required for hospitals new to NO
No burdensome inventory and storage requirements
NO supplied as a non-hypoxic gas mixture
No purging procedures or additional safety measures due to NO2 buildup
NO now available to hospitals unable to use NO cylinder systems today
Reduced training burden
Pregnant staff members not impacted
AIT does not have any expenses associated with a manufacturing facility for nitric oxide
AIT does not have any expenses associated with logistics related to nitric oxide cylinders
Hospitals will have significant cost amp logistics Advantages Our device will have significant cost Advantages
Losing the High-Pressure Cylinder is a Significant Gain
11
Circassia world-class specialty biopharma company backed by Astra Zeneca
SnapshotCircassia Pharmaceuticals plcStatus Public company traded on AIM CIR | Stock Price (02282019) GBP 35About Specialty pharmaceutical company founded in 2006 Focused on respiratory diseases based out of the UKIPO date Mar 2014Market Cap (02282019) GBP 130 M | Sales (1H 2018) GBP 284 MLoss (1H 2018) GBP 235 M | Cash in Hand (1H 2018) GBP 508 MCommercial Team US = ~200 | Total Employees ~400Major Shareholders Invesco Asset mgmt Woodford Investment mgmt AstraZeneca own about 67 combinedDirect Sales Force in United States China and certain European Countries
Area of Expertise Strategic Fit with AirNOvent
Respiratory NIOXo Used for asthma management
Tudorzao indicated for the long-term maintenance treatment of bronchospasm associated with COPD including
chronic bronchitis and emphysema
Duaklir (March 31 PDUFA)o Aclidinium bromide amp formoterol fumarate for treating COPD
Nitric Oxide NIOX is a nitric oxide measurement system for monitoring airway inflammation
AirNOvent will be a meaningful product
2018 Circassia company revenues expected to be $62m - $67m Two currently marketed products with a third expected later in 2019 Currently a small hospital presence Just rolling out commercial infrastructure in China
Exposure to US hospitals NIOX is detailed in the hospital and there is overlap in the US with top hospitals that use NO today
12
$3255 million in Total Milestones and 15-20 Royaltyo $105 million received to date
Royalties to AIT on Gross Profito5 on the first $50 million in the US (one time)o5 on the first $20 million in China (one time)o15 up to $100m annually (US amp China combined)o20 above $100m annually (US and China combined)oGross profit defined as net sales less only the cost of AirNOvent and
NO2 filters
PMA filing with FDA is anticipated in the Second Quarter 2019
US commercial launch planned First Half 2020
A Transforming Partnership ndash Transaction Details
Key Terms
High Concentration NO Delivery Opportunitiesbull Bronchiolitisbull Nontuberculous Mycobacteria (NTM)
14
Date Study Indication Primary Results
2011 Phase 1 Safety (n=10) All comers Safety bull No SAEs
2013 ndash 2014Phase 2 double blind randomized (n=43)
Bronchiolitis (all causes)
Safety amp Efficacy
bull No SAEsbull 24 hour reduction in hospital length of stay
2013 - 2014Phase 2 open label
(n=9)Cystic Fibrosis (CF)
Safety amp Efficacy
bull No SAEsbull Lowered bacterial load
2016Compassionate use Israel
(n=2)NTM in
CF patientsEfficacy
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2017Compassionate use National
Institute of Health (n=1)NTM in
CF patientEfficacy
bull No SAEsbull Improvements in clinical endpoints
2017 Pilot open label (N=9)Refractory NTM
abscessusSafety
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2018Pilot study double blind
randomized (n=67)Bronchiolitis (all causes)
Efficacybull No SAEsbull 23 hour reduction in hospital length of stay
Safety First ndash AITrsquos High Concentration NO Delivery for Lung Infections
Our Nitric Oxide Delivery System Has a Demonstrated Safety Record at a concentration of 160 ppm NO
Treatments administered patients Different clinicalsettings
Serious Adverse Events (SAEs)
related to NO
2100+ 85+ 7 0
15
Second Indication Bronchiolitis (BRO) Overview
Bronchiolitis is the leading cause of hospitalization for infants worldwide (1)
~150000 infant hospitalizations per year in the US(2)
Significant impact on the elderly from equivalent viral infections with 177000 hospitalizations per year in the US(3)
No drugs approved for the treatment of BRO patients(4)
Standard of care in the hospital is oxygen and hydration
(1) Scand J Trauma Resusc Emerg Med 2014 22 23 WHO(2) Pel letier et a l Direct medical costs of hospitalizations in the United States Pediatrics 2006(3) CDC (due to RSV only)(4) American Academy of Pediatrics
Bronchiolitis Overview amp Market Dynamics Market Size
AIT estimates US market size to be gt$2 Band projects global market to be similar size to the US market with no competition
AITrsquos goal would be to reduce length of hospitalization in infants
Elderly population trials to follow infants
16
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in length of hospital stay (LOS)
(1) httpsonlinelibrarywileycomdoiepdf101002ppul23905
2014 Trial Design and Highlights
Randomized Prospective Double-blind 43 patients (age 2-12 months) with acute
bronchiolitis (mostly due to RSV) and at least 36 weeks of gestation
N=22 Supportive Care (O₂ amp hydration) N=21 Supportive Care + 160 ppm NO for 30
minutes 5xday up to 5 days Follow up visits 2 3 amp 4 weeks post discharge Single center at Soroka University Medical
Center in Israel Data presented at ATS 2015 in an oral session Reduced length of hospital stay by ~24hrs in
patients who stayed in the hospital for at least 24 hours
No treatment related SAEs Improvements in composite endpoint
(modified Tal score) and O2 consistent with improvement in LOS
Published in the December 2017 Pediatric Pulmonology Journal(1)
17
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Design and Baseline Characteristics
Randomized 67 subjects at 6 sites in Israel with a 11 randomization between 160 ppm NO + supportive care (O2 + hydration) and supportive care alone
Subjects were 0-12 months old with acute bronchiolitis requiring hospitalization with at least 28 weeks of gestation
PE (primary endpoint) the difference in hospital length of stay (LOS)
SE (secondary endpoint) time to clinical improvement using the Modified Tal score (score ge7 and lt10 to enroll le 5 is goal)
SE the difference in time to SpO2 of gt92 SE Safety (specifically NO2 levels and
methemoglobinemia) and Tolerability Treatment was five 30 minute sessions per
day not to exceed 25 treatments All inhalations delivered by airoxygen
blender +NO via a simple mask with a minimum FiO2 of 21
DATA PRESENTED AT THE SEPTEMBER 2018 EUROPEAN RESPIRATORY SOCIETY (ERS)
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
10
Improved operating economics for the hospital
No significant capital investment required for hospitals new to NO
No burdensome inventory and storage requirements
NO supplied as a non-hypoxic gas mixture
No purging procedures or additional safety measures due to NO2 buildup
NO now available to hospitals unable to use NO cylinder systems today
Reduced training burden
Pregnant staff members not impacted
AIT does not have any expenses associated with a manufacturing facility for nitric oxide
AIT does not have any expenses associated with logistics related to nitric oxide cylinders
Hospitals will have significant cost amp logistics Advantages Our device will have significant cost Advantages
Losing the High-Pressure Cylinder is a Significant Gain
11
Circassia world-class specialty biopharma company backed by Astra Zeneca
SnapshotCircassia Pharmaceuticals plcStatus Public company traded on AIM CIR | Stock Price (02282019) GBP 35About Specialty pharmaceutical company founded in 2006 Focused on respiratory diseases based out of the UKIPO date Mar 2014Market Cap (02282019) GBP 130 M | Sales (1H 2018) GBP 284 MLoss (1H 2018) GBP 235 M | Cash in Hand (1H 2018) GBP 508 MCommercial Team US = ~200 | Total Employees ~400Major Shareholders Invesco Asset mgmt Woodford Investment mgmt AstraZeneca own about 67 combinedDirect Sales Force in United States China and certain European Countries
Area of Expertise Strategic Fit with AirNOvent
Respiratory NIOXo Used for asthma management
Tudorzao indicated for the long-term maintenance treatment of bronchospasm associated with COPD including
chronic bronchitis and emphysema
Duaklir (March 31 PDUFA)o Aclidinium bromide amp formoterol fumarate for treating COPD
Nitric Oxide NIOX is a nitric oxide measurement system for monitoring airway inflammation
AirNOvent will be a meaningful product
2018 Circassia company revenues expected to be $62m - $67m Two currently marketed products with a third expected later in 2019 Currently a small hospital presence Just rolling out commercial infrastructure in China
Exposure to US hospitals NIOX is detailed in the hospital and there is overlap in the US with top hospitals that use NO today
12
$3255 million in Total Milestones and 15-20 Royaltyo $105 million received to date
Royalties to AIT on Gross Profito5 on the first $50 million in the US (one time)o5 on the first $20 million in China (one time)o15 up to $100m annually (US amp China combined)o20 above $100m annually (US and China combined)oGross profit defined as net sales less only the cost of AirNOvent and
NO2 filters
PMA filing with FDA is anticipated in the Second Quarter 2019
US commercial launch planned First Half 2020
A Transforming Partnership ndash Transaction Details
Key Terms
High Concentration NO Delivery Opportunitiesbull Bronchiolitisbull Nontuberculous Mycobacteria (NTM)
14
Date Study Indication Primary Results
2011 Phase 1 Safety (n=10) All comers Safety bull No SAEs
2013 ndash 2014Phase 2 double blind randomized (n=43)
Bronchiolitis (all causes)
Safety amp Efficacy
bull No SAEsbull 24 hour reduction in hospital length of stay
2013 - 2014Phase 2 open label
(n=9)Cystic Fibrosis (CF)
Safety amp Efficacy
bull No SAEsbull Lowered bacterial load
2016Compassionate use Israel
(n=2)NTM in
CF patientsEfficacy
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2017Compassionate use National
Institute of Health (n=1)NTM in
CF patientEfficacy
bull No SAEsbull Improvements in clinical endpoints
2017 Pilot open label (N=9)Refractory NTM
abscessusSafety
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2018Pilot study double blind
randomized (n=67)Bronchiolitis (all causes)
Efficacybull No SAEsbull 23 hour reduction in hospital length of stay
Safety First ndash AITrsquos High Concentration NO Delivery for Lung Infections
Our Nitric Oxide Delivery System Has a Demonstrated Safety Record at a concentration of 160 ppm NO
Treatments administered patients Different clinicalsettings
Serious Adverse Events (SAEs)
related to NO
2100+ 85+ 7 0
15
Second Indication Bronchiolitis (BRO) Overview
Bronchiolitis is the leading cause of hospitalization for infants worldwide (1)
~150000 infant hospitalizations per year in the US(2)
Significant impact on the elderly from equivalent viral infections with 177000 hospitalizations per year in the US(3)
No drugs approved for the treatment of BRO patients(4)
Standard of care in the hospital is oxygen and hydration
(1) Scand J Trauma Resusc Emerg Med 2014 22 23 WHO(2) Pel letier et a l Direct medical costs of hospitalizations in the United States Pediatrics 2006(3) CDC (due to RSV only)(4) American Academy of Pediatrics
Bronchiolitis Overview amp Market Dynamics Market Size
AIT estimates US market size to be gt$2 Band projects global market to be similar size to the US market with no competition
AITrsquos goal would be to reduce length of hospitalization in infants
Elderly population trials to follow infants
16
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in length of hospital stay (LOS)
(1) httpsonlinelibrarywileycomdoiepdf101002ppul23905
2014 Trial Design and Highlights
Randomized Prospective Double-blind 43 patients (age 2-12 months) with acute
bronchiolitis (mostly due to RSV) and at least 36 weeks of gestation
N=22 Supportive Care (O₂ amp hydration) N=21 Supportive Care + 160 ppm NO for 30
minutes 5xday up to 5 days Follow up visits 2 3 amp 4 weeks post discharge Single center at Soroka University Medical
Center in Israel Data presented at ATS 2015 in an oral session Reduced length of hospital stay by ~24hrs in
patients who stayed in the hospital for at least 24 hours
No treatment related SAEs Improvements in composite endpoint
(modified Tal score) and O2 consistent with improvement in LOS
Published in the December 2017 Pediatric Pulmonology Journal(1)
17
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Design and Baseline Characteristics
Randomized 67 subjects at 6 sites in Israel with a 11 randomization between 160 ppm NO + supportive care (O2 + hydration) and supportive care alone
Subjects were 0-12 months old with acute bronchiolitis requiring hospitalization with at least 28 weeks of gestation
PE (primary endpoint) the difference in hospital length of stay (LOS)
SE (secondary endpoint) time to clinical improvement using the Modified Tal score (score ge7 and lt10 to enroll le 5 is goal)
SE the difference in time to SpO2 of gt92 SE Safety (specifically NO2 levels and
methemoglobinemia) and Tolerability Treatment was five 30 minute sessions per
day not to exceed 25 treatments All inhalations delivered by airoxygen
blender +NO via a simple mask with a minimum FiO2 of 21
DATA PRESENTED AT THE SEPTEMBER 2018 EUROPEAN RESPIRATORY SOCIETY (ERS)
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
11
Circassia world-class specialty biopharma company backed by Astra Zeneca
SnapshotCircassia Pharmaceuticals plcStatus Public company traded on AIM CIR | Stock Price (02282019) GBP 35About Specialty pharmaceutical company founded in 2006 Focused on respiratory diseases based out of the UKIPO date Mar 2014Market Cap (02282019) GBP 130 M | Sales (1H 2018) GBP 284 MLoss (1H 2018) GBP 235 M | Cash in Hand (1H 2018) GBP 508 MCommercial Team US = ~200 | Total Employees ~400Major Shareholders Invesco Asset mgmt Woodford Investment mgmt AstraZeneca own about 67 combinedDirect Sales Force in United States China and certain European Countries
Area of Expertise Strategic Fit with AirNOvent
Respiratory NIOXo Used for asthma management
Tudorzao indicated for the long-term maintenance treatment of bronchospasm associated with COPD including
chronic bronchitis and emphysema
Duaklir (March 31 PDUFA)o Aclidinium bromide amp formoterol fumarate for treating COPD
Nitric Oxide NIOX is a nitric oxide measurement system for monitoring airway inflammation
AirNOvent will be a meaningful product
2018 Circassia company revenues expected to be $62m - $67m Two currently marketed products with a third expected later in 2019 Currently a small hospital presence Just rolling out commercial infrastructure in China
Exposure to US hospitals NIOX is detailed in the hospital and there is overlap in the US with top hospitals that use NO today
12
$3255 million in Total Milestones and 15-20 Royaltyo $105 million received to date
Royalties to AIT on Gross Profito5 on the first $50 million in the US (one time)o5 on the first $20 million in China (one time)o15 up to $100m annually (US amp China combined)o20 above $100m annually (US and China combined)oGross profit defined as net sales less only the cost of AirNOvent and
NO2 filters
PMA filing with FDA is anticipated in the Second Quarter 2019
US commercial launch planned First Half 2020
A Transforming Partnership ndash Transaction Details
Key Terms
High Concentration NO Delivery Opportunitiesbull Bronchiolitisbull Nontuberculous Mycobacteria (NTM)
14
Date Study Indication Primary Results
2011 Phase 1 Safety (n=10) All comers Safety bull No SAEs
2013 ndash 2014Phase 2 double blind randomized (n=43)
Bronchiolitis (all causes)
Safety amp Efficacy
bull No SAEsbull 24 hour reduction in hospital length of stay
2013 - 2014Phase 2 open label
(n=9)Cystic Fibrosis (CF)
Safety amp Efficacy
bull No SAEsbull Lowered bacterial load
2016Compassionate use Israel
(n=2)NTM in
CF patientsEfficacy
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2017Compassionate use National
Institute of Health (n=1)NTM in
CF patientEfficacy
bull No SAEsbull Improvements in clinical endpoints
2017 Pilot open label (N=9)Refractory NTM
abscessusSafety
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2018Pilot study double blind
randomized (n=67)Bronchiolitis (all causes)
Efficacybull No SAEsbull 23 hour reduction in hospital length of stay
Safety First ndash AITrsquos High Concentration NO Delivery for Lung Infections
Our Nitric Oxide Delivery System Has a Demonstrated Safety Record at a concentration of 160 ppm NO
Treatments administered patients Different clinicalsettings
Serious Adverse Events (SAEs)
related to NO
2100+ 85+ 7 0
15
Second Indication Bronchiolitis (BRO) Overview
Bronchiolitis is the leading cause of hospitalization for infants worldwide (1)
~150000 infant hospitalizations per year in the US(2)
Significant impact on the elderly from equivalent viral infections with 177000 hospitalizations per year in the US(3)
No drugs approved for the treatment of BRO patients(4)
Standard of care in the hospital is oxygen and hydration
(1) Scand J Trauma Resusc Emerg Med 2014 22 23 WHO(2) Pel letier et a l Direct medical costs of hospitalizations in the United States Pediatrics 2006(3) CDC (due to RSV only)(4) American Academy of Pediatrics
Bronchiolitis Overview amp Market Dynamics Market Size
AIT estimates US market size to be gt$2 Band projects global market to be similar size to the US market with no competition
AITrsquos goal would be to reduce length of hospitalization in infants
Elderly population trials to follow infants
16
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in length of hospital stay (LOS)
(1) httpsonlinelibrarywileycomdoiepdf101002ppul23905
2014 Trial Design and Highlights
Randomized Prospective Double-blind 43 patients (age 2-12 months) with acute
bronchiolitis (mostly due to RSV) and at least 36 weeks of gestation
N=22 Supportive Care (O₂ amp hydration) N=21 Supportive Care + 160 ppm NO for 30
minutes 5xday up to 5 days Follow up visits 2 3 amp 4 weeks post discharge Single center at Soroka University Medical
Center in Israel Data presented at ATS 2015 in an oral session Reduced length of hospital stay by ~24hrs in
patients who stayed in the hospital for at least 24 hours
No treatment related SAEs Improvements in composite endpoint
(modified Tal score) and O2 consistent with improvement in LOS
Published in the December 2017 Pediatric Pulmonology Journal(1)
17
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Design and Baseline Characteristics
Randomized 67 subjects at 6 sites in Israel with a 11 randomization between 160 ppm NO + supportive care (O2 + hydration) and supportive care alone
Subjects were 0-12 months old with acute bronchiolitis requiring hospitalization with at least 28 weeks of gestation
PE (primary endpoint) the difference in hospital length of stay (LOS)
SE (secondary endpoint) time to clinical improvement using the Modified Tal score (score ge7 and lt10 to enroll le 5 is goal)
SE the difference in time to SpO2 of gt92 SE Safety (specifically NO2 levels and
methemoglobinemia) and Tolerability Treatment was five 30 minute sessions per
day not to exceed 25 treatments All inhalations delivered by airoxygen
blender +NO via a simple mask with a minimum FiO2 of 21
DATA PRESENTED AT THE SEPTEMBER 2018 EUROPEAN RESPIRATORY SOCIETY (ERS)
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
12
$3255 million in Total Milestones and 15-20 Royaltyo $105 million received to date
Royalties to AIT on Gross Profito5 on the first $50 million in the US (one time)o5 on the first $20 million in China (one time)o15 up to $100m annually (US amp China combined)o20 above $100m annually (US and China combined)oGross profit defined as net sales less only the cost of AirNOvent and
NO2 filters
PMA filing with FDA is anticipated in the Second Quarter 2019
US commercial launch planned First Half 2020
A Transforming Partnership ndash Transaction Details
Key Terms
High Concentration NO Delivery Opportunitiesbull Bronchiolitisbull Nontuberculous Mycobacteria (NTM)
14
Date Study Indication Primary Results
2011 Phase 1 Safety (n=10) All comers Safety bull No SAEs
2013 ndash 2014Phase 2 double blind randomized (n=43)
Bronchiolitis (all causes)
Safety amp Efficacy
bull No SAEsbull 24 hour reduction in hospital length of stay
2013 - 2014Phase 2 open label
(n=9)Cystic Fibrosis (CF)
Safety amp Efficacy
bull No SAEsbull Lowered bacterial load
2016Compassionate use Israel
(n=2)NTM in
CF patientsEfficacy
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2017Compassionate use National
Institute of Health (n=1)NTM in
CF patientEfficacy
bull No SAEsbull Improvements in clinical endpoints
2017 Pilot open label (N=9)Refractory NTM
abscessusSafety
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2018Pilot study double blind
randomized (n=67)Bronchiolitis (all causes)
Efficacybull No SAEsbull 23 hour reduction in hospital length of stay
Safety First ndash AITrsquos High Concentration NO Delivery for Lung Infections
Our Nitric Oxide Delivery System Has a Demonstrated Safety Record at a concentration of 160 ppm NO
Treatments administered patients Different clinicalsettings
Serious Adverse Events (SAEs)
related to NO
2100+ 85+ 7 0
15
Second Indication Bronchiolitis (BRO) Overview
Bronchiolitis is the leading cause of hospitalization for infants worldwide (1)
~150000 infant hospitalizations per year in the US(2)
Significant impact on the elderly from equivalent viral infections with 177000 hospitalizations per year in the US(3)
No drugs approved for the treatment of BRO patients(4)
Standard of care in the hospital is oxygen and hydration
(1) Scand J Trauma Resusc Emerg Med 2014 22 23 WHO(2) Pel letier et a l Direct medical costs of hospitalizations in the United States Pediatrics 2006(3) CDC (due to RSV only)(4) American Academy of Pediatrics
Bronchiolitis Overview amp Market Dynamics Market Size
AIT estimates US market size to be gt$2 Band projects global market to be similar size to the US market with no competition
AITrsquos goal would be to reduce length of hospitalization in infants
Elderly population trials to follow infants
16
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in length of hospital stay (LOS)
(1) httpsonlinelibrarywileycomdoiepdf101002ppul23905
2014 Trial Design and Highlights
Randomized Prospective Double-blind 43 patients (age 2-12 months) with acute
bronchiolitis (mostly due to RSV) and at least 36 weeks of gestation
N=22 Supportive Care (O₂ amp hydration) N=21 Supportive Care + 160 ppm NO for 30
minutes 5xday up to 5 days Follow up visits 2 3 amp 4 weeks post discharge Single center at Soroka University Medical
Center in Israel Data presented at ATS 2015 in an oral session Reduced length of hospital stay by ~24hrs in
patients who stayed in the hospital for at least 24 hours
No treatment related SAEs Improvements in composite endpoint
(modified Tal score) and O2 consistent with improvement in LOS
Published in the December 2017 Pediatric Pulmonology Journal(1)
17
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Design and Baseline Characteristics
Randomized 67 subjects at 6 sites in Israel with a 11 randomization between 160 ppm NO + supportive care (O2 + hydration) and supportive care alone
Subjects were 0-12 months old with acute bronchiolitis requiring hospitalization with at least 28 weeks of gestation
PE (primary endpoint) the difference in hospital length of stay (LOS)
SE (secondary endpoint) time to clinical improvement using the Modified Tal score (score ge7 and lt10 to enroll le 5 is goal)
SE the difference in time to SpO2 of gt92 SE Safety (specifically NO2 levels and
methemoglobinemia) and Tolerability Treatment was five 30 minute sessions per
day not to exceed 25 treatments All inhalations delivered by airoxygen
blender +NO via a simple mask with a minimum FiO2 of 21
DATA PRESENTED AT THE SEPTEMBER 2018 EUROPEAN RESPIRATORY SOCIETY (ERS)
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
High Concentration NO Delivery Opportunitiesbull Bronchiolitisbull Nontuberculous Mycobacteria (NTM)
14
Date Study Indication Primary Results
2011 Phase 1 Safety (n=10) All comers Safety bull No SAEs
2013 ndash 2014Phase 2 double blind randomized (n=43)
Bronchiolitis (all causes)
Safety amp Efficacy
bull No SAEsbull 24 hour reduction in hospital length of stay
2013 - 2014Phase 2 open label
(n=9)Cystic Fibrosis (CF)
Safety amp Efficacy
bull No SAEsbull Lowered bacterial load
2016Compassionate use Israel
(n=2)NTM in
CF patientsEfficacy
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2017Compassionate use National
Institute of Health (n=1)NTM in
CF patientEfficacy
bull No SAEsbull Improvements in clinical endpoints
2017 Pilot open label (N=9)Refractory NTM
abscessusSafety
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2018Pilot study double blind
randomized (n=67)Bronchiolitis (all causes)
Efficacybull No SAEsbull 23 hour reduction in hospital length of stay
Safety First ndash AITrsquos High Concentration NO Delivery for Lung Infections
Our Nitric Oxide Delivery System Has a Demonstrated Safety Record at a concentration of 160 ppm NO
Treatments administered patients Different clinicalsettings
Serious Adverse Events (SAEs)
related to NO
2100+ 85+ 7 0
15
Second Indication Bronchiolitis (BRO) Overview
Bronchiolitis is the leading cause of hospitalization for infants worldwide (1)
~150000 infant hospitalizations per year in the US(2)
Significant impact on the elderly from equivalent viral infections with 177000 hospitalizations per year in the US(3)
No drugs approved for the treatment of BRO patients(4)
Standard of care in the hospital is oxygen and hydration
(1) Scand J Trauma Resusc Emerg Med 2014 22 23 WHO(2) Pel letier et a l Direct medical costs of hospitalizations in the United States Pediatrics 2006(3) CDC (due to RSV only)(4) American Academy of Pediatrics
Bronchiolitis Overview amp Market Dynamics Market Size
AIT estimates US market size to be gt$2 Band projects global market to be similar size to the US market with no competition
AITrsquos goal would be to reduce length of hospitalization in infants
Elderly population trials to follow infants
16
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in length of hospital stay (LOS)
(1) httpsonlinelibrarywileycomdoiepdf101002ppul23905
2014 Trial Design and Highlights
Randomized Prospective Double-blind 43 patients (age 2-12 months) with acute
bronchiolitis (mostly due to RSV) and at least 36 weeks of gestation
N=22 Supportive Care (O₂ amp hydration) N=21 Supportive Care + 160 ppm NO for 30
minutes 5xday up to 5 days Follow up visits 2 3 amp 4 weeks post discharge Single center at Soroka University Medical
Center in Israel Data presented at ATS 2015 in an oral session Reduced length of hospital stay by ~24hrs in
patients who stayed in the hospital for at least 24 hours
No treatment related SAEs Improvements in composite endpoint
(modified Tal score) and O2 consistent with improvement in LOS
Published in the December 2017 Pediatric Pulmonology Journal(1)
17
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Design and Baseline Characteristics
Randomized 67 subjects at 6 sites in Israel with a 11 randomization between 160 ppm NO + supportive care (O2 + hydration) and supportive care alone
Subjects were 0-12 months old with acute bronchiolitis requiring hospitalization with at least 28 weeks of gestation
PE (primary endpoint) the difference in hospital length of stay (LOS)
SE (secondary endpoint) time to clinical improvement using the Modified Tal score (score ge7 and lt10 to enroll le 5 is goal)
SE the difference in time to SpO2 of gt92 SE Safety (specifically NO2 levels and
methemoglobinemia) and Tolerability Treatment was five 30 minute sessions per
day not to exceed 25 treatments All inhalations delivered by airoxygen
blender +NO via a simple mask with a minimum FiO2 of 21
DATA PRESENTED AT THE SEPTEMBER 2018 EUROPEAN RESPIRATORY SOCIETY (ERS)
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
14
Date Study Indication Primary Results
2011 Phase 1 Safety (n=10) All comers Safety bull No SAEs
2013 ndash 2014Phase 2 double blind randomized (n=43)
Bronchiolitis (all causes)
Safety amp Efficacy
bull No SAEsbull 24 hour reduction in hospital length of stay
2013 - 2014Phase 2 open label
(n=9)Cystic Fibrosis (CF)
Safety amp Efficacy
bull No SAEsbull Lowered bacterial load
2016Compassionate use Israel
(n=2)NTM in
CF patientsEfficacy
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2017Compassionate use National
Institute of Health (n=1)NTM in
CF patientEfficacy
bull No SAEsbull Improvements in clinical endpoints
2017 Pilot open label (N=9)Refractory NTM
abscessusSafety
bull No SAEsbull Improvements in clinical amp surrogate endpoints
2018Pilot study double blind
randomized (n=67)Bronchiolitis (all causes)
Efficacybull No SAEsbull 23 hour reduction in hospital length of stay
Safety First ndash AITrsquos High Concentration NO Delivery for Lung Infections
Our Nitric Oxide Delivery System Has a Demonstrated Safety Record at a concentration of 160 ppm NO
Treatments administered patients Different clinicalsettings
Serious Adverse Events (SAEs)
related to NO
2100+ 85+ 7 0
15
Second Indication Bronchiolitis (BRO) Overview
Bronchiolitis is the leading cause of hospitalization for infants worldwide (1)
~150000 infant hospitalizations per year in the US(2)
Significant impact on the elderly from equivalent viral infections with 177000 hospitalizations per year in the US(3)
No drugs approved for the treatment of BRO patients(4)
Standard of care in the hospital is oxygen and hydration
(1) Scand J Trauma Resusc Emerg Med 2014 22 23 WHO(2) Pel letier et a l Direct medical costs of hospitalizations in the United States Pediatrics 2006(3) CDC (due to RSV only)(4) American Academy of Pediatrics
Bronchiolitis Overview amp Market Dynamics Market Size
AIT estimates US market size to be gt$2 Band projects global market to be similar size to the US market with no competition
AITrsquos goal would be to reduce length of hospitalization in infants
Elderly population trials to follow infants
16
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in length of hospital stay (LOS)
(1) httpsonlinelibrarywileycomdoiepdf101002ppul23905
2014 Trial Design and Highlights
Randomized Prospective Double-blind 43 patients (age 2-12 months) with acute
bronchiolitis (mostly due to RSV) and at least 36 weeks of gestation
N=22 Supportive Care (O₂ amp hydration) N=21 Supportive Care + 160 ppm NO for 30
minutes 5xday up to 5 days Follow up visits 2 3 amp 4 weeks post discharge Single center at Soroka University Medical
Center in Israel Data presented at ATS 2015 in an oral session Reduced length of hospital stay by ~24hrs in
patients who stayed in the hospital for at least 24 hours
No treatment related SAEs Improvements in composite endpoint
(modified Tal score) and O2 consistent with improvement in LOS
Published in the December 2017 Pediatric Pulmonology Journal(1)
17
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Design and Baseline Characteristics
Randomized 67 subjects at 6 sites in Israel with a 11 randomization between 160 ppm NO + supportive care (O2 + hydration) and supportive care alone
Subjects were 0-12 months old with acute bronchiolitis requiring hospitalization with at least 28 weeks of gestation
PE (primary endpoint) the difference in hospital length of stay (LOS)
SE (secondary endpoint) time to clinical improvement using the Modified Tal score (score ge7 and lt10 to enroll le 5 is goal)
SE the difference in time to SpO2 of gt92 SE Safety (specifically NO2 levels and
methemoglobinemia) and Tolerability Treatment was five 30 minute sessions per
day not to exceed 25 treatments All inhalations delivered by airoxygen
blender +NO via a simple mask with a minimum FiO2 of 21
DATA PRESENTED AT THE SEPTEMBER 2018 EUROPEAN RESPIRATORY SOCIETY (ERS)
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
15
Second Indication Bronchiolitis (BRO) Overview
Bronchiolitis is the leading cause of hospitalization for infants worldwide (1)
~150000 infant hospitalizations per year in the US(2)
Significant impact on the elderly from equivalent viral infections with 177000 hospitalizations per year in the US(3)
No drugs approved for the treatment of BRO patients(4)
Standard of care in the hospital is oxygen and hydration
(1) Scand J Trauma Resusc Emerg Med 2014 22 23 WHO(2) Pel letier et a l Direct medical costs of hospitalizations in the United States Pediatrics 2006(3) CDC (due to RSV only)(4) American Academy of Pediatrics
Bronchiolitis Overview amp Market Dynamics Market Size
AIT estimates US market size to be gt$2 Band projects global market to be similar size to the US market with no competition
AITrsquos goal would be to reduce length of hospitalization in infants
Elderly population trials to follow infants
16
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in length of hospital stay (LOS)
(1) httpsonlinelibrarywileycomdoiepdf101002ppul23905
2014 Trial Design and Highlights
Randomized Prospective Double-blind 43 patients (age 2-12 months) with acute
bronchiolitis (mostly due to RSV) and at least 36 weeks of gestation
N=22 Supportive Care (O₂ amp hydration) N=21 Supportive Care + 160 ppm NO for 30
minutes 5xday up to 5 days Follow up visits 2 3 amp 4 weeks post discharge Single center at Soroka University Medical
Center in Israel Data presented at ATS 2015 in an oral session Reduced length of hospital stay by ~24hrs in
patients who stayed in the hospital for at least 24 hours
No treatment related SAEs Improvements in composite endpoint
(modified Tal score) and O2 consistent with improvement in LOS
Published in the December 2017 Pediatric Pulmonology Journal(1)
17
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Design and Baseline Characteristics
Randomized 67 subjects at 6 sites in Israel with a 11 randomization between 160 ppm NO + supportive care (O2 + hydration) and supportive care alone
Subjects were 0-12 months old with acute bronchiolitis requiring hospitalization with at least 28 weeks of gestation
PE (primary endpoint) the difference in hospital length of stay (LOS)
SE (secondary endpoint) time to clinical improvement using the Modified Tal score (score ge7 and lt10 to enroll le 5 is goal)
SE the difference in time to SpO2 of gt92 SE Safety (specifically NO2 levels and
methemoglobinemia) and Tolerability Treatment was five 30 minute sessions per
day not to exceed 25 treatments All inhalations delivered by airoxygen
blender +NO via a simple mask with a minimum FiO2 of 21
DATA PRESENTED AT THE SEPTEMBER 2018 EUROPEAN RESPIRATORY SOCIETY (ERS)
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
16
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in length of hospital stay (LOS)
(1) httpsonlinelibrarywileycomdoiepdf101002ppul23905
2014 Trial Design and Highlights
Randomized Prospective Double-blind 43 patients (age 2-12 months) with acute
bronchiolitis (mostly due to RSV) and at least 36 weeks of gestation
N=22 Supportive Care (O₂ amp hydration) N=21 Supportive Care + 160 ppm NO for 30
minutes 5xday up to 5 days Follow up visits 2 3 amp 4 weeks post discharge Single center at Soroka University Medical
Center in Israel Data presented at ATS 2015 in an oral session Reduced length of hospital stay by ~24hrs in
patients who stayed in the hospital for at least 24 hours
No treatment related SAEs Improvements in composite endpoint
(modified Tal score) and O2 consistent with improvement in LOS
Published in the December 2017 Pediatric Pulmonology Journal(1)
17
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Design and Baseline Characteristics
Randomized 67 subjects at 6 sites in Israel with a 11 randomization between 160 ppm NO + supportive care (O2 + hydration) and supportive care alone
Subjects were 0-12 months old with acute bronchiolitis requiring hospitalization with at least 28 weeks of gestation
PE (primary endpoint) the difference in hospital length of stay (LOS)
SE (secondary endpoint) time to clinical improvement using the Modified Tal score (score ge7 and lt10 to enroll le 5 is goal)
SE the difference in time to SpO2 of gt92 SE Safety (specifically NO2 levels and
methemoglobinemia) and Tolerability Treatment was five 30 minute sessions per
day not to exceed 25 treatments All inhalations delivered by airoxygen
blender +NO via a simple mask with a minimum FiO2 of 21
DATA PRESENTED AT THE SEPTEMBER 2018 EUROPEAN RESPIRATORY SOCIETY (ERS)
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
17
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Design and Baseline Characteristics
Randomized 67 subjects at 6 sites in Israel with a 11 randomization between 160 ppm NO + supportive care (O2 + hydration) and supportive care alone
Subjects were 0-12 months old with acute bronchiolitis requiring hospitalization with at least 28 weeks of gestation
PE (primary endpoint) the difference in hospital length of stay (LOS)
SE (secondary endpoint) time to clinical improvement using the Modified Tal score (score ge7 and lt10 to enroll le 5 is goal)
SE the difference in time to SpO2 of gt92 SE Safety (specifically NO2 levels and
methemoglobinemia) and Tolerability Treatment was five 30 minute sessions per
day not to exceed 25 treatments All inhalations delivered by airoxygen
blender +NO via a simple mask with a minimum FiO2 of 21
DATA PRESENTED AT THE SEPTEMBER 2018 EUROPEAN RESPIRATORY SOCIETY (ERS)
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
18
Completed Two Pilot Bronchiolitis Trials
Data from both Pilot Bronchiolitis trials demonstrated a significant reduction in LOS
2018 Trial Results Presented at ERS 2018
bull Primary endpoint of LOS calculated fromenrollment to time of hospital discharge
bull Welchrsquos t-test p=0085 ndash study was notpowered for statistical significance
bull Secondary endpoint of time to oxygen saturation of gt92 calculated from enrollment
bull Welchrsquos t-test p=0053
bull Secondary endpoint of time to modified Tal composite score of lt5 calculated from enrollment
bull Welchrsquos t-test p=020
Pivotal Study to Begin in the US in 4Q19 and Complete in 2Q20
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
19
Losing the High-Pressure Cylinder Makes Home Use a Technical Reality
Losing high pressure cylinder makes NO accessible in a number of settings
Our system is simple to use and patients can self-administer
4 simple steps
Plug in any standard electrical outlet
Insert AIT Smart Filter
Position mask on face
Press GO
Light-weight and easy to transport
Can be used with any standard electrical outlet
Potential use in both acute and chronic lung disease
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
20
Third Indication Non Tuberculous Mycobacteria (NTM)
NTM is an FDA disease area of focus with limited options Patients can die within a few years (1)
Acquired by inhalation from the environment Water thought to be the main source Warmer climates have higher infection rates Patient to patient transmission possible
How is NTM Acquired (2) Who is at risk (2)
Underlying lung disease andor genetic predisposition Cystic Fibrosis (CF) patients COPD (chronic obstructive pulmonary disease) Bronchiectasis patients Immunosuppressive therapy
NTM Market Dynamics
There are a limitednumber of players inhuman studies for NTM
Median survival for MAC is13 years while for non-MAC NTM it is 46 years (6)
Over 180k NTM cases wereestimated for 2014 in theUnited States(3)
NTM costs estimated at $17b(3)
with MABSC costs gt 2x MACcosts
37 of NTM confirmed CysticFibrosis patients in the US areMABSC (4)
AIT is initially targeting NTM abscessus (MABSC) the most aggressive and difficult to treat form of
NTM AIT expects to seek approval in NTM MAC (mycobacterium avium
complex) following MABSC approval
20 - 25 of all NTMcases in a South Koreandatabase are MABSC (5)
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf (2) Data wwwntmfactscom FDA(3) Strolloet al The Burden of Pulmonary Nontuberculous Mycobacterial Pub 27-July-2015(4) Data presented at ATS 2017 (Derek Low et al Medical University of South Carolina)
(5) Data presented at ATS 2017 (Keun Burn Chung et al Seoul National University College of Medicine) (6) Kotilainen H et al ldquoClinical Findings in Relation to Mortality in Non-Tuberculous Mycobacterial Infections
Patients with Mycobacterium Avium Complex Have Better Survival than Patients with Other Mycobacteriardquo European Journal of Clinical Microbiology amp Infectious Diseases 349 (2015)
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
21
237
448 431
301 277
0
5
10
15
20
25
30
35
40
45
50
Day 7 Day 14 Day 21 Day 51 Day 81
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)Proprietary NO formulation yielded positive clinical results in humans in its single arm pilot NTM study
6MW Mean Inc in Distance (meters) v Baseline Mean change in FEV1 from Baseline
9 CF patients with refractory MABSC were treated at 3 centers in Israel with NO added to background antibiotic therapy
160 ppm NO was given via mask for 30 min 5xday for 14 days and 3xday for 7 days
Primary endpoint of safety was met with no NO-related serious adverse events (SAEs) observed
Key secondary endpoints of 6-minute walk (6MW) and FEV1 are shown in the charts below
Bacterial load as measured by qPCR showed a 65 reduction at day 81 versus baseline
One patient was culture negative at Day 51 and Day 81
Quality-of-Life data showed positive trends on relevant questions (SF-36 used)
Tolerability not an issue as no patient requested that any treatment be stopped or not administered
3 patients treated under compassionate use experienced similar results (1 treated at NIH with generator 1 culture conversion)
17
28
41
32
-16-20
-10
00
10
20
30
40
50
Day 7 Day 14 Day 21 Day 51 Day 81
On Therapy Off Therapy On Therapy Off Therapy
Source AIT management
DATA PRESENTED IN AN ORAL SESSION AT AMERICAN THORACIC SOCIETY (ATS) 2018
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
22
Pulmonary Infections eg Non Tuberculous Mycobacteria (NTM)
NO has direct killing effect on multi-drug resistant M abscessus and P aeruginosa in vitro
Source AIT management
bull M abscessus B1 bacteria cultured in artificialsputum were treated with increasing doses ofNO (160 250 and 300ppm) for up to 10hrs
bull Time-kill curves show susceptibility of Mabscessus B1 (rough) B5 (smooth) B8 (rough)and MRD (rough) clinical isolates cultured inartificial sputum to continuous 250ppm NOtreatment All M abscessus strains show
susceptibility to NO treatment
bull P aeruginosa were cultured at 106 CFUml inartificial sputum (2ml planktonic) and treatedcontinuously with 200ppm NO for up to 10hrs
DATA PRESENTED AT THE 3RD WORLD BRONCHIECTASIS CONFERENCE IN 2018
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
23
AITrsquos Goal is to initiate a pivotal trial in United States in 2020
(1) httpswwwfdagovdownloadsDrugsNewsEventsUCM471341pdf
FDA Guidance(1)AIT Plans for Approval
FDA is asking for ldquoevidence of efficacy for a clinically meaningful outcome evaluated in adequate and well controlled trialsrdquo
Based on discussions with FDA AIT believes a placebo controlled trial with a PE of 6MWD plus relevant SE endpoints (FEV1 bacterial load in sputum culture conversion QoL safety) will be adequate for approval
Prior to a pivotal study a 12 week single arm multi-center pilot study in the US will begin in 2H19 with the endpoints listed above where patients infected with either MABSC or MAC will self-administer at home potentially at concentrations gt160 ppm
Extensive in-vitro data already exists to support the direct killing effect of NO on MABSC and more studies will be available throughout 2019 on NTM and other bacteria
AIT expects to make its NO therapy available to NTM patients in the US in 2023
Potentially other severe chronic and refractory infections such as Pseudomonas Aeruginosa can be targeted
Pulmonary Infections Non Tuberculous Mycobacteria (NTM)
Timeline amp Plan for Registration in the US
2020 2021 2022 2023
Pivotal Trial initiation planned
Pivotal Trial completion planned
FDA approval anticipated
2019
Pilot Study start anticipated at-home use 12 weeks higher concentrations
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
24
AIT Active Pipeline amp Market Size
Product IndicationDevelopment
StatusKey Dates
US Sales Potential
Worldwide Sales
Potential
AIT-PH (Pulmonary
Hypertension)In-Hospital Use
Commercial system in
development
FDA submission expected 2Q19
Launch first half 2020
gt$300m
Partnered with
gt$600m
AIT-BRO (Bronchiolitis)
Bronchiolitis Pilot phase complete
Pivotal Study expected during
20192020 Winter
Launch 2021
gt$500m gt$12b
AIT-SLI(Severe Lung
Infections)
NTM Mycobacterium
Abscessus Complex (MABSC)
9 pt study complete
2nd pilot study to have higher ppm NO and
MAC infection
2019 start for pilot study with
self-administration
at home
Launch 2023
gt$1b gt$25b
Al l dates are based on projections anticipated first launch on a global basis pending appropriate regulatory approvals Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
25
AIT Inactive Pipeline amp Status
Product Indication Development StatusWorldwide Sales
Potential
AIT-SLIVarious bacterial
infectionsPilot study initiation anticipated in 2020
Multi Billion $ Opportunities
AIT-COPD (Chronic Obstructive Pulmonary Disease)
Exacerbation caused by any type of infection
(treatment and prevention)
Proof of concept initiation anticipated in
2020
AIT-PH At-Home UseProof of concept
initiation anticipated in 2021
CF (Cystic Fibrosis)
Acute infections and Chronic Therapy
Trials to begin in 2021
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
26
Patent Portfolio
Development of this pipeline is conditional on obtaining additional financing Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential
Issued patent expirations 2019 through 2033
Pending patents if issued may extend the last expiration through 2037
AIT believes that its patent portfolio is strong and broad
The generator
The breathing circuit
NO concentration
NO action in the body
NO dosing
NO2 filter
Method of Use
gt20 Issued Patents and gt10 Pending Patents Across Major Global Markets
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
27
Financial Profile
As of February 1 2019
Cash $13 million
Debt $0
Expected Monthly Burn is$600000-$650000
Corporate HQs in New York
PPHN FDA regulatory filing anticipated 2Qcalendar 2019 with launch in 1H2020
Positive NTM data presented at ATS andWorld Bronchiectasis 2018
Positive BRO data presented at ERS 2018
Current cash runway through 1Q2020
Includes BRO trial in 201920 winter
Excludes further milestones fromcurrent or future PPHN deals
Excludes further use of stockpurchase agreement
$12m milestone associated with PPHNpartnership expected in 1H 2020
$20m stock purchase agreement in placethrough August of 2021 (~$19m remains)
Ticker AITB
Exchange OTCQB
Share Price $500 (as of March 11 2019)
Shares Outstanding 86m
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
28
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital Millennium
Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Duncan FatkinCCO
25+ yearsrsquo experience across global medical device amp biopharma companies including Becton Dickinson Zimmer Biomet amp DePuyJampJ
Strong track record of commercialization leading marketing amp sales Member of the Chartered Institute of Marketing for 30 years
Giora DavidaiCMO
Prior to industry was a pediatric nephrologist at Duke 23 yearsrsquo experience in clinical research with gt10 drugs approved
including Phase 2-IV development of Spiriva Previously worked at Boehringer Ingelheim and Glaxo
Douglas BeckCFO
Over 10 years serving as CFO for 5 companies including 3 Biotechs Has helped companies raise over $100 mill ion in equity amp debt Serves on the New York State Society of CPAs Chief Financial Officer amp
SEC committee
Frederick MontgomeryVP Medical Systems
Developed all FDA approved NO systems used by Ino Therapeutics Ikaria and Mallinckrodt
Author on over 30 NO related patents including InoPulse Previously worked at Ikaria and NitricGen
Rhona ShankerVP Regulatory Affairs
35 years of FDA experience 22 years at the Device Division of FDA with the final 6 years as an
expert device reviewer
Ali ArdakaniSVP Device amp BD
20 years of development of therapeutics amp devices including two FDA approved NO systems
Responsible for multiple drug amp device global partnerships incl CareFusion Bayer Eisai etc
Management Team
Highly experienced and successful team of industry experts
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
29
Steve LisiChairman and CEO
18 years experience as a Healthcare investor 3 years as SVP Head of Strategy and BD at Avadel (AVDL) Previously worked at Mehta and Isaly SAC Capital
Millennium Management and was a partner at Deerfield
Amir AvnielPresident amp COO
15 years of executive-level experience in finance business development and operations including MampA
Previously worked at Rosetta Genomics (Founder) Rosetta Green (sold to Monsanto) and Monsanto
Ron BentsurDirector
Director since August 2015 CEO and Director of UroGen Pharma since 2015 Previous CEO and Director of Keryx Biopharmaceuticals Previous CEO of XTL Biopharmaceuticals
Erick LuceraDirector
Director since August 2017 CFO at Valeritas Previous CFO of Viventia Bio Previous VP Corporate Development at Aratana
Yoori LeeDirector
Director since January 2018 Co-founder and President of Trio Health Advisory Group 15 years at Leerink Partners LLC
Helped found the MEDACorp network
Bill ForbesDirector
President and CEO of Vivelix Pharmaceuticals Ltd Former Chief Development Officer and Head of Medical
and RampD as Salix Pharmaceuticals Responsible for more than a dozen NDASNDA approvals
Robert F CareyDirector
Director since February 2019 Served as Executive VP and Chief Business Officer at
Horizon Pharma Previous Managing Director at JMP Securities
Board of Directors
Board of Directors with vast industry experience
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
30
Hugh OrsquoBrodovich MD
Andrew Collin MD
John P Clancy MD
Richard Malley MD
Scientific Advisory Board
Scientific Advisory Board comprised of world renowned thought leaders
Hannah Blau MD
David Greenberg MD
Prof Yossef Av-Gay PhD
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
31
AIT Therapeutics Revolutionizing the Delivery of Nitric Oxide (NO)
AIT is a medical device company that has developed a platform Nitric Oxide generator system
Experienced Management Team
Deep industry experience developing NO delivery systems
Proven experience in gaining regulatory approvals for both drugs anddevices on a global basis
Demonstrated Safety Profile
More than 2100 treatments in over 85 patients across 7 studies at NOconcentrations gt150 parts per million (ppm)
No Serious Adverse Events (SAEs) related to NO therapy
Proprietary Nitric Oxide Technology
Platform
AITrsquos propriety generator and delivery system generates NO from ambientair eliminating the need for expensive and cumbersome cylinders
AITrsquos system provides significant advantages over approved NO cylinderbased systems currently used in hospitals around the world AND may allowfor use in the home setting targeting certain respiratory conditions
First 3 Indications Address Large
Markets
Target Patient PopulationUS Sales
Potential WW Sales Potential
Launch Year
Pulmonary Hypertension (in-hospital)
gt$300m gt$600m 2020
Bronchiolitis (in-hospital) gt$500m gt$12b 2021
Severe Lung Infections (at-home) gt$1b gt$25b 2023
Al l figures are Company estimates for peak year sales Global Sales Potential includes US Sales Potential Anticipated first launch on a global basis pending appropriate regulatory approvals
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom
Transformational Therapies to Treat Lung Infections amp Pulmonary Disease
For more information contactSteve Lisi CEO
+1-516-665-8200steveait-pharmcomwwwait-pharmcom