corporate presentation april 2020 · corporate presentation april 2020. ... omega-3. 1h 2021 q4...
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www.matinasbiopharma.comNYSE AMERICAN: MTNB
Corporate PresentationApril 2020
Forward-Looking Statement
This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including those relating to the Company’s product development, clinical and regulatory timelines, market opportunity, cash flow and other statements that are predictive in nature, that depend upon or refer to future events or conditions. All statements other than statements of historical fact are statements that could be forward-looking statements. Forward-looking statements include words such as “expects,” “anticipates,” “intends,” “plans,“ “could,” “believes,” “estimates” and similar expressions. These statements involve known and unknown risks, uncertainties and other factors which may cause actual results to be materially different from any future results expressed or implied by the forward-looking statements. Forward-looking statements are subject to a number of risks and uncertainties, including, but not limited to, our ability to obtain additional capital to meet our liquidity needs on acceptable terms, or at all, including the additional capital which will be necessary to complete the clinical trials of our product candidates; our ability to successfully complete research and further development and commercialization of our product candidates; the uncertainties inherent in clinical testing; the timing, cost and uncertainty of obtaining regulatory approvals; our ability to protect the Company's intellectual property; the loss of any executive officers or key personnel or consultants; competition; changes in the regulatory landscape or the imposition of regulations that affect the Company's products; and the other factors listed under “Risk Factors” in our filings with the SEC, including Forms 10-K, 10-Q and 8-K. Investors are cautioned not to place undue reliance on such forward-looking statements, which speak only as of the date of this release. Except as may be required by law, the Company does not undertake any obligation to release publicly any revisions to such forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. Matinas BioPharma’s product candidates are all in a development stage and are not available for sale or use.
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NYSE AMERICAN: MTNB Founded: 2013Based in:
Bedminster, NJ
Company Overview
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MAT9001Cardiovascular and Metabolic Conditions
LNC PlatformLipid Nano-Crystal Delivery System
§ Potential best-in-class drug focused on severe hypertriglyceridemia with potential expansion into multi-billion-dollar market
§ Head-to-head data demonstrating superior triglyceride lowering and EPA levels against market leading drug, Vascepa®
§ Key additional head to head data from ENHANCE-IT study vs. Vascepa®
§ Clear differentiation from currently approved prescription omega-3 products
§ Safe, oral, targeted intracellular delivery of potent medicines
§ MAT2203 - Oral Amphotericin B, a broad-spectrum antifungal agent. Program financially supported by the National Institutes of Health
§ EnACT study for MAT2203 in cryptococcal meningitis represents gateway opportunity for the treatment of invasive fungal infections. Expected cohort updates during 2020.
§ Feasibility evaluations with three Big Pharma companies across multiple compounds
Cash runway ($70+ million) into fourth quarter of 2022 -through multiple data milestones and class catalysts
Key Milestones Position Company for Near-Term Value-Driving Events
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Initiated NIH-sponsored EnACT study of MAT2203 in Cryptococcal Meningitis. Cohort efficacy updates 2H 2020. Full data expected in 2H 2021.
Vascepa sNDA approval potentially opens the omega-3 class to 40+ million patients.
Commence ENHANCE-IT head-to-head study of MAT9001 vs Vascepa (n=100). Designed to further demonstrate superiority.
Expected start phase 3 program for MAT9001. Topline data expected in late 2022.
Planned end-of-phase 2 meeting with FDA for MAT9001 to discuss Phase 3 program design.
Topline results expected from MAT9001 ENHANCE-IT study, with potential to further validate MAT9001 as potential best in class prescription-only omega-3.
1H 2021
Q4 2020/Q1 2021
Q3 2020October 2019
Dec 2019
Q2 2020
MAT9001 Overview
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MAT9001
MAT9001 - Unique Prescription-Only Omega-3 Fatty AcidFirst Omega-3 Specifically Designed to Treat Hypertriglyceridemia and Dyslipidemia
MAT9001*Vascepa* Epanova*Lovaza*
EPA
DPAother
EPA
DHA
EPA
EPA
DHA
other
FREE FATTY ACIDS
Highly Bioavailable
ETHYL ESTERS
Less Bioavailable
1st Generation 2nd Gen
DPA – Most Potent
DHA – More Potent
EPA – Less Potent
Individual Omega-3 Effect on Triglyceride Lowering
other
*estimated compositionMAT9001 specifically designed
for CV applicationsRepurposed products
1
2
2
1: Drouin G et al. J Nutr Biochem 2019; 63: 186-96 / 2: Mozaffarian D et al. J Nutr 2012; 142: 614S-625S
Demonstrated Superiority of MAT9001 Over Vascepa® in a Head-to-Head Study
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MAT9001
1: Some values were not calculated because the TG concentration pre- or post-treatment was >400 mg/dL2: Response variable was not normally distributed (Shapiro-Wilk p<0.01), analysis was completed using ANCOVA after rank transformation for between treatment comparisons
-33%
-11%
-35%
-30%
-25%
-20%
-15%
-10%
-5%
0%
MAT9001
Vascepa®
Significant Reductions in Triglycerides, VLDL, Non-HDL-C, Total Cholesterol, ApoAI and ApoCIIIAdditional Significant Reductions in PCSK9
Triglyceride Reduction(Median % Change From Baseline)
p < 0.001
-33.2 -32.5
-8.8-11.3
-2.4
-911%
-8.1-4.6
-11.1
-4.3-6.2
-40
-35
-30
-25
-20
-15
-10
-5
0
Med
ian
Chan
ge, %
MAT9001
EPA-EE
P < 0.001 P < 0.001
p = 0.027p = 0.337
p = 0.116
p = 0.013
Vascepa®
Median % Changes from Pre-Treatment Values for Lipids
TG VLDL-C1 Non-HDL-C HDL-C LDL-C1,2 TC
-3.8
-15.3
-25.5
-12.3
-0.7
-10.2
-5
8.8
-30
-25
-20
-15
-10
-5
0
5
10
15
Med
ian
Chan
ge, %
MAT9001
EPA-EE
p = 0.058
p = 0.003
p < 0.001
Vascepa®
Apo B Apo AI2 Apo CIII PCSK92
p = 0.006
Median % Changes from Pre-Treatment for Apolipoproteins and PCSK9
Substantially Higher Blood Levels of EPA with MAT9001 vs. Vascepa®
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MAT9001
1: T=0 is drug administration on final day of treatment
0 2 4 6 8 10 12 14 16 18 20 22 24
400
350
300
250
200
150
100
50
0
Plas
ma
Base
line-
Adju
sted
Tota
l EPA
(μg/
mL)
Time (Hours) from Dose
MAT9001
EPA-EE
Mean Plasma Baseline-Adjusted Total EPA Concentration on Final Day of Treatment1
Potential Implications for Future CV Outcomes
Vascepa®
EPA blood levels were the only biomarker that predicted CV Outcomes in REDUCE-IT
Primary End Point by Average of EPA Level at Years 1, 2, 3 and End of Study
Hazard Ratio (95% CI): 0.85 (073–0.99)
0.74 (0.63–0.86)
Icosapent Ethyl EPA >190.6 µg/mL vs Placebo 0.63 (0.54–0.74)
MAT9001
Key Points:1. The highest EPA blood levels in
REDUCE-IT were associated with the lowest CV event rates.
2. EPA was the only biomarker that predicted outcome – Changes in TG, LDL-C, non-HDL, ApoB and hs-CRP did not.
3. TG changes may be a surrogate marker for Omega-3 blood levels
4. MAT9001 has already shown dramatically higher EPA blood levels than Vascepa in a prior study.
5. This will likely be further validated in our upcoming second h-t-h study vs. Vascepa (results in Q4 2020)
Vascepa EMDAC Panel Nov. 2019
Primary Endpoints in REDUCE-IT as a Function of Serum EPA Levels (1 year)
MAT9001 Clinical Development Plan
ENHANCE-IT Study DesignObjectives – To assess the pharmacodynamic effects of MAT9001, compared with Vascepa (icosapent ethyl, EPA ethyl esters), on triglycerides (TGs) and other lipoprotein lipids, apolipoproteins, hs-CRP, and PCSK9 in men and women with elevated TG
§ Randomized, open-label, active-control crossover design (n=100)§ MAT9001 vs Vascepa, administered as 2 g 2x/day with each of two meals ; TLC
diet§ Fasting TG 150-499 mg/dL (at least 50% with TGs >200-499 mg/dL)§ No other lipid-lowering Rx (stable-dose statins allowed)
§ Two (2) 28-day treatment periods, > 28-day washout between treatments§ Measurement of pharmacodynamic parameters, omega-3 blood levels
Primary Endpoint - Percent change from baseline to end-of-treatment in plasma TG
Secondary EndpointsTotal-C, LDL-C, VLDL-C, HDL-C, non-HDL-C, Apo A1, Apo B, Apo C3, PCSK9, hs-CRP, Omega-3 fatty acids (EPA, DHA, DPA, total) in plasma
Screening
Randomization
MAT9001
Vascepa
[ Washout ]> 28 days
Treatment(28 days)
Treatment(28 days)
MAT9001
Vascepa
n = 100
10 Endpoint - % D from baseline in plasma TG
Men and women > 18 yrsTGs 150-499 mg/dL
> 50% w/TGs 200-499 mg/dL
NCT # 04177680
Blood samples
Blood samples
Blood samples
Blood samples
MAT9001
Clinical Development Plan
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MAT9001
Stage Registration Studies [505(b)(2) Pathway]
Market Differentiation Study
Preclinical Toxicology Studies• Toxicity parameters, toxicokinetics
PK/PD Studies
Phase 1 Comparator vs. Lovaza®• Single dose comparative bioavailability• Healthy volunteers (n=36)
Drug-Drug Interaction Studies (TBD)• Simvastatin: n=50• Warfarin: n=40-50• Aspirin: n=40-50
ENHANCE-IT head-to-head MAT9001 (4g) vs. Vascepa®• Patients with TG 150-499 (n=100); crossover design• Primary endpoint: % change in TG (PD)• Additional plasma omega-3 levels
Pivotal Clinical
MAT9001 (2g or 4g) vs Placebo in SHTG• 12-week study in patients with TG 500-2000 mg/dL • Primary endpoint: % change in TG
MAT9001 Development Program Timeline
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MAT9001
Activity 2019 2020 2021 2022
IND Re-Activation
Phase 1 Comparative PK/BA vs Lovaza® (Completed – Final Study Report forthcoming)
ENHANCE-IT head-to-head vs Vascepa®
EOP2 Meeting
Phase 3 – SHTG (≥ 500mg/dL) 12 Week, Global, 3-arm
NDA Submission
d ToplineEnrollment completeFPI
ToplineFPI
FPI
Intellectual Property and Barriers to Entry
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MAT9001
Omega-3 Portfolio
22 Patents filed
2 Orange-book listable U.S. patents issued, extend to 2033
• Q4 2014: U.S. 8,906,964 • Q3 2018: U.S. 10,058,521
- 4 additional U.S. patents pending, plus opportunity for composition claims depending on outcome of USPTO debate
Additional IP to be developed as clinical development plan progresses
NCE ExclusivityThe active moiety of MAT9001 is the entire mixture of omega-3 ingredients representing a single active ingredient, which makes MAT9001 eligible for 5-year NCE exclusivity
MAT2203
MAT2203: A Novel Approach to Treating Invasive Fungal Infections
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MAT2203
Broad SpectrumAntifungal
Agent
Gold Standard Efficacy for invasive fungal
infectionsAmphotericin B
Potential to become the preferred antifungal agent for treatment of cryptococcal meningitis
4 QIDP and Fast Track Designations | Orphan Drug DesignationUp to 12 years marketing exclusivity
MAT2203 is well Tolerated
§ Two Phase 2 studies
§ Orally Administered
§ No drug-related serious adverse events reported in Phase 2
LNC Platform Technology Benefits
§ Oral bioavailability
§ Reduction in toxicity
§ Targeted delivery
EnACT: Phase 1 Safety and Tolerability in Subjects without Active Neuro-Infection
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MAT2203
• MAT2203 administered in 4 to 6 divided doses over up to 24 hrs under observation• High calcium drink and meals provided at scheduled times• Safety and tolerability recordings over 24hrs• Blood draws at 0, 6, 12, 18, 24 and 48-96 hours for PK and safety• Advance to next dosing cohort if 7 of 9 complete full dose and have Grade 2 or less AEs
A
Max Tolerated Dose Moved Forward for 1wk Multi-Day Tolerability Dose Phase (n=9)
Recruitment of 27 HIV Infected Subjects, No Meningitis, No Acute Illness, Informed Consent
Cohort 1MAT2203
1g over 24hrs
n=9
Cohort 2MAT2203
1.5g over 24hrs
n=9
Cohort 3MAT2203
2g over 24hrs
n=9
B
IV Ampho: 9 daysMAT2203 5 days
MAT2203: 9 days MAT2203: 4 weeksIV Ampho5 days
MAT2203: 12 days MAT2203: 4 weeks
MAT2203: 4 weeks
MAT2203: 2 weeks MAT2203: 4 weeks
Induction Therapy (2 wks) Early Maintenance Therapy w/ MAT2203 (4 wks)
Ø Assess MAT2203 as induction and maintenance therapy
Ø Primary endpoint: Rate of CSF fungal clearance
Ø N=100 patients receiving MAT2203 + flucytosine (5-FC) in 4 stages of escalating durations of MAT2203 and decreasing duration of IV Amphotericin B (AMB)
Ø N=40 control patients receiving standard of care (IV AMB + 5-FC)
Ø Safety and efficacy monitored throughout study by independent Data Monitoring Committee
Ø All arms to receive 5-FC during induction therapy and fluconazole during maintenance therapy
Ø All stages to include a comparator arm of IV Amp + 5-FC (induction) and fluconazole (maintenance) to be run in parallel
Ø Maintenance with Fluconazole will continue through Week 10
Stage 1n=10
Stage 2n=40
Stage 3n=10
Stage 4n=40
EnACT Study Design: Phase 2 in HIV patients with Cryptococcal Meningitis
IV Ampho2 days
Open-Label, Sequential-Cohort study Assessing Safety, Tolerability and Efficacy of MAT2203
Protocol Details
MAT2203
Lipid Nano-Crystal (LNC) Platform
LNCs Enable Safe, Targeted and Intracellular Delivery of Potent Medicines
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LNC Platform
Highly Efficient, Physiologic and Nontoxic Drug Formulation Platform
Enter cells through non-destructive, membrane fusion
Physiologically targets activated cells
Validated in multiple clinical and preclinical studies
evidence of immunogenicityNOtoxicity of drugsReduced
broad range of molecules
Ability to deliver a
Flexible administrationOral IntravenousIntramuscular Intranasal
LNC Platform Drives Value Creating Partnerships
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LNC Platform
Driving innovation and platform expansion with Big Pharma financial support and molecule expertise
Q1 2019: Signed first LNC Platform Research Evaluation of Oligonucleotide with top-10 global pharmaceutical company
Q2 2019: Signed Research Collaboration with ViiV Healthcare to Evaluate Formulation of Antiviral Drug Candidates
Q4 2019: Signed Feasibility Evaluation with Genentech, a member of the Roche Group, to formulate up to three compounds
Leadership Team
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Jerome D. Jabbour Co-Founder, Chief Executive Officer, Director
James J. Ferguson III, M.D., FACC, FAHAChief Medical Officer
Keith A. Kucinski, CPA, MBAChief Financial Officer
Theresa Matkovits, Ph.D.Chief Development Officer
Raphael J. Mannino, Ph.D.Chief Scientific Officer
Executive Officers
Board of DirectorsHerbert Conrad Chairman of the Board
Matthew A. Wikler, M.D., MBA FIDSADirector
Patrick G. LePoreVice Chairman
Adam SternDirector
Eric J. Ende, M.D., MBADirector
Jerome D. JabbourCo-Founder, Chief Executive Officer, Director
James S. ScibettaDirector
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§ Resume Phase 2 portion of EnACT study
§ EnACT cohort progression updates
§ Top-line data from EnACT study
LNC Platform
MAT9001
MAT2203
Matinas Key Development Milestones
§ Resume head-to-head ENHANCE-IT study
§ End-of-Phase 2 meeting with FDA
§ Top-line data from ENHANCE-IT study
§ Commence Phase 3 in severe hypertriglyceridemia
§ Partnership development updates
www.matinasbiopharma.comNYSE AMERICAN: MTNB
Corporate PresentationApril 2020