blu corporate presentationjuly152013vfinal
TRANSCRIPT
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Corporate Presentation
Roberto BelliniPresident and Chief Executive OfficerTwitter: @rbellini
July 15, 2013
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Forward Looking StatementCertain statements contained in this presentation, other than statements of fact that areindependently verifiable at the date hereof, may constitute forward-looking statements. Suchstatements, based as they are on the current expectations of management, inherently involvenumerous risks and uncertainties, known and unknown, many of which are beyond BELLUSHealth Inc.'s control. Such risks include but are not limited to: the ability to obtain financingimmediately in current markets, the impact of general economic conditions, general conditionsin the pharmaceutical and/or nutraceuticals industry, changes in the regulatory environment inthe jurisdictions in which the BELLUS Health Group does business, stock market volatility,fluctuations in costs, and changes to the competitive environment due to consolidation,achievement of forecasted burn rate, and that actual results may vary once the final andquality-controlled verification of data and analyses has been completed.
Consequently, actual future results may differ materially from the anticipated results expressedin the forward-looking statements. The reader should not place undue reliance, if any, on anyforward-looking statements included in this news release. These statements speak only as ofthe date made and BELLUS Health Inc. is under no obligation and disavows any intention toupdate or revise such statements as a result of any event, circumstances or otherwise, unlessrequired by applicable legislation or regulation. Please see the Company’s public fillingsincluding the Annual Information Form of BELLUS Health Inc. for further risk factors that mightaffect the BELLUS Health Group and its business
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Background and Business Model
Public company (TSX: BLU) based in Montreal, QCFocused on products in amyloid-related fields, principally AA Amyloidosis, an orphan indication affecting the kidneys
Late-stage product pipeline with fully funded business plan
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Business ModelFocused on building value for clinical stage health products in critical unmet medical needs
Financial Facts Shares outstanding (Fully
Diluted): 65M
Cash (03/31/13): ~$18M
Burn rate (monthly): <$300K
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Pipeline of Products
PHARMACEUTICALS
NUTRACEUTICAL
NRM8499Alzheimer’s disease
VIVIMIND™Memory protection
DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III
MARKETDISCOVERY PRECLINICAL PHASE I PHASE II PHASE III
NDA/MAA
KIACTA™AA amyloidosis confirmatory
Thallion acquisition announced June 18th to expand pipeline with addition of Phase II product Shigamabs for sHUS
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KIACTA™ Overview
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For Amyloid A (AA) Amyloidosis, a rare and deadly disease with no specific treatment
Market opportunity
Clinical evidence
1 Market assessment by Frankel Group in April 2009.
Partnership
Orphan population of ≈50,000 in the USA, Europe and Japan with peak annual revenues projected at $400-600M1
Phase II/III clinical trial showing statistically significant primary efficacy endpoints(p value = 0.025) and clean safety profile
Partnership with Auven Therapeutics (previously known as Celtic Therapeutics) to conduct and finance (>$50M) Phase III Confirmatory Study
Phase III Confirmatory
Study
Marketing approval based on confirming safety and efficacy of phase II/III study
>66% patients recruited
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Mechanism of Action
CHRONIC INFLAMMATION
SERUM AMYLOID APRECURSOR (SAA) PROTEIN
AA PROTEIN + GLYCOSAMINOGLYCANS(GAGs)
ORGAN DAMAGE, IN PARTICULAR TO KIDNEYS
REDUCTION IN FIBRIL FORMATION & DEPOSITION
Converts toAA Protein
Generatescytokine cascade (TNFα / IL-1 / IL-6)
and increases SAA levels
Rheumatic ConditionsInflammatory Bowel DiseaseChronic InfectionsFamilial Mediterranean Fever
KIACTA blocksAA + GAGs interaction
Systemic Amyloid A Fibril Formation & Deposition
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KIACTA™ - Targeted Opportunity
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Dia
gnos
ed A
A Pa
tient
s (0
00s)
Patient population estimated at 34,000-50,000 in the United States, Europe Top 5 and Japan1
KIACTA™ peak annual revenues projected at $400-600M1
(U.S., EU5, Japan)Strong potential for premium pricing Clear pharmacoeconomic
rationale due to high cost ofkidney disease
Comparative orphan drug pricing
1Market assessment by Frankel Group in April 2009
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FINANCIAL IMPLICATION
With global fund AuvenTherapeutics
Auven Therapeutics funding 100% of KIACTA™’s Confirmatory Phase III clinical trial
Auction process for the commercialization rights of KIACTA™ on completion of Phase III Confirmatory Study
PARTNERSHIP
US$10M in upfront payments
≥ US$50M in investments by Auven Therapeutics
Proceeds of any eventual transaction expected to be shared 50%-50% between BELLUS Health and AuvenTherapeutics
Strategic Partnership
Partnership to fund Phase III Confirmatory Study with significant upside for BELLUS shareholders
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HR 0.58 0.41 0.48 0.54 0.95
95% C.I 0.37, 0.93 0.19,0.86 0.28,0.82 0.22,1.37 0.27,3.29
P value 0.025 0.019 0.008 0.20 0.94
Graphical representation of the information in this table
• Landmark study in AA Amyloidosis: 183 patients treated for 2 years
• Composite endpoint based on patients reaching events of decreasing kidney function or death
• Statistically significant primary endpoint (Cox Proportional Hazard Ratio; p=0.025)
• Clinically meaningful treatment effect with 42% reduction in risk of reaching event
• Most sensitive componentof composite endpoint highly significant (CrCl, p < 0.01)
Composite Endpoint (Time to First Worse
Event)
Doubling Serum
Creatine
50%DecreaseCreatine
CIearance
Dialysis/ESRD Death
Num
ber o
f Pat
ient
Eve
nts
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*
*
*
KIACTA™ - Robust Clinical Results in Phase II/III
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KIACTA™ - Phase II/III Key Secondary Endpoints
HR=0.58Wald Chi Square test: p=0.025
KIACTATM
Placebo
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Key secondary endpoints show robust effect of KIACTA™
Using slope of creatinine clearance decline, calculated delayto time of dialysis is ~2 years on Kiacta versus placebo
Kaplan Meier: Time to First Worst Event Slope of Creatinine Clearance Decline
Placebo
Months of Treatment
Mea
n ∆
CrC
l(m
L/m
in/1
.73m
2 )
= 9.4mL/min/1.73 m2
after 24 months p=0.025
-10.9 ± 5.1 mL/min/1.73 m2/year
-15.6 ± 4.0 mL/min/1.73 m2/year
KIACTATM
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KIACTA™ - Phase II/III Feedback
2007 NEJM article published by lead investigators concludes that KIACTA™ slows decline of renal function in AA Amyloidosis
Agreement reached under Special Protocol Assessment with FDA and Scientific Advice with EMEA to conduct Phase III Confirmatory Study
Marketing approval based on positive result (p value <0.05) from confirmatory study with same scope of first phase II/III clinical trial
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PHASE III CONFIRMATORY STUDY183 patients in 13 countries
Composite primary endpoint based on patients reaching kidney function worsening events: Doubling serum creatinine
50% decrease in creatinine clearance
Reaching ESRD/dialysis
Death
Fixed treatment duration of 2 years
PHASE II/III STUDY230 patients in >25 countries
Composite primary endpoint (target p<0.05) based on patients reaching kidney function worsening events:
Persistent 80% increase serum creatinine
Persistent 40% decrease in creatinineclearance
Reaching ESRD/dialysis
Event driven trial to conclude on attainment of 120 events (~90% power)
KIACTA™ - Phase III Confirmatory Study
Key improvements made to increase robustness of Phase III Confirmatory Study
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Study ProgressSTUDY UPDATE (May 2013)
>65 sites in >25 countries activated
>150 patients enrolled
Recruitment expected to be completed in 1H 2014
COMPLETION
Event driven trial to complete on reaching 120 events
Study expected to be completed in 2017
Patient baseline characteristics and demographicsto date are similar to those in the first Phase III study
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Thallion Transaction - OverviewBellus to acquire Thallion for $6.3M in cash plus certain contingent value rights (CVRs):
$6.3M in cash consideration1 ($0.1904 per share) based on $7.5M net cash at closing;
100% of expected $1.4M 2016 payment (up to $0.041 per share)
5% of Shigamabs product revenue, capped at $6.5M (up to $0.181 per share)
Binding agreement and announcement on June 17th
Unanimous support from Thallion and Bellus Board of Directors
Voting support agreements in place currently for more than 24% of votes, including 14.5% from largest shareholder Jaguar Financial
Transaction to proceed by court supervised plan of arrangement requiring 66 2/3% shareholder approval
Thallion shareholder meeting planned for August 6th
Close of transaction expected shortly after shareholder meeting
1 Cash consideration to be adjusted (up and down) on a dollar for dollar based on net cash 5 days before closing
Acquisition rationale based on building pipeline with addition of Shigamabs, a complementary Phase 2 asset
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Thallion Transaction – BenefitsBenefits to Bellus:
Adds Shigamabs to pipeline, a complementary Phase II asset targeting a rare disease affecting the kidneys
Net cash of ~$1.2M from transaction provides funding for Shigamabs development
Benefits to Thallion:
Provides initial cash consideration at significant premium to stock price, 46% premium from announcement date and 90% premium from initiation of discussions
Provides additional value in CVR with potential to achieve aggregate ~$0.41 per share, 215% premium from announcement date and 310% premium from initiation of discussions
Transaction is executionally low risk and also tax efficient for Thallion shareholders
Mutually beneficial transaction for Thallion and Bellus shareholders
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Shigamabs Overview
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For STEC related Hemolytic Uremic Syndrome (sHUS), an ultra rare disease primarily affecting the kidneys
Market opportunity
Clinical evidence
Mechanism
Shigatoxin producing e.coli (STEC) is a rare food borne illness1,500-2,000 annual cases progress to sHUS in developed countries, principally in childrenPremium pricing potential
Safety in target pediatric population from 45 patient Phase 2 study
Monoclonal antibody binds and neutralizes Stx-2, the most prominent and virulent variant of STEC
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sHUS (5%-10%)
Resolve (90%-95%)
Ingestion Colonization Toxin Secretion
-4 0 2 4 6 8-2Day
Thallion intervention (prevention)
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Shigamabs Development Strategy
Development thesis to reposition Shigamabs from prevention of sHUS to treatment of sHUS
Support from key opinion leader community for downstream therapeutic approach to sHUS
Recent publication supports role of toxin neutralization for treatment of sHUS
Conduct proof of concept studies in several HUS animal models to validate approach
Clear clinical and regulatory path into Phase II proof of concept study
Only treating patients with high unmet medical need
Clear endpoint based on reduction in need for dialysis (40-50% of sHUS patients require dialysis)
Dialysis is considered clinically relevant by regulatory agencies
Bellus intervention
STEC and sHUS Disease Course
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VIVIMIND™
Nutraceutical for memory protection
Regulatory
Partnerships
Growing cash flow positive business
Partnerships for Italy (launched), Canada (launched), South Korea (regulatory), Greece (pre-launch), Middle East (pre-launch), Taiwan (regulatory) and Israel (regulatory)Pursuing creation of worldwide distributor network
Health claims include: ‘Protects the hippocampus’ and ‘Enhances cognitive function and memory’
VIVIMIND Revenues ($K)
0
100
200
300
400
500
2010 2011 2012
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BLU8499
Next generation of tramiprosate intended for the treatment of Alzheimer's disease
Market opportunity
Clinical evidence
Partnership
Large and growing epidemic currently affecting over 30M patients worldwide
Evidence of effectiveness of parent compound tramiprosate in ApoE4+ Alzheimer’s patientsSafe and well tolerated in Phase I
Partnership with Asclepios Bioresearch in September 2012 to finance development of BLU8499 into Phase 2aCommitment of ~$4M in non-dilutive capital
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Capital Structure
Basic Shares Outstanding 47M
Fully Diluted Shares Outstanding 65M
Operations funded into mid-201820
Financial Position and Capital Structure
Financial Position
Cash (March 31st, 2013) ~$18M
Burn Rate (monthly) <$300K
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Shareholder Ownership
Bellini Family ≈ 30%
Power Corporation ≈ 30%
Pharmascience ≈ 10%
Governance and Shareholders
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Board of Directors Company / Experience
Dr. Francesco Bellini (Chair)
Franklin Berger
Charles Cavell
Hélène Fortin
Pierre Larochelle
Donald Olds
Joseph Rus
Dr. Martin Tolar
Roberto Bellini
Management Title
Roberto Bellini President and Chief Executive Officer
Dr. Denis Garceau Senior Vice President, Drug Development
François Desjardins Vice President, Finance
Tony Matzouranis Vice President, Business DevelopmentLAROSE FORTIN CA Inc.
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Short-term milestones driving long-term value
Milestones
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Past ExecutionAttractive partnership with for Kiacta
Execution of global KIACTA Phase III Confirmatory Study
Cashflow positive VIVIMIND business
Partnership for BLU8499
Strong balance sheet and clean capital structure
Milestones (12 months)Completion of recruitment of KIACTA™ Phase III Confirmatory Study
Additional KIACTA™ activities:
Launch of open label extension study
Market and pricing assessment
Japan orphan drug designation
BLU 8499 Pre-Phase II package completion
VIVIMIND partnerships
Long Term Value
Results of Phase III Confirmatory Study and auction of KIACTA™
Sale or spin-out of VIVIMIND business
BLU8499 Phase IIa study results
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