Corporate Presentation (TSX: BLU)
Roberto BelliniPresident and Chief Executive OfficerTwitter: @rbellini
Winter 2014
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30 millionpeople in the United States have a RARE disease.
Source: NIH: National Institutes of Health Office of Rare Diseases
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Only about 5%of these people have a specific therapy to treat their disease.
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85-90%of rare diseases are serious or life threatening.
Regulatory advantage
Premium pricing
Market protection
Smaller clinical trials
Efficient commercialization strategies
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Small patient numbers, BIG opportunity
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At BELLUS, we are focused on developing drugs for rare diseases starting with conditions that affect the kidneys.
Fruitful 2013 leading into important milestones in 2014
Executing on Plan
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2013Lead rare disease program, KIACTA™ for AA amyloidosis in Phase III Confirmatory Study :
Recruitment on target
Japan orphan drug designation
Expand rare disease pipeline
Acquisition of Shigamabfor STEC-HUS
Research partnership for AL amyloidosis
Divestiture of non-core assets
Maintain financial health
2014Continue executing KIACTA™ plan:
Completion of recruitment
Launch of open label extension study
Market and pricing assessment
Progress pipeline projects:
Animal studies to support Shigamab Phase II
Pre-clinical proof of concept for AL amyloidosis
Continued financial stewardship
Business plan fully funded through KIACTA™ exit
OVERVIEW CAPITAL STRUCTURE
Shareholder Information
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Public company (TSX: BLU) based in Montreal, QC
Developing drugs for rare diseases
Late-stage product pipeline with fully funded business plan
Shares outstanding (Fully Diluted): 65M
Cash (09/30/13): ~$16M
Burn rate (monthly): <$300K
Shareholder makeup: 70% institutional, 30% retail
Late stage pipeline focused on developing innovative drugs for rare diseases
Pipeline of Products
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ShigamabsHUS
DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III
KIACTA™AA amyloidosis
MARKET
AL amyloidosis
Lead Phase III Product Candidate
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A rare and deadly kidney disease with no specific treatment
FOR AMYLOID A (AA) AMYLOIDOSIS
Disease and Mechanism of Action
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CHRONIC INFLAMMATION
SERUM AMYLOID APRECURSOR (SAA) PROTEIN
AA PROTEIN + GLYCOSAMINOGLYCANS(GAGs)
ORGAN DAMAGE, IN PARTICULAR TO KIDNEYS LEADING TO DIALYSIS
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
MARKET SIZEOrphan drug designation granted with market protection in the U.S. (7 years), Europe and Japan (10 years)
Formulation (Dosing Schedule) and Methods for Treating Amyloidosis with expiry in 2026
5 year patent extension can be applied to provide protection until 2031
MARKET PROTECTIONKIACTA™ peak annual revenues projected at $500 million
Clear pharmacoeconomicrationale due to high cost of kidney disease
Premium pricing for comparative rare disease drugs
Market
Independent market assessment currently underway and to be completed by Q1 2014
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Partnership to fund Phase III Confirmatory Study with significant upside for BELLUS shareholders
With global fund AuvenTherapeutics, a private equity group specialized in drug development project financing
Auven Therapeutics funding 100% of KIACTA™’s Phase III Confirmatory Study
US$10M in upfront by AuvenTherapeutics
≥ US$50M in investments by Auven Therapeutics
Proceeds of exit expected to be shared 50-50
Strategic Partnership
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FINANCIALSPARTNERSHIP
0
5
10
15
20
25
30
35
40
45
50
Placebo
KIACTA
Composite Endpoint (Time to
First Worse Event)
Doubling Serum
Creatine
50%DecreaseCreatine
CIearance
Dialysis/ESRD
Num
ber o
f Wor
se E
vent
s
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*
*
**
Strong Clinical Results in First Phase III Study
Landmark study in AA amyloidosis: 183 patientstreated for 2 years
Important benefits for patients on drug:
Statistically significant reduction in number and risk of reaching worsening kidney event
Important delay in reaching dialysis
*p<0.05 **p<0.01
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Regulatory
New England Journal of Medicine publication concludes that KIACTATM
slows decline of renal function in AA amyloidosis
Agreement reached in U.S., Europe, Japan to conduct Phase III Confirmatory Study
Approval based on achieving comparable result of first Phase III Study
Study enrolling 230 patients total with ~200 patients enrolled
Event driven trial to complete when 120 of 230 patients reach event of kidney function deterioration
Study completion expected in 2016
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Phase III Confirmatory Study
Second Rare Disease Product Candidate
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A rare disease primarily affecting the kidneys of children
FOR STEC RELATEDHEMOLYTIC UREMIC SYNDROME (SHUS),SHIGAMAB
Disease Course and Mechanism of Action
E. COLI INGESTION
GUT COLONIZATION AND SECRETION OF TOXIN INTO BLOODSTREAM
TOXIN MAY BE CARRIED BY PMNs IN BLOODSTREAMSYMPTOMS: BLOODY DIARRHEA
SHIGAMAB BINDING NEUTRALIZES TOXIN WHICH IS THEN ELIMINATED
ShigamabAntibody
Day -4 Day 0 Day 4 Day 8
TOXIN BINDS TO GB3 RECEPTORS ON KIDNEY LEADING TO STEC-HUS. OUTCOMES: -CHRONIC KIDNEY DISEASE / HYPERTENSION: 40%-ENCEPHALOPATHY / DEATH: 5%-RESOLUTION: 55%
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90%
SPONTANEOUSRESOLUTION
10%
SHIGAMAB TREATMENT
Potential for partnership in 18-24 months
Shigamab Overview
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NEXT STEPS (12 MONTHS)
MARKET OPPORTUNITY
PRE-CLINICAL AND CLINICAL
Proof of concept for treatment of sHUS in animal modelsMeetings with regulators to agree on development plan
2,000-3,000 estimated annual cases of sHUS in developed countries, principally children$100-200 million annual sales opportunity
Treatment with Shigamab led to significantly increased survival in STEC animal modelSafe and well tolerated in target pediatric population
Research Program
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A rare and deadly blood disorder
RESEARCH PROJECT FOR AMYLOID LIGHT-CHAIN (AL) AMYLOIDOSIS,DRUG CANDIDATES
Potential for pre-clinical proof-of-concept within 12 months
AL Amyloidosis Project Overview
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PARTNERSHIP
DISEASE
Partnership with Amorchem, a Montreal-based venture fund, to finance research projectObjective: identify and develop drug candidates for AL amyloidosis to pre-clinical proof-of-concept
AL amyloidosis is a blood disorder that leads to the formation of toxic amyloid fibrils and plaquesTreatment options are limited leading to death in most cases2,000-3,000 new cases are reported each year in the United States
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 Development
LAROSE FORTIN CA Inc.
Short-term milestones driving long-term value
Milestones
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Past Execution
Attractive partnership for KIACTA™
Execution of global KIACTA™ Phase III Confirmatory Study
Expansion of rare disease pipeline
Strong balance sheet and clean capital structure
2014 MilestonesContinue executing KIACTA™ plan:
Completion of recruitment
Launch of open label extension study
Market and pricing assessment
Progress pipeline projects:
Animal studies to support Shigamab Phase II
Pre-clinical proof of concept for AL amyloidosis
Long Term Value
KIACTA™ exit and results of Phase III Confirmatory Study
Shigamab partnership or proof-of -concept Phase II study results
Forward Looking Statement
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Certain 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