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Bellus corporate presentation january 2014 v final

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Bellus corporate presentation january 2014 v final

  1. 1. Corporate Presentation (TSX: BLU) Winter 2014 Roberto Bellini President and Chief Executive Officer Twitter: @rbellini
  2. 2. 30 million people in the United States have a RARE disease. Source: NIH: National Institutes of Health Office of Rare Diseases 2
  3. 3. Only about 5% of these people have a specific therapy to treat their disease. 3
  4. 4. 85-90% of rare diseases are serious or life threatening. 4
  5. 5. Small patient numbers, BIG opportunity Regulatory advantage Premium pricing Market protection Smaller clinical trials Efficient commercialization strategies 5
  6. 6. At BELLUS, we are focused on developing drugs for rare diseases starting with conditions that affect the kidneys. 6
  7. 7. Executing on Plan 2013 Lead 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 Shigamab for STEC-HUS Research partnership for AL amyloidosis Divestiture of non-core assets Maintain financial health 2014 Continue 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 Fruitful 2013 leading into important milestones in 2014 7
  8. 8. Shareholder Information OVERVIEW CAPITAL STRUCTURE Public company (TSX: BLU) based in Montreal, QC Shares outstanding (Fully Diluted): 65M Developing drugs for rare diseases Cash (09/30/13): ~$16M Late-stage product pipeline with fully funded business plan Burn rate (monthly): <$300K Shareholder makeup: 70% institutional, 30% retail Business plan fully funded through KIACTA™ exit 8
  9. 9. Pipeline of Products DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III MARKET KIACTA™ AA amyloidosis Shigamab sHUS AL amyloidosis Late stage pipeline focused on developing innovative drugs for rare diseases 9
  10. 10. Lead Phase III Product Candidate FOR AMYLOID A (AA) AMYLOIDOSIS A rare and deadly kidney disease with no specific treatment 10
  11. 11. Disease and Mechanism of Action SERUM AMYLOID A PRECURSOR (SAA) PROTEIN AA PROTEIN + GLYCOSAMINOGLYCANS KIACTA™ blocks AA + GAGs interaction (GAGs) REDUCTION IN FIBRIL FORMATION & DEPOSITION CHRONIC INFLAMMATION Generates cytokine cascade (TNFα / IL-1 / IL-6) and increases SAA levels Rheumatic Conditions Inflammatory Bowel Disease Chronic Infections Familial Mediterranean Fever Converts to AA Protein Systemic Amyloid A Fibril Formation & Deposition ORGAN DAMAGE, IN PARTICULAR TO KIDNEYS LEADING TO DIALYSIS 11
  12. 12. Market MARKET PROTECTION Orphan 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 SIZE KIACTA™ peak annual revenues projected at $500 million Clear pharmacoeconomic rationale due to high cost of kidney disease Premium pricing for comparative rare disease drugs Independent market assessment currently underway and to be completed by Q1 2014 12
  13. 13. Strategic Partnership PARTNERSHIP FINANCIALS With global fund Auven Therapeutics, a private equity group specialized in drug development project financing US$10M in upfront by Auven Therapeutics Auven Therapeutics funding 100% of KIACTA™’s Phase III Confirmatory Study Proceeds of exit expected to be shared 50-50 ≥ US$50M in investments by Auven Therapeutics Partnership to fund Phase III Confirmatory Study with significant upside for BELLUS shareholders 13
  14. 14. Strong Clinical Results in First Phase III Study 50 Placebo 45 KIACTA Number of Worse Events 40 35 30 Landmark study in AA amyloidosis: 183 patients treated for 2 years Important benefits for patients on drug: * 25 Statistically significant reduction in number and risk of reaching worsening kidney event ** 20 15 * 10 Important delay in reaching dialysis 5 0 Composite Endpoint (Time to First Worse Event) Doubling Serum Creatine 50% Decrease Creatine CIearance Dialysis/ ESRD *p<0.05 **p<0.01 14
  15. 15. 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 15
  16. 16. Phase III Confirmatory 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 16
  17. 17. Second Rare Disease Product Candidate SHIGAMAB FOR STEC RELATED HEMOLYTIC UREMIC SYNDROME (SHUS), A rare disease primarily affecting the kidneys of children 17
  18. 18. Disease Course and Mechanism of Action SHIGAMAB TREATMENT SHIGAMAB BINDING NEUTRALIZES TOXIN WHICH IS THEN ELIMINATED GUT COLONIZATION AND SECRETION OF TOXIN INTO BLOODSTREAM TOXIN MAY BE CARRIED BY PMNs IN BLOODSTREAM SYMPTOMS: BLOODY DIARRHEA Shigamab Antibody E. COLI INGESTION 10% 90% TOXIN BINDS TO GB3 RECEPTORS ON KIDNEY LEADING TO STEC-HUS. OUTCOMES: -CHRONIC KIDNEY DISEASE / HYPERTENSION: 40% -ENCEPHALOPATHY / DEATH: 5% -RESOLUTION: 55% SPONTANEOUS RESOLUTION Day -4 Day 0 Day 4 Day 8 18
  19. 19. Shigamab Overview MARKET OPPORTUNITY 2,000-3,000 estimated annual cases of sHUS in developed countries, principally children $100-200 million annual sales opportunity PRE-CLINICAL AND CLINICAL Treatment with Shigamab led to significantly increased survival in STEC animal model Safe and well tolerated in target pediatric population NEXT STEPS (12 MONTHS) Proof of concept for treatment of sHUS in animal models Meetings with regulators to agree on development plan Potential for partnership in 18-24 months 19
  20. 20. Research Program DRUG CANDIDATES RESEARCH PROJECT FOR AMYLOID LIGHT-CHAIN (AL) AMYLOIDOSIS, A rare and deadly blood disorder 20
  21. 21. AL Amyloidosis Project Overview PARTNERSHIP Partnership with Amorchem, a Montreal-based venture fund, to finance research project Objective: identify and develop drug candidates for AL amyloidosis to pre-clinical proof-of-concept DISEASE AL amyloidosis is a blood disorder that leads to the formation of toxic amyloid fibrils and plaques Treatment options are limited leading to death in most cases 2,000-3,000 new cases are reported each year in the United States Potential for pre-clinical proof-of-concept within 12 months 21
  22. 22. Governance and Shareholders Board of Directors Company / Experience Management Title Roberto Bellini Hélène Fortin Pierre Larochelle Vice President, Finance Tony Matzouranis Charles Cavell Senior Vice President, Drug Development François Desjardins Franklin Berger President and Chief Executive Officer Dr. Denis Garceau Dr. Francesco Bellini (Chair) Vice President, Business Development LAROSE FORTIN CA Inc. Shareholder Ownership Bellini Family Donald Olds Joseph Rus ≈ 30% Power Corporation ≈ 30% Pharmascience ≈ 10% Dr. Martin Tolar Roberto Bellini 22
  23. 23. Milestones 2014 Milestones 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 Continue executing KIACTA™ plan: Completion of recruitment Launch of open label extension study Market and pricing assessment Long Term Value KIACTA™ exit and results of Phase III Confirmatory Study Shigamab partnership or proof-of -concept Phase II study results Progress pipeline projects: Animal studies to support Shigamab Phase II Pre-clinical proof of concept for AL amyloidosis Short-term milestones driving long-term value 23
  24. 24. Forward Looking Statement Certain statements contained in this presentation, other than statements of fact that are independently verifiable at the date hereof, may constitute forward-looking statements. Such statements, based as they are on the current expectations of management, inherently involve numerous risks and uncertainties, known and unknown, many of which are beyond BELLUS Health Inc.'s control. Such risks include but are not limited to: the ability to obtain financing immediately in current markets, the impact of general economic conditions, general conditions in the pharmaceutical and/or nutraceuticals industry, changes in the regulatory environment in the 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 and quality-controlled verification of data and analyses has been completed. Consequently, actual future results may differ materially from the anticipated results expressed in the forward-looking statements. The reader should not place undue reliance, if any, on any forward-looking statements included in this news release. These statements speak only as of the date made and BELLUS Health Inc. is under no obligation and disavows any intention to update or revise such statements as a result of any event, circumstances or otherwise, unless required by applicable legislation or regulation. Please see the Company’s public fillings including the Annual Information Form of BELLUS Health Inc. for further risk factors that might affect the BELLUS Health Group and its business 24

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