Corporate presentation january 2013 v final


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  • Corporate presentation january 2013 v final

    1. 1. Corporate PresentationRoberto BelliniPresident and Chief Executive OfficerJanuary 2013
    2. 2. 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 and quality-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 2
    3. 3. Background and Business Model Public company (TSX: BLU) based in Montreal, QC Focused namely on the development of products in amyloid- related fields, principally AA Amyloidosis, an orphan indication affecting the kidneys Pipeline also includes product candidate for the treatment of Alzheimer’s disease BUSINESS MODEL Focused on building value for clinical stage health products in critical unmet medical needs 3
    5. 5. KIACTA™ For AA Amyloidosis, an orphan indication and a deadly disease with no treatment Orphan population of ≈50,000 in the USA, Europe Market and Japan with peak annual revenues projected at opportunity $400-600M1 1 Independent market assessment by Frankel Group in April 2009. Phase II/III clinical trial showing statistically Clinical significant primary efficacy endpoints evidence (p value = 0.025) and clean safety profile Partnership with Celtic Therapeutics to conduct Partnership and finance (>$50M) Phase III Confirmatory Study Marketing approval based on confirming safety Phase III and efficacy of phase II/III studyConfirmatory Study Actively recruiting patients 5
    6. 6. AA Amyloidosis – A Rare and Lethal Disease AA PROTEIN + REDUCTION IN SERUM AMYLOID A GLYCOSAMINOGLYCAN KIACTA blocks FIBRIL FORMATION PRECURSOR (SAA) S (GAGs) AA + GAGs interaction & DEPOSITION PROTEINCHRONICINFLAMMATION Generates Converts to cytokine cascade AA Protein (TNFα / IL-1 / IL-6) Systemic Amyloid A Fibril and increases SAA levels Formation & Deposition ORGAN DAMAGE, INRheumatic Conditions PARTICULAR TOInflammatory Bowel Disease KIDNEYSChronic Infections -Rapid deterioration ofFamilial Mediterranean Fever kidney function leading to dialysis 6
    7. 7. KIACTA™ - Targeted Opportunity Patient population estimated at 34-50,000 in the U.S., EU5 and Japan1Diagnosed AA Patients (000s) Clear pharmacoeconomic rationale for premium pricing KIACTA™ peak annual revenues projected at $400-600M1 (U.S., EU5, Japan) Orphan Drug Status in the U.S. and EU provides 7 and 10 years market exclusivity upon commercialization, respectively 1 Independent market assessment by Frankel Group in April 2009 7
    8. 8. KIACTA™ - Strategic PartnershipPARTNERSHIP FINANCIAL IMPLICATION Partnered with private equity US$10M upfront firm Celtic Therapeutics ≥ US$50M in investments by Celtic Therapeutics funding Celtic Therapeutics 100% of KIACTA™’s Phase III Confirmatory Study Proceeds of any eventual transaction expected to be Auction process for the shared 50%-50% between commercialization rights of BELLUS Health and Celtic KIACTA™ on completion of Therapeutics Phase III Confirmatory Study 8
    9. 9. KIACTA™ - Robust Clinical Results in Phase II/III • Composite endpoint basedNumber of Patient Events on patients reaching events of decreasing kidney function or death • Statistically significant * primary endpoint (Cox Graphical Proportional Hazard Ratio; representation of the p=0.025) * information in this • Cox test takes into account table event and time to event * • Most sensitive component of composite endpoint highly significant (CrCl, p < 0.01) Composite Doubling 50% Dialysis/ • Clinically meaningful treatment Endpoint (Time Decrease Death to First Worse Serum Creatine ESRD effect with 42% reduction in Creatine Event) CIearance risk of reaching event 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 9
    10. 10. KIACTA™ - Phase II/III Key Secondary EndpointsKaplan Meier: Time to First Worst Event Slope of Creatinine Clearance Decline Months of Treatment Mean ∆ CrCl (mL/min/1.73m2) KIACTATM KIACTATM -10.9 ± 5.1 mL/min/ Placebo 1.73 m2/year Placebo -15.6 ± 4.0 mL/min/ ∆= 9.4mL/min/ 1.73 m2/year 1.73 m2 HR=0.58 after 24 Wald Chi Square test: p=0.025 months p=0.025 Key secondary endpoints show robust effect of KIACTA™ Using slope of creatinine clearance decline, calculated delay to time to dialysis is ~2 years on KIACTA™ versus placebo 10
    11. 11. 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 Trial Marketing approval based on positive result (p value <0.05) from confirmatory study with same scope of first phase II/III clinical trial 11
    12. 12. KIACTA™ - Confirmatory Phase III Study PHASE IIICOMPLETED PHASE II/III STUDY CONFIRMATORY STUDY 183 patients in 13 countries 230 patients in 28 countries Statistically significant composite Composite primary endpoint primary endpoint (p=0.025) (target p<0.05) based on patients principally based on patients reaching kidney function events: reaching kidney function events:  80% increase serum creatinine  Doubling serum creatinine  40% decrease in creatinine  50% decrease in creatinine clearance clearance  Reaching ESRD/dialysis  Reaching ESRD/dialysis  Death Event driven trial to conclude on attainment of 120 events (~90% Fixed treatment duration of two power) years Key improvements made to Key improvements made to increase robustness of increase robustness of confirmatory study confirmatory study 12
    13. 13. KIACTA™ - Study Progress Recruitment1 Completion >70 sites in >25 Event driven trial to countries actively complete on reaching recruiting 120 events ~120 patients enrolled Study expected to be completed in 2017 Recruitment expected to be completed in 1H 2014 Patient baseline characteristics and demographics to date are similar to those in the first Phase III study 1 Data as of November 2012 13
    14. 14. KIACTA™ - Exit Strategy RARE DISEASE DRUGS IN THE NEWS Sarepta shares rocket up on stellar muscular dystrophy trial results (October 2012) ALEXION TO AQUIRE ENOBIA PHARMA FOR UP TO $1.08 BILLION Alnylam Gets $22.5M (December 2011) From Genzyme for Asia Rights to Novartis signs $665m option deal with Amyloidosis Drug Selexys for sickle cell disease drug (October 2012) (September 2011) BioMarin shares pop on strong pivotal data for rare disease drug (November 2012) •• Auction process at end of study to realize full value Auction process at end of study to realize full value • Partial exit also possible (commercial partnership) before Phase III data 14
    16. 16. VIVIMIND™ Nutraceutical for memory protection Health claims include: VIVIMIND Revenues ($K) Regulatory ‘Protects the hippocampus’ and ‘Enhances cognitive function and memory’ Partnerships for Italy (launched), Canada (launched), South Korea (regulatory), Greece (pre-Partnerships launch), Middle East (pre-launch), Taiwan (regulatory) and Israel (regulatory) Pursuing creation of worldwide distributor network Growing cash flow positive business 16
    17. 17. BLU8499 Next generation of tramiprosate intended for the treatment of Alzheimers disease Large and growing epidemic currently affecting Market over 30M patients worldwideopportunity Represents > $180B in annual costs in the United States alone Evidence of effectiveness of parent compound Clinical tramiprosate in ApoE4+ Alzheimer’s patients Evidence Safe and well tolerated in Phase I 17
    18. 18. BLU8499 – Asclepios Partnership Partnership with Asclepios Bioresearch in September 2012 to finance development of BLU8499 Partnership Investment of ~$4M in non-dilutive capital Parties expected to share any future proceeds approximately equally Long term animal toxicity to be completed in 2013Development Phase IIa proof of concept study in mild apoE4+ Plan Alzheimer’s disease patients Expected to begin in 2014 Focused development plan to demonstrate effectiveness in targeted patient population 18
    19. 19. Financial Position and Capital Structure Capital Structure Basic Shares Outstanding 47M Fully Diluted Shares Outstanding1 61M Financial Position Cash (as of September 30th, 2012) >$19M Burn Rate (monthly) <$300K Strategic financing completed with Pharmascience in May 2012 $17.25M total investment: $8.15M in non-dilutive capital and $9.1M for 10.4% stake Operations funded into 2018 1 Does not include stock option grants 19
    20. 20. Governance and ShareholdersBoard of Directors Company / Experience Management TitleDr. Francesco Bellini President and Chief Roberto Bellini(Chair) Executive Officer Senior Vice President, Drug Dr. Denis GarceauFranklin Berger Development François Desjardins Vice President, FinanceCharles Cavell Vice President, Business Tony Matzouranis DevelopmentHélène Fortin LAROSE FORTIN CA Inc.Pierre Larochelle Shareholder OwnershipDonald Olds Bellini Family ≈ 30%Joseph Rus Power Corporation ≈ 30%Dr. Martin Tolar Pharmascience ≈ 10%Roberto Bellini 20
    21. 21. Milestones Milestones Past Execution Long Term Value Completion of recruitment of Attractive KIACTA™ Phase III partnership with Confirmatory Study Results of Phase III Celtic for Kiacta Confirmatory Study Additional KIACTA™ and auction of Execution of global activities: KIACTA™ Kiacta Phase III Confirmatory Study Launch of open label Sale or spin-out of extension study VIVIMIND business Cashflow positive VIVIMIND business Updated market and BLU8499 Phase IIa pricing assessment study results Partnership for BLU8499 BLU 8499 Pre-Phase II package completion Strong balance sheet VIVIMIND partnerships Short-term milestones driving long-term value 21