Bloom Burton Healthcare Investor Conference
Roberto BelliniPresident and Chief Executive OfficerTwitter: @rbellini
May 2, 2016
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Forward Looking StatementsCertain 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, the impact of general economic conditions, general conditions in the pharmaceutical industry, changes in the regulatory environment in the jurisdictions in which the BELLUS Health Inc. does business, stock market volatility, fluctuations in costs, changes to the competitive environment due to consolidation, achievement of forecasted burn rate, potential payments in relation to indemnity agreements, achievement of forecasted clinical trial milestones, and that actual results may vary once the final and quality-controlled verification of data and analyses has been completed. In addition, the length of the KIACTA™ Phase III Confirmatory Study is dependent upon many factors, including clinical sites activation, patient enrollment rate, patient drop-out rate and occurrence of clinical endpoint events, and the sharing of proceeds between Auven Therapeutics and BELLUS Health Inc. from potential future revenue of KIACTA™ is dependent upon a number of factors, including the quantum of proceeds. 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 presentation. 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 BELLUS Health Inc.’s public fillings including the Annual Information Form for further risk factors that might affect BELLUS Health Inc. and its business.
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At BELLUS, we are focused on developing drugs for rare diseases starting with conditions that affect the kidneys.
Value driving rare disease pipeline fully funded through key milestones
Company Highlights
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Late-stage pipeline with 4 projects targeting rare diseases
Lead drug candidate, KIACTA, in Phase III Confirmatory Study for AA amyloidosis Rare and deadly kidney disease with no treatment
Phase II/III study completed with positive efficacy and clean safety
Similar and confirmatory Phase III study completed (Data expected in Q2 2016)
Potential peak market sales of $600M-$1B
Potential exit to commercial partner following Phase III data
Business plan fully funded through KIACTA Phase III and exit process
Late stage pipeline focused on developing innovative drugs for rare diseases
Pipeline of Products
ShigamabsHUS
DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III
KIACTA™AA amyloidosis
MARKET
AL amyloidosis
KIACTA™Sarcoidosis
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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
KIACTA designed to bind AA amyloid, slow down disease progression and delay dialysis 7
KIACTA™ – Addressable Market
Source: Navigant Consulting 2014 8
Estimated Peak Annual Sales
$600 Million-$1Billion
KIACTA Eligible Patients
10-15 Thousand
Expected Pricing Per Patient Per Year
$200-$275 Thousand
Experienced and knowledgeable partner working on lead project
Auven is a global biotech private equity group
Partnered on KIACTA project in 2010
Funding 100% of KIACTA™ project including studies in AA Amyloidosis and Sarcoidosis
≥ US$70M in investments
Overall proceeds of exit expected to be shared 50-50
KIACTA™ to be sold/partnered to commercial entity after Phase III Confirmatory Study results
Auven Therapeutics Partnership for KIACTA™
BUSINESS PLANAUVEN PARTNERSHIP
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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 worsening kidney event
Clear regulatory path to approval based on confirmatory study
Composite Endpoint (Time to
First Worse Event)
Doubling Serum
Creatinine
50%DecreaseCreatinine Clearance
Dialysis/ESRD Death
Num
ber o
f Pat
ient
Eve
nts
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*
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KIACTA™ - Robust Clinical Results in Phase II/III
*p<0.05 **p<0.01
PHASE III CONFIRMATORY STUDYKey entry criteria based on kidney function: High proteinuria (>1 g/d) or low
creatinine clearance (< 60 ml/min/1.73m2)
183 patients in 13 countries
Fixed treatment duration of 2 years 74 kidney function worsening events
Composite primary endpoint based on patients reaching kidney function worsening events Target statistical significance of p=0.01
PHASE II/III STUDYEnriched patient population High proteinuria (>1 g/d)
More patients 261 patients in >25 countries
Increased power Event driven trial to conclude on reaching
120 events
Lower statistical bar to achieve success Primary endpoint with target statistical
significance of p=0.05
KIACTA™ – Phase III Confirmatory Study
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Key improvements made to increase chance of successful study
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Study enrolled with 261 patients
Study completed with 120 events reached (January 2016)
Topline data expected in Q2 2016
Phase III Confirmatory Study
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Clean capital structure and cash runway through potential exit
Corporate
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Capital Markets (as of April 28th, 2016)
Ticker TSX: BLU
Shares (Basic) 54.7M
Shares (Fully Diluted) 65.7M
Daily Volume ~100K
Market Capitalization (FD) ~C$110M
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Finance
Cash (December 31, 2015) C$9.7M
Burn rate (monthly) <C$300K
Shareholder Ownership (FD)
Bellini Family ≈ 29%
Power Corporation ≈ 27%
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
Muriel Lortie
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.
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Potential KIACTA™ exit
Continue executing KIACTA™ for AA Amyloidosis plan:
Reach 120 event target (Q1 2016)
Top Line Data (Q2 2016)
Progress rare disease pipeline projects:
IND filing for KIACTA Phase 2 for Sarcoidosis (1H 2016)
Shigamab animal data (1H 2016)
Shigamab clinical trial design (1H 2016)
Significant news flow and value inflection point in 2016
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
Milestones
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