bellus corporate presentation april 5 2016

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Corporate Presentation (TSX: BLU) Roberto Bellini President and Chief Executive Officer Twitter: @rbellini April 2016 r

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Page 1: Bellus corporate presentation April 5 2016

Corporate Presentation (TSX: BLU)

Roberto BelliniPresident and Chief Executive OfficerTwitter: @rbellini

April 2016

r

Page 2: Bellus corporate presentation April 5 2016

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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Page 3: Bellus corporate presentation April 5 2016

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At BELLUS, we are focused on developing drugs for rare diseases starting with conditions that affect the kidneys.

Page 4: Bellus corporate presentation April 5 2016

Regulatory advantage

Premium pricing

Market protection

Smaller clinical trials

Efficient commercialization strategies

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Small patient numbers, BIG opportunity

Page 5: Bellus corporate presentation April 5 2016

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

Page 6: Bellus corporate presentation April 5 2016

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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Page 7: Bellus corporate presentation April 5 2016

Lead Phase III Product Candidate

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A rare and deadly kidney disease with no specific treatment

FOR AMYLOID A (AA) AMYLOIDOSIS

Page 8: Bellus corporate presentation April 5 2016

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 8

Page 9: Bellus corporate presentation April 5 2016

Patient Population

Source: Navigant Consulting 2014

10,000-15,000

potential KIACTATM patients in the United States and Europe

MARKET RESEARCHNavigant Consulting conducted extensive primary and secondary research including over 60 interviews with treating physicians and key opinion leaders in the United States and Europe

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Page 10: Bellus corporate presentation April 5 2016

PRICING

Orphan drug designation granted with

market protection in the U.S. (7 years),

Europe and Japan (10 years)

Intellectual property protection to 2031

PROTECTION

Disease with large unmet medical need and no specific treatment

Clear pharmaco-economic rationale due to high cost of kidney disease

Premium pricing for comparative rare disease drugs

Market Considerations

KIACTA is well positioned to achieve premium pricing in line with comparable rare disease drugs

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Drug U.S. Patients Disease Price

Vyndaqel 1,500 Transthyretin amyloid polyneuropathy $200K

Gattex 9,500 Short Bowel Syndrome $295K

Kalydeco 1,350 Cystic Fibrosis (G551D mutation) $335K

Procysbi 500 Nephropathic cystinosis $250K

Juxtapid 3,000 Familial hypercholesterolemia $250K

Jakafi 1,500 Splenomegaly $87K

COMPARABLES

Page 11: Bellus corporate presentation April 5 2016

KIACTA™ – Addressable Market

Source: Navigant Consulting 2014 11

Estimated Peak Annual Sales

$600 Million-$1Billion

KIACTA Eligible Patients

10-15 Thousand

Expected Pricing Per Patient Per Year

$200-$275 Thousand

Page 12: Bellus corporate presentation April 5 2016

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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Page 13: Bellus corporate presentation April 5 2016

POTENTIAL ACQUIRERSPharma/Big biotech with inflammation and/or nephrology franchise

Orphan disease focused biotech

Exit Strategy

Strong M&A environment for rare disease products13

Company Main Drug / Disease Stage Transaction

Synageva Kanuma/ LAL-D Registration (no sales) Acquired by Alexion in June 2015 for $8.4B

NPSGattex / Short Bowel Syndrome Market ($350M in sales) Acquired by Shire in January 2015 for

$6.2B

Scioderm Zorblisa / E. Bullosa Phase 3 (no sales)Acquired by Amicus in August 2015 for $230M upfront plus $600M in milestones

RECENT RARE DISEASE M&A

Page 14: Bellus corporate presentation April 5 2016

0

5

10

15

20

25

30

35

40

45

50

PlaceboKIACTA

Composite Endpoint (Time to

First Worse Event)

Doubling Serum

Creatinine

50%DecreaseCreatinine CIearance

Dialysis/ESRD

Num

ber o

f Wor

se E

vent

s

14

*

*

**

Strong Clinical Results in Phase II/III Study

Landmark study in AA amyloidosis: 183 patients treated for 2 years

Important benefits for patients on drug:

Statistically significant (p-value=0.025) reduction in number and risk of reaching worsening kidney event

Important delay in reaching dialysis

*p<0.05 **p<0.01

Page 15: Bellus corporate presentation April 5 2016

Clean safety profile without any important differences

between groups in Phase II/III study 15

KIACTA™ – Clean Safety Profile

Adverse Events Serious Adverse Events Discontinuations due to Adverse Events

0%

20%

40%

60%

80%

100% 98%

36%

23%

93%

42%

25%

KIACTA

Placebo

% o

f Pat

ient

s

Page 16: Bellus corporate presentation April 5 2016

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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

Marketing approval based on achieving comparable result with lower statistical bar than first Phase III Study

Page 17: Bellus corporate presentation April 5 2016

PHASE III CONFIRMATORY STUDY183 patients in 13 countries

Composite primary endpoint based on patients reaching kidney function worsening events Target statistical significance of p=0.01

Key entry criteria based on kidney function: High proteinuria (>1 g/d) or low

creatinine clearance (< 60 ml/min/1.73m2)

Fixed treatment duration of 2 years 74 kidney function worsening events

PHASE II/III STUDYMore patients 261 patients in >25 countries

Lower statistical bar to achieve success Primary endpoint with target statistical

significance of p=0.05

Enriched patient population High proteinuria (>1 g/d)

Increased power Event driven trial to conclude on reaching

120 events

KIACTA™ – Phase III Confirmatory Study

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Key improvements made to increase chance of successful study

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Page 18: Bellus corporate presentation April 5 2016

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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Page 19: Bellus corporate presentation April 5 2016

Second KIACTA™ Indication – Sarcoidosis

INDICATION

DEVELOPMENT

Chronic sarcoidosis, a rare disease that causes lung scarring and decreased lung functionKIACTA target Serum Amyloid A plays key role in triggering disease

Agreement with Mount Sinai Hospital New York to start Phase 2 proof-of-concept studyIND filing expected in 1H 2016

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Page 20: Bellus corporate presentation April 5 2016

Second Rare Disease Product Candidate

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A rare disease primarily affecting the kidneys of children

FOR STEC RELATEDHEMOLYTIC UREMIC SYNDROME (SHUS),

SHIGAMABSHIGAMAB

Page 21: Bellus corporate presentation April 5 2016

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

Shigamab Antibody

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

Page 22: Bellus corporate presentation April 5 2016

Data presented at VTEC conference September 14-16Mice rescued from shigatoxin induced weight loss and kidney injury up to 4 days post intoxication

Shigamab Overview

NEXT STEPS (12 MONTHS)

MARKET OPPORTUNITY

CLINICAL

Further animal model data in treatment of sHUSMeetings with regulators to agree on clinical development plan

2,000-3,000 estimated annual cases of sHUS in developed countries, principally children$100-200 million annual sales opportunity

Safe and well tolerated in target pediatric population

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PRE- CLINICAL

Page 23: Bellus corporate presentation April 5 2016

Clean capital structure and cash runway through potential exit

Corporate

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Capital Markets (as of April 1st, 2016)

Ticker TSX: BLU

Shares (Basic) 54.7M

Shares (Fully Diluted) 65.7M

Volume (3 month) ~100K

Market Capitalization (FD) ~C$100M

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Finance

Cash (December 31, 2015) C$9.7M

Burn rate (monthly) <C$300K

Page 24: Bellus corporate presentation April 5 2016

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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Page 25: Bellus corporate presentation April 5 2016

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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Page 26: Bellus corporate presentation April 5 2016

Connect With Us

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Read our blog @ www.bellushealth.com

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