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Corporate Presentation (TSX: BLU) Roberto Bellini President and Chief Executive Officer Twitter: @rbellini Fall 2014

Corporate Presentation Fall 2014

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Bellus Fall 2014 Corporate Presentation

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Page 1: Corporate Presentation Fall 2014

Corporate Presentation (TSX: BLU)

Roberto Bellini President and Chief Executive Officer

Twitter: @rbellini

Fall 2014

Page 2: Corporate Presentation Fall 2014

2

30 million people in the United States have a RARE disease.

Source: NIH: National Institutes of Health Office of Rare Diseases

Page 3: Corporate Presentation Fall 2014

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Only about 5%

of these people have a specific therapy

to treat their disease.

Page 4: Corporate Presentation Fall 2014

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85-90% of rare diseases are serious or life threatening.

Page 5: Corporate Presentation Fall 2014

Regulatory advantage

Premium pricing

Market protection

Smaller clinical trials

Efficient commercialization

strategies

5

Small patient numbers, BIG opportunity

Page 6: Corporate Presentation Fall 2014

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At BELLUS, we are focused on developing drugs for rare

diseases starting with conditions that affect the kidneys.

Page 7: Corporate Presentation Fall 2014

Business plan fully funded through key milestones

OVERVIEW CAPITAL STRUCTURE

Shareholder Information

7

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 (06/30/14): ~$13M

Burn rate (monthly): <$300K

Shareholder makeup:

70% institutional, 30% retail

Page 8: Corporate Presentation Fall 2014

Late stage pipeline focused on developing innovative drugs for rare

diseases

Pipeline of Products

Shigamab

sHUS

DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III

KIACTA™

AA amyloidosis

MARKET

AL amyloidosis

KIACTA™

Sarcoidosis

9

Page 9: Corporate Presentation Fall 2014

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 10: Corporate Presentation Fall 2014

Disease and Mechanism of Action

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CHRONIC

INFLAMMATION

SERUM AMYLOID A

PRECURSOR (SAA)

PROTEIN

AA PROTEIN +

GLYCOSAMINOGLYCANS

(GAGs)

ORGAN DAMAGE, IN

PARTICULAR TO

KIDNEYS LEADING TO

DIALYSIS

REDUCTION IN

FIBRIL FORMATION

& DEPOSITION

Converts to

AA Protein Generates

cytokine cascade

(TNFα / IL-1 / IL-6)

and increases SAA levels

Rheumatic Conditions

Inflammatory Bowel Disease

Chronic Infections

Familial Mediterranean Fever

KIACTA™ blocks

AA + GAGs interaction

Systemic Amyloid A Fibril

Formation & Deposition

Page 11: Corporate Presentation Fall 2014

Patient Population

Source: Navigant Consulting 2014

10,000-

15,000 potential KIACTATM

patients in the United

States and Europe

MARKET RESEARCH Navigant Consulting conducted

extensive primary and secondary

research including over 60

interviews with treating physicians

and key opinion leaders in the

United States and Europe

11

Page 12: Corporate Presentation Fall 2014

PRICING

Orphan drug designation granted with

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

Europe and Japan (10 years)

Intellectual property to 2026

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 12

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 13: Corporate Presentation Fall 2014

Experienced and knowledgeable partner working on lead project

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 through

auction process either prior to, or

after, Phase III Confirmatory Study

results

Investment bank Lazard engaged

to explore sale of KIACTA™ in

May 2014.

Auven Therapeutics Partnership for KIACTA™

BUSINESS PLAN AUVEN PARTNERSHIP

17

Page 14: Corporate Presentation Fall 2014

0

5

10

15

20

25

30

35

40

45

50

Placebo

KIACTA

Composite

Endpoint (Time to

First Worse

Event)

Doubling

Serum

Creatinine

50%

Decrease

Creatinine

CIearance

Dialysis/

ESRD

Nu

mb

er

of W

ors

e E

ve

nts

14

*

*

**

Strong Clinical Results in First Phase III Study

Landmark study in AA

amyloidosis: 183 patients

treated 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

Page 15: Corporate Presentation Fall 2014

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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

Page 16: Corporate Presentation Fall 2014

Study enrolled with ~230 patients

Trial concludes when 120 of 230

patients reach event of kidney

function deterioration

Study completion expected in 2016

Phase III Confirmatory Study

12

Page 17: Corporate Presentation Fall 2014

New KIACTA™ Indication - Sarcoidosis

INDICATION

DEVELOPMENT

Chronic sarcoidosis, a rare

disease that causes lung scarring

and decreased lung function

No specific treatment

Agreement with Mount Sinai Hospital

New York to start Phase 2 proof-of-

concept study in late 2014/early 2015

13

Page 18: Corporate Presentation Fall 2014

Second Rare Disease Product Candidate

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A rare disease

primarily affecting

the kidneys of

children

FOR STEC RELATED

HEMOLYTIC UREMIC

SYNDROME (SHUS), SHIGAMAB

Page 19: Corporate Presentation Fall 2014

Disease Course and Mechanism of Action

E. COLI INGESTION

GUT COLONIZATION AND

SECRETION OF TOXIN

INTO BLOODSTREAM

TOXIN MAY BE CARRIED

BY PMNs IN

BLOODSTREAM

SYMPTOMS: 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%

19

90%

SPONTANEOUS

RESOLUTION

10%

SHIGAMAB TREATMENT

Page 20: Corporate Presentation Fall 2014

Potential for partnership in 18-24 months

Shigamab Overview

20

NEXT STEPS (12 MONTHS)

MARKET OPPORTUNITY

CLINICAL

Proof-of-concept for treatment of sHUS in animal models

Meetings 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

Safe and well tolerated in target pediatric population

Page 21: Corporate Presentation Fall 2014

Research Program

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A rare and deadly

blood disorder

RESEARCH PROJECT FOR

AMYLOID LIGHT-CHAIN

(AL) AMYLOIDOSIS, DRUG CANDIDATES AL

AMYLOIDOSIS

Page 22: Corporate Presentation Fall 2014

Potential for pre-clinical, proof-of-concept within 12 months

AL Amyloidosis Project Overview

22

PARTNERSHIP

DISEASE

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

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

Page 23: Corporate Presentation Fall 2014

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.

Page 24: Corporate Presentation Fall 2014

Focused on achieving milestones that create value for

shareholders

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

20

Potential KIACTA™ exit

Continue executing KIACTA™ for AA

Amyloidosis plan:

Completion of recruitment

Launch of open label extension

study

Market assessment

Progress rare disease pipeline projects:

IND filing for KIACTA Phase 2 for

Sarcoidosis

Animal studies to support

Shigamab Phase II

Pre-clinical, proof-of-concept for

AL amyloidosis

12 Month Milestones

Page 25: Corporate Presentation Fall 2014

Forward Looking Statements

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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

Page 26: Corporate Presentation Fall 2014

Corporate Presentation (TSX: BLU)

Roberto Bellini President and Chief Executive Officer

Twitter: @rbellini

Fall 2014