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28
Corporate Presentation (TSX: BLU) Roberto Bellini President and Chief Executive Officer Twitter: @rbellini Spring 2015

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Page 1: Bellus corporate presentation march 23

Corporate Presentation (TSX: BLU)

Roberto BelliniPresident and Chief Executive Officer

Twitter: @rbellini

Spring 2015

Page 2: Bellus corporate presentation march 23

Forward Looking Statements

2

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 3: Bellus corporate presentation march 23

3

30 millionpeople in the United States have a RARE disease.

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

Page 4: Bellus corporate presentation march 23

4

Only about 5%of these people have a specific therapy

to treat their disease.

Page 5: Bellus corporate presentation march 23

5

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

Page 6: Bellus corporate presentation march 23

Regulatory advantage

Premium pricing

Market protection

Smaller clinical trials

Efficient commercialization

strategies

6

Small patient numbers, BIG opportunity

Page 7: Bellus corporate presentation march 23

7

At BELLUS, we are focused on developing drugs for rare

diseases starting with conditions that affect the kidneys.

Page 8: Bellus corporate presentation march 23

Business plan fully funded through key milestones

OVERVIEW KEY FINANCIALS

Shareholder Information

8

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 (12/31/14): $12.3M

Burn rate (monthly): <$300K

Page 9: Bellus corporate presentation march 23

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 10: Bellus corporate presentation march 23

Lead Phase III Product Candidate

10

A rare and deadly

kidney disease with

no specific treatment

FOR AMYLOID A (AA)

AMYLOIDOSIS

Page 11: Bellus corporate presentation march 23

Disease and Mechanism of Action

11

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 12: Bellus corporate presentation march 23

Patient Population

Source: Navigant Consulting 2014

10,000-

15,000potential KIACTATM

patients in the United

States and Europe

MARKET RESEARCHNavigant Consulting conducted

extensive primary and secondary

research including more than 60

interviews with treating physicians

and key opinion leaders in the

United States and Europe

12

Page 13: Bellus corporate presentation march 23

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 drugs13

Drug U.S.

PatientsDisease Price

Vyndaqel 1,500Transthyretin amyloid

polyneuropathy$200K

Gattex 9,500 Short Bowel Syndrome $295K

Kalydeco1,350

Cystic Fibrosis (G551D

mutation)$335K

Procysbi500 Nephropathic cystinosis $250K

Juxtapid 3,000Familial

hypercholesterolemia$250K

Jakafi1,500 Splenomegaly $87K

COMPARABLES

Page 14: Bellus corporate presentation march 23

Increasing Number of Orphan Drug Launches…

14

0

20

40

60

80

100

120

2006-2008 2009-2011 2012-2014

Rare Disease Non-Rare Disease

U.S. Drug Launches of New Molecular Entities

Source: IMS

Page 15: Bellus corporate presentation march 23

…Grabbing Increasing Share of Pharma Market

15

Page 16: Bellus corporate presentation march 23

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™

Auven Therapeutics Partnership for KIACTA™

BUSINESS PLANAUVEN PARTNERSHIP

16

Page 17: Bellus corporate presentation march 23

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

Num

ber

of W

ors

e E

vents

17

*

*

**

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

reduction in number and

risk of reaching

worsening kidney event

Important delay in

reaching dialysis

*p<0.05

**p<0.01

Page 18: Bellus corporate presentation march 23

Clean safety profile without any important differences

between groups in Phase II/III study18

KIACTA™ – Clean Safety Profile

98%

36%

23%

93%

42%

25%

0%

20%

40%

60%

80%

100%

AEs SAEs Disc due to AEs

% o

f P

ati

en

ts

KIACTA

Placebo

Page 19: Bellus corporate presentation march 23

19

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 20: Bellus corporate presentation march 23

Study enrolled with 260 patients

Trial concludes when 120 of 230 patients reach event of kidney function deterioration

More than two thirds of the events have been reached

Study completion expected in 2016

Phase III Confirmatory Study

20

Page 21: Bellus corporate presentation march 23

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 2015

21

Page 22: Bellus corporate presentation march 23

Second Rare Disease Product Candidate

22

A rare disease

primarily affecting

the kidneys of

children

FOR STEC RELATED

HEMOLYTIC UREMIC

SYNDROME (SHUS),SHIGAMABSHIGAMAB

Page 23: Bellus corporate presentation march 23

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%

23

90%

SPONTANEOUS

RESOLUTION

10%

SHIGAMAB TREATMENT

Page 24: Bellus corporate presentation march 23

Shigamab Overview

24

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 25: Bellus corporate presentation march 23

Shareholder Ownership

Bellini Family ≈ 30%

Power Corporation ≈ 30%

Pharmascience ≈ 10%

Governance and Shareholders

25

Board of Directors Company / Experience

Dr. Francesco Bellini

(Chair)

Franklin Berger

Charles Cavell

Hélène Fortin

Pierre Larochelle

Murielle Lortie

Joseph Rus

Dr. Martin Tolar

Roberto Bellini

Management Title

Roberto BelliniPresident and Chief

Executive Officer

Dr. Denis GarceauSenior Vice President, Drug

Development

François Desjardins Vice President, Finance

Tony MatzouranisVice President, Business

DevelopmentLAROSE FORTIN CA Inc.

25

Page 26: Bellus corporate presentation march 23

KEY STATS

Shareholder Information

Price (52-week range): $0.44-$1.80

Daily Volume: 20K (3 month avg)

0

50,000

100,000

150,000

200,000

250,000

300,000

$0.00

$0.20

$0.40

$0.60

$0.80

$1.00

$1.20

$1.40

$1.60

$1.80

$2.00

Feb 2014 Mar 2014 Apr 2014 May 2014 Jun 2014 Jul 2014 Aug 2014 Sep 2014 Oct 2014 Nov 2014 Dec 2014 Jan 2015

BLU 1-YR STOCK PERFORMANCE (TSX)

Price Volume

RESEARCH COVERAGE

Bloom Burton (D. Martin) - $3.00

Hybridan (D. Nathan) - $6.00

Zacks (J. Napodano) - $2.50

2626

Page 27: Bellus corporate presentation march 23

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

Focused on achieving short term milestones that drive long term

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

12 Month Milestones

27

Page 28: Bellus corporate presentation march 23