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

Bellus Corporate Presentation - February 2016

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Corporate Presentation (TSX: BLU)

Roberto BelliniPresident and Chief Executive Officer

Twitter: @rbellini

February, 2016

r

Forward Looking Statements

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, 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.2

3

At BELLUS, we are focused on developing drugs for rare

diseases starting with conditions that affect the kidneys.

Regulatory advantage

Premium pricing

Market protection

Smaller clinical trials

Efficient commercialization

strategies

4

Small patient numbers, BIG opportunity

Value driving rare disease pipeline fully funded through key

milestones

Investment Highlights

5

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 full funded through KIACTA Phase III and exit

process

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

6

Lead Phase III Product Candidate

7

A rare and deadly

kidney disease with

no specific treatment

FOR AMYLOID A (AA)

AMYLOIDOSIS

Disease and Mechanism of Action

8

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

KIACTA designed to bind AA amyloid, slow down disease

progression and delay dialysis

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 over 60

interviews with treating physicians

and key opinion leaders in the

United States and Europe

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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 drugs10

Drug U.S. Patients Disease 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

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

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

12

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

NPS

Gattex / 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

Tripex Quincair / Cystic Fibrosis Registration (no sales)

Acquired by Raptor in August 2015 for

$68M upfront plus $350M in

milestones and royalties

RECENT RARE DISEASE M&A

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

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

Clean safety profile without any important differences

between groups in Phase II/III study15

KIACTA™ – Clean Safety Profile

98%

36%

23%

93%

42%

25%

0%

20%

40%

60%

80%

100%

Adverse Events Serious Adverse Events Discontinuations due toAdverse Events

% o

f P

ati

en

ts

KIACTA

Placebo

16

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

PHASE III CONFIRMATORY STUDY

183 patients in 13 countries

Composite primary endpoint based on patients reaching kidney function worsening events

Target statistical significant 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 STUDY

More patients

261 patients in >25 countries

Lower statistical bar to achieve

success

Primary endpoint with target statistical significant 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

17

Key improvements made to increase chance of successful study17

Study enrolled with 261 patients

Study completed with 120 events

reached (January 2016)

Topline data expected in Q2 2016

Phase III Confirmatory Study

18

Second KIACTA™ Indication – Sarcoidosis

INDICATION

DEVELOPMENT

Chronic sarcoidosis, a rare

disease that causes lung scarring

and decreased lung function

KIACTA target Serum Amyloid A

plays key role in triggering

disease

Agreement with Mount Sinai Hospital

New York to start Phase 2 proof-of-

concept study

IND filing expected in 1H 2016

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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),SHIGAMABSHIGAMAB

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%

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90%

SPONTANEOUS

RESOLUTION

10%

SHIGAMAB TREATMENT

Data presented at VTEC conference September 14-16

Mice 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 sHUS

Meetings 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

Clean capital structure and cash runway through potential exit

Corporate

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Capital Markets (as of February 23th, 2015)

Ticker TSX: BLU

Shares (Basic) 54.7M

Shares (Fully Diluted) 65.7M

Market Capitalization (FD) C$76M

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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

24

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 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.

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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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Connect With Us

Follow us on Twitter: @BELLUSHealth

Join our LinkedIn group

Read our blog @ www.bellushealth.com

Join our mailing list

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