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Annual Meeting 2013 Roberto Bellini President and Chief Executive Officer May 15, 2013 1

Agm 2013 presentation en (final)

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Page 1: Agm 2013 presentation en (final)

Annual Meeting

2013

Roberto Bellini President and Chief Executive Officer

May 15, 2013

1

Page 2: Agm 2013 presentation en (final)

Forward Looking Statement

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 and/or

nutraceuticals industry, changes in the regulatory environment in the jurisdictions in which

BELLUS Health Inc. does business, stock market volatility, fluctuations in costs, and changes to

the competitive environment due to consolidation, achievement of forecasted burn rate,

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. The length of

the KIACTA Phase III Confirmatory Study is dependent upon many factors including clinical

sites activation, patient enrolment rate, patient drop out rate and occurrence of clinical endpoint

events.

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 Company and its business. 2

Page 3: Agm 2013 presentation en (final)

Business Model

3

Focused

Development

in areas of

high unmet

medical need

Strategic

Partnerships

with strong

industry

players

Identification

of New

Product

Opportunities

Page 4: Agm 2013 presentation en (final)

Focused

Development

4

Dedicated to scientific excellence

Targeted areas of high unmet medical need

Potential for strong shareholder return

Page 5: Agm 2013 presentation en (final)

Pipeline of Products

Pharmaceutical

Nutraceutical

BLU8499

Alzheimer’s

disease

VIVIMIND™

Memory protection

DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III

COMMERCIA-

LIZATION DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III

NDA/MAA

KIACTA™

AA amyloidosis

5

Page 6: Agm 2013 presentation en (final)

KIACTA™

Phase II/III clinical trial showing statistically

significant primary efficacy endpoints (p value =

0.025) and clean safety profile

Patient population estimated at 35,000 to 50,000 in

the USA, Europe and Japan1

Orphan Drug Status in the USA and Europe

provides 7 and 10 years market exclusivity upon

commercialization, respectively

KIACTA™ peak annual revenues projected at

$400-600M1 (USA, Europe, Japan)

1 Market assessment by Frankel Group in April 2009.

For AA Amyloidosis, a rare and deadly

disease with no treatment

Market

opportunity

Clinical

evidence

6

Page 7: Agm 2013 presentation en (final)

Phase III Confirmatory Study

183 patients

Statistically significant composite primary endpoint (p=0.025) principally based on patients reaching kidney function events of worsening:

Doubling serum creatinine

50% decrease in creatinine clearance

Reaching ESRD/dialysis

Death

Fixed treatment duration of 2 years

Completed Phase II/III Study

230 patients

Composite primary endpoint

(target p<0.05) based on patients

reaching kidney function events of

worsening:

Persistent 80% increase serum creatinine

Persistent 40% decrease in creatinine

clearance

Reaching ESRD/dialysis

Event driven trial to conclude on

attainment of 120 events (~90%

power)

KIACTA™ - Phase III Confirmatory Study Design

7

Key improvements made to increase robustness

of confirmatory study

Page 8: Agm 2013 presentation en (final)

KIACTA™ - Study Progress

Recruitment

>65 sites in >25

countries actively

recruiting

152 of 230 patients

enrolled

Recruitment expected

to be completed in 1H

2014

8

Completion

Event driven trial to

complete on reaching

120 events

Study expected to be

completed in 2017

Page 9: Agm 2013 presentation en (final)

9

Lower financial risk

Maintain significant upside value

Bring important external expertise

Strategic

Partnerships

Page 10: Agm 2013 presentation en (final)

Auven Partnership

Global pharmaceutical fund

Strong business expertise

from General Managing

Partner Stephen Evans-

Freke (Sugen, Royalty

Pharma)

Strong drug development

expertise from General

Managing Partner Dr. Peter

Corr (Pfizer)

Financial Implication

US$10M in upfront payments

Auven Therapeutics funding 100% of KIACTA™’s Phase III Confirmatory Study

≥ US$50M in development investments by Auven Therapeutics

Proceeds of any eventual transaction expected to be shared 50%-50% between BELLUS Health and Auven Therapeutics

Auven (previously Celtic) Therapeutics - KIACTA™ Partnership

10

Page 11: Agm 2013 presentation en (final)

KIACTA™ - Exit Strategy

11

RARE DISEASE DRUGS IN THE NEWS

Alnylam Gets $22.5M From Genzyme for Asia Rights to Amyloidosis Drug (October 2012)

Sarepta shares rocket up on

stellar muscular dystrophy trial

results (October 2012) ALEXION TO AQUIRE ENOBIA

PHARMA FOR UP TO $1.08 BILLION

(December 2011)

BioMarin shares pop on strong pivotal

data for rare disease drug (November

2012)

Sale or commercial partnership at end of study or following close

of recruitment

Novartis signs $665m option deal with

Selexys for sickle cell disease drug

(September 2011)

Page 12: Agm 2013 presentation en (final)

VIVIMIND™

Partnerships

COUNTRY / REGION PARTNER Status

ITALY

FB Health Commercial

CANADA Advanced

Orthomolecular

Research

Commercial

MIDDLE EAST Agahan Group Commercial

GREECE Integris Commercial

ISRAEL LevPharm Regulatory

TAIWAN Lifenergy Regulatory

SOUTH KOREA IL-SUNG Regulatory

12

Growing cash flow positive business

VIVIMIND Revenues ($K)

0

100

200

300

400

500

2010 2011 2012

Page 13: Agm 2013 presentation en (final)

BLU8499 – Asclepios Partnership

13

Long term animal toxicity needed

Phase IIa proof of concept study in mild

apoE4+ Alzheimer’s disease patients

Expected to begin in 2014

Development

Plan

Partnership with Asclepios Bioresearch in

September 2012 to finance development of

BLU8499 in Alzheimer’s Disease

Investment of ~$4M in non-dilutive capital

Parties expected to share future proceeds

approximately equally

Partnership

Focused development plan to demonstrate effectiveness in

targeted patient population

Page 14: Agm 2013 presentation en (final)

Shareholders and Financials

Bellini Family ≈ 30%

Power Corporation ≈ 30%

Pharmascience ≈ 10%

Float ≈ 30%

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Financial Position

Cash (December 31st,

2012) >$18M

Burn Rate (monthly) <$300K

Strong shareholder base and financial position

with cash until mid-2018

Fully

Diluted Name

Page 15: Agm 2013 presentation en (final)

15

New Opportunitie

s

Leverage existing intellectual property to start new project(s)

Leverage know how and expertise to add value to acquired project(s)

New

Opportunities

Page 16: Agm 2013 presentation en (final)

Exploring new projects leveraging existing intellectual

property

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KIACTA for AA

Amyloidosis

AL Amyloidosis

ATTR Amyloidosis

Sarcoidosis

Page 17: Agm 2013 presentation en (final)

Bellus Strengths

Expertise of team (drug

development, business

development, capital

markets)

Public vehicle

Strong shareholder base

New Project

High unmet medical need

Close to or in clinical development

Ideally in nephrology or orphan indication

Clear market relevance

New Opportunities – External Sources

17

Leverage BELLUS strengths to develop acquired

or in-licensed new projects

Page 18: Agm 2013 presentation en (final)

Attractive partnership

with Auven

Therapeutics for

KIACTA

Execution of global

KIACTA Phase III

Confirmatory Study

Cashflow positive

VIVIMIND business

Partnership for

BLU8499

Strong balance sheet

• Completion of recruitment of

KIACTA™ Phase III

Confirmatory Study

• Additional KIACTA™

activities:

- Launch of open label

extension study

- Market and pricing

assessment

- Japan orphan drug

designation

• Expansion of product pipeline

• BLU8499 Pre-Phase II package

completion

• VIVIMIND partnerships

Summary

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Past Execution

Milestones (12 months)

Long Term Value

• Results of Phase III

Confirmatory Study

and auction of

KIACTA™

• Sale or spin-out of

VIVIMIND business

• BLU8499 Phase IIa

study results

Short-term milestones driving long-term value

Page 19: Agm 2013 presentation en (final)

Annual Meeting

2013

Roberto Bellini President and Chief Executive Officer

May 15, 2013

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