blu corporate presentationjuly152013vfinal

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Corporate Presentation Roberto Bellini President and Chief Executive Officer Twitter: @rbellini July 15, 2013

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Page 1: Blu corporate presentationjuly152013vfinal

Corporate Presentation

Roberto BelliniPresident and Chief Executive OfficerTwitter: @rbellini

July 15, 2013

Page 2: Blu corporate presentationjuly152013vfinal

Forward Looking StatementCertain statements contained in this presentation, other than statements of fact that areindependently verifiable at the date hereof, may constitute forward-looking statements. Suchstatements, based as they are on the current expectations of management, inherently involvenumerous risks and uncertainties, known and unknown, many of which are beyond BELLUSHealth Inc.'s control. Such risks include but are not limited to: the ability to obtain financingimmediately in current markets, the impact of general economic conditions, general conditionsin the pharmaceutical and/or nutraceuticals industry, changes in the regulatory environment inthe 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 andquality-controlled verification of data and analyses has been completed.

Consequently, actual future results may differ materially from the anticipated results expressedin the forward-looking statements. The reader should not place undue reliance, if any, on anyforward-looking statements included in this news release. These statements speak only as ofthe date made and BELLUS Health Inc. is under no obligation and disavows any intention toupdate or revise such statements as a result of any event, circumstances or otherwise, unlessrequired by applicable legislation or regulation. Please see the Company’s public fillingsincluding the Annual Information Form of BELLUS Health Inc. for further risk factors that mightaffect the BELLUS Health Group and its business

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Background and Business Model

Public company (TSX: BLU) based in Montreal, QCFocused on products in amyloid-related fields, principally AA Amyloidosis, an orphan indication affecting the kidneys

Late-stage product pipeline with fully funded business plan

3

Business ModelFocused on building value for clinical stage health products in critical unmet medical needs

Financial Facts Shares outstanding (Fully

Diluted): 65M

Cash (03/31/13): ~$18M

Burn rate (monthly): <$300K

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Pipeline of Products

PHARMACEUTICALS

NUTRACEUTICAL

NRM8499Alzheimer’s disease

VIVIMIND™Memory protection

DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III

MARKETDISCOVERY PRECLINICAL PHASE I PHASE II PHASE III

NDA/MAA

KIACTA™AA amyloidosis confirmatory

Thallion acquisition announced June 18th to expand pipeline with addition of Phase II product Shigamabs for sHUS

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KIACTA™ Overview

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For Amyloid A (AA) Amyloidosis, a rare and deadly disease with no specific treatment

Market opportunity

Clinical evidence

1 Market assessment by Frankel Group in April 2009.

Partnership

Orphan population of ≈50,000 in the USA, Europe and Japan with peak annual revenues projected at $400-600M1

Phase II/III clinical trial showing statistically significant primary efficacy endpoints(p value = 0.025) and clean safety profile

Partnership with Auven Therapeutics (previously known as Celtic Therapeutics) to conduct and finance (>$50M) Phase III Confirmatory Study

Phase III Confirmatory

Study

Marketing approval based on confirming safety and efficacy of phase II/III study

>66% patients recruited

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Mechanism of Action

CHRONIC INFLAMMATION

SERUM AMYLOID APRECURSOR (SAA) PROTEIN

AA PROTEIN + GLYCOSAMINOGLYCANS(GAGs)

ORGAN DAMAGE, IN PARTICULAR TO KIDNEYS

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

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KIACTA™ - Targeted Opportunity

7

Dia

gnos

ed A

A Pa

tient

s (0

00s)

Patient population estimated at 34,000-50,000 in the United States, Europe Top 5 and Japan1

KIACTA™ peak annual revenues projected at $400-600M1

(U.S., EU5, Japan)Strong potential for premium pricing Clear pharmacoeconomic

rationale due to high cost ofkidney disease

Comparative orphan drug pricing

1Market assessment by Frankel Group in April 2009

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FINANCIAL IMPLICATION

With global fund AuvenTherapeutics

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

Auction process for the commercialization rights of KIACTA™ on completion of Phase III Confirmatory Study

PARTNERSHIP

US$10M in upfront payments

≥ US$50M in investments by Auven Therapeutics

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

Strategic Partnership

Partnership to fund Phase III Confirmatory Study with significant upside for BELLUS shareholders

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HR 0.58 0.41 0.48 0.54 0.95

95% C.I 0.37, 0.93 0.19,0.86 0.28,0.82 0.22,1.37 0.27,3.29

P value 0.025 0.019 0.008 0.20 0.94

Graphical representation of the information in this table

• Landmark study in AA Amyloidosis: 183 patients treated for 2 years

• Composite endpoint based on patients reaching events of decreasing kidney function or death

• Statistically significant primary endpoint (Cox Proportional Hazard Ratio; p=0.025)

• Clinically meaningful treatment effect with 42% reduction in risk of reaching event

• Most sensitive componentof composite endpoint highly significant (CrCl, p < 0.01)

Composite Endpoint (Time to First Worse

Event)

Doubling Serum

Creatine

50%DecreaseCreatine

CIearance

Dialysis/ESRD Death

Num

ber o

f Pat

ient

Eve

nts

9

*

*

*

KIACTA™ - Robust Clinical Results in Phase II/III

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KIACTA™ - Phase II/III Key Secondary Endpoints

HR=0.58Wald Chi Square test: p=0.025

KIACTATM

Placebo

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Key secondary endpoints show robust effect of KIACTA™

Using slope of creatinine clearance decline, calculated delayto time of dialysis is ~2 years on Kiacta versus placebo

Kaplan Meier: Time to First Worst Event Slope of Creatinine Clearance Decline

Placebo

Months of Treatment

Mea

n ∆

CrC

l(m

L/m

in/1

.73m

2 )

= 9.4mL/min/1.73 m2

after 24 months p=0.025

-10.9 ± 5.1 mL/min/1.73 m2/year

-15.6 ± 4.0 mL/min/1.73 m2/year

KIACTATM

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KIACTA™ - Phase II/III Feedback

2007 NEJM article published by lead investigators concludes that KIACTA™ slows decline of renal function in AA Amyloidosis

Agreement reached under Special Protocol Assessment with FDA and Scientific Advice with EMEA to conduct Phase III Confirmatory Study

Marketing approval based on positive result (p value <0.05) from confirmatory study with same scope of first phase II/III clinical trial

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PHASE III CONFIRMATORY STUDY183 patients in 13 countries

Composite primary endpoint based on patients reaching kidney function worsening events: Doubling serum creatinine

50% decrease in creatinine clearance

Reaching ESRD/dialysis

Death

Fixed treatment duration of 2 years

PHASE II/III STUDY230 patients in >25 countries

Composite primary endpoint (target p<0.05) based on patients reaching kidney function worsening events:

Persistent 80% increase serum creatinine

Persistent 40% decrease in creatinineclearance

Reaching ESRD/dialysis

Event driven trial to conclude on attainment of 120 events (~90% power)

KIACTA™ - Phase III Confirmatory Study

Key improvements made to increase robustness of Phase III Confirmatory Study

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Study ProgressSTUDY UPDATE (May 2013)

>65 sites in >25 countries activated

>150 patients enrolled

Recruitment expected to be completed in 1H 2014

COMPLETION

Event driven trial to complete on reaching 120 events

Study expected to be completed in 2017

Patient baseline characteristics and demographicsto date are similar to those in the first Phase III study

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Thallion Transaction - OverviewBellus to acquire Thallion for $6.3M in cash plus certain contingent value rights (CVRs):

$6.3M in cash consideration1 ($0.1904 per share) based on $7.5M net cash at closing;

100% of expected $1.4M 2016 payment (up to $0.041 per share)

5% of Shigamabs product revenue, capped at $6.5M (up to $0.181 per share)

Binding agreement and announcement on June 17th

Unanimous support from Thallion and Bellus Board of Directors

Voting support agreements in place currently for more than 24% of votes, including 14.5% from largest shareholder Jaguar Financial

Transaction to proceed by court supervised plan of arrangement requiring 66 2/3% shareholder approval

Thallion shareholder meeting planned for August 6th

Close of transaction expected shortly after shareholder meeting

1 Cash consideration to be adjusted (up and down) on a dollar for dollar based on net cash 5 days before closing

Acquisition rationale based on building pipeline with addition of Shigamabs, a complementary Phase 2 asset

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Thallion Transaction – BenefitsBenefits to Bellus:

Adds Shigamabs to pipeline, a complementary Phase II asset targeting a rare disease affecting the kidneys

Net cash of ~$1.2M from transaction provides funding for Shigamabs development

Benefits to Thallion:

Provides initial cash consideration at significant premium to stock price, 46% premium from announcement date and 90% premium from initiation of discussions

Provides additional value in CVR with potential to achieve aggregate ~$0.41 per share, 215% premium from announcement date and 310% premium from initiation of discussions

Transaction is executionally low risk and also tax efficient for Thallion shareholders

Mutually beneficial transaction for Thallion and Bellus shareholders

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Shigamabs Overview

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For STEC related Hemolytic Uremic Syndrome (sHUS), an ultra rare disease primarily affecting the kidneys

Market opportunity

Clinical evidence

Mechanism

Shigatoxin producing e.coli (STEC) is a rare food borne illness1,500-2,000 annual cases progress to sHUS in developed countries, principally in childrenPremium pricing potential

Safety in target pediatric population from 45 patient Phase 2 study

Monoclonal antibody binds and neutralizes Stx-2, the most prominent and virulent variant of STEC

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sHUS (5%-10%)

Resolve (90%-95%)

Ingestion Colonization Toxin Secretion

-4 0 2 4 6 8-2Day

Thallion intervention (prevention)

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Shigamabs Development Strategy

Development thesis to reposition Shigamabs from prevention of sHUS to treatment of sHUS

Support from key opinion leader community for downstream therapeutic approach to sHUS

Recent publication supports role of toxin neutralization for treatment of sHUS

Conduct proof of concept studies in several HUS animal models to validate approach

Clear clinical and regulatory path into Phase II proof of concept study

Only treating patients with high unmet medical need

Clear endpoint based on reduction in need for dialysis (40-50% of sHUS patients require dialysis)

Dialysis is considered clinically relevant by regulatory agencies

Bellus intervention

STEC and sHUS Disease Course

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VIVIMIND™

Nutraceutical for memory protection

Regulatory

Partnerships

Growing cash flow positive business

Partnerships for Italy (launched), Canada (launched), South Korea (regulatory), Greece (pre-launch), Middle East (pre-launch), Taiwan (regulatory) and Israel (regulatory)Pursuing creation of worldwide distributor network

Health claims include: ‘Protects the hippocampus’ and ‘Enhances cognitive function and memory’

VIVIMIND Revenues ($K)

0

100

200

300

400

500

2010 2011 2012

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BLU8499

Next generation of tramiprosate intended for the treatment of Alzheimer's disease

Market opportunity

Clinical evidence

Partnership

Large and growing epidemic currently affecting over 30M patients worldwide

Evidence of effectiveness of parent compound tramiprosate in ApoE4+ Alzheimer’s patientsSafe and well tolerated in Phase I

Partnership with Asclepios Bioresearch in September 2012 to finance development of BLU8499 into Phase 2aCommitment of ~$4M in non-dilutive capital

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Capital Structure

Basic Shares Outstanding 47M

Fully Diluted Shares Outstanding 65M

Operations funded into mid-201820

Financial Position and Capital Structure

Financial Position

Cash (March 31st, 2013) ~$18M

Burn Rate (monthly) <$300K

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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 DevelopmentLAROSE FORTIN CA Inc.

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Short-term milestones driving long-term value

Milestones

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Past ExecutionAttractive partnership with for Kiacta

Execution of global KIACTA Phase III Confirmatory Study

Cashflow positive VIVIMIND business

Partnership for BLU8499

Strong balance sheet and clean capital structure

Milestones (12 months)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

BLU 8499 Pre-Phase II package completion

VIVIMIND partnerships

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

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