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Management Presentation Roberto Bellini President and Chief Executive Officer Twitter: @rbellini Annual General Meeting 2015
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Page 1: Management Presentation - May 2015

Management Presentation

Roberto BelliniPresident and Chief Executive Officer

Twitter: @rbellini

Annual General Meeting 2015

Page 2: Management Presentation - May 2015

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” within the meaning of Canadian

securities legislation and regulations. Such statements, based as they are on the current expectations of

management, inherently involve numerous important risks, uncertainties and assumptions, known and

unknown, many of which are beyond BELLUS Health Inc.'s control. Such risks factors 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 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/outcomes in relation to indemnity

agreements and contingent value rights, achievement of forecasted clinical trial milestones, dependence on

Auven Therapeutics for the completion of the KIACTA™ Phase III Confirmatory Study 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

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 and events may differ materially from the anticipated results and events

expressed in the forward-looking statements. BELLUS Health Inc. believes that expectations represented by

forward-looking statements are reasonable, yet there can be no assurance that such expectations will prove

to be correct. The reader should not place undue reliance, if any, on any forward-looking statements included

in this presentation. These forward-looking statements speak only as of the date made, and BELLUS Health

Inc. is under no obligation and disavows any intention to update publicly or revise such statements as a result

of any new information, future event, circumstances or otherwise, unless required by applicable legislation or

regulation. Please see BELLUS Health Inc.’s public filings with the Canadian securities regulatory authorities,

including the Annual Information Form, for further risk factors that might affect BELLUS Health Inc. and its

business.

Page 3: Management Presentation - May 2015

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30 millionpeople in the United States have a RARE disease.

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

Page 4: Management Presentation - May 2015

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Only about 5%of these people have a specific therapy

to treat their disease.

Page 5: Management Presentation - May 2015

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

Page 6: Management Presentation - May 2015

Regulatory advantage

Premium pricing

Market protection

Smaller clinical trials

Efficient commercialization

strategies

6

Small patient numbers, BIG opportunity

Page 7: Management Presentation - May 2015

Increasing Number of Orphan Drug Launches…

7

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 8: Management Presentation - May 2015

…Grabbing Increasing Share of Pharma Market

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Almost 20% of global drug market will come from rare disease drug

sales by 2020

Worldwide Orphan Drug Sales & Share of Prescription Drug Market (2000-20)

Source: EvaluatePharma® (27 OCT 2014)

Page 9: Management Presentation - May 2015

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At BELLUS, we are focused on developing drugs for rare

diseases starting with conditions that affect the kidneys.

Page 10: Management Presentation - May 2015

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Strong Team

Diverse Product Pipeline

Focused Business Model

Develop rare

disease drugs

and

Deliver

shareholder value

Page 11: Management Presentation - May 2015

Diverse Product Pipeline

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Page 12: Management Presentation - May 2015

Diverse 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

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Page 13: Management Presentation - May 2015

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Patient Profile

Underlying inflammatory

disease causes amyloid

deposition in kidneys

50-60 years old

Median time to dialysis: 8 years

How does KIACTA work?

Inhibits formation of amyloid

fibrils and amyloid deposits

in the kidney

KIACTA™ – Treating AA Amyloidosis Patients

Page 14: Management Presentation - May 2015

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KIACTA™ - Phase II/III Data

Phase 2/3 data published in

New England Journal of Medicine

publication:

• 42% reduction in risk (p=0.025) of

worsening renal events for Kiacta patients

• “Benefits [of Kiacta] are clinically

meaningful and were evident early in the

course of treatment”

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 15: Management Presentation - May 2015

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KIACTA - Phase III Confirmatory Study Progress:

Fully enrolled with 261 patients

Completion expected in 2016

Page 16: Management Presentation - May 2015

KIACTA™ – Addressable Market

Source: Navigant Consulting 2014 16

Estimated Peak Annual Sales

$600 Million-$1Billion

KIACTA Eligible Patients

10-15 Thousand

Expected Pricing

$200-$275 Thousand

Page 17: Management Presentation - May 2015

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Patient Profile

Idiopathic rare lung condition

Chronic form causes

increasing loss of lung

function and potentially death

Next Steps

Begin proof of concept study

in 2H 2015

Second KIACTA™ Indication - Sarcoidosis

Page 18: Management Presentation - May 2015

Shigamab – Treating sHUS Patients

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Patient Profile

Triggered by ingestion of toxin producing e.coli

Immune compromised patients: most below <10 years old

Immediate kidney damage and chance of death

How does SHIGAMAB work?

Antibody that binds and neutralizes sHUS toxin

Page 19: Management Presentation - May 2015

Focused

Business

Model

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Page 20: Management Presentation - May 2015

Experienced and knowledgeable partner working on lead project

• Biotech private equity group

funding 100% of KIACTA™

project including studies in AA

Amyloidosis and Sarcoidosis

≥ US$70M in investments

• KIACTA™ to be sold either prior

to, or after, Phase III

Confirmatory Study results

Overall proceeds of exit expected

to be shared 50-50

Investment bank Lazard engaged to

explore sale of KIACTA™

Auven Therapeutics Partnership for KIACTA™

PARTNERSHIP

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Page 21: Management Presentation - May 2015

Financed through KIACTA™ Phase 3 data and exit

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Cash

$12.3M(YE 2014)

Burn Rate<$0.3M /month

Page 22: Management Presentation - May 2015

Strong

Team

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Page 23: Management Presentation - May 2015

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

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.

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Page 24: Management Presentation - May 2015

Driving

Shareholder

Value

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Page 25: Management Presentation - May 2015

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

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Page 26: Management Presentation - May 2015

Management Presentation

Roberto BelliniPresident and Chief Executive Officer

Twitter: @rbellini

Annual General Meeting 2015