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Annual Meeting
2013
Roberto Bellini
President and Chief Executive Officer
May 15, 2013
1
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
Business Model
3
Focused
Development
in areas of
high unmet
medical need
Strategic
Partnerships
with strong
industry
players
Identification
of New
Product
Opportunities
Focused
Development
4
 Dedicated to scientific excellence
 Targeted areas of high unmet medical need
 Potential for strong shareholder return
Pipeline of Products
Pharmaceutical
Nutraceutical
BLU8499
Alzheimer’s
disease
VIVIMIND™
Memory protection
DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III
COMMERCIA-
LIZATIONDISCOVERY PRECLINICAL PHASE I PHASE II PHASE III
NDA/MAA
KIACTA™
AA amyloidosis
5
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
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
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
9
 Lower financial risk
 Maintain significant upside value
 Bring important external expertise
Strategic
Partnerships
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
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 $665m option deal with
Selexys for sickle cell disease drug
(September 2011)
VIVIMIND™
Partnerships
COUNTRY / REGION PARTNER Status
ITALY FB Health Commercial
CANADA Advanced
Orthomolecular
Research
Commercial
MIDDLE EAST Agahan Group Commercial
GREECE Integris Commercial
ISRAEL LevPharm Regulatory
TAIWAN Lifenergy Regulatory
SOUTH KOREA IL-SUNG Regulatory
12
Growing cash flow positive business
VIVIMIND Revenues ($K)
0
100
200
300
400
500
2010 2011 2012
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
Shareholders and Financials
Bellini Family ≈ 30%
Power Corporation ≈ 30%
Pharmascience ≈ 10%
Float ≈ 30%
14
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
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
Exploring new projects leveraging existing intellectual
property
16
KIACTA for
AA
Amyloidosis
AL
Amyloidosis
ATTR
Amyloidosis
Sarcoidosis
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
 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
18
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
Annual Meeting
2013
Roberto Bellini
President and Chief Executive Officer
May 15, 2013
19

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Agm 2013 presentation en (final)

  • 1. Annual Meeting 2013 Roberto Bellini President and Chief Executive Officer May 15, 2013 1
  • 2. 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
  • 3. Business Model 3 Focused Development in areas of high unmet medical need Strategic Partnerships with strong industry players Identification of New Product Opportunities
  • 4. Focused Development 4  Dedicated to scientific excellence  Targeted areas of high unmet medical need  Potential for strong shareholder return
  • 5. Pipeline of Products Pharmaceutical Nutraceutical BLU8499 Alzheimer’s disease VIVIMIND™ Memory protection DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III COMMERCIA- LIZATIONDISCOVERY PRECLINICAL PHASE I PHASE II PHASE III NDA/MAA KIACTA™ AA amyloidosis 5
  • 6. 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
  • 7. 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
  • 8. 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
  • 9. 9  Lower financial risk  Maintain significant upside value  Bring important external expertise Strategic Partnerships
  • 10. 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
  • 11. 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 $665m option deal with Selexys for sickle cell disease drug (September 2011)
  • 12. VIVIMIND™ Partnerships COUNTRY / REGION PARTNER Status ITALY FB Health Commercial CANADA Advanced Orthomolecular Research Commercial MIDDLE EAST Agahan Group Commercial GREECE Integris Commercial ISRAEL LevPharm Regulatory TAIWAN Lifenergy Regulatory SOUTH KOREA IL-SUNG Regulatory 12 Growing cash flow positive business VIVIMIND Revenues ($K) 0 100 200 300 400 500 2010 2011 2012
  • 13. 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
  • 14. Shareholders and Financials Bellini Family ≈ 30% Power Corporation ≈ 30% Pharmascience ≈ 10% Float ≈ 30% 14 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
  • 15. 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
  • 16. Exploring new projects leveraging existing intellectual property 16 KIACTA for AA Amyloidosis AL Amyloidosis ATTR Amyloidosis Sarcoidosis
  • 17. 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
  • 18.  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 18 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
  • 19. Annual Meeting 2013 Roberto Bellini President and Chief Executive Officer May 15, 2013 19