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Corporate Presentation




Roberto Bellini
President and Chief Executive Officer


November 2012
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
immediately in current markets, 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 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 and
quality-controlled verification of data and analyses has been completed.


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 BELLUS Health Group and its business



                                                                                                    2
Background and Business Model
 Public company (TSX: BLU) based in Montreal, QC
 Focused namely on the development of products in amyloid-
 related fields, principally AA Amyloidosis, an orphan indication
 affecting the kidneys
 Pipeline also includes product candidate for the treatment of
 Alzheimer’s disease



                BUSINESS MODEL
            Focused on building value for clinical
            stage health products in critical unmet
                        medical needs


                                                                    3
Pipeline of Products


PHARMACEUTICALS     DISCOVERY   PRECLINICAL PHASE I   PHASE II   PHASE III   NDA/MAA

KIACTA™
AA amyloidosis


BLU8499
Alzheimer’s
disease

                                                                             COMMERCIA
NUTRACEUTICAL       DISCOVERY   PRECLINICAL PHASE I   PHASE II   PHASE III    -LIZATION

VIVIMIND™
Memory protection




                                                                                          4
KIACTA™
               For AA Amyloidosis, an orphan indication and a
               deadly disease with no treatment

                      Orphan population of ≈50,000 in the USA, Europe
   Market             and Japan with peak annual revenues projected at
 opportunity          $400-600M1
                      1 Independent   market assessment by Frankel Group in April 2009.



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

                      Partnership with Celtic Therapeutics to conduct
 Partnership          and finance (>$50M) Phase III Confirmatory
                      Study
                      Marketing approval based on confirming safety
  Phase III           and efficacy of phase II/III study
Confirmatory
   Study              Actively recruiting patients
                                                                                          5
AA Amyloidosis – A Rare and Lethal Disease




                                                  AA PROTEIN +                                   REDUCTION IN
                        SERUM AMYLOID A           GLYCOSAMINOGLYCANS   KIACTA blocks             FIBRIL FORMATION
                        PRECURSOR (SAA)           (GAGs)            AA + GAGs interaction        & DEPOSITION
                        PROTEIN

CHRONIC
INFLAMMATION
                      Generates           Converts to
                   cytokine cascade       AA Protein
                  (TNFα / IL-1 / IL-6)                               Systemic Amyloid A Fibril
               and increases SAA levels                               Formation & Deposition

                                                                                                 ORGAN DAMAGE, IN
Rheumatic Conditions                                                                             PARTICULAR TO
Inflammatory Bowel Disease                                                                       KIDNEYS
Chronic Infections                                                                               -Rapid deterioration of
Familial Mediterranean Fever                                                                     kidney function leading to
                                                                                                 dialysis




                                                                                                                         6
KIACTA™ - Targeted Opportunity

                                     Patient population estimated at
                                     34-50,000 in the U.S., EU5 and
                                     Japan1
Diagnosed AA Patients (000s)




                                     Clear pharmacoeconomic
                                     rationale for premium pricing
                                     KIACTA™ peak annual revenues
                                             ™
                                     projected at $400-600M1
                                     (U.S., EU5, Japan)
                                     Orphan Drug Status in the U.S.
                                     and EU provides 7 and 10 years
                                     market exclusivity upon
                                     commercialization, respectively


                                     1Independent   market assessment by Frankel Group in April 2009   7
KIACTA™ - Strategic Partnership


PARTNERSHIP                        FINANCIAL IMPLICATION
 With global fund Celtic            US$10M in upfront payments
 Therapeutics
                                    ≥ US$50M in investments by
 Celtic Therapeutics funding        Celtic Therapeutics
 100% of KIACTA™’s
 confirmatory phase III clinical    Proceeds of any eventual
 trial                              transaction expected to be
                                    shared 50%-50% between
 Auction process for the            BELLUS Health and Celtic
 commercialization rights of        Therapeutics
 KIACTA™ on completion of
 Phase III Confirmatory Study




                                                                 8
KIACTA™ - Robust Clinical Results in Phase II/III
                                                                        Statistical significant on
                                                                        primary endpoint
                                                                        (p value <0.05) and clinically
                                                                        meaningful treatment effect
                                                                        (42% reduction in risk)
                                                                          Calculated 2-year delay to
                                                                          dialysis for patients on
                                                                          KIACTATM
                      Number of
                                              Relative Risk
    N=183              Events                                           Clean safety profile
                    Kiacta   Placebo   HR         95% C.I.    P value
                                                                        Agreement with FDA/EMEA
Primary
composite                                                               for confirmatory phase III
                     29        45      0.58      0.37, 0.93   0.025
endpoint
(First “worse”
                                                                        clinical trial
event)
                                                                          Marketing approval based on
Doubling SCr          9        17      0.41      0.19, 0.86   0.019
                                                                          positive result (p value <0.05)
50% decrease CrCl    19        31      0.48      0.28, 0.82   0.008       from confirmatory study with
Dialysis/ESRD         7        13      0.54      0.22, 1.37    0.20       same scope of first phase III
Death                 5        5       0.95      0.27, 3.29    0.94       clinical trial

                                                                                                            9
KIACTA™ - Confirmatory Phase III Study
                                              PHASE III
COMPLETED PHASE II/III STUDY                  CONFIRMATORY STUDY
  183 patients in 13 countries                 230 patients in 28 countries

  Statistically significant composite          Composite primary endpoint
  primary endpoint (p=0.025)                   (target p<0.05) based on patients
  principally based on patients                reaching kidney function events:
  reaching kidney function events:
                                                    80% increase serum creatinine
       Doubling serum creatinine
                                                    40% decrease in creatinine
       50% decrease in creatinine                   clearance
       clearance
                                                    Reaching ESRD/dialysis
       Reaching ESRD/dialysis

       Death                                   Event driven trial to conclude on
                                               attainment of 120 events (~90%
  Fixed treatment duration of two              power)
  years

                                Key improvements made to
                                  increase robustness of
                                    confirmatory study
                                                                                    10
KIACTA™ - Study Progress


   Recruitment1                              Completion
         >70 sites in >25                       Event driven trial to
         countries actively                     complete on reaching
         recruiting                             120 events
         ~120 patients enrolled                 Study expected to be
                                                completed in 2017
         Recruitment expected
         to be completed in 1H
         2014


              Patient baseline characteristics and demographics to date are similar
              to those in the first Phase III study
  1   Data as of November 2012                                                        11
KIACTA™ - Exit Strategy
                 RARE DISEASE DRUGS IN THE NEWS


   Sarepta shares rocket up on
   stellar muscular dystrophy trial
   results (October 2012)     ALEXION TO AQUIRE ENOBIA PHARMA
              Alnylam Gets $22.5M FOR UP TO $1.08 BILLION (December
              From Genzyme for 2011)
              Asia Rights to            Novartis signs $665m option deal with
              Amyloidosis Drug          Selexys for sickle cell disease drug
              (October 2012)            (September 2011)
    BioMarin shares pop on strong pivotal data
    for rare disease drug (November 2012)



       • Auction process at end of study to realize full value
       • Partial exit also possible (commercial partnership) before
         Phase III data
                                                                                12
KIACTA™ - Providing Base Value
                                    AUCTION
                  DEVELOPMENT
  LOW RISK                       PROCESS WITH
                   COST FULLY
CONFIRMATORY                        EQUALLY
                   FUNDED BY
PHASE III STUDY                     SHARED
                     CELTIC
                                   PROCEEDS



                       +
         SIGNIFICANT SHAREHOLDER
                 VALUE BASE

                                                13
VIVIMIND™

                  Nutraceutical for memory protection

                     Canada: Protects the hippocampus
Health Claims
                     Italy: Enhances cognitive function and memory


                     Regulatory approval in Italy obtained in 2009
 Regulatory
                     NPN number issued by Health Canada in 2010
  Approval

                     Partnerships for Italy, Canada, Greece, Middle East,
                     Taiwan and Israel
Partnerships         Pursuing efforts to conclude additional partnerships
                     in other territories: creating a distributor network
                     worldwide

                Growing cash flow positive business
                                                                        14
BLU8499
              Next generation of tramiprosate intended
              for the treatment of Alzheimer's disease


                   Large and growing epidemic currently affecting
  Market           over 30M patients worldwide
opportunity        Represents > $180B in annual costs in the United
                   States alone


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




                                                                      15
BLU8499 – Asclepios Partnership

                        Partnership with Asclepios Bioresearch in
                        September 2012 to finance development of
                        BLU8499
 Partnership
                        Investment of ~$4M in non-dilutive capital
                        Parties expected to share any future proceeds
                        approximately equally

                        Long term toxicity testing expected to be
                        completed in 2013
Development             Phase IIa proof of concept study in mild apoE4+
   Plan                 Alzheimer’s disease patients
                            Expected to begin at end of 2013


               Focused development plan to demonstrate
               effectiveness in targeted patient population

                                                                          16
Financial Position and Capital Structure

              Capital Structure
             Basic Shares Outstanding             47M

             Fully Diluted Shares Outstanding1    61M

              Financial Position
             Cash (as of September 30th, 2012)    >$19M

             Burn Rate (monthly)                  <$300K


   Strategic financing completed with Pharmascience in May 2012
       $17.25M total investment: $8.15M in non-dilutive capital and $9.1M
       for 10.4% stake
   Operations funded into 2018


                                                   1Does   not include stock option grants 17
Governance and Shareholders
Board of Directors      Company / Experience     Management            Title

Dr. Francesco Bellini                                                  President and Chief
                                                 Roberto Bellini
(Chair)                                                                Executive Officer
                                                                       Senior Vice President, Drug
                                                 Dr. Denis Garceau
Franklin Berger                                                        Development

                                                 François Desjardins   Vice President, Finance
Charles Cavell
                                                                       Vice President, Business
                                                 Tony Matzouranis
                                                                       Development
Hélène Fortin            LAROSE FORTIN CA Inc.


Pierre Larochelle
                                                 Shareholder                     Ownership

Donald Olds                                      Bellini Family                   ≈ 30%

Joseph Rus                                       Power Corporation                ≈ 30%

Dr. Martin Tolar                                 Pharmascience                    ≈ 10%

Roberto Bellini


                                                                                                     18
Milestones

                                       Milestones
 Past Execution                                                 Long Term Value
                                 Completion of recruitment of
  Attractive                     KIACTA™ Phase III
  partnership with               Confirmatory Study             Results of Phase III
  Celtic for Kiacta                                             Confirmatory Study
                                 Additional KIACTA™             and auction of
  Execution of global            activities:                    KIACTA™
  Kiacta Phase III
  Confirmatory Study                 Launch of open label       Sale or spin-out of
                                     extension study            VIVIMIND business
  Cashflow positive
  VIVIMIND business                  Updated market and         BLU8499 Phase IIa
                                     pricing assessment         study results
  Partnership for
  BLU8499                        Submission of BLU8499
                                 Phase IIa package
  Strong balance
  sheet                          VIVIMIND partnerships




                      Short-term milestones driving long-term value
                                                                                       19
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Corporate Presentation

  • 1. Corporate Presentation Roberto Bellini President and Chief Executive Officer November 2012
  • 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 immediately in current markets, 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 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 and quality-controlled verification of data and analyses has been completed. 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 BELLUS Health Group and its business 2
  • 3. Background and Business Model Public company (TSX: BLU) based in Montreal, QC Focused namely on the development of products in amyloid- related fields, principally AA Amyloidosis, an orphan indication affecting the kidneys Pipeline also includes product candidate for the treatment of Alzheimer’s disease BUSINESS MODEL Focused on building value for clinical stage health products in critical unmet medical needs 3
  • 4. Pipeline of Products PHARMACEUTICALS DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III NDA/MAA KIACTA™ AA amyloidosis BLU8499 Alzheimer’s disease COMMERCIA NUTRACEUTICAL DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III -LIZATION VIVIMIND™ Memory protection 4
  • 5. KIACTA™ For AA Amyloidosis, an orphan indication and a deadly disease with no treatment Orphan population of ≈50,000 in the USA, Europe Market and Japan with peak annual revenues projected at opportunity $400-600M1 1 Independent market assessment by Frankel Group in April 2009. Phase II/III clinical trial showing statistically Clinical significant primary efficacy endpoints evidence (p value = 0.025) and clean safety profile Partnership with Celtic Therapeutics to conduct Partnership and finance (>$50M) Phase III Confirmatory Study Marketing approval based on confirming safety Phase III and efficacy of phase II/III study Confirmatory Study Actively recruiting patients 5
  • 6. AA Amyloidosis – A Rare and Lethal Disease AA PROTEIN + REDUCTION IN SERUM AMYLOID A GLYCOSAMINOGLYCANS KIACTA blocks FIBRIL FORMATION PRECURSOR (SAA) (GAGs) AA + GAGs interaction & DEPOSITION PROTEIN CHRONIC INFLAMMATION Generates Converts to cytokine cascade AA Protein (TNFα / IL-1 / IL-6) Systemic Amyloid A Fibril and increases SAA levels Formation & Deposition ORGAN DAMAGE, IN Rheumatic Conditions PARTICULAR TO Inflammatory Bowel Disease KIDNEYS Chronic Infections -Rapid deterioration of Familial Mediterranean Fever kidney function leading to dialysis 6
  • 7. KIACTA™ - Targeted Opportunity Patient population estimated at 34-50,000 in the U.S., EU5 and Japan1 Diagnosed AA Patients (000s) Clear pharmacoeconomic rationale for premium pricing KIACTA™ peak annual revenues ™ projected at $400-600M1 (U.S., EU5, Japan) Orphan Drug Status in the U.S. and EU provides 7 and 10 years market exclusivity upon commercialization, respectively 1Independent market assessment by Frankel Group in April 2009 7
  • 8. KIACTA™ - Strategic Partnership PARTNERSHIP FINANCIAL IMPLICATION With global fund Celtic US$10M in upfront payments Therapeutics ≥ US$50M in investments by Celtic Therapeutics funding Celtic Therapeutics 100% of KIACTA™’s confirmatory phase III clinical Proceeds of any eventual trial transaction expected to be shared 50%-50% between Auction process for the BELLUS Health and Celtic commercialization rights of Therapeutics KIACTA™ on completion of Phase III Confirmatory Study 8
  • 9. KIACTA™ - Robust Clinical Results in Phase II/III Statistical significant on primary endpoint (p value <0.05) and clinically meaningful treatment effect (42% reduction in risk) Calculated 2-year delay to dialysis for patients on KIACTATM Number of Relative Risk N=183 Events Clean safety profile Kiacta Placebo HR 95% C.I. P value Agreement with FDA/EMEA Primary composite for confirmatory phase III 29 45 0.58 0.37, 0.93 0.025 endpoint (First “worse” clinical trial event) Marketing approval based on Doubling SCr 9 17 0.41 0.19, 0.86 0.019 positive result (p value <0.05) 50% decrease CrCl 19 31 0.48 0.28, 0.82 0.008 from confirmatory study with Dialysis/ESRD 7 13 0.54 0.22, 1.37 0.20 same scope of first phase III Death 5 5 0.95 0.27, 3.29 0.94 clinical trial 9
  • 10. KIACTA™ - Confirmatory Phase III Study PHASE III COMPLETED PHASE II/III STUDY CONFIRMATORY STUDY 183 patients in 13 countries 230 patients in 28 countries Statistically significant composite Composite primary endpoint primary endpoint (p=0.025) (target p<0.05) based on patients principally based on patients reaching kidney function events: reaching kidney function events: 80% increase serum creatinine Doubling serum creatinine 40% decrease in creatinine 50% decrease in creatinine clearance clearance Reaching ESRD/dialysis Reaching ESRD/dialysis Death Event driven trial to conclude on attainment of 120 events (~90% Fixed treatment duration of two power) years Key improvements made to increase robustness of confirmatory study 10
  • 11. KIACTA™ - Study Progress Recruitment1 Completion >70 sites in >25 Event driven trial to countries actively complete on reaching recruiting 120 events ~120 patients enrolled Study expected to be completed in 2017 Recruitment expected to be completed in 1H 2014 Patient baseline characteristics and demographics to date are similar to those in the first Phase III study 1 Data as of November 2012 11
  • 12. KIACTA™ - Exit Strategy RARE DISEASE DRUGS IN THE NEWS Sarepta shares rocket up on stellar muscular dystrophy trial results (October 2012) ALEXION TO AQUIRE ENOBIA PHARMA Alnylam Gets $22.5M FOR UP TO $1.08 BILLION (December From Genzyme for 2011) Asia Rights to Novartis signs $665m option deal with Amyloidosis Drug Selexys for sickle cell disease drug (October 2012) (September 2011) BioMarin shares pop on strong pivotal data for rare disease drug (November 2012) • Auction process at end of study to realize full value • Partial exit also possible (commercial partnership) before Phase III data 12
  • 13. KIACTA™ - Providing Base Value AUCTION DEVELOPMENT LOW RISK PROCESS WITH COST FULLY CONFIRMATORY EQUALLY FUNDED BY PHASE III STUDY SHARED CELTIC PROCEEDS + SIGNIFICANT SHAREHOLDER VALUE BASE 13
  • 14. VIVIMIND™ Nutraceutical for memory protection Canada: Protects the hippocampus Health Claims Italy: Enhances cognitive function and memory Regulatory approval in Italy obtained in 2009 Regulatory NPN number issued by Health Canada in 2010 Approval Partnerships for Italy, Canada, Greece, Middle East, Taiwan and Israel Partnerships Pursuing efforts to conclude additional partnerships in other territories: creating a distributor network worldwide Growing cash flow positive business 14
  • 15. BLU8499 Next generation of tramiprosate intended for the treatment of Alzheimer's disease Large and growing epidemic currently affecting Market over 30M patients worldwide opportunity Represents > $180B in annual costs in the United States alone Evidence of effectiveness of parent compound Clinical tramiprosate in ApoE4+ Alzheimer’s patients Evidence Safe and well tolerated in Phase I 15
  • 16. BLU8499 – Asclepios Partnership Partnership with Asclepios Bioresearch in September 2012 to finance development of BLU8499 Partnership Investment of ~$4M in non-dilutive capital Parties expected to share any future proceeds approximately equally Long term toxicity testing expected to be completed in 2013 Development Phase IIa proof of concept study in mild apoE4+ Plan Alzheimer’s disease patients Expected to begin at end of 2013 Focused development plan to demonstrate effectiveness in targeted patient population 16
  • 17. Financial Position and Capital Structure Capital Structure Basic Shares Outstanding 47M Fully Diluted Shares Outstanding1 61M Financial Position Cash (as of September 30th, 2012) >$19M Burn Rate (monthly) <$300K Strategic financing completed with Pharmascience in May 2012 $17.25M total investment: $8.15M in non-dilutive capital and $9.1M for 10.4% stake Operations funded into 2018 1Does not include stock option grants 17
  • 18. Governance and Shareholders Board of Directors Company / Experience Management Title Dr. Francesco Bellini President and Chief Roberto Bellini (Chair) Executive Officer Senior Vice President, Drug Dr. Denis Garceau Franklin Berger Development François Desjardins Vice President, Finance Charles Cavell Vice President, Business Tony Matzouranis Development Hélène Fortin LAROSE FORTIN CA Inc. Pierre Larochelle Shareholder Ownership Donald Olds Bellini Family ≈ 30% Joseph Rus Power Corporation ≈ 30% Dr. Martin Tolar Pharmascience ≈ 10% Roberto Bellini 18
  • 19. Milestones Milestones Past Execution Long Term Value Completion of recruitment of Attractive KIACTA™ Phase III partnership with Confirmatory Study Results of Phase III Celtic for Kiacta Confirmatory Study Additional KIACTA™ and auction of Execution of global activities: KIACTA™ Kiacta Phase III Confirmatory Study Launch of open label Sale or spin-out of extension study VIVIMIND business Cashflow positive VIVIMIND business Updated market and BLU8499 Phase IIa pricing assessment study results Partnership for BLU8499 Submission of BLU8499 Phase IIa package Strong balance sheet VIVIMIND partnerships Short-term milestones driving long-term value 19