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Corporate Presentation




Roberto Bellini
President and Chief Executive Officer


September 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
 Late-stage product pipeline




                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 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$35M 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 clinical trial




                                                                 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
endpoint
(First “worse”
                     29        45      0.58      0.37, 0.93   0.025
                                                                        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
                                               CONFIRMATORY PHASE III
COMPLETED PHASE II/III STUDY                   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 2                 power)
  years

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


   Recruitment1                              Completion
         >70 sites in 26                        Event driven trial to
         countries actively                     complete on reaching
         recruiting                             120 events
         90 patients enrolled                   Study expected to be
                                                completed in 2H 2015
         Recruitment expected
         to be completed in 2H
         2013


             Patient baseline characteristics and demographics to date are similar
             to those in the first Phase III study
  1   Data as of May 10, 2012                                                        11
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

                                                12
BLU8499 (previously NRM8499)
               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




                                                                       13
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

                                                                          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
                     and Israel
Partnerships         Pursuing efforts to conclude additional partnerships
                     in other territories: creating a distributor network
                     worldwide

                Growing cash flow positive business
                                                                            15
Financial Position and Capital Structure
               Capital Structure
              Basic Shares Outstanding                  47M

              Fully Diluted Shares Outstanding1         61M

               Financial Position
              Cash (as of June 30th, 2012)              >$21M

              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 beyond 2016
    Kiacta Phase 3 data expected in 2015
    Additional funds can be used for new potential projects
                                                         1Does   not include stock option grants 16
Governance and Shareholders
Board of Directors      Company / Experience     Management            Title

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

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


Pierre Larochelle
                                                 Shareholder                    Ownership

Donald Olds                                      Bellini Family                   ≈ 30%

Joseph Rus                                       Power Corporation                ≈ 30%

Dr. Martin Tolar                                 Pharmascience                    ≈ 10%

Roberto Bellini


                                                                                                  17
Milestones

                                    2012 Milestones
 Past Execution                                                  Long Term Value
                                 Financing
  Attractive                     Continued execution of          Results of confirmatory
  partnership with               KIACTA Confirmatory phase       phase III clinical trial
  Celtic for Kiacta              III clinical trial:             and auction of
                                                                 KIACTA™
  Execution of global                Launch of Japan sites
  Kiacta Phase III                   and receipt of Japanese     Sale or spin-out of
  confirmatory study                 orphan drug designation     nutraceutical business

  Building cashflow              Partnership for BLU8499 for     BLU8499 Phase IIa
  positive VIVIMIND              Phase IIa study                 study results
  business
                                     Financial partner with no
                                     shareholder dilution

                                 Large regional partnership
                                 for VIVIMIND™



                      Short-term milestones driving long-term value
                                                                                          18
Corporate presentation   september 2012 v final v3

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Corporate presentation september 2012 v final v3

  • 1. Corporate Presentation Roberto Bellini President and Chief Executive Officer September 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 Late-stage product pipeline 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 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$35M 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 clinical trial 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 endpoint (First “worse” 29 45 0.58 0.37, 0.93 0.025 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 CONFIRMATORY PHASE III COMPLETED PHASE II/III STUDY 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 2 power) years Key improvements made to increase robustness of confirmatory study 10
  • 11. KIACTA™ - Study Progress Recruitment1 Completion >70 sites in 26 Event driven trial to countries actively complete on reaching recruiting 120 events 90 patients enrolled Study expected to be completed in 2H 2015 Recruitment expected to be completed in 2H 2013 Patient baseline characteristics and demographics to date are similar to those in the first Phase III study 1 Data as of May 10, 2012 11
  • 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 12
  • 13. BLU8499 (previously NRM8499) 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 13
  • 14. 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 14
  • 15. 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 and Israel Partnerships Pursuing efforts to conclude additional partnerships in other territories: creating a distributor network worldwide Growing cash flow positive business 15
  • 16. Financial Position and Capital Structure Capital Structure Basic Shares Outstanding 47M Fully Diluted Shares Outstanding1 61M Financial Position Cash (as of June 30th, 2012) >$21M 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 beyond 2016  Kiacta Phase 3 data expected in 2015  Additional funds can be used for new potential projects 1Does not include stock option grants 16
  • 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, Dr. Denis Garceau Franklin Berger Drug Development François Desjardins Vice President, Finance Charles Cavell Vice President, Business Tony Matzouranis LAROSE FORTIN CA Inc. Development Hélène Fortin Pierre Larochelle Shareholder Ownership Donald Olds Bellini Family ≈ 30% Joseph Rus Power Corporation ≈ 30% Dr. Martin Tolar Pharmascience ≈ 10% Roberto Bellini 17
  • 18. Milestones 2012 Milestones Past Execution Long Term Value Financing Attractive Continued execution of Results of confirmatory partnership with KIACTA Confirmatory phase phase III clinical trial Celtic for Kiacta III clinical trial: and auction of KIACTA™ Execution of global Launch of Japan sites Kiacta Phase III and receipt of Japanese Sale or spin-out of confirmatory study orphan drug designation nutraceutical business Building cashflow Partnership for BLU8499 for BLU8499 Phase IIa positive VIVIMIND Phase IIa study study results business Financial partner with no shareholder dilution Large regional partnership for VIVIMIND™ Short-term milestones driving long-term value 18