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Corporate Presentation




Roberto Bellini
President and Chief Executive Officer


August 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

Eprodisate
(KIACTA™)
AA amyloidosis

NRM8499
Alzheimer’s
disease

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

Homotaurine 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 confirmatory phase III clinical trial

                         Marketing approval based on confirming safety
Confirmatory             and efficacy of phase II/III study
   phase III
 clinical trial          Actively recruiting patients
                                                                                             5
AA Amyloidosis
 Rare and life-threatening disease
 Associated with chronic inflammatory diseases
   65% of patients are rheumatoid arthritis patients
 AA protein accumulates in major organs, mainly kidneys, leading to:
   Rapid and significant deterioration of kidney function, and
   Eventual progress to dialysis



 Kiacta slows the deterioration of kidney function by preventing amyloid A
 protein fibril formation, accumulation and deposition
   Orally bioavailable small molecule


                   Na+-03S                     S03-Na+


                                                                             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
                       Events                                           Clean safety profile
                    NC-503   Placebo   HR         95% C.I.    P value

Primary
                                                                        Agreement with FDA/EMEA
composite                                                               for confirmatory phase III
                      29       45      0.58      0.37, 0.93   0.025
                                                                        clinical trial
endpoint
(First “worse”
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
VIVIMIND™

                  Nutraceutical for memory protection

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


                     Regulatory approvals in Italy and Spain obtained
 Regulatory          in 2009
  Approval
                     NPN number issued by Health Canada in 2010

                     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
                                                                            13
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 USA
                          alone

                          Completed in 67 young and elderly healthy
                          subjects
   Phase I                Safe and well tolerated at the intended
clinical trial            therapeutic dose
                          Better gastrointestinal tolerability and
                          pharmacokinetic profile than tramiprosate

                 Company seeking a potential partnership to
                 pursue development process
                                                                           14
Strategic Partnership and Financing


Pharmascience                         Strategic Investment
  Leading Canadian                       $17.25M total investment:
  manufacturer of generic drugs
                                           $8.15M in non-dilutive capital
  Private company                          $9.1M for 10.4% stake
  Annual sales >$700M                    1 board member
  R&D budget >$30M
  More than 1,300 employees




              Pharmascience transaction closed on May 25th

                                                                             15
Financial Position and Capital Structure

              Capital Structure
              Basic Shares Outstanding               47M

              Fully Diluted Shares Outstanding       61M

              Financial Position
              Cash (as of May 31st, 2012)            >$20M

              Burn Rate (monthly)                    <$300K


   Operations funded beyond 2016
    Kiacta Phase 3 data expected in 2015
    Additional funds can be used for new potential projects



                                                               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
                                 Continued execution of
  Attractive                     KIACTA Confirmatory phase       Results of confirmatory
  partnership with               III clinical trial:             phase III clinical trial
  Celtic for Kiacta                                              and auction of
                                     Launch of Japan sites       KIACTA™
  Execution of global                and receipt of Japanese
  Kiacta Phase III                   orphan drug designation     Sale or spin-out of
  confirmatory study                                             nutraceutical business
                                 Partnership for NRM8499 for
  Building cashflow              Phase 2 study                   NRM8499 Phase 2
  positive VIVIMIND                                              study results
  business                           Financial partner with no
                                     shareholder dilution
  Fully financed
  business plan                  Large regional partnership
                                 for VIVIMIND™




                      Short-term milestones driving long-term value
                                                                                          18
Corporate presentation   august 2012

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Corporate presentation august 2012

  • 1. Corporate Presentation Roberto Bellini President and Chief Executive Officer August 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 Eprodisate (KIACTA™) AA amyloidosis NRM8499 Alzheimer’s disease COMMERCIA NUTRACEUTICAL DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III -LIZATION Homotaurine 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 confirmatory phase III clinical trial Marketing approval based on confirming safety Confirmatory and efficacy of phase II/III study phase III clinical trial Actively recruiting patients 5
  • 6. AA Amyloidosis Rare and life-threatening disease Associated with chronic inflammatory diseases  65% of patients are rheumatoid arthritis patients AA protein accumulates in major organs, mainly kidneys, leading to:  Rapid and significant deterioration of kidney function, and  Eventual progress to dialysis Kiacta slows the deterioration of kidney function by preventing amyloid A protein fibril formation, accumulation and deposition  Orally bioavailable small molecule Na+-03S S03-Na+ 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 Events Clean safety profile NC-503 Placebo HR 95% C.I. P value Primary Agreement with FDA/EMEA composite for confirmatory phase III 29 45 0.58 0.37, 0.93 0.025 clinical trial endpoint (First “worse” 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. VIVIMIND™ Nutraceutical for memory protection Canada: Protects the hippocampus Health Claims Italy: Enhances cognitive function and memory Regulatory approvals in Italy and Spain obtained Regulatory in 2009 Approval NPN number issued by Health Canada in 2010 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 13
  • 14. 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 USA alone Completed in 67 young and elderly healthy subjects Phase I Safe and well tolerated at the intended clinical trial therapeutic dose Better gastrointestinal tolerability and pharmacokinetic profile than tramiprosate Company seeking a potential partnership to pursue development process 14
  • 15. Strategic Partnership and Financing Pharmascience Strategic Investment Leading Canadian $17.25M total investment: manufacturer of generic drugs  $8.15M in non-dilutive capital Private company  $9.1M for 10.4% stake Annual sales >$700M 1 board member R&D budget >$30M More than 1,300 employees Pharmascience transaction closed on May 25th 15
  • 16. Financial Position and Capital Structure Capital Structure Basic Shares Outstanding 47M Fully Diluted Shares Outstanding 61M Financial Position Cash (as of May 31st, 2012) >$20M Burn Rate (monthly) <$300K Operations funded beyond 2016  Kiacta Phase 3 data expected in 2015  Additional funds can be used for new potential projects 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 Continued execution of Attractive KIACTA Confirmatory phase Results of confirmatory partnership with III clinical trial: phase III clinical trial Celtic for Kiacta and auction of Launch of Japan sites KIACTA™ Execution of global and receipt of Japanese Kiacta Phase III orphan drug designation Sale or spin-out of confirmatory study nutraceutical business Partnership for NRM8499 for Building cashflow Phase 2 study NRM8499 Phase 2 positive VIVIMIND study results business Financial partner with no shareholder dilution Fully financed business plan Large regional partnership for VIVIMIND™ Short-term milestones driving long-term value 18