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Bloom Burton Healthcare
Investor Conference
Roberto Bellini
President and Chief Executive Officer
Twitter: @rbellini
May 2, 2016
r
Forward Looking Statements
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 industry,
changes in the regulatory environment in the jurisdictions in which the BELLUS Health Inc.
does business, stock market volatility, fluctuations in costs, changes to the competitive
environment due to consolidation, achievement of forecasted burn rate, potential payments in
relation to indemnity agreements, 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. In addition, the length of the KIACTA™ Phase III Confirmatory Study is
dependent upon many factors, including clinical sites activation, patient enrollment rate, patient
drop-out rate and occurrence of clinical endpoint events, and the sharing of proceeds between
Auven Therapeutics and BELLUS Health Inc. from potential future revenue of KIACTA™ is
dependent upon a number of factors, including the quantum of proceeds.
Consequently, actual future results 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 presentation. 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 BELLUS Health Inc.’s public fillings
including the Annual Information Form for further risk factors that might affect BELLUS Health
Inc. and its business.
2
3
At BELLUS, we are focused on developing drugs for rare
diseases starting with conditions that affect the kidneys.
Value driving rare disease pipeline fully funded through key
milestones
Company Highlights
4
Late-stage pipeline with 4 projects targeting rare diseases
Lead drug candidate, KIACTA, in Phase III Confirmatory Study for
AA amyloidosis
 Rare and deadly kidney disease with no treatment
 Phase II/III study completed with positive efficacy and clean safety
 Similar and confirmatory Phase III study completed (Data expected in Q2
2016)
 Potential peak market sales of $600M-$1B
 Potential exit to commercial partner following Phase III data
Business plan fully funded through KIACTA Phase III and exit
process
Late stage pipeline focused on developing innovative drugs for
rare diseases
Pipeline of Products
Shigamab
sHUS
DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III
KIACTA™
AA amyloidosis
MARKET
AL amyloidosis
KIACTA™
Sarcoidosis
5
Lead Phase III Product Candidate
6
A rare and deadly
kidney disease with
no specific treatment
FOR AMYLOID A (AA)
AMYLOIDOSIS
Disease and Mechanism of Action
7
CHRONIC
INFLAMMATION
SERUM AMYLOID A
PRECURSOR (SAA)
PROTEIN
AA PROTEIN +
GLYCOSAMINOGLYCANS
(GAGs)
ORGAN DAMAGE, IN
PARTICULAR TO
KIDNEYS LEADING TO
DIALYSIS
REDUCTION IN
FIBRIL FORMATION
& DEPOSITION
Converts to
AA Protein
Generates
cytokine cascade
(TNFα / IL-1 / IL-6)
and increases SAA levels
Rheumatic Conditions
Inflammatory Bowel Disease
Chronic Infections
Familial Mediterranean Fever
KIACTA™ blocks
AA + GAGs interaction
Systemic Amyloid A Fibril
Formation & Deposition
KIACTA designed to bind AA amyloid, slow down disease
progression and delay dialysis 7
KIACTA™ – Addressable Market
Source: Navigant Consulting 2014 8
Estimated Peak Annual Sales
$600 Million-$1Billion
KIACTA Eligible Patients
10-15 Thousand
Expected Pricing Per Patient Per Year
$200-$275 Thousand
Experienced and knowledgeable partner working on lead project
Auven is a global biotech private
equity group
Partnered on KIACTA project in
2010
Funding 100% of KIACTA™ project
including studies in AA Amyloidosis
and Sarcoidosis
≥ US$70M in investments
Overall proceeds of exit expected
to be shared 50-50
KIACTA™ to be sold/partnered to
commercial entity after Phase III
Confirmatory Study results
Auven Therapeutics Partnership for KIACTA™
BUSINESS PLANAUVEN PARTNERSHIP
9
HR 0.58 0.41 0.48 0.54 0.95
95% C.I 0.37, 0.93 0.19,0.86 0.28,0.82 0.22,1.37 0.27,3.29
P value 0.025 0.019 0.008 0.20 0.94
Graphical representation
of the information in this
table
Landmark study in AA
Amyloidosis: 183 patients
treated for 2 years
 Composite endpoint based on
patients reaching events of
decreasing kidney function or
death
Statistically significant
primary endpoint (Cox
Proportional Hazard Ratio;
p=0.025)
Clinically meaningful
treatment effect with 42%
reduction in risk of reaching
worsening kidney event
Clear regulatory path to
approval based on
confirmatory study
Composite
Endpoint (Time to
First Worse
Event)
Doubling
Serum
Creatinine
50%
Decrease
Creatinine
Clearance
Dialysis/
ESRD
Death
NumberofPatientEvents
10
*
*
**
KIACTA™ - Robust Clinical Results in Phase II/III
*p<0.05
**p<0.01
PHASE III CONFIRMATORY STUDY
Key entry criteria based on
kidney function:
 High proteinuria (>1 g/d) or low
creatinine clearance (< 60
ml/min/1.73m2)
183 patients in 13 countries
Fixed treatment duration of 2
years
 74 kidney function worsening events
Composite primary endpoint
based on patients reaching
kidney function worsening
events
 Target statistical significance of
p=0.01
PHASE II/III STUDY
Enriched patient population
 High proteinuria (>1 g/d)
More patients
 261 patients in >25 countries
Increased power
 Event driven trial to conclude on reaching
120 events
Lower statistical bar to achieve
success
 Primary endpoint with target statistical
significance of p=0.05
KIACTA™ – Phase III Confirmatory Study
11
Key improvements made to increase chance of successful study
11
Study enrolled with 261 patients
Study completed with 120 events
reached (January 2016)
Topline data expected in Q2 2016
Phase III Confirmatory Study
12
Clean capital structure and cash runway through potential exit
Corporate
13
Capital Markets (as of April 28th, 2016)
Ticker TSX: BLU
Shares (Basic) 54.7M
Shares (Fully Diluted) 65.7M
Daily Volume ~100K
Market Capitalization (FD) ~C$110M
13
Finance
Cash (December 31, 2015) C$9.7M
Burn rate (monthly) <C$300K
Shareholder Ownership (FD)
Bellini Family ≈ 29%
Power Corporation ≈ 27%
Pharmascience ≈ 10%
Governance and Shareholders
14
Board of Directors Company / Experience
Dr. Francesco Bellini
(Chair)
Franklin Berger
Charles Cavell
Hélène Fortin
Pierre Larochelle
Muriel Lortie
Joseph Rus
Dr. Martin Tolar
Roberto Bellini
Management Title
Roberto Bellini
President and Chief Executive
Officer
Dr. Denis Garceau
Senior Vice President, Drug
Development
François Desjardins Vice President, Finance
Tony Matzouranis
Vice President, Business
Development
LAROSE FORTIN CA Inc.
14
Potential KIACTA™ exit
Continue executing KIACTA™ for AA
Amyloidosis plan:
Reach 120 event target (Q1 2016)
Top Line Data (Q2 2016)
Progress rare disease pipeline projects:
IND filing for KIACTA Phase 2 for
Sarcoidosis (1H 2016)
Shigamab animal data (1H 2016)
Shigamab clinical trial design (1H
2016)
Significant news flow and value inflection point in 2016
Milestones
Past Execution
Attractive partnership
for KIACTA™
Execution of global
KIACTA™ Phase III
Confirmatory Study
Expansion of rare
disease pipeline
Strong balance sheet
and clean capital
structure
Milestones
15
Connect With Us
Follow us on Twitter: @BELLUSHealth
Join our LinkedIn group
Read our blog @ www.bellushealth.com
Join our mailing list

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BBHC Confirms Positive KIACTA Data

  • 1. Bloom Burton Healthcare Investor Conference Roberto Bellini President and Chief Executive Officer Twitter: @rbellini May 2, 2016 r
  • 2. Forward Looking Statements 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 industry, changes in the regulatory environment in the jurisdictions in which the BELLUS Health Inc. does business, stock market volatility, fluctuations in costs, changes to the competitive environment due to consolidation, achievement of forecasted burn rate, potential payments in relation to indemnity agreements, 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. In addition, the length of the KIACTA™ Phase III Confirmatory Study is dependent upon many factors, including clinical sites activation, patient enrollment rate, patient drop-out rate and occurrence of clinical endpoint events, and the sharing of proceeds between Auven Therapeutics and BELLUS Health Inc. from potential future revenue of KIACTA™ is dependent upon a number of factors, including the quantum of proceeds. Consequently, actual future results 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 presentation. 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 BELLUS Health Inc.’s public fillings including the Annual Information Form for further risk factors that might affect BELLUS Health Inc. and its business. 2
  • 3. 3 At BELLUS, we are focused on developing drugs for rare diseases starting with conditions that affect the kidneys.
  • 4. Value driving rare disease pipeline fully funded through key milestones Company Highlights 4 Late-stage pipeline with 4 projects targeting rare diseases Lead drug candidate, KIACTA, in Phase III Confirmatory Study for AA amyloidosis  Rare and deadly kidney disease with no treatment  Phase II/III study completed with positive efficacy and clean safety  Similar and confirmatory Phase III study completed (Data expected in Q2 2016)  Potential peak market sales of $600M-$1B  Potential exit to commercial partner following Phase III data Business plan fully funded through KIACTA Phase III and exit process
  • 5. Late stage pipeline focused on developing innovative drugs for rare diseases Pipeline of Products Shigamab sHUS DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III KIACTA™ AA amyloidosis MARKET AL amyloidosis KIACTA™ Sarcoidosis 5
  • 6. Lead Phase III Product Candidate 6 A rare and deadly kidney disease with no specific treatment FOR AMYLOID A (AA) AMYLOIDOSIS
  • 7. Disease and Mechanism of Action 7 CHRONIC INFLAMMATION SERUM AMYLOID A PRECURSOR (SAA) PROTEIN AA PROTEIN + GLYCOSAMINOGLYCANS (GAGs) ORGAN DAMAGE, IN PARTICULAR TO KIDNEYS LEADING TO DIALYSIS REDUCTION IN FIBRIL FORMATION & DEPOSITION Converts to AA Protein Generates cytokine cascade (TNFα / IL-1 / IL-6) and increases SAA levels Rheumatic Conditions Inflammatory Bowel Disease Chronic Infections Familial Mediterranean Fever KIACTA™ blocks AA + GAGs interaction Systemic Amyloid A Fibril Formation & Deposition KIACTA designed to bind AA amyloid, slow down disease progression and delay dialysis 7
  • 8. KIACTA™ – Addressable Market Source: Navigant Consulting 2014 8 Estimated Peak Annual Sales $600 Million-$1Billion KIACTA Eligible Patients 10-15 Thousand Expected Pricing Per Patient Per Year $200-$275 Thousand
  • 9. Experienced and knowledgeable partner working on lead project Auven is a global biotech private equity group Partnered on KIACTA project in 2010 Funding 100% of KIACTA™ project including studies in AA Amyloidosis and Sarcoidosis ≥ US$70M in investments Overall proceeds of exit expected to be shared 50-50 KIACTA™ to be sold/partnered to commercial entity after Phase III Confirmatory Study results Auven Therapeutics Partnership for KIACTA™ BUSINESS PLANAUVEN PARTNERSHIP 9
  • 10. HR 0.58 0.41 0.48 0.54 0.95 95% C.I 0.37, 0.93 0.19,0.86 0.28,0.82 0.22,1.37 0.27,3.29 P value 0.025 0.019 0.008 0.20 0.94 Graphical representation of the information in this table Landmark study in AA Amyloidosis: 183 patients treated for 2 years  Composite endpoint based on patients reaching events of decreasing kidney function or death Statistically significant primary endpoint (Cox Proportional Hazard Ratio; p=0.025) Clinically meaningful treatment effect with 42% reduction in risk of reaching worsening kidney event Clear regulatory path to approval based on confirmatory study Composite Endpoint (Time to First Worse Event) Doubling Serum Creatinine 50% Decrease Creatinine Clearance Dialysis/ ESRD Death NumberofPatientEvents 10 * * ** KIACTA™ - Robust Clinical Results in Phase II/III *p<0.05 **p<0.01
  • 11. PHASE III CONFIRMATORY STUDY Key entry criteria based on kidney function:  High proteinuria (>1 g/d) or low creatinine clearance (< 60 ml/min/1.73m2) 183 patients in 13 countries Fixed treatment duration of 2 years  74 kidney function worsening events Composite primary endpoint based on patients reaching kidney function worsening events  Target statistical significance of p=0.01 PHASE II/III STUDY Enriched patient population  High proteinuria (>1 g/d) More patients  261 patients in >25 countries Increased power  Event driven trial to conclude on reaching 120 events Lower statistical bar to achieve success  Primary endpoint with target statistical significance of p=0.05 KIACTA™ – Phase III Confirmatory Study 11 Key improvements made to increase chance of successful study 11
  • 12. Study enrolled with 261 patients Study completed with 120 events reached (January 2016) Topline data expected in Q2 2016 Phase III Confirmatory Study 12
  • 13. Clean capital structure and cash runway through potential exit Corporate 13 Capital Markets (as of April 28th, 2016) Ticker TSX: BLU Shares (Basic) 54.7M Shares (Fully Diluted) 65.7M Daily Volume ~100K Market Capitalization (FD) ~C$110M 13 Finance Cash (December 31, 2015) C$9.7M Burn rate (monthly) <C$300K
  • 14. Shareholder Ownership (FD) Bellini Family ≈ 29% Power Corporation ≈ 27% Pharmascience ≈ 10% Governance and Shareholders 14 Board of Directors Company / Experience Dr. Francesco Bellini (Chair) Franklin Berger Charles Cavell Hélène Fortin Pierre Larochelle Muriel Lortie Joseph Rus Dr. Martin Tolar Roberto Bellini Management Title Roberto Bellini President and Chief Executive Officer Dr. Denis Garceau Senior Vice President, Drug Development François Desjardins Vice President, Finance Tony Matzouranis Vice President, Business Development LAROSE FORTIN CA Inc. 14
  • 15. Potential KIACTA™ exit Continue executing KIACTA™ for AA Amyloidosis plan: Reach 120 event target (Q1 2016) Top Line Data (Q2 2016) Progress rare disease pipeline projects: IND filing for KIACTA Phase 2 for Sarcoidosis (1H 2016) Shigamab animal data (1H 2016) Shigamab clinical trial design (1H 2016) Significant news flow and value inflection point in 2016 Milestones Past Execution Attractive partnership for KIACTA™ Execution of global KIACTA™ Phase III Confirmatory Study Expansion of rare disease pipeline Strong balance sheet and clean capital structure Milestones 15
  • 16. Connect With Us Follow us on Twitter: @BELLUSHealth Join our LinkedIn group Read our blog @ www.bellushealth.com Join our mailing list