Roberto Bellini presented an overview of BELLUS Health Inc. Key points included:
- BELLUS is focused on developing products for amyloid-related diseases including its lead product KIACTA for AA amyloidosis currently in Phase III trials.
- KIACTA has shown statistically significant results in reducing kidney function decline in Phase II/III trials. A partnership with Auven Therapeutics is funding the Phase III confirmatory trial.
- BELLUS is acquiring Thallion which adds the antibody Shigamabs targeting sHUS to its pipeline. Shigamabs has completed Phase II trials and the strategy is to reposition it for treatment of sHUS.
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risk to help physicians proactively manage patient health. The
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‘GeneType for Colorectal Cancer’, are clinically validated risk
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development pipeline includes new tests for COVID-19, Type 2
diabetes, cardiovascular disease, prostate cancer, and melanoma.
Listed on the ASX in 2000 and NASDAQ in 2005, Genetic
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commercialization of genetic risk assessment technology for 20 years.
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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 on products in amyloid-
related fields, principally AA
Amyloidosis, an orphan
indication affecting the kidneys
Late-stage product pipeline with
fully funded business plan
3
Business Model
Focused on building value for clinical stage health
products in critical unmet medical needs
Financial Facts
Shares outstanding (Fully
Diluted): 65M
Cash (03/31/13): ~$18M
Burn rate (monthly): <$300K
5. KIACTA™ Overview
5
For Amyloid A (AA) Amyloidosis, a rare and
deadly disease with no specific treatment
Market
opportunity
Clinical
evidence
1 Market assessment by Frankel Group in April 2009.
Partnership
Orphan population of ≈50,000 in the USA, Europe
and Japan with peak annual revenues projected at
$400-600M1
Phase II/III clinical trial showing statistically
significant primary efficacy endpoints
(p value = 0.025) and clean safety profile
Partnership with Auven Therapeutics (previously
known as Celtic Therapeutics) to conduct and
finance (>$50M) Phase III Confirmatory Study
Phase III
Confirmatory
Study
Marketing approval based on confirming safety
and efficacy of phase II/III study
>66% patients recruited
6. 6
Mechanism of Action
CHRONIC
INFLAMMATION
SERUM AMYLOID A
PRECURSOR (SAA)
PROTEIN
AA PROTEIN +
GLYCOSAMINOGLYCANS
(GAGs)
ORGAN DAMAGE, IN
PARTICULAR TO
KIDNEYS
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
7. KIACTA™ - Targeted Opportunity
7
DiagnosedAAPatients(000s)
Patient population estimated at
34,000-50,000 in the United States,
Europe Top 5 and Japan1
KIACTA™ peak annual revenues
projected at $400-600M1
(U.S., EU5, Japan)
Strong potential for premium
pricing
Clear pharmacoeconomic
rationale due to high cost of
kidney disease
Comparative orphan drug pricing
1Market assessment by Frankel Group in April 2009
8. FINANCIAL IMPLICATION
With global fund Auven
Therapeutics
Auven Therapeutics funding
100% of KIACTA™’s
Confirmatory Phase III
clinical trial
Auction process for the
commercialization rights of
KIACTA™ on completion of
Phase III Confirmatory Study
PARTNERSHIP
US$10M in upfront payments
≥ US$50M in investments by
Auven Therapeutics
Proceeds of any eventual
transaction expected to be
shared 50%-50% between
BELLUS Health and Auven
Therapeutics
Strategic Partnership
Partnership to fund Phase III Confirmatory Study with significant
upside for BELLUS shareholders 8
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
event
• Most sensitive component
of composite endpoint highly
significant (CrCl, p < 0.01)
Composite
Endpoint (Time
to First Worse
Event)
Doubling
Serum
Creatine
50%
Decrease
Creatine
CIearance
Dialysis/
ESRD
Death
NumberofPatientEvents
9
*
*
*
KIACTA™ - Robust Clinical Results in Phase II/III
10. KIACTA™ - Phase II/III Key Secondary Endpoints
HR=0.58
Wald Chi Square test: p=0.025
KIACTATM
Placebo
10
Key secondary endpoints show robust effect of KIACTA™
Using slope of creatinine clearance decline, calculated delay
to time of dialysis is ~2 years on Kiacta versus placebo
Kaplan Meier: Time to First Worst Event Slope of Creatinine Clearance Decline
Placebo
Months of Treatment
Mean∆CrCl(mL/min/1.73m2)
= 9.4mL/min/
1.73 m2
after 24 months
p=0.025
-10.9 ± 5.1 mL/min/
1.73 m2/year
-15.6 ± 4.0 mL/min/
1.73 m2/year
KIACTATM
11. KIACTA™ - Phase II/III Feedback
2007 NEJM article published by lead investigators concludes that
KIACTA™ slows decline of renal function in AA Amyloidosis
Agreement reached under Special Protocol Assessment with FDA and
Scientific Advice with EMEA to conduct Phase III Confirmatory Study
Marketing approval based on positive result (p value <0.05) from confirmatory study with
same scope of first phase II/III clinical trial
11
12. PHASE III CONFIRMATORY STUDY
183 patients in 13 countries
Composite primary endpoint
based on patients reaching
kidney function worsening
events:
Doubling serum creatinine
50% decrease in creatinine clearance
Reaching ESRD/dialysis
Death
Fixed treatment duration of 2
years
PHASE II/III STUDY
230 patients in >25 countries
Composite primary endpoint (target
p<0.05) based on patients reaching
kidney function worsening events:
Persistent 80% increase serum creatinine
Persistent 40% decrease in creatinine
clearance
Reaching ESRD/dialysis
Event driven trial to conclude on
attainment of 120 events (~90%
power)
KIACTA™ - Phase III Confirmatory Study
Key improvements made to increase robustness of Phase III
Confirmatory Study 12
13. Study Progress
STUDY UPDATE (May 2013)
>65 sites in >25 countries
activated
>150 patients enrolled
Recruitment expected to be
completed in 1H 2014
COMPLETION
Event driven trial to
complete on reaching 120
events
Study expected to be
completed in 2017
Patient baseline characteristics and demographics
to date are similar to those in the first Phase III study 13
14. Thallion Transaction - Overview
Bellus to acquire Thallion for $6.3M in cash plus certain contingent value rights (CVRs):
$6.3M in cash consideration1 ($0.1904 per share) based on $7.5M net cash at closing;
100% of expected $1.4M 2016 payment (up to $0.041 per share)
5% of Shigamabs product revenue, capped at $6.5M (up to $0.181 per share)
Binding agreement and announcement on June 17th
Unanimous support from Thallion and Bellus Board of Directors
Voting support agreements in place currently for more than 24% of votes, including 14.5% from largest
shareholder Jaguar Financial
Transaction to proceed by court supervised plan of arrangement requiring 66 2/3%
shareholder approval
Thallion shareholder meeting planned for August 6th
Close of transaction expected shortly after shareholder meeting
1 Cash consideration to be adjusted (up and down) on a
dollar for dollar based on net cash 5 days before closing
Acquisition rationale based on building pipeline with addition of
Shigamabs, a complementary Phase 2 asset 14
15. Thallion Transaction – Benefits
Benefits to Bellus:
Adds Shigamabs to pipeline, a complementary Phase II asset targeting a rare disease
affecting the kidneys
Net cash of ~$1.2M from transaction provides funding for Shigamabs development
Benefits to Thallion:
Provides initial cash consideration at significant premium to stock price, 46% premium
from announcement date and 90% premium from initiation of discussions
Provides additional value in CVR with potential to achieve aggregate ~$0.41 per share,
215% premium from announcement date and 310% premium from initiation of
discussions
Transaction is executionally low risk and also tax efficient for Thallion shareholders
Mutually beneficial transaction for Thallion and Bellus
shareholders 15
16. Shigamabs Overview
16
For STEC related Hemolytic Uremic Syndrome
(sHUS), an ultra rare disease primarily affecting
the kidneys
Market
opportunity
Clinical
evidence
Mechanism
Shigatoxin producing e.coli (STEC) is a rare food
borne illness
1,500-2,000 annual cases progress to sHUS in
developed countries, principally in children
Premium pricing potential
Safety in target pediatric population from 45 patient
Phase 2 study
Monoclonal antibody binds and neutralizes Stx-2,
the most prominent and virulent variant of STEC
17. sHUS
(5%-10%)
Resolve
(90%-95%)
Ingestion Colonization Toxin Secretion
-4 0 2 4 6 8-2
Day
Thallion intervention
(prevention)
17
Shigamabs Development Strategy
Development thesis to reposition Shigamabs from prevention of sHUS to treatment of sHUS
Support from key opinion leader community for downstream therapeutic approach to sHUS
Recent publication supports role of toxin neutralization for treatment of sHUS
Conduct proof of concept studies in several HUS animal models to validate approach
Clear clinical and regulatory path into Phase II proof of concept study
Only treating patients with high unmet medical need
Clear endpoint based on reduction in need for dialysis (40-50% of sHUS patients require dialysis)
Dialysis is considered clinically relevant by regulatory agencies
Bellus
intervention
STEC and sHUS Disease Course
18. VIVIMIND™
Nutraceutical for memory protection
Regulatory
Partnerships
Growing cash flow positive business
Partnerships for Italy (launched), Canada
(launched), South Korea (regulatory), Greece (pre-
launch), Middle East (pre-launch), Taiwan
(regulatory) and Israel (regulatory)
Pursuing creation of worldwide distributor network
Health claims include:
‘Protects the
hippocampus’ and
‘Enhances cognitive
function and memory’
VIVIMIND Revenues ($K)
0
100
200
300
400
500
2010 2011 2012
18
19. BLU8499
Next generation of tramiprosate intended
for the treatment of Alzheimer's disease
Market
opportunity
Clinical
evidence
Partnership
Large and growing epidemic currently affecting over
30M patients worldwide
Evidence of effectiveness of parent compound
tramiprosate in ApoE4+ Alzheimer’s patients
Safe and well tolerated in Phase I
Partnership with Asclepios Bioresearch in
September 2012 to finance development of
BLU8499 into Phase 2a
Commitment of ~$4M in non-dilutive capital
19
20. Capital Structure
Basic Shares Outstanding 47M
Fully Diluted Shares Outstanding 65M
Operations funded into mid-2018
20
Financial Position and Capital Structure
Financial Position
Cash (March 31st, 2013) ~$18M
Burn Rate (monthly) <$300K
21. Shareholder Ownership
Bellini Family ≈ 30%
Power Corporation ≈ 30%
Pharmascience ≈ 10%
Governance and Shareholders
21
Board of Directors Company / Experience
Dr. Francesco Bellini
(Chair)
Franklin Berger
Charles Cavell
Hélène Fortin
Pierre Larochelle
Donald Olds
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.
22. Short-term milestones driving long-term value
Milestones
22
Past Execution
Attractive
partnership with for
Kiacta
Execution of global
KIACTA Phase III
Confirmatory Study
Cashflow positive
VIVIMIND business
Partnership for
BLU8499
Strong balance
sheet and clean
capital structure
Milestones (12 months)
Completion of recruitment of
KIACTA™ Phase III
Confirmatory Study
Additional KIACTA™
activities:
Launch of open label
extension study
Market and pricing
assessment
Japan orphan drug
designation
BLU 8499 Pre-Phase II
package completion
VIVIMIND partnerships
Long Term Value
Results of Phase III
Confirmatory Study
and auction of
KIACTA™
Sale or spin-out of
VIVIMIND business
BLU8499 Phase IIa
study results