Top Banner
Corporate Presentation (TSX: BLU) Roberto Bellini President and Chief Executive Officer Twitter: @rbellini Winter 2014
25
Welcome message from author
This document is posted to help you gain knowledge. Please leave a comment to let me know what you think about it! Share it to your friends and learn new things together.
Transcript
Page 1: Bellus corporate presentation january 2014 v final

Corporate Presentation (TSX: BLU)

Roberto BelliniPresident and Chief Executive OfficerTwitter: @rbellini

Winter 2014

Page 2: Bellus corporate presentation january 2014 v final

2

30 millionpeople in the United States have a RARE disease.

Source: NIH: National Institutes of Health Office of Rare Diseases

Page 3: Bellus corporate presentation january 2014 v final

3

Only about 5%of these people have a specific therapy to treat their disease.

Page 4: Bellus corporate presentation january 2014 v final

4

85-90%of rare diseases are serious or life threatening.

Page 5: Bellus corporate presentation january 2014 v final

Regulatory advantage

Premium pricing

Market protection

Smaller clinical trials

Efficient commercialization strategies

5

Small patient numbers, BIG opportunity

Page 6: Bellus corporate presentation january 2014 v final

6

At BELLUS, we are focused on developing drugs for rare diseases starting with conditions that affect the kidneys.

Page 7: Bellus corporate presentation january 2014 v final

Fruitful 2013 leading into important milestones in 2014

Executing on Plan

7

2013Lead rare disease program, KIACTA™ for AA amyloidosis in Phase III Confirmatory Study :

Recruitment on target

Japan orphan drug designation

Expand rare disease pipeline

Acquisition of Shigamabfor STEC-HUS

Research partnership for AL amyloidosis

Divestiture of non-core assets

Maintain financial health

2014Continue executing KIACTA™ plan:

Completion of recruitment

Launch of open label extension study

Market and pricing assessment

Progress pipeline projects:

Animal studies to support Shigamab Phase II

Pre-clinical proof of concept for AL amyloidosis

Continued financial stewardship

Page 8: Bellus corporate presentation january 2014 v final

Business plan fully funded through KIACTA™ exit

OVERVIEW CAPITAL STRUCTURE

Shareholder Information

8

Public company (TSX: BLU) based in Montreal, QC

Developing drugs for rare diseases

Late-stage product pipeline with fully funded business plan

Shares outstanding (Fully Diluted): 65M

Cash (09/30/13): ~$16M

Burn rate (monthly): <$300K

Shareholder makeup: 70% institutional, 30% retail

Page 9: Bellus corporate presentation january 2014 v final

Late stage pipeline focused on developing innovative drugs for rare diseases

Pipeline of Products

9

ShigamabsHUS

DISCOVERY PRECLINICAL PHASE I PHASE II PHASE III

KIACTA™AA amyloidosis

MARKET

AL amyloidosis

Page 10: Bellus corporate presentation january 2014 v final

Lead Phase III Product Candidate

10

A rare and deadly kidney disease with no specific treatment

FOR AMYLOID A (AA) AMYLOIDOSIS

Page 11: Bellus corporate presentation january 2014 v final

Disease and Mechanism of Action

11

CHRONIC INFLAMMATION

SERUM AMYLOID APRECURSOR (SAA) PROTEIN

AA PROTEIN + GLYCOSAMINOGLYCANS(GAGs)

ORGAN DAMAGE, IN PARTICULAR TO KIDNEYS LEADING TO DIALYSIS

REDUCTION IN FIBRIL FORMATION & DEPOSITION

Converts toAA Protein

Generatescytokine cascade

(TNFα / IL-1 / IL-6) and increases SAA levels

Rheumatic ConditionsInflammatory Bowel DiseaseChronic InfectionsFamilial Mediterranean Fever

KIACTA™ blocksAA + GAGs interaction

Systemic Amyloid A Fibril Formation & Deposition

Page 12: Bellus corporate presentation january 2014 v final

MARKET SIZEOrphan drug designation granted with market protection in the U.S. (7 years), Europe and Japan (10 years)

Formulation (Dosing Schedule) and Methods for Treating Amyloidosis with expiry in 2026

5 year patent extension can be applied to provide protection until 2031

MARKET PROTECTIONKIACTA™ peak annual revenues projected at $500 million

Clear pharmacoeconomicrationale due to high cost of kidney disease

Premium pricing for comparative rare disease drugs

Market

Independent market assessment currently underway and to be completed by Q1 2014

12

Page 13: Bellus corporate presentation january 2014 v final

Partnership to fund Phase III Confirmatory Study with significant upside for BELLUS shareholders

With global fund AuvenTherapeutics, a private equity group specialized in drug development project financing

Auven Therapeutics funding 100% of KIACTA™’s Phase III Confirmatory Study

US$10M in upfront by AuvenTherapeutics

≥ US$50M in investments by Auven Therapeutics

Proceeds of exit expected to be shared 50-50

Strategic Partnership

13

FINANCIALSPARTNERSHIP

Page 14: Bellus corporate presentation january 2014 v final

0

5

10

15

20

25

30

35

40

45

50

Placebo

KIACTA

Composite Endpoint (Time to

First Worse Event)

Doubling Serum

Creatine

50%DecreaseCreatine

CIearance

Dialysis/ESRD

Num

ber o

f Wor

se E

vent

s

14

*

*

**

Strong Clinical Results in First Phase III Study

Landmark study in AA amyloidosis: 183 patientstreated for 2 years

Important benefits for patients on drug:

Statistically significant reduction in number and risk of reaching worsening kidney event

Important delay in reaching dialysis

*p<0.05 **p<0.01

Page 15: Bellus corporate presentation january 2014 v final

15

Regulatory

New England Journal of Medicine publication concludes that KIACTATM

slows decline of renal function in AA amyloidosis

Agreement reached in U.S., Europe, Japan to conduct Phase III Confirmatory Study

Approval based on achieving comparable result of first Phase III Study

Page 16: Bellus corporate presentation january 2014 v final

Study enrolling 230 patients total with ~200 patients enrolled

Event driven trial to complete when 120 of 230 patients reach event of kidney function deterioration

Study completion expected in 2016

16

Phase III Confirmatory Study

Page 17: Bellus corporate presentation january 2014 v final

Second Rare Disease Product Candidate

17

A rare disease primarily affecting the kidneys of children

FOR STEC RELATEDHEMOLYTIC UREMIC SYNDROME (SHUS),SHIGAMAB

Page 18: Bellus corporate presentation january 2014 v final

Disease Course and Mechanism of Action

E. COLI INGESTION

GUT COLONIZATION AND SECRETION OF TOXIN INTO BLOODSTREAM

TOXIN MAY BE CARRIED BY PMNs IN BLOODSTREAMSYMPTOMS: BLOODY DIARRHEA

SHIGAMAB BINDING NEUTRALIZES TOXIN WHICH IS THEN ELIMINATED

ShigamabAntibody

Day -4 Day 0 Day 4 Day 8

TOXIN BINDS TO GB3 RECEPTORS ON KIDNEY LEADING TO STEC-HUS. OUTCOMES: -CHRONIC KIDNEY DISEASE / HYPERTENSION: 40%-ENCEPHALOPATHY / DEATH: 5%-RESOLUTION: 55%

18

90%

SPONTANEOUSRESOLUTION

10%

SHIGAMAB TREATMENT

Page 19: Bellus corporate presentation january 2014 v final

Potential for partnership in 18-24 months

Shigamab Overview

19

NEXT STEPS (12 MONTHS)

MARKET OPPORTUNITY

PRE-CLINICAL AND CLINICAL

Proof of concept for treatment of sHUS in animal modelsMeetings with regulators to agree on development plan

2,000-3,000 estimated annual cases of sHUS in developed countries, principally children$100-200 million annual sales opportunity

Treatment with Shigamab led to significantly increased survival in STEC animal modelSafe and well tolerated in target pediatric population

Page 20: Bellus corporate presentation january 2014 v final

Research Program

20

A rare and deadly blood disorder

RESEARCH PROJECT FOR AMYLOID LIGHT-CHAIN (AL) AMYLOIDOSIS,DRUG CANDIDATES

Page 21: Bellus corporate presentation january 2014 v final

Potential for pre-clinical proof-of-concept within 12 months

AL Amyloidosis Project Overview

21

PARTNERSHIP

DISEASE

Partnership with Amorchem, a Montreal-based venture fund, to finance research projectObjective: identify and develop drug candidates for AL amyloidosis to pre-clinical proof-of-concept

AL amyloidosis is a blood disorder that leads to the formation of toxic amyloid fibrils and plaquesTreatment options are limited leading to death in most cases2,000-3,000 new cases are reported each year in the United States

Page 22: Bellus corporate presentation january 2014 v final

Shareholder Ownership

Bellini Family ≈ 30%

Power Corporation ≈ 30%

Pharmascience ≈ 10%

Governance and Shareholders

22

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.

Page 23: Bellus corporate presentation january 2014 v final

Short-term milestones driving long-term value

Milestones

23

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

2014 MilestonesContinue executing KIACTA™ plan:

Completion of recruitment

Launch of open label extension study

Market and pricing assessment

Progress pipeline projects:

Animal studies to support Shigamab Phase II

Pre-clinical proof of concept for AL amyloidosis

Long Term Value

KIACTA™ exit and results of Phase III Confirmatory Study

Shigamab partnership or proof-of -concept Phase II study results

Page 24: Bellus corporate presentation january 2014 v final

Forward Looking Statement

24

Certain statements contained in this presentation, other than statements of fact that areindependently verifiable at the date hereof, may constitute forward-looking statements. Suchstatements, based as they are on the current expectations of management, inherently involvenumerous risks and uncertainties, known and unknown, many of which are beyond BELLUSHealth Inc.'s control. Such risks include but are not limited to: the ability to obtain financingimmediately in current markets, the impact of general economic conditions, general conditionsin the pharmaceutical and/or nutraceuticals industry, changes in the regulatory environment inthe 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 andquality-controlled verification of data and analyses has been completed.

Consequently, actual future results may differ materially from the anticipated results expressedin the forward-looking statements. The reader should not place undue reliance, if any, on anyforward-looking statements included in this news release. These statements speak only as ofthe date made and BELLUS Health Inc. is under no obligation and disavows any intention toupdate or revise such statements as a result of any event, circumstances or otherwise, unlessrequired by applicable legislation or regulation. Please see the Company’s public fillingsincluding the Annual Information Form of BELLUS Health Inc. for further risk factors that mightaffect the BELLUS Health Group and its business

Page 25: Bellus corporate presentation january 2014 v final