This corporate presentation by Roberto Bellini of BELLUS Health Inc. discusses the company's focus on developing drugs for rare diseases. The lead product candidate is KIACTA for AA amyloidosis, a rare kidney disease with no existing treatment. Key highlights include:
- KIACTA showed positive results in a Phase III trial, significantly reducing kidney function decline. A Phase III confirmatory study is ongoing.
- If approved, KIACTA could achieve premium pricing like other orphan drugs, with over $70M in funding from partner Auven Therapeutics.
- The company also has a preclinical antibody, Shigamab, targeting STEC-related HUS, a rare kidney disease
Genetic Technologies Limited is a diversified molecular diagnostics
company developing tools for the prediction and assessment of cancer
risk to help physicians proactively manage patient health. The
Company’s lead products, ‘GeneType for Breast Cancer’ and
‘GeneType for Colorectal Cancer’, are clinically validated risk
assessment tests that are first in their class. The Company’s
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
Technologies has been a leader in the development and
commercialization of genetic risk assessment technology for 20 years.
Genetic Technologies Limited is a diversified molecular diagnostics
company developing tools for the prediction and assessment of cancer
risk to help physicians proactively manage patient health. The
Company’s lead products, ‘GeneType for Breast Cancer’ and
‘GeneType for Colorectal Cancer’, are clinically validated risk
assessment tests that are first in their class. The Company’s
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
Technologies has been a leader in the development and
commercialization of genetic risk assessment technology for 20 years.
Can-Fite BioPharma Ltd. (NYSE American: CANF) is an advanced clinical stage drug development company with a platform technology that addresses multi-billion-dollar markets in the treatment of autoimmune inflammatory diseases including Rheumatoid Arthritis and Psoriasis, and liver diseases including advanced liver cancer and NASH. Can-Fite’s drugs have an excellent safety profile with experience in over 1,000 patients. Can-Fite’s intellectual property portfolio consists of 13 patent families issued and pending. Piclidenoson and Namodenoson have been out-licensed in select territories with approximately $18 million received to date. Visit CANFinfo.com to learn more.
Genetic Technologies Limited is a diversified molecular diagnostics company
developing tools for the prediction and assessment of cancer risk to help physicians
proactively manage patient health. The Company’s lead products, ‘GeneType for
Breast Cancer’ and ‘GeneType for Colorectal Cancer’, are clinically validated risk
assessment tests that are first in their class. The Company’s development pipeline
includes new tests for Type 2 diabetes, cardiovascular disease, prostate cancer, and
melanoma. Listed on the ASX in 2000 and NASDAQ in 2005, Genetic Technologies
has been a leader in the development and commercialization of genetic risk
assessment technology for 20 years.
CytoDyn is a biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a key role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and in immune-mediated illnesses, such as GvHD and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in the first quarter of 2020 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients and plans to initiate a registration-directed study of leronlimab monotherapy indication, which if successful, could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, results from a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients, with some patients on leronlimab monotherapy remaining virally suppressed for more than five years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is at www.cytodyn.com.
Lantern Pharma is a clinical stage biotechnology company focused on leveraging artificial intelligence (“A.I.”), machine learning and genomic date to streamline the drug development process and to identify patients who will benefit from their targeted oncology therapies. Their portfolio of therapies consists of compounds that others have tried, but failed, to develop into an approved commercialized drug. Additionally, they develop new compounds with the assistance of their A.I. platform (RADR) and biomarker driven approach. The Company is currently developing four therapeutic programs.
Nutriband is a transdermal focused pharmaceutical company based in the United States with a goal to improve the safety, comfort and efficacy of existing drugs using transdermal technologies. The Company is developing a pharmaceutical pipeline to improve drug delivery technologies and capabilities for patients, physicians, and payers through transdermal delivery technologies. Nutriband’s lead product is its AVERSA® technology. AVERSA® abuse deterrent transdermal technology incorporates aversive agents to prevent the Abuse, DiVERsion, MiSuse and Accidental exposure of drugs with abuse potential, such as opioids. The Company’s first application for AVERSA® is an abuse deterrent fentanyl transdermal patch which it is developing to provide clinicians and patients a safe extended-release patch for chronic pain. The goal is to make opioid based pain treatments safe for all who need them. AVERSA technology has received patent protection in the European Union, Australia, Japan, Mexico, Russia with patent prosecution in the US and Canada. Nutriband has made sure to target all large applicable markets and its global patent protection has opened up many opportunities for the Company to make a big impact on the safety profile of drugs globally.
Global orphan drug market outlook 2018Rajesh Sarma
“Global Orphan Drug Market Outlook 2018” research report by KuicK Research comprehensive insight on following developments related to global orphan drug market:
Global & Regional Orphan Drug Market Overview
Orphan Drug Designation Criteria Across Key Markets
Market Specific Reimbursement Policy & Regulatory Framework
Orphan Drug Pipeline by Phase, Orphan Designated Disease & Country
Competitive Landscape
Can-Fite BioPharma Ltd. (NYSE American: CANF) is an advanced clinical stage drug development company with a platform technology that addresses multi-billion-dollar markets in the treatment of autoimmune inflammatory diseases including Rheumatoid Arthritis and Psoriasis, and liver diseases including advanced liver cancer and NASH. Can-Fite’s drugs have an excellent safety profile with experience in over 1,000 patients. Can-Fite’s intellectual property portfolio consists of 13 patent families issued and pending. Piclidenoson and Namodenoson have been out-licensed in select territories with approximately $18 million received to date. Visit CANFinfo.com to learn more.
Genetic Technologies Limited is a diversified molecular diagnostics company
developing tools for the prediction and assessment of cancer risk to help physicians
proactively manage patient health. The Company’s lead products, ‘GeneType for
Breast Cancer’ and ‘GeneType for Colorectal Cancer’, are clinically validated risk
assessment tests that are first in their class. The Company’s development pipeline
includes new tests for Type 2 diabetes, cardiovascular disease, prostate cancer, and
melanoma. Listed on the ASX in 2000 and NASDAQ in 2005, Genetic Technologies
has been a leader in the development and commercialization of genetic risk
assessment technology for 20 years.
CytoDyn is a biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a key role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and in immune-mediated illnesses, such as GvHD and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in the first quarter of 2020 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients and plans to initiate a registration-directed study of leronlimab monotherapy indication, which if successful, could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, results from a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients, with some patients on leronlimab monotherapy remaining virally suppressed for more than five years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is at www.cytodyn.com.
Lantern Pharma is a clinical stage biotechnology company focused on leveraging artificial intelligence (“A.I.”), machine learning and genomic date to streamline the drug development process and to identify patients who will benefit from their targeted oncology therapies. Their portfolio of therapies consists of compounds that others have tried, but failed, to develop into an approved commercialized drug. Additionally, they develop new compounds with the assistance of their A.I. platform (RADR) and biomarker driven approach. The Company is currently developing four therapeutic programs.
Nutriband is a transdermal focused pharmaceutical company based in the United States with a goal to improve the safety, comfort and efficacy of existing drugs using transdermal technologies. The Company is developing a pharmaceutical pipeline to improve drug delivery technologies and capabilities for patients, physicians, and payers through transdermal delivery technologies. Nutriband’s lead product is its AVERSA® technology. AVERSA® abuse deterrent transdermal technology incorporates aversive agents to prevent the Abuse, DiVERsion, MiSuse and Accidental exposure of drugs with abuse potential, such as opioids. The Company’s first application for AVERSA® is an abuse deterrent fentanyl transdermal patch which it is developing to provide clinicians and patients a safe extended-release patch for chronic pain. The goal is to make opioid based pain treatments safe for all who need them. AVERSA technology has received patent protection in the European Union, Australia, Japan, Mexico, Russia with patent prosecution in the US and Canada. Nutriband has made sure to target all large applicable markets and its global patent protection has opened up many opportunities for the Company to make a big impact on the safety profile of drugs globally.
Global orphan drug market outlook 2018Rajesh Sarma
“Global Orphan Drug Market Outlook 2018” research report by KuicK Research comprehensive insight on following developments related to global orphan drug market:
Global & Regional Orphan Drug Market Overview
Orphan Drug Designation Criteria Across Key Markets
Market Specific Reimbursement Policy & Regulatory Framework
Orphan Drug Pipeline by Phase, Orphan Designated Disease & Country
Competitive Landscape
Merck & co., inc. barclays final 3-10-2015Marcel Brussee
What does Merck's $10B buyback plan say about pharma M&A?
Adding $10 billion to its stock buyback program might mean Merck & Co. ($MRK) wants to keep investors happy as its sales continue to shrink. That would be the typical explanation. But The Wall Street Journal sees a different possibility--one the rest of the industry might want to pay attention to.
Source: http://www.fiercepharma.com/story/what-does-mercks-10b-buyback-plan-say-about-pharma-ma/2015-03-26?utm_campaign=SocialMedia
The problem is that we have a health industry that is not concerned with food…
…and a food industry that is not concerned with health.
For further information on how to invest please contact:
Andrew Haythorpe
Founder, Managing Director
Mobile: +61407 737 973
Email: a@bettercells.com
Skype: andrewhaythorpe
Global orphan drug market future outlook 2020Rajesh Sarma
"Global Orphan Drug Market Future Outlook 2020" report highlights:
Global Orphan Drug Market Overview
Global Orphan Drug Market Segment Analysis
FDA & EMA Regulation for Clinical Trials Orphan Designated Drugs
Orphan Drug Designation Criteria & Reimbursement Policy by Region
Comprehensive Insight on Global Orphan Drugs Clinical Pipeline & Patent Analysis by Company, Country, Indication & Phase
Global Orphan Drugs Clinical Pipeline: 697 Drugs
Majority Orphan Drugs in Phase-II Trials: 249 Drugs
Marketed Orphan Drugs: 274 Drugs
Soligenix is a late-stage biopharmaceutical company specializing in developing and commercializing products to treat rare diseases with unmet medical needs. The Company's primary focus is on its Specialized BioTherapeutics business segment, which is responsible for the development of HyBryte™, a novel photodynamic therapy that uses safe visible light for the treatment of cutaneous T-cell lymphoma (CTCL). The Company has successfully completed a Phase 3 study for HyBryte™ and is now negotiating a confirmatory Phase 3 pivotal study design with the US Food & Drug Administration, while also evaluating/pursuing potential HyBryte™ marketing approval ex-U.S. with the successfully completed Phase 3 study.
OneMedForum New York Company Presentation: Soligenix, Inc., a late-stage biopharmaceutical company, focuses on the development of products to treat life-threatening side effects of cancer treatments. Learn More at: http://www.onemedplace.com/database/list/cid/14016
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Personal Brand Statement:
As an Army veteran dedicated to lifelong learning, I bring a disciplined, strategic mindset to my pursuits. I am constantly expanding my knowledge to innovate and lead effectively. My journey is driven by a commitment to excellence, and to make a meaningful impact in the world.
1. Corporate Presentation (TSX: BLU)
Roberto Bellini
President and Chief Executive Officer
Twitter: @rbellini
Winter 2015
2. Forward Looking Statements
2
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
3. 3
30 million
people in the United States have a RARE disease.
Source: NIH: National Institutes of Health Office of Rare Diseases
4. 4
Only about 5%
of these people have a specific therapy
to treat their disease.
7. 7
At BELLUS, we are focused on developing drugs for rare
diseases starting with conditions that affect the kidneys.
8. Business plan fully funded through key milestones
OVERVIEW KEY FINANCIALS
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 (12/31/14): $12.3M
Burn rate (monthly): <$300K
9. 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
9
10. Lead Phase III Product Candidate
10
A rare and deadly
kidney disease with
no specific treatment
FOR AMYLOID A (AA)
AMYLOIDOSIS
11. Disease and Mechanism of Action
11
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
12. Patient Population
Source: Navigant Consulting 2014
10,000-
15,000
potential KIACTATM
patients in the United
States and Europe
MARKET RESEARCH
Navigant Consulting conducted
extensive primary and secondary
research including over 60
interviews with treating physicians
and key opinion leaders in the
United States and Europe
12
13. PRICING
Orphan drug designation granted with
market protection in the U.S. (7 years),
Europe and Japan (10 years)
Intellectual property to 2026
PROTECTION
Disease with large unmet medical need
and no specific treatment
Clear pharmaco-economic
rationale due to high cost of kidney
disease
Premium pricing for comparative rare
disease drugs
Market Considerations
KIACTA is well positioned to achieve premium pricing in line with
comparable rare disease drugs 13
Drug
U.S.
Patients
Disease Price
Vyndaqel 1,500
Transthyretin amyloid
polyneuropathy
$200K
Gattex 9,500 Short Bowel Syndrome $295K
Kalydeco
1,350
Cystic Fibrosis (G551D
mutation)
$335K
Procysbi
500 Nephropathic cystinosis $250K
Juxtapid 3,000
Familial
hypercholesterolemia
$250K
Jakafi
1,500 Splenomegaly $87K
COMPARABLES
14. Experienced and knowledgeable partner working on lead project
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 through
auction process either prior to, or
after, Phase III Confirmatory Study
results
Investment bank Lazard engaged
to explore sale of KIACTA™
Auven Therapeutics Partnership for KIACTA™
BUSINESS PLANAUVEN PARTNERSHIP
14
15. 0
5
10
15
20
25
30
35
40
45
50
Placebo
KIACTA
Composite
Endpoint (Time to
First Worse
Event)
Doubling
Serum
Creatinine
50%
Decrease
Creatinine
CIearance
Dialysis/
ESRD
NumberofWorseEvents
15
*
*
**
Strong Clinical Results in First Phase III Study
Landmark study in AA
amyloidosis: 183 patients
treated 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
16. 16
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
17. Study enrolled with 260 patients
Trial concludes when 120 of 230
patients reach event of kidney function
deterioration. More than two thirds of
the events have been reached
Study completion expected in 2016
Phase III Confirmatory Study
17
18. New KIACTA™ Indication - Sarcoidosis
INDICATION
DEVELOPMENT
Chronic sarcoidosis, a rare
disease that causes lung scarring
and decreased lung function
No specific treatment
Agreement with Mount Sinai Hospital
New York to start Phase 2 proof-of-
concept study in 2015
18
19. Second Rare Disease Product Candidate
19
A rare disease
primarily affecting
the kidneys of
children
FOR STEC RELATED
HEMOLYTIC UREMIC
SYNDROME (SHUS),
SHIGAMABSHIGAMAB
20. Disease Course and Mechanism of Action
E. COLI INGESTION
GUT COLONIZATION AND
SECRETION OF TOXIN
INTO BLOODSTREAM
TOXIN MAY BE CARRIED
BY PMNs IN
BLOODSTREAM
SYMPTOMS: BLOODY
DIARRHEA
SHIGAMAB BINDING
NEUTRALIZES TOXIN
WHICH IS THEN
ELIMINATED
Shigamab
Antibody
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%
20
90%
SPONTANEOUS
RESOLUTION
10%
SHIGAMAB TREATMENT
21. Shigamab Overview
21
NEXT STEPS (12 MONTHS)
MARKET OPPORTUNITY
CLINICAL
Proof-of-concept for treatment of sHUS in animal models
Meetings 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
Safe and well tolerated in target pediatric population
22. 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
Murielle 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.
22
23. KEY STATS
Shareholder Information
23
Price (52-week range): $0.44-$1.80
Daily Volume: 20K (3 month avg)
0
50,000
100,000
150,000
200,000
250,000
300,000
$0.00
$0.20
$0.40
$0.60
$0.80
$1.00
$1.20
$1.40
$1.60
$1.80
$2.00
Feb 2014 Mar 2014 Apr 2014 May 2014 Jun 2014 Jul 2014 Aug 2014 Sep 2014 Oct 2014 Nov 2014 Dec 2014 Jan 2015
BLU 1-YR STOCK PERFORMANCE (TSX)
Price Volume
ANALYSTS
Bloom Burton (D. Martin) - $3.00
Hybridan (D. Nathan) - $6.00
Zacks (J. Napodano) - $2.50
24. Potential KIACTA™ exit
Continue executing KIACTA™ for AA
Amyloidosis plan:
Completion of recruitment
Launch of open label extension
study
Market assessment
Progress rare disease pipeline projects:
IND filing for KIACTA Phase 2 for
Sarcoidosis
Animal studies to support
Shigamab Phase II
Pre-clinical, proof-of-concept for
AL amyloidosis
Focused on achieving short term milestones that drive long term
value for shareholders
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
12 Month Milestones
24