julie phillips, ceo - biodiem · 2015. 2. 11. · rp (orphan eye disease) foundation fighting...
TRANSCRIPT
s Therapies for major infectious
diseases and related cancers
Julie Phillips, CEO www.biodiem.com
(ASX:BDM)
Presentation overview
1. Compelling investment case
2. Recent achievements
3. Strong global partnering network
4. Robust product portfolio
5. Development priorities
6. Why invest in BioDiem?
2
A compelling investment case
BioDiem has an extensive technology portfolio targeting multiple infectious diseases and cancers, supported by:
• Existing license income with strong growth.
• Existing licenses to WHO, Serum Institute of India & Changchun BCHT Biotech Co, China.
A strong pipeline of products with high value disease targets, including:
• Large markets: influenza, schistosomiasis, hepatitis, TB.
• High-value niche markets: fungal diseases, MRSA, sexually transmitted diseases, viral-related cancers.
• Influenza vaccine on the market.
Global network with leading research institutions and companies
• Including WHO, National Institutes of Health (USA), PATH (Program for Appropriate Technology in Health), Centres for Disease Control and Prevention (US), VIVALIS, and the Institute of Experimental Medicine.
• Partnering strategy accelerates development, lowers cost, while retaining IP control. 3
Multiple products targeting multiple diseases
Product Disease Targets Current Partners Development Status
LAIV Vaccine (Influenza)
Influenza – Seasonal & Pandemic
WHO SII (India) BCHT (China) IEM (Russia)
Marketed with license revenues of A$1.4m year to date Phase II (cell-based production technology)
Bird flu IEM/WHO Clinical trial commenced in Russia
LAIV Vector (Vaccine delivery) Vaccine development VIVALIS First stage of development project underway
BDM-I (Antimicrobial)
Bacterial infections (MRSA, tuberculosis)
US government backed research institutions Success in activity screening program
Fungal infections (Aspergillus, Scedosporium, yeasts
and others)
US government backed research institutions
University of Sydney
Positive screening results in difficult to treat fungi, patents granted for major indications
Parasitic diseases (schistosomiasis, others) QIMR program Success in activity screening program
SAVINE (Custom vaccines)
Nasopharyngeal carcinoma (NPC), tuberculosis In-house Seeking partner for more advanced data in animals
BDM-E (eye disease) RP (orphan eye disease)
Foundation Fighting Blindness University of Miami
Animal model data support activity in retinal disease, safety shown at doses used (in Phase I/II study).
New vaccine technologies
Dengue fever, hepatitis, various targets
University of Canberra, Australian National University Promising early-stage laboratory results 4
Recent achievements
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Influenza vaccine licensed to WHO
and Indian biotech, SII.
AUG -‐-‐-‐-‐ DEC JAN FEB MAR APR MAY
2012
SAVINE vaccine technology acquired
Influenza vaccine
licensed to Chinese
biotech, BCHT
VIVALIS: first commercial
partner signed for LAIV vector
project
$1.4 million received YTD
for LAIV influenza
vaccine licenses
BDM-‐I patents granted in US: STIs / protozoal
infec[on
In-‐license of dengue &
hepa[[s vaccine technologies
A #me of accelera#ng ac#vity..
JUN
Global partnering/commercialisation network
• Successful partnering model supports :
-‐ QLD Ins[tute of Medical Research -‐ Australian Na[onal University -‐ University of Canberra
BCHT
Serum Ins[tute
Ins[tute of Experimental Medicine
VIVALIS
WHO
Centres for Disease Control
PATH
Na[onal Ins[tutes of
Health
USAMRIID BioReliance
– Ongoing product development – Growth in royalty revenues – Reduced development costs – Retention of full control of IP
Focused development plan
Three core development programs
Target Product
1. Seasonal and pandemic flu vaccines LAIV vaccine – licensed in multiple countries
2. Vaccine development SAVINE, LAIV vector, flavivirus and hepatitis virus technologies for designing new vaccines
3. Infectious disease therapies • Fungal disease: Aspergillosis • Bacterial disease: MRSA • Parasites: Schistosomiasis
BDM-I antimicrobial
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Flu vaccines: the LAIV vaccine A revenue-‐genera1ng cornerstone
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A growing business in flu vaccines
• Current major revenue generator and growth business.
• LAIV licensed to WHO as part of Global Pandemic Influenza Action Plan.
• BioDiem has signed sublicenses with the Serum Institute of India Ltd (SII), and Changchun BCHT Biotechnology Co. (BCHT) of China.
• H1N1 (pandemic) influenza vaccine launched in India by SII in July 2010 (Nasovac™).
− Exclusive licence signed with Serum Institute of India for private sector sales in India.
• Currently in production for seasonal influenza
• Non-exclusive license signed with Serum Institute for Mexico, Argentina, Peru, South Africa, Bangladesh, Bhutan, Nepal, Pakistan and Sri Lanka.
• BCHT deal for Chinese private sector market signed in February 2012
YTD flu vaccine licensing revenue of A$1.4 million
Flu vaccines
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Competitive advantages
Live Attenuated Influenza Virus: LAIV
Advantages:
1. Needle-free nasal delivery: no trained personnel, blood/sharps precautions necessary.
2. Extensive clinical and market experience (>100m doses) in Russia with egg-based vaccine has established efficacy and safety in >500,000 adults and 140,000 children.
3. High yields in egg-based production; can be manufactured in cell culture to meet pandemic need without reliance on eggs, such as during a bird flu pandemic.
4. New licenses for LAIV are in negotiation.
BioDiem’s technology advantages allow it to compete in a market dominated by Big Pharma
Flu vaccines
New vaccines: Designing custom therapies
Our LAIV vector and a range of other versatile technologies for designing new
vaccines
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Versatile proprietary platform technologies
• Opportunity to target multiple infectious diseases and related cancers
• Licensing model to be pursued targeting other vaccine developers
• Complementary technologies acquired, broadening disease target range
LAIV Vector: A viral vector can deliver a customised protein into the body to produce a Protective or Boosted immune response to fight a disease e.g. nasopharyngeal cancer.
SAVINE: the “scrambled antigen vaccine” technology allows design of customised proteins e.g. NPC SAVINE for Epstein Barr virus-related diseases.
One in six cancers is linked to a virus infection
New vaccines
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A new partnership
• May 2012: BioDiem collaborating with VIVALIS SA for LAIV vector technology plan
• VIVALIS: A leading French vaccine technology provider
• Initial phase of collaboration is to investigate synergy of LAIV and Vivalis’ proprietary cell line, EB66®
• Successful results could lead to new agreement to develop BioDiem’s LAIV as a vector in Vivalis’ proprietary cell line.
New vaccines
Next steps: Establishing proof of concept and designing customised virus-based vaccines
Case study for success:
• Partnering and acquisition strategy to build vaccine development expertise paid off for Crucell, which was acquired by Johnson & Johnson in 2011 for $US 2.3 billion.
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Path to commercialisation
Test LAIV and cell line system (first
stage) (2012)
Develop vector system with
commercial partners (2013)
Package vector product for sale or
out license (2014)
New vaccines
Goal: Confirm feasibility of LAIV vector and license to vaccine developers. Progress: 1. Vivalis S.A. (NYSE Euronext Paris: VLS, France) agreement signed to test
in EB66 proprietary cell line 2. Discussion with other cell line owners ongoing Platform technology multiple new vaccine possibilities for cancers and
infectious diseases 14
Infectious diseases: the BDM-I antimicrobial
Multiple targets and accelerated development program
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BDM-I broad spectrum antimicrobial
• BDM-I’s broad activity could mean claiming a share of several major markets.
• Microorganisms being targeted include bacteria, fungi, and parasites.
• Increasing resistance to antibiotics is a major concern for healthcare systems worldwide.
• Significant opportunity to target high value diseases.
• Big Pharma are returning to this space, and looking to acquire
• Pipelines are running dry so innovative products are in high demand
Next steps: An accelerated development program with rapid market access for life-threatening diseases
Infectious diseases
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Antibiotic resistant bacteria • Methicillin-resistant Staphylococcus aureus
• Cause serious and difficult-to-treat illness, most common in hospitals.
• Increasingly of concern with more frequent multi-resistance.
• Total antibiotic market worth US$42 billion in 2009.
• New effective treatments have potential for extensive use. Major concern for hospitals
• Few treatments with more resistance to standard therapy.
• Rapid spread; patient isolation
• Carrier-status of staff as source of cross infection.
• Increases patient stay, treatment cost, risk of death.
Results to date • Successful lab screening studies show effect of BDM-I at
concentrations achievable in the body.
MRSA: a major issue for hospitals Infectious diseases: bacterial
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Fungal infection • Deep-seated invasive infection of body cavities and organs
• Can be rapidly fatal or cause chronic morbidity for patients with weak immune systems
• Existing treatments can cause serious side effects and the fungus is increasingly resistant.
• Antifungal market worth US$9.4 billion in 2010.
• High-value entry for effective antifungals in life-threatening disease.
Results to date • Activity of BDM-I has been shown in screening studies by Australian
and US research laboratories
Aspergillosis: an invasive fungus Infectious diseases: fungal
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Chronic parasitic infection • Water-living parasites which enter through the skin.
• Cause ongoing damage to organs.
• Particularly affects children in developing countries.
• Can stunt growth and mental development.
• Can increase risk of bladder cancer.
Widespread population at risk • More than 200 million people infected.
• Range of parasite places 700 million at risk.
Potential to capture leading treatment position • A “neglected disease” largely affecting developing countries.
• Few competitors offers opportunity for effective treatment.
Results to date • Successful screening studies show positive BDM-I effect on adult parasites
Schistosomiasis (bilharzia) Infectious diseases: parasites
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Infectious diseases
Next steps for BDM-I
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BDM-I has delivered a range of exciting results at world-class research facilities. The variety of possible indications gives the asset considerable scope for producing significant value to shareholders. BioDiem will build on our strong results to date by: 1. Working with our international partners to progress research
into animal models of the disease
2. Maintaining our model of collaborative research with minimal outlay by BioDiem while retaining control of IP
3. Seeking discussion with potential licensing partners for the technology
New vaccine technologies: hepatitis target
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Infectious diseases
Hepatitis program
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This new technology will use a new form of the hepatitis D virus to target hepatitis D, B and possibly C infections. Hepatitis D
• Currently no vaccines available. • 20% mortality. Liver transplant for severe cases.
Hepatitis B • Approx. 800,000-1.4m chronically infected in US. • Currently no complete cure. Existing treatments cost US$5k-$35K p.a.
Hepatitis C
• The most common bloodborne infection in the US. • Currently no vaccines available. New “triple cocktail” treatment achieves 80% cure
and costs $60K per patient treatment.
Next steps: Collaborative R&D program in place with hepatitis D as first
demonstration target.
New vaccine technologies: dengue fever target
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Dengue fever & related diseases
• Technology licenced from Australian National University in June 2012.
• Demonstration of vaccine effect in dengue fever model
• No current vaccines on the market
• Possible extension into other dangerous disease targets including:
Next Steps: Outlicence or joint collaboration for development for disease targets.
Infectious diseases
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West Nile virus Yellow fever
Japanese encephalitis
Tick-borne encephalitis
Murray Valley encephalitis
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A closer look at these diseases
Dengue Japanese encephalitis West Nile
Carrier (vector) Aedes mosquito Culex mosquito, occasionally Aedes mosquito
Culex mosquito, occasionally Aedes mosquito and others
Global cases/year 100 million 50,000 low
Mass disease in endemic regions
Not frequent, but oCen severe
Niche disease, low incidence
Infectious diseases
Adapted from Datamonitor : Stakeholder Opinions: Tropical Diseases, Dec 2010
• Mosquito-borne diseases of increasing interest due to climate-change induced habitat expansion. The size of populations at risk of exposure is increasing.
Orphan eye disease: BDM-E
A high-value niche market on track for out-licensing
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Tackling genetic eye disease A pre-packaged asset for sale
• FDA granted Orphan Drug status to BDM-E compound for treatment of genetic eye disease retinitis pigmentosa (RP).
• BioDiem’s studies show BDM-E has biological effect in inflammation and eye disease models. New intellectual property generated from these studies.
• Clinical usage: no safety concerns at dosage used.
• Results will be presented in July 2012 at International eye research conference.
• Research collaboration with Uni of Miami and Foundation Fighting Blindness to evaluate the potential of BDM-E to treat retinitis pigmentosa
• The study will examine the protective ability of BDM-E in a non-clinical disease model which mimics RP.
Next Steps: Completion of data package. On track for sale or out license within 12
months
Eye diseases
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Anti-infectives market, US$53 billion in 2011
Operating within the largest healthcare markets
Vaccine market
US$20.2 billion in
2009
Antibiotics market US$42
billion in 2009
Antifungals
market, US$9.4 billion
in 2010
Company Vaccine sales 2010
Compound annual growth rate %
(2004-‐2009), Datamonitor 2010
Key products
$6.75 billion 21.5% Infant combina1ons, hepa11s, influenza, HPV, rotavirus
$5.01 billion 18.5% Infant combina1ons, meningococcal vaccines, influenza
$3.55 billion 25.3% HPV, rotavirus, MMR-‐V vaccines
$3.67 billion 26.7% (2004-‐08) Pneumococcal vaccines
$2.92 billion 36.4% (2006-‐09) Influenza, meningococcal vaccines
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Financials and Board
As at 30 April 2012
Market Cap $8.17m
52 week range $0.07 - 0.18
Cash $1.32m
Shares 102,095,554
Shareholders 919
Listed Options 12,519,008
Revenue YTD $1.4m
Chairman: Hugh Morgan, AC • President of BCA from 2003-2005. • Chairman of the Order of Australia Association Foundation • Lafarge International Advisory Board member • Former Director of the Board of the Reserve Bank of Australia
CEO: Julie Phillips • Extensive experience in clinical and regulatory affairs • Previous Chief Executive Officer and Director of Australian
biotech start-ups • Board member of the CRC for Asthma and Airways
Non-executive Director: Dr Larisa Rudenko • Head of Virology at Institute of Experimental Medicine • Developed Russian clinical trials for LAIV • World expert in LAIV • Published more than 225 papers and 42 patents • Honoured Scientist of the Russian Federation
Non-executive Director: Dr Arthur Li • Deputy Chairman of The Bank of East Asia • Emeritus Professor of Surgery at The Chinese University of Hong Kong • Numerous Australian and HK appointments
Non-executive Director: Don Brooks • Former Licensing Senior Counsel at Merck & Co. • Former General Counsel for EntreMed • Formerly outside Counsel to HGS, MedImmune and Genetics Therapy • Numerous North American biotech consultancy roles
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Mul1ple inflec1on points on the horizon
Flu vaccine licensing
BDM-‐I (An1microbial)
New vaccines (vector)
VIVALIS collabora1on first stage results
Commercial development of vector system
Package vector product for sale or out license
2012 – 2013
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Enter animal models for major indica1ons
Secure out licensing deal for
technology
Secure further IP protec1on
Collaborate with partners to develop to market
Cell-‐based LAIV
agreement Securing of new licensees in new markets
Con1nue building revenue growth
Why invest in BioDiem?
• BioDiem has successfully licensed its flu vaccine to the largest markets in the world.
• A proven track record of new license growth, e.g. BCHT (China) and the Serum Institute of India.
• Exposure to multiple opportunities for disease treatments with high market need.
• Global partnering strategy with research leaders accelerates development and delivers more for each research and development dollar.
• Potential to engineer multiple new vaccines from BioDiem’s technologies.
• The research suggests exciting potential for BDM-I in multiple acute and chronic infectious diseases with a chance for accelerated regulatory approval.
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Therapies for major infectious
diseases and related cancers
Julie Phillips, CEO www.biodiem.com
(ASX:BDM)
Appendices
BDM-I: Work in progress
MIC Testing Results from NIH NIAID’s1 In Vitro Assessment for Antimicrobial Activity Service2
Pathogen MIC (µg/mL)
E. faecalis (drug resistant) 8
C. jejuni 0.5
S. aureus 4
MRSA 8
A. fumigatus 8
The results are encouraging and BioDiem will discuss with NIAID the potential to use NIAID’s Animal Models of Infectious Disease Service3 to further evaluate BDM-I’s activity.
1 (NIAID) is the National Institute of Allergy and Infectious Diseases, an institute of the National Institutes of Health (NIH). 2 http://www.niaid.nih.gov/LabsAndResources/resources/dmid/invitro/Pages/invitro.aspx 3 http://www.niaid.nih.gov/labsandresources/resources/dmid/animalmodels/Pages/default.aspx 34