Developing vaccines and vaccine technologiestechnologies

Julie Phillips, CEO

jphillips@biodiem.com

61 3 9613 4100+61 3 9613 4100

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BioDiem: OverviewBioDiem: OverviewListed on ASX in 2004 (ASX:BDM)

Income from milestone payments & royalties

Key technology: Live Attenuated Influenza Virus (LAIV) technologyLicenced from the Institute of Experimental Medicine (IEM), St Petersburg.

LAIV Influenza vaccines:LAIV Influenza vaccines:• marketed for seasonal/pandemic flu• egg & cell-based manufacturing methodegg & cell based manufacturing method

LAIV Vector: • In research in design of infectious disease and cancer vaccinesIn research in design of infectious disease and cancer vaccines

Other technologiesBDM E for retinal disease BDM–E: for retinal disease

BDM–I: antimicrobial

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BioDiem Ltd: Experienced Board and Management

H gh Morgan AC Pre io s board member of Reser e Bank and pre io s chairman of B siness Co ncil of Hugh Morgan ACChairman

Previous board member of Reserve Bank and previous chairman of Business Council of Australia.

Prof. Larisa RudenkoNon executive director

Head of the Virology department of the Institute of Experimental Medicine and a leading expert in live attenuated influenza vaccine technology working closely with the CDC and Non-executive director p gy g yWHO and other world-recognised influenza groups.

Don BrooksNon-executive director

Previous Senior Counsel-Licensing at Merck & Co., Inc. Served as Counsel to a U.S. law firm representing clients in the biotechnology industry, as well as serving as an advisor to firms in the biotechnolog and the pharmace tical ind str in general firms in the biotechnology and the pharmaceutical industry in general.

Prof. Arthur LiNon-executive director

Deputy Chairman of The Bank of East Asia and Emeritus Professor of Surgery of The Chinese University of Hong Kong. Previous board member of GlaxoWellcome plc.

Julie PhillipsExecutive director, CEO Multinational pharma background in regulatory affairs, clinical trials and pricing

Richard WadleyCFO

Previous Company Secretary and Chief Financial Officer at a number of life sciencecompanies.

Cathy CroppP j t M

Over 20 years experience in international quality/compliance, manufacturing and drug, bi l i l d i d l t tProjects Manager biologicals and vaccine development management.

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BioDiem: Financials

Capital Structure & Financials (30/06/2011)

Market Cap $8 97mMarket Cap $8.97m

52 wk Range $0.07 - 0.21

Cash $2.58M

Shares 101,984,443

Shareholders 921

Options 398 946Options 398,946

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BioDiem: Focus on Vaccines

+ +VIRUS CELL LINE ANTIGENVACCINE + +

LAIV Mammalian Disease-specificcell line proteins p

Vaccines markets are attractive: • Global market size in 2009: US$22.7b (US$36.5b in 2013)

• High growth market: CAGR 2008-2013: 13%

• High level of unmet medical need ( >40 human disease pathogens without effective vaccines )

• Therapeutic and prophylactic vaccine applicationsTherapeutic and prophylactic vaccine applications

• High barriers to entry with little generic competition

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BioDiem: PipelineBioDiem: Pipeline

R h P li i l Ph I & II Ph III M k t dVaccine

Research Preclinical Phase I & II Phase III Marketed

Seasonal & P d i

Cell-based productionPandemic Influenzavaccines

Egg-based productionLicenced to the World Health Organisation for Developing CountriesLicenced to the Serum Institute of India for certain developing country private markets

LAIV vectorplatform

Feasibility studies, then target indications of nasopharyngeal carcinoma (EBV-related) and RSV

Non-vaccine PipelineBDM-IAntimicrobialAntimicrobial

BDM-E * Retinal disease

FDA Orphan Drug Designation ”retinitis pigmentosa”* Has been in clinical trial retinitis pigmentosaF

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LAIV Influenza vaccines: LAIV Influenza vaccines: Preparation of reassortant vaccine strains

PB1PA

PB2

PAHANP

XXNAMNSNS

DonorDonorstrainstrain

tsts, ca, , ca, attatt

6:2 vaccine6:2 vaccinereassortantreassortanttsts, ca, , ca, attatt

CurrentCurrentcirculating viruscirculating virus

nonnon--tsts, not, not--ca, nonca, non--attatt

((Master donor strains: A/Leningrad/134/17/57 (H2N2), B/USSR/60/69)Master donor strains: A/Leningrad/134/17/57 (H2N2), B/USSR/60/69)

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LAIV Influenza vaccines: cold-adapted, temperature-sensitive

Normal flu grows at 37 ºC (normal body temperature)

BioDiem Master Strains grow at 25 ºCg

Temperature sensitivity means they do not reprod ce in l ngs at normaldo not reproduce in lungs at normal body temperature

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LAIV Influenza VaccinesLAIV Influenza Vaccines

• Needle-free delivery (intranasal) – no trained personnel, blood/sharps precautions necessary

I d b d i • Induces broad immune response – mucosal, systemic and cell-mediated responses

• High yield in egg & cell based production • High yield in egg & cell-based production – to meet pandemic need; not reliant on eggs (e.g avian ‘flu outbreak)

• Extensive clinical trials and on-market experience (>100m doses) Extensive clinical trials and on market experience (>100m doses) with egg-manufactured vaccine has established efficacy and safety. Safety demonstrated in >500,000 adults and 140,000 children >3yrs

• Phase II trial completed: clinical experience with cell-manufactured vaccine

• H1N1 pandemic vaccine launched in India (Nasovac™) – July 2010p ( ) y

• Extensive work on pandemic applications (H5N1) via CDC and PATH relationships

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LAIV Influenza VaccinesLAIV Influenza Vaccines

Opportunity: Additional licensees for manufacture and sale of LAIV influenza (intranasal) ( )

vaccines, both egg- and cell-based production methods.

• Cell-based manufactured vaccines:– intranasal influenza vaccine (seasonal/pandemic)– GMP materials (master seed virus, master cell bank)

Ph II d l i l d d i– Phase II development materials and documentation• Egg-based manufactured vaccines:

intranasal influenza vaccine (seasonal/pandemic)– intranasal influenza vaccine (seasonal/pandemic)– technical dossiers; know-how & expert assistance.

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LAIV – Viral Vector PlatformLAIV Viral Vector PlatformUsing the LAIV as a vector to design new vaccines

- Infectious disease vaccines e.g. RSVg- Cancer vaccines e.g. nasopharyngeal carcinoma (NPC)Income to be derived from sale of technology licences

Viral Vectors: • Many viruses in use as vectors are too weak and do not generate strong immune responses.

Potential Advantages of LAIV as a viral vector: • Demonstrated safety profile (Use in Russia and European clinical trials)• Intranasal administration; broader immune response• Intranasal administration; broader immune response• Potential to induce stronger immune response than inactivate viruses

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LAIV viral vectorLAIV – viral vector

Opportunity: Partnerships for co development investment and complementary Partnerships for co-development, investment and complementary

technologies

- GMP LAIV material and GMP cell line availableG a e a a d G ce e a a ab e- Specific field of use and territories available

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BDM-I : AntimicrobialBDM I : Antimicrobial• Novel synthetic compound active against a broad range

of pathogenic micro-organisms, including– G –ve: Haemophilus influenzae, Neisseria gonorrhoeae, Proteus vulgaris.

– G +ve: Staphylococcus aureus, Streptococcus pyogenes, Clostridium perfringens

– Fungi: Aspergillus fumigatus, Trichophyton rubru, Candida albicans

– Protozoa: Trichomonas vaginalis, Plasmodium falciparum

– Other: Mycobacterum tuberculosis

• Target indications: Serious bacterial and invasive fungal disease (high value niche)

• Patents are granted in the US, Australia, Singapore, South Africa, Russia and China. Pending in Europe, Brazil, Canada, Japan, and Malaysia.

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BDM-I : dossier to dateBDM-I : dossier to date• CMC: CMC:

– Synthetic pathway and drug substance manufactured (GLP); – stability up to 3 years;

physicochemical profiling and preliminary drug product formulation – physicochemical profiling and preliminary drug product formulation studies for iv and oral administration

• Preclinical: – in vitro MICs: fungi, bacteria and protozoa– Pharmacokinetics: serum assay development, studies in rat, mouse

( l d i d i i t ti )(oral and iv administration)– Toxicology: non-GLP in rat, nude mouse; GLP single dose po and iv in rat,

repeat dose in rat; GLP genetic toxicology study p ; g gy y

• Mechanism of action• Additional expanded sensitivity testing underway, including schistosomiasisp y g y gFor

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BDM I BDM-I Opportunity: Opportunity:

Sale/outlicence.Potential co-development with investment and complementary Potential co development with investment and complementary

technologies (e.g. drug delivery technologies)

Multiple routes of administration: Multiple routes of administration: - Oral, Topical, Pulmonary, Systemic, eye/ear dropsGLP materialGLP materialWorld-wide rights.F

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BDM-E – Retinal DiseaseBDM E Retinal Disease• A synthetic tetrapeptide in development for retinal diseases; ageing

G t d t t i j k t• Granted patents in major markets• Orphan Drug designation by FDA for Retinitis Pigmentosa

• Safety results in clinical trial (n = 192) 10microgram/day sc x 10 days

• BDM-E has effect in:– In vitro studies of cell proliferation & apoptosis; inflammation– Several animal models of human retinal diseases, including genetic

models of retinitis pigmentosamodels of retinitis pigmentosa

• BioDiem holds worldwide licence to BDM-E (except Russia and CIS)

Opportunity: Sale/outlicence of BDM-E for continued development in retinal diseases and other

degenerative diseases

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BDM E: animal model studiesBDM-E: animal model studiesAnimal Model Clinical Disease Modeled Status

Campbell Rats Genetic model of Retinitis Pigmentosa Completed+ve effect+ve effect

Rd/rd mice Genetic model of Retinitis Pigmentosa CompletedffRd/rd mice Genetic model of Retinitis Pigmentosa +ve effect

Ph ti d t ti Model of Retinitis Pigmentosa & Dry Age- CompletedPhotic damage to mouse retina ode o e s g e osa & y gerelated Retinal Degeneration

Co p e ed+ve effect

Retinopathy of Prematurity (ROP) Model of human ROP and proliferative Completedin mice retinopathies +ve effect

Retinopathy of diabetic rats Model of human Diabetic Retinopathy Completed+ ff tp y p y +ve effect

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Summary: Licensing Opportunitiesy g ppTechnology/Product Existing Licences Licenses available

LAIV I fl i EU USA JLAIV Influenza vaccine- cell based manufacturing method

EU, USA, JapanOther countries except Russia and CIS.

• Licensed to WHO for public markets

LAIV Influenza vaccine- egg based manufacturing method

in developing countries

• Royalty-bearing licence with Serum Institute for private market in India

EU, USA, JapanOther countries except Russia and CIS.

and other developing countries

LAIV vectorCo-development and Partnering licenses LAIV vector

Platform technology Partnering licenses (GMP LAIV material and cell line available)- Specific field of use and territories available

BDM-I – Antimicrobial Worldwide rights (sale, outlicence or co-development)

BDM-E – Retinitis Pigmentosa & Worldwide rights( l tli d l t)BDM E Retinitis Pigmentosa &

other retinal diseases (sale, outlicence or co-development)(except Russia & CIS)

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Developing vaccines and vaccine Developing vaccines and vaccine technologiesg

www.biodiem.com

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