22 February 2008
Antisense Therapeutics To Present at Sachs Associates 7th Annual North America Forum for Investing and Partnering in Biotech
Antisense Therapeutics Limited (ASX: ANP) today announced that the Company is scheduled to present at Sachs Associates 7th Annual North America Forum for Investing and Partnering in Biotech taking place on February 25-26 in Boston at the Fairmont Copley Plaza. Mark Diamond, Antisense Therapeutics Chief Executive Officer, will give a presentation on February 25, at 11:30 a.m. ET on the Company and its lead compound, ATL1102, which is currently in Phase IIa clinical trials as a treatment for multiple sclerosis (MS) and has recently been licensed to Teva Pharmaceuticals Industries Ltd. Trial results are anticipated by Mid 2008. The presentation will be followed by a question and answer session and a live webcast may be accessed at http://www.investorcalendar.com/CEPage.asp?ID=126176. A copy of the presentation is attached. For more information about Sachs Associates 7th Annual North America Forum for Investing and Partnering in Biotech, please visit the Sachs Associates website at www.sachsforum.com. About Antisense Therapeutics Limited Antisense Therapeutics Limited (ASX: ANP) is an Australian publicly listed biopharmaceutical drug discovery and development company. Its mission is to create, develop and commercialise novel antisense pharmaceuticals for large unmet markets. ANP has two drugs in development and two drugs in pre-clinical research. Its lead drug, ATL1102, is in the advanced stages of a Phase IIa trial as a potential treatment of multiple sclerosis. About ATL1102 ATL1102 is a second generation antisense inhibitor of CD49d, a subunit of VLA-4 (Very Late Antigen-4), and is currently in Phase IIa clinical trials as a treatment for MS. In inflammation, white blood cells (leukocytes) move out of the bloodstream into the inflamed tissue, for example, the CNS in MS, and the lung airways in asthma. The inhibition of VLA-4 may prevent white blood cells from entering sites of inflammation, thereby halting progression of the disease. VLA-4 is a clinically validated target in the treatment of MS. Antisense inhibition of VLA-4 has demonstrated positive effects in a number of animal models of inflammatory disease including MS (Myers et al. J Neuroimmunol 160, p12-24, 2005). Website: www.antisense.com.au Contact Information The Global Consulting Group (Andrea Costa) (646) 284-9461, [email protected] Mark Diamond, Managing Director, Antisense Therapeutics Limited +61 3 9827 8999 Market Connect Pty. Ltd. (Simon Watkin) +61 3 9863 7797 or 0413 153 272
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7TH ANNUAL NORTH AMERICA FORUM FOR INVESTING AND PARTNERING IN BIOTECH
25 – 26 February 2008, Fairmont Copley Plaza, Boston
Sachs Associates
Antisense Therapeutics LtdMelbourne, Australia
ASX: ANP
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Forward Looking Statements
This presentation contains forward-looking statements regarding the company’s business and the therapeutic and commercial potential of its technologies and products in development. Any statement describing the company’s goals, expectations, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those risks or uncertainties inherent in the process of developing technology and in the process of discovering, developing and commercialising drugs that can be proven to be safe and effective for use as human therapeutics, and in the endeavour of building a business around such products and services. Actual results could differ materially from those discussed in this presentation. Factors that could cause or contribute to such differences include, but are not limited to, those discussed in the Antisense Therapeutics Limited Annual Report for the year ended 30 June 2007, copies of which are available from the company or at www.antisense.com.au.
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ANP: Investment Fundamentals
• Collaboration with world leader in antisense technology– Isis Pharmaceuticals Inc (NASDAQ:ISIS)
• Lead compound ATL1102 • Licensed to Teva Pharmaceutical Industries• Phase IIa MS trial – fully enrolled; results due Mid’08• Target (VLA-4) clinically validated by Tysabri®
• Product pipeline with significant commercial potential– MS, asthma, diabetic retinopathy, acromegaly, prostate
cancer
• Early partnering strategy– Project validation and revenues
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Business Strategy
• Leverage from Isis antisense technology development- Mature and well characterised platform technology - Isis - one antisense drug on market; 18 in development; licensing
deals with Big Pharma- ANP’s exclusive licenses cover range of potential
targets/applications
• Utilise technology know-how to fast track projectdevelopment
• Grow pipeline of new antisense therapeutics
• Derive revenue from early partnering strategy– ATL1102 licensed to Teva PharmaceuticalsF
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ATL1103 vision, acromegalys.c. injection Toxicology & Clinical supplies
ATL1102 asthmainhaled Preclinical Efficacy
Teva have Option
ATL1101 prostate cancers.c. injection Preclinical Research
ProductProduct StatusStatus
s.c. injectionATL1102 multiple sclerosis Clinical Phase IIa
Licensed to Teva
DiseaseDisease
Product Research & Development PipelineF
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How antisense technology works…
....Blocks disease-causing proteins from being produced
Gene( )
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Second Generation Chemistryfrom proof of concept to versatile drug platform
• Second Generation chemistry (2’-MOE) brings all the strengths of first generation chemistry and addresses the limitations:
• 10 – 15 fold increase in potency• 5 – 20 fold increase in duration of action• Marked decrease in toxicities & increase in therapeutic index• S.C. dosing once a week or less frequently
• First generation chemistry was important for proof-of-concept in man. Identified the strengths and limitations of the technology.
Strengths LimitationsSelectivity PotencyRapid ID of leads Duration of actionLocal applications Chronic toxicities
I.V. dosing (for systemic)
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Disease & Market
• Life-long chronic disease of the central nervous system- No cure: goal is to reduce the severity and frequency of relapses and
to stop disease progression
• Global drug sales of >US$6bn and forecast to grow to US$10bn by 2012
• Beta-interferon first line therapy- Dose limiting side effects (flu-like symptoms)- Not effective in all patients- Longer term efficacy benefits uncertain - Neutralising antibodies formed which reduce clinical effectiveness
• Need for more effective drug with less side effects
Lead product: ATL1102 for Multiple Sclerosis
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Product
• 2nd generation antisense inhibitor of VLA-4 protein
• VLA-4 is a clinically validated target in MS (Tysabri®)– Tysabri® monoclonal antibody (mAb) to VLA-4
– Most effective MS drug to date for treatment of relapsing-remitting MS– Twice as effective as interferon at reducing relapse rates
• Antisense inhibition of VLA-4 has demonstrated positive effects in work undertaken to date
– Compelling animal data in MS animal studies comparable to VLA-4 mAb– Data published in peer reviewed scientific journal – Phase I study confirmed ATL1102 to be safe and well tolerated
• Patents granted in US, Europe, Australia and Japan; pending in Canada
ATL1102 for Multiple Sclerosis
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ATL1102 for Multiple SclerosisPhase IIa MS trial
• Assess safety & efficacy in 77 patients with relapsing-remitting MS
• Multi-centre, randomised, double-blinded, placebo-controlled
• Study fully enrolled
• Dosing: 2 arms – treatment and placebo; subcutaneous injection, twice per week over 8 weeks
• Trial supervised by independent safety monitoring board composedof international neurology experts
• Trial results due Mid’2008
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ATL1102 for Multiple SclerosisLicensed to Teva Pharmaceutical Industries
• Teva Pharmaceutical Industries (Teva)
– Top 20 global pharmaceutical company
– Market leading MS therapy; Copaxone®
• Deal
– Teva have an exclusive, world-wide license to ATL1102
– Teva to fund and perform future development beyond Phase IIa trial
– Potential milestone payments up to US$100M for MS indication
– Low double digit royalty
– ANP share milestone payments and royalty income with Isis
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Pipeline: ATL1103 for growth & sight disorders
• Antisense inhibitor to the Growth Hormone receptor (GHr)
• GH action is mediated through IGF-I hormone
- Acromegalics have elevated levels of both GH and IGF-I
- IGF-I is associated with clinical improvement in retinopathy
reduces IGFreduces IGF--I in bloodI in blood
ATL1103ATL1103blocks GH action on liverblocks GH action on liver
adapted from Le Roith et al. Endocr Rev 22, 53-74 (2001)
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Pipeline: ATL1103 for growth & sight disorders
Activity of GHr antisense confirmed in animal models • Successfully suppressed circulating IGF-I levels in mice
and primates• Significantly reduced retinal neo-vascularisation (new
blood vessels) in mouse animal model of retinopathyWilkinson-Berka, Lofthouse, Jaworski, Ninkovic, Tachas, Wraight: Mol Vis 13, 1529-38 (2007)
• Data on suppression of circulating IGF-1 levels in mice published in peer reviewed scientific journalTachas, Lofthouse, Wraight, et al.: J Endocrinol 189, 147-54 (2006)
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ATL1103 active in primates
Hepatic GHr mRNA levels
• ATL1103 is active in non-human primates• Significant suppression of hepatic target mRNA• Pharmacologically relevant suppression of circulating IGF-I• Pharmacological paradigm demonstrated in primates
Antisense Therapeutics Ltd. proprietary data
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ATL1103 for growth & sight disorders
Key features of ATL1103 development program• GHr target is clinically validated in acromegaly• GHr is expressed in liver which is a target organ for
antisense drug distribution• Ability to test for drug activity (serum IGF-I is clinical
endpoint ) in early human studies • Limited competition• Potential dosing, administration and cost advantages
– Development path has reduced risk
• Manufactured drug for pre-clinical toxicology studies
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Pipeline: Inhaled ATL1102 for asthmaProduct• Inhaled VLA-4 antisense
- Positive effects demonstrated in acute asthma model (mouse) - Drug active at low inhaled doses- Key asthma indicators suppressed
» airway hyperresponsiveness» lung eosinophilia» airway mucous accumulation
• Existing pre-clinical and clinical data on ATL1102 in MS would support clinical development of inhaled ATL1102 in asthma
• Teva Pharmaceuticals have an option to inlicense
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ATL1101 for prostate cancer
• Potent, specific, 2nd gen ASO of IGF-IR active in human cancer cells
• Exclusive licenses to Isis chemistry for IGF-IR; IP protection to ≥ 2023
• Mouse toxicology studies completed
• Extensive research literature supports anti-tumour role for IGF-IR inhibition
• Target of interest to Pharma• Imclone, Pfizer, Insmed (Phase II); Amgen, Hoffman-LaRoche, Sanofi-Aventis (Phase I);
Merck & Co, others (preclinical)
• Research collaboration with leader in prostate cancer therapy • Prof. Martin Gleave (Uni. Brit. Columbia)
• Clinical trials confirm activity of 2nd gen ASO in prostate tumour• Phase I: Oncogenex OGX-011 (Chi et al., Gleave, 2005, J Natl Cancer Inst 97, 1287-96);• Phase II: Oncogenex OGX-011 (Oncogenex Press Release 2 Jun 2007)
• Adds further diversification & value to R&D product pipelineFor
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Looking forward (2008)
• ATL1102 for MS- Complete Phase IIa trial- Report results of trial (Forecast Mid’08)
• Pipeline- ATL1103 - next step is pre-clinical tox studies- Pre-clinical research on ATL1101 in prostate cancer
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Antisense Therapeutics Limited
Key Shareholders
• Circadian Technologies 19.2%• Firebird (US fund) 11.2%• Syngene * 10.2% • Isis Pharmaceuticals 7.5%
• Market Capitalisation A$32M
* 42% owned by Circadian
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