Benitec Biopharma Inc. · NASDAQ:BNTC
Benitec Biopharma — Investor Presentation (October 2012)
29 slides from Benitec Biopharma's investor presentation, October 2012
Slide 1: Benitec Biopharma's ddRNAi technology: The next therapeutic revolution?
Benitec Biopharma’s ddRNAi technology The next therapeutic revolution? October 2012 Benitec Biopharma Ltd 1 www.benitec.com
Slide 2: Forward looking statements
This presentation contains forward looking statements that involve risks and uncertainties. Although we believe that the expectations reflected in the forward looking statements are reasonable at this time, Benitec Biopharma can give no assurance that these expectations will prove to be correct. Act
Slide 3: Benitec Biopharma Ltd (ASX:BLT) aim and strategy
Benitec Biopharma Ltd (ASX:BLT) Aim: To create appropriate return on investment by commercializing CSIRO gene silencing technology. Strategy: • To execute commercial partnerships with global pharmaceutical companies. • To demonstrate the broad applicability of our technology. Copyright © Benitec Bio
Slide 4: Company Overview
Company Overview • ASX:BLT • Dominant global IP • Technology: • “Turns off” (silences) disease-causing genes • Induces patients’ cells to continuously manufacture the silencing RNA • A single administration. • Selected pipeline programs fulfill critical unmet medical needs. Copyright © Benitec Bioph
Slide 5: How ddRNAi Technology works
How ddRNAi Technology works Copyright © Benitec Biopharma Ltd, 2012. 5 www.benitec.com
Slide 6: Copyright © Benitec Biopharma Ltd, 2012. www.benitec.com
Copyright © Benitec Biopharma Ltd, 2012. 6 www.benitec.com
Slide 7: Benitec has developed the equivalent of a gene silencing penknife
Benitec has developed the equivalent of a gene silencing penknife • ddRNAi can silence any gene • Programs showcase the breadth of potential therapeutic applications Copyright © Benitec Biopharma Ltd, 2012. 7 www.benitec.com
Slide 8: Pathways to Revenue
Pathways to Revenue • Advance in-house pipeline programs to the clinic then seek to partner • Out-license other targets for ddRNAi technology to biotech and pharma companies Benitec Biopharma Ltd 8 www.benitec.com
Slide 9: Strategy to build value
Strategy to build value ddRNAi technology Human therapeutics Research tools Long-term value Immediate opportunities Developing own Joint License ddRNAi to Exploit platform as a pipeline and licensing ventures/ co- biotechs/pharmas for research tool through drugs to pharma after development wide rang
Slide 10: Pipeline and Licensed Programs
Pipeline and Licensed Programs Indication Partners/ Collaborators Discovery Pre-clinical Clinical HIV/AIDS Calimmune Hepatitis C Addition from Tacere University of New South Drug resistant lung cancer Wales Cancer-associated Stanford University neuropathic pain Hepatitis B Biomics Biotechnologies Oc
Slide 11: Hepatitis C Market Opportunity
Hepatitis C Market Opportunity • There are over 170 million HCV infected people worldwide • There are three genotypes (GT) – 75% of all cases are genotype 1 (GT1) – GT 2 & 3 are comparatively well-served • Current therapies use a combination of interferon, ribavirin and protease inhibitors – interfe
Slide 12: Hepatitis C is a "hot" therapeutic area
Hepatitis C is a “hot” therapeutic area Nov 2011: Gilead paid $11 billion to buy Pharmasset Jan 2012: BMS paid $2.5 billion to buy Inhibitex and its INX-189 drug Achillion’s CEO projected HCV treatments would fetch $20 billion by the end of this decade. "Our goal is to be a leader in hepatitis C, an
Slide 13: Hepatitis C Asset Acquisition
Hepatitis C Asset Acquisition • Benitec Biopharma announced on October 11th 2012 that it will acquire Tacere Therapeutics, and all non-cash assets, in an all share transaction. • Opportunity to acquire Tacere complements and expands ddRNAi pipeline Copyright © Benitec Biopharma Limited, 2012 www.ben
Slide 14: Description of Key Asset – TT034
Description of Key Asset – TT034 • A “one shot monotherapy cure” • Stand alone or in combination • Comprises three shRNAs targeting three separate regions on the virus genome • Inhibits HCV resistance development, high efficacy. • Mainly targeted to genotype 1 • Ready to enter a first in man phase I
Slide 15: Clinical trial opportunity in 2013
Clinical trial opportunity in 2013 Key next steps: – Meeting with the NIH Recombinant Advisory Committee – Final pre-IND meeting with FDA – Filing of the IND – Dosing of first patient Copyright © Benitec Biopharma Limited, 2012 www.benitec.com 15
Slide 16: Hepatitis B
Hepatitis B • More than 2,000 million people alive today have been infected with HBV at some time in their lives • ~350 million carriers Global Business Intelligence Hepatitis Market to 2017 USA: • Over 1.25 million people living with the consequences of chronic HBV • Over 60,000 new cases per year
Slide 17: Cancer-Associated Pain
Cancer-Associated Pain • Approximately 65% of all cancer patients experience pain • 11.7 million people in the US with cancer Copyright © Benitec Biopharma Ltd, 2012. 17 www.benitec.com
Slide 18: Identifying the key pain gene target
Identifying the key pain gene target PKCγ-Mediated Pain Inhibition in Rats • Increased PKCg found in the spinal cord in cancer pain One dose of PKCg-silencing ddRNAi construct 6 weeks Dose- No dependent toxicity increase in pain threshold • Switching off PKCg also overcomes morphine tolerance Copyri
Slide 19: Target product profile – Nervarna
Target product profile – Nervarna™ • Terminally ill cancer patients • Intractable neuropathic pain • Morphine ineffective • Single intrathecal injection of a single shRNA targeting PKCg in a lentiviral vector • Long term pain relief on its own or in conjunction with opioids. Collaboration with Stanf
Slide 20: Drug-resistant lung cancer
Drug-resistant lung cancer low$βIII'tubulin$ • Non-Small Cell Lung Cancer (NSCLC) is theβIII'tubulin$ high$ one of the most common cancers: 1.6 M new cases per year globally • Dismal prognosis, with a high proportion becoming resistant to conventional drug therapy within a short period of time Overa
Slide 21: Target product profile – Tribetarna
Target product profile – Tribetarna™ • Non-small cell lung cancer patients • Resistant to chemotherapy – DNA damaging agents and tubulin binding agents • Single intravenous injection of a triple shRNA targeting bIII-tubulin in a non-viral vector • Adjunct to chemotherapy – restoration of tumour sens
Slide 22: Delivery and silencing of bIII-tubulin gene
Delivery and silencing of bIII-tubulin gene expression in NSCLC tumours in vivo Treated mouse with bIII tubulin is orthotopic lung tumors 120 decreased by JetPEi specifically around 50% in delivers the gene 100 lung tumours in construct to lung cells the orthotopic 80 model The uptake of the 60 vect
Slide 23: OPMD (oculopharyngeal muscular dystrophy)
OPMD (oculopharyngeal muscular dystrophy) • No effective treatment exists • Symptoms: swallowing difficulties leading to choking and death OPMD is an orphan disease • 1 in 100 000 • Caused by mutation in PABPN1 gene Benitec Biopharma Approach: ddRNAi-based silencing of mutant gene and replacement wi
Slide 24: Big Pharma are doing big deals in Benitec's program areas
Big Pharma are doing big deals in Benitec’s program areas Phase I/II clinical trials are Benitec Biopharma’s next significant inflection point. Companies Condition Stage Deal When Xenon / Pain Phase A $646 million deal – undisclosed Jan Genentech II upfronts and milestones. 2012 Enanta / Hep C Phase
Slide 25: Other potential applications
Other potential applications… • Infectious diseases • Multiple cancer types • Cardiovascular disease • Huntington’s disease • Alzheimer’s • Autoimmune Hepbarna™ • Stem cells • Genetic diseases Pabparna™ Copyright © Benitec Biopharma Ltd, 2012. 25 www.benitec.com
Slide 26: Outlicensed projects utilising Benitec's ddRNAi technology
Outlicensed projects utilising Benitec Biopharma’s ddRNAi technology HIV/AIDS - Calimmune, USA Retinitis Pigmentosa – Genable, Ireland Benitec Biopharma Ltd 26 www.benitec.com
Slide 27: Experienced Leadership Team
Experienced Leadership Team Dr Michael Graham CSO Mel Bridges Peter Francis Peter Carl Stubbings Iain Ross NED Chairman Greg West French Chief Business NED John Chiplin CFO CEO Officer NED Copyright © Benitec Biopharma Ltd, 2012. 27 www.benitec.com
Slide 28: In summary
In summary: • Potent long-lasting gene silencing platform technology from CSIRO • Multiple patent protection world wide • Proven pre-clinical and clinical efficacy and safety • Broad pipeline in multiple very high value therapeutic areas Benitec Biopharma Limited 28 www.benitec.com
Slide 29: Contact Information
Contact Information For further information, please contact: Dr Peter French Chief Executive Officer Benitec Biopharma Ltd. Phone: +61 (0)412 457 595 E-mail: [email protected] www.benitec.com Benitec Biopharma video: http://www.youtube.com/watch?v=KYRRNgziRpQ Copyright © Benitec Biopharma Ltd, 201
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