Gene Silencing: A quiet revolution in healthcare

Transcription

1 Gene Silencing: A quiet revolution in healthcare February 2014 Benitec Ltd 1

2 Forward looking statement 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. Actual results could differ materially from those anticipated. Reasons may include risks associated with drug development and manufacture, risks inherent in the regulatory processes, delays in clinical trials, risks associated with patent protection, future capital needs or other general risks or factors. This document does not constitute an offer, solicitation or recommendation in relation to the subscription, purchase or sale of securities in any jurisdiction. Neither this presentation nor anything in it will form any part of any contract for the acquisition of securities. 2

3 Benitec investment case Developing a novel gene silencing technology ddrnai a treatment and single shot cure for a range of diseases Infectious Diseases (eg. Hepatitis C & B and HIV ) Cancer (eg. drug resistant Non Small Cell Lung Cancer) Ocular Diseases (eg. Retinitis Pigmentosa) Genetic Diseases (eg. Huntington s) Currently poised to commence Phase I/IIa clinical trials in two major diseases: Hepatitis C (TT-034) Drug-resistant lung cancer (Tribetarna ) Currently earning revenues from a number of licensing deals Protected by a dominant, global patent estate - over 100 patents covering ddrnai and specific disease targets 3

4 Company Financial Snapshot Key Financials Share Price as at close of trade 24 February 2014: Market Capitalisation (fully diluted) as at 12 February 2014: Issued Securities as at 12 February 2014: Ordinary shares Options Cash balance at 31 December 2013: Monthly Burn Rate for 6 months to 31 December 2013 (excluding clinical trial costs): ASX:BLT AUD $1.85 AUD $158M 85,365,991 9,987,513 AUD $5.2M AUD 0.345M Benitec Biopharma Limited (ASX Code: BLT) (12 Month Share Price Performance) 24 th February - Announced AUD$31.5M Capital Raise to US Institutional Investors 4

5 ddrnai Technology The next revolution in gene silencing DNA-directed RNA interference (ddrnai) is superior to traditional RNAi approaches because Specific delivery to target cells Fewer side effects Produces much longer lasting effects Multiple therapy in a single molecule - can be engineered to silence a specific gene, multiple sites on a gene or multiple genes Other gene silencing technologies Eg. Alnylam & Arrowhead ddrnai technology Eg. Benitec 5

6 In-house and Licensed Programs Focus Indication Licensees/Collaborators Discovery Pre-clinical Clinical Hepatitis C Infectious Disease HIV/AIDS Licensed to Calimmune Licensed Hepatitis B Biomics Biotechnology (JV) Non Small Cell* Lung Cancer University of New South Wales (RC) Cancer Cancer Associated Pain Stanford University (RC) Cancer Vaccines Licensed to Regen BioPharma Licensed Ocular Disease AMD** Retinitis Pigmentosa Licensed to Genable Licensed Out-licensed Genetic Disease OPMD*** Huntington s Disease Royal Holloway London University (RC) Licensed to uniqure Licensed Out-licensed Out-licensed RC = research collaboration JV = joint venture *and other chemotherapy-resistant cancers **Age-Related Macular Degeneration ***Oculopharyngeal Muscular Dystrophy, an orphan disease 6

7 Focusing on HCV: TT-034 Benitec s in-house programs focusing on Hepatitis C (TT-034) TT-034 is a Disruptive Technology in a market that will remain very large TT-034 is an RNAi therapeutic that is intended as a one-shot-cure Eliminates long treatment courses and patient compliance issues Very low toxicity in animal studies Potential for combination with small molecule therapies for enhanced efficacy Clinically relevant doses of TT-034 produce sustained levels of HCV inhibition without toxicity Competitively priced and offers significant clinical and compliance advantages Tacere Therapeutics, Inc. June

8 MOA of TT-034 Against HCV ITR A shrna-22 B shrna-19 C shrna-6 ITR Exportin 5 TT-034 DNA HCV (+) RNA shrna Dicer Viral proteins capsid replication complex HCV (-) RNA RISC HCV (+) RNA packaging Viral RNA No packaging No capsid No replication complex HCV Tacere Therapeutics, Inc. June

9 Hepatitis C Phase I/IIa Clinical Trial US-based open-label dose-escalation Phase I/IIa trial Regulatory activity: Protocol reviewed & approved by NIH RAC June 2013 US FDA IND filed December 6, 2013 Trial initiated January 12, currently enrolling Trial sites Duke University (Dr Keyur Patel) University of California, San Diego (Dr David Wyles) Design Efficacy and safety study Patients who have failed triple therapy for HCV 5 dose cohorts Interim reads 9

10 TT-034 Trial Endpoints Primary Endpoints (Safety): Incidence of adverse events Changes in clinical parameters Secondary Endpoints (Efficacy): Sustained reduction in HCV viral load in the blood Assessment of TT-034 levels in liver biopsy Assessment of shrna expression in liver biopsy shrna expression levels in serum (exosomes) Tacere Therapeutics, Inc. June

11 TT-034 Phase I/IIa Dose Cohorts Cohort Dose (vg/kg) Dose escalation step (log 10) Total No subjects Dosing scheme for subjects Starting dose 2 Sequential (1+1) 6 week Observation period per subject and between cohorts before dose escalation Sequential and parallel (1+2) Sequential and parallel (1+2) Sequential and parallel (1+2) Sequential and parallel (1+2) 6 week 6 week 10 weeks 10 weeks DSMB review after first patient in each cohort and between cohorts Extensive safety monitoring during 24 weeks observation

12 2013 Highlights May: Expansion of Benitec Board with appointment of ex-cephalon CEO Kevin Buchi June: NIH s Recombinant DNA Advisory Committee (RAC) unanimously approves TT-034 trial design for Hepatitis C June: Licensee Calimmune commences Phase I/IIa clinical trials in HIV July: Completed $10.7 million equity capital financing and secured funding for next stage of lead programs July: Completed 25:1 consolidation of issued securities Nov: Appointment of CEO Dr Peter French to Board as Managing Director Dec: Filed IND for TT-034 with US FDA (review completed and trial initiated Jan-14) 12

13 Upcoming milestones and value driving events Hepatitis C first-in-man Phase I/IIa trial to commence dosing in Q Interim safety data Q2 & Q3 Interim efficacy data Q3 & Q4 Advancement of lung cancer program Toxicology studies to be undertaken in 2014 Phase I/IIa clinical trial aimed to commence in late 2014 Progress on Calimmune s HIV/AIDS clinical trial throughout 2014 Additional licensing agreements for other disease areas Progress on other pipeline programs as resources allow 13

14 Commercially-focused Management and Board Management MD and CEO: Peter French, MBA, PhD CSIRO, St Vincent s, Cryosite founder. CSO: Michael Graham, PhD Inventor of ddrnai technology CSIRO, Benitec founder CBO: Carl Stubbings, BSc Panbio, Quest Diagnostics, Focus Diagnostics SVP R&D: David Suhy, PhD Tacere Therapeutics, Avocel, Antara Biosciences, PPD Discovery CFO: Greg West, CA Price Waterhouse, Bankers Trust, Deutsche Bank, NZI Board Chairman: Peter Francis, LLB, Grad Dip. (Intellectual Property) Partner at Francis Abourizk Lightowlers Directors: Mel Bridges, BAppSc, FAICD Tissue Therapies, Alchemia, PanBio John Chiplin, PhD Polynoma, Arana, ITI Life Science Fund Iain Ross, BSc, CH.D. Silence Therapeutics, Tissue Therapies, Ark Therapeutics Kevin Buchi Cephalon, Teva, Mesoblast, Tetralogic 14

15 Contact Information Carl Stubbings Chief Business Officer Benitec Biopharma Ltd. Phone: +61 (0)