Gene Silencing - ASX · Company Financial Snapshot 4 Key Financials ASX:BLT Share Price as at close...
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Benitec Ltd www.benitec.com 1 Gene Silencing: A quiet revolution in healthcare March 2014
Transcript of Gene Silencing - ASX · Company Financial Snapshot 4 Key Financials ASX:BLT Share Price as at close...
Benitec Ltd
www.benitec.com
1
Gene Silencing: A quiet revolution in
healthcare
March 2014
Copyright © Benitec Biopharma Ltd,
www.benitec.com
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.
Forward looking statement
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Copyright © Benitec Biopharma Ltd,
www.benitec.com
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
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Benitec investment case
Copyright © Benitec Biopharma Ltd,
www.benitec.com
Company Financial Snapshot
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Key Financials ASX:BLT
Share Price as at close of trade 24 February 2014:
AUD $1.85
Market Capitalisation (fully diluted) as at 12 February 2014:
AUD $158M
Issued Securities as at 12 February 2014:
Ordinary shares
Options
85,365,991
9,987,513
Cash balance at 31 December 2013: AUD $5.2M
Monthly Burn Rate for 6 months to 31 December 2013 (excluding clinical trial costs):
AUD 0.345M
Benitec Biopharma Limited (ASX Code: BLT) (12 Month Share Price Performance)
24th February - Announced AUD$31.5M Capital Raise to US Institutional Investors
Copyright © Benitec Biopharma Ltd,
www.benitec.com
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
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ddRNAi Technology The next revolution in gene silencing
Other gene silencing technologies
Eg. Alnylam & Arrowhead
ddRNAi technology Eg. Benitec
Copyright © Benitec Biopharma Ltd,
www.benitec.com
In-house and Licensed Programs
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*and other chemotherapy-resistant cancers **Age-Related Macular Degeneration ***Oculopharyngeal Muscular Dystrophy, an orphan disease
Out-licensed
Out-licensed
Out-licensed
Indication Licensees/Collaborators Discovery Pre-clinical Clinical
Hepatitis C
HIV/AIDS Licensed to Calimmune
Hepatitis B Biomics Biotechnology (JV)
Non Small Cell* Lung Cancer
University of New South Wales (RC)
Cancer Associated Pain
Stanford University (RC)
Cancer Vaccines
AMD**
Retinitis Pigmentosa
Licensed to Genable
OPMD*** Royal Holloway London University (RC)
Huntington’s Disease
Licensed to uniQure
Focus
Infectious Disease
Cancer
Ocular Disease
Genetic Disease
Licensed to Regen BioPharma
Licensed
Licensed
Licensed
Licensed
RC = research collaboration JV = joint venture
Copyright © Benitec Biopharma Ltd,
www.benitec.com
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 2013 | 7
Copyright © Benitec Biopharma Ltd,
www.benitec.com
DNA
HCV (+) RNA
HCV
capsid
replication complex
packaging
HCV (-) RNA
HCV (+) RNA
Viral proteins
TT-034
shRNA
B shRNA-19 C shRNA-6 ITR A shRNA-22 ITR
RISC
Dicer
Exportin 5
Viral RNA No capsid
No replication complex
No packaging
MOA of TT-034 Against HCV
Tacere Therapeutics, Inc. June 2013 | 8
Copyright © Benitec Biopharma Ltd,
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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, 2014- 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
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Hepatitis C Phase I/IIa Clinical Trial
Copyright © Benitec Biopharma Ltd,
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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 2013 | 10
Copyright © Benitec Biopharma Ltd,
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TT-034 Phase I/IIa Dose Cohorts
• DSMB review after first patient in each cohort and between cohorts
• Extensive safety monitoring during 24 weeks observation
Cohort Dose (vg/kg) Dose
escalation
step (log 10)
Total No
subjects
Dosing scheme for
subjects
Observation period per subject
and between cohorts before dose
escalation
1 4.00 × 1010 Starting dose 2 Sequential (1+1) 6 week
2 1.25 × 1011 0.5 3 Sequential and
parallel (1+2)
6 week
3 4.00 × 1011 0.5 3 Sequential and
parallel (1+2)
6 week
4 1.25 × 1012 0.5 3 Sequential and
parallel (1+2)
10 weeks
5 4.00 × 1012 0.5 3 Sequential and
parallel (1+2)
10 weeks
Copyright © Benitec Biopharma Ltd,
www.benitec.com
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)
2013 Highlights
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Upcoming milestones and value driving events
Hepatitis C first-in-man Phase I/IIa trial to commence dosing in Q1 2014
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
Copyright © Benitec Biopharma Ltd,
www.benitec.com
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
Commercially-focused Management and Board
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Copyright © Benitec Biopharma Ltd,
www.benitec.com
Contact Information
Carl Stubbings Chief Business Officer
Benitec Biopharma Ltd. Phone: +61 (0)423666374
E-mail [email protected] www.benitec.com
