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...

Page 1: Gene Silencing - ASX · Company Financial Snapshot 4 Key Financials ASX:BLT Share Price as at close of trade 24 February 2014: AUD $1.85 Market Capitalisation (fully diluted) as at

Benitec Ltd

www.benitec.com

1

Gene Silencing: A quiet revolution in

healthcare

March 2014

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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

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Copyright © Benitec Biopharma Ltd,

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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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Contact Information

Carl Stubbings Chief Business Officer

Benitec Biopharma Ltd. Phone: +61 (0)423666374

E-mail [email protected] www.benitec.com

http://www.benitec.com

RNA silencing:

RNA silencing:

Silencing The Past

Silencing The Past

RAPPORT DE CAPITALISATION

RAPPORT DE CAPITALISATION

$1.85 Million for Carbon Sequestration Research Selected … · 2019-01-18 · $1.85 Million for Carbon Sequestration Research With a $1.85 million grant from the New York-based Doris

$1.85 Million for Carbon Sequestration Research Selected … · 2019-01-18 · $1.85 Million for Carbon Sequestration Research With a $1.85 million grant from the New York-based Doris

EMERGENCY CALLS...Crowsnest Pass 2.25 Drumheller 2.25 Picked-up $1.85 1.85 4.05 6.40 2.90 1.85 1.85 Mailed Fort McMurray $2.30 Letfibridge a District 3.90 Medicine Hat a District 3.90

EMERGENCY CALLS...Crowsnest Pass 2.25 Drumheller 2.25 Picked-up $1.85 1.85 4.05 6.40 2.90 1.85 1.85 Mailed Fort McMurray $2.30 Letfibridge a District 3.90 Medicine Hat a District 3.90

DODATAK P E T A K 30.11 · ---------------------------------- 1.85 1.85 153.5 2.20 1.60 77.5 1.85 1.85 78.5 1.85 1.85 79.5 1.85 1.85 77.5 1.85 1.85 1.90 14.0 1.80 1.85 1.95 14.0 1.80

DODATAK P E T A K 30.11 · ---------------------------------- 1.85 1.85 153.5 2.20 1.60 77.5 1.85 1.85 78.5 1.85 1.85 79.5 1.85 1.85 77.5 1.85 1.85 1.90 14.0 1.80 1.85 1.95 14.0 1.80

Sure silencing

Sure silencing

IRB 2600ID-15/1.85

IRB 2600ID-15/1.85

Posttranscriptional gene silencing

Posttranscriptional gene silencing

Note de capitalisation

Note de capitalisation

Gene Silencing Techniques

Gene Silencing Techniques

Gene Silencing

Gene Silencing

Capitalisation éducation inclusive

Capitalisation éducation inclusive

LISTED INVESTMENT COMPANIES.€¦ · LIC Investment Profiles Domestic Equity Large Capitalisation 22 Large/Medium Capitalisation 25 Medium/Small Capitalisation 36 Small/Micro Capitalisation

LISTED INVESTMENT COMPANIES.€¦ · LIC Investment Profiles Domestic Equity Large Capitalisation 22 Large/Medium Capitalisation 25 Medium/Small Capitalisation 36 Small/Micro Capitalisation

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GENE SILENCING: CONCEPTS, APPLICATIONS, AND … · ABSTRACT: RNA interference, transcriptional gene silencing, virus induced gene silencing, and micro RNAs comprise a series of mechanisms

GENE SILENCING: CONCEPTS, APPLICATIONS, AND … · ABSTRACT: RNA interference, transcriptional gene silencing, virus induced gene silencing, and micro RNAs comprise a series of mechanisms

Transgene silencing

Transgene silencing

Evaluation capitalisation 59

Evaluation capitalisation 59

Silencing RNA techniques

Silencing RNA techniques

RNA Silencing Poster

RNA Silencing Poster