BENITEC CEO PRESENTATION AT EUROPEAN BIOTECH CONFERENCE

Transcription

1 BENITEC CEO PRESENTATION AT EUROPEAN BIOTECH CONFERENCE Brussels, Belgium, 20 May 2014: Benitec Biopharma Limited (ASX:BLT) is pleased to advise that Managing Director and CEO, Dr Peter French will deliver a presentation by invitation at the Pharmaceutical & Biotech Finance Leaders Forum, held in Brussels on 20 May at 2.50pm local time. The topic of the presentation will be Managing a global pipeline in a virtual biotech company. Dr French s presentation will cover: How to identify and contract with potential collaboration partners. Different types of deal structures to maximise limited finances whilst advancing the pipeline. Managing global partners to keep the programs on track. Leveraging partnerships to identify additional sources of funding. A copy of the presentation is attached. The conference will be attended by representatives from a number of pharmaceutical companies as well as smaller biotech companies and investors. For more information, please contact the persons below or visit the Company s website at. Company Carl Stubbings Chief Business Officer Tel: +61 (2) cstubbings@benitec.com Investor relations Jane Lowe Buchan Consulting Tel: +61 (2) jlowe@buchanwe.com.au About Benitec Biopharma Limited: Benitec Biopharma Limited is an ASX- listed biotechnology company (ASX Code: BLT) based in Sydney, Australia. The company has a pipeline of in- house and partnered therapeutic programs based on its patented gene- silencing technology, ddrnai. Benitec is developing treatments for chronic and life- threatening human conditions such as Hepatitis C, Hepatitis B, wet age- related macular degeneration, cancer- associated pain, drug resistant lung cancer and oculopharyngeal muscular dystrophy based on this technology. In addition, Benitec has licensed ddrnai technology to other biopharmaceutical companies who are progressing their programs towards the clinic for applications including HIV/AIDS, retinitis pigmentosa and Huntington s disease. For more information on Benitec refer to the Company s website at.

2 Managing a global pipeline in a virtual biotech company Dr Peter French Goal: Demonstrate different types of deal structures to maximize limited finances whilst advancing the pipeline

3 Forward looking statement This presenta,on contains forward looking statements that involve risks and uncertain,es. Although we believe that the expecta,ons reflected in the forward looking statements are reasonable at this,me, Benitec Biopharma can give no assurance that these expecta,ons will prove to be correct. Actual results could differ materially from those an,cipated. Reasons may include risks associated with drug development and manufacture, risks inherent in the regulatory processes, delays in clinical trials, risks associated with patent protec,on, future capital needs or other general risks or factors. This document does not cons,tute an offer, solicita,on or recommenda,on in rela,on to the subscrip,on, purchase or sale of securi,es in any jurisdic,on. Neither this presenta,on nor anything in it will form any part of any contract for the acquisi,on of securi,es. 1

4 Benitec Biopharma: Company Profile Australian based biotechnology company (ASX: BLT) Unique proprietary gene silencing technology (>100 patents) DNA Directed RNA interference (ddrnai) has poten,al to provide one shot cures Unlike current an.retroviral therapies, (shrna)- based gene therapy may be poten.ally cura.ve, reversing the virus s damage (to) the immune system, with a single or few administra.ons. 1 There is no ques.on a one-.me treatment for chronic hepa..s C would be an outstanding advance. 2 1 Ringpis et al, PLoS ONE 7(12): e doi: /journal.pone US- based Hep C specialist, May

5 Benitec s Field: RNA interference (RNAi) 3

6 Benitec s technology: ddrnai 1. The ddrnai construct is delivered to the target cell by a gene therapy vector, where it is transported to the nucleus 1 Benitec technology ddrnai DNA construct 2. Construct con,nuously produces short hairpin RNAs (shrnas), which move into the cytoplasm and are processed into sirnas 3. The sirnas are processed via the RISC complex with one strand binding the matching sequence on the target mrna, ac,va,ng its degrada,on and silencing the target gene sirna enzyme complex (RISC) 3 Enzyma'c cleavage of hairpin loop by DICER Target mrna cleaved 2 shrna Con6nually expressed Protein Gene 4

7 Advantages of ddrnai over sirna Long term expression of shrna provides a poten,al single- shot cure Specific delivery to target cells via gene therapy vectors Fewer side effects Las6ng benefits dsrna generated con,nuously for the life of the cell Mul6ple therapy in a single molecule can be engineered to silence a specific gene, mul,ple sites on a gene or mul,ple genes Protected by a dominant, global patent estate - over 100 patents covering ddrnai and specific disease targets 5

8 Dominant Global IP Position ddrnai pioneer Dominant interna6onal IP posi6on in RNAi human therapeu6cs Core U.S. and U.K. plahorm patents granted in 2003 Cover method for silencing any gene in any cell using ddrnai Patents granted in Australia, Canada, Czech Republic, Great Britain, Hong Kong, New Zealand, Singapore, South Africa, and United States Most of the IP in (ddrnai) is owned by Benitec Benitec lays claim to a seminal US patent that describes gene'c constructs for delaying or repressing the expression of a target gene - Nature Biotechnology, Nego'a'ng the RNAi patent thicket (March 2007) 6

9 ddrnai: One weapon: Many possible targets The ddrnai pla]orm technology has the poten,al to treat a wide range of chronic and life- threatening diseases by silencing target genes Infec,ous Diseases Cancer Gene,c Diseases Ocular Diseases 7

10 Corporate strategy: June 2010 Develop proprietary RNAi posi6on in infec6ous diseases and cancer Increase therapeu6c licenses for non- core areas IP prosecu6on and maintenance Prosecute core pla]orm (Graham) patent claims globally Maintain current patent prosecu,on of program IP Develop and file new program IP 8

11 Corporate strategy: June 2010 But in June 2010, we had X X X X No office 1.5 home- based employees in two different ci6es A global patent estate under challenge at the USPTO $0.6 m in cash So how do we do it? 9

12 Developing ddrnai: A challenge without staff, lab or cash Op6ons: In- house lab Research collabora6on License in Joint venture Sub- license Acquisi6on X ü ü ü ü ü u6lised a combina6on of these approaches 10

13 Deal structure example: Research, collaboration and licence-in Program: Drug- resistant lung cancer Collabora6on Partner: Children s Cancer Ins6tute Australia, UNSW Sydney, Australia

14 Research collaboration: Drug-resistant non-small cell lung cancer Tribetarna : Silencing the βiii- tubulin gene to restore sensi,vity to chemotherapy With clinical success in lung cancer, this approach can be developed to target other cancers that express high levels of βiii- tubulin (e.g. breast, ovarian, gastric 1 ) 1. Bri.sh Journal of Cancer (2010) 102:

15 Research collaborator and Licensor: CCIA and UNSW How did BLT identify and contract with CCIA/ UNSW? CCIA published papers supporting beta III tubulin as a target associated with drug resistance University tech transfer arm approached Benitec Description of relationship All lab work done at UNSW BLT pays for the work as a fee for service BLT has exclusive license from UNSW on the target gene How does BLT leverage CCIA/UNSW s past contacts/funding sources? BLT licensed UNSW patents on using RNAi to silence beta III tubulin How does BLT manage CCIA/UNSW to keep program on track? Regular teleconferences, reports and face-to-face meetings with researchers CCIA researchers Maria Kavallaris and Josh McCarroll 13

16 Deal structure example: Joint venture Program: Hepa66s B Partner: Biomics Biotechnologies, Nantong, China 14

17 Join venture: Hepatitis B Hepbarna : Three shrnas that target three highly conserved regions on the HBV DNA polymerase gene and cross- silence the HBsAg A combina,on therapy of a nucleoside inhibitor and Hepbarna is a poten,al powerful therapeu,c and even a cura,ve approach. Scope of Project Prepare a library of sirna sequences across the en,re length of the HBV polymerase genome (AyW subtype) Select those with the greatest knockdown efficacy! Develop shrna constructs based on the candidate sirnas Op,mise hepatocyte expression of constructs Package constructs into delivery vectors Test constructs in vivo To undertake appropriate toxicity and biodistribu,on studies! 15

18 Join venture partner: Biomics Biotechnologies How did BLT identify and contract with Biomics? Private company in China developed high throughput sirna screening Biomics MD visited Benitec in Australia How does BLT leverage Biomics past contacts/ funding sources? Chinese government funding and regulatory environment being explored Description of relationship Lab work done at Biomics Each party contributes towards the costs of the program and shares IP and revenues How does BLT manage Biomics to keep program on track? Regular teleconferences, reports and occasional visits 16

19 Deal structure example: Acquisition Programs: - Hepa66s C - Wet age- related macular degenera6on Company: Tacere Therapeu6cs Inc, California USA 17

20 Acquisition: Hepatitis C therapeutic TT- 034 HCV Genome Ø TT- 034 expresses three shrnas targe,ng three separate, highly conserved regions on the HCV virus genome Ø Delivered with AAV8 vector Ø Comprehensive preclinical safety and efficacy data generated by Pfizer and Tacere Therapeu,cs Co- developed with Pfizer, providing preclinical data at big- pharma standards Returned from Pfizer with closure of research site in Sandwich, UK Ø Ini,ated an open- label dose- escala,on Phase I/IIa study in 14 genotype 1 HCV pa,ents First dosing is expected in H1 2014; 5 dose cohorts at two US trial sites 24- week safety and efficacy endpoints, with interim data expected within months 18

21 Acquisition: Wet AMD Wet age- related macular degenera6on (AMD) Ø Current standard- of- care is repeated intraocular injec,ons using an an,- VEGF- a Ab (Lucen,s or Avas,n) Benitec s acquired wet AMD program: shmir construct delivered by a modified vector to all layers of the re,na Cell- specific promoters allow for controlled expression Good efficacy data has been demonstrated TT- 211 TT- 211 may not only halt AMD development but could reverse it ager one intravitreal injec6on 19

22 Acquisition target: Tacere Therapeutics How did BLT iden,fy and contract with Tacere? Tacere was an exclusive licensee of Benitec s Private company developed TT- 034 with Pfizer Acquired for shares in Benitec. Now a fully owned subsidiary of BLT How does BLT leverage Tacere s past contacts/ funding sources? Data from Pfizer collabora,on formed basis of IND applica,on Informs design and development of HBV program How does BLT manage Tacere to keep program on track? Acquisi,on included SVP R&D Dr David Suhy based in USA who oversees local opera,ons. 20

23 Deal structure example: Sub-licence Program: HIV/AIDS Licensee: Calimmune Inc, USA 21

24 Sub-licencee: Calimmune Deal structure: Non- exclusive license to Calimmune for use of ddrnai for up to three targets in HIV/AIDS Annual fees, milestones, and royal,es on sales Calimmune have commenced a Phase I/IIa trial in HIV/AIDS 22

25 In summary, the virtual company model: RESEARCH COLLABORATION JOINT VENTURE SUB- LICENSE Posi6ves No capital overheads or associated staff costs Can bring in specialist providers as projects require on an on- demand basis Easy to terminate Unity of purpose Greater access to more exper,se Reduced (shared) costs No cost to advance ddrnai Revenues from fees on milestones Nega6ves Rely on external providers who may have other priori,es Academic/commercial nexus Lack of control,melines Communica,on- working across regions with varying cultures,,me zones and regulatory requirements Maintaining a unified message and approach Sharing of revenue Lack of sole control Communica,on can be difficult Limited informa,on No control over the program 23

26 Benitec Biopharma: end 2013 Utilising a combination of approaches Focus Indica6on Licensees/Collaborators Discovery Pre- clinical Clinical Approach Hepa66s C Acquired Infec6ous Disease HIV/AIDS Calimmune Hepa66s B Biomics Biotechnology Licensed Joint venture Cancer Lung Cancer* Pain Cancer Vaccines UNSW Stanford University Regen BioPharma Research collabora'on Research collabora'on Licensed Ocular Disease Gene6c Disease AMD** Re6ni6s Pigmentosa OPMD*** Hun6ngton s Disease Genable Royal Holloway London University uniqure Out- licensed Out- licensed Out- licensed Acquired Licensed Research collabora'on Licensed *and other chemotherapy- resistant cancers **Age- Related Macular Degenera'on ***Oculopharyngeal Muscular Dystrophy, an orphan disease Sub- licensed program In- house program 24

27 Transformational funding event: February th February - announced AUD$31.5M capital raise to US ins,tu,onal investors Key Metrics April 2013* April 2014 Share Price as at close of trade 8 April: Market Capitalisa,on as at 8 April: Issued Securi,es as at 8 April: Ordinary shares Op,ons AUD $0.325 AUD $1.745 $ 14.4 million $ million 44,270,686 17,794, ,115,987 18,477,742 Benitec Biopharma Limited (ASX Code: BLT) (12 Month Share Price Performance) * Reflects post consolida,on data 25

28 Use of funds: advancing the pipeline Indica6on Proof of Concept Pre Clinical Phase I/IIa Phase IIb Hepa,,s C Hepa,,s B Age- related macular degenera,on Lung cancer Pain & OPMD Laboratory establishment Other 26

29 Developing ddrnai: 2014 Op6ons: In- house lab Research collabora6on License in Joint venture Sub- license Acquisi6on ü ü ü ü ü ü Will con6nue to u6lise a combina6on of these approaches to advance ddrnai into the clinic 27

30 Contact information Dr Peter French CEO & Managing Director Phone: +61 (0) E- mail: