Benitec Biopharma Inc. · NASDAQ:BNTC
Benitec Biopharma — Investor Presentation (June 2022)
28 slides from Benitec Biopharma's investor presentation, June 2022
Slide 1: Corporate Presentation
C O R P O R AT E P R E S E N TAT I O N June 2022
Slide 2: Safe Harbor Statement
Safe Harbor Statement This presentation contains "forward-looking statements" within the meaning of section 27A of the US Securities Act of 1933 and section 21E of the US Securities Exchange Act of 1934. Benitec has tried to identify such forward-looking statements by use of such words as "expects,"
Slide 3: Company Highlights
Company Highlights • DNA-directed RNA interference (ddRNAi) platform combines gene therapy with RNA interference to simultaneously Novel ddRNAi silence & replace disease-causing genes permanently following a single administration Technology Platform • ddRNAi platform can potentially treat diseases t
Slide 4: DNA-directed RNA Interference (ddRNAi) Platform Simultaneously Silences & Replaces Disease-Causing Genes
DNA-directed RNA Interference (ddRNAi) Platform Simultaneously Silences & Replaces Disease-Causing Genes Benitec’s technology simultaneously silences mutant proteins and delivers wild type replacement genes to restore normal cell function Competitive Advantage Lead Candidate BB-301 For some genetic
Slide 5: ddRNAi Produces Constant Levels of shRNA Expression in Target Tissues to Permanently Silence Genes
ddRNAi Produces Constant Levels of shRNA Expression in Target Tissues to Permanently Silence Genes Traditional Gene Silencing BENITEC: Permanent Gene Silencing Advantages of Permanent Gene Silencing • Combines RNA interference with gene therapy delivery • Long-term therapeutic potential from a singl
Slide 6: Executive Team
Executive Team Expertise in Gene Therapy Development, Biological Manufacturing, and Capital Allocation Jerel A. Banks, M.D., Ph.D. Megan Boston Claudia Kloth, Ph.D. CEO and Executive Chairman Executive Director SVP of Manufacturing • Healthcare investment • CEO and Managing Director of • 20+ years o
Slide 7: BB-301: Gene Therapy for Oculopharyngeal Muscular Dystrophy (OPMD)
BB-301: Gene Therapy for Oculopharyngeal Muscular Dystrophy (OPMD), Debilitating, Progressive Disease with No Approved Therapeutic Options • Rare, autosomal dominant, monogenic disease (caused by mutant PABPN1 gene) • Estimated prevalence of 15k adults (40-60 years old) in Europe, Canada, Israel, an
Slide 8: Genetic Basis of OPMD: Trinucleotide Repeat Expansion at PABPN1 Exon 1
Genetic Basis of OPMD: Trinucleotide Repeat Expansion at PABPN1 Exon 1 WT ATG (GCG)6 -------------(GCA)3 GCG GGG GCT GCG.. MUT ATG (GCG)6 (GCG)1-7 (GCA)3 GCG GGG GCT GCG…-- PABPN1 In OPMD Ubiquitous factor that promotes interaction • Genetic mutation results in trinucleotide repeat expansion within
Slide 9: Preclinical Results: A17 Mouse Model
PRECLINICAL RESULTS: A17 MOUSE MODEL
Slide 10: BB-301 Silenced and Replaced Mutant PABPN1 Over a Broad Range of Doses in Murine Models
BB-301 Silenced and Replaced Mutant PABPN1 Over a Broad Range of Doses in Murine Models Transgenic Genome of the A17 Mouse Model Study Design BB-301 was injected into the Tibialis Anterior (TA) muscle of 10 week old-to-12 week old A17 mice (a transgenic mouse model for OPMD), and 14-weeks post admin
Slide 11: BB-301 Restored Muscle Strength to Wild Type Levels in Murine Models
BB-301 Restored Muscle Strength to Wild Type Levels in Murine Models • Varying levels of inhibition of PABPN1 expression, when coupled with partial replacement of wild type PABPN1 significantly: • reduced INIs • increased muscle strength • corrected disease phenotype • Statistically significant impr
Slide 12: CTA-Enabling and IND-Enabling Studies for BB-301
CTA-Enabling and IND-Enabling Studies for BB-301 • 8-week Pilot Dosing study in Beagle dogs to confirm the transduction efficiency of Pilot study to BB-301 following direct intramuscular injection into the pharyngeal muscles via the use of an open surgical approach determine dosing • The pharyngeal
Slide 13: Preclinical Results: Pilot Dosing Study in Beagle Dogs
PRECLINICAL RESULTS: PILOT DOSING STUDY IN BEAGLE DOGS
Slide 14: Successful, Dose-dependent Transduction of BB-301 in Target Tissue
Successful, Dose-dependent Transduction of BB-301 in Target Tissue Avg. Reported BB-301 Copies Per Cell 6.00 5.66 5.00 5.12 The data demonstrate biologically significant, 4.00 dose-dependent levels of BB-301 tissue transduction 3.00 3.15 (i.e., delivery of the multi- 2.70 functional genetic 2.00 2.0
Slide 15: Broad-based, Dose-dependent Expression of siRNA13 in Target Tissue
Broad-based, Dose-dependent Expression of siRNA13 in Target Tissue Avg. Reported siRNA13 Copies Per Cell 600,000 500,000 400,000 300,000 200,000 100,000 0 1+E13 vg/mL 3+E13 vg/mL Low 3+E13 vg/mL High 1+E13 vg/mL 3+E13 vg/mL Low 3+E13 vg/mL High (Hypopharyngeal Volume Volume (Thyropharyngeal Volume V
Slide 16: Broad-based, Dose-dependent Expression of siRNA17 in Target Tissue
Broad-based, Dose-dependent Expression of siRNA17 in Target Tissue Avg. Reported siRNA17 Copies Per Cell 120,000 100,000 80,000 60,000 40,000 20,000 0 1+E13 vg/mL 3+E13 vg/mL Low 3+E13 vg/mL High 1+E13 vg/mL 3+E13 vg/mL Low 3+E13 vg/mL High (Hypopharyngeal Volume Volume (Thyropharyngeal Volume Volum
Slide 17: Broad-based, Dose-dependent Expression of coPABPN1 in Target Tissue
Broad-based, Dose-dependent Expression of coPABPN1 in Target Tissue Avg. Reported coPABPN1 Copies Per Cell 90.00 80.00 70.00 60.00 50.00 40.00 30.00 20.00 10.00 0.00 1+E13 vg/mL 3+E13 vg/mL Low Volume 3+E13 vg/mL High Volume 1+E13 vg/mL 3+E13 vg/mL Low Volume 3+E13 vg/mL High Volume (Hypopharyngeal
Slide 18: Consistent Inhibition of Wild Type PABPN1 at All Dose Levels
Consistent Inhibition of Wild Type PABPN1 at All Dose Levels Avg. Reported % Inhibition of wtPABPN1 90 Analysis at 8-weeks 80 revealed durable and 70 biologically significant 60 levels of target gene knock-down within the 50 pharyngeal muscle cells 40 30 The average level of 20 inhibition observed 1
Slide 19: BB-301 Clinical Program
BB-301 Clinical Program OPMD Natural History Phase 1b/2a Treatment Dosing (NH) Study Study 26 Weeks 1 day 52 Weeks Characterization of OPMD patient disposition at Open-label, dose escalation study in subjects with OPMD baseline assessing: • Primary endpoints: safety and tolerability • Rates of progr
Slide 20: Reproducible Quantitative Radiographic Measures to Assess Baseline Natural History and Phase 1b/2a Endpoints
Reproducible Quantitative Radiographic Measures to Assess Baseline Natural History and Phase 1b/2a Treatment Study Endpoints Quantitative radiographic measures of global swallowing function, pharyngeal constrictor muscle function, and swallowing efficiency using Videofluoroscopic Swallowing Studies
Slide 21: Upcoming Clinical and Regulatory Milestones
Upcoming Clinical and Regulatory Milestones 2H 2022 1Q 2023 1H 2023 2H 2023 Clinical Trial Phase 1b/2a BB-301 Application and Treatment Study initial Investigational New clinical data disclosures Drug Application for key efficacy and safety Filings required to endpoints initiate the Phase OPMD Natur
Slide 22: Broad Intellectual Property Portfolio
Broad Intellectual Property Portfolio • OPMD Family 4 anticipated expiry February 2040 OPMD-related • OPMD Family 3 anticipated expiry October 2039 intellectual property • OPMD Family 2 anticipated expiry December 2037 • OPMD Family 1 anticipated expiry April 2037 AAV-related • AAV Family 1 anticipa
Slide 23: Financial Summary
Financial Summary NasdaqCM Listed: BNTC Shares Outstanding Cash & Cash Equivalents • Exclusively Nasdaq listed; re- • 8,171,690 as of 03/31/22 • $8.6mm as of 03/31/22 domiciled in April 2020 23
Slide 24: Appendix
BIOPHARMA silencing genes for life’
Slide 25: BB-301 Drove Dose-Dependent shRNA Expression in A17 Mouse Model
BB-301 Drove Dose-Dependent shRNA Expression in A17 Mouse Model (Analysis Performed 14-weeks after Administration) 25
Slide 26: BB-301 Inhibited PABPN1 Expression and Restored Near Wild Type Levels of coPABPN1 in A17 Mouse Model
BB-301 Inhibited PABPN1 Expression and Restored Near Wild Type Levels of coPABPN1 in A17 Mouse Model (Analysis Performed 14-weeks after Administration) Relative coPABPN1 Expression ((coPABPN1/GAPDH)/A17 Saline) 26
Slide 27: BB-301 Drove Dose-Dependent Resolution of Intranuclear Inclusions in A17 Mouse Model
BB-301 Drove Dose-Dependent Resolution of Intranuclear Inclusions in the Injected Muscles in A17 Mouse Model (Analysis Performed 14-weeks after Administration) 27
Slide 28: Thank You
THANK YOU C o n t a c t I n f o : wwindham@soleburytrout.com
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