Benitec Biopharma Inc. · NASDAQ:BNTC
Benitec Biopharma — Investor Presentation (January 2023)
28 slides from Benitec Biopharma's investor presentation, January 2023
Slide 1: Corporate Presentation
Ooi H T IN E M < CORPORATE PRESENTATION
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 silence & replace Novel ddRNAi Technology disease-causing genes permanently following a single administration Platform • ddRNAi platform can potentially treat diseases t
Slide 4: 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 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 professional • CEO and Managing Director of
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 between • Genetic mutation results in trinucleotide repeat expansion
Slide 9: Preclinical Results: Murine Models
PRECLINICAL RESULTS: MURINE MODELS
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 Murine Model Study Design BB-301 was injected into the Tibialis Anterior (TA) muscle of 10 week old-to-12 week old mice, and 14-weeks post administration each A17 cohort was anestheti
Slide 11: BB-301 Restored Muscle Force to Wild Type Levels in Murine Models
BB-301 Restored Muscle Force 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 function • corrected disease phenotype • Statistically significant improv
Slide 12: IND-Enabling and CTA-Enabling Studies for BB-301
IND-Enabling and CTA-Enabling Studies for BB-301 • 8-week Pilot Dosing study in Beagle dogs to confirm the transduction efficiency of BB-301 Pilot study to following direct intramuscular injection into the pharyngeal muscles via the use of an open surgical approach determine optimal • The pharyngeal
Slide 13: Preclinical Results: Pilot Dosing Study in Beagles
PRECLINICAL RESULTS: PILOT DOSING STUDY IN BEAGLES
Slide 14: Successful, Dose-dependent, Transduction of BB-301 in Target Tissues
Successful, Dose-dependent, Transduction of BB-301 in Target Tissues Avg. Reported BB-301 Copies Per Cell 6.00 5.66 5.00 5.12 The data demonstrate biologically significant, dose- 4.00 dependent levels of BB-301 tissue transduction (i.e., 3.00 3.15 delivery of the multi- 2.70 functional genetic const
Slide 15: Broad-based, Dose-dependent, Expression of siRNA13 in Target Tissues
Broad-based, Dose-dependent, Expression of siRNA13 in Target Tissues 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 Muscle) Volume Volume (Thyropharyngea
Slide 16: Broad-based, Dose-dependent, Expression of siRNA17 in Target Tissues
Broad-based, Dose-dependent, Expression of siRNA17 in Target Tissues 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 Vol
Slide 17: Broad-based, Dose-dependent, Expression of coPABPN1 in Target Tissues
Broad-based, Dose-dependent, Expression of coPABPN1 in Target Tissues 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 (Hypopharyngea
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 50 knock-down within the pharyngeal muscle cells 40 30 The average level of 20 inhibition observed a
Slide 19: BB-301 Clinical Program
BB-301 Clinical Program OPMD Natural History Dosing Phase 1b/2a Treatment (NH) Study Study 26 Weeks 1 day 52 Weeks Characterization of OPMD patient disposition at baseline Open-label, dose escalation study in subjects with OPMD assessing: • Primary endpoints: safety and tolerability • Rates of progr
Slide 20: Quantitative Radiographic Measures Employed to Assess Key Outcome Measures
Quantitative Radiographic Measures Employed to Assess Key Outcome Measures at Baseline, During Natural History Study, and During Phase 1b/2a Treatment Study Quantitative radiographic measures of global swallowing function, pharyngeal constrictor muscle force generation, and swallowing efficiency usi
Slide 21: Clinical and Regulatory Milestones
Clinical and Regulatory Milestones 2H 2022 1Q 2023 Mid-2023 2H 2023 Investigational New Phase 1b/2a BB-301 Drug Application and Treatment Study initial Clinical Trial clinical data disclosures for Application Filings key efficacy and safety required to initiate the endpoints Phase 1b/2a Treatment OP
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- • 10,855,710 as of 09/30/22 • $16.5mm as of 09/30/22 domiciled in April 2020 23
Slide 24: Appendix
BILOPHARMA silencing genes for life Appendix
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
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
T HA NK YOU C o n t a c t I n f o : wwindham@soleburystrat.com
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