Benitec Biopharma Inc. · NASDAQ:BNTC
Benitec Biopharma — Investor Presentation (July 2023)
28 slides from Benitec Biopharma's investor presentation, July 2023
Slide 1: Corporate Presentation
SBENITEC H RM 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 Novel ddRNAi silence & replace disease-causing genes permanently following a single administration Technology 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 wildtype replacement genes to restore normal cell function Competitive Advantage Lead Candidate BB-301 For some genetic d
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 • 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: Intramuscular Injection of BB-301 in the Murine A17 Model
PRECLINICAL RESULTS: INTRAMUSCULAR INJECTION OF BB-301 IN THE MURINE A17 MODEL
Slide 10: BB-301 Silenced and Replaced PABPN1 Over a Broad Range of Doses in Murine Model
BB-301 Silenced and Replaced PABPN1 Over a Broad Range of Doses in the Core Murine Model of OPMD (A17 Mouse Model) 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 ea
Slide 11: BB-301 Restored Muscle Force to Wildtype Levels in Murine Models
BB-301 Restored Muscle Force to Wildtype Levels in Murine Models • Varying levels of inhibition of PABPN1 expression, when coupled with partial replacement of wildtype PABPN1, significantly: • reduced INIs • increased muscle force generation • corrected disease phenotype • Statistically significant
Slide 12: IND-Enabling Studies for BB-301
IND-Enabling Studies for BB-301 Pilot study to • 8-week Pilot Dosing study in Beagle dogs to confirm the transduction efficiency of optimize dosing BB-301 following direct intramuscular injection into the pharyngeal muscles via the use of an open surgical approach procedure and • The pharyngeal musc
Slide 13: Preclinical Results: BB-301 Pilot Dosing Study in Beagle Dogs
PRECLINICAL RESULTS: BB-301 PILOT DOSING STUDY IN BEAGLE DOGS
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, 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
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 Volume Volume (Thyropharyngeal Volume
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 Wildtype PABPN1 at All Dose Levels
Consistent Inhibition of Wildtype 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 10
Slide 19: BB-301 Clinical Development Program: IND Application Approved
BB-301 Clinical Development Program: IND Application for BB-301 Approved to Proceed by the U.S. FDA in June 2023 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 wi
Slide 20: Quantitative Radiographic Measures 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 function, and swallowing efficiency using Video
Slide 21: Clinical and Regulatory Milestones
Clinical and Regulatory Milestones 1H 2023 2Q 2023 2H 2023 2H 2023 Investigational New BB-301 Phase 1b/2a Drug (IND) Treatment Study Application Filing preliminary clinical data required to initiate disclosures for key safety the BB-301 Phase and efficacy endpoints 1b/2a Treatment for the first subj
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- • 27,981,161 as of 03/31/23 • $6.55mm as of 03/31/23 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 Wildtype Levels of coPABPN1
BB-301 Inhibited PABPN1 Expression and Restored Near Wildtype 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@soleburystrat.com
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