Benitec Biopharma Inc. · NASDAQ:BNTC
Benitec Biopharma — Investor Presentation (January 2024)
23 slides from Benitec Biopharma's investor presentation, January 2024
Slide 1: Corporate Presentation January 2024
CORPORATE PRESENTATION J anuary 2024 ©2023 Benitec Biopharma | Proprietary & Confidential
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: Investment Highlights
Investment Highlights Novel Technology Platform • Benitec’s DNA-directed RNA interference (ddRNAi) platform combines gene therapy with RNA interference (RNAi) to simultaneously silence & replace disease-causing genes permanently, following a single administration • Platform has application in diseas
Slide 4: Experienced and Efficient Management
Experienced and Efficient Management Jerel A. Banks, MD, PhD Megan Boston Claudia Kloth, PhD CEO & Executive Chairman Executive Director SVP of Manufacturing • Healthcare investment professional with • CEO & managing director of multiple ASX- • Over 20 years of cGMP manufacturing & process over 15 y
Slide 5: ddRNAi Platform Enables Both Permanent Silencing AND Replacement of Mutated Genes
ddRNAi Platform Enables Both Permanent Silencing AND Replacement of Mutated Genes in the Target Tissue Limitations Advantages of Current siRNA of the ddRNAi Technologies: Platform: • Requires repeated administration • Long-term therapeutic potential from a single administration • Enables only transi
Slide 6: OPMD: A Progressive, Chronic Disease With No Approved Therapeutics
OPMD: A Progressive, Chronic Disease With No Approved Therapeutics • Oculopharyngeal muscular dystrophy (OPMD) is an autosomal dominant, chronic, myopathic disorder characterized by ptosis (drooping of the upper eyelid) and progressive dysphagia (loss of the ability to swallow) due to impairment of
Slide 7: BB-301 Simultaneously Silences Mutant PABPN1 & Delivers Wildtype PABPN1
BB-301 Simultaneously Silences Mutant PABPN1 & Delivers Wildtype PABPN1 To Restore Normal Myocyte Function PABPN1 in OPMD Design of Lead Candidate * BB-301 • PABPN1 is a ubiquitous protein that controls the length of mRNA poly(A) tails, mRNA export from the nucleus & alternative poly(A) site usage.
Slide 8: BB-301: Overview of Gene Therapy for OPMD
BB-301: Overview of Gene Therapy for OPMD BB-301 targets the genetic cause of the disease by both silencing the mutant PABPN1 gene and delivering a wildtype PABPN1 gene to restore production of functional protein with a single intramuscular administration. Orphan drug designation has been granted fo
Slide 9: BB-301 Clinical Development Program in OPMD
BB-301 Clinical Development Program in OPMD Characterization of OPMD Natural BB-301 Phase 1b/2a Open- Endpoints OPMD subject disposition History Study Dosing label Dose Escalation (1 Day) at baseline assessing: (26wk) Study (NCT06185673) • Primary: (52wk) Safety and tolerability • Rates of progressi
Slide 10: Outcome Measures for the OPMD Natural History Study and BB-301 Phase 1b/2a
Outcome Measures for the OPMD Natural History Study and the BB-301 Phase 1b/2a Clinical Study (NCT06185673) Videofluoroscopic Swallowing Study Assessments Clinical Assessments Global Swallowing Pharyngeal Constrictor Other Assessments Swallowing Efficiency Function Muscle Function Dynamic Imaging Gr
Slide 11: Key Preclinical & IND-Enabling Studies
Key Preclinical & IND-Enabling Studies 11 ©2024 Benitec Biopharma | Proprietary & Confidential
Slide 12: BB-301 Silenced and Replaced PABPN1 Over a Broad Range of Doses in A17 Mouse Model
BB-301 Silenced and Replaced PABPN1 Over a Broad Range of Doses in the A17 Mouse Model of OPMD “Silence” “Replace” BB-301 Dose (vg) Inhibition of PABPN1 coPABPN1 Expression Varying levels of inhibition of PABPN1 7.50 x 1011 86% 63% expression, when coupled with partial 2.50 x 1011 75% 26% replacemen
Slide 13: BB-301 Restored Muscle Strength to Wildtype Levels in A17 Model
BB-301 Restored Muscle Strength to Wildtype Levels in A17 Model At 14-weeks post intramuscular administration of BB-301, statistically significant improvements in muscle strength and complete phenotypic correction were achieved at doses ≥ 5.00 x 1010 vg Muscle Response Muscle Response (150 Hz) (10-1
Slide 14: Dose-Dependent Resolution of INIs in Injected Muscles of A17 Mouse Model
Dose-Dependent Resolution of INIs in Injected Muscles of A17 Mouse Model of OPMD Immunofluorescence Staining of INIs in Mouse Muscle Tissue Quantitative Analysis of INIs in Mouse Muscle Tissue Heavy accumulation of Absence of INIs in Very Few PABPN1 INIs PABPN1 INIs in A17 Normal Mouse Muscle in BB-
Slide 15: IND-Enabling Studies for BB-301
IND-Enabling Studies for BB-301 Pilot Dosing Study in Beagle Dogs Toxicology Study in Beagle Dogs • 8-week study to confirm the transduction 12-week GLP Toxicology and Biodistribution study efficiency of BB-301 following direct in Beagle dogs intramuscular injection into the pharyngeal muscles via t
Slide 16: Dose-Dependent BB-301 Tissue Transduction in Beagle Dogs
Dose-Dependent BB-301 Tissue Transduction Observed 8 Weeks Following Direct Intramuscular Injection in Beagle Dogs Copies of BB-301 (average copies per cell) Hypopharyngeal Thyropharyngeal 5.66 BB-301 Dose (vg/mL) Muscle 5.12 Muscle 3.00 x 1013 vg/ml 5.12 5.66 High Volume 3.00 x 1013 vg/ml 3.15 2.70
Slide 17: Dose-Dependent Expression of coPABPN1 in Beagle Dogs
Dose-Dependent Expression of coPABPN1 Observed 8 Weeks Following Direct Intramuscular Injection of BB-301 in Beagle Dogs Copies of coPABPN1 (average copies per cell) Hypopharyngeal Thyropharyngeal 5.66 BB-301 Dose (vg/mL) Muscle 5.12 Muscle 3.00 x 1013 vg/ml 61.69 77.26 High Volume 3.00 x 1013 vg/ml
Slide 18: Dose-Dependent Expression of siRNA13, siRNA17 in Beagle Dogs
Dose-Dependent Expression of siRNA13, siRNA17 Observed 8 Weeks Following Direct Intramuscular Injection of BB-301 in Beagle Dogs siRNA13 siRNA17 Hypopharyngeal Thyropharyngeal Hypopharyngeal Thyropharyngeal Muscle Muscle Muscle Muscle BB-301 Dose (vg/mL) average copies per cell average copies per ce
Slide 19: Dose-Dependent Inhibition of PABPN1 in Beagle Dogs
Dose-Dependent Inhibition of PABPN1 Observed 8 Weeks Following Direct Intramuscular Injection of BB-301 in Beagle Dogs Average Reported % Inhibition of wtPABPN1 Hypopharyngeal Thyropharyngeal BB-301 Dose (vg/mL) 5.66 Muscle Muscle 3.00 x 1013 vg/ml 5.12 High Volume 83% 82% 3.00 x 1013 vg/ml 74% 64%
Slide 20: Broad Intellectual Property Portfolio
Broad Intellectual Property Portfolio OPMD-Related Intellectual Property AAV-Related Intellectual Property • OPMD Family 1 anticipated expiry of April AAV Family 1 anticipated expiry of 2037 August 2038 • OPMD Family 2 anticipated expiry of December 2037 • OPMD Family 3 anticipated expiry of October
Slide 21: Financial Summary
Financial Summary NasdaqCM Listed: BNTC Exclusively Nasdaq listed; re-domiciled in April 2020 Shares Outstanding 2,592,434 as of January 3, 2024 Cash & Equivalents $25.9 million as of September 30, 2023 21 ©2024 Benitec Biopharma | Proprietary & Confidential
Slide 22: Clinical and Regulatory Milestones
Clinical and Regulatory Milestones 2 Q 2023 4Q 2023 1H 2024 Mid-2024 2H 2024 BB-301 IND BB-301 Phase 1b/2a BB-301 Phase 1b/2a Application Clinical Trial Clinical Trial Approved to (NCT06185673) (NCT06185673) Proceed June 2023 Continued subject Continued subject enrollment and enrollment and evaluati
Slide 23: For additional information contact
Fo r a d d i t i o na l i nf o r m a t i o n c o nt a c t : Irina Koffler LifeSci Advisors, LLC 917-734-7387 ikoffler@lifesciadvisors.com ©2023 Benitec Biopharma | Proprietary & Confidential
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