Benitec Biopharma Announces Late Breaking Oral Abstract Presentation on BB-301 Phase 1b/2a Clinical Study at the 29th Annual Congress of the World Muscle Society

Rhea-AI Summary

Benitec Biopharma Inc. (NASDAQ: BNTC) announced that a Late Breaking Abstract on BB-301 has been accepted for oral presentation at the 29th Annual Congress of the World Muscle Society in Prague, Czech Republic, from October 8-12, 2024. The presentation, scheduled for October 12, 2024, will focus on interim clinical data from a Phase 1b/2a study evaluating the safety and clinical activity of BB-301, an AAV9-based gene therapy for Oculopharyngeal Muscular Dystrophy (OPMD) with dysphagia. Professor Milan R. Amin, M.D., from the New York University Grossman School of Medicine, will present the findings. This presentation represents a significant milestone in Benitec's development of novel genetic medicines using their proprietary 'Silence and Replace' DNA-directed RNA interference (ddRNAi) platform.

Positive

• Late Breaking Abstract on BB-301 accepted for oral presentation at a major scientific conference
• Phase 1b/2a clinical study progress for BB-301 gene therapy in OPMD
• Potential advancement in treatment for Oculopharyngeal Muscular Dystrophy

Negative

• None.

HAYWARD, Calif., Sept. 18, 2024 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or the “Company”), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary “Silence and Replace” DNA-directed RNA interference ("ddRNAi") platform, today announced that a Late Breaking Abstract on BB-301 was accepted for oral presentation at the 29^th Annual Congress of the World Muscle Society, taking place in Prague, Czech Republic from October 8-12, 2024. The Late Breaking Abstract entitled “Interim Clinical Data Summary: A Phase 1b/2a Open-label, Dose Escalation Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of an AAV9-based gene therapy (BB-301) Administered to Subjects with Oculopharyngeal Muscular Dystrophy (OPMD) with Dysphagia” will be presented on October 12, 2024.

Late Breaking Oral Presentation Title: Interim Clinical Data Summary: A Phase 1b/2a Open-label, Dose Escalation Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of an AAV9-based gene therapy (BB-301) Administered to Subjects with Oculopharyngeal Muscular Dystrophy (OPMD) with Dysphagia

Presenter: Professor Milan R. Amin, M.D., Department of Otolaryngology-Head and Neck Surgery, New York University Grossman School of Medicine, Director, New York University Langone Voice Center

Date of Conference Session: Saturday, October 12, 2024

For more information on the 29^th Annual Congress of the World Muscle Society, please click here.

Full details from the oral presentation will follow in a future press release that will be posted to the Company’s News & Events section on the Investor page of the Benitec website.

About OPMD

OPMD is a rare progressive muscle-wasting disease caused by a mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene, for which there is currently no effective drug therapy. The disease is characterized by swallowing difficulties (dysphagia), limb weakness and eyelid drooping (ptosis). Dysphagia worsens over time and can lead to chronic choking, regurgitation, aspiration pneumonia, and in severe cases, death. Available clinical and surgical interventions are limited in scope and effectiveness and do not address the underlying progressive muscle weakness.

About BB-301

BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against mutant PABPN1. The two siRNAs are modeled into microRNA backbones to silence expression of faulty mutant PABPN1, while allowing expression of the codon-optimized PABPN1 to replace the mutant with a functional version of the protein. We believe the silence and replace mechanism of BB-301 is uniquely positioned for the treatment of OPMD by halting mutant expression while providing a functional replacement protein.

About Benitec Biopharma Inc.

Benitec Biopharma Inc. (“Benitec” or the “Company”) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary “Silence and Replace” DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview of the Company can be found on Benitec’s website at www.benitec.com.

Forward Looking Statements

Except for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec’s plans to develop and potentially commercialize its product candidates, the timing of completion of pre-clinical and clinical trials, the timing of the availability of data from our clinical trials, the timing and sufficiency of patient enrollment and dosing in clinical trials, the timing of expected regulatory filings, the clinical utility and potential attributes and benefits of ddRNAi and Benitec’s product candidates, the intellectual property position, and other forward-looking statements.

These forward-looking statements are based on the Company’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities; the Company’s ability to protect and enforce its patents and other intellectual property rights; the Company’s dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Company’s products and the products of the Company’s collaboration partners; the acceptance of the Company’s products and the products of the Company’s collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the Company’s ability to satisfy its capital needs through increasing its revenue and obtaining additional financing, given market conditions and other factors, including our capital structure; our ability to continue as a going concern; the length of time over which the Company expects its cash and cash equivalents to be sufficient to execute on its business plan; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company’s reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements.

Investor Relations Contact:

Irina Koffler
LifeSci Advisors
Tel: (917) 734-7387
ikoffler@lifesciadvisors.com

FAQ

What is the significance of Benitec Biopharma's (BNTC) Late Breaking Abstract presentation at the World Muscle Society Congress?

The presentation represents a significant milestone in Benitec's development of BB-301, a gene therapy for Oculopharyngeal Muscular Dystrophy (OPMD). It will provide interim clinical data from a Phase 1b/2a study, potentially demonstrating progress in the treatment of this rare genetic disorder.

When and where will Benitec Biopharma (BNTC) present the interim clinical data for BB-301?

Benitec will present the interim clinical data for BB-301 on October 12, 2024, at the 29th Annual Congress of the World Muscle Society in Prague, Czech Republic.

What is BB-301 and what condition does it target for Benitec Biopharma (BNTC)?

BB-301 is an AAV9-based gene therapy developed by Benitec Biopharma. It targets Oculopharyngeal Muscular Dystrophy (OPMD) with dysphagia, a rare genetic disorder affecting muscles of the upper eyelids and throat.

What phase of clinical trials is Benitec Biopharma's (BNTC) BB-301 currently in?

BB-301 is currently in a Phase 1b/2a open-label, dose escalation study to evaluate its safety and clinical activity in subjects with Oculopharyngeal Muscular Dystrophy (OPMD) with dysphagia.