Benitec Biopharma Announces Acceptance of Late- Breaking Oral Abstract for the BB-301 Phase 1b/2a Clinical Study at the Muscular Dystrophy Association Clinical and Scientific Conference

Rhea-AI Summary

Benitec Biopharma (NASDAQ: BNTC) announced the acceptance of a late-breaking oral abstract for their BB-301 Phase 1b/2a Clinical Treatment Study at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference. The presentation, scheduled for March 19, 2025, will provide interim updates on the first three subjects treated with BB-301 for Oculopharyngeal Muscular Dystrophy (OPMD) with moderate dysphagia.

The company previously reported in October 2024 that the first two subjects showed durable, clinically meaningful improvements in swallowing function following BB-301 treatment. The therapy, based on Benitec's proprietary 'Silence and Replace' DNA-directed RNA interference platform, aims to address dysphagia, a severe, life-threatening complication of OPMD. Additional clinical updates for enrolled subjects are planned for Q4 2025.

Positive

• Interim data shows durable, clinically meaningful improvements in swallowing function
• Successful safety profile reported for first two treated subjects
• Acceptance of late-breaking presentation at major scientific conference

Negative

• data available (only from first two subjects)
• Full efficacy results not yet available

Insights

Clinical Research Analyst

The acceptance of a late-breaking oral presentation at the prestigious MDA conference carries significant weight in the clinical research community. Late-breaking abstracts are typically reserved for groundbreaking developments that emerge after standard submission deadlines, suggesting the data's novelty and potential importance.

The planned presentation of interim data from three subjects is particularly noteworthy in the context of rare disease trials. For OPMD, which affects approximately 1 in 100,000 people, even small patient cohorts can provide meaningful efficacy signals. The previously reported durable improvements in swallowing function from October 2024 are especially significant because:

• Durability is a critical metric in gene therapy, as it indicates potential long-term therapeutic benefits and reduced need for repeated treatments
• Dysphagia (difficulty swallowing) represents one of the most severe complications of OPMD, leading to malnutrition, aspiration pneumonia, and increased mortality risk
• Current treatment options are to surgical interventions that often provide only temporary relief

The strategic timing of data releases, with this update in March and additional updates planned for Q4 2025, demonstrates a well-structured clinical development program. This systematic approach to data disclosure helps maintain investor interest while providing regular validation points for the technology platform.

The focus on treating dysphagia specifically represents a smart strategic choice, as it addresses a clear unmet medical need with measurable outcomes. Success in this indication could position BB-301 as a first-in-class therapy for OPMD, potentially capturing the entire addressable market for this rare disease.

-Interim clinical study updates for the first three Subjects treated with BB-301 in the Phase 1b/2a Clinical Treatment Study to be presented as a late-breaking oral presentation at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference on March 19, 2025-

HAYWARD, Calif., Feb. 14, 2025 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “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 the acceptance of a late-breaking oral abstract for the BB-301 Phase 1b/2a Clinical Treatment Study ongoing in Subjects diagnosed with Oculopharyngeal Muscular Dystrophy (OPMD) with moderate dysphagia. Interim clinical study updates for the first three Subjects will be discussed in an oral presentation at the Muscular Dystrophy Association Clinical and Scientific Conference on March 19, 2025 at 1:15 pm Central Time.

“In October of 2024 we shared interim clinical study data which demonstrated durable, clinically meaningful improvements in swallowing function for the first two Subjects safely treated with BB-301, and we are excited to share updated interim clinical study data with caregivers and families in the OPMD community during the Muscular Dystrophy Association Clinical and Scientific Conference in March,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “Dysphagia is a severe, life-threatening complication of OPMD, and our goal is to develop a safe and efficacious treatment which meaningfully reduces the burden of dysphagia. Additional clinical updates for Subjects enrolled in the BB-301 Phase 1b/2a Clinical Treatment Study are planned for the fourth calendar quarter of this year.”

Late-Breaking Oral Abstract Presentation:

An interim study update for the Phase 1b/2a Clinical Treatment Study of BB-301 in OPMD subjects with moderate dysphagia will be presented in a late-breaking oral presentation entitled “Interim Study Update for the BB-301 Gene Therapy Phase 1b/2a First in Human Trial in Subjects with Oculopharyngeal Muscular Dystrophy with Dysphagia” at 1:15 pm Central Time on March 19^th at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference in room Coronado ABCD.

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 causative gene for OPMD). 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 commercialize its product candidates, the timing of the 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, and the clinical utility and potential attributes and benefits of ddRNAi and Benitec’s product candidates, 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: the success of our plans to develop and potentially commercialize our product candidates; the timing of the completion of preclinical studies and clinical trials; the timing and sufficiency of patient enrollment and dosing in any future clinical trials; the timing of the availability of data from our clinical trials; the timing and outcome of regulatory filings and approvals; the development of novel AAV vectors; our potential future out-licenses and collaborations; the plans of licensees of our technology; the clinical utility and potential attributes and benefits of ddRNAi and our product candidates, including the potential duration of treatment effects and the potential for a “one shot” cure; our intellectual property position and the duration of our patent portfolio; expenses, ongoing losses, future revenue, capital needs and needs for additional financing, and our ability to access additional financing given market conditions and other factors, including our capital structure; the length of time over which we expect our cash and cash equivalents to be sufficient to execute on our business plan; 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 and other regulatory developments; 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 impact of, and our ability to remediate, the identified material weakness in our internal controls over financial reporting; 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, LLC
(917) 734-7387
ikoffler@lifesciadvisors.com

FAQ

What are the interim results of BNTC's BB-301 Phase 1b/2a trial for OPMD?

The interim results showed durable, clinically meaningful improvements in swallowing function for the first two subjects treated with BB-301, with a favorable safety profile.

When will BNTC present the next clinical updates for BB-301?

Benitec will present interim updates on March 19, 2025, at the Muscular Dystrophy Association Conference, with additional updates planned for Q4 2025.

What condition does BNTC's BB-301 gene therapy treat?

BB-301 treats Oculopharyngeal Muscular Dystrophy (OPMD) with moderate dysphagia, a severe condition affecting swallowing function.

How many subjects have been treated in BNTC's BB-301 trial so far?

The press release indicates that interim data will be presented for the first three subjects treated in the Phase 1b/2a trial.