Benitec Biopharma Provides Operational Updates

Rhea-AI Summary

Benitec Biopharma (NASDAQ: BNTC) has announced significant progress in its Phase 1b/2a Clinical Treatment Study for BB-301, its gene therapy treatment. The company successfully completed treatment of the sixth and final subject in Cohort 1 with a low dose of BB-301 in April 2025.

The Independent Data Safety Monitoring Board (DSMB) has completed its review of all six subjects in Cohort 1 and recommended continuation of subject enrollment. Following this positive recommendation, enrollment for Cohort 2 is expected to begin in Q4 2025. The company reports encouraging results regarding BB-301's safety profile, particularly noting the benefits of their local direct intramuscular delivery method.

Positive

• Successful completion of Cohort 1 treatment with positive safety profile
• DSMB approval to continue enrollment, validating safety data
• Local intramuscular delivery enables lower doses compared to systemic administration

Negative

• None.

Insights

Biotechnology Clinical Trials Expert

Benitec's BB-301 gene therapy shows favorable safety in first cohort, enabling progression to second cohort, a positive milestone for their OPMD treatment program.

Benitec Biopharma has reached an important milestone in their clinical development program for BB-301, their gene therapy candidate for Oculopharyngeal Muscular Dystrophy (OPMD). The company has successfully completed dosing of all six subjects in Cohort 1 with the low dose of BB-301, with the final subject treated in April 2025. More significantly, the independent Data Safety Monitoring Board (DSMB) has reviewed the safety data for all six subjects and recommended continuation of the Phase 1b/2a clinical trial.

This DSMB recommendation represents a critical safety validation for Benitec's gene therapy approach. In early-stage clinical trials, especially for novel gene therapies, safety is the primary concern. The positive DSMB review indicates no significant safety signals were detected that would warrant pausing or terminating the trial.

The company highlights their direct intramuscular delivery method as potentially advantageous from a safety perspective. This local administration approach allows for lower doses compared to systemic delivery methods used in other gene therapy programs. This could potentially reduce off-target effects and systemic toxicity issues that have challenged other gene therapy candidates.

With this safety milestone achieved, Benitec now plans to begin enrollment for Cohort 2 in Q4 2025. This progression through the dose-escalation protocol is a fundamental step in determining both the safety profile across multiple dose levels and identifying potential efficacy signals. The company has indicated that additional clinical updates for Cohort 1 subjects are planned for Q4 2025, which may provide initial insights into efficacy measures beyond the current safety data.

-In April 2025 the Sixth and Final Subject of Cohort 1 was Safely Treated with the Low Dose of BB-301 in the Phase 1b/2a Clinical Treatment Study (NCT06185673)-

-Independent Data Safety Monitoring Board Review Has Been Completed for All Six Subjects Enrolled into Cohort 1, and the Data Safety Monitoring Board Recommended Continuation of Subject Enrollment for the Phase 1b/2a Clinical Treatment Study-

-Following the Positive Data Safety Monitoring Board Recommendation, Enrollment of Cohort 2 is Expected to Begin in Q4 2025-

HAYWARD, Calif., July 09, 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 recommendation of the independent Data Safety Monitoring Board (DSMB) to continue enrollment of the Phase 1b/2a Clinical Treatment Study (NCT06185673) following completion of the comprehensive review of safety information for all six Subjects enrolled into Cohort 1. Following the positive DSMB recommendation, enrollment of Cohort 2 is expected to begin in Q4 2025.

In accordance with the Protocol for the BB-301 Phase 1b/2a Clinical Treatment Study, a meeting of the DSMB was convened following the completion of the 28-day post BB-301 dosing visit for the sixth Subject enrolled into Cohort 1. The DSMB recommended the continuation of Subject enrollment for the Phase 1b/2a Clinical Treatment Study. Following the positive DSMB recommendation, enrollment of Cohort 2 is expected to begin in Q4 2025.

“We are extremely thankful and humbled to have the opportunity to continue our collaborative development work for BB-301 with the OPMD patient community and the OPMD clinical community,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “The sixth and final Subject of Cohort 1 was safely treated with the low dose of BB-301 in April 2025 and, following the favorable DSMB recommendation, we look forward to beginning the enrollment of Cohort 2 in the fourth calendar quarter of this year. We continue to be encouraged by the benign safety profile of BB-301 associated with our local route of direct intramuscular delivery, as this method of administration enables the use of lower doses of our gene therapy agent relative to the doses employed for other gene therapy programs which rely on systemic routes of administration. Additional clinical study updates for Subjects enrolled in Cohort 1 are planned for the fourth calendar quarter of this year.”

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 is the current status of Benitec's (BNTC) BB-301 clinical trial?

The trial has completed Cohort 1 with six subjects safely treated with low-dose BB-301, and received DSMB approval to continue enrollment. Cohort 2 enrollment is expected to begin in Q4 2025.

When did BNTC complete treatment of the final subject in Cohort 1?

The sixth and final subject of Cohort 1 was treated with the low dose of BB-301 in April 2025.

What was the outcome of BNTC's Data Safety Monitoring Board review?

The DSMB completed its review of all six subjects in Cohort 1 and recommended continuation of subject enrollment for the Phase 1b/2a Clinical Treatment Study.

What advantages does BB-301's delivery method offer according to Benitec?

BB-301 uses a local direct intramuscular delivery method, which enables lower doses compared to systemic administration routes used in other gene therapy programs.

When will Benitec (BNTC) provide the next clinical study updates?

Additional clinical study updates for Subjects enrolled in Cohort 1 are planned for Q4 2025.