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Benitec Biopharma Announces Promising Interim Results for BB-301 Trial

Benitec Biopharma announced "promising" interim clinical results from the BB-301 Phase 1b/2a first-in-human study evaluating low dose and high dose BB-301 treatment for Oculopharyngeal Muscular Dystrophy with moderate dysphagia. Interim and long-term clinical results for patients enrolled into Cohort 1, and interim clinical results for the first patient enrolled into Cohort 2 in the ongoing clinical trial will be presented as a late-breaking poster presentation at the Muscular Dystrophy Association, MDA, Clinical and Scientific Conference, in Orlando, Florida on March 9, 2026. "We are strongly encouraged by the 100% response rate and the depth and durability of the responses that have been observed for all patients treated with BB-301 to date," said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. "We are incredibly excited to share these interim clinical results which demonstrate positive, clinically meaningful improvements across the most critical radiographic, functional, and patient-reported assessments of swallowing function. With no currently approved treatments for OPMD patients, the results presented today represent an important step towards the management of the unmet medical need that exists in the OPMD community."

Benitec CEO Highlights Positive Progress in BB-301 Clinical Development

"We continue to be encouraged by the benign safety profile and the durability of efficacy demonstrated in our BB-301 clinical development program," said Jerel Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. "We look forward to engaging with the U.S. Food and Drug Administration (FDA) in mid-2026 to confirm the BB-301 pivotal study design and continuing to present interim clinical results at future medical conferences. I want to sincerely thank our investigators, our clinical advisors, and-most importantly-the patients and families who have made this progress possible."

Benitec Biopharma BB-301 Clinical Trial Patient Shows Positive Outcomes

Benitec Biopharma announced that the first patient treated in Cohort 1 of the BB-301 Phase 1b/2a clinical study evaluating BB-301 for the treatment of dysphagia in oculopharyngeal muscular dystrophy, OPMD, has completed the 24-month post-treatment assessment. At the 24-month follow-up timepoint, Patient 1 continued to demonstrate robust, disease-modifying outcomes. At the 24-month follow-up timepoint, Patient 1 demonstrated deepening improvements in post-swallow pharyngeal residue as compared to the final pre-treatment timepoint and the 12-month post-treatment follow-up timepoint as assessed by x-ray-based swallowing studies. Additionally, Patient 1 experienced deepening improvements in total dysphagic symptom burden as assessed by the Sydney Swallow Questionnaire, SSQ. The first 4 patients in Cohort 1 have now completed the 12-month statistical follow-up period for the Phase 1b/2a study, and all 4 Completers continued to demonstrate durable response to BB-301. All 4 Cohort 1 Completers met the pre-specified statistical criteria for response to BB-301 defined by Benitec which require improvement across 2 or more of the 5 categories of assessment comprising the Responder Analysis.