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Benitec Biopharma Announces Positive Interim Phase 1b/2a Results for High Dose BB-301 and Continued Durable Improvements for Low Dose BB-301 Treatment at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference
- Oculopharyngeal Muscular Dystrophy (OPMD) Patients treated with low dose BB-301 and high dose BB-301 experienced significant improvements in throat closure,
About this update from Benitec Biopharma Inc.
[{"type":"text","content":"- Oculopharyngeal Muscular Dystrophy (OPMD) Patients treated with low dose BB-301 and high dose BB-301 experienced significant improvements in throat closure, throat emptying, and total dysphagic symptom burden - OPMD Patients treated with low dose BB-301 experienced highly durable improvements, with clinical and radiographic improvements continuing to deepen two years post BB-301 treatment -The first OPMD Patient treated with high dose BB-301 experienced an extraordinarily robust dose-response at an early interim follow-up time-point, indicating the continued potential for BB-301 to achieve disease-modifying outcomes for OPMD patients with dysphagia - BB-301 is the only clinical-stage therapeutic in development designed to treat dysphagia in patients with OPMD HAYWARD, Calif., March 09, 2026 (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 promising interim clinical results from the BB-301 Phase 1b/2a first-in-human study (NCT06185673) evaluating low dose and high dose BB-301 treatment for Oculopharyngeal Muscular Dystrophy (OPMD) with moderate dysphagia. Interim and long-term clinical results for patients enrolled into Cohort 1 (low dose BB-301 ), and interim clinical results for the first patient enrolled into Cohort 2 (high dose BB-301) 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 medic...