Benitec Reports Durable 24-Month BB-301 Benefits In Phase 1b/2a OPMD Dysphagia Study

Benitec Biopharma has reported encouraging long-term data from its BB-301 Phase 1b/2a trial in patients with oculopharyngeal muscular dystrophy (OPMD), a rare genetic disorder characterized by progressive swallowing difficulties. The first patient in Cohort 1 has now completed a 24-month follow-up after treatment, demonstrating sustained and deepening clinical benefits, including improved throat function and reduced swallowing difficulty compared to both pre-treatment and 12-month results.

X-ray–based swallowing studies confirmed continued improvements in pharyngeal muscle performance and in the amount of food or liquid left behind after swallowing. Measures such as throat closure (PhAMPC), post-swallow residue in the valleculae (NRRSv), and total pharyngeal residue (TPR) all showed durable and, in some cases, increasing benefit over time, indicating that BB-301 may be modifying the underlying disease process rather than providing only short-term symptom relief.

“Progressive dysphagia is the most severe, life-threatening complication of OPMD, and we are extremely excited to observe safe, durable, disease-modifying outcomes for the patients treated with BB-301,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec Biopharma Inc. “Durable improvements in the dysphagic symptom burden can have a profound impact on the lives of patients living with OPMD, and we remain singularly focused on advancing BB-301 through development to improve the lives of all OPMD patients. 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. We extend our deepest gratitude to the patients and families participating in the clinical study and to the investigators and clinical teams for their dedication to advancing new treatment options.”