Benitec Biopharma reported that all six patients treated in Cohort 1 of its Phase 1b/2a study of BB‑301 for oculopharyngeal muscular dystrophy (OPMD) met the formal statistical criteria for response, yielding a 100 % response rate. The study documented significant and sustained improvements in dysphagic symptom burden, post‑swallow residue, time to consume liquid volumes, and pharyngeal closure.
The U.S. Food and Drug Administration granted BB‑301 Fast Track designation, a status that can accelerate review and approval for drugs addressing serious conditions with unmet medical needs. BB‑301 also received Orphan Drug Designation from both the FDA and the European Medicines Agency, underscoring the rarity of OPMD and the lack of approved therapies.
The Phase 1b/2a trial is an open‑label, dose‑escalation study. In addition to the six patients in Cohort 1, the first patient in Cohort 2 was successfully treated in the fourth quarter of 2025. Earlier interim data from the first two subjects showed durable, clinically meaningful improvements in swallowing function, supporting the platform’s dual‑action mechanism of silencing mutant PABPN1 and replacing it with a wild‑type copy.
Benitec’s proprietary Silence and Replace DNA‑directed RNA interference platform underpins BB‑301’s therapeutic strategy. The company plans to meet with the FDA in 2026 to confirm the pivotal study design for BB‑301. Dr. Sharon Mates was appointed to Benitec’s Board of Directors on November 2, 2025, bringing significant leadership experience in biotechnology. A webcast detailing the six treated patients is scheduled for November 3 at 8:00 am EST.
Benitec Biopharma is headquartered in Hayward, California. The positive interim results and Fast Track designation position BB‑301 as a potential breakthrough treatment for OPMD and validate the company’s ddRNAi platform for future applications.
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.