Benitec Biopharma Inc. (NASDAQ:BNTC) shares soared 20% on Monday after the biotech company announced positive interim results from its ongoing Phase 1b/2a clinical trial of BB-301 and confirmed that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the therapy.
The clinical-stage gene therapy company reported that all six patients in Cohort 1 of the study met formal statistical criteria for response, resulting in a 100% response rate. BB-301 is designed to treat Oculopharyngeal Muscular Dystrophy (OPMD) with dysphagia, a severe swallowing disorder that affects approximately 97% of OPMD patients.
Following treatment, patients showed notable improvements across several clinical measures, including reduced symptom burden, lower post-swallow residue accumulation, shorter liquid consumption times, and improved pharyngeal closure during swallowing.
“Progressive dysphagia is a severe, life-threatening complication of OPMD which impacts 97% of OPMD patients, often leading to serious health risks, such as chronic choking, malnutrition, aspiration pneumonia, and death. We are excited by the profound effect that BB-301 can potentially have on this progressive disease as demonstrated by the interim clinical trial results for Cohort 1, where 100% of patients were responders,” said Jerel A. Banks, Executive Chairman and CEO of Benitec Biopharma.
The FDA’s Fast Track designation follows the agency’s review of the interim clinical data. BB-301 had already received Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA).
Looking ahead, Benitec plans to meet with the FDA in 2026 to finalize the pivotal study design for BB-301. The company also confirmed that the first patient in Cohort 2 was successfully dosed with BB-301 during the fourth quarter of 2025, marking continued momentum for the program.