Benitec says BB-301 pivotal trial stays on track for mid-2027 start after FDA meeting

Benitec Biopharma Inc. said on October 5, 2026 that it received formal written feedback from the FDA following a Type C meeting held in the third quarter of 2026 regarding its planned pivotal study of BB-301, an investigational gene therapy for Oculopharyngeal Muscular Dystrophy (OPMD)-related dysphagia.

The company and the FDA are continuing to discuss the design of a planned open-label, single-arm pivotal study, and the FDA has asked for more available data, including findings from Benitec's own OPMD Natural History Study.¹ The agency agreed to review that information on a timeline that would let Benitec lock in the final pivotal study design by the first quarter of 2027.¹

Benitec said the overall timeline for the pivotal study remains unchanged, with patient enrollment still expected to start in mid-2027.¹

Separately, the company plans to share updated clinical data from its ongoing Phase 1b/2a study of BB-301 at the European Society of Gene and Cell Therapy Annual Congress in Hamburg, Germany, running October 27 to 30, 2026. The late-breaking poster presentation will include 12-month follow-up results from all six patients in Cohort 1 and interim follow-up data from the three patients enrolled in Cohort 2.¹

BB-301 is designed as a single-administration therapy targeting the muscles involved in swallowing. It uses a modified AAV9 vector to deliver a bifunctional construct that both silences mutant PABPN1 (the gene linked to OPMD) and replaces it with a codon-optimized, functional version, using microRNA-based siRNAs.¹ The FDA has granted BB-301 both Orphan Drug and Fast Track designations, while the European Medicines Agency has granted it Orphan Drug Designation.¹ The therapy is currently being tested under trial registration NCT06185673.

OPMD currently has no approved treatments and affects close to 15,000 patients across North America, Europe, and Israel, according to the company.¹