Benitec's BB-301 Gene Therapy Shows Swallowing Gains in All Nine OPMD Patients

Benitec Biopharma Inc. (BNTC) said Wednesday interim Phase 1b/2a results showed swallowing improvements in all nine patients receiving BB-301, an investigational gene therapy for oculopharyngeal muscular dystrophy, a rare inherited disorder with no approved therapies. All nine patients met formal response criteria. At month three, throat-emptying response rates were 100% in the high-dose group versus 67% in the low dose, while timed water-drinking response rates were 67% versus 17%. Both groups recorded 100% response rates on the Sydney Swallow Questionnaire and 67% for throat closing. The open-label, dose-escalation trial covered six low-dose patients followed for 12 months and three high-dose patients with three to nine months of follow-up. Low-dose improvements remained durable and no serious adverse events occurred, the company said. Findings were presented at the European Society of Gene and Cell Therapy Congress. Benitec plans a pivotal study in mid-2027. BNTC traded down 0.69% at $8.62.