Benitec Biopharma reports first patient completed 24-month BB-301 follow-up

Benitec Biopharma announced that the first patient in Cohort 1 of the BB-301 Phase 1b/2a study completed a 24-month post-treatment assessment with continued improvements in swallowing measures. The company also reported that the first four Cohort 1 patients completed the 12-month statistical follow-up and met its pre-specified responder criteria.

Benitec Biopharma announced that the first patient treated in Cohort 1 of the BB-301 Phase 1b/2a clinical study completed a 24-month post-treatment assessment. The study is evaluating BB-301 for treatment of dysphagia in oculopharyngeal muscular dystrophy (OPMD). At 24 months the company reported continued, disease-modifying outcomes for Patient 1, with improvements noted on clinical swallowing assessments. The first four patients in Cohort 1 have completed the 12-month statistical follow-up period and were reported as meeting the trial's pre-specified response criteria. Key results: - Patient 1 completed the 24-month post-treatment assessment and was reported to show continued, disease-modifying outcomes. - X-ray-based swallowing studies showed deepening improvements in post-swallow pharyngeal residue for Patient 1 versus pre-treatment and the 12-month follow-up. - Patient 1 also showed deepening improvement in total dysphagic symptom burden as measured by the Sydney Swallow Questionnaire (SSQ). - The first four Cohort 1 patients completed the 12-month statistical follow-up period and were reported to demonstrate a durable response to BB-301. - All four Cohort 1 completers met the company's pre-specified statistical criteria for response, defined as improvement across two or more of five assessment categories in the Responder Analysis. Summary: Benitec reported sustained functional and symptom improvements for Patient 1 at 24 months and consistent responder outcomes among the first four Cohort 1 patients at 12 months. The company states that all four completers met its pre-specified responder criteria. Undetermined at this time.