Benitec Biopharma receives FDA clearance of IND for BB-301

Benitec Biopharma announced the U.S. Food and Drug Administration has cleared its Investigational New Drug application for BB-301, its silence and replace gene therapy for the treatment of Oculopharyngeal Muscular Dystrophy-related Dysphagia. Subjects from Benitec’s ongoing NH study will be eligible for rollover onto the Phase 1b/2a clinical study of BB-301 for the treatment of OPMD-related Dysphagia after 6 months of baseline data collection. Following a 1-day dosing procedure for BB-301, each study subject will be evaluated for the same radiographic and clinical outcome measures as were evaluated during the NH study, including quantitative radiographic swallowing studies to facilitate objective assessments of swallowing safety, swallowing efficiency, and functional performance of the pharyngeal muscles underlying the OPMD-related Dysphagia. Currently, there are 13 subjects enrolled into the NH study, with each subject having the potential to rollover onto the Phase 1b/2a clinical dosing study for BB-301. Interim safety and efficacy data is expected to become available from the BB-301 Phase 1b/2a study approximately every 90 days following the dosing of each subject.