Benitec Biopharma’s BB-301 shows efficacy in Phase 1b/2a study of OPMD

Benitec Biopharma announced continued durable improvements in the radiographic assessments of swallowing efficiency and the subject-reported outcome instrument as assessed at the 180-day timepoint following the administration of the low-dose of BB-301 to the study’s first subject treated in the BB-301 Phase 1b/2a study in Oculopharyngeal Muscular Dystrophy, or OPMD. BB-301 has been granted Orphan Drug designation by the FDA and EMA Committee for Orphan Medicinal Products. OPMD is a rare progressive muscle-wasting disease for which there is currently no effective drug therapy. The disease is characterized by swallowing difficulties, limb weakness and eyelid drooping. Benitec plans to provide additional analyses of the interim clinical study data at upcoming medical conferences and company-sponsored Research and Development-focused webcasts in 1Q25. The company is poised to dose the third subject in Q3 and remains on track to report additional interim clinical study results in 1Q25. At the 90-day post-dose assessment following the administration of the low-dose of BB-301, Subject 1 demonstrated improvements in key videofluoroscopic swallowing study assessments which correlated with the observation of similar levels of improvement in the Sydney Swallow Questionnaire vs. the pre-dose average values recorded for Subject 1 during the OPMD Natural History Study, indicating an improvement in swallowing function as reported by Subject 1. The post-dose average values for TPR remained meaningfully reduced. Regarding the BB-301 safety profile observed to date, acid reflux or heartburn has been reported previously in April 2024 at the time of the Research and Development Day webcast. No Serious Adverse Events have been observed for the two subjects that have received the low-dose of BB-301.