Benitec Biopharma reports interim trial data for subject treasted with BB-301

Benitec Biopharma announces positive interim clinical data from the 90-day timepoint following the administration of BB-301 to the study’s first subject treated in the BB-301 Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study in Oculopharyngeal Muscular Dystrophy. BB-301 has been granted Orphan Drug designation by the US Food and Drug Administration and European Medicines Agency Committee for Orphan Medicinal Products. BB-301 Interim Clinical Study Results: During the OPMD Natural History Study, which represents the pre-dose observational period for each subject, Subject 1 experienced progressive worsening of dysphagia as demonstrated by the results of the videofluoroscopic swallowing studies, the cold water timed drinking test, and the key subject-reported outcome measure. At the 90-day timepoint following the administration of BB-301, Subject 1 demonstrated improvements in key videofluoroscopic assessments which correlated with the observation of similar improvement in the key subject-reported outcome measure as compared to the average values for the respective assessments completed during the pre-dose observational period. The most significant VFSS improvements at Day 90 were observed for swallowing tasks centered on the evaluation of pharyngeal constrictor muscle function and swallowing efficiency in the context of the consumption of thin liquids, solid foods and thick, non-solid foods. The VFSS improvements correlated with an improvement in the key subject-reported outcome measure the Sydney Swallow Questionnaire, indicating an improvement in swallowing function as reported by Subject Regarding the BB-301 safety profile observed to date, no Serious Adverse Events have been observed for the two subjects that have received BB-301.