Benitec Biopharma Inc. announced the enrollment of the first oculopharyngeal muscular dystrophy (OPMD) patient into the OPMD natural history phase of the BB-301 clinical development program. The OPMD Natural History (NH) Study represents the 6-month pre-treatment observation period for each OPMD subject prior to the administration of BB-301 for the treatment of OPMD-related dysphagia. Upon the completion of 6-months of radiographic and clinical assessments required for the NH Study, participants will be eligible for enrollment into the BB-301 Phase 1b/2a treatment study in which BB-301 will be administered. The OPMD NH Study will facilitate the characterization of OPMD patient disposition at baseline and assess subsequent rates of progression of dysphagia via the use of the following quantitative radiographic measures (i.e., videofluoroscopic swallowing studies or "VFSS"), with the VFSS outlined below collectively providing objective assessments of swallowing safety, swallowing efficiency, and the functional capacity of the specific pharyngeal constrictor muscles underlying the progression of dysphagia in OPMD patients: Total Phaharyngeal Residue %(C2-4)2, Pharyngeal Area at Maximum Constriction (PhAMPC), Dynamic Imagining Grade of Swallowing Toxicity Scale (DIGEST), Vallecular Residue %(C2-4)2, Pyriform Sinus Residue %(C2-4)2, and Other Pharyngeal Residue %(C2-4). Normalized Residue Ratio Scale (NRRSv, NRRSp). Pharyngeal Construction Ratio (PCR). The study will also employ clinical measures of global swallowing capacity and oropharyngeal dyspgia, along with two distinct patient-reported outcome instruments targeting the assessment of oropharyngeal dysphagia. Upon the achievement of 6-months of follow-up in the NH Study, participants will be eligible for enrollment into the
planned BB-301 Phase 1b/2a treatment study.