Benitec Biopharma Reports Positive Interim Clinical Results for Three Subjects Treated with BB-301 in Phase 1b/2a Study to be Presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference

Core Insights - Benitec Biopharma Inc. has reported durable and clinically significant improvements in swallowing function for subjects treated with BB-301, a gene therapy for Oculopharyngeal Muscular Dystrophy (OPMD) [1][3][19] - The interim clinical study results will be presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference, highlighting the efficacy of BB-301 in reducing dysphagic symptom burden [1][2] Summary of Clinical Results - Subject 1 showed a 37% reduction in Total Pharyngeal Residue for Thin Liquid, an 18% reduction for Solid Food, and a 29% reduction for Thick Liquids, with a 41% reduction in total dysphagic symptom burden 12 months post-treatment [8][9] - Subject 2 experienced a significant reduction in pathologic sequential swallows, achieving a clinically normal swallowing profile with a 91% reduction in total dysphagic symptom burden, resulting in an SSQ score of 68 units [10][12] - Subject 3 achieved complete resolution of pathologic sequential swallows and a 68% reduction in total dysphagic symptom burden, with an SSQ score of 70 units indicative of a clinically normal swallowing profile at 3 months post-treatment [13][15] Study Design and Methodology - The study is a Phase 1b/2a, open-label, dose escalation trial assessing the safety and clinical activity of BB-301 in OPMD patients [3][19] - Key assessment methods include videofluoroscopic swallowing study (VFSS) for swallowing efficiency and effectiveness, and the Sydney Swallow Questionnaire (SSQ) for patient-reported outcomes [2][5] Company Overview - Benitec Biopharma Inc. is focused on developing novel genetic medicines using its proprietary "Silence and Replace" DNA-directed RNA interference platform, targeting chronic and life-threatening conditions like OPMD [19]