Benitec Biopharma Provides Positive Long-Term Clinical Study Results for BB-301 Phase 1b/2a Clinical Trial Demonstrating Robust Efficacy and Continued Durability of Response

Core Insights - Benitec Biopharma Inc. announced positive results from the 24-month follow-up of the first patient treated with BB-301 in a clinical study for oculopharyngeal muscular dystrophy (OPMD), showing robust disease-modifying outcomes [1][3][4] Group 1: Clinical Study Results - Patient 1 demonstrated deepening improvements in post-swallow pharyngeal residue and total dysphagic symptom burden at the 24-month follow-up compared to both pre-treatment and 12-month follow-up assessments [1][3][4] - All four patients in Cohort 1 completed the 12-month follow-up, with each showing durable responses to BB-301, meeting pre-specified statistical criteria for improvement [1][4][8] - Patient 1 exhibited a 27% improvement in Pharyngeal Area at Maximum Constriction (PhAMPC) at the 12-month mark, which was maintained at the 24-month follow-up [5] - Patient 1 showed a 35% reduction in post-swallow residue in the vallecular region at 12 months, improving to a 60% reduction at 24 months [7] - Total Pharyngeal Residue (TPR) improved by 32% at 12 months and 39% at 24 months, indicating enhanced swallowing efficiency over time [7] Group 2: Patient-Reported Outcomes - The Sydney Swallow Questionnaire (SSQ) total score indicated a 64% reduction in total dysphagic symptom burden at 12 months, improving to a 78% reduction at 24 months [7] - The Responder Analysis framework was utilized to evaluate the efficacy of BB-301, with all four patients classified as formal responders based on their improvements across multiple assessment categories [6][8] Group 3: Company Overview and Future Plans - Benitec Biopharma is focused on developing novel genetic medicines using its proprietary "Silence and Replace" platform, targeting chronic and life-threatening conditions like OPMD [10][11] - The company plans to engage with the U.S. Food and Drug Administration (FDA) in mid-2026 to confirm the pivotal study design for BB-301 and will present interim clinical results at future medical conferences [2]