Benitec Biopharma Announces Positive Interim Phase 1b/2a Results for High Dose BB-301 and Continued Durable Improvements for Low Dose BB-301 Treatment at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference

Core Insights - Benitec Biopharma Inc. announced promising interim clinical results for BB-301, a treatment for Oculopharyngeal Muscular Dystrophy (OPMD) with moderate dysphagia, showing significant improvements in swallowing function [2][3] Clinical Study Results - Patients treated with low dose BB-301 showed durable improvements in throat closure, throat emptying, and total dysphagic symptom burden, with effects continuing to deepen two years post-treatment [1][11] - The first patient treated with high dose BB-301 exhibited a robust dose-response at an early follow-up, indicating potential for disease-modifying outcomes [1][6] - Interim results for low dose BB-301 showed a 100% response rate among study completers, while high dose BB-301 demonstrated significantly improved results across all assessments [8][10] Efficacy Assessments - Key efficacy parameters included the Sydney Swallow Questionnaire (SSQ), pharyngeal area at maximum constriction (PhAMPC), total pharyngeal residue (TPR), and normalized residue ratio scale-valleculae (NRRSv) [5] - Low dose BB-301 resulted in a ~7% reduction in total dysphagic symptom burden (SSQ) compared to a ~68% reduction with high dose BB-301 [10] - Improvements in throat closure (PhAMPC) were ~8% for low dose versus ~19% for high dose, and overall throat emptying (TPR) showed a ~6% worsening for low dose compared to a ~44% improvement for high dose [10] Background Information - OPMD is a rare degenerative muscle disorder affecting nearly 15,000 patients in North America, Europe, and Israel, with no currently approved therapies available [13] - BB-301 is a novel gene therapy designed to treat OPMD by silencing the expression of faulty mutant PABPN1 while providing a functional replacement [14]