BB-301

May 14, 2026: Benitec Biopharma Inc. (NASDAQ: BNTC) reported fiscal 3Q results (quarter ended March 31, 2026) and provided BB-301 (gene therapy) program updates in an ongoing Phase 1b/2a study for oculopharyngeal muscular dystrophy (OPMD)-related dysphagia. A late-breaking abstract presented at the March 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference reported durable responses with low-dose BB-301 at 12 and 24 months post-treatment and further improvement in depth of response with high-dose BB-301 at 3 months post-treatment; both doses were described as disease-modifying. All six Cohort 1 patients completed 12-month follow-up with robust responses. Cohort 2 enrollment is ongoing; Patient 1 and Patient 2 were safely treated with high-dose BB-301, with no treatment-related severe adverse events observed.

Upcoming catalysts: Phase 1b/2a interim results were selected for oral presentation at the 2026 ASGCT Annual Meeting on May 15 (Boston). The company expects to engage the FDA “mid-year” to formalize the BB-301 pivotal study design and plans updated interim data in 2H 2026.

Financial: cash and cash equivalents of ~$184.8M as of March 31, 2026; quarterly net loss $11.9M ($(0.24)/share).