Four patients enrolled in the BB-301 Phase 1b/2a treatment study completed the 12-month follow-up period, with all showing a positive response to BB-301. Patient 1 continued to show improved symptoms at the 24-month mark. An update on Cohort 2 is expected in mid-2026, with an FDA meeting for pivotal study design planned mid-year.

Benitec Biopharma announced financial results for the second fiscal quarter, highlighting the safety and efficacy of BB-301. They plan to engage with the FDA in mid-2026 to confirm the pivotal study design. Key milestones for BB-301 development were outlined, including the Responder Analysis for Study Completers.

BB-301 is a genetic medicine under development that utilizes a silence and replace mechanism to address Oculopharyngeal Muscular Dystrophy-related Dysphagia. Benitec Biopharma is focused on advancing genetic medicines using their proprietary ddRNAi platform. Financial results showed total expenses of $13.4 million, with significant progress in research and development.

The Responder Analysis for Study Completers showed that all four Cohort 1 patients were responders to BB-301, demonstrating a durable response. Patient 1 of Cohort 1 continued to show improvements at the 24-month follow-up. Enrollment for the BB-301 study is ongoing, with updates on Cohort 2 expected in mid-2026.

Benitec Biopharma is a clinical-stage biotechnology company developing genetic medicines. BB-301 offers a potential one-time treatment for genetic conditions. Financially, the company reported total expenses of $13.4 million for the quarter ended December 31, 2025. They aim to engage with the FDA to finalize study design in mid-2026.

Read more at GlobeNewswire: Benitec Biopharma Releases Second Quarter 2026 Financial