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Benitec Biopharma BB-301 Clinical Trial Results Show 100% Response Rate

Benitec Biopharma announced interim clinical results from the ongoing BB-301 Phase 1b/2a first-in-human study for Patients treated with high dose BB-301 and continued durable improvements for Patients treated with the low dose of BB-301. The clinical results observed across both dose cohorts of the BB-301 first-in-human study continue to demonstrate robust and durable improvements in swallowing function in dysphagic Patients with OPMD, with a 100% response rate observed across all Patients treated to date. The updated analyses include 12-month post-treatment follow-up results for all six Patients enrolled in Cohort 1 and the available post-treatment follow-up results for all three Patients enrolled in Cohort 2. Clinical and fluoroscopic results were available for the final 12-month post-treatment follow-up time-point of the study for all Cohort 1 Patients. Clinical and fluoroscopic results were available for the 9-month post-treatment follow-up time-point for the first Patient enrolled in Cohort 2 and for the 3-month post-treatment follow-up time-point for the second and third Patients enrolled in Cohort 2. For Cohort 1, all six Patients are formal Responders to BB-301. For Cohort 2, interim clinical results are available, and all three Patients are formal Responders to BB-301. Evaluations of dose-related improvements were carried out. Full Cohort comparisons were carried out at the 3-month post-treatment time-point, as complete clinical and fluoroscopic results were available for the total Patient populations of the respective cohorts at this post-treatment time-point. A larger proportion of Cohort 2 Patients exhibited improvements in throat emptying ability and functional swallowing capacity at Month 3 post-BB-301 administration. Overall, 67% of Cohort 1 Patients and 100% of Cohort 2 Patients met the statistical criteria for response for improved throat emptying ability at the 3-month post-treatment time-point. Overall,17% of Cohort 1 Patients and 67% of Cohort 2 Patients met the statistical criteria for response for improved functional swallowing capacity as assessed by Cold-Water Timed Drinking Test at the 3-month post-treatment time-point. Improvements in total Sydney Swallow Questionnaire score and throat closing ability for Cohort 1 and Cohort 2 Patients were equivalent at Month 3 post-BB-301 administration. All the patients in Cohort 1 and Cohort 2 met the statistical criteria for response for improved total SSQ score at the 3-month post-treatment time-point. Overall, 67% of Patients in Cohort 1 and Cohort 2 met the statistical criteria for response for improved throat closing ability at the three-month post-treatment time-point. There have been no Serious Adverse Events in any patient treated with BB-301 to date.

Benitec Receives Formal FDA Feedback, Plans to Start BB-301 Pivotal Study in Mid-2027

Benitec announced formal FDA feedback following a Type C meeting in Q3. Discussions on an open-label, single-arm pivotal study for BB-301 are ongoing. The FDA requested additional available data, including data from the Benitec-sponsored OPMD Natural History Study, and agreed to review that information on a timeline that would allow Benitec to confirm final pivotal study design in Q1 2027. Patient enrollment for the BB-301 pivotal study is planned to begin in mid-2027. Benitec will present interim Phase 1b/2a results as a late-breaking poster at the European Society of Gene and Cell Therapy Annual Congress in Hamburg on October 27 to 30, including 12-month follow-up for all six Cohort 1 patients and interim follow-up for all three Cohort 2 patients. "We greatly appreciate the collaboration from the FDA and are encouraged by the constructive nature of our discussions" said Jerel Banks, executive chairman and CEO of Benitec.

Benitec Biopharma Files $200M Mixed Securities Shelf

Benitec Biopharma files $200M mixed securities shelf