Benitec reports positive interim high-dose BB-301 results, durable low-dose benefit in OPMD trial

Benitec Biopharma Inc. announced on October 28, 2026 positive interim results from its Phase 1b/2a first-in-human study of BB-301 (NCT06185673) in patients with Oculopharyngeal Muscular Dystrophy (OPMD), reporting continued benefit in the low-dose cohort and new data from the high-dose cohort. Across both dose cohorts, the company said patients continue to show robust and durable improvements in swallowing function, with a 100% response rate observed in all patients treated to date.¹

The updated analysis covered 12-month post-treatment follow-up for all six Cohort 1 (low dose) patients and interim follow-up for all three Cohort 2 (high dose) patients, with fluoroscopic results available at 9 months for the first Cohort 2 patient and at 3 months for the second and third Cohort 2 patients.¹ All six Cohort 1 patients and all three Cohort 2 patients were classified as formal responders to BB-301.¹

At the three-month mark, a comparison between cohorts found a larger share of high-dose patients improved on measures of throat emptying and functional swallowing capacity. 67% of Cohort 1 patients and 100% of Cohort 2 patients met the statistical criteria for response on throat emptying ability, as assessed by videofluoroscopic evaluation of post-swallow food and liquid residue.¹ On the Cold-Water Timed Drinking Test, 17% of Cohort 1 patients and 67% of Cohort 2 patients met the response criteria for functional swallowing capacity.¹ Both cohorts showed 100% response on total Sydney Swallow Questionnaire score, while 67% of patients in each cohort met criteria for improved throat closing ability.¹

No serious adverse events have occurred in any patient treated with BB-301 to date.¹ The data were presented in a late-breaking poster at the European Society of Gene and Cell Therapy (ESGCT) Annual Congress. BB-301 holds Orphan Drug Designation from the European Medicines Agency as well as Orphan Drug and Fast Track Designations from the U.S. FDA.¹ Benitec's chief executive, Jerel A. Banks, said the company is working toward starting a pivotal study in mid-2027.