BB-301 Hits 100% Response Rate Across Both Dose Cohorts in OPMD Study
BNTC is trading near its 52-week low of $8 (10% above the low).
Summary
Benitec reported positive interim Phase 1b/2a data for BB-301 in OPMD-related dysphagia: all nine patients treated across both dose cohorts are formal responders, and no serious adverse events have occurred in any patient to date. The high dose looks better on the harder endpoints at month 3 — 100% of Cohort 2 patients met the response criteria for improved throat emptying versus 67% in Cohort 1, and 67% versus 17% on the Cold-Water Timed Drinking Test. Cohort 1 now has 12-month follow-up for all six patients, showing the effect is durable, while Cohort 2 follow-up ranges from 3 to 9 months. This is the core de-risking event for the program ahead of the pivotal study the company says will start in mid-2027, and it follows the October 5 8-K disclosing FDA feedback requesting additional natural history work on the pivotal design. The company ended FY2026 with $180M in cash, so funding the pivotal trial is not the near-term constraint. Watch for the full pivotal study design and any further FDA interaction before the mid-2027 start.
At the time of this announcement, BNTC was trading at $8.80 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $302.8M. The 52-week trading range was $8.00 to $16.76. This news item was assessed with positive market sentiment and an importance score of 8 out of 10. Source: GlobeNewswire.