Benitec Biopharma said on October 28, 2026, that all nine patients treated in its early-stage BB-301 trial had responded to the experimental gene therapy for swallowing difficulties associated with oculopharyngeal muscular dystrophy, or OPMD.
Key Takeaways
- The interim analysis covered six patients given a low dose and three given a high dose.
- A larger proportion of high-dose patients showed improvements on two swallowing measures at three months.
- Benitec reported no serious adverse events among treated patients.
BB-301 trial results by dose
Benitec said all six low-dose patients and all three high-dose patients met its criteria as formal responders. The company reported 12-month follow-up results for every patient in the low-dose group. In the high-dose group, one patient had nine months of follow-up and two had three months.
At three months, a larger proportion of high-dose patients met the criteria for improvement in throat emptying and in a timed drinking test. Both dose groups had the same response rates on a swallowing questionnaire and a measure of throat closing ability.
The findings are interim results from a first-in-human, open-label Phase 1b/2a study. The small groups and differing lengths of follow-up limit what can be concluded about the treatment’s longer-term effects.
| Item | Details |
|---|---|
| Throat emptying at three months | 67% of low-dose patients and 100% of high-dose patients met the improvement criteria. |
| Timed drinking test at three months | 17% of low-dose patients and 67% of high-dose patients met the improvement criteria. |
Treatment and access
OPMD is a progressive muscle disorder that can cause severe swallowing difficulties. Benitec said there are no approved therapies for the condition.
BB-301 is designed to suppress a faulty gene and provide a functional replacement. It is administered to throat muscles in a one-time procedure. Under Benitec’s expanded access policy, the investigational treatment is currently available only through participation in its clinical trials.
Next milestone
Benitec plans to advance toward a pivotal BB-301 study in mid-2027.
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