Benitec Biopharma reported positive interim results from its early-stage study of BB-301, an investigational treatment for swallowing problems caused by oculopharyngeal muscular dystrophy (OPMD). The update includes follow-up on six patients given a low dose and initial results for three given a higher dose.
All nine patients met the study’s criteria for being formal responders. At the three-month assessment, a higher proportion of patients in the high-dose group showed improvement on two swallowing measures, though the study is small and the high-dose follow-up remains limited.
Key details
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Study size and follow-up: The update covers all six patients in the low-dose group, who have reached 12 months after treatment, and all three in the high-dose group. The first high-dose patient has follow-up through nine months; the other two have reached three months.
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Response across both groups: All six low-dose patients and all three high-dose patients were classified as formal responders. This overall response designation does not mean every patient improved on every measure.
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Three-month results favored the high dose on two measures:
- For throat emptying, measured by residue remaining after swallowing, 100% of high-dose patients met the response criteria, compared with 67% of low-dose patients.
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For functional swallowing capacity, measured by a timed cold-water drinking test, the figures were 67% for the high-dose group and 17% for the low-dose group.
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Other three-month measures were similar between groups: All patients in both groups met the response criteria for improvement in total score on the Sydney Swallow Questionnaire. For throat-closing ability, 67% in each group met the criteria.
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Safety: Benitec reported no serious adverse events among patients treated with BB-301 to date.
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Next development milestone: Chief Executive Jerel A. Banks said the company is advancing toward a pivotal study in mid-2027.
What the results mean
The update provides an early signal that BB-301 may improve swallowing function, including on some measures where the high-dose group had a larger share of responders at three months. But the high-dose results come from only three patients, and follow-up for two of them is still just three months. The reported 12-month follow-up applies to the low-dose group; the release does not provide detailed 12-month response rates for individual measures.
OPMD is a progressive disorder, and the company says there are no approved therapies for it. The results support continued development of BB-301, but they are interim findings from a nine-patient, early-stage study rather than evidence from a pivotal trial.