RegulatoryCompany release

Benitec reports interim BB-301 data in OPMD dysphagia, with no serious adverse events

🇺🇸 Benitec Biopharma Inc. (BNTC) · Wed 28 Oct 2026

All nine patients in the first-in-human Phase 1b/2a study are formal responders, and the high-dose cohort showed larger three-month gains in swallowing measures.

Benitec Biopharma announced on 28 October 2026 interim results from its ongoing first-in-human Phase 1b/2a study of BB-301, a gene therapy for patients with oculopharyngeal muscular dystrophy (OPMD) who have swallowing difficulty (dysphagia).

According to the company, all nine patients across the low-dose and high-dose cohorts are formal responders. Patients in the high-dose cohort showed larger gains at three months in throat emptying and drinking capacity than those in the low-dose cohort.

Benitec said no serious adverse events have been reported to date. The data were presented as a late-breaking poster at the ESGCT congress. The figures are interim and come from a small, early-phase study.

BB-301 holds Orphan Drug and Fast Track designations from the US Food and Drug Administration and Orphan designation from the European Medicines Agency. The company plans to begin a pivotal study in mid-2027.

For context, Fast Track is a US programme intended to speed the development and review of products for serious conditions with unmet medical need.

What it means for your team

  • The company has set mid-2027 as the planned start of a pivotal study, so regulatory teams tracking OPMD should watch for details on trial design and endpoints.
  • The safety picture is limited to nine patients in an early study; pharmacovigilance teams should treat the absence of serious adverse events as preliminary.
  • The higher-dose cohort showed larger three-month improvements, which may inform dose selection discussions as the programme advances.
  • Existing FDA Orphan and Fast Track designations and EMA Orphan designation are relevant to future development and market-access planning in both regions.

Company's press release (SEC filing): Benitec Biopharma Inc. ↗

Article written by VigiEye from the official source (Benitec Biopharma Inc.). Claims are the company's own. AI-assisted; check the source before acting.

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