Benitec Biopharma Inc. (BNTC), Wednesday, announced positive interim results from its Phase 1b/2a clinical study of BB-301, an investigational gene therapy for Oculopharyngeal Muscular Dystrophy or OPMD.
The study, which is a single-arm, open-label, sequential dose-escalation trial, evaluated the safety and efficacy of BB-301 in three subjects with OPMD.
The interim results showed that all three subjects experienced clinically significant improvements in swallowing function and reductions in dysphagic symptom burden.
Subject 1 demonstrated a 41 percent reduction in dysphagic symptoms at 12 months post-treatment, with significant improvements in swallowing efficiency and effectiveness.
Subject 2 achieved a clinically normal swallowing profile after 12 months, with a 91 percent reduction in total dysphagic symptom burden.
Subject 3 showed significant improvements after 3 months, achieving a clinically normal swallowing profile and a 68% reduction in dysphagic symptoms.
The Phase 1b/2a study is ongoing, with the sixth and final subject of Cohort 1 expected to receive treatment in the second quarter 2025.
The therapy has shown a favorable safety profile, with no severe adverse events reported.
Benitec plans to continue enrolling additional subjects at higher doses later this year and anticipates further positive results.
If successful, BB-301 could provide a much-needed treatment option for OPMD, a rare disease characterized by progressive dysphagia with no effective drug therapies currently available.
Benitec is progressing with the development of BB-301 with hopes of addressing the underlying causes of OPMD.
Currently, BNTC is trading at $15.50 down by 0.13 percent on the Nasdaq.
For comments and feedback contact: editorial@rttnews.com
Business News
June 12, 2026 17:14 ET Major central bank action was the focus this week in economic news. The European Central Bank became the first major central bank to move in response to the rising inflationary pressures in the backdrop of the conflict in the Middle East. In North America, the U.S. inflation and trade data as well as Canada’s central bank decision gained attention. The Chinese trade data was the main news in Asia.