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.
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