Benitec Biopharma Reports Positive Interim Results For BB-301 Gene Therapy In OPMD Clinical Trial

(RTTNews) - Benitec Biopharma Inc. (BNTC), a clinical-stage biotechnology company, Wednesday announced sustained improvements in swallowing function and reduced dysphagic symptoms following the administration of a low dose of BB-301 in three participants of its Phase 1b/2a clinical trial - NCT06185673 for Oculopharyngeal Muscular Dystrophy or OPMD.

The interim results, presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference in Dallas, Texas, highlight the potential efficacy of BB-301.

The study assessed swallowing efficiency and effectiveness using videofluoroscopic swallowing studies or VFSS and the Sydney Swallow Questionnaire or SSQ. Findings showed clinically meaningful improvements in all three participants, with two achieving swallowing function within normal clinical parameters. These results were compared to pre-treatment data gathered from the Benitec-sponsored OPMD Natural History Study.

Benitec's CEO, Dr. Jerel A. Banks, expressed gratitude to the participants and their families, emphasizing optimism for continued benefits as the trial progresses. The sixth and final participant in Cohort 1 is set to receive BB-301 in the second quarter of 2025, with enrollment for the next, higher-dose phase expected later in the year.

OPMD is a rare, late-onset degenerative muscle disorder primarily affecting swallowing. With no existing drug therapies, treatment options are currently limited to surgical and dietary interventions. BB-301, an investigational gene therapy, aims to address the underlying cause of the disease.

The study's methodology involved blinded assessments to ensure objective evaluation of VFSS and SSQ results. Preliminary findings demonstrated significant reductions in dysphagic symptoms, with Subject 1 showing a 41 percent reduction in symptom burden, Subject 2 achieving a 91 percent reduction, and Subject 3 experiencing a 68 percent reduction within three months post-treatment.

With five participants having safely received the low-dose BB-301, the trial continues to advance, marking a potential breakthrough in gene therapy for OPMD.

BNTC is currently trading at $15.26 or 1.71% lower on the Nasdaq Capital Market.