BB-301 Demonstrates Sustained Therapeutic Benefits in Extended Clinical Assessment for Oculopharyngeal Muscular Dystrophy

Benitec Biopharma Inc. (BNTC) has revealed compelling extended follow-up data from its Phase 1b/2a trial of BB-301, a gene therapy candidate targeting dysphagia symptoms associated with oculopharyngeal muscular dystrophy (OPMD). The findings underscore the treatment’s lasting impact on swallowing function and patient quality of life.

Extended Durability Confirmed in Lead Patient Cohort

A landmark 24-month assessment of the first patient enrolled in Cohort 1 reveals sustained therapeutic benefit beyond the initial treatment period. The patient exhibited progressive amelioration of post-swallow pharyngeal residue—a key indicator of swallowing difficulty—coupled with measurable reduction in overall dysphagic symptom burden. Notably, these improvements persisted and even deepened when compared to both baseline pre-treatment status and the 12-month checkpoint, validated through objective x-ray-based swallowing evaluations and patient-reported outcomes on the Sydney Swallow Questionnaire (SSQ).

Robust Response Rates Across Patient Population

Building on this single-patient milestone, the complete Cohort 1 data set—encompassing four enrolled patients—achieved full response status at the 12-month statistical assessment period. Each participant met Benitec’s pre-defined efficacy criteria, which mandate demonstrable improvement across at least two of five standardized swallowing assessment categories. This consistent responder classification across the entire early cohort suggests BB-301 delivers reproducible clinical benefit rather than isolated cases of exceptional response.

Additionally, a separate patient assessment at the 12-month interval reaffirmed potent disease-modifying activity, reinforcing the durability signal evident in the broader Cohort 1 population.

Market Context

BNTC stock activity on January 9 reflected modest pressure, closing at $11.84 per share, representing a $0.34 decrease or 2.79% decline from the previous session.