Benitec Reports Positive Phase 1b/2a Results For BB-301 In Oculopharyngeal Muscular Dystrophy

(RTTNews) - Benitec Biopharma Inc. (BNTC) announced continued durable improvements in swallowing following administration of the low-dose of BB-301 in the study's first two subjects treated in the BB-301 Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study in Oculopharyngeal Muscular Dystrophy or OPMD.

According to the company, two Subjects received the lowest dose of gene therapy BB-301 (1.2e13 vg/subject), with no significant adverse events reported. At baseline, Subject 1 exhibited more severe dysphagic symptoms compared to Subject 2, as assessed by pre-dose SSQ and TPR results. However, both subjects experienced significant clinical benefits, as indicated by their post-dose SSQ scores and TPR results.

The SSQ Total Scores and Sub-Scores demonstrated a strong correlation with the VFSS TPR results. Subject 1 showed clinically meaningful improvements in the post-dose SSQ Total Score and Sub-Scores by Day 270, which were driven by corresponding reductions in VFSS TPR values. Meanwhile, Subject 2 achieved clinically meaningful improvements in the post-dose SSQ Total Score and Sub-Scores by Day 180, reaching a score indicative of a normal swallowing profile, primarily due to a reduction in the frequency of pathologic low-volume sequential swallows.

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