Benitec Biopharma Inc. (BNTC) on Monday, announced encouraging interim results from its Phase 1b/2a clinical study evaluating BB-301, a gene therapy for Oculopharyngeal Muscular Dystrophy.
The early-stage trial reported a 100 percent response rate among treated patients, with improvements in swallowing function and dysphagia symptoms.
Initial results also showed stronger clinical improvements in the first patient receiving the high-dose treatment compared with the low-dose cohort. No treatment-related serious adverse events were reported.
High-dose BB-301 reduced dysphagia symptom burden by about 68%, compared with roughly 7 percent with the low dose. Throat closure improved by about 19 percent versus 8 percent with low dose, while throat emptying improved by 44 percent compared with a 6 percent worsening in the low-dose group. Residue in the vallecular region improved by about 57 percent with high dose versus 3 percent with low dose.
BB-301 uses the company's DNA-directed RNA interference platform to silence the mutated gene responsible for OPMD while delivering a functional replacement. The findings were presented at the Muscular Dystrophy Association Clinical and Scientific Conference 2026.
BNTC is currently trading at $12.77 up $1.43 or 12.65 percent on the Nasdaq.
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