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Investing.com -- Benitec Biopharma Inc. (NASDAQ:BNTC) stock rose 20% on Monday after the company announced positive interim clinical results for its BB-301 gene therapy treatment and received Fast Track designation from the FDA.
The clinical-stage biotechnology company reported that all six patients enrolled in Cohort 1 of its Phase 1b/2a clinical trial for BB-301 met formal statistical criteria for response, representing a 100% response rate. BB-301 is being developed to treat Oculopharyngeal Muscular Dystrophy (OPMD) with dysphagia, a severe swallowing disorder that affects 97% of OPMD patients.
Following the administration of BB-301, patients demonstrated significant improvements across multiple clinical measures, including reduced dysphagic symptom burden, decreased post-swallow residue accumulation, shorter time required to consume liquids, and improved pharyngeal closure during swallowing.
"Progressive dysphagia is a severe, life-threatening complication of OPMD which impacts 97% of OPMD patients, often leading to serious health risks, such as chronic choking, malnutrition, aspiration pneumonia, and death. We are excited by the profound effect that BB-301 can potentially have on this progressive disease as demonstrated by the interim clinical trial results for Cohort 1, where 100% of patients were responders," said Jerel A. Banks, Executive Chairman and CEO of Benitec Biopharma.
The FDA’s Fast Track designation follows the agency’s review of the interim data. BB-301 has also previously received Orphan Drug Designation from both the FDA and European Medical Association.
Benitec plans to meet with the FDA in 2026 to confirm the pivotal study design for BB-301. The company also reported that the first patient in Cohort 2 was successfully treated with BB-301 in the fourth quarter of 2025.
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