Benitec gets FDA Fast Track for BB-301 in OPMD; 100% response rate in Cohort 1

Benitec Biopharma Inc.

FDA granted Fast Track Designation to BB-301 for OPMD with dysphagia; BB-301 also has Orphan Drug Designation from FDA and EMA.
All six Cohort 1 patients met statistical response criteria (100% response rate) with significant improvements in dysphagia measures.
First Cohort 2 patient treated in Q4 2025; Benitec plans FDA meeting in 2026 to confirm pivotal study design.
Appointed Dr. Sharon Mates to board; she was co-founder/CEO of Intra-Cellular Therapies (acquired by JNJ for ~$14.6B in 2025).

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