Clinical TrialMay 14, 2026, 08:02 AM
Benitec Biopharma BB-301 Shows Durable Responses; $184.8M Cash
AI Summary
Benitec Biopharma announced positive interim clinical study results for BB-301, its gene therapy for OPMD-related dysphagia. The low dose demonstrated durable responses at 12 and 24 months, while the high dose showed further improvements at 3 months. The company is advancing Cohort 2 enrollment and plans to engage the FDA mid-year for pivotal study design. Benitec reported a Q3 2026 net loss of $11.9 million and held $184.8 million in cash as of March 31, 2026, positioning it to complete the pivotal study.
Key Highlights
- BB-301 low dose showed durable responses at 12 and 24 months post-treatment.
- High dose BB-301 demonstrated further improvements in depth of response at 3 months.
- BB-301 Phase 1b/2a interim results selected for oral presentation at ASGCT on May 15.
- Cohort 2 enrollment advanced with two patients safely treated with high dose BB-301.
- Company on track to engage FDA mid-year to formalize BB-301 pivotal study design.
- Cash and cash equivalents were $184.8 million as of March 31, 2026.
- Q3 2026 net loss was $11.9 million, or $(0.24) per basic and diluted share.
- Total expenses for Q3 2026 decreased to $13.6 million from $15.3 million YoY.