Stoke Therapeutics at Cantor conference: dravet data, cash runway

Stoke Therapeutics (STOK) presented data on STK-002, an experimental treatment for Autosomal Dominant Optic Atrophy (ADOA), at the American Academy of Ophthalmology 2026 Annual Meeting. The Phase 1 OSPREY study completed dosing in the first two cohorts, with initial safety and efficacy data expected in 2027. STK-002 targets the OPA1 gene, which causes 65-90% of ADOA cases. The study aims to assess safety, tolerability, and visual function improvements.

Stoke Therapeutics (STOK) met with the FDA to align on its NDA submission for zorevunersen, a treatment for Dravet syndrome. The FDA provided feedback on data requirements and timing. Stoke plans to submit data in H1 2027 and complete the NDA submission in H2 2027. The Phase 3 EMPEROR study is ongoing, with enrollment completed in multiple regions. Stoke collaborates with Biogen (BIIB) for development and commercialization.

Stoke Therapeutics (STOK) completed a meeting with the FDA to align on the planned U.S. NDA for zorevunersen, an investigational treatment for Dravet syndrome. The Phase 3 EMPEROR study will assess changes in cognition and behavior using four Vineland-3 subdomains. Data submissions are expected in the first half of 2027, with a Phase 3 readout anticipated in Q3 2027. The company plans to complete the NDA submission in the second half of 2027.

Stoke Therapeutics (STOK) aligned with the FDA on its U.S. NDA plan for zorevunersen, including a multicomponent Vineland-3 analysis. The company aims to complete the NDA in H2 2027, following a Phase 3 readout expected in Q3 2027. The FDA meeting was a mid-cycle regulatory de-risking step for Stoke's lead program.

Stoke Therapeutics (STOK) appointed Bo Cumbo to its board, replacing Edward Kaye, who retired. Cumbo has extensive biopharma experience. Stoke is developing zorevunersen for Dravet syndrome, with potential FDA approval in 2028. It partners with Biogen (BIIB) for global commercialization, retaining U.S., Canada, and Mexico rights.

Stoke Therapeutics (STOK) announced key milestones, including a phase III readout for Dravet syndrome within 12 months and progress in ADOA trials. Zorevunersen showed sustained 75% seizure reduction and functional gains, with long-term efficacy supported by OLE data. The EMPEROR study is well-powered, and the company's commercial strategy leverages robust clinical evidence.