Stoke Therapeutics, Inc. (STOK) Aligns With FDA on U.S. NDA Plan for Zorevunersen

Stoke Therapeutics (STOK) discussed Q3 earnings, highlighting plans for zorevunersen's NDA submission in 2027. The FDA supports a multicomponent endpoint for cognition/behavior. Stoke estimates 38,000 Dravet syndrome patients in key markets. STK-002 for ADOA is in Phase I trials. Q3 net loss was $73.6M, with $358.8M cash. Stoke expects funding into early 2028.

Stoke Therapeutics (STOK) reported Q3 2026 progress, highlighting zero dropouts in its EMPEROR Phase 3 trial for zorevunersen in Dravet syndrome. The company beat revenue estimates but reported a wider-than-expected loss. Stoke has $358.8M in cash, enough to fund operations into early 2028. The trial enrolled 162 patients, exceeding the target, with strong patient retention and statistical significance in endpoints. The FDA has accepted the study's multicomponent approach for regulatory filing.

Stoke Therapeutics, Inc. (STOK) filed an SEC Form 8-K — Results of Operations and Financial Condition. EX 99.1 Stoke Therapeutics Announces Third Quarter 2026 Financial Results and Provides Business Updates –Phase 3 EMPEROR study of zorevunersen, an investigational medicine for Dravet syndrome: ~110 patients through Week 28 primary endpoint, ~20 patients through Week 52 and all co

Stoke Therapeutics reported progress in its Phase 3 EMPEROR study for zorevunersen, an investigational drug for Dravet syndrome, with 110 patients reaching the Week 28 primary endpoint. The company plans to submit data to the FDA in the first half of 2027 and complete the NDA submission in the second half. Additionally, the Phase 1 OSPREY study for STK-002, targeting ADOA, is ongoing with initial data expected in H1 2027.

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.