Stoke Therapeutics, Inc.STOK

Stoke Therapeutics, Inc. (STOK) is focused on neurology. The company's drug zorevunersen is in Phase 3 development. The Phase 3 EMPEROR Study (NCT06872125) for zorevunersen is a global, double-blind, sham-controlled study. The primary analysis population for the EMPEROR Study includes 162 patients from the U.S., U.K., and Japan. An additional cohort of approximately 30 patients is being enrolled in Europe (Germany, France, Spain, and Italy). Enrollment for the EMPEROR study (Zorevunersen) was completed in 10 months, with 162 patients enrolled from the U.S., U.K., and Japan. Approximately 50 patients have completed 28 weeks of treatment in the EMPEROR study (Zorevunersen) in the U.S., UK, and Japan. The primary endpoint for the Phase 3 EMPEROR Study (NCT06872125) is measured at Week 28, and the secondary endpoint is measured at Week 52. Zorevunersen has been granted Orphan Drug Designation, Rare Pediatric Disease Designation, and Breakthrough Therapy Designation. The company anticipates a pre-NDA meeting for zorevunersen in the U.S. in H2 2026, with rolling U.S. NDA submission initiation in Q1 2027 and completion in H2 2027. Data readout for the EMPEROR study (zorevunersen) is anticipated in Q3 2027.

Stoke Therapeutics is also developing STK-002, which is being studied in the OSPRAY study. The first cohort of the OSPRAY study (STK-002) included 3 patients, with no serious or severe safety events reported. Initial data for the OSPRAY study (STK-002) is anticipated in H1 2027, with safety and efficacy results anticipated in H1 2027. The dosing of the second cohort for the OSPRAY study (STK-002) is expected in August 2026. The incidence of ADOA is 1 in 10,000 people in Denmark and 1 in 30,000 people globally. OPA1 variants cause 65% to 90% of ADOA cases, with 400 OPA1 variants causing ADOA.