Stoke Therapeutics completes enrollment of 162-patient Phase 3 EMPEROR trial for zorevunersen

Stoke Therapeutics announced on June 30, 2026 that it had completed the enrollment of 162 patients into the Phase 3 EMPEROR study of zorevunersen.¹ Zorevunersen is being developed as a treatment for Dravet syndrome, and the company described it as a first-in-class potential disease-modifying treatment for the condition.¹

Stoke said it plans to initiate a rolling New Drug Application submission to the U.S. Food and Drug Administration in the first quarter of 2027.¹ The company added that a Phase 3 data readout is anticipated in the third quarter of 2027 to complete the rolling U.S. NDA submission in the second half of 2027.¹

On trial progress, Stoke reported that approximately 50 out of the 162 patients in the U.S., UK and Japan are through 28 weeks of treatment, the time point at which the primary endpoint of change in major motor seizure frequency is measured.¹ Separately, an additional cohort of approximately 30 patients in Europe is currently enrolling, and the last patient is expected to enroll in August.¹ The company said no patients have discontinued treatment in the study.¹

The trial, registered as NCT06872125, is a global, double-blind, sham-controlled study.¹ Its primary endpoint is change in major motor seizure frequency measured at Week 28, although the study will remain blinded and all data will be analyzed at the end of the 52-week treatment period given that secondary endpoints measuring cognition and behavior will be assessed at Week 52.¹

Stoke also noted that site activation is underway in China, where zorevunersen was recently granted Breakthrough Therapy Status,¹ though data from the additional patients in Europe and China are not planned for inclusion in the U.S. NDA submission to the FDA.¹ The company has a partnership with Biogen, under which Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico, while Biogen receives exclusive rest of world commercialization rights.¹