Stoke Therapeutics Announces Completion of Enrollment of 162 Patients Into the Phase 3 EMPEROR Study of Zorevunersen, an Investigational Medicine for the Treatment of Dravet Syndrome

About this update from Stoke Therapeutics, Inc.

Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine and has a lead investigational medicine, zorevunersen, in development as a first-in-class potential disease-modifying treatment for Dravet syndrome. The Company today announced the completion of enrollment of 162 patients into the Phase 3 EMPEROR study of zorevunersen. Stoke plans to initiate a rolling New Drug Application (NDA) submission to the U.S. Food and Drug Administration (FDA) in the first quarter of 2027. 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. The EMPEROR study is progressing, and 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. An additional cohort of approximately 30 patients in Europe is currently enrolling, and the last patient is expected to enroll in August. No patients have discontinued treatment in the study. “The rapid enrollment of the Phase 3 EMPEROR study reflects the severity of Dravet syndrome and the potential of zorevunersen to address the underlying genetic cause of the disease, resulting in reduced seizure burden and the opportunity for more neurotypical development,” said Ian F. Smith, Chief Executive Officer and Director of Stoke Therapeutics. “With five years of clinical data for zorevunersen and strong awareness among patients, families and investigators, we completed enrollment in just 10 months and are on track for a Phase 3 data readout in the third quarter of 2027. We expect to complete our rolling NDA submission shortly thereafter based on these data, and we continue to build our organization and capabilities to deliver zorevunersen to all patients in the U.S. who may benefit following a potential FDA approval and U.S. launch by early 2028.” “The Dravet Syndrome Foundation was founded to advance understanding of this devastating disease and fight for better treatments. We are deeply gratified to have played a role in designing and delivering clinical studies to create potential new medicines, from early natural history studies of Dravet syndrome through to the successful enrollment of this first-of-its-kind Pha...

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