Stoke Therapeutics Completes Phase 3 Enrollment for Dravet Syndrome Drug Zorevunersen — Stoke Therapeutics, Inc.

Event summary

Stoke Therapeutics has completed enrollment of 162 patients in the Phase 3 EMPEROR study for zorevunersen, an investigational treatment for Dravet syndrome.
The company plans to initiate a rolling New Drug Application (NDA) submission to the FDA in Q1 2027, with a data readout expected in Q3 2027.
Approximately 50 patients have completed 28 weeks of treatment, and no patients have discontinued treatment in the study.
An additional cohort of about 30 patients is currently enrolling in Europe, with the last patient expected to enroll in August 2026.

The big picture

Stoke Therapeutics' completion of Phase 3 enrollment for zorevunersen marks a critical milestone in the development of a potential first-in-class treatment for Dravet syndrome, a severe developmental and epileptic encephalopathy. The rapid enrollment reflects the unmet medical need and the potential of RNA medicine to address the underlying genetic cause of the disease. The strategic collaboration with Biogen positions Stoke to leverage global commercialization capabilities if regulatory approval is achieved.

What we're watching

Regulatory Pathway: Whether the rolling NDA submission will meet FDA expectations and secure approval by early 2028.
Clinical Efficacy: The impact of zorevunersen on major motor seizure frequency and neurodevelopmental outcomes in Dravet syndrome patients.
Commercial Strategy: How Stoke Therapeutics and Biogen will position zorevunersen in a market with no approved disease-modifying therapies for Dravet syndrome.