Stoke and Biogen Present Long-Term Data on Zorevunersen for Dravet Syndrome — Stoke Therapeutics, Inc.

Event summary

Stoke Therapeutics and Biogen presented 4-year data on zorevunersen at the 16th European Epilepsy Congress, showing sustained cognitive and behavioral improvements in Dravet syndrome patients.
The Phase 3 EMPEROR study results are anticipated in Q3 2027.
Zorevunersen demonstrated durable seizure reductions and a generally well-tolerated safety profile over 5 years of treatment.
New exploratory analyses showed effects on severe seizure types, a key risk factor for sudden unexpected death in epilepsy (SUDEP).
77% of patients from the Phase 1/2a studies continued into open-label extension studies.

The big picture

Stoke Therapeutics and Biogen's presentation of long-term data on zorevunersen underscores the potential of RNA medicine to address the underlying genetic cause of Dravet syndrome. The sustained improvements in cognition, behavior, and seizure reduction highlight the growing interest in disease-modifying therapies for rare neurological disorders. The strategic collaboration between Stoke and Biogen positions zorevunersen as a potential first-in-class treatment, with significant implications for patients and the broader epilepsy market.

What we're watching

Regulatory Pathway: Whether the positive long-term data will accelerate regulatory reviews and approvals for zorevunersen.
Clinical Efficacy: The pace at which zorevunersen can demonstrate disease-modifying potential in larger, controlled studies.
Commercial Strategy: How Stoke and Biogen will position zorevunersen in a market with limited disease-modifying therapies for Dravet syndrome.