On July 10, 2025, Stoke Therapeutics, Inc. and Biogen Inc. announced the presentation of data from an analysis of zorevunersen, an investigational medicine for Dravet syndrome, at the 16th European Paediatric Neurology Society (EPNS) Congress. The analysis, which informed the design of the Phase 3 EMPEROR study, evaluated the potential effects of the Phase 3 zorevunersen dosing regimen.
The data showed improvements in multiple measures of cognition and behavior at Week 68, contrasting with outcomes observed in natural history data for patients treated with standard of care medicines. Previously reported data from Phase 1/2a and open-label extension (OLE) studies of zorevunersen demonstrated substantial and durable reductions in major motor seizure frequency and continuing improvements in cognition and behavior through two years of treatment.
These findings reinforce the potential of zorevunersen as a first-in-class disease-modifying treatment for Dravet syndrome, a complex neurodevelopmental disorder with significant impacts on patients and their families. The data support the inclusion of key secondary endpoints assessing cognition and behavior in the ongoing Phase 3 EMPEROR study, aiming to address the underlying genetic cause of the disease.
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