05 Mar Publication of data showing Zorevunersen effects on Dravet Syndrome patients
Stoke Therapeutics, a biotechnology company that works on a proposed treatment for Dravet Syndrome, announced the publication of first data that shows the potential for disease modification in Dravet Syndrome.
The company plans to position the investigational medicine zorevunersen as potentially the first disease-modifying medicine for the treatment of Dravet Syndrome. Zorevunersen was granted FDA Breakthrough Therapy Designation, a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically-significant endpoint(s).
Stoke announced the data from studies of zorevunersen were publiched in The New England Journal of Medicine (NEJM). The publication includes results from two completed Phase 1/2a and ongoing open-label extension (OLE) studies that demonstrate, for the first time, the potential for disease modification in people living with Dravet Syndrome, the company said in a stock exchange announcement. “These data showed substantial and durable reductions in seizures and improvements across multiple measures of cognition and behavior that began in the Phase 1/2a treatment period and continued through three additional years of treatment in the OLEs. The effects were shown in people treated with zorevunersen on top of standard of care anti-seizure medicines (ASMs),” Stoke said.
The company has already started the Phase 3 EMPEROR Study, a global, double-blind, sham-controlled study evaluating the efficacy, safety and tolerability of zorevunersen in children ages 2 to <18 with Dravet Syndrome, with a confirmed variant in the SCN1A gene not associated with gain-of-function. Stoke expects to complete enrollment of approximately 150 patients in the United States, United Kingdom and Japan in Q2 2026, with a data readout on track for mid-2027 to support the submission of a New Drug Application (NDA) to the FDA. At least 20 additional patients are expected to enroll in Germany, Spain, France and Italy starting in the second quarter of 2026.
The proposed study will evaluate two loading doses of 70mg followed by two maintenance doses of 45mg over 52-weeks compared to sham in children and adolescents ages 2 to <18 with Dravet syndrome. The primary endpoint will be reduction in major motor seizure frequency. Key secondary endpoints will include improvements in cognition and behavior as measured primarily by Vineland-3.
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More details:
The NEJM publication
Stoke Therapeutics news release