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Stoke Therapeutics And Biogen Report NEJM Data Showing Zorevunersen May Modify Disease In Dravet Syndrome, Supporting Phase 3 EMPEROR Study

Benzinga·03/04/2026 22:07:11
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–Results show that targeting the underlying cause of Dravet syndrome with zorevunersen may improve outcomes for people with this rare, devastating genetic neurodevelopmental disease–

–Data support ongoing global Phase 3 EMPEROR study–

BEDFORD, Mass. and CAMBRIDGE, Mass., March 04, 2026 (GLOBE NEWSWIRE) --  Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine, and Biogen Inc. (NASDAQ:BIIB), today announced the publication of data from studies of the investigational medicine zorevunersen 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. 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).

Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) in which people experience severe and recurrent seizures and a plateauing in their neurodevelopment around the age of two. Over time, children with Dravet syndrome fall further and further behind their neurotypical peers in their ability to achieve developmental milestones. There are currently no approved disease-modifying medicines to treat Dravet syndrome.