The New England Journal of Medicine has published data from Stoke Therapeutics and Biogen demonstrating for the first time the potential for disease modification in Dravet syndrome using the investigational medicine zorevunersen. The publication includes results from Phase 1/2a and ongoing open-label extension studies showing substantial and durable reductions in seizures and improvements across multiple measures of cognition and behavior.
Dravet syndrome is a severe developmental and epileptic encephalopathy in which children experience severe and recurrent seizures and developmental plateau around age two. Most cases result from mutations in the SCN1A gene leading to insufficient levels of NaV1.1 protein. Currently, there are no approved disease-modifying medicines to treat Dravet syndrome. The study included 81 patients ages 2 to 18 with highly refractory Dravet syndrome who received zorevunersen on top of standard anti-seizure medicines. Substantial reductions in seizures were observed and continued through three years of treatment in the open-label extension studies, with the most substantial reductions among patients treated with initial doses of 70 mg.
Study Results and Safety Profile
Improvements in communication, motor skills, socialization, daily living, and quality of life continued through three additional years of treatment. Helen Cross, corresponding author of the publication and The Prince of Wales’s Chair of Childhood Epilepsy at University College London, stated that “the improvements in cognition, behavior and quality of life seen in these studies suggest we may be changing the course of the disease.” Zorevunersen has been generally well tolerated across the Phase 1/2a and open-label extension studies, with more than 800 doses administered to 81 patients. The most common treatment-related adverse event was cerebrospinal fluid protein elevations with a 44% incidence in the open-label extension studies, though no related clinical manifestations were observed. A Phase 3 study called EMPEROR is underway, with completion of enrollment of approximately 150 patients expected in the second quarter of 2026 and data readout anticipated for mid-2027 to support a New Drug Application to the FDA.
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