Biogen and Stoke Present New Zorevunersen Data Supporting Phase 3 Dravet Syndrome Study
Biogen and Stoke Therapeutics, Inc. (Nasdaq: STOK) today presented new data supporting the design and dosing strategy of the ongoing Phase 3 EMPEROR study of zorevunersen, an investigational, first-in-class disease-modifying therapy for Dravet syndrome, at the 16th European Paediatric Neurology Society (EPNS) Congress.
The analysis evaluated the cognitive and behavioral impact of zorevunersen through Week 68, using a dosing regimen aligned with EMPEROR (two 70mg loading doses, two 45mg maintenance doses). Results showed improvements in multiple measures of cognition and behavior, contrasting with outcomes from the BUTTERFLY natural history study of patients on standard of care.
“These findings give early evidence that this genetically targeted approach could address the underlying cause of Dravet syndrome and improve cognitive and behavioral outcomes,” said Dr. Andreas Brunklaus, study investigator and pediatric neurologist at the Royal Hospital for Children in Glasgow.
Earlier data from Phase 1/2a (ADMIRAL) and the LONGWING open-label extension studies showed durable reductions in seizure frequency and behavioral gains over two years. Zorevunersen was generally well tolerated across all studies.
The latest analysis also guided the selection of key secondary endpoints in EMPEROR, specifically five sub-domains from the Vineland-3 Adaptive Behavior Scales: Receptive and Expressive Communication, Interpersonal Relationships, Coping Skills, and Personal Skills.
“Most Dravet patients continue to struggle with seizures and have no treatments that address the disease’s cognitive burden,” said Dr. Katherine Dawson, Head of Therapeutics Development at Biogen. “We are committed to advancing zorevunersen as a potential breakthrough for this underserved population.”
Dravet syndrome is a rare developmental and epileptic encephalopathy marked by severe seizures and lasting cognitive and behavioral impairments. Approximately 38,000 people are estimated to be living with the condition across the U.S., U.K., EU4, and Japan.
