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Stoke Therapeutics and Biogen Present New Data at the 54th Child Neurology Society (CNS) Annual Meeting that Support the Potential of Zorevunersen as a Disease-Modifying Medicine for Dravet Syndrome
– An analysis designed to evaluate the potential effects of the Phase 3 zorevunersen dosing regimen showed continuing improvements in cognition and behavior
About this update from Stoke Therapeutics, Inc.
[{"type":"text","content":"\n– An analysis designed to evaluate the potential effects of the Phase 3 zorevunersen dosing regimen showed continuing improvements in cognition and behavior at 2 years; results contrast with minimal change in natural history –\n\n\n– Improvements in overall clinical status at 3 years in the open-label extension studies reported by clinicians and caregivers in 95% of patients treated with zorevunersen –\n\n\n BEDFORD, Mass., & CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nStoke 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 presentation of longer-term follow-up analyses from the ongoing open-label extension (OLE) studies of zorevunersen that support the potential of zorevunersen as a disease-modifying medicine for Dravet syndrome. These new results were presented at the 54th Child Neurology Society (CNS) Annual Meeting.\n\n\nNew two-year data from an analysis that was initially performed to understand the potential effects of the Phase 3 dosing regimen on cognition and behavior showed continuing improvements at two years. These results contrast with findings from a two-year natural history study in which patients with Dravet syndrome who were treated with standard of care showed minimal changes in cognition and behavior. In addition, the companies presented three-year results from analyses of Clinical and Caregiver Global Impression of Change (CGI-C and CaGI-C) scales measuring overall clinical status, which complement previously presented EQ-VAS caregiver-reported quality of life improvements. Caregivers and clinicians separately reported similar improvements in overall clinical status in 95% of patients treated with zorevunersen (n=19).\n\n\n“Data from the ongoing open-label extension studies are helping to shape our understanding of the long-term effects of zorevunersen on seizures, cognition, behavior and overall functioning, which together support the potential for disease modification,” said Kelly Knupp, MD, MSCS, Professor of Pediatrics and Neurology at the University of Colorado Anschutz and the Dravet Program Director and Epilepsy Program Lead at Children's Hospital Colorado. “The natural history data continue to provide important context for the effects we are seeing in ...