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– 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 –
– 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 –
CAMBRIDGE, Mass. and BEDFORD, Mass., Oct. 09, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (NASDAQ:BIIB) and Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine, 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.
New 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).