Stoke, Biogen presents new data from OLE studies of zorevunersen

Stoke Therapeutics (STOK) and Biogen (BIIB) announced the presentation of longer-term follow-up analyses from the ongoing open-label extension 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 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 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. “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 patients treated with zorevunersen. Improvements in cognition and behavior are not something we see in patients with Dravet syndrome, and rarely do we see such strong correlation between caregiver and clinician-reported outcomes. Together, these data are starting to paint a fuller picture of the potential impact disease modification could have on a patient’s overall health and daily living.”