Stoke Therapeutics presents Zorevunersen data at EEC

Stoke Therapeutics announced highlights from presentations at the 15th European Epilepsy Congress, or EEC, related to the company’s work to develop the first disease-modifying medicine for Dravet syndrome. Zorevunersen data showing substantial and sustained reductions in seizures and meaningful improvements in multiple measures of cognition and behavior were presented for the first time in a scientific forum. New data from an analysis of patients treated in the Phase 1/2a ADMIRAL study showed improvements in cognition and behavior during the first year of treatment with additional increases demonstrated as treatment continued. In addition, data from a two-year natural history study presented at the meeting showed that despite treatment with standard-of-care antiseizure medications, patients with Dravet syndrome continued to have high seizure rates and plateaued in their neurodevelopment, resulting in a widening gap in development compared to their neurotypical peers. MONARCH and ADMIRAL Phase 1/2a studies: Single and multiple doses of zorevunersen up to 70mg were generally well tolerated. Patients treated with 2 or 3 doses of 70mg of zorevunersen experienced median seizure reductions of 85% at 3 months and 74% at 6 months after the last dose, compared to baseline. Multiple doses of zorevunersen led to meaningful improvements in multiple measures of cognition and behavior within the first year of treatment, including receptive communication, interpersonal relationships and gross motor skills. SWALLOWTAIL and LONGWING open label extension studies: Safety findings among patients who continued treatment were consistent with the findings from the Phase 1/2a studies, except for a greater incidence of cerebrospinal fluid protein elevation. Durable reductions in convulsive seizure frequency were observed throughout the course of treatment. In addition, data indicated meaningful improvements in multiple measures of cognition and behavior over the first year of continued dosing of zorevunersen. BUTTERFLY: Compared to their neurotypical peers, adaptive functioning and neurodevelopment in patients with Dravet syndrome generally plateaued, resulting in a widening developmental gap over time. Seizure rates remained high over 24 months despite treatment with standard-of-care antiseizure medications.