Stoke Therapeutics(US:STOK)2026-01-14 00:30Zorevunersen for Dravet Syndrome - Stoke Therapeutics is advancing zorevunersen as a potential disease-modifying therapy for Dravet Syndrome[9] - Phase 3 study (EMPEROR) enrollment is expected to complete in Q2 2026 with data readout in mid-2027 to support NDA[12, 38] - Approximately 330 patients have been identified globally for the EMPEROR study, with ~60 in screening and ~60 randomized[12, 39] - Data from ongoing OLE studies show continuing improvements in cognition and behavior over 36 months[24] - Zorevunersen demonstrated significant reduction in major motor seizure frequency at 6 months compared to natural history data, with an 8248% reduction compared to a 2043% reduction in the natural history group (P=00181)[26] - In OLE studies, 86% of patients experienced CSF protein elevation, classified as a TEAE in 45%[31] STK-002 for Autosomal Dominant Optic Atrophy (ADOA) - A Phase 1 study of STK-002 for ADOA has been initiated in the UK and Europe[11] Financial Status and Collaboration - Stoke Therapeutics had approximately $3917 million in cash, cash equivalents, and marketable securities as of December 31, 2025, with funding anticipated into 2028[12] - A strategic collaboration with Biogen exists for the development and commercialization of zorevunersen, with Biogen holding exclusive commercialization rights outside of the US, Canada, and Mexico[48] - The Biogen collaboration includes $165 million upfront, shared development costs, and potential milestone payments of $385 million[49] Market Opportunity - There are approximately 38,000 patients with Dravet syndrome across 7 major markets[40, 44]