Business
Stoke Therapeutics Reports First Quarter 2025 Financial Results and Provides Business Updates
– Regulatory alignment achieved for Phase 3 EMPEROR study of zorevunersen, a potential first-in-class disease-modifying medicine for Dravet syndrome; Study
About this update from Stoke Therapeutics, Inc.
[{"type":"text","content":"\n– Regulatory alignment achieved for Phase 3 EMPEROR study of zorevunersen, a potential first-in-class disease-modifying medicine for Dravet syndrome; Study start anticipated in 2Q 2025 –\n\n– Zorevunersen collaboration with Biogen brings leading capabilities for commercializing high value, disease-modifying medicines for rare genetic diseases; Stoke retains full rights in the United States, Canada, and Mexico –\n\n– As of March 31, 2025, the Company had $380.3 million in cash, cash equivalents, and marketable securities, anticipated to fund operations beyond 2H 2027 Phase 3 data and into launch readiness projected to mid-2028 –\n\n BEDFORD, Mass.--(BUSINESS WIRE)--\nStoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine and has a lead investigational medicine, zorevunersen, in development as a first-in-class potential disease-modifying treatment for Dravet syndrome. The Company today reported financial results for the first quarter ended March 31, 2025, and provided business updates.\n\n“The first quarter was defined by strong execution as we advance zorevunersen into Phase 3 development for Dravet syndrome,” said Ian F. Smith, Interim Chief Executive Officer and Director of Stoke Therapeutics. “With regulatory alignment on a global Phase 3 study, a strategic collaboration with Biogen, and a balance sheet that takes the company to launch readiness, we are well-positioned to deliver the first disease-modifying therapy for Dravet syndrome to patients worldwide who need it while also continuing to invest in our pipeline.”\n\nRecent Program Highlights and Upcoming Milestones\n\n\nThe Company plans to initiate EMPEROR in the second quarter of 2025 with an expected data readout in the second half of 2027, which is anticipated to support global regulatory filings.\n\n\nIn February, the Company entered into a collaboration with Biogen to develop and commercialize zorevunersen for the treatment of Dravet syndrome for which Stoke received $165 million upfront. The parties will share external clinical development costs for zorevunersen (30 percent Biogen; 70 percent Stoke). Additionally, Stoke may receive up to $385 million in development and commercial milestone payments and be eligible to receive tiered royalties ranging from low ...