Stoke Therapeutics Reports First Quarter 2025 Financial Results and Provides Business Updates

About this update from Stoke Therapeutics, Inc.

– 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 – – 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 – – 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 – BEDFORD, Mass. --(BUSINESS WIRE)-- Stoke 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. “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.” Recent Program Highlights and Upcoming Milestones The 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. In 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 double digits to high teens on potential net sales in the Biogen territory. Stoke retains exclusive rights for zorevunersen in the United States , Canada , and Mexico . Biogen receives exclusive rest of world commercialization rights. (For full details, see the press release ). In January, the Company announced plans to initiate a global Phase 3 registrational study of zorevunersen (EMPEROR) for the treatment of Dravet syndrome following successful alignment with the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA) and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA). The study design is based on positive clinical data from Phase 1/2a and open label extension studies that showed continuing improvements in cognition and behavior in addition to substantial and durable reductions in major motor seizure frequency. The study will evaluate two loading doses of 70mg followed by two maintenance doses of 45mg over 52 weeks compared to sham in children and adolescents ages 2 to

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