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Stoke Therapeutics Announces Updates to Timelines for the Completion of Enrollment and a Phase 3 Data Readout from the EMPEROR Study of Zorevunersen for the Treatment of Dravet Syndrome
— Company now expects to complete enrollment of 150 patients in Q2 2026, with a Phase 3 data readout in mid-2027; Rolling NDA submission planned to initiate
About this update from Stoke Therapeutics, Inc.
[{"type":"text","content":"\n— Company now expects to complete enrollment of 150 patients in Q2 2026, with a Phase 3 data readout in mid-2027; Rolling NDA submission planned to initiate in first half 2027—\n\n\n— Discussions with FDA ongoing following recent multidisciplinary meeting; Company continues to explore potential for expedited development, registration and delivery of zorevunersen to patients —\n\n\n— Company to present at 44th Annual J.P. Morgan Healthcare Conference on Tuesday, January 13, 2026 —\n\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 with Biogen (Nasdaq: BIIB) as a first-in-class potential disease-modifying treatment for Dravet syndrome. Today, the Company announced accelerated timelines for the completion of enrollment and a Phase 3 data readout from the EMPEROR study. Completion of enrollment of 150 patients is now expected in the second quarter of 2026. This enrollment progress puts the Phase 3 EMPEROR study on track for a data readout in mid-2027 that is anticipated to support the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA). The Company plans to initiate a rolling NDA submission in the first half of 2027.\n\n\nIn addition, as part of zorevunersen’s Breakthrough Therapy Designation, a multidisciplinary meeting was held with the FDA to discuss the ongoing clinical development of zorevunersen, including the exploration of potential expedited regulatory pathways. No immediate changes to the zorevunersen development program were agreed to at the meeting. The FDA has requested additional information, and discussions between the Company and the Agency are ongoing regarding potential opportunities to expedite the development, registration and delivery of zorevunersen to patients.\n\n\n“The rate of enrollment in the Phase 3 EMPEROR study is highly encouraging and supports the significant need for a disease-modifying treatment for Dravet syndrome. The accelerated timing for completion of enrollment of 150 patients, in addition to Breakthrough Therapy Designation, positions us to initiate a rolling NDA submission in the first half of 2027 resulting in the potential to delive...