– Phase 3 EMPEROR study of zorevunersen, a first-in-class potential disease-modifying medicine for Dravet syndrome, on track to initiate in 2Q 2025 –
– As of December 31, 2024, the Company had $246.7 million in cash, cash equivalents, and marketable securities; together with the $165 million upfront and eligible proceeds from Biogen collaboration anticipated to fund operations to mid-2028 –
BEDFORD, Mass., March 18, 2025–(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 full year ended December 31, 2024 and provided business updates.
“Recent milestones – including Breakthrough Therapy Designation, positive data supporting our Phase 3 dosing regimen and global regulatory alignment – have catalyzed the Dravet community and put us on track to initiate EMPEROR in the second quarter,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “The potential of zorevunersen to change the course of Dravet syndrome by addressing its underlying cause is becoming increasingly recognized. Our recent collaboration with Biogen brings complementary expertise in neurology and the global commercialization of high-value medicines for rare genetic diseases – expertise that will help us deliver faster for patients and maximize value. With a strong financial position, we are well-capitalized to advance through Phase 3 and prepare for launch readiness.”
Recent Program Highlights and Additional Corporate Updates
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Separately today, the Company announced that Dr. Edward M. Kaye has decided to step down as Chief Executive Officer of Stoke Therapeutics. After seven years of successful leadership and taking the company from a startup to a late-stage clinical development company, Dr. Kaye will transition to an advisory role and continue to serve as a Director.
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In February, the Company entered into a collaboration with Biogen to develop and commercialize zorevunersen for the treatment of Dravet syndrome. The Company retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights.
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In January, the Company announced plans to initiate a global Phase 3 registrational study of zorevunersen (EMPEROR) following successful alignment with regulatory agencies in the United States, Europe, and Japan.
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In December, the Company shared new positive data from the open-label extension (OLE) studies of zorevunersen in children and adolescents with Dravet syndrome at American Epilepsy Society (AES) 2024 Annual Meeting.
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In December, the Company announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for zorevunersen for the treatment of Dravet syndrome with a confirmed mutation, not associated with gain-of-function, in the SCN1A gene.
