Business
Stoke Therapeutics Reports Second Quarter Financial Results and Provides Business Updates
– FDA removes Partial Clinical Hold as company advances toward a Phase 3 registrational study of zorevunersen (STK-001) in children and adolescents with
About this update from Stoke Therapeutics, Inc.
[{"type":"text","content":"\n– FDA removes Partial Clinical Hold as company advances toward a Phase 3 registrational study of zorevunersen (STK-001) in children and adolescents with Dravet syndrome –\n\n\n– Company to provide an update on Phase 3 registrational plans for zorevunersen in the second half of 2024 –\n\n\n– Company on track to initiate the Phase 1 study (OSPREY) of STK-002 for the treatment of Autosomal Dominant Optic Atrophy (ADOA) this year –\n\n\n– As of June 30, 2024, Company had $282.0 million in cash, cash equivalents, and marketable securities –\n\n\n BEDFORD, Mass.--(BUSINESS WIRE)--\nStoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine, today reported financial results for the second quarter of 2024 and provided business updates including those related to zorevunersen (STK-001), the Company’s proprietary antisense oligonucleotide (ASO) which is in development by Stoke as the first potential medicine to address the genetic cause of Dravet syndrome.\n\n\n“The landmark data announced earlier this year provide strong support for zorevunersen as the first potential disease-modifying medicine for the treatment of Dravet syndrome by showing marked reductions in seizures and improvements in cognition and behavior in a heavily treated patient population,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “We continue to receive strong support and hear a great sense of urgency for zorevunersen among the Dravet syndrome community, including families, caregivers and clinicians. We thank the FDA for working with us to remove the Partial Clinical Hold and look forward to continuing our discussions with them and with other global regulatory agencies toward the goal of agreeing on a single, global Phase 3 registrational study design by year-end.”\n\n\nRecent Program Highlights and Upcoming Milestones\n\n\n\nToday, the Company announced that the U.S. Food and Drug Administration (FDA) has removed the Partial Clinical Hold on zorevunersen.\n\n\n\nThe Company plans to share previously presented positive data from patients treated in the Phase 1/2a and open label extension (OLE) studies of zorevunersen in children and adolescents with Dravet syndrome at the 15th European Epilepsy Congress (EEC), September 7 – 11, 2024, in Rom...