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The New England Journal of Medicine Publishes First Data to Demonstrate the Potential for Disease Modification in Dravet Syndrome
–Results show that targeting the underlying cause of Dravet syndrome with zorevunersen may improve outcomes for people with this rare, devastating genetic
About this update from Stoke Therapeutics, Inc.
[{"type":"text","content":"\n–Results show that targeting the underlying cause of Dravet syndrome with zorevunersen may improve outcomes for people with this rare, devastating genetic neurodevelopmental disease–\n\n\n–Data support ongoing global Phase 3 EMPEROR study–\n\n\n BEDFORD, Mass. & CAMBRIDGE, 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, and Biogen Inc. (NASDAQ: BIIB), today announced the publication of data from studies of the investigational medicine zorevunersen in The New England Journal of Medicine (NEJM). The publication includes results from two completed Phase 1/2a and ongoing open-label extension (OLE) studies that demonstrate, for the first time, the potential for disease modification in people living with Dravet syndrome. These data showed substantial and durable reductions in seizures and improvements across multiple measures of cognition and behavior that began in the Phase 1/2a treatment period and continued through three additional years of treatment in the OLEs. The effects were shown in people treated with zorevunersen on top of standard of care anti-seizure medicines (ASMs).\n\n\nDravet syndrome is a severe developmental and epileptic encephalopathy (DEE) in which people experience severe and recurrent seizures and a plateauing in their neurodevelopment around the age of two. Over time, children with Dravet syndrome fall further and further behind their neurotypical peers in their ability to achieve developmental milestones. There are currently no approved disease-modifying medicines to treat Dravet syndrome.\n\n\n“These data mark a potential turning point in the treatment of Dravet syndrome,” said Helen Cross, MB ChB, Ph.D., corresponding author of the NEJM publication and The Prince of Wales’s Chair of Childhood Epilepsy and Director of University College London Great Ormond Street Institute of Child Health, Honorary Consultant in Paediatric Neurology at Great Ormond Street Hospital. “While reducing seizures is still critical, the improvements in cognition, behavior and quality of life seen in these studies suggest we may be changing the course of the disease and therefore the lives of patients and their families.”\n\n\n“Dravet syndrome arrives without warning for families, just as it did...