Stoke Therapeutics Announces Data That Support the Potential for STK-001 to be the First Disease-Modifying Medicine for the Treatment of Patients with Dravet Syndrome
May 21, 2024
Press Release by Stoke Therapeutics
Stoke Therapeutics, Inc. released data from two open-label Phase 1/2a studies and two open-label extension studies of children and adolescents ages 2 to 18 with Dravet syndrome who were treated with STK-001. Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures, beginning within the first year of life. Dravet syndrome is difficult to treat and has a poor long-term prognosis. The effects of the disease often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system, and mood disorders. There are currently no approved disease-modifying therapies for people living with Dravet syndrome. Data from these new studies showed clinically meaningful effects, including substantial and durable reductions in convulsive seizure frequency and improvements in multiple measures of cognition and behavior that support the potential for disease modification. These changes were observed among a highly refractory group of patients. The improvements in skills like communication, behavior, socialization, and movement distinguish this approach from anything seen to date and mark the entry into a new era in the treatment of Dravet syndrome.