Stoke Therapeutics, a biotechnology company focused on RNA medicine, will present data at the 2024 American Epilepsy Society (AES) Annual Meeting in Los Angeles, California, from December 6th to 10th. A virtual event for investors and research analysts will also be held on December 9th at 8:30 am EST, featuring discussions led by clinicians and patient advocates.
The company is developing zorevunersen, a potential first-in-class disease-modifying treatment for Dravet syndrome. New data from a Phase 1/2 study will be presented, showcasing results from nine patients who received initial 70mg doses of zorevunersen followed by 45mg maintenance doses in an open-label extension (OLE) study. Additional data from 73 patients in the OLE studies will also be presented, demonstrating consistent and durable seizure reduction and continuous improvement across various Vineland-3 subdomains over 24 months. Treatment was generally well-tolerated.
Stoke will host a symposium for clinicians on December 8th from 9:00 PM to 12:00 AM EST. This symposium will examine new data from the Dravet syndrome program and discuss the need for disease-modifying therapies that address more than just seizure reduction, focusing on the behavioral, cognitive, and seizure-related impacts of the disease.
The investor and analyst virtual event on December 9th, from 8:30 AM to 9:30 AM EST, will feature leading clinicians and patient advocates discussing the impacts of Dravet syndrome, the current treatment landscape, the latest zorevunersen data, and the potential real-world benefits of a disease-modifying treatment. A question-and-answer session for research analysts will also be included.
Dravet syndrome, a severe and progressive genetic epilepsy, is characterized by frequent, prolonged seizures starting in infancy. It often leads to intellectual disability, developmental delays, movement and balance issues, speech disturbances, growth defects, sleep problems, autonomic nervous system disruptions, and mood disorders. This disease is classified as a developmental and epileptic encephalopathy and carries a higher risk of Sudden Unexpected Death in Epilepsy (SUDEP) than the general epilepsy population. Currently, there are no approved disease-modifying treatments. Dravet syndrome affects approximately one in 16,000 babies, regardless of geographic location or ethnicity.
Zorevunersen, an investigational antisense oligonucleotide (ASO), aims to increase NaV1.1 protein expression by utilizing the healthy copy of the SCN1A gene. This action is intended to restore physiological NaV1.1 levels, thus reducing seizures and non-seizure comorbidities. Zorevunersen has received orphan drug designation from both the FDA and EMA, and rare pediatric disease designation from the FDA for Dravet syndrome.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
