Core Insights - Stoke Therapeutics and Biogen announced longer-term follow-up analyses from ongoing open-label extension studies of zorevunersen, indicating its potential as a disease-modifying medicine for Dravet syndrome [1] Company Overview - Stoke Therapeutics is a biotechnology company focused on restoring protein expression through RNA medicine [1] - Biogen Inc. is a biotechnology company that collaborates with Stoke Therapeutics on the zorevunersen studies [1] Product Development - The ongoing studies of zorevunersen are aimed at evaluating its effectiveness in treating Dravet syndrome, a severe form of epilepsy [1] - The new results from the studies support the potential of zorevunersen as a disease-modifying treatment [1]

Core Insights - Stoke Therapeutics has appointed Ian F. Smith as Chief Executive Officer, who has been serving as Interim CEO since March 2025 and has been a board member since 2023 [1][3] - The company is advancing its lead investigational medicine, zorevunersen, in a global Phase 3 study for Dravet syndrome, a severe developmental and epileptic encephalopathy [2][8] - The collaboration with Biogen Inc. for zorevunersen allows Stoke to retain exclusive rights in the U.S., Canada, and Mexico, while Biogen holds rights for the rest of the world [2] Company Leadership - Ian F. Smith is recognized for his deep experience in rare disease drug development and has a strong track record in building and leading teams [3][4] - Arthur Tzianabos, who has been on the board since 2018, will resume his role as Chairman after serving as Executive Chairman during the CEO search [1][6] Clinical Development - The pivotal Phase 3 EMPEROR study for zorevunersen has commenced, contributing to the company's growth and value creation for shareholders [3] - Stoke is also expanding its pipeline with a second clinical program targeting Autosomal Dominant Optic Atrophy [3] Company Background - Stoke Therapeutics focuses on restoring protein expression through RNA medicine, utilizing its proprietary TANGO approach to develop antisense oligonucleotides [8] - The company is headquartered in Bedford, Massachusetts, and aims to address diseases caused by a loss of approximately 50% of normal protein levels [8]

Core Insights - Zorevunersen shows potential as the first disease-modifying treatment for Dravet syndrome, with significant reductions in seizure frequency and improvements in cognitive and behavioral outcomes over three years of treatment [2][3][12] Efficacy Results - In Phase 1/2a studies, patients receiving initial doses of 70 mg of zorevunersen experienced a median seizure reduction of 84.8% and an increase of eight seizure-free days per 28 days at three months post-treatment [4] - In open-label extension studies, 94% of eligible patients continued treatment, with 77% remaining after three years, sustaining reductions in major motor seizure frequency [5] - Improvements in quality of life were noted, with an 18-point increase in EuroQol Visual Analog Scale (EQ-VAS) scores over three years [6] Safety Profile - Zorevunersen was generally well tolerated, with treatment-emergent adverse events (TEAEs) reported in 30% of patients in Phase 1/2a studies and 53% in open-label extension studies [7] - The most common TEAE was elevated cerebrospinal fluid (CSF) protein levels, occurring in 14% of patients in Phase 1/2a and 44% in open-label extension studies [7] Study Design - The Phase 1/2a studies involved 81 patients aged 2 to 18 with refractory Dravet syndrome, focusing on safety, pharmacokinetics, and seizure frequency [9] - The ongoing EMPEROR Phase 3 study aims to further evaluate zorevunersen's efficacy and safety in a larger population [13] Company Background - Biogen and Stoke Therapeutics are collaborating on the development of zorevunersen, which has received orphan drug designation and Breakthrough Therapy Designation from the FDA [12][14] - Stoke Therapeutics focuses on restoring protein expression through RNA medicine, with zorevunersen being their first investigational product [16]