
Core Insights - Stoke Therapeutics is advancing the development of zorevunersen (STK-001) for Dravet syndrome, aiming to be the first disease-modifying therapy for this genetic epilepsy [1][3] - The FDA granted Breakthrough Therapy designation to zorevunersen in December 2024, highlighting its potential significance [1] - Stoke Therapeutics' shares have increased by 94.7% over the past year, contrasting with a 13.6% decline in the industry [2] Company Developments - The company has received alignment from global regulatory agencies regarding the phase III EMPEROR study design for zorevunersen [3] - The EMPEROR study will involve a randomized, double-blind trial comparing two loading doses of 70mg and two maintenance doses of 45mg over 52 weeks in children and adolescents aged 2 to under 18 with Dravet syndrome [4] - The phase III study is set to commence in mid-2025 across the United States, the United Kingdom, Europe, and Japan [4] Future Prospects - Management is focused on zorevunersen as a potential first disease-modifying treatment for Dravet syndrome, while also developing another candidate, STK-002, for autosomal dominant optic atrophy [5] - The company has a Zacks Rank of 2 (Buy), with narrowed estimates for 2025 loss per share from $2.49 to $2.43 over the past 60 days [7] - Positive regulatory updates and the successful development of zorevunersen are expected to support further stock gains in 2025 [7]