Stoke Therapeutics Prepares for Phase 3 Study of Zorevunersen for Dravet Syndrome

Stoke Therapeutics, Inc. (NASDAQ: STOK) recently announced plans to launch a Phase 3 study of zorevunersen, aiming to develop the first disease-modifying treatment for Dravet syndrome. The company disclosed this information in a Form 8-K filing with the Securities and Exchange Commission.

The Phase 3 study, named EMPEROR, will assess zorevunersen’s efficacy in reducing major motor seizure frequency in children and adolescents aged 2 to under 18 with Dravet syndrome. Stoke Therapeutics has finalized the study protocol following productive engagements with regulatory bodies such as the U.S. Food and Drug Administration (FDA) and the European Medicines Association (EMA).

The EMPEROR trial will involve the evaluation of two loading doses of 70mg followed by two maintenance doses of 45mg over a 52-week period compared to a sham treatment. The primary endpoint will be a reduction in major motor seizure frequency, with key secondary endpoints focusing on improvements in cognition and behavior, primarily measured by Vineland-3.

Stoke Therapeutics anticipates initiating the Phase 3 study by mid-2025, with data expected by the end of 2027, subject to enrollment and study timelines. The company is evaluating the commercial potential of zorevunersen for over 38,000 patients with Dravet syndrome across seven major markets.

To support this initiative, Stoke Therapeutics recently conducted a virtual event for investors and analysts, providing details on the Phase 3 study design and potential commercial opportunities for zorevunersen. The company’s leadership expressed optimism about the drug’s ability to address the unmet medical needs of patients with Dravet syndrome.

The filing also included information on the regulatory alignment with global agencies and the anticipated benefits of zorevunersen, emphasizing the significance of developing a disease-modifying therapy for Dravet syndrome. Stoke Therapeutics’ commitment to advancing zorevunersen underscores its dedication to improving the lives of individuals living with this debilitating condition.

This article was generated by an automated content engine and was reviewed by a human editor prior to publication. For additional information, read Stoke Therapeutics’s 8K filing here.

Stoke Therapeutics Company Profile

Stoke Therapeutics, Inc, an early-stage biopharmaceutical company, develops medicines to treat the underlying causes of severe genetic diseases in the United States. The company utilizes its proprietary targeted augmentation of nuclear gene output to develop antisense oligonucleotides to selectively restore protein levels.