Stoke Therapeutics Announces Global Phase 3 EMPEROR Study Design for Zorevunersen in Dravet Syndrome
Stoke Therapeutics has announced the design of its global Phase 3 EMPEROR study for zorevunersen in Dravet syndrome, following alignment with regulatory agencies14.
The study aims to enroll approximately 150 patients with Dravet syndrome aged 2 to <18 years who have SCN1A variants14.
EMPEROR will be a randomized, double-blind, sham-controlled trial lasting 60 weeks (8-week baseline period followed by 52-week treatment period)4.
The dosing regimen will consist of two loading doses of 70mg followed by two maintenance doses of 45mg over 52 weeks14.
The primary endpoint is the reduction in major motor seizure frequency compared to sham treatment4.
Secondary endpoints include durability of seizure reduction and improvements in behavior and cognition, measured primarily by Vineland-3 subdomains14.
The study is expected to start in mid-2025, with data anticipated by the end of 20274.
Zorevunersen has received FDA Breakthrough Therapy Designation, potentially expediting its development and review process4.
Previous clinical data showed an 87% median reduction in convulsive seizure frequency at month eight in patients treated with a similar dosing regimen4.
The EMPEROR study will be conducted globally, including sites in the United States, United Kingdom, European Union, and Japan24.
Sources:
1. https://www.neurologylive.com/view/stoke-announces-phase-3-emperor-registrational-study-zorevunersen-dravet-syndrome
2. https://www.stoketherapeutics.com/patients-families/clinical-studies/
4. https://investor.stoketherapeutics.com/news-releases/news-release-details/stoke-therapeutics-announces-alignment-global-regulatory