09 Jan Phase 3 of Zorevunersen Study

Stoke Therapeutics, a biotechnology company that works on a proposed treatment for Dravet Syndrome, announced this week that, following successful interactions with the FDA, EMA and PMDA, it has finalized its EMPEROR Phase 3 study protocol. The proposed study will evaluate reductions in major motor seizure frequency as well as improvements in behavior and cognition in children and adolescents ages 2 to <18 years old, the company said.

The trial, which will therefore be developed in the US, the UK, Europe and Japan, will involve globally 150 patients with a confirmed variant in the SCN1A gene not associated with a gain of function. Stoke plans to start the Phase 3 study in mid-2025.

The study will be a global, randomized, double-blind, sham-controlled trial, that means that some patients will receive treatment while some others (control arm) will undergo a sham procedure without receiving treatment. At its completion, if the outcome of the trial is successful, both groups of patients will be offered ongoing treatment with the treatment as part of an OLE study.

The proposed study will evaluate two loading doses of 70mg followed by two maintenance doses of 45mg over 52-weeks compared to sham in children and adolescents ages 2 to <18 with Dravet syndrome. The primary endpoint will be reduction in major motor seizure frequency. Key secondary endpoints will include improvements in cognition and behavior as measured primarily by Vineland-3.

The company plans to position zorevunersen as potentially the first disease-modifying medicine for the treatment of Dravet Syndrome. Zorevunersen was recently granted FDA Breakthrough Therapy Designation, a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically-significant endpoint(s).

At the moment, neither the hospital sites that will be involved in the trial nor the timeframe for the start-up are yet known, which, taking into account the different administrative procedures, could be quite long.

Keep following us, as usual we will keep you updated on the main advances in the understanding and treatment of Dravet Syndrome.