Stoke Therapeutics will be looking towards a Phase III study after the FDA released the partial clinical hold. Image credit: Shutterstock / Vitalii Vodolazskyi.

Stoke Therapeutics has revealed it will move to Phase III trials of its Dravet syndrome drug zorevunersen after the US Food and Drug Administration (FDA) releases its clinical hold.

Stoke Therapeutics said it will be discussing the Phase III registrational plans for zorevunersen with the agency in the second half of 2024.

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The company’s stock price increased by 5.6% following the announcement, closing at $14.23 on 6 August and opening at $15.02 on 7 August, before restoring to normal levels during the day.

Massachusetts-based Stoke Therapeutics previously announced positive efficacy data from its Phase I/IIa MONARCH trial (NCT04442295) and open-label extension (OLE) SWALLOWTAIL trial (NCT04740476) of zorevunersen in children and adolescents with Dravet syndrome.

Despite positive efficacy signals of reduction in convulsive seizure frequency across all three cohorts, one-third of the patients suffered adverse events, most commonly CSF protein elevations, vomiting and irritability. Of those patients, 22% suffered serious adverse events but just one case was deemed related to the study drug.

Stoke Therapeutics’ CEO Dr Edward Kaye said: “We thank the FDA for working with us to remove the partial clinical hold and look forward to continuing our discussions with them and with other global regulatory agencies toward the goal of agreeing on a single, global Phase III registrational study design by year-end.”

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Zorevunersen sodium acts as an SCN1A activator. SCN1A gene generates part of a sodium channel called Nav1.1 that regulates sodium’s entry into cells. The right amount of sodium is crucial to cell communication in the brain. The therapeutic candidate increases the production of Scn1a messenger RNA and the Nav1.1 protein it helps generate without affecting non-sodium ion channels.

Zorevunersen has been granted orphan drug designation by the FDA and the EMA, and rare paediatric disease designation by the FDA.

Dravet syndrome landscape

Dravet syndrome is a severe and progressive genetic epilepsy characterised by frequent, prolonged, and refractory seizures, beginning within the first year of life. Dravet syndrome is difficult to treat and has a poor long-term prognosis. Disease effects go beyond seizures and often include intellectual disability, developmental delays, movement, and balance issues.

Patients with Dravet syndrome typically need a combination of anti-seizure medications (ASMs) to help control or reduce seizures. Despite this, the disease remains one of the most treatment-resistant diseases and good seizure control is rare, with very few patients living seizure-free.