Stoke Therapeutics, Inc., based in Bedford, Massachusetts, has announced significant updates regarding its ongoing clinical trial for zorevunersen, a potential treatment for Dravet syndrome. This investigational medicine is being developed in collaboration with Biogen and is positioned as a first-in-class disease-modifying therapy. The company revealed on January 11, 2026, that it has accelerated timelines for both patient enrollment and the expected data readout from the EMPEROR study.
The company anticipates that enrollment of 150 patients will now be completed by the second quarter of 2026. This progress enables the Phase 3 EMPEROR study to remain on schedule for a data readout projected for mid-2027. The results are expected to support the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA).
Key Milestones for Zorevunersen Development
The accelerated timeline reflects Stoke Therapeutics’ commitment to advancing its clinical programs. The company plans to initiate a rolling NDA submission in the first half of 2027, which would facilitate the potential for zorevunersen to reach the market sooner for patients suffering from Dravet syndrome.
Dravet syndrome is a severe form of epilepsy that typically begins in the first year of life. It is characterized by frequent seizures and developmental delays, making effective treatments essential for improving patients’ quality of life. The EMPEROR study aims to evaluate the safety and efficacy of zorevunersen in this vulnerable patient population.
Stoke Therapeutics has positioned itself at the forefront of RNA medicine, focusing on restoring protein expression to treat various diseases. The collaboration with Biogen enhances the development capabilities and resources available for zorevunersen, potentially accelerating the path to market.
As the clinical trial progresses, both companies remain optimistic about the outcomes and their implications for patients with Dravet syndrome. Stakeholders are closely monitoring these developments, as they may represent a significant advancement in treatment options for this challenging condition.
In summary, Stoke Therapeutics is making swift progress in its clinical endeavors, with the EMPEROR study set to provide critical data in mid-2027, paving the way for potentially transformative treatment options for individuals affected by Dravet syndrome.
