## Stoke Therapeutics Accelerates Dravet Syndrome Drug Timelines, Eyes 2027 NDA Submission
**Bedford, MA** – Stoke Therapeutics, Inc. (Nasdaq: STOK) is advancing its lead investigational therapy, zorevunersen, for Dravet syndrome, a severe form of epilepsy. The company announced updated timelines today, projecting the completion of patient enrollment in its pivotal Phase 3 EMPEROR study for the second quarter of 2026. This accelerated enrollment pace positions the study for a mid-2027 data readout, anticipated to support a New Drug Application (NDA) submission to the U.S. Food and Drug Administration (FDA). Stoke Therapeutics plans to initiate a rolling NDA submission in the first half of 2027.
Zorevunersen, developed in collaboration with Biogen (Nasdaq: BIIB), is a first-in-class RNA medicine targeting the underlying cause of Dravet syndrome. The drug is designed to restore the expression of the NaV1.1 protein, which is deficient in patients with this condition due to mutations in the *SCN1A* gene. Current treatments often fall short, with a significant percentage of patients experiencing less than a 50% reduction in seizure frequency.
The company also reported on its ongoing dialogue with the FDA following a recent multidisciplinary meeting. While no immediate changes to the zorevunersen development program were decided, the FDA has requested additional information. Stoke Therapeutics is actively engaged with the agency to explore avenues for expediting the development, registration, and delivery of zorevunersen to patients who currently have limited therapeutic options.
“The rapid enrollment in our Phase 3 EMPEROR study is highly encouraging and underscores the urgent need for a disease-modifying treatment for Dravet syndrome,” said Ian F. Smith, CEO of Stoke Therapeutics. “The accelerated timelines for enrollment completion and the Breakthrough Therapy Designation place us in a strong position to initiate a rolling NDA submission in the first half of 2027, potentially bringing zorevunersen to patients sooner than anticipated.”
Smith further commented on the FDA meeting, stating, “Our recent interaction with the FDA was productive. We appreciate their engagement in understanding Dravet syndrome and its profound impact on patients and their families. We have provided the requested additional information and are committed to exploring all opportunities to expedite the availability of zorevunersen.”
As of January 9, 2026, investigators have identified nearly 330 potential study candidates globally. Approximately 60 patients are currently undergoing the initial screening period, with another 60 having progressed to randomization and initial dosing.
**Financial Outlook**
Stoke Therapeutics reported approximately $391.7 million in cash, cash equivalents, and marketable securities as of December 31, 2025. This financial standing, coupled with anticipated proceeds from its collaboration with Biogen, is expected to support the company’s operations through 2028.
**Presentation at J.P. Morgan Healthcare Conference**
Ian F. Smith is scheduled to present at the 44th Annual J.P. Morgan Healthcare Conference on Tuesday, January 13, 2026, at 7:30 p.m. Eastern Time. A live webcast of the presentation will be accessible via the Investors & News section of Stoke’s website.
**Understanding Dravet Syndrome**
Dravet syndrome is a rare and severe form of developmental and epileptic encephalopathy (DEE). It is characterized by frequent, prolonged seizures, along with significant cognitive and behavioral challenges. Genetic mutations, most commonly in the *SCN1A* gene, disrupt the normal electrical activity in the brain, leading to insufficient NaV1.1 protein. Even with current anti-seizure medications, many patients do not achieve adequate seizure control. The condition carries a heightened risk of sudden unexpected death in epilepsy (SUDEP), with a substantial mortality rate before adulthood. While occurring globally, an estimated 38,000 individuals in the U.S., UK, EU-4, and Japan are living with Dravet syndrome, for whom no disease-modifying therapies are currently approved.
**About Zorevunersen**
Zorevunersen is an investigational antisense oligonucleotide therapy designed to address the root cause of Dravet syndrome. By enhancing functional NaV1.1 protein production from the healthy copy of the *SCN1A* gene, it aims to significantly reduce seizure frequency and improve neurodevelopmental outcomes. The therapy has received orphan drug designation from both the FDA and the European Medicines Agency (EMA), as well as rare pediatric disease designation and Breakthrough Therapy Designation from the FDA for Dravet syndrome associated with specific *SCN1A* gene mutations. The collaboration with Biogen grants Stoke Therapeutics exclusive rights in the U.S., Canada, and Mexico, while Biogen holds commercialization rights for the rest of the world.
**The EMPEROR Study**
The EMPEROR Phase 3 study (NCT06872125) is a global, double-blind, sham-controlled trial evaluating the efficacy, safety, and tolerability of zorevunersen in children aged 2 to 18 years. The study is being conducted across the U.S., UK, and Japan, with planned expansion into Germany, Spain, France, and Italy in the second quarter of 2026. Participants are randomized to receive either zorevunersen via intrathecal administration or a sham comparator for 52 weeks. The primary endpoint focuses on the change in major motor seizure frequency from baseline. Key secondary endpoints include the durability of effect and improvements in behavior and cognition, assessed using validated scales. More information can be found at emperorstudy.com.
**Stoke Therapeutics: Pioneering RNA Medicine**
Stoke Therapeutics (Nasdaq: STOK) is a biotechnology company focused on developing RNA-based medicines to restore protein expression. Their proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach utilizes antisense oligonucleotides to selectively increase natural protein levels. Zorevunersen, their lead candidate for Dravet syndrome, is a testament to this technology. Stoke’s initial pipeline targets central nervous system and ocular diseases caused by approximately 50% loss of protein function (haploinsufficiency), with potential applications extending to other organs and systems.
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**Forward-Looking Statements:** This press release contains forward-looking statements regarding the potential of zorevunersen, clinical trial progress, regulatory interactions, and financial projections. These statements are subject to various risks and uncertainties, including the ability to achieve regulatory approval and commercial success, as detailed in Stoke Therapeutics’ filings with the Securities and Exchange Commission.
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**Contact:**
Susan Willson
Vice President, Corporate Communications
[email protected]
415-509-8202
Doug Snow
Director, Communications & Investor Relations
[email protected]
508-642-6485
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