Stoke Therapeutics Moves Forward with Groundbreaking Phase 3 Study

Stoke Therapeutics and the Journey Ahead

Stoke Therapeutics, Inc. (Nasdaq: STOK), a pioneering biotechnology company, has announced a significant milestone in its commitment to addressing Dravet syndrome, a severe epilepsy condition. By aligning with global regulatory agencies, Stoke has set the stage for the Phase 3 EMPEROR study of zorevunersen, which could potentially be the first disease-modifying medicine for this challenging disorder.

Understanding Dravet Syndrome

Dravet syndrome is a complex developmental and epileptic condition that manifests early in life, often within the first year. Children diagnosed with this disorder face a multitude of challenges, including frequent seizures and associated cognitive impairments, which can hinder their development and quality of life. Current treatment options focus primarily on seizure reduction, often leaving significant neurodevelopmental issues unaddressed.

Regulatory Alignment and Study Design

Following productive discussions with authorities like the FDA, EMA, and PMDA, Stoke has finalized the design for the EMPEROR study. This pivotal Phase 3 study intends to treat children and adolescents aged 2 to under 18 with a regimen comprising two loading doses of 70mg, followed by two maintenance doses of 45mg over a 52-week treatment period. The primary goal of this study is to measure the reduction of major motor seizure frequency, while key secondary endpoints will evaluate cognitive and behavioral improvements as assessed using the Vineland-3 tool.

CEO’s Vision for Change

Edward M. Kaye, M.D., CEO of Stoke Therapeutics, expressed his enthusiasm about this alignment and the accompanying momentum from the scientific and medical community. He emphasized that the collaboration between clinicians, patient organizations, and regulatory agencies fuels a collective commitment to find effective solutions for managing Dravet syndrome.

The Role of Zorevunersen

Zorevunersen is distinguished as an investigational new medicine currently undergoing clinical tests. It’s designed to upregulate expression of the NaV1.1 protein by targeting the non-mutant variant of the SCN1A gene. The hope is that this innovative approach will significantly mitigate seizure frequency and address associated comorbidities that patients endure.

Clinical Results and Future Prospects

Support from clinical data has laid strong foundations for the anticipated Phase 3 study. In prior Phase 1/2a studies, patients receiving two or three doses of 70mg exhibited an impressive 87% median reduction in convulsive seizure frequency after eight months. Participants also showed ongoing enhancements in cognition and behavior metrics through two years of treatment. These promising outcomes have been observed even among patients on existing anti-seizure medications.

Global Impact and Enrollment Insights

Scheduled to take place in numerous regions including the US, UK, EU, and Japan, the EMPEROR study expects to enroll around 150 participants diagnosed with Dravet syndrome. This global reach reflects Stoke’s determination to understand and improve treatment frameworks for diverse populations facing this condition.

Expected Milestones

The EMPEROR study is designed for a duration of 60 weeks, which includes an initial 8-week baseline phase, followed by a continuous treatment period. Stoke anticipates having data from this crucial study by late 2027, contingent upon timely patient enrollment and adherence to planned study timelines.

Continuing Engagement with Stakeholders

Stoke Therapeutics plans to conduct ongoing community engagement, particularly with families and clinicians, providing opportunities for continued involvement via open-label extensions for patients eligible for long-term treatment with zorevunersen.

A Look at Stoke Therapeutics

Founded with the mission of leveraging RNA medicine to restore protein expression, Stoke Therapeutics is increasingly recognized for its TANGO approach targeting protein levels critical to health. This focus underscored the company’s commitment to innovative therapies, especially for serious conditions like Dravet syndrome. The proactive development and testing ethos within the company promise a bright horizon for both researchers and patients.

Frequently Asked Questions

What is Dravet syndrome?

Dravet syndrome is a severe form of epilepsy presenting in the first year of life, characterized by frequent seizures and significant developmental challenges.

What is zorevunersen and its purpose?

Zorevunersen is an investigational treatment aiming to modify the disease processes underlying Dravet syndrome and improve patient outcomes.

What does the EMPEROR study entail?

The EMPEROR study is a Phase 3 clinical trial investigating the efficacy of zorevunersen in reducing seizure frequency among children with Dravet syndrome.

When is the EMPEROR trial expected to begin?

The Phase 3 study is planned for initiation around mid-2025, following regulatory approvals and patient enrollment.

Who will participate in the EMPEROR study?

The study is set to enroll approximately 150 patients aged 2 to under 18 diagnosed with Dravet syndrome.

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