Stoke Therapeutics Unveils New Insights on Zorevunersen for Dravet Syndrome

Stoke Therapeutics to Showcase Zorevunersen at AES 2024

Stoke Therapeutics, Inc. (Nasdaq: STOK), a pioneering biotechnology company focused on enhancing protein expression through RNA medicine, is set to exhibit critical findings at the American Epilepsy Society (AES) 2024 Annual Meeting, scheduled for December 6 – 10. This meeting offers a platform for revealing the promising developments surrounding zorevunersen, a treatment that could be the first disease-modifying therapy for Dravet syndrome.

Overview of Zorevunersen

Zorevunersen is an innovative investigational medicine aiming to address the genetic underpinnings of Dravet syndrome—a severe form of epilepsy that manifests within the first year of life. This condition is characterized by frequent seizures and significant comorbidities that severely impact patient quality of life. Stoke Therapeutics believes zorevunersen has the potential to make a lasting impact by restoring normal NaV1.1 protein levels, thereby reducing the incidence of seizures and associated complications.

Presentation of New Data

At AES 2024, Stoke Therapeutics is set to present newly collected data from recent studies involving patients with Dravet syndrome. Speaking on behalf of the company, Chief Medical Officer Dr. Barry Ticho expressed enthusiasm about the data’s potential implications for treatment options. Data drawn from patients who were already receiving optimal anti-seizure medications and then switched to zorevunersen revealed promising results, reinforcing the potential of this treatment as a viable disease-modifying option.

Phase 1/2 Study Insights

Findings will include data from nine patients treated in a Phase 1/2 study who received initial doses of 70 mg of zorevunersen followed by a maintenance dose of 45 mg. Moreover, ongoing open-label extensions included an expanded group of 73 patients, with results showing consistent seizure reduction and improvements in cognitive function over a two-year period. This data suggests not only durability in treatment effects but also the medicine's overall tolerability in the patient population.

Stoke Therapeutics Presentations at AES

The details of Stoke's presentations at the meeting are as follows:

• Title: Zorevunersen (STK-001) Demonstrates Potential for Disease Modification Including Reductions in Seizures and Improvements in Cognition and Behavior in Children and Adolescents with Dravet Syndrome
  Poster Session Date & Time: Sunday, December 8 at 12:00 PM PST
  Oral Presentation Date & Time: Monday, December 9 at 3:15 PM PST
  Presenter: Dr. Linda Laux, Northwestern University Feinberg School of Medicine, Chicago
• Title: Patients with Dravet Syndrome in Open-Label Extension Studies of Zorevunersen (STK-001) Have Durable Reductions in Seizure Frequency and Clinically Meaningful Improvements in Cognition and Behavior
  Poster Session Date & Time: Sunday, December 8 at 12:00 PM PST
  Presenter: Dr. Joseph Sullivan, University of California San Francisco
• Title: Small Changes on the Vineland-3 are Meaningful to Caregivers of Patients with Dravet Syndrome
  Poster Session Date & Time: Sunday, December 8 at 12:00 PM PST
  Presenter: Ms. Carrie Condon, Clinical Science at Stoke Therapeutics
• Title: Spectral EEG Analysis Demonstrates Decreased Slow-wave Activity in Patients with Dravet Syndrome after Treatment with Zorevunersen, an Antisense Oligonucleotide
  Poster Session Date & Time: Monday, December 9 at 12:00 PM PST
  Presenter: Mr. Nigel Colenbier, Epilog, Clouds of Care NV

Symposium and Virtual Events

Moreover, Stoke Therapeutics will engage clinicians in a corporate symposium, addressing the advancements in Dravet syndrome treatment and the need for therapies that extend beyond seizure management. Titled "The Potential of Disease-Modifying Treatments for Dravet Syndrome: Entering a New Era," this session will emphasize significant impacts on patient behavior and cognition.

Additionally, Stoke is hosting a virtual event that will delve into Dravet syndrome's effects and the treatment landscape, including promising insights on zorevunersen. This session aims to facilitate Q&A discussions with leading experts and patient advocates.

Understanding Dravet Syndrome

Dravet syndrome is notoriously challenging to manage, often resulting in extensive comorbidities that impair the quality of life for both patients and caregivers. The disease's progression includes not only seizures but also significant cognitive and developmental hurdles. With one in 16,000 births affected, the reach and impact of this condition extend across diverse populations.

About Stoke Therapeutics

Stoke Therapeutics is committed to addressing critical unmet needs in healthcare through innovative RNA medicine solutions. By utilizing their TANGO approach to develop antisense oligonucleotides, the company is at the forefront of creating potential treatments for genetic disorders. As they advance zorevunersen through clinical trials, Stoke continues to explore additional therapies targeting haploinsufficiencies across various conditions.

Frequently Asked Questions

What is zorevunersen?

Zorevunersen is an investigational medicine being developed to treat Dravet syndrome by restoring NaV1.1 protein levels in the body.

What are the upcoming events for Stoke Therapeutics?

Stoke Therapeutics will present at the AES 2024 Annual Meeting from December 6-10 and will host both a symposium and a virtual event.

Why is zorevunersen significant?

Zorevunersen is potentially the first disease-modifying therapy for Dravet syndrome, aiming to address not only seizures but also associated cognitive and behavioral challenges.

What is Dravet syndrome?

Dravet syndrome is a severe form of genetic epilepsy characterized by frequent, prolonged seizures that typically begin in infancy.

How is Stoke Therapeutics advancing its research?

Stoke Therapeutics utilizes a proprietary approach to develop therapies aimed at restoring protein expression, with several candidates in clinical trials.

About Investors Hangout

Investors Hangout is a leading online stock forum for financial discussion and learning, offering a wide range of free tools and resources. It draws in traders of all levels, who exchange market knowledge, investigate trading tactics, and keep an eye on industry developments in real time. Featuring financial articles, stock message boards, quotes, charts, company profiles, and live news updates. Through cooperative learning and a wealth of informational resources, it helps users from novices creating their first portfolios to experts honing their techniques. Join Investors Hangout today: https://investorshangout.com/

Disclaimer: The content of this article is solely for general informational purposes only; it does not represent legal, financial, or investment advice. Investors Hangout does not offer financial advice; the author is not a licensed financial advisor. Consult a qualified advisor before making any financial or investment decisions based on this article. The author's interpretation of publicly available data shapes the opinions presented here; as a result, they should not be taken as advice to purchase, sell, or hold any securities mentioned or any other investments. The author does not guarantee the accuracy, completeness, or timeliness of any material, providing it "as is." Information and market conditions may change; past performance is not indicative of future outcomes. If any of the material offered here is inaccurate, please contact us for corrections.