Stoke Therapeutics Receives Breakthrough Therapy Designation for Zorevunersen in the Treatment of Dravet Syndrome

Stoke Therapeutics, Inc. (NASDAQ: STOK) has recently announced significant developments in its pursuit of treating Dravet syndrome through zorevunersen. On December 4, 2024, Stoke Therapeutics revealed that it had obtained Breakthrough Therapy Designation from the U.S. Food & Drug Administration (FDA) for zorevunersen, a product intended for addressing Dravet syndrome—a severe epilepsy disorder with a genetic basis.

Additionally, on December 6, 2024, Stoke Therapeutics released new data from analysis of patients treated with zorevunersen, showcasing positive findings from Phase 1/2a and open-label extension (OLE) studies. The results demonstrated substantial improvements in reducing convulsive seizure frequency as well as enhancements in cognition and behavior in patients undergoing treatment.

The data unveiled in both the Breakthrough Therapy Designation and new analysis reflect a promising outlook for zorevunersen. The Company has been focusing on providing a potential disease-modifying therapy for Dravet syndrome, aiming to tackle the underlying causes of the condition and enhance patients’ quality of life.

These recent advancements have led Stoke Therapeutics to consider embarking on a Phase 3 registrational study to further validate the efficacy of zorevunersen. The Company remains dedicated to addressing neurological disorders, aiming to bring innovative solutions to market.

While these developments show substantial promise, Stoke Therapeutics emphasizes that any forward-looking statements come with inherent risks and uncertainties. The Company continues to work diligently to navigate these challenges and pursue its goals.

For further details on the recent advancements at Stoke Therapeutics and the potential implications for Dravet syndrome treatment, investors and stakeholders are welcome to attend the virtual event scheduled for Monday, December 9, 2024.

This press release contains forward-looking statements and interested parties are encouraged to stay updated on Stoke Therapeutics’ ensuing progress and future developments.

This article was generated by an automated content engine and was reviewed by a human editor prior to publication. For additional information, read Stoke Therapeutics’s 8K filing here.

Stoke Therapeutics Company Profile

Stoke Therapeutics, Inc, an early-stage biopharmaceutical company, develops medicines to treat the underlying causes of severe genetic diseases in the United States. The company utilizes its proprietary targeted augmentation of nuclear gene output to develop antisense oligonucleotides to selectively restore protein levels.