Stoke Therapeutics Advances Zorevunersen for Dravet Syndrome with Positive Long-Term Study Results

What's Happening?

Stoke Therapeutics has announced positive findings from long-term open-label extension studies of zorevunersen, an investigational antisense oligonucleotide, in children and adolescents with Dravet syndrome. The studies show substantial and durable reductions in convulsive seizure frequency over three years of treatment, alongside improvements in cognition and behavior. Zorevunersen aims to treat Dravet syndrome by increasing NaV1.1 protein production in brain cells from the non-mutated copy of the SCN1A gene. The drug has received orphan drug designation from the FDA and EMA, as well as rare pediatric disease designation and Breakthrough Therapy Designation for treating Dravet syndrome. Stoke Therapeutics is collaborating with Biogen to develop and commercialize zorevunersen.

Why It's Important?

The development of zorevunersen represents a significant advancement in the treatment of Dravet syndrome, a severe neurodevelopmental disorder characterized by frequent seizures and cognitive impairments. Current treatments often fail to adequately control seizures, impacting the quality of life for patients and caregivers. Zorevunersen's potential to modify the disease and improve neurodevelopmental outcomes could offer a new therapeutic option for the estimated 16,000 individuals living with Dravet syndrome in the U.S. The collaboration with Biogen enhances the drug's commercial prospects, potentially expanding access to this innovative treatment.

What's Next?

Stoke Therapeutics plans to continue the clinical development of zorevunersen, with the first patient dosed in the global Phase 3 EMPEROR study. The company aims to present additional data at upcoming medical congresses in 2025. Stoke is also advancing STK-002, another antisense oligonucleotide, into a Phase 1 study for Autosomal Dominant Optic Atrophy, expanding its approach to new disease areas. The company anticipates funding operations through mid-2028, supported by its financial position and strategic collaborations.