Zorevunersen

Stoke Therapeutics announced a webcast on January 7, 2025, to discuss regulatory alignment for a Phase 3 study of zorevunersen, a potential first disease-modifying treatment for Dravet syndrome. Zorevunersen aims to restore protein levels to treat this severe genetic epilepsy, leveraging RNA medicine and Stoke's TANGO approach.

Stoke Therapeutics aligns with FDA, EMA, and PMDA on Phase 3 EMPEROR study design for zorevunersen, targeting Dravet syndrome. The study aims to reduce major motor seizure frequency and improve cognition and behavior in children and adolescents. FDA Breakthrough Therapy designation accelerates development, with Phase 3 expected to start mid-2025.

Stoke Therapeutics finalized the Phase 3 EMPEROR study design for zorevunersen, aiming to treat Dravet syndrome by reducing seizures and improving cognition and behavior. The study, set to start mid-2025, targets children and adolescents, with data expected by end of 2027. Zorevunersen has shown promise in reducing seizure frequency and improving patient outcomes.

Stoke Therapeutics aligns with FDA, EMA, and PMDA for a Phase 3 study of zorevunersen, aiming to treat Dravet syndrome by reducing seizures and improving cognition and behavior in children and adolescents. The study, set to begin mid-2025, targets a 52-week treatment period with data expected by end of 2027.

Stoke Therapeutics announced new OLE study data showing zorevunersen, at 70mg initial doses followed by 45mg maintenance, significantly reduces convulsive seizure frequency in Dravet Syndrome patients, supporting its potential as a disease-modifying treatment.

Leerink analyst Marc Goodman initiated coverage on Stoke Therapeutics with an Outperform rating and an $18 price target, highlighting the unmet need for Dravet syndrome treatments. STK-001, a disease-modifying therapy, is seen as a potential blockbuster for its first-in-class approach.