Stoke Therapeutics

Stoke Therapeutics aligns with FDA, EMA, and PMDA for Phase 3 EMPEROR study of zorevunersen, targeting Dravet syndrome in children. The study aims to reduce major motor seizure frequency and improve cognition and behavior, with Phase 3 initiation planned for mid-2025. Zorevunersen received FDA Breakthrough Therapy designation, indicating potential for significant improvement over existing treatments.

Stoke Therapeutics announced alignment with FDA, EMA, and PMDA on the Phase 3 EMPEROR study design for zorevunersen, targeting Dravet syndrome. The study will assess seizure frequency reduction and cognitive/behavioral improvements in children and adolescents. FDA Breakthrough Therapy designation accelerates development, with Phase 3 initiation planned for mid-2025.

Stoke Therapeutics finalized the Phase 3 EMPEROR study protocol for zorevunersen, targeting Dravet syndrome treatment. The study, starting mid-2025, will assess seizure reduction and cognitive improvements in children and adolescents. Zorevunersen, with FDA Breakthrough Therapy Designation, showed an 87% median seizure reduction in earlier studies and was well tolerated.

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.

Stoke Therapeutics develops medicines for severe genetic diseases using antisense oligonucleotides. Its lead candidates, STK-002 and STK-001, target autosomal dominant optic atrophy and Dravet syndrome, respectively. The company collaborates with Acadia Pharmaceuticals on RNA-based treatments for neurodevelopmental diseases, headquartered in Bedford, MA.

Stoke Therapeutics, Inc., founded in 2014 by Isabel Aznarez and Adrian R. Krainer, focuses on developing treatments for genetic diseases, targeting tissues like the CNS, eye, kidney, and liver. Based in Bedford, MA.

FDA grants Breakthrough Therapy designation to Stoke Therapeutics' zorevunersen (STK-001) for Dravet syndrome, showing potential as a disease-modifying therapy. Shares rose 12.1% on Dec. 4. Zorevunersen demonstrated substantial reductions in seizure frequency and cognitive improvements in phase I/IIa and OLE studies. Stoke is discussing a global phase III study with FDA and other agencies, with an update expected by 2024-end.

Stoke Therapeutics received FDA Breakthrough Therapy Designation for zorevunersen, its Dravet syndrome treatment, potentially expediting its development. Clinical trials showed significant seizure reduction and cognitive improvements. The company plans a global Phase 3 study, with an update expected by year-end. Zorevunersen targets the genetic cause of Dravet syndrome, aiming to restore normal protein levels.

Stoke Therapeutics received FDA Breakthrough Therapy Designation for zorevunersen, targeting Dravet syndrome with SCN1A gene mutations. Clinical trials showed significant seizure reduction and cognitive improvements. Plans for a Phase 3 study are underway, aiming to address the disease's root cause beyond current treatments.