Zorevunersen

Stoke Therapeutics received FDA Breakthrough Therapy Designation for zorevunersen, targeting Dravet syndrome. Clinical trials show significant seizure reduction and cognitive improvements. Plans for a Phase 3 study are underway, aiming to address the disease's root cause by restoring NaV1.1 protein levels.

Stoke Therapeutics received FDA Breakthrough Therapy Designation for zorevunersen, targeting Dravet syndrome with SCN1A gene mutations. Clinical trials show zorevunersen reduces seizures and improves cognition, aiming to be the first disease-modifying treatment. A Phase 3 study update is expected by year-end.

Stoke Therapeutics is advancing RNA-based drugs, focusing on up-regulating proteins to treat rare neurological diseases like Dravet syndrome. After overcoming safety issues, they are preparing for a phase 3 trial of zorevunersen, aiming to improve cognition and behavior in patients. Stoke emphasizes innovation in RNA science, addressing manufacturing and patient access challenges, and is collaborating with Acadia Pharmaceuticals for further development.

Stoke Therapeutics plans to share seizure, cognition, and behavior data from zorevunersen studies by year-end, aiming for a Phase 3 trial. With $269.2M in cash as of September 30, 2024, the company focuses on Dravet syndrome treatment, delaying STK-002's Phase 1 study. Financials show increased R&D and G&A expenses.

Stoke Therapeutics plans Phase III trials for Dravet syndrome drug zorevunersen after FDA lifts clinical hold. Positive efficacy data from earlier trials noted, despite adverse events. Zorevunersen increases SCN1A gene activity, crucial for brain cell communication. FDA and EMA granted orphan and rare paediatric disease designations. Dravet syndrome is a severe, treatment-resistant epilepsy with poor prognosis.

Alignment with FDA, EMA, and PMDA for EMPEROR study. Zorevunersen's one-year study to assess seizure frequency reduction and cognitive-behavioral improvements in children aged 2+.

Stoke Therapeutics reported positive data from STK-001 studies in Dravet syndrome patients, showing significant seizure reduction and cognitive improvements. The FDA approved a dosing regimen, and the company plans regulatory discussions for a registrational study. STOK stock surged following the update.

STK-001, Stoke Therapeutics' treatment for Dravet syndrome, shows promise in reducing seizures in children and adolescents, with Phase 1/2a trials indicating safety and efficacy. Interim results suggest a shift towards syndrome management, with further data expected to inform Phase 3 trials. STK-001 aims to restore NaV1.1 levels, potentially improving quality of life.