Zorevunersen

Stoke Therapeutics announced a webcast on January 7, 2025, to discuss Phase 3 study alignment for zorevunersen, a potential first disease-modifying treatment for Dravet syndrome. Zorevunersen, an ASO, aims to restore NaV1.1 protein levels, reducing seizures and comorbidities in Dravet syndrome patients.

Stoke Therapeutics announced a webcast to discuss global regulatory alignment for a Phase 3 study of zorevunersen, potentially the first disease-modifying treatment for Dravet syndrome. The webcast is scheduled for January 7, 2025, accessible via Stoke's website. Zorevunersen aims to restore protein levels to treat Dravet syndrome, with FDA and EMA designations.

Stoke Therapeutics announced a webcast on January 7, 2025, to discuss Phase 3 study results of zorevunersen for Dravet syndrome, aiming to be the first disease-modifying treatment. Zorevunersen, an antisense oligonucleotide, targets the SCN1A gene to restore protein levels, reducing seizures and comorbidities.

In December 2024, the FDA made several key decisions: granted breakthrough therapy to STK-001 for Dravet syndrome, provided guidance for CNM-Au8 in ALS, aligned on AMT-130's accelerated approval for Huntington's, designated tolebrutinib for nrSPMS, placed a hold on PGN-EDO51 for DMD, and approved tirzepatide for OSA and obesity.

Skorpios Trust reduced its stake in Stoke Therapeutics Inc by 2,000,000 shares at $11.50 each, a -22.46% change, yet retains 6,906,181 shares, 13.04% of its portfolio. This strategic adjustment reflects a cautious approach amidst Stoke's mixed financial indicators.

STK-001, an antisense oligonucleotide targeting SCN1A gene, shows promise in reducing seizures and improving cognition in Dravet syndrome patients. Presented at AES 2024, it demonstrated up to 85% seizure reduction with sustained effects. Safety profile is encouraging, with a breakthrough therapy designation from FDA. Plans include a phase 3 trial aiming for rapid, lasting benefits and potential disease-modifying therapy for epilepsy.

Stoke Therapeutics secures FDA Breakthrough Therapy Designation for zorevunersen, an investigational treatment for Dravet syndrome, showing significant seizure reduction and cognitive improvements in clinical studies, positioning it as a potential first-in-class disease-modifying therapy.

The FDA granted breakthrough therapy designation to Stoke Therapeutics' STK-001 for Dravet syndrome, designed to upregulate NaV1.1 protein expression. Over 600 doses have been administered, with some patients on the drug for over 3 years. Phase 3 registrational study plans are expected by year-end. Clinical data from MONARCH and ADMIRAL trials show significant seizure reduction and improved quality of life.