Stoke Therapeutics Advances Dravet Syndrome Drug to Phase III Trials After FDA Lifts Partial Clinical Hold

Clinical Hold Lifted:
The FDA has removed the partial clinical hold on Stoke Therapeutics' Dravet syndrome drug, zorevunersen (STK-001), allowing the company to proceed with Phase III trials.

Phase III Plans:
Stoke Therapeutics is planning to discuss Phase III registrational plans with the FDA in the second half of 2024.

Drug Mechanism:
Zorevunersen acts as an SCN1A activator, increasing the production of Scn1a messenger RNA and the Nav1.1 protein, crucial for sodium channel function in the brain.

Designations:
Zorevunersen has been granted orphan drug designation by the FDA and EMA, and rare pediatric disease designation by the FDA.

Clinical Trials:
Positive efficacy data from Phase I/IIa MONARCH and open-label extension SWALLOWTAIL trials showed reduction in convulsive seizure frequency, despite some adverse events.

Stock Performance:
Stoke Therapeutics' stock price increased by 5.6% following the announcement, reflecting investor optimism about the drug's potential.

Future Milestones:
The company anticipates completing Phase 1/2a studies in 2023 to initiate a Phase 3 program in 2024.