Stoke Therapeutics

Biogen and Stoke Therapeutics Collaborate to Advance Zorevunersen for Dravet Syndrome

5 months ago

• Biogen and Stoke Therapeutics will collaborate to develop and commercialize zorevunersen for Dravet syndrome outside the U.S., Canada, and Mexico. • Stoke's Phase 3 EMPEROR study of zorevunersen, expected to begin in mid-2025, aims to reduce seizure frequency and improve cognition and behavior in children with Dravet syndrome. • Zorevunersen, an antisense oligonucleotide targeting the SCN1A gene, has shown promising results in earlier trials, including an 87% median reduction in convulsive seizure frequency. • Biogen will provide Stoke with an upfront payment of $165 million, potential milestone payments up to $385 million, and tiered royalties on net sales in Biogen's territory.

Zorevunersen Receives FDA Breakthrough Therapy Designation for Dravet Syndrome

6 months ago

• Stoke Therapeutics' zorevunersen has received FDA Breakthrough Therapy Designation for Dravet syndrome with confirmed SCN1A gene mutation. • Clinical data from Phase 1/2a and open-label extension studies demonstrated substantial seizure reduction and improvements in cognition and behavior. • Zorevunersen is a novel antisense oligonucleotide designed to upregulate NaV1.1 protein expression, addressing the underlying cause of Dravet syndrome. • Stoke plans to provide an update on its global Phase 3 registrational study plans by the end of the year.

Stoke Therapeutics Advances Dravet Syndrome Treatment with Zorevunersen

7 months ago

• Stoke Therapeutics is progressing zorevunersen (STK-001) for Dravet syndrome, with ongoing discussions with regulatory agencies for a global Phase 3 study. • Recent data from clinical studies of zorevunersen demonstrate substantial and sustained effects across multiple measures of Dravet syndrome. • The FDA has removed the partial clinical hold on higher doses of zorevunersen, allowing for continued chronic dosing in ongoing trials. • Stoke Therapeutics reported $269.2 million in cash, cash equivalents, and marketable securities as of September 30, 2024, ensuring operations are funded through at least the end of 2025.

Stoke Therapeutics' STK-001 Shows Promising Results in Dravet Syndrome Trials

a year ago

• Stoke Therapeutics' STK-001 demonstrated substantial reductions in convulsive seizure frequency in children and adolescents with Dravet syndrome. • The treatment also showed improvements in cognition and behavior, suggesting potential disease modification in this patient population. • The FDA has allowed Stoke to proceed with a dosing regimen of 70mg followed by 45mg, paving the way for registrational studies. • Stoke Therapeutics is preparing to meet with regulatory agencies to discuss a registrational study based on the encouraging data.