Ed Kaye, Stoke Therapeutics CEO

Stoke teas­es ear­ly ef­fi­ca­cy da­ta for pos­si­ble PhI­II dose in lead Dravet syn­drome pro­gram

Late last year, Stoke Ther­a­peu­tics put out ear­ly da­ta for its lead pro­gram, not­ing a po­ten­tial ef­fi­ca­cy sig­nal in a hand­ful of Dravet syn­drome pa­tients. Now, the biotech is back with a first look at re­sults from the dose it may ul­ti­mate­ly take in­to a piv­otal study.

Ze­ro­ing in on a sub­group of six pa­tients ex­am­ined at the high­est dose so far — who had all re­ceived three dos­es — Stoke ob­served a me­di­an 55% re­duc­tion in con­vul­sive seizure fre­quen­cy. The re­duc­tion was seen be­tween four weeks af­ter pa­tients got their first 45 mg dose and three months af­ter re­ceiv­ing their third. They were among a to­tal of 55 pa­tients who had re­ceived at least one dose of the drug at any lev­el.

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