Ed Kaye, Stoke Therapeutics CEO

Stoke touts ear­ly signs of ef­fi­ca­cy for Dravet syn­drome drug

Two and a half years af­ter dri­ving his an­ti­sense oligonu­cleotide plat­form to Wall Street, Stoke Ther­a­peu­tics CEO Ed Kaye is paint­ing a fuller pic­ture of the com­pa­ny’s first clin­i­cal da­ta. And though the tri­al wasn’t pow­ered to de­tect sta­tis­ti­cal sig­nif­i­cance, Kaye says the read­out shows ear­ly signs of ef­fi­ca­cy in kids with a rare, drug-re­sis­tant form of epilep­sy

STK-001 was well-tol­er­at­ed in sin­gle and mul­ti­ple dos­es in 22 Dravet syn­drome pa­tients be­tween the ages of 2 and 18 years old, Stoke an­nounced on Fri­day. What’s more, 12 of the 17 evalu­able pa­tients at the time (70.6%) saw re­duc­tions from base­line in con­vul­sive seizure fre­quen­cy, ac­cord­ing to the com­pa­ny.

Endpoints News

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.