BioCentury
ARTICLE | Clinical News

Edison's EPI-743 misses Rett syndrome endpoint

September 4, 2014 12:49 AM UTC

Edison Pharmaceuticals Inc. (Mountain View, Calif.) said EPI-743 missed the primary endpoint of improving the Rett Syndrome Clinical Severity Scale score vs. placebo in a Phase II trial. The compound did meet a secondary endpoint of increasing head circumference from baseline by 0.75 cm vs. 0.29 cm for placebo (p=0.05). Edison said it is evaluating next steps for EPI-743 in the indication, including a potential Phase III program.

Rett syndrome is a rare neurodevelopmental disorder that results in slow brain growth, seizures, loss of motor function and speech, and impaired respiratory function. ...