BioCentury
ARTICLE | Clinical News

Vatiquinone alpha-tocotrienol quinone: Phase II data

September 8, 2014 7:00 AM UTC

A double-blind, Italian Phase II trial in 24 female Rett syndrome patients ages 2.5-8 showed that thrice-daily 15 mg/kg EPI-743 missed the primary endpoint of improving the Rett Syndrome Clinical Severity Scale score from baseline to 6 months vs. placebo (p-value not disclosed). EPI-743 did meet the secondary endpoint of increasing head circumference from baseline to 6 months vs. placebo (0.75 vs. 0.29 cm, p=0.05). EPI-743 was well tolerated with no treatment-related serious adverse events reported. Edison said it will evaluate next steps for EPI-743 in the indication, including a potential Phase III program, in the next several months. Rett syndrome is a rare neurodevelopmental disorder that results in slow brain growth, seizures, loss of motor function and speech, and impaired respiratory function. ...