Rett syndrome phase 3 trial meets primary, secondary efficacy endpoints
A once daily oral liquid dose of up to 30mg ANAVEX2-73 (blarcamesine) demonstrated statistically significant and clinically meaningful reductions in symptoms with related changes in potential biomarkers (GABA and L-AAA) of disease pathology improvement over placebo, in adult women with Rett syndrome, according to top-line results from the Phase 3 randomized, double-blind, placebo-controlled AVATAR trial.
Rett syndrome is a chronic central nervous system disease caused by a spontaneous mutation of MECP2 gene.
“The outcome of this trial has confirmed the promising results of the early lower-dose study in adults with Rett syndrome,” said Professor Terence O’Brien, MD, FRACP, Chair of Medicine and Head, The Central Clinical School, Monash University, Program Director of Alfred Brain & Deputy Director of Research at Alfred Health and Principal Investigator of the study, in a statement. “ANAVEX®2-73 was not only safe but it also demonstrated clinically meaningful improvements in multiple common areas of impairment, which are known to impair the quality of life of girls and women affected by the disorder.”
There are currently no FDA-approved drugs for Rett syndrome. ANAVEX®2-73 has Fast Track designation, Rare Pediatric Disease designation and Orphan Drug designation from the FDA for the treatment of Rett syndrome. Anavex plans to meet with the FDA to discuss the approval pathway.
Read the full press release here.