Primary and all secondary efficacy and safety endpoints met for the treatment of adult patients with Rett syndrome, with consistent improvements in RSBQ AUC (p = 0.037), ADAMS (p = 0.010) and CGI-I (p = 0.037) response
Efficacy endpoints demonstrated statistically significant and clinically meaningful reductions in Rett syndrome symptoms with related changes in potential biomarkers (GABA and L-AAA) of disease pathology
Key milestone met to advance regulatory approval pathway for adult patients with Rett syndrome
anavex.com/post/anavex-2-73...
Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) disorders, today reported positive top-line results from the Phase 3 randomized, double-blind, placebo-controlled AVATAR trial of ANAVEX®2-73 (blarcamesine) in adult female patients with Rett syndrome and demonstrated a statistically significant improvement over placebo for the primary efficacy endpoint as well as for all the secondary efficacy endpoints.
Anavex’s lead drug candidate, ANAVEX®2-73 (blarcamesine), successfully completed a Phase 2a clinical trial for Alzheimer’s disease and recently a Phase 2 proof-of-concept study in Parkinson’s disease dementia and a Phase 2 study in adult patients with Rett syndrome.