Anavex Life Sciences Announces ANAVEX2-73 (Blarcamesine) Biomarker Correlated with Efficacy Endpoints in Placebo-Controlled U.S. Phase 2 Clinical Trial for the Treatment of Adult Patients with Rett Syndrome

ANAVEX2-73 treatment resulted in significant increase in the expression of the SIGMAR1 mRNA biomarker that significantly correlated with improvements in the two primary clinical efficacy endpoints RSBQ (p = 0.035) and CGI-I (p = 0.029)

Data strengthens milestone to advance regulatory approval pathway for adult patients with Rett syndrome and continued development in other autism spectrum disorders

NEW YORK, June 21, 2021 (GLOBE NEWSWIRE) -- 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 predictive biomarker of response established with SIGMAR1 mRNA expression correlates significantly with responses in primary clinical efficacy endpoints from the U.S. Phase 2 randomized, double-blind, placebo-controlled trial of ANAVEX2-73 (blarcamesine) in adult female patients with Rett syndrome.

ANAVEX2-73 activates the sigma-1 receptor (SIGMAR1). Data suggests that activation of SIGMAR1 results in the restoration of complete housekeeping function within the body and is pivotal to restoring neural cell homeostasis and promoting neuroplasticity.¹ Recent independent findings strengthen the understanding of the beneficial effect of SIGMAR1 activation as compensatory mechanism to chronic CNS diseases.²

Rett syndrome is a chronic CNS disease caused by a spontaneous mutation of one gene, MECP2. This study demonstrates for the first-time that a biomarker correlates with clinical efficacy in Rett syndrome. ANAVEX2-73 treatment resulted in increases in the mRNA expression of SIGMAR1, the gene coding for the receptor targeted by ANAVEX2-73, which correlated with clinical efficacy as measured by both primary efficacy endpoints (ITT population), namely RSBQ (p = 0.035) and CGI-I (p = 0.029).

In addition, prespecified patients with WT SIGMAR1 in the clinical trial demonstrated a clinically meaningful and statistically significant 14.5-point (p = 0.009) improvement over placebo in the RSBQ total score, the trial’s key efficacy endpoint. This magnitude of the improvement with ANAVEX2-73 compares favorably to published data currently in clinical development, which reported an average difference of 4.4 points in RSBQ total score versus placebo, despite an advantage of higher dose and lower age compared to ANAVEX2-73-RS-001 trial.³