Anavex Life Sciences Announces Completion of ANAVEX()2-73 (blarcamesine) EXCELLENCE Phase 2/3 Rett Syndrome Clinical Trial

NEW YORK, June 06, 2023 (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 announced the completion of dosing of all participants of the placebo-controlled EXCELLENCE Phase 2/3 study ANAVEX2-73-RS-003 in pediatric patients with Rett syndrome. The Company expects to announce topline results from this study in the second half of this year.

ANAVEX2-73 (blarcamesine) is an orally available, small-molecule activator of the sigma-1 receptor (SIGMAR1) which, data suggest, is pivotal to restoring cellular homeostasis and promoting neuroplasticity.¹

The completion of the randomized, placebo-controlled EXCELLENCE Phase 2/3 study ANAVEX2-73-RS-003 for the treatment of 92 pediatric patients with Rett syndrome ages ≥ 5 years to 17 (inclusive) was preceded by the successful completion of both placebo-controlled Phase 2 U.S. (ANAVEX2-73-RS-001)², and Phase 3 AVATAR (ANAVEX2-73-RS-002)³ studies in adult patients with Rett syndrome.

The multi-center, double-blind clinical EXCELLENCE study (ANAVEX2-73-RS-003)⁴ in pediatric patients is measuring safety, tolerability, and efficacy of daily oral ANAVEX2-73 (blarcamesine) doses or placebo. After completing the double-blind study, eligible participants are able to join a voluntary open-label extension study of ANAVEX2-73 (blarcamesine).

In communication with the FDA, the Company received the Agency’s input on the study endpoints, which were utilized in this clinical study. The Rett Syndrome Behavior Questionnaire (RBSQ) total score and Clinical Global Impression Improvement Scale (CGI-I) score are co-primary endpoints in the statistical analysis plan with specified linear mixed-effects models for repeated measures (MMRM) as the primary analysis methods.

ANAVEX2-73 (blarcamesine) had previously received Fast Track designation, Rare Pediatric Disease designation and Orphan Drug designation from the FDA for the treatment of Rett syndrome.

“We would like to thank the investigators and clinical site coordinators as well as all the participating families for their dedication to this clinical study completed with ANAVEX2-73 (blarcamesine),” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “Rett syndrome is a devastating, non-inherited genetic post-natal progressive neurodevelopmental disorder that occurs almost exclusively in girls and leads to severe impairments, affecting nearly every aspect of the child’s life, hence we continue our fast-paced development program of ANAVEX2-73 (blarcamesine) in Rett syndrome.”