Anavex Life Sciences Corp. announced the completion of dosing of all participants of the placebo-controlled EXCELLENCE Phase 2/3 study ANAVEX(R) 2-73-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.

ANAVEX(R) 2-73 (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. The completion of the randomized, placebo-controlled EXCELLENCE Phase 2/3 study ANAVEX(R) 2-73-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. (ANAVEX(R) 2-73-RS-001)(2), and Phase 3 AVATAR (ANAVEX(R) 2-73-RS-002)(3) studies in adult patients with Rett syndrome. The multi-center, double-blind clinical EXCELLENCE study (ANAVEX(R) 2-73-RS-003)(4) in pediatric patients is measuring safety, tolerability, and efficacy of daily oral ANAVEX(R) 2-73 (blarcamesine) doses or placebo.

After completing the double-blind study, eligible participants are able to join a voluntary open-label extension study of ANAVEX(R) 2-73 (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.

ANAVEX(R) 2-73 (blarcamesine) had previously received Fast Track designation, Rare Pediatric Disease designation and Orphan Drug designation from the FDA for the treatment of Rett syndrome.