Phase III AVATAR trial of ANAVEX 2-73 shows significant improvement in Rett Syndrome.

Convenient once daily oral liquid doses of up to 30mg ANAVEX 2-73 was well tolerated with very good medication compliance. Rett syndrome is a chronic CNS disease caused by a spontaneous mutation of one gene, MECP2.

ANAVEX 2-73 activates the sigma-1 receptor (SIGMAR1). Data suggests that activation of SIGMAR1 results in the restoration of homeostatic function within the body and is pivotal to restoring neural cell balance and the promotion of neuroplasticity. Recent independent findings strengthen the understanding of the beneficial effects of SIGMAR1 activation as a compensatory mechanism to chronic CNS diseases. In the primary endpoint, RSBQ AUC, ANAVEX 2-73 induced a statistically significant and clinical meaningful improvement in 72.2% of patients as compared to 38.5% on placebo; (p = 0.037) with a Cohen’s d effect size of 1.91 (very large). The secondary efficacy endpoints also demonstrated statistically significant and clinical meaningful improvements.

For the ADAMS, a measure of emotional behavior symptoms, a significantly higher proportion of ANAVEX 2-73 treated adult patients with Rett syndrome (52.9%) than placebo-treated patients (8.3%) showed improvement (p = 0.010), which corresponded to a Cohen’s d effect size of 0.609 (large). For the CGI-I, more patients achieved clinically meaningful CGI-I response over the treatment duration in the ANAVEX 2-73-treated group (72.2%) than in the placebo group (38.5%); (p = 0.037) with a Cohen’s d effect size of 1.91 (very large).

Based on the results in this Phase III study (ANAVEX 2-73-RS-002)3 and the prior successful U.S. Phase III (ANAVEX 2-73-RS-001)4 study in adult patients with Rett syndrome, Anavex is planning to meet with FDA to discuss the approval pathway.