Anavex Life Sciences Says Rett Syndrome Trial Meets Primary & Secondary Goals

Anavex Life Sciences Corp AVXL reported topline data from the Phase 3 AVATAR trial of ANAVEX2-73 (blarcamesine) in adult female patients with Rett syndrome.

• The data demonstrated a statistically significant improvement over placebo for the primary efficacy endpoint and all the secondary efficacy endpoints. 
• Convenient once-daily oral liquid doses of up to 30mg ANAVEX 2-73 were well tolerated. 
• STAT News' Adam Feuerstein Tweets '...Anavex changed the primary and secondary endpoints of this Rett study on January 18, allowing it to claim success when the drug most likely failed. This press release is entirely misleading.'
• Rett syndrome is a chronic CNS disease caused by a spontaneous mutation of one gene, MECP2.
• Related: Anavex Life's Rett Syndrome Candidate Shows Promising Biomarker Correlating Efficacy Data.
• In the primary endpoint, Rett Syndrome Behaviour Questionnaire (RSBQ) AUC, ANAVEX 2-73 induced a statistically significant and clinically meaningful improvement in 72.2% of patients compared to 38.5% on placebo. 
• For 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).
• The placebo-controlled EXCELLENCE Phase 2/3 pediatric Rett syndrome study (ANAVEX 2-73-RS-003) is currently ongoing and is evaluating ANAVEX 2-73 for Rett syndrome patients ages 5 to 17.
• Also See: Anavex Life's Neurodegenerative Disease Candidate Meets Primary Endpoint In Early-Stage Study.
• Price Action: AVXL shares are down 21.20% at $10.30 during the market session on the last check Tuesday.