Blarcamesine Reduces Rett Syndrome Symptoms 

Blarcamesine (Anavex2-73; Anavex Life Sciences Corp, New York, NY), once daily oral liquid doses of up to 30mg, demonstrated statistically significant reductions in Rett syndrome symptoms with related changes in potential biomarkers, GABA and L-Alpha-aminoadipic acid (L-AAA), of disease pathology. The study results showed consistent improvements in Rett Syndrome Behavioral Quotient (RSBQ) AUC (P=.037), ADAMS (P=.010) and Clinical Global Impressions scale (CGI) (P=.037) response. Blarcamesine induced a statistically significant and clinical meaningful improvement in 72.2% of participants as compared with 38.5% on placebo; (P=.037) with a Cohen’s d effect size of 1.91.

For the Anxiety, Depression, and Mood (ADAM) Scale, a significantly higher proportion of participants with Rett syndrome treated with blarcamesine (52.9%) than placebo-treated participants (8.3%) showed improvement (P= .010). This data corresponds to a Cohen’s d effect size of 0.609. 

Professor Terence O’Brien, MD, FRACP, chair of Medicine and Head, The Central Clinical School, Monash University, program director of Alfred Brain & deputy director of Research at Alfred Health and principal investigator of the study commented: “The outcome of this trial has confirmed the promising results of the early lower-dose study in adults with Rett syndrome. Anavex2-73 was not only safe, but it also demonstrated clinically meaningful improvements in multiple common areas of impairment, which are known to impair the quality of life of girls and women affected by the disorder.”

Professor Paramala J Santosh, MBBS, Dip NB (Psych), MD, PhD, FRCPsych, Developmental Neuropsychiatry & Psychopharmacology, Department of Child and Adolescent Psychiatry at King's College London added: “These exciting results indicate a high likelihood of marked improvements in younger, usually more drug-responsive, patients with Rett syndrome, such as those participating in the ongoing pediatric EXCELLENCE study.”

Blarcamesine demonstrated dose-related significant improvement in overall Quality of Life (QoL) measured with Child Health Questionnaire - Parent Form 50 (CHQ-PF50) (P= 0.030).

Data suggest that activation of sigma-1 receptor (SIGMAR1) results in the restoration of homeostatic function within the body. The activation is pivotal to restoring neural cell balance and the promotion of neuroplasticity.