Anavex life sciences announces publication of foundational data for anavex®2-73 (blarcamesine) in rett syndrome

Anavex life sciences corp. announced that preclinical data of anavex®2-73 (blarcamesine) in rett syndrome were published in the peer-reviewed journal, pharmacology, biochemistry and behavior. rett syndrome, a rare genetic neurological disorder that afflicts mostly girls, is caused by a genetic defect associated with severe physical and cognitive impairments. the study evaluated doses of anavex®2-73 (blarcamesine) in heterozygous mecp2 mice, a validated animal model for the disease, which resulted in improvements in motor, sensory, and autonomic phenotypes related to the disease. these included motor coordination and balance, acoustic and visual responses, and other improvements. anavex®2-73 (blarcamesine) also showed a good safety profile. the study, anavex®2-73 (blarcamesine), a sigma-1 receptor agonist, ameliorates neurologic impairments in a mouse model of rett syndrome, served as a proof-of-concept for the ongoing phase 2 anavex2-73-rs-001 study (nct03758924) as well as for upcoming pediatric studies. preliminary results from the first cohort of this study were recently presented at the 6th annual european rett syndrome conference. the data described significant improvements in the two global efficacy endpoints: the rett syndrome behaviour questionnaire (rsbq) total score and the clinical global impression – improvement (cgi-i); as well as in the rsbq hand behaviours and the rsbq breathing abnormalities subscale scores. efficacy signals on both caregiver- and clinician-based measures of severity also correlated with plasma changes of key biomarkers related to disease pathogenesis.
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