The first patient has been dosed in a Phase 2/3 clinical trial, EXCELLENCE, evaluating Anavex Life Sciences’ (NASDAQ:AVXL) lead drug Anavex 2-73 (blarcamesine) in patient with Rett syndrome, a rare inherited neurological disorder occurring almost exclusively in girls that leads to severe impairments.
The primary endpoints of the 69-subject study are the changes from baseline to end of treatment in two metrics, Rett Syndrome Behavior Questionnaire and CGI-I score.
The estimated completion date is July 2021.
Orally available blarcamesine is a sigma-1 receptor (S1R) agonist. S1Rs play a key role in the modulation of neurotransmission. It is also being investigated for the potential treatment of Parkinson’s disease dementia and mild-to-moderate Alzheimer’s disease.
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