Anavex Life Sciences (NASDAQ:AVXL) has received confirmation from the FDA that its Investigational New Drug application (IND) is now open for ANAVEX2-73 for the treatment of Rett syndrome, a rare and catastrophic neurodevelopmental disease.
ANAVEX2-73 activates the Sigma-1 receptor protein, which serves as a molecular chaperone and functional modulator involved in restoring homeostasis. It has already received orphan drug designation for the treatment of Rett syndrome.
Pharmacokinetic and dose finding will be investigated in a total of 15 patients over a 7-week treatment period including ANAVEX2-73-specific genomic precision medicine biomarkers.
AVXL up 34% premarket.
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