Anavex Life Sciences (NASDAQ:AVXL) dosed the first patient in its Phase 2 trial of ANAVEX2-73 for the treatment of Rett syndrome.
Rett syndrome is a genetic neurodevelopmental disorder caused by mutations in the MECP2 gene, and is characterized by severe physical and intellectual impairments. The only treatment for Rett syndrome is symptomatic; there is currently no cure.
ANAVEX2-73, which has received FDA orphan drug designation, is an orally-administered liquid designed to activate the sigma-1 receptor protein, which is involved in restoring homeostasis.
“We have made great effort to develop a patient-centric clinical trial, using our learnings from broad dialogue with the Rett syndrome community,” Christopher Missling, Anavex’s president and CEO, said in a statement.
The trial will enroll some 30 patients 18 years of age and older to receive ANAVEX2-73 once daily, or placebo, for up to seven weeks.
