The trial (NCT03758924) assessing the safety, tolerability, and efficacy of Anavex 2-73 in 15 adults (18 and older) with Rett syndrome is going on at three clinical sites in the U.S. One site, the University of Alabama at Birmingham, is currently recruiting. More information on enrollment can be found on the trial’s official page.
Participants will be randomly assigned to take an oral solution of Anavex 2-73 or a placebo daily for seven weeks. Those who complete the study and show a good safety and tolerability profile will be eligible to continue to receive Anavex 2-73 in a 12-week open-label extension study.
“We are very pleased to report the first patient dosing of the ANAVEX 2-73 Rett syndrome Phase 2 study in the U.S.,” Christopher U. Missling, PhD, president and chief executive officer of Anavex, said in a press release. “This is an important step toward achieving clinical data for the third indication for ANAVEX 2-73 also incorporating genomic precision medicine biomarkers.”
Anavex 2-73 is an engineered small molecule that can modulate several cellular processes relevant to neurodegenerative and neurodevelopmental diseases through the activation of the sigma-1 receptor (S1R) protein. The investigational medicine is thought to help restore cellular balance by targeting failed protein folding, oxidative stress, mitochondrial dysfunction, and inflammation.
Preclinical studies with a genetically induced Rett syndrome mouse model — the animals carried genetic mutations on the MECP2 gene, leading to neurological symptoms similar to those observed in humans — showed that treatment with Anavex 2-73 significantly improved motor and reflex response to external auditory stimulation, compared with placebo. Treated mice also showed improved balance and gait patterns.
This Phase 2 trial is part of a large program testing Anavex 2-73 for Rett syndrome, including a placebo-controlled study to evaluate the therapy’s safety and efficacy when administered for three months.