Anavex Proceeding with Phase 2 Trial to Assess Anavex 2-73 for Rett Syndrome

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by Alice Melao |

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Anavex 2-73 trial

After the U.S. Food and Drug Administration recently approved an investigational new drug application for Anavex 2-73, Anavex Life Sciences is proceeding with its Phase 2 clinical trial of the medication in Rett syndrome patients, the company announced.

The trial (NCT03758924) will assess the safety, tolerability, and efficacy of the investigational therapy in approximately 15 adults (18 years old and older) with Rett syndrome. Participants will be randomly assigned to take an oral solution of Anavex 2-73 or a placebo daily for seven weeks.

Researchers will test changes in patients’ behavior, sleeping patterns, frequency of seizures, as well as anxiety, depression, and mood. They will also assess treatment efficacy, based on the levels of Anavex 2-73-specific biomarkers. All patients who participate in the Phase 2 trial and show a good safety and tolerability profile will be eligible to continue treatment with Anavex 2-73 in an open-label extension study.

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.

Anavex received orphan drug designation from the FDA for Anavex 2-73 as a potential Rett syndrome treatment. Currently, the company is also exploring its potential in Alzheimer’s disease and Parkinson’s disease.

With the support of, the company studied Anavex 2-73 in a genetically induced Rett syndrome mouse model. The animals had genetic mutations on the MECP2 gene that led to neurological symptoms similar to those observed in humans.

The preclinical studies demonstrated 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.

“Administration of Anavex 2-73 results in both significant and dose-related improvements in an array of behavioral paradigms in the MECP2 Rett syndrome disease model,” the researchers wrote in the study. “These behavioral paradigms measure different aspects of muscular coordination, balance, motor learning, and muscular strengths, some of the core deficits observed in Rett syndrome.”

Anavex recently appointed Walter E. Kaufmann, MD, as chief medical officer of the company. Kaufmann was the founder and leader of RettSearch, the international consortium of Rett syndrome clinical researchers, and co-author of the current diagnostic guidelines for the disorder.

He currently leads the Rett Syndrome Molecular Biomarkers Working Group for the International Rett Syndrome Foundation and the Rett Syndrome Behaviour Questionnaire Working Group.

With Kaufmann’s “extensive expertise in Rett syndrome, and other related disorders,” the company expects to advance the clinical program of Anavex 2-73 in “both neurodevelopmental and neurodegenerative diseases,” Christopher U. Missling, PhD, president and CEO of Anavex, said in a press release.