First Patient Dosed in Phase 2 Trial of Experimental Rett Syndrome Treatment

According to a story from, the biopharmaceutical company Anavex Life Sciences Corp. has just announced that the first patient has been dosed in its phase 2 clinical trial, which is testing the company’s experimental product candidate ANAVEX®2-73 as a treatment for Rett syndrome. Anavex is dedicated to the development of therapies for a variety of neurodevelopmental and neurodegenerative diseases.

About Rett Syndrome

Rett syndrome is a brain disorder that become evident early in the lives of its female patients. The disease is caused by a genetic mutation that affects the MECP2 gene. This gene is found on the X chromosome. Boys who have this mutation die soon after being born, so Rett syndrome exclusively affects girls. It occurs as a spontaneous mutation in the vast majority of cases, and is rarely inherited from parents. Symptoms include sleeping issues, difficulty speaking, poor coordination, scoliosis, seizures, small head size, slow growth, and repetitive movements. There is no cure for Rett syndrome, and management focuses on maintaining function and alleviating symptoms. Life expectancy for patients is around 40 years. Death often occurs sponatenously, and is often linked to brainstem malfunction, gastric perforation, or cardiac arrest. To learn more about Rett syndrome, click here.

About The Clinical Trial

The phase 2 trial is expected to include around 30 Rett syndrome patients aged 18 or older. This study will measure the safety and efficacy of a liquid formulation of Anavex2-73. All patients who participate in the trial will be able to continue treatment in an open label extension following the conclusion of the trial. The drug has earned Orphan Drug designation as a treatment for Rett syndrome from the US Food and Drug Administration (FDA). Anavex also plans to develop Anavex2-73 to treat Alzheimer’s and dementia associated with Parkinson’s disease.

About ANAVEX®2-73

The experimental drug Anavex2-73 functions to restore homeostatic states to cells by targeting the muscarinic and sigma-1 receptors. In preclinical research, this therapy demonstrated the potential to halt or reverse the course of Alzheimer’s disease. The drug has also displayed potential efficacy in treating epilepsy, depression, and amnesia in animal models.

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