Independent Data Review Gives Go-Ahead for Rett Syndrome Clinical Trial

According to a story from Financial Buzz, the biopharmaceutical company Anavex Life Sciences Corp. has recently announced that the Independent Data Safety Monitoring Board (DSMB) has completed its review of the company’s preliminary phase 2 clinical trial data. This trial is testing the company’s experimental drug candidate blarcamesine as a treatment for the rare genetic disorder Rett syndrome. Anavex is focused on the development of therapies for neurological diseases such as Parkinson’s disease, Rett syndrome, Alzheimer’s disease, and others.

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 the 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 this rare disease, click here.

About The Trial

The board recommended that the trial continue to proceed without any modifications. The goal of these reviews of the data is the protection of patient interests and ensuring the integrity of the data findings in the trial. The clinical trial included a total of 15 Rett syndrome patients who were treated with blarcamesine (also known as ANAXEX 2-73) over a period of seven weeks. All participants in this placebo controlled study are eligible to receive the treatment in an open label extension period.

About ANAVEX 2-73 (Blarcamesine)

Blarcamesine is a liquid, orally available medication that activates a protein called the Sigma-1 receptor (S1R). This protein plays a role in the maintenance of homeostasis, which is the steady state of internal chemical and physical conditions that are maintained by the body. The drug has shown some potential in the treatment of Alzheimer’s in the clinical trial setting and positive effects in mouse models of Rett syndrome. Blarcamesine has also earned Orphan Drug designation for Rett syndrome from the US Food and Drug Administration (FDA).


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