Anavex Life Sciences has dosed the first patient in the Phase II/III EXCELLENCE clinical trial of ANAVEX 2-73 (blarcamesine) for the treatment of Rett syndrome.

Rett syndrome is a rare, non-inherited, genetic, progressive neurodevelopmental disease. It is known to mostly occur in girls and causes severe impairments.

The disorder develops due to mutations in the MECP2 gene. Rett syndrome currently lacks cure and treatment is symptomatic.

ANAVEX 2-73 is designed to activate the Sigma-1 receptor (S1R) protein, which acts as a molecular chaperone and functional regulator associated with restoring homeostasis.

In a mouse model of Rett syndrome, the drug candidate led to improvements at young ages of about six weeks and in adulthood of seven months. The mouse model had a heterozygous Mecp2-null mutation with neurological impairments similar to the human disorder.

Chronic oral administration of ANAXEX 2-73 demonstrated significant dose-related improvements in various motor, sensory and autonomic phenotypes related to Rett syndrome.

The Phase II/III EXCELLENCE study will examine a once-daily oral liquid ANAVEX 2-73 formulation to treat Rett syndrom in at least 69 patients over a 12-week treatment period.

Anavex said that the trial design includes genomic biomarkers of response discovered in the drug candidate’s Phase IIa Alzheimer’s disease (AD) study.

All participants in the EXCELLENCE trial will be eligible to receive ANAVEX 2-73 as part of a voluntary open label extension protocol.

Anavex Life Sciences president and CEO Christopher Missling said: “We are delighted to expand the ANAVEX 2-73 treatment programme for Rett syndrome to include pediatric patients. This is the third ongoing clinical study within the Anavex Rett syndrome programme.

“In addition to Rett syndrome, Anavex has ongoing clinical development programmes for ANAVEX 2-73 for the treatment of Alzheimer’s disease and Parkinson’s disease dementia.”

In October last year, the company launched a long-term extension study for patients with early Alzheimer’s disease and treated with ANAVEX 2-73.