US Patent Issued for Anavex 2-73 for Treatment of Neurodevelopmental Disorders, Including MS

Anavex Life Sciences has received a U.S. patent covering the use of Anavex 2-73 (blarcamesine) for the treatment of several neurodegenerative and neurodevelopmental conditions, including multiple sclerosis (MS) and Rett syndrome.

Issued by the U.S. Patent and Trademark Office, the new patent (no. 10,507,196) covers therapeutic methods based on Anavex 2-73 that are aimed at treating a wide variety of conditions involving the brain and affecting development. In addition to MS and Rett syndrome, some of the other conditions included are autism, Angelman syndrome, and cerebral palsy.

The company expects the patent to remain in force until at least 2037.

“We are extremely pleased with the continued development of the patent portfolio for Anavex 2-73 (blarcamesine),” Christopher U. Missling, PhD, president and CEO of Anavex, said in a press release. “This new issuance of the U.S. patent continues to expand the breadth and depth of our intellectual property, and is another step in the development of a robust patent portfolio” related to the drug, he said.

Anavex 2-73 is an orally available, small-molecule activator of a protein called the sigma-1 receptor (S1R), which is essential to ensure correct protein folding within nerve cells and to promote neuroplasticity — the brain’s ability to adapt or rewire following damage to preserve function.

The activation of S1R mediated by Anavex 2-73 is also thought to decrease mitochondria dysfunction, oxidative stress, and inflammation in the brain, potentially benefiting people with conditions like MS that damage the nervous system.

Mitochondria are the cell compartments responsible for the production of energy in the body. Oxidative stress refers to cellular damage that occurs as a result of having high levels of oxidant molecules.

Anavex 2-73 has been granted orphan drug status and rare pediatric disease designation by the U.S. Food and Drug Administration for the treatment of Rett syndrome.

The therapy was found to have beneficial effects in a mouse model of Rett, improving the animals’ reflexes and walking patterns. In addition, early data from an ongoing Phase 2 clinical trial (NCT03758924) testing Anavex 2-73 in adults with Rett suggested that the therapy was safe and effective at reducing disease symptoms.

Another ongoing Phase 2 trial, AVATAR (NCT03941444), is investigating the safety, tolerability, and efficacy of Anavex 2-73 in adults with Rett. A third study, called EXCELLENCE (Anavex-2-73-RS-003) is planned to start soon. That study will assess the effects of the medication in approximately 69 children with Rett. More information about planned clinical trials of Anavex 2-73 in Rett syndrome can be found here.

Anavex 2-73 has shown neuroprotective, anti-amnesic, anti-depressant, and anticonvulsant properties in several animal models, evidence of its potential to treat disorders affecting the central nervous system, according to the company.