New US Patent Granted Covering Rett Therapy Anavex 2-73 Until 2037

Anavex's oral therapy aims to lessen severity of MECP2 gene disorders

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by Patricia Inácio, PhD |

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Anavex Life Sciences has been granted another U.S. patent for its lead investigational oral therapy Anavex 2-73 (blarcamesine), being developed to treat neurodevelopmental and neurological disorders, including Rett syndrome, that are driven by mutations in the MECP2 gene.

This latest of a series of patents to Anavex is expected to remain in force until at least 2037, though patent term extensions are possible.

“We are pleased with the continued development of the patent portfolio for ANAVEX 2-73 (blarcamesine). This latest patent will fortify our robust patent portfolio relating to ANAVEX 2-73,” Christopher U. Missling, PhD, president and CEO of Anavex, said in a company press release.

Anavex 2-73 is a small molecule designed to activate a protein receptor called SIGMAR1. By activating this receptor, the therapy is expected to restore nerve cell balance and boost neuroplasticity — the brain’s flexibity to change and adapt to new information and injury.

It works by lessening inflammation in the brain, reducing protein misfolding, and easing problems in mitochondria, known as the powerhouses of cells. All are implicated in Rett syndrome.

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Oral Anavex 2-73 Safely Eases Rett Symptoms, Severity in Phase 3 Trial

Issued by the U.S. Patent and Trademark Office (USPTO), patent number 11,446,275 covers the use of Anavex 2-73 and its analogs, or similar compounds.

These compounds have different administration routes: oral, transdermal or through the skin, and parenteral, meaning non-orally, such as via intravenous (into-the-vein), intramuscular, or under-the-skin (subcutaneous) administration.

The therapy is designed for diseases linked with abnormalities in the MeCP2 protein, which include — but are not limited to — Rett syndrome, Angelman syndrome, and Williams syndrome.

Other related disorders include cerebral palsy, autism spectrum disorder, and multiple sclerosis.

The analogs covered in the patent include Anavex 1-41 and Anavex 19-144, both targeting the SIGMAR1.

Overall the new patent “demonstrates our strong overall commitment to protecting the innovation and commercial opportunity of our entire product portfolio,” Missling said.

In a Phase 2 trial (NCT03758924) involving 31 women with Rett, treatment with a low dose (5 mg) of Anavex 2-73 was significantly superior to a placebo at lessening behavioral symptoms and disease severity.

A Phase 3 trial, dubbed AVATAR (NCT03941444), involved 33 women with Rett, ages 18–45, who were on stable treatment for at least four months. Each was randomly assigned to Anavex 2-73 — administered as an oral solution up to 30 mg — or a placebo solution, given every day for seven weeks.

Top-line data showed that the trial met both its primary and secondary goals, with Anavex 2-73 easing the behavioral features and severity of Rett syndrome, while improving patients’ quality of life.

Anavex 2-73 has been granted fast track, rare pediatric disease, and orphan drug designations from the U.S Food and Drug Administration for the treatment of Rett syndrome. All are designed to speed the development and regulatory approval of therapy candidates.

The therapy also is being tested in people with Alzheimer’s disease and individuals with Parkinson’s disease dementia.

Data from a Phase 2b/3 study (NCT03790709) in Alzheimer’s disease, completed in June, is expected later this year.