top of page

Anavex Life Sciences Announces Notice of Allowance for U.S. Patent Application with Expanding Claims Covering ANAVEX®2-73 (blarcamesine) for the Treatment of Rett Syndrome and other Neurodevelopmental Disorders

Issuance of Its Newest U.S. Patent Will Strengthen Anavex’s Compounds Intellectual Property Portfolio


NEW YORK – November 30, 2020

Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) disorders, today announced it received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for its patent application number 16/717,921 expanding coverage of treatment methods for ANAVEX®2-73 (blarcamesine) and related sigma-1 receptor agonist drug candidates, for treating a range neurodevelopmental disorders including Rett syndrome, autism spectrum disorder, Angelman syndrome, cerebral palsy and multiple sclerosis, among other indications.

Data suggests, that activation of the sigma-1 receptor is pivotal to restoring neural cell homeostasis and promoting neuroplasticity.[1]

This newest U.S. patent is expected to remain in force at least until 2037, not including any patent term extensions. Together with U.S. Patent No. 10,507,196 granted to Anavex in January 2020, the new patent will expand claim coverage for use of Anavex’s sigma-1 receptor agonists in oral, transdermal and parenteral compositions as well as for both pediatric and adult patients.

“This patent allowance by the USPTO is not only another important milestone in protecting the commercial potential of ANAVEX®2-73 (blarcamesine) and Anavex’s other lead compounds, but along with other recent allowances, demonstrates our strong overall commitment to protecting the innovation and commercial opportunity of our entire product portfolio,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex.

ANAVEX®2-73 (blarcamesine) is currently evaluated for Rett syndrome in one completed and two ongoing placebo-controlled clinical studies. In the U.S. Phase 2 study (ANAVEX®2-73-RS-001) [2] adult Rett syndrome patients were treated with low-dose ANAVEX®2-73 (blarcamesine) oral liquid formulation, while the subsequent international AVATAR Phase 2 adult Rett syndrome study (ANAVEX®2-73-RS-002)[3] and EXCELLENCE Phase 2/3 pediatric Rett syndrome study (ANAVEX®2-73-RS-003)[4] is treating patients with high-dose ANAVEX®2-73 (blarcamesine) oral liquid formulation. ANAVEX®2-73 (blarcamesine) had previously received Fast Track designation, Rare Pediatric Disease designation and Orphan Drug designation from the FDA for the treatment of Rett syndrome.

About Rett Syndrome

Rett syndrome is a devastating, non-inherited genetic post-natal progressive neurodevelopmental disorder that occurs almost exclusively in girls and leads to severe impairments, affecting nearly every aspect of the child’s life: their ability to speak, walk, eat and easily breathe. The hallmark of Rett syndrome is near constant repetitive hand movements while awake. The disease is characterized by normal early growth and development (6 to 18 months) followed by a slowing of development, loss of purposeful use of the hands, distinctive hand movements, autistic features, slowed brain and head growth, ataxia, seizures and intellectual disability.

Rett syndrome is caused by mutations in the MECP2 gene and strikes all racial and ethnic groups. The disease occurs worldwide in approximately one in every 10,000 to 15,000 live births. There is currently no cure for Rett syndrome.

About Anavex Life Sciences Corp.

Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company dedicated to the development of differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, pain and various types of cancer. Anavex’s lead drug candidate, ANAVEX®2-73 (blarcamesine), recently completed successfully a Phase 2a clinical trials for Alzheimer’s disease and a Phase 2 proof-of-concept study in Parkinson’s disease dementia. ANAVEX®2-73 (blarcamesine) is an orally available drug candidate that restores cellular homeostasis by targeting sigma-1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer’s disease. ANAVEX®2-73 (blarcamesine) also exhibited anticonvulsant, anti-amnesic, neuroprotective and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson’s Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX®2-73 (blarcamesine) for the treatment of Parkinson’s disease. ANAVEX®3-71, which targets sigma-1 and muscarinic receptors, is a promising clinical stage drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimer’s disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid and tau pathologies. In preclinical trials, ANAVEX®3-71 has shown beneficial effects on mitochondrial dysfunction and neuroinflammation. Further information is available at You can also connect with the company on Twitter, Facebook and LinkedIn.

Forward-Looking Statements

Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks set forth in the Company’s most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Anavex Life Sciences Corp. undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.

For Further Information:

Anavex Life Sciences Corp.
Research & Business Development
Toll-free: 1-844-689-3939

Investors & Media:


[1] Advances in Experimental Medicine and Biology Volume 964 (2017) Sigma Receptors: Their Role in Disease and as Therapeutic Targets.

[2] Identifier: NCT03758924

[3] Identifier: NCT03941444

[4] Identifier: NCT04304482

bottom of page