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Anavex Life Sciences Issued New U.S. Patent for ANAVEX®2-73 Treatment of Neurodevelopmental Disorders including Rett Syndrome and Multiple Sclerosis


NEW YORK – January 8, 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 that the United States Patent and Trademark Office (USPTO) granted U.S. Patent No. 10,507,196 to support Anavex’s leading drug candidate, ANAVEX®2-73 (blarcamesine) for the treatment of neurodevelopmental disorders including Rett syndrome, and multiple sclerosis.

Anavex’s newest patent is expected to remain in force at least until 2037, not including any patent term extensions. It covers methods of treatment for neurodevelopmental disorders including Rett syndrome, autism spectrum disorder, Angelman syndrome, and cerebral palsy, among others, and also treatment for multiple sclerosis, using ANAVEX®2-73 (blarcamesine). ANAVEX®2-73 (blarcamesine) is currently in several clinical studies for Rett syndrome, including the U.S. Phase 2 Rett syndrome study[1] and the AVATAR Rett syndrome study[2], with the international EXCELLENCE Rett syndrome study of ANAVEX®2-73 (blarcamesine) in pediatric patients scheduled to initiate early 2020. The ‘196 patent adds important coverage for ANAVEX®2-73 (blarcamesine) treatments for Rett syndrome and multiple sclerosis.

“We are extremely pleased with the continued development of the patent portfolio for ANAVEX®2-73 (blarcamesine). 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 relating to ANAVEX®2-73 (blarcamesine),” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex.

ANAVEX®2-73 (blarcamesine) is an orally available, small-molecule activator of the sigma-1 receptor which, data suggest, is pivotal to restoring neural cell homeostasis and promoting neuroplasticity.[3]

About and Rett Syndrome and Multiple Sclerosis

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.

Multiple sclerosis (MS) is a chronic inflammatory-neurodegenerative disease of the central nervous system (CNS), resulting in the accumulation of irreversible disabilities such as muscle weakness, visual disturbances and other neurological impairments. MS is the most common autoimmune disorder of the CNS, and worldwide, ~2.3 million people are affected. Most people experience their first symptoms of MS between the ages of 20 and 40. Currently there is no cure for MS and many medications have serious side effects and some carry significant risks.[4] There is still a huge unmet need for agents directly interacting with the central nervous system: Regeneration, axonal protection, neuronal protection and remyelination.

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 a successful Phase 2a clinical trial for Alzheimer’s disease. 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 preclinical 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.

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[1] Identifier: NCT03758924

[2] Identifier: NCT03941444

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


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