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Anavex Life Sciences Announces Publication of Foundational Data for ANAVEX®2-73 (blarcamesine) in Rett Syndrome

Published in Pharmacology Biochemistry and Behavior, Results Highlight Improvements in Multiple Motor, Sensory, and Autonomic Phenotypes Associated with Rett Syndrome

Study Provided Proof-of-Concept for Ongoing ANAVEX®2-73 (blarcamesine) Phase 2 Trials


NEW YORK – November 6, 2019

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) diseases, today announced that preclinical data of ANAVEX®2-73 (blarcamesine) in Rett syndrome were published in the peer-reviewed journal, Pharmacology, Biochemistry and Behavior.

Rett syndrome, a rare genetic neurological disorder that afflicts mostly girls, is caused by a genetic defect associated with severe physical and cognitive impairments. The study evaluated doses of ANAVEX®2-73 (blarcamesine) in heterozygous MECP2 mice, a validated animal model for the disease, which resulted in improvements in motor, sensory, and autonomic phenotypes related to the disease. These included motor coordination and balance, acoustic and visual responses, and other improvements. ANAVEX®2-73 (blarcamesine) also showed a good safety profile.

“Testing novel drugs that can safely improve the symptoms of Rett syndrome is a high priority,” said Walter E Kaufmann, M.D., Chief Medical Officer of Anavex and corresponding author of the paper. “ANAVEX®2-73 (blarcamesine) is a sigma-1 receptor agonist and muscarinic receptor modulator with a strong safety record. The drug’s ability to enhance calcium homeostasis and improve the mitochondrial and cellular functions that underlie pathological processes and compensatory mechanisms in Rett syndrome, make ANAVEX®2-73 (blarcamesine) a promising candidate.”

The study, “ANAVEX®2-73 (blarcamesine), a Sigma-1 receptor agonist, ameliorates neurologic impairments in a mouse model of Rett syndrome,” served as a proof-of-concept for the ongoing Phase 2 ANAVEX2-73-RS-001 study (NCT03758924) as well as for upcoming pediatric studies. Preliminary results from the first cohort of this study were recently presented at the 6th Annual European Rett Syndrome Conference. The data described significant improvements in the two global efficacy endpoints: the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score and the Clinical Global Impression – Improvement (CGI-I); as well as in the RSBQ Hand Behaviours and the RSBQ Breathing Abnormalities subscale scores. Efficacy signals on both caregiver- and clinician-based measures of severity also correlated with plasma changes of key biomarkers related to disease pathogenesis.

“We look forward to completing the double-blind, placebo-controlled Phase 2 ANAVEX2-73-RS-001 study, followed by the double-blind, placebo-controlled Phase 2 ANAVEX2-73-RS-002 (AVATAR) study,” said Christopher U. Missling, Ph.D., Chief Executive Officer of Anavex. “We also expect to initiate the double-blind, placebo-controlled clinical trial, ANAVEX2-73-RS-003 (EXCELLENCE) in pediatric patients with Rett syndrome. Anavex is excited about our progress to date, as well as our pursuit of a much-needed treatment option for patients with Rett syndrome.”

The full paper can be accessed online at: (

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 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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