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Anavex Life Sciences Announces Exceeding of Enrollment Target for ANAVEX®2-73 (blarcamesine)

EXCELLENCE Phase 2/3 Rett Syndrome Clinical Trial


NEW YORK – February 2, 2023

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 the total enrollment of 92 patients with Rett syndrome for the ANAVEX®2-73 (blarcamesine) EXCELLENCE Phase 2/3 study in Rett syndrome patients ages ≥ 5 years to 17 (inclusive). This exceeds the original enrollment target and the Company expects to announce topline results from this study in 2H 2023.


ANAVEX®2-73 is an orally available, small-molecule activator of the sigma-1 receptor (SIGMAR1) which, data suggest, is pivotal to restoring neural cell homeostasis and promoting neuroplasticity.[1]


The enrollment completion of the randomized, placebo-controlled EXCELLENCE Phase 2/3 study ANAVEX®2-73-RS-003 for the treatment of pediatric patients with Rett syndrome was preceded by the successful completion of both placebo-controlled Phase 2 U.S. (ANAVEX®2-73-RS-001)[2], and Phase 3 AVATAR (ANAVEX®2-73-RS-002)[3]studies in adult patients with Rett syndrome.


The multi-center, double-blind clinical EXCELLENCE study (ANAVEX®2-73-RS-003)[4] in pediatric patients is measuring safety, tolerability, and efficacy of daily oral ANAVEX®2-73 doses or placebo. After completing the double-blind study, eligible participants are able to join a voluntary open-label extension study of ANAVEX®2-73. In communication with the FDA, we received their input on the endpoints, which were utilized in this study.


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.


“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,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “We are excited to complete enrollment in this multicenter study with ANAVEX®2-73 in Rett syndrome and look forward to reporting the results later this year.”


About Rett Syndrome

Rett syndrome is a rare, non-inherited genetic postnatal 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 even breathe easily. The hallmark of Rett syndrome is near constant repetitive hand movements while awake. It 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, slowed brain and head growth, problems with walking, seizures and intellectual disability. There is currently no cure for Rett syndrome and treatment of the disorder is symptomatic. Management of symptoms is done through a multidisciplinary approach utilizing medication for motor difficulties, breathing irregularities and control of seizures through anticonvulsant drugs. Rett syndrome is caused by mutations in the MECP2 gene and strikes all racial and ethnic groups and occurs worldwide in approximately one in every 10,000 to 15,000 live female births.

About Anavex Life Sciences Corp.

Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company dedicated to the development of novel 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), has successfully completed a Phase 2a and recently a Phase 2b/3 clinical trial for Alzheimer's disease, a Phase 2 proof-of-concept study in Parkinson's disease dementia, and both a Phase 2 and a Phase 3 study in adult patients with Rett syndrome. ANAVEX®2-73 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 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 for the treatment of Parkinson's disease. ANAVEX®3-71, which targets sigma-1 and M1 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 www.anavex.com. You can also connect with the company on Twitter, Facebook, Instagram, 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:

Andrew J. Barwicki Investor Relations Tel: 516-662-9461


[1]Advances in Experimental Medicine and Biology Volume 964 (2017) Sigma Receptors: Their Role in Disease and as Therapeutic Targets. [2] ClinicalTrials.gov Identifier: NCT03758924 [3] ClinicalTrials.gov Identifier: NCT03941444 [4] ClinicalTrials.gov Identifier: NCT04304482


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