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Anavex Life Sciences Announces Completion of ANAVEX®2-73 U.S. Phase 2 Rett Syndrome Clinical Trial

NEW YORK – September 10, 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) diseases, today announced the completion of the ANAVEX®2-73 (blarcamesine) U.S. Phase 2 study in Rett syndrome. The Company expects to announce topline results from this study in the next quarter.

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.[1]

The ANAVEX®2-73 (blarcamesine) U.S. Phase 2 study enrolled 31 patients with Rett syndrome and all patients completed the 7-week trial.

The multi-center, double-blind clinical study at 8 sites across U.S. measured safety, tolerability, and efficacy of daily oral ANAVEX®2-73 (blarcamesine) doses or placebo. After completing the trial, eligible participants were able to join a voluntary open-label 12-week extension study of ANAVEX®2-73 (blarcamesine).

Anavex previously reported interim data of the PART A intensive PK sub-cohort in the first 6 patients of the U.S. Phase 2 Rett syndrome study in which ANAVEX®2-73 (blarcamesine) that demonstrated significant improvements in the two global efficacy endpoints, the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score and the Clinical Global Impression – Improvement (CGI-I). ANAVEX®2-73 (blarcamesine) also significantly improved the RSBQ Hand Behaviours and RSBQ Breathing Abnormalities subscales. In addition, efficacy signals on both caregiver- and clinician-based measures of severity correlated with levels of glutamate, a key biomarker of disease pathogenesis.

This study is one of three ongoing clinical studies in Anavex’s Rett Syndrome Program: U.S. RTT (ANAVEX®2-73-RS-001)[2], AVATAR (ANAVEX®2-73-RS-002)[3] and EXCELLENCE (ANAVEX®2-73-RS-003)[4]. 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.

“We would like to thank the investigators and clinical site coordinators as well as all the participating families for their dedication to this first US-based clinical study completed with ANAVEX®2-73 (blarcamesine)”, said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “Since there is currently no approved agent to treat this devastating disease, we continue our fast-paced development programs of ANAVEX®2-73 (blarcamesine) in Rett syndrome.”

About Rett Syndrome

Rett syndrome is a rare, non-inherited genetic postnatal progressive neurodevelopmental disorder that occurs predominantly 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 relatively 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 non-pharmacological therapies, and drugs for seizure control, motor difficulties, breathing irregularities, and abnormal behaviors. Rett syndrome is mainly 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®2-73 (blarcamesine)

ANAVEX®2-73 (blarcamesine) activates the Sigma-1 receptor (S1R) protein, which serves as a molecular chaperone and functional modulator involved in restoring cellular homeostasis. In a Phase 2a Alzheimer’s disease (AD) study, ANAVEX®2-73 showed dose dependent improvement in exploratory endpoints of cognition (MMSE) and activities of daily living (ADCS-ADL). The ANAVEX®2-73 Phase 2 Rett syndrome study design includes genomic biomarkers of response identified in the ANAVEX®2-73 Phase 2a AD study. Studies of ANAVEX®2-73 (blarcamesine) in a mouse model of Rett syndrome, carrying a heterozygous Mecp2-null mutation and presenting with neurological impairments that mimic the human disorder, demonstrated improvements at both young ages (~6 weeks) and adulthood (7 months). Chronic oral administration of ANAVEX®2-73 (blarcamesine) led to significant improvements in multiple motor, sensory, and autonomic phenotypes of relevance to Rett syndrome, including motor coordination and balance, auditory and visual responses, hindlimb clasping (resembling the characteristic repetitive hand movements), and episodes of apnea. Notably, these changes were dose related and also evident in animals at an advanced stage of pathology. These experiments were sponsored by

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.

For Further Information:

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

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



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