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Anavex Life Sciences Announces Initiation of the EXCELLENCE ANAVEX®2-73-RS-003 Clinical Study in Pediatric Patients with Rett Syndrome


NEW YORK – September 4, 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 the approval to initiate the ANAVEX®2-73-RS-003 clinical study of approximately 69 pediatric patients with Rett syndrome, aged 5 to 18, called EXCELLENCE (ANAVEX®2-73 (blarcamesine) in a Double-Blind, Randomized, Placebo-Controlled Safety and Efficacy Study in Pediatric Patients with Rett Syndrome). The EXCELLENCE study is designed as an international study with the first approval by the Australian Human Research Ethics Committee and scheduled to initiate within the next quarters.

Safety and efficacy will be investigated in approximately 69 patients over a 12-week treatment period including ANAVEX®2-73-specific precision medicine biomarkers using a convenient once-daily oral liquid ANAVEX®2-73 (blarcamesine) formulation to treat Rett syndrome. All patients who participate in the study will be eligible to receive ANAVEX®2-73 (blarcamesine) under a voluntary open label extension protocol. This study is part of the planned Anavex Rett Syndrome Program including ongoing and further studies of ANAVEX®2-73 (blarcamesine) in wider age range and other geographic regions.

For parents who wish to learn more about the EXCELLENCE pediatric Rett syndrome clinical study, please visit the website,

Previously, administration of ANAVEX®2-73 resulted in both significant and dose-related improvements in an array of neurologic paradigms in the Mecp2 heterozygous Rett syndrome mouse model at different ages. These paradigms measured different sensory, motor, and autonomic abnormalities of relevance to Rett syndrome, including motor coordination and balance; gait abnormalities; hindlimb clasping, a model of the characteristic hand stereotypies in individuals with Rett syndrome; acoustic startle response; and, in older animals, visual acuity and breathing abnormalities. This data was presented at scientific meetings and is scheduled to be published in a peer-reviewed journal. Anavex is utilizing innovative strategies in trial design in order to effectively and efficiently translate preclinical and clinical findings into an exploration of ANAVEX®2-73 (blarcamesine) as a potential therapeutic in Rett syndrome.

Rett syndrome, which is seen almost exclusively in girls, is a severe genetic disorder in which the brain does not mature properly and there is progressive neurologic impairment that leads to shorter lifespan and diminished quality of life. It affects multiple neurologic aspects, including ability to communicate, ability to walk and other intellectual functions, hand function, and also to certain extent breathing abnormalities, behavioral problems, and seizures.

“This study highlights Anavex’ commitment to all families of young girls with Rett syndrome to be able to offer a potential treatment to a broader age range complementing the ongoing clinical studies in the U.S. and the AVATAR study in adult patients with Rett syndrome,” said Christopher U Missling, PhD, Chief Executive Officer of Anavex. “There are currently no approved therapies for Rett syndrome and we are looking forward to working with the Rett syndrome community worldwide to developing transformative therapies for people and their families living with 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 M1 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

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