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Anavex Life Sciences Announces First Patient Dosed in EXCELLENCE Phase 2/3 Clinical Trial of ANAVEX®2-73 (Blarcamesine) in Patients with Rett Syndrome


NEW YORK – July 1, 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 diseases including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, today announced that the first patient was dosed in the EXCELLENCE Phase 2/3 ANAVEX®2-73-RS-003 clinical trial for the treatment of Rett syndrome with ANAVEX®2-73 (blarcamesine).

The Phase 2/3 study will use a convenient once-daily oral liquid ANAVEX®2-73 (blarcamesine) formulation to treat Rett syndrome. Safety and efficacy will be investigated in at least 69 patients over a 12-week treatment period including ANAVEX®2-73-specific precision medicine biomarkers. All patients who participate in the study will be eligible to receive ANAVEX®2-73 (blarcamesine) under a voluntary open label extension protocol.

This represents the 3rd ongoing clinical study in Anavex’s Rett Syndrome Program: U.S. RTT (ANAVEX®2-73-RS-001)[1], AVATAR (ANAVEX®2-73-RS-002)[2] and EXCELLENCE (ANAVEX®2-73-RS-003)[3].

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.

For parents who wish to learn more about the EXCELLENCE pediatric Phase 2/3 Rett syndrome clinical study, please visit the website,

Rett Syndrome Association of Australia (RSAA) will make the necessary bookings and pay for them directly so as not to burden and leave participants and their families out of pocket for major expenses whilst directly participating in the EXCELLENCE trial. Please contact Claude Buda, RSAA President, directly by phone or email for any inquiries:

Claude Buda, RSAA President

Phone: +61 (0)411 089 156


“We are delighted to expand the ANAVEX®2-73 (blarcamesine) treatment program for Rett syndrome to include pediatric patients. This is the 3rd ongoing clinical study within the Anavex Rett syndrome program,” said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. “In addition to Rett syndrome, Anavex has ongoing clinical development programs for ANAVEX®2-73 (blarcamesine) for the treatment of Alzheimer’s disease[4] and Parkinson’s disease dementia[5].”

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®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 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 ANAXEX®2-73 (blarcamesine) led to significant improvements in multiple motor, sensory, and autonomic phenotypes of relevance to Rett syndrome, including motor coordination and balance, acoustic and visual responses, hindlimb clasping (resembling the characteristic hand stereotypies), 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


Investors & Media:


[1] Identifier: NCT03758924

[2] Identifier: NCT03941444

[3] Identifier: NCT04304482

[4] Identifier: NCT03790709

[5] Identifier: NCT03774459

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