Anavex Life Sciences Announces Fast Track Designation Granted by U.S. FDA for ANAVEX®2-73 (blarcamesine) Clinical Development Program for the Treatment of Rett Syndrome
NEW YORK – February 3, 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) disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ANAVEX®2-73 (blarcamesine) clinical development program for the treatment of Rett syndrome.
FDA Fast Track is a program designed to facilitate and expedite the development and review of a new drug to address unmet medical need in the treatment of a serious and life-threatening condition for which it demonstrates the potential to address unmet medical needs for such a disease or condition. The purpose of the program is to get important new therapies to the patients earlier in order to address the unmet medical needs in the treatment serious and life-threatening diseases.
In the U.S. Phase 2 Rett syndrome study to date, ANAVEX®2-73 (blarcamesine) demonstrated significant improvements of the two global efficacy endpoints, the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score and the Clinical Global Impression – Improvement (CGI-I).
At the 6th Annual European Rett Syndrome Conference, Anavex presented data demonstrating also that ANAVEX®2-73 (blarcamesine) significantly improved the RSBQ Hand Behaviours and the RSBQ Breathing Abnormalities scores. In addition, efficacy signals on both caregiver- and clinician-based measures of severity correlated with a key biomarker related to disease pathogenesis (Glutamate levels).
This is one of three clinical studies in Anavex’s Rett Syndrome Program: U.S. RTT (ANAVEX®2-73-RS-001), AVATAR (ANAVEX®2-73-RS-002) and EXCELLENCE (ANAVEX®2-73-RS-003).
“With no currently approved agents to treat Rett syndrome, patients and their physicians have an urgent need for new therapeutic options,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “We view this FDA Fast Track designation as continued support that ANAVEX®2-73 (blarcamesine) has the potential to address this unmet need. Importantly, Fast Track designation provides a number of important advantages that could expedite the development and review of ANAVEX®2-73 (blarcamesine).”
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.
About FDA Fast Track Designation
Fast Track is a program designed to facilitate the expedited development and review of a new drug alone or in combination with other drugs to treat serious or life-threatening conditions for which there is a demonstration of the potential to address an unmet medical need. The purpose is to advance new drugs earlier for patients who need them. Fast Track addresses a broad range of serious conditions.
A drug that receives Fast Track designation is eligible for some or all of the following:
More frequent meetings and interactions with the review team at the FDA to discuss the drug’s development and ensure collection of appropriate data needed to support drug approval as well as to discuss accelerated approval, the structure and content of an NDA, and other critical issues
More frequent written communications from FDA about such things as the design of the proposed clinical trials and use of biomarkers
Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met
Rolling Review, which means that a drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, rather than waiting until every section of the marketing application is completed before the entire application can be reviewed. BLA or NDA review usually does not begin until the drug company has submitted the entire application to the FDA.
About Rett Syndrome
Rett syndrome is a devastating, 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, autistic features, slowed brain and head growth, ataxia, seizures and intellectual disability. There is currently no cure for Rett syndrome. 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 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.
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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 Advances in Experimental Medicine and Biology Volume 964 (2017) Sigma Receptors: Their Role in Disease and as Therapeutic Targets.