Shared clinical phenotypes and underlying pathophysiology of Autism-Related Disorders such as Fragile X and Rett syndrome, indicates that ANAVEX 2-73 could be of interest to investigate clinically in these disorders
NEW YORK, NY – June 6, 2016 – 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, other central nervous system (CNS) diseases, pain and various types of cancer, today announced positive data of ANAVEX 2-73 in an exploratory study in a Fragile X syndrome model presented at the Gordon Research Conference for Fragile X and Autism-Related Disorders, being held June 5-10, 2016 in Mount Snow, VT.
Fragile X syndrome is the most common form of inherited intellectual disability and the most frequent single gene cause of autism.
“The ANAVEX 2-73 data in an array of behavioral paradigms in a validated mouse model of Fragile X is very encouraging. The results are promising for both Fragile X syndrome and Autism Spectrum Disorders, since there is an overlap in the clinical as well as in the underlying molecular pathology of the two disorders. We would be very interested in continuing the work with ANAVEX 2-73,” said Michael Tranfaglia, MD, Medical Director, Chief Scientific Officer of FRAXA Research Foundation.
Dosing of ANAVEX 2-73 for 14 days in a Fragile X syndrome mouse model was followed by a battery of behavioral tests that are linked to pathophysiological signs of Autism-related disorders and Fragile X syndrome: hyper-locomotion, associative learning, and marble burying. Treatment with ANAVEX 2-73 significantly improved all behaviors tested. ANAVEX 2-73 significantly reversed the hyperactivity and impairment in learning and memory (p≤0.0001), to the same levels observed in vehicle-treated wild-type mice. ANAVEX 2-73 also yielded a partial effect in the marble burying experiment (p≤0.05). The study was sponsored by FRAXA, via the FRAXA Drug Validation Initiative, and performed by Fraunhofer Chile Research, Santiago, Chile.
“Our previous results in Rett syndrome, together with the new data in another neurodevelopmental disorder, Fragile X, support our hypothesis that ANAVEX 2-73 has a mechanism of action that could be of interest to investigate clinically in rare disorders like Rett syndrome, Fragile X syndrome, and other Autism Spectrum Disorders, in addition to neurodegenerative diseases such as Alzheimer’s disease,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex.
The poster titled “Sigma-1 Receptor Agonists as Potential Treatment Options for Autism Spectrum Disorders: Pre-clinical Studies with ANAVEX 2-73 in a Fragile X Model” is being presented at the Gordon Research Conference for Fragile X and Autism-Related Disorders, in Mount Snow, VT, June 6-7, 2016, and will be available on the publications page of the Anavex website.
About Fragile X Syndrome and Autism Spectrum Disorders
Fragile X syndrome is the most common form of inherited intellectual disability and the most frequent single gene cause of autism, affecting approximately 1 in 4,000 males and 1 in 6,000 females. The disorder is caused by the unstable expansion of a CGG repeat in the FMR1 gene and abnormal methylation, which results in suppression of FMR1 transcription and decreased protein levels in the brain. The average age of Fragile X syndrome diagnosis of boys and girls are, 35 to 37 months and 42 months, respectively. Behavioral abnormalities, including Autism Spectrum Disorder, are common.
Autism Spectrum Disorder is a behavioral diagnosis while Fragile X syndrome is a medical/genetic diagnosis. Many studies have evaluated the Fragile X syndrome-Autism Spectrum Disorders link over the past decade. Since many children with Fragile X syndrome are interested in social interactions, they may not meet the diagnostic criteria for Autism Spectrum Disorders, even though they exhibit some features of Autism Spectrum Disorders such as poor eye contact, shyness, social anxiety, hand-flapping and sensory issues. Autism is much more common in boys with Fragile X syndrome than in girls with Fragile X syndrome. According to the CDC, a national parent survey found that 46% of males and 16% of females with Fragile X syndrome have been diagnosed or treated for Autism Spectrum Disorders.
FRAXA’s mission is to find effective treatments and a cure for children and adults with Fragile X and related disorders such as autism. FRAXA has funded over $25,000,000 in biomedical research, yielding discoveries which can change the lives of all families struggling with Fragile X. FRAXA Research Foundation is a non-profit, 501(c)3 organization which is dedicated to funding biomedical research for improved treatment and a cure for Fragile X, the leading inherited cause of intellectual disability and autism.
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 diseases including Alzheimer’s disease, other central nervous system (CNS) diseases, pain and various types of cancer. Anavex’s lead drug candidate, ANAVEX 2-73, is currently in a Phase 2a clinical trial for Alzheimer’s disease. ANAVEX 2-73 is an orally available drug candidate that targets sigma-1 and muscarinic receptors and successfully completed Phase 1 with a clean safety profile. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer’s disease. It has also exhibited anticonvulsant, anti-amnesic, neuroprotective and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy and others. The Michael J. Fox Foundation (MJFF) for Parkinson’s Research has awarded Anavex a research grant to develop ANAVEX 2-73 for the treatment of Parkinson’s disease to fully fund a preclinical study, which could justify moving ANAVEX 2-73 into a Parkinson’s disease clinical trial. ANAVEX 3-71, also targeting sigma-1 and M1 muscarinic receptors, is a promising preclinical drug candidate demonstrating disease modifications against the major Alzheimer’s hallmarks in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid and tau pathologies, and also with beneficial effects on neuroinflammation and mitochondrial dysfunctions. Further information is available at www.anavex.com.
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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