NEW YORK, June 08, 2022 (GLOBE NEWSWIRE) — Anavex Life Sciences Corp. (“Anavex” or the “Company”) (AVXL), a clinical-stage biopharmaceutical company developing differentiated therapies 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 the peer-reviewed publication in American Journal of Medical Genetics identify a blood biomarker to assess the treatment effect of ANAVEX®2-73 in fragile X syndrome, which will also be included in the planned clinical trial of ANAVEX®2-73 in fragile X syndrome.
Fragile X syndrome (FXS) is the most common form of inherited intellectual disability and the most common monogenic cause of autism spectrum disorder with an estimated population of approximately 62,500 in the United States and 1,088,500 in the world.1 Currently, there is no approved treatment for fragile X syndrome.
The results demonstrate that the therapeutic effect of ANAVEX®2-73 resulting in reversal of hyperactivity, restoration of associative learning, and reduction of anxiety in a mouse model of fragile X syndrome are also associated with improvements in major blood signaling biomarkers, which are also measurable in patients with fragile X syndrome.
Previously Reported Improvements in Cognitive and Behavioral Paradigms in a Mouse Model of Fragile X Syndrome, Following ANAVEX®2-73, are associated with parallel improvements in peripheral lymphocyte signaling abnormalities (ie, decreased activated/phosphorylated Akt and ERK).2 Fragile X syndrome is characterized by multiple cell signaling abnormalities, including increased activation of the PI3K/Akt/mTOR and MAPK/ERK pathways. These have been demonstrated in brain samples from mouse models as well as in patient lymphocytes.
This publication, “Brain cell signaling abnormalities are detected in blood in a mouse model of fragile X syndrome and corrected by the Sigma-1 receptor agonist Blarcamesine”, is one of the first studies showing that key signaling abnormalities can also be detected and corrected in mouse lymphocytes, providing a solid basis for the use of lymphocyte signaling biomarkers in clinical trials involving ANAVEX®2-73. The study was supported by the FRAXA Research Foundation.
“Assessments of lymphocyte cell signaling in mouse models of fragile X syndrome are feasible and support corresponding assessments in affected individuals,” said Walter E. Kaufmann, MD, Anavex Chief Scientific Officer and corresponding author. of publishing. “These assays have the potential to monitor treatment response, particularly for drugs that correct multiple pathway defects such as the sigma-1 receptor agonist ANAVEX®2-73. The implications of this work extend beyond FXS to most neurological disorders associated with abnormal cell signaling.
The data suggest that activation of the sigma-1 receptor is essential for restoring neural cell homeostasis and promoting neuroplasticity.3
“These additional biomarker results in fragile X syndrome provide further evidence of the potential to broaden the therapeutic profile of ANAVEX®2-73 in the largest portion of the autism spectrum disorder addressable market, fragile X syndrome,” said Christopher U Missling, PhD, President and CEO of Anavex. “We look forward to initiating a double-blind, placebo-controlled Phase 2/3 ANAVEX study®2-73 fragile X syndrome study. This is further proof of the potential of ANAVEX®2-73 as a precision medicine technology platform.”
The document can be viewed online at: https://onlinelibrary.wiley.com/doi/10.1002/ajmg.a.62853
Anavex Life Sciences’ product portfolio platform includes an orally available small molecule drug candidate ANAVEX®2-73 for the treatment of Alzheimer’s disease, Parkinson’s disease and Rett’s syndrome and ANAVEX®3-71 for frontotemporal dementia.
About Fragile X Syndrome and Autism Spectrum Disorders
Fragile X syndrome is the most common form of inherited intellectual disability and the most common monogenic cause of autism, affecting approximately 1 in 4,000 men and 1 in 6,000 women. The disorder is caused by the expansion instability of a CGG repeat in the FMR1 gene that leads to abnormal methylation and suppression of FMR1 transcription with the resulting decrease in protein levels in the brain and other tissues. The average age of diagnosis of fragile X syndrome in boys and girls is 35-37 months and 42 months, respectively. Behavioral abnormalities, including autism spectrum disorders, are common.
Autism spectrum disorder is a behavioral diagnosis, while fragile X syndrome is a medical/genetic diagnosis. Numerous studies have assessed the link between fragile X syndrome and autism spectrum disorders over the past decades. Because many children with fragile X syndrome are interested in social interactions, they may not meet the diagnostic criteria for autism spectrum disorder, even if they exhibit certain characteristics such as poor eye contact, shyness, social anxiety, hand flapping and sensory issues. Autism is much more common in boys than in girls with fragile X syndrome. According to the CDC, a national survey of parents found that 46% of men and 16% of women with fragile X syndrome have been diagnosed or treated for autism spectrum disorder.
About the FRAXA Research Foundation
FRAXA’s mission is to find effective treatments and ultimately a cure for fragile X syndrome. FRAXA directly funds research grants and fellowships at top universities around the world. FRAXA partners with biomedical and pharmaceutical companies large and small to bridge the gap between research discoveries and real treatments. Treatments for fragile X syndrome are likely to help people with autism, Alzheimer’s disease and other brain disorders. More information is available at www.fraxa.org.
About Anavex Life Sciences Corp.
Anavex Life Sciences Corp. (AVXL) is a publicly traded biopharmaceutical company dedicated to developing novel therapies for the treatment of neurodegenerative and neurodevelopmental disorders, including Alzheimer’s disease, Parkinson’s disease, Rett’s syndrome and other diseases of the neurological system. central nervous system (CNS), pain, and various types of cancer. Anavex’s lead drug candidate, ANAVEX®2-73 (blarcamesine), successfully completed a Phase 2a clinical trial in Alzheimer’s disease, a Phase 2 proof-of-concept study in Parkinson’s disease dementia, and a Phase 2 and Phase 3 in adult patients with Rett syndrome. ANAVEX®2-73 is an orally available drug candidate that restores cellular homeostasis by targeting sigma-1 and muscarinic receptors. Preclinical studies have demonstrated its potential to halt and/or reverse the progression of Alzheimer’s disease. ANAVEX®2-73 has also shown anticonvulsant, anti-amnesic, neuroprotective, and antidepressant properties in animal models, indicating its potential to treat other CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson’s Disease Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX®2-73 for the treatment of Parkinson’s disease. ANAVEX®3-71, which targets sigma-1 and muscarinic M1 receptors, is a promising clinical-stage drug candidate demonstrating disease-modifying activity against key hallmarks of Alzheimer’s disease in transgenic mice (3xTg-AD) , 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 www.anavex.com. You can also connect with the company on Twitter, Facebook, Instagram and LinkedIn.
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 these statements due to a variety of 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. assumes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.
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3 Advances in Experimental Medicine and Biology Volume 964 (2017) Sigma Receptors: Their Role in Disease and as Therapeutic Targets.