Anavex Life Sciences Announces Translational Biomarker Data for ANAVEX®2-73 (blarcamesine) in Fragile X Syndrome (Major Cause of Autism) at the 19th NFXF International Fragile X Conference

ANAVEX^®2-73 corrects directly to humans’ translatable EEG biomarkers in a model of Fragile X Syndrome (FXS)

Therapeutic potential to address behavioral and cognitive deficits in individuals with neurodevelopmental disorders

Plans advancing to initiate a clinical trial in individuals with FXS

NEW YORK, July 30, 2024 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of Alzheimer's disease, Parkinson's disease, schizophrenia, neurodevelopmental, neurodegenerative, and rare diseases, including Rett syndrome and Fragile X syndrome (FXS), a major cause of autism, today reported positive preclinical results in directly to humans’ translatable biomarkers for individuals with FXS for ANAVEX^®2-73 (blarcamesine), in a disease model of Fragile X syndrome (FXS).

Fragile X syndrome (FXS) is the most common form of inherited intellectual disability and the most frequent single gene cause of autism spectrum disorder with an estimated population of approximately 62,500 in the US and 1,088,500 worldwide.¹ It is characterized by a range of cognitive, behavioral, and sensory challenges, including learning difficulties, anxiety, hyperactivity, and sensitivity to sensory stimuli. At present, there is no approved treatment for Fragile X syndrome.

The findings presented at the 19^th NFXF International Fragile X Conference reports positive results in directly to humans’ translatable electroencephalogram (EEG) biomarkers present in both individuals with FXS and animal models of FXS.

The study presented investigated the effects of ANAVEX^®2-73 on brain activity in a mouse model of FXS using electroencephalography (EEG), a non-invasive technique that measures electrical signals in the brain. EEG recordings from mice treated with ANAVEX^®2-73 showed significant dose-dependent improvements in several key biomarkers of brain function compared to untreated mice.

Key results were found in the auditory steady state response (ASSR) EEG test, which are of particular importance to the treatment of CNS disorders. In FXS and other CNS disorders, the brain is unable to synchronize sensory stimuli like sounds appropriately, driving sensory overload and hypersensitivity in affected individuals. In the current study, ANAVEX^®2-73 robustly enhances neural synchrony in response to sounds in the frontal and auditory cortices of the brain in a dose dependent manner.

Dysfunction of cells that regulate the balance of excitatory-inhibitory signaling has been implicated in these characteristic hypersensitivities of various neurodevelopmental disorders, including FXS. This new data demonstrates that ANAVEX^®2-73 restores this balance and improves neuronal connectivity, which is directly translatable to humans and demonstrates the therapeutic potential of ANAVEX^®2-73 to address behavioral, sensory, and cognitive abnormalities in individuals with FXS.

ANAVEX^®2-73 has previously demonstrated preclinical efficacy in an animal model of FXS published in the scientific journals Nature Scientific Reports² and the American Journal of Medical Genetics³. These previous studies highlighted the ability of ANAVEX^®2-73 to ameliorate behavioral deficits and fluid biomarker features of FXS. Dose-dependent treatment effect of ANAVEX^®2-73 resulted in reversal of hyperactivity, restoration of associative learning and reduction of anxiety in a mouse model of Fragile X syndrome, which were also associated with improvements in key blood signaling biomarkers, which are measurable in patients with Fragile X syndrome as well.

Based on these results, the Company plans to initiate a clinical trial to evaluate the safety and efficacy of ANAVEX®2-73 in individuals with FXS.

“These additional non-invasive biomarker findings in Fragile X syndrome provide further evidence of potential to expand the therapeutic profile of ANAVEX^®2-73 into the largest portion of addressable market of autism spectrum disorder, Fragile X syndrome,” said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. “We look forward to initiating a double-blind, placebo-controlled ANAVEX^®2-73 study in Fragile X syndrome. This is further evidence of the potential of ANAVEX^®2-73 as a platform technology of precision medicine."

The poster presentation is available on the Investors section of the Company’s website at www.anavex.com.

Anavex Life Sciences’ product portfolio platform includes orally available small molecule drug candidate ANAVEX^®2-73 for the treatment of Alzheimer’s disease, Parkinson’s disease, Rett syndrome and ANAVEX^®3-71 for schizophrenia.

About Fragile X Syndrome and Autism Spectrum Disorder

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 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 Fragile X syndrome diagnosis for 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 link between Fragile X syndrome and autism spectrum disorder over the last few decades. Since many children with Fragile X syndrome are interested in social interactions, they may not meet the diagnostic criteria for autism spectrum disorder, even though they exhibit some features 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 parent survey found that 46% of males and 16% of females with Fragile X Syndrome have been diagnosed or treated for autism spectrum disorder.

About Anavex Life Sciences Corp.

Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company dedicated to the development of novel therapeutics for the treatment of neurodegenerative, neurodevelopmental, and neuropsychiatric disorders, including Alzheimer's disease, Parkinson's disease, Rett syndrome, schizophrenia and other central nervous system (CNS) diseases, pain, and various types of cancer. Anavex's lead drug candidate, ANAVEX^®2-73 (blarcamesine), has successfully completed a Phase 2a and a Phase 2b/3 clinical trial for Alzheimer's disease, a Phase 2 proof-of-concept study in Parkinson's disease dementia, and both a Phase 2 and a Phase 3 study in adult patients and one Phase 2/3 study in pediatric patients with Rett syndrome. ANAVEX^®2-73 is an orally available drug candidate that restores cellular homeostasis by targeting SIGMAR1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer's disease. ANAVEX^®2-73 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 for the treatment of Parkinson's disease. We believe that ANAVEX^®3-71, which targets SIGMAR1 and M1 muscarinic receptors, is a promising clinical stage 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 www.anavex.com. You can also connect with the Company on Twitter, Facebook, Instagram, 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
Email: info@anavex.com

Investors:
Andrew J. Barwicki
Investor Relations
Tel: 516-662-9461
Email: andrew@barwicki.com

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¹ https://fragilex.org/understanding-fragile-x/fragile-x-101/prevalence/
² Reyes ST, Deacon RMJ, Guo SG, et al. Effects of the sigma-1 receptor agonist blarcamesine in a murine model of fragile X syndrome: neurobehavioral phenotypes and receptor occupancy. Sci Rep. 2021;11(1):17150. Published 2021 Aug 25. doi:10.1038/s41598-021-94079-7
³ Cogram P, Deacon RMJ, Klamer D, et al. Brain cell signaling abnormalities are detected in blood in a murine model of Fragile X syndrome and corrected by Sigma-1 receptor agonist Blarcamesine. Am J Med Genet A. 2022;188(8):2497-2500. doi:10.1002/ajmg.a.62853