Pre-treatment with blarcamesine entirely prevented Aβ-induced cognitive decline
Confirmed significant biomarker response in hippocampus
NEW YORK – August 20, 2025
Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company focused on developing innovative treatments for Alzheimer’s disease, Parkinson’s disease, schizophrenia, neurodevelopmental, neurodegenerative, and rare diseases, including Rett syndrome, and other central nervous system (CNS) disorders, today reported a peer-reviewed publication in the journal Neuroscience Letters, titled “Prevention of memory impairment and hippocampal injury with blarcamesine in an Alzheimer’s disease model.”[1]
This study shows that pre-treatment with blarcamesine prevented amyloid beta-induced memory impairment and brain oxidative injury, suggesting that blarcamesine is an attractive candidate for Alzheimer’s disease pharmacological prevention.
“This preclinical study is exciting since it clearly demonstrates a preventative effect of blarcamesine in Alzheimer’s pathology and potentially might be able to prevent onset of Alzheimer’s disease in healthy individuals,” said Tangui Maurice, PhD, Research Director at University of Montpellier, France, and author of the publication. “These findings support the future direction of clinical trials with the objective of addressing blarcamesine’s potential as a safe and effective pharmacologic agent — applied as a convenient once-daily oral pill — for the prevention of Alzheimer’s disease.”
While placebo-controlled mice developed significant amyloid toxicity in the brain after injection of the toxic Aβ25-35 peptide, animals pre-treated with blarcamesine showed significant protection, with reduced vulnerability to Aβ25-35-induced oxidative stress and reduced susceptibility to learning and memory deficits.
The mechanistic confirmation that blarcamesine restores impaired autophagy through SIGMAR1 activation — acting upstream of amyloid and tau pathologies — has previously been established in both in vitro and in vivo studies. Specifically, blarcamesine demonstrated enhanced autophagic flux in human cells and in C. elegans, increased proteostasis capacity, and promotion of autophagosome biogenesis, cargo reception, and lysosome fusion.[2]
SIGMAR1 has emerged as a key therapeutic target in neurodegenerative disease. Its activation enhances autophagy, facilitating degradation of amyloid-beta precursor protein (APP) and normalizing Aβ production.[3] In addition to amyloid regulation, SIGMAR1 activation supports neurogenesis, reduces oxidative stress by lowering reactive oxygen species (ROS), suppresses neuroinflammation, and mitigates Aβ-induced toxicity. SIGMAR1 also helps maintain endoplasmic reticulum (ER) integrity and modulates intracellular calcium signaling.[4] Collectively, these effects contribute to restored cellular homeostasis, rebalanced neuronal function, and enhanced neuroplasticity.[5]
“The findings in this publication further support the potential beneficial effect of blarcamesine in Alzheimer’s disease prevention. With once-daily oral blarcamesine administration — and hence preserved autophagy function through upstream SIGMAR1 activation — downstream pathological manifestations of amyloid beta might be prevented,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex.
The paper can be accessed online at: https://pubmed.ncbi.nlm.nih.gov/40784611/
This release discusses investigational uses of an agent in development and is not intended to convey conclusions about efficacy or safety. There is no guarantee that any investigational uses of such product will successfully complete clinical development or gain health authority approval. About Alzheimer’s Disease.
Alzheimer’s disease is a progressive degenerative brain disorder that gradually destroys memory and cognitive function. An estimated 7.2 million Americans currently have Alzheimer’s dementia. The disease is the most common cause of dementia among older adults and may rank as the third leading cause of death in the U.S. In 2020, Alzheimer’s and other dementias cost the nation approximately $781 billion; costs could rise to $1.1 trillion by 2050.[6] Globally, more than 50 million people live with dementia, a number expected to reach 152 million by 2050.[7] Nearly 10 million new cases are diagnosed each year, representing one case every 3 seconds and intensifying caregiving burdens worldwide.[8]
About Anavex Life Sciences Corp.
Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company developing novel therapeutics for neurodegenerative, neurodevelopmental, and neuropsychiatric disorders, including Alzheimer’s disease, Parkinson’s disease, schizophrenia, Rett syndrome, and other CNS diseases. Its lead candidate, ANAVEX®2-73 (blarcamesine), has completed multiple clinical trials across Alzheimer’s disease, Parkinson’s disease dementia, and Rett syndrome. ANAVEX®3-71, targeting SIGMAR1 and M1 muscarinic receptors, is also in clinical development and has demonstrated disease-modifying effects in Alzheimer’s models.
More information is available at www.anavex.com. Follow the Company on Twitter, Facebook, Instagram, and LinkedIn.
Forward-Looking Statements
Statements in this press release that are not strictly historical are forward-looking statements based on current expectations and involve risks and uncertainties. Actual results may differ materially due to various factors, including risks detailed in the Company’s most recent Annual Report on Form 10-K filed with the SEC. Anavex undertakes no obligation to update these statements after the date of this release.
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
References
[1] Maurice, Tangui. “Prevention of memory impairment and hippocampal injury with blarcamesine in an Alzheimer’s disease model.” Neuroscience Letters. Aug. 2025.
[2] Christ, M.G. et al. “Sigma-1 Receptor Activation Induces Autophagy and Increases Proteostasis Capacity In Vitro and In Vivo.” Cells 8,3 (2019): 211.
[3] Jaeger P.A. et al. “Regulation of amyloid precursor protein processing by the Beclin 1 complex.” PLOS One 5(6): e11102 (2010).
[4] Nguyen L. et al. “Role of sigma-1 receptors in neurodegenerative diseases.” Journal of Pharmacological Sciences 127(1):17-29 (2015); plus additional referenced studies.
[5] Advances in Experimental Medicine and Biology, Vol. 964 (2017). Sigma Receptors: Their Role in Disease and as Therapeutic Targets.
[6] https://www.nia.nih.gov/health/alzheimers; https://www.alz.org/alzheimers-dementia/facts-figures
[7] Alzheimer’s Disease International. World Alzheimer Report 2019. https://www.alz.co.uk/research/WorldAlzheimerReport2019.pdf
[8] AARP. 2020 Report: Caregiving in the U.S. https://www.aarp.org/content/dam/aarp/ppi/2020/05/full-report-caregiving-in-the-united-states.pdf