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AEX-1 is a small molecule drug candidate developed by Aexon Labs for the treatment of Alzheimer's disease. It features a multi-target mechanism of action, functioning as a Sigma-1 receptor (S1R) agonist, a Cathepsin H (CTSH) inhibitor, and an Orexin receptor 1 (OX1R) agonist. This polypharmacological approach is designed to address complex pathological features of Alzheimer's, including tau protein abnormalities, neuroinflammation, and synaptic dysfunction. By targeting S1R, the molecule aims to provide neuroprotection and improve protein homeostasis, while its activity on CTSH and OX1R is intended to further modulate inflammatory responses and support neuronal health. AEX-1 is currently in the discovery stage of development.
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