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AEX-4 is a preclinical small molecule drug candidate developed by Aexon Labs for the treatment of synucleinopathies and REM sleep behavior disorder (RBD). It features a unique dual mechanism of action, acting as an orexin-1 receptor (OX1R) agonist while simultaneously inhibiting cathepsin B (CTSB) and cathepsin H (CTSH). This approach is designed to counteract the downregulation of OX1R associated with alpha-synuclein pathology, which is a known driver of REM sleep behavior disorder. By enhancing orexin signaling and modulating cathepsin-mediated proteolytic pathways, AEX-4 aims to provide neuroprotective effects in conditions such as Parkinson's disease, Dementia with Lewy Bodies, and Multiple System Atrophy.
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