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ATH-097 is an orally bioavailable small molecule inhibitor of proprotein convertase subtilisin/kexin type 9 (PCSK9), currently in early-stage clinical development by Atheron Therapeutics for the treatment of hypercholesterolemia and atherosclerotic cardiovascular disease (ASCVD). PCSK9 is a protein that binds to the low-density lipoprotein receptor (LDLR) on the surface of hepatocytes, promoting its degradation and thereby reducing the clearance of LDL cholesterol from the bloodstream. By inhibiting the interaction between PCSK9 and LDLR, ATH-097 aims to increase the density of LDLRs on the cell surface, leading to significant reductions in circulating LDL cholesterol levels. As an oral therapy, ATH-097 is designed to provide a more convenient alternative to existing injectable PCSK9 inhibitors, such as monoclonal antibodies and siRNA-based treatments.
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