CAT-2003

CAT-2003 is an investigational, orally administered **single-molecule SMART Linker conjugate** developed by Catabasis Pharmaceuticals for dyslipidemias, including hypertriglyceridemia, hypercholesterolemia, and chylomicronemia. It covalently links eicosapentaenoic acid and niacin and is designed for intracellular cleavage by fatty acid amide hydrolase in the endoplasmic reticulum. The resulting intracellular delivery inhibits maturation of sterol regulatory element-binding protein 1 and sterol regulatory element-binding protein 2, reducing expression of lipid-synthesis and lipid-regulatory genes including PCSK9 and HMGCR, while increasing low-density lipoprotein receptor protein expression and LDL uptake. CAT-2003 completed Phase 2 studies but is not an approved medicine and is no longer an active development program.

Other names

CAT 2003CAT2003CAT-2003