PRD001

PRD001 is a **first-in-class, orally available small molecule** that acts as a **selective inhibitor of SOAT2 (sterol O-acyltransferase 2, formerly known as ACAT2)**. It is being developed for the treatment of **homozygous familial hypercholesterolemia (HoFH)** and **metabolic dysfunction-associated steatotic liver disease (MASH/MASLD)**. PRD001 is unique in that it specifically inhibits SOAT2, in contrast to prior efforts that targeted SOAT1, SOAT1/2, or both, and represents the first clinical evaluation of an SOAT2-selective inhibitor in humans. Mechanistically, the drug controls three core pathways of lipid metabolism: **cholesterol synthesis in the liver, cholesterol absorption in the small intestine, and the uptake of blood LDL-C**, promoting a substantial reduction in LDL-C levels **independent of LDL receptor activity**. Preclinical animal models demonstrated efficacy in lowering blood and liver lipid levels, suppressing fatty liver disease and atherosclerosis progression, without significant adverse events. The drug is currently in a first-in-human Phase 1 clinical trial to evaluate safety, tolerability, pharmacokinetics, and early efficacy markers including LDL-C reduction and liver fat changes as measured by MRI-PDFF[1][3][5][6].