WS016 (Waterstone Pharmaceuticals)

WS016 is an investigational oral small molecule inhibitor of proprotein convertase subtilisin/kexin type 9 (PCSK9) developed by Waterstone Pharmaceutical for the treatment of hyperlipidemia, including primary hypercholesterolemia and mixed dyslipidemia. The drug functions by blocking the interaction between PCSK9 and the low-density lipoprotein receptor (LDLR), which prevents the degradation of LDLR and facilitates the removal of LDL cholesterol from the blood. WS016 is part of a new class of oral PCSK9 inhibitors aimed at providing a more convenient alternative to injectable monoclonal antibodies. It has received FDA clearance for Phase 3 clinical trials in the United States and is also undergoing Phase 3 development in China.