LCL161

LCL161 is an orally bioavailable small molecule and a second mitochondrial-derived activator of caspases (SMAC) mimetic that functions as an inhibitor of apoptosis protein (IAP) antagonist. It binds to IAP family members such as X chromosome-linked inhibitor of apoptosis protein (XIAP), cellular inhibitor of apoptosis protein 1 (cIAP1), and cellular inhibitor of apoptosis protein 2 (cIAP2) with high affinity, promoting their degradation and preventing them from inhibiting caspases. By antagonizing these proteins, which are often overexpressed in cancer cells to evade apoptosis, LCL161 restores apoptotic signaling pathways and induces programmed cell death in tumor cells. The drug has demonstrated potent antitumor activity in preclinical models across a range of solid tumors and hematologic malignancies. It has been investigated primarily for the treatment of cancers including breast cancer, ovarian cancer, leukemia, multiple myeloma, myelofibrosis, small cell lung cancer, hepatocellular carcinoma (HCC), esophageal carcinoma, and other neoplasms. Developed by Novartis Pharmaceuticals.

Other names

SMAC mimetic LCL161