LBW242 is a 3-mer and Smac mimetic and is an effective and orally active proapoptotic IAP inhibitor. LBW242 has activity against multiple myeloma and potentiates TRAIL- and anticancer drug-mediated cell death of ovarian cancer cells. LBW242 displays effects on mutant FLT3-expressing cells.

LBW242 partially inhibits the growth of mutant FLT3-expressing lines, MV4;11 at 1 μM. LBW242 kills cells in a manner strictly dependent on caspases, and death is accompanied by PARP cleavage, Annexin positivity, and accumulation of cells in sub-G1. LBW242 is a 3-mer and Smac mimetic, based on the ability of the NH2-terminal seven amino acids of Smac to neutralize the BIR3 domain of X-chromosome-linked IAP. LBW242 (0-0.1 μM; 3 days) inhibits a panel of PKC412-sensitive or PKC412-resistant, mutant FLT3-expressing Ba/F3 lines The IC50 values ranged from 0.5 to >1 μM[1].

LBW242 (50 mg/kg; p.o.; daily for 10 days) decreases tumor burden[1].