MK-4101 is a potent, orally bioavailable and brain penetrating Hedgehog pathway (Hh) inhibitor that inhibits Smoothened (Smo) with IC50 of 1.1 uM.
MK-4101 inhibits Hh signaling in a reporter gene assay in cancer cells with IC50 of 1-1.5 uM, also inhibited the proliferation of medulloblastoma cells derived from neonatally irradiated Ptch1−/+ mice in vitro with IC50 of 0.3 uM.
MK-4101 demonstrate a robust antitumor activity against Hh-driven tumors in vivo, shows potentiation for the treatment of medulloblastoma and BCC.
