LEQ506

LEQ506 is an orally bioavailable small-molecule antagonist of the Smoothened (SMO) receptor, a key component of the Hedgehog signaling pathway. By selectively binding to and inhibiting SMO, LEQ506 suppresses Hedgehog pathway activation, which can inhibit tumor cell growth in cancers driven by this pathway. It is considered a second-generation SMO inhibitor with potent activity (IC50s of 2 nM in humans and 4 nM in mice). LEQ506 has demonstrated the ability to penetrate the blood–brain barrier and has been investigated primarily for advanced solid tumors, recurrent or refractory medulloblastoma (a brain cancer), and locally advanced or metastatic basal cell carcinoma. The drug was developed by Novartis and has undergone Phase I clinical trials[1][2][3][4][5][6].