OTS167

OTS167 is an orally available small molecule inhibitor targeting maternal embryonic leucine zipper kinase (MELK), a serine/threonine kinase highly upregulated in various cancers and associated with cancer cell survival, invasiveness, and maintenance of cancer stem cells. By binding to MELK, OTS167 prevents its phosphorylation and activation, thereby inhibiting downstream signaling pathways involved in tumor growth and survival. Preclinical studies have demonstrated potent antitumor activity across multiple cancer types including breast, lung, pancreatic, prostate cancers, neuroblastoma, and small cell lung cancer. The drug has shown the ability to suppress tumor growth in xenograft models by reducing proliferation markers and affecting key molecules such as DEPDC1 and FOXM1 within the MELK pathway. Developed as a first-in-class anti-cancer agent by OncoTherapy Science for oral administration, OTS167 has reached phase II clinical trials for hematologic malignancies (AML, ALL) and advanced myeloid neoplasms (MDSs/MPNs/CML), as well as phase I trials for solid tumors like breast cancer[1][2][3][5][6][7].

Other names

MELK inhibitor OTS167