NMS-1286937 (onvansertib), a pyrazoloquinazoline, is a third generation PLK1 ATP-competitor with an in vitro IC50 of 36 nmol/L, and had a strong cytotoxic effect in AML cells, for which it was originally registered by FDA as orphan drug (149). The gene discussed is PLK1; the disease is acute myeloid leukemia.