AP232 is a selective inhibitor of U2AF1-UHM with an IC50 value of 7.96 μM. It shows 2.8 to 24 times selectivity over other UHM-containing proteins. AP232 exhibits anti-leukemia activity and demonstrates heightened effectiveness in cell lines with splicing factor mutations. It can induce G2/M and G1 phase arrest in leukemia cells, impair lysosomal acidification, and inhibit autophagy (autophagy). AP232 is applicable in cancer research, particularly for leukemia studies.

AP232 inhibits the U2 auxiliary factor homolog motif of U2AF1 (U2AF1-UHM) with an IC50 value of 7.96 μM and demonstrates 2.8-24 times selectivity over other UHM-containing proteins (RBM39-UHM, SPF45-UHM, PUF60-UHM, U2AF2-UHM). At a concentration of 40 μM, AP232 stabilizes endogenous full-length U2AF1 and reduces its sensitivity to proteinase K in K562 cells. AP232 also inhibits the growth of six leukemia cell lines (MV4-11, NKM-1, K562, MOLM13, HNT-34, MONO-MAC1) at concentrations ranging from 1-1000 μM over 72 hours. Furthermore, AP232 at 5-30 μM disrupts the cell cycle of leukemia cells within 24-48 hours. A concentration of 20 μM over 2 hours decreases the proportion of functional lysosomes in NKM-1, K562, and MV4-11 cells. Additionally, AP232 at 10-30 μM over 48 hours inhibits autophagy in NKM-1 and K562 cells.