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

AP232 dihydrochloride is an inhibitor of the U2 auxiliary factor homology motif of U2AF1 (U2AF1-UHM), with an IC50 value of 7.96 μM, demonstrating a selectivity of 2.8-24 times over other UHM-containing proteins (RBM39-UHM, SPF45-UHM, PUF60-UHM, U2AF2-UHM). At a concentration of 40 μM, it stabilizes endogenous full-length U2AF1, reducing its sensitivity to proteinase in K562 cells. AP232 dihydrochloride (1-1000 μM, 72 h) inhibits the proliferation of six leukemia cell lines (MV4-11, NKM-1, K562, MOLM13, HNT-34, MONO-MAC1). Furthermore, concentrations of 5-30 μM over 24-48 hours disrupt the cell cycle in leukemia cells. At 20 μM for 2 hours, it lowers the proportion of functional lysosomes in NKM-1, K562, and MV4-11 cells. Finally, AP232 dihydrochloride at 10-30 μM for 48 hours inhibits autophagy in NKM-1 and K562 cells.