Pexmetinib is primarily investigated for its potential in treating myeloid leukemias, particularly chronic myeloid leukemia (CML) and acute myeloid leukemia (AML). It functions by inhibiting specific tyrosine kinases, including p38α and Tie-2, which play roles in cancer cell proliferation and survival. By targeting these pathways, pexmetinib helps suppress the growth of malignant hematopoietic cells and may overcome resistance to other kinase inhibitors. It has shown activity in preclinical models against both proliferating and quiescent leukemia stem cells, suggesting potential for targeting disease reservoirs that are often resistant to standard therapies. Pexmetinib has been studied in early-phase clinical trials to evaluate its safety and efficacy, especially in patients with advanced or treatment-resistant myeloid malignancies.