Researchers and clinicians are intensifying efforts to develop menin inhibitors as a targeted therapy for KMT2A‑rearranged acute leukemia. The development addresses a genetically defined, treatment‑resistant form of leukemia that disproportionately affects pediatric and young adult patients and carries a poor prognosis. Early translational work and preclinical data have shown that blocking the menin–MLL interaction can reverse leukemogenic transcriptional programs in KMT2A‑driven tumors. Clinical programs are advancing toward early‑phase testing; investigators and trial sponsors are focusing on safety, target engagement and combination strategies with standard chemotherapy and other epigenetic agents.