• Ziftomenib, a selective menin inhibitor, demonstrates promising clinical activity in relapsed/refractory AML patients, especially those with _NPM1_ mutations or _KMT2A_ rearrangements.
• The KOMET-001 trial revealed a 25% complete remission rate in patients with _KMT2A_ rearrangement or _NPM1_ mutations at the 600 mg dose, with a 35% complete remission rate in _NPM1_ mutated patients.
• Ziftomenib exhibits a manageable safety profile, with the most common severe adverse events including anemia, febrile neutropenia, and pneumonia, and a low incidence of _MEN1_ mutation development.
• Ongoing clinical studies are exploring ziftomenib in combination with other therapies, such as 7+3 induction chemotherapy and azacytidine/venetoclax, to enhance its effectiveness in AML treatment.