Ziftomenib with venetoclax and azacitidine for relapsed/refractory NPM1-mutated acute myeloid leukemia.

Ziftomenib combined with venetoclax and azacitidine achieves a 46% composite complete remission rate in relapsed/refractory NPM1-mutated AML patients at the 600 mg dose.

• Why it matters: Relapsed/refractory NPM1-mutated AML has limited treatment options and poor prognosis, highlighting the need for more effective therapies.
• What they did: The phase 1 KOMET-007 trial evaluated 67 adults with R/R AML receiving ziftomenib plus standard venetoclax/azacitidine, focusing on safety and efficacy at different doses.
• The result: The combination was well tolerated, with deep MRD negativity in many patients and median response duration of 8.6 months, supporting further development of this regimen.