Oncology Brothers: Practice-Changing Cancer Discussions FDA Approval of Ziftomenib (KOMET-001) in NPM1 mut Acute Myeloid Leukemia (AML) – Dr. Eunice Wang
Nov 17, 2025
Dr. Eunice Wang, a leading hematologist-oncologist at Roswell Park Comprehensive Cancer Center, shares her expertise on the recent FDA approval of Ziftomenib for NPM1-mutant acute myeloid leukemia (AML). She discusses the promising results from the KOMET-001 trial, highlighting a nearly 30% response rate and improved survival for patients. Dr. Wang dives into the mechanism of action of menin inhibitors, management of side effects, and the importance of patient sequencing. She also contrasts Ziftomenib with other treatments, paving the way for future therapies.
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Targeting A Shared Oncogenic Program
- Menin inhibitors target a shared transcriptional program in KMT2A-rearranged and NPM1-mutant AML driven by menin.
- Blocking menin disrupts oncogenic transcription, induces differentiation, and yields clinical responses.
First Patient Treated At Roswell Park
- Roswell Park treated the very first patient worldwide with ziftomenib in relapsed refractory acute leukemia.
- In KOMET-001 expansion, ziftomenib produced CR/CRh and overall responses in heavily pretreated NPM1-mutant patients.
Delayed Responses But Meaningful Survival
- Responses to ziftomenib were seen after a delayed period (median ~2 months) consistent with differentiation rather than cytotoxicity.
- Responders had markedly longer median overall survival (~16.4 months) versus non-responders.