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KOMET-001 was the registration trial for ziftomenib: a quarter of heavily pretreated patients with NPM1-mutated AML reached complete remission on a once-daily pill.
KOMET-001, trial NCT04067336 sponsored by Kura Oncology and reported in 2025, was the registration study for ziftomenib, in which about a quarter of heavily pretreated patients with relapsed or refractory NPM1-mutated acute myeloid leukaemia reached complete remission on a once-daily tablet. In 112 adults on 600 mg daily the complete remission rate was 23 percent and the overall response rate 33 percent, including patients who had received venetoclax and FLT3 inhibitors, and the FDA approved the drug on 13 November 2025. OnCo links it to acute myeloid leukaemia, menin and the NPM1 mutation as targets and to ziftomenib. Frontline combinations in KOMET-007 show much higher remission rates, as expected for an earlier line, and whether menin inhibitors belong there is the open question.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
112 treated.
| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Complete remissionprimary | Ziftomenib | 112 | 23% | - | - | link |
| Overall response rate | Ziftomenib | - | 33% | - | - | - |
| Overall survival (median) | Ziftomenib | - | 6.6 months | - | - | - |
Shares Kura Oncology, Ziftomenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia, Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET).
Shares NPM1 mutation, Ziftomenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia, Menin.
Shares Ziftomenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia, Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Acute myeloid leukaemia.
Shares Kura Oncology, NPM1 mutation, Ziftomenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia.
Shares NPM1 mutation, Ziftomenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia, Menin.
Shares NPM1 mutation, Ziftomenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia, Menin.
Shares NPM1 mutation, Ziftomenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia, Acute myeloid leukaemia.
Shares NPM1 mutation, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia, Menin, Epigenetic therapy roadmap: loosening silenced genes → mutation-specific enzymes → editing the epigenome.