KOMET-001
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.
112 adults with relapsed/refractory NPM1-mutated AML: CR 23%, ORR 33%, median OS 6.6 months. Approved 13 November 2025. Responses included patients with prior venetoclax and FLT3 inhibitors. Frontline combinations (KOMET-007) show much higher remission rates, as expected for an earlier line.
- 23 out of 100 people had no sign of cancer on scans or tests with Ziftomenib.
- There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
- This is a surrogate endpoint: it measures the cancer being controlled or absent on scans and tests, which often, but not always, translates into living longer.
- 33 out of 100 people had their tumour shrink with Ziftomenib.
- There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
- A response rate counts how many people had their tumours shrink by a set amount; shrinking is encouraging but does not by itself show that people live longer or feel better.
- Median 6.6 months with Ziftomenib.
- A median is a midpoint: half the people did better than this and half did worse.
- Overall survival counts deaths from any cause, so it is the most direct measure of whether a treatment helps people live longer.
- These results apply to the people the trial enrolled: Relapsed/refractory NPM1-mutated AML: ziftomenib 600 mg daily monotherapy. People in a different situation may not see the same effect.
- The trial selected people by a biomarker (NPM1); the result should not be assumed for people whose cancer does not have it.
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 participants enrolled.
| 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 | — | — | — |
Pages like this
not linked directly; found by shared links- PairingMenin inhibitor + venetoclax + azacitidine
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- PersonTimothy J. Ley
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