NPM1 mutation
NPM1 is the most common mutation in adult leukaemia. It moves a nuclear protein into the cytoplasm and, it turns out, makes the leukaemia dependent on menin. This dossier gathers the 1 product (1 approved), 3 trials, 0 pathways and 0 resistance routes in the corpus that involve it, with external identifiers so it can be joined to UniProt, ChEMBL, Open Targets and the rest of biology.
Biology
Nucleophosmin is a nucleolar chaperone; the mutant acquires a nuclear export signal, delocalising to the cytoplasm and dysregulating HOX gene expression via menin-dependent chromatin binding.
- Adult AML (~30%)
- Normal-karyotype AML (50-60%)
Elsewhere: identifiers and databases
Built from HGNC, Ensembl, UniProt and ChEMBL idsHow common it is, by cancer
Full matrix →| Cancer | Prevalence | Measure | Note | Source |
|---|---|---|---|---|
| Acute myeloid leukaemia | 30% | mutation | ~50-60% of cytogenetically normal AML |
Approximate, population-level figures; the measure column says what was counted. Ranges show the midpoint as a bar.
Products by modality and phase
Browse products →| Modality | Approved |
|---|---|
| Small molecule 1 |
Trials
Evidence ranking →| Trial | Phase | Status | Setting | Result | Products |
|---|---|---|---|---|---|
| myeloMATCH NCT05564390 | platform | Recruiting | Newly diagnosed AML and MDS: genomic screening at diagnosis assigns patients to tiered, biomarker-defined sub-studies from induction through MRD-guided consolidation and maintenance | ||
| KOMET-001 NCT04067336 | 1/2 | Positive | Relapsed/refractory NPM1-mutated AML: ziftomenib 600 mg daily monotherapy | CR 23%, ORR 33%, median OS 6.6 months. | |
| AUGMENT-101 NCT04065399 | 1/2 | Positive | Relapsed/refractory KMT2A-rearranged or NPM1-mutated acute leukaemia: revumenib monotherapy | CR+CRh 22.8%, ORR 63.2% in KMT2Ar cohort. |
Resistance routes that involve this target
Unaddressed routes →The resistance atlas has no route that names this target.
Pathways where it is a node
Pathway-to-drug matrix →No pathway diagram carries this target as a node.
Companion diagnostics and assays
Assay registry →No companion diagnostic in the registry measures this target.
Preclinical models
All models →No model entry for this target yet; check the cancer entries on the models page.
Open questions
All open questions →No open questions recorded for this target yet. Suggest one.
Ideas and companies
Key papers and the live literature
Preprints →- AUGMENT-101: revumenib, the first menin inhibitor, in relapsed leukaemias driven by KMT2A rearrangement or NPM1 mutation · Nature 2023
Query for this target: (TITLE:"NPM1 mutation" OR ABSTRACT:"NPM1 mutation" OR TITLE:"NPM1" OR ABSTRACT:"NPM1") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about NPM1 mutation, not a curated reading list.
Export
The dossier as machine-readable JSON: identifiers from HGNC, Ensembl, UniProt and ChEMBL, products with status, trials, pathways, hotspots, open questions and assays. The full entity record is in the open API at /api/v1/entities/npm1.json. Licence CC BY 4.0.