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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), 5 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.
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.
| 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.
| Modality | Approved |
|---|---|
| Small molecule 1 |
| Trial | Setting | Result | Products | ||
|---|---|---|---|---|---|
| 3 | Recruiting | Phase 3 Randomized, Double-blind, Placebo-controlled Studies Assessing Ziftomenib in Combination With Either Standard of Care Nonintensive (Venetoclax+Azacitidine) or Intensive (7+3) Therapy in Patients With Untreated NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia | - | ||
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 | - | |
| 2 | Recruiting | A Phase 2, Multicenter, Open-Label Study of Ziftomenib Monotherapy in Japanese Patients With Relapsed or Refractory Acute Myeloid Leukemia With NPM1 Mutation | - | ||
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. |
No recorded escape route names this target.
No pathway diagram carries this target as a node.
No companion diagnostic in the registry measures this target.
No model entry for this target yet; check the cancer entries on the models page.
No open questions recorded for this target yet. Suggest one.
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.
The dossier as machine-readable JSON, at /api/v1/dossiers/npm1.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-NC 4.0.