Protein / target

Nucleophosmin

NPM1P06748Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
2
Approved medicines
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

Core promoter sequence-specific DNA binding

Strongest disease association

dyskeratosis congenita

Genetic literature evidence · score 0.61

Therapeutic maturity

Clinically validated target

2 approved medicines against this target

Druggability

Small molecule

Open Targets tractability · Approved Drug

Clinical development

2 approved

30 linked trials

Derived from structured UniProt, Open Targets and literature data on this page.

Protein profile

UniProt 2026_02

Canonical identity and biological annotation from UniProt.

Function

Involved in diverse cellular processes such as ribosome biogenesis, centrosome duplication, protein chaperoning, histone assembly, cell proliferation, and regulation of tumor suppressors p53/TP53 and ARF. Binds ribosome presumably to drive ribosome nuclear export. Associated with nucleolar ribonucleoprotein structures and bind single-stranded nucleic acids. Acts as a chaperonin for the core histones H3, H2B and H4. Stimulates APEX1 endonuclease activity on apurinic/apyrimidinic (AP) double-stranded DNA but inhibits APEX1 endonuclease activity on AP single-stranded RNA. May exert a control of APEX1 endonuclease activity within nucleoli devoted to repair AP on rDNA and the removal of oxidized rRNA molecules. In concert with BRCA2, regulates centrosome duplication. Regulates centriole duplication: phosphorylation by PLK2 is able to trigger centriole replication. Negatively regulates the activation of EIF2AK2/PKR and suppresses apoptosis through inhibition of EIF2AK2/PKR autophosphorylation. Antagonizes the inhibitory effect of ATF5 on cell proliferation and relieves ATF5-induced G2/M blockade (PubMed:22528486). In complex with MYC enhances the transcription of MYC target genes (PubMed:25956029). May act as chaperonin or cotransporter in the nucleolar localization of transcription termination factor TTF1 (By similarity)

Subcellular location

Nucleus, nucleolusNucleus, nucleoplasmCytoplasm, cytoskeleton, microtubule organizing center, centrosome
Domains and Gene Ontology detail (62)

Gene Ontology

  • Ccentrosome
  • Ccytoplasm
  • Ccytosol
  • Cfocal adhesion
  • Cgranular component
  • Clarge ribosomal subunit
  • Cmembrane
  • Cnuclear speck
  • Cnucleolus
  • Cnucleoplasm
  • Cnucleus
  • Cprotein-containing complex

294 aa · 33 kDa · 3 isoforms

Biological roles

Reactome v97

What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.

Transcriptional regulationUniProt · GO · ReactomeKinase signallingUniProt · GOApoptosis & cell deathGO
View supporting evidence

Transcriptional regulation

  • ·Involved in diverse cellular processes such as ribosome biogenesis, centrosome duplicati…
  • ·DNA-binding transcription factor binding
  • ·transcription coactivator activity
  • ·positive regulation of DNA-templated transcription

Kinase signalling

  • ·Involved in diverse cellular processes such as ribosome biogenesis, centrosome duplicati…
  • ·protein kinase binding
  • ·protein kinase inhibitor activity

Apoptosis & cell death

  • ·negative regulation of apoptotic process
View underlying pathways (10)

Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.

Interaction neighbourhood

STRING v12.0

Proteins with the strongest functional or physical association. Node size and line weight reflect STRING confidence; hover or select a partner to inspect one association.

NCLCDKN2ATP53ALKAGO2HJURPH4C6RPL5CTCFMDM2NPM1

10 strongest partners — larger node and heavier line mean higher confidence

Functional and physical associations from STRING v12.0. Only associations with this protein are drawn — partner-to-partner links are not part of this evidence.

Drugs targeting this protein

1

Whether each drug engages this protein directly or through a complex, and how many other targets are recorded for it. Direct binders with few recorded targets are listed first.

crizotinib
ApprovedInhibitor

NPM/ALK (Nucleophosmin/ALK tyrosine kinase receptor) inhibitor

Appears in clinical studies involving non-small cell lung carcinoma, non-small cell lung carcinoma, non-small cell lung carcinoma, neoplasm

Direct interaction with this protein · 1 of 6 recorded protein targets

ChEMBL mechanism, action type and target identity. Open Targets clinical status and indications.

Translational evidence

Open Targets 26

Why this target matters therapeutically — disease associations, drugs in development, tractability and safety, from Open Targets.

Strongest genetic associations

Human genetic evidence — the most direct causal link between this target and a disease.

dyskeratosis congenita0.61

Genetic literature · overall 0.51

acute myeloid leukemia0.30

Genetic literature · overall 0.66

Highest-confidence therapeutic associations

Diseases where a drug acting on this target has already reached clinical development.

non-small cell lung carcinoma0.97

Clinical · overall 0.66

neoplasm0.83

Clinical · overall 0.53

cancer0.49

Clinical · overall 0.68

anaplastic large cell lymphoma0.18

Clinical · overall 0.41

Highest overall evidence

Remaining associations by Open Targets' aggregated evidence score.

neurodegenerative disease0.55

Pathway

HIV infectious disease0.54

Pathway

Hodgkins lymphoma0.38

Somatic mutation

lymphoid neoplasm0.37

Somatic mutation

Show all associations
cancer0.68
non-small cell lung carcinoma0.66
acute myeloid leukemia0.66
neurodegenerative disease0.55
HIV infectious disease0.54
neoplasm0.53
dyskeratosis congenita0.51
anaplastic large cell lymphoma0.41
Hodgkins lymphoma0.38
lymphoid neoplasm0.37

Open Targets ranks 661 associations for this target and we store the top 10 by aggregated score. The long tail is largely literature co-mention and RNA-expression evidence, not evidence that this target drives those diseases.

Known drugs · 2 total

CRIZOTINIBApproval

non-small cell lung carcinoma · non-small cell lung carcinoma · non-small cell lung carcinoma

CERITINIBApproval

non-small cell lung carcinoma · non-small cell lung carcinoma · non-small cell lung carcinoma

Tractability

SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · Druggable FamilyPR · UniProt UbiquitinationPR · Database UbiquitinationPR · Half-life DataPR · Small Molecule Binder

Clinical trials

30

Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein.

Show remaining trials (24)

ACTIVE_NOT_RECRUITING · via crizotinib · NCT02465060

ACTIVE_NOT_RECRUITING · via crizotinib · NCT06357975

ClinicalTrials.gov via the drug-target graph.

Forefront confidence

Synthesis

Our own weighting of the evidence behind each disease association. Human genetics and clinical evidence count for most; literature co-mention counts for little, because two entities sharing an abstract is not evidence that one drives the other.

acute myeloid leukemiaWell supported
0.83
agreement 0.740.91
Somatic mutation37%Pathway21%Genetic literature17%Literature11%Animal model8%Clinical6%Geneticdup

Open Targets aggregate 0.66 · 6 independent evidence families · 1 not counted as duplicate

non-small cell lung carcinomaWell supported
0.81
agreement 0.690.93
Clinical68%Somatic mutation28%Literature4%

Open Targets aggregate 0.66 · 3 independent evidence families

neoplasmModerately supported
0.68
agreement 0.520.83
Clinical82%Literature18%

Open Targets aggregate 0.53 · 2 independent evidence families

cancerModerately supported
0.67
agreement 0.540.80
Pathway44%Clinical41%Literature15%

Open Targets aggregate 0.68 · 3 independent evidence families

dyskeratosis congenitaModerately supported
0.55
agreement 0.410.69
Genetic91%Literature9%Genetic literaturedup

Open Targets aggregate 0.51 · 2 independent evidence families · 1 not counted as duplicate

This ranking differs from Open Targets' own: re-weighting moves genetically-evidenced diseases above more heavily co-mentioned ones. The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the underlying per-type scores are shown above so the calculation can be checked.

What's happening now

6

Recent activity around this target, drawn from one canonical event stream. Every item is reached through a drug that targets this protein, so each event is news about that drug rather than about the protein directly.

  1. New publication2024-05-31
    Lorlatinib Versus Crizotinib in Patients With Advanced <i>ALK</i>-Positive Non-Small Cell Lung Cancer: 5-Year Outcomes From the Phase III CROWN Study.

    Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2024 · 254 citations · Europe PMC · via crizotinib

  2. New publication2021-09-16
    Brigatinib Versus Crizotinib in ALK Inhibitor-Naive Advanced ALK-Positive NSCLC: Final Results of Phase 3 ALTA-1L Trial.

    Journal of thoracic oncology : official publication of the International Association for the Study of Lung Cancer · 2021 · 300 citations · Europe PMC · via crizotinib

  3. Safety communication2015-11-12

    Drug Safety Update: Crizotinib (Xalkori▼): risk of cardiac failure

    mhra · safety · mhra · via crizotinib

  4. New publication2014-12-01
    First-line crizotinib versus chemotherapy in ALK-positive lung cancer.

    The New England journal of medicine · 2014 · 2,470 citations · Europe PMC · via crizotinib

  5. Regulatory approval2012-10-23

    Approval: Xalkori (EMA)

    ema · regulatory · ema · via crizotinib

  6. New publication2012-01-03
    ROS1 rearrangements define a unique molecular class of lung cancers.

    Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2012 · 1,121 citations · Europe PMC · via crizotinib

Objective event titles are shown unmodified; the event kind and significance line are derived from structured fields. Forefront AttentionEvent stream aggregating Europe PMC Regulatory filings ClinicalTrials.gov.