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    BIOMARKER:

    NPM1 mutation

    i
    Other names: Nucleophosmin (Nucleolar Phosphoprotein B23, Numatrin), Testicular Tissue Protein Li 128, Nucleolar Protein NO38, Numatrin, NPM1, Nucleophosmin 1, Nucleophosmin/Nucleoplasmin Family, Member 1, Nucleolar Phosphoprotein B23
    Entrez ID:
    4869
    Related biomarkers:
    Expression
    Mutation
    Fusion
    Others
    Related tests:
    22h
    Revumenib in Combination With 7+3 + Midostaurin in AML (clinicaltrials.gov)
    P1, N=22, Recruiting, Richard Stone, MD | Trial completion date: Mar 2027 --> Mar 2028 | Trial primary completion date: Mar 2026 --> Mar 2027
    Trial completion date • Trial primary completion date
    |
    TP53 (Tumor protein P53) • FLT3 (Fms-related tyrosine kinase 3) • ABL1 (ABL proto-oncogene 1) • NPM1 (Nucleophosmin 1) • DNMT3A (DNA methyltransferase 1) • RUNX1 (RUNX Family Transcription Factor 1) • SF3B1 (Splicing Factor 3b Subunit 1) • ASXL1 (ASXL Transcriptional Regulator 1) • KMT2A (Lysine Methyltransferase 2A) • SRSF2 (Serine and arginine rich splicing factor 2) • CREBBP (CREB binding protein) • BCOR (BCL6 Corepressor) • U2AF1 (U2 Small Nuclear RNA Auxiliary Factor 1) • STAG2 (Stromal Antigen 2) • MECOM (MDS1 And EVI1 Complex Locus) • NUP214 (Nucleoporin 214) • GATA2 (GATA Binding Protein 2) • KAT6A (Lysine Acetyltransferase 6A) • ZRSR2 (Zinc Finger CCCH-Type, RNA Binding Motif And Serine/Arginine Rich 2)
    |
    TP53 mutation • FLT3-ITD mutation • NPM1 mutation • Chr del(5q)
    |
    midostaurin • daunorubicin • Revuforj (revumenib)
    1d
    REVEAL-ND NPM1: Study of Revumenib in Combination With Intensive Chemotherapy in Newly Diagnosed Acute Myeloid Leukemia (AML) With a NPM1 Mutation (clinicaltrials.gov)
    P3, N=468, Recruiting, Syndax Pharmaceuticals
    Trial initiation date
    |
    NPM1 (Nucleophosmin 1)
    |
    NPM1 mutation
    |
    cytarabine • Revuforj (revumenib)
    1d
    The interaction between NPMc+ and Orai1 induces abnormal calcium influx to facilitate leukemogenesis. (PubMed, FEBS J)
    These findings uncover a novel mechanism in which NPMc+ interacts with Orai1, disrupting calcium homeostasis and promoting AML progression. This presents a promising therapeutic strategy targeting the NPMc+/Orai1-mediated calcium imbalance in NPM1-mutated AML.
    Journal
    |
    NPM1 (Nucleophosmin 1)
    |
    NPM1 mutation
    2d
    Myeloid dermatosis with features of sweet syndrome and leukemia cutis: a case report. (PubMed, Ann Hematol)
    Prompt immunophenotypic evaluation and genomic testing of myeloid dermatoses, particularly those with blastoid cells, can be critical in distinguishing SS from LC to provide significant insight on prognosis and therapeutic options. This case highlights the clinical and histopathologic spectrum of myeloid dermatoses and further emphasizes the relevance of skin biopsies in the diagnosis of cutaneous involvement by hematologic malignancies, such as myeloid neoplasms.
    Journal
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase) • NPM1 (Nucleophosmin 1) • CD34 (CD34 molecule)
    |
    NPM1 mutation
    2d
    DSP-5336-101: A Phase 1/2 Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia (Horizen-1) (clinicaltrials.gov)
    P1/2, N=606, Recruiting, Sumitomo Pharma America, Inc. | N=362 --> 606
    Enrollment change
    |
    FLT3 (Fms-related tyrosine kinase 3) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • NPM1 (Nucleophosmin 1) • KMT2A (Lysine Methyltransferase 2A)
    |
    FLT3-ITD mutation • FLT3 mutation • NPM1 mutation
    |
    Venclexta (venetoclax) • Xospata (gilteritinib) • azacitidine • enzomenib (DSP-5336)
    7d
    Discovery and preclinical activity of the menin-KMT2A inhibitor ziftomenib in acute leukemia models. (PubMed, Blood)
    We next assessed ziftomenib against four MEN1 (gene encoding menin) mutants (T349M, M327I, G331R, G331D) associated with clinical resistance to another menin inhibitor revumenib. The crystal structures of ziftomenib in complex with menin wild-type, T349M or G331R mutants revealed a similar binding mode of ziftomenib to these menin variants, rationalizing potent inhibitory activity towards these mutants. Ziftomenib has recently received FDA approval for adult patients with NPM1-mutated acute myeloid leukemia and continues to be evaluated clinically in leukemias with NPM1 or KMT2A alterations, both as monotherapy and in combinations.
    Preclinical • Journal
    |
    NPM1 (Nucleophosmin 1) • KMT2A (Lysine Methyltransferase 2A) • NUP98 (Nucleoporin 98 And 96 Precursor 2) • HOXA9 (Homeobox A9) • MEIS1 (Meis Homeobox 1) • MEN1 (Menin 1) • HOXB2 (Homeobox B2)
    |
    NPM1 mutation • KMT2A rearrangement
    |
    Revuforj (revumenib) • Komzifti (ziftomenib)
    7d
    Maintenance Therapy of Hypomethylating Agent (HMA) in Favorable Risk Acute Myeloid Leukemia (AML) Patients (clinicaltrials.gov)
    P2, N=77, Recruiting, The First Affiliated Hospital of Soochow University | Trial primary completion date: Dec 2025 --> Dec 2026
    Trial primary completion date
    |
    NPM1 (Nucleophosmin 1) • CEBPA (CCAAT Enhancer Binding Protein Alpha)
    |
    NPM1 mutation
    |
    azacitidine • decitabine
    8d
    BY002 IIT Study in R/R Acute Leukemia (clinicaltrials.gov)
    P1, N=18, Recruiting, The First Affiliated Hospital of Soochow University | Trial completion date: Dec 2026 --> Jul 2027 | Initiation date: Nov 2025 --> Mar 2026 | Trial primary completion date: Jun 2026 --> Mar 2027
    Trial completion date • Trial initiation date • Trial primary completion date • First-in-human
    |
    NPM1 (Nucleophosmin 1) • KMT2A (Lysine Methyltransferase 2A)
    |
    NPM1 mutation • KMT2A rearrangement
    8d
    AUGMENT-101: A Study of Revumenib in R/R Leukemias Including Those With an MLL/KMT2A Gene Rearrangement or NPM1 Mutation (clinicaltrials.gov)
    P1/2, N=447, Recruiting, Syndax Pharmaceuticals | N=67 --> 447
    Enrollment change
    |
    NPM1 (Nucleophosmin 1) • KMT2A (Lysine Methyltransferase 2A) • NUP98 (Nucleoporin 98 And 96 Precursor 2)
    |
    NPM1 mutation • KMT2A rearrangement
    |
    Revuforj (revumenib) • Tybost (cobicistat)
    10d
    Twenty Years of Therapeutic Leukocytapheresis in Newly Diagnosed Acute Myeloid Leukemia: Insights From A Single Center. (PubMed, J Clin Apher)
    LCP remains a valuable therapeutic option for patients with HL in newly diagnosed AML. Our long-term experience supports its safety and efficacy, particularly in symptomatic patients, as a bridge to definitive therapy regardless of treatment intensity eligibility.
    Retrospective data • Journal
    |
    FLT3 (Fms-related tyrosine kinase 3) • NPM1 (Nucleophosmin 1) • KMT2A (Lysine Methyltransferase 2A)
    |
    FLT3-ITD mutation • NPM1 mutation • KMT2A rearrangement
    10d
    GFH009X2101: Study of SLS009 (Formerly GFH009) a Potent Highly Selective CDK9 Inhibitor in Patients With Hematologic Malignancies and High-Risk Newly Diagnosed AML (clinicaltrials.gov)
    P1/2, N=160, Recruiting, Sellas Life Sciences Group | Trial completion date: Dec 2025 --> Dec 2027 | Trial primary completion date: Dec 2025 --> Dec 2026
    Trial completion date • Trial primary completion date
    |
    PD-L1 (Programmed death ligand 1) • KRAS (KRAS proto-oncogene GTPase) • FLT3 (Fms-related tyrosine kinase 3) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • NPM1 (Nucleophosmin 1) • SF3B1 (Splicing Factor 3b Subunit 1) • ASXL1 (ASXL Transcriptional Regulator 1) • SRSF2 (Serine and arginine rich splicing factor 2) • BCOR (BCL6 Corepressor) • U2AF1 (U2 Small Nuclear RNA Auxiliary Factor 1) • STAG2 (Stromal Antigen 2) • DDX41 (DEAD-Box Helicase 41) • ZRSR2 (Zinc Finger CCCH-Type, RNA Binding Motif And Serine/Arginine Rich 2)
    |
    TP53 mutation • KRAS mutation • FLT3-ITD mutation • Chr del(17p) • IDH1 mutation • IDH2 mutation • FLT3 mutation • TP53 wild-type • NPM1 mutation • KRAS wild-type • Chr del(11q) • ASXL1 mutation • SF3B1 mutation • EZH2 mutation • NRAS wild-type • SRSF2 mutation • IDH wild-type
    |
    Venclexta (venetoclax) • azacitidine • tambiciclib (SLS009)
    13d
    DNMT3B Controls Enhancer-Linked Chromatin and Cell Cycle Networks in Acute Myeloid Leukemia. (PubMed, Cancers (Basel))
    DNMT3B functions as a context-dependent epigenetic regulator linking enhancer-associated chromatin organization with proliferative control and apoptotic resistance in AML. DNMT3B-directed epigenetic perturbation remodels cis-regulatory circuitry and is associated with increased venetoclax responsiveness, supporting DNMT3B-governed networks as a candidate co-targeting axis in high-risk AML.
    Journal • IO biomarker
    |
    NPM1 (Nucleophosmin 1) • DNMT3A (DNA methyltransferase 1) • MCL1 (Myeloid cell leukemia 1) • BCL2L1 (BCL2-like 1) • CEBPA (CCAAT Enhancer Binding Protein Alpha) • DNMT3B (DNA Methyltransferase 3 Beta)
    |
    NPM1 mutation
    |
    Venclexta (venetoclax)