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    DRUG CLASS:

    Menin-MLL inhibitor

    Related drugs:
    14h
    CAR T cells derived from a novel, high-affinity anti-CLL-1 monoclonal antibody exhibit a significant anti-AML effect. (PubMed, Cancer Immunol Immunother)
    Additionally, treatment with revumenib, a menin inhibitor, increased CLL-1 expression in AML cells harboring mixed-lineage leukemia (MLL) fusion genes or the NPM1 mutation, making them more susceptible to 2-23 CAR T cell-mediated cytotoxicity in vitro. These findings suggest that the clinical efficacy of CAR T cells derived from the novel, high-affinity anti-CLL-1 mAb 2-23 should be tested in patients with CLL-1-positive AML and also that combining 2-23 CAR T cells with revumenib may benefit some patients with CLL-1low/- AML.
    Journal
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    NPM1 (Nucleophosmin 1)
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    NPM1 mutation
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    Revuforj (revumenib)
    3d
    A Phase Ia/Ib Trial of Revumenib Combined With Cytarabine, Daunorubicin, and Gemtuzumab Ozogamicin (GO) in Frontline and Relapsed /Refractory Pediatric Acute Leukemia Patients (clinicaltrials.gov)
    P1, N=32, Not yet recruiting, M.D. Anderson Cancer Center | Initiation date: Dec 2025 --> Dec 2027
    Trial initiation date
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    KMT2A (Lysine Methyltransferase 2A) • NUP98 (Nucleoporin 98 And 96 Precursor 2)
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    cytarabine • Mylotarg (gemtuzumab ozogamicin) • daunorubicin • Revuforj (revumenib)
    3d
    Targeting menin in T-lineage acute lymphoblastic leukemia. (PubMed, Mol Cancer Ther)
    We tested menin inhibitors (ziftomenib, revumenib, VTP-50469) in 14 primary T-ALL samples and 8 cell lines, representing HOXA-high and HOXA-low genotypes. In conclusion, a subset of T-ALL, defined by high p-MEF2C S222, is sensitive to menin inhibition. Combining ziftomenib with CDK or ERK inhibition offers synergistic efficacy, supporting biomarker-driven clinical trials of this strategy in relapsed/refractory T-ALL.
    Journal
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    KMT2A (Lysine Methyltransferase 2A) • MEIS1 (Meis Homeobox 1) • CDK1 (Cyclin-dependent kinase 1) • MEF2C (Myocyte Enhancer Factor 2C)
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    Revuforj (revumenib) • Komzifti (ziftomenib) • VTP-50469
    5d
    How [14C]-DSP-5336 is Absorbed, Broken Down, and Removed From the Body After a Single Oral Dose in Patients With Advanced Blood Cancers (clinicaltrials.gov)
    P1, N=8, Not yet recruiting, Sumitomo Pharma America, Inc.
    New P1 trial
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    enzomenib (DSP-5336)
    10d
    BN104-101: A Study of BN104 in the Treatment of Acute Leukemia (clinicaltrials.gov)
    P1/2, N=66, Active, not recruiting, Institut de Recherches Internationales Servier (I.R.I.S.) | Trial completion date: Jun 2027 --> Dec 2027 | Trial primary completion date: Jun 2026 --> Dec 2026
    Trial completion date • Trial primary completion date
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    NPM1 (Nucleophosmin 1) • KMT2A (Lysine Methyltransferase 2A) • NUP98 (Nucleoporin 98 And 96 Precursor 2) • MEIS1 (Meis Homeobox 1)
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    NPM1 mutation • KMT2A rearrangement • KMT2A mutation • MLL mutation
    10d
    KO-MEN-017: Studies testing Ziftomenib together with standard treatments in adults with newly diagnosed acute myeloid leukemia (AML) that has specific genetic changes (NPM1 or KMT2A) (2025-521314-25-00)
    P2/3, N=411, Not yet recruiting, Kura Oncology Inc.
    New P2/3 trial
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    FLT3 (Fms-related tyrosine kinase 3) • NPM1 (Nucleophosmin 1) • KMT2A (Lysine Methyltransferase 2A)
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    Venclexta (venetoclax) • cytarabine • azacitidine • Komzifti (ziftomenib)
    11d
    Overcoming Menin inhibitor resistance in AML cells with combinations including BET proteins and dual BRG1/BRM inhibitor. (PubMed, Blood)
    Compared to each drug alone, co-treatment with SNDX-5613 (revumenib) and FHD-286 or OTX015 and FHD-286 significantly reduced the in vivo AML burden and improved survival of the immune depleted mice, without inducing significant toxicity, in the xenograft models of MITR and MI-resistant PD MLL1-r AML cells. These findings highlight novel, targeted, drug combinations that overcome MI resistance in AML cells with MLL1-r or mtNPM1.
    Journal
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    NPM1 (Nucleophosmin 1) • SMARCA4 (SWI/SNF related, matrix associated, actin dependent regulator of chromatin, subfamily A, member 4) • CREBBP (CREB binding protein) • HOXA9 (Homeobox A9) • MEIS1 (Meis Homeobox 1) • BRD4 (Bromodomain Containing 4) • SMARCA2 (SWI/SNF Related, Matrix Associated, Actin Dependent Regulator Of Chromatin, Subfamily A, Member 2)
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    NPM1 mutation • MLL rearrangement • MLL mutation
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    Revuforj (revumenib) • birabresib (OTX015) • camibirstat (FHD-286)
    13d
    STRIVE: Revumenib Treatment in Combination with FLA Chemotherapy in Paediatric Refractory/Relapsed KMT2A-r, NUP98-r or NPM1-mut Acute Myeloid Leukemia (AML) (2025-521390-14-00)
    P1/2, N=31, Not yet recruiting, GPHS Deutsche Paediatrisch-haematologische Studiengesellschaft mbH
    New P1/2 trial
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    NPM1 (Nucleophosmin 1) • KMT2A (Lysine Methyltransferase 2A) • NUP98 (Nucleoporin 98 And 96 Precursor 2)
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    NPM1 mutation
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    cytarabine • Revuforj (revumenib) • fludarabine IV
    13d
    KO-MEN-017: Studies testing Ziftomenib together with standard treatments in adults with newly diagnosed acute myeloid leukemia (AML) that has specific genetic changes (NPM1 or KMT2A) (2025-521314-25-00)
    P2/3, N=144, Not yet recruiting, Kura Oncology Inc.
    New P2/3 trial
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    FLT3 (Fms-related tyrosine kinase 3) • NPM1 (Nucleophosmin 1) • KMT2A (Lysine Methyltransferase 2A)
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    Venclexta (venetoclax) • cytarabine • azacitidine • Komzifti (ziftomenib)
    18d
    Phase 1/1b Trial Of Olutasidenib And Ziftomenib For NPM1 And IDH1 Co-Mutated Acute Myeloid Leukemia (clinicaltrials.gov)
    P1, N=20, Not yet recruiting, M.D. Anderson Cancer Center
    New P1 trial
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    IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • NPM1 (Nucleophosmin 1)
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    NPM1 mutation
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    Komzifti (ziftomenib) • Rezlidhia (olutasidenib)
    18d
    D8620C00001: A trial to learn how safe AZD3632 is, how well it works, and how it moves throughout the body over time in adults with acute leukemia and myelodysplastic syndrome with HOX gene overexpression (2025-521299-76-00)
    P1/2, N=17, Recruiting, AstraZeneca AB
    New P1/2 trial
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    NPM1 (Nucleophosmin 1) • RUNX1 (RUNX Family Transcription Factor 1) • KMT2A (Lysine Methyltransferase 2A) • ETV6 (ETS Variant Transcription Factor 6) • CREBBP (CREB binding protein) • NUP98 (Nucleoporin 98 And 96 Precursor 2) • NUP214 (Nucleoporin 214) • CDX2 (Caudal Type Homeobox 2)
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    NPM1 mutation • MLL mutation
    18d
    HOVON 181 AML: A study to investigate the effect of adding bleximenib to standard-of-care treatment with intensive chemotherapy in patients with a specific type of acute myeloid leukemia (AML). (2025-522767-15-00)
    P2/3, N=271, Recruiting, Hemato-Oncologie voor Volwassenen Nederland (Hovon) Stichting
    New P2/3 trial
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    NPM1 (Nucleophosmin 1) • KMT2A (Lysine Methyltransferase 2A)
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    NPM1 mutation
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    bleximenib (JNJ-6617)