{
  "abstract": "Aim: Bleximenib is a potent, selective menin inhibitor with activity in NPM1-mutated (NPM1m) or KMT2A-rearranged (KMT2Ar) acute myeloid leukemia (AML) Previous data have shown an acceptable safety and efficacy profile in participants (pts) with newly diagnosed (ND) NPM1m or KMT2Ar AML treated with Bleximenib (blex) in combination with intensive chemotherapy (IC). We now report updated safety and efficacy data (clinical cut-off: July 2025) from this combination treatment in IC-eligible ND AML pts. Methods: In the Phase 1b ALE1002 study (NCT05453903), Cohort C1 received standard 7+3 induction (cytarabine plus daunorubicin or idarubicin) with blex 30–100 mg BID starting on Day 4 and continued through count recovery. Patients achieving CR received up to 4 consolidation cycles with intermediate-dose cytarabine plus blex; those not undergoing allo-HSCT could continue blex for up to 12 months. The safety population included all patients receiving blex 30–100 mg BID with 7+3. Relative dose intensity (RDI) was defined as received/planned blex dose per 28-day cycle. The ITT efficacy population included all NPM1m or KMT2Ar patients treated with blex 100 mg BID plus 7+3, regardless of study discontinuation before first response assessment. Responses were assessed per ELN criteria. Results: The safety analysis set included 44 ND AML pts median age 57.0 years; 52.3% female; 56.8% NPM1m, 43.2% KMT2A; 15.9% FLT3 co-mutations; ELN risk classification: 44.2% favorable, 27.9% intermediate, 27.9% adverse. The median duration of follow-up was 6.3 months (range, 1.31–22.51). All 44 pts had ≥1 treatment-emergent adverse event (TEAE, all grades), with the most common being thrombocytopenia (79.5%), neutropenia (72.7%), diarrhea (70.5%), nausea (68.2%), anemia, and febrile neutropenia (both 63.6%). The majority of cytopenia TEAEs were Grade 3/4, consistent with an IC backbone. Of the 44 pts, the 30- and 60-day mortality was 0/44 (0%) and 1/44 (2.3%), respectively. There was no differentiation syndrome (DS) observed. Three TEAEs of QT prolongation were reported, all of which were Grade 1/2 and resolved without blex interruption. Among 37 pts achieving composite CR (cCR=CR + CR with partial hematologic recovery [CRh] + CR with incomplete hematologic recovery [CRi]), the median time from Day 1 of induction to platelet count recovery was 32.0 days (22.0–82.0), and the median time to neutrophil count recovery was 30.0 days (21.0–71.0). During induction, the median RDI of blex was 100% with no required dose reductions of co-agents. Of 24 pts (NPM1m, n=15; KMT2Ar, n=9) in the ITT efficacy dataset receiving blex 100 mg BID in combination with ‘7+3’, overall response rate (ORR; ≥partial response) was 95.8%, cCR was 87.5%, and CR/CRh was 75%. Responses were similar across mutational subtypes. Median time to CR in the 100 mg BID group was 28 days (range, 21–36) and this was similar to the median time to first response. Median duration of response was not reached. Four pts receiving blex 100 mg BID in combination with ‘7+3’ proceeded to allogenic transplant. Conclusions: In ND NPM1m or KMT2Ar AML, the safety profile of blex + ‘7+3’, including count recovery, was consistent with a ‘7+3’ IC backbone, with no DS adverse events and no QTc prolongation signal observed. Combined with early efficacy, the clinical data are supportive of a planned Phase 3 study blex + ‘7+3’ in IC-eligible ND AML pts harboring KMT2Ar or NPM1m.",
  "abstract_shown": true,
  "affiliations": [
    "7Universitätsklinikum Ulm, Ulm, Germany, Germany",
    "Princess Margaret Cancer Centre, Toronto, Canada",
    "26University Cancer Institute Toulouse Oncopole, Toulouse, France",
    "University College London Hospitals NHS Foundation Trust and UCL/NIHR Clinical Research Facility, London, United Kingdom",
    "City of Hope National Medical Center, Duarte, CA, United States",
    "Clínica Universidad de Navarra, Navarra, Spain",
    "Johnson & Johnson USA, United States",
    "START Madrid-FJD Early Phase Unit, University Hospital Fundación Jiménez Díaz, IIS-FJD, Madrid, Spain",
    "Johnson & Johnson Spring House USA, United States",
    "Institut Universitaire du Cancer de Toulouse Oncopole (IUCT-Oncopole) and the Centre Hospitalier Universitaire (CHU) de Toulouse, France",
    "Johnson & Johnson, United State",
    "Centro Oncológico, Hospital de Investigación Humanitas, Italy",
    "Janssen R&D US, United States",
    "Novant Health Cancer Institute in Winston-Salem, North Carolina, United States",
    "Hospital Clínic de Barcelona, Barcelona, Spain",
    "Massachusetts General Hospital, Harvard Medical School, Boston, MA, United States",
    "Johnson & Johnson Spring House, United States",
    "Institute Food and Nutrition, Wageningen, The Netherlands",
    "Aix-Marseille Université, Inserm, CNRS, Institut Paoli-Calmettes, Centre de Recherche en Cancérologie de Marseille, Marseille, France",
    "Hospital de la Santa Creu i Sant Pau, Barcelona, Spain",
    "University Hospital Vall d’Hebron, and Experimental Hematology, Vall d’Hebron Institute of Oncology, Barcelona, Spain",
    "Centre Hospitalier Universitaire de Tours, CIC INSERM U1415, Tours, France",
    "14MD Anderson Cancer Center, University of Texas, Houston, TX, United States",
    "Universitätsklinikum Leipzig, Leipzig, Germany; 16Montefiore Einstein Comprehensive Cancer Center, Bronx, NY, United States",
    "Montefiore Einstein Comprehensive Cancer Center, Bronx, NY, United States",
    "Charité – Universitätsmedizin Berlin, Berlin, Germany",
    "Johnson & Johnson Innovative Medicine, Spring House, PA, United States",
    "Janssen Research & Development, LLC, High Wycombe, United Kingdom",
    "ASST Grande Ospedale Metropolitano Niguarda, Milan, Italy",
    "Johnson & Johnson Innovative Medicine, Cambridge, MA, United States",
    "Sant’Orsola-Malpighi Polyclinic, Bologna, Italy",
    "Janssen Research & Development, LLC, Beerse, Belgium",
    "Hospital Universitario de Leipzig, Germany",
    "Vall d’Hebron Institute of Oncology, Barcelona, Spain",
    "Department of Internal Medicine V; Heidelberg University Hospital, Heidelberg, Germany",
    "6Johnson & Johnson Innovative Medicine, Spring House, PA, United States",
    "The Christie NHS Foundation Trust and University of Manchester, Manchester, United Kingdom",
    "Johnson & Johnson Raritan, United States",
    "Université de Paris Cité Paris, France",
    "Westmead Hospital and University of Sydney, Sydney, Australia"
  ],
  "article_type": null,
  "arxiv": null,
  "author_total": 51,
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      "name": "Marie Luise HüTTER-KRÖNKE"
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      "name": "Christina LOEFGREN"
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  "bibtex": "https://research.draconitic.com/paper/cbhs9kk6mtrf6.bib",
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  "doi": "10.1016/j.htct.2026.107848",
  "fulltext_url": "http://www.sciencedirect.com/science/article/pii/S2531137926015956",
  "id": "cbhs9kk6mtrf6",
  "issns": [
    "2531-1379",
    "2531-1387"
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  "landing_url": "https://doi.org/10.1016/j.htct.2026.107848",
  "language": "en",
  "licence": "CC-BY",
  "licence_label": "CC BY",
  "licence_source": "journal",
  "licence_url": "https://creativecommons.org/licenses/by/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "lcc",
      "RC633-647.5"
    ],
    [
      "openalex",
      "2720|Acute Myeloid Leukemia Research"
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  "openalex": "W7223105645",
  "pdf_url": null,
  "pmcid": null,
  "pmid": null,
  "published": "2026-10-23",
  "publisher": "Elsevier",
  "ris": "https://research.draconitic.com/paper/cbhs9kk6mtrf6.ris",
  "sources": [
    {
      "first_seen": "2026-10-30T13:43:26Z",
      "native_id": "7a06ac5aab3e44edb1234e9c035f4fc2",
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  "subjects": [
    {
      "field": "medicine",
      "origin": "lcc:RC633-647.5",
      "subfield": "endocrinology-metabolism"
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  ],
  "title": "BLEXIMENIB IN COMBINATION WITH INTENSIVE CHEMOTHERAPY: A PHASE 1B STUDY IN NEWLY DIAGNOSED ACUTE MYELOID LEUKEMIA WITH KMT2A OR NPM1 ALTERATIONS",
  "title_text": "BLEXIMENIB IN COMBINATION WITH INTENSIVE CHEMOTHERAPY: A PHASE 1B STUDY IN NEWLY DIAGNOSED ACUTE MYELOID LEUKEMIA WITH KMT2A OR NPM1 ALTERATIONS",
  "type": "peer-reviewed",
  "updated_at": "2026-10-30T13:43:26Z",
  "url": "https://research.draconitic.com/paper/cbhs9kk6mtrf6",
  "venue": "Hematology, Transfusion and Cell Therapy",
  "versions": []
}