Targeting Menin in T-Lineage Acute Lymphoblastic Leukemia
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DOI: 10.1182/blood-2024-201741
Abstract: Relapsed/refractory (R/R) T-lineage acute lymphoblastic leukemia (T-ALL) has a dismal prognosis with 10,000 nanomolar (nM). Concomitantly, we performed RNA-seq to assess expression of HOXA and downstream targets of menin inhibition. There was no correlation between… read more here.
Keywords: hoxa; menin inhibition; ziftomenib; leukemia ... See more keywords
APAL2020K/ITCC-101: A Phase I Trial of the Menin Inhibitor Ziftomenib in Combination with Chemotherapy in Children with Relapsed/Refractory KMT2A-rearranged, NUP98-rearranged, or NPM1-mutant Acute Leukemias
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DOI: 10.1182/blood-2024-204583
Abstract: Background and Significance: Aberrant expression of HOX family and MEIS1 genes, commonly found in KMT2A-rearranged, NUP98-rearranged, or NPM1-mutant acute leukemias, leads to arrested differentiation and leukemia development. HOX family genes are crucial regulators of normal… read more here.
Keywords: trial; ziftomenib; chemotherapy; leukemia ... See more keywords
A Phase I Study Investigating the Combination of the Ziftomenib, Venetoclax and Azacitidine (ZiVA) in Pediatric Relapsed and Refractory Acute Leukemias
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DOI: 10.1182/blood-2024-208146
Abstract: Background and Significance: KMT2A-rearanged (KMT2A-r) and NPM1-mutated (NPM1-m) leukemia cells exist in an arrested, undifferentiated state mediated by the KMT2A-menin complex. Ziftomenib, an oral inhibitor of the KMT2A-menin interaction, has shown efficacy in adults. KMT2A-rearanged… read more here.
Keywords: acute leukemias; combination; combination ziftomenib; ziftomenib ... See more keywords
Ziftomenib in combination with venetoclax and azacitidine in relapsed/refractory NPM1-m or KMT2A-r acute myeloid leukemia: Updated phase 1a/b safety and clinical activity results from KOMET-007
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DOI: 10.1182/blood-2025-764
Abstract: Introduction: Leukemogenesis is driven by nucleophosmin 1 mutations(NPM1-m) or lysine methyltransferase 2A rearrangements(KMT2A-r) in ~35–40% of acute myeloid leukemia (AML) cases. Nearly half of AML patients will develop relapsed/refractory (R/R) disease within a year, with read more here.
Keywords: combination; ziftomenib; response; clinical activity ... See more keywords
Ziftomenib in combination with venetoclax and azacitidine in newly diagnosed NPM1-m acute myeloid leukemia: Phase 1b results from KOMET-007
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DOI: 10.1182/blood-2025-766
Abstract: Introduction: Nucleophosmin 1 mutations (NPM1-m) drive leukemogenesis in approximately 30% of acute myeloid leukemia (AML) cases. Ziftomenib is a potent, highly selective, oral, investigational menin inhibitor that has demonstrated clinical activity as both monotherapy and… read more here.
Keywords: newly diagnosed; combination; ziftomenib; response ... See more keywords
Ziftomenib in relapsed/refractory (R/R) NPM1 -mutant acute myeloid leukemia (AML): Phase 1b/2 clinical activity and safety results from the pivotal KOMET-001 study.
Sign Up to like & getrecommendations! Published in 2025 at "Journal of Clinical Oncology"
DOI: 10.1200/jco.2025.43.16_suppl.6506
Abstract: 6506 Background: NPM1 -m drives leukemogenesis in ~30% of AML. Despite current risk stratification, nearly half will have R/R disease within a year, after which outcomes are poor with read more here.
Keywords: ziftomenib; clinical activity; pivotal komet; komet 001 ... See more keywords