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Page 1
Menin inhibition with revumenib for NPM1-mutated relapsed or refractory acute myeloid leukemia: the AUGMENT-101 study.
Arellano ML, Thirman MJ, DiPersio JF, Heiblig M, Stein EM, Schuh AC, Žučenka A, de Botton S, Grove CS, Mannis GN, Papayannidis C, Perl AE, Issa GC, Aldoss I, Bajel A, Dickens DS, Kühn MWM, Mantzaris I, Raffoux E, Traer E, Amitai I, Döhner H, Greco C, Kovacsovics T, McMahon CM, Montesinos P, Pigneux A, Shami PJ, Stone RM, Wolach O, Harpel JG, Chudnovsky Y, Yu L, Bagley RG, Smith AR, Blachly JS. Arellano ML, et al. Blood. 2025 Aug 28;146(9):1065-1077. doi: 10.1182/blood.2025028357. Blood. 2025. PMID: 40332046 Free PMC article. Clinical Trial.
The prognosis for relapsed or refractory (R/R) nucleophosmin 1-mutated (NPM1m) acute myeloid leukemia (AML) is poor and represents an urgent unmet medical need. Revumenib, a potent, selective menin inhibitor, was recently ap …
The prognosis for relapsed or refractory (R/R) nucleophosmin 1-mutated (NPM1m) acute myeloid leukemia ( …
Ziftomenib in Relapsed or Refractory NPM1-Mutated AML.
Wang ES, Montesinos P, Foran J, Erba H, Rodríguez-Arbolí E, Fedorov K, Heiblig M, Heidel FH, Altman JK, Baer MR, Ades L, Pettit K, Peterlin P, Papayannidis C, Berthon C, Walter RB, Shah MV, Balasubramanian S, Khawandanah M, Salamero Garcia O, Bergeron J, Madanat YF, Roboz GJ, Ulrickson M, Redner RL, McCloskey J, Pigneux A, de la Fuente Burguera A, Mitra A, Soifer HS, Tabachri M, Zhang Z, Riches M, Corum D, Leoni M, Issa GC, Fathi AT; KOMET-001. Wang ES, et al. J Clin Oncol. 2025 Nov;43(31):3381-3390. doi: 10.1200/JCO-25-01694. Epub 2025 Sep 25. J Clin Oncol. 2025. PMID: 40997296 Free PMC article. Clinical Trial.
PURPOSE: Ziftomenib-a potent, highly selective, oral menin inhibitor-was well tolerated and demonstrated encouraging clinical activity as monotherapy for relapsed/refractory NPM1-mutated (NPM1-m) and KMT2A-rearranged AML in …
PURPOSE: Ziftomenib-a potent, highly selective, oral menin inhibitor-was well tolerated and demonstrated encouraging cl …
Combination Strategies with Menin Inhibitors for Acute Leukemia.
Issa GC, Cai SF, Bataller A, Kantarjian HM, Stein EM. Issa GC, et al. Blood Cancer Discov. 2025 Nov 3;6(6):547-560. doi: 10.1158/2643-3230.BCD-24-0212. Blood Cancer Discov. 2025. PMID: 40900001 Review.
Menin inhibitors are targeted therapies for the treatment of genetically defined subsets of acute leukemia. The menin inhibitor revumenib is approved for the treatment of relapsed or refractory leukemia with rearrangement of KMT2A. In addition, multipl …
Menin inhibitors are targeted therapies for the treatment of genetically defined subsets of acute leukemia. The menin inhibitor
Azacitidine, Venetoclax, and Revumenib for Newly Diagnosed NPM1-Mutated or KMT2A-Rearranged AML.
Zeidner JF, Lin TL, Welkie RL, Curran E, Koenig K, Stock W, Madanat YF, Swords R, Baer MR, Blum W, Stein EM, Olin RL, Schiller G, Nichols A, Odenike O, Traer E, Lachowiez C, Duong VH, Hochman MJ, Cai SF, Smith C, Stefanos M, Martycz M, Huang Y, Rosenberg L, Marcus S, Chen TL, Yocum AO, Druker BJ, Levine RL, Borate U, Byrd JC, Mims AS. Zeidner JF, et al. J Clin Oncol. 2025 Aug 10;43(23):2606-2615. doi: 10.1200/JCO-25-00914. Epub 2025 Jun 12. J Clin Oncol. 2025. PMID: 40504618 Free PMC article. Clinical Trial.
PURPOSE: Azacitidine and venetoclax is a standard frontline treatment regimen for newly diagnosed older adults with AML; however, long-term outcomes remain poor. Revumenib is an oral menin inhibitor with clinical activity in AML patients with nucleopho …
PURPOSE: Azacitidine and venetoclax is a standard frontline treatment regimen for newly diagnosed older adults with AML; however, lon …
Acute myeloid leukemias with UBTF tandem duplications are sensitive to menin inhibitors.
Barajas JM, Rasouli M, Umeda M, Hiltenbrand R, Abdelhamed S, Mohnani R, Arthur B, Westover T, Thomas ME 3rd, Ashtiani M, Janke LJ, Xu B, Chang TC, Rosikiewicz W, Xiong E, Rolle C, Low J, Krishan R, Song G, Walsh MP, Ma J, Rubnitz JE, Iacobucci I, Chen T, Krippner-Heidenreich A, Zwaan CM, Heidenreich O, Klco JM. Barajas JM, et al. Blood. 2024 Feb 15;143(7):619-630. doi: 10.1182/blood.2023021359. Blood. 2024. PMID: 37890156 Free PMC article.
UBTF tandem duplications (UBTF-TDs) have recently emerged as a recurrent alteration in pediatric and adult acute myeloid leukemia (AML). UBTF-TD leukemias are characterized by a poor response to conventional chemotherapy and a transcriptional signature …
UBTF tandem duplications (UBTF-TDs) have recently emerged as a recurrent alteration in pediatric and adult acute myeloid le
Targeting the Menin-KMT2A interaction in leukemia: Lessons learned and future directions.
Perner F, Gadrey JY, Armstrong SA, Kühn MWM. Perner F, et al. Int J Cancer. 2026 Jan 15;158(2):342-356. doi: 10.1002/ijc.35332. Epub 2025 Jan 30. Int J Cancer. 2026. PMID: 39887730 Free PMC article. Review.
Moreover, non-rearranged KMT2A-complexes have been demonstrated to be crucial for disease development and maintenance in NPM1-mutated and NUP98-rearranged leukemia, expanding the spectrum of genetic disease subtypes that are dependent on KMT2A. Recent advances in the devel …
Moreover, non-rearranged KMT2A-complexes have been demonstrated to be crucial for disease development and maintenance in NPM1-mutated …
A 2024 Update on Menin Inhibitors. A New Class of Target Agents against KMT2A-Rearranged and NPM1-Mutated Acute Myeloid Leukemia.
Candoni A, Coppola G. Candoni A, et al. Hematol Rep. 2024 Apr 18;16(2):244-254. doi: 10.3390/hematolrep16020024. Hematol Rep. 2024. PMID: 38651453 Free PMC article. Review.
Menin inhibitors are new and promising agents currently in clinical development that target the HOX/MEIS1 transcriptional program which is critical for leukemogenesis in histone-lysine N-methyltransferase 2A-rearranged (KMT2Ar) and in NPM1-mutated (NPM1mut) acute leukemias …
Menin inhibitors are new and promising agents currently in clinical development that target the HOX/MEIS1 transcriptional program which is c …
Insights into KMT2A rearrangements in acute myeloid leukemia: from molecular characteristics to targeted therapies.
Zehtabcheh S, Soleimani Samarkhazan H, Asadi M, Zabihi M, Parkhideh S, Mohammadi MH. Zehtabcheh S, et al. Biomark Res. 2025 May 13;13(1):73. doi: 10.1186/s40364-025-00786-y. Biomark Res. 2025. PMID: 40361241 Free PMC article. Review.
Acute myeloid leukemia (AML) with KMT2A rearrangements (KMT2A-r) represents a highly aggressive and prognostically unfavorable subtype of leukemia, often resistant to standard treatments and associated with high relapse rates. KMT2A-r, found in 3-10% o
Acute myeloid leukemia (AML) with KMT2A rearrangements (KMT2A-r) represents a highly aggressive and prognostical
Therapeutic Implications of Menin Inhibitors in the Treatment of Acute Leukemia: A Critical Review.
Canichella M, Papayannidis C, Mazzone C, de Fabritiis P. Canichella M, et al. Diseases. 2025 Jul 19;13(7):227. doi: 10.3390/diseases13070227. Diseases. 2025. PMID: 40710017 Free PMC article. Review.
Acute myeloid leukemia (AML) with NPM1m-which accounts for approximately 30% of AML cases and AML or acute lymphoblastic leukemia (ALL) with KMT2Ar-and is present in 5-10% of cases, shares a common pathogenetic mechanism: the aberrant act
Acute myeloid leukemia (AML) with NPM1m-which accounts for approximately 30% of AML cases and AML
Effective Menin inhibitor-based combinations against AML with MLL rearrangement or NPM1 mutation (NPM1c).
Fiskus W, Boettcher S, Daver N, Mill CP, Sasaki K, Birdwell CE, Davis JA, Takahashi K, Kadia TM, DiNardo CD, Jin Q, Qi Y, Su X, McGeehan GM, Khoury JD, Ebert BL, Bhalla KN. Fiskus W, et al. Blood Cancer J. 2022 Jan 11;12(1):5. doi: 10.1038/s41408-021-00603-3. Blood Cancer J. 2022. PMID: 35017466 Free PMC article.
Treatment with Menin inhibitor (MI) disrupts the interaction between Menin and MLL1 or MLL1-fusion protein (FP), inhibits HOXA9/MEIS1, induces differentiation and loss of survival of AML harboring MLL1 re-arrangement (r) and FP, or expressing mutant (mt)-N
Treatment with Menin inhibitor (MI) disrupts the interaction between Menin and MLL1 or MLL1-fusion protein (FP), inhibits HOXA …
65 results