Ziftomenib in Relapsed or Refractory NPM1-Mutated AML.

doi:10.1200/JCO-25-01694

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Article

Ziftomenib in Relapsed or Refractory NPM1-Mutated AML.

Journal of clinical oncology : official journal of the American Society of Clinical Oncology - 1 Nov 2025

Wang Eunice S, Montesinos Pau, Foran James, Erba Harry, Rodríguez-Arbolí Eduardo, Fedorov Kateryna, Heiblig Maël, Heidel Florian H, Altman Jessica K, Baer Maria R, Ades Lionel, Pettit Kristen, Peterlin Pierre, Papayannidis Cristina, Berthon Céline, Walter Roland B, Shah Mithun V, Balasubramanian Suresh, Khawandanah Mohamad, Salamero Garcia Olga, Bergeron Julie, Madanat Yazan F, Roboz Gail J, Ulrickson Matthew, Redner Robert L, McCloskey James, Pigneux Arnaud, de la Fuente Burguera Adolfo, Mitra Amitava, Soifer Harris S, Tabachri Marilyn, Zhang Zijing, Riches Marcie, Corum Daniel, Leoni Mollie, Issa Ghayas C, Fathi Amir T

Abstract excerpt

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 the KOMET-001 phase I trial. METHODS: In the registration-enabling phase II part of KOMET-001, patients with relapsed/refractory NPM1-m AML received ziftomenib 600 mg once daily. The primary...

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Identifiers

PMID: 40997296
DOI: 10.1200/JCO-25-01694
PMCID: PMC12573682

Genes

NPM1
KMT2A

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026.

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