The goal of the Phase 2 registration-enabling expansion is to further evaluate the anti-tumor activity and tolerability of ziftomenib in patients with NPM1-mutant (NPM1-m) relapsed or refractory (R/R) acute myeloid leukemia (AML).


Ziftomenib, an oral investigational drug candidate, is a novel compound targeting the menin-KMT2A (MLL) interaction for treatment of genetically defined AML patients with high unmet need. In preclinical models, ziftomenib inhibits the KMT2A (MLL) protein complex and has downstream effects on HOXA9/MEIS1 expression.

  • Epigenetic modifications by the menin-KMT2A (MLL) complex lead to expression of HOXA9/MEIS1, which leads to proliferation, stemness and differentiation block.1
  • These KMT2A (MLL) rearrangements alter normal histone methyltransferase function of KMT2A (MLL), resulting in sustained high HOX levels and blockage of hematopoietic differentiation, ultimately leading to acute leukemia.1-3
  • Mutant NPM1 is also dependent on the interaction between menin and wild-type KMT2A (MLL) to drive leukemogenic gene expression.1
  • Ziftomenib is an investigational small molecule inhibitor of the menin-KMT2A (MLL) complex that may inhibit the survival, growth, and proliferation of certain kinds of leukemia cells as demonstrated in vitro.4


have NPM1-m with or without other gene mutations5,6


Banner MD Anderson Cancer Center

Gilbert, Arizona, United States, 85234

Centre Hospitalier Lyon Sud

Pierre-Bénite, France, 69495

CHU de Bordeaux

Bordeaux, France, 33000

CHU de Lille

Lille, France, 59000

CHU de Nantes

Nantes, France, 44000

Duke Cancer Institute

Durham, North Carolina, United States, 27710

Fred Hutchinson Cancer Research Center

Seattle, Washington, United States, 98109

Hackensack University Medical Center

Hackensack, NJ 07601

Harold C. Simmons Comprehensive Cancer Center – UT Southwestern Medical Center

Dallas, Texas, United States, 75390
Contact: Yasmeen Akhtar, MBBS,MS,CCRP

Hopital Saint Louis

Paris, France, 75475

Hospital Universitari i Politecnic La Fe

Valencia, Spain, 46026

Hospital Universitari Vall d’Hebron

Barcelona, Spain 08035

Hospital Universitario Central de Asturias

Oviedo, Spain, 33011

Hospital Universitario Sanchinarro

Madrid, Spain, 28050

Hospital Universitario Virgen del Rocio

Sevilla, Spain, 41013

Indiana University Melvin and Bren Simon Comprehensive Cancer Center

Indianapolis, Indiana, United States, 46202
Contact: Jill Weisenbach, RN

Institut Gustave Roussy

Villejuif, France, 94800

Institute of Hematology and Medical Oncology “L. and A. Seragnoli”

Emilia-Romagna, Italy, 40138

Karmanos Cancer Institute

Detroit, Michigan, United States, 48201

Massachusetts General Hospital

Boston, Massachusetts, United States, 02114

Mayo Clinic

Rochester, Minnesota, United States, 55905

Mayo Clinic

Jacksonville, Florida, United States, 32224

Mayo Clinic

Phoenix, Arizona, United States, 85054

MD Anderson Cancer Center

Madrid, Spain, 28033

MD Anderson Cancer Center

Houston, Texas, United States, 77030

Northwestern University

Chicago, Illinois, United States, 60611

Roswell Park Comprehensive Cancer Center

Buffalo, New York, United States, 14203

The Mount Sinai Hospital

New York, New York, United States, 10029

UCLA Bowyer Oncology Center

Los Angeles, California, United States, 90095

Universitat de Barcelona

Barcelona, Spain 08035

University of Maryland Greenebaum Comprehensive Cancer Center

Baltimore, Maryland, United States, 21201

University of Michigan Hospitals

Ann Arbor, Michigan, United States, 48109

UPMC Hillman Cancer Center

Pittsburg, PA, United States, 15232

Vanderbilt-Ingram Cancer Center

Nashville, Tennessee, United States, 37232

Weill Cornell

New York, NY 10021

For more information, please email: KO-MEN-001@kuraoncology.com.
To connect with a medical science liaison (MSL), please email: medicalaffairs@kuraoncology.com.


  • 1.Kühn MW, Song E, Feng Z et al. Targeting chromatin regulators inhibits leukemogenic gene expression in NPM1 mutant leukemia. Cancer Discov. 2016;6(10):1166-1181. 2. Klossowski S, Miao H, Kempinska K, et al. Menin inhibitor MI-3454 induces remission in MLL1-rearranged and NPM1-mutated models of leukemia. J Clin Invest. 2020;130(2):981-997. 3. Chan AKN, Chen CW. Rewiring the epigenetic networks in MLL-rearranged leukemias: epigenetic dysregulation and pharmacological interventions. Front Cell Dev Biol. 2019;7:81. 4. Burrows F, Wu T, Kessler L, et al. A novel small molecule menin-MLL inhibitor for potential treatment of MLL-rearranged leukemias and NPM1/DNMT3A-mutant AML [abstract]. In: Proceedings of the AACR EORTC International Conference: Molecular Targets and Cancer Therapeutics; 2017 Oct 26-30; Philadelphia, PA. Philadelphia (PA): AACR; Mol Cancer Ther. 2018;17(1 Suppl): Abstract nr LB-A27. 5. Thiede C, Koch S, Creutzig E, et al. Prevalence and prognostic impact of NPM1 mutations in 1485 adult patients with acute myeloid leukemia (AML). Blood. 2006;107(10):4011-4020. 6. Prata PH, Bally C, Prebet T, et al. NPM1 mutation is not associated with prolonged complete remission in acute myeloid leukemia patients treated with hypomethylating agents. Haematologica. 2018;103(10):e455-e457.