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-m 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 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 mutations 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. National Cancer Institute. What Are Clinical Trials? Reviewed February 4, 2020. Accessed October 19, 2021. www.cancer.gov/aboutcancer/treatment/clinical-trials/what-are-trials 2. Vigneswaran N, Williams MD. Epidemiologic trends in head and neck cancer and aids in diagnosis. Oral Maxillofac Surg Clin North Am. 2014;26(2):123-141. 3. Sung H, Ferlay J, Siegel R, et al. Global cancer statistics 2020: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin. 2021;71(3):209-249. 4. American Cancer Society. Cancer Facts & Figures 2021. Atlanta, GA: American Cancer Society; 2021. 5. National Cancer Institute. Head and neck cancers. Reviewed March 29, 2017. Accessed June 17, 2021. https:// www.cancer.gov/types/head-and-neck/head-neck-fact-sheet
    6. Malone E, Siu LL. Precision medicine in head and neck cancer: myth or reality? Clin Med Insights Oncol. 2018;12:1179554918779581. 7. Braig F, Voigtlaender M, Schieferdecker A, et al. Liquid biopsy monitoring uncovers acquired RAS-mediated resistance to cetuximab in a substantial proportion of patients with head and neck squamous cell carcinoma. Oncotarget. 2016;7(28):42988-42995. 8. Koontongkaew S. The tumor microenvironment contribution to development, growth, invasion and metastasis of head and neck squamous cell carcinomas. J Cancer. 2013;4(1):66-83. 9. American Cancer Society. Genes and cancer. Revised June 25, 2014. Accessed June 17, 2021. https://www.cancer.org/cancer/cancer-causes/genetics/genesand-cancer.html 10. National Cancer Institute Dictionary of Cancer Terms. Overexpress. Accessed June 17, 2021. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/overexpress 11. Burrows F, Shivani M, Wang Z, et al. Antitumor activity of tipifarnib and PI3K pathway inhibitors in HRASassociated head and neck squamous cell carcinoma. Poster presented at: 32nd EORTC-NCI-AACR Symposium; October 24-25 2020; virtual. 12. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Head and Neck Cancers V.3.2021. © National Comprehensive Cancer Network, Inc. 2021. All rights reserved. Accessed October 19, 2021. To view the most recent and complete version of the guideline, go online to NCCN.org. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way. 13. Cancer.Net. Head and neck cancer: types of treatment. October 2019. Accessed June 17, 2021. https://www.cancer.net/cancer types/headand-neck-cancer/types-treatment 14. American Cancer Society. How chemotherapy drugs work. Revised November 22, 2019. Accessed June 17, 2021. https://www.cancer.org/treatment/treatments-and-side-effects/treatment-types/chemotherapy/how-chemotherapy-drugs-work.html 15. Chow LQM. Head and neck cancer. N Engl J Med. 2020;382(1):60-72.