Final Landmark Clinical Trial Data Demonstrates Deep, Durable Responses to Menin Inhibition in Acute Leukemia Patients

BUFFALO, N.Y. — Final data from a landmark clinical trial in patients with an aggressive form of leukemia were published today in the Journal of Clinical Oncology. The results of the phase 1B/2 KOMET-001 clinical trial co-led by Eunice Wang, MD, Chief of Leukemia at Roswell Park Comprehensive Cancer Center, show that ziftomenib produced deep, durable responses in patients with recurrent or treatment-resistant acute myeloid leukemia (AML) with NPM1 mutations.

“Ziftomenib is a potent menin inhibitor with proven efficacy for the treatment of adult patients with NPM1-mutant acute myeloid leukemia who have failed multiple lines of therapy,” says Dr. Wang, Co-Principal Investigator on the study. “I am proud to say that Roswell Park patients were among the first in the world to be treated with ziftomenib, and we are continuing to treat patients with this agent in combination with chemotherapy in the newly diagnosed setting.”

About 30% of all AML patients have the NPM1 mutation. The disease tends to relapse or resist treatment in about half of those patients within a year of treatment, at which point outcomes are poor. Of 92 patients enrolled in the KOMET-001 study between January 2023 and May 2024, 23% achieved a complete response with ziftomenib, including 67% who had no detectable disease. 

Currently there are no approved targeted treatments for more than half of AML patients, so they are treated with chemotherapy or other cell-killing therapies, which produce subpar clinical outcomes. 

Based on results of the international phase IB/2 clinical trial (NCT04067336) co-led by Dr. Wang and sponsored by Kura Oncology Inc., the U.S. Food and Drug Administration is considering approval of ziftomenib as treatment for patients with relapsed or refractory AML with an NPM1 mutation.

Dr. Wang first presented the findings at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting in June.

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