Ziftomenib (Komzifti, KO-539) is a selective and potent small molecule inhibitor of menin first approved by the U.S. Food and Drug Administration on November 13, 2025.
Ziftomenib is the first and only once-daily targeted therapy approved for adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML). Importantly, the approval was granted with no Boxed Warning indicating a potential best-in-class safety profile.
Starting in 2010, the Therapy Acceleration Program® (TAP) committed over $6 million to support a promising University of Michigan research project led by Jolanta Grembecka, PhD, to develop new treatments for patients with a rare and lethal subtype of leukemia. Through TAP, chemists were engaged to improve the properties that produced lead compounds that exhibited potent anti-leukemic activity. In 2014, Blood Cancer United introduced Kura Oncology to the project that ultimately led to Kura Oncology completing a licensing agreement with the University of Michigan to continue to develop these molecules. The KOMET-001 clinical study that led to approval by the FDA was supported in part by TAP.