Acute myeloid leukemia, a rare and aggressive cancer affecting bone marrow and blood, has seen a breakthrough in treatment options. Professors Jolanta Grembecka and Tomasz Cierpicki of the Medical School have developed a promising drug, providing new hope for patients. KOMZIFTI™ (ziftomenib), a once-daily oral therapy, has recently been approved by the U.S. Food and Drug Administration for relapsed or refractory NPM1-mutant acute myeloid leukemia.
The innovative drug, discovered by Grembecka and Cierpicki, was developed in collaboration with Kura Oncology and is being commercialized with Kyowa Kirin. Grembecka, a Richard and Susan Rogel Professor of Cancer Therapeutics, remarked, “Seeing our pioneering work on menin inhibitors evolve into an FDA-approved treatment for leukemia patients is extremely rewarding. It is remarkable to witness the direct impact of our research, which resulted in a drug that benefits leukemia patients. This is an achievement we could only dream of, and now it’s a reality.”
Research led by Grembecka and Cierpicki, in collaboration with College of Pharmacy researchers, including Duxin Sun, resulted in foundational discoveries licensed to Kura Oncology in 2014. They developed first-in-class small molecule inhibitors targeting menin, a protein involved in leukemia cell growth. These inhibitors disrupt protein-protein interactions, allowing leukemia cells to mature into normal white blood cells. This innovative approach provides new hope for AML patients with NPM1 mutations, who often do not respond to standard therapies.
Cierpicki, also a professor of biophysics, noted, “To develop menin inhibitors, we had to pioneer an entirely new area of research — producing the human protein, creating robust biochemical assays, conducting high-throughput screening and solving the crystal structure of menin.” The skepticism in the industry regarding targeting protein-protein interactions did not deter their efforts.
Clinical trials for ziftomenib in AML patients began in 2019, initiated by Kura Oncology. Recently, Kura partnered with Kyowa Kirin to expand global development and commercialization. Grembecka and Cierpicki’s research journey started at the University of Virginia before they joined U-M in 2009. Their work has been funded by the National Institutes of Health, American Cancer Society, and Blood Cancer United.
Kelly Sexton, associate vice president for research – innovation partnerships and economic impact, emphasized the importance of federal support for science, stating, “This FDA approval highlights U-M’s commitment to translating research discoveries into medical breakthroughs that serve patients and advance the public good.” Ongoing clinical trials are exploring KOMZIFTI in combination with other therapies for leukemia and solid tumors.
The University of Michigan, along with Cierpicki and Grembecka, holds a financial interest in Kura Oncology. The technology was licensed to Kura by Innovation Partnerships.
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