KOMZIFTI™ (ziftomenib), the first and only once-daily oral therapy for patients with relapsed or refractory NPM1-mutant acute myeloid leukemia, recently received approval by the U.S. Food and Drug Administration. The drug was discovered by Grembecka and Cierpicki in collaboration with Kura Oncology and is being developed and commercialized by Kura Oncology and Kyowa Kirin.
“Acute myeloid leukemia is a very aggressive blood cancer with poor clinical outcomes,” said Grembecka, also the Richard and Susan Rogel Professor of Cancer Therapeutics. “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, the Charles R. Walgreen Jr Professor of Pharmacy, professor of pharmaceutical sciences and director of the Pharmacokinetic and Mass Spectrometry Core in the College of Pharmacy, resulted in foundational discoveries that were licensed to Kura Oncology in 2014 with the help of Innovation Partnerships.
The research team identified first-in-class small molecule inhibitors blocking the protein, menin. These inhibitors were designed to disrupt the protein-protein interactions that drive leukemia cell growth and survival, allowing the cells to mature into white blood cells instead of cancerous ones. This is a transformative approach for AML patients with NPM1 mutations, who often do not respond to standard therapies.
For more details see: https://record.umich.edu/articles/leukemia-treatment-based-on-u-m-research-gets-fda-approval/