The Clinical Leukemia Service at Roswell Park Comprehensive Cancer Center helped lead the first clinical trial of the experimental oral drug ziftomenib in patients with acute myeloid leukemia (AML).
The results of the KOMET-001 study, published in The Lancet Oncology, show that the drug — part of a class of targeted therapies known as menin inhibitors — produced a partial or complete response in about a third of patients, all of whom had received two or more prior therapies.
All 83 patients enrolled in the study experienced disease progression after receiving between 2 and 9 prior therapies. The most pronounced results were seen in patients with NPM1 mutations and KMT2A rearrangements — profiles associated with poorer clinical outcomes.
The clinical trial was conducted by investigators at 22 hospitals in France, Italy, Spain and the U.S. from 2019-2022.
In the clinical trial, when patients with an NPM1 mutation were treated with the recommended 600 mg. daily dose of ziftomenib, 35% (7 of 20) achieved complete remission. Based on study results, earlier this year the FDA granted Breakthrough Therapy designation to the drug, a recognition intended to speed its development and review for FDA approval.
Roswell Park Comprehensive Cancer Center release on Newswise
