Roswell Park-Led Clinical Trial Paves Way for FDA Approval of Targeted Drug for Aggressive Leukemia

U.S. FDA approves new drug application for ziftomenib following international study co-led by Dr. Eunice Wang

BUFFALO, N.Y. — The U.S. Food and Drug Administration has approved a new drug to treat an aggressive form of leukemia, based on the results of a clinical trial co-led by Eunice Wang, MD, Chief of Leukemia at Roswell Park Comprehensive Cancer Center.

The FDA recently approved use of the oral therapy ziftomenib as treatment for adults with acute myeloid leukemia (AML) carrying an NPM1 mutation. The approval is based on findings from the pivotal phase 1/2 KOMET-001 trial, for which Dr. Wang served as global Principal Investigator.

“I am proud to say that Roswell Park patients were the first in the world to be treated with ziftomenib, and we are continuing to advance this development of this drug for AML patients with clinical trials investigating this agent in combination with chemotherapy in the newly diagnosed and relapsed/ refractory setting,” says Dr. Wang. Clinical trial findings were published in print in November in the prestigious Journal of Clinical Oncology with Dr. Wang as first author.

Ziftomenib works by blocking menin, a protein that fuels the growth of leukemia cells. Dr. Wang and fellow physicians from the Roswell Park leukemia service led the phase 1/2 study, and remain significantly involved in ongoing clinical trials of this targeted treatment in combination with chemotherapies. Up to one-third of patients newly diagnosed with AML have this genetic subtype and could potentially be candidates for this treatment.

In that study of 92 adults with relapsed or refractory acute myeloid leukemia (AML) carrying an NPM1 mutation, all of whom had received multiple lines of prior therapy, 23% achieved a complete response, demonstrating no evidence of disease after treatment on the study. Dr. Wang and co-authors reported in that publication: “Ziftomenib demonstrated significant clinical benefit and deep responses in patients with heavily pretreated, relapsed/ refractory AML,” and Ziftomenib was well tolerated with a safety profile consistent with previous studies.

Clinical trials provide access to the very latest therapies in cancer care. Information about clinical trials currently available at Roswell Park is available at roswellpark.org/clinical trials.