Roswell Park-Led Study Shows Promise for New Targeted Combination in People With Relapsed AML

New research co-led by a Roswell Park Comprehensive Cancer Center leukemia expert highlights a promising investigational treatment approach using the targeted therapy ziftomenib for patients with a difficult-to-treat form of acute myeloid leukemia (AML). The findings, published in the journal Blood, show encouraging results from a three-drug combination therapy in patients whose leukemia has returned or not responded to prior treatment.

The study focuses on patients with NPM1-mutated AML, a subtype that can be especially challenging to treat once standard therapies stop working. Roswell Park was part of an international clinical trial evaluating the addition of ziftomenib (brand name Komzifti), an oral targeted therapy, to two commonly used treatments, venetoclax and azacitidine.

The team’s findings show strong responses to the combination: Among patients who had not previously received venetoclax, nearly 90% responded to treatment and 70% achieved a complete or near-complete remission. Many of these responses were deep, meaning no measurable cancer cells were detected with highly sensitive testing. In patients who had received venetoclax before, about half responded.

Importantly, responses occurred quickly — within about four weeks, on average — and in many cases lasted for several months. The treatment was generally well tolerated, with low rates of serious side effects.

“These results are highly encouraging, particularly in the context of an aggressive leukemia for which limited treatment options exist,” says Eunice Wang, MD, Chief of Leukemia at Roswell Park Comprehensive Cancer Center and first author of the study. “We observed deep and durable responses with this three-drug combination, along with a safety profile that supports continued study.”

The findings come from the ongoing phase 1a/b KOMET-007 clinical trial (NCT05735184), sponsored by Kura Oncology. While ziftomenib is currently approved by the U.S. Food and Drug Administration as a single-agent treatment for certain patients with relapsed or refractory AML, its use in combination with other therapies remains investigational. Dr. Wang and Roswell Park have also contributed significantly on an additional clinical trial examining the efficacy of ziftomenib in combination with other therapies in patients with newly diagnosed AML, to be presented later this month at the 2026 European Hematology Association Congress. 

Roswell Park leukemia patients were among the first in the world to be treated with ziftomenib through the cancer center’s Early Phase Leukemia Clinical Trials program, made possible by support from the Roswell Park Alliance Foundation. For a list of currently enrolling clinical trials available at Roswell Park, see roswellpark.org/clinical-trials.