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).

By
August 26, 2026
3 min

Roswell Park Comprehensive Cancer Center

Objective:

To evaluate the efficacy of a three-drug combination therapy including ziftomenib for patients with relapsed acute myeloid leukemia (AML).

Approach:

Study Design: International clinical trial assessing the combination of ziftomenib, venetoclax, and azacitidine in patients with NPM1-mutated AML.

Key Findings:

Nearly 90% of previously untreated patients who had not received venetoclax responded to the treatment.
70% of these patients achieved a complete or near-complete remission.
Responses occurred quickly, within about four weeks on average.
Treatment was generally well tolerated with low rates of serious side effects.

Limitations:

The use of ziftomenib in combination with other therapies remains investigational.
The study primarily focused on patients with NPM1-mutated AML.

Sources:

Blood Journal

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

Objective:

Approach:

Key Findings:

Limitations:

Sources:

Original Source(s)

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

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