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

Overview

A recent study led by Roswell Park demonstrates that a three-drug combination therapy involving ziftomenib, venetoclax, and azacitidine shows response rates in patients with relapsed acute myeloid leukemia (AML).

Background

Acute myeloid leukemia (AML) is a challenging hematological malignancy, particularly in its relapsed form. The NPM1-mutated subtype of AML is known for its poor prognosis and limited treatment options once standard therapies fail. The exploration of new combination therapies is critical for improving outcomes in these patients.

Data Highlights

Key Findings

• The study assessed a three-drug combination of ziftomenib, venetoclax, and azacitidine.
• Nearly 90% of previously untreated patients with NPM1-mutated AML responded to the treatment.
• 70% of these patients achieved a complete or near-complete remission.
• Responses were observed quickly, typically within four weeks.
• The treatment was generally well tolerated with low rates of serious side effects.

Clinical Implications

The findings indicate that the combination of ziftomenib with venetoclax and azacitidine may be relevant for patients with relapsed NPM1-mutated AML.

Conclusion

The study presents data on a new combination therapy for relapsed AML.

Related Resources & Content

1. Roswell Park Comprehensive Cancer Center, Roswell Park-Led Study Shows Promise for New Targeted Combination in People With Relapsed AML, 2026 -- Roswell Park-Led Study Shows Promise for New Targeted Combination in People With Relapsed AML
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8. Kura Oncology and Kyowa Kirin Announce Publication in Blood of Ziftomenib plus Venetoclax / Azacitidine Combination in Patients with R/R NPM1-m AML
9. Kura Oncology and Kyowa Kirin to Present Updated Frontline Ziftomenib / 7+3 Combination Data at EHA 2026 Congress - Kyowa Kirin
10. Roswell Park-Led Study Shows Promise for New Targeted Combination in People With Relapsed AML | Roswell Park Comprehensive Cancer Center - Buffalo, NY