What question did this study set out to answer?

To assess the clinical activity and tolerability of ziftomenib in combination with venetoclax and azacitidine in adults with relapsed/refractory NPM1-mutated acute myeloid leukemia.

June 4, 2026Blood

Ziftomenib with venetoclax and azacitidine in relapsed/refractory NPM1-mutated acute myeloid leukemia

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Authors

EWEunice S. WangRoswell Park Comprehensive Cancer Center HEHarry P. ErbaDuke University AZAmer M. ZeidanYale University

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Implication

Randomized trial investigates ziftomenib with venetoclax/azacitidine improving outcomes in R/R NPM1-m AML, suggesting strong potential benefits.

Key Points

To assess the clinical activity and tolerability of ziftomenib in combination with venetoclax and azacitidine in adults with relapsed/refractory NPM1-mutated acute myeloid leukemia.
Phase 1a and 1b trial design with adult patients having R/R NPM1-m AML.
Patients received ziftomenib at varying doses combined with standard doses of venetoclax and azacitidine.
Sixty-seven patients treated; median age 66, with different prior therapy histories.
In patients treated with ziftomenib 600 mg, composite complete remission rate was 46% (22/48).
CRc rates in venetoclax-naïve patients were 70% (16/23) and in -exposed patients 24% (6/25).
Median duration of response was 8.6 months; median overall survival was not reached.

Cite This Study

Wang et al. (2026) studied this question.

synapsesocial.com/papers/6a21164cd499ed480b16f43f https://doi.org/10.1182/blood.2026034043

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Implication

Randomized trial investigates ziftomenib with venetoclax/azacitidine improving outcomes in R/R NPM1-m AML, suggesting strong potential benefits.

Ziftomenib with venetoclax and azacitidine in relapsed/refractory NPM1-mutated acute myeloid leukemia

Discussion

Member takes

Implication

Key Points

Cite This Study

Ziftomenib with venetoclax and azacitidine in relapsed/refractory NPM1-mutated acute myeloid leukemia

Discussion

Member takes

Implication

Key Points

Cite This Study

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