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ASCO 2023 | Phase I study of ziftomenib with venetoclax or venetoclax/azacitidine or 7+3 in R/R AML

Amer Zeidan, MBBS, Yale University, New Haven, CT, comments on the efficacy of ziftomenib as a monotherapy in relapsed/refractory (R/R) acute myeloid leukemia (AML) with NPM1 mutations or MLL rearrangement, discussing an ongoing Phase I study (NCT04067336) evaluating ziftomenib in combination with venetoclax or venetoclax/azacitidine or standard induction chemotherapy in R/R AML.

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Transcript (edited for clarity)

So, one of the important trials that are being planned is the use of a menin inhibitor called ziftomenib. So this drug actually had very nice data in the refractory lab setting for patients with acute myeloid leukemia who had NPM1 or MLL rearrangement, and the data suggested high response rate, but most importantly as a monotherapy. So I think the next step was to combine this drug with our standard treatments, such as azacitidine and venetoclax for older unfit patients or with intensive chemotherapy...

So, one of the important trials that are being planned is the use of a menin inhibitor called ziftomenib. So this drug actually had very nice data in the refractory lab setting for patients with acute myeloid leukemia who had NPM1 or MLL rearrangement, and the data suggested high response rate, but most importantly as a monotherapy. So I think the next step was to combine this drug with our standard treatments, such as azacitidine and venetoclax for older unfit patients or with intensive chemotherapy. So, we actually worked with a company to design a trial that addresses this aspect where patients with refractory leukemia will receive ziftomenib with azacitidine and venetoclax if they have NPM1 or MLL rearrangement. But also importantly, we are going to test this combination in the frontline setting with 7+3 or with azacitidine and venetoclax. So I think this is a next step for menin inhibitors in general to be tested in the frontline setting because hopefully we are going to see augmentation of the responses and improved outcomes.

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