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ASH 2023 | Preliminary results of tagraxofusp in R/R CD123-positive AML: GIMEMA AML2020

Paola Minetto, MD, PhD, IRCCS Ospedale Policlinico San Martino, Genoa, Italy, presents the preliminary findings of the Phase II GIMEMA AML2020 trial (NCT04342962), which is investigating the use of tagraxofusp in patients with relapsed/refractory (R/R) CD123-positive acute myeloid leukemia (AML). The results are encouraging in this high-risk population, although some patients have discontinued treatment due to toxicity. This interview took place at the 65th ASH Annual Meeting and Exposition, held in San Diego, CA.

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Transcript (edited for clarity)

The GIMEMA AML2020 trial is the first European trial investigating the activity of the immunotoxin tagraxofusp in the context of acute myeloid leukemia. The population of patients treated with tagraxofusp were very poor prognosis patients because we are talking about relapsed and refractory AML patients who did not have any other available options for their treatment...

The GIMEMA AML2020 trial is the first European trial investigating the activity of the immunotoxin tagraxofusp in the context of acute myeloid leukemia. The population of patients treated with tagraxofusp were very poor prognosis patients because we are talking about relapsed and refractory AML patients who did not have any other available options for their treatment. Of course, patients should express CD123 on AML blast to be enrolled. And we enrolled a small cohort of patients, we’re talking about 23 patients. Enrollment is going to be finished in a few months. And we had some encouraging results. We were able to achieve a few complete remission responses in a few patients, which was quite unexpected considering the very high-risk population. Other patients had to interrupt treatment because of non-response or toxicity, but we are collecting interesting information about the mechanism of action of tagraxofusp in this context and the management of toxicity profile. And this could be a very important experience for upcoming trials exploring combinations of standard treatments in association with tagraxofusp in the context of AML treatment.

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