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ASH 2025 | An update on the ENERGIZE-T trial of mitapivat in transfusion-dependent thalassemia
Maria Domenica Cappellini, MD, University of Milan, Milan, Italy, provides an update on the ENERGIZE-T trial (NCT04770779), a Phase III open-label double-blind placebo-controlled study of mitapivat in patients with transfusion-dependent thalassemia (TDT). Prof. Cappellini notes that mitapivat significantly affects the metabolism of red cells in thalassemia, resulting in a meaningful reduction in transfusion dependence and ultimately in iron accumulation. The agent has also been shown to improve the quality of life (QoL) of patients, with individuals reporting improved well-being. This interview took place at the 67th ASH Annual Meeting and Exposition, held in Orlando, FL.
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Transcript
ENERGIZE-T is a phase 3 randomized double-blind trial in adult patients with thalassemia transfusion dependent. Mitapivat is an oral drug inducing pyruvate kinase that has a significant impact on the metabolism of thalassemia red cells increasing the production of ATP, because, as you know, the thalassemic red cells are under stress and they need more energy. So the expectation from the trial was reduction of transfusion burden more than 50% compared to baseline, and this primary endpoint has been achieved significantly when comparing the result to placebo...
ENERGIZE-T is a phase 3 randomized double-blind trial in adult patients with thalassemia transfusion dependent. Mitapivat is an oral drug inducing pyruvate kinase that has a significant impact on the metabolism of thalassemia red cells increasing the production of ATP, because, as you know, the thalassemic red cells are under stress and they need more energy. So the expectation from the trial was reduction of transfusion burden more than 50% compared to baseline, and this primary endpoint has been achieved significantly when comparing the result to placebo. And it’s quite significant because 50% of transfusion reduction is meaning a lot of units over the year, which impacts also on accumulation of iron. Moreover, we have data related to the study in terms of quality of life improvement and patients reported a better well-being in some different items of the tool we used. But particularly, we also performed the six-minute walk test, which can be evaluated, measured concretely, and there was a significant increase on walk compared to baseline in those patients treated with mitapivat. So the results are very, very encouraging, and we are just waiting for the approval to be able to use mitapivat in a larger cohort of patients than those included in the trial.
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