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EHA 2024 | Updates from the ENERGIZE study of mitapivat in non-transfusion-dependent alpha-/beta-thalassemia
Ali Taher, MD, PhD, FRCP, American University of Beirut Medical Center, Beirut, Lebanon, provides an update on the Phase III ENERGIZE trial (NCT04770779) investigating the safety and efficacy of mitapivat in patients with non-transfusion-dependent (NTD) alpha- or beta-thalassemia. The results are positive, with the study meeting its primary and secondary endpoints. This interview took place at the 29th Congress of the European Hematology Association (EHA) in Madrid, Spain.
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Transcript
I’ve just finished presenting it in the plenary session. The ENERGIZE trial is a trial that is a randomized, double-blind, placebo-controlled trial. A global trial from 17 countries, 18 sites and this trial is done for the first time on alpha and beta thalassemia to show the efficacy and safety of mitapivat in this group of non-transfusion-dependent thalassemia patients...
I’ve just finished presenting it in the plenary session. The ENERGIZE trial is a trial that is a randomized, double-blind, placebo-controlled trial. A global trial from 17 countries, 18 sites and this trial is done for the first time on alpha and beta thalassemia to show the efficacy and safety of mitapivat in this group of non-transfusion-dependent thalassemia patients.
The results were very positive. The majority of the patients increased their hemoglobin by more than one gram per deciliter between weeks 12 to 24, which is the primary outcome of the study, and around 42% increased by this one gram per deciliter. The secondary outcome was related to quality of life and the FACIT-Fatigue, which is a patient reported outcome data that would include to look into the fatigue related to chronic illnesses. It was much better in the mitapivat arm.
So this drug, mitapivat, that has showed efficacy and safety, it’s an oral drug, in both alpha and beta thalassemia, non-transfusion-dependent thalassaemia. So this is the first time we will have a modality of treatment for this group of patients.
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Disclosures
Consultancy: Bristol-Myers Squibb (Celgene), Vifor, Pharmacosmos, Agios, Novo Nordisk; Research funding: Bristol-Myers Squibb (Celgene), Pharmacosmos, Agios: Consultancy.
