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ASCAT 2024 | Update on the ENERGIZE trial of mitapivat in NTD alpha- and beta-thalassemia
Ali Taher, MD, PhD, FRCP, American University of Beirut Medical Center, Beirut, Lebanon, presents updated results from 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 agent has shown encouraging safety and efficacy, with the study reaching its primary endpoint. This interview took place at the 19th Annual Scientific Conference of the Academy for Sickle Cell and Thalassaemia (ASCAT 2024) in London, UK.
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Transcript
For the mitapivat, I presented the ENERGIZE. And the ENERGIZE is a randomized double-blind placebo-controlled trial for alpha and beta non-transfusion-dependent thalassemia. The primary outcome in this group of patients was to increase hemoglobin by more than 1 gram per deciliter from week 12 to 24, and the study reached its primary outcome. Also, I presented a key secondary outcome, which is the FACIT, which is a marker or a score that we measure the fatigue in chronic illnesses...
For the mitapivat, I presented the ENERGIZE. And the ENERGIZE is a randomized double-blind placebo-controlled trial for alpha and beta non-transfusion-dependent thalassemia. The primary outcome in this group of patients was to increase hemoglobin by more than 1 gram per deciliter from week 12 to 24, and the study reached its primary outcome. Also, I presented a key secondary outcome, which is the FACIT, which is a marker or a score that we measure the fatigue in chronic illnesses. And it’s usually the higher the score, the less fatigue. And I also presented that in mitapivat group of patients, those that were on mitapivat had a higher score or less fatigue than those that were not receiving the treatment. And I presented the safety and it was as safe as the placebo group. So I ended this presentation by mentioning that mitapivat is a drug that is safe and efficacious in adults with alpha and beta non-transfusion dependent thalassemia.
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Disclosures
Consultancy: Novo Nordisk, Bristol-Myers Squibb (Celgene), Vifor, Pharmacosmos, Agios; Research funding: Bristol-Myers Squibb (Celgene), Vifor, Pharmacosmos, Agios.
