From GlobeNewswire:

<2024 marked a major breakthrough in thalassemia treatment with Agios Pharmaceuticals' successful Phase 3 study of mitapivat. The study showed a significant increase in hemoglobin response rate compared to the placebo, as well as key improvements in fatigue and hemoglobin concentration. The results indicate a promising oral treatment for non-transfusion-dependent alpha- and beta-thalassemia. Dr. Sarah Gheuens, Agios' chief medical officer and head of R&D, expressed gratitude to the patients, collaborators, and investigators involved in this milestone. The study's data bring hope for a comprehensive thalassemia treatment, highlighting the potential of mitapivat to meet the unmet needs of patients with non-transfusion-dependent thalassemia globally. Additionally, the study demonstrated potential for mitapivat to be the first oral therapy for all non-transfusion-dependent thalassemia patients, as confirmed by Dr. Ali Taher, Professor of Medicine at the American University of Beirut Medical Center. The study's promising results suggest a potential oral treatment option for over half of all thalassemia cases, addressing a significant unmet medical need. Following the success of the ENERGIZE study, Agios is moving forward with the fully enrolled Phase 3 ENERGIZE-T study in adults with transfusion-dependent alpha- or beta-thalassemia. The company intends to file for regulatory approval of mitapivat as a thalassemia treatment by the end of 2024, incorporating data from both studies. The Phase 3 ENERGIZE study enrolled 194 patients, with 130 on mitapivat and 64 on placebo, and demonstrated a significant increase in hemoglobin response compared to the placebo. 42.3% of patients on mitapivat achieved a hemoglobin response, demonstrating the potential of this oral treatment for thalassemia patients.


Read more: Agios Announces Phase 3 ENERGIZE Study of Mitapivat Met