Agios Announces Phase 3 ENERGIZE Study of Mitapivat Met Primary Endpoint and Both Key Secondary Endpoints in Adults with Non-Transfusion-Dependent Alpha- or Beta-Thalassemia

–Mitapivat Demonstrated a Statistically Significant Increase in Hemoglobin Response Rate Compared to Placebo –

– Statistical Significance Also Achieved for Key Secondary Endpoints of Change From Baseline in Both FACIT-Fatigue Score and Hemoglobin Concentration –

– ENERGIZE is the First Phase 3 Study to Demonstrate Efficacy of an Oral Treatment for
Non-Transfusion-Dependent Alpha- and Beta-Thalassemia –

– Agios to Host Investor Webcast Event Today at 8:00 a.m. ET–

CAMBRIDGE, Mass., Jan. 03, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism and pioneering therapies for rare diseases, today announced that the global Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent (NTD) alpha- or beta-thalassemia achieved its primary endpoint of hemoglobin response. Statistical significance was also achieved for both key secondary endpoints associated with change from baseline in FACIT-Fatigue Score and hemoglobin concentration.

“The results of the Phase 3 ENERGIZE study underscore the potential of mitapivat to be a meaningful treatment option for adults with non-transfusion dependent alpha- or beta-thalassemia. All subgroup analyses favored the mitapivat treatment arm compared to placebo,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios. “We are grateful to all of the patients who participated in this trial, our collaborators, study investigators and advisors in the patient and clinical communities for their partnership in achieving this milestone. These data bring us one step closer to a treatment for all thalassemia patients, and we look forward to the ENERGIZE-T readout mid-year.”

“The results of the ENERGIZE study support the potential of mitapivat to be the first oral therapy for all NTD thalassemia patients, including those with alpha- or beta-thalassemia,” said Ali Taher, M.D., Ph.D., Professor of Medicine, Hematology & Oncology and Director – Naef K. Basile Cancer Institute, American University of Beirut Medical Center in Beirut, Lebanon. “For NTD thalassemia patients across the globe, there are currently no approved oral treatments, and NTD thalassemia has consistently been associated with morbidity and mortality if left untreated. NTD thalassemia represents over half of clinically significant forms of thalassemia, so there is a tremendous unmet need. Based on the data reported to date, mitapivat has the potential to be a foundational treatment option for the thalassemia community.”