Agios Pharmaceuticals to Present New Data on Mitapivat at European Hematology Congress

Agios Pharmaceuticals announced that new data on mitapivat, an oral pyruvate kinase activator, will be featured in oral and poster presentations during the 31st European Hematology Association Congress in Stockholm, Sweden, June 11-14, 2026. Select presentations at EHA 2026 will include: An oral presentation during the Plenary Abstracts Session on detailed efficacy and safety data from the global 52-week, randomized, double-blind, placebo-controlled RISE UP Phase 3 trial of mitapivat in patients aged 16 years or older with sickle cell disease. Results demonstrate that treatment with mitapivat significantly improved hemoglobin response and reduced markers of hemolysis. Additional analyses, including measures of clinical benefit and patient-reported outcomes that were not previously disclosed in the company's November 2025 topline report, will also be presented. The RISE UP abstract was one of only six selected for this distinguished plenary session from thousands of submissions. An oral presentation on long-term data from the ENERGIZE Phase 3 trial of mitapivat in adult patients with non-transfusion-dependent alpha- or beta-thalassemia. Of the 192 patients who received at least one dose of mitapivat or placebo in the double-blind period of ENERGIZE, 95% opted to transition into the corresponding open-label extension period, during which all patients receive mitapivat. The results show the durability of mitapivat treatment, with clinically meaningful hemoglobin improvements sustained for up to 127 weeks across the double-blind and OLE periods. During the OLE, nearly half of non-responders who switched from placebo achieved a hemoglobin improvement, supporting a consistent treatment effect with mitapivat. Additionally, about one-third of non-responders who received mitapivat during the double-blind period achieved a hemoglobin improvement during the OLE, suggesting a potential benefit of continued long-term treatment. A related poster will highlight a subset of patients with higher baseline hemoglobin levels in ENERGIZE. Among these patients, 38.9% in the mitapivat arm achieved a hemoglobin response in the double-blind period, compared with 0% in the placebo arm. Additionally, patients in the mitapivat arm showed a 5.1-point improvement in patient-reported fatigue scores compared with 0.8 points among those in the placebo arm, as measured by the least squares mean change from baseline in Functional Assessment of Chronic Illness Therapy-Fatigue from Week 12 to Week 24. An oral presentation on the collaborator-led SATISFY Phase 2 trial of mitapivat in 24 patients with one of three rare hemolytic anemias: hereditary spherocytosis, hereditary xerocytosis, or congenital dyserythropoietic anemia type II. Treatment resulted in sustained hemoglobin improvements over 56 weeks. Notably, patients who achieved a hemoglobin response also showed significant decreases in liver iron content, an important marker of downstream complications in hemolytic anemias.