Agios to Present New Mitapivat Data in Rare Blood Disorders at 67th ASH Annual Meeting and Exposition

Core Insights - Agios Pharmaceuticals is set to present new data on mitapivat, an oral pyruvate kinase activator, at the 67th American Society of Hematology Annual Meeting in December 2025, highlighting its potential for treating rare blood disorders [1][2]. Group 1: Presentation Highlights - The presentations will showcase clinical and preclinical data supporting mitapivat's therapeutic potential for thalassemia, sickle cell disease, and pyruvate kinase deficiency, which are rare and life-threatening blood disorders [2]. - A total of 10 presentations and publications will be shared at ASH 2025, including significant findings from the ENERGIZE-T Phase 3 trial and other studies [3][5]. Group 2: ENERGIZE-T Trial Results - In the ENERGIZE-T trial, 77.8% of patients with alpha-thalassemia in the mitapivat arm achieved the primary endpoint of transfusion reduction, compared to 0% in the placebo arm [5]. - Long-term results showed that patients who achieved transfusion independence had a mean duration of 30.5 weeks without transfusions, with a maximum of 84.3 weeks [5]. Group 3: Pediatric and Preclinical Findings - The ACTIVATE-Kids Phase 3 trial demonstrated a higher hemoglobin response rate in children with pyruvate kinase deficiency treated with mitapivat compared to the placebo group [5]. - Preclinical data indicated that mitapivat protects against cardiomyopathy in a mouse model of beta-thalassemia, potentially linked to its activation of the PKM2 isoform [5]. Group 4: Advisory Council Research - Research from Agios-supported advisory councils revealed key knowledge gaps about thalassemia and the impact of fatigue on patients with rare blood disorders [5]. - A global patient survey identified the importance of regular monitoring in non-transfusion-dependent patients and highlighted cognitive impairment as a significant issue related to fatigue [5].