Agios Pharmaceuticals presents updated PYRUKYND data

Agios Pharmaceuticals reported new data from the ongoing long-term extension period of the Phase 2 open-label study of PYRUKYND, a first-in-class, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase enzymes, in adults with non-transfusion dependent alpha- or beta-thalassemia. Data from the study were featured in a poster presentation at the 64th American Society of Hematology Annual Meeting and Exposition, hosted Dec. 10-13, 2022, in New Orleans. Consistent with previously reported data, durable improvements in hemoglobin concentration and markers of hemolysis and ineffective erythropoiesis were observed for up to 72 weeks of treatment in both alpha- and beta-thalassemia patients. Additionally, markers of iron homeostasis remained stable or improved through Week 72. PYRUKYND was well tolerated, and the safety profile was consistent with previous studies. Agios also presented data at ASH further elucidating the burden of disease and unmet needs in alpha- and beta-thalassemia.