Agios Pharmaceuticals, Inc, presented the updated results from the Phase 2 thalassemia in an oral presentation at the European Hematology Association (EHA) Annual Congress. The ongoing, open-label Phase 2 study is evaluating the efficacy, safety, pharmacokinetics and pharmacodynamics of mitapivat treatment in adults with non-transfusion-dependent α- and β-thalassemia who have a baseline hemoglobin (Hb) concentration of ≤10 g/dL. The trial is fully enrolled with 20 patients and includes a 24-week core period followed by a 2-year extension period for eligible participants. All patients were treated with an initial dose of mitapivat 50 mg twice daily followed by a dose-level increase to 100 mg twice daily at the week 6 visit based on safety evaluations and Hb concentrations. Treatment with Mitapivat induced Hb increase of ≥1.0 g/dL from baseline in 12 of 13 (92%) evaluable patients, including 4 of 4 (100%) α-thalassemia patients and 8 of 9 (88.9%) patients with β-thalassemia, during weeks 4-12. Sever of 8 (88%) β-thalassemia patients achieved sustained primary and Hb response in ≥2 assessments during weeks 12-24. Furthermore, preliminary results for markers of hemolysis and erythropoiesis demonstrated improvements that were consistent with the Hb increase. “Our focus now is to advance the development of mitapivat for these patients as quickly and efficiently as possible. By the end of the year, we expect to finalize a robust pivotal development plan that spans both α-and β-thalassemia, as well as transfusion dependent and non-transfusion dependent patients, with a goal of initiating a pivotal program in 2021” said Chris Bowden, chief medical officer at Agios. More information can be found here.