Agios: Updated Clinical Proof-of-Concept Data from the Phase 2 Study of Mitapivat in Thalassemia at the European Hematology Association Annual Congress

Agios Pharmaceuticals, Inc, announced that the updated results from the Phase 2 thalassemia study will be presented at the European Hematology Association (EHA) Annual Congress being held virtually June 11-14, 2020. Phase 2 trial of Mitapivat (AG-38) involves patients with non-transfusion-dependent thalassemia. Mitapivat is an investigational, first-in-class, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase-R (PKR) enzymes. The Phase 2 study has enrolled 12 (nine with β-thalassaemia and three with α-thalassaemia) of the intended 17 patients. The primary endpoint has been set as a haemoglobin increase of ≥1.0 g/dL from baseline in at least one assessment during weeks 4-12. The β-thalassaemia patients were treated with 50 mg of Mitapivat twice daily for the first six weeks and escalated to 100 mg twice daily, and all patients remain on treatment for 12.4 to 34.3 weeks. Seven of eight efficacy evaluable patients achieved a hemoglobin increase of 1.0 g/dL, and for responders the mean hemoglobin increase from baseline was 1.76 g/dL (range, 0.9–3.3 g/dL) during weeks 4-12. More information can be found here.