Agios presents positive results of PK treatment "mitapivat" in both α- and β-thalassaemia

Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), presented positive results from its Phase 2 proof-of-concept study of the oral pyruvate kinase (PK) activator "mitapivat" for non-transfusion-dependent thalassaemia (NTDT). This is the first clinical study of a PK activator in thalassaemia and the first drug trial in α-thalassaemia. The Phase 2 trial of mitapivat evaluated its efficacy, safety, pharmacokinetics and pharmacodynamics in 20 adult patients with either α- or β-NTDT and ≤10 g/dL baseline haemoglobin (Hb) levels. A sustained increase in Hb levels ≥1.0 g/dL from baseline was reported in 80% of the treated patients, together with improvements in hemolysis and ineffective erythropoiesis, and mild adverse drug reactions similar to previous studies in healthy volunteers and patients with PK deficiency. Agios plans to initiate two Phase 3 studies of mitapivat, ENERGIZE and ENERGIZE-T, in not regularly transfused and regularly transfused adults with thalassaemia. More info: Agios press release