Single-arm phase 2 study | Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent alpha-thalassaemia or beta-thalassaemia.

Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent α-thalassaemia or β-thalassaemia: an open-label, multicentre, phase 2 study – The Lancet (link to abstract – $ for full-text)

Commentary: PK Modulator Shows Promise in Thalassemia — In small study, mitapivat increased hemoglobin in 80% of non-transfusion-dependent patients – MedPage Today (free registration required)