PKR activator mitapivat treatment of thalassemia: continued improvement in patient hemoglobin levels

Agios Pharmaceuticals recently reported on Phase II studies showing that its experimental oral drug mitapivat (PKR activator) significantly improved hemoglobin (Hb) levels and the associated markers associated with red blood cell generation"This study demonstrates the potential for the use of PKR activators in hemoglobin diseases such as thalassemia and sickle cell disease," said Chris Bowden, Chief Medical Officer of Agios, a Phase II study of patients with non-transfusion-dependent alpha and beta thalassemiaongoing Phase II clinical trials aimed at assessing the effectiveness and safety of mitapivat treatment baseline Hb concentrations of 10 g / dL or lower for non-transfusion-dependent alpha and beta thalassemiaEach of the 20 patients received an initial dose of 50 mg of mitapivat twice a day and then increased the dose level to 100 mg twice a day at 6 weeksAs of March 3, the Hb concentration in 13 patients increased by at least 1.0 g/ dL from the baselinethalassemias is an inherited blood disorder that causes hemoglobin synthesis disorders, and its symptoms can vary according to type, to varying degrees from asymptomatic to severeCommonly thalassemia is accompanied by a typical symptom of anemia, namely low levels of red blood cells