Cancer drugmaker Agios offers promising data from two studies testing blood disorder drug

With two cancer drugs on the market, the spotlight is on Agios’ rare metabolic disease franchise led by mitapivat, its experimental drug under development for a range of conditions including pyruvate kinase deficiency, thalassemia, and sickle cell disease.

At the European Hematology Association Annual Congress (EHA), the company issued two updates on the effect of mitapivat — a drug designed to target pyruvate kinase-R (PKR), an enzyme involved in the conversion of sugar, or glucose, into energy that is critical for the survival of red blood cells.