Agios’ Phase III Data Move It Closer To First Therapy For Rare Anemia

With two approved leukemia drugs that target mutated isocitrate dehydrogenase (IDH), Agios Pharmaceuticals, Inc. is moving closer to adding a major revenue driver with its first-in-class allosteric activator of mutated pyruvate kinase receptor enzymes. Top-line Phase III data for mitapivat reported on 1 December support submission for US and EU approval to treat a rare form of anemia, and the drug may be applicable in larger anemia indications.

Data from the 80-patient, pivotal Phase III ACTIVATE study of mitapivat in pyruvate kinase deficiency patients who are not regularly treated with blood transfusions showed statistical significance for the primary...