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...