Agios Pharmaceuticals said Tuesday that its experimental drug mitapivat increased hemoglobin levels in patients with a rare form of anemia — achieving the primary goal of a Phase 3 clinical trial.
Mitapivat is the most important and closely followed medicine in Agios’ research pipeline because of its potential to treat a range of rare blood diseases defined by the destruction or malfunction of oxygen-carrying red blood cells. The positive study results announced Tuesday involved adults with pyruvate kinase deficiency, or PK deficiency. Agios is also developing mitapivat as a potential treatment for patients with sickle cell disease and two types of thalassemia.
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