STAT+: Agios drug for rare form of anemia achieves main goal of late-stage clinical trial
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.
Continue to STAT+ to read the full story…
