Agios readies FDA filing as anemia drug clears another phase 3

A second phase 3 trial of Agios Pharmaceuticals’ mitapivat has hit its primary endpoint in patients with pyruvate kinase deficiency, keeping the biotech on track to file for approval on both sides of the Atlantic in the coming months.

The latest clinical readout comes from a single-arm study that gave the oral, small-molecule allosteric activator of pyruvate kinase-R enzymes to 27 regularly transfused patients with PK deficiency. Agios compared the performance of the patients during the 24-week fixed dose period—which followed 16 weeks of dose optimization—to historical data to show the effect of mitapivat on transfusion burden.