Agios Pharma (AGIO) Confirms Proof-of-Concept for Mitapivat (AG-348) Based on Preliminary Phase 2 Results in Patients with Non-Transfusion-Dependent Thalassemia

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Go back to Agios Pharma (AGIO) Confirms Proof-of-Concept for Mitapivat (AG-348) Based on Preliminary Phase 2 Results in Patients with Non-Transfusion-Dependent Thalassemia

Agios Pharma (AGIO) Reports Updated Data for Mitapivat from Extension Phase of DRIVE PK Study in Patients with Pyruvate Kinase Deficiency

December 9, 2019 10:13 AM EST

Agios Pharmaceuticals, Inc. (NASDAQ: AGIO) today reported new data from the extension phase of the DRIVE PK Phase 2 study of mitapivat (AG-348) in adults with pyruvate kinase (PK) deficiency at the 2019 American Society of Hematology (ASH) Annual Meeting. Mitapivat is an investigational, first-in-class, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase-R (PKR) enzymes that directly targets the underlying metabolic defect in PK deficiency, a rare, potentially debilitating, hemolytic anemia. In addition, data was shared from the Agios-sponsored Natural History Study of PK deficiency that detailed the comorbidities and... More