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Agios Establishes Proof-of-Concept for Mitapivat in Non-transfusion-dependent Thalassemia Based on Preliminary Phase 2 Results

a?? Treatment with Mitapivat Induced Hemoglobin Increase of a??1.0 g/dL in 7 of 8 Evaluable Patients a??
a?? Safety Profile Consistent with Previously Published Phase 2 Data for Mitapivat in Patients with Pyruvate Kinase Deficiency a??a?? Additional Data for the Phase 2 Study of Mitapivat in Thalassemia to be Presented at a Medical Meeting in the First Half of 2020 a??a?? Company to Host ASH Investor Event and Webcast Monday, December 9 atA 8:00 p.m. ETA a??CAMBRIDGE, Mass., Dec. 08, 2019 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced that clinical proof-of-concept has been established based on a preliminary analysis of the Phase 2 trial of mitapivat (AG-348) in patients with non-transfusion-dependent thalassemia. 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.
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