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Agios Pharmaceuticals (AGIO) Initiates Phase 1 Study Of AG-348, A First-in-class PKR Activator, For Pyruvate Kinase Deficiency


4/17/2014 9:30:24 AM

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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the fields of cancer metabolism and inborn errors of metabolism (IEM), today announced dose administration of AG-348 in a Phase 1 study in healthy volunteers. AG-348 is an orally available, potent, selective small molecule activator of pyruvate kinase-R (PK-R), a metabolic enzyme, which, when mutated, leads to pyruvate kinase (PK) deficiency, a rare, inherited hemolytic anemia. There is substantial unmet need among patients with PK deficiency as there is no therapy available to treat the underlying disease. AG-348 is wholly owned by Agios, with the company maintaining full worldwide development and commercialization rights.

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