Agios Pharmaceuticals (AGIO) Initiates Multiple Ascending Dose Trial In Healthy Volunteers Of AG-348 For The Potential Treatment Of PK Deficiency, A Rare, Hemolytic Anemia
6/9/2014 8:32:00 AM
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the fields of cancer metabolism and inborn errors of metabolism (IEMs), today announced the initiation of a multiple ascending dose (MAD) Phase 1 trial of AG-348 in healthy volunteers. This study is designed to characterize the safety, tolerability, pharmacokinetics and pharmacodynamics of increasing doses of AG-348 for 14 days. AG-348 is a novel, first-in-class, orally available activator of pyruvate kinase-R (PKR) for the treatment of pyruvate kinase (PK) deficiency, a rare, hemolytic anemia. This trial was initiated based on data from the ongoing single ascending dose (SAD) Phase 1 trial, in which AG-348 has been determined to be well tolerated to date. The SAD study began in April 2014 and has completed dosing of more than half of the planned cohorts. Data from both studies are expected to be presented at a medical conference in 2015.
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