Agios Pharmaceuticals Announces Orphan Drug Designation of AG-348 for Treatment of Pyruvate Kinase Deficiency

Agios Pharmaceuticals, Inc.

, a leader in the fields of cancer metabolism and rare genetic disorders of metabolism, today announced that the United States Food and Drug Administration (FDA) has granted the company orphan drug designation for its investigational medicine AG-348 for the treatment of pyruvate kinase (PK) deficiency, a rare form of hemolytic anemia. AG-348, a first-in-class, orally available activator of pyruvate kinase-R (PKR) enzymes, met its primary endpoints in two Phase 1 healthy volunteer studies – a single ascending dose study and multiple ascending dose study. In addition, data presented in December 2014 at the 54^th Annual Meeting of the American Society of Hematology (ASH) provided early proof-of-mechanism for AG-348. Based on these findings, Agios plans to initiate a Phase 2 clinical trial in patients with PK deficiency in the first half of 2015. "We are pleased to achieve another milestone in the clinical program for AG-348, the first medicine in development designed to treat the underlying cause of PK deficiency," said Chris Bowden, M.D., chief medical officer of Agios. "PK deficiency can result in lifelong medical problems and is an example of our focus on underserved diseases with significant medical needs." The FDA's Office of Orphan Drug Products grants orphan status to support development of medicines for underserved patient populations, or rare disorders that affect fewer than 200,000 people in the U.S. This designation provides certain benefits, including market exclusivity upon regulatory approval, if received, exemption of FDA application fees and tax credits for qualified clinical trials.