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news.Agios Pharmaceuticals has received orphan drug designation from the US Food and Drug Administration (FDA) for its investigational medicine AG-348 to treat pyruvate kinase (PK) deficiency, a rare form of hemolytic anemia.
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AG-348 is a first-in-class, orally available activator of pyruvate kinase-R (PKR) enzymes and the medicine met its primary endpoints in two Phase I healthy volunteer trials including a single ascending dose study and multiple ascending dose study.
The company said that data presented at the 54th Annual Meeting of the American Society of Hematology (ASH) in December 2014 provided early proof-of-mechanism for AG-348.
Based on the data, the company intends to begin a Phase II clinical trial in patients with PK deficiency in the first half of 2015.
Agios chief medical officer Dr Chris Bowden said: "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.
"PK deficiency can result in lifelong medical problems and is an example of our focus on underserved diseases with significant medical needs."
The company intends to report final results from the Phase I multiple ascending dose trial at a medical conference in mid-2015.