Stock Update (NASDAQ:AGIO): Agios Pharmaceuticals Inc Announces Orphan Drug Designation of AG-348 for Treatment of Pyruvate Kinase Deficiency

Agios Pharmaceuticals Inc (Nasdaq:AGIO), 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 54th 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. (Original Source)

Shares of Agios Pharmaceuticals opened today at $102.65 and are currently trading up at $102.94. AGIO has a 1-year high of $138.85 and a 1-year low of $31.42. The stock's 50-day moving average is $108.43 and it's 200-day moving average is $93.93.

On the ratings front, Agios Pharmaceuticals (NASDAQ: AGIO) has been the subject of a number of recent research reports. In a report issued on February 18, MLV & Co. analyst Arlinda Lee maintained a Buy rating on AGIO, with a price target of $130, which implies an upside of 26.6% from current levels. Separately, on the same day, Citigroup's Yaron Werber maintained a Hold rating on the stock and has a price target of $132.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Arlinda Lee and Yaron Werber have a total average return of 9.0% and 28.0% respectively. Lee has a success rate of 60.0% and is ranked #945 out of 3546 analysts, while Werber has a success rate of 80.7% and is ranked #88.

In total, 2 research analysts have assigned a Hold rating and 2 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $102.65 which is 31.3% above where the stock opened today.

Agios Pharmaceuticals Inc is engaged in the development of medicines to treat cancer metabolism and inborn errors of metabolism, which are a subset of orphan genetic metabolic diseases.

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