Agios Pharmaceuticals Inc (NASDAQ:AGIO), a leader in the fields of cancer metabolism and rare genetic disorders of metabolism, announced that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for AG-120 for treatment of patients with acute myelogenous leukemia (AML). AG-120 is an oral, first-in-class IDH1 mutant inhibitor being evaluated in a Phase 1 clinical trial in patients with advanced hematologic malignancies that carry an IDH1 mutation.

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. Orphan drug designation provides to Agios certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDAapplication fees and tax credits for qualified clinical trials.

“Receiving orphan drug designation for AG-120 is an important milestone as we continue to move this program to late-stage development,” said Chris Bowden, M.D. chief medical officer of Agios. “We are pleased with the progress we are making in the clinic and look forward to presenting new data from our ongoing Phase 1 study of AG-120 at the Congressof the European Hematology Association later this week. We believe that AG-120, which is on track to initiate multiple expansion cohorts in the next month, has the potential to play a significant role in shifting the treatment paradigm for IDH1-mutant positive hematologic cancers from the conventional chemotherapy approach.”

AML, a cancer of blood and bone marrow characterized by rapid disease progression, is the most common acute leukemia in adults. Undifferentiated blast cells proliferate in the bone marrow rather than mature into normal blood cells. AML incidence significantly increases with age, and according to the American Cancer Society the median age is 66. Less than 10 percent of U.S. patients are eligible for bone marrow transplant, and the vast majority of patients do not respond to chemotherapy and progress to relapsed/refractory AML. The five-year survival rate for AML is approximately 20 to 25 percent. IDH1 mutations are present in about 6 to 10 percent of AML cases. (Original Source)

Shares of Agios closed today at $121.60, up $2.19 or 1.83%. AGIO has a 1-year high of $138.85 and a 1-year low of $33.01. The stock’s 50-day moving average is $111.11 and its 200-day moving average is $110.03.

On the ratings front, Agios has been the subject of a number of recent research reports. In a report issued on June 7, Roth Capital analyst Debjit Chattopadhyay reiterated a Hold rating on AGIO, with a price target of $114, which represents a potential downside of 6.3% from where the stock is currently trading. Separately, on May 8, MLV & Co.’s Arlinda Lee maintained a Buy rating on the stock and has a price target of $130.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Debjit Chattopadhyay and Arlinda Lee have a total average return of 32.2% and 5.8% respectively. Chattopadhyay has a success rate of 70.5% and is ranked #15 out of 3619 analysts, while Lee has a success rate of 52.7% and is ranked #1130.

Overall, 3 research analysts have assigned a Hold rating and one research analyst has given a a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $115.67 which is -4.9% under 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.