Agios Pharmaceuticals Inc (NASDAQ:AGIO), a leader in the fields of cancer metabolism and rare genetic disorders of metabolism, announced new data from the dose-escalation phase and expansion cohorts from the ongoing Phase 1 study evaluating single agent AG-221, a first-in-class, oral, selective, potent inhibitor of mutant isocitrate dehydrogenase-2 (IDH2), in advanced hematologic malignancies. The data will be presented at the 20th Congress of the European Hematology Association (EHA) taking place June 11-14, 2015 in Vienna.

Data as of May 1, 2015 from 177 patients (104 in dose escalation and 73 from the first four expansion cohorts) with advanced hematologic malignancies treated with single agent AG-221 showed durable clinical activity and a favorable safety profile. More than half of the 177 patients remain on treatment. The study had an overall response rate of 40 percent (63 of 158 response-evaluable patients, using the criteria below) and a complete remission rate of 16 percent (26 of 158 response-evaluable patients). Patients responding to AG-221 continue to show durable clinical activity on treatment for more than 15 months, with an estimated 76 percent of responders staying on treatment for six months or longer. The overall safety profile observed was consistent with previously reported data with more than 100 additional patients treated as of the last analysis.

This new data reflects responses in the evaluable population, which includes all patients with a pre-AG-221 screening assessment and day 28 or later response assessment or an earlier discontinuation for any reason. Patients with a screening assessment who were still on treatment, but had not reached the day 28 disease assessment, were excluded.

“The clinical profile of AG-221 continues to be impressive from the perspectives of response rate, durability, safety and unique mechanism of action,” said Courtney DiNardo, M.D., lead investigator and assistant professor, leukemia atUniversity of Texas MD Anderson Cancer Center. “Additionally, it is encouraging to see early proof-of-concept in myelodysplastic syndrome (MDS) and untreated acute myeloid leukemia (AML) given the need for more effective therapies for these patients.”

“As the data from the AG-221 study continue to mature, we are compiling a robust dataset to quickly move this program into global registration studies later this year in collaboration with Celgene,” said Chris Bowden, M.D., chief medical officer of Agios. “We are excited about the speed of enrollment we’ve seen to date in our four expansion cohorts and are on track to enroll our recently announced fifth expansion cohort of 125 patients with relapsed and/or refractory AML. With this progress, we are executing on our strategy to combine speed and breadth to reach people with hematologic malignancies in urgent need of better treatments.” (Original Source)

Shares of Agios Pharmaceuticals closed yesterday at $122.59 . 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 $112.05 and its 200-day moving average is $110.21.

On the ratings front, Agios Pharmaceuticals 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 implies a downside of 7.0% from current levels. 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 33.9% and 6.2% respectively. Chattopadhyay has a success rate of 73.1% and is ranked #10 out of 3623 analysts, while Lee has a success rate of 55.4% and is ranked #1096.

The street is mostly Neutral on AGIO stock. Out of 4 analysts who cover the stock, 3 suggest a Hold rating and one recommend to Buy the stock. The 12-month average price target assigned to the stock is $115.67, which reflects a potential downside of -5.6% from last closing price.

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.