Agios Experimental Leukemia Drug Sees Success

Biotech firm Agios held a press conference detailing the success of their experimental leukemia drug, AG-221. The drug, which is taken in pill form, was able to render cancer undetectable in three patients in a company study.

Researchers leading the trial studied how Ag-221 affected patients with acute myeloid leukemia (AML) whose tumors have a specific gene mutation — isocitrate dehydrogenase-2 (IDH2). The results of the company's study were released at the American Association for Cancer Research's yearly meeting and were presented by Dr. Eytan Stein. Of the ten AML patients who were evaluated, three could not continue treatment because of the disease's progression. Six of the seven patients left in the study saw their conditions improve significantly, with three patients showing no signs of cancer cells left in the blood. Two other patients were close to remission, though they still showed depleted platelet levels.

Agios' CEO, David Schenkein, called the results 'very encouraging.' “The observation of complete remissions, reduction of 2HG, preliminary favorable safety profile and effects on cellular differentiation provide proof-of-principle for AG-221’s novel mechanism of action. These findings corroborate the use of precision medicine in genetically defined patient populations and demonstrate the potential of targeting cancer metabolism to develop transformative medicines for patients,” he said.

Larger studies will need to be done in order to conclude if the Agios drug is fit for public use. Industry experts believe that it is still years from reaching any markets. Still, researchers are optimistic about its future. “I would say this data is kind of unheard of,” said Dr. Stein. While patients who experience complete remission usually have to go on for bone marrow transplants, Stein says, “For this agent I think all bets are off about whether [a bone marrow transplant] would be necessary or not.”