Wow. Agios’ Experimental Leukemia Drug Makes Cancer Undetectable In Three Patients

In In The News by Barbara Jacoby

Thumbnail for 5488By: Matthew Harper


Agios, a biotechnology company based in Cambridge, Mass., has seemed in some ways a sign of the overexcitement of the recent biotech boom. After all, the company, despite an experienced management team, a July public stock offering that raised more than $100 million, and great scientific ideas, hasn’t been able to show a single result from testing its drugs in humans.

That changed this morning at a press conference for the annual meeting of the American Association for Cancer Research. Eytan Stein, a doctor at Memorial Sloan-Kettering Cancer Center in New York, presented the results of the first study of Agios’ experimental pill AG-221 in patients with acute myeloid leukemia whose tumors have a mutation in a specific gene.

Ten AML patients were available for evaluation. Three of those had to stop taking the drug because they became sick due to their disease. Of the seven who were left, six saw their disease measurably improve. Three of them had all signs of cancer cells in their blood disappear — called a complete remission. Two more were close, but still had depleted platelet levels, which Stein says usually come up over time if they are already rising.

“This is I would say this data is kind of unheard of,” Stein said at the press conference. Usually, these patients who get a complete remission will go on to have a bone marrow transplant, he said, which is considered the treatment most likely to lead to a cure. But he volunteered that might not be so for patients on AG-221. “For this agent I think all bets are off about whether [a bone marrow transplant] would be necessary or not,” he said.

It’s too soon to tell exactly how effective AG-221 is, and bigger studies will have to be done. A larger version of the current study might get the medicine approved in AML patients who have failed every other option, but moving earlier in the disease will require studies in which AG-221 is compared to a placebo. This drug is years from the market.

But the results are obviously very encouraging. For Agios chief executive David Schenkein, these results are a big step toward proving the scientific ideas behind his company — targeted medicines based on the metabolic processes of cells — can result in drugs that are invented and brought to market significantly more quickly than is the norm in pharma. A more complete version of the Agios data will be presented later in the day at the AACR meeting.