Agios shares spike on enthusiastic response to small, early-stage leukemia study

Agios Pharmaceuticals was the biotech canary in the coal mine when it went public last year. Not only did it survive its IPO, it thrived, helping lure dozens of other biotechs into the market with solid proof that investors would buy into a drug developer with no human data in the portfolio. On Sunday, Agios added some notable evidence from a very small, early-stage study that its lead drug worked. And its stock ($AGIO) shot up again, spiking more than 25% on evidence that the early promise could pay off.

AG-221, an inhibitor of the mutated IDH2 protein, is in a dose-escalating Phase I trial right now, recruiting older patients with acute myeloid leukemia or myelodysplastic syndrome which contain the mutation. Of the 10 patients initially treated with AG-221, three died of their disease and 7 were evaluable. Of those 7, three achieved complete remission, two patients achieved complete remission with incomplete platelet recovery and one patient demonstrated a partial response.

For early-stage data, it was a striking example of a biotech taking a successful proof-of-principle a step forward, an important advance for the Cambridge, MA-based biotech. The carefully coordinated campaign around these early results also achieved considerable traction among the media outlets covering news at the American Association for Cancer Research meeting in San Diego, perhaps in part because there wasn't much competition in this particular conference.

Agios CEO David Schenkein

Agios, then, chose well and was rewarded.

"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," noted Agios CEO David Schenkein. "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."

Schenkein was part of Millennium's Velcade crew before he went on to work at Genentech. Then he rejoined some of his colleagues at Millennium who went on to set up Third Rock, which funded the company--with Arch and Flagship--and dived into cancer metabolism. A big partnership with Celgene ($CELG) which included a $130 million upfront just seemed natural. 

- here's the release