UPDATED: With early data looming, Celgene grabs rights to Agios' leukemia drug

Not long after Agios ($AGIO) was established, Celgene ($CELG) leaned in early to grab options on the preclinical cancer drug programs in the biotech's pipeline, paying $130 million upfront to land the deal and extending their option arrangement last year with another $20 million payment. This morning, with Phase I data on a lead program due for public review this weekend, Celgene pounced early, agreeing to exercise its option on global rights to the leukemia drug and providing a package of milestones worth up to $120 million to obtain the rights on AG-221.

Investigators have been testing the drug for advanced hematologic malignancies, including acute myeloid leukemia. Celgene, which has one of the busiest deal teams working in cancer, will have global commercial rights to the drug, though Cambridge, MA-based Agios has retained "a portion" of U.S. marketing rights in the deal.

Agios is a specialist in cancer metabolism, focusing on targets that play a big role in feeding tumors. And Celgene's option deal was seen as a foundational relationship that tightly bound the biotech upstart to a major player in oncology. Celgene also paid $20 million back in 2011 to keep the relationship open.

"Agios' AG-221 candidate is simultaneously advancing convergent fields, including cancer metabolism, epigenetics and precision medicine. The emerging Phase I clinical data validate the preclinical and mechanistic work on IDH2 mutations in AML, and most importantly, advance a highly promising drug candidate for treatment of molecularly selected patients," said Dr. Thomas Daniel, president of research and early development at Celgene.

AG-221 is an IDH2 mutant inhibitor, one of two enzymes (along with IDH1, which Agios is targeting with AG-120) that play a role in the development of hematologic and solid tumor malignancies. Agios discovered both drugs in its own labs. On Saturday, investigators will reveal Phase I results at the European Hematology Association meeting in Milan, Italy, which were clearly impressive enough to persuade Celgene to make its move.

David Schenkein

One of the reasons Agios was able to pull off a successful IPO last year, despite the absence of any human data in its portfolio, was that the biotech is run by David Schenkein, who was part of Millennium's Velcade crew before he went on to work at Genentech, heading up cancer research at the oncology powerhouse. 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 dove into cancer metabolism. That kind of impressive resume was not ignored.

"They're a great partner and they'll add a lot of value," Schenkein says about Celgene in an interview with FierceBiotech. From here Celgene will pay all development costs, with Agios working alongside. And while the two companies are still hammering out exactly what Agios' role will be in any future commercialization effort in the U.S., the main goal will be to prep for a launch of its second program, where Agios plans to take a big step toward its goal of creating a full-fledged biopharma company with an independent commercial arm.

- here's the release

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