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Agios and Celgene: Anatomy of an Ultra-Valuable Biotech Marriage

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Agios put together a scientific advisory board of more than a dozen experts in cancer metabolism, including David Sabatini of the Whitehead Institute and MIT, Charles Sawyers of Memorial Sloan, and Homer Pearce, an Eli Lilly retiree.

Then the company locked up as much intellectual property as it could in cancer metabolism, Starr says, while at the same time building a discovery platform that merged two separate disciplines: oncology and metabolism. “To discover what those metabolic pathways are doing in cancer, we needed to build joint insight,” Starr says. “A big pharma company might have expertise in both cancer and metabolism, but it’s difficult for them to merge the two. When you’re a new company, it’s more straightforward.”

Knowing that Agios would eventually need a partner to help finance human trials, the startup team started talking to Big Pharma companies early. “In our first month, when we had only about five employees, we were already talking to them,” he says.

The company also worked hard in its early days to get on the scientific radar by submitting papers to medical journals. It had its first paper published in Nature in November 2009. The research described how a mutated gene called IDH1 produced a metabolite, which in turn seemed to contribute to some malignancies, including glioma, an aggressive and common form of brain cancer. “That showed the potential partners we were talking to that this vision could become reality,” Starr says. “It opened a lot of eyes.”

Celgene’s Daniel says his team has been wowed by a procession of research Agios has published since then, as well as what he describes as “a large body of unpublished data.” The research suggests that the metabolite produced by IDH1 may influence epigenetics—the molecular changes in cells that turn genes on or off without affecting the underlying DNA. Celgene is one of the leading makers of epigenetic drugs for cancer, with two such products: azacitidine (Vidaza) and romidepsin (Istodax). “We are interested in how epigenetic modifiers can be therapeutically valuable,” he says. And Celgene is already thinking about how drugs that target cancer metabolism might be useful in combination with its already marketed products, he adds.

Agios’s Schenkein—who joined the company in May 2009, after a stint at Genentech (now owned by Roche)—calls Celgene “an ideal partner.” The two companies meet once a quarter during a joint research committee meeting, when scientists from Celgene offer guidance and feedback on the data being generated. But virtually all of the research is done at Agios, with the startup free to make the final decisions about how to proceed with clinical candidates. The financing, adds Schenkein, has allowed Agios to build its ranks from 20 employees in the summer of 2009 to 60 today. “That allows us to go after multiple targets simultaneously,” he says.

Although the Celgene partnership will continue to focus exclusively on cancer, Agios’ scientists are already thinking about how their insights into cellular metabolism might be applied to other diseases. Schenkein declines to elaborate, except to say they’ll be ready to talk about those targets “shortly.”

All in all, adds Starr, Agios’ careful startup strategy has landed it in an enviable position in biotech. “This company,” he says, “has lots and lots of options.”

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