Epizyme, Riding High on Two Big Pharma Deals, Zeroes in on Personalized Cancer Therapy

3/29/11Follow @xconomy

Big Pharma companies are not exactly known for being nimble, but they have made a couple of quick bam-bam moves in succession to land deals with Cambridge, MA-based Epizyme.

The company made news in January when it pocketed $20 million upfront, and lined up another $630 million in development milestones, through a drug discovery partnership with London-based GlaxoSmithKline (NYSE:GSK). Two months later, Epizyme delivered an encore, with a new partnership worth $6 million upfront and another $200 million in milestones from Japan-based Eisai Pharmaceuticals, to develop cancer drugs against a specific biological target. Suddenly, it was time to look for a new office with double the space.

Those are all pretty encouraging signs of progress for a biotech company that’s barely three years old. It’s also an indication of enthusiasm for epigenetics, an emerging field of science that looks at how certain genes can be turned on or off without altering the underlying DNA. Instead of using chemotherapy that kills all kinds of fast-dividing cells, or a targeted cancer drug that rifles in on one specific mutant protein, Epizyme’s idea is to turn off the activity of a specific enzyme that basically makes it possible for multiple genes and proteins to spin out of control in cancer patients.

“These genes are really part of an orchestra,” says Epizyme’s chief business officer, Jason Rhodes. “We can influence the entire orchestra.”

The proof, like with anything in biotech, will be in the clinical trials, which aren’t here yet. But the Epizyme approach has some obvious appeal in that it’s going after biological targets that have only been well characterized, and clearly implicated in cancer, for a few years. The epigenetic targets are also enzymes, which means that scientists ought to be able to synthesize conventional small-molecule drugs that bind very specifically with these 3-D structures, Rhodes says.

It will still probably be “a couple years” before Epizyme finishes all the work it needs to bring its first drug into clinical trials, CEO Robert Gould says. And, like you’d expect from a scientist, he knows a lot of things can still go wrong: “There’s still a long, rocky road between where we are to the clinic,” he says. Plus, Epizyme isn’t the only company out there in hot pursuit of epigenetic drug targets—Cambridge, MA-based Constellation Pharmaceuticals is one notable competitor.

From left: Jason Rhodes, chief business officer of Epizyme, and Robert Gould, president and CEO of Epizyme.

In some ways, Epizyme represents a “quintessential biotech story,” says Richard Pops, the CEO of Alkermes (NASDAQ: ALKS) and a member of the startup’s board. Epizyme, he says, has all the classic ingredients of exciting science, top-notch people involved, and experienced VCs willing to push a project way out ahead of Big Pharma. While that might be exciting for a while, it doesn’t last, which is partly why the last few months have been important at Epizyme.

“As it came together we saw it as a higher risk/return company than was fashionable at the time,” Pops says. “Now, with the progress and validation of the deals, it is becoming more and more concrete.”

The GlaxoSmithKline deal was particularly important, both to bring in cash and to help the company build out its pipeline to hedge its bets. The Glaxo deal is set up to support research against a certain number of biological targets, which aren’t being disclosed, for the treatment of cancer and other diseases. But Epizyme made sure it didn’t give away the entire store to Glaxo. Epizyme got support for its research, while retaining full ownership of its two most advanced drug development programs.

One of those programs is directed … Next Page »

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