Epizyme - 2011 Fierce 15

Based: Waltham, MA
Founded: 2008
Web: epizyme.com
CEO: Robert Gould

The Scoop: Many cancer drug developers have put their faith in compounds focused on one misfit gene. However, Epizyme's technology might enable the firm to influence several cancer-related genes with a single small molecule--as well as affect genes that have evaded previous drug regimens. That's part of the promise of the firm's research into targeting certain epigenetic enzymes, which are like conductors whose activities affect entire sections of genes among the proverbial genetic orchestras involved in cancer. Over the past year, the firm has struck lucrative partnerships with several outfits--including major drug players Eisai and GlaxoSmithKline ($GSK)--to help underwrite its research in the hot class of epigenetic enzymes called histone methyltransferases. 

What Makes It Fierce: Epigenetics, which involves causes of altered gene expression other than changes in the DNA code, made the cover of Time magazine last year for its importance in understanding diseases. But Epizyme, which has raised $54 million in two venture rounds, deserves credit for significant progress in transforming epigenetic research into potential drugs. Even more, the company has banked more than $30 million in upfront cash from a sting of partnership deals this year.

Since Robert Gould (pictured) took the top spot at the biotech in early 2010, the business has grown up from a venture-funded research effort to a company with financial ties to Big Pharma outfits and disease groups. He joined the biotech around the same time as Jason Rhodes, a former dealmaker for Alnylam ($ALNY), who's been driving the successful partnership activities as chief business officer.

They've built nicely on the foundation developed with the help of chief scientist Robert Copeland and early CEO Kazumi Shiosaki, the managing director at MPM Capital who's still on the upstart's board of directors. The company also boasts high-profile academic co-founders H. Robert Horvitz, a Nobel Prize-winning biology professor at MIT, and Yi Zhang, a professor of biochemistry at the University of North Carolina Chapel Hill.

Talking to FierceBiotech during this summer's World Epigenetics Summit in Boston, Gould explained there's been intense interest in the company among clinicians even though the firm hasn't begun human testing of its drugs. That's because its compounds are being designed to serve very specific patient populations based on their genetics.

Think of kids with a rare and lethal form of leukemia. The firm is now advancing a small-molecule blocker of an epigenetic enzyme known as DOT1L, which is a culprit in mixed lineage leukemia (MLL), a deadly and small subset of acute leukemias. While the cancer develops in adults, it's notorious for poor prognoses in children. And, in a mouse study published in Cancer Cell in July, researchers showed that Epizyme's small molecule iced MLL cells without having the same killer effects on non-MLL leukemia cells.    

"We had a clinical investigators meeting at the ASH (American Society of Hematology) meeting last December to educate the people who treat these patients [with MLL]," Gould said, "and we had all the world's leading leukemia docs who are treating patients in the same room...it's that specific of a population."     

The Leukemia & Lymphoma Society agreed in June 2011 to provide up to $7.5 million to bankroll Epizyme's drug for MLL through Phase I. Big drugmakers have joined the party, too. Epizyme's research deal with GlaxoSmithKline announced in January 2011 brought the biotech $20 million upfront and gives the London-based drug giant the ability to develop drugs against a certain number of HMT enzyme targets for cancer and other diseases.

Epizyme's pact with Eisai, which was revealed in March, brought a lesser sum at the front end ($6 million). However, the Japanese powerhouse also agreed to fund all R&D costs for drugs involved in the deal through human proof-of-concept studies. That partnership focuses on molecules against the EZH2 enzyme, which plays a role in certain forms of lymphoma and other cancers.  

Epizyme is working toward completing the preclinical work necessary to advance its molecules against EZH2 and DOT1L, its lead projects, into initial clinical trials. Its chiefs wouldn't give a specific date on the start of human trials, but the nature of the IND-enabling studies they're doing now could land the firm's first candidate in the clinic sometime in 2012.

Venture backers: Amgen Ventures, Astellas Venture Management, Bay City Capital, Kleiner Perkins Caufield and Byers, MPM Capital and New Enterprise Associates.

Epizyme - 2011 Fierce 15

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