The Most Intriguing New Businesses

New Weapons in Medicine


When Kazumi Shiosaki is explaining the science behind her biotech startup, Epizyme, to investors, she uses a piano analogy. The keys, she says, are like genes: They're silent until fingers on the keyboard pick out a tune.

Genes, of course, determine how our bodies change over time. But they only spring into action when prompted by another set of biochemical factors—separate from DNA—known as the epigenome. Epizyme scientists believe they may be able to control disease-related genes by aiming new drugs at these factors.

The science of epigenetics is highly complex, but investors seem to get the picture. On Oct. 7, privately held Epizyme, based in Cambridge, Mass., announced that it had raised $32 million from a diverse group, including the venture-investing units of biotech giant Amgen (AMGN) and Japanese drugmaker Astellas Pharma. It was an impressive feat in a market that's hostile to biotech startups. The amount of venture capital raised by biotechs in the third quarter of 2009 dropped 30% year-over-year, to $759 million, according to San Francisco financial-services firm Burrill & Co.

"NEW BIOLOGICAL PLATFORM"Epizyme is in the spotlight mainly for its promising research on cancer. Its insights stem from an understanding of how DNA wraps around proteins that control how genes create cells, tissues, and organs. Its new drugs are designed to cripple enzymes that go awry in this process, contributing to cancers of the lung, breast, prostate, and more. Someday they might be tested against Alzheimer's disease, inflammation, and obesity as well. "We believe epigenetic targets present a fundamental new biological platform that will allow us to get into [such] indications," says Shiosaki, Epizyme's CEO and a managing director for its lead investor, MPM Capital.

Epizyme faces a daunting hurdle: It must prove that its drugs can hit their exotic targets without harming healthy tissue. "Some epigenetic changes are supposed to be there—they support life," says Susan K. Murphy, assistant professor at Duke University Medical Center.

Shiosaki's background prepared her for such challenges. A PhD chemist, she began her career in drug discovery at Abbott Laboratories (ABT) and Millennium Pharmaceuticals before moving to MPM Capital. In 2007 she organized an academic presentation on emerging cancer research and realized the field of epigenetics had come of age. MPM formed Epizyme under Shiosaki's command, and she recruited a slate of other drug-industry veterans, including chief scientific officer Robert A. Copeland, who came from GlaxoSmithKline. The team impressed Shinya Yano, CEO of investor Astellas Venture Management. Though it's still early days, he says, "Epizyme has a lot of talented scientists."

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