Despite being forthcoming about its big-name venture backers—MPM Capital and Kleiner Perkins Caufield & Byers—Cambridge, MA-based Epizyme has been one of several biotech startups in the Boston that are being demure about the details of their technology. So I was intrigued when the company contacted me recently to let me know that it was lifting the curtains on its bold plans to make “epigenetic” drugs to combat blood cancer and tumors. And two founders also filled me in on the genesis of the company, which began during an MPM meeting in South San Francisco nearly two years ago.
Epigenetics involves enzymes and other molecules in the body that turn genes on and off without changing the underlying DNA code. Whitehouse Station, NJ-based drug giant Merck (NYSE:[[ticker:MRK]]) markets an early FDA-approved epigenetic treatment, the lymphoma drug vorinostat (Zolinza), which works by inhibiting one such type of enzyme, called histone deacetylases. Now Epizyme is embarking on an effort to discover drugs that target a different class of epigenetic enzymes known as histone methyltransferases (HMTs), company CEO Kazumi Shiosaki, who is also a managing director at MPM, tells me.
Epizyme is initially focused on developing drugs that knock out certain ones of these enzymes that are believed to either turn off cancer-fighting genes or activate genes that promote tumor survival. There are an estimated 70 HMTs, Shiosaki says, and among them are also potential targets for treating inflammation, neurological disorders, and other diseases. Yet it’s still early days for the company, which is in the process of selecting chemical compounds that could be developed as drugs to home in on these epigenetic enzymes. And there are several years of work to do before the firm tests the drugs in humans, the CEO adds.
“We have a very good understanding of the fundamental biochemistry that these enzymes operate under,” Shiosaki says, adding that this understanding puts the company in a strong position to identify drugs that target HMT enzymes.
Indeed, Epizyme has licensed technology from the lab of company co-founder Yi Zhang, a professor of biochemistry at the University of North Carolina at Chapel Hill, who has done extensive research on histone methyltransferase enzymes. The company is working on building an estate of patents related to these enzymes, though company officials believe they have competition on this front. Constellation Pharmaceuticals, a fellow epigenetics startup in Cambridge, was co-founded by another scientist who has been studying this enzyme class, Danny Reinberg, a professor of biochemistry at