Emerging Drug Developer: Epizyme

Epizyme plots a pioneering path in new drug field

Two years ago, MPM Capital was hosting a meeting of its scientific advisory board when Yi Zhang, a professor of biochemistry at the University of North Carolina Chapel Hill, came in to outline the work he was doing in epigenetics. Kazumi Shiosaki, PhD, a managing director at MPM, vividly recalls his impact.

"Zhang," she says, "brought the house down."

And Shiosaki quickly built a new biotech house around that original idea.

In short order Kleiner Perkins Caufield and Byers, which has forged a reputation for its taste in disruptive new technologies, was on board as a co-investor in the first round for the newly-minted Epizyme. Shiosaki stepped in as the start-up CEO, a role she has played several times before, and the company got started as a stealth developer. Last spring Epizyme stepped out from behind the curtains. And last week the fledgling biotech wrapped a $32 million Series B-with Bay City Capital, Amgen Ventures, and Astellas Venture Management stepping in for the first time--designed to carry the company into 2011.

In a nutshell, Epizyme is developing drugs that target gene-regulating enzymes which are responsible for triggering diseases. Much as Merck's Kolinza targets histone deacetylases, Epizyme is focused on histone methyltransferases (HMTs), which play a role in spurring cancer as well as inflammation, metabolic diseases, neurodegenerative ailments and other conditions.

"Epigenetics has been around for some time," says Shiosaki. The therapeutic possibilities attached to being able to control gene expression are well understood. Enzymes are "intrinsically druggable. A small molecule can be developed to control that enzyme." And, she adds, researchers at Epizyme have conducted some promising experiments showing how epigenetics can work to fight various cancers--one of the hottest fields in drug development today.

"We now have a number of potent inhibitors that appear to be very specific for our HMT of choice," adds Shiosaki. And the company is ramping up work that is ultimately designed to provide proof-of-concept data in cancer.

But don't ask for a timeline. Epizyme operated in stealth mode for more than a year before unveiling its approach. And Shiosaki is still reluctant to detail its R&D blueprint. "We have a very aggressive plan," she says, adding only that it will be several years before one of its therapies can be approved for human use.

For now, Epizyme is focused on identifying two lead programs, along with three to five other earlier-stage programs that can follow in the pipeline. But because the Epizyme platform is broadly focused, the developer also expects to be able to line up collaborations with pharma companies at an early stage of its corporate development.

"There's tremendous interest in Epizyme," says the CEO. "We've certainly started an early-stage dialogue with pharma companies. They've called us to learn more. And, patting ourselves a little on the back, pharma companies have been impressed with our progress in a short period of time."

Right now, says Shiosaki, Big Pharma is just not able to internally develop transformational drugs. The vision behind Epizyme is to do just that. And it can potentially play the role of offering up partnerships on new therapies that can shake up entire drug markets. 

"I think we were spot on, in that pharma was very interested in a dialogue with Epizyme," says Shiosaki. "I think they know they can't do this kind of innovative research in their own companies."

One other company, Constellation Pharmaceuticals, has also been pioneering new work in epigenetics. Shiosaki says that it was just a coincidence that the two biotechs got started at just about the same time. But she's not alarmed by the presence of another early-stage developer in the epigenetics field. The science has broad application, she notes, and there's plenty of room for more than two players.

"If Epizyme was the only one working here, I might have thought that maybe we were too early," says Shiosaki. "Other companies will be started in this space." 

These are still early days at the fledgling biotech company, and ultimately there are many directions for the developer to go. Whether that eventually involves an M&A deal, an IPO or something else, says Shiosaki, is still to be seen.

"I think a company, to be very successful, should not have one option but many."

Over the next year Shiosaki expects Epizyme to boost its ranks from the 16 people who work there today to the low to mid-20s. And it will continue to outsource much of its work as it stays focused on getting the maximum bang for its buck. As an investor and an officer, that's something that Shiosaki never loses sight of.

Emerging Drug Developer: Epizyme
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