CellCentric builds on a global epigenetics platform

Every time development pioneers try to bust down the door to a new sphere of drug discovery, a lot of time and money goes into building the therapeutic platform. And they have to carefully weigh the potential they're exploring against the investment it takes to push into novel territory.

For CellCentric, the start-up calculations were daunting. Six years ago "we realized that epigenetics was an important emerging space, but where it was going was not completely clear," says Dr. Will West, the CEO.

So back in 2004, the founders of the company decided to build a platform specializing in cancer targets with the help of some 30 scientists working at leading institutions around the world. Rather than hire a bevy of experts, they collaborated with them through a ‘hub and spoke' system that could deliver tidbits of knowledge through its research web. Those bits and pieces of the epigenetics puzzle were pieced together into new drug development programs by the small staff at the headquarters in Cambridge, UK.

Last week, Takeda Pharmaceutical inked a $200 million deal to in-license one of CellCentric's preclinical programs, a sum that included an unspecified upfront payment and a schedule of milestones.

"It's a target that's particularly relative to cancer," says West, "though it may have other therapeutic potential as well. The money coming into the business is cash, which is quite transformational for us. We will consult and advise, but there's no obligation to carry out discovery for them."

From this stage, Takeda scientists will take over the preclinical research work, and then hand over the most promising molecules to its Millennium subsidiary in Boston, which handles global oncology development.

This is a big step for CellCentric and another stride for the entire field of epigenetics. In a nutshell, the science centers on gene expression; regulating aberrations that trigger diseases like cancer, inflammatory diseases, neurodegenerative diseases and so on.

"We're sort of a fairly virtual operation," says West. "In terms of going forward, we will take our own programs forward but will also collaborate and work on new licenses. We are sitting on a unique platform of knowledge in epigenetics."

They're not alone. In the U.S., Constellation Pharmaceuticals has jumped into the field, garnering $32 million in mid-2008 from a group of venture capital companies interested in backing a start-up with its own ties to top investigators in the field. Last October SuperGen announced a $375 million epigenetics pact with GlaxoSmithKline, with $5 million of that arriving upfront. And the NIH has demonstrated its interest by earmarking $190 million for research work in the field.

Duke University's Randy Jirtle likens epigenetics to body software, orchestrating the action of the genome hardware.

"You're looking at the book of life, how it's read and how you can change it," Jirtle told the Washington Post late last year.

CellCentric's West acknowledges that the bulk of the money it's getting from Takeda is back-ended, which you'd expect from a licensing deal involving a preclinical cancer program. But the initial flow of money allows the developer to start thinking bigger.

"We've been fairly nimble in the early stages of development," says West. "We haven't built a big, expensive organization." The staff totals 10 right now, and eventually that could hit 20 - though West isn't really thinking much beyond that. West singles out a trio of in-house experts for coordinating the cutting-edge work: Nessa Carey, director of exploratory research; Neil Pegg, director of discovery research and David Knowles, chief scientific officer.

"We are working on seven targets and 11 others that we're interested in," says the CEO. "To take it forward ourselves would be too much. We're not a typical biotech planning on going into Phase II or Phase III."

The company's strength is in its intellectual property, and that makes CellCentric a likely candidate for a buyout at some point--if it continues to deliver promising science. - John Carroll (twitter | email)