Receptos, a San Diego-based biotech startup formed by a number of Biogen Idec veterans, has nailed down $25 million in venture capital to develop a vivid new way to look at certain protein structures on cells that it hopes will ultimately take a lot of the guesswork out of drug discovery.
The cash infusion is coming from Venrock Associates, Arch Venture Partners, Flagship Ventures, and Lilly Ventures. Bill Rastetter, a partner at Venrock who was the CEO of Idec Pharmaceuticals before it merged into Cambridge, MA-based Biogen Idec, has joined Receptos as CEO.
The big idea for the fledgling company comes from the labs of Ray Stevens and Hugh Rosen at The Scripps Research Institute in San Diego. The Scripps scientists have developed a process for capturing the first high-resolution crystal structure images of certain spaghetti-like targets on cells, known as G-protein coupled receptors. These are highly complex proteins that snake inside and outside of cells. This architecture, and the lack of great 3-D imaging to see them in their full glory, makes them especially tricky to develop specific drugs against. Even so, an estimated 30 to 40 percent of all pharmaceuticals worldwide hit these GPCR targets, including best-sellers for depression, allergies, and high blood pressure. Because the new method of capturing images of these proteins holds the potential for creating more specific new drugs against these targets, it was called one of the top 10 breakthroughs of the year in 2007 by Science magazine.
“It’s sort of like if you can’t look under the hood of a car, how can you know what’s wrong?” Stevens says. “What Receptos does is enable scientists to see the binding sites on the targets, to develop more effective new drugs against them.” He adds that this has implications far beyond any one drug, that this method will be applied over and over against a number of targets. “The potential is enormous,” Stevens says.
Rosen, a co-founder and former Merck executive director, called the new technique a “quantum leap” that will clear the way for more data-driven discovery of new drugs.
This isn’t meant to say any of this will be easy. Part of the challenge with characterizing GPCRs is that scientists using current methods are required to have the natural occurring, or synthetic, ligand that binds with the receptor. Receptos still has to operate within that limitation. But what the company has developed of value is a 15-step, reproducible process to make high-resolution crystal images of the known GPCRs, which gives scientists much more information about the target they are developing drugs against, Stevens says.
Once drug developers have the crystal structure figured out, then they have a solid template to design drugs with the desired ability to turn on or off a certain function related to disease. While this ability to capture crystal structures has eluded scientists looking at GPCRs in the past, it’s a standard part of the toolbox for researchers looking at tyrosine kinases—which have since become one of the hot targets in cancer biology.
So what does Receptos plan to do with this new platform for discovering drugs? A few things. It plans to form partnerships with other drug companies that want this same kind of information at their fingertips, says Marcus Boehm, a co-founder and vice president of chemistry. That will help pay the bills in the early days for what it really has in store, and that’s creating a pipeline of its own proprietary drugs.
“We’re not a virtual entity. We’re a serious engine,” Boehm says, for pumping out new drug candidates for clinical trials. Chrysa Mineo, the company’s vice president of business development, says the strategy is to develop “first-in-class, best-in-class” new therapies.
Receptos already has about 20 employees, led by Rastetter. Boehm was formerly VP of chemistry at Conforma Therapeutics until that company was acquired by Biogen Idec in 2006, and he stayed at Biogen after that. Mineo is a 20-year biotech veteran who got her start at Amgen and most recently worked on partnerships for San Diego-based Neurocrine Biosciences and its GPCR neurology drug candidates. Robert Peach, the vice president of biology and co-founder, was most recently the senior director of oncology discovery at Biogen Idec.
With all those connections to Biogen, the world’s largest maker of multiple sclerosis drugs, it shouldn’t come as a surprise that Receptos has picked a lead drug candidate for that particular neurodegenerative disease. The goal will be to develop an oral pill for MS that will reduce immune system flare-ups that damage nerves, while preserving the function of the nerves, Boehm says. That drug is being slated for clinical trials next year.
It was really the combination of the top-tier management team led by Rastetter, the scientific platform from Stevens (a proven entrepreneur who co-founded Syrrx and MemRx), and the fact that it has a drug poised to enter clinical trials that enabled Receptos to win so much funding in a difficult financial climate, Stevens says. “We have them all pulled together here,” he says.
Of course, since Stevens published that highly acclaimed paper in 2007, other groups around the world have sought to improve the characterization of GPCR drug targets as well. U.K.-based Heptares Therapeutics is also pursuing new methods of getting a better look at these targets. Last month, the Novartis Option Fund bought an option on the technology that could be worth more than $200 million over time. “We have a tremendous respect for Heptares,” Rosen says.
All this work is still at a very early stage, so there’s obviously a lot more science to talk about than medical evidence that says its drugs are on the right track. Those questions will come later, but for now, the Receptos group sounds jazzed about how anything’s possible.
“It’s the combination of technology, with the human capital, that really makes this interesting,” Mineo says. Rosen adds: “It’s worth investing our time, and that’s the most precious resource of all that we can give.”
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