Plexxikon - 2010 Fierce 15

Based: Berkeley, CA
Founded: 2000
CEO: Peter Hirth

The Scoop: With pharma focused on new frontline therapies, developers are relying on new technology to advance a fresh series of best-in-class therapies--anything that looks like a me-too approach won't get pushed. And Plexxikon hit paydirt when it collected a $60 million upfront from Roche in early 2009 pact covering its PLX-5568, an experimental therapy for a rare genetic kidney disease as well as additional undisclosed programs. Altogether the developer has rounded up a whopping $185 million in non-dilutive partnership funding, a stellar record in an industry that thrives on that kind of cash.

What Makes it Fierce: 5568 wasn't Roche's first deal with Plexxikon. The Swiss company had also committed itself to PLX-4032, an oral drug that targets the cancer-causing BRAF mutation, which triggers about half of all melanoma cases. The partners needed to reformulate the drug in the course of an early-stage study. But in August researchers unveiled some stunning results. In the melanoma extension cohort of the study, nearly all patients showed some response; 81 percent of patients had tumor shrinkage of at least 30 percent.

"This is the most important breakthrough in melanoma, ever," the University of Pennsylvania's Lynn Schuchter told USA Today.

Ask Plexxikon CEO Peter Hirth how big the company is and he replies: "It would be better to ask how small the company is." Plexxikon has big ambitions, but it has kept its staff to a lean-and-mean 42. "We operate very much as a virtual company," he says, with service providers expected to pick up the slack wherever possible.

A relatively small staff hasn't stopped Plexxikon from becoming a highly productive company. "We've paid taxes for the last three years in a row," says Kathy Glaub, the president of Plexxikon. And anyone in the biotech industry knows that is no small feat for a developer with no commercial products to generate revenue.

Two pivotal studies are underway for 4032, with results due later this year and in the first quarter of 2011. And Plexxikon has retained rights to a co-promotion role for itself in the U.S. market, leaving it at the threshold of a potential commercial launch that could prove transformative. But 4032 is just one of five clinical programs that Plexxikon tracks on its web site, with other experimental therapies being tested for Type 2 diabetes, rheumatoid arthritis, pain and kidney disease. "We've worked hard to have a unique business model," says Glaub.

Plexxikon has been largely funded through collaborations and $67 million in venture cash. "Going forward, we don't necessarily see changing our business model much," says Glaub. "Once we get our first product in the market, we'll have royalties coming in and steady revenue. We will be able to take things farther in the clinic."

"We have the makings of a very successful company," adds the president. "We have a validated engine, actually a very nice pipeline, most of which we don't talk about. With a commercial launch in the next year, we're a really different opportunity."

Venture Backers: Advanced Technology Ventures, Alta Partners, Astellas Venture Capital, CW Ventures, Daiwa SMBC Capital, GIMV, Kumho Asiana Group, Pappas Ventures and Walden International.

Plexxikon - 2010 Fierce 15

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