Based: San Diego
Founded: 2006
Website: www.ventirx.com
Co-Founders: Michael Kamdar, CBO, and Robert Hershberg, CMO

The Scoop: VentiRx started the year with a venture capital bang, adding $25 million to its bank account and bringing its total to more than $50 million. That's a solid record for a virtual biotech company with some committed backers--several of whom made a point of communicating their strong feelings directly to FierceBiotech earlier in the year.

What Makes it Fierce: VentiRx helps define the new breed of lean-and-mean in biotech. When the developer pulled the wraps off its last venture round in January, co-founder Michael Kamdar--there is no CEO, by design, at this virtual company operated by a 12-person staff--asserted that the company was well set to make it through to the next big funding event, whether that be a partnership, a buyout or an IPO.

It's using its money to advance small molecule drugs that spur Toll-like receptors--specifically TLR8--to rev up an immune response to fight off disease. And it set its first sights on cancer--a big target for a little company to tackle. In the lab, VentiRx has seen signs that its TLR8-targeted therapy can spark antibody dependent cellular cytotoxicity, making it a potential add-on for existing blockbusters. Its researchers have also come up with a nasal spray version of its TLR8 stimulator to fight allergies, essentially hijacking the body's immune response from the cascade of events that trigger an allergy attack.

VentiRx's TL8 niche falls in a wedge between TLR7 and TLR9, where other discovery units are already at work. It's no accident that AstraZeneca, which is already focused on TLR7, jumped on board VentiRx's wagon through the investment arm of its subsidiary MedImmune.

"It's actually worked out quite well," says Kamdar. "I have an extensive business and finance background, Rob (Hershberg) is a PhD." Hershberg, the CMO, had worked on TLR4 at Corixa, Kamdar on TLR7 at Anadys. And they both thought that TLR8 was the way to go forward.

"From our standpoint, TLR8 was fairly underappreciated," says Kamdar. The target couldn't be tested in rodent models, forcing researchers to start with larger species that cost a lot more to work with. And the literature has since been catching up to VentiRx's work, with signs that they may be on the best path to treat asthma, allergic rhinitis and various cancers.

On the cancer side VentiRx has gathered some early stage data demonstrating disease stabilization in about 25 percent of patients and a Phase Ib/II will examine its efficacy in combination with chemotherapy and monoclonal antibody therapy. In vitro data points to their approach as a promising additive to therapies like Rituxan, Erbitux and Herceptin.

VentiRx researchers are teamed with the University of Pennsylvania on a mouse study where the mice were treated after getting an ovarian cancer tumor transplant. Those results will be presented later this year, says Kamdar, adding that "we're looking at moving forward in ovarian cancer."

Following up on positive preclinical data on dogs, which indicated that a single dose of their intranasal therapy had a chance of replacing daily allergy meds, researchers launched a Ib study in allergic patients in an allergen exposure chamber in Austria. Data from 75 patients was "very encouraging, with subjective and objective signs of efficacy." Another Ib study will produce fresh data in the fourth quarter and VentiRx expects it can deliver "data next year that show results across the board on efficacy and tolerability."

Venture backers: Arch Venture Partners, Domaine Associates, Frazier Healthcare Ventures and MedImmune Ventures.