Emerging Drug Developer: Transave

Transave takes precise aim at lung infections

Transave’s fortunes will rise or fall based on the success of its drug delivery technology. And with a fresh, $35 million venture round in its pocket, the biotech’s current trajectory is up.

“Our focus is on pulmonary delivery technology,” says CEO Tim Whitten. “We take existing or generic drugs, reformulate them into next-generation pulmonary delivery technology--using liposomal technology--and dramatically change the product profile.”

It’s not a new area of drug development. But there are new ways to stand out in it.

“We’re highly efficient in how much drug we can pack into the lipid,” says the biotech exec. “That reduces the volume that you have to inhale through a nebulizer in the mouth.” The particle size is also ideal for inhaling in the lungs, he adds. In the case of Transave’s lead therapy, Arikace, 65 percent of the drug is delivered into the upper airways and 35 percent to 40 percent goes into the lower areas.

“For Arikace (a form of the antibiotic amikacin that is enclosed in nanocapsules of lipid called liposomes), those liposomes are designed to penetrate the mucus that cystic fibrosis patients and other patients have. The mucoid barrier allows Pseudomonas lung infections to grow unfettered,” adds Whitten. Transave’s approach is to get the liposomes through the mucus barrier as well as the biofilm created by the Pseudomonas to the site of the infection and release the drug on target, something like a militarily precise bombing mission.

Adds Whitten: “That could give us the opportunity to deliver potentially break-through efficacy in patients with mucus or biofilm. We would position this as a first-line therapy. There are a couple of therapies in CF. TOBI (Tobramycin Inhalation Solution) is the leader in the U.S. for inhalation into lungs for Pseudomonas.”

That business model has attracted a lineup of venture capital groups. Quaker BioVentures and Bessemer Venture Partners led the fourth round for the Monmouth Junction, NJ-based biotech. Also participating were TVM Capital, Prospect Venture Partners, Fidelity Biosciences, Forbion Capital Partners and Easton Capital.

Transave’s intellectual property originated with research that was developed at the Liposome Company in nearby Princeton, NJ--which Transave purchased after the developer was bought out by Elan--and then further developed on its own.

“I joined the company four to six months after the Series C round had closed,” says Whitten, an experienced marketer and former senior VP of commercial operations and development at Pharmacyclics. “It was readily apparent that we would need a fourth round to achieve proof-of- concept data. We will have proof-of-concept data in two indications and get into Phase III a year from now.

“Right now, I’m running the company assuming we’re going to take this all the way to commercialization,” he adds. That’s possible because launching a product like Arikace would only require a U.S. sales force of up to 24 sales reps, a business model that’s already been tested by the likes of Genentech.

“But,” says Whitten, “we’re also open to different alternatives as we go ahead.

“We’re anticipating filing with the FDA in 2011. We have funding that will take us into the second half of 2009. I think when we get our proof-of-concept data we have several options: mezzanine financing, a partnership, a public offering. And we’re open to all those. We’ll make those decisions toward the end of this year.”

There are other decisions ahead as well. Whitten says a third indication for Arikace--which he’s not yet revealing--will probably be added for clinical development.

“We also have one other compound which has completed several Phase Ib-type of trials,” says Whitten. “We’ll likely license that compound; pulmonary delivery for cancers that affect the lung.”

Researchers at Transave--which has 45 staffers--are also quietly beavering away in a new field with star potential.

“We believe that we have the potential to be the preferred pulmonary delivery technology for RNAi,” says Whitten. “We’re working toward proof-of-concept in that regard. For now, we’ve stayed pretty much under the radar screen. We have three collaborations in various stages in terms of pulmonary delivery of RNAi.”

But none are being made public--yet.