By Luke Timmerman

People with pulmonary fibrosis have a pretty raw deal. Scientists don’t really know what causes this lung-damaging disease, and there’s no really effective FDA-approved treatment. The disease makes it hard to breathe, and kills an estimated 40,000 people a year. That’s a bigger killer than some far better known culprits like prostate cancer.

A few drug companies have tried to fill this void with little to show for it yet. But San Diego-based Amira Pharmaceuticals says it is hot on the trail of creating a new small-molecule drug against a new target that it thinks could represent a big step forward. This program is in very early stages—too early to say anything about potential effectiveness—but I figured I’d listen because this company’s scientific team worked together at Merck’s former San Diego operation and played important roles in developing montelukast sodium (Singulair), the asthma drug that generates $4 billion a year in revenue.

Amira was started in 2005 by a trio of scientists—Peppi Prasit, Jilly Evans, and John Hutchinson—who worked together for years at Merck until that company closed its San Diego operation. I got the update on what’s new from CEO Bob Baltera, who came to Amira after a 17-year run at Amgen, while that company became the world’s largest biotech. He surprised me a bit, because instead of talking about the company’s two lead programs in development-including one in clinical trials for respiratory diseases that’s in a partnership with GlaxoSmithKline—Baltera mostly wanted to talk about a promising new protein target for pulmonary fibrosis characterized by Andrew Tager and colleagues at Massachusetts General Hospital. The Amira team is at work developing the best drug candidate it can to hit this new target.

“This is a grievous illness, and Amira is out at the forefront,” Baltera says.

There’s still a lot of work to do, maybe 12 to 18 months’ worth, before Amira will have a drug candidate that can enter clinical trials, Baltera says.

Tager’s research was described in January 2008 in Nature Medicine. Scientists found that mice who lack a certain protein called LPA1 were protected from fibrosis and death after being exposed to likely triggers of the disease.

Many of the first responders on Sept. 11, 2001 in New York are coming down with pulmonary fibrosis, so anything that’s remotely successful is likely to generate its fair share of attention. Baltera likes it for a couple of business reasons. The first is that he doesn’t see any direct competitors that he’s aware of, unlike the more crowded battlefield for Amira’s experimental drug that’s made to block a target called DP2 for asthma and chronic obstructive pulmonary disease. (However, Gilead Sciences, InterMune, Novartis, and Actelion are a few competitors trying to tackle pulmonary fibrosis in different ways.)

Pulmonary fibrosis also happens to be a disease that affects a small enough number of people that a small company like Amira, with 50 employees, can foresee building a small specialty sales force to market it without the help of a massive pharmaceutical partner. “We’ll keep this one for ourselves,” he says.

The company’s philosophy with this program, like others, is to shoot for a once-daily pill so that it can have a reasonable shot at being a commercial success, Baltera says. This philosophy from the founders comes from “battle scars” accumulated at Merck, when they discovered that science wasn’t the only thing that matters in drug development. “It’s easy to get on your scientific high horse and forget about what patients really need, and what payers really need,” Baltera says. “You’ve got to be practical in this business.”

With a meaningful milestone on this program still more than a year away, I wondered how Amira is managing the business. The company has done some belt-tightening in terms of keeping resources focused on its most “high-value” programs, Baltera says. The company raised $25 million in a Series B round in March 2007 from Novo A/S, Prospect Venture Partners, Avalon Ventures, and Versant Ventures. If that cash, along with undisclosed amounts it has gotten from its partnership with Glaxo, can stretch out long enough to get to some of those meaningful milestones, then the story could get much more interesting.

Luke Timmerman is the National Biotechnology Editor for Xconomy.
Email: ltimmerman@xconomy.com
Tel: 206-624-2374