Amira Mixes Small Biotech Irreverence, With Big Pharma Rigor, To Create New Lung Drugs

Xconomy San Diego — 

Amira Pharmaceuticals looks and feels like a typical small biotech company, with the sort of irreverent sense of humor that you don’t often find at a Big Pharma shop. While CEO Bob Baltera showed me around the Amira offices on my last visit to San Diego, he opened up a storage room where employees had stashed away some belongings.

There was an old turkey baster, next to a bench press.

“That’s how we burn off a few calories around here before Thanksgiving,” Baltera cracked.

Amira may be able to joke around and have fun, but it’s actually borrowed more than a few pages from the old Big Pharma drug development playbook. The company was founded in 2005 by three scientists—Peppi Prasit, Jilly Evans, and John Hutchinson—who worked together for years at Merck before the giant pharma company closed its San Diego operation. As I described in a feature last March, they played important roles in developing montelukast sodium (Singulair), the asthma drug that generates $4 billion a year in revenue, and they resolved to try to do it again at their own company in San Diego. They’ve had some success already, raising a little more than $28 million in venture capital, building a staff of 50 employees, and scoring a partnership with GlaxoSmithKline.

But what I found most interesting when I met with Baltera was how Amira has combined some of the best cultural features of biotech and pharma in one place. That means while softball trophies are one prominent display of how the team literally plays together, Amira also insists on being a little extra careful and cautious, like a Big Pharma company, when it comes to building a body of evidence to support new treatments.

Bob Baltera

Bob Baltera

What does that mean? For one thing, Amira scientists identify not just one lead drug candidate to block a particular target, but they identify a lead and backup contender that generally follows six months behind in development. That way if a safety issue emerges with the lead candidate, the entire program won’t have to go in the trash. And by running experiments on both molecules, Amira hopes to answer whether the problem was in picking a dangerous target, or whether it was just something with the molecule that needs a minor tweak. From a business perspective, this means Baltera isn’t betting the company on a single molecule, which biotech startups often do.

“We try to sell a drug development program, not a molecule,” Baltera says.

This definitely costs more money, significantly more, to run simultaneous clinical trials of different molecules, Baltera says. He concedes that investors have occasionally asked why the company really needs to spend money on two candidates instead of one. Amira hasn’t really had to aggressively defend the strategy during the downturn, because the Glaxo partnership has provided enough support that it hasn’t had to raise money since Baltera arrived in July 2007. But he insists that going the extra mile mitigates the company’s risk, and helps establish credibility when it comes to forming collaborations with pharma.

“If there’s uncertainty in Phase I, it means we can’t go to Phase II,” Baltera says. “The data is what the data is. This is a pharmaceutical style small-molecule strategy. You pick the best one and go forward.”

While the Glaxo partnership provides essential cash to keep the lights on, Amira is trying to build its company around a very early-stage drug program for idiopathic pulmonary fibrosis. This is a fatal disease, and scientists don’t know what causes it. There aren’t any FDA-approved therapies at the moment. A couple of other companies, Brisbane, CA-based Intermune and Switzerland-based Actelion Pharmaceuticals, are further along with pulmonary fibrosis treatments, this is uncharted territory from a regulatory standpoint, meaning it’s hard to say exactly what the FDA will demand from a new therapy.

That sounds like the kind of risky bet that you’d expect from a biotech company. One of Amira’s scientific collaborators, Andrew Tager of Massachusetts General Hospital, has published data that suggests a drug which blocks a target called LPA1 might be useful for pulmonary fibrosis, and Amira has developed a drug, tested it in mice, and seen some promising results. Since this is a relatively uncommon disease, the clinical trials, and commercialization push could conceivably be handled by a small company like Amira, Baltera says. Amira has the incentive to do this since it has 100 percent of the commercial rights, Baltera notes.

This year will be a critical test for Amira’s strategy. It expects to have its lead candidate ready for its first clinical trial, by mid-to-late 2010, Baltera says. It will have a backup candidate right behind that. The first results from people should be available about a year later. If all goes well, then it can start taking a serious look at testing it for other fibrotic scarring conditions, like cirrhosis of the liver and certain kidney diseases. It might even be time by then to start whispering about an initial public offering, he says.

“It’s going to be all hands on deck around here in 2010,” Baltera says.