Emerging Drug Developer: Alnara Pharmaceuticals

Fledgling Alnara plots a 2010 market launch

As chief scientific officer for Altus Pharmaceuticals, Alexey Margolin, PhD, grew intimately familiar with ALTU-135, an experimental pancreatic enzyme replacement therapy the developer tested in patients with cystic fibrosis and chronic pancreatitis.

But by the time he had gone off and started his own company, Alnara Pharmaceuticals, in the fall of 2008, Margolin had thought all of that work was behind him. Just a few months later, though, the late-stage drug dropped back into his lap, and it would have a profound effect on the newly-launched biotech company.

"It certainly wasn't planned," says Margolin. "When I left Altus I thought I would never work on this program again."

Altus was doing a Phase III on the drug--liprotamase--when the company "pretty much ran out of money," he adds. "They couldn't raise the funds, and they couldn't just stop. The Cystic Fibrosis Foundation held the IP according to their agreement. Altus had to return the program to the foundation."

So Alnara, which had only a skeleton fulltime staff, stepped in.

"I think the reason they chose us was because of our intimate knowledge and commitment," says the CEO. "As you can imagine, the program was very complex, in Phase III, if someone else had come in there would have been another year of delay." And the foundation's main goal is to get the drug approved and in the hands of patients as swiftly as possible.

Margolin is confident now that the company can complete and submit the NDA for liprotamase this quarter. And he has the money in the bank, as well as the data, needed to complete the process. New investor MPM Capital led a $35 million Series B for Alnara last week, joined by current investors Third Rock Ventures, Frazier Healthcare and Bessemer Venture Partners.

Liprotamase is designed to treat pancreatic insufficiency, a condition which afflicts people with CF and other ailments. Patients can't properly digest and absorb fat, protein, and carbohydrates, preventing them from getting the nutrition they need. And Alnara believes that liprotamase can swiftly become the preferred, front-line drug for pancreatic insufficiency.

There is no pediatric formulation of liprotamase for kids (and adults unable to swallow capsules) and so that is what we have as a second program," says Margolin. "It looks like Alka-Seltzer. You put this tablet in a small amount of water and it disintegrates quickly so kids take it easily. The plan is to start a pivotal trial this year and file a supplemental NDA this year."

With only about 120 treatment centers for cystic fibrosis in the U.S., Margolin thinks Alnara can successfully commercialize the therapy on its own with a small sales staff of 20 to 25 people. Outside the U.S. market, he adds, Alnara will likely line up a partner for commercialization.

Adds Margolin: "There has been plenty of interest. As soon as the NDA is filed we will look at all the opportunities carefully."

Alnara has already begun to make some of the key appointments it will need to fill this year. The developer ended 2009 with a fulltime staff of only 10, relying on some 30 contract employees to carry much of the work load. More staff positions will now need to be filled as the fledgling developer makes a quick leap into commercialization.

"We've already brought on some of the skill sets we'll need," says Bob Gallotto, the chief business officer. "We'll bring together the infrastructure on the marketing and sales side early on. Then we'll wait to hire all the reps until we see approval."

Lee Brettman, MD, joined Alnara as chief medical officer in October. Brettman was the founder and CEO of Dynogen and is a veteran of Millenium, Vertex and other developers where he shepherded several products to an approval.

Liprotamase is getting the lion's share of the attention now, but Margolin still has two preclinical programs in place that will grow more important to Alnara after the new drug is launched.

Both those programs "are based on the same approach we discussed more than a year ago," the CEO tells me. "This non-systemic delivery of protein therapeutics--all proteins are delivered orally but stay in the gut--this principle hasn't changed." But it will probably be next year before one of those programs can be moved into the clinic.

For now, Alnara's small staff is staying focused on liprotamase.

Emerging Drug Developer: Alnara Pharmaceuticals

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