Emerging Drug Developer: Alnara Pharmaceuticals

On a wing, a theory and $20M in venture capital

When I caught up with Alexey Margolin a few days ago to talk about his new company, Alnara Pharmaceuticals, he was the only staffer on the payroll. There was no company office and not even a big technology licensing deal to discuss.

But he did have a hefty $20 million Series A round to bank on from some of the brand names in venture finance. The round was co-led by Frazier Healthcare Ventures and Third Rock Ventures, and included co-investor Bessemer Venture Partners. And he has two strong believers on the board -- Sirtris Pharmaceuticals CEO Christoph Westphal and Sirtris co-founder, Rich Aldrich - who have forged a sterling reputation for pushing the envelope in drug development.

Margolin also won't be flying solo for long.

Later this week, Margolin expects to be joined by the first of about 10 people he is recruiting to form Alnara's core clinical development team as he gets busy developing non-systemic, oral protein therapeutics. Margolin - the former chief scientific officer of Altus Pharmaceuticals -- has set out to prove that protein therapeutics can be developed to stay and do their work in the gut, and that could help revolutionize the field.

"I left Altus more than a year ago," says Margolin, where he worked on a similar program in non-systemic protein therapeutics. "I took some rest, though not for long, and started talking to people. I've known Aldrich and Westphal for a long time. We started shooting ideas around, talking about what will work, what won't work, and then we started our fundraising activity.

"It's pretty simple," says Margolin about the company's technology. "If you look at therapeutic proteins, (which are currently administered intravenously or subcutaneously) all attempts to deliver orally or other ways pretty much fail."

But if you can deliver protein therapeutics orally, so they will stay in the gut, they can do the work they need to do - "especially for metabolic diseases."

Margolin likes to use high cholesterol as an example. "We don't want proteins in the blood stream. We want them to stay in the gut," where the cholesterol can be most effectively targeted. To do that, the company will explore pills, capsules and liquid formulations.

"We have identified five or six programs where we've been doing very intensive market research, just to identify the lead program," says the newly minted CEO. "We'll be closing on it very soon. This money should bring us to clinical proof-of-concept and we should be able to advance a second therapeutic into preclinical development.

"I think one of the beauties of our approach, a reason why the investors like it, is the reduced risk of the whole development program. We start with molecules with known activity, reformulate and deliver them. For example, there are enzymes that can potentially work in these indications. Even if you need a new enzyme, there are well known techniques" to create them.

"My philosophy is to always have a pipeline," he adds. "Of course, you're limited by money." But by focusing on taking the first program to proof-of-concept, the company can validate its approach and, based on the results, add new programs.

Right now, Margolin says he's looking for office space. He'd like to stay in Cambridge, but that's expensive. At the very least, he says, he'll work out a lease in the greater Boston area.

But don't look for any sudden spending splurges.

"I think we should be very careful," says Margolin.

Emerging Drug Developer: Alnara Pharmaceuticals
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