Emerging Drug Developer: Ascent Therapeutics

A fledgling biotech launches a new development platform

For a newly-sprouted biotech company, Ascent Therapeutics has some deep scientific roots.

The technology dates back to the late ‘90s, when the company's founding scientists--Athan Kuliopulos and Lidija Covic, both at Tufts Medical Center--started to push the envelope on G protein coupled receptor drug development. They came up with Pepducin technology--a new structure that enables Pepducin lipopeptides to more effectively modulate a well-researched drug target.

"They put in patents in 2000 and 2001 and started to try and raise money," says CEO Frederick Jones, MD. "They got some experienced biotech execs, including Mike Webb in 2006, and closed the Series A in October, 2007.

"The company operated in stealth mode for the next year," adds Jones. "The first six months was the process of taking a clean-slate look at all GPCRs and finding the most attractive targets for this kind of technology. The next six months was spent getting into the lab and getting a critical mass of people on board. We moved into lab space in the first of April, got critical mass over the summer and by October made the formal announcement. The other thing was that investors decided they wanted a say in the executive management team and I came on board in August and we brought in the head of R&D (Stephen Hunt, a veteran of Pfizer's Research Technology Center) in September.

"Pepducins is a name invented by Kuliopulos. It describes a new class of molecules which we're developing to modulate GPCRs. The molecules are short peptides based on intracellular loops of GPCR targets. Each molecule has a short lipid moiety, which allows molecules to anchor in the cell membrane. It gets caught up in the inner layer of the cell membrane, and the peptide portion dangles out and interacts with portions of the GPCRs. It's an entirely novel way of interacting with these receptors."

That kind of platform technology offers a wide range of targets.

"GPCRs are a well validated family of drug targets," says Jones. "Forty percent of currently approved drugs target GPCRs. On the other hand, it's a very large family with several hundred members and only a small number have been able to be drugs. We hope to greatly expand the GPCR space. There are 15 different GPCRs that the founding scientists in our labs have targeted to create Pepducins and in every instance they have ID'd active Pepducins against receptors, so it appears to be a very robust technology."

But then, a small biotech with 15 employees can't pursue 15 or 20 programs. So the initial goal has been to narrow their focus to a manageable number of internal programs and get started teaming up with other developers.

"They did narrow down the focus to a small number of targets; inflammation, oncology and some cardiovascular indications," says Jones. "And we're actually concentrating efforts on two of those targets. They are mostly applicable in inflammatory disorders.

"We plan to start a very active business development effort and look for collaborators who could complement the programs we have going on," he adds. "We ideally would continue our high-priority projects on our own. We're well funded to do that well into 2010. We'd be looking for funded research collaborations to move along projects that otherwise would sit on the shelf, monetize those, and let other companies get familiar with this brand new platform."

Adds Jones: "Convincing people this stuff can really work is a challenge. Sometimes they have to play with it in their own hands."

Cambridge, MA-based Ascent has already inked a $200 million licensing option pact with the Novartis Option Fund. On December 8 Ascent announced that it had gained an unspecified upfront fee and Novartis obtained an option against a specific GPCR target. Novartis Option Fund also participated in the company's Series A, which raised $19 million. Healthcare Ventures and TVM Capital participated in the round.    

"It's clear that even since I got here things have changed considerably in the financial world," says Jones. "And conditions are much different now from when investors made their original commitment. As a platform company, the question now is how much do you invest in broadening the platform versus putting all your bets on one program and moving that as far as you can or into clinical trials. And that's the sort of decision-making spectrum we're looking at."

By the middle of 2010 Ascent should have a program ready to go into Phase I and a strong focus on a second program, he adds. "It should take 18 months in Phase I, two years, two-and-a-half years in Phase II. So we're probably three to four years from getting through that first Phase II study.

"This is such an attractive platform, which is traditionally pretty well valued by pharma in general," sums up the CEO. "If we continue to make progress with the lead projects, we'd be in good position to raise additional capital. At this point, that's the path we expect to take. Business development becomes even more important in this environment."

Emerging Drug Developer: Ascent Therapeutics

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