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Ambit, Buoyed by Astellas Deal, Attempting Rare Switch from Platform to Drug Developer

Ambit Biosciences made news last Friday when it pulled in $40 million through a partnership with Japan-based Astellas Pharma. We had the basic gist out fast that morning, but I gathered a lot more detail and insight during a follow-up conversation with Ambit CEO Scott Salka.

It’s been a frenetic year at Ambit. The company entered 2009 with some encouraging, albeit preliminary Phase I clinical trial data on a leukemia drug. It is closing the year with the same drug that’s now in a pivotal study that—if successful—could form the basis for FDA approval. Along the way, Ambit added a Big Pharma partner with the money and manpower to really test its hypothesis in a big way in the coming year.

It’s been quite a journey for Ambit, a company that has reinvented itself, not once, but twice. As I described a couple months ago, Ambit was born in the genomics heyday of May 2000, and scrapped a couple of its previous business plans that were built around platform technologies it intended to offer to Big Pharma companies. As Salka points out, lots of companies have tried to make the switch from being a platform technology provider into a drug developer, but few have been able to pull it off. Millennium Pharmaceuticals is one company that raised a pile of money in the genomics days, but then became a success when it acquired a drug and further developed it into a billion-dollar molecule. Millennium was sold to Japan-based Takeda Pharmaceuticals last year for $8.8 billion.

“I really can’t think of many companies that have made the transition from a platform into a therapeutic company,” Salka says. Millennium made the transition, although Salka notes that it did that through acquiring a drug candidate.

While Ambit still has a long way to go before it can claim the distinction of turning its own platform into a product engine, it is at least in the game. “This is a big step toward the realization that we can transform the company,” Salka says.

Ambit pushed hard to prepare for the Astellas deal, Salka says. It had to have meetings with the FDA to discuss the protocol for a major clinical trial of 180 patients, recruit the clinical sites, get approval from local institutional review boards that monitor patient safety, and manufacture and ship enough drug to supply the trial sites. The company, which has about 85 employees, considered it a “stretch goal” to get the trial up and running by the end of 2009.

By securing the deal with Astellas, Ambit will get to hire another “half dozen” or so people for its clinical development team, Salka says. Part of their work will be on AC220, the lead drug candidate for acute myeloid leukemia (linked to a specific mutation of the FLT3 kinase), but some of this new capability also will go toward building up Ambit’s pipeline. The company has a drug that’s intended to block multiple variations of HER kinase, and a JAK2 kinase inhibitor for myelodysplastic syndrome that’s scheduled to enter clinical trials in the first half of 2010. Another version of a FLT3 kinase inhibitor that’s being designed for chronic use in patients with autoimmune disease is being prepared for the clinic as well, Salka says.

So as hard as Ambit pushed this year to be in a position to secure the Astellas deal, Salka doesn’t sound like he’s ready to take a rest.

“This deal really allows us to push the rest of the pipeline as fast as possible,” Salka says.