Emerging Drug Developer: Ambit Biosciences

Ambit Biosciences has taken another important step forward. In the middle of last month, CEO Scott Salka announced that the biotech had garnered close to $50 million in its fourth round. That money should be enough to get the company through 2009, a key transition period for Ambit.

“We’re expecting at least clinical proof of concept and completion of that milestone with one compound by the end of ‘09.” Salka tells FierceBiotech, which named Ambit a Fierce 15 company in 2006.

Until now, most of San Diego-based Ambit’s news has centered on a series of technology deals with an A-list of drug developers. The company’s KinomeScan has ginned tens of millions of dollars in revenue for the emerging biotech. The high throughout technology screens small molecules against hundreds of human kinases, detailing how each binds to a target in the search for kinase inhibitors.

But the technology isn’t just up for hire. Ambit has been using it to launch its own drug candidates. And on the heels of the latest venture round, Salka has been talking up the early positive indications researchers have been seeing for their lead candidate – AC220, an FLT3 inhibitor for acute myeloid leukemia.

AC220 has the potential to be a “significant improvement over first generation therapies,” says Salka.

“We’re seeing kind of what we were expecting,” he adds. The pharmacokinetics indicates that the drug can work as a once daily, orally delivered drug given at a relatively low dose with relatively few side effects. And he says that researchers will keep on going until they find how high they can safely take the dosage.

Once Phase I is complete early next year, says Salka, Ambit will take AC220 straight into Phase II, which should last about a year. Several mid-stage studies are planned in different subsets of AML patients. One of the trials will “likely” focus on a particular mutation found among AML patients. And Ambit has other programs that it intends to get into the clinic as well.

“If all things go well, we can be in a position to wrap Phase II in ‘09 and move into Phase III, finishing in 2010,” adds Salka. “That would be exceptionally fast.”

But fast has always been Salka’s goal. In 2005, Ambit raised $31 million in its third round. And at that time the biotech expected to raise one more round to drive it through Phase II.

“We have an outstanding group of partners: Bristol-Myers Squibb, Roche, GlaxoSmithKline, Cephalon, and we do screening for a lot of other companies besides,” adds Salka. “Any of those companies are likely suspects for a partner for more advanced clinical development. And we’re always talking and looking to expand the people who utilize the technology.”

Ambit has also been steadily adding new kinases to the system, growing to 353 last August. And that number is expected to grow past 400 soon.

At the end of October, Bristol-Myers Squibb forged its second expansion of its screening deal with Ambit. “So far,” says Salka, “all those deals have generated $45 million in non-dilutive cash, a significant amount of money for a young company like Ambit.” In the latest venture round – which brought its venture total to $106 million -- Apposite Capital LLP led the way with MedImmune Ventures and OrbiMed Advisors joining in.

Salka and Ambit researchers are still exploring possible endpoints for company trials.

“Progression-free survival is one possibility,” says Salka. “These patients tend to relapse pretty quickly. One likely area is to look at patients who responded to standard regimens and add a kinase inhibitor and look at progression-free survival and time to relapse.” Salka also noted that some of these goals could require a longer amount of time for a clinical trial to deliver results as quickly as he would like.

What happens after ‘09 is still a matter of some discussion at Ambit. If you ask Salka if an IPO, acquisition or new financing round is a possibility, he’ll say yes.

For now, Ambit is keeping all of its options open.