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San Diego’s Ambit Biosciences Raises $25M to Advance Leukemia Drug

Xconomy San Diego — 

Ambit Biosciences has gone through several course changes in the 12 years since it was founded. But Ambit CEO Michael Martino says the San Diego company would not be announcing $25 million raised from existing investors today unless Ambit was making significant progress on its current trajectory.

“What the financing represents is a huge vote of confidence by all the Series D investors who have invested in this Series E round,” Martino said in a phone interview. In a statement today, Ambit says the $25 million is the first tranche of $50 million in a preferred stock financing that will be used mostly to advance its anti-cancer drug quizartinib (designated previously as AC220) for acute myeloid leukemia (AML).

As Luke explained a few years ago, Ambit has been working to develop a group of small-molecule drugs that block certain enzymes called kinases, and quizartinib is Ambit’s lead drug candidate. The company says it has completed a 333-paitent, mid-stage trial of quizartinib in relapsed AML patients with the FLT3 kinase mutation, a particularly aggressive form of the disease. About one-third of AML patients, or 4,000 new cases a year, are estimated to have the FLT3 kinase mutation.

Ambit plans to disclose the results of its trial in December, at the American Society of Hematology’s (ASH) 54th Annual Meeting in Atlanta, GA. During our call, Martino sought to preview the results without saying too much. He referred to quizartinib as “truly a potential game-changer,” saying presentations and poster sessions scheduled for the meeting demonstrate Ambit’s “distinct competency” in developing oral kinase inhibitors that are both highly potent and highly selective, and that exquisitely target the FLT3 kinase mutation.

Martino, who joined Ambit a year ago from San Diego-based CareFusion (NYSE: CFN), said the company plans to also use some proceeds from the financing to develop other drug candidates, including AC430, a potential drug for treating autoimmune diseases like rheumatoid arthritis.

Since joining Ambit, Martino said he has refocused and prioritized the company’s work on earlier stage drugs in its pipeline, which included discontinuing work on the anti-cancer compound (AC480) and returning it to the licensor, Bristol-Myers Squibb.

Martino says Ambit has rebuilt its relationship with Japan’s Astellas Pharma, and he has rebuilt Ambit’s top management, recruiting Athena Countouriotis from Pfizer to serve as chief medical officer. The company now has a total of about 45 employees. Before moving to San Diego in 2010, Martino was the CEO of Seattle’s Arzeda and Sonus Pharmaceuticals.

Before April, when Ambit closed on $13 million in bridge financing, Martino said the company had raised $163 million. But keep in mind, Martino said, that Ambit was founded in 2000 and spent more than seven years developing a services business that screened drug candidates against certain protein targets. When he joined the company, Martino said Ambit was looking for a leader with a demonstrated ability to bring a framework and process to drug development, drive commitment, and raise additional capital.

New York-based OrbiMed Advisors, an existing investor, led the financing announced today. Other existing investors joined in the round, including Aisling Capital, Apposite Healthcare, Roche Ventures, GrowthWorks, MedImmune Ventures, Forward Ventures, GIMV, and Radius Ventures.

As part of the deal, David Bonita, a principal at OrbiMed, is joining Ambit’s board. In the company’s statement, Bonita calls the recent data from quizartinib’s Phase 2 trial “very promising.”