Biotech companies run into walls all the time, and either reinvent themselves or die. If they’re fortunate, they get one shot at a turnaround. San Diego-based Ambit Biosciences is getting a third chance, and this time it has some hard data, not just a flashy concept, to offer.
Ambit got started in May 2000, just when the bubble was starting to pop on the Internet and genomics stock bubbles. Ambit’s original plan was to take some technology out of Yale University to build a database of all the proteins in the body and sell the information to drug companies.
When that business model fell out of favor just seven months later, Ambit re-started around the idea of screening ideal drug candidates against certain protein targets. Two multi-billion dollar drugs for diabetes and neurological conditions, metformin and gabapentin, were losing patent protection, and Ambit thought it could develop more effective second-generation treatments if it could discover how they really interacted with protein targets. It raised $20 million on that effort in 2001 and 2002, before it flamed out the next year.
“At the end of the second incarnation, we had nothing. It didn’t work,” says Scott Salka, Ambit’s CEO. “We had two strikes against us, we were standing at the plate, and had hardly any money. We could fold up our tent and go home, or salvage something.”
Ambit’s next chance came with an emerging class of drugs known as kinase inhibitors. They have been a hot area for prospective cancer drugs over the past decade, and block certain enzymes called kinases. Novartis’ imatinib (Gleevec) achieved breakthrough status (and multi-billion-dollar sales) as an inhibitor of a very specific kinase involved in chronic myeloid leukemia. Other pioneering kinase inhibitors, like Pfizer’s sunitinib (Sutent) appeared to work well against kidney cancer even though it blocked several variations in the family of kinases. That stirred debate about whether it was better to be more selective to certain targets, or less, in developing new compounds. That created demand among drug companies to do in-depth selectivity studies, Salka says. As a result, drug companies lined up for services from Ambit, which could screen large numbers of kinases for the best possible drug to block them.
That core skill of Ambit’s was good enough to win support from Roche, Bristol-Myers Squibb, GlaxoSmithKline, Pfizer, Johnson & Johnson, and Cephalon, and has provided some cash to keep the doors open of a fledgling biotech company for nine years. It’s also enabled Ambit to raise a total of $105 million from a list of investors that now stretches 14 deep.
But just like genomics was the hot new thing that faded in 2000, it’s hard to get investors excited about a biotech business that collects fees for services from Big Pharma. The big potential, as always, is in developing new drugs for deadly diseases.
While Ambit was screening kinases for Big Pharma customers, it was also building up a portfolio of kinase drug candidates of its own. The company’s lead asset of the moment is AC220 for acute myeloid leukemia. It’s a malignancy that strikes 13,000 people each year in the U.S., mostly elderly, who have a short life expectancy and no realistic treatment options beyond chemotherapy. With little notice, Ambit is gearing up this month to start a pivotal clinical trial of this product and is even in partnership talks. If this drug can match striking results seen in an earlier study, then Ambit could have this product on the market by the first half of 2011, Salka says.
“It’s a great story for San Diego biotech,” Salka says. “Hopefully we can deliver the goods.”
Ambit drew some attention for its drug last December at the American Society of Hematology meeting in San Francisco. That was where researchers reported that 16 of the first 54 patients with relapsed forms of acute myeloid leukemia had tumor shrinkage
