[Updated 3/12/15 9:55 am.] Even after the FDA refused to approve the anti-cancer drug tipifarnib in 2005, some experts could see a tantalizing glimmer of potential for patients with acute myeloid leukemia (AML) and other forms of cancer.
[Updated to add Kura’s presence in Cambridge, MA] Now the oral drug is getting a second chance at Kura Oncology, a new San Diego biopharmaceutical company led by Troy Wilson, a serial life sciences entrepreneur. Kura is springing onto the scene as a fully formed public company, after Wilson secured a $60 million investment accompanied by a reverse merger—an unusual route that enables Kura to publicly list its shares as quickly as possible. The company also has an office in Cambridge, MA, Wilson said.
Wilson wants to apply the concepts of “precision medicine” to determine if tipifarnib can be used to treat some people with certain types of AML and solid tumor cancers. The drug was shelved before advances in genomic sequencing, which are now being applied to help drug researchers identify the genetic traits of patients who respond well to certain drugs. “Tipifarnib was sort of the right drug at the wrong time,” Wilson said yesterday in a phone interview.
Wilson’s most notable success was San Diego’s Intellikine, which advanced a portfolio of anti-cancer drugs targeting the PI3 kinase pathway. Japan’s Takeda Pharmaceuticals acquired Intellikine at the end of 2011 in a deal valued at $310 million. Since then, Wilson has founded two other life sciences startups, Wellspring Biosciences and Avidity NanoMedicines.
The underlying concept of precision medicine, which has made national headlines this year thanks to a big push by the National Institutes of Health and President Obama, is to optimize healthcare—in the form of drugs or other measures—that is most effective for a patient based on his or her individual genetic traits. Kura intends to take advantage of advances in next-generation sequencing as well as emerging information about cancer genetics to identify the patients most likely to benefit from tipifarnib, Wilson said.
Kura is going to apply tipifarinib to AML, a fast-spreading cancer in which the bone marrow makes abnormal blood cells known as myeloblasts. In the United States, about 19,0000 new cases of AML are diagnosed each year, with the average patient facing an overall survival rate of 25 percent after five years.
Johnson & Johnson (NYSE: JNJ) discovered tipifarnib, which binds to a particular enzyme (farnesyl protein transferase), preventing it from activating Ras oncogenes and inhibiting the growth of certain types of cancer cells. The company tested the drug in a wide variety of cancers, with mostly disappointing results in colorectal, lung (both non-small cell and small cell), brain, pancreatic, and urothelial cancers.
According to Wilson, about 10 percent of AML patients responded completely to tipifarnib in J&J’s mid-stage clinical trials. The drug also showed promise in patients with peripheral T-cell lymphoma.
But the results for a few patients was not enough to warrant FDA approval, and in 2005, the FDA rejected J&J’s new drug application for tipifarnib, saying “you need to know which patients will respond,” according to Wilson.
Kura has licensed tipfarnib from J&J’s Janssen Pharmaceutica for development and commercialization. The company also raised $60 million through a private placement of its common stock to a group of institutional investors, led by EcoR1 Capital. Fidelity Management & Research, ARCH Venture Partners, Boxer Capital of Tavistock Life Sciences, Partner Fund Management and other healthcare investors also participated.
In conjunction with the private financing, Kura acquired a defunct public company in a reverse merger that will enable Kura to register its stock on the Over-the-Counter Markets within the next couple months.
In addition to holding a doctorate in biorganic chemistry, Wilson has a law degree and experience in corporate financing and securities. But he said the idea for a private stock placement followed by a reverse merger with a public shell corporation was suggested by the investors, who preferred to make their investment in a public company.
Over time, Wilson said the company would likely move from the OTC Markets to the Nasdaq or New York Stock Exchange, which have more rigorous listing requirements.
In forming Kura, Wilson said he has pulled together many senior managers from Intellikine and elsewhere, including the chief scientific officer, Yi Liu; senior vice president of chemistry and pharmaceutical sciences; Pingda Ren, and chief medical officer, Antonio Gualberto.
“We’ve got the cash and we’ve got the people,” Wilson said. “Now we have to execute.”