Onyx Preps for Dash to FDA, After Myeloma Drug Helps Sickest of the Sick

7/26/10Follow @xconomy

Tony Coles has been pushing for two years to re-fashion Onyx Pharmaceuticals into more than a one-drug company, and now he might have the data to make that happen.

The Emeryville, CA-based biotech company (NASDAQ: ONXX) is announcing today that its drug candidate for multiple myeloma was able to at least partially shrink tumors for about one-fourth of patients who were so sick they had essentially run out of other options. Those fortunate enough to respond to therapy stayed in remission a median time of 7.4 months. Side effects—such as pneumonia, anemia, and depletion of infection-fighting white blood cells—were consistent with findings from a previous study. The evidence from the latest trial of 266 patients was strong enough that Onyx plans to use the data to seek FDA approval for its drug, carfilzomib, before the end of this year.

Onyx has been built on the back of one hit drug for kidney and liver cancer, sorafenib (Nexavar), which generated $843 million in sales last year for Onyx and its partner, Germany-based Bayer. Onyx has been seeking to diversify its portfolio for years, and made an aggressive move last October when it agreed to acquire South San Francisco-based Proteolix for as much as $535 million to obtain whole ownership of carfilzomib. About 20,000 people get diagnosed with multiple myeloma, a cancer of the bone marrow, each year in the U.S., and about 10,000 people die from the disease annually, according to the American Cancer Society.

Multiple myeloma therapy is dominated today by Cambridge, MA-based Millennium: The Takeda Oncology Company and Summit, NJ-based Celgene. Onyx is hoping this new set of results in the sickest patients will help it capture some significant share of a worldwide market that’s worth an estimated $4 to $5 billion a year, and still growing.

“This data really validates our growth strategy, and the acquisition of Proteolix,” Coles says.

Tony Coles

Tony Coles

Only a portion of the data is being made public to investors today, and much more detailed information will have to come later at a medical meeting or in a peer-reviewed journal. So there is plenty of room for skeptics to question the results being announced today, or at least reserve judgment for a later time.

That said, here’s the gist of what Onyx is reporting. This trial enrolled 266 patients who had previously relapsed and stopped responding to a median of five prior courses of therapy that usually included Millennium’s bortezomib (Velcade), Celgene’s lenalidomide (Revlimid), and dexamethasone, a common anti-inflammatory drug. Based on historical records, patients this sick were expected to respond to therapy about 11 percent of the time, and have a life expectancy of about six to 10 months.

These patients’ last hope was essentially to enroll in the Onyx study, in which they got regular intravenous infusions with carfilzomib. The Onyx drug, like Millennium’s treatment, is what’s known as a proteasome inhibitor. Millennium blazed this trail almost a decade ago, showing that if you can make a drug to inhibit these enzymes that act as a cellular garbage disposal, then you might block the release of certain proteins that cancer cells secrete to grow and resist conventional chemotherapies.

Because the patients who enrolled in this study were extremely sick, and had already cycled through all the other therapies, the trial was designed to simply enroll patients on the Onyx drug without randomly assigning them to a placebo or any other drug that could offer a comparison.

Heading into this study, Onyx said it was hoping to see at least … Next Page »

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