Onyx Pharmaceuticals is betting that it can keep growing on the strength of a new cancer drug that’s supposed to compete with a blockbuster from Cambridge, MA-based Millennium: The Takeda Oncology Company. Today, Onyx is presenting key data to gird for the market battle.
Onyx, the Emeryville, CA-based biotech company (NASDAQ: [[ticker:ONXX]]), is reporting today on a clinical trial of 266 patients that shows its lead cancer drug in development, carfilzomib, shrank tumors by at least half in 24 percent of the sickest patients with multiple myeloma, a deadly cancer of the bone marrow. About one-third of these patients saw what was considered a clinical benefit, while two-thirds didn’t. The remissions lasted a median time of 8.3 months, and patients had a median overall survival time of 15.5 months.
These patients weren’t randomly assigned to another treatment option, so it’s impossible to say how much better this drug is compared to another round of currently available therapies. But previous studies suggest that patients this sick-whose disease had only worsened after a median of five rounds of therapy—had a life expectancy of about six to nine months, says David Siegel, a myeloma specialist at Hackensack University Medical Center in New Jersey, and an investigator on the study. He is presenting the data today at the American Society of Hematology meeting in Orlando, FL.
“The patients in this trial were rapidly approaching the end of their lives, and we saw some incredibly deep and durable responses in patients for whom we had no reason to expect that,” says Siegel. “Unabashedly, I love this drug and I can’t wait for it to be approved.”
About 20,000 patients are diagnosed in the U.S. with myeloma each year, and about 10,000 die from it, according to the American Cancer Society.
Onyx’s findings shouldn’t be much of a surprise to investors, since the company reported on the basic overall tumor shrinkage rate back in July. What’s new and meaningful today is how long the remissions lasted, the survival times, and the side effects.
Before going into the clinical details, first a little background. Like Millennium’s $1 billion blockbuster, bortezomib (Velcade), the Onyx drug is 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, you could fight cancer a novel way. The idea was to block the release of certain proteins that cancer cells secrete to grow and resist conventional chemotherapies.The Millennium drug has shown an impressive ability to shrink tumors of multiple myeloma patients, and its wide adoption, along with a different product from Celgene, lenalidomide (Revlimid), is widely credited among doctors with helping make myeloma more like a chronic disease than a near-term death sentence.
The catch, and there is always is one in the pharma business, is in the side effect profile. Most patients on the Millennium drug get
