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

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partial tumor shrinkage in a “high-teens” percentage of patients, and a duration of remission of at least four to six months, Coles says. While those measurements are important to the FDA and physicians, the study also looked at other key goals such as how long the drug could keep tumors from spreading (what’s known as progression-free survival) and the gold standard of cancer drug development—overall survival time. Data on progression-free survival and overall survival times isn’t being released today, and will have to come out at a future medical meeting, Coles says.

Even with just a few leading indicators being released today, an important patient advocacy group cheered the result.

“These are noteworthy results in this refractory population,” says Susan Kelley, chief medical officer of the Norwalk, CT-based Multiple Myeloma Research Consortium. The consortium helped enroll more than half the patients in the study.

Even though Onyx doesn’t yet have data from the third and final phase of clinical trials usually required for FDA approval, Coles says it has good reason to take its existing database to U.S. drug regulators for a review later this year. That’s because the FDA has published guidelines that say it will consider reviewing non-randomized studies for certain blood cancers, as long as the treatment is for a patient population that truly doesn’t have other options, and the drug appears to have a good balance of safety and effectiveness, Coles says.

The FDA has lived up to its word recently, granting approval to a couple of other companies in a similar situation, Coles says. Westminster, CO-based Allos Therapeutics (NASDAQ: ALTH) and Cambridge, MA-based Gloucester Pharmaceuticals both won clearance from the FDA to start selling drugs for blood cancers that hadn’t gone through traditional randomized studies, he says.

Still, Onyx needs to run a bigger study to fulfill the sales potential of its treatment for multiple myeloma. The company is currently enrolling patients in a 700-patient trial, called Aspire, which will randomly assign patients to a combination of Celgene’s lenalidomide (Revlimid), low-dose dexamethasone, and Onyx’s carfilzomib, or just the first two drugs alone. This study is designed to enroll patients with an earlier stage of disease who have a better prognosis and therefore ought to have better odds of responding to treatment. A posting on clinicaltrials.gov says that Onyx expects to have data on the primary goal of the study by October 2011, although Coles says he hasn’t provided a timeline for when investors should expect results.

If Onyx can persuade the FDA that this first trial is good enough to earn a place on the market for the sickest of the sick, and the next trial provides compelling proof that the drug is useful at an earlier stage of disease, then this could be a big story for years to come for Onyx. Getting clearance in both patient populations could be enough for carfilzomib to generate $1 billion in peak annual worldwide sales, Coles says.

While it may not be a cure, the results do represent an additional piece of good news for patients with multiple myeloma, who have benefitted immensely from innovative new treatments of the past decade, according to Kelley of the Multiple Myeloma Research Consortium. Millennium and Celgene have set the bar much higher than ever before for multiple myeloma, so Onyx clearly has had its work cut out.

“It’s incredible how much this field has evolved. We hope to see it [multiple myeloma] become more like a chronic disease,” Kelley says.

Onyx plans to discuss the data and business implications on a conference call with analysts this morning at 8 am Eastern/5 am Pacific. To listen to a live webcast, click here.

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