Amgen preps for swift FDA review as Kyprolis/Revlimid combo stymies myeloma

Amgen CEO Robert Bradway

Amgen ($AMGN) put out the word Monday morning that Kyprolis--provided in a combo package with Celgene's ($CELG) Revlimid--beat out Revlimid alone in a head-to-head Phase III study for multiple myeloma, hitting the primary endpoint and offering the Big Biotech a shot at expanding its franchise for the drug at a critical moment in the company's history.

The headline numbers included a 26.3-month median average for disease-free progression for the combination compared to 17.6 months for Revlimid. That may be good enough for an accelerated approval for the drug for an expanded patient population, according to Amgen. The full slate of data will be reviewed at ASH later in the year.

Amgen shares spiked about 4% in premarket trading.

There was a big hitch, though. Investigators say that while data for a key secondary endpoint on overall survival had not fully matured, the trend favored the Kyprolis/Revlimid combo but did not achieve statistical significance, leaving no clear evidence of a survival benefit.

Amgen picked up Kyprolis (carfilzomib) in its $10.4 billion buyout of Onyx, setting course for an expanded approval as it pursued a slate of new studies and looked to build its new oncology franchise. That's a key part of the company's late-stage pipeline effort, which includes a pair of recent applications and now a major retrenchment in R&D as Amgen shutters its R&D hub in Seattle and cuts up to 2,900 staffers.

Amgen needs Celgene's Revlimid to beat another Celgene drug--Pomalyst, which has been gobbling up market share for hard-to-treat patients running out of options.

"Coupled with our recent U.S. regulatory submissions for ivabradine and talimogene laherparepvec and our upcoming regulatory submissions for evolocumab and blinatumomab, our pipeline continues to show notable progress," boasted CEO Robert Bradway in a statement.

Amgen is heavily focused on its late-stage efforts right now, which has been forcing up R&D costs as the biotech absorbed Onyx's research operations. When Amgen bought Onyx about a year ago, Kyprolis had been approved as a third-line therapy for patients who had already failed two treatments. Expanding on that approval is a key strategy of Amgen's. And success in this study marks another step down a path that investigator say could one day make myeloma into a disease that can be managed indefinitely.

"In the treatment of patients with multiple myeloma, periods of remission become shorter following each treatment regimen, underscoring the need for new options. The results of the ASPIRE study demonstrate that Kyprolis can significantly extend the time patients live without their disease progressing," said Dr. Pablo Cagnoni, president of Onyx Pharmaceuticals. "The ability of novel therapies to produce deep and durable responses may, one day, transform this uniformly fatal disease to one that is chronic and manageable."

- here's the release

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