Survival miss for Amgen's Kyprolis may hurt approval chances in Europe

A Phase III late-stage myeloma miss for Amgen's Kyprolis didn't shock analysts, who said the failure was more or less expected when the company announced trial results Wednesday. But without proof that the med prolongs life in those patients, getting Kyprolis by European regulators could be a tough sell.

In FOCUS, a 315-person trial of patients with an advanced form of multiple myeloma blood cancer, Kyprolis missed its primary endpoint, failing to extend survival compared with standard care. The study also revealed an increase in the incidence of kidney-related adverse events compared with the trial's control arm and the drug's label.

ISI Group analyst Mark Schoenebaum said in a note to investors that he did not believe expectations had been high, noting that "questions have been raised about how the design of the trial might handicap its success." RBC Capital Markets analyst Michael Yee told Reuters the renal side effects were "not a big concern" given that the trial enrolled very sick patients.

But as Reuters notes, Amgen ($AMGN) intended the FOCUS study to show that Kyprolis helped patients live longer--a factor European regulators usually require before green-lighting a med. And even if it does nab an approval, cost watchdogs might come down hard on the therapy without that survival win.

The way Sanford Bernstein analyst Geoffrey Porges sees it, the findings place Kyprolis--acquired in last year's $10.4 billion buyout of Onyx Pharmaceuticals--at "a significant regulatory and competitive disadvantage" to Pomalyst, Celgene's ($CELG) multiple myeloma contender, he wrote in a note (as quoted by the news service).

Amgen, on the other hand, said in a statement that it thinks it has the data it needs to get the drug approved across the pond, thanks to recently announced positive results from a study that looked at Kyprolis in patients with earlier-stage disease.

"While it is unfortunate that the FOCUS study did not meet its primary endpoint of overall survival, we believe the results from the recent positive ASPIRE Phase III clinical trial will be sufficient to support regulatory submissions around the world," Onyx President Pablo Cagnoni said in a statement.

- read Amgen's release
- get more from Reuters

Special Reports: Top 20 orphan drugs by 2018 - Kyprolis | Top 15 Drug Launch Superstars - Kyprolis - Onyx Pharmaceuticals | Pharma's top 10 M&A deals of 2013 - Amgen/Onyx

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