EU watchdogs demand info on AstraZeneca's disputed Brilinta trial

Last week, AstraZeneca ($AZN) succinctly disclosed that Department of Justice investigators are digging into its data on the blood thinner Brilinta. Now, European regulators are adding their shovels to the effort. If they're not satisfied with their findings, AstraZeneca's hopes for pumping up Brilinta sales could be in vain.

At issue is the PLATO trial, a pivotal study that helped lay the foundation for Brilinta's regulatory approval. The head-to-head trial against megablockbuster clot-fighter Plavix helped support a superiority claim on Brilinta's FDA label when its was approved in 2011. That gave AstraZeneca some extra marketing ammo. And that was important, given the fact that the Sanofi ($SNY) and Bristol-Myers Squibb ($BMY) drug--the standard of care--was about to go off patent. The study also gave AstraZeneca some pricing support; in setting a premium price for the drug in Europe, the company cited PLATO data as justification.

But how justified was it, really? The European Medicines Agency (EMA) said yesterday that it asked AstraZeneca for more information after the Justice Department's probe came to light. "The EMA takes seriously any information that may have an impact on the profile of a drug's benefits and risks," an EMA spokeswoman told Reuters. "At the moment we are at the point of trying to understand what is going on."

PLATO has come under fire before. Researchers have pointed to flaws in the study, with some suggesting that the data appeared to be fudged to benefit Brilinta. Last summer, a report in the International Journal of Cardiology said an independent group's review of PLATO data yielded a more negative picture than AstraZeneca's analysis.

The researchers also noted that almost half of the favorable results for Brilinta came from just two Eastern European countries, Hungary and Poland. Plus, some unfavorable outcomes in the Brilinta arm appeared to have been set aside or soft-pedaled, they said. If regulators determine that the study included rigged data, the consequences could be dire, Bernstein analyst Tim Anderson said earlier this week.

In fact, the probe could end up forcing the drug off the market, Anderson figures. So far, that threat is low-risk, he emphasizes. But with consensus models projecting $1.6 billion in sales for Brilinta--a drug with a 70% margin--the doomsday scenario would claw out 15% of the company's expected profits.

The news comes at an awkward time for AstraZeneca and CEO Pascal Soriot, who has singled out the drug for a marketing push. Brilinta has never really gained hold in the marketplace, but Soriot has said he sees that as a problem in execution rather than a problem with the drug itself. The company has rolled out new consumer advertising and beefed up its sales force to support Brilinta, and last week Soriot said he expects the sales to really start rolling in next quarter. Already, sales have grown to $75 million for the third quarter of this year, compared with $24 million last year.

Soriot said last week that he's confident in the PLATO data, citing the "strong academic group" involved in the trial. The PLATO researchers themselves have fired back at their critics. It's likely to take some time for the DoJ and EMA to come to any conclusions. Till then, Soriot says, the Brilinta marketing program won't be hindered by the probes.

- see the Reuters news

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