DOJ clears AstraZeneca for a U.S. Brilinta takeoff

Prosecutors end clinical trial probe with a whimper, planning no action against AZ

August 19, 2014 | By Tracy Staton

AstraZeneca CEO Pascal Soriot is now living in the best of all possible worlds, at least where Brilinta's U.S. marketing is concerned. The Department of Justice closed out its probe of a key Brilinta trial--and without further ado. No additional label warnings. And certainly no forced withdrawal from the U.S. market.

That means AstraZeneca ($AZN) can get back to the business of turning the underperforming blood thinner into a blockbuster-to-be. Brilinta, you'll recall, is one of Soriot's pet projects--and one of the company's 5 "growth platforms" for rebuilding sales as patents expire.

"[W]e are committed to delivering the full potential of this important medicine," Soriot said in a Tuesday statement.

The DOJ probe came at a terrible time for Soriot: AstraZeneca had just ramped up its Brilinta marketing efforts. And it saw some results. "We felt we were moving in the right direction," Soriot said earlier this year. "We saw some nice feedback, some nice movement, until the DOJ investigation impacted us."

For safety's sake, the company stopped its promotions while the DOJ did its investigatory work. That meant Soriot had to mothball the Brilinta sales charts he'd been trotting out to show its potential in the U.S., where its market share is weakest.

Now, the hundreds of U.S. salespeople who'd joined the Brilinta field force can rev up their promo engines again. And as AstraZeneca's Tom Keith-Roach tells Reuters, doctors may now be more receptive to their case. "I think physicians, particularly in the U.S., will now see that a line has been drawn underneath any potential controversy surrounding this trial,"said Keith-Roach, who heads up Brilinta commercial efforts.

The Justice Department had been investigating allegations of data-tampering in the PLATO trial, a keystone of Brilinta's FDA approval app. The 18,000-patient study pitted Brilinta (ticagrelor) against Plavix (clopidogrel), marketed by Sanofi ($SNY) and Bristol-Myers Squibb ($BMY)--and now off patent. Overall, a Brilinta-aspirin combo beat Plavix plus aspirin at preventing heart attack, stroke or death.

In the U.S., however, the results weren't so good. FDA staffers put that down to the high-dose aspirin used by some U.S. Brilinta patients, which could have interfered with Brilinta's work. Later, scientists questioned the PLATO data for other reasons, saying that patients monitored by the company showed more benefits from taking Brilinta than did those monitored by independent researchers. They also pointed out that the trial patient population was skewed toward Eastern Europe.

The Justice Department decided to sift the evidence for itself, launching a probe last October. Nine months later, Soriot announced the result with PR-language gusto: "We have always had absolute confidence in the integrity of the trial and we are proud of the important benefit Brilinta offers to patients around the world suffering from acute coronary syndrome," he said in the statement.

When the DOJ stepped in to probe the trial, analysts expected catastrophe--or at least calamity. AstraZeneca might have to add warnings to the drug's label, at a time when it was just starting to gain new traction in the U.S. It might even have to pull the drug off the market completely, undermining Soriot's turnaround efforts.

Contrary to that worst-case scenario is this best-case result. Now, the Brilinta force has to prove itself. With $3.5 billion in sales as a target, they have a tough road ahead. The drug brought in $283 million in 2013 sales worldwide, and as Soriot said earlier this year, Brilinta's long-term success hinges on "an acceleration in the U.S." Stay tuned.