Pricing, Results

Heart attack while on Repatha? Amgen will give you your money back

Washington, DC —Amgen has overcome one of the toughest challenges in cardio research, outlining a clear health benefit for its PCSK9 cholesterol drug Repatha. But before payers get a chance to gnaw at the numbers being put on display today, looking for holes or finding fault with the level of benefit, the pharma giant wants to send them a simple message: it’s ready to offer a negotiated truce to get them to drop their defenses and pull out of the trenches.

After finding access to Repatha blocked and locked by blanket payer rejections, corralling this drug far from the mainstream, Amgen $AMGN wants to parlay.

If payers will just consider the benefits Amgen is demonstrating today and take down their elaborate barriers, company execs say they are not only willing to offer a refund when the drug fails, they’ll also negotiate annual payment caps and risk sharing deals with insurers so payers can be sure to keep control of their budgets.

Sean Harper, EVP Research and Development

Drug refunds have begun to make their way into the system, but they’re still rare enough to make this a standout proposal for a manufacturer trying to establish a big drug.

“We will work with payers if they’re willing to just stop blocking patients from getting the drug,” Sean Harper, head of R&D at Amgen, tells me.

Harper and his team showed up at American College of Cardiology conference in Washington DC on Friday to lay their case out with some pioneering data. Studying this drug in 27,564 patients over two years, researchers say, Repatha was able to lower a composite of cardio risks by an average of 20%. And the improvement increased with time, growing from a 16% risk advantage in year one to 25% after 12 months.

That’s large enough to hit key endpoints, but it’s also small enough to leave many analysts wondering if this drug can ever make it to big blockbuster status, which Amgen is certain it deserves.

Breaking down the data, which everyone will do, researchers noted a 27% reduction in the risk of heart attacks, a 21% risk reduction for stroke and a 22% reduction on coronary revascularization. But significantly there was no impact on mortality or unstable angina.

This was the first time that a trial has shown a clear cardio benefit from PCSK9 inhibition.

Sums up Harper: “This is huge that we can do this.”

Investors, though, were clearly not impressed. Amgen’s shares were down 6% after the headlines hit. And this Tweet should give you a flavor for what’s driving the skepticism:

Umer Raffat at EvercoreISI noted that most of investors were looking for a mortality benefit and were clearly disappointed in not seeing one. Amgen’s bullishness on the data ahead of the details also inflated expectations. And the same bitter taste also extended to the rest of the players in the field, including Regeneron $REGN and Sanofi $SNY, which both got hit, as well as The Medicines Company {$MDCO -20%} which has a next-gen therapy in the clinic it’s been seeing success with.

Baird’s Brian Skorney called it a let down. He added:

The primary endpoint just hit stat sig with a HR of 0.85 (below expectations), while the MACE secondary endpoint saw a 20% risk reduction (in-line). Even more disappointing, CV death failed to separate from placebo. Though we do think that today’s data is evidence that PCSK9 inhibition works, the benefit isn’t as profound as investors were looking for and payers are likely to leverage this disappointment.

Amgen has bet big on Repatha, bankrolling several huge studies to demonstrate the drug’s ability to dramatically slash LDL levels, which its investigators say is clearly a toxin. Now it has a win on cardio outcomes to make its point to physicians and a large potential patient population. But the trial success is not big enough by itself to get payers to stop rejecting prescriptions “over and over and over again,” as Harper describes the treatment to date.

Joshua J. Ofman, Amgen

“The process on utilization management has been broken,” says Amgen value management chief Joshua J. Ofman. Ever since 2013’s introduction of Sovaldi payers have been upping their game, figuring out new ways to stop their members from getting access to new drugs like this. And in Repatha’s case, payers are in overdrive, forcing physicians to go back and file over and over again.

The numbers Amgen is reporting today are also being pitched as far more conservative than what patients see in the real world. All the patients in the study got the best standard of care, which is something you won’t see in the real world. And Amgen is happy to negotiate its next round of contracts based on real-world expectations.

So now Amgen says they’ll guarantee a lower risk of heart attack and refund the cost of the drug for any patient who has a heart attack. They’ll still come out well ahead if they can reach the at-risk population whose LDL levels cannot be controlled by cheap statins.

Amgen’s full court press on Repatha has included a coordinated attack on its big rival Praluent from Regeneron and Sanofi. At one point recently a judge’s ruling threatened to push the competing drug off the market as Amgen — a major league litigator — pressed its argument that Praluent violated its PCSK9 patents. But the partners managed to block that, for now.

Next up: Cardio data from Regeneron and Sanofi. So far, these drugs have appeared very similar, and it wouldn’t surprise many observers if their competitor comes on around the same score. But every data point will get careful scrutiny as the giants block and tackle in search of every small advantage, while hoping to achieve a breakthrough on marketing that can finally generate some big numbers.

So far, it’s not looking good.


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