Amgen rolls out top-line Repatha data in another bid for PCSK9 uptake

Repatha pack and injector

Amgen just scored some new data that could help its PCSK9 cholesterol fighter Repatha gain traction with payers and doctors. In a late-stage trial, Repatha proved that it could not only lower LDL cholesterol, but it also affects plaque buildup in the arteries, a key cause of cardiovascular disease.

Detailed results from the GLAGOV trial will be presented in November at the American Heart Association’s annual scientific meeting. For now, the California-based biotech could only say that Repatha met its primary and secondary goals in the study, which used imaging to monitor atherosclerotic buildup in the blood vessels of the heart.

Amgen needs all the help it can get to persuade payers to cover Repatha, one of two PCSK9 drugs that hit the market last year to great fanfare. The drugs, list priced at more than $14,000 per year, were expected to quickly capture sales because of their ability to lower bad cholesterol dramatically.

But payers have raised prior authorization hurdles to limit uptake of the meds, which also include Sanofi and Regeneron’s Praluent, and physicians have been slow to adopt them as well. So far, the drugs’ sales have disappointed the companies--and investors--at €33 million for the first half of the year for Praluent, and $43 million for Repatha.

Proof that the drugs not only lower LDL cholesterol, but also improve clinical outcomes, is key to more widespread adoption. Last month, Sanofi and Regeneron unveiled data showing that, for patients with extremely high cholesterol, Praluent therapy helped reduce pricey treatments that siphon cholesterol out of their blood.

Now, Amgen has its own salvo, thanks to the imaging study top-lined today. Atherosclerosis is “the major underlying cause” of cardiovascular disease, which is still a leading cause of death worldwide, the company noted Tuesday. If Repatha has proven helpful at dealing with plaque, then payers and doctors have one less reason to resist it.

“[O]ne year after the FDA approved Repatha, nearly two-thirds of patients prescribed Repatha are still being denied access,” Amgen R&D chief Sean Harper said in a statement. “We are concerned that many patients with uncontrolled LDL cholesterol levels continue to face challenges in accessing a medicine that we now know has a positive impact on plaque burden.”

Repatha and Praluent are both in outcomes studies to test whether they have a significant effect on cardiovascular complications such as heart attack and stroke, and the first hints from those studies are expected early next year with top-line results from Amgen’s FOURIER study.

Amgen hopes the data can then be presented at the American College of Cardiology meeting in March. First results from the Praluent outcomes trial, ODYSSEY, are not expected till the end of 2017.

To make a big difference with doctors and insurers, the outcomes data will need to be strong--really strong, analysts report. To change payers' stance and to really boost adoption of the drugs, they'll need to see "substantial benefit in the outcomes trials, including overall survival," Leerink Partners told investors earlier this year. What's substantial? "[C]loser to 50% than 25%," Leerink's Geoffrey Porges quoted some payers as saying.

Regeneron CEO Len Schleifer, for one, thinks that’s possible. The companies "powered their study to detect what they consider to be a clinically meaningful composite risk reduction of 15%," Porges has said, citing comments at Leerink’s investor conference. Schleifer "believes that a much greater effect size is likely, and anticipates the outcomes improvement will be proportional to the absolute LDL reduction.”

Meanwhile, Pfizer is getting closer to market with its own PCSK9 competitor. By the time the Repatha and Praluent outcomes data hit, Pfizer's bococizumab could make its debut. And Pfizer has planned all along to bring bococizumab to market along with cardiovascular outcomes data. By Pfizer’s rough estimate, given that it’s a CV events-driven trial, we could see those results during the second half of next year.

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