|Amgen R&D Chief Sean Harper|
Amgen ($AMGN) is batting 1.000 in its ambitious clinical program for the promising PCSK9 cholesterol drug evolocumab (AMG 145), unveiling a 5th successful Phase III study as it prepares for an FDA application that could give it first-mover status in a blockbuster field.
In its latest top-line readout, evolocumab met its co-primary endpoints of lowering baseline and mean LDL cholesterol rates over 12 weeks in patients with heterozygous familial hypercholesterolemia, a common genetic disorder affecting one out of every 300 to 500 people, Amgen said. The company is keeping exact results under wraps but said the latest trial fell in line with an earlier Phase II study in which the drug lowered "bad" cholesterol by as much as 56% in the same patient population.
Now, with 5 of evolocumab's planned 13 Phase III studies in the books, Amgen is on pace to file for approval this year, Bloomberg reports, and if all goes according to plan, the drug could hit the market between 6 months and one year before its closest competitor, Regeneron ($REGN) and Sanofi's ($SNY) alirocumab. Those two are pursuing a 12-study effort of their own with plans for an FDA application in 2015.
A swift approval for evolocumab would put Amgen in pole position in a market analysts say could top out at $10 billion a year, and many figure the drug will hit the $1 billion mark on its own by 2018. Unsurprisingly, evolocumab is the top late-stage priority at Amgen's multibillion-dollar R&D unit, as research chief Sean Harper bears down on what looks like a surefire clinical success.
"My race is really getting this to patients as fast as we possibly can," Harper told Bloomberg. "It's a substantial global unmet need."
But the fate of every PCSK9 hopeful is dependent on a warm reception from physicians and payers, which, thanks to a fair amount of skepticism over patient outcomes, isn't guaranteed. While the FDA accepts reductions in LDL cholesterol and blood pressure as surrogate endpoints for long-term cardiovascular benefit, the powers that be in reimbursement and prescription may choose to hold out for data from time-consuming outcomes studies before embracing PCSK9 blockers over tried, true and cheap statins.
- read the release
- check out Bloomberg's story