Amgen goes 6-for-6 in Phase III, leading the race for a new cardio blockbuster

Amgen's ($AMGN) promising cardio drug aced its 6th late-stage study, the company said, beating out statins in patients with a cholesterol-boosting genetic disorder and widening the drugmaker's lead in a potentially lucrative new field.

In its latest Phase III trial, Amgen said evolocumab (AMG 145) met its primary endpoint of significantly lowering LDL cholesterol in 49 patients with homozygous familial hypercholesterolemia, a rare ailment that lifts "bad" cholesterol levels to dangerous highs in adolescents. Evolocumab works by blocking the protein PCSK9 and thus helping the body clear LDL cholesterol from the blood, and Amgen said its latest study was the first to test out the pathway in patients with the disease, which affects about one in a million people.

The top-line success comes on the heels of 5 Phase III wins for evolocumab, and Amgen is working through a total of 14 studies on the drug with eyes on filing an FDA application this year. If everything goes according to plan, the California biotech will be the first PCSK9 player on the market, getting first crack at what could be a $10 billion market and beating out Regeneron ($REGN) and Sanofi's ($SNY) alirocumab and Pfizer's ($PFE) bococizumab (RN-316).

Meanwhile, a dustup over the safety of PCSK9 blockers has led some to dial back their enthusiasm over the new class of drugs. On March 7, Sanofi revealed that the FDA is looking into some potential neurocognitive side effects tied to PCSK9 therapies, and the agency asked alirocumab's developers to keep tabs on the issue through their 12-study Phase III program. Amgen later said it got the same notice from regulators, leading spooked investors to dump shares in all three companies and worry that the FDA might hold out for long-term outcomes data before approving any PCSK9 drugs.

But those concerns have mostly blown over. As Amgen pointed out, fewer than 1% of patients reported cognitive impairment in a year-long study of evolocumab, and regulators have long been aware of such side effects in statins, noting that they tend to clear right up once patients stop taking the drugs. The agency has said it will consider reductions in LDL cholesterol as surrogate endpoints for cardiovascular improvement when considering PCSK9 inhibitors, and analysts don't believe regulators are going to change their tune over what sound like minor cognitive issues.

And so Amgen and its multibillion-dollar R&D shop remain in the lead, plugging along with their top clinical prospect with hopes of marketing a PCSK9 drug between 6 months and a year ahead of their nearest rivals. The company has 8 more late-stage studies to report out as it makes its way to the FDA, and analysts figure evolocumab, if approved, can bring in $1 billion a year by 2018.

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