Amgen's PCSK9 drug hits PhIII mark in the race for a new cardio blockbuster

Amgen's ($AMGN) PCSK9-blocking evolocumab hit its primary endpoints in a Phase III study, the company said, bringing down bad cholesterol and stirring fresh hope for the company as it races with Pfizer ($PFE), Sanofi ($SNY) and others to cash in with a new approach to cardiovascular treatment.

In a study of 614 patients taking no other drugs, the former AMG 145 lowered average low-density lipoprotein (LDL) cholesterol at a rate consistent with its mid-stage data, Amgen said. That data, from a 12-week Phase II study, found that patients getting the highest doses of evolocumab charted an average 51% reduction in bad cholesterol on its own. For comparison, Pfizer's competing RN316 cut LDL cholesterol by a mean of 56% at its top dosage after 12 weeks, and Sanofi and Regeneron's ($REGN) alirocumab brought it down 47% on average after 24 weeks.

Amgen is keeping detailed results under wraps until a later medical meeting, but the top-line data put the Thousand Oaks, CA, company right in line with its competitors. Amgen is in the midst of a 13-trial Phase III program to prove evolocumab can safely and effectively bring down bad cholesterol, all while Sanofi and Regeneron plow through a 12-study effort and Pfizer prepares to spend big on a 22,000-patient outcomes program.

At stake is pole position in what the industry's most optimistic analysts say could be a $10 billion market, and while Sanofi and Regeneron would appear to lead the way, a Phase III surprise from any of the competitors could easily change the game.

The whole field of PCSK9 developers got some welcome news last month when the FDA announced it would continue to accept reductions in LDL cholesterol and blood pressure as surrogate endpoints for long-term benefit, dashing fears that each company would need to supply sweeping--and costly--outcomes data to prove its drug improved cardiovascular health.

However, what works for the agency may not sway payers who would prefer to shell out for cheap statins than newfangled monoclonal antibodies. The sky-high projections for the novel treatments depend on a blockbuster reception, but the industry's gatekeepers might hold out for long-term results before letting PCSK9 developers reap their rewards.

The latest release is Amgen's first Phase III readout for the drug, which works by inhibiting a protein that reduces the liver's ability to remove bad cholesterol from the blood. In the trial, the most common side effects were headache, diarrhea, nausea and urinary tract infection, the company said.

- read the statement

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