Amgen Prepares Cholesterol Drug for Review After Fifth Study

Jan 31, 2014 4:13 pm ET

(Updates share prices in seventh paragraph.)

Jan. 31 (Bloomberg) -- Amgen Inc. completed a fifth late- stage study of its experimental anti-cholesterol medicine, leading competitors that are trying to bring a new class of therapies to a market estimated to be worth billions.

The new treatments target a protein called PCSK9 that’s associated with high levels of LDL, or “bad,” cholesterol. Amgen’s drug, evolocumab, met the study goals, enabling the company to proceed with an application for approval this year from regulators, Thousand Oaks, California-based Amgen said yesterday in a statement.

That puts Amgen, the world’s largest biotechnology company by revenue, ahead by six months to a year of its closest rival in the drug class, a compound being developed by Sanofi and Regeneron Pharmaceuticals Inc., according to Eric Schmidt, an analyst with Cowen & Co. He estimates Amgen’s drug may target as many as 17 million patients in the U.S., Europe and Japan.

“My race is really getting this to patients as fast as we possibly can,” Sean Harper, head of research and development at Amgen, said in a telephone interview. “It’s a substantial global unmet need.”

Analysts estimate Amgen’s drug could generate more than $1 billion in sales in 2018, and getting to market before competitors may tip the scales in the company’s favor.

Regeneron plans to file for approval in the U.S. in 2015, the Tarrytown, New York-based drugmaker told investors earlier this month at the JPMorgan Chase & Co. health-care conference. Pfizer Inc., Roche Holding AG, Merck & Co., Alnylam Pharmaceuticals Inc. and Eli Lilly & Co. also have PCSK9 inhibitors under development.

Amgen declined 1.6 percent to $118.95 at the close in New York, while Regeneron fell 1.5 percent to $288.59.

New Target

The medicines work differently from statins, another cholesterol-lowering class of drugs that include the world’s former top-seller, Lipitor. The new treatments are designed to help people who can’t tolerate statins or who don’t get their cholesterol under control with older medicines.

“In a way, this is a salvage therapy, being provided to patients already availing themselves to all available therapy and who still have a substantial risk of a cardiovascular event,” Harper said.

The medicines have shown to cut cholesterol in studies, though they haven’t yet completed broader trials to prove they would help people avoid heart attacks or death. The Food and Drug Administration said last year it won’t require companies to finish those studies before filing for approval.

Heart disease is the leading cause of death worldwide, killing more than 600,000 Americans every year, according to the U.S. Centers for Disease Control and Prevention. High levels of bad LDL cholesterol, which can form fatty plaque, raises the risk that the arteries will clog and cause a heart attack.

The data Amgen presented yesterday were in patients with heterozygous familial hypercholesterolemia, an inherited disorder that affects 1 in 300 to 500 people worldwide, according to the company. Amgen said the study met its goals and that further data will be presented at a medical meeting.

--With assistance from Michelle Fay Cortez in Minneapolis and Drew Armstrong in New York. Editors: Andrew Pollack, Ben Livesey