By Bill Berkrot
Jan 23 A cholesterol fighter developed by Amgen
Inc from a highly promising new class of medicines
significantly lowered levels of "bad" LDL cholesterol in a late
stage trial in patients unable to tolerate statin drugs, the
company said on Thursday.
The results marked the third successful Phase III test of
the drug evolocumab reported by Amgen in recent months - this
one in a patient population among the most in need of
An estimated five to 20 percent of heart patients are
intolerant of statin medications for lowering cholesterol due to
side effects such as muscle weakness or fatigue, Amgen said.
Evolocumab belongs to a class of medicine called PCSK9
inhibitors that work by blocking a protein that reduces the
liver's ability to remove LDL cholesterol from the blood.
Based on dramatic LDL lowering demonstrated by Amgen and
other companies in earlier studies, PCSK9 inhibitors could be
the most important new heart drugs to come along in several
years with multibillion-dollar sales potential.
The injected medicines are likely to be used in high risk
heart patients unable to lower their LDL levels sufficiently
with high doses of widely used statin drugs, such as Pfizer's
Lipitor, and in those unable to take statins.
Many patients remain at high risk of heart attack and stroke
"despite the use of all available therapies," Amgen research and
development chief Sean Harper said in a telephone interview.
"The ability to treat those individuals with a completely
distinct mechanism and see this very large effect size of
dropping LDL cholesterol by roughly 50 percent, that's a pretty
big deal for those patients," Harper said.
The company expects during the current quarter to have
results of its final two Phase III studies - one in patients
already taking high doses of statins and one in patients
genetically predisposed to dangerously high cholesterol levels.
Amgen will provide details of its latest 307-patient Phase
III study, called GAUSS-2, which compared evolocumab to Merck &
Co's Zetia (ezetimibe), at an upcoming medical meeting.
However, the world's largest biotech company said the
percentage of cholesterol lowering was consistent with those
observed in a similar, smaller Phase II trial. In that study,
evolocumab led to 51 percent reduction in LDL levels and 63
percent when combined with Zetia versus a 15 percent reduction
seen with Zetia alone.
"It's very unusual to have a therapeutic that addresses one
of the underlying factors that drive the greatest mortality risk
that exists in Western countries, and increasingly in developing
countries - cardiovascular disease driven by atherosclerosis,"
The company anticipates that some of its global filings
seeking approval of the drug will occur this year.
"We don't come across therapeutics like this very often in
this industry," Harper said. "It is very exciting, and as we get
each data set our confidence slowly builds that we have the
efficacy and the safety profile that will make this an important
Regeneron Pharmaceuticals Inc, in partnership with
Sanofi, is also in Phase III testing of a rival drug.
Pfizer is developing its own PCSK9 inhibitor.
Amgen shares were down about 0.6 percent at $123.55 on
Nasdaq, roughly in line with declines in the broader market.