By Ransdell Pierson
Oct 16 A new type of cholesterol drug being
developed by Regeneron Pharmaceuticals Inc and Sanofi
SA, when used by itself, cut levels of "bad" LDL
cholesterol almost in half in the first of a dozen late-stage
trials of the injectable medicine.
The experimental drug, called alirocumab, is from a
promising new class of injectable cholesterol fighters also
being developed by Amgen Inc and other drugmakers. They
work by blocking a naturally occurring protein called PCSK9 and
could each garner annual sales of $3 billion or more, if
approved, according to some industry analysts.
In earlier mid-stage studies, when combined with standard
oral cholesterol drugs called statins such as Pfizer Inc's
Lipitor, alirocumab and Amgen's AMG145 slashed levels of
LDL cholesterol by close to 70 percent more than statins alone.
But the 103-patient Phase III study described on Wednesday
involved patients who took alirocumab by itself, or who took
only Merck & Co Inc's cholesterol fighter Zetia
(ezetimibe) - a pill often used by patients who cannot tolerate
Although patients enrolled in the Regeneron/Sanofi trial had
high cholesterol levels, they were deemed to have only moderate
cardiovascular risk due to the absence of many other risk
Regeneron and Sanofi said LDL cholesterol levels fell 47
percent in those who gave themselves injections of low-dose
alirocumab every two weeks over a 24-week period. Cholesterol
levels fell 15.6 percent in those taking daily 10-milligram
doses of Zetia over the same period.
George Yancopoulos, Regeneron's research chief, said no
worrisome side effects were seen in the study and in earlier
trials of alirocumab.
"What we're seeing in first Phase III trial of our drug is
in line with what we saw in Phase II" trials, Yancopoulos said
in an interview. "The good news here is there were no surprises,
and that it supports the good efficacy and safety profile we've
seen to date."
In the study, called Odyssey Mono, patients taking
alirocumab started out with a low 75-milligram dose every two
weeks. But their doses were increased to 150 milligrams at week
12 if their LDL levels at week eight were above 70.
About three-fourths of the patients remained on the low dose
of alirocumab throughout the study because they got their LDL
levels down to below 70 - an aggressive LDL target - by the
eighth week of the study.
"It shows that a low dose, when used as a monotherapy, can
be quite effective," Yancopoulos said.
Yancopoulos said Regeneron and Sanofi hope to seek
regulatory approvals by late 2015 for their drug, for use by
itself and with statins, after data from the remaining 11
late-stage trials of alirocumab are released. An estimated
23,000 patients are being studied in the various Phase III
The Regeneron research chief said Amgen's rival drug might
reach market soon after alirocumab, or even before. But he
predicted neither drug would have much of an advantage by
getting approved first.
"But we're hoping to have bragging rights for the first
approval," Yancopoulos said.
Regeneron and Sanofi are hoping their new medicine will be a
compelling treatment option for the estimated 1 million
Americans who cannot tolerate statins and up to another 5
million Americans who have failed to drive down their
cholesterol levels to desired levels with statins.
Analysts believe strong demand from outside the United
States could also propel blockbuster sales of alirocumab and
At an investor conference in September, Sanofi's CEO Chris
Viehbacher said the French drugmaker could look at nearly
doubling its stake in Regeneron, voicing confidence in the
drug's success. Sanofi holds about 16 percent of Regeneron.
Deutsche Bank analysts said in a report last month that the
new drug could be priced at around $15 per day, comparable to
the cost of injectable diabetes drugs known as GLP-1s, such as
Novo Nordisk's Victoza.