By Bill Berkrot and Ransdell Pierson
DALLAS, Nov 19 (Reuters) - Amgen Inc's experimentalheart medicine from a closely watched new class of drugs calledPCSK9 inhibitors lowered "bad" LDL cholesterol 52 percent afterone year with no major increase in serious adverse side effectscompared with standard drugs, such as statins, according to datafrom a study.
The clinical trial of the drug, evolocumab, marks the firstdata looking at 52 weeks of use for the new class of injectablebiotech medicines seen as potentially the biggest advance in thefield of cholesterol therapy in many years.
Several companies are developing the drugs, which work byblocking a protein that prevents the liver from removing LDLcholesterol from blood. In addition to Amgen, Pfizer Inc and Regeneron Pharmaceuticals Inc in partnership withSanofi are in advanced stages of testing PCSK9inhibitors.
They are most likely to be used in patients who cannottolerate statins or those whose LDL cholesterol remains too higheven after being treated with high potency statins.
"The results from the Osler study are encouraging asevolocumab may offer a potential treatment option for patientswho cannot control their cholesterol levels," said Dr. MichaelKoren, the study's lead investigator, who presented the data onTuesday at the American Heart Association (AHA) scientificmeeting in Dallas.
The 1,104-patient trial tested 420 milligrams of evolocumabinjected once a month in addition to standard therapy withstatins or other medicines versus standard of care drugs alone.
After 52 weeks, those who took the Amgen drug saw their LDLlevels drop 52 percent on top of statins, which was consideredby researchers to be highly statistically significant.
Levels of other blood lipids also moved in the beneficialdirection, researchers said. Triglycerides went down about 9percent and "good" HDL went up about 9 percent.
Patients in the Osler extension study had previouslycompleted participation in one of four 12-week Phase II studiesof the drug.
Serious adverse side effects occurred in 7.1 percent ofpatients treated with evolocumab and standard care compared with6.3 percent for those who got standard therapy alone.
Side effects such as elevated liver enzymes were similar -1.8 percent for evolocumab versus 1.6 percent in the controlgroup - and a measure of potential kidney problems was 1 percentcompared with 1.9 percent.
The serious side effects were not felt to be related toevolocumab, said Koren, of the Jacksonville Center for ClinicalResearch in Florida. He added that adverse heart events, such asheart attacks, were slightly higher in the standard of carepatients.
"It did wonderfully," Koren said of the Amgen drug, "Thisgives us a much greater comfort level about possible safetysignals."
That may play a pivotal role in whether drugs from thisclass can be approved without long-term, "outcomes" studies thatdefinitively show whether they can reduce the risk of heartattacks, strokes and deaths.
New cholesterol management guidelines released last week bythe AHA and American College of Cardiology only backed therapywith medicines that had demonstrated a reduction in heart risk,such as statins.
That called into question whether the U.S. Food and DrugAdministration would still be willing to approve drugs based onso-called surrogate end points, such as lowering LDL levels.
An FDA official last week said the agency might be willingto approve the PCSK9 drugs without outcomes data if they did notshow any unexpected toxicities.
"I know with fairly good confidence that The FDA believes inLDL," Koren said.
"There's tremendous excitement," he said of the new class ofdrugs. "This is the biggest thing to happen to lipid treatmentsince statins."
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