Amgen reported positive top-line results from the Phase III trial assessment of evolocumab. The drug met its co-primary endpoints in statin intolerant patients with high cholesterol.
The company is developing evolocumab as an investigational fully human monoclonal antibody which works to inhibit proprotein convertase subtilisin/kexin type 9 (PCSK9). PCSK9 is a protein that makes it difficult for the liver to remove low-density lipoprotein cholesterol (LDL-C) from the blood.
Over 71 million adults in the US have high LDL-C, according to the Centers for Disease Control and Prevention (CDC). Elevated LDL-C is a known major risk factor for cardiovascular disease and is usually treated with statins. However, 5% to 15% of patients are unable to tolerate statins because of muscle-related side effects.
Evolocumab is designed to bind to and inhibit PCSK9 from forming bonds with LDL receptors on the surface of the liver. Without PCSK9, more LDL receptors are available on the liver surface to remove LDL-C from the blood.
Evolocumab underwent the Phase III of GAUSS-2 (Goal Achievement After Utilizing an Anti-PCSK9 Antibody in Statin Intolerant Subjects-2) trial and showed results consistent with those observed in Phase II of the GAUSS study. The drug achieved percent reduction from baseline in LDL-C at week 12 and mean from baseline at weeks 10 and 12. 307 patients participated in the study. Patients with high cholesterol and unable to tolerate effective doses of at least two statins due to muscle-related side effects were particularly targeted.
Amgen R&D executive vice president Sean E. Harper, M.D., said, “"We are pleased to continue to see promising results from the Phase III studies in our comprehensive development program for evolocumab… The positive GAUSS-2 results suggest that evolocumab may offer a new lipid-lowering treatment to meet an important medical need for high-risk patients who cannot tolerate effective doses of statins."