Positive Results Reported From Late-Stage Trial of Evolocumab in Patients With Familial Hypercholesterolemia
Study meets co-primary endpoints of LDL cholesterol reduction (January 31)
The phase III RUTHERFORD-2 (RedUction of LDL-C with PCSK9 InhibiTion in HEteRozygous Familial HyperchOlesteRolemia Disorder Study-2) trial evaluating evolocumab (Amgen) in combination with statins and other lipid-lowering therapies in patients with heterozygous familial hypercholesterolemia (HeFH) has met its co-primary endpoints: the percent reduction from baseline in low-density lipoprotein cholesterol (LDL-C) at week 12, and the mean percent reduction from baseline in LDL-C at weeks 10 and 12.
The mean percent reductions in LDL-C, or “bad” cholesterol, were consistent with the results observed for the same doses in the phase II RUTHERFORD trial of evolocumab compared with placebo.
Evolocumab is an investigational fully human monoclonal antibody that inhibits proprotein convertase subtilisin/kexin type 9 (PCSK9), a protein that reduces the liver’s ability to remove LDL-C from the blood.
The RUTHERFORD-2 trial evaluated the safety, tolerability, and efficacy of evolocumab in 329 HeFH patients on a stable dose of statin and other lipid-lowering therapies. The patients were randomly assigned to one of four treatment groups to compare subcutaneous evolocumab (140 mg every 2 weeks or 420 mg monthly) with subcutaneous placebo (every 2 weeks or monthly).
The most common adverse events included nasopharyngitis (8.6% with evolocumab vs. 4.6% with placebo), contusion (4.1% vs. 0.9%), back pain (3.6% vs. 0.9%), nausea (3.6% vs. 0.9%), influenza (3.2% vs. 0%), and myalgia (2.7% vs. 0%).
Source: Amgen; January 31, 2014.