Topline results from a phase 3 trial evaluating the efficacy of evinacumab (Regeneron) in the treatment of patients with homozygous familial hypercholesterolemia (HoFH) showed that the investigational angiopoietin-like 3 (ANGPTL3) antibody reduced low-density lipoprotein (LDL) cholesterol by 49% compared with placebo.

In the ELIPSE HoFH trial, patients with HoFH (N=65) were randomized to receive evinacumab intravenously (n=43) every 4 weeks or placebo (n=22); patients in the evinacumab treatment group were on the following lipid-lowering therapies: statins (98%), PCSK9 inhibitors (81%), ezetimibe (75%), LDL apheresis (33%), lomitapide (26%). The primary efficacy end point of the study was the percent change in calculated LDL-C from baseline to week 24.

Results showed that at week 24, patients in the evinacumab group had a 47% reduction in LDL-C, compared with a 2% increase in the placebo arm (absolute change in LDL-C from baseline was 132mg/dL; P<.0001). Moreover, 47% of evinacumab-treated patients achieved LDL-C levels of <100mg/dL vs 23% of the placebo group (nominal P=.0203). With regard to safety, the most common treatment-associated adverse events included influenza-like illness and rhinorrhea.

Commenting on the study results, George D. Yancopoulos, MD, PhD, President and Chief Scientific Officer of Regeneron said, “On average, evinacumab reduced patients’ LDL cholesterol in half and was generally well-tolerated in the trial. These results raise the potential that evinacumab may have value for other patients with severe, refractory hypercholesterolemia, where we have a trial ongoing.”

Related Articles

According to the Company, additional data from the trial will be presented at a future medical meeting, with regulatory submission expected sometime in 2020.

Evinacumab is also being evaluated in patients with persistent hypercholesterolemia, despite receiving maximally-tolerated lipid-modifying therapy, as well as in patients with severe hypertriglyceridemia at risk for acute pancreatitis

For more information visit