Evinacumab, which is a human monoclonal antibody inhibitor of angiopoietin-like 3 (ANGPTL3), appears to have a dramatic effect on low-density lipoprotein (LDL) cholesterol levels in patients with homozygous familial hypercholesterolemia (HoFH) with little or no LDL receptor activity. The phase 3 ELIPSE trial showed that evinacumab reduced LDL cholesterol levels in HoFH patients by an average of 47%.
On October 5, the results of the post-hoc analysis of this study were presented at the European Atherosclerosis Society 2020. The analysis included 10 patients with less than 2% functional LDL receptor activity. The average age of the patients was 34 years. At 24 weeks, evinacumab was associated with a 53.5%% reduction in LDL cholesterol levels over baseline. The patients who received placebo experienced an 18.8% rise over baseline, yielding a reduction of 72.3% for evinacumab vs placebo (P = .005).
The authors note that the study did not reveal any serious side effects of the drug. However, the main limitation of this study is a short-term observation period therefore, it is premature to talk about the safety of long-term therapy with evinacumab.
