TARRYTOWN, N.Y., Aug. 19, 2020 /PRNewswire/ —

Patients with homozygous familial hypercholesterolemia (HoFH) suffer from a severe form of early cardiovascular disease and are inadequately served by currently available medications

Adding evinacumab to other lipid-lowering therapies cut bad cholesterol levels in half in patients with HoFH, including for the most difficult-to-treat patients who had nearly non-existent LDL-receptor activity

Evinacumab is currently under Priority Review with the FDA; decision expected by February 11, 2021

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the New England Journal of Medicine (NEJM) published positive results from the Phase 3 trial of evinacumab in 65 patients with homozygous familial hypercholesterolemia (HoFH). Evinacumab is an investigational medicine that binds to and blocks the function of angiopoietin-like 3 (ANGPTL3), and is the first medicine of its kind to show efficacy in patients with HoFH – including patients with little to no low-density lipoprotein (LDL) receptor function.

Patients with HoFH have severely elevated levels of bad cholesterol (low-density lipoprotein cholesterol, or LDL-C), which increases their risk for premature atherosclerotic disease and cardiac events as early as their teenage years. Treatment guidelines recommend early and intensive LDL-C lowering, but patients with HoFH are less responsive (or unresponsive) to standard lipid-lowering therapies, including statins and PCSK9 (proprotein convertase subtilisin/kexin type 9) inhibitors.

As initially announced in a topline press release, the trial met its primary endpoint, showing that patients who added evinacumab to other lipid-lowering therapies (n=43) reduced their LDL-C from baseline by 49% compared to lipid-lowering therapies alone (placebo, n=22) at week 24 (47% reduction evinacumab, 2% increase placebo, p<0.0001). At the same time point, evinacumab-treated patients also decreased LDL-C from baseline by 132 mg/dL compared to placebo (135 mg/dL reduction evinacumab, 3 mg/dL reduction placebo, p<0.0001). As discussed in the NEJM publication, genetic loss of ANGPTL3 has been associated with additional lipid-lowering effects, including lowered triglycerides, apolipoprotein B (ApoB), HDL and non-HDL cholesterol, and total cholesterol. Evinacumab treatment mirrored these lipid-lowering effects.