Regeneron Presents Favorable Interim Data From Stage 2 Proof-Of-Concept Trial Of Evinacumab

Regeneron Pharmaceuticals, Inc. REGN revealed that positive preliminary results from an ongoing proof-of-concept trial of evinacumab (REGN1500) in patients with Homozygous Familial Hypercholestrolemia (HoFH) were presented at the 84th European Atherosclerosis Society (EAS) Congress in Innsbruck, Austria. According to the company, the interim data from the first four patients treated in this trial demonstrated that evinacumab added to current lipid-lowering therapy reduced low-density lipoprotein cholesterol (LDL-C) levels by an additional 55 percent at week 4 compared to baseline.

Regeneron Pharmaceuticals said Evinacumab was an investigational monoclonal antibody to angiopoietin-like protein 3 (ANGPTL3). The company revealed that ANGPTL3 acted as an inhibitor of lipoprotein lipase and endothelial lipase, and appeared to play a central role in lipoprotein metabolism.

The company's VP, Program Direction, Bill Sasiela, commented, "HoFH patients are not as responsive to traditional lipid lowering therapies like statins, and use of some new treatment options can be limited by safety and tolerability concerns. Preliminary data from this ongoing study show that evinacumab reduced LDL-C when combined with other lipid-lowering therapies, and suggests that ANGPTL3 is a new and exciting target that we look forward to exploring further in additional clinical trials."

Regeneron said HoFH was the most severe form of hypercholesterolemia. While rare, occurring in approximately 1-2 people per million, untreated patients could have "bad cholesterol" or LDL-C levels ranging between 500 and 1000 mg/dL, compared to normal LDL-C levels of fewer than 130 mg/dL. As a result of these high levels of LDL-C, patients with HoFH were at an extreme risk of premature cardiovascular disease. Without treatment, patients typically present with signs and symptoms of atherosclerotic cardiovascular disease before the age of 20.

The company indicated that Evinacumab was generally well tolerated and that there were no adverse events leading to discontinuation. The company said that the most common drug-related adverse events were injection-site reactions, which were mild in severity.