Regeneron Pharmaceuticals, Inc. (REGN) said that positive preliminary results from an ongoing proof-of-concept study of evinacumab, REGN1500, in patients with Homozygous Familial Hypercholestrolemia or HoFH were presented at the 84th European Atherosclerosis Society (EAS) Congress in Innsbruck, Austria.
HoFH is the most severe form of hypercholesterolemia. While rare, occurring in approximately 1 to 2 people per million, untreated patients can have "bad cholesterol" or LDL-C levels ranging from 500 to 1000 mg/dL, compared to normal LDL-C levels of less than 130 mg/dL.
The ongoing single-arm, open label, proof-of-concept study is targeting to enroll up to 8 patients with HoFH. The interim data from the first 4 patients treated in this study showed that evinacumab added to current lipid-lowering therapy reduced low-density lipoprotein cholesterol or LDL-C levels by an additional 55 percent at week 4 compared to baseline.
Evinacumab is an investigational monoclonal antibody to angiopoietin-like protein 3 or ANGPTL3. ANGPTL3 acts as an inhibitor of lipoprotein lipase and endothelial lipase, and appears to play a central role in lipoprotein metabolism.
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