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. Due to these high levels of LDL-C, patients with HoFH are 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.
“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,” said Bill Sasiela, Ph.D., Vice President, Program Direction, Regeneron. “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.”
The ongoing single-arm, open label, proof-of-concept study is targeting to enroll up to 8 patients with HoFH. Study patients’ diagnosis is confirmed both genetically and phenotypically. Current lipid-lowering therapy was maintained from 4 weeks before baseline through the 26-week treatment and observation period. Patients on LDL apheresis within 4 weeks prior to screening were excluded. For the 4 patients reported here, all were receiving maximum doses of statin plus ezetimibe with one patient additionally receiving lomitapide 20 mg. Evinacumab was dosed as a single 250 mg subcutaneous injection at baseline and subsequently, at week 2, as a 15 mg/kg intravenous (IV) infusion. The primary endpoint of the study was the mean percent change in LDL-C levels from baseline to week 4.
After 4 weeks of treatment and 2 weeks after administration of the 15 mg/kg IV dose, the mean reduction in LDL-C in the 4 patients was 55 percent (primary endpoint of the study). Among these 4 patients, the percent reductions in LDL-C ranged from 25 to 90 percent. Overall, the patients enrolled in the study saw their mean LDL-C levels fall from 331mg/dL at baseline to 175mg/dL at week 4.
Evinacumab was generally well tolerated and there were no adverse events leading to discontinuation. The most common drug-related adverse events were injection-site reactions, which were mild in severity.
About Regeneron Pharmaceuticals, Inc.
Regeneron (REGN) is a leading science-based biopharmaceutical company based in Tarrytown, New York that discovers, invents, develops, manufactures, and commercializes medicines for the treatment of serious medical conditions. Regeneron commercializes medicines for high LDL cholesterol, eye diseases, and a rare inflammatory condition and has product candidates in development in other areas of high unmet medical need, including oncology, rheumatoid arthritis, asthma, atopic dermatitis, pain, and infectious diseases. For additional information about the company, please visit www.regeneron.com or follow @Regeneron on Twitter.
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