Regeneron Pharmaceuticals Inc.'s experimental drug was effective in treating symptoms of patients with a genetic disorder when used with conventional therapy in a late-stage study.
The ongoing phase 3 study, dubbed Elipse, is evaluating efficacy and safety of evinacumab in patients with homozygous familial hypercholesterolemia, who are already receiving lipid-lowering therapies.
Homozygous familial hypercholesterolemia is a rare condition characterized by high levels of bad cholesterol — also known as low-density lipoprotein, or LDL, cholesterol — that increases the risk of premature cardiovascular disease and a heart attack. The disorder is estimated to affects about 1,300 people in the U.S., according to the company.
The drug is being tested in 65 patients aged 12 or older in a randomized, parallel-group trial. Of these, 43 patients received 15 milligram/kilogram of evinacumab and 22 were given placebo every four weeks.
Results at week 24 of the study showed that evinacumab, when added to current lipid-lowering therapies, reduced LDL cholesterol in patients by 49% compared to placebo.
The company noted that reduction in LDL cholesterol levels was observed from the first assessment at week two, and was maintained throughout the 24-week treatment period. Evinacumab also reduced apolipoprotein B and non-high-density lipoprotein, or non-HDL, cholesterol, as well as total cholesterol compared to placebo.
About 66% of the patients who received evinacumab and 81% of placebo patients experienced an adverse event during the study.
Regeneron plans to submit data from the study to the regulatory authorities, starting with the U.S. Food and Drug Administration in 2020.
The Tarrytown, N.Y.-based company noted that it will present the detailed results of the study at a future medical meeting.