Regeneron has announced positive topline results from its Phase III trial of evinacumab in patients with homozygous familial hypercholesterolemia – an inherited form of high cholesterol.

The company states that adding the investigational angiopoietin-like 3 (ANGPTL3) antibody reduced LDL cholesterol by 49% in patients with the disorder, compared to lipid-lowering therapies alone.

The data shows that on average patients entered the trial with LDL cholesterol levels of 255 mg/dL, despite treatment with other lipid-lowering therapies, including maximally-tolerated statins, PCSK9 (proprotein convertase subtilisin/kexin type nine) inhibitors, ezetimibe, LDL apheresis and lomitapide.

47% of pateints achieved LDL cholesterol levels less than 100 mg/dL compared to 23% for placebo and similar levels of LDL cholesterol-lowering were also observed in the most difficult-to-treat patients who often don't respond to certain other therapies

In 2017, the US Food and Drug Administration (FDA) granted Breakthrough Therapy designation for evinacumab for the treatment of hypercholesterolemia in patients with homozygous familial hypercholesterolemia.

The results also show that evinacumab was generally well-tolerated, and all patients completed the six-month treatment period.

"Currently HoFH patients face limited choices in reducing their LDL cholesterol, including therapies that are time-consuming like LDL apheresis, or that may have side effect concerns. Despite recent therapeutic advances, there is still a significant unmet need to lower the LDL cholesterol of many patients with HoFH. On average, evinacumab reduced patients' LDL cholesterol in half and was generally well-tolerated in the trial," said George D. Yancopoulos, president and chief scientific officer of Regeneron.

He also said that the results “raise the potential that evinacumab may have value for other patients with severe, refractory hypercholesterolemia, where we have a trial ongoing."

The drug is an investigational, fully-human, monoclonal antibody that specifically binds to angiopoietin-like protein 3 (ANGPTL3). ANGPTL3 acts as an inhibitor of lipoprotein lipase and endothelial lipase, and appears to play a central role in lipoprotein metabolism.

It is currently being studied in patients with homozygous familial hypercholesterolemia (Phase III), refractory hypercholesterolemia (Phase III) and severe hypertriglyceridemia (Phase III).