Evinacumab is an investigational monoclonal antibody to angiopoietin-like protein 3 (ANGPTL3).
ANGPTL3 claimed to serve an inhibitor of lipoprotein lipase and endothelial lipase, in addition to playing a key role in lipoprotein metabolism.
The company is presently planning a phase 3 trial after positive interim phase 2 results for evinacumab in May 2016.
Regeneron’s proof-of-concept study had demonstrated that evinacumab added to current lipid-lowering therapy reduced low-density lipoprotein cholesterol (LDL-C) levels by an additional 55% at week four compared against baseline, the company said.
The single-arm, open label and proof-of-concept study had recruited up to 8 patients with HoFH, who were diagnosed genetically and phenotypically
Regeneron fixed the primary endpoint of the trial as the mean percent change in LDL-C levels from baseline to week four.
The mean reduction in LDL-C in the 4 patients was 55%, after four weeks of treatment and two weeks after administration of the 15 mg/kg IV dose. The percent reductions in LDL-C ranged from 25% to 90% among four patients.
Though rare HoFH disease can occur one to two people per million and those suffering from it can have a risk of premature cardiovascular disease.
Untreated patients can have LDL cholesterol levels ranging from 500 to 1000 mg/dL, compared to normal LDL cholesterol levels of less than 130 mg/dL.
Breakthrough Therapy designation is given to a drug to speed up the development and review of drugs that can treat life-threatening diseases.
Image: The FDA campus at New Hampshire Ave in Silver Spring, Maryland. Photo: courtesy of The U.S. Food and Drug Administration.