Armed with stellar — if niche — PhIII data, Regeneron steers 'breakthrough' cholesterol drug to post-PCSK9 waters
Two years after nabbing a breakthrough therapy designation for evinacumab, Regeneron has a slate of stellar Phase III data to buttress its case for offering the antibody to patients whose LDL cholesterol are so out of control that they may need something even stronger than PCSK9 inhibitors.
The drug treats a rare condition called homozygous familial hypercholesterolemia, or HoFH, in which patients have mutations in both copies of either PCSK9, LDLR or APOB genes. Regeneron scientists found two decades ago that the angiopoietin-like protein 3 (ANGPTL3) blocks lipoprotein lipase and endothelial lipase from regulating lipoprotein metabolism. By inhibiting this protein, Regeneron’s theory goes, they can unleash the enzymes to bring down the dangerously high levels of LDL cholesterol.
Unlock this article instantly by becoming a free subscriber.
You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.
