PCSK9 inhibitor alirocumab outperforms Merck’s Zetia as a monotherapy in first Phase III trial reported, a result that has relevance for the large population of statin-intolerant patients, according to the drug’s sponsors.

A monthly dose of Amgen’s PCSK9 inhibitor lowers LDL significantly with a good tolerability profile in two Phase II studies. Reporting results for the RUTHERFORD trial in the journal Circulation, investigators compare AMG 145 favorably for heterozygous familial hypercholesterolemia to a range of rivals, including Sanofi’s REGN 727, Genzyme’s mipomersen and Aegerion’s lomitapide.

Statins represent a high standard of care for managing high blood cholesterol, bolstered by a strong evidence base, but new targets like PCSK9 and CETP are nevertheless stealing the spotlight nowadays. Whether new drugs, if they make it to market, will be used beyond narrow, targeted population segments is uncertain and will depend on results from large-scale outcomes studies showing significant efficacy and, importantly, reassurance of a solid safety profile.