Safety signal dampens reception for mipomersen antisense.

摘要

In February, Isis announced its cholesterol-lowering antisense therapy, mipomersen, had met its endpoints in a phase 3 trial of 124 patients with heterozygous familial hypercholesterolemia (FH) and coronary artery disease. The news ought to have sent share prices soaring, as mipomersen, if approved, could rival blockbuster statin drugs. Instead, lingering concerns related to high liver enzyme levels in some trial participants—a safety signal that had been noted in previous trials—dampened investor enthusiasm. But even if elevated liver enzymes make it less likely that mipomersen will supplant statins in major indications like coronary artery disease, efficacy data for the antisense therapy in patients with homozygous FH remain compelling.