Genzyme and Isis meet cholesterol trial endpoint

US companies Genzyme and Isis Pharmaceuticals have achieved the primary endpoint in a Phase III study of mipomersen in patients with homozygous  familial hypercholesterolemia, with subjects achieving a 25% reduction  in low-density lipoprotein cholesterol after 26 weeks, compared to just  3% for placebo.

In addition, all three secondary endpoints were also met: apolipoprotein  B, total cholesterol and non-high-density lipoprotein cholesterol (all  p=0.001). Though the patients in the trial were on maximally-tolerated  statins and other lipid-lowering therapies, their average LDL-C at  baseline was greater than 400mg/dL.

The randomized, double-blind, placebo-controlled study enrolled 51  patients, aged 12 and older. Subjects were randomized 2:1 to receive a  200mg dose of mipomersen or placebo via weekly injections for 26 weeks.  A regulatory filing is anticipated in the second half of next year.