Isis Pharmaceuticals announced that the FOCUS FH Phase 3 study of Kynamro (mipomersen sodium) met its primary efficacy endpoint in patients with severe heterozygous familial hypercholesterolemia (severe HeFH).

FOCUS FH was a multicenter, randomized, placebo-controlled, double-blind, parallel-group study, followed by an open-label continuation. Cohort 1 included patients with severe HeFH with low density lipoprotein-cholesterol (LDL-C) ≥200mg/dL plus coronary heart disease or LDL-C ≥300mg/dL. Cohort 2 included patients with HeFH with LDL-C ≥160mg/dL and <200mg/dL plus coronary heart disease. Within each cohort, patients were randomized 2:1 to either 200mg once weekly, 70mg thrice weekly, or placebo for a 60 week duration. The primary efficacy endpoint evaluated was the LDL-C percent change from baseline to week 61 for cohort 1 and each dose regimen.

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Results from FOCUS FH showed a statistically significant reduction in LDL-C after 60 weeks of Kynamro 200mg once weekly injections compared to placebo. Genzyme, Isis’ partner company, will provide a more in depth review of the safety and efficacy data at a future medical meeting.

Kynamro is an oligonucleotide inhibitor of apolipoprotein B-100 synthesis indicated as an adjunct to lipid-lowering medications and diet to reduce LDL-C, apolipoprotein B (apo B), total cholesterol (TC), and non-high density lipoprotein-cholesterol (non-HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH).

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