Phase III trial of Kynamro (mipomersen sodium) meets endpoint in severe HeFH- Isis Pharmaceuticals

Isis Pharmaceuticals has announced that the FOCUS FH Phase III study of Kynamro (mipomersen sodium) in patients with severe heterozygous familial hypercholesterolemia (severe HeFH) met its primary efficacy endpoint, a statistically significant reduction in LDL-cholesterol after 60 weeks of treatment of once weekly injections of 200 mg of Kynamro compared to placebo. LDL-cholesterol reduction was similar to that observed in previous phase III studies.

In addition, based on the data available for review, the safety profile of Kynamro observed in the FOCUS FH trial was similar to the safety profile reported in previous phase III studies. Genzyme will provide a more in depth review of the safety and efficacy data at a future medical meeting.

Comment: FOCUS FH was a multicenter, randomized, placebo-controlled, double-blind, parallel-group study that enrolled 310 patients aged 18 and older, followed by an open-label continuation. Upon completion of the 60 week blinded treatment, patients had the option to enter the open label continuation period for 26 weeks and receive the full dose regimen of Kynamro according to the dosing schedule they were randomized to during the blinded treatment phase. The trial was conducted at 131 sites worldwide. The primary efficacy endpoint evaluated was the LDL-C percent change from baseline to week 61 for cohort 1 and each dose regimen.