SAR 236553 success in Phase II study for heFH

Sanofi and Regeneron have announced results from study 1003 of SAR 236553/REGN 727, a subcutaneously administered, fully-human antibody targeting PCSK9 (proprotein convertase subtilisin/kexin type 9) intended to treat Heterozygous Familial Hypercholesterolemia (heFH). The trial randomized 77 patients with heFH whose LDL-cholesterol (LDL-C) levels remained uncontrolled on statin therapy with or without ezetimibe. Across the four different dosing regimens tested, patients receiving SAR236553 / REGN727 for 12 weeks achieved a mean LDL-C reduction from baseline of 28.9% to 67.9%, compared to 10.7% in patients receiving placebo (p<0.05 .in addition in the most intense dose regimen tested where the greatest ldl-c reduction was observed 150 milligrams mg every two weeks 93.8 of patients achieved ldl-c levels lower than 100 mg dl 2.59 mmol l compared to 13.3 of patients on placebo and 81.3 reached ldl-c levels lower than 70 mg dl 1.81 mmol l compared to none on placebo.the results from this study were published online in the lancet and also presented at a late-breaking oral session at the 80th european atherosclerosis society congress eas in milan italy. positive top-line results from this study were announced in november 2011.see stein ea et al. a randomized double-blind placebo-controlled trial of the safety and efficacy of a monoclonal antibody to pcsk9 regn727 sar236553 in heterozygous familial hypercholesterolemia patients on a stable statin dose with or without ezetimibe therapy. nct:01266876. presented at the 80th eas congress milan italy may 26 2012. abstract 1398.the companies intend to initiate a global phase iii program with sar236553 regn727 in june. this will be the first phase iii program of an investigational drug targeting pcsk9.>