Updated on 28 May 2012
Positive results: Phase II trials to treat familial heFH show new hope
Singapore: Sanofi and Regeneron Pharmaceuticals revealed additional positive results from a phase II trial of SAR236553/REGN727 (Study 1003, NTC01266876) in patients with heterozygous familial hypercholesterolemia (heFH).
The candidate is a subcutaneously administered, fully-human antibody that targets proprotein convertase subtilisin/kexin type 9 (PCSK9) in clinical development. The research was published online in The Lancet, and was also presented at a late-breaking oral session at the 80th European Atherosclerosis Society Congress (EAS) in Milan, Italy.
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 the candidate for 12 weeks achieved a mean LDL-C reduction from baseline of 28.9 percenrt to 67.9 percent as compared to 10.7 percent in patients receiving placebo (p < 0.05).
There were no serious adverse events (SAE) on active treatment, while a single SAE was recorded for a patient in the placebo group. There were no elevations in liver function tests (LFT) and no cases of elevated creatinine kinase (CK) were reported. The most common adverse event reported was injection-site reaction. Sanofi and Regeneron also revealed that they intend to initiate a global phase III program with the candidate in June. This will be the first phase III program of an investigational drug targeting PCSK9.