Sanofi and Regeneron Pharmaceuticals have announced additional positive results from the Phase 2 trial of SAR236553 / REGN727 in patients with heterozygous familial hypercholesterolemia (heFH).
SAR236553 / REGN727 is a fully human monoclonal antibody that acts by inhibiting PCSK9, a determinant of circulating LDL-C levels in the blood, thereby increasing the number of free LDL receptors that can bind to circulating LDL-C and clear it from the bloodstream.
The study was a double-blind, placebo-controlled, dose-finding trial that randomised 77 heFH patients to either placebo or one of four active dose regimens of 150mg at four-week intervals or 150mg at two-week intervals.
In the trial, the primary objective was to evaluate the efficacy of various subcutaneous doses and dosing regimens of SAR236553 / REGN727 on LDL-C in patients with heFH.
At baseline, all patients had LDL-C greater than or equal to 100mg/dL and were on stable daily statin therapy with or without ezetimibe, for at least six weeks prior to screening.
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, according to the study.
Metabolic and Atherosclerosis Research Center, US, director and principal investigator of the study Evan Stein said heterozygous familial hypercholesterolemia is a common, serious and often undiagnosed cause of early heart disease.
"These data suggest that SAR236553 / REGN727 may provide a new option, on top of existing therapies, to lower LDL-cholesterol and finally reach LDL-C goals for these difficult-to-treat patients," Stein added.
Regeneron chief scientific officer and Regeneron Laboratories president, George Yancopoulos, said the data, along with recently presented data in patients with hypercholesterolemia, further support the company's belief that blocking PCSK9 with its antibody has the potential to offer a mechanism for lowering LDL-cholesterol in a broad range of patients.
The companies also said that based on discussions with the US and European regulatory authorities, they intend to commence a global Phase 3 programme with SAR236553 / REGN727 in June 2012.
Sanofi Global Research & Development president Elias Zerhouni said the Phase 3 programme will include patients with a high unmet medical need, such as patients with familial hypercholesterolemia or with elevated cardiovascular risk who cannot reach their LDL-cholesterol goals with current standard therapies.