Sanofi and Regeneron Pharmaceuticals Inc. announced that the first Phase 2 study with alirocumab, an investigational monoclonal antibody targeting PCSK9 (proprotein convertase subtilisin/kexin type 9), in Japanese patients met its primary endpoint. The results demonstrated that the mean low-density lipoprotein-cholesterol (LDL-C, or "bad" cholesterol) percentage reduction from baseline to week 12, the primary efficacy endpoint of the study, was significantly greater in patients randomized to receive one of three doses of alirocumab administered every other week (Q2W)- 150 milligrams (mg), 75 mg, and 50 mg, in combination with statin therapy, compared to patients receiving placebo.
At Week 12, the mean percentage reduction in LDL-C from baseline in patients receiving alirocumab 50 mg Q2W was 55%, alirocumab 75 mg Q2W was 62% and alirocumab 150 mg Q2W was 72%, compared to 3% in the placebo group (p<0.0001 vs. placebo for all treatment arms). All patients in each of the alirocumab groups achieved LDL-C levels of less than 100 mg/dL, compared to 8% of patients in the placebo group.
Treatment emergent adverse events (TEAEs) in this study were reported by 52% of patients in the alirocumab 50 mg group, 48% of patients in the 75 mg group, and 64% of patients in the 150 mg group, compared to 32% in the placebo group. The most frequently reported TEAEs were nasopharyngitis, injection site reaction, back pain, cystitis and ligament sprain.
"We are delighted with the findings from the first Phase 2 trial with alirocumab in Japanese patients. Not only was alirocumab shown, in this study, to significantly reduce LDL-C in this patient population, the results of this study also demonstrate the potential efficacy of alirocumab at a range of doses," said Jay Edelberg, head of the PCSK9 Development and Launch Unit, Sanofi Group.
"Hypercholesterolemia is a growing problem in Japan and many patients are poorly-controlled on statins," commented George Yancopoulos, chief scientific officer of Regeneron and president of Regeneron Laboratories. "The results from this trial support the efficacy and safety of alirocumab at a range of doses in Japanese patients."
Date: April 1, 2014