Phase III trial of Praluent (alirocumab) cut LDL-C in patients with heterozygous familial hypercholesterolemia- Sanofi / Regeneron Pharma

Sanofi and Regeneron Pharmaceuticals have announced detailed positive results from ODYSSEY ESCAPE, a Phase III trial which evaluated Praluent (alirocumab) Injection in patients with an inherited form of high cholesterol known as heterozygous familial hypercholesterolemia (HeFH) who require regular weekly or bi-weekly apheresis treatment. Apheresis is a procedure similar to kidney dialysis where bad (LDL) cholesterol is removed from the blood, and is usually reserved for high-risk patients with very high cholesterol unable to achieve their cholesterol-lowering goals on any other therapy.

The trial demonstrated that adding Praluent to existing therapy reduced LDL cholesterol by approximately 50 percent from baseline (compared to 2 percent increase for placebo). Praluent significantly reduced the need for apheresis treatment by 75 percent compared to placebo, the primary endpoint of the study. Despite being treated with apheresis and entering ODYSSEY ESCAPE with very high LDL cholesterol levels (4.7 millimoles/liter [mmol/L] or 181 milligrams/deciliter [mg/dL]), nearly two-thirds (63 percent) of patients treated with Praluent no longer required apheresis therapy after six weeks of receiving Praluent.

At this same time point, the average LDL cholesterol level among the Praluent-treated group was 2.3 mmol/L (90 mg/dL), compared to 4.8 mmol/L (185 mg/dL) in the placebo group. European guidelines recommend LDL cholesterol target levels between 1.8-3.0 mmol/L (70-115 mg/dL), depending on cardiovascular risk. Results will be presented at a Hot Line session at the ESC Congress 2016 in Rome, Italy.