Phase III ODYSSEY HoFH trial of Praluent meets primary endpoint in homozygous familial hypercholesterolemia.- Regeneron Pharma

Regeneron Pharma has presented Phase III ODYSSEY HoFH trial results from a late-breaking presentation, demonstrating the effect of Praluent (alirocumab) in patients with homozygous familial hypercholesterolemia (HoFH). The trial met its primary endpoint, with Praluent-treated patients experiencing a 36% reduction in LDL-C at week 12 compared to placebo (27% reduction Praluent, 9% increase placebo, p<0.0001).

At the same time point, compared to baseline Praluent-treated patients also experienced that average LDL-C levels decreased by 72 mg/dL compared to placebo (63 mg/dL reduction Praluent, 9 mg/dL increase placebo). More than half (57%) reduced their LDL-C by at least 30% and more than a quarter (27%) reduced their LDL-C by at least half, compared to 4% and 0% for placebo, respectively (p=0.0010 and p=0.0017). No serious AEs, permanent treatment discontinuations or deaths were reported during the double-blind treatment period. During the double-blind treatment period, the AE that occurred in at least 5% of patients, and more commonly with Praluent, was diarrhea (7% Praluent, 0% placebo).

AEs that occurred in at least 5% of patients, and more commonly with placebo, were upper respiratory tract infection (4% Praluent, 8% placebo) and headache (4% Praluent, 8% placebo). Regeneron plans to submit these data as the basis of regulatory submissions with the FDA in the second quarter of 2020. Data were presented at the American College of Cardiology’s Annual Scientific Session together with World Congress of Cardiology (ACC.20).