Data from the interim analysis of the ongoing ORION-1 study, which is evaluating The Medicines Company’s investigational first-in-class PCSK9 synthesis inhibitor, confirm the significant and durable low density lipoprotein (LDL) cholesterol reduction with the drug that was demonstrated up to day 90 in the preceding Phase 1 study. Additionally, the PCSK9si was well tolerated and no material safety issue was observed in the Day 90 interim analysis of unblinded safety data.
ORION-1 is a placebo-controlled, double-blind, randomised, dose-finding Phase 2 trial of single or multiple subcutaneous injections of PCSK9si in a total of 501 patients with atherosclerotic cardiovascular disease or related risk equivalents (eg., diabetes and familial hypercholesterolemia) and elevated LDL cholesterol despite maximum tolerated doses of LDL cholesterol lowering therapies. The trial compares the effect of different doses of PCSK9si and evaluates the potential for dosing no more than two, three or four times per year.
John JP Kastelein (professor of Medicine and chairman, Department of Vascular Medicine, Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands), says: “The thoughtful and robust design of the ORION-1 study has delivered highly impressive interim data. The potential for management of hypercholesterolemia with two or three low volume injections per year could open a new, transformative and affordable pathway for patients and physicians in the treatment of atherosclerotic cardiovascular disease.”
A press release reports that the interim analysis of Day 90 follow-up for all 501 patients, as well as top-line data from Day 180 follow-up for up to 200 patients, will be presented in a late-breaking Clinical Trials session at the 2016 American Heart Association (12–16 November, New Orleans, USA).