The Medicines Company (MDCO) has reported positive topline results from its Phase II trial of single or multiple subcutaneous injections of PCSK9 synthesis inhibitor (PCSK9si).
PCSK9si is an investigational proprotein convertase subtilisin / kexin type 9 (PCSK9) synthesis inhibitor for the treatment of hypercholesterolemia.
This is a genetically validated protein regulator of LDL receptor metabolism and acts by inhibiting PCSK9 synthesis in the liver.
Hypercholesterolemia is a condition where very high levels of cholesterol are found in the blood.
The placebo-controlled, double-blind, randomised, dose-finding trial, ORION-1, involved a total of 501 patients with atherosclerotic cardiovascular disease (ASCVD) or ASCVD-risk equivalents such as diabetes and familial hypercholesterolemia and elevated LDL-C.
Patients were administered different doses of PCSK9si, and the potential for dosing no more than two, three or four times per year was evaluated.
MDCO CEO Clive Meanwell said: “We are highly encouraged by the strength and consistency of the day 90 efficacy and safety data from ORION-1, which build significantly on the promise generated by the preceding Phase I study of PCSK9si.
“This compelling interim data affirms PCSK9si’s highly competitive profile and validate PCSK9si’s game-changing potential. We look forward to presenting results from the ORION-1 study, including day 180 follow-up for up to 200 patients, at the AHA meeting on 15 November.”
The Medicines Company and Alnylam Pharmaceuticals are collaborating in the advancement of PCSK9si, under an agreement signed in early 2013.
As per the agreement, Alnylam completed certain pre-clinical studies and the Phase I clinical study of PCSK9si.