The Medicines Company Advances Clinical Development of Investigational First-in-Class PCSK9 Synthesis Inhibitor for Atherosclerotic Cardiovascular Disease (ASCVD)

11 Jan 2016

Initiates sites and begins enrolling patients in dose-finding Phase 2 study

Study will evaluate effectiveness of ALN-PCSsc on LDL-C levels with quarterly and bi-annual dosing regimens to determine dose and dose regimen for anticipated Phase III

PARSIPPANY, NJ--(BUSINESS WIRE)--Jan. 11, 2016-- The Medicines Company (NASDAQ:MDCO) today announced that it has initiated study sites and begun enrolling patients in the ORION-1 Phase 2 study to compare the effect of different doses of ALN-PCSsc given as subcutaneous injections in a quarterly or bi-annual dosing regimen in subjects with atherosclerotic cardiovascular disease (ASCVD) or risk equivalent ASCVD and elevated LDL-C. ALN-PCSsc is an investigational RNA interference (RNAi) therapeutic targeting PCSK9 – a genetically validated protein regulator of LDL receptor metabolism. In contrast to anti-PCSK9 monoclonal antibodies (MAbs) that bind to PCSK9 in blood, ALN-PCSsc is a first-in-class investigational medicine that acts by turning off PCSK9 synthesis in the liver.

The ORION-1 study is a Phase 2, placebo-controlled, double-blind, randomized trial in 480 subjects with atherosclerotic cardiovascular disease or ASCVD-risk equivalents (e.g., diabetes and familial hypercholesterolemia) and elevated LDL-C despite maximum tolerated doses of LDL-C lowering therapies to evaluate the efficacy, safety, and tolerability of ALN-PCSsc injection(s).

“The results from the Phase 1 study of ALN-PCSsc were impressive, and we are excited to see the rapid progression of this therapy into the next phase of its development,” said John J.P. Kastelein, MD, PhD, Professor of Medicine, Department of Vascular Medicine at the Academic Medical Center (AMC) of the University of Amsterdam and Chairman of the Executive Committee for ORION-1. “We look forward to the expected completion of this Phase 2 trial by the end of 2016 to confirm what may be an advantageous dosing regimen for patients at risk for cardiovascular events due to elevated LDL-C.”

“We are very pleased with the rapid development progress for ALN-PCSsc, which has moved from Phase 1 to Phase 2 in a few weeks,” said Clive Meanwell, MD, PhD, Chief Executive Officer, The Medicines Company. “We remain very excited about the prospects of having a bi-annual dosing treatment available for LDL-C reduction which has the potential to improve adherence and address a major unmet medical need.”

“We are pleased to have successfully completed the transition of ALN-PCSsc to our partners at The Medicines Company,” said John Maraganore, PhD, Chief Executive Officer of Alnylam Pharmaceuticals, Inc. “Given our world-leading expertise in RNAi therapeutics, we look forward to providing all necessary support to enable MDCO efforts, including in areas such as regulatory and manufacturing.”

The Medicines Company and Alnylam Pharmaceuticals are collaborating in the advancement of ALN-PCSsc per the companies’ agreement formed in early 2013. Under the terms of the agreement, Alnylam completed certain pre-clinical studies and the Phase 1 clinical study, with The Medicines Company leading and funding the development of ALN-PCSsc from Phase 2 forward as well as potential commercialization.

About Hypercholesterolemia

Hypercholesterolemia is a condition characterized by very high levels of cholesterol in the blood which is known to increase the risk of coronary artery disease, the leading cause of death in the U.S. Some forms of hypercholesterolemia can be treated through dietary restrictions, lifestyle modifications (e.g., exercise and smoking cessation) and medicines such as statins. However, a large proportion of patients with hypercholesterolemia are not achieving adequate LDL-C levels with currently available therapies such as statins, including genetic familial hypercholesterolemia (FH) patients, acute coronary syndrome patients, high-risk patient populations (e.g., patients with coronary artery disease, diabetes, symptomatic carotid artery disease, etc.) and other patients that are statin intolerant. Severe forms of hypercholesterolemia are estimated to affect more than 500,000 patients worldwide, and as a result, there is a significant need for novel therapeutics to treat patients with hypercholesterolemia whose disease is inadequately managed by existing therapies.

About ALN-PCSsc

ALN-PCSsc, an investigational product not approved for commercial use in any market, is a subcutaneously administered RNAi therapeutic that utilizes Alnylam Pharmaceuticals’ proprietary Enhanced Stabilization Chemistry (ESC)-GalNAc-siRNA conjugate delivery platform. ESC-GalNAc-siRNA conjugates are designed to achieve targeted delivery of RNAi therapeutics to hepatocytes through uptake by the asialoglycoprotein receptor, and enable subcutaneous dosing with increased potency and durability and a wide therapeutic index.

About The Medicines Company

The Medicines Company's purpose is to save lives, alleviate suffering and contribute to the economics of healthcare by focusing on 3000 leading acute/intensive care hospitals worldwide. Its vision is to be a leading provider of solutions in three areas: serious infectious disease care, acute cardiovascular care and surgery and perioperative care. The company operates in the Americas, Europe and the Middle East, and Asia Pacific regions with global centers today in Parsippany, NJ, USA and Zurich, Switzerland.

Forward-Looking Statements

Statements contained in this press release about The Medicines Company that are not purely historical, and all other statements that are not purely historical, may be deemed to be forward-looking statements for purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Without limiting the foregoing, the words "believes," "anticipates," "expects," “hopes” and “potential” and similar expressions, are intended to identify forward-looking statements. These forward-looking statements involve known and unknown risks and uncertainties that may cause the Company's actual results, levels of activity, performance or achievements to be materially different from those expressed or implied by these forward-looking statements. Important factors that may cause or contribute to such differences include whether ALN-PCSsc will advance in the clinical trials process on a timely basis or at all, whether physicians, patients and other key decision makers will accept clinical trial results, whether the Company will make regulatory submissions for ALN-PCSsc on a timely basis or at all, whether its regulatory submissions will receive approvals from regulatory agencies on a timely basis or at all, the Company's ability to successfully compete with potential competitors which may discover, develop or commercialize competing products more successfully than we do and such other factors as are set forth in the risk factors detailed from time to time in the Company's periodic reports and registration statements filed with the Securities and Exchange Commission including, without limitation, the risk factors detailed in the Company's Quarterly Report on Form 10-Q filed with the SEC on November 9, 2015, which are incorporated herein by reference. The Company specifically disclaims any obligation to update these forward-looking statements

Source: The Medicines Company

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