PARSIPPANY, N.J.--(BUSINESS WIRE)--The Medicines Company (NASDAQ: MDCO) today announced that the target of 400 patients randomized in the ORION-9 trial has been exceeded in 10 weeks – considerably ahead of its anticipated recruitment goal of 24 weeks.
ORION-9 is a double-blind, randomized multi-national Phase III trial designed to confirm the effectiveness and safety of inclisiran in heterozygous familial hypercholesterolemia patients. In the trial, patients are randomized to inclisiran 300 mg or placebo given subcutaneously on days 1, 90, 270 and 450 – then followed to the end of the study at 540 days (18 months). ORION-9 is one of four pivotal phase III trials with similar designs to confirm the safety and effectiveness of inclisiran and potentially provide key data required for NDA and MAA submission in the United States and Europe, respectively.
The Company announced completion of enrollments in the first pivotal Phase III study, ORION-11, ahead of schedule on January 25, 2018.
The third pivotal Phase III study is ORION-10, which is actively enrolling 1500 ASCVD patients in the United States and Canada, is expected to complete enrollment during the first half of 2018. The fourth pivotal Phase III study, ORION-5, in up to 60 patients with homozygous familial hypercholesterolemia (HoFH) is expected to begin enrolling patients later in 2018 and to provide data for HoFH as part of a comprehensive LDL-C lowering program in a planned NDA and MAA submission. On January 23, 2018, the U.S. Food and Drug Administration (FDA) granted orphan drug designation for inclisiran for the treatment of HoFH.
“Completion of enrollment in ORION-9 is another key milestone on our way to confirming the excellent safety and efficacy data for inclisiran,” said David Kallend, MB BS, Chief Medical Officer of The Medicines Company. “We look forward to completing the follow-up of ORION-9 and the enrollment and follow-up of other pivotal trials, so that we can review the data in the second half of 2019 as part of the anticipated NDA and MAA submissions. We believe that the rapid pace of enrollment in ORION-11 and ORION-9, as well as the quality of data emerging from this and other trials, reflect the design of the studies, the effective operational support of excellent clinical sites, and the apparent ease of use of the protocol and study medication.”
ORION-9 is a Phase III, placebo-controlled, double-blind, randomized study of ~400 heterozygous familial hypercholesterolemia patients with elevated LDL-C levels above 100 mg/dL despite maximum tolerated doses of LDL-C lowering therapies including statins. The primary endpoint of the study is LDL-C reduction from baseline to day 510. The trial also evaluates the safety and tolerability of inclisiran.
About heterozygous familial hypercholesterolemia
According to the Familial Hypercholesterolemia Foundation, familial hypercholesterolemia (FH) is an inherited disorder that leads to aggressive and premature cardiovascular disease. This includes problems like heart attacks, strokes, and even narrowing of heart valves. For individuals with FH, although diet and lifestyle are important, they are not the cause of high LDL. In FH patients, genetic mutations make the liver incapable of metabolizing (or removing) excess LDL. The result is very high LDL levels which can lead to premature cardiovascular disease (CVD). Heterozygous FH occurs in 1 in 250 people worldwide. In the United States alone, an estimated 1.3 million people live with FH. Yet only 10% of them are diagnosed. FH runs in families. If one parent has FH, each child has a 50% chance of having FH. If left untreated, men have a 50% rise of having a heart attack by age 50. Untreated women have a 30% risk by age 60. 1 in 160,000 to 1 in 1 million people have HoFH. For more information go to https://thefhfoundation.org
Inclisiran is an investigational GalNAc-conjugated RNA interference therapeutic which inhibits the synthesis of PCSK9 protein in liver cells, thereby reducing liver cell LDL-receptor turnover, and lowering plasma LDL-C.
The Medicines Company and Alnylam Pharmaceuticals, Inc. are collaborating in the advancement of inclisiran pursuant to the terms of their 2013 agreement. Under the terms of that agreement, Alnylam completed certain pre-clinical studies and the Phase I clinical study, with The Medicines Company leading and funding the development of inclisiran from Phase II forward, as well as potential commercialization.
About The Medicines Company
The Medicines Company is a biopharmaceutical company driven by an overriding purpose – to save lives, alleviate suffering and contribute to the economics of healthcare. The Company’s goal is to create transformational solutions to address the most pressing healthcare needs facing patients, physicians and providers in cardiovascular care. The Company is headquartered in Parsippany, New Jersey.
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" 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 clinical trials for inclisiran will advance on a timely basis, or at all, or succeed in achieving their specified endpoints; whether physicians will prescribe and patients will use inclisiran, if it becomes available; whether the Company will make additional regulatory submissions for inclisiran on a timely basis, or at all; whether the Company’s regulatory submissions will receive approvals from regulatory agencies on a timely basis, or at all; and such other factors as are set forth in the risk factors detailed in the Company's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 9, 2017, which are incorporated herein by reference. The Company specifically disclaims any obligation to update these forward-looking statements.