Market Overview

The Medicines Company Reaches Enrollment Target for ORION-9 Ahead of Schedule

  • ORION-9, a Phase III confirmatory clinical trial in heterozygous
    familial hypercholesterolemia patients randomized more than 400
    patients in 10 weeks – 14 weeks ahead of schedule

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

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."

About ORION-9

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

About inclisiran

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.

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" 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.

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