—Inclisiran demonstrated significant and sustained reductions in
LDL-C and high standards of safety and tolerability—
—Optimal starting dose regimen (300 mg injection administered on
Day-1 and Day-90) lowered LDL-C by average of 52.6% (64 mg/dL) and
up to 81% (122 mg/dL) at Day-180, and by time-adjusted mean of
>50% (63 mg/dL) for the six-month period from Day-90 through
Day-270; every patient displayed significant response and mean
LDL-C reductions over this time period were practically
constant—
—Robust ORION-1 data reaffirm inclisiran’s potential to address
unmet needs with highly-differentiated, infrequent, low-volume
dosing regimen of two or three injections per year—
—No material safety issues on inclisiran in ORION-1; overall
incidence of adverse events for inclisiran was similar to
placebo—
—The Company is actively preparing to advance inclisiran into
comprehensive, global Phase III development – initially focused on
United States and Europe—
—ORION-1 study presented today in Late-Breaking Clinical Trials
session at American College of Cardiology’s 66th Annual Scientific
Session and published in The New England Journal of Medicine—
—The Company to host conference call at 4:30 p.m., Eastern Time,
today—
The Medicines Company (NASDAQ: MDCO) and Alnylam
Pharmaceuticals, Inc. (NASDAQ: ALNY) today announced positive final
results from the ORION-1 Phase II study of inclisiran, an
investigational, first-in-class PCSK9 synthesis inhibitor being
developed for the treatment of hypercholesterolemia. The results
are being presented today in the “Late-Breaking Clinical
Trials - Featured Clinical Research 1” session at the American
College of Cardiology’s 66th Annual Scientific Session, ACC.17, and
have been published in the March 17, 2017 online issue of The New
England Journal of Medicine.
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The detailed data from ORION-1 showed that inclisiran delivered
significant and sustained reductions of LDL-C and high standards of
safety and tolerability. Remarkably, every patient who received the
two dose starting regimen displayed a significant response and the
mean LDL-C reductions over time were practically constant.
Inclisiran was well tolerated and no material safety issue was
observed, including no investigational drug-related elevation of
liver enzymes and no neuropathy, change in renal function,
thrombocytopenia, or anti-drug antibodies.
“The groundbreaking results from ORION-1 are compelling and
affirm inclisiran’s unique and highly-differentiated attributes,
and its game-changing potential to address the unmet needs of
millions of at-risk, often non-adherent, patients worldwide who
continue to struggle with high cholesterol given the limitations of
available therapies,” said Clive Meanwell, M.D., Ph.D., Chief
Executive Officer of The Medicines Company. “We are singularly
focused on thoughtfully and aggressively advancing inclisiran into
Phase III development, including the initiation of ORION-4, which
we expect to include cardiovascular outcomes in high-risk primary
and secondary prevention patients with an average baseline LDL-C of
approximately 130 mg/dL.”
John J.P. Kastelein, M.D., Ph.D., Professor of Medicine and
Chairman of the Department of Vascular Medicine at
the Academic Medical Center of the University
of Amsterdam, continued, “We are pleased to see the complete
results from this comprehensive 497 patient Phase II study.
Inclisiran’s universal and practically constant effect is
unprecedented in my experience of over 30 years of dyslipidemia
clinical trials. The unique dosing regimen virtually eliminates
variability in LDL-C levels over time and inclisiran may,
therefore, solve one of the most vexing challenges of
cardiovascular medicine – namely, how to make sure everyone
responds to treatment.”
David Kallend, MBBS, Vice President and Global Medical Director
of The Medicines Company, added, “The results from ORION-1
have the potential to significantly advance the treatment paradigm
with robust and durable knockdown of LDL-C and a convincing and
highly-reassuring safety and tolerability profile. The data support
the selection of 300 mg as the optimal dose, as well as our
conclusion that a two dose starting regimen, followed by dosing two
times per year, constitutes a highly-differentiated and competitive
treatment for patients with hypercholesterolemia. We believe the
strength of the data will enable a quick and efficient transition
to Phase III development.”
John Maraganore, Ph.D., Chief Executive Officer of Alnylam,
added, “We are delighted with these final data from the ORION-1
study and impressed by the progress made by investigators and our
partner, The Medicines Company. Importantly, as the largest study
yet to be performed for an RNAi therapeutic, we believe the results
provide compelling support for the positive safety and tolerability
profile of our investigational medicines.”
In ORION-1, the mean baseline LDL-C was approximately 130 mg/dL
among 497 randomized and treated patients. The optimal starting
dose regimen (300 mg injection administered on Day-1 and Day-90)
achieved a mean LDL-C reduction of 52.6% and up to 81% at Day-180,
and a time-adjusted mean of >50% for the six month period from
Day-90 through Day-270. For all dose groups, at all time points,
differences in the primary (LDL-C) and secondary (PCSK9) endpoints
between inclisiran and placebo were statistically significant (p
<0.0001).
The overall incidence of treatment emergent adverse events was
76% in both patients randomized to placebo and patients randomized
to inclisiran, with no significant difference between inclisiran
doses. Injection site reactions associated with inclisiran were
infrequent (observed in 6.5% of patients given the two dose
starting regimen and 3.8% of patients given the one dose starting
regimen), mild or moderate, and transient.
The results from the Phase II study of inclisiran, published in
today’s online issue of The New England Journal of Medicine, can be
found here.
Webcast Information for Late-Breaking Clinical Trial
Presentations
Live audio and video of the “Late-Breaking Clinical Trials -
Featured Clinical Research 1” presentation by ORION-1’s principal
investigator, Kausik K. Ray, M.D., MPhil (Cantab), FRCP, Professor
of Public Health, Imperial College London, will be webcast over the
internet at 1:30 p.m., Eastern Time, today. The live webcast may be
accessed from the “Investors-Events/Presentations” section of The
Medicines Company website.
Conference Call and Webcast Details
The Medicines Company will host a conference call and webcast
today at 4:30 p.m., Eastern Time. The conference call may be
accessed as follows:
U.S./Canada: (877) 359-9508
International: (224) 357-2393 Conference ID: 60380330
The live webcast may be accessed in the
“Investors-Events/Presentations” section of The Medicines Company
website.
A taped replay of the conference call will be archived and
available for approximately one week. The replay may be accessed as
follows:
U.S./Canada: (855) 859-2056
International: (404) 537-3406 Conference ID: 60380330
A replay of the webcast will also be archived and available
after the conference call.
About ORION-1
ORION-1 is a placebo-controlled, double-blind, randomized Phase
II study of single or multiple subcutaneous injections of
inclisiran in a total of 501 patients with atherosclerotic
cardiovascular disease (ASCVD) or ASCVD-risk equivalents
(e.g., diabetes and familial
hypercholesterolemia) and elevated LDL-C despite maximum tolerated
doses of LDL-C lowering therapies. The study compares the effect of
different doses of inclisiran and evaluates the potential for an
infrequent dosing regimen. The primary endpoint of the study is the
percentage change in LDL-C from baseline at Day-180.
About Inclisiran
Inclisiran (formerly known as PCSK9si and ALN-PCSsc) is an
investigational GalNAc-conjugated RNAi therapeutic targeting PCSK9
– a genetically validated protein regulator of LDL receptor
metabolism – being developed for the treatment of
hypercholesterolemia. In contrast to anti-PCSK9 monoclonal
antibodies (MAbs) that bind to PCSK9 in blood, inclisiran is a
first-in-class investigational medicine that acts by turning off
PCSK9 synthesis in the liver.
The Medicines Company and Alnylam Pharmaceuticals, Inc. are
collaborating in the advancement of inclisiran pursuant to their
2013 agreement. Under the terms of the 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 mission is
to create transformational solutions to address the most pressing
healthcare needs facing patients, physicians and providers in three
critical therapeutic areas: serious infectious disease care,
cardiovascular care and surgery and perioperative care. The Company
is headquartered in Parsippany, New Jersey, with global innovation
centers in California and Switzerland.
About Alnylam Pharmaceuticals
Alnylam is a biopharmaceutical company developing novel
therapeutics based on RNA interference, or RNAi. The company is
leading the translation of RNAi as a new class of innovative
medicines. Alnylam's pipeline of investigational RNAi therapeutics
is focused in 3 Strategic Therapeutic Areas (STArs): Genetic
Medicines, with a broad pipeline of RNAi therapeutics for the
treatment of rare diseases; Cardio-Metabolic Disease, with a
pipeline of RNAi therapeutics toward genetically validated,
liver-expressed disease targets for unmet needs in cardiovascular
and metabolic diseases; and Hepatic Infectious Disease, with a
pipeline of RNAi therapeutics that address the major global health
challenges of hepatic infectious diseases. In early 2015, Alnylam
launched its "Alnylam 2020" guidance for the advancement and
commercialization of RNAi therapeutics as a whole new class of
innovative medicines. Specifically, by the end of 2020, Alnylam
expects to achieve a company profile with 3 marketed products, 10
RNAi therapeutic clinical programs – including 4 in late stages of
development – across its 3 STArs. The company's demonstrated
commitment to RNAi therapeutics has enabled it to form major
alliances with leading companies including Ionis, Novartis, Roche,
Takeda, Merck, Monsanto, The Medicines Company, and Sanofi Genzyme.
In addition, Alnylam holds an equity position in Regulus
Therapeutics Inc., a company focused on discovery, development, and
commercialization of microRNA therapeutics. Alnylam scientists and
collaborators have published their research on RNAi therapeutics in
over 200 peer-reviewed papers, including many in the world's top
scientific journals such as Nature, Nature Medicine, Nature
Biotechnology, Cell, New England Journal of
Medicine, and The Lancet. Founded in 2002, Alnylam
maintains headquarters in Cambridge, Massachusetts. For more
information about Alnylam's pipeline of investigational RNAi
therapeutics, please visit www.alnylam.com.
The Medicines Company Forward-Looking Statements
Statements contained in this press release 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," “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 in the clinical process on a timely basis,
or at all, or succeed in achieving their specified endpoints;
whether physicians, patients and other key decision makers will
accept clinical trial results; whether the Company will make
regulatory submissions for inclisiran on a timely basis, or at all;
whether its 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 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 Annual Report on Form 10-K filed with
the Securities and Exchange Commission on March 1,
2017, which are incorporated herein by reference. The Company
specifically disclaims any obligation to update these
forward-looking statements.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam's future
expectations, plans and prospects, including without limitation,
Alnylam's views with respect to the potential for RNAi
therapeutics, including inclisiran, its expectations regarding the
timing of clinical studies, its expectations regarding scientific
and regulatory support for inclisiran, its expectations regarding
its STAr pipeline growth strategy, and its “Alnylam 2020” guidance
for the advancement and commercialization of RNAi therapeutics,
constitute forward-looking statements for the purposes of the safe
harbor provisions under The Private Securities Litigation Reform
Act of 1995. Actual results and future plans may differ materially
from those indicated by these forward-looking statements as a
result of various important risks, uncertainties and other factors,
including, without limitation, Alnylam's ability to discover and
develop novel drug candidates and delivery approaches, successfully
demonstrate the efficacy and safety of its product candidates, the
pre-clinical and clinical results for its product candidates, which
may not be replicated or continue to occur in other subjects or in
additional studies or otherwise support further development of
product candidates for a specified indication or at all, actions or
advice of regulatory agencies, which may affect the design,
initiation, timing, continuation and/or progress of clinical trials
or result in the need for additional pre-clinical and/or clinical
testing, delays, interruptions or failures in the manufacture and
supply of our product candidates, obtaining, maintaining and
protecting intellectual property, Alnylam's ability to enforce its
intellectual property rights against third parties and defend its
patent portfolio against challenges from third parties, obtaining
and maintaining regulatory approval, pricing and reimbursement for
products, progress in establishing a commercial and ex-United
States infrastructure, competition from others using technology
similar to Alnylam's and others developing products for similar
uses, Alnylam's ability to manage its growth and operating
expenses, obtain additional funding to support its business
activities, and establish and maintain strategic business alliances
and new business initiatives, Alnylam's dependence on third parties
for development, manufacture and distribution of products, the
outcome of litigation, the risk of government investigations, and
unexpected expenditures, as well as those risks more fully
discussed in the "Risk Factors" filed with Alnylam's most recent
Annual Report on Form 10-K filed with the Securities and
Exchange Commission (SEC) and in other filings that Alnylam
makes with the SEC. In addition, any forward-looking
statements represent Alnylam's views only as of today and should
not be relied upon as representing its views as of any subsequent
date. Alnylam explicitly disclaims any obligation, except to the
extent required by law, to update any forward-looking
statements.
The scientific information referenced in this news release
relating to inclisiran is preliminary and investigative. Inclisiran
has not been approved by the U.S. Food and Drug Administration,
European Medicines Agency, or any other regulatory authority and no
conclusions can or should be drawn regarding its safety or
effectiveness.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20170317005437/en/
The Medicines
CompanyMedia:Meg Langan, 973-290-6319Vice
Presidentmargaret.langan@themedco.comInvestors:Krishna
Gorti, M.D., 973-290-6122Vice President, Investor
Relationskrishna.gorti@themedco.comorAlnylam PharmaceuticalsInvestors and
Media:Christine Regan Lindenboom, 617-682-4340Vice
Presidentclindenboom@alnylam.comInvestors:Josh Brodsky,
617-551-8276Associate Directorjbrodsky@alnylam.com
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