— Pivotal trials will enroll approximately
1,500 subjects randomized to inclisiran versus approximately 1,500
subjects randomized to placebo, with a primary endpoint of LDL-C
and an 18-month study period —
— NDA submission anticipated at or around the
end of 2019 —
The Medicines Company (NASDAQ:MDCO) and Alnylam Pharmaceuticals,
Inc. (NASDAQ:ALNY) today announced that The Medicines Company has
agreed with the U.S. Food and Drug Administration (FDA) on plans
for the Phase III clinical program for inclisiran, which is
designed to support the submission of a New Drug Application (NDA).
The Company has received final, End-of-Phase II meeting minutes
from the FDA.
The Phase III program will comprise clinical trials in subjects
with atherosclerotic cardiovascular disease (ASCVD) and familial
hypercholesterolemia (FH), and will collectively enroll
approximately 3,000 subjects randomized to treatment with
inclisiran (1,500) or placebo (1,500).
The primary endpoint for all pivotal trials will be LDL-C change
from baseline.
Subjects will be studied for 18 months. The dose of inclisiran
will be 300 mg given subcutaneously on day-1, day-90 and then
every six months thereafter. Subjects will receive a total of four
doses of inclisiran during the 18-month study period.
Although not part of the first NDA, The Medicines Company will
also perform a cardiovascular outcomes trial in approximately
14,000 subjects with ASCVD and/or risk equivalents, such as
diabetes, to determine the effects of LDL-C lowering with
inclisiran on cardiovascular outcomes. The design of the outcomes
trial has also been agreed with the FDA and the primary efficacy
endpoint of the trial will be a composite of coronary heart disease
death, non-fatal myocardial infarction and fatal and non-fatal
ischemic stroke. These endpoints have been demonstrated to be
modifiable in previous, similar outcomes trials. The duration of
the outcomes trial will be long enough to accumulate a sufficient
number of events to provide overwhelming statistical power to
ascertain treatment group differences and maximize the clinical
effect size associated with LDL-C lowering. Assuming success,
results of the outcomes trial will be submitted to the FDA as a
supplemental NDA.
“We are grateful to the FDA for its expertise, advice and
support. Cardiovascular disease is a serious threat to the health
of Americans, and it is clear that the FDA is committed to
facilitating the development and approval of effective and safe
drugs to address this important public health problem,” said Clive
Meanwell, M.D., Ph.D., Chief Executive Officer of The Medicines
Company. “Based on data from our Phase II ORION-1 study and the
previous Phase I study – both published in The New England Journal
of Medicine – we are confident that inclisiran reduces LDL-C
meaningfully. We have also been highly encouraged by the safety
data in these prior studies. Furthermore, inclisiran’s
highly-differentiated dosing schedule has the potential to
transform the burden placed on millions of patients who so badly
need to lower LDL-C levels. We anticipate completing the LDL-C
lowering program quickly and expect to submit an NDA for ASCVD and
FH at or around the end of 2019. We believe our agreement with the
FDA on the Phase III clinical development program for inclisiran is
highly favorable and adds significantly to inclisiran’s strategic
value.”
Dr. Meanwell added, “Although not required for the NDA, we will
also perform an aggressive cardiovascular outcomes trial in
high-risk subjects with ASCVD and/or risk-equivalents, such as
diabetes. We believe that positive outcomes data, with primary
outcome clinical effects greater than those reported for anti-PCSK9
monoclonal antibodies, will drive a high level of competitiveness
in the worldwide market – which we expect to become very
large.”
“The Medicines Company’s agreement with the FDA supports a clear
path forward for further inclisiran development and regulatory
review. Indeed, we’re pleased with the excellent progress made by
our colleagues at The Medicines Company to advance this potential,
innovative medicine to patients in need, and we intend to fully
support them in their efforts,” said John M. Maraganore, Ph.D.,
Chief Executive Officer of Alnylam. “Moreover, we believe the
planned development path for inclisiran positions this promising
potential medicine to contribute meaningfully to our Alnylam 2020
goals.”
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 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 (NASDAQ: ALNY) is leading the translation of RNA
interference (RNAi) into a whole new class of innovative medicines
with the potential to transform the lives of patients who have
limited or inadequate treatment options. Based on Nobel
Prize-winning science, RNAi therapeutics represent a powerful,
clinically validated approach for the treatment of a wide range of
debilitating diseases. Founded in 2002, Alnylam is delivering on a
bold vision to turn scientific possibility into reality, with a
robust discovery platform and deep pipeline of investigational
medicines, including three product candidates that are in
late-stage development or will be in 2017. Looking forward, Alnylam
will continue to execute on its “Alnylam 2020” strategy of building
a multi-product, commercial-stage biopharmaceutical company with a
sustainable pipeline of RNAi-based medicines. For more information
about our people, science and pipeline, please visit
www.alnylam.com and engage with us on Twitter at @Alnylam.
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 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 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.
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version on businesswire.com: http://www.businesswire.com/news/home/20170426005497/en/
The Medicines CompanyMediaMeg Langan,
973-290-6319Vice
Presidentmargaret.langan@themedco.comorInvestorsKrishna
Gorti, M.D., 973-290-6122Vice President, Investor
Relationskrishna.gorti@themedco.comorAlnylam
PharmaceuticalsInvestors and MediaChristine Regan
Lindenboom, 617-682-4340Vice
Presidentclindenboom@alnylam.comorInvestorsJosh Brodsky,
617-551-8276Associate Directorjbrodsky@alnylam.com
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