ORION-9 study met all primary and secondary efficacy
endpoints
Inclisiran demonstrated durable and potent efficacy and
excellent safety in a patient population where cardiovascular
disease is most severe
Detailed data from ORION-9 will be presented in a
late-breaking science session at the American Heart Association
Scientific Sessions in Philadelphia
The Medicines Company (NASDAQ: MDCO) today announced positive
topline results for the ORION-9 Phase 3 clinical study in patients
with Heterozygous Familial Hypercholesterolemia (HeFH). ORION-9 met
all primary and secondary endpoints, and inclisiran demonstrated
durable and potent efficacy and was well-tolerated with excellent
safety that was generally well-balanced between the treatment
groups. Furthermore, there were no treatment-related liver or renal
laboratory abnormalities.
Detailed efficacy, tolerability and safety data from ORION-9
will be presented at the American Heart Association (AHA)
Scientific Sessions in Philadelphia on Monday, November 18, 9:24am
EST, during Late Breaking Science VI: New Frontiers in Lipid
Therapy. The company will also present data from the ORION-10 study
in ASCVD patients at the AHA congress on Saturday, November 16,
11:06 am EST, during Late Breaking Science I: Outside the Box: New
Approaches to CVD Risk Reduction.
“The devastating cumulative effects of high LDL-C are more
pronounced in people with FH, many of whom do not reach their
treatment goals despite having significantly increased risk of
cardiovascular disease,” said Mark Timney, Chief Executive Officer
of The Medicines Company. “The results of ORION-9 strongly support
inclisiran’s potential in this patient population where the disease
severity is highest.”
FH is an inherited condition that causes high levels of LDL-C
and leads to early onset of heart disease. Approximately one in 250
people in the world – 1.3 million people in the United States –
have FH, but over 90% have not been properly diagnosed. HeFH is the
most common form of FH. Homozygous FH (HoFH), the most severe form,
is rarer and affects approximately 1 in 300,000 individuals
worldwide. HoFH causes extremely high LDL-C resulting in aggressive
heart disease at a very young age.
“It is particularly gratifying to see a successful outcome in a
patient population where baseline LDL-C levels are significantly
higher and unmet needs are so profound,” said Peter Wijngaard,
Ph.D., Chief Development Officer of The Medicines Company. “We
thank the patients and clinical sites who participated in this
study, and we look forward to sharing detailed results during AHA
and submitting data to a peer-reviewed journal.”
Regulatory submissions for inclisiran are anticipated to occur
in the U.S. in the fourth quarter of 2019 and in Europe in the
first quarter of 2020. Patients who have completed their respective
Phase 3 studies are now enrolling into ORION-8, an open-label,
long-term extension study in which patients completing ORION-9,
ORION-10 and ORION-11 will receive inclisiran for three years to
generate longer-term efficacy, safety and tolerability data for
inclisiran.
Background on Pivotal Phase 3 LDL-C Lowering Studies
The ORION program is studying the efficacy and safety of
inclisiran in patients with ASCVD and FH, with ORION-9, ORION-10
and ORION-11 comprising the pivotal Phase 3 LDL-C lowering
studies.
ORION-9 is a pivotal Phase 3, placebo-controlled, double-blind,
randomized study to evaluate the efficacy, safety and tolerability
of inclisiran sodium 300 mg administered subcutaneously in 482
patients with clinical or genetic evidence of HeFH and elevated
LDL-C, despite maximum tolerated dose of LDL-C-lowering therapies
(e.g., a statin or ezetimibe). The primary endpoints are percentage
change in LDL-C from baseline to day 510 (17 months) and
time-adjusted percentage change in LDL-C from baseline between day
90 (three months) and up to day 540 (18 months). Key secondary
endpoints include the mean absolute change at Day 510 (17 months),
the average absolute reduction from Day 90 (three months) up to Day
540 (18 months), and changes in other lipids and lipoproteins. The
international study was conducted at 54 sites in eight countries.
Each study participant received inclisiran sodium 300 mg
administered as a subcutaneous injection initially, again at three
months and then every six months thereafter. The majority of study
participants are taking inclisiran or placebo in addition to
existing lipid-lowering therapy with a maximally tolerated statin
(with or without ezetimibe).
ORION-10 is a pivotal Phase 3, placebo-controlled, double-blind,
randomized study to evaluate the efficacy, safety and tolerability
of inclisiran sodium 300 mg administered subcutaneously in 1,561
participants with ASCVD and elevated LDL-C, despite maximum
tolerated dose of LDL-C-lowering therapies (e.g., a statin or
ezetimibe). The primary endpoints are percentage change in LDL-C
from baseline to day 510 (17 months) and time-adjusted percentage
change in LDL-C from baseline after day 90 (three months) and up to
day 540 (18 months). The majority of study participants are taking
inclisiran or placebo in addition to existing lipid-lowering
therapy with a maximally tolerated statin (with or without
ezetimibe). Key secondary endpoints include the mean absolute
change at Day 510 (17 months), the average absolute reduction from
Day 90 (three months) up to Day 540 (18 months), and changes in
other lipids and lipoproteins. The study was conducted at 145 sites
in the United States. Each study participant received inclisiran
sodium 300 mg administered as a subcutaneous injection initially,
again at three months and then every six months thereafter. The
majority of study participants are taking inclisiran or placebo in
addition to existing lipid-lowering therapy with a maximally
tolerated statin (with or without ezetimibe).
ORION-11 is a pivotal Phase 3, placebo-controlled, double-blind,
randomized study to evaluate the efficacy, safety, and tolerability
of inclisiran sodium 300 mg administered subcutaneously in 1,617
patients with ASCVD or ASCVD-risk equivalents and elevated LDL-C
despite maximum tolerated dose of statin therapy (with or without
ezetimibe). In ORION-11, twice-yearly dosing with inclisiran sodium
300 mg met all primary and secondary efficacy endpoints, was
well-tolerated and demonstrated an excellent safety profile.
Detailed study results were presented during a late-breaking
science session at the European Society of Cardiology’s ESC
Congress 2019 in Paris on September 2 [click here for news
release].
About Inclisiran
Inclisiran, the first and only cholesterol-lowering therapy in
the siRNA (small-interfering RNA or “sir-nah”) class, is The
Medicines Company’s investigational twice-yearly therapy in Phase 3
clinical development to evaluate its ability to reduce low-density
lipoprotein cholesterol (also known as LDL-C). As a siRNA,
inclisiran harnesses the body’s natural process of RNA interference
to specifically prevent production of the PCSK9 protein in the
liver which enhances the liver’s ability to remove LDL-C from the
bloodstream, thereby lowering LDL-C levels. Inclisiran is not yet
approved for use by the FDA or any other regulatory authority. The
Medicines Company obtained global rights to develop, manufacture
and commercialize inclisiran under a license and collaboration
agreement with Alnylam Pharmaceuticals.
Commercial Opportunity
Nearly 60 million people with ASCVD or FH across the U.S., the
largest European countries, China and Japan are currently treated
with lipid-lowering therapies to manage cardiovascular risk. More
than 70 percent of these patients are not achieving LDL-C treatment
goals with current therapies, and approximately two-thirds of
patients do not adhere to available first-line cholesterol-lowering
treatments after one year. This implies a population of more than
40 million people who could potentially benefit from the
investigational candidate inclisiran in the aforementioned
countries alone. Inclisiran is the first cholesterol-lowering siRNA
with the potential to deliver potent and durable lowering of LDL-C
levels via twice-yearly dosing that can help address two critical
unmet needs – additional LDL-C lowering and poor adherence to
therapy.
About The Medicines Company
The Medicines Company (NASDAQ: MDCO) is a biopharmaceutical
company with a singular, relentless focus on addressing the
greatest global healthcare challenge and burden today –
cardiovascular disease. Our purpose is to halt the deadly
progression of atherosclerosis and the cardiovascular risk created
by high levels of LDL-C. The Company is headquartered in
Parsippany, New Jersey. For more information, please visit
www.themedicinescompany.com and follow us on Twitter @MDCONews and
LinkedIn.
Forward-Looking Statements
Statements contained in this press release that are not purely
historical, including, but not limited to, statements about the
Company, the proposed offering described herein and the use of
proceeds therefrom, are 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,” “plans,” “expects,” “should,” 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 the ability of
the Company to effectively develop inclisiran; whether inclisiran
will advance in the clinical trials process on a timely basis or at
all, or succeed in achieving its specified endpoints; whether the
Company will make regulatory submissions for inclisiran on a timely
basis; whether its regulatory submissions will receive approvals
from regulatory agencies on a timely basis or at all; the extent of
the commercial success of inclisiran, if approved; the strength,
durability and life of the Company’s patent protection for
inclisiran and whether the Company will be successful in extending
exclusivity; 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 SEC, including,
without limitation, the risk factors detailed in the Company's
Quarterly Report on Form 10-Q filed with the SEC on July 24, 2019.
The Company specifically disclaims any obligation to update these
forward-looking statements.
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version on businesswire.com: https://www.businesswire.com/news/home/20190925005450/en/
Investor Relations Krishna Gorti, M.D. Investor Relations
+1 973 290 6122 krishna.gorti@themedco.com
Media Inquiries Michael Blash Communications +1 973 290
6100 michael.blash@themedco.com
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