Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the
leading RNAi therapeutics company, today provided perspective on
the positive complete results from the ORION-11 Phase 3 study of
inclisiran, an RNAi therapeutic in development for the treatment of
hypercholesterolemia. These results were presented earlier
today by Alnylam’s partner The Medicines Company and
collaborators at the European Society of Cardiology’s ESC Congress
2019 in Paris, France.
“The ORION-11 results represent a landmark event for Alnylam and
RNAi therapeutics. With over 1,600 patients enrolled, ORION-11 is
the largest ever randomized, double-blind, placebo-controlled study
of any RNA-based medicine. The results from the study demonstrate
significant efficacy for a drug administered subcutaneously once
every six months and a very encouraging safety profile, with
predominantly mild and transient injection site reactions as the
only reported drug-related finding,” said John Maraganore, Ph.D.,
Chief Executive Officer of Alnylam. “These results further support
the safety of our RNAi therapeutics platform and provide the
largest demonstration to date suggesting that there is no
systematic evidence for a platform-specific safety signal.
Consequently, these results greatly strengthen our conviction for
the future potential of RNAi therapeutics in large population
diseases. Moreover, the pharmacology of RNAi therapeutics, as
infrequently administered medicines, creates what we believe to be
a very attractive profile for the treatment of common diseases.
Finally, assuming a positive regulatory review, the significant
inclisiran royalties of up to 20 percent will provide yet another
source of relatively near-term revenues and will support Alnylam’s
transition toward a self-sustainable financial profile for
continued and future growth.”
ORION-11 now becomes the third positive Phase 3 study for an
RNAi therapeutic and the second in 2019 alone. Assuming ORION-9 and
-10 are similarly positive and following regulatory submissions and
review, Alnylam expects inclisiran could reach the market around
late 2020.
A video of Dr. Maraganore commenting on the implications of the
ORION-11 results can be viewed on the Company’s website,
www.alnylam.com.
About Inclisiran
Inclisiran, the first cholesterol-lowering therapy in the RNAi
therapeutics class, is an investigational therapy in Phase 3
clinical development to evaluate its ability to lower low-density
lipoprotein cholesterol (also known as LDL-C) through twice-yearly
dosing. 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. In Phase
2 studies, inclisiran provided clinically significant LDL-C
reductions greater than 50 percent in addition to the effects of
statins and/or ezetimibe, and LDL-C reductions were sustained
throughout the six-month dosing interval. The safety and efficacy
of inclisiran were evaluated in the ORION-11 Phase 3 study with
positive results; these results have not been evaluated by the FDA,
the EMA or any other health authority. Inclisiran is not yet
approved for use by the FDA or any other regulatory authority. In
February 2013 Alnylam granted global rights under a license and
collaboration agreement to The Medicines Company to develop,
manufacture, and commercialize inclisiran.
About RNAi
RNAi (RNA interference) is a natural cellular process of gene
silencing that represents one of the most promising and rapidly
advancing frontiers in biology and drug development today. Its
discovery has been heralded as “a major scientific breakthrough
that happens once every decade or so,” and was recognized with the
award of the 2006 Nobel Prize for Physiology or Medicine. By
harnessing the natural biological process of RNAi occurring in our
cells, a new class of medicines, known as RNAi therapeutics, is now
a reality. Small interfering RNA (siRNA), the molecules that
mediate RNAi and comprise Alnylam's RNAi therapeutic platform,
function upstream of today’s medicines by potently silencing
messenger RNA (mRNA) – the genetic precursors – that encode for
disease-causing proteins, thus preventing them from being made.
This is a revolutionary approach with the potential to transform
the care of patients with genetic and other diseases.
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 people afflicted with
rare genetic, cardio-metabolic, hepatic infectious, and central
nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning
science, RNAi therapeutics represent a powerful, clinically
validated approach for the treatment of a wide range of severe and
debilitating diseases. Founded in 2002, Alnylam is delivering on a
bold vision to turn scientific possibility into reality, with a
robust discovery platform. Alnylam’s first commercial RNAi
therapeutic is ONPATTRO® (patisiran) approved in the U.S., EU,
Japan and Canada. Alnylam has a deep pipeline of investigational
medicines, including five product candidates that are in late-stage
development. 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 to address the needs of patients
who have limited or inadequate treatment options. Alnylam employs
over 1,200 people worldwide and is headquartered in Cambridge, MA.
For more information about our people, science and pipeline, please
visit www.alnylam.com and engage with us on Twitter at
@Alnylam or on LinkedIn.
Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam's future
expectations, plans and prospects, including, without limitation,
the potential implications of the ORION-11 results for patients,
Alnylam's views with respect to the safety of RNAi therapeutics,
its views regarding the future potential of RNAi therapeutics in
large population diseases, its expectations regarding the timing of
results to be reported by The Medicines Company from the ORION-9
and ORION-10 studies of inclisiran and the regulatory review of an
NDA for inclisiran, its expectations regarding the receipt of
royalties from The Medicines Company on sales of inclisiran, if
approved by regulatory authorities, its plans to achieve a
self-sustainable financial profile for continued and future growth,
and expectations regarding 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, Alnylam’s
and its partners’ ability to successfully demonstrate the efficacy
and safety of its product candidates, including inclisiran, 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 its 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, Alnylam’s
and its partners’ ability to obtain and maintain regulatory
approval, pricing and reimbursement for products, including
inclisiran, progress in establishing a commercial and ex-United
States infrastructure, successfully launching, marketing and
selling its approved products globally, Alnylam’s ability to
successfully expand the indication for ONPATTRO in the future,
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 Quarterly Report on Form 10-Q 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20190902005039/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media) 617-682-4340
Josh Brodsky (Investors) 617-551-8276
Alnylam Pharmaceuticals (NASDAQ:ALNY)
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