– Expected Top-line Data Readout Remains
Unchanged in Mid-2017 –
Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi
therapeutics company, announced today that the Data Monitoring
Committee (DMC) for the Phase 3 APOLLO study of patisiran in
patients with hereditary ATTR amyloidosis with polyneuropathy
(hATTR-PN) met on October 7, 2016 and recommended continuation of
the trial without modification. The APOLLO DMC met at the request
of the Company following the decision – announced on October 5,
2016 – to discontinue development of revusiran for the treatment of
hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM). The DMC
will continue to meet periodically per their remit to monitor the
overall safety of patisiran in the APOLLO study through its
completion.
The APOLLO study has completed enrollment of 225 patients at 44
sites in 19 countries, between December 2013 and January 2016.
“As part of our vigilance around patient safety, we felt it was
important to take immediate action and requested that the APOLLO
DMC convene to evaluate accumulated safety data from the
randomized, placebo-controlled Phase 3 study of patisiran,” said
Akshay Vaishnaw, M.D., Ph.D., Executive Vice President of R&D
and Chief Medical Officer at Alnylam. “We’re pleased to learn of
the DMC’s recommendation that dosing can continue in APOLLO, and we
look forward to the top-line data readout from that study expected
in mid-2017. There is substantial unmet need in hATTR-PN and we are
committed to advancing patisiran through development in hopes of
bringing a new and needed treatment option to patients.”
About ATTR Amyloidosis
ATTR amyloidosis is a progressively debilitating and often fatal
disease caused by deposition of transthyretin (TTR) in peripheral
tissues. TTR protein is produced primarily in the liver and is
normally a carrier of vitamin A. In hereditary ATTR amyloidosis
(hATTR), mutations in TTR cause abnormal amyloid proteins to
accumulate and damage body organs and tissue, such as the
peripheral nerves and heart, resulting in intractable peripheral
sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy.
Hereditary ATTR amyloidosis represents a major unmet medical need
with significant morbidity and mortality; hATTR amyloidosis with
polyneuropathy (hATTR-PN) – also known as familial amyloidotic
polyneuropathy (FAP) – affects approximately 10,000 people
worldwide. hATTR-PN patients have a life expectancy of 5 to 15
years from symptom onset, and the only approved treatment options
for early stage disease are liver transplantation and tafamidis
(approved in Europe, certain countries in Latin
America and Japan, where it is approved for all stages of
disease). There is a significant need for novel therapeutics to
treat patients with ATTR amyloidosis.
Sanofi Genzyme Alliance
In January 2014, Alnylam and Sanofi Genzyme, the specialty
care global business unit of Sanofi, formed an alliance to
accelerate and expand the development and commercialization of RNAi
therapeutics across the world. The alliance is structured as a
multi-product geographic alliance in the field of rare diseases.
Alnylam retains product rights in North
America and Western Europe, while Sanofi Genzyme obtained
the right to access certain programs in Alnylam's current and
future Genetic Medicines pipeline in the rest of the world (ROW)
through the end of 2019, together with certain broader
co-development/co-commercialization rights and global rights for
certain products. In the case of patisiran, Alnylam will advance
the product in North America and Western Europe,
while Sanofi Genzyme will advance the product in the ROW.
About RNAi
RNAi (RNA interference) is a revolution in biology, representing
a breakthrough in understanding how genes are turned on and off in
cells, and a completely new approach to drug discovery and
development. Its discovery has been heralded as "a major scientific
breakthrough that happens once every decade or so," and represents
one of the most promising and rapidly advancing frontiers in
biology and drug discovery today which was awarded the 2006 Nobel
Prize for Physiology or Medicine. RNAi is a natural process of gene
silencing that occurs in organisms ranging from plants to mammals.
By harnessing the natural biological process of RNAi occurring in
our cells, the creation of a major new class of medicines, known as
RNAi therapeutics, is on the horizon. Small interfering RNA
(siRNA), the molecules that mediate RNAi and comprise Alnylam's
RNAi therapeutic platform, target the cause of diseases by potently
silencing specific mRNAs, thereby preventing disease-causing
proteins from being made. RNAi therapeutics have the potential to
treat disease and help patients in a fundamentally new way.
About LNP Technology
Alnylam has licenses to Arbutus Biopharma LNP intellectual
property for use in RNAi therapeutic products using LNP
technology.
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.
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 the continued development of patisiran in
the ongoing APOLLO Phase 3 study, its expectations regarding the
expected timing for reporting top-line data from its APOLLO Phase 3
study of patisiran, its expectations regarding the safety and
tolerability of its products in clinical development, including
patisiran, and 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, including actions by regulators concerning
product candidates in addition to Alnylam’s decision to discontinue
development of revusiran, 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, 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
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.
The scientific information discussed in this news release
relating to Alnylam’s investigational therapeutics is preliminary
and investigative. None of Alnylam’s investigational therapeutics
have 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 the safety or
effectiveness of these therapeutics.
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version on businesswire.com: http://www.businesswire.com/news/home/20161010005305/en/
Alnylam Pharmaceuticals, Inc.Investors and
Media:Christine Regan Lindenboom, 617-682-4340orInvestors:Josh
Brodsky, 617-551-8276
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