– Company Remains on Track to Report Top Line
Data in Early 2018 –
Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi
therapeutics company, today announced that it has completed
enrollment in its ENDEAVOUR Phase 3 study with revusiran, an
investigational RNAi therapeutic targeting transthyretin (TTR) for
the treatment of hereditary ATTR amyloidosis with cardiomyopathy
(hATTR-CM), also known as familial amyloidotic cardiomyopathy
(FAC). The ENDEAVOUR trial enrolled 206 patients with hATTR-CM,
above the enrollment target of 200 patients. The Company expects to
report data from the study in early 2018.
“ATTR amyloidosis is a progressive, life-threatening, orphan
disease with limited treatment options. These patients often have a
poor prognosis due to the rapid progression of their disease, and
new therapeutic options are needed,” said Eric Green, Vice
President, General Manager, TTR Program. “We’re excited to have now
completed enrollment in our ENDEAVOUR Phase 3 study, ahead of
expectations, and look forward to reporting data in early
2018."
The ENDEAVOUR Phase 3 trial is a randomized, double-blind,
placebo-controlled, global study designed to evaluate the efficacy
and safety of revusiran in patients with hATTR-CM. The co-primary
endpoints of the study are the change compared to baseline in
six-minute walk distance (6-MWD) and the percent reduction in TTR
level between placebo- and revusiran-treated patients at 18 months.
Secondary endpoints include a composite endpoint of cardiovascular
(CV) mortality and cardiovascular hospitalization, New York Heart
Association (NYHA) class, Kansas City Cardiomyopathy Questionnaire
(KCCQ), and all-cause mortality. The trial was designed to enroll
up to 200 hATTR-CM patients with a documented TTR mutation,
including V122I or other mutations, in addition to amyloid deposits
as identified by biopsy or technetium scan. Patients were
randomized 2:1, revusiran to placebo, with revusiran administered
subcutaneously at 500 mg daily for five days then weekly for 18
months.
All patients completing the ENDEAVOUR Phase 3 study may be
eligible to enroll in a Phase 3 open-label extension (OLE) study
(ENDEAVOUR-OLE).
Alnylam recently presented 12-month results from an ongoing
Phase 2 OLE study of revusiran in patients with TTR cardiac
amyloidosis. These data were presented at the XV International
Symposium on Amyloidosis, held July 3 – 7, 2016, in Uppsala,
Sweden.
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 and hATTR amyloidosis with cardiomyopathy (hATTR-CM) –
also known as familial amyloidotic cardiomyopathy (FAC) – is
estimated to affect at least 40,000 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).
hATTR-CM is fatal within 2.5 to 5 years of diagnosis and treatment
is currently limited to supportive care. Wild-type ATTR amyloidosis
(wtATTR) – previously called senile systemic amyloidosis (SSA) – is
a non-hereditary form of TTR cardiac amyloidosis caused by
idiopathic deposition of wild-type TTR; its prevalence is generally
unknown, but is associated with advanced age. 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 revusiran, Alnylam and Sanofi
Genzyme will co-develop/co-commercialize 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 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 revusiran, its expectations regarding the
presentation of clinical data from its ENDEAVOUR Phase 3 trial for
revusiran, 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, 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
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/20160808005257/en/
Alnylam Pharmaceuticals, Inc.Investors and MediaChristine Regan
Lindenboom, 617-682-4340orInvestorsJosh Brodsky, 617-551-8276
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