– Initiates Free Screening Support for Genetic
Mutations Associated with Familial Amyloidotic Polyneuropathy (FAP)
in Transthyretin-Mediated Amyloidosis (ATTR) as Initial Effort
under Alnylam Assist –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi
therapeutics company, announced today that it has launched Alnylam
Assist™, a program dedicated to providing support to patients,
families, and caregivers. Alnylam is committed to developing RNAi
therapeutics for a number of genetic diseases and intends to
implement certain aspects of Alnylam Assist broadly across its
pipeline of novel investigational therapies in development.
“Alnylam Assist is a broad-based initiative dedicated to
providing support to patients, families, and caregivers affected by
certain genetic diseases. As an initial effort under this program,
we are sponsoring third party laboratory screening for people in
the United States who are at a higher risk for carrying genetic
mutations known to be associated with transthyretin-mediated
familial amyloidotic polyneuropathy. Screening is an important step
as early diagnosis of this disease can assist patients in getting
the help and support they need,” said Pritesh Gandhi, Vice
President, Medical Affairs at Alnylam. “At Alnylam, we are
committed to developing RNAi therapeutics for certain genetic
diseases and through Alnylam Assist, our goal is to support people
affected by these diseases.”
Familial amyloidotic polyneuropathy (FAP) is a rare, inherited,
genetic disease caused by mutations in the transthyretin (TTR) gene
resulting in accumulation of TTR amyloid deposits in various
tissues, such as the peripheral nerves. FAP is a progressive
condition; it remains underdiagnosed and has limited treatment
options. Alnylam is committed to advancing the diagnosis and
treatment of FAP. To facilitate accurate and early diagnosis,
Alnylam Assist offers free third party laboratory screening in the
United States for people experiencing clinical signs and symptoms
consistent with this condition, as well as for family members of
patients previously diagnosed with FAP. For more information about
Alnylam Assist and the FAP free screening program, please visit
www.alnylam.com/patients.
“Tremendous advances are being made in FAP research,” said
Natacha T. Pires, M.B.B.S., Director, Medical and Public Affairs of
The Neuropathy Association. “This free screening program offered by
Alnylam gives patients and their physicians access to laboratory
testing for genetic mutations known to be associated with this
serious, life-threatening disease. This screening is expected to
help enable earlier diagnosis and enhance a patient’s knowledge of
the disorder and how it might affect them and their family members.
It will also allow them to initiate discussions with their
healthcare provider about their care, and potentially enable them
to consider participating in ongoing clinical trials.”
About Alnylam Assist™
Alnylam is committed to developing RNAi therapeutics for genetic
diseases and through Alnylam Assist, is dedicated to providing
support to patients, families, and caregivers. Alnylam intends to
implement certain aspects of Alnylam Assist broadly across its
pipeline of novel investigational therapies in development.
About Transthyretin-Mediated Amyloidosis
Transthyretin (TTR)-mediated amyloidosis (ATTR) is an inherited,
progressively debilitating, and often fatal disease caused by
mutations in the TTR gene. TTR protein is produced primarily in the
liver and is normally a carrier of vitamin A. 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. ATTR represents a major unmet
medical need with significant morbidity and mortality; familial
amyloidotic polyneuropathy (FAP) affects approximately 10,000
people worldwide and familial amyloidotic cardiomyopathy (FAC) is
estimated to affect at least 40,000 people worldwide. FAP 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). FAC is fatal
within 2.5 to 5 years of diagnosis and treatment is currently
limited to supportive care. 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 TTR amyloid
polyneuropathy and/or cardiomyopathy.
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 with a core focus on RNAi therapeutics as genetic
medicines, including programs as part of the company’s “Alnylam
5x15™” product strategy. Alnylam’s genetic medicine programs are
RNAi therapeutics directed toward genetically defined targets for
the treatment of serious, life-threatening diseases with limited
treatment options for patients and their caregivers. These include:
patisiran (ALN-TTR02), an intravenously delivered RNAi therapeutic
targeting transthyretin (TTR) for the treatment of TTR-mediated
amyloidosis (ATTR) in patients with familial amyloidotic
polyneuropathy (FAP); ALN-TTRsc, a subcutaneously delivered RNAi
therapeutic targeting TTR for the treatment of ATTR in patients
with TTR cardiac amyloidosis, including familial amyloidotic
cardiomyopathy (FAC) and senile systemic amyloidosis (SSA);
ALN-AT3, an RNAi therapeutic targeting antithrombin (AT) for the
treatment of hemophilia and rare bleeding disorders (RBD); ALN-CC5,
an RNAi therapeutic targeting complement component C5 for the
treatment of complement-mediated diseases; ALN-AS1, an RNAi
therapeutic targeting aminolevulinic acid synthase-1 (ALAS-1) for
the treatment of hepatic porphyrias including acute intermittent
porphyria (AIP); ALN-PCS, an RNAi therapeutic targeting PCSK9 for
the treatment of hypercholesterolemia; ALN-AAT, an RNAi therapeutic
targeting alpha-1 antitrypsin (AAT) for the treatment of AAT
deficiency-associated liver disease; ALN-HBV, an RNAi therapeutic
targeting the hepatitis B virus (HBV) genome for the treatment of
HBV infection; ALN-TMP, an RNAi therapeutic targeting TMPRSS6 for
the treatment of beta-thalassemia and iron-overload disorders;
ALN-ANG, an RNAi therapeutic targeting angiopoietin-like 3
(ANGPTL3) for the treatment of genetic forms of mixed
hyperlipidemia and severe hypertriglyceridemia; ALN-AC3, an RNAi
therapeutic targeting apolipoprotein C-III (apoCIII) for the
treatment of hypertriglyceridemia; ALN-AGT, an RNAi therapeutic
targeting angiotensinogen (AGT) for the treatment of hypertensive
disorders of pregnancy (HDP), including preeclampsia; ALN-GO1, an
RNAi therapeutic targeting glycolate oxidase (GO) for the treatment
of primary hyperoxaluria type 1 (PH1); and other programs yet to be
disclosed. As part of its “Alnylam 5x15” strategy, as updated in
early 2014, the company expects to have six to seven genetic
medicine product candidates in clinical development – including at
least two programs in Phase 3 and five to six programs with human
proof of concept – by the end of 2015. The company’s demonstrated
commitment to RNAi therapeutics has enabled it to form major
alliances with leading companies including Merck, Medtronic,
Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, Cubist,
GlaxoSmithKline, Ascletis, Monsanto, and The Medicines Company. In
early 2014, Alnylam and Genzyme, a Sanofi company, formed a
multi-product geographic alliance on Alnylam's genetic medicine
programs in the rare disease field. Specifically, Alnylam will lead
development and commercialization of programs in North America and
Europe, while Genzyme will develop and commercialize products in
the rest of world. In addition, Alnylam and Genzyme will co-develop
and co-commercialize ALN-TTRsc in North America and Europe. In
March 2014, Alnylam acquired Sirna Therapeutics, a wholly owned
subsidiary of Merck. 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,
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 expectations regarding the Alnylam Assist program, its
views with respect to the potential for RNAi therapeutics,
including patisiran for the treatment of FAP, its expectations
regarding its “Alnylam 5x15” product strategy, and its plans
regarding 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 may differ materially from those indicated by
these forward-looking statements as a result of various important
factors, including, without limitation, Alnylam’s ability to manage
operating expenses, Alnylam’s ability to discover and develop novel
drug candidates and delivery approaches, successfully demonstrate
the efficacy and safety of its drug candidates, the pre-clinical
and clinical results for its product candidates, which may not
support further development of product candidates, actions of
regulatory agencies, which may affect the initiation, timing and
progress of clinical trials, obtaining, maintaining and protecting
intellectual property, Alnylam’s ability to enforce its patents
against infringers and defend its patent portfolio against
challenges from third parties, obtaining regulatory approval for
products, competition from others using technology similar to
Alnylam’s and others developing products for similar uses,
Alnylam’s ability to 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, marketing, sales and
distribution of products, the outcome of litigation, 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 to
update any forward-looking statements.
Alnylam Pharmaceuticals, Inc.Cynthia Clayton,
617-551-8207Vice President, Investor Relations andCorporate
CommunicationsorMedia:SpectrumLiz Bryan, 202-955-6222 x2526
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