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.
Copyright Business Wire 2014