Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), a leading RNAi
therapeutics company, announced today that the European Medicines
Agency (EMA) Committee for Orphan Medicinal Products (COMP) has
adopted a positive opinion recommending ALN-TTRsc for designation
as an orphan medicinal product for the treatment of transthyretin
(TTR)-mediated amyloidosis (ATTR).
“We are very pleased to have received a positive opinion from
the EMA COMP on our application for Orphan Drug Designation for
ALN-TTRsc,” said Saraswathy (Sara) Nochur, Ph.D., Senior Vice
President, Regulatory Affairs and Quality Assurance at Alnylam. “We
believe RNAi therapeutics represent a promising new approach for
the treatment of ATTR, with the potential to make a meaningful
impact for patients with this progressive and debilitating disease.
We look forward to sharing Phase 2 clinical data from our ALN-TTRsc
program later in the year, and, assuming continued positive
results, we plan to advance to a Phase 3 pivotal trial in ATTR
patients with TTR cardiac amyloidosis by the end of the year.”
ALN-TTRsc is currently in a pilot Phase 2 clinical trial for the
treatment of ATTR patients with TTR cardiac amyloidosis; this study
is aimed at evaluating the tolerability of ALN-TTRsc in
approximately 15 patients. In addition, the study will assess
preliminary clinical activity as measured by knockdown of serum TTR
levels and additional exploratory tests, such as cardiac imaging
(including echocardiography and cardiac MRI), circulating cardiac
biomarkers (NT-proBNP and troponins T and I), 6-minute walk test,
New York Heart Association (NYHA) classification, and measures of
heart failure symptoms and quality of life (Kansas City
Cardiomyopathy Questionnaire and EQ-5D QOL). The company expects to
present data from the Phase 2 trial in late 2014. Patients
completing the Phase 2 trial will be eligible to participate in an
open-label extension (OLE) study for further assessment of general
tolerability and clinical activity with long-term dosing; the
ALN-TTRsc Phase 2 OLE study is expected to be initiated in
mid-2014. Assuming positive results, Alnylam expects to begin a
Phase 3 trial in TTR cardiac amyloidosis patients by the end of
2014.
Orphan Drug Designation by the European Commission provides
regulatory and financial incentives for companies to develop and
market therapies that treat a life-threatening or chronically
debilitating condition affecting no more than five in 10,000
persons in the European Union (EU), and where no satisfactory
treatment is available. In addition to a 10-year period of
marketing exclusivity in the EU after product approval, Orphan Drug
Designation provides incentives for companies seeking protocol
assistance from the EMA during the product development phase, and
direct access to centralized marketing authorization.
In January 2014, Genzyme and Alnylam 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 Genzyme obtains the right to access Alnylam’s current “5x15”
and future genetic medicines pipeline in the rest of the world
(ROW), including co-development/co-commercialization and/or global
product rights for certain programs. In the case of ALN-TTRsc,
Alnylam and Genzyme are co-developing and co-commercializing the
product in North America and Western Europe, while Genzyme will
advance the product in the ROW.
About Transthyretin-Mediated Amyloidosis
Transthyretin (TTR)-mediated amyloidosis (ATTR) is an inherited,
progressively debilitating, and fatal disease caused by mutations
in the TTR gene. TTR protein is produced primarily in the liver and
is normally a carrier for retinol binding protein. 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)
affects at least 40,000 people worldwide. FAP patients have a life
expectancy of five to 15 years from symptom onset, and the only
approved treatment options for early stage disease are liver
transplantation, and tafamidis (approved in Europe). The mean
survival for FAC patients is approximately 2.5 years, and there are
no approved therapies. 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
5x15TM” 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 aminolevulinate 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 liver disease; 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; 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, The Medicines Company, and
Genzyme, a Sanofi company. In January 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, the 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 its "Alnylam 5x15" product
strategy, Alnylam’s views with respect to the potential for RNAi
therapeutics, including ALN-TTRsc for the treatment TTR cardiac
amyloidosis, its expectations with respect to the timing and
success of its clinical trials, the expected timing of additional
clinical trials, 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 Annual Report on Form 10-K 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 and Corporate
CommunicationsorMedia:SpectrumAmanda Sellers, 202-955-6222
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