Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), a leading RNAi
therapeutics company, announced today that the United States Food
& Drug Administration (FDA) has granted Orphan Drug Designation
to revusiran, an investigational RNAi therapeutic, for the
treatment of transthyretin (TTR)-mediated amyloidosis (ATTR
amyloidosis). Revusiran is currently in Phase 3 development for the
treatment of Familial Amyloidotic Cardiomyopathy (FAC), one of the
predominant clinical manifestations of ATTR amyloidosis.
"We are very pleased to have received Orphan Drug Designation
from the FDA for revusiran, a key program in our Genetic Medicines
pipeline. We believe RNAi therapeutics represent a promising new
approach for the treatment of ATTR amyloidosis, with the potential
to make a meaningful impact for patients with this progressive and
debilitating disease," said Saraswathy (Sara) Nochur, Ph.D.,
Senior Vice President, Regulatory Affairs and Quality Assurance at
Alnylam. "We look forward to the continued advancement of
revusiran, including enrollment in our ENDEAVOUR Phase 3 trial in
ATTR amyloidosis patients with FAC. In addition, we continue dosing
TTR cardiac amyloidosis patients in our Phase 2 open-label
extension study with revusiran, and plan to present initial data
from that study in late 2015.”
Revusiran is currently enrolling FAC patients in the ENDEAVOUR
Phase 3 trial, a randomized, double-blind, placebo-controlled,
global study designed to evaluate the efficacy and safety of
revusiran in patients with FAC. The co-primary endpoints of the
study are the change compared to baseline in 6-minute walk distance
(6-MWD) and the percent reduction in serum TTR between placebo- and
revusiran-treated patients at 18 months. Secondary endpoints
include a composite endpoint of cardiovascular mortality and
cardiovascular hospitalization, New York Heart Association (NYHA)
class, Kansas City Cardiomyopathy Questionnaire (KCCQ), CV
mortality, CV hospitalization and all-cause mortality. The trial is
designed to enroll up to 200 FAC patients with a documented TTR
mutation, including V122I or other mutations, in addition to
amyloid deposits as identified by biopsy. Patients are being
randomized 2:1, revusiran:placebo, with revusiran administered
subcutaneously at 500 mg daily for five days then weekly for 18
months. The trial design was informed by natural history data which
showed a mean decline of 140 meters in 6MWD over an 18-month period
in FAC patients with mild-to-moderate heart failure. The ENDEAVOUR
study was designed with 90% power to detect as little as a 39%
difference in the 18-month change from baseline for 6-MWD between
treatment groups, with a significance level of p < 0.05. An
unblinded interim analysis for futility may be conducted when 50%
of patients reach 18 months. All patients completing the ENDEAVOUR
Phase 3 study will be eligible to enroll in a Phase 3 open-label
extension (OLE) study.
The FDA Office of Orphan Products Development (OOPD)
mission is to advance the evaluation and development of products
that demonstrate promise for the diagnosis and/or treatment of rare
diseases or conditions. The Orphan Drug Act provides incentives for
sponsors to develop products for rare diseases. The Orphan Drug
Designation program provides orphan status to drugs and biologics
which are defined as those intended for the safe and effective
treatment, diagnosis or prevention of rare diseases/disorders that
affect fewer than 200,000 people in the U.S.
In April 2014, the European Medicines Agency (EMA) Committee for
Orphan Medicinal Products (COMP) adopted a positive opinion
recommending revusiran for designation as an orphan medicinal
product for the treatment of ATTR amyloidosis.
In January 2014, Alnylam and Genzyme, a Sanofi company, 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 certain programs in Alnylam’s current and future Genetic
Medicines pipeline in the rest of the world (ROW), including
co-development/co-commercialization and/or global product rights in
certain defined instances. In the case of revusiran, Alnylam and
Genzyme will co-develop/co-commercialize the product in North
America and Western Europe, while Genzyme will advance the product
in the ROW.
About ATTR
Transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis) 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. 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 Merck, Medtronic,
Novartis, Biogen, Roche, Takeda, Kyowa Hakko Kirin, Cubist,
GlaxoSmithKline, Ascletis, Monsanto, The Medicines Company, and
Genzyme, a Sanofi company. 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 for the treatment of FAC, its
expectations with respect to the design, timing, and success of its
clinical trials, including with revusiran, its expectations
regarding the presentation of data from clinical trials with
revusiran, its expectations regarding the potential market
opportunity for revusiran, its expectations regarding its STAr
pipeline growth 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.
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Alnylam Pharmaceuticals, Inc.Michael Mason,
617-551-8327Vice President, Finance and
TreasurerorMedia:SpectrumLiz Bryan, 202-955-6222 ext. 2526
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