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Alnylam Receives Orphan Drug Designation from U.S. Food & Drug Administration for ALN-AT3, an RNAi Therapeutic for the Treatment

  Alnylam Receives Orphan Drug Designation from U.S. Food & Drug
  Administration for ALN-AT3, an RNAi Therapeutic for the Treatment of
  Hemophilia

Business Wire

CAMBRIDGE, Mass. -- August 14, 2013

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics
company, announced today that the U.S. Food & Drug Administration (FDA) has
granted an Orphan Drug Designation to ALN-AT3 as a therapeutic for the
treatment of hemophilia B. Alnylam is developing ALN-AT3, a subcutaneously
administered RNAi therapeutic targeting antithrombin (AT), for the treatment
of hemophilia – including hemophilia A, hemophilia B, and hemophilia A or B
with “inhibitors” – and other Rare Bleeding Disorders (RBD).

“We are very pleased to have received Orphan Drug Designation from the FDA for
ALN-AT3, a key program in our ‘Alnylam 5x15’ product development and
commercialization strategy. We believe that our subcutaneously delivered RNAi
therapeutic represents an innovative approach for the management of hemophilia
and has great potential to make a meaningful impact in the treatment of this
often debilitating bleeding disorder,” said Saraswathy (Sara) Nochur, Ph.D.,
Senior Vice President, Regulatory Affairs and Quality Assurance at Alnylam.
“We look forward to advancing this important program towards the clinic in the
months to come.”

At the recent Congress of the International Society on Thrombosis and
Haemostasis, Alnylam presented pre-clinical data demonstrating that ALN-AT3
can normalize thrombin generation and improve hemostasis in hemophilia mice
and can fully correct thrombin generation in a non-human primate (NHP)
hemophilia “inhibitor” model. ALN-AT3 utilizes the company’s proprietary
GalNAc conjugate delivery platform, enabling subcutaneous dose administration.
Alnylam plans to file an investigational new drug (IND) application for
ALN-AT3 in the fourth quarter of 2013 and initiate a Phase I clinical trial in
early 2014.

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. OOPD 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.

About Hemophilia and Rare Bleeding Disorders (RBD)

Hemophilias are hereditary disorders caused by genetic deficiencies of various
blood clotting factors, resulting in recurrent bleeds into joints, muscles,
and other major internal organs. Hemophilia A is defined by loss-of-function
mutations in factor VIII, and there are greater than 40,000 people in the U.S.
and E.U. Hemophilia B, defined by loss-of-function mutations in factor IX,
affects greater than 9,500 people in the U.S. and E.U. Other Rare Bleeding
Disorders (RBD) are defined by congenital deficiencies of other blood
coagulation factors, including Factors II, V, VII, X, and XI, and there are
about 1,000 people worldwide with a severe bleeding phenotype. Standard
treatment for people with hemophilia involves replacement of the missing
clotting factor either as prophylaxis or on-demand therapy. However, as many
as one third of people with hemophilia A will develop an antibody to their
replacement factor – a very serious complication; these 'inhibitor' subjects
become refractory to standard replacement therapy. There exists a small subset
of people with hemophilia who have co-inherited a prothrombotic mutation, such
as factor V Leiden, antithrombin deficiency, protein C deficiency, and
prothrombin G20210A. People with hemophilia that have co-inherited these
prothrombotic mutations are characterized as having a later onset of disease,
lower risk of bleeding, and reduced requirements for factor VIII or factor IX
treatment as part of their disease management. There exists a significant need
for novel therapeutics to treat hemophilia and RBD.

About Antithrombin (AT)

Antithrombin (AT, also known as “antithrombin III” and “SERPINC1”) is a liver
expressed plasma protein and member of the “serpin” family of proteins that
acts as an important endogenous anticoagulant by inactivating factor Xa and
thrombin. AT plays a key role in normal hemostasis, which has evolved to
balance the need to control blood loss through clotting with the need to
prevent pathologic thrombosis through anticoagulation. In hemophilia, the loss
of certain procoagulant factors (Factor VIII and Factor IX, in the case of
hemophilia A and B, respectively) results in an imbalance of the hemostatic
system toward a bleeding phenotype. In contrast, in thrombophilia (e.g.,
factor V Leiden, protein C deficiency, antithrombin deficiency, amongst
others), certain mutations result in an imbalance in the hemostatic system
toward a thrombotic phenotype. Since co-inheritance of prothrombotic mutations
may ameliorate the clinical phenotype in hemophilia, inhibition of AT defines
a novel strategy for improving hemostasis.

About RNA Interference (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
toward genetically defined targets for the treatment of serious,
life-threatening diseases with limited treatment options for patients and
their caregivers. These include: 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 familial amyloidotic cardiomyopathy (FAC);
ALN-AT3, an RNAi therapeutic targeting antithrombin (AT) for the treatment of
hemophilia and rare bleeding disorders (RBD); ALN-AS1, an RNAi therapeutic
targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of porphyria
including acute intermittent porphyria (AIP); ALN-PCS, an RNAi therapeutic
targeting PCSK9 for the treatment of hypercholesterolemia; ALN-TMP, an RNAi
therapeutic targeting TMPRSS6 for the treatment of beta-thalassemia and
iron-overload disorders; ALN-AAT, an RNAi therapeutic targeting
alpha-1-antitrypsin (AAT) for the treatment of AAT deficiency liver disease;
and ALN-CC5, an RNAi therapeutic targeting complement component C5 for the
treatment of complement-mediated diseases, amongst other programs. As part of
its “Alnylam 5x15^TM” strategy, the company expects to have five RNAi
therapeutic products for genetically defined diseases in clinical development,
including programs in advanced stages, on its own or with a partner by the end
of 2015. Alnylam has additional partnered programs in clinical or development
stages, including ALN-RSV01 for the treatment of respiratory syncytial virus
(RSV) infection and ALN-VSP for the treatment of liver cancers. The company’s
leadership position on RNAi therapeutics and intellectual property have
enabled it to form major alliances with leading companies including Merck,
Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, Cubist,
Ascletis, Monsanto, Genzyme, and The Medicines Company. In addition, Alnylam
holds an equity position in Regulus Therapeutics Inc., a company focused on
discovery, development, and commercialization of microRNA therapeutics.
Alnylam has also formed Alnylam Biotherapeutics, a division of the company
focused on the development of RNAi technologies for applications in biologics
manufacturing, including recombinant proteins and monoclonal antibodies.
Alnylam’s VaxiRNA™ platform applies RNAi technology to improve the
manufacturing processes for vaccines; GlaxoSmithKline is a collaborator in
this effort. Alnylam scientists and collaborators have published their
research on RNAi therapeutics in over 100 peer-reviewed papers, including many
in the world’s top scientific journals such as Nature, Nature Medicine, Nature
Biotechnology, and Cell. Founded in 2002, Alnylam maintains headquarters in
Cambridge, Massachusetts. For more information, please visit www.alnylam.com.

About “Alnylam 5x15™”

The “Alnylam 5x15” strategy, launched in January 2011, establishes a path for
development and commercialization of novel RNAi therapeutics toward
genetically defined targets for the treatment of diseases with high unmet
medical need. Products arising from this initiative share several key
characteristics including: a genetically defined target and disease; the
potential to have a major impact in a high unmet need population; the ability
to leverage the existing Alnylam RNAi delivery platform; the opportunity to
monitor an early biomarker in Phase I clinical trials for human proof of
concept; and the existence of clinically relevant endpoints for the filing of
a new drug application (NDA) with a focused patient database and possible
accelerated paths for commercialization. By the end of 2015, the company
expects to have five such RNAi therapeutic programs in clinical development,
including programs in advanced stages, on its own or with a partner. The
“Alnylam 5x15” programs include: 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 familial amyloidotic cardiomyopathy (FAC);
ALN-AT3, an RNAi therapeutic targeting antithrombin (AT) for the treatment of
hemophilia and rare bleeding disorders (RBD); ALN-AS1, an RNAi therapeutic
targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of porphyria
including acute intermittent porphyria (AIP); ALN-PCS, an RNAi therapeutic
targeting PCSK9 for the treatment of hypercholesterolemia; ALN-TMP, an RNAi
therapeutic targeting TMPRSS6 for the treatment of beta-thalassemia and
iron-overload disorders; ALN-AAT, an RNAi therapeutic targeting
alpha-1-antitrypsin (AAT) for the treatment of AAT deficiency liver disease;
and ALN-CC5, an RNAi therapeutic targeting complement component C5 for the
treatment of complement-mediated diseases, amongst other programs. Alnylam
intends to focus on developing and commercializing certain programs from this
product strategy itself in North and South America, Europe, and other parts of
the world; these include ALN-TTR, ALN-AT3, ALN-AS1, and ALN-CC5.

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-AT3 for the treatment of
hemophilia and Rare Bleeding Disorders, its expectations with respect to the
timing and success of its clinical and pre-clinical trials, the expected
timing of regulatory filings, including its plan to file an IND application
and initiate clinical trials for ALN-AT3, its plans to seek a partner for
certain ‘Alnylam 5x15’ programs, 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 current report on Form 10-Q filed with the
Securities and Exchange Commission (SEC) on August 9, 2013 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.

Contact:

Alnylam Pharmaceuticals, Inc.
Cynthia Clayton, 617-551-8207
Vice President, Investor Relations and Corporate Communications
or
Spectrum
Amanda Sellers (Media), 202-955-6222 x2597
 
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