Alnylam to Present Data from its RNAi Therapeutic Programs Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated

  Alnylam to Present Data from its RNAi Therapeutic Programs Targeting
  Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis (ATTR) at
  XIVth International Symposium on Amyloidosis (ISA)

 – Presentations Include Results of 283-Patient Natural History Study of ATTR
          Patients with Familial Amyloidotic Polyneuropathy (FAP) –

ISA XIV

Business Wire

CAMBRIDGE, Mass. -- March 21, 2014

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics
company, announced today that its scientists and collaborators will present
pre-clinical and clinical data with patisiran (ALN-TTR02) and ALN-TTRsc – RNAi
therapeutics targeting transthyretin (TTR) for the treatment of TTR-mediated
amyloidosis (ATTR) – at the XIV^th International Symposium on Amyloidosis
(ISA) being held April 27 – May 1, 2014 in Indianapolis, Indiana. Amongst
other new data, the company presentations include results from a 283-patient
natural history study of ATTR patients with familial amyloidotic
polyneuropathy (FAP) that document the rate of disease progression.

Presentations and posters from Alnylam scientists and collaborators at the
meeting include:

  *an oral presentation titled “Neuropathy progression rate in patients with
    familial amyloidotic polyneuropathy” in the “ATTR: Diagnosis and
    pathogenesis” plenary session being held on Wednesday, April 30 from 11:30
    a.m. – 1:00 p.m. ET;
  *an oral presentation titled “Further analysis of phase II trial of
    patisiran, a novel RNAi therapeutic for the treatment of familial
    amyloidotic polyneuropathy” in the “ATTR: Prognosis and therapy” plenary
    session being held on Wednesday, April 30 from 2:30 p.m. – 4:30 p.m. ET;
    and,
  *a poster titled “Preclinical Evaluation of RNAi Therapeutics for the
    Treatment of ATTR: An Update” on Wednesday, April 30.

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 as
genetic medicines, including programs as part of the company’s “Alnylam
5x15^TM” 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 agreed to acquire 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.

About “Alnylam 5x15™” and Genetic Medicines

The “Alnylam 5x15” strategy, launched in January 2011, establishes a path for
development and commercialization of novel RNAi therapeutics as genetic
medicines. Alnylam’s genetic medicine programs are RNAi therapeutics directed
toward genetically defined targets for the treatment of diseases with high
unmet medical need. These programs share several key characteristics
including: a genetically defined target and disease expressed in the liver;
the potential to have a major impact in a high unmet need population; the
ability to leverage the existing Alnylam RNAi platform with clinically proven
delivery to the liver; the opportunity to monitor an early biomarker in Phase
1 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.
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 “Alnylam 5x15” programs include: patisiran (ALN-TTR02),
an intravenously delivered RNAi therapeutic targeting transthyretin (TTR) in
development for the treatment of TTR-mediated amyloidosis (ATTR) in patients
with familial amyloidotic polyneuropathy (FAP); ALN-TTRsc, a subcutaneously
delivered RNAi therapeutic targeting TTR in development 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) in development for the treatment of
hemophilia and rare bleeding disorders (RBD); ALN-CC5, an RNAi therapeutic
targeting complement component C5 in development for the treatment of
complement-mediated diseases; ALN-AS1, an RNAi therapeutic targeting
aminolevulinate synthase-1 (ALAS-1) in development for the treatment of
hepatic porphyrias including acute intermittent porphyria (AIP); ALN-PCS, an
RNAi therapeutic targeting PCSK9 in development 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 in development 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. In 2014, Alnylam and Genzyme, a Sanofi
company, formed a multi-product geographic alliance on Alnylam’s genetic
medicine programs. 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.

Alnylam Forward-Looking Statements

Various statements in this press release concerning Alnylam’s future
expectations, plans and prospects, including without limitation, Alnylam’s
views with respect to the potential for RNAi therapeutics, including patisiran
(ALN-TTR02) and ALN-TTRsc, and its plans to present pre-clinical and clinical
data regarding its RNAi therapeutic programs targeting TTR for the treatment
of ATTR, 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 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.

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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