Alnylam’s McSwiggen Patent Upheld in European Opposition Proceedings

  Alnylam’s McSwiggen Patent Upheld in European Opposition Proceedings

Business Wire

CAMBRIDGE, Mass. -- April 7, 2014

Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), a leading RNAi therapeutics
company, announced today that the European Patent Office (EPO) has upheld the
McSwiggen EP 1423406 (’406) patent in oral opposition proceedings in Munich,
Germany. The McSwiggen patent estate broadly describes chemical modifications
of RNAi therapeutics that the company believes are critical for achieving
“drug-like” properties in siRNA, the molecules that mediate RNAi. The patent
is owned by Alnylam, and was recently obtained through the company’s
acquisition of Sirna Therapeutics from Merck. The McSwiggen patent family
comprises a core component of Alnylam’s overall intellectual property (IP)
estate for the advancement of RNAi therapeutics as a new class of medicines.

“We are pleased with the European Patent Office’s decision to uphold the ’406
patent from our McSwiggen patent family, a key component of the IP estate we
recently obtained through our acquisition of Sirna Therapeutics from Merck.
This patent has broad and significant claims describing chemical modifications
of RNAi therapeutics, which we believe are critical for the development and
commercialization of all RNAi therapeutics,” said Laurence Reid, Ph.D., Senior
Vice President and Chief Business Officer of Alnylam. “Our IP estate remains a
cornerstone in our efforts to advance RNAi therapeutics to patients in need.”

The McSwiggen patent family includes 21 granted or issued patents around the
world, including patents in the U.S. (7,923,547; 7,956,176; 7,989,612;
8,202,979; 8,232,383; 8,236,944; 8,242,257; 8,268,986; 8,273,866) and the EU
(1423406; 1458741; 1627061; 2278004; 2287306), and generally describes the
chemical modification of siRNA. The claims for the McSwiggen ’406 patent cover
compositions for siRNA, including, in general terms, a chemically modified
double-stranded RNA that down regulates expression of a target gene by RNAi,
wherein:

  *each strand is independently 18 to 24 nucleotides in length and each
    duplex comprises 17 to 23 base pairs; and
  *10 or more pyrimidine nucleotides of the sense and/or antisense strand are
    chemically modified with 2'-deoxy, 2'-OMe, or 2'F with one or more
    phosphorothioate and/or a terminal cap at the 5', 3' or both, present in
    the same or different strand.

Granted claims of the ’406 patent as well as other granted Alnylam owned or
licensed patents are provided on the company’s website, and in aggregate
broadly cover siRNA and their use in a wide range of lengths from 15 to 49
nucleotides, and chemical modifications with naturally or non-naturally
occurring nucleotides, including, for example acyclic nucleotides such as
those termed “unlocked nucleobase analogs.” In addition, Alnylam’s owned or
licensed patents broadly cover delivery of RNAi therapeutics, including those
that employ GalNAc-siRNA conjugate technology. Finally, Alnylam’s IP estate
also includes patents that broadly cover siRNA toward a wide range of disease
targets.

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^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 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, 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 views with regard
to the scope of its IP estate and Alnylam’s expectations regarding its
“Alnylam 5x15” product strategy, 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.

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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