Alnylam Pharmaceuticals, Inc. : Alnylam Earns Milestone Payment from GlaxoSmithKline in VaxiRNA Collaboration on Flu Vaccine Production
07/30/2012| 08:10am US/Eastern

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Alnylam
Pharmaceuticals, Inc. (Nasdaq: ALNY), a
leading RNAi therapeutics company, announced today that it has earned a
development milestone payment from GlaxoSmithKline (GSK), totaling $3.2
million. The milestone payment is related to progress in the companies'
collaboration on the use of VaxiRNA? technology for the production of
GSK influenza vaccine. Alnylam's VaxiRNA platform uses RNAi technology
for the enhanced manufacturing of vaccine products.
"We have made important progress with GSK in advancing Alnylam's VaxiRNA
technology, and are pleased to have achieved a key milestone in our
collaborative effort," said Rachel Meyers, Ph.D., Vice President,
Research and RNAi Lead Development. "At Alnylam, we continue to pioneer
innovative applications of our RNAi platform that could have a
transformative impact in medicine and the biopharmaceutical industry at
large. Our VaxiRNA platform is representative of this strategy, where
RNAi technology is being used to increase virus titers in the production
of vaccine products. With GSK, we are applying RNAi technology with the
aim to enhance influenza vaccine production in cell culture. We look
forward to continuing our efforts in this strategic collaboration."
Alnylam's VaxiRNA platform applies siRNA for the silencing of specific
genes that limit or prevent efficient manufacturing of vaccine antigens,
including those produced in cell culture and in eggs. The VaxiRNA
platform stems from work Alnylam has performed as part of its Alnylam
Biotherapeutics efforts, where RNAi technology has been applied to
improve the manufacturing of biotherapeutic products, such as
recombinant proteins and monoclonal antibodies. Alnylam and GSK formed
their collaboration in the fourth quarter of 2011. The effort is focused
initially on influenza vaccine production in cell culture systems. Under
the terms of the agreement, GSK will provide research funding and
certain milestone payments to Alnylam, such as the $3.2 million received
this quarter. If successfully applied in the manufacturing of commercial
product, Alnylam will receive payments on unit product sales of
commercialized vaccine product. In addition, GSK has obtained an option
for VaxiRNA applications toward two additional vaccine products.
About VaxiRNA?
Alnylam is applying RNAi technology to improve the manufacturing
processes for vaccines in an effort called VaxiRNA. With VaxiRNA,
Alnylam is using siRNA that silence specific genes in vaccine production
systems, such as cells or eggs, which limit or prevent the efficient
manufacture of vaccine products. New innovations in vaccine
manufacturing are needed to enable the scale and speed of global
immunization for a number of pathogens. In 2011, Alnylam formed a
VaxiRNA partnership with GlaxoSmithKline for influenza vaccine
production.
About RNA Interference (RNAi)
RNAi (RNA interference) represents a breakthrough in understanding how
genes are turned on and off in cells, and adds a new approach to drug
discovery and development. Its discovery represents a promising and
rapidly advancing frontier 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. This process can be used to enhance the manufacturing
of vaccines or medicines. In addition, 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 might be
possible in the future. 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 for the treatment of genetically defined
diseases, including ALN-TTR for the treatment of transthyretin-mediated
amyloidosis (ATTR), ALN-AT3 for the treatment of hemophilia, ALN-PCS for
the treatment of severe hypercholesterolemia, ALN-HPN for the treatment
of refractory anemia, and ALN-TMP for the treatment of
hemoglobinopathies. As part of its "Alnylam 5x15TM" 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, ALN-VSP for the treatment of liver cancers, and
ALN-HTT for the treatment of Huntington's disease. 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, and Ascletis. In addition, Alnylam and Isis co-founded
Regulus Therapeutics Inc., a company focused on discovery, development,
and commercialization of microRNA therapeutics; Regulus has formed
partnerships with GlaxoSmithKline and Sanofi. 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.
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 VaxiRNA to apply RNAi
technology to improve the manufacturing process for vaccines, its
expectations regarding the receipt of research funding, milestone
payments and payments on unit product sales under its collaboration with
GSK, as well as Alnylam's expectations with respect to 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 successfully apply RNAi technology to improve the
manufacturing process for vaccines, including in connection with its GSK
collaboration, demonstrate the efficacy and safety of its drug
candidates in pre-clinical and human clinical trials, and establish and
maintain strategic business alliances and new business initiatives, as
well as those risks more fully discussed in the "Risk Factors" section
of its most recent quarterly report on Form 10-Q on file with the
Securities and Exchange Commission. 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
does not assume any obligation to update any forward-looking statements.

Alnylam Pharmaceuticals, Inc.
Cynthia Clayton, 617-551-8207
Vice
President, Investor Relations and
Corporate Communications
or
Spectrum
Amanda
Sellers (Media), 202-955-6222 x2597
© Business Wire 2012
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