–Marks First Product Opt-In since Formation of
Landmark 2014 Alliance for Global Advancement of RNAi Therapeutics
as Genetic Medicines–
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi
therapeutics company, and Genzyme, a Sanofi company (EURONEXT:SAN
and NYSE:SNY), today announced that Genzyme has elected to opt into
Alnylam’s investigational ALN-AT3 hemophilia program for
development and potential future commercialization in territories
outside of North America and Western Europe. This marks the first
product from Alnylam’s Genetic Medicines pipeline to which Genzyme
has opted in since the formation of the companies’ global alliance
in January 2014, and the third product opt-in overall. Genzyme’s
opt-in decision was based on encouraging clinical data from the
Phase 1 trial of ALN-AT3, including positive interim data that were
presented at the International Society on Thrombosis and
Haemostasis (ISTH) 2015 Congress in June 2015.
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“Our collaboration with Genzyme is a key part of our strategy to
advance RNAi therapeutics to global markets. Genzyme’s proven track
record in developing and commercializing therapies for rare
diseases makes them an ideal partner to advance an innovative
medicine for the treatment of hemophilia. Accordingly, we are very
pleased that they have elected to opt into the ALN-AT3 program, the
first product opt-in since formation of our landmark alliance,”
said John Maraganore, Ph.D., Chief Executive Officer of Alnylam.
“We believe that ALN-AT3 holds tremendous promise as a new
investigational medicine for the management of hemostasis in
hemophilia and rare bleeding disorders. We look forward to working
with Genzyme to advance ALN-AT3 as a potential new treatment option
for people with hemophilia around the world.”
“ALN-AT3 represents a unique and promising new approach for the
potential treatment of hemophilia,” said David P. Meeker, M.D.,
President and CEO of Genzyme, a Sanofi company. “We are excited to
expand our clinical stage pipeline of treatments for patients with
rare diseases, and to broaden our relationship with Alnylam.”
In January 2014, Alnylam and Genzyme 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,
i.e., programs in Alnylam’s Genetic Medicine Strategic Therapeutic
Area. Alnylam retains product rights in North
America and Western Europe, while Genzyme obtained the
right to access certain programs in Alnylam's current and future
Genetic Medicines pipeline in the rest of the world (ROW) through
the end of 2019, together with certain broader
co-development/co-commercialization rights and global rights for
certain products. Alnylam maintains development and
commercialization control for all programs in its territory.
ALN-AT3 is the third Alnylam product for which Genzyme has
exercised its opt-in right, the first two occurring at the close of
the deal in early 2014 for patisiran and revusiran, investigational
RNAi therapeutics for the treatment of transthyretin-mediated
amyloidosis. In the case of ALN-AT3, Genzyme has elected presently
to opt into the program for its ROW rights. Genzyme retains its
future opt-in right to co-develop and co-promote ALN-AT3 with
Alnylam in North America and Western Europe. Specifically, Genzyme
has the right to either co-develop and co-promote ALN-AT3
in Alnylam's territory – with Alnylam maintaining development and
commercialization control – or to maintain its ROW rights
for ALN-AT3 and, if exercised by Genzyme, obtain a global license
to ALN-AS1, Alnylam’s investigational RNAi therapeutic for the
treatment of acute hepatic porphyrias. Genzyme will exercise this
selection right upon completion of human proof-of-concept for the
ALN-AS1 program, which is expected to occur in 2016.
Per the 2014 agreement, Alnylam will receive R&D funding for
programs where Genzyme has elected to opt in for development and
commercialization. For "regional" programs where Genzyme will
develop and commercialize in their ROW territory, such as patisiran
and ALN-AT3 as currently structured, Genzyme pays 20% of global
development costs. In the case of ALN-AT3, such cost sharing is
expected to begin in January 2016. For "co-develop/co-promote"
programs such as revusiran (and possibly ALN-AT3 in the future),
Genzyme pays 50% of global development costs. For "global" programs
(e.g., possibly ALN-AS1 if selected), Genzyme will pay 100% of
global development costs. In addition, Alnylam is eligible to
receive milestones totaling up to $75 million per product
for regional and co-develop/co-promote programs. In the case of
global Genzyme programs, Alnylam is eligible to receive up
to $200 million in milestones per product. Finally,
Alnylam is also eligible to receive tiered double-digit royalties
up to 20% on net sales on all products commercialized by Genzyme in
its territories. In the case of Genzyme's co-develop/co-promote
products in the Alnylam territory, the parties will share profits
equally and Alnylam will book net sales revenues.
About ALN-AT3 for HemophiliaHemophilias are hereditary
disorders caused by genetic deficiencies of various blood clotting
factors, resulting in recurrent bleeds into joints, muscles, and
other major internal organs. 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 severe hemophilia A will develop an
antibody to their replacement factor. These ‘inhibitor’ subjects
become refractory to standard replacement factor therapy, and
therefore are significantly more complicated to manage and have
poorer clinical outcomes.
ALN-AT3 is an investigational, subcutaneously administered RNAi
therapeutic for the treatment of hemophilia and other rare bleeding
disorders. ALN-AT3 is aimed at correcting coagulation defects by
knockdown of antithrombin (AT) – an important endogenous
anticoagulant. AT acts as a “brake” on the production of thrombin,
a protein essential for the formation of a blood clot. ALN-AT3 is
being evaluated in a Phase 1 study in people with
moderate-to-severe hemophilia. A pivotal Phase 3 clinical
trial is planned to start in mid-2016.
About RNAiRNAi (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 PharmaceuticalsAlnylam 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.
About Genzyme, a Sanofi CompanyGenzyme has pioneered the
development and delivery of transformative therapies for patients
affected by rare and debilitating diseases for over 30 years. We
accomplish our goals through world-class research and with the
compassion and commitment of our employees. With a focus on rare
diseases and multiple sclerosis, we are dedicated to making a
positive impact on the lives of the patients and families we serve.
That goal guides and inspires us every day. Genzyme’s portfolio of
transformative therapies, which are marketed in countries around
the world, represents groundbreaking and life-saving advances in
medicine. As a Sanofi company, Genzyme benefits from the reach and
resources of one of the world’s largest pharmaceutical companies,
with a shared commitment to improving the lives of patients. Learn
more at www.genzyme.com.
Genzyme® is a registered trademark of Genzyme Corporation. All
rights reserved.
About SanofiSanofi, a global healthcare leader,
discovers, develops and distributes therapeutic solutions focused
on patients’ needs. Sanofi has core strengths in the field of
healthcare with seven growth platforms: diabetes solutions, human
vaccines, innovative drugs, consumer healthcare, emerging markets,
animal health and the new Genzyme. Sanofi is listed in Paris
(EURONEXT: SAN) and in New York (NYSE: SNY).
Alnylam Forward Looking StatementsVarious 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 ALN-AT3
for the treatment of hemophilia and rare bleeding disorders,
expectations regarding the reporting of data from clinical studies,
including completion of human proof-of-concept for ALN-AS1, its
expectations regarding Genzyme's participation in the development
and commercialization of RNAi therapeutics, its expectations
regarding the receipt of potential R&D payments, development,
regulatory and sales milestones and royalties from
Genzyme, expectations regarding its STAr pipeline growth
strategy, and its plans regarding commercialization of RNAi
therapeutics, including ALN-AT3, 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 be
replicated or continue to occur in other subjects or in additional
studies or otherwise 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 manage operating expenses,
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.
Sanofi Forward Looking StatementsThis press release
contains forward-looking statements as defined in the Private
Securities Litigation Reform Act of 1995, as amended.
Forward-looking statements are statements that are not historical
facts. These statements include projections and estimates and their
underlying assumptions, statements regarding plans, objectives,
intentions and expectations with respect to future financial
results, events, operations, services, product development and
potential, and statements regarding future performance.
Forward-looking statements are generally identified by the words
"expects", "anticipates", "believes", "intends", "estimates",
"plans" and similar expressions. Although Sanofi's management
believes that the expectations reflected in such forward-looking
statements are reasonable, investors are cautioned that
forward-looking information and statements are subject to various
risks and uncertainties, many of which are difficult to predict and
generally beyond the control of Sanofi, that could cause actual
results and developments to differ materially from those expressed
in, or implied or projected by, the forward-looking information and
statements. These risks and uncertainties include among other
things, the uncertainties inherent in research and development,
future clinical data and analysis, including post marketing,
decisions by regulatory authorities, such as the FDA or the EMA,
regarding whether and when to approve any drug, device or
biological application that may be filed for any such product
candidates as well as their decisions regarding labelling and other
matters that could affect the availability or commercial potential
of such product candidates, the absence of guarantee that the
product candidates if approved will be commercially successful, the
future approval and commercial success of therapeutic alternatives,
the Group's ability to benefit from external growth opportunities,
trends in exchange rates and prevailing interest rates, the impact
of cost containment policies and subsequent changes thereto, the
average number of shares outstanding as well as those discussed or
identified in the public filings with the SEC and the AMF made by
Sanofi, including those listed under "Risk Factors" and "Cautionary
Statement Regarding Forward-Looking Statements" in Sanofi's annual
report on Form 20-F for the year ended December 31, 2014. Other
than as required by applicable law, Sanofi does not undertake any
obligation to update or revise any forward-looking information or
statements.
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version on businesswire.com: http://www.businesswire.com/news/home/20151001005526/en/
Alnylam Pharmaceuticals, Inc.Michael Mason,
617-551-8327Vice President, Finance & TreasurerorSpectrumLiz
Bryan (Media), 202-955-6222 x2526orSanofi Media
RelationsJack Cox, +33 (0)1 53 77 46 46orSanofi Investor
RelationsSébastien Martel, +33 (0)1 53 77 45 45orGenzyme
Media RelationsBo Piela, 617-768-6579
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