CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam
Pharmaceuticals, Inc. (Nasdaq: ALNY) and Genzyme,
a Sanofi company (EURONEXT: SAN and NYSE: SNY), announced today that
they have formed an exclusive alliance to develop and commercialize RNAi
therapeutics targeting transthyretin (TTR) for the treatment of
transthyretin-mediated amyloidosis (ATTR) in Japan and other
Asia-Pacific countries. ATTR is a rare, debilitating, hereditary disease
that damages the nervous system and heart, resulting in a life
expectancy of 5 to 15 years.
“As we work to build our pipeline through both internal research and
development, and through external collaborations, we look forward to
working with Alnylam on this important program.”
“Our ALN-TTR program holds promise as a breakthrough therapy for the
treatment of ATTR, a debilitating orphan disease. As the lead program in
our ‘Alnylam 5x15’ product strategy, we also view this program as a key
part of building Alnylam for the future,” said John Maraganore, Ph.D.,
Chief Executive Officer of Alnylam. “In this important collaboration,
Genzyme will advance our ALN-TTR program with their proven capabilities
in the Japanese and broader Asian market, while we maintain our plans to
develop and commercialize this potential breakthrough medicine in the
U.S., Europe, and rest of world. In addition, a key part of the value
proposition in this alliance for Alnylam is the potential for
significant royalty payments on sales of products.”
ATTR is an endemic disease in Japan, with a significant number of
patients carrying the V30M TTR mutation which leads to onset of a severe
form of ATTR known as familial amyloidotic polyneuropathy (FAP).
Together, Alnylam and Genzyme intend to maximize the value of ALN-TTR
worldwide by developing the program in FAP and other ATTR indications,
such as familial amyloidotic cardiomyopathy (FAC) and senile systemic
amyloidosis (SSA). Alnylam’s ALN-TTR program currently includes
ALN-TTR02, which is in a Phase II clinical trial, and ALN-TTRsc, a
subcutaneously administered RNAi therapeutic in late stage pre-clinical
development.
Under the terms of the agreement, Genzyme will make an upfront cash
payment of $22.5 million to Alnylam. The agreement also includes
development milestone payments and tiered royalties expected to yield an
effective rate in the mid-teens to mid-twenties on Genzyme’s sales of
ALN-TTR products in their territory. In addition, each party will be
responsible for the development and commercialization activities in
their respective territories.
“We are encouraged by Alnylam’s progress with their ALN-TTR program and
are excited by the potential for this innovative drug candidate to make
a difference in the lives of patients with ATTR. The results to date
demonstrate impressive clinical activity and support advancement of this
promising therapeutic into pivotal studies and toward the market,” said
David Meeker, M.D., President and Chief Executive Officer of Genzyme.
“As we work to build our pipeline through both internal research and
development, and through external collaborations, we look forward to
working with Alnylam on this important program.”
Recently, Alnylam presented positive
clinical results from its ALN-TTR02 Phase I trial demonstrating
robust and unprecedented knockdown of serum TTR protein levels of up to
94%; the overall results were highly significant (p<0.00001 by ANOVA).
Suppression of TTR, the disease-causing protein in ATTR, was found to be
rapid, dose dependent, durable, and specific after just a single dose.
The drug was generally safe and well tolerated in this Phase I study.
Alnylam is currently enrolling patients in a Phase II multi-dose study
of ALN-TTR02 in ATTR patients and aims to initiate a Phase III pivotal
study of ALN-TTR02 by the end of 2013.
About ATTR
Transthyretin (TTR)-mediated amyloidosis (ATTR) is a hereditary,
systemic disease caused by mutations in the TTR gene. TTR protein is
produced primarily in the liver and is normally a carrier for thyroid
hormones and retinol binding proteins. Mutations in TTR cause abnormal
amyloid proteins to accumulate and damage body organs and tissue, such
as the peripheral nerves and heart, resulting in intractable peripheral
sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy. ATTR
represents a major unmet medical need with significant morbidity and
mortality; FAP affects approximately 10,000 people worldwide and FAC
affects at least 40,000 people worldwide. FAP patients have a mean life
expectancy of five to 15 years from symptom onset, and the only
treatment options for early stage disease are liver transplantation and
tafamidis (approved in Europe). As a result, there is a significant need
for novel therapeutics to treat patients who have inherited mutations in
the TTR gene.
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 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, Ascletis, Monsanto and Genzyme. 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, Sanofi,
AstraZeneca and Biogen Idec. 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,
statements regarding Alnylam’s views with respect to the potential for
RNAi therapeutics, including the potential for ALN-TTR02 and ALN-TTRsc,
its expectations regarding the receipt upfront, and potential
development milestone and royalty payments under the Genzyme agreement,
its expectations regarding the market opportunity for ALN-TTR, including
in Japan, its expectations with respect to the timing and success of its
clinical trials for ALN-TTR02, including the possible initiation of a
Phase III pivotal trial, and the expected timing of an IND filing for
ALN-TTRsc, 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, Genzyme’s ability to
successfully advance ALN-TTR02 and/or ALN-TTRsc in Japan and other Asian
countries including China, Australia, and India, as well as Alnylam’s
ability to develop ALN-TTR02 and/or ALN-TTRsc in the rest of the world,
resulting in the potential payment of development milestones and
royalties to Alnylam, Alnylam’s ability to successfully demonstrate the
efficacy and safety of its drug candidates, the pre-clinical and
clinical results for these product candidates, which may not support
further development of such product candidates, actions of regulatory
agencies, which may affect the initiation, timing and progress of
clinical trials for such product candidates, obtaining, maintaining and
protecting intellectual property, obtaining regulatory approval for
products, competition from others using technology similar to Alnylam’s
and others developing products for similar uses, and Alnylam’s ability
to establish and maintain strategic business alliances, including its
collaboration with Genzyme, 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.
About Genzyme, a Sanofi Company
Genzyme 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.
Sanofi Forward Looking Statements
This 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, 2011. Other than as required by applicable
law, Sanofi does not undertake any obligation to update or revise any
forward-looking information or statements.