PARIS and SOUTH SAN FRANCISCO, Calif., Sept. 17, 2014 /PRNewswire/ -- Sanofi (EURONEXT:
SAN and NYSE: SNY) and MyoKardia, Inc., a privately-held
company leading the development of precision therapies for genetic
heart disease, announced today a worldwide collaboration to
discover and develop first-of-its-kind targeted therapeutics for
heritable heart diseases known as cardiomyopathies, the most common
forms of heart muscle disease. The collaboration builds upon
MyoKardia's pioneering science, which hopes to correct the
disruptive effects that disease mutations have on heart muscle
contraction.
The collaboration, representing one of the largest research and
development commitments to genetic forms of cardiomyopathy,
encompasses three MyoKardia programs. Two of these programs are
focused on hypertrophic cardiomyopathy (HCM) and the other is
focused on dilated cardiomyopathy (DCM). The collaboration provides
up to $200 million in equity
investments, milestone payments and research and development
services through 2018, of which $45
million has already been received in an upfront licensing
fee and an initial equity investment. In addition, Sanofi and
MyoKardia will equally share development costs on the HCM programs
following initial demonstration of efficacy in patients, with
Sanofi fully covering the development costs of the DCM
program.
The collaboration is an outgrowth of Sanofi's Sunrise
initiative, a strategic partnership model that seeks to invest in
early stage opportunities that align with Sanofi's expert
development and commercialization abilities. The commitment of
resources from Sanofi will accelerate and broaden MyoKardia's basic
disease research and will support the shared commitment of bringing
desperately needed therapies to patients.
"This collaboration illustrates Sanofi's research and
development philosophy for Sunrise projects. It combines in a
meaningful way the unique expertise in rare and cardiovascular
diseases of our top scientists with that of the best innovators in
the world, like MyoKardia's founders and scientists, to achieve
real breakthroughs in medicine," said Dr. Elias Zerhouni, President Global R&D for
Sanofi.
"MyoKardia's research represents the first hope for targeted
treatments that address the primary cause of each patient's
disease," said Tassos Gianakakos, Chief Executive Officer,
MyoKardia. "By genetically defining HCM and DCM into several
underlying rare genetic diseases, MyoKardia's candidate therapies
have the potential to be developed far more efficiently than
traditional cardiovascular drugs. The collaboration extends
MyoKardia's competitive advantage, allowing advancement of a broad
portfolio of important treatments for patients".
Within the collaboration, MyoKardia will drive research and
worldwide development activities through early human efficacy
studies. Thereafter, MyoKardia will lead worldwide development and
U.S. commercial activities for the two HCM programs, where it has
retained product rights, and Sanofi will lead global development
and commercial activities for DCM where it has obtained worldwide
rights, and ex-U.S. regulatory and commercial activities to the two
HCM programs where it has ex-U.S. commercialization rights. Sanofi
also has the option to co-promote in the U.S. for potential
expanded cardiovascular diseases outside of the genetically
targeted indications for either of the HCM programs, with MyoKardia
having the option to co-promote the DCM program in the U.S.
"We are very excited to embark on this deep collaboration
with MyoKardia, to identify first-in-class, life-changing therapies
for HCM and DCM patients," said Dr. Kathy Bowdish, Vice President Global R&D and
Head of Sunrise. "This opportunity allows us to advance our
collective knowledge and bench strength in this exciting and
emerging field. This creative collaboration truly demonstrates
Sanofi's open innovation model."
About Genetic Cardiomyopathies
HCM and DCM are types
of heritable heart diseases that are caused by mutations in the
genes of the proteins that are primarily responsible for the
contraction of the heart muscle. HCM is the leading cause of sudden
cardiac death in young adults, and DCM, the leading genetic illness
requiring heart transplantation, together are believed to affect
nearly 800,000 children and adults in the U.S. alone. Available
treatments for patients suffering from these forms of heart disease
have been approved for other illnesses and only treat the symptoms
of their disease. As their disease progresses, it can require
invasive procedures including heart transplantation, and can lead
to sudden cardiac death and complications associated with heart
failure, including stroke.
HCM is believed to be the most common heritable cardiovascular
disease, thought to affect one in 500 people in the general
population. HCM is characterized by an abnormal thickening and
stiffening of the walls of the heart's main pumping chamber, the
left ventricle. Disease-causing HCM mutations make it difficult for
the heart to relax and refill with fresh blood between beats,
causing shortness of breath and sometimes dizziness, fainting or
chest pain. About one-fourth of HCM patients experience an abnormal
heart rhythm called atrial fibrillation that profoundly increases
the risk of potentially debilitating or even fatal stroke.
Genetically-defined DCM, estimated to occur in one in 2,500
people in the general population, is a condition in which the heart
becomes enlarged and weakened, and is unable to pump enough blood
to the rest of the body. DCM is also known to be associated with
harmful blood clots, which can cause strokes, and with irregular
and harmful heartbeats and sudden cardiac death. More than 20
percent of heart transplantations are performed every year for
patients with genetic DCM.
About Sanofi
Sanofi, an integrated 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, and consumer healthcare, emerging
markets, animal health and the new Genzyme. Sanofi is listed in
Paris (EURONEXT: SAN) and in
New York (NYSE: SNY).
To learn more about Sunrise, please
visit www.sanofi-sunrise.com
About MyoKardia
MyoKardia, Inc. is pioneering a
precision medicine approach to treat patients with genetic heart
disease. By combining leading-edge cardiovascular genetics with
recent advances in heart muscle biochemistry, MyoKardia seeks to
make a meaningful difference in the lives of people suffering from
these diseases. Leveraging powerful genetic insights MyoKardia
intends to classify these heritable cardiovascular diseases into a
series of underlying rare genetic disorders, enabling physicians to
treat patients based on their unique genetic and clinical profiles.
MyoKardia has helped organize an international patient registry,
the Sarcomeric Human Cardiomyopathy Registry (SHaRe) to help in
this effort. As a result, MyoKardia's therapeutic candidates have
the potential to be developed far more efficiently than traditional
cardiovascular drugs – matching the appropriate therapy to each
patient. Launched in 2012, MyoKardia was founded by world-class
experts in cardiovascular disease, cardiac muscle biology and
genetics, and is funded by leading healthcare investor Third Rock
Ventures. For more information, please visit www.myokardia.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, 2013. Other than as required by
applicable law, Sanofi does not undertake any obligation to update
or revise any forward-looking information or statements.
Contacts:
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Sanofi
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Media
Relations
Laurence
Bollack
Tel: +33 (0) 1 53 77
46 46
E-mail:
mr@sanofi.com
Global R&D
Communications
Amy Ba, Ph.
D.
Tel: +1
617-665-4851
E-mail:
amy.ba@sanofi.com
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Investor
Relations
Sebastien
Martel
Tel: +33 (0)1 53 77
45 45
E-mail:
IR@sanofi.com
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MyoKardia
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Sheryl
Seapy
Pure
Communications
sheryl@purecommunicationsinc.com
Tel. :
949-903-4750
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SOURCE Sanofi