CAMBRIDGE, Mass. & CARLSBAD, Calif.--(BUSINESS WIRE)--Genzyme,
a Sanofi company (EURONEXT: SAN and NYSE: SNY), and Isis Pharmaceuticals
Inc. (NASDAQ: ISIS), announced today that new two-year data from a phase
3 long-term extension study of KYNAMROTM (mipomersen sodium)
were presented at the XVI International Symposium on Atherosclerosis in
Sydney, Australia. In the study, patients treated with KYNAMROTM
for two years maintained robust reductions in LDL-C and all other Apo B
containing atherogenic lipoproteins measured with a safety profile
consistent with the phase 3 studies of KYNAMROTM.
“These positive and clinically meaningful results represent a
significant addition to the KYNAMROTM clinical development
program”
Data presented today included 141 patients who enrolled in the study
after having completed one of the three phase 3 studies: homozygous FH,
severe hypercholesterolemia, or the heterozygous FH. These studies were
six months long and required patients to be on stable maximally
tolerated lipid-lowering therapy throughout the study. To date, 63
patients remain on or have completed treatment with 40 patients
consenting to participate for another two years of treatment - a total
of four years of treatment.
In this study, sustained reductions in LDL-C with a mean reduction of 28
percent in LDL-C at six months and at two years were observed in
patients treated with KYNAMROTM. Changes in liver fat were
observed in some patients where the overall median change stabilized and
then declined with continued treatment. The median change from baseline
in percent liver fat increased from 5 percent at 26 weeks, to 13 percent
at 52 weeks, and returned to 5 percent at 104 weeks. Results represent
those patients in the extension study at each assessment period: 60
patients at 26 weeks, 31 patients at 52 weeks, and 39 patients at 104
weeks. The median change reflects both patients who continued at full
dose as well as those with dose adjustments and dose interruptions. Upon
treatment discontinuation, changes in liver fat returned towards normal.
“These data show that robust LDL-C reductions are seen in patients
treated for 2 years and more. Liver fat may increase in some patients
but in this long term study the median fat fraction is seen to stabilize
and decline with time as measured by MRI,” said study
investigator Raul D. Santos, M.D., Ph.D., Director of the Lipid Clinic
of the Heart institute, Instituto do Coração, Hospital das Clínicas, São
Paulo, Brazil. “These results are encouraging and support the
potential for effective and safe long-term use in patients with the most
aggressive forms of FH.”
Genzyme and Isis have completed the four phase 3 studies that are
included in the European submission and will be included in the U.S.
submission. In these studies, the most commonly observed adverse events
were injection site reactions and flu-like symptoms. The long-term data
demonstrate a safety and efficacy profile consistent with phase 3
findings.
"These positive and clinically meaningful results represent a
significant addition to the KYNAMROTM clinical development
program,” said Vice President and General Manager of Genzyme’s
Cardiovascular Business, Paula Soteropoulos. “We look forward to
continuing to work with regulatory authorities to bring this treatment
to market for such a severe and life-threatening disease.”
KYNAMRO™ is the registered trade name submitted to health authorities
for investigational agent mipomersen. Genzyme submitted for EU marketing
approval of KYNAMROTM for the treatment of patients with
homozygous FH (HoFH) and severe heterozygous FH (Severe HeFH) in July
2011. Genzyme also expects to submit an NDA for U.S. approval for the
HoFH indication by the end of March 2012.
Webcast
At 4:00 p.m. Eastern Time today, Isis will host a live conference call
webcast and slide presentation with Dr. Raul D. Santos to discuss KYNAMROTM
data presented at the International Symposium on Atherosclerosis.
Interested parties may listen to the call by dialing 866.761.0749 and
refer to the passcode “ISIS 2012” or access the webcast with or without
audio at www.isispharm.com.
A webcast replay will be available for a limited time at the same
address.
About KYNAMROTM (mipomersen sodium)
KYNAMROTM is a first-in-class Apo B synthesis inhibitor
currently in late-stage development for patients with homozygous
familial hypercholesterolemia (HoFH) and severe heterozygous familial
hypercholesterolemia (Severe HeFH) to further reduce LDL cholesterol
(LDL-C) in patients already maintaining a stable regimen of maximally
tolerated lipid-lowering therapies. It is intended to reduce LDL-C by
preventing the formation of atherogenic lipoproteins, the particles that
carry cholesterol through the bloodstream. KYNAMROTM acts by
blocking the production of, apolipoprotein B (Apo B), the protein that
provides the structural core for these atherogenic particles, including
LDL and lipoprotein-a (Lp(a)).
About Familial Hypercholesterolemia (FH)
FH is a genetic disease that results in elevated LDL-C levels and family
patterns of increased risk of premature heart disease and heart
disease-related death. FH patients have inherited abnormalities in liver
cells that are responsible for clearing LDL particles from the blood. FH
is autosomal dominant, which means that all first-degree relatives of FH
patients have a 50 percent chance of having the disease as well, making
early detection through family screening critically important.
The most severe FH patients have LDL-C levels that are two to four times
higher than recommended levels, even when taking multiple
cholesterol-lowering medications. These people, who are characterized as
having severe FH, include: those who have inherited the disease from
both parents (HoFH) and those who have inherited it from only one
parent, and have a particularly severe form of the disease (Severe HeFH)
defined as those people who are maximally treated and still have LDL-C
greater than 200 mg/dL (5.1 mmol) with coronary heart disease or greater
than 300 mg/dL (7.1 mmol) without coronary heart disease. People with
HoFH may have aggressive heart disease beginning in childhood, and even
with today’s therapies remain at significant risk of cardiovascular
events. Learn more at www.FHJourneys.com.
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.
Genzyme® and KYNAMROTM are registered trademarks
of Genzyme Corporation. All rights reserved.
About Sanofi
Sanofi, a global and diversified 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).
About Isis Pharmaceuticals, Inc.
Isis is exploiting its leadership position in antisense technology to
discover and develop novel drugs for its product pipeline and for its
partners. Isis' broad pipeline consists of 25 drugs to treat a wide
variety of diseases with an emphasis on cardiovascular, metabolic and
severe and rare/neurodegenerative diseases, and cancer. Isis' partner,
Genzyme, plans to commercialize Isis' lead product, KYNAMRO, following
regulatory approval, which is expected in 2012. Isis' patents provide
strong and extensive protection for its drugs and technology. Additional
information about Isis is available at www.isispharm.com.
Isis Pharmaceuticals® is a registered trademark of Isis
Pharmaceuticals, Inc.
Sanofi Forward Looking Statement
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.
Isis Forward Looking Statement
This press release includes forward-looking statements regarding
Isis’ collaboration with Genzyme Corporation, its financial and business
development activities, and the development, activity, therapeutic and
commercial potential and safety of KYNAMRO in treating patients with
high cholesterol. Any statement describing Isis’ goals, expectations,
financial or other projections, intentions or beliefs is a
forward-looking statement and should be considered an at-risk statement.
Such statements are subject to certain risks and uncertainties,
particularly those inherent in the process of discovering, developing
and commercializing drugs that are safe and effective for use as human
therapeutics, and in the endeavor of building a business around such
drugs. Isis’ forward-looking statements also involve assumptions that,
if they never materialize or prove correct, could cause its results to
differ materially from those expressed or implied by such
forward-looking statements. Although Isis’ forward-looking statements
reflect the good faith judgment of its management, these statements are
based only on facts and factors currently known by Isis. As a result,
you are cautioned not to rely on these forward-looking statements. These
and other risks concerning Isis’ programs are described in additional
detail in Isis’ annual report on Form 10-K for the year ended December
31, 2011, which is on file with the SEC. Copies of this and other
documents are available from the Company.