fda gives thumbs up to antisense !! , page-18

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30 May 2012
Antisense Technology Update
? ANP technology partner, Isis Pharmaceuticals Ltd. announces that the U.S. Food and Drug
Administration (FDA) has accepted for filing the New Drug Application (NDA) for KYNAMRO™
(mipomersen sodium) for the treatment of patients with homozygous familial
hypercholesterolemia (HoFH)
Antisense Therapeutics Limited’s (ASX:ANP) technology partner and major shareholder, Isis
Pharmaceuticals, Inc. (NASDAQ:ISIS) has made an announcement to the market in the US (last night
Australian time) concerning their 2nd generation antisense drug mipomersen (KYNAMROTM) -
“Genzyme and Isis Announce Filing of U.S. NDA for KYNAMRO™ (mipomersen sodium) in
Homozygous Familial Hypercholesterolemia.” Genzyme submitted an application for U.S.
marketing approval of KYNAMRO™ for the treatment of patients with HoFH in March 2012. The
application will be subject to a standard review and will have a Prescription Drug User Fee Act
(PDUFA) date of January 29, 2013 (the time by which the FDA is to complete its review).
Progress in the regulatory approval process for KYNAMROTM is a further validation milestone for the
antisense technology platform. ANP’s three drugs in its development pipeline: ATL1101 for Prostate
Cancer, ATL1102 for Multiple Sclerosis, Stem Cell Mobilisation and Asthma and ATL1103 for Growth
(Acromegaly), Cancer and Diabetes Related Disorders were all accessed by ANP via its collaboration
with Isis. These compounds, like KYNAMRO™, are all 2nd generation antisense drugs.
In July 2011, Genzyme submitted an application for EU marketing approval of KYNAMROTM for the
treatment of patients with HoFH and severe heterozygous FH (Severe HeFH).
The Isis announcement follows.
Antisense Therapeutics Limited (ASX: ANP) (ASX: ANP) is an Australian publicly listed biopharmaceutical drug
discovery and development company. Its mission is to create, develop and commercialise second generation antisense
pharmaceuticals for large unmet markets. ANP has 4 products in its development pipeline. ATL1102 (injection) has
successfully completed a Phase II efficacy and safety trial, significantly reducing the number of brain lesions in patients
with multiple sclerosis. ATL1103 is a second-generation antisense drug designed to block GHr production and thereby lower
blood IGF-I levels and is in clinical development as a potential treatment for growth, cancer and diabetes associated
disorders. ATL1102 (inhaled) is at the pre-clinical research stage as a potential treatment for asthma. ATL1101 is a secondgeneration
antisense drug at the pre-clinical stage being investigated as a potential treatment for prostate cancer.
Contact Information: Website: www.antisense.com.au
Managing Director: Mark Diamond +61 (3) 9827 8999
Investor Relations: Simon Watkin +61 (0) 413 15327
Press Release
Genzyme and Isis Announce Filing of U.S. NDA for KYNAMRO™ (mipomersen
sodium) in Homozygous Familial Hypercholesterolemia
Isis earns $25 million milestone payment
CAMBRIDGE, Mass. & CARLSBAD, Calif.--(BUSINESS WIRE)--
Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), and Isis
Pharmaceuticals Inc. (ISIS), announced today that the U.S. Food and Drug Administration
(FDA) has accepted for filing the New Drug Application (NDA) for KYNAMRO™ (mipomersen
sodium) for the treatment of patients with homozygous familial hypercholesterolemia
(HoFH). The NDA filing with the FDA triggers a $25 million milestone payment to Isis from
Genzyme.
"The NDA filing with the FDA represents a significant achievement in the development of
KYNAMRO™ and our efforts to get this important new drug to the market for patients who
are at high-risk of a cardiovascular event,” said David Meeker, M.D., President and CEO,
Genzyme. “We look forward to continuing the review process with the U.S. and EU
regulatory authorities to bring KYNAMRO™ to patients in need.”
Genzyme submitted an application for U.S. marketing approval of KYNAMRO™for the
treatment of patients with HoFH in March 2012. The application will be subject to a standard
review and will have a Prescription Drug User Fee Act (PDUFA) date of January 29, 2013. In
July 2011, Genzyme submitted an application for EU marketing approval of KYNAMROTM for
the treatment of patients with HoFH and severe heterozygous FH (Severe HeFH).
“We believe that KYNAMRO™ has the potential to bring real benefit to patients in the United
States with HoFH who are unable to adequately control their LDL-C with currently available
treatments,” said B. Lynne Parshall, Chief Operating Officer and CFO of Isis. “The
successes of our joint development efforts for KYNAMRO™ are evident in the significant
progress made in bringing this important new drug to the regulatory agencies for review. We
are pleased to have earned the first regulatory milestone payment for KYNAMRO™ from this
collaboration.”
The FDA submission for KYNAMRO™ is supported by the largest clinical trial conducted to
date in the HoFH patient population. In the randomized, double-blind, placebo controlled,
multi-center trial, significant reductions were observed in all atherogenic lipoproteins
evaluated (including LDL-C, Apo B and Lp(a)) for patients receiving KYNAMRO™ who are
already receiving a regimen of maximally tolerated lipid lowering therapies including statins.
Three patients (12 percent) treated with KYNAMRO™ withdrew due to adverse events.
Consistent with other studies evaluating KYNAMRO™, commonly observed adverse events
included mild to moderate injection site reactions and flu-like symptoms, as well as
elevations in liver transaminases.
KYNAMRO™ is the registered trade name submitted to health authorities for investigational
agent mipomersen.
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, and who require additional, significant lipid lowering therapy. 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.
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 (BSAC) and in New York (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, KYNAMROTM, 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.
Genzyme® and KYNAMRO™ are registered trademarks of Genzyme Corporation. All rights
reserved.
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.
Isis Forward Looking Statement
This press release includes forward-looking statements regarding Isis’ collaboration with
Genzyme, a Sanofi company, and the development, activity, therapeutic benefit and safety
of KYNAMRO™ in treating patients with high cholesterol. Any statement describing Isis’
goals, expectations, financial or other projections, intentions or beliefs, including the planned
commercialization of KYNAMRO, 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 and its most recent quarterly report on Form 10-Q, which
are on file with the SEC. Copies of these and other documents are available from the