Broad New Patent Granted
CAMBRIDGE, Mass. and CARLSBAD, Calif., Aug. 6 /PRNewswire-FirstCall/ --
Genzyme Corp. (Nasdaq: GENZ) and Isis Pharmaceuticals, Inc. (Nasdaq: ISIS)
today announced that they have begun a phase 3 study of mipomersen in
patients with heterozygous familial hypercholesterolemia (heFH), a genetic
disorder that causes exceptionally high levels of LDL cholesterol. It is
the first of four new trials the companies plan to initiate by the end of
this year, and the second late-stage study of mipomersen, a novel
lipid-lowering drug that utilizes antisense technology.
The companies also announced that the United States Patent and
Trademark Office granted the patent entitled "Antisense Modulation of
Apolipoprotein B (apoB) Expression," U.S. Patent No. 7,407,943. The patent
broadly covers the use of antisense compounds targeting the apoB messenger
RNA except a ribozyme.
"We are making excellent progress in implementing our comprehensive
development plan for mipomersen," stated John P. Butler, president of
Genzyme's renal, endocrine and cardiovascular business units. "The start of
the heFH study puts us on track to meet our goal of beginning four new
mipomersen studies this year. In addition, the recent patent issuance
increases the overall value of mipomersen, which we believe has enormous
potential to help patients."
The new trial will evaluate the safety and efficacy of mipomersen in
patients who have heFH and coronary artery disease. It is a randomized,
double-blind, placebo-controlled study taking place at approximately 30
sites in the U.S. and Canada, with an anticipated total enrollment of
around 100 patients. Patients on a stable dose of other lipid-lowering
agents are being randomized 2:1 to receive a 200 mg dose of mipomersen or
placebo weekly for 26 weeks. The primary endpoint will be percent reduction
in LDL cholesterol, and data are expected to be available in 2010. The
trial will add to the body of clinical data on mipomersen and the overall
product profile.
"This is an exciting moment for mipomersen, combining the initiation of
the phase 3 trial in heFH with a very broad patent allowance that expands
the patent coverage for the apoB franchise," said Stanley Crooke, chairman
and chief executive officer of Isis. "This comprehensive patent covers
methods of inhibiting apoB by targeting anywhere on the messenger RNA
including the site to which mipomersen binds, and all therapeutic uses that
might result from lowering apoB."
Mipomersen Development Plan
The initial indication sought for mipomersen will be for patients with
homozygous FH, and enrollment in a phase 3 trial in this patient population
is expected to be completed by the end of this year. Data are expected to
be available in mid-2009 and a U.S. filing for this indication is
anticipated during the second half of 2010.
Genzyme and Isis plan to begin three additional trials evaluating
mipomersen's safety and efficacy in reducing LDL cholesterol in high-risk
patients during the second half of 2008. These trials will include: one for
apheresis-eligible patients, and two for high-risk, high cholesterol
patients. All three have anticipated trial designs that include a 2:1
randomization ratio of a 200 mg dose of mipomersen or placebo weekly for 26
weeks.
These trials will continue to build the body of clinical evidence
around mipomersen's value in managing very high risk patients. Data from
the trials will also inform the design of the morbidity and mortality
outcome study for potential expansion of mipomersen's label to include a
broader group of at-risk, high cholesterol patients on maximally tolerated,
currently available therapies.
Following the finalization in June of the mipomersen license and
collaboration agreement between Genzyme and Isis, the mipomersen IND and
all regulatory authority has been transferred to Genzyme. Now that this
transition has taken place, the companies are looking forward to Genzyme
beginning discussions with the FDA and regulatory authorities in Europe,
where the development path for mipomersen may differ from that in the U.S.
New Patent
The patent entitled "Antisense Modulation of Apolipoprotein B (apoB)
Expression," U.S. Patent No. 7,407,943 is the first allowance in a series
of broad filings protecting the therapeutic use of targeting apoB for the
lowering of all atherogenic lipids, including LDL cholesterol and
triglycerides. The patent covers the use of both single-stranded and
double-stranded (siRNA) antisense drugs complementary to any site of the
mRNA of human apoB regardless of their chemistry or antisense mechanism of
action. The allowance provides broad protection of the Isis-Genzyme apoB
franchise, including mipomersen and potential future follow-on compounds.
About Mipomersen
Mipomersen is a second-generation antisense drug currently in phase 3
development that reduces the production of apoB-100, a protein critical to
the synthesis and transport of "bad" cholesterol.
In phase 2 studies, mipomersen, a weekly injectable therapeutic, was
observed to reduce cholesterol and other atherogenic lipids beyond
reductions achieved with standard lipid-lowering drugs, enabling more
patients to achieve LDL-C targets. In 2008 Genzyme and Isis completed a
licensing agreement that provides Genzyme with exclusive worldwide rights
to mipomersen, which was discovered and initially developed by Isis.
About Genzyme
One of the world's leading biotechnology companies, Genzyme is
dedicated to making a major positive impact on the lives of people with
serious diseases. Since 1981, the company has grown from a small start-up
to a diversified enterprise with more than 10,000 employees in locations
spanning the globe and 2007 revenues of $3.8 billion. In 2007, Genzyme was
chosen to receive the National Medal of Technology, the highest honor
awarded by the President of the United States for technological innovation.
With many established products and services helping patients in nearly
90 countries, Genzyme is a leader in the effort to develop and apply the
most advanced technologies in the life sciences. The company's products and
services are focused on rare inherited disorders, kidney disease,
orthopaedics, cancer, transplant, and diagnostic testing. Genzyme's
commitment to innovation continues today with a substantial development
program focused on these fields, as well as immune disease, infectious
disease, and other areas of unmet medical need.
Genzyme's press releases and other company information are available at
http://www.genzyme.com and by calling Genzyme's investor information line
at 1-800-905-4369 within the United States or 1-678-999-4572 outside the
United States.
About Isis Pharmaceuticals, Inc.
Isis is exploiting its expertise in RNA to discover and develop novel
drugs for its product pipeline and for its partners. The Company has
successfully commercialized the world's first antisense drug and has 18
drugs in development. Isis' drug development programs are focused on
treating cardiovascular and metabolic diseases. Isis' partners are
developing antisense drugs invented by Isis to treat a wide variety of
diseases. Ibis Biosciences, Inc., Isis' majority-owned subsidiary, is
developing and commercializing the Ibis T5000(TM) Biosensor System, a
revolutionary system to identify infectious organisms. Isis is a joint
owner of Regulus Therapeutics LLC, a joint venture focused on the
discovery, development and commercialization of microRNA therapeutics. As
an innovator in RNA-based drug discovery and development, Isis is the owner
or exclusive licensee of over 1,500 issued patents worldwide. Additional
information about Isis is available at http://www.isispharm.com.
Genzyme Safe Harbor Statement
This press release contains forward-looking statements, including
without limitation, statements concerning mipomersen's safety and benefits
for patients with high cholesterol, the development plan for mipomersen and
FDA's requirements for its approval. These statements are subject to risks
and uncertainties that could cause actual results to differ materially from
those forecasted. These risks and uncertainties include, among others: the
timing of further discussions with FDA regarding the approval of
mipomersen; the timing and content of submissions to and decisions made by
the FDA relating to mipomersen; further analysis of clinical trial data;
the results of other studies; the actual efficacy and safety of mipomersen;
and the risks and uncertainties described in Genzyme's SEC reports filed
under the Securities Exchange Act of 1934, including the factors discussed
under the caption "Risk Factors" in Genzyme's Quarterly Report on Form 10-Q
for the quarter ended March 31, 2008. Genzyme cautions investors not to
place substantial reliance on the forward-looking statements contained in
this press release. These statements speak only as of today's date and
Genzyme undertakes no obligation to update or revise the statements.
Genzyme(R) is a registered trademark of Genzyme Corporation. All rights
reserved.
Isis Safe Harbor 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
potential and safety of mipomersen 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, including those statements
that are described as Isis' goals or projections. 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, in developing and
commercializing systems to identify infectious organisms that are effective
and commercially attractive, and in the endeavor of building a business
around such products. 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, 2007, and
its quarterly report on Form 10-Q for the quarter ended March 31, 2008,
which are on file with the SEC. Copies of these and other documents are
available from the Company.
Isis Pharmaceuticals is a registered trademark of Isis Pharmaceuticals,
Inc. Ibis Biosciences and Ibis T5000 are trademarks of Ibis Biosciences,
Inc. Regulus Therapeutics is a trademark of Regulus Therapeutics LLC.
Genzyme Contacts:
Patrick Flanigan (Investors) Erin Emlock (Media)
617-768-6563 617-768-6923
Isis Pharmaceuticals' Contacts:
Kristina Lemonidis (Investors) Amy Blackley, Ph.D. (Media)
760-603-2490 760-603-2772
SOURCE Genzyme Corp.; Isis Pharmaceuticals, Inc.
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Related links:
http://www.genzyme.com
http://www.isispharm.com
http://www.prnewswire.com/comp/113803.html/
CONTACT:
Genzyme Contacts, Investors, Patrick
Flanigan, +1-617-768-6563, or Media, Erin Emlock,
+1-617-768-6923; Isis Pharmaceuticals' Contacts, Investors,
Kristina Lemonidis, +1-760-603-2490, or Media, Amy Blackley,
Ph.D., +1-760-603-2772
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