CAMBRIDGE, Mass. & CARLSBAD, Calif.--(BUSINESS WIRE)--Jan. 12, 2009--Genzyme Corp. (Nasdaq: GENZ) and Isis Pharmaceuticals, Inc. (Nasdaq:
ISIS) today announced that they have begun two new studies of
mipomersen, a novel lipid-lowering drug in late-stage development, and a
third is currently screening patients. These three trials will provide
additional data on mipomersen in high-risk patient populations. The new
studies, together with ongoing trials, will also substantially increase
the size of the database of patients treated with mipomersen over the
next 18 months.
The trial currently screening patients will evaluate the safety and
efficacy of mipomersen in patients with severe hypercholesterolemia who
are on a maximally tolerated lipid-lowering regimen (previously referred
to as apheresis-eligible patients). This phase 3 study includes patients
who are not on LDL cholesterol apheresis but who have such severely
elevated LDL-C levels that they are eligible for the procedure, and will
enroll up to 75 patients.
In addition, the companies recently initiated a phase 3 trial in
hypercholesterolemic patients on maximally tolerated dosages of statins
who are at high risk for coronary heart disease. This study will enroll
up to 180 patients. The companies have also begun a new phase 2 study in
high-risk, high-cholesterol patients who are intolerant to statins,
which will enroll up to 30 patients.
All three new trials are double-blind, placebo-controlled studies in
which patients will be randomized 2:1 to receive a 200 mg dose of
mipomersen or placebo weekly for 26 weeks, with percent change in LDL-C
as the primary endpoint.
The companies in August began a phase 3 trial of mipomersen in patients
with heterozygous familial hypercholesterolemia (heFH), a genetic
disorder that causes severely elevated LDL-C levels.
"With the start of these studies, we will have begun four new mipomersen
trials since we formed our collaboration with Isis," stated John P.
Butler, president of Genzyme's renal, endocrine, and cardiovascular
business units. "We are very excited about the progress we are making on
our development plan to learn more about mipomersen's use in lipid
management for patients with the highest unmet medical need."
"Together with our partners at Genzyme we have made great strides in
moving mipomersen forward, creating the momentum to take us into 2009
and beyond. Data from these new studies should greatly enhance our
understanding of the safety and efficacy of mipomersen as we move it
toward an NDA," said Stanley T. Crooke, Chairman and CEO of Isis
Pharmaceuticals. "We continue to believe that the Genzyme/Isis team is
ideal to bring this exciting new drug to patients in need."
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 complete. Top-line data are expected to be available in
mid-2009, and the submission of a U.S. marketing application for this
indication is anticipated during the second half of 2010. Genzyme
continues to engage in discussions with regulatory authorities outside
the U.S. regarding the development path for mipomersen.
Data from the four new studies of mipomersen will continue to build the
body of clinical evidence around the treatment's value in managing
high-risk, high-cholesterol patients. Data are expected to be available
before the companies begin an outcome study of mipomersen, and will help
inform that trial's design. The study, anticipated to begin in mid-2010,
is intended to support potential expansion of mipomersen's label to
include a broader group of at-risk, high cholesterol patients.
About Mipomersen
Mipomersen is a first-in-class apo-B synthesis inhibitor currently in
phase 3 development. It is intended to reduce LDL-C by preventing the
formation of atherogenic lipoproteins. In phase 2 studies, mipomersen, a
weekly injectable therapeutic, was observed to reduce LDL-C beyond
reductions achieved with standard lipid-lowering drugs, enabling more
patients to achieve LDL-C targets. It was also observed to reduce
triglycerides, lipoprotein(a), and serum apo-B, all generally accepted
risk factors for cardiovascular disease. 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 immune disease, and diagnostic
testing. Genzyme's commitment to innovation continues today with a
substantial development program focused on these fields, as well as
cardiovascular disease, neurodegenerative diseases, and other areas of
unmet medical need.
Genzyme's press releases and other company information are available at 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 19
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. Isis is a joint owner of Regulus Therapeutics Inc., a joint
venture focused on the discovery, development and commercialization of
microRNA therapeutics. Isis also has made significant innovations beyond
human therapeutics resulting in products that other companies, including
Abbott, are commercializing. As an innovator in RNA-based drug discovery
and development, Isis is the owner or exclusive licensee of over 1,600
issued patents worldwide. Additional information about Isis is available
at www.isispharm.com.
Genzyme Safe Harbor Statement
This press release contains forward-looking statements regarding
Genzyme's business plans and strategies including, without limitation,
statements about the potential uses and benefits of mipomersen; the data
expected from the mipomersen clinical studies; the expected timing of
the clinical study results; plans for regulatory filings; and future
clinical study plans and potential uses of the data from such clinical
studies. 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 actual timing
and results of the clinical studies; Genzyme's ability to accurately
understand and predict the outcome and impact of its clinical studies
related to mipomersen; Genzyme's ability to continue to support its
clinical and other development efforts related to mipomersen; the actual
efficacy and safety of mipomersen; the outcome of discussions with
regulatory authorities regarding clinical studies 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 September 30, 2008. Genzyme cautions
investors not to place undue reliance on the forward-looking statements
contained in this press release. These statements speak only as of the
date of this press release 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. 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
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 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 Company.
In this press release, unless the context requires otherwise, "Isis"
refers to Isis Pharmaceuticals and its subsidiaries and joint venture.
Isis Pharmaceuticals is a registered trademark of Isis Pharmaceuticals,
Inc. Regulus Therapeutics is a trademark of Regulus Therapeutics Inc.
CONTACT: Genzyme Contacts:
Patrick Flanigan (Investors), 617-768-6563
or
Erin Emlock (Media), 617-768-6923
or
Isis Pharmaceuticals' Contacts:
Kristina Lemonidis (Investors), 760-603-2490
or
Amy Blackley, Ph.D. (Media), 760-603-2772
Source: Genzyme Corp.
