Conference call to be held today at 10 a.m. EDT
CAMBRIDGE, Mass., and CARLSBAD, Calif., June 24 /PRNewswire-FirstCall/
-- Genzyme Corp. (Nasdaq: GENZ) and Isis Pharmaceuticals, Inc. (Nasdaq:
ISIS) today announced the finalization of the license and collaboration
agreement for mipomersen. The collaboration provides Genzyme with exclusive
worldwide rights to mipomersen, a novel lipid-lowering drug discovered and
developed by Isis that is in phase 3 clinical development. During the
second half of this year, enrollment is expected to be completed in a
pivotal study of mipomersen in homozygous familial hypercholesterolemia,
and a new trial in apheresis-eligible patients is expected to begin.
As part of the agreement, Isis will receive a $175 million license fee
for mipomersen. In February, Isis received a $150 million payment from
Genzyme to purchase 5 million shares of Isis common stock at $30 per share.
The companies have updated the deal terms so that Isis will contribute
up to $50 million in additional development funding for mipomersen,
bringing Isis' development funding commitment up to $125 million.
Thereafter Isis and Genzyme will share development costs equally. The
initial Isis development funding commitment and the shared funding will end
when the program is profitable. In exchange for this additional
contribution, Isis has the opportunity to receive $75 million in milestone
payments early.
"Mipomersen is an innovative treatment that has the potential to change
the standard of care for severely ill patients whose needs cannot be
addressed by current cholesterol-lowering therapies," said Henri A.
Termeer, Genzyme's chairman and chief executive officer. "This treatment is
an important addition to Genzyme's robust late-stage pipeline. We will
manage the clinical development of mipomersen within our current R&D budget
and financial guidance."
Over the next 30 days, the companies will transition the mipomersen IND
and all regulatory authority to Genzyme. As the sponsor of mipomersen,
Genzyme will take the lead on discussions with regulatory agencies and
filings. In response to guidance received from the FDA, the companies have
modified the initial development plan for mipomersen, subject to further
discussions with the agency.
The key changes to the plan include:
-- The addition of clinical studies of mipomersen in apheresis-eligible
patients.
-- Consolidation of the planned filings for heterozygous FH patients
and other high-risk, high cholesterol patients into a single registration
in the U.S.
-- Acceleration of the planned outcome study so that it can be used to
support the above mentioned consolidated U.S. filing.
"Mipomersen is an important drug that demonstrates the power and
precision of antisense drugs. We believe now, as we did in January, that
Genzyme is the ideal partner for mipomersen," said Stanley Crooke, Chairman
and Chief Executive Officer of Isis. "We will continue to work with Genzyme
on a development plan that is responsive to the FDA and other regulatory
agencies, and maximizes the value of the drug. In addition, we look forward
to exploring new areas of therapeutic opportunity with Genzyme in CNS and
certain rare diseases as part of this alliance."
Deal Terms
As a result of the changes in the development plan and consistent with
the premise of the transaction in which the companies are sharing the value
of mipomersen, the following changes to the original financial terms of the
deal have been made:
-- Isis will contribute up to the first $125 million in development
funding, reflecting an additional contribution of up to $50 million.
Thereafter Isis and Genzyme will share development costs equally. The
initial Isis development funding commitment and the shared funding will end
when the program is profitable. In exchange for this additional
contribution, Isis has the opportunity to receive certain milestone
payments early.
-- $75 million of the $150 million milestone associated with the
heterozygous FH indication (the portion related to U.S. registration) may
be accelerated, to be paid $25 million at annual product revenue of $250
million and $50 million at annual product revenue of $500 million. The $75
million milestone for European approval of the heterozygous FH indication
remains the same.
All other financial terms of the transaction remain unchanged. As part
of the agreement, Isis will receive a total of $325 million in up-front
payments from Genzyme, including the $175 million license fee and the $150
million February 2008 stock purchase.
Isis is eligible to receive up to $750 million in commercial milestone
payments, in three increments of $250 million, when annual net revenues
meet $3 billion, $4 billion, and $5 billion for two consecutive years.
Isis is also eligible to receive up to $825 million in development and
regulatory milestone payments, which are broken out as follows:
-- Total milestones related to homozygous FH $50 million
-- Total milestones related to heterozygous FH $150 million
-$75 million for U.S. approval (may be accelerated
based on earlier achievement of sales targets)
-$75 million for E.U. approval -- Total milestones related to approvals of a first Non-FH indication
$375 million
-- Total milestones related to approvals of a
follow-on product $250 million
Total $825 million
Isis and Genzyme will allocate responsibility for funding development
expenses as described above. Genzyme will be responsible for funding sales
and marketing expenses until revenues are sufficient to cover them.
Genzyme and Isis will share mipomersen profits, beginning with a 70/30
Genzyme/Isis split. This split will adjust on a sliding scale, reaching
50/50 when annual revenues reach $2 billion. As part of the strategic
relationship, Genzyme will also have preferred access to future Isis drugs
for CNS and certain rare diseases.
Mipomersen Development Plan
The initial indication sought for mipomersen will be for patients with
homozygous FH, and a phase 3 trial in this patient population is ongoing. A
U.S. filing for this indication is expected during the second half of 2010.
The companies also plan to study mipomersen's use in other very high risk
patient groups, including apheresis-eligible patients. Data from a trial in
apheresis-eligible patients is expected before the filing for the initial
homozygous FH indication.
LDL apheresis is a blood-filtering procedure that targets "bad"
cholesterol, and is indicated for individuals for whom diet and maximum
drug therapy has either been ineffective or not tolerated. Specific LDL
levels defining apheresis eligibility vary by country. Many patients who
are eligible, however, choose not to undergo apheresis due to its negative
impact on quality of life. The procedure is painful and inconvenient,
requiring patients to go to apheresis centers for treatment once every one
to two weeks. Apheresis can cost $75,000 to $150,000 per year.
The trial in apheresis-eligible patients is an important addition to
the mipomersen development program. Like homozygous FH patients, apheresis-
eligible patients are characterized by extremely high LDL levels and are
not able to be managed by existing therapies. There are an estimated 10,000
- 15,000 apheresis-eligible patients in the United States and Europe.
Genzyme and Isis expect to begin three additional trials of mipomersen
in high-risk patients during the second half of this year. These trials
will include: one for heterozygous FH patients, and two for high-risk, high
cholesterol patients. These trials will continue to build the body of
clinical evidence around mipomersen's value in managing very high risk
patients.
Data from these five trials will also inform the design of the
morbidity and mortality outcome study for potential expansion of
mipomersen's label to include a larger group of at-risk, high cholesterol
patients. In addition to the ongoing discussions with the FDA, plans are
underway to engage in discussions with regulatory authorities in Europe,
where the development path for mipomersen may differ from that in the U.S.
About Mipomersen
Mipomersen is a second-generation antisense drug that reduces the
production of apoB-100, a protein critical to the synthesis and transport
of "bad" cholesterol. Mipomersen is currently in phase 3 development for
patients with homozygous familial hypercholesterolemia, a disease which
creates a greatly increased risk of premature cardiovascular disease (CVD)
and CVD-related death. 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.
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.
Conference Call Information
Genzyme will host a conference call with Isis today at 10:00 a.m.
Eastern. To participate in the call, please dial 1-210-234-0010 and use
passcode "Genzyme." The replay number for the call is 1-203-369-0239, and
the replay will be available until midnight on July 1. Today's call will
also be Webcast on the investor events section of http://www.genzyme.com.
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 Corporation; 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:
Investors, Patrick Flanigan of Genzyme,
+1-617-768-6563, or Kristina Lemonidis of Isis, +1-760-603-2490,
or Media, Erin Emlock of Genzyme, +1-617-768-6923, or Amy
Blackley, Ph.D., of Isis, +1-760-603-2772
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