Biogen Idec and Isis Pharmaceuticals Announce Global Collaboration for Antisense Program Targeting Myotonic Dystrophy
-- Biogen Idec has Option to Develop and Commercialize Promising Compound for Most Common Form of Muscular Dystrophy --
--Isis Expands its Rare Disease Franchise to Include Myotonic Dystrophy Type 1--
WESTON, Mass. & CARLSBAD, Calif.--(BUSINESS WIRE)--Jun 29, 2012 - Biogen Idec (NASDAQ: BIIB) and Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) today announced that they have entered into an exclusive, worldwide option and collaboration agreement under which the companies will develop and commercialize a novel antisense drug for the treatment of myotonic dystrophy type 1 (DM1), which is also known as Steinert disease.
DM1, the most common form of muscular dystrophy in adults, is a genetic neuromuscular disease characterized by progressive muscle atrophy, weakness and disabling muscle spasms. It is caused by a genetic defect in the dystrophia myotonica-protein kinase (DMPK) gene in which a sequence of three nucleotides repeats extensively, creating an abnormally long toxic RNA, which accumulates in the cell and prevents the production of proteins needed for normal cellular function. Isis' DM1 antisense program is being developed to correct the underlying genetic defect that causes DM1.
Isis will receive an upfront payment of $12 million and is responsible for the discovery of a lead antisense drug candidate targeting DMPK for the treatment of DM1. Isis is eligible to receive up to $59 million in milestone payments associated with the clinical development of the DMPK-targeting drug prior to licensing. Biogen Idec has the option to license the drug from Isis up through the completion of the Phase 2 trial. Isis could receive up to another $200 million in a license fee and regulatory milestone payments. In addition, Isis will receive double-digit royalties on sales of the drug. Isis will be responsible for global development of the drug through the completion of Phase 2 clinical trials, with Biogen Idec providing advice on the clinical trial design and regulatory strategy. If Biogen Idec exercises its option, it will assume global development, regulatory and commercialization responsibilities.
“Myotonic dystrophy is a debilitating neuromuscular disease that often affects entire families,” said Steven H. Holtzman, Executive Vice President of Corporate Development at Biogen Idec. “The unmet need is great, and there are currently no therapies to slow or stop progression of the disease. Myotonic dystrophy has an identifiable genetic cause, the program fits with our mission to bring innovative therapies to patients with serious neurologic diseases, and Isis' antisense compound has the potential to make a real difference. This collaboration, which is our second with Isis, reflects the tremendous respect we have for their scientific leadership and expertise in antisense technology.”
“Biogen Idec is a world leader in neurodegenerative diseases,” said B. Lynne Parshall, J.D., Chief Operating Officer, Chief Financial Officer and Secretary from Isis. “This collaboration allows us to expand our pipeline of drugs for rare and severe diseases with Biogen Idec's additional resources and support. It also complements our new alliance with Biogen Idec for our Phase 1 program in spinal muscular atrophy, or SMA. As with SMA, we are using our antisense technology in a unique manner to treat another devastating disease. Biogen Idec is an ideal partner for these programs with its expertise in neurodegenerative disease and global reach to help bring these therapies successfully to patients who have no treatment options.”
“DM1 is a progressive disease that leads to the gradual loss of muscle function,” said Charles A. Thornton, M.D., School of Medicine at University of Rochester Medical Center. “In the later stages it causes problems with breathing and walking. DM1 usually affects several people in a family, cutting across the generations to affect infants, young adults, and older members of a family at the same time. Genetic testing can aid in identifying people at risk of developing DM1, but there are no treatments available to delay the onset of symptoms or slow down the progression. Because the disease-causing substance in DM1 is a toxic RNA, the disease is a good target for Isis' antisense approach to selectively target the toxic RNA within the cell, removing it and restoring normal cell function.”
This collaboration follows on a worldwide option and collaboration agreement between Biogen Idec and Isis, which was announced in January, to develop and commercialize Isis' antisense investigational drug, ISIS-SMNRx, for the treatment of spinal muscular atrophy (SMA).
About DM1
DM1 is a genetic neuromuscular disease characterized by disabling
muscle spasms and progressive muscle wasting and weakness. DM1 also
affects many other organs within the body and patients with DM1 can
experience insulin insensitivity, cataracts and infertility. DM1 is
estimated to effect approximately 150,000 patients in the US,
Europe and Japan. It is passed from parent to child with each
subsequent generation experiencing more severe disease earlier in
life. The chance of a child inheriting DM1 is 50 percent if one
parent carries the genetic defect. DM1 is caused by a defect in the
DMPK gene that produces an increase in the number of triplet
repeats, CTG, within the gene. This DNA expansion produces an
abnormally large RNA that accumulates in cells, including muscle
cells, and prevents production of proteins essential for normal
cellular function. The severity and age of onset of DM1 correlates
with the number of triplet repeats, which increases from one
generation to the next. There are no disease-modifying therapies
for patients with DM1 and current treatments are intended to manage
symptoms and minimize disability.
About Antisense and RNase H
An antisense mechanism is a process when a nucleic acid binds to a
target RNA forming a duplex molecule. The formation of this duplex
molecule prevents the RNA from functioning normally and producing a
specific protein. Using antisense, Isis' drugs can reduce the
production of disease-causing proteins that are largely
inaccessible to traditional drug discovery approaches. The majority
of Isis' antisense drugs in development activate a cellular enzyme
called RNase H. Upon activation, RNase H seeks out and destroys the
duplex mRNA, inhibiting a cell's production of a specific protein.
Treating DM1 offers a unique opportunity for antisense. The genetic
defect in the DMPK gene creates a toxic RNA rather than a
disease-causing protein. The toxic RNA accumulates within the
nucleus of the cell and prevents the production of proteins
essential for normal cellular function. RNase H activation occurs
within the cell nucleus and therefore Isis' antisense drugs present
a specific therapeutic strategy for treating DM1. In preclinical
studies, Isis' antisense drugs have demonstrated potent and
selective reduction of toxic DMPK RNA.
About Biogen Idec
Through cutting-edge science and medicine, Biogen Idec discovers,
develops and delivers to patients worldwide innovative therapies
for the treatment of neurodegenerative diseases, hemophilia and
autoimmune disorders. Founded in 1978, Biogen Idec is the world's
oldest independent biotechnology company. Patients worldwide
benefit from its leading multiple sclerosis therapies, and the
company generates nearly $5 billion in annual revenues. For product
labeling, press releases and additional information about the
company, please visit
www.biogenidec.com.
About Isis Pharmaceuticals
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, severe and rare 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.
Biogen Idec Safe Harbor Statement
This press release contains forward-looking statements, including
statements about product development and commercialization. These
forward-looking statements may be accompanied by such words as
"anticipate," "believe," "estimate," "expect," "forecast,"
"intend," "may," "plan," "will" and other words and terms of
similar meaning. You should not place undue reliance on these
statements. Drug development and commercialization involve a high
degree of risk. Factors which could cause actual results to differ
materially from current expectations include the risk that adverse
safety events may occur, regulatory authorities may require
additional information or may fail to approve any potential new
therapy, product reimbursement may be limited or unavailable, there
may be problems with manufacturing processes, intellectual property
rights may not be adequately protected, and the other risks and
uncertainties that are described in the Risk Factors section of
Biogen Idec Inc.'s most recent annual or quarterly report and in
other reports Biogen Idec Inc. has filed with the SEC. These
statements are based on current beliefs and expectations and speak
only as of the date of this press release. Biogen Idec Inc. does
not undertake any obligation to publicly update any forward-looking
statements.
Isis Safe Harbor Statement
This press release includes forward-looking statements regarding
Isis' strategic alliance with Biogen Idec, and the discovery,
development, activity, therapeutic potential, safety and
commercialization of an antisense drug targeting DMPK for the
treatment of DM1. 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 Company.
Isis Pharmaceuticals® is a registered trademark of Isis Pharmaceuticals, Inc. KYNAMRO™ is a trademark of Genzyme Corporation.
Contact: MEDIA CONTACTS:
Biogen Idec
Amanda Brown Galgay, 781-464-3260
Senior Manager, Public Affairs
or
Isis Pharmaceuticals
Amy Blackley, 760-603-2772
Associate Director, Corporate Communications
or
INVESTOR CONTACTS:
Biogen Idec
Ben Strain, 781-464-2442
Senior Manager, Investor Relations
or
Isis Pharmaceuticals
Kristina Lemonidis, 760-603-2490
Director, Investor Relations
Posted: June 2012
