CARLSBAD, Calif., March 6, 2015 /PRNewswire/ -- Isis
Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has
earned a $9 million milestone payment
from Biogen Idec related to advancing the ongoing pivotal Phase 3
study evaluating ISIS-SMNRx in infants with spinal
muscular atrophy (SMA).
ENDEAR, a Phase 3 study of ISIS-SMNRx, is a
randomized, double-blind, sham-procedure controlled thirteen month
study in approximately 110 infants diagnosed with SMA. The
study will evaluate the efficacy and safety of a 12 mg dose of
ISIS-SMNRx with a primary endpoint of survival or time
to permanent ventilation. Additional efficacy endpoints are
also included in the study. For further study information,
please visit www.clinicaltrials.gov and search for
ISIS-SMNRx or the identifier number NCT02193074 or visit
the ISIS-SMNRx study site at www.smastudy.com.
ABOUT ISIS-SMNRx
ISIS-SMNRx is designed to alter the splicing of a
closely related gene (SMN2) to increase production of fully
functional SMN protein. The FDA granted orphan drug status
and fast track designation to ISIS-SMNRx for the
treatment of patients with SMA. Isis is currently in
collaboration with Biogen Idec to develop and potentially
commercialize the investigational compound, ISIS-SMNRx,
to treat all types of SMA. Under the terms of the
January 2012 agreement, Isis is
responsible for global development and Biogen Idec has the option
to license the compound. Isis is conducting two Phase 3
studies with agreement from the FDA for special protocol
assessments, or SPAs. A SPA is a written agreement between the FDA
and a drug sponsor intended to confirm that the clinical trial
protocol is adequate to meet current scientific and regulatory
requirements for a potential new drug application.
Isis acknowledges support from the following organizations for
ISIS-SMNRx: Muscular Dystrophy Association, SMA
Foundation, Cure SMA and intellectual property licensed from Cold
Spring Harbor Laboratory and the University of
Massachusetts Medical School.
ABOUT SMA
SMA is a severe genetic disease that affects approximately
30,000-35,000 patients in the United
States, Europe and
Japan. SMA is caused by a
loss of, or defect in, the survival motor neuron 1 (SMN1) gene
leading to a decrease in the survival motor neuron (SMN) protein.
SMN is critical to the health and survival of nerve cells in
the spinal cord responsible for neuromuscular growth and function.
One in 50 people, the equivalent of about 6 million people in
the United States, are carriers of
a defective SMN1 gene, which is unable to produce fully functional
SMN protein. Carriers experience no symptoms and do not develop the
disease. However, when both parents are carriers, there is a
one in four chance that their child will have SMA. The
severity of SMA correlates with the amount of SMN protein.
Infants with Type I SMA, the most severe form of the disease,
produce very little SMN protein and have a life expectancy of less
than two years. Children with Type II have greater amounts of SMN
protein but still have a shortened lifespan and are never able to
stand independently. Children with Type III have a normal
lifespan but accumulate life-long physical disabilities as they
grow.
ABOUT ISIS and BIOGEN IDEC
Biogen Idec and Isis have established four collaborations
focused on leveraging antisense technology to advance the treatment
of neurological and neuromuscular disorders. This alliance
combines Isis' expertise in antisense technology to evaluate
potential neurological targets and discover antisense drugs with
Biogen Idec's capability to develop therapies for neurological
disorders. Isis is primarily responsible for drug discovery
and early development of antisense therapies. Biogen Idec has
the option to license each antisense program at a particular stage
in development. Current development-stage programs include
antisense drugs to treat patients with spinal muscular atrophy
(SMA), ISIS-SMNRx, myotonic dystrophy type 1 (DM1),
ISIS-DMPKRx, and two undisclosed neurodegenerative
diseases, ISIS-BIIB3Rx and ISIS-BIIB4Rx.
ABOUT ISIS PHARMACEUTICALS, INC.
Isis is exploiting its leadership position in RNA-targeted
technology to discover and develop novel drugs for its product
pipeline and for its partners. Isis' broad pipeline consists
of 38 drugs to treat a wide variety of diseases with an emphasis on
cardiovascular, metabolic, severe and rare diseases, including
neurological disorders, and cancer. Isis' partner, Genzyme,
is commercializing Isis' lead product, KYNAMRO®, in
the United States and other
countries for the treatment of patients with homozygous FH.
Isis has numerous drugs in Phase 3 development in severe/rare
diseases and cardiovascular diseases. These include
ISIS-APOCIIIRx, a drug Isis is developing through its
wholly owned subsidiary, Akcea Therapeutics, to treat patients with
severely high triglycerides, such as patients with familial
chylomicronemia syndrome; ISIS-TTRRx, a drug Isis is
developing with GSK to treat patients with the polyneuropathy form
of TTR amyloidosis; and, ISIS-SMNRx, a drug Isis is
developing with Biogen Idec to treat infants and children with
spinal muscular atrophy, a severe and rare neuromuscular
disease. Isis' patents provide strong and extensive
protection for its drugs and technology. Additional
information about Isis is available at www.isispharm.com.
ISIS PHARMACEUTICALS' FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding
Isis' alliance with Biogen Idec, the discovery, development,
activity, therapeutic and commercial potential and safety of
ISIS-SMNRx and the discovery, development and
therapeutic potential of an antisense drug for the treatment of
spinal muscular atrophy. 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. 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,
2014, which is on file with the SEC. Copies of this
and other documents are available from the Company.
In this press release, unless the context requires otherwise,
"Isis," "Company," "we," "our," and "us" refers to Isis
Pharmaceuticals and its subsidiaries.
Isis Pharmaceuticals® is a registered trademark of
Isis Pharmaceuticals, Inc. Akcea Therapeutics™ is a trademark
of Isis Pharmaceuticals, Inc. KYNAMRO® is a
registered trademark of Genzyme Corporation.
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SOURCE Isis Pharmaceuticals, Inc.