CARLSBAD, Calif., July 14, 2015 /PRNewswire/ -- Isis
Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has
earned a $2.15 million milestone
payment from Biogen related to advancing the ongoing pivotal Phase
3 study (CHERISH) evaluating ISIS-SMNRx in children with
spinal muscular atrophy (SMA). To date, Isis
has generated more than $120
million in payments from Biogen related to the development
of ISIS-SMNRx.
CHERISH, a Phase 3 study of ISIS-SMNRx, is a
randomized, double-blind, sham-procedure controlled fifteen month
study in approximately 120 children who are non-ambulatory with SMA
between the ages of 2 to 12. The study will evaluate the
efficacy and safety of 12 mg doses of ISIS-SMNRx with a
primary endpoint of a change in the Hammersmith Functional Motor
Scale-Expanded (HFMSE), a validated method to measure changes in
muscle function in patients with SMA. 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 SMA
SMA is a severe genetic disease that affects approximately
30,000 to 35,000 patients in the United
States, Europe and
Japan. There are no approved treatments for SMA. The
disease 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 that are responsible for
neuromuscular growth and function. One in 50 people, the
equivalent of about six 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.
Natural history studies have been conducted in patients with
SMA. Type I is the most severe form of SMA and most infants
with Type I SMA die in infancy. In a 2009 paper by
Rudnik-Schöneborn[i], the median age for event-free survival in
infants with Type I SMA was 6.1 months. In a contemporaneous
study published in 2014 by the Pediatric Neuromuscular Clinical
Research group (PNCR)[ii], the median age for event-free survival
in infants with two copies of SMN2 was 10.5 months. The
severity of SMA correlates with the amount of SMN protein.
Infants with Type I SMA 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 walk. Children with Type III
have a normal lifespan but accumulate life-long physical
disabilities as they grow.
ABOUT ISIS-SMNRx
ISIS-SMNRx is designed to alter the splicing of SMN2,
a gene that is closely related to SMN1, to increase production of
fully functional SMN protein. The United States Food and Drug
Administration granted orphan drug status and fast track
designation to ISIS-SMNRx for the treatment of patients
with SMA. Isis is currently collaborating with Biogen to
develop and potentially commercialize the investigational compound,
ISIS-SMNRx, for the treatment of SMA. Under the
terms of the January 2012 agreement,
Isis is responsible for global development and Biogen has the
option to license the compound. In addition to the pivotal
studies described below, Biogen is operationalizing two Phase 2
studies (NURTURE & EMBRACE) to augment the ongoing Phase 3
program.
Isis is conducting two Phase 3 studies of
ISIS-SMNRx. One Phase 3 study, ENDEAR, in infants
with SMA and a second Phase 3 study, CHERISH, in children with
SMA. The ENDEAR study 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 ISIS-SMNRx with a primary
endpoint of event-free survival. The CHERISH study is a
randomized, double-blind, sham-procedure controlled fifteen month
study in approximately 120 non-ambulatory children with SMA.
The study will evaluate the efficacy and safety of
ISIS-SMNRx with a primary endpoint of a change in
Hammersmith Functional Motor Scale-Expanded.
For further study information, please visit
www.clinicaltrials.gov and search for ISIS-SMNRx or
visit the ISIS-SMNRx study site at www.smastudy.com.
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 ISIS and BIOGEN
Isis and Biogen have a broad strategic alliance 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's capability to develop therapies for neurological
disorders. Isis is primarily responsible for drug discovery
and early development of antisense therapies. Biogen 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. In addition to these four drugs,
Isis and Biogen have numerous opportunities to evaluate additional
targets for the development of drugs to treat neurological
disorders.
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
volanesorsen, a drug Isis is developing and plans to commercialize
through its wholly owned subsidiary, Akcea Therapeutics, to treat
patients with familial chylomicronemia syndrome and familial
partial lipodystrophy; ISIS-TTRRx, a drug Isis is
developing with GSK to treat patients with the polyneuropathy and
cardiomyopathy forms of TTR amyloidosis; and ISIS-SMNRx,
a drug Isis is developing with Biogen 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, 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, 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," "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.
[i] Rudnik-Schöneborn S, Berg C, Zerres K, et al.
Genotype-phenotype studies in infantile spinal muscular atrophy
(SMA) type 1 in Germany:
implications for clinical trials and genetic counselling.
Clin Genet.
2009;76(2):168-78.
[ii] Finkel RS et al. Observational study of spinal muscular
atrophy type I and implications for clinical trials. Neurology.
2014 Aug 26;83(9):810-7.
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SOURCE Isis Pharmaceuticals, Inc.