CARLSBAD, Calif., July 21, 2015 /PRNewswire/ -- Isis
Pharmaceuticals, Inc. (NASDAQ: ISIS) today announced that it has
initiated a Phase 1/2a clinical study of ISIS-HTTRx in
patients with Huntington's disease (HD). ISIS-HTTRx is
the first therapy to enter clinical development that is designed to
directly target the cause of the disease by reducing the production
of the protein responsible for HD. HD is a rare genetic
neurological disease in which patients experience deterioration of
both mental abilities and physical control. Presently, there are no
disease-modifying treatments for HD, with current therapies focused
only on treating disease symptoms.
ISIS-HTTRx has been granted orphan drug
designation by the European Medicines Agency for the treatment of
patients with HD. Orphan drug designation is granted to
products designed to diagnose, prevent or treat life-threatening or
very serious conditions that affect not more than five in 10,000
persons in the European Union.
"Although the toxic protein produced from the huntingtin (HTT)
gene in HD patients has been a target of interest for many years,
no therapies have advanced to clinical trials to treat the
underlying cause of the disease. Our antisense technology has
enabled us to discover and develop ISIS-HTTRx, the first
therapeutic approach designed to treat the genetic cause of HD.
Together with Roche, we are committed to investigating this
approach to treat patients with HD, a devastating disease that
typically affects generations of families," said C. Frank Bennett, Ph.D., senior vice president
of research at Isis Pharmaceuticals.
"Initiating the clinical study of ISIS-HTTRx in
patients with HD is the first step in developing a treatment that
could significantly impact a patient's disease. It is also an
important milestone in our collaboration with Roche. As we advance
this program, we will continue to benefit from Roche's scientific
expertise in developing therapeutics for neurodegenerative
conditions," said B. Lynne Parshall,
chief operating officer of Isis Pharmaceuticals.
The randomized, placebo-controlled, dose escalation Phase 1/2a
clinical study will evaluate the safety and activity of
ISIS-HTTRx in patients with early stage
HD. In this study, ISIS-HTTRx
will be administered intrathecally as an injection
directly into the cerebral spinal fluid. Intrathecal administration
of antisense drugs has been shown to be well tolerated in multiple
clinical studies in patients.
"The initial development of this antisense drug for Huntington's
disease came out of a longstanding productive partnership between
Isis and CHDI, and its advancement now to clinical trial is
testament to Isis' perseverance and scientific expertise," said
Robi Blumenstein, president of CHDI
Management, which oversees the activities of CHDI Foundation, a
nonprofit research organization exclusively dedicated to the
development of therapies that will slow the progression of HD.
"It's exciting that therapeutic candidates grounded in the biology
of Huntington's disease are finally making their way to clinical
trial."
ABOUT ISIS and ROCHE
Roche and Isis are collaborating
to develop antisense drugs to treat HD. The alliance combines Isis'
antisense expertise with Roche's scientific knowledge in developing
neurodegenerative therapeutics. With the initiation of the Phase
1/2a study for ISIS-HTTRx, Isis earned a $22 million milestone payment from Roche. To
date, Isis has earned $52 million in
upfront and milestone payments from its relationship with Roche and
is eligible to earn additional milestone payments as the drug
progresses in development, as well as royalties on sales of
ISIS-HTTRx if it is commercialized. Roche has the option
to license ISIS-HTTRx from Isis through the completion
of the Phase 1/2a study. Prior to option exercise, Isis is
responsible for the discovery and development of
ISIS-HTTRx. If Roche exercises its option, it will
assume responsibility for global development, regulatory and
commercialization activities for the drug.
CHDI Foundation, Inc. provided financial and scientific support
to Isis' HD drug discovery program through a development
collaboration with Isis. Over time, CHDI will be reimbursed for its
support of Isis' program out of milestone payments received by
Isis.
ABOUT ISIS-HTTRx and Huntington's Disease
ISIS-HTTRx is a Gen. 2.0+ antisense drug in development
for the treatment of Huntington's disease. ISIS-HTTRx
is designed to reduce the production of all forms
of the huntingtin protein, which is the protein responsible for HD.
As such, ISIS-HTTRx offers a unique approach to
treat all patients with HD. HD is a rare genetic, progressive
neurological disease resulting in deterioration in mental abilities
and physical control. HD is referred to as a triplet repeat
disorder, and is one of a large family of genetic diseases in which
certain gene sequences are mistakenly repeated. In HD, the gene
that encodes for the HTT protein contains a trinucleotide sequence
that is repeated in the gene more than 36 times. The resulting HTT
protein is toxic and gradually damages neurons in the brain.
Symptoms of HD usually appear between the ages of 30 to 50 years,
and continually worsen over a 10 to 25 year period. Ultimately, the
weakened individual succumbs to pneumonia, heart failure or other
complications. Presently, there is no effective disease modifying
treatment, and current approaches only focus on managing the
severity of some disease symptoms.
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 Roche, the development, activity, therapeutic
potential, commercial potential and safety of
ISIS-HTTRx. 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.
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SOURCE Isis Pharmaceuticals, Inc.