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-HTT
Rx in patients with Huntington's disease (HD). ISIS-HTT
Rx
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-HTT
Rx 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-HTT
Rx, 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-HTT
Rx 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-HTT
Rx in patients with early stage HD. In this study, ISIS-HTT
Rx 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-HTT
Rx, 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-HTT
Rx if it is commercialized. Roche has the option to license ISIS-HTT
Rx
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-HTT
Rx. 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-HTT
Rx is a Gen. 2.0+ antisense drug in development for the treatment of Huntington's disease. ISIS-HTT
Rx is
designed to reduce the production of all forms of the huntingtin
protein, which is the protein responsible for HD. As such, ISIS-HTT
Rx 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.
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