CARLSBAD, Calif., June 22, 2017 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS) today announced the completion
of enrollment in the Phase 1/2a randomized, placebo-controlled,
dose escalation study of IONIS-HTTRx in patients with
Huntington's disease (HD). Dosing in the final patient cohort
continues, and Ionis plans to report top-line results from this
study around year-end 2017. The safety and tolerability profile of
IONIS-HTTRx in the completed cohorts of the Phase 1/2a
study supports its continued development. Patients who complete the
Phase 1/2a study will be eligible to participate in an open-label
extension (OLE) study that Ionis plans to initiate in the next
several months. Roche, Ionis' partner for this drug, continues to
advance and support the program. IONIS-HTTRx is the
first therapy in clinical development targeting the cause of HD by
reducing the production of the toxic mutant huntingtin protein
(mHTT) from the mutated huntingtin gene.
"We are encouraged by the safety profile of
IONIS-HTTRx we have observed to date in the completed
dosing cohorts in the Phase 1/2a study. Upon completion and full
analysis of this study, the next step for this program will be to
conduct a study to investigate whether decreasing mutant huntingtin
protein with IONIS-HTTRx can slow the progression of
this terrible disease," said C. Frank
Bennett, Ph.D., senior vice president of research at Ionis
Pharmaceuticals. "We believe that IONIS-HTTRx, which is
designed to reduce the production of all forms of the huntingtin
(HTT) protein – the known cause of HD, represents the most
promising opportunity to address this significant unmet medical
need. Together with our partners at Roche, we are committed to
developing IONIS-HTTRx, which has the potential to
transform the treatment of HD."
ABOUT IONIS-HTTRx and HUNTINGTON'S
DISEASE (HD)
IONIS-HTTRx is an antisense
drug in development for the treatment of HD.
IONIS-HTTRx is designed to reduce the production of
all forms of the huntingtin (HTT) protein, which in its mutated
variant (mHTT) is responsible for HD. As such,
IONIS-HTTRx offers a unique approach to treat all
patients with HD, irrespective of their individual HTT mutation.
IONIS-HTTRx has been granted orphan
drug designation by the U.S. Food and Drug Administration
(FDA) and by the European Medicines Agency (EMA) for the treatment
of patients with HD.
HD is a rare genetic, progressive, neurodegenerative disease
resulting in deterioration in mental abilities and physical
control. In the U.S., there are approximately 30,000 symptomatic
patients and more than 200,000 people at-risk of inheriting HD. 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 mHTT 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 for HD
available, and current approaches only focus on managing some of
the disease symptoms.
ABOUT IONIS/ROCHE COLLABORATION
Roche and
Ionis are collaborating to develop antisense drugs to treat HD. The
alliance combines Ionis' antisense expertise
with Roche's knowledge in clinical development of
anti-neurodegenerative therapeutics. To date, Ionis has
earned $55 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
IONIS-HTTRx if it is
commercialized. Roche has the option to license
IONIS-HTTRx from Ionis through the completion of
the Phase 1/2a study. Prior to option exercise, Ionis is
responsible for the discovery and development of
IONIS-HTTRx. If Roche exercises its option, it will
assume responsibility for global clinical development, regulatory
and commercialization activities for the drug.
CHDI Foundation, Inc. provided financial and scientific
support to Ionis' HD drug discovery program through a development
collaboration with Ionis. Over time, CHDI will be reimbursed for
its support of Ionis' program out of milestone payments received by
Ionis.
ABOUT IONIS PHARMACEUTICALS, INC.
Ionis is the leading
company in RNA-targeted drug discovery and development focused on
developing drugs for patients who have the highest unmet medical
needs, such as those patients with severe and rare diseases. Using
its proprietary antisense technology, Ionis has created a large
pipeline of first-in-class or best-in-class drugs, with over three
dozen drugs in development. SPINRAZA® (nusinersen) is a
drug that has been approved in the U.S. and Europe for the treatment of spinal muscular
atrophy (SMA) in pediatric and adult patients. Biogen is
responsible for commercialization of SPINRAZA. Drugs that have
successfully completed Phase 3 studies include volanesorsen, a drug
Ionis is developing and plans to commercialize through its
subsidiary, Akcea Therapeutics, to treat patients with either
familial chylomicronemia syndrome or familial partial
lipodystrophy; and inotersen (IONIS-TTRRx), a drug Ionis
is developing with GSK to treat patients with TTR amyloidosis. Both
drugs are progressing toward regulatory filings in the second half
of 2017. Ionis' patents provide strong and extensive protection for
its drugs and technology. Additional information about Ionis is
available at www.ionispharma.com.
IONIS' FORWARD-LOOKING STATEMENT
This press release
includes forward-looking statements regarding Ionis' alliance with
Roche and the development, activity, therapeutic potential,
commercial potential and safety of IONIS-HTTRx. Any
statement describing Ionis' 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. Ionis' 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 Ionis'
forward-looking statements reflect the good faith judgment of its
management, these statements are based only on facts and factors
currently known by Ionis. As a result, you are cautioned not to
rely on these forward-looking statements. These and other risks
concerning Ionis' programs are described in additional detail in
Ionis' annual report on Form 10-K for the year ended December 31, 2016, 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,
"Ionis," "Company," "we," "our," and "us" refers to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals,
Inc. Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals,
Inc. SPINRAZA® is a registered trademark of Biogen.
To view the original version on PR Newswire,
visit:http://www.prnewswire.com/news-releases/enrollment-in-phase-12a-study-of-ionis-htt-rx-in-patients-with-huntingtons-disease-completed-and-open-label-extension-study-to-open-in-2h-2017-300477959.html
SOURCE Ionis Pharmaceuticals, Inc.