CARLSBAD, Calif., Dec. 11, 2017 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that Roche has
exercised its option to license IONIS-HTTRx following
the completion of a Phase 1/2a randomized, placebo-controlled, dose
escalation study of IONIS-HTTRx in people with
Huntington's disease (HD). Roche will now be responsible for all
development and commercial activities. IONIS-HTTRx is
the first therapy in clinical development designed to target the
underlying cause of HD by reducing the production of the toxic
mutant huntingtin protein (mHTT). In conjunction with the decision
to license IONIS-HTTRx, Ionis earned a $45 million license fee from Roche.
In the Phase 1/2a study, dose-dependent reductions of mHTT were
observed in participants treated with IONIS-HTTRx. In
addition, the safety and tolerability profile of
IONIS-HTTRx observed in the Phase 1/2a study supports
continued development. Ionis and Roche plan to present results from
this study at medical conferences in the first half of 2018 and
plan to submit the study results for publication in a peer-reviewed
medical journal. Ionis and Roche have also recently initiated an
open-label extension (OLE) study for patients who completed the
Phase 1/2a study.
"We are encouraged by the performance of IONIS-HTTRx
in the Phase 1/2a clinical study. The dose-dependent reductions of
mHTT we observed in the study substantially exceeded our
expectations and we were equally encouraged by the safety profile
of the drug," said Dr. C. Frank
Bennett, senior vice president of research at Ionis
Pharmaceuticals. "We are grateful to the patients and investigators
participating in this study. We could not have reached this
important milestone without their commitment and that of the
broader HD community."
"We are extremely pleased that to have reached this important
milestone in our collaboration with Roche to discover and develop a
therapy for people with Huntington's disease," said B. Lynne Parshall, chief operating officer at Ionis
Pharmaceuticals. "This is our second antisense drug targeting a
neurodegenerative disease to demonstrate a positive impact on a
disease target in the CNS."
"The results of this trial are of ground-breaking importance for
Huntington's disease patients and families. For the first time, a
drug has lowered the level of the toxic disease-causing protein in
the nervous system, and the drug was safe and well tolerated. The
key now is to move quickly to a larger trial to test whether
IONIS-HTTRx slows disease progression," said Dr.
Sarah Tabrizi, professor of clinical
neurology, director of the University College London's Huntington
Centre and the global lead investigator on the Phase 1/2a
study.
Roche is now responsible for all IONIS-HTTRx
development, regulatory and commercialization activities and costs.
The two companies will work together to transition the ongoing OLE
to Roche which will be responsible for managing this study and all
future studies.
This transaction is subject to clearances under the
Hart-Scott-Rodino Antitrust Improvements Act.
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 individuals
with symptomatic HD and more than 200,000 people at risk of having
inherited 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 trinucleotide
sequence in the gene that encodes for the HTT protein is repeated
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, and current products focus only on managing 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.
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)
has been approved in global markets for the treatment of
spinal muscular atrophy (SMA). Biogen is responsible for
commercializing SPINRAZA. Drugs that have successfully completed
Phase 3 studies include inotersen, an antisense drug Ionis is
developing to treat patients with hereditary TTR amyloidosis
(hATTR), and volanesorsen, an antisense drug discovered by Ionis
and co-developed by Ionis and Akcea Therapeutics to treat
patients with either familial chylomicronemia syndrome or familial
partial lipodystrophy. Akcea, an affiliate of Ionis, is a
biopharmaceutical company focused on developing and commercializing
drugs to treat patients with serious cardiometabolic diseases
caused by lipid disorders. If approved, volanesorsen will be
commercialized through Ionis' affiliate, Akcea. Inotersen filings
for marketing approval have been submitted in the U.S. and
EU. Volanesorsen filings for marketing approval have been
submitted in the U.S., EU, and Canada. 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.
SOURCE Ionis Pharmaceuticals, Inc.