CARLSBAD, Calif., Oct. 13, 2017 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS) today announced that it has
initiated a Phase 1/2a clinical study of IONIS-MAPTRx in
patients with mild Alzheimer's disease (AD). Ionis earned a
$10 million milestone payment from
Biogen related to the initiation of this study.
IONIS-MAPTRx is an antisense drug designed to
selectively reduce the production of microtubule-associated protein
tau (MAPT), or tau protein, in the brain. Tau misfolding leads to
the accumulation of neurofibrillary tangles inside brain cells.
These tangles are a hallmark feature of a spectrum of
neurodegenerative diseases known as tauopathies, which include AD
and some forms of frontotemporal dementia (FTD). AD is the most
common form of dementia, accounting for an estimated 70% of cases.
FTD is a rare form of early-onset dementia resulting from neuronal
damage in the frontal and temporal lobes of the brain.
"In contrast to amyloid plaques that may begin to deposit in the
brain for up to 20 years before the onset of AD, tau deposits are
spatially and temporally associated with the brain regions where
atrophy occurs and neurocognitive deficits originate.
IONIS-MAPTRx is designed to reduce the production of all
of the many forms of tau in all regions of the brain," said
C. Frank Bennett, Ph.D., senior vice
president of research at Ionis Pharmaceuticals.
The three-month randomized, placebo-controlled, dose escalation
Phase 1/2a clinical study will evaluate the safety and activity of
IONIS-MAPTRx in approximately 44 patients with mild AD.
In the study, IONIS-MAPTRx is administered as a
once-monthly intrathecal injection directly into the cerebral
spinal fluid, similar to the way SPINRAZA® (nusinersen)
is administered. Biogen has an option to develop and
commercialize IONIS-MAPTRx.
"IONIS-MAPTRx is another exciting program within our
strategic collaboration with Biogen," said B. Lynne Parshall, chief operating officer of
Ionis Pharmaceuticals. "Targeting MAPT represents a unique
opportunity to address the unmet need in both rare and more
prevalent tauopathies. The first clinical study with
IONIS-MAPTRx is in patients with AD, however, in
parallel, we also plan to develop IONIS-MAPTRx for
patients with FTD, a rare disease population. This strategy has the
potential to accelerate the drug's path to market."
ABOUT IONIS AND BIOGEN
Ionis and Biogen have a broad
strategic collaboration combining Biogen's expertise in neurology
with Ionis' leadership in antisense technology to develop novel
therapies to treat neurological disorders. SPINRAZA, an antisense
drug developed with Ionis Pharmaceuticals' proprietary antisense
technology is the first commercial drug from this collaboration.
SPINRAZA is approved in global markets for the treatment of
patients with spinal muscular atrophy (SMA). Biogen is responsible
for commercialization of SPINRAZA. Also under this collaboration,
Ionis and Biogen are developing IONIS-SOD1Rx for
amyotrophic lateral sclerosis (ALS), and IONIS-BIIB5Rx,
IONIS-BIIB6Rx, and IONIS-BIIB7Rx to treat
undisclosed neurodegenerative diseases.
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 TTR amyloidosis, 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. Volanesorsen filings for marketing
approval have been submitted in the U.S., EU and Canada. Inotersen is progressing toward
regulatory filings for marketing authorization. Ionis' patents
provide strong and extensive protection for its drugs and
technology. Additional information about Ionis is available at
www.ionispharma.com.
IONIS PHARMACEUTICALS' FORWARD-LOOKING STATEMENT
This
press release includes forward-looking statements regarding Ionis'
strategic relationship with Biogen and the development, activity,
therapeutic potential, commercial potential and safety of
IONIS-MAPTRx.
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.
View original content with
multimedia:http://www.prnewswire.com/news-releases/ionis-pharmaceuticals-initiates-clinical-study-of-ionis-mapt-rx-in-patients-with-alzheimers-disease-300536094.html
SOURCE Ionis Pharmaceuticals, Inc.