CARLSBAD, Calif., April 20, 2021 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS) today announced it has
initiated a pivotal clinical study of ION373 in patients with
Alexander disease, a rare and generally fatal neurological
disorder. Alexander disease patients experience progressive
deterioration, leading to severe disability and loss of
independence. Childhood onset is associated with more serious
disease, with patients rarely surviving beyond adolescence.
Alexander disease is caused by a genetic mutation that leads to
overproduction and toxic accumulation of glial fibrillary acidic
protein (GFAP) in the brain. ION373 is an investigational antisense
medicine designed to reduce the level of GFAP.
ION373 is one of Ionis' wholly owned medicines that the company
plans to commercialize as part of its strategy to develop and
commercialize rare disease medicines primarily focused on its core
neurological and cardiometabolic franchises.
"Initiation of a pivotal study of ION373 marks an important step
toward bringing a novel disease modifying therapy to Alexander
disease patients afflicted by this devastating disorder. The
ability to begin our clinical program with a pivotal study serves
as a testament to how Ionis' long-standing collaboration with the
patient, medical and research communities can lay the foundation
for an aggressive clinical path," said C. Frank Bennett, Ph.D., Ionis' chief scientific
officer and franchise leader for neurological programs. "This study
is yet another example of the power of Ionis' antisense technology
to target the root causes of neurological diseases, delivering
potentially transformative treatments to patients, and futures to
families who previously had little hope."
Alexander disease has been estimated to occur in about one in
one million births. ION373 has received orphan drug designation and
rare pediatric disease designation from the U.S. Food and Drug
Administration (FDA). The European Medicines Agency (EMA) has also
granted orphan drug status to ION373.
The Phase 2/3 study of ION373 is a multi-center, double-blind,
placebo-controlled, multiple-ascending dose (MAD) study in up to 58
patients with Alexander disease. Patients will receive ION373 or
placebo for a 60-week period, after which all patients in the study
will receive ION373 for a 60-week open-label treatment period.
Learn more about the pivotal trial of ION373 at:
About Ionis' Neurology Franchise
The Ionis neurology franchise addresses all major brain regions
and central nervous system cell types and currently has three Phase
3 studies ongoing with 11 medicines in clinical development, three
of which are wholly owned. Ionis is leading the way in treating the
root causes of many neurological diseases and developing antisense
medicines for common diseases like Alzheimer's and Parkinson's as
well as rare diseases like amyotrophic lateral sclerosis (ALS) and
Alexander disease. Ionis' marketed neurological disease medicines
include SPINRAZA®, the global foundation of care for
spinal muscular atrophy (SMA), commercialized by Biogen, and
TEGSEDI®, the first and only self-administered,
subcutaneous treatment for the polyneuropathy of hereditary ATTR
amyloidosis in adults.
About Alexander disease
Alexander disease is a rare neurological condition characterized
as a leukodystrophy, or a disease affecting the myelin sheath (the
fatty insulation that protects a nerve fiber and supports signal
conduction). It is caused by overproduction and toxic accumulation
of GFAP in astrocytes. Clinically, Alexander disease presents with
a range of symptoms that may include seizures, motor and cognitive
development delay, encephalopathy, weakness, spasticity,
pseudobulbar/bulbar symptoms and macrocephaly. Age of disease onset
is variable, occurring from birth through young adulthood, with
childhood onset generally more severe. There are treatments that
can relieve symptoms, but there is no disease modifying therapy yet
available to patients.
About Ionis Pharmaceuticals
For more than 30 years, Ionis has been the leader in
RNA-targeted therapy, pioneering new markets and changing standards
of care with its novel antisense technology. Ionis currently has
three marketed medicines and a premier late-stage pipeline
highlighted by industry-leading neurological and cardiometabolic
franchises. Our scientific innovation began and continues with the
knowledge that sick people depend on us, which fuels our vision of
becoming one of the most successful biotechnology companies.
To learn more about Ionis visit www.ionispharma.com and follow
us on twitter @ionispharma.
Ionis' Forward-looking Statement
This press release includes forward-looking statements regarding
Ionis' business, Ionis' technologies, ION373 and other products in
development. 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, including those related to the impact COVID-19 could
have on our business, and including but not limited to those
related to our commercial products and the medicines in our
pipeline, and particularly those inherent in the process of
discovering, developing and commercializing medicines that are safe
and effective for use as human therapeutics, and in the endeavor of
building a business around such medicines. 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, 2020, which is on
file with the SEC. Copies of this 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
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SOURCE Ionis Pharmaceuticals, Inc.