- Zilganersen is the first investigational medicine in clinical
development for adults and children living with Alexander disease,
an ultra-rare neurological condition
CARLSBAD, Calif., Oct. 1, 2024
/PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS)
announced today that the U.S. Food and Drug
Administration (FDA) has granted zilganersen Fast Track
designation for the treatment of children and adults with an
ultra-rare, progressive and ultimately fatal neurological disorder
known as Alexander disease (AxD). Topline data from the pivotal
study of zilganersen is expected in the second half of 2025. The
FDA grants investigational medicines Fast Track designation to
facilitate the development and expedite the review of medicines
that demonstrate the potential to treat serious conditions and fill
an unmet medical need.
"With no approved treatments available for people living with
AxD, receiving this Fast Track designation for zilganersen reflects
the seriousness of this ultra-rare disease and the significant
unmet need for treatment in this patient population,"
said Eugene Schneider, M.D., executive vice president and
chief clinical development officer at Ionis. "Zilganersen was
designed to address the underlying cause of disease and help
improve the functioning of people living with AxD. We look forward
to a data readout next year and working closely with the FDA to
potentially bring forward the first approved AxD treatment."
The pivotal Phase 1-3 study of zilganersen in adults and
children living with AxD completed enrollment earlier this year
across 13 sites in eight countries. More information on the study
(NCT04849741) is available at www.clinicaltrials.gov.
About Alexander Disease (AxD)
AxD is an ultra-rare, progressive and ultimately fatal type of
leukodystrophy, which are a group of genetic disorders that affect
the brain's white matter. AxD is estimated to occur in
approximately one in one million to one in three million people
worldwide and usually leads to death within 14 - 25 years after
symptom onset. AxD can present throughout life as loss of
independence and lack of ability to control muscles for swallowing,
airway protection and purposeful movements, though the impact of
AxD can vary depending on factors like age of onset. Diagnosing AxD
is based on a combination of clinical presentation, brain magnetic
resonance imaging (MRI) findings and genetic testing. AxD is caused
by changes in the glial fibrillary acidic protein (GFAP) gene that
lead to a build-up of abnormal proteins in cells, causing
progressive damage to the nervous system. There are no medicines
approved for people with AxD, and current treatments focus on
managing their symptoms.
About Zilganersen (ION373)
Zilganersen is an investigational antisense oligonucleotide
medicine being developed as a potential treatment for people with
genetically confirmed Alexander disease (AxD). Zilganersen is
designed to stop the excess glial fibrillary acidic protein (GFAP)
production that accumulates because of disease-causing variants in
the GFAP gene. In 2020, the U.S. Food and Drug
Administration (FDA) granted zilganersen Orphan Drug
designation and Rare Pediatric Disease designation. In
addition, the European Medicines Agency (EMA) granted
zilganersen Orphan Drug designation in 2019.
About Ionis Pharmaceuticals, Inc.
For three decades,
Ionis has invented medicines that bring better futures to people
with serious diseases. Ionis currently has five marketed medicines
and a leading pipeline in neurology, cardiology, and other areas of
high patient need. As the pioneer in RNA-targeted medicines, Ionis
continues to drive innovation in RNA therapies in addition to
advancing new approaches in gene editing. A deep understanding of
disease biology and industry-leading technology propels our work,
coupled with a passion and urgency to deliver life-changing
advances for patients.
To learn more about Ionis, visit Ionis.com and follow us on X
(Twitter) and LinkedIn.
Ionis Forward-looking
Statements
This press release includes forward-looking
statements regarding Ionis' business, and the therapeutic and
commercial potential of Ionis' commercial medicines, zilganersen,
additional medicines in development and technologies. 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 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. Except as required by law, we undertake no obligation to
update any forward-looking statements for any reason. 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 Dec. 31, 2023, and most recent
Form 10-Q, which are on file with the SEC. Copies of these and
other documents are available at www.Ionis.com.
In this press release, unless the context requires otherwise,
"Ionis," "Company," "we," "our" and "us" all refer to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a registered trademark of Ionis
Pharmaceuticals, Inc.
Zilganersen is an investigational medicine that has not been
approved for the treatment of any disease by regulatory
authorities.
Ionis Pharmaceuticals Investor Contact: D. Wade Walke, Ph.D. - IR@ionis.com -
760-603-2331
Ionis Pharmaceuticals Media Contact: Hayley Soffer - Media@ionis.com -
760-603-4679
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SOURCE Ionis Pharmaceuticals, Inc.