CARLSBAD, Calif., June 13,
2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.
(Nasdaq: IONS), the leader in RNA-targeted therapeutics, announced
today that the U.S. Food and Drug Administration (FDA) has granted
orphan drug designation and rare pediatric disease designation to
ION582, an investigational antisense medicine for the treatment of
Angelman syndrome, a rare neurogenetic disorder caused by the loss
of function of the maternally inherited Ubiquitin Protein Ligase
E3A (UBE3A) gene. ION582 targets UBE3A.
Angelman syndrome, which affects an estimated one in 12,000 to
20,000 people globally,1 presents early in life
with profound and severe developmental delays in motor, language
and cognitive functioning, seizures and ataxia. It is a
neuro-developmental disorder that generally does not improve
following symptom onset in early childhood, resulting in complete
dependence on a caregiver. Currently, there are no
disease-modifying treatments for Angelman syndrome.
"Receiving FDA orphan drug designation for ION582 reflects
the important and urgent need for delivering an effective treatment
to patients living with Angelman syndrome. Ionis is committed to
working closely with regulators, clinical investigators, patients
and caregivers to advance this novel treatment and make it
available to those who need it," said C. Frank Bennett, Ph.D., executive vice president,
chief scientific officer and franchise leader for neurological
programs at Ionis.
Under the FDA's Orphan Drug Act, orphan drug status provides
incentives, including tax credits, grants and waiver of certain
administrative fees for clinical trials, and seven years of market
exclusivity following drug approval. The FDA defines a rare
pediatric disease as a serious or life-threatening disease that
primarily affects individuals from birth to 18 years of age. Under
the FDA's rare pediatric disease designation and voucher programs,
if Ionis receives marketing approval for ION582 for Angelman
syndrome, the company may qualify for a voucher that can be
redeemed to receive a priority review of a subsequent marketing
application for a different product.
ION582 is being evaluated in a Phase 1/2, open-label,
dose-escalation clinical study in up to approximately 44
participants with Angelman syndrome. For more information on the
HALOS Study (NCT05127226), visit clinicaltrials.gov.
About Ionis Pharmaceuticals,
Inc.
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 cardiovascular and neurological
franchises. Our scientific innovation began and continues with the
knowledge that sick people depend on us, which fuels our vision of
becoming a leading, fully integrated biotechnology company.
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 and the therapeutic and commercial potential of
Ionis' technologies, ION582 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, 2021, and the most
recent Form 10-Q quarterly filing, 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.
1 Mertz LG, Christensen R, Vogel I, Hertz JM,
Nielsen KB, Gronskov K, Ostergaard JR. Angelman syndrome in
Denmark. birth incidence, genetic
findings, and age at diagnosis. Am J Med Genet A.
2013;161A:2197–203.
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SOURCE Ionis Pharmaceuticals, Inc.