CARLSBAD, Calif., Nov. 7, 2021 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted
therapies, announced today that positive results from the Phase 2
study of its investigational antisense medicine, donidalorsen
(formerly IONIS-PKK-LRx), will be presented at the
American College of Asthma, Allergy & Immunology (ACAAI) Annual
Scientific Meeting in New Orleans
and via livestream, November 4-8. The
Phase 2 study results support the clinical profile of donidalorsen
as a potential, best-in-class prophylactic treatment for patients
with hereditary angioedema (HAE), and underscore Ionis' commitment
to advancing antisense technology to target the root cause of
diseases.
HAE is a rare and potentially fatal autosomal dominant disease
that results in recurrent, painful attacks of swelling affecting
the hands, feet, limbs, face, abdomen, larynx and trachea.
Donidalorsen is an investigational antisense medicine designed to
reduce the production of prekallikrein, which plays a key role in
the activation of inflammatory mediators associated with acute
attacks of HAE. Donidalorsen was developed using Ionis' advanced
LIgand-Conjugated Antisense (LICA)
technology.
Topline results of the Phase 2 study, reported earlier this
year, showed that donidalorsen met its primary and all secondary
endpoints, achieving significant reductions in the number of
attacks suffered by patients with hereditary angioedema (HAE)
compared to placebo. These data support advancing donidalorsen into
Phase 3 development, which Ionis plans to initiate this year.
"A Phase 2 Study Evaluating an Antisense Oligonucleotide to
Prekallikrein in Patients with Hereditary Angioedema" will
be presented on Sunday, Nov. 7 at
3:03 p.m. CT (recorded presentation)
by Danny Cohn, M.D., Ph.D.,
Department of Vascular Medicine, Amsterdam Universities Medical
Center, Amsterdam, The
Netherlands. Dr. Cohn, a primary investigator in the study,
will present results of the Phase 2 study during Oral Abstracts
Session 2B
(Angioedema/urticaria, food allergy, allergy diagnostics and
immunotherapy). Additional details can be found on the
ACAAI website.
About Hereditary Angioedema
HAE is a rare genetic
disease that is characterized by rapid and painful attacks of
inflammation in the hands, feet, limbs, face, abdomen, larynx and
trachea. HAE affects approximately 20,000 patients in the United States and Europe and can be fatal if swelling occurs in
the larynx. In patients with frequent or severe attacks, doctors
may use prophylactic treatment approaches to prevent and reduce the
severity of HAE attacks.
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 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, and
the therapeutic and commercial potential of Ionis' technologies,
donidalorsen 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, 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.
Ionis Media Relations Contact:
760-603-4679
Ionis Investor Relations Contact:
760-603-2331
View original content to download
multimedia:https://www.prnewswire.com/news-releases/phase-2-study-results-of-ionis-novel-antisense-treatment-for-hereditary-angioedema-to-be-presented-at-acaai-annual-meeting-301418049.html
SOURCE Ionis Pharmaceuticals, Inc.