- Phase 2 open-label study data reinforce donidalorsen's
potential to be a best-in-class prophylactic treatment for patients
living with hereditary angioedema
- Ionis is preparing to launch donidalorsen, one of its three
near-term commercial opportunities
CARLSBAD, Calif., Feb. 21,
2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.
(Nasdaq: IONS) today announced additional positive interim data
from a Phase 2 open-label extension (OLE) study of donidalorsen, an
investigational antisense medicine for the treatment of patients
with hereditary angioedema (HAE). Positive interim data presented
in November 2022 showed that
treatment with donidalorsen resulted in an overall sustained mean
reduction in HAE attack rates of 95% from baseline. In the latest
update, patients treated for one year with donidalorsen showed a
clinically meaningful 24-point mean improvement in their Angioedema
Quality of Life (AE-QoL) total score relative to baseline with
improvements observed in all domains. An improvement of 6 points or
more is considered clinically meaningful1.
These long-term, open-label data will be presented at the
American Academy of Allergy Asthma & Immunology (AAAAI) Annual
Meeting in San Antonio, Texas,
February 24-27. Additional details
can be found on the AAAAI website.
"The improvement in quality of life demonstrated in patients
treated with donidalorsen for one year were clinically meaningful
and further support our belief in this medicine's potential to be a
best-in-class prophylactic treatment for patients with HAE," said
Richard S. Geary, Ph.D., executive
vice president and chief development officer at Ionis. "We continue
to be pleased with the progress of the Phase 3 OASIS study of
donidalorsen, which remains on track to complete enrollment this
year."
Poster titles:
- Poster #399: Phase 2 Open-Label Extension Study with
Donidalorsen in Patients with Hereditary Angioedema: Updated
Interim Analysis with Quality-of-Life Data
- Poster #400: Phase 2 Open-Label Extension Study with
Donidalorsen Treatment in Patients with Hereditary Angioedema:
Pharmacodynamic Data
- Poster #412: The Impact of Donidalorsen Taken Every 8
Weeks in Patients with Hereditary Angioedema
About the Phase 2 OLE Study
Patients who completed the blinded, placebo-controlled Phase 2
study were eligible for enrollment in the OLE study. There were 20
Type 1 or Type 2 HAE patients in the Phase 2 study, and 17 (85%)
entered the OLE. Following a 13-week fixed-dose period where
participants received subcutaneous donidalorsen 80 mg every four
weeks, eight patients switched to subcutaneous donidalorsen 80 mg
every eight weeks.
About Hereditary Angioedema (HAE)
HAE is a rare and potentially fatal genetic disease
characterized by rapid and painful attacks of inflammation in the
hands, feet, limbs, face, abdomen, larynx, and trachea. HAE is
estimated to affect more than 20,000 patients in
the U.S. and Europe and can be fatal if
swelling occurs in the larynx. In patients with frequent or severe
attacks, doctors frequently use prophylactic treatment approaches
to prevent and reduce the severity of HAE attacks.
About Donidalorsen
Donidalorsen is an investigational antisense medicine that uses
Ionis'
advanced LIgand-Conjugated Antisense
(LICA) technology and is designed to reduce the production of
prekallikrein, or PKK, to treat patients with
HAE. PKK plays an important role in the activation of
inflammatory mediators associated with acute attacks of HAE.
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 promising 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 to
become the leader in genetic medicine, utilizing a multi-platform
approach to discover, develop and deliver life-transforming
therapies.
To learn more about Ionis visit www.ionispharma.com and follow
us on Twitter @ionispharma.
Ionis' Forward-looking Statements
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 Dec. 31, 2021, and the most
recent Form 10-Q quarterly filing, which are on file with the
Securities and Exchange Commission. 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 Weller K, et al. Allergy. 2016;71(8):
1203-1209.
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SOURCE Ionis Pharmaceuticals, Inc.