- Interim results show sustained reduction in hereditary
angioedema attacks and no new safety signals in patients treated
for one year
CARLSBAD, Calif., Nov. 13,
2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.
(Nasdaq: IONS) today presented positive results from a Phase 2
open-label extension (OLE) study evaluating the safety and efficacy
of its investigational antisense medicine, donidalorsen, in
patients with hereditary angioedema (HAE), a rare and devastating
inflammatory disease. Interim data after all patients completed 1
year of treatment in the study showed a sustained reduction in HAE
attacks and no new safety signals following treatment with
donidalorsen. Treatment with donidalorsen resulted in an overall
sustained mean reduction in HAE attack rates of 95% from baseline.
For patients treated with donidalorsen, 99.6% of study days were
HAE attack-free.
The data were presented at the American College of Allergy,
Asthma & Immunology (ACAAI) Annual Scientific Meeting in
Louisville, Ky. Additional details
can be found on the ACAAI website.
"Today's data further enhance donidalorsen's profile and
potential to provide significant sustained protection from attacks
for people living with hereditary angioedema," said Richard S. Geary, Ph.D., executive vice
president and chief development officer at Ionis. "The positive
results of the Phase 2 OLE are encouraging as we continue
evaluating donidalorsen, a potential best-in-class medicine, in the
ongoing OASIS Phase 3 program."
Patients completing the 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.
Patients who remained on donidalorsen 80 mg every 4 weeks had a
mean reduction in attack rate of 95.3% and 98.3%, from Week 1
(after first dose) and Week 5 (after second dose), respectively.
Patients receiving donidalorsen 80 mg every eight weeks experienced
a mean reduction in attack rate of 75.6% from baseline and the mean
monthly attack rate was 0.28. Five of these patients remained
attack free over the one-year duration of this analysis, and three
patients returned to 80 mg every four weeks.
No serious adverse events were reported in the OLE study and no
treatment-emergent adverse events (TEAEs) led to study
discontinuation. There were no clinically relevant abnormalities in
any laboratory measurements.
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 affects
approximately 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
may 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. HAE is a rare genetic disease
characterized by rapid and painful attacks of inflammation in the
hands, feet, limbs, face, abdomen, larynx, and trachea. HAE 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 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 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 December 31, 2021, and the most
recent Form 10-Q quarterly filing, which is 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.
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SOURCE Ionis Pharmaceuticals, Inc.