CARLSBAD, Calif., Nov. 18, 2021 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted
therapies, announced today initiation of OASIS-HAE, the
registrational study in the donidalorsen (formerly
IONIS-PKK-LRx) Phase 3 clinical program. 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 hereditary angioedema (HAE). Donidalorsen uses Ionis'
LIgand-Conjugated Antisense, or LICA,
"Initiating the Phase 3 program for donidalorsen moves us one
step closer to bringing a potential best-in-class prophylactic
treatment to market for people with HAE globally experiencing
recurrent painful and severe HAE attacks," said Kenneth Newman, M.D., M.B.A., Ionis' vice
president of clinical development and leader of the immunology and
pulmonology franchise. "Advancing this program underscores our
commitment to the HAE patient community to deliver transformative
OASIS-HAE is a double-blind, randomized, placebo-controlled
registrational study in up to 84 patients with hereditary
angioedema, Types 1 and 2. Patients will be randomized to receive
monthly or bi-monthly subcutaneous donidalorsen for 25 weeks.
Following the placebo-controlled portion of the study, patients may
enter the 52-week open-label extension study.
New Phase 2 data recently presented at ACAAI highlighted an
overall reduction in moderate to severe attacks, and a reduction in
these attacks starting with the second dose. For the final
month of the study, all donidalorsen treated patients were
attack-free. In this study, donidalorsen was safe and well
For more information on the OASIS-HAE clinical study please
About Donidalorsen Phase 2 study
In the Phase 2 clinical study, 20 adults with Type 1 or Type 2
HAE were randomized and received either donidalorsen 80mg (n=14) or
placebo (n=6) subcutaneously once monthly for 17 weeks. The primary
endpoint was the reduction of monthly HAE attacks compared to
placebo. Secondary endpoints included the reduction of monthly
attacks in weeks five to 17, reduction in the number of moderate or
severe attacks in weeks one to 17, the number of moderate or severe
attacks in weeks five to 17 and the number of attacks requiring
acute therapy in weeks five to 17. The majority of adverse events
during the study were mild with a frequency that was similar
between groups. The most common treatment-emergent adverse events
(TEAEs) were headache and nausea, which were seen more frequently
in the placebo arm compared to the active treatment arm.
About Hereditary Angioedema
HAE is a rare and potentially fatal 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 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 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
View original content to download
SOURCE Ionis Pharmaceuticals, Inc.