– Data continue to demonstrate
donidalorsen potential to deliver significant and sustained
reductions in HAE attacks up to three years with monthly or every
two-month dosing
CARLSBAD, Calif., Oct. 3, 2024
/PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS)
announced today that it will present new three-year data from the
Phase 2 open-label extension (OLE) study as well as additional
results from the pivotal Phase 3 OASIS and OASISplus studies of
donidalorsen, the company's investigational RNA-targeted
prophylactic medicine for hereditary angioedema (HAE). Results will
be presented at the 2024 American College of Allergy, Asthma &
Immunology (ACAAI) Annual Scientific Meeting in Boston, Massachusetts.
New data at ACAAI demonstrate donidalorsen significantly reduced
HAE attacks, translating to high levels of disease control and
significant and clinically meaningful improvements in
quality-of-life across multiple measures in a vast majority of
patients, sustained up to three years. Presentations include:
- Phase 2 Open-Label Extension of Donidalorsen In
Patients with Hereditary Angioedema: A Week 197 Analysis
- e-Poster Presentation: October 25,
2024 (Monitor 21, Exhibit Hall A)
- Presenting Author: Michael
Manning
- Impact of Donidalorsen on Patient-Reported Outcomes:
Results from the Phase 3 OASIS-HAE Study
- e-Poster Presentation: October 25,
2024 (Monitor 21, Exhibit Hall A)
- Presenting Author: Aaron Yarlas
- Treatment Of Hereditary Angioedema: Safety, Efficacy,
and Patient Preference after Switching to Donidalorsen (OASISplus
Study)
- e-Poster Presentation: October 25,
2024 (Monitor 21, Exhibit Hall A)
- Presenting Author: Danny Cohn
- Donidalorsen for Hereditary Angioedema: Results from the
OASISplus Open-Label Extension Study
- e-Poster Presentation: October 25,
2024 (Monitor 21, Exhibit Hall A)
- Presenting Author: Raffi Tachdjian
- Donidalorsen Exposure-Response Analysis: Hereditary
Angioedema Attack Rate versus Plasma Prekallikrein Concentration
Relationship
- e-Poster Presentation: October 25,
2024 (Monitor 19, Exhibit Hall A)
- Presenting Author: Pratap
Singh
- Drug Patterns, Clinical Outcomes, and Economic Costs among
Hereditary Angioedema Patients Initiating Long-Term
Prophylaxis
- e-Poster Presentation: October 25,
2024 (Monitor 19, Exhibit Hall A)
- Presenting Author: William R. Lumry
Ionis previously reported positive results from
the Phase 3 OASIS-HAE and OASISplus studies. Data from both studies
were presented at the 2024 European Academy of
Allergy and Clinical Immunology (EAACI) Annual Congress
in Valencia, Spain and results from OASIS-HAE were
published in The New England Journal of Medicine. The
company also previously reported positive two-year results from the
Phase 2 OLE study last year.
About Hereditary Angioedema (HAE)
HAE is a rare and
potentially life-threatening genetic condition that involves
recurrent attacks of severe swelling (angioedema) in various parts
of the body, including the hands, feet, genitals, stomach, face
and/or throat. HAE is estimated to affect more than 20,000 patients
in the U.S. and Europe. In the
U.S., doctors frequently use prophylactic treatment approaches to
prevent and reduce the severity of HAE attacks in
patients.
About Donidalorsen
Donidalorsen is an
investigational RNA-targeted medicine designed to target
prekallikrein (PKK), which plays an important role in activating
inflammatory mediators associated with acute attacks of hereditary
angioedema (HAE). By reducing the production of PKK, donidalorsen
could be an effective prophylactic approach to preventing HAE
attacks, if approved.
Donidalorsen is an investigational medicine that has not been
approved for the treatment of any disease by regulatory
authorities.
About Ionis Pharmaceuticals, Inc.
For three
decades, Ionis has invented medicines that bring better futures to
people with serious diseases. Ionis currently has five marketed
medicines and a leading pipeline in neurology, cardiology, and
other areas of high patient need. As the pioneer in RNA-targeted
medicines, Ionis continues to drive innovation in RNA therapies in
addition to advancing new approaches in gene editing. A deep
understanding of disease biology and industry-leading technology
propels our work, coupled with a passion and urgency to deliver
life-changing advances for patients. To learn more about Ionis,
visit Ionis.com and follow us on X
(Twitter) and LinkedIn.
Forward-looking Statements
This press release includes
forward-looking statements regarding Ionis' business and the
therapeutic and commercial potential of our commercial medicines,
donidalorsen, additional medicines in development and technologies.
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 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. Except as required by law, we
undertake no obligation to update any forward-looking statements
for any reason. 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,
2023, and most recent Form 10-Q, 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" all refer to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a registered trademark
of Ionis Pharmaceuticals, Inc.
Ionis Pharmaceuticals Investor Contact:
D.
Wade Walke, Ph.D.
– IR@ionis.com – 760-603-2331
Ionis Pharmaceuticals Media Contact:
Hayley
Soffer – media@ionis.com – 760-603-4679
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SOURCE Ionis Pharmaceuticals, Inc.