CARLSBAD, Calif., March 29, 2021 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that data from
a Phase 2 clinical study of IONIS-PKK-LRx met its
primary and secondary endpoints, achieving significant reductions
in the number of attacks suffered by patients with hereditary
angioedema (HAE) compared to placebo. The study demonstrated a mean
reduction of 90% in the number of monthly HAE attacks in weeks one
to 17 of the study (p <0.001) and a mean reduction of 97% in the
number of monthly HAE attacks in weeks five to 17 (p=0.003). In
weeks five to 17, 92% of patients treated with
IONIS-PKK-LRx were attack-free compared to 0% in the
placebo group (p <0.001).
IONIS-PKK-LRx was developed using Ionis' advanced
ligand-conjugated antisense (LICA) technology.
IONIS-PKK-LRx is one of Ionis' wholly owned medicines to
treat rare diseases.
Hereditary angioedema is a rare autosomal dominant disease that
results in recurrent, painful attacks of swelling affecting the
arms, legs, face, intestinal track and airway.
IONIS-PKK-LRx is an investigational antisense medicine
designed to reduce the production of prekallikrein, or PKK, which
plays a key role in the activation of inflammatory mediators
associated with acute attacks of HAE. Without preventive treatment,
HAE attacks can be frequent and severe and, in some patients,
life-threatening. Physicians have long prescribed prophylactic
treatment approaches, including C1-INH replacement therapies and
more recently inhibitors of plasma kallikrein, to prevent and
reduce the severity of HAE attacks. Despite these available
therapies, patients with HAE may experience breakthrough
attacks.
"These topline Phase 2 study results support a profile for
IONIS-PKK-LRx as a potential best-in-class prophylactic
treatment for patients with HAE, with excellent efficacy, safety
and tolerability along with the convenience of once per month low
volume subcutaneous injections," said Kenneth Newman, M.D., M.B.A., Ionis' vice
president of clinical development and leader of the pulmonology and
immunology franchise. "These results highlight the potential
benefits and advantages of IONIS-PKK-LRx for the
treatment of hereditary angioedema and more broadly underscore the
power of Ionis' antisense technology to target the root causes of
rare diseases like HAE."
In the Phase 2 clinical study, 20 adults with Type 1 or Type 2
HAE were randomized and received either IONIS-PKK-LRx
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. Ionis expects to present a full analysis of
its Phase 2 study of IONIS-PKK-LRx at a medical
conference later this year.
Ionis will host a series of webinars on rare diseases for which
the company is developing potential treatments. The first webinar,
on hereditary angioedema, is scheduled for Monday, April 5. Visit www.ionispharma.com for
more information and to register for the webinar.
About Ionis Pharmaceuticals
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-PKK-LRx and Ionis' technologies and 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, which is on
file with the SEC. Copies of this 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.
View original content to download
multimedia:http://www.prnewswire.com/news-releases/ionis-reports-positive-topline-phase-2-study-results-of-its-novel-antisense-treatment-for-hereditary-angioedema-301257232.html
SOURCE Ionis Pharmaceuticals, Inc.