- Phase 2b RE-THINC ESRD study
of fesomersen met its primary endpoint in patients with end-stage
renal disease on hemodialysis
- Fesomersen demonstrated substantial and statistically
significant reductions in Factor XI activity levels
- Fesomersen, a novel Factor XI antisense inhibitor designed
to prevent thrombosis, was safe and well-tolerated in the study
with up to 48-week treatment
CARLSBAD, Calif., July 28,
2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.
(Nasdaq: IONS), today announced positive topline results of the
Phase 2b RE-THINC ESRD study of
fesomersen, formerly IONIS-FXI-LRx, in patients with
end-stage renal disease (ESRD) on hemodialysis. In the study,
fesomersen achieved its primary outcome measure of no increase in
incidence of major bleeding and clinically relevant non-major
bleeding as compared to placebo. Data from the study show that
fesomersen, administered monthly at 40 mg, 80 mg and 120 mg for up
to 48 weeks, was safe and well-tolerated. Fesomersen also
demonstrated substantial and statistically significant reductions
in Factor XI activity levels.
Data from the study are consistent with the clinical profile
seen across Ionis' other LICA programs, further validating how
advancements in the company's LIgand-Conjugated
Antisense technology position Ionis to deliver potentially
transformative treatments for a range of unmet medical needs. The
RE-THINC ESRD study was conducted by Bayer, which licensed
fesomersen from Ionis. Data from the study will be presented at an
upcoming medical meeting.
"For decades, anticoagulants have been a therapeutic mainstay in
the treatment and prevention of thrombosis. However, they can be
associated with increased bleeding risk, which can lead to major,
sometimes fatal, bleeding events," said Sanjay Bhanot, M.D., Ph.D., senior vice
president, chief medical officer and metabolic and liver franchise
leader at Ionis. "The results of the RE-THINC ESRD study
demonstrate fesomersen's potential as a novel anti-thrombotic
treatment for cardiovascular and renal disease patients."
An estimated 17.9 million people die from cardiovascular disease
each year, representing 32% of all deaths
worldwide.1 Of these deaths, 85% are due to heart
attacks and strokes, which are often caused by blood clots
(thrombosis) that block blood vessels in the heart or brain.
Fesomersen is an investigational antisense medicine designed by
Ionis to reduce the production of Factor XI, a protein produced in
the liver that is an important component of the coagulation
pathway. Factor XI is an attractive target for an anti-thrombotic
medicine because of its potential to separate anti-thrombotic
activity from bleeding risk. The RE-THINC ESRD study focused on
ESRD patients because they have a significantly high risk of
thromboembolic events and also a very high bleeding risk.
In prior clinical studies evaluating the safety and efficacy of
the non-LICA version of fesomersen, IONIS-FXIRx,
dose-dependent inhibition of Factor XI activity was demonstrated,
which was associated with significant reductions in clotting events
and no increase in major bleeding events. IONIS-FXIRx
was the first anti-thrombotic in development to demonstrate
potential to separate anti-thrombotic activity from bleeding
risk.
About the RE-THINC ESRD
Study
RE-THINC ESRD (NCT04534114) is a randomized, double-blind,
placebo-controlled study evaluating the safety, pharmacokinetics
and pharmacodynamics of multiple doses of fesomersen in 307
patients with end-stage renal disease on hemodialysis. Study
participants were randomized to 1 of 3 cohorts receiving 40 mg, 80
mg or 120 mg of subcutaneously administered fesomersen or placebo
every four weeks for up to 48 weeks. The primary endpoint of the
study was incidence of major bleeding and clinically relevant
non-major bleeding.
About Thrombosis
Thrombosis is the formation of blood clots inside blood vessels.
Blood clots can obstruct blood flow to prevent sufficient oxygen
flow to tissues and organs. In addition, clot fragments can break
off from the blood clot and travel to occlude other parts of the
circulation. Thrombosis is responsible for many heart attacks and
strokes and is the leading cause of morbidity and mortality
worldwide. Current anti-thrombotic treatments include
anticoagulants such as warfarin, Factor Xa inhibitors and thrombin
inhibitors. Although these therapies are effective at lowering the
risk of thrombosis, they can place patients at significant risk of
bleeding because they target factors required for normal
coagulation.
About Fesomersen
Fesomersen, (formerly IONIS-FXI-LRx) is an
investigational antisense medicine designed by Ionis to reduce the
production of Factor XI, a clotting factor produced in the liver
that is an important component of the coagulation pathway. High
levels of Factor XI increase the risk of blood clot formation
inside blood vessels (thrombosis), which can cause heart attacks
and strokes. Alternatively, individuals deficient in Factor XI have
a lower incidence of thrombosis-related events and little to no
increase in bleeding risk. This makes Factor XI an attractive
target for an anti-thrombotic medicine because of the potential to
separate anti-thrombotic activity from bleeding risk. Although
currently available anticoagulants reduce the risk of thrombosis,
these anticoagulants are associated with increased bleeding risk at
therapeutic doses, which can lead to major, sometimes fatal
bleeding events.
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, fesomersen 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 World Health Organization;
https://www.who.int/news-room/fact-sheets/detail/cardiovascular-diseases-(cvds)
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SOURCE Ionis Pharmaceuticals, Inc.