- Positive
Phase 2b data from RE-THINC ESRD study of fesomersen in
patients on hemodialysis presented at Kidney Week 2022
- Ionis to regain
rights to fesomersen from Bayer
CARLSBAD, Calif., Nov. 4, 2022
/PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today
announced positive results from the Phase 2b RE-THINC
ESRD study of fesomersen (formerly IONIS-FXI-LRx), an
investigational antisense medicine designed to reduce the
production of Factor XI (FXI) for the prevention of thrombosis,
were presented by Bayer at the American Society of Nephrology's
(ASN) Kidney Week 2022. The RE-THINC ESRD study evaluated
fesomersen in patients with end-stage renal disease (ESRD) on
hemodialysis.
In the study, fesomersen achieved its primary endpoint,
demonstrating no increase in the incidence of the composite of
major bleeding and clinically relevant non-major (CRNM) bleeding
with 24 weeks of treatment. Fesomersen also achieved
dose-dependent and sustained median reductions in steady-state FXI
levels of 53.1%, 72.2% and 86.6% in the 40 mg, 80 mg, and 120 mg
doses of fesomersen, respectively, administered once every 4 weeks.
Incidences of dialysis circuit clotting and AV-access thrombosis
diminished significantly with decreasing FXI levels, both of which
were exploratory efficacy endpoints. Fesomersen showed favorable
safety and tolerability with 48 weeks of treatment in this
study.
"We are very pleased with the efficacy and safety data seen in
the Phase 2b study of fesomersen in
patients with ESRD, which we believe supports continued advancement
of this potential novel anti-thrombotic therapy for patients with
renal and cardiovascular diseases," said Richard S. Geary,
Ph.D., executive vice president and chief development officer at
Ionis. "We thank Bayer for their partnership in the development of
fesomersen. We are focused on getting fesomersen into the hands of
a new partner to deliver it to the market and patients in
need."
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 each of 3 dose cohorts or placebo
administered subcutaneously every four weeks for up to 48
weeks. The RE-THINC ESRD study was conducted by Bayer, which
licensed fesomersen from Ionis.
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 December 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.
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SOURCE Ionis Pharmaceuticals, Inc.