- Companies jointly developing and commercializing eplontersen
in the U.S.
- AstraZeneca adds Latin
America as part of its exclusive rights for rest of
world
CARLSBAD, Calif., July 28,
2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.
(Nasdaq: IONS) today announced it has expanded its existing
collaboration with AstraZeneca to include exclusive rights for
AstraZeneca to commercialize eplontersen in Latin America. Ionis previously granted
AstraZeneca exclusive rights to commercialize eplontersen in all
other countries outside the U.S. Ionis and AstraZeneca will
continue to jointly develop and commercialize eplontersen in the
U.S. The companies are successfully advancing eplontersen by
combining Ionis' industry-leading expertise in RNA-targeted
therapeutics and deep knowledge of transthyretin amyloidosis (ATTR)
with AstraZeneca's global cardiovascular commercial
capabilities.
Eplontersen is an investigational antisense medicine discovered
by Ionis that reduces the production of transthyretin protein (TTR)
to treat ATTR, a systemic, progressive, and fatal disease.
"The positive clinical results from our Phase 3 NEURO-TTRansform
study, combined with eplontersen's self-administration profile,
reinforce eplontersen's potential to be an important and
differentiated new treatment option for patients with ATTR," said
Brett P. Monia Ph.D., chief executive officer of Ionis. "With FDA
review of eplontersen for ATTR polyneuropathy already underway and
plans to file for regulatory approval in the EU and other countries
later this year, today's agreement underscores our shared
commitment to ensuring that this much needed treatment is made
available to patients around the world."
AstraZeneca paid Ionis $20 million
to license eplontersen in Latin
America.
Under the terms of the collaboration agreement, Ionis is
eligible to receive up to $3.6
billion in milestone and other payments. The collaboration
includes territory-specific cost-sharing provisions. Ionis is also
eligible to earn royalties in the range of low double-digit to
mid-20s percentage depending on region.
Ionis and AstraZeneca are seeking regulatory approval for
eplontersen for the treatment of hereditary transthyretin-mediated
amyloid polyneuropathy (ATTRv-PN) in the U.S. and plan to seek
regulatory approval in the EU and other parts of the world. The
U.S. Food and Drug Administration accepted the New Drug Application
for eplontersen for the treatment of ATTRv-PN with a PDUFA action
date of Dec. 22, 2023. Eplontersen
was granted Orphan Drug Designation in the U.S.
Eplontersen is currently being evaluated in the Phase 3
CARDIO-TTRansform study (NCT04136171) for transthyretin-mediated
amyloid cardiomyopathy (ATTR-CM), a systemic, progressive and fatal
condition that typically leads to progressive heart failure and
often death within three to five years from disease onset.
About Eplontersen
Eplontersen is an investigational
LIgand-Conjugated Antisense (LICA) medicine
designed to inhibit the production of TTR protein. Eplontersen is
being developed as a monthly self-administered subcutaneous
injection to treat all types of ATTR. ATTR amyloidosis is a
systemic, progressive and fatal disease in which patients
experience multiple overlapping clinical manifestations caused by
the inappropriate formation and aggregation of TTR amyloid deposits
in various tissues and organs, including peripheral nerves, heart,
intestinal tract, eyes, kidneys, central nervous system, thyroid
and bone marrow. The progressive accumulation of TTR amyloid
deposits in these tissues and organs leads to organ failure and
eventually death.
About Transthyretin Amyloidosis (ATTR)
Transthyretin amyloidosis (ATTR) is a systemic, progressive and
fatal disease in which patients experience multiple overlapping
clinical manifestations caused by the inappropriate formation and
aggregation of TTR amyloid deposits in various tissues and organs,
including peripheral nerves, heart, intestinal tract, eyes,
kidneys, central nervous system, thyroid and bone marrow. The
progressive accumulation of TTR amyloid deposits in these tissues
and organs leads to organ failure and eventually death.
Hereditary transthyretin-mediated amyloid polyneuropathy
(ATTRv-PN) is caused by the accumulation of misfolded mutated TTR
protein in the peripheral nerves. Patients with ATTRv-PN experience
ongoing debilitating nerve damage throughout their body resulting
in the progressive loss of motor functions, such as walking. These
patients also accumulate TTR in other major organs, which
progressively compromises their function. The damage from misfolded
TTR protein accumulation leads to disability within five years of
diagnosis and is generally fatal within a decade.
Transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) is an
underdiagnosed and fatal disease. It is caused by the accumulation
of misfolded TTR protein in the cardiac muscle. Patients experience
ongoing debilitating heart damage resulting in progressive heart
failure, which results in death within three to five years from
disease onset. ATTR cardiomyopathy includes both the genetic and
wild-type form of the disease.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been a leader in RNA-targeted
therapy, pioneering new markets and changing standards of care.
Ionis currently has four marketed medicines and a promising
late-stage pipeline highlighted by cardiovascular and neurological
franchises. Our scientific innovation began and continues with the
knowledge that sick people depend on us, which fuels our vision to
become the leader in genetic medicine, utilizing a multi-platform
approach to discover, develop and deliver life-transforming
therapies.
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
eplontersen, Ionis' technologies 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 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, 2022, 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" all refer to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a registered trademark of Ionis
Pharmaceuticals, Inc.
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SOURCE Ionis Pharmaceuticals, Inc.