CARLSBAD, Calif., Dec. 7, 2021 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted
therapies, today announced it has entered into a strategic
collaboration agreement with AstraZeneca to develop and
commercialize eplontersen, Ionis' investigational antisense
medicine for the treatment of transthyretin amyloidosis (ATTR).
Eplontersen, formerly known as IONIS-TTR-LRx, is
designed to reduce the production of transthyretin, or TTR protein,
to treat ATTR, a systemic, progressive and fatal disease. It
uses Ionis' advanced LIgand-Conjugated
Antisense (LICA) technology.
The companies will develop a global strategy for developing,
manufacturing and commercializing eplontersen. A breakdown of
responsibilities of the collaboration includes, but is not limited
to:
- Ionis will continue to lead the conduct of the global Phase 3
clinical trials in patients with hereditary ATTR amyloidosis
(hATTR) with polyneuropathy (NEURO-TTRansform) and cardiomyopathy
(CARDIO-TTRansform).
- Ionis will manufacture and supply eplontersen for the ongoing
clinical trials and process qualifications. AstraZeneca will be
responsible for commercial supply, with transition timing to be
agreed by both companies.
- Ionis and AstraZeneca will have shared responsibility for
medical affairs and commercial activities in the U.S.
- AstraZeneca will have an exclusive license for eplontersen
outside the U.S. except certain countries in Latin America.
"We believe that bringing together Ionis' industry-leading
experience in RNA-targeted therapeutics and deep knowledge of the
TTR amyloidosis market with AstraZeneca's global scale and
leadership in cardiovascular drug development and commercialization
will enable faster and deeper market penetration for the benefit of
patients," said Brett P. Monia,
Ph.D., chief executive officer of Ionis. "In addition to being the
best strategy to maximize patient and shareholder value for
eplontersen, this agreement also represents a key step in
bolstering our commercial organization as we prepare to launch
multiple products."
Hereditary ATTR amyloidosis (hATTR) with polyneuropathy is
expected to be the first indication for which the companies will
seek regulatory approval for eplontersen, with the potential to
file a new drug application (NDA) with the U.S. Food and Drug
Administration by the end of 2022.
Under the terms of the agreement, Ionis will receive a
$200 million upfront payment, up to
$485 million in development and
approval milestones, and up to $2.9
billion in sales-related milestone payments. The
collaboration includes territory-specific development, commercial
and medical affairs cost-sharing provisions. Ionis is also eligible
to earn royalties in the range of low double-digit to mid-20s
percentage depending on region.
The agreement is expected to become effective by the end of
2021, subject to the satisfaction of requirements of the
Hart-Scott-Rodino Antitrust Improvements Act of 1976.
Webcast
Ionis will conduct a webcast on Dec.
7 at 10 a.m. Eastern Time to
discuss this announcement and related activities. Interested
parties may access the webcast here. A webcast replay will be
available for a limited time at the same address.
About eplontersen
Eplontersen (formerly known as IONIS-TTR-LRx) is an
investigational antisense medicine developed with Ionis'
LIgand-Conjugated Antisense (LICA) technology
that is designed to reduce the production of transthyretin, or TTR
protein, to treat all types of TTR amyloidosis (ATTR), a systemic,
progressive, and fatal disease. In patients with ATTR, both the
mutant and wild type (wt) TTR protein builds up as fibrils in
tissues, such as the peripheral nerves, heart, intestinal tract,
eyes, kidneys, central nervous system, thyroid and bone marrow. The
presence of TTR fibrils interferes with the normal functions of
these tissues. As the TTR protein fibrils enlarge, more tissue
damage occurs and the disease worsens, resulting in poor quality of
life and eventually death. Ionis is currently evaluating
eplontersen through its CARDIO-TTRansform Phase 3 study for the
treatment of patients with hereditary and wild-type cardiomyopathy
(ATTR-CM) and its NEURO-TTRansform Phase 3 study for the treatment
of patients with hereditary transthyretin-mediated amyloid
polyneuropathy (hATTR-PN).
About TTR Amyloidosis (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.
Polyneuropathy due to hATTR is caused by the accumulation of
misfolded mutated TTR protein in the peripheral nerves. Patients
with polyneuropathy due to hATTR 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
compromise their function and eventually leading to death within
five to fifteen years of disease onset.
ATTR cardiomyopathy 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 3 to 5 years from disease onset. ATTR
cardiomyopathy includes both the genetic and wild-type form of the
disease.
Worldwide, there are an estimated 300,000 –
500,0001,2 patients with ATTR.
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 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.
Forward-looking Statements
This press release includes forward-looking statements regarding
Ionis' business and the therapeutic and commercial potential of
Ionis' technologies, eplontersen 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, 2020, and the most
recent Form 10-Q quarterly filing, which are on file with the SEC.
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 Mohamed-Salem L, et al. Prevalence of wild type
ATTR assessed as myocardial uptake in bone scan in the elderly
population. Int J Cardiol. 2018 Nov
1;270:192-196. doi: 10.1016/j.ijcard.2018.06.006.
2 Cuscaden C, et al. Estimation of prevalence of
transthyretin (ATTR) cardiac amyloidosis in an Australian
subpopulation using bone scans with echocardiography and clinical
correlation. J Nucl Cardiol. 2020 May
8. doi: 10.1007/s12350-020-02152-x.
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SOURCE Ionis Pharmaceuticals, Inc.