- CARDIO-TTRansform is the largest, most comprehensive ATTR-CM
study with more than 1,400 patients enrolled
- Eplontersen is currently under U.S. FDA review for
ATTRv-polyneuropathy, with ATTR-CM representing a second, larger
potential patient population
CARLSBAD, Calif., July 31, 2023 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (Nasdaq: IONS) today announced the
completion of enrollment in the Phase 3 CARDIO-TTRansform
cardiovascular outcomes study of eplontersen in patients with
transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a large
and growing patient population at risk of life-threatening
cardiovascular (CV) events. The study enrolled more than 1,400
patients.
Eplontersen is an investigational antisense medicine designed to
inhibit the production of transthyretin and slow the progression of
cardiomyopathy for people living with hereditary or wild-type
ATTR-CM.
"CARDIO-TTRansform is the largest and most comprehensive ATTR-CM
study ever conducted. The evaluation of eplontersen in this broad,
diverse patient population will enable robust and clinically
meaningful analysis of the composite endpoint of CV mortality and
recurrent CV events," said Eugene
Schneider, M.D., executive vice president and chief clinical
development officer for Ionis. "It will also allow us to
gather data from important patient subsets, including patients
with or without stabilizer therapy and those with or without
hereditary disease within the evolving ATTR-CM landscape. We look
forward to reporting results as early as the first half of
2025."
As part of a global development and commercialization agreement,
Ionis and AstraZeneca are seeking regulatory approval for
eplontersen for the treatment of transthyretin-mediated amyloid
polyneuropathy (ATTRv-PN) in the U.S. and plan to seek regulatory
approval in Europe 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.
For more information on the CARDIO-TTRansform study, please
visit www.clinicaltrials.gov (NCT04136171).
About the CARDIO-TTRansform Study
CARDIO-TTRansform is a global, double-blind, randomized,
placebo-controlled Phase 3 cardiovascular outcome study in more
than 1,400 patients with ATTR cardiomyopathy (ATTR-CM). It is
designed to compare eplontersen to placebo in patients with both
wild-type ATTRwt-CM and hereditary ATTRv-CM who are either naïve to
treatment or on a currently available standard of care. The primary
composite endpoint is cardiovascular (CV) mortality and recurrent
CV clinical events comparing the two study arms up to week 140.
Secondary endpoints include the change from baseline in the
6-minute walk test (6MWT) and the Kansas City Cardiomyopathy
Questionnaire (KCCQ) scores at week 121, as well as the rates of CV
mortality, CV clinical events and all-cause mortality at week
140.
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-mediated Amyloid Cardiomyopathy
(ATTR-CM)
Transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) is an
underdiagnosed and potentially 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-CM includes both the genetic
and wild-type form of the disease. Worldwide, there are an
estimated 300,000 – 500,0001,2 patients with
ATTR-CM.
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.
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.