- Topline Phase 3 NEURO-TTRansform results show eplontersen
continued to halt neuropathy disease progression and improve
quality of life through 85 weeks
- Data further strengthen eplontersen's differentiated
profile, positioning it to be an important potential treatment for
patients with ATTRv-PN
CARLSBAD, Calif., July 10,
2023 /PRNewswire/ -- Ionis Pharmaceuticals (Nasdaq:
IONS) today announced positive topline, 85-week data from the Phase
3 NEURO-TTRansform study in patients with hereditary
transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
Ionis and AstraZeneca's eplontersen continued to show
sustained improvements in measures of neuropathy disease and a
favorable safety and tolerability profile.
An overview of key results from the 85-week topline analysis is
available here. At 85 weeks, eplontersen continued to show a
sustained reduction in serum TTR concentration compared to baseline
and continued to halt disease progression as measured by the
modified Neuropathy Impairment Score +7 (mNIS+7). Eplontersen also
showed continued improvement on the Norfolk Quality of Life
Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) compared to
baseline. Results from the NEURO-TTRansform primary analysis at 66
weeks were presented earlier this year.
"These positive findings further strengthen eplontersen's
efficacy and safety profile, underscoring its potential to be an
important, differentiated advancement for patients with this
progressive, debilitating and fatal disease," said Eugene Schneider, M.D., Ionis' executive vice
president and chief clinical development officer. "A substantial
number of eplontersen-treated patients showed improvement in
neuropathy impairment and quality of life through 19 months of
treatment. ATTRv-PN continues to be an underserved patient
population and we look forward to working with regulatory
authorities to bring this important new, self-administered
treatment to patients."
Results from the 85-week exploratory analysis of
NEURO-TTRansform will be submitted for presentation at an upcoming
medical meeting. As part of a global development and
commercialization agreement, Ionis and AstraZeneca are seeking
regulatory approval for eplontersen for the treatment of 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.
Eplontersen is currently being evaluated in the Phase 3
CARDIO-TTRansform study 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 Hereditary Transthyretin-Mediated Amyloid
Polyneuropathy (ATTRv-PN)
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.
About the NEURO-TTRansform Study
NEURO-TTRansform is a global, open-label, randomized trial
evaluating the efficacy and safety of eplontersen in patients with
ATTRv-PN at week 35, week 66 and week 85. The final analysis
comparing eplontersen to an external placebo group was completed at
week 66. All patients were then followed on treatment until week 85
and evaluated four weeks after the last dose in an end-of-trial
assessment. Following treatment and the end-of-trial assessments,
patients were eligible to enter an open-label extension study to
continue receiving eplontersen once every four weeks or enter a
20-week post-treatment evaluation period. For more information on
the NEURO-TTRansform study, please visit:
https://clinicaltrials.gov/ct2/show/NCT04136184
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.