CARLSBAD, Calif., July 29, 2021 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted
therapies, today announced it has completed enrollment of
NEURO-TTRansform, the company's Phase 3 study of eplontersen,
formerly AKCEA-TTR-LRx, for people with hereditary
transthyretin-mediated amyloid polyneuropathy (hATTR-PN). The
company has exceeded the enrollment target in NEURO-TTRansform and
expects to register more than 160 patients in the study.
Eplontersen is an investigational antisense medicine
designed to inhibit production of transthyretin (TTR), a
protein that forms the amyloid deposits that cause debilitating
nerve damage throughout the body. Eplontersen utilizes Ionis'
advanced LIgand Conjugated Antisense (LICA)
technology. In a Phase 1 clinical trial, patients treated with
eplontersen experienced reductions in TTR of up to 94 percent.
"The speed of enrollment in our pivotal NEURO-TTRansform study
speaks to the urgent need for new treatment options for people
living with hATTR amyloidosis," said Brett
P. Monia, Ph.D., Ionis' chief executive officer. "Because
eplontersen utilizes Ionis' LICA technology, it offers the
advantages of high potency, convenient, infrequent dosing and an
attractive safety and tolerability profile. We remain dedicated to
advancing eplontersen as rapidly as possible and look forward to
announcing results from a formal interim analysis by mid-year 2022,
with NDA filing to potentially follow by year-end 2022."
Ionis is also studying eplontersen in the CARDIO-TTRansform
Phase 3 study in patients with hereditary and wild-type
For more information on the NEURO-TTRansform study, please
About the NEURO-TTRansform Study
NEURO-TTRansform is a global, open-label, randomized study
evaluating the efficacy and safety of eplontersen in patients
with polyneuropathy due to hATTR amyloidosis. The study has
enrolled adult patients with stage 1 or stage 2 polyneuropathy and
will be compared to the historical placebo arm from the
TEGSEDI® (inotersen) NEURO-TTR registrational study that Ionis
completed in 2017. There will be a formal interim analysis at week
35 analyzing the co-primary efficacy endpoints of percent change
from baseline in serum TTR concentration and change from baseline
The final primary endpoint analysis will be completed at week 66
and all patients will be followed until week 85 when they will have
the option to transition into the open label extension
The co-primary efficacy endpoints at week 66 are:
- Percent change from baseline in serum TTR concentration
- Change from baseline in the modified Neuropathy Impairment
Score +7 (mNIS+7), a measure of neuropathic disease
- Change from baseline in the Norfolk Quality of Life
Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN)
Eplontersen is an antisense drug that uses Ionis' advanced
Ligand Conjugated Antisense, or LICA,
technology designed to inhibit the production of the
transthyretin (TTR) protein at its source. Eplontersen is in
development to treat a broad population of patients with both
hereditary and wild-type forms of transthyretin amyloidosis, or
About Hereditary Transthyretin (hATTR) Amyloidosis
hATTR amyloidosis is a severe, progressive, and life-threatening
disease caused by the abnormal formation of the TTR protein and
aggregation of TTR amyloid deposits in various tissues and organs
throughout the body, including in peripheral nerves, the heart and
intestinal tract. The progressive accumulation of TTR amyloid
deposits in these organs often leads to intractable peripheral
sensorimotor neuropathy, autonomic neuropathy, and/or
cardiomyopathy, as well as other disease manifestations. hATTR
amyloidosis causes significant morbidity and progressive decline in
quality of life, severely impacting activities of daily living. The
disease often progresses rapidly and can lead to premature death.
The median survival is 4.7 years following diagnosis.
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.
Ionis' Forward-looking Statement
This press release includes forward-looking statements regarding
Ionis' business, 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
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
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SOURCE Ionis Pharmaceuticals, Inc.