– Results across NEURO-TTRansform subgroups
show consistent benefit in neuropathy impairment and improved
quality of life, regardless of patient segmentation –
CARLSBAD, Calif., May 23, 2024
/PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS)
announced today that it will present new subgroup analyses from
Ionis and AstraZeneca's Phase 3 NEURO-TTRansform study of WAINUA™
(eplontersen) at the 2024 International Symposium on Amyloidosis
(ISA) in Rochester, Minnesota,
May 26-30. WAINUA was approved by the
U.S. Food and Drug Administration (FDA) in December 2023 for the treatment of the
polyneuropathy of hereditary transthyretin-mediated amyloidosis in
adults, commonly referred to as hATTR-PN or ATTRv-PN.
Notable presentations include:
- Neuropathy Impairment and Nutritional Status with
Eplontersen in Patients with Hereditary Transthyretin-Mediated
Amyloidosis
- Abstract ID 174: May 29, 2024,
10:00-10:30 am ET (poster
presentation 1) and 2:45-3:45 pm ET
(poster presentation 2)
- Presenting Author: Jonas
Wixner
- Eplontersen for Hereditary Transthyretin Amyloidosis with
Polyneuropathy: An Exploratory Analysis in Patients with the V30M
TTR Variant and Early-Onset or Late-Onset Disease
- Abstract ID 230: May 29, 2024,
10:00-10:30 am ET (poster
presentation 1) and 2:45-3:45 pm ET
(poster presentation 2)
- Presenting Author: Julian D.
Gillmore
- Eplontersen for Hereditary Transthyretin Amyloidosis with
Polyneuropathy: An Exploratory Analysis of Treatment Effect in Male
and Female Patients
- Abstract ID 202: May 29, 2024,
10:00-10:30 am ET (poster
presentation 1) and 2:45-3:45 pm ET
(poster presentation 2)
- Presenting Author: Márcia Waddington
Cruz
As part of a global development and
commercialization agreement, AstraZeneca and Ionis are
commercializing WAINUA for the treatment of ATTRv-PN in
the U.S. and are seeking regulatory approval
in Europe and other parts of the world.
Eplontersen is currently being evaluated in the Phase 3
CARDIO-TTRansform study for adults with 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. The
CARDIO-TTRansform Phase 3 study is fully enrolled with more than
1,400 patients – making it the largest study in this patient
population to date.
About WAINUA™ (eplontersen)
WAINUA™ (eplontersen) is a
LIgand-Conjugated Antisense (LICA) medicine
designed to inhibit the production of transthyretin, or TTR
protein. WAINUA has been approved in the U.S. for the
treatment of the polyneuropathy of hereditary
transthyretin-mediated amyloidosis in adults (also referred to as
ATTRv-PN). Please see full Prescribing Information.
INDICATION for WAINUA™ (eplontersen)
WAINUA injection
for subcutaneous use 45 mg is indicated for treatment of the
polyneuropathy of hereditary transthyretin-mediated amyloidosis in
adults.
IMPORTANT SAFETY INFORMATION for WAINUA™
(eplontersen)
WARNINGS AND PRECAUTIONS
Reduced Serum Vitamin A
Levels and Recommended Supplementation WAINUA leads to a
decrease in serum vitamin A levels. Supplement with recommended
daily allowance of vitamin A. Refer patient to an ophthalmologist
if ocular symptoms suggestive of vitamin A deficiency occur.
ADVERSE REACTIONS
Most common adverse reactions (≥9% in WAINUA-treated patients)
were vitamin A decreased (15%) and vomiting (9%).
Please see link to U.S. Full Prescribing
Information for WAINUA.
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.
About Hereditary Transthyretin-Mediated Amyloid
Polyneuropathy (ATTRv-PN)
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 Ionis Pharmaceuticals, Inc.
For three decades, Ionis has invented medicines that bring
better futures to people with serious diseases. Ionis currently has
five marketed medicines and a leading pipeline in neurology,
cardiology, and other areas of high patient need. As the pioneer in
RNA-targeted medicines, Ionis continues to drive innovation in RNA
therapies in addition to advancing new approaches in gene editing.
A deep understanding of disease biology and industry-leading
technology propels our work, coupled with a passion and urgency to
deliver life-changing advances for patients. To learn more about
Ionis, visit Ionispharma.com and follow us on X (Twitter)
and LinkedIn.
Forward-looking Statements
This press release includes forward-looking statements regarding
eplontersen, Ionis' business, and the therapeutic and commercial
potential of Ionis' commercial medicines, additional medicines in
development and technologies. 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 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. Except as required by
law, we undertake no obligation to update any forward-looking
statements for any reason. 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,
2023, and most recent Form 10-Q, which are on file with
the SEC. Copies of these and other documents are available
at www.ionispharma.com.
Ionis Pharmaceuticals® is a registered trademark of Ionis
Pharmaceuticals, Inc. WAINUATM is a registered trademark
of the AstraZeneca group of companies.
Ionis Pharmaceuticals Investor Contact: D. Wade Walke, Ph.D. - info@ionisph.com -
760-603-2331; Ionis Pharmaceuticals Media Contact:
Daria Binder -
CorporateCommunications@ionisph.com - 760-603-4679
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SOURCE Ionis Pharmaceuticals, Inc.