- Recommendation based on NEURO-TTRansform Phase 3 results
showing WAINZUA demonstrated consistent and sustained benefit
improving neuropathy impairment and quality of life versus
placebo
CARLSBAD, Calif., Oct. 21,
2024 /PRNewswire/ -- Ionis Pharmaceuticals,
Inc. (Nasdaq: IONS) announced today that Ionis' and
AstraZeneca's WAINZUA (eplontersen) has been recommended for
approval by the Committee for Medicinal Products for Human Use
(CHMP) in the European Union (EU) for the treatment of hereditary
transthyretin-mediated amyloidosis in adult patients with stage 1
or stage 2 polyneuropathy, commonly referred to as hATTR-PN or
ATTRv-PN. If approved by the European Commission, WAINZUA will be
the only approved medicine in the EU for the treatment of ATTRv-PN
that can be self-administered monthly via an auto-injector.
The CHMP based its opinion on the positive NEURO-TTRansform
Phase 3 trial which showed that through 66 weeks, patients treated
with WAINZUA demonstrated consistent and sustained benefit on the
co-primary endpoints of serum transthyretin (TTR) concentration and
neuropathy impairment measured by modified Neuropathy Impairment
Score +7 (mNIS+7), and key secondary endpoint of quality of life
(QoL) on the Norfolk Quality of Life Questionnaire-Diabetic
Neuropathy (Norfolk QoL-DN) versus external placebo. WAINZUA
continued to demonstrate a favorable safety and tolerability
profile throughout the NEURO-TTRansform trial.
ATTRv-PN is a debilitating disease that leads to peripheral
nerve damage with motor disability within five years of diagnosis
and, without treatment, is generally fatal within a decade. WAINZUA
is a once-monthly RNA-targeted medicine designed to reduce the
production of TTR protein at its source.
"Hereditary transthyretin-mediated amyloidosis with
polyneuropathy remains a progressive and debilitating disease in
Europe and other parts of the
world, despite currently available medicines," said Brett P. Monia, Ph.D., chief executive officer,
Ionis. "The CHMP recommendation is an important step toward making
WAINZUA available in Europe,
which, if approved, will be the only medicine in the EU for the
treatment of transthyretin-mediated amyloidosis with polyneuropathy
that can be self-administered monthly via an auto-injector. We are
proud to partner with AstraZeneca whose global leadership and
expertise positions our alliance to rapidly and effectively bring
WAINZUA to many people living with hereditary
transthyretin-mediated amyloidosis with polyneuropathy in
Europe, pending the EMA's
decision."
In December 2023, eplontersen was
approved in the U.S. for the treatment of ATTRv-PN, under the brand
name WAINUA™ (eplontersen). WAINUA is now gaining approvals in
additional countries worldwide, including Canada. As part of a global development and
commercialization agreement, AstraZeneca and Ionis are
commercializing WAINUA in the U.S. The companies are seeking
regulatory approval in Europe and
other parts of the world where AstraZeneca has exclusive rest of
world commercialization and development rights. Eplontersen has
also been granted Orphan Drug Designation in the U.S. and in the EU
for the treatment of transthyretin-mediated amyloidosis (ATTR).
Eplontersen is also currently being evaluated in the global
CARDIO-TTRansform Phase 3 study for the treatment of adults with
ATTR-CM. The trial is fully enrolled with more than 1,400 patients
– making it the largest, most comprehensive study to date in this
patient population. More information on the CARDIO-TTRansform study
(NCT04136171) is available at www.clinicaltrials.gov.
About TTR Amyloidosis
ATTR is caused by the accumulation of liver-derived misfolded
TTR protein in tissues, such as the heart and the peripheral
nerves, causing organ damage and failure. ATTR then causes
complications, leading to cardiovascular, neurological and renal
diseases such as heart failure and chronic kidney disease. There
are both hereditary (ATTRv) and non-hereditary (wild-type) forms of
ATTR. ATTR is a rapidly progressive and fatal disease that requires
timely recognition of symptoms. ATTR has several phenotypes
including ATTR-cardiomyopathy (CM), which predominantly impacts the
heart, potentially leading to heart failure, ATTR-polyneuropathy
(PN), which predominantly affects the peripheral nervous system and
mixed phenotype, where patients experience symptoms of both.
Worldwide, there are an estimated 300,000 – 500,000 patients with
ATTR-CM and about 10,000 – 40,000 patients with ATTRv-PN.
About NEURO-TTRansform
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, which is still ongoing.
Full results from the NEURO-TTRansform trial were published in
The Journal of the American Medical Association (JAMA)
demonstrating the benefit of eplontersen across the spectrum of
ATTRv-PN at 35, 66 and 85 weeks of treatment.
About WAINZUA / WAINUA™ (eplontersen)
Eplontersen is a once-monthly RNA-targeted medicine that
provides upstream suppression of transthyretin (TTR) production and
is designed to precisely target and reduce the production of TTR
protein at its source in the liver.
WAINZUA has been approved in the U.S. and Canada under the brand name WAINUA™
(eplontersen) for the treatment of the polyneuropathy of hereditary
transthyretin-mediated amyloidosis in adults, commonly referred to
as hATTR-PN or ATTRv-PN. Please see link for full U.S. Prescribing
Information. WAINZUA is not yet approved for any indication in
Europe.
U.S. 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 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 Ionis.com and follow us on X (Twitter) and
LinkedIn.
Ionis 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.Ionis.com.
Ionis Pharmaceuticals® is a registered trademark of Ionis
Pharmaceuticals, Inc. WAINUA™ is a trademark of AstraZeneca
plc.
Ionis Investor Contact:
D. Wade Walke, Ph.DD.
IR@ionis.com 760-603-2331
Ionis Media Contact:
Daria Binder
media@ionis.com 760-603-4679
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