- 35-week data included in the filing demonstrated a
statistically significant and clinically meaningful change from
baseline for co-primary and secondary endpoints compared to
external placebo group
- Eplontersen previously granted Orphan Drug Designation for
transthyretin-mediated amyloidosis
- FDA assigns PDUFA action date of Dec.
22, 2023
CARLSBAD, Calif., March 7,
2023 /PRNewswire/ -- Ionis Pharmaceuticals (Nasdaq:
IONS) today announced that the U.S. Food and Drug Administration
(FDA) has accepted for review a New Drug Application (NDA) for
eplontersen, an investigational antisense medicine for the
treatment of people living with hereditary transthyretin-mediated
amyloid polyneuropathy (ATTRv-PN). The application has been given a
Prescription Drug User Fee Act (PDUFA) action date of Dec. 22, 2023.
In its acceptance letter, the FDA stated that it has not
identified any review issues and did not make any additional data
requests. The FDA also noted that it is not planning to hold an
advisory committee meeting to discuss the application.
Patients with ATTRv-PN experience ongoing debilitating nerve
damage throughout their body resulting in the progressive loss of
motor function. These patients accumulate TTR in other major
organs, which progressively compromises their function and
eventually leads to death within five to fifteen years of disease
onset.
"We are excited by today's FDA acceptance of our NDA filing as
it brings Ionis and our partner, AstraZeneca, one step closer to
making eplontersen available to patients with ATTR polyneuropathy,"
said Eugene Schneider, M.D.,
executive vice president and chief clinical development officer at
Ionis. "Significant reductions in TTR protein levels were
observed during the NEURO-TTRansform 35-week interim analysis.
Overall, the interim analysis demonstrated eplontersen has the
potential to make a positive impact on disease progression and
improve quality of life in a substantial number of patients."
The NDA is based on results from the global Phase 3
NEURO-TTRansform study presented at the International Symposium on
Amyloidosis (ISA). In the 35-week interim analysis, eplontersen
demonstrated a statistically significant and clinically meaningful
change from baseline for its co-primary and key secondary endpoints
compared to the external placebo group. In the study, eplontersen
achieved a significant mean reduction (p<0.0001) in the
co-primary endpoint of serum transthyretin (TTR) concentration
compared to baseline. Eplontersen also demonstrated a significant
treatment effect on the co-primary endpoint of modified Neuropathy
Impairment Score +7 (mNIS+7), a measure of neuropathic disease
progression, with a statistically significant difference in mean
change from baseline versus the external placebo group
(p<0.0001). The study met its key secondary endpoint of change
from baseline in the Norfolk Quality of Life Questionnaire-Diabetic
Neuropathy (Norfolk QoL-DN), showing that treatment with
eplontersen significantly improved patient-reported quality of life
compared to the external placebo group (p<0.0001). Eplontersen
also demonstrated a favorable safety and tolerability profile.
Eplontersen is an investigational medicine designed to reduce
the production of transthyretin (TTR) protein to treat both
hereditary and non-hereditary forms of ATTR amyloidosis (ATTR). In
December 2021, Ionis and AstraZeneca
entered into a strategic collaboration to develop and commercialize
eplontersen. Eplontersen is being jointly developed and
commercialized by both companies in the U.S. and will be developed
and commercialized in the rest of the world by AstraZeneca, with
the exception of Latin America. In
January 2022, eplontersen was granted
Orphan Drug Designation in the U.S. by the FDA.
In addition to the NEURO-TTRansform study, eplontersen is also
currently being evaluated in the global Phase 3 CARDIO-TTRansform
study for transthyretin amyloid cardiomyopathy (ATTR-CM), a
systemic, progressive and fatal condition that leads to progressive
heart failure and 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 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 with
its novel antisense technology. Ionis currently has three 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
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 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. 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, which is on file
with the Securities and Exchange Commission. Copies of this and
other documents are available from the Company.
In this press release, unless the context requires otherwise,
"Ionis," "Company," "we," "our," and "us" refers to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a trademark of Ionis
Pharmaceuticals, Inc.
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SOURCE Ionis Pharmaceuticals, Inc.