- Eplontersen showed statistically significant and
clinically meaningful improvements in mNIS+7 and Norfolk
QoL-DN
- Eplontersen demonstrated a favorable safety
profile
- Ionis and AstraZeneca expect to file a New Drug
Application this year
CARLSBAD, Calif., June 21,
2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.
(Nasdaq: IONS) today announced positive topline results from a
35-week interim analysis of the Phase 3 NEURO-TTRansform study of
Ionis and AstraZeneca's eplontersen in patients with hereditary
transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
Eplontersen demonstrated a statistically significant and clinically
meaningful change from baseline for the co-primary endpoints of
percent change in serum transthyretin (TTR) concentration and the
modified Neuropathy Impairment Score +7 (mNIS+7), a measure of
neuropathic disease progression, versus the historical placebo
group. Eplontersen also met its key secondary endpoint of change
from baseline in the Norfolk Quality of Life Questionnaire-Diabetic
Neuropathy (Norfolk QoL-DN), showing treatment with eplontersen
significantly improved patient-reported quality of life versus the
historical placebo group.
In the study, eplontersen demonstrated a favorable safety and
tolerability profile with no specific concerns. The study data are
consistent with the clinical profile seen across Ionis' other LICA
programs, further validating how advancements in the company's
LIgand-Conjugated Antisense technology
position Ionis to deliver potentially transformative treatments for
a range of unmet medical needs.
Based on these study results, the companies will seek regulatory
approval for eplontersen for ATTRv-PN and plan to file a new drug
application with the U.S. Food and Drug Administration this
year.
"These encouraging data reinforce the safety profile of
eplontersen and demonstrate clear evidence of its potential to
provide much needed therapeutic benefit to patients living with
hereditary transthyretin-mediated amyloid
polyneuropathy," said Teresa
Coelho, M.D., a neurologist and neurophysiologist at
Hospital Santo António, Centro Hospitalar Universitário do
Porto, Portugal and an
investigator for the NEURO-TTRansform study.
"We are pleased that the data in the NEURO-TTRansform study
demonstrate eplontersen had a positive impact on disease
progression, including improvement in neuropathy impairment and
quality of life in a substantial number of patients. These highly
statistically significant and clinically meaningful results put us
on the cusp of providing a new therapeutic option for
polyneuropathy patients living with this debilitating and fatal
disease," said Eugene Schneider,
M.D., Ionis' executive vice president and chief clinical
development officer. "We are grateful to the patients, families and
clinicians who are participating in NEURO-TTRansform. Without their
commitment the eplontersen program would not have progressed as
successfully as it has."
The data from the 35-week interim analysis will be presented at
an upcoming medical meeting later this year.
As part of a global development and commercialization agreement
between Ionis and AstraZeneca, 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).
Eplontersen was granted Orphan Drug Designation in the U.S. It
is also currently being evaluated in the Phase 3 CARDIO-TTRansform
study for amyloid transthyretin cardiomyopathy (ATTR-CM), a
systemic, progressive and fatal condition that leads to progressive
heart failure and death within four years from diagnosis.
For more information on the NEURO-TTRansform study, please
visit: https://clinicaltrials.gov/ct2/show/NCT04136184.
About the NEURO-TTRansform
Study
NEURO-TTRansform is a global, open-label, randomized study
evaluating the efficacy and safety of eplontersen in patients with
hereditary transthyretin-mediated amyloid polyneuropathy
(ATTRv-PN). The study has enrolled adult patients with stage 1 or
stage 2 polyneuropathy and will compare efficacy of eplontersen to
the historical placebo arm from the TEGSEDI® (inotersen)
NEURO-TTR registrational study that Ionis completed in 2017. 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 study.
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
progression
- Change from baseline in the Norfolk Quality of Life
Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN)
About Eplontersen
Eplontersen is an investigational antisense medicine that uses
Ionis' advanced LIgand-Conjugated Antisense,
or LICA, technology designed to inhibit the production of the
transthyretin (TTR) protein at its source. Eplontersen, which is
planned to be delivered to patients via an autoinjector, is in
development to treat all types of ATTR, a systemic, progressive and
fatal disease.
About Hereditary Transthyretin
Amyloidosis (ATTRv)
Hereditary transthyretin amyloidosis (ATTRv) 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. Hereditary transthyretin 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 cardiovascular and neurological
franchises. Our scientific innovation began and continues with the
knowledge that sick people depend on us, which fuels our vision of
becoming a leading, fully integrated biotechnology company.
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 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 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, 2021, 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
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.