BOSTON and CARLSBAD, Calif., Oct.
30, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.
(NASDAQ: IONS) and its wholly owned subsidiary, Akcea Therapeutics,
received the Prix Galien USA Award
for the Best Biotechnology Product in 2020 in recognition of
TEGSEDI, the first and only self-administered, subcutaneous
treatment for the polyneuropathy of hereditary ATTR amyloidosis in
adults. This Prix Galien award recognizes outstanding
achievements in biomedicine that improve the human condition and
was presented at a virtual ceremony on Thursday, Oct. 29.
"We are thrilled to accept this award for TEGSEDI – a treatment
that has already provided substantial benefit for those
who have been devastated with the polyneuropathy associated with
hereditary transthyretin amyloidosis," said Brett P. Monia, Ph.D., chief executive
officer at Ionis, and a founding scientist at Ionis who led
the discovery and development of TEGSEDI. "Our vision in the
discovery and development of TEGSEDI was to improve the lives of
patients living with the devastating effects of this rare genetic
disease that impacts the lives of generations of families. I would
like to thank our teams at Akcea and Ionis for their tireless work
and dedication in developing TEGSEDI and giving hope to patients
worldwide."
The international Prix Galien Award is presented annually by the
Galien Foundation. The Prix Galien USA Committee
comprises renowned leaders from the biomedical industry and
academia, who together are responsible for evaluating
nominees. To qualify, each candidate must be U.S. Food and
Drug Administration-approved for marketing within the last five
years and demonstrate significant potential to advance human health
worldwide. Since its inception in 1970, the Prix Galien is
considered the biopharmaceutical and medical industries' equivalent
of the Nobel prize.
ABOUT HEREDITARY TRANSTHYRETIN (hATTR)
AMYLOIDOSIS
Hereditary ATTR 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. Hereditary ATTR 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. Additional information
on hereditary ATTR amyloidosis, including a full list of
organizations supporting the hATTR amyloidosis community
worldwide, is available
at www.hattrchangethecourse.com or by
visiting www.hATTRGuide.com.
ABOUT TEGSEDI® (INOTERSEN)
TEGSEDI was approved by the U.S. Food and Drug Administration (FDA)
for the treatment of the polyneuropathy of hereditary
transthyretin-mediated (hATTR) amyloidosis in adults. TEGSEDI,
discovered and developed by Ionis Pharmaceuticals, is the world's
first and only subcutaneous RNA-targeting drug designed to reduce
the production of human transthyretin (TTR) protein. TEGSEDI also
received marketing authorization in the European Union and
Canada for the treatment of stage
1 or stage 2 polyneuropathy in adult patients with hereditary
transthyretin amyloidosis.
The approval is based on data from the NEURO-TTR study that was
a Phase 3 randomized (2:1), double-blind, placebo-controlled,
15-month, international study in 172 patients
with hATTR amyloidosis with symptoms of polyneuropathy.
In NEURO-TTR, TEGSEDI demonstrated significant improvement compared
to placebo in measures of neuropathy and quality of life as
measured by the modified Neuropathy Impairment Score +7 (mNIS+7)
and in the Norfolk Quality of Life Questionnaire-Diabetic
Neuropathy (Norfolk QOL-DN) total score. Patients treated with
TEGSEDI experienced similar benefit regardless of subgroups such as
age, sex, race, region, Neuropathy Impairment Score (NIS), Val30Met
mutation status, and disease stage.
The approval is also based on data from the NEURO-TTR Open Label
Extension (OLE) that is an ongoing study for patients who completed
the NEURO-TTR study, designed to evaluate the long-term efficacy
and safety of TEGSEDI.
For TEGSEDI's full prescribing information, please
visit www.TEGSEDI.com.
About Akcea Therapeutics, Inc.
Akcea Therapeutics, Inc., is a wholly owned subsidiary
of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in
RNA therapeutics. Akcea commercializes
TEGSEDI® (inotersen) and
WAYLIVRA® (volanesorsen), and with Ionis is
advancing a mature pipeline of novel medicines discovered by Ionis
and based on Ionis' proprietary antisense technology. TEGSEDI is
approved in the U.S., E.U., Canada and Brazil, and
WAYLIVRA is approved in the E.U. For more information
about Akcea please visit www.akceatx.com.
ABOUT IONIS PHARMACEUTICALS, INC.
As the leader in RNA-targeted drug discovery and development, Ionis
has created an efficient, broadly applicable, drug discovery
platform called antisense technology that can treat diseases where
no other therapeutic approaches have proven effective. Our drug
discovery platform has served as a springboard for actionable
promise and realized hope for patients with unmet needs. We created
the first and only approved treatment for children and adults with
spinal muscular atrophy as well as the world's first RNA-targeted
therapeutic approved for the treatment of polyneuropathy in adults
with hereditary transthyretin amyloidosis. Our sights are set on
all the patients we have yet to reach with a pipeline of more than
40 novel medicines designed to potentially treat a broad range of
disease, including neurological, cardio-renal, metabolic,
infectious, and pulmonary diseases. To learn more about Ionis
visit www.ionispharma.com and follow us on Twitter
@ionispharma.
FORWARD-LOOKING STATEMENT
This press
release includes forward-looking statements regarding the
therapeutic and commercial potential of TEGSEDI (inotersen) and
Ionis' technologies and products in development, including the
business of Akcea Therapeutics, Inc., Ionis' wholly owned
subsidiary. 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 December 31, 2019, 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.
Ionis Pharmaceuticals® is a trademark of Ionis
Pharmaceuticals, Inc. Akcea
Therapeutics® TEGSEDI▼® are
trademarks of Akcea Therapeutics, Inc.
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SOURCE Akcea Therapeutics, Inc.; Ionis Pharmaceuticals, Inc.