- SOD1-ALS affects approximately 2% of people living with ALS
worldwide1
- If approved, tofersen would be the world's first treatment
to target a genetic cause of ALS
- EMA acceptance follows FDA's acceptance of tofersen NDA
earlier this year
CARLSBAD, Calif., Dec. 5, 2022
/PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today
announced that the European Medicines Agency (EMA) has accepted the
marketing authorization application (MAA) for review of tofersen,
an investigational medicine for the treatment of superoxide
dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). SOD1-ALS is
a progressive and uniformly fatal disease that affects fewer than
1,000 people across Europe.2
The EMA is the second regulatory agency to accept review of a
marketing application for tofersen following U.S. Food and Drug
Administration (FDA) acceptance earlier this year. The FDA has set
a PDUFA date for tofersen of April 25,
2023. There are currently no treatments targeted for
SOD1-ALS.
"EMA acceptance of the tofersen MAA is a significant development
for people and families battling SOD1-ALS," said C. Frank Bennett, Ph.D., executive vice president,
chief scientific officer and franchise leader for neurological
programs at Ionis. "If approved, tofersen will be the world's first
treatment that targets a genetic cause of ALS. Tofersen also
strengthens Ionis' platform strategy to target other neurological
diseases, including other forms of ALS."
The MAA includes results from the Phase 3 VALOR study, its open
label extension (OLE) study, a Phase 1 study in healthy volunteers
and a Phase 1/2 study evaluating ascending dose levels. Also
included are the most current 12-month integrated results from
VALOR and the OLE study that were recently published in The New
England Journal of Medicine.
Biogen announced that it will maintain its early access program
for tofersen, which is now available in 34 countries. Biogen also
announced that it will continue to actively engage with other
regulators around the world and will provide updates when
appropriate.
About Tofersen
Tofersen is an antisense medicine being evaluated for the
potential treatment of SOD1-ALS. Tofersen binds to SOD1 mRNA,
allowing for its degradation by RNase-H in an effort to reduce
synthesis of SOD1 protein production. In addition to the ongoing
open label extension of VALOR, tofersen is being studied in the
Phase 3 ATLAS study designed to evaluate whether tofersen can delay
clinical onset when initiated in presymptomatic individuals with a
SOD1 genetic mutation and biomarker evidence of disease activity.
Biogen licensed tofersen from Ionis under a collaborative
development and license agreement.
About Amyotrophic Lateral
Sclerosis and SOD1-ALS
Amyotrophic lateral sclerosis (ALS) is a rare, progressive and
fatal neurodegenerative disease that results in the loss of motor
neurons in the brain and the spinal cord that are responsible for
controlling voluntary muscle movement. People with ALS experience
muscle weakness and atrophy, causing them to lose independence as
they steadily lose the ability to move, speak, eat, and eventually
breathe. Average life expectancy for people with ALS is three to
five years from time of symptom onset. Patients with some SOD1
mutations have an even shorter life expectancy.
Multiple genes have been implicated in ALS. Genetic testing
helps determine if a person's ALS is associated with a genetic
mutation, even in individuals without a family history of the
disease. Currently, there are no genetically targeted treatment
options for ALS. Mutations in the SOD1 gene are responsible for
approximately 2% of the estimated 168,000 people who have ALS
globally (SOD1-ALS).
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, tofersen 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
Securities and Exchange Commission. 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.
1 Brown CA, Lally C, Kupelian V, Flanders WD.
Estimated Prevalence and Incidence of Amyotrophic Lateral Sclerosis
and SOD1 and C9orf72 Genetic Variants. Neuroepidemiology.
2021;55(5):342-353. doi: 10.1159/000516752. Epub 2021 Jul
9.
2 Brown CA, Lally C, Kupelian V, Flanders WD.
Estimated Prevalence and Incidence of Amyotrophic Lateral Sclerosis
and SOD1 and C9orf72 Genetic Variants. Neuroepidemiology.
2021;55(5):342-353. doi: 10.1159/000516752. Epub 2021 Jul
9.
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SOURCE Ionis Pharmaceuticals, Inc.