- If approved, tofersen will be the first treatment targeting
a genetic cause of ALS and the next marketed Ionis-discovered
antisense medicine
- FDA decision expected by April 25,
2023
CARLSBAD, Calif., March 22,
2023 /PRNewswire/ -- Ionis (Nasdaq: IONS) today
announced the outcome of the U.S. Food and Drug Administration's
(FDA) Peripheral and Central Nervous System Drugs Advisory
Committee meeting on tofersen, an investigational antisense
medicine for the treatment of people with superoxide dismutase 1
(SOD1) amyotrophic lateral sclerosis (ALS). SOD1-ALS is a rare
genetic form of ALS that leads to the loss of everyday functions
and ultimately death.
On the question, "Is the available evidence sufficient to
conclude that a reduction in plasma neurofilament light chain (NfL)
concentration in tofersen-treated patients is reasonably likely to
predict clinical benefit of tofersen for treatment of patients with
SOD1-ALS?", the committee voted unanimously yes (9 yes to 0 no) for
consideration of a potential accelerated approval. On the second
question, "Does the clinical data from the placebo-controlled study
and available long-term extension study results, with additional
supporting results from the effects on relevant biomarkers (i.e.,
changes in plasma NfL concentration and/or reductions in SOD1),
provide substantial evidence of the effectiveness of tofersen in
the treatment of patients with SOD1-ALS?", the committee voted 3
(yes), 5 (no) and 1 (abstain) for consideration of a potential
traditional approval.
Additionally, the committee discussed both of these topics and
reached consensus that the benefit-risk profile was favorable based
on the review of the totality of data for tofersen in people with
SOD1-ALS.
"We are encouraged by the outcome of today's advisory committee
meeting. If approved, tofersen would be the first medicine
targeting a known cause of familial ALS, a devastating
neurodegenerative disease that diminishes motor function and leads
to death within two to five years of diagnosis," said C.
Frank Bennett, Ph.D., executive vice
president and chief scientific officer at Ionis. "Tofersen is the
lead investigational medicine from our innovative pipeline of
neurological therapies designed to target the root cause of
intractable diseases. We are diligently advancing these programs to
potentially benefit patients in need."
Advisory Committees provide non-binding recommendations for
consideration by the FDA. The New Drug Application for tofersen for
the treatment of SOD1-ALS was submitted to the FDA for
consideration under accelerated approval. The FDA is continuing its
review of tofersen with a Prescription Drug User Fee Act (PDUFA)
action date of April 25, 2023.
About Tofersen
Tofersen is an investigational antisense medicine being
evaluated as a treatment for SOD1-ALS. In people with this form of
the disease, mutations in their SOD1 gene cause their bodies to
create a toxic form of SOD1 protein. This toxic protein causes
motor neurons to degenerate, resulting in progressive muscle
weakness. Tofersen is designed to bind to SOD1 mRNA and reduce SOD1
protein production.
In addition to the ongoing open label extension of the Phase 3
VALOR study, 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. Mean life expectancy for people with ALS is between three
to five years from time of symptom onset.
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. SOD1-ALS is diagnosed in approximately 2% of all
ALS cases, impacting about 330 people in the U.S.1 While
there are medications approved for broad ALS, no available
treatments target a genetic mutation associated with ALS.
Approximately 5%-10% of people with ALS are thought to have a
genetic form of the disease;2 however, they may not have
a known family history of the disease.
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.
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, 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 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.
1Brown 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.
2National Institute of Neurological Disorders and
Stroke. Amyotrophic Lateral Sclerosis (ALS) Fact Sheet. Available
at:
https://www.ninds.nih.gov/amyotrophic-lateral-sclerosis-als-fact-sheet.
Accessed: January 2023.
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SOURCE Ionis Pharmaceuticals, Inc.