Biogen Provides Update on FDA Advisory Committee Meeting on
Tofersen for SOD1-ALS
Biogen Inc. (Nasdaq: BIIB) announced today 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 product for the treatment of superoxide dismutase 1
(SOD1) amyotrophic lateral sclerosis (ALS).
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.
“After hearing the moving experiences of the ALS
community and reviewing the totality of data, the Committee voted
that reductions of neurofilament are reasonably likely to predict
clinical benefit of tofersen. If approved, tofersen would
potentially represent a major advance for people living with
SOD1-ALS,” said Priya Singhal, M.D., M.P.H, Executive Vice
President and Head of Development and interim Head of Research and
Global Safety and Regulatory Sciences at Biogen. “We thank the FDA
for convening this important discussion. Most importantly, we are
grateful to all the people with SOD1-ALS who participated in our
tofersen studies, and their caregivers, families, study
investigators and the entire community, without whom this
scientific progress could not have been made.”
FDA 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 action date of April 25, 2023.
About TofersenTofersen is an
antisense oligonucleotide (ASO) 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 Pharmaceuticals, Inc. under a
collaborative development and license agreement.
About Amyotrophic Lateral Sclerosis and
SOD1-ALSAmyotrophic lateral sclerosis (ALS) is an
ultra-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.1
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 percent
of all ALS cases, impacting about 330 people in the United States.2
While there are medications approved for broad ALS, no available
treatments target a genetic mutation associated with ALS.
Approximately 5-10 percent of people with ALS are thought to have a
genetic form of the disease;1 however, they may not have a known
family history of the disease.
Biogen’s Continuous Commitment to
ALSFor over a decade, Biogen has been committed to
advancing ALS research to provide a deeper understanding of all
forms of the disease. The company has continued to invest in and
pioneer research despite making the difficult decision to
discontinue a late-stage ALS asset in 2013. Biogen has applied
important learnings to its portfolio of assets for genetic and
other forms of ALS, with the goal of increasing the probability of
bringing a potential therapy to patients in need. These applied
learnings include evaluating genetically validated targets in
defined patient populations, pursuing the most appropriate modality
for each target and employing sensitive clinical endpoints. Today,
the company has a pipeline of investigational drugs being evaluated
in ALS, including tofersen and BIIB105.
About BiogenFounded in 1978,
Biogen is a leading global biotechnology company that has pioneered
multiple breakthrough innovations including a broad portfolio of
medicines to treat multiple sclerosis, the first approved treatment
for spinal muscular atrophy, and two co-developed treatments to
address a defining pathology of Alzheimer’s disease. Biogen is
advancing a pipeline of potential novel therapies across neurology,
neuropsychiatry, specialized immunology and rare diseases and
remains acutely focused on its purpose of serving humanity through
science while advancing a healthier, more sustainable and equitable
world.
We routinely post information that may be important
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Biogen Safe Harbor This news
release contains forward-looking statements, including statements
about results from the Phase 3 VALOR study of tofersen or its OLE;
the potential clinical effects of tofersen; the potential benefits,
safety and efficacy of tofersen; the clinical development program
for tofersen; the potential approval of tofersen; the
identification and treatment of ALS; our research and development
program for the treatment of ALS; the potential of our commercial
business and pipeline programs, including tofersen; and risks and
uncertainties associated with drug development and
commercialization. These forward-looking statements may be
accompanied by words such as “aim,” “anticipate,” “believe,”
“could,” “estimate,” “expect,” “forecast,” “intend,” “may,” “plan,”
“potential,” “possible,” “will,” “would” and other words and terms
of similar meaning. Drug development and commercialization involve
a high degree of risk and only a small number of research and
development programs result in commercialization of a product.
Results in early stage clinical trials may not be indicative of
full results or results from later stage or larger scale clinical
trials and do not ensure regulatory approval. You should not place
undue reliance on our forward-looking statements.
These statements involve risks and uncertainties
that could cause actual results to differ materially from those
reflected in such statements, including without limitation,
uncertainty of success in the development and potential
commercialization of tofersen; the risk that we may not fully
enroll our clinical trials or enrollment will take longer than
expected; unexpected concerns may arise from additional data,
analysis or results obtained during our clinical trials; regulatory
authorities may require additional information or further studies,
or may fail or refuse to approve or may delay approval of our drug
candidates, including tofersen; the occurrence of adverse safety
events; the risks of unexpected hurdles, costs or delays; failure
to protect and enforce our data, intellectual property and other
proprietary rights and uncertainties relating to intellectual
property claims and challenges; product liability claims; and the
direct and indirect impacts of the ongoing COVID-19 pandemic on our
business, results of operations and financial condition. The
foregoing sets forth many, but not all, of the factors that could
cause actual results to differ from our expectations in any
forward-looking statement. Investors should consider this
cautionary statement, as well as the risk factors identified in our
most recent annual or quarterly report and in other reports we have
filed with the U.S. Securities and Exchange Commission. These
statements speak only as of the date of this news release.
We do not undertake any obligation to publicly
update any forward-looking statements.
References:
- National 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: March 2023.
- 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.
MEDIA CONTACT:BiogenJack Cox+ 1 210 544
7920public.affairs@biogen.com |
INVESTOR CONTACT:BiogenMike Hencke+1 781 464
2442IR@biogen.com |
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