Biogen Inc. (Nasdaq: BIIB) today announced that the U.S. Food and
Drug Administration (FDA) has accepted a New Drug Application (NDA)
for tofersen, an investigational drug for superoxide dismutase 1
(SOD1) amyotrophic lateral sclerosis (ALS). The application has
been granted priority review and given a Prescription Drug User Fee
Act action date of January 25, 2023. The FDA has noted that it is
currently planning to hold an Advisory Committee meeting for this
application, on a yet-to-be determined date. The average life
expectancy for people with ALS is three to five years from time of
symptom onset. There is currently no treatment targeted for
SOD1-ALS.2
“The available data show that tofersen has the potential to make
a meaningful difference for people with SOD1-ALS,” said Priya
Singhal, M.D., M.P.H., Head of Global Safety and Regulatory
Sciences and Interim Head of R&D at Biogen. “Pursuing the FDA’s
accelerated approval pathway offers the potential to make tofersen
available to people living with this fatal, neurodegenerative
disease as quickly as possible. If approved, tofersen will be the
first treatment to target a genetic cause of ALS and we hope this
will pave the way for further advances in this relentless
disease.”
Biogen is seeking approval of tofersen under the FDA’s
accelerated approval pathway, based on the use of neurofilament as
a surrogate biomarker that is reasonably likely to predict clinical
benefit. Neurofilaments are normal proteins found in healthy
neurons, that are increased in blood and cerebrospinal fluid when
damage has been done to neurons or their axons and are a marker of
neurodegeneration. In ALS, higher levels of neurofilaments
have been found to predict more rapid decline in clinical function
and shortened survival.3 Tofersen study results suggest reductions
in neurofilament preceded and predicted slowing of decline in
measures of clinical and respiratory function, strength, and
quality of life. Biogen is committed to ongoing data generation and
finalizing the confirmatory data package with the FDA.
“The 12-month results showed that individuals with SOD1-ALS who
started tofersen earlier experienced a slower rate of decline in
clinical and respiratory function, strength and quality of life.
These are critical measures for people living with this devastating
disease,” said Timothy Miller, M.D., Ph.D., principal investigator
of VALOR and ALS Center co-Director at Washington University School
of Medicine, St. Louis. “For people in my clinic living with
SOD1-ALS, tofersen may meaningfully slow the rapid progression of
their disease and the impact it has on their lives.”
The tofersen NDA included results from a Phase 1 study in
healthy volunteers, a Phase 1/2 study evaluating ascending dose
levels, the Phase 3 VALOR study, and the open label extension (OLE)
study. Also included are the most current 12-month integrated
results from VALOR and the OLE study, recently presented at the
European Network to Cure ALS (ENCALS) annual meeting.
As previously reported in October 2021, VALOR, a six-month Phase
3 randomized study, did not meet the primary endpoint of change
from baseline to week 28 in the Revised Amyotrophic Lateral
Sclerosis Functional Rating Scale. However, trends of reduced
disease progression across multiple secondary and exploratory
endpoints were observed. The 12-month integrated data showed that
earlier initiation of tofersen led to sustained reductions in
neurofilament, a marker of neurodegeneration and slowed decline
across multiple efficacy endpoints.
In the 12-month data, the most common adverse events (AEs) in
participants receiving tofersen in VALOR and the OLE study were
headache, procedural pain, fall, back pain and pain in extremities.
Most AEs in both VALOR and the OLE were mild to moderate in
severity. Serious neurologic events including myelitis,
radiculitis, aseptic meningitis, and papilledema, were reported in
6.7 percent of participants receiving tofersen in VALOR and its
OLE.
During the FDA review period Biogen will maintain its early
access program for tofersen, now with participants in over a dozen
countries. The open-label extension and Phase 3 ATLAS study in
presymptomatic individuals with a SOD1 genetic mutation remain
ongoing. Biogen is actively engaging with other regulators around
the world and will provide updates when appropriate.
About TofersenTofersen is an antisense drug
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
Pharmaceuticals, Inc. under a collaborative development and license
agreement.
About Amyotrophic Lateral Sclerosis and
SOD1-ALSAmyotrophic 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.2
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 percent of the estimated 168,000 people who have
ALS globally (SOD1-ALS).1 Life expectancy in SOD1-ALS varies
widely with some patients surviving less than a year.4
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 BiogenAs pioneers in neuroscience, Biogen
discovers, develops, and delivers worldwide innovative therapies
for people living with serious neurological diseases as well as
related therapeutic adjacencies. One of the world’s first global
biotechnology companies, Biogen was founded in 1978 by Charles
Weissmann, Heinz Schaller, Sir Kenneth Murray, and Nobel Prize
winners Walter Gilbert and Phillip Sharp. Today, Biogen has a
leading portfolio of medicines to treat multiple sclerosis, has
introduced the first approved treatment for spinal muscular
atrophy, and developed the first and only approved treatment to
address a defining pathology of Alzheimer’s disease. Biogen is also
commercializing biosimilars and focusing on advancing one of the
industry’s most diversified pipelines in neuroscience that will
transform the standard of care for patients in several areas of
high unmet need.
In 2020, Biogen launched a bold 20-year, $250 million initiative
to address the deeply interrelated issues of climate, health, and
equity. Healthy Climate, Healthy Lives™ aims to eliminate fossil
fuels across the company’s operations, build collaborations with
renowned institutions to advance the science to improve human
health outcomes, and support underserved communities.
We routinely post information that may be important to investors
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Biogen Safe Harbor This news release
contains forward-looking statements, including statements made
pursuant to the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995, 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 these statements
or the scientific data presented.
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 are based on our current
beliefs and expectations and speak only as of the date of this news
release.
We do not undertake any obligation to publicly update any
forward-looking statements, whether as a result of new information,
future developments or otherwise.
References:
- Brown CA, Lally C, Kupelian V,
Flanders WD. Estimated Prevalence and Incidence of Amyotrophic
Lateral Sclerosis and SOD1 and C9orf72 Genetic Variants.
Neuroepidemiology. 2021
- Brown RH, Al-Chalabi A. Amyotrophic
Lateral Sclerosis. N Engl J Med. 2017 Jul 13
- Thompson AG et al. Multicentre
appraisal of amyotrophic lateral sclerosis biofluid biomarkers
shows primacy of blood neurofilament light chain. Brain
Commun. 2022;4(1):fcac029. Published 2022 Feb 9.
doi:10.1093/braincomms/fcac029
- Bali T, et al. Defining SOD1 ALS
natural history to guide therapeutic clinical trial design. J
Neurol Neurosurg Psychiatry. 2017 Feb
MEDIA
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