The New England Journal of Medicine Publishes Final Results from
Phase 1/2 Study of Tofersen for a Genetic Form of ALS
Biogen Inc. (Nasdaq: BIIB) today announced that positive
results from a Phase 1/2 study of tofersen (BIIB067) for the
potential treatment of superoxide dismutase 1 (SOD1) amyotrophic
lateral sclerosis (ALS) were published in The New England Journal
of Medicine. A mutation in the SOD1 gene is believed to be a
genetic driver of disease in approximately two percent of all ALS
cases.
“By evaluating genetically validated targets such as SOD1 in
defined populations, we believe we can more quickly identify how to
treat this devastating disease,” said Toby Ferguson, M.D., Ph.D.,
Vice President and Head of the Neuromuscular Development Unit at
Biogen. “Biogen is committed to furthering ALS research in an
effort to potentially bring a therapy to people living with this
rapidly progressing neurological condition.”
Biogen continues to invest in ALS clinical development despite
discontinuing the dexpramipexole program in 2013 due to
disappointing results from the Phase 3 EMPOWER study. The company
has applied important learnings from the EMPOWER study to its broad
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.
The Phase 1/2 Study ResultsThe Phase 1/2 study
of tofersen was a randomized, placebo-controlled, single- and
multiple-ascending dose study that evaluated the safety,
pharmacokinetics, pharmacodynamics and exploratory efficacy
endpoints in individuals living with SOD1-ALS. In the multiple
ascending dose (MAD) portion of the study, participants with
SOD1-ALS were randomized to receive tofersen (20 mg, 40 mg, 60 mg
or 100 mg) or placebo for 12 weeks.
The primary objective of the MAD portion of the study was to
evaluate the safety, tolerability and pharmacokinetics of tofersen.
The most commonly reported adverse events in people who received
one or more doses of tofersen (n=38) were headache, procedural
pain, post-lumbar puncture syndrome and falls. Five tofersen- and
two placebo-treated people experienced serious adverse events. One
death occurred in the placebo group during the trial due to
respiratory failure secondary to ALS and 2 deaths occurred in the
tofersen group during a follow up period due to pulmonary embolism
and respiratory failure (20 mg and 60 mg group, respectively).
“The data published in The New England Journal of Medicine are
an important step in understanding the potential of tofersen and
genetic disease drivers as targets for ALS,” said Timothy Miller,
M.D., Ph.D., co-principal investigator and ALS Center Director at
Washington University School of Medicine, St. Louis. “We are
encouraged by these study results and will continue to evaluate the
efficacy and safety of tofersen as a potential treatment for
SOD1-ALS.”
The secondary outcome was the change from baseline in
cerebrospinal fluid (CSF) SOD1 protein concentration. Treatment
with tofersen 100 mg (n=10) over a 3-month period resulted in a 36
percent reduction of SOD1 concentration compared to 3 percent in
the placebo group (n=12).
Exploratory measures demonstrated numerical trends towards
slowing of clinical decline as measured by the ALS Functional
Rating Scale Revised (ALSFRS-R) as well as slow vital capacity and
muscle strength measured by handheld dynamometer (HHD) compared to
placebo. The mean change in ALSFRS-R score from baseline to day 85
was -1.19 in the tofersen 100 mg group compared to 5.63 in the
placebo group on a 48-point scale. Across exploratory clinical
measures, separation from the placebo group was primarily driven by
the fast-progressing subgroup.
“SOD1-ALS is a neurological disease with no treatment options
that reliably slow or halt its rapid progression,” said Merit
Cudkowicz, M.D., co-principal investigator and the director of the
Sean M. Healey & AMG Center for ALS at Massachusetts General
Hospital. “We are dedicated to this research with the goal of
bringing a new treatment option to this community with great unmet
need.”
A Phase 3 study, VALOR, is currently ongoing and will assess the
efficacy and safety of tofersen versus placebo in adults with
SOD1-ALS.
About Tofersen Tofersen is an antisense
oligonucleotide (ASO) 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. Tofersen demonstrated proof-of-biology and
proof-of-concept in a Phase 1/2 study. Biogen licensed tofersen
from Ionis Pharmaceuticals, Inc. under a collaborative development
and license agreement.
About Amyotrophic Lateral Sclerosis Amyotrophic
lateral sclerosis (ALS) is a progressive neurodegenerative disease
that results in the loss of motor neurons in the brain and the
spinal cord that is responsible for controlling voluntary muscle
movement. People with ALS may experience a gradual weakening of
muscles, causing them to lose their strength and ability to speak,
move and eventually breathe. There is a growing body of evidence
that mutations within multiple genes are believed to cause the
disease. SOD1-ALS, which accounts for two percent of all ALS cases,
is a subset of ALS. Genetic testing may help determine if a
person’s ALS is due to a SOD1 gene mutation, even in individuals
without a family history of the disease.
About BiogenAt Biogen, our mission is clear: we
are pioneers in neuroscience. Biogen discovers, develops and
delivers worldwide innovative therapies for people living with
serious neurological and neurodegenerative 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, Kenneth Murray and Nobel Prize winners
Walter Gilbert and Phillip Sharp. Today Biogen has the leading
portfolio of medicines to treat multiple sclerosis, has introduced
the first approved treatment for spinal muscular atrophy,
commercializes biosimilars of advanced biologics and is focused on
advancing research programs in multiple sclerosis and
neuroimmunology, Alzheimer’s disease and dementia, neuromuscular
disorders, movement disorders, ophthalmology, immunology,
neurocognitive disorders, acute neurology and pain.
We routinely post information that may be important to investors
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Biogen Safe Harbor Statement 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 1/2 study of tofersen; the potential clinical
effects of tofersen; the potential benefits, safety and efficacy of
tofersen; the clinical development program for 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
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Results in early stage clinical trials may not be indicative of
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undue reliance on these statements or the scientific data
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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
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may delay approval of our drug candidates, including tofersen; the
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