Biogen to Present New Interim Data from Its Phase 1/2 Clinical
Study of Tofersen (BIIB067) for the Potential Treatment of a
Subtype of Familial Amyotrophic Lateral Sclerosis (ALS)
Biogen Inc. (Nasdaq: BIIB) announced today interim results of
a phase 1/2 study of tofersen, an antisense oligonucleotide (ASO)
being studied for the potential treatment of amyotrophic lateral
sclerosis (ALS) in adults with a confirmed superoxide dismutase 1
(SOD1) mutation. The data will be presented at the American Academy
of Neurology Annual Meeting (AAN) in Philadelphia, PA (May 4-10,
2019).
“The interim results of this study, which achieved
proof-of-biology and proof-of-concept, support the initiation of a
phase 3 clinical trial to confirm the efficacy and safety of
tofersen in SOD1-ALS patients and further demonstrate the potential
of ASOs to target the genetic driver of disease,” said Michael
Ehlers, M.D., Ph.D., executive vice president, research and
development at Biogen. “We are committed to bringing a potential
breakthrough therapy to patients with ALS and we are expediting our
efforts with the aim of addressing this urgent unmet need.”
The Phase 1/2 study of tofersen in SOD1-ALS is a randomized,
placebo-controlled, single- and multiple-ascending dose study
evaluating the safety, tolerability, pharmacokinetics,
pharmacodynamics and exploratory efficacy endpoints of tofersen in
70 patients with ALS. In the multiple ascending dose portion of the
study, 50 participants with SOD1 mutations were randomized to
receive tofersen (20 mg, 40 mg, 60 mg, or 100 mg) or placebo for 12
weeks.
In the interim analysis, treatment with tofersen 100 mg (n=10)
over a three-month period resulted in a statistically significant
lowering of SOD1 protein levels in the cerebrospinal fluid
(p=0.002) and a numerical trend towards slowing of clinical decline
as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R),
slow vital capacity and muscle strength compared to placebo (n=12).
Specifically, the mean change in ALSFRS-R score from baseline to
Day 85 was -1.1 in the tofersen 100 mg group compared to -5.3 in
the placebo group, on a 48-point scale. Across clinical measures,
separation from the placebo group was most apparent in patients
with fast progressing disease. The safety and tolerability profile
in this analysis supports continued development of tofersen in ALS.
The data will be presented by study co-principal investigator
Timothy M. Miller, M.D., Ph.D., of the Washington University School
of Medicine in St. Louis, MO on May 7, 2019 at 11:45 a.m. ET as a
late breaking oral presentation.
“ALS is a devastating disease and there are currently no
therapeutic options that reliably slow or halt its rapid
progression,” said Merit Cudkowicz, M.D., co-principal investigator
and the director of the Healey Center at Massachusetts General
Hospital. “We are encouraged by the positive results of this phase
1/2 study of tofersen in patients with SOD1-ALS and are excited to
advance this clinical program to a phase 3 trial to further
investigate its therapeutic potential.”
In March 2019 the first patient was dosed in the Phase 3 VALOR
study of tofersen in adults with ALS with a confirmed SOD1
mutation. The VALOR study aims to assess the efficacy and safety of
tofersen versus placebo. The primary endpoint of this study is an
analysis based on the ALSFRS-R score, which is a validated rating
instrument that monitors the progression of disability in patients
with ALS. Biogen is collaborating with regulators to further define
the scope of the clinical data package required to support the
registration of tofersen.
ALS is a rare and fatal neurodegenerative disease characterized
by motor neuron loss in the brain and spinal cord that is
responsible for controlling voluntary muscle movement. Symptoms may
vary depending on the location of the motor neuron failure and may
include limb weakness, difficulty breathing and trouble speaking
and swallowing. There is a growing body of evidence that mutations
within multiple genes are believed to cause ALS. ALS with SOD1
mutations is a rare subtype of familial ALS and accounts for
approximately two percent of all ALS cases.
About tofersen Tofersen is an antisense
oligonucleotide (ASO) being developed for the treatment of ALS with
SOD1 mutations. Tofersen binds to SOD1 mRNA, allowing for its
degradation by RNase-H and reduction of SOD1 protein production.
This is thought to decrease the toxicity of mutant SOD1 and
potentially provide therapeutic benefit through improved survival
and function to people with ALS with SOD1 mutations. 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 Biogen At 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, and today has the leading
portfolio of medicines to treat multiple sclerosis, has introduced
the first and only approved treatment for spinal muscular atrophy,
and is focused on advancing neuroscience research programs in MS
and neuroimmunology, Alzheimer’s disease and dementia, movement
disorders, neuromuscular disorders, acute neurology, neurocognitive
disorders, pain, and ophthalmology. Biogen also commercializes
biosimilars of advanced biologics.
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
potential of our commercial business and pipeline programs,
including tofersen; the anticipated benefits and potential of our
collaboration arrangements with Ionis; 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 other unexpected
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risks. The foregoing sets forth many, but not all, of the factors
that could cause actual results to differ from our expectations in
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of the date of this press release.
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