- BIIB078, an investigational antisense oligonucleotide
for C9orf72-associated amyotrophic lateral sclerosis
(ALS), did not show clinical benefit; clinical program will
be discontinued
- Biogen and Ionis remain committed to their decade-long
pursuit of advancing ALS research and developing therapies for all
forms of this progressive and fatal neurodegenerative
disease
CAMBRIDGE, Mass. and CARLSBAD, Calif., March 28,
2022 /PRNewswire/ -- Biogen Inc. (Nasdaq: BIIB) and
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced topline
results from the Phase 1 study of BIIB078 (IONIS-C9Rx),
an investigational antisense oligonucleotide (ASO) for people with
C9orf72-associated amyotrophic lateral sclerosis (ALS).
In this Phase 1 study, BIIB078 was generally well-tolerated. The
adverse events (AEs) were mostly mild to moderate in severity and
occurred at a similar rate across BIIB078 and placebo groups. The
most common AEs were fall, procedural pain and headache.
BIIB078 did not meet any secondary efficacy endpoints and it did
not demonstrate clinical benefit. In the dose cohorts up to 60 mg
there were no consistent differences between the BIIB078 group and
the placebo group. Participants in the BIIB078 90 mg dose cohort
trended toward a greater decline than those in the placebo group
across secondary endpoints. Based on these results, the BIIB078
clinical development program will be discontinued, including its
ongoing open-label extension study.
"We are incredibly grateful for the selfless commitment of the
individuals with ALS who participated in the study, and the
community's dedication to advancing research for this devastating
disease," said Toby Ferguson, M.D.,
Ph.D., Vice President and Head of the Neuromuscular Development
Unit at Biogen. "While these were not the results we were hoping
for, they are clear and will inform future research across our
broad pipeline of investigational ALS therapies. We remain focused
on pioneering new treatments that will positively impact people
living with this debilitating disease."
"C9orf72-associated ALS is a complex genetic form of ALS and
there are multiple mechanisms by which the scientific community
believes the C9orf72 gene causes disease. We designed
BIIB078 to test the prevailing hypothesis that the mechanisms of
disease for C9orf72-associated ALS were caused by toxicity
associated with the repeat containing RNA and corresponding
dipeptides. Unfortunately, this Phase 1 study did not support the
hypothesis, suggesting that the disease mechanism is much more
complex. While these results do not support further development of
BIIB078, we anticipate they will provide valuable learnings that
lead to a deeper understanding of this form of ALS," said C.
Frank Bennett, Executive Vice
President, Chief Scientific Officer and Franchise Leader for
Neurological Programs at Ionis.
This Phase 1 study was a randomized, placebo-controlled,
dose-escalating trial to evaluate BIIB078 administered
intrathecally to adults (n=106) with C9orf72-associated ALS. Within
each of the six study treatment cohorts, participants were
randomized to receive BIIB078 or placebo (3:1 ratio). The primary
objective of the study was to assess safety and tolerability.
Secondary efficacy endpoints included ALS Functional Rating
Scale–Revised, Slow Vital Capacity, Hand-Held Dynamometry, and the
Iowa Oral Pressure Instrument.
The companies will present the BIIB078 Phase 1 data at a future
scientific forum.
Biogen's Continuous Commitment to ALS
For 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 several investigational drugs being evaluated in ALS,
including tofersen, BIIB105 and BIIB100.
About Biogen
As 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 is providing the first and only
approved treatment to address a defining pathology of Alzheimer's
disease. Biogen is also commercializing biosimilars and focusing on
advancing the industry's most diversified pipeline 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.
The company routinely post information that may be important to
investors on our website at www.biogen.com. To learn more,
please visit www.biogen.com and follow Biogen on social
media
– Twitter, LinkedIn, Facebook, YouTube.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been the leader in RNA-targeted
therapy, pioneering new markets and changing standards of care with
its novel antisense technology. Ionis currently has three marketed
medicines and a premier late-stage pipeline highlighted by
industry-leading cardiovascular and neurological franchises. Our
scientific innovation began and continues with the knowledge that
sick people depend on us, which fuels our vision of becoming a
leading, fully integrated biotechnology company.
To learn more about Ionis, visit www.ionispharma.com and follow
us on Twitter @ionispharma.
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 study of BIIB078; the
potential clinical effects of BIIB078; the potential benefits,
safety and efficacy of BIIB078; the clinical development program
for BIIB078; 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 BIIB078;
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
BIIB078; 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 BIIB078; 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.
Ionis Forward-looking
Statement
This press release includes forward-looking
statements regarding Ionis' business, and the therapeutic and
commercial potential of Ionis' technologies, IONIS-C9Rx
(BIIB078) 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 those related
to the impact COVID-19 could have on our business, and 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 December 31, 2021, and the most
recent Form 10-Q quarterly filing, which are on file with the SEC.
Copies of these and other documents are available from the
Company.
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SOURCE Ionis Pharmaceuticals, Inc.