Biogen and Ionis Announce Topline Phase 1 Study Results of
Investigational Drug in C9orf72 Amyotrophic Lateral Sclerosis
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 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 several investigational drugs being evaluated in ALS,
including tofersen, BIIB105 and BIIB100.
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 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 StatementThis 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 StatementThis 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.
MEDIA
CONTACTS:BiogenAshleigh Koss+ 1 908 205
2572public.affairs@biogen.comIonisDavid Polk+ 1
760 603 4679info@ionisph.com |
INVESTOR
CONTACTS:BiogenMike Hencke+1 781 464
2442IR@biogen.com IonisJennifer
Capuzelo+1 760 603 2331info@ionisph.com |
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