- Development of BIIB105, an investigational antisense
oligonucleotide for amyotrophic lateral sclerosis (ALS), will be
discontinued based on data from the Phase 1/2 ALSpire study
- Biogen and Ionis continue their long-standing commitment to
developing therapies for ALS given the devastating impact of this
progressive, fatal neurodegenerative condition
CARLSBAD, Calif. and CAMBRIDGE, Mass., May 16, 2024
/PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and
Biogen Inc. (Nasdaq: BIIB) announced the decision to terminate
development of BIIB105 (ION541) an investigational antisense
oligonucleotide (ASO) for amyotrophic lateral sclerosis (ALS) based
on topline results from the Phase 1/2 ALSpire study. BIIB105 was
designed to reduce expression of ataxin-2 (ATXN2) protein and
demonstrated statistically significant cerebrospinal fluid (CSF)
ATXN2 protein reductions in the study. However, over the 6-month
placebo-controlled period, treatment with BIIB105 did not result in
a reduction in levels of plasma neurofilament light chain (NfL), a
marker of neurodegeneration and neuronal damage. Additionally,
BIIB105 did not demonstrate an impact on clinical outcome measures
of function, breathing and strength.
"While BIIB105 lowered ATXN2 protein, it did not reduce
neurofilament, which gives us confidence that BIIB105 did not slow
the disease process," said Stephanie
Fradette, Pharm.D., Head of the Neuromuscular Development
Unit at Biogen. "We are deeply grateful for the contributions of
the study participants and remain committed to developing
treatments that can meaningfully change the disease trajectory for
people living with ALS."
"We are very appreciative of the people with ALS and
investigators who participated in this study and were critical to
advancing our scientific understanding of ALS," said Frank Bennett, Ph.D., executive vice president
and chief scientific officer of Ionis. "Ionis continues to be
committed to the ALS community and is advancing our Phase 3
ulefnersen program for people with the genetic form of the disease
known as FUS-ALS."
Longer-term biomarker and efficacy data from the
open-label-extension were similar to those seen during the 6-month
placebo-controlled treatment period, with sustained reductions in
ATXN2 but no impact on NfL or clinical outcome measures over 40+
weeks of follow up. No evidence of benefit was observed in any
subgroup evaluated, including those participants with a Poly-CAG
expansion in the ATXN2 gene.
The Phase 1/2 study was a randomized, placebo-controlled,
dose-escalating trial to evaluate BIIB105 administered
intrathecally to adults (n=99) with ALS. Participants were
randomized to receive BIIB105 or placebo (3:1 or 2:1 ratio) for 3
to 6 months. Participants who completed the placebo-controlled
period were eligible to enroll in the open-label extension.
During the 6-month placebo-controlled portion of the study, the
most common adverse events (AEs) in BIIB105 treated participants
were procedural pain, headache and fall. AEs leading to study
discontinuation were higher in the BIIB105 group (8.3%) compared
with the placebo group (3.6%).
Analyses of data from the study are ongoing to further
understanding of the underlying disease process and effects of
BIIB105. The companies will present the BIIB105 Phase 1/2 data at
the upcoming European Network to Cure ALS (ENCALS) meeting in
Stockholm, Sweden in June.
About Ionis' Neurology Franchise
Ionis' neurology
franchise addresses all major brain regions and central nervous
system cell types and currently has three Phase 3 studies ongoing
with 11 therapies in clinical development, several of which Ionis
plans to commercialize directly. Ionis is discovering and
developing potential treatments for many neurological diseases for
which there are few or no disease modifying treatments, including
common diseases like Alzheimer's and Parkinson's as well as rare
diseases such as amyotrophic lateral sclerosis (ALS) and Alexander
disease. Ionis has discovered and developed three commercially
available neurological disease medicines, including
SPINRAZA® (nusinersen), the first approved treatment for
spinal muscular atrophy, WAINUATM (eplontersen), a
medicine to treat hereditary transthyretin-mediated amyloid
polyneuropathy (ATTRv-PN), and QALSODY® (tofersen) for
SOD1-ALS.
About Ionis Pharmaceuticals, Inc.
For three decades,
Ionis has invented medicines that bring better futures to people
with serious diseases. Ionis currently has five marketed medicines
and a leading pipeline in neurology, cardiology, and other areas of
high patient need. As the pioneer in RNA-targeted medicines, Ionis
continues to drive innovation in RNA therapies in addition to
advancing new approaches in gene editing. A deep understanding of
disease biology and industry-leading technology propels our work,
coupled with a passion and urgency to deliver life-changing
advances for patients.
To learn more about Ionis, visit Ionispharma.com and
follow us on X (Twitter) and LinkedIn.
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. In addition to QALSODY, the
first treatment to target a genetic cause of ALS, which was
discovered by Ionis, the company has a robust discovery pipeline
including efforts to address TDP43 pathology for the broad ALS
population. TDP43 pathology is seen in 97% of ALS cases and is
considered a hallmark of the disease.
About Biogen
Founded in 1978, Biogen is a leading
biotechnology company that pioneers innovative science to deliver
new medicines to transform patients' lives and to create value for
shareholders and our communities. We apply deep understanding of
human biology and leverage different modalities to advance
first-in-class treatments or therapies that deliver superior
outcomes. Our approach is to take bold risks, balanced with return
on investment to deliver long-term growth.
We routinely post information that may be important to investors
on our website at www.biogen.com. Follow us on social
media - Facebook, LinkedIn, X, YouTube.
Ionis Forward-looking
Statements
This press release includes forward-looking
statements regarding Ionis' business, financial guidance and the
therapeutic and commercial potential of our commercial medicines,
additional medicines in development and technologies. 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 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. Except as required by law, we undertake
no obligation to update any forward-looking statements for any
reason. 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,
2023, and most recent Form 10-Q, which are on file with the
Securities and Exchange Commission. Copies of these and other
documents are available from the Company.
In this press release, unless the context requires otherwise,
"Ionis," "Company," "we," "our" and "us" all refer to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a registered trademark
of Ionis Pharmaceuticals, Inc. QALSODY® is a
registered trademark of Biogen. WAINUATM is a registered
trademark of the AstraZeneca group of companies.
Biogen Safe Harbor
This news release contains
forward-looking statements, about the treatment of ALS; the
anticipated benefits and potential of Biogen's collaboration
arrangements with Ionis; the potential of Biogen's commercial
business and pipeline programs; and risks and uncertainties
associated with drug development and commercialization. These
statements may be identified by words such as "aim," "anticipate,"
"believe," "could," "estimate," "expect," "forecast," "intend,"
"may," "plan," "possible," "potential," "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
studies may not be indicative of full results or results from later
stage or larger scale clinical studies and do not ensure regulatory
approval. You should not place undue reliance on these
statements.
These statements involve risks and uncertainties that could
cause actual results to differ materially from those reflected in
such statements, including without limitation unexpected concerns
that may arise from additional data, analysis or results obtained
during clinical studies; the occurrence of adverse safety events;
risks of unexpected costs or delays; the risk of other unexpected
hurdles; regulatory submissions may take longer or be more
difficult to complete than expected; regulatory authorities may
require additional information or further studies, or may fail or
refuse to approve or may delay approval of Biogen's drug
candidates, including lecanemab; actual timing and content of
submissions to and decisions made by the regulatory authorities
regarding lecanemab; uncertainty of success in the development and
potential commercialization of lecanemab; failure to protect and
enforce Biogen's data, intellectual property and other proprietary
rights and uncertainties relating to intellectual property claims
and challenges; product liability claims; and third party
collaboration risks, 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 Biogen's expectations in
any forward-looking statement. Investors should consider this
cautionary statement as well as the risk factors identified in
Biogen's most recent annual or quarterly report and in other
reports Biogen has filed with the U.S. Securities and Exchange
Commission. These statements speak only as of the date of this news
release. Biogen does not undertake any obligation to publicly
update any forward-looking statements.
MEDIA
CONTACT(S):
Ionis
Hayley
Soffer
+1 760 603
4679
CorporateCommunications@ionisph.com
Biogen
Jack Cox
+ 1 781 464
3260
public.affairs@biogen.com
|
INVESTOR
CONTACT(S):
Ionis
D. Wade Walke,
Ph.D.
+1 760 603
2331
info@ionisph.com
Biogen
Chuck Triano
+1 781 464
2442
IR@biogen.com
|
View original content to download
multimedia:https://www.prnewswire.com/news-releases/ionis-and-biogen-announce-topline-phase-12-study-results-of-investigational-drug-in-amyotrophic-lateral-sclerosis-302147377.html
SOURCE Ionis Pharmaceuticals, Inc.