New Data from Biogen’s Investigational Antisense Oligonucleotide (ASO) Targeting Tau Shows Promise for Potential New Generation of Treatments in Early Alzheimer’s Disease
25 Outubro 2023 - 11:51AM
Biogen Inc. (Nasdaq: BIIB) reported new Phase 1b clinical data from
the study of BIIB080, an investigational antisense oligonucleotide
(ASO) therapy targeting tau, in mild Alzheimer’s disease (AD). The
data showed favorable trends on multiple exploratory endpoints of
cognition and activities of daily living in AD (n=46), building
upon prior results which showed a reduction of tau protein in the
cerebral spinal fluid (CSF t-tau) and tau positron emission
tomography (PET) across brain regions. The late-breaking results
were presented at the 2023 Clinical Trials on Alzheimer’s Disease
(CTAD) meeting held in Boston, MA from October 24-27.
In AD, both tau and amyloid beta are linked to disease
progression.1 Tau protein can form tangles which progressively
accumulate in brain regions involved in cognition.2 The
accumulation of pathological tau tangles has been shown to lead to
neuronal loss. ASO therapies are seen as promising tools for
modulating production of disease-associated proteins. Currently,
the ASO approach underpins more than 60 treatments approved or in
clinical trials for a variety of disease areas including a range of
cancers, viral illnesses, and genetic conditions.3
“This is the first time we’ve seen both strong target engagement
and favorable trends on clinical outcomes with a novel mechanism
targeting tau,” said Priya Singhal, M.D., M.P.H., Head of
Development at Biogen. “While these are preliminary findings, we
are excited about these results and continue to enroll the Phase 2
CELIA study. We believe defeating Alzheimer’s disease will take
different approaches and we are committed to exploring the
targeting of tau as a new generation of treatment.”
In results presented at CTAD, favorable trends were observed on
the global Clinical Dementia Rating Sum of Boxes (CDR-SB),
Mini-Mental State Exam (MMSE) cognitive scales and Functional
Activities Questionnaire (FAQ) at week 100 in groups treated with a
high-dose of BIIB080 (n=16). The results build on data presented at
the International Conference on Alzheimer’s and Parkinson’s Disease
(ADPDTM 2023) which showed that direct targeting of tau protein
production had a substantial impact on tau biomarkers, reducing
total and phosphorylated tau in the CSF and aggregated tau
pathology as measured by PET across all brain regions assessed. The
favorable trends suggest a potential link between a reduction in
tau PET pathology and clinical outcomes.
Treatment was generally well tolerated throughout the study. The
majority of adverse events were mild or moderate in severity, of
which the most common were headache, back pain, pain in extremity,
post-lumbar puncture syndrome and procedural pain.
BIIB080 is designed to target microtubule-associated protein tau
(MAPT) mRNA and reduce production of tau protein. The Phase 1b
trial and its long-term extension study (LTE) were designed to
assess the safety and tolerability of multiple doses in patients
with mild dementia due to AD. Participants were randomized to
placebo or to four dose cohorts receiving 10mg once every 4 weeks
[Q4W], 30mg Q4W, 60mg Q4W or 115mg Q12W. In the LTE, all
participants received 60mg or 115mg every 12 weeks.
Recruitment for the Phase 2 CELIA study (NCT05399888),
evaluating the potential for this ASO targeting tau to slow the
worsening of mild cognitive impairment or mild dementia due to AD,
is ongoing at sites across North America, Europe and Asia
Pacific.
In December 2019, Biogen exercised a license option with Ionis
and obtained a worldwide, exclusive, royalty-bearing license to
develop and commercialize BIIB080.
About Antisense Therapies Antisense therapies
are designed to seek out, bind to and destroy a mRNA in a highly
specific manner, so that the amount of disease-causing protein is
dramatically decreased. Antisense therapies can also treat diseases
caused by too little protein by increasing the production of the
protein, thereby restoring the protein to normal levels.
About BiogenFounded in 1978, Biogen is a
leading global biotechnology company that has pioneered multiple
breakthrough innovations including a broad portfolio of medicines
to treat multiple sclerosis, the first approved treatment for
spinal muscular atrophy, and two co-developed treatments to address
a defining pathology of Alzheimer’s disease. Biogen is advancing a
pipeline of potential novel therapies across neurology,
neuropsychiatry, specialized immunology and rare diseases and
remains acutely focused on its purpose of serving humanity through
science while advancing a healthier, more sustainable and equitable
world.
We routinely post information that may be important to investors
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Biogen Safe Harbor This news release
contains forward-looking statements, about the potential of
BIIB080, including related to the Phase 2 CELIA study; 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
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or may fail or refuse to approve or may delay approval of Biogen's
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uncertainties relating to intellectual property claims and
challenges; product liability claims; third party collaboration
risks; and the direct and indirect impacts of the ongoing COVID-19
pandemic on Biogen's 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 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.References:
- BrightFocus Foundation. Tau Protein and Alzheimer’s Disease:
What’s the Connection?
https://www.brightfocus.org/alzheimers/article/tau-protein-and-alzheimers-disease-whats-connection.
Accessed September 2023.
- Moumné et al. Oligonucleotide Therapeutics: From Discovery and
Development to Patentability. Pharmaceutics 2022, 14(2),
260; https://doi.org/10.3390/pharmaceutics14020260
- Alzheimer’s Association. Tau Topic Sheet.
https://www.alz.org/media/Documents/alzheimers-dementia-tau-ts.pdf.
Accessed October 2023.
MEDIA CONTACT:BiogenJack Cox+ 1
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INVESTOR CONTACT:BiogenChuck
Triano+1 781 464 2442IR@biogen.com |
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