CAMBRIDGE, Mass. and
CARLSBAD, Calif., July 26, 2021 /PRNewswire/ -- Biogen Inc.
(Nasdaq: BIIB) and Ionis Pharmaceuticals, Inc. (Nasdaq: IONS)
announced today that topline data from a Phase 1b placebo-controlled, multiple ascending dose
clinical study showed that BIIB080/IONIS-MAPTRx met its
primary objective of safety and tolerability in patients with mild
Alzheimer's disease. The study demonstrated robust time and dose
dependent lowering of tau protein in cerebrospinal fluid (CSF) over
the three-month treatment period and sustained reductions during
the six-month post-treatment period.
In patients receiving BIIB080, there were dose-dependent
decreases in the concentration of total-tau in CSF eight weeks
post-last dose (Day 141) with a mean percentage reduction of 30
percent, 40 percent and 49 percent in the low, medium and high dose
groups treated every four-weeks, respectively, and 42 percent in
the group treated every 12 weeks. Total-tau in the CSF continued to
decline 16 weeks post-last dose in patients treated with BIIB080 in
the high dose four-week and 12-week dose groups, showing a 55
percent and 49 percent mean reduction from baseline, respectively.
CSF was not collected 16 weeks post-last dose in the low and medium
four-week dose groups. There were similar dose-dependent decreases
in the levels of phosphorylated tau. All participants (n=46)
completed the Multiple Ascending Dose (MAD) period and 43
participants completed the Post-Treatment (PT) period (3
participants voluntarily withdrew). These data were presented in a
poster session at the 2021 Alzheimer's Association International
Conference (AAIC) held virtually and in Denver, Colo., July
26 – 30.
"There is clearly an urgent need to develop and deliver
effective treatments for Alzheimer's disease, a devastating
disorder for which there currently are limited therapeutic options.
We are encouraged by the topline results from this study of
BIIB080, which demonstrate the potential of Ionis' antisense
technology to successfully target what we believe is a root cause
of Alzheimer's disease," said C. Frank
Bennett, Ph.D., Ionis' chief scientific officer and
franchise leader for neurological programs. Dr. Bennett added,
"These study results support further investigation of BIIB080 for
the treatment of Alzheimer's disease and suggest that
antisense-mediated suppression of tau protein may be a feasible
therapeutic approach for other tauopathies."
"Biogen is deeply committed to the development of novel
treatments for patients with Alzheimer's disease. This commitment
extends across multiple modalities, including antisense
oligonucleotides, as with BIIB080," said Alfred Sandrock, Jr., M.D., Ph.D., Head of
Research and Development at Biogen. "Biogen is encouraged by the
results of this trial, and we look forward to our continued
research in future clinical studies with this promising
investigational asset."
Alzheimer's disease is a progressive neurodegenerative disorder
characterized by cognitive and functional decline resulting in
significant disability. Until recently, treatment was limited to
management of symptoms. BIIB080 is an investigational antisense
therapy designed to target microtubule-associated protein tau
(MAPT) mRNA and prevent production of tau protein. Growing evidence
suggests that aggregated, hyperphosphorylated tau may be a key
driver of neurodegeneration in Alzheimer's disease as well as other
tauopathies including progressive supranuclear palsy and
frontotemporal degeneration. In preclinical studies in MAPT
transgenic mice, MAPT-targeted antisense treatment demonstrated
robust tau-lowering in CNS tissues and prevention and reversal of
disease.
The primary objective of the Phase 1b first-in-human study was to assess safety and
tolerability of multiple intrathecal (IT) bolus administrations of
BIIB080. The study was divided into two parts: Part 1, a MAD study
of 46 patients with mild Alzheimer's disease comprising a
three-month Treatment Evaluation Period and a six-month PT period;
Part 2, an open label long-term extension study comprising a
12-month Treatment Evaluation Period and a four- or six-month PT
period. Four ascending dose cohorts were enrolled sequentially and
randomized 3:1 to IT bolus administrations of BIIB080 or placebo.
Patients aged 50-74 years with mild Alzheimer's disease and
confirmed amyloid positivity (via CSF) at screening were considered
eligible. Part 1 is now complete; Part 2 is currently ongoing
(EudraCT: 2016-002713-22; NCT03186989).
The characteristics of patients at baseline were representative
of relatively younger, mild Alzheimer's disease patients and were
generally similar across trial groups. All adverse events were mild
to moderate in severity with no serious adverse events occurring in
any patients that received BIIB080. There were no deaths,
dose-limiting adverse events or dosing discontinuations.
About Ionis' Neurology Franchise
The Ionis neurology franchise addresses all major brain regions
and central nervous system cell types and currently has three Phase
3 studies ongoing with 11 medicines in clinical development, three
of which are wholly owned. Ionis is leading the way in treating
root causes of many neurological diseases and developing antisense
medicines for common diseases like Alzheimer's and Parkinson's as
well as rare diseases like amyotrophic lateral sclerosis (ALS) and
Alexander disease. Ionis' marketed neurological disease medicines
include SPINRAZA®, a global foundation of care for
spinal muscular atrophy (SMA), commercialized by Biogen, and
TEGSEDI®, the first and only self-administered,
subcutaneous treatment for the polyneuropathy of hereditary ATTR
amyloidosis in adults.
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. Today Biogen has the leading
portfolio of medicines to treat multiple sclerosis, has introduced
the first approved treatment for spinal muscular atrophy,
commercializes biosimilars of advanced biologics and is focused on
advancing research programs in multiple sclerosis and
neuroimmunology, Alzheimer's disease and dementia, neuromuscular
disorders, movement disorders, ophthalmology, neuropsychiatry,
immunology, acute neurology and neuropathic
pain.
We routinely post information that may be important to investors
on our website at www.biogen.com. Follow us on social media
– Twitter, LinkedIn, Facebook, YouTube.
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, relating to the
potential clinical effects of BIIB080; the potential benefits,
safety and efficacy of BIIB080; the results of the Phase
1b study of BIIB080; the clinical
development program for BIIB080; the identification and treatment
of Alzheimer's disease; the potential of Biogen's commercial
business and pipeline programs, including BIIB080; the anticipated
benefits and potential of Biogen's collaboration arrangements with
Ionis; and risks and uncertainties associated with drug development
and commercialization. These forward-looking statements may be
accompanied by such words as "aim," "anticipate," "believe,"
"could," "estimate," "expect," "forecast," "goal," "intend," "may,"
"plan," "potential," "possible," "prospect," "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 unexpected concerns that may arise from
additional data, analysis or results obtained during clinical
trials; the occurrence of adverse safety events; risks of
unexpected costs or delays; the risk of other unexpected hurdles;
uncertainty of success in the development and potential
commercialization of BIIB080; 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; third party collaboration
risks; the direct and indirect impacts of the ongoing COVID-19
pandemic on Biogen's business, results of operations and financial
condition; and any other risks and uncertainties that are described
in other reports Biogen has filed with the U.S. Securities and
Exchange Commission. These statements are based on Biogen's current
beliefs and expectations and speak only as of the date of this news
release. Biogen does not undertake any obligation to publicly
update any forward-looking statements, whether as a result of new
information, future developments or otherwise.
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 neurological and cardiometabolic
franchises. Our scientific innovation began and continues with the
knowledge that sick people depend on us, which fuels our vision of
becoming one of the most successful biotechnology companies.
To learn more about Ionis visit www.ionispharma.com and follow
us on twitter @ionispharma.
Ionis' Forward-looking Statement
This press release includes forward-looking statements regarding
Ionis' business and the therapeutic and commercial potential of
Ionis' technologies, IONIS-MAPTRx/BIIB080 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, 2020, 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.
In this press release, unless the context requires otherwise,
"Ionis," "Company," "we," "our," and "us" refers to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a trademark of Ionis
Pharmaceuticals, Inc.
BIIB080 is licensed to Biogen.
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SOURCE Ionis Pharmaceuticals, Inc.; Biogen Inc.