- Phase 1b study showed
dose-dependent and sustained reduction of tau protein in
cerebrospinal fluid through open-label long-term extension
(LTE)
- IONIS-MAPTRx reduced aggregated tau pathology
across all brain composites assessed starting as early as week 25
through week 100
CARLSBAD, Calif., March 29,
2023 /PRNewswire/ -- Ionis Pharmaceuticals,
Inc. (Nasdaq: IONS) today reported that its partner Biogen
presented new Phase 1b clinical data
showing that IONIS-MAPTRx (BIIB080) reduced soluble tau
protein in cerebrospinal fluid (CSF) in a dose-dependent and
sustained manner in patients with early-stage Alzheimer's disease
(AD). IONIS-MAPTRx also reduced aggregated tau
pathology, as measured by positron emission tomography (PET) in all
brain composites assessed. The primary endpoint of the Phase
1b study (25 weeks) and open-label
long-term extension study (through week 100) was safety and
tolerability, with biomarker data as an exploratory endpoint. The
results were presented today at the International Conference on
Alzheimer's and Parkinson's Diseases (AD/PD™ 2023).
![Ionis logo with tagline (PRNewsfoto/Ionis Pharmaceuticals, Inc.) Ionis logo with tagline (PRNewsfoto/Ionis Pharmaceuticals, Inc.)](https://mma.prnewswire.com/media/1990217/IONIS_A_Genetic_Medicines_Company_Logo_v1.jpg)
In patients with AD, tau protein can form "tangles" that
progressively accumulate in brain regions involved in cognition.
The accumulation of pathological tau tangles has been shown to
promote neuronal damage and death. IONIS-MAPTRx is an
investigational antisense medicine discovered by Ionis that is
being developed by Biogen. It is designed to target
microtubule-associated protein tau (MAPT) mRNA and prevent
production of tau protein.
"This Phase 1b clinical study of
IONIS-MAPTRx is the first to demonstrate this magnitude
of a reduction of tau pathology across important brain
regions. These encouraging findings are the latest example of
the potential of the Ionis RNA-targeting platform to selectively
modulate proteins linked to CNS disease," said Eugene Schneider, M.D., executive vice president
and chief clinical development officer at Ionis. "Ionis is
pioneering potential therapies for serious neurological diseases
with high unmet need. In addition to Alzheimer's disease, our
clinical stage neurology programs include ATTR polyneuropathy, ALS,
Alexander disease, Parkinson's disease and Angelman syndrome."
The Phase 1b trial and its
open-label long-term extension (LTE) were designed to evaluate the
safety and tolerability of multiple dose levels of
IONIS-MAPTRx in patients with mild AD (n=46). In this
study, the majority of adverse events were mild or moderate in
severity, of which the most common were headache, back pain, and
post-lumbar puncture syndrome (PLPS). The results showed that
IONIS-MAPTRx reduced biomarkers of soluble tau in CSF
(t-tau and p-tau181) in a dose-dependent and sustained manner, with
all dose groups showing approximately a 60% reduction from baseline
CSF tau levels by the end of the LTE. IONIS-MAPTRx
impacted aggregated tau pathology as measured by PET as early as
week 25 and up to the end of the LTE at week 100, including in
patients who began on placebo and received IONIS-MAPTRx
treatment starting at week 25 in the LTE. By the end of the LTE,
IONIS-MAPTRx reduced tau pathology relative to baseline
in all dose groups across all brain composites assessed.
The Phase 2 CELIA study of IONIS-MAPTRx (NCT05399888)
is in progress and currently recruiting participants in the
U.S.
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 12 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' marketed
neurological disease medicines include SPINRAZA®, a
global foundation of care for spinal muscular atrophy (SMA),
commercialized by Biogen, and TEGSEDI®, for hereditary
transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been a leader in RNA-targeted
therapy, pioneering new markets and changing standards of care.
Ionis currently has three marketed medicines and a promising
late-stage pipeline highlighted by cardiovascular and neurological
franchises. Our scientific innovation began and continues with the
knowledge that sick people depend on us, which fuels our vision to
become the leader in genetic medicine, utilizing a multi-platform
approach to discover, develop and deliver life-transforming
therapies.
To learn more about Ionis visit www.ionispharma.com and follow
us on Twitter @ionispharma.
Ionis' Forward-looking Statements
This press release includes forward-looking statements regarding
Ionis' business and the therapeutic and commercial potential of
Ionis' technologies, IONIS-MAPTRx 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 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 Dec. 31, 2022, which is on file
with the Securities and Exchange Commission. Copies of this 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.
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SOURCE Ionis Pharmaceuticals, Inc.