CARLSBAD, Calif., April 5, 2021 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS) today announced the
initiation of a Phase 3 clinical trial of ION363 in patients with
amyotrophic lateral sclerosis (ALS) with mutations in the fused
in sarcoma gene (FUS). Patients with a mutation in the
FUS gene develop a rare form of ALS, referred to as FUS-ALS,
which is the most common cause of juvenile-onset ALS. There is
substantial evidence that mutations in the FUS gene are
responsible for a toxic gain of function that can lead to rapid,
progressive loss of motor neurons in patients with FUS-ALS. ION363
is an investigational antisense medicine targeting the FUS RNA to
reduce the production of the FUS protein. Antisense-mediated
reduction of mutant FUS protein in a FUS-ALS mouse model prevents
motor neuron loss. By targeting the root cause of FUS-ALS, ION363
has the potential to reduce or prevent disease progression in
FUS-ALS patients.
ALS is a rare, rapidly progressing and fatal neurodegenerative
disorder that affects approximately 55,000 people
globally.i FUS-ALS is the third most common genetic
cause of ALS. People with ALS experience muscle weakness, loss of
movement, and difficulty breathing and swallowing, resulting in a
severely declining quality of life and eventually death.
"There is an urgent need for novel treatments for all forms of
ALS, a devastating disease that affects far too many patients and
their families. Advancement of ION363 to a pivotal trial is the
latest example of the power of Ionis' antisense technology to
potentially target the root causes of neurological diseases," said
C. Frank Bennett, Ph.D., Ionis'
chief scientific officer and franchise leader for neurological
programs. "Driven by our experience in developing medicines for
motor neuron diseases such as ALS and spinal muscular atrophy and
our intimate connection to the ALS patient community, Ionis made
the decision to advance ION363 to the clinic and, ultimately, to
the market because we believe we are uniquely positioned to make it
available to patients living with FUS-ALS."
The trial will be led by Neil
Shneider, M.D., Ph.D., director of Columbia University's Eleanor and Lou Gehrig ALS
Center. Ionis began collaborating with Dr. Shneider when the
company learned of his efforts to develop a treatment for
Jaci Hermstad, an Iowa woman living with FUS-ALS. Ionis shared
its research and expertise with Dr. Shneider, resulting in an
experimental treatment designed specifically for Jaci. Inspired by
Jaci's spirit and courage, Ionis made the decision to invest in
clinical studies so that many more patients can gain access to
ION363, also known as "jacifusen." Since Jaci, several FUS-ALS
patients have received treatment with ION363 under Dr. Shneider's
investigator-initiated study through the U.S. Food and Drug
Administration's expanded access pathway, sometimes called
"compassionate use."
"FUS-ALS is an atypically aggressive form of the disease,
involving the youngest of ALS patients. Building on our expanded
access program, a controlled clinical trial is the best way to
demonstrate the efficacy of ION363 and to make this therapeutic
available to all patients who could potentially benefit from it,"
said Dr. Shneider.
The Phase 3 trial of ION363 is a global, multi-center study in
up to 64 patients. Part one of the trial will consist of patients
randomized to receive a multi-dose regimen of ION363 or placebo for
29 weeks, followed by part two, which will be an open-label period
in which all patients in the trial will receive ION363 for 73
weeks.
Learn more about the Phase 3 trial of ION363 at:
https://clinicaltrials.gov/ct2/show/NCT04768972?term=ion363&draw=2&rank=1
Ionis' other leading investigational medicines to treat ALS are
tofersen (BIIB067), IONIS-C9Rx (BIIB078) and ION541
(BIIB105).
About Ionis' Neurology Franchise
The Ionis neurology franchise addresses all major brain regions
and central nervous system types and currently has three Phase 3
studies ongoing with eight medicines in clinical development, five
of which are wholly owned. Ionis is leading the way in treating the
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®, the 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 Ionis Pharmaceuticals
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, Ionis' technologies, tofersen,
IONIS-C9Rx, ION541, ION363 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, which is on
file with the SEC. 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.
Tofersen, IONIS-C9Rx and ION541 are partnered with
Biogen.
i France,
Germany, Italy, Japan,
Spain, the United Kingdom and the United States.
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SOURCE Ionis Pharmaceuticals, Inc.