Avidity supports World Duchenne Awareness Day,
International Myotonic Dystrophy Awareness Day, FSHD Society Walk
& Roll to Cure FSHD and Global Genes Week in RARE
Avidity reported groundbreaking data in all
three clinical programs addressing rare muscular dystrophies:
myotonic dystrophy type 1 (DM1), facioscapulohumeral muscular
dystrophy (FSHD) and Duchenne muscular dystrophy amenable to
exon 44 skipping (DMD44)
SAN
DIEGO, Sept. 5, 2024 /PRNewswire/ -- Avidity
Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company
committed to delivering a new class of RNA therapeutics called
Antibody Oligonucleotide Conjugates (AOCs™), today announced it is
joining with patient and advocacy communities to raise awareness
during National Muscular Dystrophy Awareness Month, an annual
observance that takes place every September to support families who
are impacted by neuromuscular diseases. During the month, Avidity
is engaging with patient communities to support muscular dystrophy
initiatives, including World Duchenne Awareness Day, International
Myotonic Dystrophy Awareness Day, FSHD Society Walk & Roll to
Cure FSHD, and Global Genes Week in RARE to build broader awareness
of muscular dystrophies.
"We are proud to stand alongside patient communities and their
families this Muscular Dystrophy Awareness Month, raising awareness
for those impacted by debilitating muscle disorders, who are facing
limited or no treatment options," said Sarah Boyce, president and chief executive
officer at Avidity. "This year, we reported unprecedented data
across our muscular dystrophy programs - DM1, FSHD and DMD44 -
and advanced our DM1 program into a global Phase 3 study. We are
deeply grateful to the muscular dystrophy community for their
ongoing support as we work together to advance these programs as
expeditiously as possible. At Avidity, we are committed to
profoundly improving people's lives by revolutionizing a new class
of targeted RNA therapeutics."
Avidity has demonstrated groundbreaking data across all three
rare neuromuscular disease clinical development programs:
del-desiran™ for myotonic dystrophy type 1 (DM1),
del-brax™ for facioscapulohumeral muscular dystrophy
(FSHD) and del-zota™ for Duchenne muscular dystrophy
amenable to exon 44 skipping (DMD44). The company also initiated
the global Phase 3 HARBOR™ study for DM1 and is advancing
additional candidates from their DMD franchise following their
positive del-zota™ data. Avidity is working to accelerate the
development of these important potential therapies to address the
unmet need of people living with these serious rare neuromuscular
diseases.
In support of National Muscular Dystrophy Awareness Month,
Avidity is engaging with several leading advocacy groups and
patient communities in a range of activities, including:
- Supporting the 2024 MDF Gala, taking place Saturday, September 14 in Los Angeles;
- Participating in the Jett Foundation's Stronger than Duchenne
World Duchenne Awareness Day celebration, including virtual and
in-person events in the Boston
area on September 7;
- Recognizing International Myotonic Dystrophy Awareness Day on
September 15 as a proud member of the
Global Alliance for Myotonic Dystrophy Awareness;
- Supporting the 2024 Global Genes Week in RARE event taking
place September 25-28 in Kansas City, MO, including the RARE Health
Equity Forum and RARE Advocacy Summit, one of the world's largest
gatherings of rare disease patients, caregivers, advocates and
healthcare professionals;
- Partnering with the FSHD Society for its 2024 Walk & Roll
to Cure FSHD in San Diego on
September 21 and additional locations
across the U.S., the only international annual event focused solely
on funding research progress for FSHD; and
- Presenting at the Defeat Duchenne Canada 2024 Family Forum
taking place September 21 in
Ontario, an annual event where
families and caregivers gather to learn more about the latest
advancements in research, clinical trials, and advocacy
initiatives.
About Myotonic Dystrophy Type 1
Myotonic dystrophy
type 1 (DM1) is an underrecognized, progressive and often fatal
disease caused by a triplet-repeat in the DMPK gene, resulting in a
toxic gain of function mRNA. The disease is highly variable with
respect to severity, presentation and age of onset, however all
forms of DM1 are associated with high levels of disease burden and
may cause premature mortality. DM1 primarily affects skeletal and
cardiac muscle, however patients can suffer from a constellation of
manifestations including myotonia and muscle weakness, respiratory
problems, fatigue, hypersomnia, cardiac abnormalities, severe
gastrointestinal complications, and cognitive and behavioral
impairment. Currently, there are no approved treatments for people
living with DM1.
About Duchenne muscular dystrophy (DMD)
Duchenne muscular dystrophy (DMD) causes a lack of functional
dystrophin that leads to stress and tears of muscle cell membranes,
resulting in muscle cell death and the progressive loss of muscle
function. The dystrophin protein maintains the integrity of muscle
fibers and acts as a shock absorber through its role as the
foundation of a group of proteins that connects the inner and outer
elements of muscle cells. People living with DMD suffer from
progressive muscle weakness that typically starts at a very young
age. Over time, people with Duchenne will develop problems walking
and breathing, and eventually, the heart and respiratory muscles
will stop working. Those living with the condition often require
special aid and assistance throughout their lives and have
significantly shortened life expectancy. While there are treatments
approved to treat people with DMD, there remains a very high unmet
need. DMD is a monogenic, X-linked, recessive disease that
primarily affects males, with one in 3,500 to 5,000 boys born
worldwide having Duchenne.
About Facioscapulohumeral Muscular Dystrophy
(FSHD)
Facioscapulohumeral muscular dystrophy (FSHD) is a
rare, progressive, and variable hereditary muscle-weakening
condition marked by significant pain, fatigue, and disability. It
is characterized by progressive and often asymmetric skeletal
muscle loss that initially causes weakness in muscles in the face,
shoulders, arms and trunk and progresses to weakness in muscles in
the lower body. FSHD is an autosomal dominant disease caused by the
aberrant expression of the DUX4 (double homeobox 4) gene in the
skeletal muscle, which activates genes that are toxic to muscle
cells and leads to a series of downstream events that result in
skeletal muscle wasting and compromised muscle function. Skeletal
muscle weakness results in physical limitations throughout the
whole body, including an inability to lift arms for more than a few
seconds, loss of ability to show facial expressions and serious
speech impediments. These symptoms cause many people affected by
FSHD to become dependent on the use of a wheelchair for mobility.
Currently, there are no approved treatments for people living with
FSHD.
About Avidity
Avidity Biosciences, Inc.'s mission is to profoundly improve
people's lives by delivering a new class of RNA therapeutics -
Antibody Oligonucleotide Conjugates (AOCs™). Avidity is
revolutionizing the field of RNA with its proprietary AOCs, which
are designed to combine the specificity of monoclonal antibodies
with the precision of oligonucleotide therapies to address targets
and diseases previously unreachable with existing RNA therapies.
Utilizing its proprietary AOC platform, Avidity demonstrated the
first-ever successful targeted delivery of RNA into muscle and is
leading the field with clinical development programs for three rare
muscle diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular
dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD).
Avidity is broadening the reach of AOCs with its advancing and
expanding pipeline including programs in cardiology and immunology
through internal discovery efforts and key partnerships. Avidity is
headquartered in San Diego, CA. For more information about our AOC
platform, clinical development pipeline and people, please
visit www.aviditybiosciences.com and engage with us
on LinkedIn and X.
Forward-Looking Statements
Avidity cautions readers
that statements contained in this press release regarding matters
that are not historical facts are forward-looking statements. These
statements are based on the company's current beliefs and
expectations. Such forward-looking statements include, but are not
limited to, statements regarding: the characterization of data
associated with del-desiran™, del-brax™ and del-zota™ within their
respective clinical studies, and the impact of such data on the
advancement of the respective product candidates; Avidity's plans
and expectations to advance its clinical programs, and the timing
thereof; and Avidity's platform, planned operations and
programs.
The inclusion of forward-looking statements
should not be regarded as a representation by Avidity that any of
these plans will be achieved. Actual results may differ from those
set forth in this press release due to the risks and uncertainties
inherent in Avidity's business and beyond its control, including,
without limitation: preliminary results of a clinical trial are not
necessarily indicative of final results; further analysis of
existing clinical data and analysis of new data may lead to
conclusions different from those established as of the respective
data cutoff dates in Avidity's clinical trials, and such data may
not meet Avidity's expectations; unexpected adverse side effects
to, or inadequate efficacy of, Avidity's product candidates that
may delay or limit their development, regulatory approval and/or
commercialization; Avidity may not be able to resolve the partial
clinical hold related to del-desiran™; later developments with the
FDA and other global regulators that could be inconsistent with the
feedback received to date regarding Avidity's clinical trials;
Avidity's approach to the discovery and development of product
candidates based on its AOC™ platform is unproven; potential delays
in the commencement, enrollment, data readouts and completion of
preclinical studies or clinical trials; Avidity's dependence on
third parties in connection with preclinical and clinical testing
and product manufacturing; legislative, judicial and regulatory
developments in the United States
and foreign countries; Avidity could exhaust its available capital
resources sooner than it currently expects; and other risks
described in Avidity's Annual Report on Form 10-K for the fiscal
year ended December 31, 2023 and
subsequent filings with the SEC. Avidity cautions readers not to
place undue reliance on these forward-looking statements, which
speak only as of the date hereof, and the company undertakes no
obligation to update such statements to reflect events that occur
or circumstances that arise after the date hereof. All
forward-looking statements are qualified in their entirety by this
cautionary statement, which is made under the safe harbor
provisions of the Private Securities Litigation Reform Act of
1995.
Investor Contact:
Mike MacLean
(619) 837-5014
investors@aviditybio.com
Media Contact:
Navjot Rai
(619) 837-5016
media@aviditybio.com
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SOURCE Avidity Biosciences, Inc.
