SAN
DIEGO, Feb. 29, 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 its
support for Rare Disease Day®, highlighting the
importance of bringing awareness to the devastating impact rare
diseases have on patients, families and caregivers worldwide.
"Today, on Rare Disease Day, we are proud to join the global
community in raising awareness for people living with rare
diseases," said Sarah Boyce,
president and chief executive officer at Avidity. "Recently, we had
the privilege of hosting individuals affected by rare muscle
diseases at Avidity. Their journeys, filled with challenges,
determination, and unwavering hope, exemplify the urgency of our
mission to profoundly improve people's lives by revolutionizing a
new class of targeted RNA therapeutics. At Avidity, we are
committed to listening, learning, and partnering with the patient
and advocacy community and look forward to our continued
collaboration as we advance our three rare muscle disease clinical
programs for DM1, DMD44 and FSHD."
Avidity has three distinct rare disease programs in clinical
development: AOC 1001 for the treatment of myotonic dystrophy type
1 (DM1) currently in the MARINA open-label extension (MARINA-OLE™)
trial and on-track to be studied in the global Phase 3 HARBOR™
trial planned to initiate mid-2024; AOC 1044 in the Phase 1/2
EXPLORE44™ trial for the treatment of Duchenne muscular dystrophy
(DMD) mutations amenable to exon 44 skipping (DMD44), and AOC 1020
in the Phase 1/2 FORTITUDE™ trial for the treatment of
facioscapulohumeral muscular dystrophy (FSHD).
"Millions of Americans live with rare diseases, yet a staggering
95% have no approved treatments," stated Annie Kennedy, Chief of Policy, Advocacy, and
Patient Engagement at EveryLife Foundation for Rare Diseases. "It's
essential that we highlight the importance of patient voices and
advocate for policies that drive research. This can lead to
breakthrough therapies that offer significant improvements for
patients and their families, bringing hope to the rare disease
community."
Rare Disease Day takes place on the last day of February each
year with the goal to raise awareness of the impact of rare
diseases worldwide. EURORDIS established Rare Disease Day in 2008
and coordinates with more than 70 national alliance patient
organizations each year to honor those living with rare diseases as
well as their families and caregivers.
About Myotonic Dystrophy Type 1 (DM1)
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: plans to initiate a
global Phase 3 trial for people living with DM1; plans for
the progression of Avidity's clinical programs for AOC 1001, AOC
1044 and AOC 1020, and the timing thereof; the potential of
Avidity's product candidates to treat rare diseases and Avidity's
efforts to bring them to people suffering from applicable diseases;
the potential of AOCs to target a range of different cells and
tissues beyond the liver; and Avidity's pipeline 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:
Avidity may not be able to resolve the partial clinical hold
related to the serious adverse event which occurred in the Phase
1/2 MARINA® trial, which may result in delays in the
clinical development of AOC 1001; additional data related to
Avidity's current clinical programs that continues to become
available may be inconsistent with the data produced as of the
respective data cutoff dates, further analysis of existing data and
analysis of new data may lead to conclusions different from those
established as of the date hereof, 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, or may result in clinical holds which may not be
timely lifted (if at all), recalls or product liability claims;
Avidity is early in its development efforts; Avidity's approach to
the discovery and development of product candidates based on its
AOC platform is unproven, and the company does not know whether it
will be able to develop any products of commercial value; potential
delays in the commencement, enrollment, data readouts and
completion of preclinical studies or clinical trials; the success
of its preclinical studies and clinical trials for the company's
product candidates; Avidity's dependence on third parties in
connection with preclinical and clinical testing and product
manufacturing; Avidity may not realize the expected benefits of its
collaborations; regulatory developments in the United
States and foreign countries; Avidity could exhaust its
available capital resources sooner than it currently expects and
fail to raise additional needed funds; and other risks described in
Avidity's Annual Report on Form 10-K for the fiscal year
ended December 31, 2023, filed with the Securities and
Exchange Commission (SEC) on February 28, 2024. 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:
Geoffrey
Grande, CFA
(619) 837-5014
investors@aviditybio.com
Media Contact:
Navjot Rai
(619) 837-5016
media@aviditybio.com
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SOURCE Avidity Biosciences, Inc.