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 joins with MDF community leaders,
patients, caregivers, and legislators in educational briefing to
highlight the latest scientific advancements in myotonic dystrophy
research and development
Company advancing clinical development
programs for three types of muscular dystrophy – DM1, DMD and FSHD
– with planned data readouts over the next 12 months
SAN
DIEGO, Sept. 7, 2023 /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 that
it is joining with patient communities to raise awareness during
National Muscular Dystrophy Awareness Month, an annual observance
that takes place every September to support families across the
U.S. who are impacted by neuromuscular diseases. Today, Avidity
will be joining Myotonic Dystrophy Foundation (MDF) community
leaders, patients, caregivers, and legislators to raise awareness
of myotonic dystrophy and the importance of advancing research to
develop new treatments for the disease as part of a special MDF
Advocacy Day on Capitol Hill in Washington, D.C. In addition, Avidity is
engaging with patient communities to support World Duchenne
Awareness Day, International Myotonic Dystrophy Awareness Day, FSHD
Society Walk & Roll to Cure FSHD, and Global Genes Week in
RARE, all taking place during National Muscular Dystrophy Awareness
Month.
"We are proud to be uniting with patient communities and their
families this month to raise awareness of muscular dystrophy.
Today, we are joining MDF as part of an educational briefing on
Capitol Hill and also, recognizing World Duchenne Awareness Day.
Later this month, we will be engaging with members of the
FSHD community and Global Genes," said Sarah Boyce, president and chief executive
officer at Avidity. "At Avidity, we are developing a new class of
treatments and advancing three distinct clinical development
programs for three types of muscular dystrophy – DM1, DMD and FSHD.
Many people living with these devastating muscle disorders have
limited or no treatment options. It is important that we work
together as a community to bring promising new treatments to
patients in need as quickly as possible."
Avidity is currently advancing three muscular dystrophy clinical
programs: AOC 1001 for the treatment of myotonic dystrophy type 1
(DM1), AOC 1044 for the treatment of Duchenne muscular dystrophy in
people with mutations amenable to exon 44 skipping (DMD44), and AOC
1020 for the treatment of facioscapulohumeral muscular dystrophy
(FSHD).
"National Muscular Dystrophy Awareness Month is an opportunity
for members of the myotonic dystrophy community to unite to help
patients and families affected by all types of muscular dystrophy,"
said Tanya Stevenson, EdD, MPH,
Chief Executive Officer at the Myotonic Dystrophy Foundation
(MDF). "Together with Avidity, MDF is proud to have the
opportunity to educate and engage with key members of Congress, and
representatives from the National Institutes of Health and the
Congressionally Directed Medical Research Program, to raise new
levels of awareness about myotonic dystrophy, educate about the
impact of the disease on the quality of life, and to provide
updates on the latest scientific developments." MDF is committed to
helping patients and families by supporting and connecting the
myotonic dystrophy community, providing resources and
advocating for care, and accelerating research toward
treatments and a cure.
In support of National Muscular Dystrophy Awareness Month,
Avidity will be engaging in various activities with patient and
advocacy communities, including:
- Joining leaders from the advocacy community and Congress to
discuss advances in myotonic dystrophy research in an
educational briefing on Capitol Hill organized by the MDF to be
held September 7 beginning at
12:30 p.m. ET in the 385 Senate
Russell Building in Washington,
D.C.
- Participating at the 2023 MDF Annual Conference, taking place
September 7-9, 2023.
- Supporting the Jett Foundation's Stronger than Duchenne World
Duchenne Awareness Day event, taking place on September 7, 2023.
- Recognizing International Myotonic Dystrophy Awareness Day on
September 15 as a proud member of the
Global Alliance for Myotonic Dystrophy Awareness.
- Participating at the 2023 Global Genes Week in RARE event
taking place September 18-21,
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 their 2023 Walk & Roll
to Cure FSHD in San Diego on
September 30 and additional locations
throughout the country, the only international annual event focused
solely on funding progress for FSHD.
Avidity's lead program, AOC 1001, is currently being studied in
the MARINA open-label extension (MARINA-OLE™) trial in adults
living with DM1. The company plans to share a first look at data
from the MARINA-OLE study in the first half of 2024, while in
parallel, planning for a Phase 3 study. Avidity is advancing AOC
1044 in the Phase 1/2 EXPLORE44™ trial in people living with DMD44
and plans to report data from healthy volunteers in the EXPLORE44
trial in the fourth quarter of 2023. The company is also advancing
AOC 1020 in the Phase 1/2 FORTITUDE™ trial in people living with
FSHD with data from a preliminary assessment in approximately half
of the participants in the FORTITUDE trial planned for the first
half of 2024.
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 Twitter.
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 anticipated timing of release of data from the MARINA-OLE™,
EXPLORE44™ and FORTITUDE™ trials; plans for a Phase 3 study for AOC
1001; plans for the progression of 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 to treat cardiac and immunological
diseases; the continued advancement of programs with collaboration
partners; and Avidity's plans to expand its AOC platform and to
invest in its 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, 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 participant data related to AOC 1001 that continues to
become available may be inconsistent with the data produced as of
the most recent date cutoff, and further analysis of existing data
and analysis of new data may lead to conclusions different from
those established as of such date cutoff; 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 additional clinical
holds which may not be timely lifted, 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, 2022, filed with the Securities and
Exchange Commission (SEC) on February 28, 2023, and in
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:
Geoff
Grande
(858) 401-7900
investors@aviditybio.com
Media Contact:
Navjot
Rai
(858) 401-7900
media@aviditybio.com
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SOURCE Avidity Biosciences, Inc.