SAN
DIEGO, Oct. 3, 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 that
the U.S. Food and Drug Administration (FDA) has removed the partial
clinical hold on delpacibart etedesiran (del-desiran/AOC 1001), an
investigational treatment designed to address the root cause of
myotonic dystrophy type 1 (DM1).
Del-desiran is being evaluated in the ongoing Phase 3
HARBOR™ trial in patients with DM1, an underrecognized,
progressive and often fatal neuromuscular disease with no approved
therapies.
Del-desiran has received Breakthrough Therapy, Orphan Drug and
Fast Track designations by the FDA and Orphan designation by the
European Medicines Agency (EMA).
About Del-desiran (AOC 1001)
Del-desiran (AOC 1001), Avidity's lead product candidate
utilizing its AOC platform, is designed to address the root cause
of DM1 by reducing levels of a disease-related mRNA called DMPK.
Del-desiran consists of a proprietary monoclonal antibody that
binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA
targeting DMPK mRNA. Del-desiran is currently being assessed in the
global Phase 3 HARBOR™ trial and in the ongoing MARINA-OLE™ trial
in people with DM1. Long-term data from the MARINA-OLE trial showed
reversal of disease progression in people living with DM1 across
multiple endpoints including video hand opening time (vHOT) as a
measure of hand function and myotonia, muscle strength and
activities of daily living when compared to END-DM1 natural history
data. Del-desiran has received Breakthrough Therapy, Orphan Drug
and Fast Track designations by the U.S. Food and Drug
Administration (FDA) and Orphan designation by the European
Medicines Agency (EMA).
About Myotonic Dystrophy Type 1
Myotonic dystrophy
type 1 (DM1) is an underrecognized, autosomal dominantly
inherited, 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 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 advancement of
del-desiran™ and the timing thereof; the characterization of
data associated with del-desiran™, and the impact of such data on
its advancement; the design, goals, timelines and expectations
related to the global Phase 3 HARBOR™ trial of del-desiran; 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; the FDA lifting the partial clinical hold
related to del-desiran does not foreclose the possibility of
additional issues of safety 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.