Avidity initiating global Phase
3 HARBOR™ study for delpacibart etedesiran this
quarter
Delpacibart etedesiran data from
MARINA-OLE™ showed reversal of disease progression in multiple
functional measures in DM1 compared to END-DM1 natural history
data
SAN
DIEGO, May 8, 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
granted Breakthrough Therapy designation to delpacibart etedesiran
(AOC 1001), the company's lead clinical development program, for
the treatment of myotonic dystrophy type 1 (DM1). Delpacibart
etedesiran, abbreviated as del-desiran, is an investigational
treatment designed to address the root cause of DM1, an
underrecognized, progressive and often fatal neuromuscular disease
with no approved therapies.
"We are pleased that the FDA has granted Breakthrough Therapy
designation to del-desiran for myotonic dystrophy type 1,
underscoring the potential of del-desiran to be an effective
treatment and the urgency of bringing this treatment to people
living with DM1," said Sarah Boyce,
president and chief executive officer at Avidity. "Initiation is
underway for our global Phase 3 HARBOR™ study as we focus on
rapidly advancing del-desiran for people living with DM1, who
currently have no treatment options to address the underlying cause
of this devastating rare muscle disease."
Avidity is initiating the global pivotal HARBOR™ study of
del-desiran this quarter. The primary endpoint in the Phase 3
HARBOR trial is video hand opening time (vHOT) and key secondary
endpoints include muscle strength as measured by hand grip strength
and quantitative muscle testing (QMT) total score, and activities
of daily living as measured by DM1-Activ. Avidity recently reported
positive long-term MARINA-OLE™ data demonstrating reversal of
disease progression in adults living with DM1 across multiple
endpoints including vHOT, muscle strength and DM1-Activ when
compared to natural history data.
In addition to receiving FDA Breakthrough Therapy designation,
del-desiran has previously been granted Orphan Drug and Fast Track
designations by the FDA and Orphan designation by the European
Medicines Agency (EMA) for the treatment of DM1.
About the Phase 2 MARINA-OLE™ Study
MARINA-OLE™ is an open-label, multi-center trial designed to
evaluate the long-term safety and tolerability
of del-desiran (AOC 1001) in participants with DM1 who
were previously enrolled in the MARINA® Phase 1/2
trial. This trial will continue to evaluate the safety,
tolerability, PK, PD, and efficacy of del-desiran in
participants enrolled in the randomized, placebo-controlled, Phase
1/2 MARINA clinical trial. Participants enrolled in the MARINA-OLE
study receive quarterly doses of del-desiran regardless
of whether they received active treatment or placebo in the MARINA
study. The total duration of active treatment
with del-desiran in the MARINA-OLE study is approximately
24 months. Once patients have completed active treatment, there
will be a nine-month safety follow-up period. Avidity may extend
active treatment beyond 24 months at a future timepoint. For more
information on this study click here or
visit http://www.clinicaltrials.gov and search for
NCT05479981.
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. In preclinical
studies, del-desiran successfully delivered siRNAs to
muscle cells, resulting in durable, dose-dependent reductions of
DMPK RNA across a broad range of muscles including skeletal,
cardiac, and smooth muscles. Del-desiran is currently in
Phase 1/2 development with the completed
MARINA® trial and the ongoing
MARINA-OLE™ trial in adults with DM1. The U.S. Food and Drug
Administration (FDA) and European Medicines Agency (EMA) have
granted Orphan Designation for del-desiran and the FDA has
granted del-desiran Fast Track Designation.
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 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 implications of Breakthrough Therapy designation for the
advancement of del-desiran; the global pivotal Phase 3 HARBOR™
trial for del-desiran, the timing of its initiation and key
endpoints to be used therein; the MARINA-OLE™ study, its design,
goals, dosage amounts and timelines; the potential of Avidity's
product candidates, including del-desiran, 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; and Avidity's plans to expand
its AOC platform and to invest in its pipeline programs. This press
release also contains estimates and other statistical data made by
independent parties and by us. This data involves a number of
assumptions and limitations, and the reader is cautioned not to
give undue weight to such estimates.
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;
additional participant data related to del-desiran that
continues to become available may be inconsistent with the data
produced as of the date hereof, and further analysis of existing
data and analysis of new data may lead to conclusions different
from those established as of the date hereof; 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; Avidity's dependence on third parties in connection with
preclinical and clinical testing and product manufacturing;
regulatory developments in the United States and foreign
countries; 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, 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:
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.