SAN DIEGO, Feb. 20, 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 Rare
Pediatric Disease designation to AOC 1044, the company's
investigational therapy for the treatment of Duchenne muscular
dystrophy (DMD) in people living with mutations amenable to exon 44
skipping (DMD44). AOC 1044 is being assessed in the Phase 1/2
EXPLORE44™ trial for people living with DMD44 and is the first of
multiple AOCs the company is developing for DMD. In addition to
receiving Rare Pediatric Disease Designation, AOC 1044 has been
granted Orphan Designation by the FDA and the European Medicines
Agency (EMA), and Fast Track Designation by the FDA.
DMD is a rare genetic condition that is characterized by
progressive muscle damage and weakness due to the loss of
dystrophin protein that typically starts at a very young age.
Currently, there are no therapies approved targeting exon 44.
"We are pleased that the FDA has granted Rare Pediatric Disease
designation to AOC 1044, adding to the Orphan Drug and Fast Track
designations already granted. The effects of DMD44 are devastating,
with symptoms often starting in childhood. These designations by
the FDA underscore the urgent need for innovative treatments and
validate the potential of AOC 1044 to address the unmet need of
people living with Duchenne muscular dystrophy," said Steve Hughes, M.D., chief medical officer at
Avidity. "We recently shared healthy volunteer data of AOC 1044
from our Phase 1/2 EXPLORE44 trial demonstrating unprecedented
delivery of therapeutic oligonucleotide in skeletal muscle and
consistent exon skipping in healthy volunteers, and we look forward
to sharing data from that study in people living with DMD44 later
this year. We remain steadfast in our commitment to advancing
science and improving the lives of people and their families
affected by this devastating condition."
In December 2023, Avidity reported
positive AOC 1044 data in healthy volunteers from the EXPLORE44
trial. AOC 1044 is designed to deliver phosphorodiamidate
morpholino oligomers (PMO) to skeletal muscle and heart tissue to
specifically skip exon 44 of the dystrophin gene to enable
dystrophin production. AOC 1044 delivered unprecedented
concentrations of PMO in skeletal muscle with up to 50-times
greater concentrations of PMO in skeletal muscle following a single
dose compared to peptide conjugated PMOs in healthy volunteers. AOC
1044 was well tolerated, demonstrated statistically significant
exon 44 skipping compared to placebo of up to 1.5% in healthy
volunteers after a single dose of 10 mg/kg AOC 1044 and increased
exon skipping in all participants. Avidity plans to provide a first
look at AOC 1044 data in people living with DMD44 in 2H 2024.
The FDA defines a "rare pediatric disease" as a serious or
life-threatening disease in which the serious or life-threatening
manifestations primarily affect individuals aged from birth to 18
years. Under the FDA's Rare Pediatric Disease Priority Review
Voucher program, a sponsor who receives an approval for a drug or
biologic for a rare pediatric disease may qualify for a voucher
that can be redeemed to receive a priority review of a subsequent
marketing application for a different product.
The EXPLORE44™ Phase 1/2 Trial of AOC 1044
The
EXPLORE44 trial is a randomized, placebo-controlled, double-blind,
Phase 1/2 clinical trial to evaluate AOC 1044 in healthy volunteers
and participants with DMD mutations amenable to exon 44 skipping
(DMD44). EXPLORE44 will evaluate the safety, tolerability,
pharmacokinetics, and pharmacodynamic effects of single and
multiple ascending doses of AOC 1044 administered intravenously.
EXPLORE44 is expected to enroll approximately 40 healthy volunteers
and 24 participants with DMD44, ages seven to 27 years old. The
EXPLORE44 trial will assess exon skipping and dystrophin protein
levels in participants with DMD44. Participants with DMD44 will
have the option to enroll into an extension study. For more
information about the EXPLORE44 trial, visit the EXPLORE44
study website or
visit http://www.clinicaltrials.gov and search for
NCT05670730.
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 AOC 1044
AOC 1044 is designed to deliver phosphorodiamidate morpholino
oligomers (PMOs) to skeletal muscle and heart tissue to
specifically skip exon 44 of the dystrophin gene to enable
dystrophin production in people living with Duchenne muscular
dystrophy with mutations amenable to exon 44 skipping (DMD44). DMD
is characterized by progressive muscle degeneration and weakness
due to alterations of a protein called dystrophin that protects
muscle cells from injury during contraction. AOC 1044 consists of a
proprietary monoclonal antibody that binds to the transferrin
receptor 1 (TfR1) conjugated with a PMO targeting exon 44. AOC 1044
is currently in Phase 1/2 development as part of the EXPLORE44™
trial for the treatment of DMD mutations amenable to exon 44
skipping. Data from the Phase 1/2 EXPLORE44 trial showed that AOC
1044 delivered unprecedented concentrations of PMO in skeletal
muscle with up to 50-times greater concentrations of PMO in
skeletal muscle following a single dose compared to peptide
conjugated PMOs in healthy volunteers. AOC 1044 was well tolerated,
demonstrated statistically significant exon 44 skipping compared to
placebo of up to 1.5% in healthy volunteers after a single dose of
10 mg/kg AOC 1044 and increased exon skipping in all
participants.
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 anticipated timing of release
of data from the EXPLORE44™ trial; the characterization of data
associated with AOC 1044 in healthy volunteers; the impact of
such data on the advancement of AOC 1044; the significance of
designations by the FDA; the additional AOCs Avidity is developing
for DMD; the design and goals of the EXPLORE44™ trial and the
dosages of AOC 1044 to be administered therein; the number and type
of participants to enroll in the EXPLORE44 trial; an extension
study for EXPLORE44 participants; expectations related to the
EXPLORE44 trial and AOC 1044; the potential of Avidity's product
candidates to treat rare diseases and Avidity's efforts to bring
them to people suffering from applicable diseases; and the
potential of AOCs to target a range of different cells and tissues
beyond the liver, and to treat cardiac and immunological
diseases.
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; 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; 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, and the success of such
studies and trials; 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, 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.