SAN
DIEGO, Aug. 15, 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
the U.S. Food and Drug Administration (FDA) has granted Orphan Drug
designation to AOC 1044, the company's investigational therapy in
development for the treatment of Duchenne muscular dystrophy (DMD)
in people with mutations amenable to exon 44 skipping (DMD44). 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.
![(PRNewsfoto/Avidity Biosciences, Inc.) (PRNewsfoto/Avidity Biosciences, Inc.)](https://mma.prnewswire.com/media/974628/Avidity_Biosciences_Logo.jpg)
AOC 1044 is being assessed in the Phase 1/2 EXPLORE44™ clinical
trial for people living with DMD44 and is the first of multiple
AOCs in development at Avidity for the treatment of DMD. Avidity
plans to share results from the healthy volunteer portion of the
EXPLORE44 trial in the fourth quarter of 2023 and is now enrolling
participants living with DMD44 into the study. In April 2023, AOC 1044 received FDA Fast Track
designation for the treatment of DMD44.
"We are pleased that the FDA has granted both Orphan Drug and
Fast Track designation to AOC 1044, highlighting the importance of
advancing new treatments for people living with DMD," said
Steve Hughes, M.D., chief medical
officer at Avidity. "There are currently no treatment options that
target the underlying cause of DMD44. AOC 1044 is designed to
specifically skip exon 44 of the dystrophin gene to enable the
production of functional dystrophin protein. We look forward to
advancing AOC 1044 in clinical development and bringing this very
important treatment to patients as quickly and safely as
possible."
Avidity's proprietary AOCs are designed to combine the
specificity of monoclonal antibodies (mAbs) with the precision of
oligonucleotide therapies to target the root causes of diseases
previously untreatable with RNA therapeutics. In the case of DMD,
the disease is caused by a genetic mutation that prevents the body
from producing the dystrophin protein, which protects muscle cells
from injury during contraction. The lack of functional dystrophin
leads to stress and tears of muscle cell membranes, resulting in
muscle cell death, inflammation, and progressive loss of muscle
function. AOC 1044 is designed to deliver phosphorodiamidate
morpholino oligomers (PMOs) to skeletal muscle and heart tissue.
The PMOs circumvent the mutation by causing exon 44 of the
dystrophin gene to be skipped, which enables production of
functional dystrophin protein.
The FDA's Office of Orphan Products Development grants orphan
status to support the development of medicines for rare disorders
that affect fewer than 200,000 people in the U.S. Orphan Drug
designation provides certain benefits, including market exclusivity
upon regulatory approval, exemption of FDA application fees, and
tax credits for qualified clinical trials.
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.
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. In a
preclinical model of DMD, a murine active AOC produced durable exon
skipping and functional dystrophin protein in skeletal muscle and
heart tissue following a single intravenous dose. 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.
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 progression of
clinical programs for AOC 1001, AOC 1044, and AOC 1020 and the
timing thereof; the potential of AOC 1044 to treat people with
DMD44; the number and characteristics of participants enrolling in
the EXPLORE44™ trial and the timing thereof; the design, goals and
prospects for success of the ongoing EXPLORE44 trial; the prospect
of a related extension study and the ability of EXPLORE44
participants to enroll therein; the reporting of data from healthy
volunteers in the EXPLORE44 study and the timing thereof; AOC
1044's potential to address unmet needs in patients with DMD44 and
to treat the underlying cause of DMD44; expectations for Avidity's
interactions with the FDA; the implications of Orphan Drug
designation; Avidity's development of multiple AOCs™ to treat DMD;
and the potential to broaden the reach of AOCs beyond skeletal
muscle tissues. 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 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; Avidity's
ability to resolve the partial clinical hold related to the Phase
1/2 MARINA™ trial of AOC 1001; potential delays in the
commencement, enrollment and completion of clinical trials;
unexpected adverse side effects to, or inadequate efficacy of, its
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, recalls or product
liability claims; the success of its preclinical studies and
clinical trials for the company's product candidates; the results
of preclinical studies and early clinical trials are not
necessarily predictive of future results; Avidity's dependence on
third parties in connection with preclinical testing and product
manufacturing; regulatory developments in the United States and foreign countries,
including acceptance of INDs and similar foreign regulatory filings
and the proposed design of future clinical trials; disruption to
its operations, including the ability to enroll eligible
participants in its clinical trials, from the COVID-19 pandemic or
the war in Ukraine; Avidity could
exhaust its available capital resources sooner than it currently
expects and be unable 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:
Mike
MacLean
(858) 401-7900 x550
investors@aviditybio.com
Media Contact:
Navjot
Rai
(858) 401-7900 x550
media@aviditybio.com
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SOURCE Avidity Biosciences, Inc.