SAN
DIEGO, Jan. 18, 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 Fast Track designation to AOC 1020 for the treatment of
facioscapulohumeral muscular dystrophy (FSHD). FSHD is a serious,
rare, hereditary muscle-weakening condition marked by life-long,
progressive loss of muscle function that causes significant pain,
fatigue, and disability. AOC 1020 is being studied in the Phase 1/2
FORTITUDE™ clinical trial in adults with FSHD and is the
company's second muscle-targeting small interfering RNA (siRNA) AOC
in clinical development. Avidity plans to share data from a
preliminary assessment of AOC 1020 in approximately half of study
participants from the FORTITUDE trial in the first half of
2024.
Fast Track designation enables more frequent interactions with
the FDA to expedite the development and review process for drugs
intended to treat serious or life-threatening conditions and that
demonstrate the potential to address unmet medical needs.
Currently, there are no FDA-approved treatments for people living
with FSHD.
"The FDA Fast Track designation for AOC 1020 reinforces the
importance of finding an effective treatment to help people living
with FSHD, a devastating and debilitating muscular dystrophy
disorder with no treatment options," said Steve Hughes, M.D., chief medical officer at
Avidity. "AOC 1020 is designed to directly target the
disease-causing gene, DUX4, to address the underlying cause of
FSHD. We look forward to working collaboratively with the FDA to
bring the first RNA therapy directly targeting DUX4 to patients as
quickly 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 cause of diseases
previously untreatable with RNA therapeutics. AOC 1020 consists of
a proprietary mAb that binds to the transferrin receptor 1 (TfR1)
conjugated with a siRNA targeting double homeobox 4 (DUX4) mRNA.
The abnormal expression of DUX4 protein leads to changes in gene
expression in muscle cells that are associated with the life-long,
progressive loss of muscle function in patients with FSHD.
Avidity has three distinct rare disease programs in the clinic.
In addition to AOC 1020, the company is also evaluating AOC 1001 in
the Phase 1/2 MARINA™ and MARINA open-label extension
(MARINA-OLE™) clinical trials for the treatment of
myotonic dystrophy type 1 (DM1) and AOC 1044 in the Phase 1/2
EXPLORE44™ trial for the treatment of Duchenne muscular
dystrophy (DMD) mutations amenable to exon 44 skipping (DMD44).
The FORTITUDE™ Phase
1/2 Trial of AOC 1020 in Adults with FSHD
The FORTITUDE™ trial is a randomized,
placebo-controlled, double-blind, Phase 1/2 clinical trial designed
to evaluate AOC 1020 in approximately 70 adult participants
with FSHD. FORTITUDE will evaluate the safety, tolerability,
pharmacokinetics, and pharmacodynamics of AOC 1020 administered
intravenously, with the primary objective being the safety and
tolerability of AOC 1020 in FSHD patients. Activity of AOC 1020
will be assessed using key biomarkers, including magnetic resonance
imaging (MRI) measures of muscle volume and composition. Though the
Phase 1/2 trial is not statistically powered to assess functional
benefit, it will explore the clinical activity of AOC 1020
including measures of mobility and muscle strength as well as
patient reported outcomes and quality of life measures.
Participants will have the option to enroll in an open-label
extension study at the end of the treatment period in the FORTITUDE
study.
About Facioscapulohumeral Muscular
Dystrophy (FSHD)
Facioscapulohumeral muscular dystrophy (FSHD) is characterized
by progressive and often asymmetric skeletal muscle loss that
typically causes weakness initially in muscles in the face,
shoulders, arms and trunk and progresses to weakness in muscles in
the lower body. FSHD is an autosomal dominant genetic disease. The
abnormal expression of DUX4 (double homeobox 4) leads to a series
of downstream events that result in skeletal muscle wasting and
progressive loss of muscle function, 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 AOC 1020
AOC 1020 is designed to treat the underlying cause of FSHD,
which is caused by the abnormal expression of a gene called double
homeobox 4 or DUX4. The abnormal expression of DUX4 protein leads
to changes in gene expression in muscle cells that are associated
with the life-long, progressive loss of muscle function in patients
with FSHD. AOC 1020 aims to reduce the expression of DUX4 mRNA and
DUX4 protein in muscles in patients with FSHD. AOC 1020 consists of
a proprietary monoclonal antibody that binds to the transferrin
receptor 1 (TfR1) conjugated with a siRNA targeting DUX4 mRNA. In
preclinical studies, a single intravenous dose with the murine
version of AOC 1020 prevented development of muscle weakness
demonstrated by three functional assays - treadmill running, in
vivo force and compound muscle action potential. AOC 1020 is
currently in Phase 1/2 development as part of the
FORTITUDE™ trial in adults 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's
proprietary AOCs are designed to combine the specificity of
monoclonal antibodies with the precision of oligonucleotide
therapies to target the root cause of diseases previously
untreatable with RNA therapeutics. Avidity's advancing and
expanding pipeline has three programs in clinical development. AOC
1001 is designed to treat people with myotonic dystrophy type 1
(DM1) and is currently in Phase 1/2 development with the ongoing
MARINA™ and MARINA-OLE™ trials. AOC
1020 is designed to treat people living with facioscapulohumeral
muscular dystrophy (FSHD) and is currently in Phase 1/2 development
with the FORTITUDE™ trial. AOC 1044 is designed for
people with Duchenne muscular dystrophy (DMD) mutations amenable to
exon 44 skipping and is currently in Phase 1/2 development with the
EXPLORE44™ trial. AOC 1044 is the first of multiple
AOCs the company is developing for DMD. Avidity is also broadening
the reach of AOCs beyond muscle tissues through both internal
discovery efforts and key partnerships as the company continues to
deliver on the RNA revolution. Avidity is headquartered in San
Diego, CA. For more information about our science, 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 1020, and
AOC 1044 and the timing thereof; the potential of AOC 1020 to treat
people with FSHD, the enrollment of participants in the
FORTITUDE™ trial, the success of the ongoing FORTITUDE
trial and the reporting of data from the preliminary assessment of
the FORTITUDE study and the timing thereof; AOC 1020's
potential to address unmet needs in patients with FSHD and to treat
the underlying cause of FSHD; expectations for Avidity's
interactions with the FDA; 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 the
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; potential delays in the
commencement, enrollment and completion of clinical trials;
unexpected adverse side effects 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, 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 from the COVID-19
pandemic or the war in Ukraine;
and other risks described in prior press releases and in filings
with the Securities and Exchange Commission (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 exist 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:
Kathleen
Gallagher
(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.