Avidity is currently enrolling participants in
the Phase 1/2 FORTITUDE™ study of AOC 1020 for the
treatment of FSHD
Company anticipates data from a preliminary
assessment in half of the participants in the FORTITUDE study in 1H
2024
SAN
DIEGO, June 20, 2023 /PRNewswire/ -- Avidity
Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company
committed to delivering a new class of RNA therapeutics called
Antibody Oligonucleotide Conjugates (AOCs™), joins with the global
community of patients, caregivers, and healthcare providers in
support of World Facioscapulohumeral Muscular Dystrophy (FSHD) Day.
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. Currently, there
are no approved therapies for the treatment of FSHD.
![(PRNewsfoto/Avidity Biosciences, Inc.) (PRNewsfoto/Avidity Biosciences, Inc.)](https://mma.prnewswire.com/media/974628/Avidity_Biosciences_Logo.jpg)
FSHD is an autosomal dominant disease caused by the aberrant
expression of the DUX4 (double homeobox 4) gene in the skeletal
muscle, which activates genes that are toxic to muscle cells and
leads to a series of downstream events that result in skeletal
muscle wasting and compromised muscle function. FSHD affects both
sexes equally, with onset typically in teenage and young adult
years.
"We are grateful for our partnership with the global FSHD
community as we collectively work together to advance AOC 1020 for
the treatment of FSHD," said Sarah
Boyce, president and chief executive officer at Avidity
Biosciences. "By directly targeting DUX4, AOC 1020 is designed to
reduce the devastating effects of skeletal muscle wasting and
progressive loss of muscle function in people living with FSHD. We
are currently enrolling adults with FSHD into the AOC 1020 Phase
1/2 FORTITUDE™ study. We plan to share a preliminary assessment
from the FORTITUDE study in the first half of next year."
Avidity is currently enrolling participants in FORTITUDE, an
ongoing randomized, placebo-controlled, double-blind, Phase 1/2
clinical trial to evaluate the investigational therapy AOC 1020 in
the treatment of FSHD. For more information about the FORTITUDE
trial, visit the FORTITUDE study website or visit
http://www.clinicaltrials.gov and search for NCT05747924.
"As the world's largest research-focused patient organization
for FSHD, our mission is to find treatments and a cure for FSHD
while empowering our families affected with FSHD, one of the most
prevalent forms of muscular dystrophy," said Mark Stone, chief executive officer of FSHD
Society. "We have set an extraordinary goal to ensure that the
first-ever FSHD therapy would be approved by 2025. This can only be
accomplished by the FSHD Community – patients & families,
industry, and the medical community – resourcing and working
together towards this goal. We are proud to partner with companies
like Avidity who are developing much needed treatments with the aim
to provide hope to families affected by FSHD."
Every June 20th, people around the world join in activities
to raise awareness for FSHD through World FSHD Day and to recognize
patients and families around the world who are affected by FSHD. At
the 30th Annual FSHD Society International Research Congress (FSHD
IRC) held in Milan, Italy, Avidity
presented oral and poster presentations on AOC 1020 preclinical
data, FORTITUDE study design and Health Economics and Economics
Outreach (HEOR) data demonstrating disease burden among people
living with FSHD. The meeting is organized by the FSHD Society, the
world's largest research-focused patient organization for FSHD. In
addition, Avidity participated in a panel discussion at the 2023
World FSHD Alliance Leadership Summit. The World FSHD Alliance is a
network of patient advocacy groups from more than 25 countries
around the world. Avidity also serves on the Global Task Force of
Project Mercury, an open collaboration spearheaded by the FSHD
Society to bring stakeholders from across the globe together
to overcome the challenges that could slow or
prevent effective therapies from getting to patients
everywhere.
About the Phase 1/2 FORTITUDE trial
The FORTITUDE
trial is a randomized, placebo-controlled, double-blind, Phase 1/2
clinical trial designed to evaluate single and multiple doses of
AOC 1020 in approximately 70 adult participants with
facioscapulohumeral muscular dystrophy (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. For more information about the
FORTITUDE trial, visit the FORTITUDE study website or visit
http://www.clinicaltrials.gov and search for NCT05747924.
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
people 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.
The U.S. Food and Drug Administration (FDA) and the European
Medicines Agency (EMA) have granted Orphan designation for AOC 1020
and the FDA has granted AOC 1020 Fast Track designation.
About Facioscapulohumeral Muscular Dystrophy
(FSHD)
Facioscapulohumeral muscular dystrophy (FSHD) is
characterized by progressive and often asymmetric skeletal muscle
loss that initially causes weakness in muscles in the face,
shoulders, arms and trunk and progresses to weakness in muscles in
the lower body. FSHD is an autosomal dominant disease caused by the
aberrant expression of the DUX4 (double homeobox 4) gene in the
skeletal muscle, which activates genes that are toxic to muscle
cells and leads to a series of downstream events that result in
skeletal muscle wasting and compromised muscle function. Skeletal
muscle weakness results in physical limitations throughout the
whole body, 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 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 the FORTITUDE™
trial 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 FORTITUDE trial and the reporting of data
from the preliminary assessment of the FORTITUDE trial and the
timing thereof; goals and expectations related to the FORTITUDE
trial; biomarker utilization and other methodologies related to the
FORTITUDE trial; the design and expected impact of AOC 1020; and
AOC 1020's potential to address unmet needs in patients with FSHD.
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 clinical
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; Avidity could exhaust its available capital resources
sooner than it currently expects and fail to raise additional
needed capital; and other risks described in our Annual Report on
Form 10-K for the 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:
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.