AOC 1020 is Avidity's second siRNA antibody
oligonucleotide conjugate (AOC™) entering Phase 1/2 studies
Company now has three distinct rare
disease programs in clinical development - myotonic dystrophy type
1 (DM1), facioscapulohumeral muscular dystrophy (FSHD), and
Duchenne muscular dystrophy (DMD)
Volume 4 of virtual investor and analyst
series on Monday, October 3, 2022
at 10:00 a.m. ET
SAN
DIEGO, Sept. 29, 2022 /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 the
Phase 1/2 FORTITUDE™ clinical trial of AOC 1020 in adults with
facioscapulohumeral muscular dystrophy (FSHD). FSHD is a rare,
hereditary muscle-weakening condition marked by life-long,
progressive loss of muscle function and causes significant pain,
fatigue, and disability. AOC 1020 is the second muscle-targeting
small interfering RNA (siRNA) AOC from Avidity's pipeline to
advance into clinical development.
Earlier this week, Avidity announced that the U.S. Food and Drug
Administration (FDA) cleared the company's investigational new drug
(IND) applications of AOC 1020 for FSHD and AOC 1044 for the
treatment of Duchenne muscular dystrophy (DMD) mutations amenable
to exon 44 skipping (DMD44). The company has now advanced three
programs - DM1, FSHD and DMD44 – into clinical development in a
14-month period.
"Advancing AOC 1020 into the Phase 1/2 FORTITUDE study is a
significant milestone for the FSHD community and our proprietary
AOC platform. People living with FSHD have no approved treatments
and experience life-long, progressive loss of muscle function
leading to fatigue and disability," said Sarah Boyce, president and chief executive
officer of Avidity. "AOC 1020, our second siRNA AOC, is designed to
directly target the disease-causing gene, DUX4, with the goal of
treating the underlying biological cause of FSHD. We now have three
clinical programs in development for three distinct rare diseases
that have no approved therapies."
FSHD affects approximately 16,000-38,000 people in the
United States alone. It is an
autosomal dominant disease caused by the abnormal expression of the
gene DUX4 (double homeobox 4) that leads to skeletal muscle wasting
and progressive loss of muscle function, with symptoms often
beginning in adolescence and early adulthood.
"We are grateful for companies like Avidity that are working to
address a significant unmet need in the FSHD community," said
Mark A. Stone, chief executive
officer at FSHD Society. "Patients as well as their caregivers and
families live with the burden of this devastating disease every day
and are in desperate need of treatment options that can improve
quality of life. As FSHD is a progressive disease, the impact is
debilitating and often results in an inability to do everyday
activities like brushing teeth or getting dressed. There is a long
road ahead, but today marks an important step and gives hope to
everyone in our community impacted by FSHD."
In addition to AOC 1020 and AOC 1044, Avidity is developing AOC
1001 for the potential treatment of myotonic dystrophy type 1
(DM1). AOC 1001 is being evaluated in the Phase 1/2
MARINA™ trial and the MARINA open label-extension study
(MARINA-OLE™). Earlier this week, Avidity announced that the FDA
placed a partial clinical hold on new participant enrollment in the
Phase 1/2 MARINA trial. All current participants, whether they are
on AOC 1001 or placebo, may continue in their current dosing cohort
and roll over into the MARINA-OLE where they will receive AOC 1001
as planned.
Avidity remains on track to conduct a preliminary assessment of
safety, tolerability and key biomarkers in approximately half of
the study participants in the Phase 1/2 MARINA trial in the fourth
quarter of 2022.
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 68 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.
Video Webcast Information – Monday,
October 3, 2022
The company is hosting Volume 4 of
their virtual investor and analyst series on Monday, October 3, 2022 beginning at 10 a.m. ET / 7 a.m.
PT to further discuss the AOC 1020 clinical development
program. The event is a live video webcast and can be accessed here
or from the "Events and Presentations" page in the "Investors"
section of Avidity's website. A replay of the webcast will be
archived on Avidity's website following the event.
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, meaning a single copy of the disease-associated gene, DUX4
(double homeobox 4), is enough to cause the disease. The abnormal
expression of DUX4 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 now has three programs in clinical
development. The company's lead product candidate, AOC 1001, is
designed to treat people with myotonic dystrophy type 1 (DM1). AOC
1001 is currently in Phase 1/2 development with the ongoing MARINA™
trial and MARINA-OLE™ in adults with DM1. The next programs in the
company's advancing and expanding pipeline are AOC 1020, designed
to treat people living with FSHD currently in Phase 1/2 development
with the ongoing FORTITUDE™ trial, and AOC 1044, the lead of
three programs for the treatment of 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 initiation of clinical trials
of AOC 1020 and AOC 1044 in patients with FSHD and DMD,
respectively, and the timing thereof; AOC 1020's potential to
address unmet needs in FSHD and to treat the underlying biological
cause of FSHD; expectations for Avidity's interactions with the
FDA, the ongoing investigation into the underlying cause of the SAE
for the affected patient, and the anticipated impact of, and
Avidity's ability to resolve, the partial clinical hold and resume
enrollment in and complete the MARINA study, and to conduct and
present data from the preliminary assessment of the MARINA study
and the timing thereof; 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; Avidity
may not be able to resolve the partial clinical hold and the
analysis related to the underlying cause of the SAE may result in
delays in the MARINA study or an inability to compete the study;
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.