LA JOLLA, Calif., Aug. 2, 2021 /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) cleared the
company to proceed with the Phase 1/2
MARINA™ clinical trial of AOC 1001 in
adults with myotonic dystrophy type 1 (DM1).
AOC 1001, Avidity's lead program utilizing its AOC platform, is
designed to address the root cause of DM1 by reducing levels of
DMPK, the disease-related mRNA. AOC 1001 consists of a proprietary
monoclonal antibody that binds to the transferrin receptor 1 (TfR1)
conjugated with a small interfering RNA (siRNA) that targets DMPK
mRNA.
"The FDA clearance to proceed with our Phase 1/2 clinical trial
for AOC 1001 is a significant milestone for Avidity as we move into
the clinic with our first AOC engineered to deliver to skeletal
muscle," said Sarah Boyce, president
and chief executive officer. "We are grateful to Dr. Johnson for
joining us on today's call to discuss the current treatment of
people living with DM1 and the important research that he and the
Myotonic Dystrophy Clinical Research Network are conducting to
further understand this progressive disease. We are continuing to
engage with the DM1 patient, advocate and physician community as we
actively work to get the trial up and running."
The MARINA™ Phase 1/2 Trial of AOC
1001 in Adults with DM1 and Recent Highlights
The MARINA trial is a randomized, double-blind,
placebo-controlled, Phase 1/2 clinical trial expected to enroll
approximately 44 adults with DM1. The primary objective of this
study is to evaluate the safety and tolerability of single and
multiple ascending doses of AOC 1001 administered intravenously.
The MARINA trial will assess the activity of AOC 1001 across key
biomarkers, including spliceopathy, a key biomarker for DM1, and
knockdown of DMPK mRNA, the disease-related mRNA responsible for
DM1. Though the Phase 1/2 trial is not powered to assess functional
benefit, it will explore the clinical activity of AOC 1001
including measures of mobility and muscle strength as well as
patient reported outcomes and quality of life measures. Patients
will have the option to enroll in an open label extension study at
the end of the post-treatment period. In the second half of 2022,
Avidity plans to conduct a preliminary assessment of safety,
tolerability and key biomarkers in approximately half of the study
participants.
Recently, the FDA also granted Orphan Drug Designation to AOC
1001 for the treatment of DM1. The FDA grants Orphan Drug
Designation to novel drugs that seek to treat a rare disease or
condition and, if the drug is approved for the designated orphan
indication, provides 7 years of market exclusivity, along with
certain financial incentives, including tax credits, opportunities
for grant funding towards clinical trial costs and FDA user-fee
waivers.
Today's Video Webcast Information
The company is hosting Volume 2 of their virtual investor and
analyst series today August 2, 2021
beginning at 8:30 am ET to further
discuss the AOC 1001 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.
The management team will be joined by Dr. Nicholas E. Johnson, MD, MSCI, FAAN. Dr. Johnson
is one of the principal investigators in END-DM1, an ongoing
natural history study being run by the Myotonic Dystrophy Clinical
Research Network (DMCRN) and will be the lead investigator in the
Phase 1/2 trial for AOC 1001. Dr. Johnson is an associate
professor, division chief of neuromuscular, and vice chair of
research in the department of neurology at Virginia Commonwealth University.
About Myotonic Dystrophy Type 1 and AOC 1001
Myotonic dystrophy type 1 (DM1) is an underrecognized,
progressive and often fatal disease caused by a triplet-repeat on
the DMPK gene, resulting in a toxic gain of function mRNA. The
disease is highly variable with respect to severity, presentation
and age of onset, however all forms of DM1, are associated with
high levels of disease burden and may cause premature mortality.
DM1 primarily affects skeletal and cardiac muscle, however patients
can suffer from a constellation of manifestations including
myotonia and muscle weakness, respiratory problems, fatigue,
hypersomnia, cardiac abnormalities, severe gastrointestinal
complications, and cognitive and behavioral impairment. Currently,
there are no treatments for patients living with DM1.
AOC 1001, Avidity's lead program utilizing its AOC platform, is
designed to address the root cause of DM1 by reducing levels of a
disease-related mRNA. AOC 1001 consists of a proprietary monoclonal
antibody that binds to the transferrin receptor 1 (TfR1) conjugated
with a siRNA targeting DMPK mRNA. In preclinical studies, AOC 1001
successfully delivered siRNAs to muscle cells, resulting in a
durable, dose-dependent reductions of DMPK RNA across a broad range
of muscles including skeletal, cardiac, and smooth muscles. In
preclinical studies, AOC 1001 had a favorable safety profile that
supports advancement into the clinic. The FDA has cleared Avidity
to proceed with the Phase 1/2 MARINA study of AOC 1001 in adults
with DM1. FDA has granted Orphan Drug Designation for AOC 1001 for
the treatment of DM1.
About Avidity Biosciences
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
lead product candidate, AOC 1001, is designed to treat myotonic
dystrophy type 1 (DM1). The FDA has cleared Avidity to proceed
with the Phase 1/2 MARINA™ trial of AOC
1001 in adults with myotonic dystrophy type 1 (DM1). Its advancing
and expanding pipeline also includes programs in
facioscapulohumeral muscular dystrophy (FSHD), Duchenne Muscular
Dystrophy (DMD), muscle atrophy and Pompe disease. The company is
planning for AOC 1044, the lead of three programs for the treatment
of DMD, and its AOC FSHD program to enter the clinic in 2022.
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 La Jolla, 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 a clinical trial of AOC 1001 in patients with
DM1; the potential benefits associated with Orphan Drug Designation
for approved drugs; plans for its other programs to enter the
clinic 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 and all of its development programs are in the
preclinical or discovery stage; 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;
disruption to its operations from the COVID-19 pandemic; risks that
the benefits associated with Orphan Drug Designation may not be
realized, including that Orphan Drug exclusivity may not
effectively protect a product from competition and that such
exclusivity may not be maintained; 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; unexpected adverse side effects or inadequate
efficacy of its product candidates that may limit their
development, regulatory approval and/or commercialization, or may
result in recalls or product liability claims; 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; risks related to integration of new management
personnel; 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.
Contacts:
Company:
Kathleen Gallagher
(858) 401-7900
kath.gallagher@aviditybio.com
Media and Investors:
Amy Conrad
Juniper Point
(858) 366-3243
amy@juniper-point.com
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SOURCE Avidity Biosciences, Inc.