LA JOLLA, Calif., Aug. 24, 2021 /PRNewswire/ -- Avidity
Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company
committed to delivering a new class of RNA therapeutics called
Antibody Oligonucleotide Conjugates (AOCs™), announced that the
European Commission (EC) has granted orphan designation for AOC
1001. The U.S. Food and Drug
administration also granted orphan drug designation to AOC 1001
earlier this summer.
The U.S. Food and Drug Administration (FDA) cleared Avidity to
proceed with the Phase 1/2 MARINATM trial of AOC 1001 in
adults with myotonic dystrophy type 1 (DM1) in the United States.
"We are pleased that both the EC and the FDA have granted AOC
1001 Orphan Designation, further validating AOCs as a powerful new
class of drugs that can bring benefit to people suffering from
untreated and undertreated diseases," said Sarah Boyce, president and chief executive
officer of Avidity. "AOC 1001 is designed to address the root
cause of DM1 and has the potential to be a first-in-class treatment
for people living with this debilitating rare disease."
The EC grants orphan designation to drugs and biologics intended
for the treatment, diagnosis or prevention of rare,
life-threatening or chronically debilitating diseases or conditions
that impact fewer than 5 in 10,000 patients in the European Union.
Orphan designation allows companies certain benefits, including
reduced regulatory fees, clinical protocol assistance, research
grants and 10 years of market exclusivity following regulatory
approval.
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 MARINATM study of AOC 1001
in adults with DM1. FDA and EMA have granted Orphan Designation for
AOC 1001.
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 (AOCsTM). 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 MARINATM trial of AOC 1001 in adults with 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
myotonic dystrophy type 1 and the potential for AOC 1001 to be a
treatment for DM1, and whether the FDA or EMA will grant AOC 1001
years of market exclusivity if approved, or significant development
incentives, including reduced regulatory fees or clinical protocol
assistance, research grants. 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; 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.
Company and Investor Contact:
Kathleen Gallagher
(858) 401-7900
kath.gallagher@aviditybio.com
Media Contact:
Cherise
Adkins
cadkins@spectrumscience.com
(301) 267-4161
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SOURCE Avidity Biosciences, Inc.