LA JOLLA, Calif., Oct. 18, 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) has granted Fast Track
Designation to its lead program, AOC 1001, for the treatment of
myotonic dystrophy type 1 (DM1).
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.
"DM1 is an underrecognized, progressive and often fatal disease
with no therapeutic options. Fast Track designation for AOC 1001
underscores this unmet need and allows us to expeditiously work
with FDA to potentially deliver this first-in-class therapy to
people living with DM1 as quickly as possible," said Sarah Boyce, president and CEO of Avidity.
About AOC 1001 and the Phase 1/2
MARINA™ Trial
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 called DMPK. 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 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 and the FDA has
granted AOC 1001 Fast Track Designation.
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. 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. For more information on this study click
here or visit http://www.clinicaltrials.gov and search for
NCT05027269.
About Myotonic Dystrophy Type 1
Myotonic dystrophy type 1 (DM1) is an underrecognized,
progressive and often fatal disease caused by a triplet-repeat in
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.
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 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 our
current beliefs and expectations. Such forward-looking statements
include, but are not limited to, statements regarding: the
potential of AOC 1001 in people with myotonic dystrophy type 1 and
the initiation of a clinical trial, and the timing thereof. The
inclusion of forward-looking statements should not be regarded as a
representation by Avidity that any of our plans will be achieved.
Actual results may differ from those set forth in this press
release due to the risks and uncertainties inherent in our
business, including, without limitation: we are early in our
development efforts and many of our development programs are in the
preclinical or discovery stage; our approach to the discovery and
development of product candidates based on our AOC platform is
unproven, and we do not know whether we will be able to develop any
products of commercial value; potential delays in the commencement,
enrollment and completion of clinical trials; disruption to
our operations from the COVID-19 pandemic; the success of our
preclinical studies and clinical trials for our product candidates;
the results of preclinical studies and early clinical trials are
not necessarily predictive of future results; our dependence on
third parties in connection with preclinical testing and product
manufacturing; unexpected adverse side effects or inadequate
efficacy of our 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 our proposed design of future
clinical trials; risks related to integration of new
personnel; and other risks described in our prior press releases
and in our 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 we undertake 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:
Cherise
Adkins
(301) 267-4161
cadkins@spectrumscience.com
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SOURCE Avidity Biosciences, Inc.