LA JOLLA, Calif., April 16, 2021 /PRNewswire/ -- Avidity
Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company
pioneering a new class of oligonucleotide-based therapies called
Antibody Oligonucleotide Conjugates (AOCs™), today
announced that preclinical data for their lead candidate, AOC 1001
in development for myotonic dystrophy type 1 (DM1), will be
presented at the American Academy of Neurology (AAN) 2021 Virtual
Annual Meeting being held April
17-22, 2021.
The abstract on AOC 1001 was chosen by the AAN Science Committee
as one of 11 abstracts to be highlighted in the Emerging Science
Session.
"We are honored to be part of the Emerging Science session at
such a prestigious meeting and are grateful to the AAN Science
Committee for highlighting AOC 1001. AOC 1001 is targeting DM1, a
complex disease with variability from patient to patient and no
approved treatments," said Art Levin, Ph.D., Chief Scientific
Officer. "Our data presented at AAN show that AOC 1001 is
highly potent with demonstrated activity in the nucleus and
cytoplasm and that single doses of AOC 1001 produced 75% reductions
in DMPK mRNA expression that were maintained for months
post-dosing. These results further solidify AOC 1001 as a promising
program with the potential to be the first therapy for people
living with DM1."
Details of the oral presentation are below:
Title: "Optimization of AOC-1001, an
antibody-oligonucleotide conjugate targeting the underlying cause
of myotonic dystrophy type 1"
Date: Sunday, April 18, 2021 at 3:45 p.m. ET
Session: Emerging Science
Presenter: Barbora Malecova, Ph.D., Associate Director,
Biology at Avidity
Avidity scientists have demonstrated activity and potency of
siRNAs against the dystrophy protein kinase (DMPK) gene in muscle
cells derived from patients with DM1. These data showed significant
reductions in DMPK mRNA levels in the nucleus and in the cytoplasm.
Treatment with Avidity's therapeutic agent were able to correct the
incorrect splicing in these DM1 cells, moving the splicing
signature closer to that of healthy cells and indicating the
potential to target the underlying cause of DM1. In addition,
Avidity scientists have shown the robust, durable activity of AOC
1001 in vivo in a broad range of muscles of non-human
primates including cardiac muscle. These data demonstrated a
75% reduction in DMPK mRNA levels in muscles following a single
dose of AOC 1001.
Avidity's abstract is available on the AAN meeting website.
Following regulatory clearance, the company plans to begin a Phase
1/2 study for AOC 1001 in the second half of 2021.
About Avidity
Avidity Biosciences, Inc. is driven to
change lives with a new class of therapies called Antibody
Oligonucleotide Conjugates (AOCs) that are designed to overcome
current limitations of oligonucleotide therapies in order to treat
a wide range of serious diseases. Avidity's proprietary AOC
platform combines the tissue selectivity of monoclonal antibodies
and the precision of oligonucleotide therapies to access previously
undruggable tissue and cell types and more effectively target
underlying genetic drivers of diseases. Avidity's lead product
candidate, AOC 1001, is designed to treat myotonic dystrophy type
1, and its other muscle programs are focused on the treatment of
Duchenne muscular dystrophy, facioscapulohumeral muscular
dystrophy, Pompe disease and muscle atrophy. In addition to its
muscle franchise, Avidity has research efforts focused on immune,
cardiac and other cell types.
Avidity is headquartered in La Jolla, CA. For more
information about Avidity's science, pipeline and people,
please visit www.aviditybiosciences.com and engage with
Avidity on LinkedIn.
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 current beliefs and expectations. Such
forward-looking statements include, but are not limited to,
statements regarding: Avidity's evolution to a clinical-stage
company; Avidity's plans to conduct clinical trials of AOC 1001 in
patients with DM1 and the expected timing thereof; 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: the company is early in
development efforts and all of its development programs are
currently in the preclinical or discovery stage; the company's
approach to the discovery and development of product candidates
based on the 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 the company's
operations from the COVID-19 pandemic; the success of its
preclinical studies and clinical trials for its product candidates;
the results of preclinical studies and early clinical trials are
not necessarily predictive of future results; its 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 the company's prior press
releases and in its 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:
Mike MacLean
(858) 401-7900
mikemaclean@aviditybio.com
Media and Investors:
Amy
Conrad
Juniper Point
(858) 366-3243
amy@juniper-point.com
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SOURCE Avidity Biosciences, Inc.