LA JOLLA, Calif., Nov. 4, 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 first participants in the Phase 1/2 MARINA trial have been
dosed with Avidity's lead AOC product candidate, AOC 1001, marking
the first time a person has been dosed with an AOC.
"This is a first for this new class of drugs and it is a
significant milestone for the DM1 community, the Avidity team and
the RNA field," said Sarah Boyce,
president and CEO of Avidity. "AOCs have the potential to expand
the possibilities of how we can treat diseases and our goal is to
deliver meaningful drugs to patients as quickly as possible. AOC
1001's MARINA trial will offer a first glimpse of proof-of-concept
data for the AOC platform to better inform the development path for
DM1 as well as future treatments for other diseases with limited
therapeutic options."
AOCs are designed to combine the proven technology of monoclonal
antibodies with the precision and potency of oligonucleotide
therapies to access previously untreatable tissue and cell types.
AOC 1001 consists of a proprietary monoclonal antibody that binds
to the transferrin receptor 1 (TfR1) conjugated with a small
interfering RNA (siRNA). It is designed to address the root cause
of DM1 by targeting DMPK, the disease-related mRNA.
Tanya Stevenson, EdD, MPH, CEO of
the Myotonic Dystrophy Foundation in the U.S. stated, "This
milestone represents years of research to advance treatment for
DM1. Opportunities for the myotonic dystrophy community to
participate in research, like the MARINA trial, are critical
because the knowledge gained may help lead to earlier diagnosis,
improved quality of life and, ultimately, a treatment. The MARINA
trial offers much needed advancement in the treatment of DM1 and
may also advance therapies for other repeat expansion diseases. We
are more hopeful, encouraged and excited than ever before."
"The AOC platform was developed entirely in-house at Avidity.
Our team has created this new technology through years of
engineering and following the data to optimize each component of
our AOCs," said Art Levin,
Ph.D., Avidity's CSO. "This is a turning point for Avidity and the
RNA field as AOCs target other cells and tissues that were
previously unreachable. Avidity's AOCs are designed to expand
the reach of oligonucleotide therapeutics to treat a broader range
of diseases."
The AOC 1001 Phase 1/2 MARINA trial is enrolling adults with
DM1. The first doses in the MARINA trial were administered to
patients at Virginia Commonwealth
University and University of
Rochester Medical Center (NY). For more information on the
MARINA trial, including a full list of participating sites, visit
www.clinicaltrials.gov.
About AOC 1001 and the Phase 1/2 MARINATM
Trial
AOC 1001, Avidity's lead product candidate 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. AOC 1001 has commenced
clinical testing with the ongoing Phase 1/2 MARINATM
trial in adults with DM1. The U.S. Food and Drug Administration
(FDA) and European Medicines Agency (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 begin to assess the activity of AOC 1001
across key biomarkers, including spliceopathy, an important
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
(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 patients with
myotonic dystrophy type 1 (DM1). AOC 1001 has commenced clinical
testing with the ongoing Phase 1/2 MARINATM trial in
adults with DM1. It's 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 timing of commencing clinical
trials and generating clinical trial data; the potential for the
MARINATM study to inform the development path for DM1 as
well as future treatments for other diseases; the potential of AOC
1001 to treat patients with DM1; and the broad potential of AOCs to
treat rare and serious diseases. 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; 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; 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 &
Investor Contact:
Kath Gallagher
kath.gallagher@aviditybio.com
858-401-7900 ext 550
|
Media
Contact: Cherise
Adkins cadkins@spectrumscience.com (301)
267-4161
|
View original content to download
multimedia:https://www.prnewswire.com/news-releases/avidity-announces-first-person-dosed-with-an-antibody-oligonucleotide-conjugate-aoc-301415970.html
SOURCE Avidity Biosciences, Inc.