Avidity expands its leading RNA
delivery technology into precision cardiology with two new
wholly-owned development candidates targeting rare genetic
cardiomyopathies: AOC 1086 to treat PLN Cardiomyopathy and AOC 1072
to treat PRKAG2 Syndrome
AOC 1072 and AOC 1086 preclinical data
demonstrated robust siRNA delivery to the heart and targeted
knockdown with potent reduction of approximately 80% in cardiac PLN
mRNA and PRKAG2 mRNA
Avidity introduces next-generation technology
innovations demonstrating improved siRNA delivery in skeletal
muscle and increased durability in preclinical studies
AOC 1072 preclinical data will be presented at
American Heart Association (AHA) Scientific Sessions 2024
Volume 11 of virtual investor and analyst
series today, Tuesday, Nov. 12 at
8:00 a.m. ET
SAN
DIEGO, Nov. 12, 2024 /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 it has
expanded beyond rare skeletal muscle disorders and is opening up a
new therapeutic field, precision cardiology, to address the root
cause of genetic diseases of the heart. Avidity is advancing its
first two new wholly-owned precision cardiology development
candidates targeting rare genetic cardiomyopathies: AOC 1086
targeting PLN (phospholamban) cardiomyopathy and AOC 1072 targeting
PRKAG2 (Protein Kinase AMP-activated non-catalytic subunit Gamma 2)
Syndrome. In addition, Avidity shared preclinical data from
next-generation technology innovations demonstrating improved siRNA
delivery and increased durability.
"Today marks a key milestone for Avidity as we leverage the
broad utility of our proprietary AOC platform to address the
underlying cause of genetic heart diseases in precision
cardiology," said Sarah Boyce, president and chief executive
officer at Avidity Biosciences. "We are proud to be the first
company to successfully deliver siRNA directly to skeletal muscle
and now, we have demonstrated that we can deliver siRNA against
targets in the heart in preclinical studies. With continued
innovations to our AOC technology, we are expanding on the
possibilities of RNA delivery. These advancements further our
mission to profoundly improve people's lives by revolutionizing a
new class of targeted RNA therapeutics."
AOC 1072 and AOC 1086 are designed to deliver siRNA directly to
the heart muscle to knock down specific disease-causing genetic
mutations that cause rare genetic cardiomyopathies. In preclinical
studies, AOC 1086 and AOC 1072 demonstrated robust siRNA delivery
to the heart muscle and potent targeted knockdown of
approximately 80% in cardiac PLN mRNA and PRKAG2 mRNA. AOC 1086 and
AOC 1072 were well tolerated without any effect on
electrocardiogram parameters for evaluating cardiac safety in
preclinical studies. Preclinical data from AOC 1072 targeting
PRKAG2 will be presented at the American Heart Association (AHA)
Scientific Sessions 2024 on November
16 in Chicago,
Illinois.
Avidity also shared a first-look at next-generation technology
innovations, including siRNA modifications and evolved antibody
engineering, which, in preclinical studies provided up to 30-fold
increase in siRNA delivery in skeletal muscle, and greater
durability with sustained target inhibition for three months.
Advancements in siRNA delivery and greater durability allow the
opportunity for less frequent dosing and improved patient
convenience.
Video Webcast Information
The company is hosting
Volume 11 of its investor and analyst event series on November
12, 2024, beginning at 8:00 a.m. ET to discuss
preclinical data on AOC 1072 and AOC 1086, along with platform
innovations. The virtual event will be available via 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.
About PLN Cardiomyopathy
PLN (Phospholamban)
Cardiomyopathy is an autosomal dominant, progressive cardiac
disease caused by a mutation on the PLN gene. The most common
disease variant is the R14del, causing a buildup of toxic protein
aggregates that trigger cardiomyocyte death leading to dilation of
the heart chambers, arrhythmias (irregular heartbeats), sudden
cardiac arrest and heart failure. There are no FDA-approved
disease-modifying therapies for PLN cardiomyopathy, and current
standard of care focuses on symptom management with medications or
invasive treatments including pacemakers or implantable
cardioverter defibrillator (ICD). Patients who develop progressive
heart failure may require a heart transplant.
About PRKAG2 Syndrome
PRKAG2 (Protein Kinase
AMP-activated non-catalytic subunit Gamma 2) Syndrome is an
autosomal dominant, progressive cardiac disease caused by a
mutation on the PRKAG2 gene that results in increased AMP-activated
protein kinase (AMPK) activity. This causes excessive cardiac
glycogen storage in the heart, leading to cardiac hypertrophy
(thickening of heart muscle) and multiple different types of
arrhythmias (irregular heartbeats), including Wolff-Parkinson-White
syndrome, atrial fibrillation, and cardiac conduction system
disease. As a result, patients with PRKAG2 syndrome are at an
increased risk of experiencing sudden cardiac arrest or heart
failure. There are no FDA-approved disease-modifying therapies for
PRKAG2 Syndrome, and current standard of care focuses on symptom
management with medications or invasive treatments including
pacemakers or implantable cardioverter defibrillator (ICD).
Patients who develop progressive heart failure may require a heart
transplant.
About Avidity
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 is revolutionizing the field of RNA with its
proprietary AOCs, which are designed to combine the specificity of
monoclonal antibodies with the precision of oligonucleotide
therapies to address targets and diseases previously unreachable
with existing RNA therapies. Utilizing its proprietary AOC
platform, Avidity demonstrated the first-ever successful targeted
delivery of RNA into muscle and is leading the field with clinical
development programs for three rare neuromuscular diseases:
myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD)
and facioscapulohumeral muscular dystrophy (FSHD). Avidity is also
advancing two wholly-owned precision cardiology development
candidates addressing rare genetic cardiomyopathies. Avidity is
broadening the reach of AOCs with its advancing and expanding
pipeline including programs in cardiology and immunology through
internal discovery efforts and key partnerships. Avidity is
headquartered in San Diego, CA.
For more information about our AOC platform, clinical development
pipeline and people, please visit
www.aviditybiosciences.com and engage with us on
LinkedIn and X.
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 ability of our AOC
technology to develop precision cardiology therapeutics; the
ability of AOC 1072 to target PRKAG2 Syndrome and the ability of
AOC 1086 to target PLN cardiomyopathy; our ability to deliver siRNA
to the heart; safety and tolerability results of our precision
cardiology product candidates; the design and capabilities of AOC
1072 and AOC 1086; the effectiveness of AOC 1072 and AOC 1086; our
next generation technology and its potential impact; the improved
delivery and durability associated with our next generation
technology; the design and capabilities of our next generation
technology; and plans and objectives of management for future
operations. 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
Avidity's business and beyond its control, including, without
limitation: the results of preclinical studies are not necessarily
indicative of final results; further analysis of existing
preclinical data and analysis of new data may lead to conclusions
different from those established as of the date hereof, and such
data may not meet Avidity's expectations; preclinical data related
to Avidity's precision cardiology programs and next generation
technology may not support the filing or approval of any IND;
unexpected adverse side effects to, or inadequate efficacy of,
Avidity's product candidates that may delay or limit their
development, regulatory approval and/or commercialization;
Avidity's approach to the discovery and development of product
candidates based on its AOC™ platform is unproven; potential delays
in the commencement, enrollment, data readouts and completion of
preclinical studies or clinical trials; Avidity's dependence on
third parties in connection with preclinical and clinical testing
and product manufacturing; legislative, judicial and regulatory
developments in the United States
and foreign countries; Avidity could exhaust its available capital
resources sooner than it currently expects; and other risks
described in Avidity's Annual Report on Form 10-K for the fiscal
year ended December 31, 2023 and
subsequent filings with the 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 arise 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.
Investor Contact:
Mike
MacLean
(619) 837-5014
investors@aviditybio.com
Media Contact:
Navjot
Rai
(619) 837-5016
media@aviditybio.com
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SOURCE Avidity Biosciences, Inc.