Avidity Biosciences Announces New Precision Cardiology Development Candidates to Treat Rare Genetic Cardiomyopathies and Provides First Look at Next-Generation Technology Innovations
Avidity Biosciences (Nasdaq: RNA) has expanded into precision cardiology with two new development candidates: AOC 1086 for PLN Cardiomyopathy and AOC 1072 for PRKAG2 Syndrome. Preclinical studies showed approximately 80% reduction in cardiac PLN mRNA and PRKAG2 mRNA through direct siRNA delivery to the heart. The company also revealed next-generation technology innovations demonstrating up to 30-fold increase in siRNA delivery to skeletal muscle and three-month sustained target inhibition in preclinical studies. Both candidates were well-tolerated with no effect on cardiac safety parameters. AOC 1072 data will be presented at the AHA Scientific Sessions 2024.
Avidity Biosciences (Nasdaq: RNA) ha ampliato il proprio campo nella cardiologia di precisione con due nuovi candidati in sviluppo: AOC 1086 per la cardiomiopatia da PLN e AOC 1072 per la sindrome da PRKAG2. Gli studi preclinici hanno mostrato una riduzione di circa l'80% dell'mRNA cardiaco di PLN e di PRKAG2 grazie alla somministrazione diretta di siRNA al cuore. L'azienda ha anche svelato innovazioni tecnologiche di nuova generazione che dimostrano un aumento fino a 30 volte nella somministrazione di siRNA ai muscoli scheletrici e un'inibizione sostenuta del bersaglio per tre mesi negli studi preclinici. Entrambi i candidati sono stati ben tollerati senza effetti sui parametri di sicurezza cardiaca. I dati su AOC 1072 saranno presentati alle AHA Scientific Sessions 2024.
Avidity Biosciences (Nasdaq: RNA) se ha expandido en la cardiología de precisión con dos nuevos candidatos en desarrollo: AOC 1086 para la cardiomiopatía por PLN y AOC 1072 para el síndrome PRKAG2. Los estudios preclínicos mostraron una reducción de aproximadamente el 80% en el mRNA cardiaco de PLN y PRKAG2 mediante la entrega directa de siRNA al corazón. La compañía también reveló innovaciones tecnológicas de próxima generación que demuestran un aumento de hasta 30 veces en la entrega de siRNA a los músculos esqueléticos y una inhibición sostenida del objetivo durante tres meses en los estudios preclínicos. Ambos candidatos fueron bien tolerados sin efectos en los parámetros de seguridad cardiaca. Los datos de AOC 1072 se presentarán en las AHA Scientific Sessions 2024.
Avidity Biosciences (Nasdaq: RNA)는 정밀 심장 의학 분야로 확장하여 두 가지 새로운 개발 후보인 AOC 1086 (PLN 심근병증)과 AOC 1072 (PRKAG2 증후군)을 발표했습니다. 전임상 연구 결과, 심장에 직접 siRNA를 전달하여 PLN 및 PRKAG2의 심장 mRNA를 약 80% 감소시키는 결과를 보였습니다. 이 회사는 또한 다음 세대 기술 혁신을 발표하여 골격근에 대한 siRNA 전달을 최대 30배 증가시키고, 전임상 연구에서 3개월 동안 지속적인 목표 억제를 달성했습니다. 두 후보 모두 안전한 심장 파라미터에 영향을 미치지 않고 잘 견디었습니다. AOC 1072에 대한 데이터는 AHA 과학 세션 2024에서 발표될 예정입니다.
Avidity Biosciences (Nasdaq: RNA) s'est étendu dans la cardiologie de précision avec deux nouveaux candidats en développement : AOC 1086 pour la cardiomyopathie associée à PLN et AOC 1072 pour le syndrome PRKAG2. Des études précliniques ont montré une réduction d'environ 80 % de l'ARNm cardiaque PLN et PRKAG2 grâce à une livraison directe de siRNA au cœur. L'entreprise a également révélé des innovations technologiques de nouvelle génération démontrant une augmentation jusqu'à 30 fois de la livraison de siRNA aux muscles squelettiques et une inhibition ciblée soutenue pendant trois mois dans les études précliniques. Les deux candidats ont été bien tolérés sans effet sur les paramètres de sécurité cardiaque. Les données sur AOC 1072 seront présentées lors des AHA Scientific Sessions 2024.
Avidity Biosciences (Nasdaq: RNA) hat sich in die präzise Kardiologie mit zwei neuen Entwicklungskandidaten erweitert: AOC 1086 für PLN-Kardiomyopathie und AOC 1072 für das PRKAG2-Syndrom. Vorläufige Studien zeigten eine Reduktion von etwa 80% der kardialen PLN-mRNA und PRKAG2-mRNA durch direkte siRNA-Verabreichung an das Herz. Das Unternehmen enthüllte auch innovative Technologien der nächsten Generation, die eine bis zu 30-fache Steigerung der siRNA-Abgabe an die Skelettmuskulatur und eine dreimonatige anhaltende Zielinhibierung in präklinischen Studien demonstrieren. Beide Kandidaten wurden gut vertragen, ohne die Parameter der kardialen Sicherheit zu beeinträchtigen. Daten zu AOC 1072 werden auf den AHA Scientific Sessions 2024 präsentiert.
- Successful preclinical results showing ~80% reduction in target mRNA for both drug candidates
- 30-fold increase in siRNA delivery efficiency in skeletal muscle
- Extended durability with 3-month sustained target inhibition
- Positive safety profile with no cardiac safety concerns in preclinical studies
- None.
Insights
The expansion into precision cardiology with two new development candidates represents a significant technological advancement. The 80% reduction in cardiac PLN mRNA and PRKAG2 mRNA in preclinical studies is particularly impressive, demonstrating strong potential efficacy. The next-generation technology showing 30-fold increase in siRNA delivery and three-month durability could be transformative for treatment protocols.
The company's ability to successfully deliver siRNA to both skeletal muscle and heart tissue positions them uniquely in the RNA therapeutics space. This dual-tissue targeting capability, combined with the improved delivery efficiency and duration, could significantly expand their addressable market beyond rare diseases. The well-tolerated safety profile without cardiac safety concerns in preclinical studies is encouraging for future clinical development.
This pipeline expansion strengthens Avidity's competitive position in the RNA therapeutics market. The company's
The move into cardiac indications diversifies the company's portfolio risk while leveraging existing technology infrastructure, potentially creating operational efficiencies. The robust preclinical data could accelerate partnerships or licensing opportunities, providing additional revenue streams. However, investors should note that significant clinical development costs lie ahead before commercialization.
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
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
"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
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
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
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.
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