Avidity Biosciences Reports Third Quarter 2024 Financial Results and Recent Highlights
Avidity Biosciences reported its Q3 2024 financial results and recent highlights. The company is advancing three key clinical trials: del-zota for DMD44, del-brax for FSHD, and del-desiran for DM1. The Phase 1/2 EXPLORE44 trial for del-zota showed a 25% increase in dystrophin production and reduced creatine kinase levels. Enrollment for the Phase 3 HARBOR trial for del-desiran is on track, with the FDA lifting a partial clinical hold. Del-brax's biomarker cohort in the FORTITUDE study shows promising results. Avidity raised $345.1 million in a public offering and reported $1.6 billion in cash and marketable securities. Collaboration revenue was $2.3 million for Q3 2024. R&D expenses increased to $77.2 million, and G&A expenses rose to $23.3 million due to expanded operations. A webcast event on November 12, 2024, will showcase precision cardiology candidates and next-gen technology innovations.
Avidity Biosciences ha riferito i suoi risultati finanziari del terzo trimestre 2024 e gli ultimi sviluppi. L'azienda sta portando avanti tre studi clinici chiave: del-zota per DMD44, del-brax per FSHD e del-desiran per DM1. Lo studio di fase 1/2 EXPLORE44 per del-zota ha mostrato un aumento del 25% nella produzione di distrofina e una riduzione dei livelli di creatina chinasi. L'arruolamento per lo studio di fase 3 HARBOR per del-desiran è in linea con le tempistiche, con la FDA che ha revocato un'ostativa clinica parziale. Il cohort di biomarcatori di del-brax nello studio FORTITUDE mostra risultati promettenti. Avidity ha raccolto 345,1 milioni di dollari in un'offerta pubblica e ha riportato 1,6 miliardi di dollari in contanti e titoli negoziabili. I ricavi da collaborazioni sono stati di 2,3 milioni di dollari per il terzo trimestre 2024. Le spese per R&D sono aumentate a 77,2 milioni di dollari, mentre le spese G&A sono salite a 23,3 milioni di dollari a causa dell'espansione delle operazioni. Un evento in webcast il 12 novembre 2024 presenterà i candidati per la cardiologia di precisione e le innovazioni tecnologiche di nuova generazione.
Avidity Biosciences informó sobre sus resultados financieros del tercer trimestre de 2024 y los últimos avances. La compañía está avanzando en tres ensayos clínicos clave: del-zota para DMD44, del-brax para FSHD y del-desiran para DM1. El ensayo de fase 1/2 EXPLORE44 para del-zota mostró un aumento del 25% en la producción de distrofina y niveles reducidos de creatina quinasa. La inscripción para el ensayo de fase 3 HARBOR para del-desiran avanza según lo previsto, con la FDA levantando un impedimento clínico parcial. La cohorte de biomarcadores de del-brax en el estudio FORTITUDE muestra resultados prometedores. Avidity recaudó 345,1 millones de dólares en una oferta pública y reportó 1,6 mil millones de dólares en efectivo y valores negociables. Los ingresos por colaboración fueron de 2,3 millones de dólares para el tercer trimestre de 2024. Los gastos en I+D aumentaron a 77,2 millones de dólares, y los gastos generales y administrativos aumentaron a 23,3 millones de dólares debido a la expansión de las operaciones. Un evento en webcast el 12 de noviembre de 2024 presentará candidatos para la cardiología de precisión e innovaciones tecnológicas de próxima generación.
아비디티 바이오사이언스는 2024년 3분기 재무 결과와 최근 소식을 보고했습니다. 이 회사는 세 가지 주요 임상 시험을 진행하고 있습니다: del-zota는 DMD44에, del-brax는 FSHD에, del-desiran은 DM1에 해당합니다. del-zota에 대한 1/2상 EXPLORE44 시험에서는 디스트로핀 생산이 25% 증가하고 크레아틴 키나아제 수치가 감소했습니다. del-desiran에 대한 3상 HARBOR 시험의 등록은 순조롭게 진행 중이며, FDA는 부분 임상 보류를 해제했습니다. FORTITUDE 연구에서 del-brax의 바이오마커 코호트는 유망한 결과를 보여줍니다. 아비디티는 공모를 통해 3억 4,510만 달러를 모금했으며, 현금 및 유동성 증권으로 16억 달러를 보고했습니다. 2024년 3분기의 협력 수익은 230만 달러였습니다. R&D 비용은 7720만 달러로 증가했으며, 운영 확장으로 인해 일반 및 관리 비용은 2330만 달러로 증가했습니다. 2024년 11월 12일에 열리는 웹캐스트 이벤트는 정밀 심장학 후보 및 차세대 기술 혁신을 소개합니다.
Avidity Biosciences a publié ses résultats financiers pour le troisième trimestre de 2024 et a présenté ses dernières avancées. La société avance dans trois essais cliniques clés : del-zota pour DMD44, del-brax pour FSHD et del-desiran pour DM1. L'essai de phase 1/2 EXPLORE44 pour del-zota a montré une augmentation de 25 % de la production de dystrophine et une réduction des niveaux de créatine kinase. L'inscription à l'essai de phase 3 HARBOR pour del-desiran est en bonne voie, la FDA levant une suspension clinique partielle. La cohorte de biomarqueurs de del-brax dans l'étude FORTITUDE montre des résultats prometteurs. Avidity a levé 345,1 millions de dollars lors d'une offre publique et a déclaré 1,6 milliard de dollars en liquidités et titres négociables. Les revenus de collaboration se sont élevés à 2,3 millions de dollars pour le troisième trimestre de 2024. Les dépenses de R&D ont augmenté à 77,2 millions de dollars, et les dépenses générales et administratives ont atteint 23,3 millions de dollars en raison de l'expansion des opérations. Un événement en webcast le 12 novembre 2024 présentera des candidats pour la cardiologie de précision et des innovations technologiques de nouvelle génération.
Avidity Biosciences hat seine Finanzzahlen für das 3. Quartal 2024 und aktuelle Fortschritte veröffentlicht. Das Unternehmen entwickelt drei wichtige klinische Studien: del-zota für DMD44, del-brax für FSHD und del-desiran für DM1. Die Phase 1/2 EXPLORE44-Studie für del-zota zeigte einen Anstieg der Dystrophinproduktion um 25 % und eine Verringerung der Kreatinkinasewerte. Die Einschreibung für die Phase-3-Studie HARBOR für del-desiran läuft planmäßig, nachdem die FDA eine teilweise klinische Aussetzung aufgehoben hat. Die Biomarker-Kohorte von del-brax in der FORTITUDE-Studie zeigt vielversprechende Ergebnisse. Avidity sammelte 345,1 Millionen Dollar in einer öffentlichen Emission und meldete 1,6 Milliarden Dollar in bar und handelbaren Wertpapieren. Die Kooperationsumsätze betrugen im 3. Quartal 2024 2,3 Millionen Dollar. Die F&E-Ausgaben stiegen auf 77,2 Millionen Dollar, und die allgemeinen und Verwaltungskosten erhöhten sich auf 23,3 Millionen Dollar aufgrund der erweiterten Geschäftstätigkeiten. Eine Webcast-Veranstaltung am 12. November 2024 wird Kandidaten für die präzise Kardiologie und Innovationen der nächsten Generation präsentieren.
- Positive del-zota data from Phase 1/2 EXPLORE44 trial for DMD44.
- Enrollment on track for del-desiran Phase 3 HARBOR trial in DM1.
- FDA lifted partial clinical hold on del-desiran.
- Strong cash position of $1.6 billion.
- Raised $345.1 million in a public offering.
- Increased R&D expenses to $77.2 million for Q3 2024.
- Higher G&A expenses of $23.3 million for Q3 2024.
Enrollment in global Phase 3 HARBOR™ study for del-desiran in DM1 is on track
Avidity initiated biomarker cohort for del-brax FORTITUDE™ study for FSHD; pursuing a potential accelerated approval path for del-brax
Reported positive del-zota data from Phase 1/2 EXPLORE44™ trial for DMD44
Avidity to provide a first look at precision cardiology candidates and a glimpse at next-generation technology innovations via webcast event November 12, 2024
"We are executing three potentially registrational clinical trials in three rare diseases where there are limited or no therapeutic options available. We reported positive del-zota data from the 5 mg/kg cohort of our Phase 1/2 EXPLORE44™ trial for DMD44 and have initiated enrollment in the EXPLORE44-OLE™. In addition, enrollment is on track for del-desiran's global Phase 3 HARBOR™ trial in DM1 and we initiated the biomarker cohort of our FORTITUDE™ study in FSHD, marking a key step in our strategy to pursue a potential accelerated approval path for del-brax," said Sarah Boyce, president and chief executive officer at Avidity. "As we build for the future, we look forward to sharing our exciting innovations in precision cardiology and a glimpse at our next-generation technology. We continue to build our global commercial infrastructure to provide potential new therapies to people living with these serious rare diseases as quickly as possible."
"We were pleased to complete an additional upsized public offering in August following positive clinical data from our EXPLORE44 program. With a strong cash position of approximately
Recent Highlights
Del-zota (AOC 1044) for DMD44
- In August, Avidity reported positive initial del-zota data from the 5 mg/kg cohort of the Phase 1/2 EXPLORE44™ trial in people living with DMD44 demonstrating unsurpassed delivery to skeletal muscle, unprecedented, unadjusted increase of
25% in near full-length dystrophin production with a profound reduction in creatine kinase levels to near normal, and robust exon 44 skipping. Del-zota demonstrated favorable safety and tolerability with most treatment emergent adverse events mild or moderate. - The Phase 1/2 EXPLORE44 trial is fully enrolled and ongoing.
- In addition to the participants rolling over from the Phase 1/2 EXPLORE44 trial, Avidity has begun enrolling 10-15 new participants in the EXPLORE44 Open-label Extension study (OLE).
Del-brax (AOC 1020) for FSHD
- In October, Avidity announced the initiation of the biomarker cohort in the Phase 1/2 FORTITUDE™ trial of del-brax. 2 mg/kg of del-brax will be administered every six weeks, designed to ensure continuous suppression of DUX4.
- In June of this year, Avidity reported positive initial del-brax 2 mg/kg data at four months from the Phase 1/2 FORTITUDE trial demonstrating unprecedented and consistent reductions of greater than
50% in DUX4 regulated genes, mean reductions of25% or greater in novel circulating biomarker and creatine kinase, trends of functional improvement, and favorable safety and tolerability in people living with FSHD. - Avidity remains on track to initiate the functional cohort in the FORTITUDE study in the first half of 2025.
- In July, the first participants from FORTITUDE began to roll over to the FORTITUDE Open-Label Extension (OLE) trial. All participants that complete FORTITUDE are eligible to enroll in the FORTITUDE-OLE™ trial.
Del-desiran (AOC 1001) for DM1
- Enrollment for the global Phase 3 HARBOR™ trial is ongoing and on track.
- In October, the
U.S. Food and Drug Administration (FDA) removed the partial clinical hold on del-desiran.
Pipeline Advancements and Organizational Highlights
- In November, Avidity plans to provide a first look at precision cardiology candidates. In addition, Avidity plans to share a glimpse at next-generation technology innovations.
- In August, Avidity announced it plans to advance additional candidates from its DMD franchise following robust del-zota data; Exon 45 is currently in IND-enabling studies.
- Kathleen Gallagher was promoted to chief program officer in September 2024.
Third Quarter 2024 Financial Results
- Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled
as of September 30, 2024, which reflects a gross$1.6 billion raise from a public offering.$345.1 million - Collaboration Revenue: Collaboration revenues of
for the third quarter of 2024 and$2.3 million for the first nine months of 2024 primarily relate to Avidity's research collaboration and license partnership with Bristol Myers Squibb. Collaboration revenues of$7.9 million for the third quarter of 2023 and$2.8 million for the first nine months of 2023 primarily related to Avidity's research collaboration and license partnership with Eli Lilly and Company.$7.4 million - Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were
for the third quarter of 2024 compared with$77.2 million for the third quarter of 2023, and$47.7 million for the first nine months of 2024 compared with$208.0 million for the first nine months of 2023. The increases were primarily driven by the advancement of del-desiran, del-brax and del-zota, as well as internal and external costs related to the expansion of the company's overall research capabilities.$138.2 million - General and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs and patent filing and maintenance fees. These expenses were
for the third quarter of 2024 compared with$23.3 million for the third quarter of 2023, and$13.7 million for the first nine months of 2024 compared with$57.9 million for the first nine months of 2023. The increases were primarily due to higher personnel costs to support the company's expanded operations.$38.1 million
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 muscle diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). 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: a potential accelerated pathway for registration for del-brax, and the potential for all of Avidity's current clinical trials to be registrational; Avidity's next-generational technology innovations and the timing of its announcement; the anticipated announcement of lead precision cardiology program targets, including the timing thereof; Avidity's plans to build a global commercial infrastructure and capabilities; plans for the advancement of DMD programs beyond DMD44; plans for the initiation of the functional cohort in the FORTITUDE™ trial and the timing thereof; the characterization of data associated with Avidity's product candidates in their respective clinical trials, the conclusions drawn therefrom, and the impact of such data on the advancement of the respective product candidates; enrollment statuses of Avidity's clinical programs; the status of the HARBOR™ study; Avidity's platform, planned operations and programs; and Avidity's cash position and runway.
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: preliminary results of a clinical trial are not necessarily indicative of final results; further analysis of existing clinical data and analysis of new data may lead to conclusions different from those established as of the respective data cutoff dates in Avidity's clinical trials, and such data may not meet Avidity's expectations; Avidity's biomarker and planned functional cohorts in the FORTITUDE study may not support the registration of del-brax; 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; later developments with the FDA and other global regulators that could be inconsistent with the feedback received to date regarding Avidity's clinical trials; Avidity's approach to the discovery and development of product candidates based on its AOC™ platform is unproven and may not produce any products of commercial value; potential delays in the commencement, enrollment, data readouts and completion of preclinical studies or clinical trials; the success of its preclinical studies and clinical trials for the company's product candidates; Avidity's dependence on third parties in connection with preclinical and clinical testing and product manufacturing; Avidity may not realize the expected benefits of its collaborations; legislative, judicial and regulatory developments in
Investor Contact:
Mike MacLean
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investors@aviditybio.com
Media Contact:
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Avidity Biosciences, Inc. Selected Condensed Consolidated Financial Information (in thousands, except per share data) (unaudited)
| |||||||
Statements of Operations | Three Months Ended September 30, | Nine Months Ended September 30, | |||||
2024 | 2023 | 2024 | 2023 | ||||
Collaboration revenue | $ 2,336 | $ 2,818 | $ 7,924 | $ 7,367 | |||
Operating expenses: | |||||||
Research and development | 77,197 | 47,714 | 207,968 | 138,151 | |||
General and administrative | 23,273 | 13,729 | 57,902 | 38,071 | |||
Total operating expenses | 100,470 | 61,443 | 265,870 | 176,222 | |||
Loss from operations | (98,134) | (58,625) | (257,946) | (168,855) | |||
Other income, net | 17,736 | 6,267 | 37,901 | 17,078 | |||
Net loss | $ (80,398) | $ (52,358) | $ (220,045) | $ (151,777) | |||
Net loss per share, basic and diluted | $ (0.65) | $ (0.71) | $ (2.08) | $ (2.11) | |||
Weighted-average shares outstanding, basic and diluted | 123,375 | 74,097 | 105,902 | 71,987 |
Balance Sheets | September 30, | December 31, | |
Assets | |||
Current assets: | |||
Cash, cash equivalents and marketable securities | $ 1,588,593 | $ 595,351 | |
Prepaid and other assets | 33,273 | 15,956 | |
Total current assets | 1,621,866 | 611,307 | |
Property and equipment, net | 9,493 | 8,381 | |
Restricted cash | 2,795 | 295 | |
Right-of-use assets | 6,299 | 8,271 | |
Other assets | 318 | 301 | |
Total assets | $ 1,640,771 | $ 628,555 | |
Liabilities and Stockholders' Equity | |||
Current liabilities: | |||
Accounts payable and other liabilities | $ 71,673 | $ 52,315 | |
Deferred revenue, current portion | 19,660 | 28,365 | |
Total current liabilities | 91,333 | 80,680 | |
Lease liabilities, net of current portion | 3,797 | 6,213 | |
Deferred revenue, net of current portion | 42,261 | 40,898 | |
Total liabilities | 137,391 | 127,791 | |
Stockholders' equity | 1,503,380 | 500,764 | |
Total liabilities and stockholders' equity | $ 1,640,771 | $ 628,555 |
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SOURCE Avidity Biosciences, Inc.
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