Avidity Biosciences Reports Second Quarter 2024 Financial Results and Recent Highlights
Avidity Biosciences (Nasdaq: RNA) reported positive Q2 2024 results and significant progress in its clinical trials. Key highlights include:
1. Positive del-zota data showing a 25% increase in dystrophin production for DMD44 patients.
2. Initiation of the global Phase 3 HARBOR™ trial for del-desiran in DM1 patients.
3. Breakthrough Therapy designation for del-desiran from the FDA.
4. Positive del-brax data with >50% reduction in DUX4 regulated genes for FSHD patients.
5. Plans to accelerate registrational cohorts for del-brax.
6. Announcement of lead precision cardiology program target planned for Q4 2024.
7. Strong financial position with $1.3 billion cash on hand.
Financials: Q2 2024 collaboration revenue was $2.0 million, R&D expenses increased to $63.9 million, and G&A expenses rose to $20.7 million.
Avidity Biosciences (Nasdaq: RNA) ha riportato risultati positivi per il secondo trimestre del 2024 e ha fatto significativi progressi nei suoi studi clinici. Tra i punti salienti:
1. Dati positivi sul del-zota che mostrano un aumento del 25% nella produzione di distrofina per i pazienti DMD44.
2. Inizio della sperimentazione globale di Fase 3 HARBOR™ per il del-desiran nei pazienti affetti da DM1.
3. Designazione di Terapia Innovativa per il del-desiran da parte della FDA.
4. Dati positivi sul del-brax con una riduzione superiore al 50% dei geni regolati da DUX4 per i pazienti FSHD.
5. Piani per accelerare i gruppi registrativi per il del-brax.
6. Annuncio del programma di cardiologia di precisione principale previsto per il quarto trimestre del 2024.
7. Posizione finanziaria robusta con 1,3 miliardi di dollari in contante disponibili.
Finanza: nel secondo trimestre del 2024, i ricavi da collaborazione sono stati di 2,0 milioni di dollari, le spese per R&S sono aumentate a 63,9 milioni di dollari e le spese generali e amministrative sono salite a 20,7 milioni di dollari.
Avidity Biosciences (Nasdaq: RNA) reportó resultados positivos en el segundo trimestre de 2024 y un progreso significativo en sus ensayos clínicos. Los puntos destacados incluyen:
1. Datos positivos del del-zota que muestran un aumento del 25% en la producción de distrofina para los pacientes DMD44.
2. Inicio del ensayo global de Fase 3 HARBOR™ para el del-desiran en pacientes con DM1.
3. Designación de Terapia Innovadora para el del-desiran por parte de la FDA.
4. Datos positivos del del-brax con una reducción superior al 50% en los genes regulados por DUX4 para los pacientes con FSHD.
5. Planes para acelerar los grupos de registro para el del-brax.
6. Anuncio del programa principal de cardiología de precisión planeado para el cuarto trimestre de 2024.
7. Fuerte posición financiera con 1.3 mil millones de dólares en efectivo disponible.
Finanzas: los ingresos por colaboración del segundo trimestre de 2024 fueron de 2.0 millones de dólares, los gastos en I+D aumentaron a 63.9 millones de dólares y los gastos generales y administrativos subieron a 20.7 millones de dólares.
Avidity Biosciences (Nasdaq: RNA)는 2024년 2분기 긍정적인 결과와 임상 시험에서의 상당한 진행을 보고했습니다. 주요 하이라이트에는:
1. DMD44 환자를 위한 dystrophin 생산이 25% 증가한 긍정적인 del-zota 데이터.
2. DM1 환자를 위한 del-desiran의 글로벌 3상 HARBOR™ 시험 시작.
3. FDA로부터 del-desiran에 대한 혁신 치료제 지정.
4. FSHD 환자를 위한 DUX4 조절 유전자에서 50% 이상의 감소를 보인 긍정적인 del-brax 데이터.
5. del-brax의 등록 집단 가속화 계획.
6. 2024년 4분기로 예정된 주요 정밀 심장 치료 프로그램 목표 발표.
7. 13억 달러의 현금을 보유한 강력한 재무 상태.
재무사항: 2024년 2분기 협업 수익은 200만 달러였고, R&D 비용은 6390만 달러로 증가했으며, 일반 관리 비용은 2070만 달러로 증가했습니다.
Avidity Biosciences (Nasdaq: RNA) a rapporté des résultats positifs pour le deuxième trimestre de 2024 et des progrès significatifs dans ses essais cliniques. Les points clés incluent:
1. Des données positives sur le del-zota montrant une augmentation de 25 % de la production de dystrophine pour les patients DMD44.
2. Lancement de l'essai mondial de Phase 3 HARBOR™ pour le del-desiran chez les patients DM1.
3. Attribution de la désignation de Thérapie Innovante pour le del-desiran par la FDA.
4. Des données positives sur le del-brax avec une réduction de plus de 50 % des gènes régulés par DUX4 chez les patients FSHD.
5. Plans pour accélérer les cohortes d'enregistrement pour le del-brax.
6. Annonce du programme principal de cardiologie de précision prévu pour le quatrième trimestre 2024.
7. Position financière solide avec 1,3 milliard de dollars en liquidités.
Finances : au deuxième trimestre 2024, les revenus de collaboration s'élevaient à 2,0 millions de dollars, les dépenses R&D ont augmenté à 63,9 millions de dollars et les dépenses générales et administratives ont atteint 20,7 millions de dollars.
Avidity Biosciences (Nasdaq: RNA) hat positive Ergebnisse für das zweite Quartal 2024 berichtet und bedeutende Fortschritte in seinen klinischen Studien erzielt. Wesentliche Highlights sind:
1. Positive del-zota-Daten zeigen eine 25%ige Steigerung der Dystrophin-Produktion bei DMD44-Patienten.
2. Beginn der globalen Phase 3 HARBOR™-Studie für del-desiran bei DM1-Patienten.
3. Durchbruch-Therapie-Bezeichnung für del-desiran von der FDA.
4. Positive del-brax-Daten mit über 50% Reduktion der von DUX4 regulierten Gene bei FSHD-Patienten.
5. Pläne zur Beschleunigung von Registrierungsgruppen für del-brax.
6. Ankündigung des führenden Programms für präzise Kardiologie, das für das vierte Quartal 2024 geplant ist.
7. Starke Finanzlage mit 1,3 Milliarden Dollar Bargeld.
Finanzen: Die Zusammenarbeitseinnahmen im 2. Quartal 2024 betrugen 2,0 Millionen Dollar, die F&E-Ausgaben stiegen auf 63,9 Millionen Dollar und die allgemeinen Verwaltungskosten stiegen auf 20,7 Millionen Dollar.
- Positive del-zota data showing 25% increase in dystrophin production and reduction of creatine kinase levels in DMD44 patients
- Initiation of global Phase 3 HARBOR™ trial for del-desiran in DM1 patients
- FDA Breakthrough Therapy designation for del-desiran
- Positive del-brax data with >50% reduction in DUX4 regulated genes in FSHD patients
- Strong cash position of $1.3 billion
- Successful upsized public offering in June 2024
- Increased R&D expenses from $42.6 million in Q2 2023 to $63.9 million in Q2 2024
- Increased G&A expenses from $12.3 million in Q2 2023 to $20.7 million in Q2 2024
Insights
Avidity Biosciences' Q2 2024 results reveal significant progress in their clinical pipeline and a strong financial position. The company reported
R&D expenses increased to
The positive clinical data across multiple programs, particularly for del-zota and del-brax, could potentially translate into significant future revenue streams if these therapies reach commercialization. However, investors should note that the path to market approval in biotech is often long and uncertain.
Avidity's Q2 results showcase impressive clinical progress across multiple programs. The del-zota data in DMD44 patients is particularly noteworthy, demonstrating a
The del-brax data in FSHD patients is equally promising, showing consistent reductions of over
The FDA's Breakthrough Therapy designation for del-desiran in DM1 underscores the potential impact of Avidity's AOC platform. As the company expands into precision cardiology, it demonstrates the versatility of their technology across different therapeutic areas. However, it's important to remember that early-stage clinical data, while promising, doesn't guarantee ultimate regulatory approval or commercial success.
Avidity's Q2 results position the company as a strong player in the RNA therapeutics space. The positive clinical data across multiple rare disease programs could potentially disrupt current treatment paradigms in DMD, FSHD and DM1. The company's AOC platform appears to be delivering on its promise of targeted RNA delivery, which could give Avidity a competitive edge in the crowded biotech landscape.
The planned expansion into precision cardiology signals Avidity's ambition to tackle larger market opportunities beyond rare diseases. This move could significantly expand the company's addressable market and attract broader investor interest. However, it also means facing potentially stiffer competition from established players in the cardiovascular space.
Investor sentiment is likely to be positive given the clinical progress and strong cash position. The upsized public offering suggests strong market confidence in Avidity's potential. However, as with any clinical-stage biotech, risks remain. The company will need to successfully navigate late-stage clinical trials and regulatory approvals to realize the full potential of its pipeline. The market will be closely watching for the announcement of the lead precision cardiology target in Q4 2024, which could be a significant catalyst for the stock.
Avidity reports positive del-zota (AOC 1044) data; demonstrated
Initiated global Phase 3 HARBOR™ trial and began administration of del-desiran in people living with DM1; del-desiran received FDA Breakthrough Therapy designation
Avidity plans to accelerate initiation of del-brax (AOC 1020) registrational cohorts after reporting unprecedented and consistent reductions in DUX4 regulated genes, trends of functional improvement and favorable safety and tolerability in people living with FSHD in Phase 1/2 FORTITUDE™ trial
Announcement of our lead precision cardiology program target planned for Q4 2024
Cash on hand of approximately
"The last few months have been incredible for Avidity, but more importantly, for people living with DM1, FSHD and DMD. We received breakthrough designation for del-desiran, initiated the global Phase 3 HARBOR™ trial, reported unprecedented data from the del-brax FORTITUDE™ trial and earlier today, shared data from del-zota's Phase 1/2 EXPLORE44™ trial demonstrating significant increases in dystrophin production and exon 44 skipping for people living with DMD44," said Sarah Boyce, president and chief executive officer at Avidity. "All of this progress is due to our AOC platform which has proven we can target a range of genetic diseases. We plan to expand beyond rare neuromuscular diseases as we share the first target from our precision cardiology pipeline in Q4 2024. These achievements, accomplished in conjunction with our patient communities, accelerate our vision of profoundly improving people's lives by revolutionizing RNA delivery."
"We successfully closed an upsized public offering in June, our second successful equity raise this year. Our cash position of
Recent Highlights
Del-zota (AOC 1044)
- In August, Avidity reported positive initial del-zota 5mg/kg patient data from the Phase 1/2 EXPLORE44™ trial in people living with DMD44 demonstrating unsurpassed delivery in skeletal muscle, unprecedented increase in dystrophin production, robust exon 44 skipping, and a profound reduction in creatine kinase
- In the Phase 1/2 EXPLORE44 trial, del-zota data from 10 participants at the four-month time period after three doses of 5mg/kg del-zota (PMO dose), or placebo every six weeks, demonstrated:
- Unsurpassed delivery of PMO of 200 nM in skeletal muscle
- Statistically significant
37% increase in exon 44 skipping and up to66% exon 44 skipping compared to baseline - Statistically significant increase of
25% of normal in dystrophin production and restored total dystrophin up to54% of normal - Profound reduction in creatine kinase as levels were reduced to near normal with greater than
80% reduction compared to baseline
- Safety and tolerability for 25 participants was assessed across two dose levels (5 mg/kg and 10 mg/kg) from the Phase 1/2 EXPLORE44 trial. Del-zota demonstrated favorable safety and tolerability with most treatment emergent adverse events (AEs) mild or moderate in participants with DMD44
- Enrollment is now complete for the Phase 1/2 EXPLORE44 trial. Avidity plans to enroll additional patients in the EXPLORE44 Open-label Extension study (OLE)
- Avidity introduced delpacibart zotadirsen as the approved international nonproprietary name of AOC 1044, abbreviated as del-zota
Del-desiran (AOC 1001)
- In June, Avidity initiated and began administration of del-desiran in the global Phase 3 HARBORTM trial in people living with myotonic dystrophy type 1 (DM1)
- The FDA granted del-desiran Breakthrough Therapy designation in May 2024 for DM1
Del-brax (AOC 1020)
- Avidity reported positive initial del-brax data from the Phase 1/2 FORTITUDE™ trial demonstrating unprecedented and consistent reductions of greater than
50% in DUX4 regulated genes, trends of functional improvement, and favorable safety and tolerability in people living with facioscapulohumeral muscular dystrophy (FSHD) - In the Phase 1/2 FORTITUDE trial, del-brax 2 mg/kg data from 12 participants at the four-month time period demonstrated:
- Greater than
50% mean reductions in DUX4 regulated genes across multiple panels for DUX4 regulated gene expression in muscle - All participants treated with del-brax showed reductions greater than
20% in DUX4 regulated genes - Mean reductions of
25% or greater in a novel circulating biomarker and creatine kinase - Trends of functional improvements including increased strength in upper and lower limb muscles, and muscle function as measured by reachable workspace (RWS) compared to placebo and the ReSolve natural history study
- Trends of improvement in patient and clinician reported outcomes
- Greater than
- A four-month look at the safety and tolerability for all 39 participants across two dose levels (2 mg/kg and 4 mg/kg) from the Phase 1/2 FORTITUDE trial demonstrated favorable safety and tolerability with all adverse events (AEs) mild or moderate, no serious adverse events and no discontinuations
- Completed enrollment for the Phase 1/2 FORTITUDE trial. Avidity plans to accelerate the initiation of registrational cohorts with the biomarker cohort planned in 2H 2024 and functional cohort planned in 1H 2025.
- Avidity introduced delpacibart braxlosiran as the approved international nonproprietary name of AOC 1020, abbreviated as del-brax
Pipeline Advancements
- Avidity plans to announce its lead precision cardiology program target in Q4 2024
Organizational Highlights
- Announced the appointment of Simona Skerjanec, M.Pharm, MBA to its board of directors in May 2024
- Announced the appointment of John B. Moriarty, Jr., J.D., as Chief Legal Officer and Corporate Secretary in August 2024
Upcoming Milestones
- Upcoming anticipated milestones include:
- In Q4 2024, announcing lead precision cardiology program target
- Accelerating the initiation of registrational cohorts in FORTITUDE™ trial:
- Biomarker cohort planned for 2H 2024
- Functional cohort planned for 1H 2025
Second Quarter 2024 Financial Results
- Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled
$1.3 billion $461 million - Collaboration Revenue: Collaboration revenues of
$2.0 million $5.6 million $2.3 $4.5 million - Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were
$63.9 million $42.6 million $130.8 million $90.4 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
$20.7 million $12.3 million $34.6 million $24.3 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 focused on 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: the anticipated announcement of a lead precision cardiology program target, including the timing thereof; the potential to develop first-in-class and best-in-class treatments; plans to accelerate the initiation of registrational cohorts in the FORTITUDE™ trial and the timing thereof; the characterization of data associated with del-brax from the FORTITUDE study and del-zota with the EXPLORE44™ study, and the impact of such data on the advancement of the respective product candidates; Avidity's DMD franchise; Avidity's plans to become an integrated global biopharmaceutical company; Avidity's platform, planned operations and programs; and Avidity's financial position, cash balance and expected cash 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 planned additional 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; Avidity may not be able to resolve the partial clinical hold related to del-desiran; 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; 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
(619) 837-5014
investors@aviditybio.com
Media Contact:
Navjot Rai
(619) 837-5016
media@aviditybio.com
Avidity Biosciences, Inc. | |||||||
Selected Condensed Financial Information | |||||||
(in thousands, except per share data) | |||||||
(unaudited) | |||||||
Statements of Operations | Three Months Ended June 30, | Six Months Ended June 30, | |||||
2024 | 2023 | 2024 | 2023 | ||||
Collaboration revenue | $ 2,045 | $ 2,316 | $ 5,588 | $ 4,549 | |||
Operating expenses: | |||||||
Research and development | 63,940 | 42,672 | 130,772 | 90,437 | |||
General and administrative | 20,731 | 12,278 | 34,629 | 24,342 | |||
Total operating expenses | 84,671 | 54,950 | 165,401 | 114,779 | |||
Loss from operations | (82,626) | (52,634) | (159,813) | (110,230) | |||
Other income, net | 11,833 | 5,609 | 20,165 | 10,811 | |||
Net loss | $ (70,793) | $ (47,025) | $ (139,648) | $ (99,419) | |||
Net loss per share, basic and | $ (0.65) | $ (0.66) | $ (1.44) | $ (1.40) | |||
Weighted-average shares | 106,928 | 71,390 | 97,070 | 70,914 | |||
Balance Sheets | June 30, | December 31, | |
Assets | |||
Current assets: | |||
Cash, cash equivalents and marketable securities | $ 1,299,046 | $ 595,351 | |
Prepaid and other assets | 29,755 | 15,956 | |
Total current assets | 1,328,801 | 611,307 | |
Property and equipment, net | 8,498 | 8,381 | |
Restricted cash | 2,795 | 295 | |
Right-of-use assets | 6,967 | 8,271 | |
Other assets | 364 | 301 | |
Total assets | $ 1,347,425 | $ 628,555 | |
Liabilities and Stockholders' Equity | |||
Current liabilities: | |||
Accounts payable and other liabilities | $ 57,515 | $ 52,315 | |
Deferred revenue, current portion | 26,697 | 28,365 | |
Total current liabilities | 84,212 | 80,680 | |
Lease liabilities, net of current portion | 4,617 | 6,213 | |
Deferred revenue, net of current portion | 37,560 | 40,898 | |
Total liabilities | 126,389 | 127,791 | |
Stockholders' equity | 1,221,036 | 500,764 | |
Total liabilities and stockholders' equity | $ 1,347,425 | $ 628,555 |
View original content to download multimedia:https://www.prnewswire.com/news-releases/avidity-biosciences-reports-second-quarter-2024-financial-results-and-recent-highlights-302218648.html
SOURCE Avidity Biosciences, Inc.
FAQ
What were Avidity Biosciences' (RNA) key clinical trial results in Q2 2024?
How much cash does Avidity Biosciences (RNA) have on hand as of Q2 2024?
What is Avidity Biosciences' (RNA) next major announcement planned for Q4 2024?
What regulatory milestone did Avidity Biosciences (RNA) achieve for del-desiran in Q2 2024?
