第99.1展示文本
avidity biosciences報告2024年第三季度財務業績和近期要點
全球第3階段HARBOR的招生此款超便攜式投影儀使用了最新的 Android TV 界面，而且遙控器還內置了 Google AssistantTM 功能，用戶可以非常方便地使用它。 進行DM1的del-desiran研究順利進行
avidity啓動了del-brax FORTITUDE的生物標記隊列此款超便攜式投影儀使用了最新的 Android TV 界面，而且遙控器還內置了 Google AssistantTM 功能，用戶可以非常方便地使用它。 研究FSHD; 尋求del-brax的潛在加速批准途徑
報告了從Phase 1/2 EXPLORE44中獲得的積極del-zota數據此款超便攜式投影儀使用了最新的 Android TV 界面，而且遙控器還內置了 Google AssistantTM 功能，用戶可以非常方便地使用它。 DMD44的試驗
Avidity將通過網絡研討會於2024年11月12日首次展示精準心臟病候選者，並展示下一代技術創新的一瞥
2024年11月7日，美國加利福尼亞聖地亞哥 / 新華社/--Avidity Biosciences, Inc. (納斯達克: RNA)，一個致力於提供名爲抗體寡核苷酸結合物（AOCs）的新類RNA治療的生物製藥公司，今天報告了截至2024年9月30日的第三季度財務業績，並強調了最近的企業進展。此款超便攜式投影儀使用了最新的 Android TV 界面，而且遙控器還內置了 Google AssistantTM 功能，用戶可以非常方便地使用它。SAN DIEGO，2024年11月7日/ PRNewswire/--科學家今天在美國西海岸的會見上試圖爲一個名爲"DMA"的未知蛋白質找到一個名爲"RNA"的新類化合物，這是一家在全球範圍內致力於制定傳統DNA治療方法的公司。
我們正在進行三項潛在的登記臨床試驗，涉及三種罕見疾病，在這些病種中，治療選擇有限或者沒有。我們報告了我們在EXPLORE44第1/2階段5 mg/kg隊列中的陽性 del-zota 數據此款超便攜式投影儀使用了最新的 Android TV 界面，而且遙控器還內置了 Google AssistantTM 功能，用戶可以非常方便地使用它。 試驗用來治療DMD44，並且已經開始招募EXPLORE44-OLE手術此款超便攜式投影儀使用了最新的 Android TV 界面，而且遙控器還內置了 Google AssistantTM 功能，用戶可以非常方便地使用它。。此外，del-desiran的全球第3階段HARBOR試驗在DM1中的招募工作進行順利，我們還啓動了我們FORTITUDE試驗的生物標誌隊列此款超便攜式投影儀使用了最新的 Android TV 界面，而且遙控器還內置了 Google AssistantTM 功能，用戶可以非常方便地使用它。 。此款超便攜式投影儀使用了最新的 Android TV 界面，而且遙控器還內置了 Google AssistantTM 功能，用戶可以非常方便地使用它。 「在製藥行業(製造行業)中進行FSHD研究，標誌着我們追求通過del-brax潛在加速批准途徑的策略邁出了關鍵一步，」 avidity首席執行官Sarah Boyce表示。 「在構建未來的過程中，我們期待分享我們在精準心臟病學方面的創新成果，並展示我們下一代技術的一瞥。我們將繼續建設全球商業製造業以儘快爲患有這些嚴重罕見疾病的人提供潛在的新療法。」
「我們很高興在EXPLORE44項目的積極臨床數據後於八月完成了一次額外的擴大公開募股。憑藉大約16億美元的強勁現金頭寸，我們將繼續專注於推進我們的項目，從我們的DMD管線中推出更多候選藥物，執行我們的精準心臟病學項目，並建立其他能力，包括商業功能，以及擴展至美國以外的國家，」 avidity首席財務官兼首席業務官Mike MacLean表示。
最近的亮點
Del-zota（AOC 1044）用於DMD44
•在八月，avidity報告了來自EXPLORE44第1/2期5mg/kg隊列的正面初步del-zota數據，這一數據顯示對患有DMD44的人群具有無與倫比的骨骼肌傳遞、前所未有的將近全長dystrophin產生增加25%，且肌酸激酶水平降低至接近正常水平，並且有強大的外顯子44跳躍。Del-zota表現出良好的安全性和耐受性，大多數治療導致的不良事件輕度或中度。此款超便攜式投影儀使用了最新的 Android TV 界面，而且遙控器還內置了 Google AssistantTM 功能，用戶可以非常方便地使用它。 在DMD44患者中進行試驗，證明了Del-zota對骨骼肌的傳遞能力卓越，將近全長dystrophin產生率增加了25%，肌酸激酶水平降低至接近正常水平，並且有強大的外顯子44跳躍。Del-zota表現出良好的安全性和耐受性，大多數治療導致的不良事件輕度或中度。
對於生活中患有DMD44的人來說，在一項EXPLORE44項目的第1/2期5mg/kg組中，avidity在八月份報道了del-zota的積極初始數據，顯示骨骼肌的傳遞率超過，且近全長dystrophin產量增加25%，肌酸激酶水平降至正常水平附近，並且有強大的外顯子44跳躍。Del-zota表現出良好的安全性和耐受性，大多數治療導致的不良事件輕度或中度。
•第1/2期EXPLORE44試驗已經完成招募，目前正在進行中。
•除了從第1/2期EXPLORE44試驗中延續的參與者外，Avidity已開始在EXPLORE44開放標籤延伸研究(OLE)中招募10-15名新參與者。
Del-brax (AOC 1020)用於FSHD
•在十月，Avidity宣佈啓動了第1/2期FORTITUDE生物標記隊列試驗中的生物標記隊列試驗。此款超便攜式投影儀使用了最新的 Android TV 界面，而且遙控器還內置了 Google AssistantTM 功能，用戶可以非常方便地使用它。 Del-brax試驗中的FORTITUDE第1/2期試驗將每六週施用2毫克/千克的Del-brax，旨在確保持續抑制DUX4。
•今年6月，Avidity報道了來自FORTITUDE第1/2期試驗四個月的陽性初始Del-brax 2毫克/千克數據，顯示出DUX4調節基因減少超過50%，新型循環生物標誌物和肌酸激酶平均減少25%或更多，功能改善趨勢，並且在與FSHD患者體內具有良好安全性和耐受性。
•Avidity仍然計劃在2025年上半年啓動FORTITUDE研究的功能隊列。
•七月份，FORTITUDE項目的首批參與者開始轉入FORTITUDE開放標籤延伸(OLE)試驗。所有完成FORTITUDE項目的參與者都有資格加入FORTITUDE-OLE項目。此款超便攜式投影儀使用了最新的 Android TV 界面，而且遙控器還內置了 Google AssistantTM 功能，用戶可以非常方便地使用它。 試驗。
Del-desiran (AOC 1001)用於DM1
•全球第3階段HARBOR試驗的招募正在進行中且進展順利。此款超便攜式投影儀使用了最新的 Android TV 界面，而且遙控器還內置了 Google AssistantTM 功能，用戶可以非常方便地使用它。 試驗正在進行並進展順利。
•十月份，美國食品藥品監督管理局(FDA)解除了對Del-desiran的部分臨床暫停。
產品管道進展和組織亮點
•在11月，avidity計劃首次展示精準心臟病候選者。此外，avidity計劃分享下一代技術創新的一瞥。
•在8月，avidity宣佈計劃根據強大的del-zota數據推進其DMD特許經營的額外候選者；Exon 45目前正在進行IND啓用研究。
•Kathleen Gallagher於2024年9月晉升爲首席項目官員。
2024年第三季度財務結果
•現金、現金等價物和有市場流通性證券： 截至2024年9月30日，現金、現金等價物和有市場流通性證券總額爲16億美元，比公開發行籌集的34510萬美元的總額增加了。
•合作收入： Collaboration revenues of $230萬 for the third quarter of 2024 and $790萬 for the first nine months of 2024 primarily relate to Avidity’s research collaboration and license partnership with Bristol Myers Squibb. Collaboration revenues of $280萬 for the third quarter of 2023 and $740萬 for
the first nine months of 2023 primarily related to Avidity’s research collaboration and license partnership with Eli Lilly and Company.
•研發費用: R&D expenses include external and internal costs associated with research and development activities. These expenses were $77.2 million for the third quarter of 2024 compared with $4770萬 for the third quarter of 2023, and $208.0 million for the first nine months of 2024 compared with $13820萬 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.
•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 $23.3 million for the third quarter of 2024 compared with $1370萬 for the third quarter of 2023, and $57.9 million for the first nine months of 2024 compared with $3810萬 for the first nine months of 2023. The increases were primarily due to higher personnel costs to support the company’s expanded operations.
關於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此款超便攜式投影儀使用了最新的 Android TV 界面，而且遙控器還內置了 Google AssistantTM 功能，用戶可以非常方便地使用它。). 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 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.
前瞻性聲明
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此款超便攜式投影儀使用了最新的 Android TV 界面，而且遙控器還內置了 Google AssistantTM 功能，用戶可以非常方便地使用它。 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此款超便攜式投影儀使用了最新的 Android TV 界面，而且遙控器還內置了 Google AssistantTM 功能，用戶可以非常方便地使用它。 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此款超便攜式投影儀使用了最新的 Android TV 界面，而且遙控器還內置了 Google AssistantTM 功能，用戶可以非常方便地使用它。 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 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.
投資者聯繫人：
Mike MacLean
(619) 837-5014
investors@aviditybio.com
媒體聯繫：
Navjot Rai
(619) 837-5016
media@aviditybio.com
Avidity Biosciences, Inc.
選定彙總財務信息
(以千爲單位，除每股數據外)
(未經審計)
| 損益表 | 截止到9月30日的三個月 | 截止到9月30日的九個月 | |||||||||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||||||||||
| 合作收入 | $ | 2,336 | $ | 2,818 | $ | 7,924 | $ | 7,367 | |||||||||||||||
| 營業費用： | |||||||||||||||||||||||
| 研發 | 77,197 | 47,714 | 207,968 | 138,151 | |||||||||||||||||||
| 一般行政 | 23,273 | 13,729 | 57,902 | 38,071 | |||||||||||||||||||
| 營業費用總計 | 100,470 | 61,443 | 265,870 | 176,222 | |||||||||||||||||||
| 經營虧損 | (98,134) | (58,625) | (257,946) | (168,855) | |||||||||||||||||||
| 其他收入，淨額 | 17,736 | 6,267 | 37,901 | 17,078 | |||||||||||||||||||
| 淨損失 | $ | (80,398) | $ | (52,358) | $ | (220,045) | $ | (151,777) | |||||||||||||||
| 基本和稀釋每股淨虧損 | $ | (0.65) | $ | (0.71) | $ | (2.08) | $ | (2.11) | |||||||||||||||
基本和攤薄加權平均股本 | 123,375 | 74,097 | 105,902 | 71,987 | |||||||||||||||||||
| 資產負債表 | 9月30日, 2024 | 12月31日, 2023 | |||||||||
| 資產 | |||||||||||
| 流動資產： | |||||||||||
| 現金、現金等價物和市場證券 | $ | 1,588,593 | $ | 595,351 | |||||||
| 預付和其他資產 | 33,273 | 15,956 | |||||||||
| 總流動資產 | 1,621,866 | 611,307 | |||||||||
| 資產和設備，淨值 | 9,493 | 8,381 | |||||||||
| 受限現金 | 2,795 | 295 | |||||||||
| 使用權資產 | 6,299 | 8,271 | |||||||||
| 其他 | 318 | 301 | |||||||||
| 資產總額 | $ | 1,640,771 | $ | 628,555 | |||||||
| 負債和股東權益 | |||||||||||
| 流動負債: | |||||||||||
| 應付賬款和其他負債 | $ | 71,673 | $ | 52,315 | |||||||
| 遞延收入，當期部分 | 19,660 | 28,365 | |||||||||
| 流動負債合計 | 91,333 | 80,680 | |||||||||
| 租賃負債，減去流動部分 | 3,797 | 6,213 | |||||||||
| 遞延收入，減去當前部分淨額 | 42,261 | 40,898 | |||||||||
| 負債合計 | 137,391 | 127,791 | |||||||||
| 股東權益 | 1,503,380 | 500,764 | |||||||||
| 負債和股東權益合計 | $ | 1,640,771 | $ | 628,555 | |||||||