小核酸疗法
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行业周报:减重新靶点初显锋芒,小核酸疗法前景可期-20260111
KAIYUAN SECURITIES· 2026-01-11 10:12
医药生物 ——行业周报 2026 年 01 月 11 日 投资评级:看好(维持) 行业走势图 -12% 0% 12% 24% 36% 48% 2025-01 2025-05 2025-09 2026-01 医药生物 沪深300 数据来源:聚源 相关研究报告 《"十五五"谋篇新蓝图,脑机接口进入 产 业 化 快 车 道 — 行 业 深 度 报 告 》 -2026.1.6 《创新驱动下,2025 年医药各细分赛 道牛股频出—行业周报》-2026.1.4 《瑞博生物通过港交所 IPO 聆讯,上 市在即—行业周报》-2025.12.28 减重新靶点初显锋芒,小核酸疗法前景可期 随着 WAVE 与 Arrowhead 小核酸临床数据相继读出,国内外正加速布局 INHBE 与 ALK7 两个靶点。INHBE 主要采用 GalNAc 技术进行肝靶向递送,海外已有 4 款药物进入临床阶段,国内大睿生物、圣因生物同靶点产品已进入 I 期临床,先 衍生物刚刚 IND 获批。ALK7 靶点小核酸药物涉及到肝外靶向,技术壁垒相对更 高,整体竞争格局相对更好;海外仅 Arrowhead 一款产品进入临床阶段,国内石 药集团、圣因生物、 ...
医药周报20251213:看好2026年四重潜在催化下的小核酸行情-20251213
Guolian Minsheng Securities· 2025-12-13 11:16
Investment Rating - The report maintains a "Buy" rating for the small nucleic acid sector, anticipating significant catalysts by 2026 [5]. Core Views - The report emphasizes three main investment themes for 2026: innovation, international expansion, and turnaround opportunities. It suggests focusing on innovative drugs, biotech companies that have gone global, and supply chain strategies [2][21]. - The small nucleic acid therapy market is expected to experience substantial growth, with FDA-approved drugs like Inclisiran and Vutrisiran projected to reach sales of $754 million and $971 million respectively by 2024 [3][16]. Summary by Sections 1. Small Nucleic Acid Market Outlook - The report highlights the commercial growth potential of small nucleic acid therapies, with significant sales forecasts for drugs like Inclisiran and Vutrisiran, and a projected market size of $30 billion for Novartis's xRNA therapies [3][16]. - The business development (BD) trend for siRNA therapies is improving, with notable deals such as a $4.165 billion agreement with Novartis [3][20]. - Clinical catalysts are expected, with Arrowhead's ARO-DIMER-PA set to announce trial results in H2 2026 [4][20]. - Increased capital recognition is noted, with companies like Rebio Biotech preparing for IPOs, indicating a positive market sentiment [4][20]. 2. Market Review and Trends - The report reviews the recent performance of the pharmaceutical sector, noting a 1.04% decline in the A-share pharmaceutical index, underperforming compared to the ChiNext and CSI 300 indices [24][28]. - The overall trading volume in the pharmaceutical sector was 423.35 billion yuan, accounting for 4.38% of the total market, below the historical average of 7.13% [43][24]. - The report identifies the best-performing sub-sector as medical services II, while the worst was pharmaceutical commerce II [35][38]. 3. Investment Recommendations - The report recommends focusing on high-quality siRNA companies in China, such as Frontier Biotech, Yuyuan Pharmaceutical, and others, as they are positioned for rapid development in the small nucleic acid space [7][21]. - It suggests monitoring the clinical progress of various siRNA drugs in development, particularly those targeting metabolic diseases [22][21].
海外小核酸行业动态跟踪Alnylam:业绩增长势头强劲,研发管线布局丰富
Ping An Securities· 2025-12-11 06:28
Investment Rating - The industry investment rating is "Outperform" compared to the market, indicating an expected performance greater than the CSI 300 index by more than 5% over the next six months [49]. Core Insights - Alnylam has shown strong revenue growth, with total revenue reaching $1.249 billion in Q3 2025, a year-over-year increase of 149%. Net product revenue was $851 million, up 103%, and revenue from collaborations and royalties surged by 393% to $398 million [2][7]. - The TTR product line is experiencing rapid growth, with Q3 2025 revenue of $724 million, a 135% increase year-over-year. The company has raised its full-year revenue guidance for TTR products to between $2.475 billion and $2.525 billion [16][29]. - Alnylam's research pipeline is robust, with seven drugs in Phase 3 clinical trials, including Nucresiran, which is expected to offer a better dosing regimen. The company is also exploring siRNA therapies in chronic disease areas [38][40]. Summary by Sections 1. Q3 2025 Performance Overview - In Q3 2025, Alnylam's total revenue was $1.249 billion, reflecting a 149% increase from the previous year. The net product revenue was $851 million, up 103%, while collaboration and royalty revenues reached $398 million, a 393% increase [2][7]. 2. Rapid Growth of TTR Products - The TTR product revenue for Q3 2025 was $724 million, marking a 135% year-over-year increase. The company has adjusted its full-year revenue guidance for TTR products to $2.475 billion to $2.525 billion, up from the previous estimate of $2.175 billion to $2.275 billion [16][29]. 3. Rich R&D Pipeline - Alnylam has a diverse R&D pipeline with seven drugs in Phase 3 trials. Nucresiran, a new generation TTR product, is expected to provide a more effective dosing schedule. The company is also advancing siRNA therapies in chronic disease management [38][40].
“A+H”队伍再扩容,三只松鼠、晶澳科技、赛力斯、剑桥科技拟赴港上市
Cai Jing Wang· 2025-04-29 09:42
Group 1: A+H Listing Expansion - The Hong Kong stock market is experiencing a surge in listing applications, with 14 companies submitting applications between April 25 and 28, including industry leaders seeking A+H listings [1] - Three Squirrels aims to become the first A+H snack brand, reporting a projected revenue of 10.622 billion yuan and a net profit of 408 million yuan for 2024, with 70% of revenue coming from online channels [2] - JA Solar has submitted its application for an A+H listing, reporting a projected revenue of 70.121 billion yuan for 2024, but facing a net loss of 4.656 billion yuan due to asset impairment losses [3][4] - Seres has successfully turned a profit in 2024, with a revenue of 1.45176 billion yuan and a net profit of 5.946 billion yuan, driven by increased sales of electric vehicles [5][6] Group 2: Companies Transitioning to Hong Kong - Rebio Biotech has submitted its application to the Hong Kong Stock Exchange after previously attempting to list on the A-share market, reporting a net loss of 437.3 million yuan in 2023 [8][9] - Daji Group, which focuses on innovative radiation surgery solutions, has also shifted its listing application to Hong Kong after previously applying for the Sci-Tech Innovation Board [10][11] - Hongyeji has submitted its application to the Hong Kong Stock Exchange after previously attempting to list on the Shenzhen Stock Exchange, reporting revenues of approximately 1.228 billion yuan in 2022 [12]
瑞博生物冲刺港交所:靶向FXI抗血栓siRNA药物临床II期,IPO前估值35.8亿元
IPO早知道· 2025-04-27 12:38
RBD4059为全球首款且临床开发进展最快的抗血栓siRNA药物。 本文为IPO早知道原创 作者|罗宾 瑞博生物目前拥有 6 款自研管线处于临床试验阶段,涉及 7 种适应症,其中 4 款处于 2 期临床试验 中,一款已进入 III 期临床试验。公司还有 20 个计划推进到临床开发阶段的临床前项目。 来源:公司招股书 核心产品 RBD4059 (靶向 FXI 的 siRNA )为全球首款、也是临床开发进展最快的用于治疗血栓性 疾病的 siRNA 药物 ,通过选择性抑制 FXI , RBD4059 可在不显著增加出血风险(传统抗凝剂的常 见局限性)的情况下降低血栓形成的风险,同时以低频率的给药产生持久的疗效,提高患者的依从 性。 同时,公司还在推进两款关键产品 RBD5044 及 RBD1016 。 RBD5044 是全球第二个进入临床开发 的靶向 APOC3 的 siRNA 。 APOC3 是一种在脂质代谢中起关键作用的蛋白质。而 RBD1016 凭借 其对 HBsAg 的强效和持久作用,是未来实现 CHB 功能性治愈的联合方法的重要支柱疗法,及治疗 CHD 的领先 siRNA 候选药物。 公司经过近二十年的专 ...
医健IPO解码丨科创板折戟后转战港股,瑞博生物能否借小核酸风口打开估值空间?
2 1 Shi Ji Jing Ji Bao Dao· 2025-04-27 10:58
Group 1: Company Overview - Suzhou Ribo BioTechnology Co., Ltd. (Ribo Bio) submitted its main board listing application to the Hong Kong Stock Exchange on April 25, marking a significant capital move after the termination of its Sci-Tech Innovation Board application in 2021 [1] - Founded in 2007, Ribo Bio is a global leader in the research and development of small nucleic acid drugs, particularly focusing on siRNA therapies, aiming to innovate treatment methods for cardiovascular, metabolic, kidney, and liver diseases [1][2] - The company has established a self-developed technology platform that covers all key aspects of small nucleic acid drug development, from drug delivery to chemical modification and multi-target drug design, supporting the entire lifecycle from early research to commercialization [1] Group 2: Market and Industry Trends - The small nucleic acid drug industry is rapidly evolving, with increasing competition from multinational companies and leading biotech firms developing similar or competing siRNA drugs [3] - The global small nucleic acid drug market is projected to grow from $2.7 billion in 2019 to $4.6 billion in 2023, with a compound annual growth rate (CAGR) of 14.3%, and is expected to reach $46.7 billion by 2033 at a CAGR of 26.1% [4] Group 3: Product Pipeline and Development - Ribo Bio has one of the largest siRNA drug pipelines globally, with six self-developed drug assets in clinical trials targeting seven indications related to cardiovascular, metabolic, kidney, and liver diseases, four of which are in Phase 2 trials [4] - The core product RBD4059, targeting FXI, is the world's first siRNA drug for treating thrombotic diseases, which is a leading cause of death globally, claiming over 10 million lives annually [6] - Ribo Bio is also advancing two key products, RBD5044 and RBD1016, which are significant in the treatment of lipid metabolism disorders and chronic hepatitis B, respectively [6] Group 4: Financial Performance - As of the end of 2023 and 2024, Ribo Bio reported net losses of RMB 437.3 million and RMB 281.5 million, respectively, with no revenue from commercial product sales [7][8] - The company anticipates substantial R&D expenditures as it progresses its clinical development plans, with R&D costs constituting 79.5% and 75.0% of total operating expenses for 2023 and 2024, respectively [9][10]