靖因药业由OrbiMed Entities及Creacion Ventures创立。自成立以来，公司已完成三轮融资，包括A轮融资、B轮融资及B2轮融资，募集资金总额约为1.44亿美 元。最新一轮融资后，其估值为2.53亿美元（约为人民币18亿元）。 瑞财经 刘治颖 9月28日，Sirius Therapeutics-B（ 靖因药业）向港交所递交上市申请，联席保荐人为高盛、海通国际、HSBC。 招股书显示，靖因药业是一家全球临床阶段生物技术公司，致力于充分发挥siRNA疗法的临床及商业价值。公司正重点推进三大潜在重磅产品管线组合：凝 血功能障碍类疾病、心脏代谢疾病与肥胖症。这些产品管线组合的布局由以下主力管线产品为代表： SRSD107作为靖因药业的核心产品，是一款潜在同类首创的靶向凝血因子XI的siRNA药物，目前正于欧洲开展II期多中心临床试验，并计划在中国及澳大利 亚╱新西兰启动另一个II期试验；该产品通过与CRISPR Therapeutics（纳斯达克：CRSP）建立的全球共同开发及商业化合作推进。SRSD216是一款潜在同类 最佳靶向Lp(a)的siRNA，当前同步在中国与美国进行IIa期试验；及S ...

siRNA疗法能够从核酸层面解决疾病机制，可以针对传统技术难以靶向的"无法成药"蛋白。siRNA疗法 为慢性病管理带来范式转变，具有更长持久性、更高依从性、良好安全性及更低药物相互作用的优势。 公司的三大核心产品管线专注于凝血功能障碍类疾病、心脏代谢疾病与肥胖症。SRSD107是一款潜在同 类首创靶向凝血因子XI的siRNA药物，已在欧洲开展II期临床试验。SRSD216是一款潜在同类最佳靶向 Lp(a)的siRNA，正在中国和美国进行IIa期试验。SRSD384是一种针对肥胖症的INHBE靶向候选药物， 拥有令人信服的临床前数据，正积极推进IND申报。 靖因药业是一家全球临床阶段生物技术公司，致力于开发siRNA疗法，并已提交港交所上市申请，高 盛、海通国际、HSBC为其联席保荐人。 公司自主研发的PEPR平台涵盖化学修饰、靶点与序列优化、递送技术等，并正在研究肝外递送系统， 以拓展siRNA作用范围。 siRNA疗法市场前景广阔，预计将从2024年的24亿美元增长到2040年的503亿美元，年复合增长率为 20.9%。 ...

据港交所9月28日披露，靖因药业向港交所主板提交上市申请书，高盛、海通国际、HSBC为其联席保荐人。 siRNA疗法的开发标志着医学领域直接从核酸层面解决疾病机制的根本性转变。这种上游机制使得siRNA可针对人体 几乎所有蛋白质研发抑制性药物，包括传统技术难以靶向的"无法成药"蛋白。 siRNA疗法为慢性病管理带来范式转变，具备三项确定性优势。即更长持久性与更高依从性、良好的安全性特征、更 低的药物相互作用与肾功能损伤。 公司正重点推进三大潜在重磅产品管线组合包括，凝血功能障碍类疾病、心脏代谢疾病与肥胖症，这些领域的战略选 择基于其巨大的市场潜力及公司提供变革性siRNA解决方案的能力。2024年，这些治疗领域各自拥有至少一款全球年 度销售额超100亿美元的三大畅销药物及年销售额达数十亿美元的其他多种药物。 主力管线产品代表上，SRSD107作为公司核心产品，是一款潜在同类首创的靶向凝血因子XI的siRNA药物，目前正于 欧洲开展II期多中心临床试验，并计划在中国及澳大利亚/新西兰启动另一个II期试验；SRSD216是一款潜在同类最佳 靶向Lp(a)的siRNA，当前同步在中国与美国进行IIa期试验；及SRS ...

公司正重点推进三大潜在重磅产品管线组合：凝血功能障碍类疾病、心脏代谢疾病与肥胖症，这些领域 的战略选择基于其巨大的市场潜力及公司提供变革性siRNA解决方案的能力。2024年，这些治疗领域各 自拥有至少一款全球年度销售额超100亿美元的三大畅销药物及年销售额达数十亿美元的其他多种药 物。这些产品管线组合的布局由以下主力管线产品为代表：SRSD107作为公司的核心产品，是一款潜在 同类首创的靶向凝血因子XI的siRNA药物，目前正于欧洲开展II期多中心临床试验，并计划在中国及澳 大利亚╱新西兰启动另一个II期试验；该产品通过与CRISPR Therapeutics(纳斯达克：CRSP)建立的全球 共同开发及商业化合作推进。SRSD216是一款潜在同类最佳靶向Lp(a)的siRNA，当前同步在中国与美国 进行IIa期试验；及SRSD384是一款针对肥胖症的INHBE靶向候选药物，拥有令人信服的临床前数据， 目前正积极推进IND申报。 公司由一支跨太平洋领导团队引领，汇聚全球战略视野、深厚的siRNA技术专长以及将候选药物从发现 推进至商业化的成熟能力。公司由OrbiMed Entities及Creacion Ve ...

Core Viewpoint - Suzhou Rebio Biotechnology Co., Ltd. is attempting to re-enter the capital market by submitting a prospectus to the Hong Kong Stock Exchange after failing to launch on the STAR Market four years ago, despite being one of the earliest companies in China to focus on small RNA drugs [1][3] Group 1: Company Overview - Founded in 2007, Rebio focuses on developing innovative therapies for cardiovascular, metabolic, renal, and liver diseases, particularly siRNA therapies [1] - The company has six self-developed drug pipelines currently in clinical trials, with four in Phase II and one in Phase III [1] Group 2: R&D Progress and Challenges - The fastest progressing pipeline, RBD1007, completed international Phase II/III research data analysis in 2021 but has not yet resulted in a marketable product [3] - Previous key pipelines RBD4988 and RBD1006 are not mentioned in the latest prospectus, indicating they may have "failed" [3] - The new core pipelines include RBD4059 for thrombotic diseases, RBD5044 for hypertriglyceridemia, and RBD1016 for chronic hepatitis B and D, all currently in Phase II, facing significant uncertainty regarding clinical efficacy and market viability [3][4] Group 3: Market Competition - Rebio's R&D progress is lagging behind competitors, with its core product RBD4059 in Phase II while similar products from Novartis have reached Phase III [4] - Ionis's APOC3-targeted drug has already been launched, raising doubts about Rebio's "Best-in-Class" potential for RBD5044, which has yet to release clinical data [4] Group 4: Financial Performance and Valuation - Rebio's valuation has fluctuated significantly, from 456 million yuan in 2015 to 3.15 billion yuan in 2020, peaking at 4.87 billion yuan in 2024, but has since dropped back to 3.58 billion yuan, equivalent to its valuation five years ago [5][6] - In 2023, the company reported revenue of only 44,000 yuan, which is expected to rise to 143 million yuan in 2024 through a partnership with Boehringer Ingelheim, but 90% of this revenue relies on a single partner, indicating a fragile income structure [6] - Total R&D expenses for 2023 and 2024 are projected to be 590 million yuan, with cumulative net losses reaching 718 million yuan, and cash reserves expected to be only 168 million yuan by the end of 2024, against interest-bearing loans of 399 million yuan, putting significant pressure on the company's cash flow [6] Group 5: Strategic Partnerships and Risks - Rebio has frequently utilized licensing agreements to alleviate financial pressure, including a deal with Boehringer Ingelheim worth over $2 billion and a licensing agreement for RBD7022 in Greater China for over 700 million yuan [7] - While these partnerships provide short-term cash flow, they may compromise future value, as the agreements do not clearly define patent ownership improvements, potentially limiting Rebio's share in future developments based on its core delivery technology [7]

Financial Data and Key Metrics Changes - In Q1 2025, the company achieved total net product revenue of $612 million, representing a 70% growth year-over-year [6][38] - Elevitus sales reached $375 million, marking a 180% increase compared to the same quarter last year [6][38] - The PMO franchise generated $237 million, up 5% year-over-year [6][38] - The company revised its net product revenue guidance for 2025 to a range of $2.3 billion to $2.6 billion, reflecting a 37% increase from 2024 at the midpoint [7][42] Business Line Data and Key Metrics Changes - The PMO franchise grew by 5%, while Elevitus saw a significant increase of 180% in sales [6][38] - Collaboration and other revenues amounted to $133 million, primarily from Roche [38] Market Data and Key Metrics Changes - The company noted that 60% of current revenue comes from top sites, which are experiencing high demand and long wait times for appointments [12] - The administrative process for gene therapy has become more complex, leading to longer turnaround times for infusions [10][18] Company Strategy and Development Direction - The company is focusing on addressing administrative challenges and enhancing site capacity to improve patient access to Elevitus [19] - A comprehensive promotional campaign for Elevitus was launched to increase awareness and understanding among healthcare providers and patients [20] - The company aims to expand its treatment network by engaging more community sites to alleviate capacity issues at top centers [19] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the challenges faced in Q1 2025, including a severe flu season and a tragic safety event that impacted patient infusions [5][16] - The company remains optimistic about long-term demand for Elevitus, expecting a recovery in sales starting in the summer [17][64] - Management emphasized the importance of education and communication to address concerns stemming from the recent safety event [52][60] Other Important Information - The company reported a GAAP net loss of $448 million for Q1 2025, primarily due to increased R&D expenses related to a collaboration with Arrowhead [41] - The company has a strong cash position with $647 million in cash and equivalents, plus an additional $600 million available through a revolving credit facility [36][42] Q&A Session Summary Question: What has been the biggest driver of potential downside pressure leading to revised guidance? - Management indicated that it is a mix of all three factors: cycle times, administrative processes, and the safety event, with cycle times being the most mechanically impactful [47][48] Question: How does the company direct patients to sites with more capacity? - Management clarified that it is more about focus and education rather than the number of sites, emphasizing the need to engage secondary sites more effectively [57][58] Question: What percentage of sales does Elevitus represent in the new guidance? - Management confirmed that the revision in total net product revenue was exclusively related to Elevitus, which is the primary driver of the guidance change [66] Question: What are the expectations regarding the potential label update for Elevitus? - Management stated that a labeling supplement was submitted in April, with a target review date set for no later than Q4 2025 [76]