报告导读： 小核酸药物行业近期 BD 和数据催化密集，随着技术平台的成熟和适应症的不 断拓展，属于小核酸药物的治疗时代将逐步开启，我们重点关注 siRNA 药物在常见病大适 应症的商业价值。 本订阅号所载内容仅面向国泰海通证券研究服务签约客户。因本资料暂时无法设置访问限制，根据《证 券期货投资者适当性管理办法》的要求，若您并非国泰海通证券研究服务签约客户，为保证服务质量、 控制投资风险，还请取消关注，请勿订阅、接收或使用本订阅号中的任何信息。我们对由此给您造成的 不便表示诚挚歉意，非常感谢您的理解与配合！如有任何疑问，敬请按照文末联系方式与我们联系。 技术升级和产品获批推动平台成熟， siRNA 药物已从"罕见病"过渡到"常见病"。 小核酸药物是继小分子和抗体药物后的第三浪，具有靶点拓展能力强、研发 延展性强、药物作用长效且不易耐药等特性。小核酸药物的种类多样，其中 siRNA 药物的安全性和长效性更好，已成为国内药企的开发重点。自 2016 年 起，随着 GalNac 递送系统的升级和完全修饰的应用， siRNA 药物进入快速发展期，在罕见病领域验证其价值，目前逐步进入了常见病的研发兑现阶段，在 心血管疾病 ...

智通财经APP获悉，国泰海通发布研报称，小核酸药物作为继小分子和抗体药物后的第三浪，正通过技 术迭代实现从罕见病向常见病的跨越。随着GalNac递送系统升级和完全修饰技术的成熟，siRNA药物在 心血管疾病、慢乙肝、减重等大适应症领域进入研发兑现阶段，有望诞生重磅品种。中国企业可在龙头 的技术基础上快速迭代，在敲降效果、给药周期、注射方式等多方面取得差异化优势。该行认为未来的 破局或在于对新疾病领域，特别是过去难以成药的靶点的探索，以及肝外递送系统的突破。 国泰海通主要观点如下： 平台化特点显著，关注适应症的卡位和肝外递送的突破 当前的siRNA药物的产业化进程在于靶点和生物学机制清晰的常见病的商业化，虽然siRNA药物在搭建 好底层技术平台后分子PCC的速度非常快，但基于其长效化特性，该行认为该机制的药物仍然具有先发 优势(临床时间长)，率先卡位的企业仍然能获得较好的商业化收益或者对外合作的可能性。因GalNac递 送系统的效率和特异性高，且专利有突破空间，该行认为这个机制的药物在致病基因或机制明确的疾病 和肝内靶点的内卷程度会非常高，未来的破局或在于对新疾病领域，特别是过去难以成药的靶点的探 索，以及肝外 ...

Core Viewpoint - The strategic collaboration between 舶望制药 and Novartis aims to develop multiple cardiovascular products, with 舶望制药 receiving an upfront payment of $160 million and potential milestone payments totaling up to $5.2 billion [1][2] Group 1: Collaboration Details - 舶望制药 will grant Novartis rights to two early-stage siRNA molecules outside of China for treating severe hypertriglyceridemia (sHTG) and mixed dyslipidemia, along with priority negotiation rights for the BW-00112 (ANGPTL3) product, which is currently in Phase II clinical trials in the US and China [1] - A separate siRNA candidate in preclinical research will have exclusive licensing rights granted to Novartis outside of China, with plans to initiate Phase I clinical trials in 2026 [2] - The new agreement builds on a previous collaboration where 舶望制药 and Novartis established licensing agreements for multiple siRNA drugs, with milestone payments not exceeding $4.165 billion [2] Group 2: Company Vision and Market Position - 舶望制药 aims to become a global biotechnology company, focusing on developing best-in-class siRNA therapies and building a top-tier clinical development team across various regions [2] - The collaboration is expected to enhance 舶望制药's innovation engine and strengthen its influence in the liver and extrahepatic siRNA therapy market [2] - Novartis recognizes the long-acting siRNA technology as a significant advancement in the prevention and treatment of cardiovascular diseases, indicating a strong commitment to addressing unmet clinical needs through this partnership [2]

Core Viewpoint - 舶望制药 is considering an IPO in Hong Kong, aiming to raise approximately $300 million, potentially becoming the largest biopharmaceutical IPO in Hong Kong since 2024, aside from 恒瑞医药 [1] Company Overview - 舶望制药 is a clinical-stage biotechnology company focused on developing next-generation siRNA drugs to provide better treatment options for global patients [1] - The company utilizes its industry-leading RADS platform technology, which offers enhanced activity, durability, and safety in RNA molecules, to establish a diverse pipeline of siRNA candidates [1] Product Pipeline - 舶望制药's product pipeline addresses a wide range of indications, including cardiovascular diseases, rare diseases, viral infections, metabolic diseases, and central nervous system disorders [1] - Key products in clinical phase II include cardiovascular drugs BW-01 and BW-02, rare disease drug PKK, and hepatitis B drug HBV [1] Investment Background - 舶望制药 has undergone several rounds of financing, with investors including 杏泽资本, 正心谷资本, CPE源峰, 道远资本, 三一创新投资, and 金沙江联合创投 [1]

Core Viewpoint - 舶望制药 is considering an IPO in Hong Kong, aiming to raise approximately $300 million, potentially becoming the largest biopharmaceutical IPO in Hong Kong since 2024, following 恒瑞医药 [1] Company Overview - 舶望制药 is a clinical-stage biotechnology company focused on developing next-generation siRNA drugs to provide better treatment options for global patients [1] - The company utilizes its industry-leading RADS platform technology, which offers stronger activity, durability, and safety in RNA molecules, to establish a diverse pipeline of siRNA candidates [1] Product Pipeline - The product pipeline of 舶望制药 covers a wide range of indications, including cardiovascular diseases, rare diseases, viral infections, metabolic diseases, central nervous system disorders, heart, muscle, and fat [1] - Key products in clinical phase II include cardiovascular drugs BW-01 and BW-02, rare disease drug PKK, and hepatitis B drug HBV [1] Investment and Financing - 舶望制药 has undergone several rounds of financing, with investors including 杏泽资本, 正心谷资本, CPE源峰, 道远资本, 三一创新投资, and 金沙江联合创投 [1]

Market Performance - A-shares saw significant gains, with the Shanghai Composite Index rising by 1.7%, the Shenzhen Component increasing by 4.55%, and the ChiNext Index surging by 8.58%[1] - The Hong Kong market experienced a decline, with the Hang Seng Index closing down 0.98% at 25,270.07 points, and the Hang Seng Tech Index falling by 0.59%[1] Economic and Policy Developments - U.S. President Trump's envoy announced that U.S. leaders agreed to provide security assurances to Ukraine, similar to NATO's collective defense clause[12] - Trump indicated that semiconductor tariffs could reach as high as 300%, with an announcement expected in the coming weeks[12] Corporate Highlights - JD Health led the Hang Seng Index with a notable performance, reflecting strong interest in the healthcare sector[1] - The semiconductor sector showed strength amid ongoing discussions about tariffs and trade policies[1] Investment and Financial Trends - The total trading volume in the Hong Kong market surged to 31.27 billion HKD, indicating heightened trading activity[1] - OpenAI plans to invest trillions in AI infrastructure, signaling a significant commitment to technology development[12] Economic Indicators - China's economic performance in July showed the worst monthly results of the year, raising expectations for further stimulus measures from the government[12] - Fixed asset investment recorded its largest decline since the onset of the COVID-19 pandemic, highlighting the impact of high tariffs and domestic demand issues[12]

Group 1 - The core viewpoint is that starting from 2024, Chinese companies are accelerating the overseas expansion of their siRNA pipelines, while also proactively laying out the siRNA sector domestically, leading to increased transactions [1][4] - Novartis reported a significant increase in sales for its long-acting siRNA drug Leqvio, with sales rising from $0.12 million in 2021 to $7.54 million in 2024, and achieving $5.55 million in H1 2025, representing a 66% increase [2] - The development of siRNA drugs is complex and involves multiple dimensions such as nucleotide modification, sequence screening, target gene selection, delivery vector optimization, and breakthroughs in non-liver targeting, indicating substantial room for improvement [1][4] Group 2 - The siRNA drug development landscape is expanding, with a shift from rare diseases to chronic disease indications like hyperlipidemia, hepatitis B, and obesity, suggesting a rapidly opening market space [3][4] - In 2023, there were 13 global siRNA drug transactions, with Chinese companies leading in transaction numbers, indicating a strong presence in the siRNA market [4] - Recommended stocks benefiting from the siRNA sector include Yuyuan Pharmaceutical, Innovent Biologics, East China Pharmaceutical, and others, highlighting potential investment opportunities [5]

Financial Performance - The company's revenue decreased by 21.9% from RMB 8,983 million in 2024 Q1 to RMB 7,015 million in 2025 Q1[19] - Gross profit decreased by 27.6% from RMB 6,495 million in 2024 Q1 to RMB 4,705 million in 2025 Q1[19] - Gross profit margin decreased by 5.2 percentage points from 72.3% in 2024 Q1 to 67.1% in 2025 Q1[19] - Reported profit attributable to shareholders of the company decreased by 8.4% from RMB 1,613 million in 2024 Q1 to RMB 1,478 million in 2025 Q1[19] - Underlying profit attributable to shareholders of the company decreased by 18.2% from RMB 1,724 million in 24Q1 to RMB 1,411 million in 25Q1[19] - Finished drugs revenue decreased by 27.3% from RMB 7,561 million in 24Q1 to RMB 5,500 million in 25Q1[21] - Bulk vitamin C revenue increased by 25.0% from RMB 486 million in 24Q1 to RMB 608 million in 25Q1[21] - Bulk antibiotics revenue increased by 3.3% from RMB 449 million in 24Q1 to RMB 464 million in 25Q1[21] R&D and Pipeline - The company has 8 R&D platforms and over 200 innovative drugs and new formulations under development[4] - R&D expenses increased by 11.4% from RMB 1,169 million in 2024 Q1 to RMB 1,302 million in 2025 Q1[19] - The company has 5 R&D centers located in China and the U S[99] Business Development - Up to May 2025, a total of HK$300 million has been repurchased[8]

Core Viewpoint - The innovative pharmaceutical industry in China is experiencing a "triple resonance" of policy support, industry upgrades, and performance recovery, leading to a restructuring of capital market valuation logic [1] Group 1: Business Development (BD) Collaborations - In May 2025, China’s pharmaceutical industry saw significant BD announcements, including a $60.5 billion collaboration between 3SBio and Pfizer for the SSGJ-707 project [2] - Following this, Sinopharm announced a licensing agreement with Chengdu Guowei Biopharmaceutical for the AGT-siRNA drug GW906, with potential payments totaling up to 5.5 billion yuan [3] - The collaboration between Sinopharm and Guowei aims to enhance the company's pipeline in chronic disease treatment, particularly for primary hypertension [3] Group 2: Market Trends and Innovations - The siRNA technology is highlighted as a frontier in global innovative drug development, with companies like Alnylam leading the market [3][4] - The small nucleic acid market is driven by increasing penetration of existing drugs and unique advantages over traditional small molecules and monoclonal antibodies [5][6] - The success rate of siRNA drugs from project initiation to clinical phase III is approximately 60%, significantly higher than traditional drugs [6] Group 3: Valuation and Market Dynamics - The valuation logic for innovative drugs is evolving, focusing on "technology scarcity × market space × threat of alternative therapies" [7] - The average R&D cost for Chinese innovative drugs is 40%-60% lower than that of Western counterparts, with a reduced timeline from IND to BLA of 1.8 years [8] - In 2024, 228 BD transactions were recorded, with a 36% increase from 2023, indicating a growing trend in domestic and international collaborations [9] Group 4: Strategic Partnerships and Challenges - Large multinational companies are increasingly engaging in early-stage Chinese innovation projects, recognizing the potential for cost-effective assets and market opportunities [10] - The challenges in BD collaborations often stem from valuation discrepancies, with domestic firms focusing on R&D potential while foreign firms prioritize market size and regulatory predictability [10] - Companies are advised to enhance their core value and innovation capabilities while also focusing on intellectual property protection to navigate the complexities of international markets [11][12] Group 5: Future Outlook - The transformation of Chinese innovative pharmaceutical companies from "price takers" to "rule makers" is underway, emphasizing the importance of continuous technological innovation and rational value recognition [13]

Core Viewpoint - The article discusses the potential investment opportunities in small-cap biotech companies with market capitalizations below 3 billion, suggesting that a market recovery could lead to significant stock price increases for these companies [2][5]. Group 1: Market Trends - Many biotech stocks with market caps below 3 billion have experienced significant price increases since early 2025, indicating a trend of recovery from previous low valuations [2][5]. - The article highlights that the majority of small-cap biotech companies have cash reserves below the 600 million safety line, making it difficult for them to transition from negative to positive cash flow [5]. Group 2: Company Analysis - **Yiming Oncology (宜明昂科)**: Focused on CD47, currently in Phase 3 clinical trials. The company has 700 million in cash, sufficient for about two years of R&D [6]. - **Zhuanxin Biotech (荃信生物)**: Engaged in autoimmune therapies, currently has negligible cash reserves but aims to survive through business development (BD) models [8]. - **Jiahua Biotech (嘉和生物)**: Recently merged with Yiteng, has 1.1 billion in cash, positioning it to potentially return to the mid-cap biotech sector [10]. - **Tengsheng Biopharma (腾盛博药)**: Focused on hepatitis treatments, has 2.4 billion in net cash, but is reducing expenses and focusing on clinical trials [11]. - **Sanyecao Biotech (三叶草生物)**: Previously a star in the COVID vaccine space, now facing financial difficulties with only 500 million in cash [13]. - **Zhaoke Ophthalmology (兆科眼科)**: Has 1.3 billion in cash, focusing on eye care products, with a relatively low annual R&D expenditure [14]. - **Deqi Pharmaceuticals (德琪医药)**: Has seen a significant decrease in cash reserves from 3 billion to 700 million, indicating a need for better cash management [16]. - **Hualing Pharmaceuticals (华领医药)**: A stable company with 900 million in cash, focusing on diabetes treatments and low R&D spending [17].