股 票 研 究 股票研究 /[Table_Date] 2025.10.10 小核酸，大时代，靶向治疗新纪元 [Table_Industry] 医药 siRNA 药物行业深度报告 | [姓名table_Authors] | 电话 | 邮箱 | 登记编号 | | --- | --- | --- | --- | | 彭娉(分析师) | 021-23185619 | pengping@gtht.com | S0880525040080 | | 余文心(分析师) | 021-38676666 | yuwenxin@gtht.com | S0880525040111 | 本报告导读： 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。 投资要点： [Table_Report] 相关报告 医药《景气延续，继续推荐创新药械产业链》 2025.10.09 医药《集采优化，持续看好创新药械产业链》 2025.09.21 医药《继续看好创新药械产业链》2025.09.14 医药《设备更新政策持续落地，医疗 ...

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 ...

Group 1 - The report highlights a marginal recovery in China's manufacturing PMI in September, indicating a rebound in production activities, with new orders and export orders showing upward trends [1] - The report suggests three main investment directions based on economic recovery and market liquidity: breakthroughs in technology AI, resonance between domestic and international markets, and the continued rise of undervalued assets [1] - In the A-share market, major indices maintained upward momentum in September, with the ChiNext index rising over 12% and net inflows from southbound funds reaching 172.65 billion yuan [3] Group 2 - The global small nucleic acid drug market is projected to grow at a compound annual growth rate of 26.1%, reaching 46.7 billion USD by 2033, driven by technological advancements and increasing clinical approvals [10] - The report details the clinical progress of small nucleic acid drugs in various therapeutic areas, indicating significant potential for growth in this sector [10] - The report recommends focusing on both international and domestic companies involved in small nucleic acid drugs, highlighting key players in the market [10] Group 3 - The report on Lian Microelectronics indicates a stable revenue growth of 14.18% year-on-year for H1 2025, driven by technological innovation and an integrated supply chain [12] - Despite the revenue growth, the company reported a significant increase in net losses, indicating ongoing profitability challenges [12] - The report emphasizes the importance of capacity expansion and integrated industry chain advantages for the company's stable operations [12] Group 4 - The report on Solon Technology highlights the opportunities created by electrification and domestic substitution trends in the nylon pipeline sector, with potential revenue growth driven by increased demand from electric vehicles [18] - The company has established long-term partnerships with over 30 automotive manufacturers, positioning itself well in the market [18] - Revenue projections for 2025-2027 indicate a growth trajectory, with expected revenues of 1.57 billion, 1.96 billion, and 2.39 billion yuan respectively [18] Group 5 - The report on Yuheng Pharmaceutical shows a decline in revenue by 9.97% in H1 2025, while net profit increased by 7.56%, indicating effective cost management [37] - The company has signed promotional agreements for new drug products, enhancing its market presence and potential revenue streams [37] - The introduction of Pemabet tablets is expected to strengthen the company's product pipeline and competitive position in the cardiovascular sector [38]

Core Viewpoint - Sanofi Pharmaceutical has signed a subscription agreement with four investors, including Huaxi Biotechnology, to issue approximately 17.35 million new shares at a price of HKD 12 per share, raising around HKD 208 million, which is crucial for addressing its commercialization challenges and funding needs [2][6]. Group 1: Investment Details - The share placement represents about 16.50% of the current issued share capital and is expected to account for approximately 14.16% of the total share capital post-placement [2]. - The subscription price reflects a discount of approximately 19.84% compared to Sanofi Pharmaceutical's closing price of HKD 14.97 per share on September 5 [2]. - Huaxi Biotechnology will hold a 9.44% stake in Sanofi Pharmaceutical, becoming its second-largest shareholder after the transaction [2][6]. Group 2: Company Background and Challenges - Sanofi Pharmaceutical, established in 2007 and listed on the Hong Kong Stock Exchange in 2021, focuses on RNAi technology for drug development, with its lead candidate STP705 targeting two tumor indications [2][6]. - The company has not yet commercialized any products and has been operating at a loss, with net losses narrowing from USD 216 million in 2021 to USD 50 million in 2024, but still recording a loss of USD 3.49 million in the first half of 2025 [6]. - As of June 30, 2025, the company's current assets were only USD 14 million, highlighting the need for sufficient funding to support clinical pipelines and market preparations [6]. Group 3: Strategic Implications for Huaxi Biotechnology - Huaxi Biotechnology's investment is not merely financial but a strategic move to position itself in the small nucleic acid and RNAi technology sectors, with potential applications in targeted fat reduction [7][8]. - The investment aims to open new growth avenues for Huaxi Biotechnology, which has faced declining revenues, with a 19.57% year-on-year drop in the first half of 2025 [8]. - The collaboration is expected to enhance clinical progress for Sanofi Pharmaceutical's core pipeline while also allowing Huaxi Biotechnology to explore further investments and partnerships if market and technology validations are successful [7][8]. Group 4: Market Context and Future Outlook - The investment reflects Huaxi Biotechnology's response to increasing competition in the cosmetic market, particularly in traditional areas like hyaluronic acid, which has seen a decline in growth [8][10]. - The strategic partnership aims to leverage Sanofi Pharmaceutical's innovative technology to create synergies, although challenges remain in integrating the distinct business models and technologies of both companies [14]. - The success of this collaboration will depend on the clinical validation of Sanofi Pharmaceutical's technology and the ability to commercialize products effectively [7][14].

Core Viewpoint - Jingyin Pharmaceutical has officially filed for an IPO in Hong Kong, with its prospectus now publicly available, marking a significant step in its growth strategy [1]. Company Overview - Jingyin Pharmaceutical was established in 2021 and focuses on leveraging the clinical and commercial potential of siRNA therapies. The company has developed a diverse product pipeline with three main candidates: FXI siRNA, Lp(a) siRNA, and INHBE siRNA, targeting coagulation disorders, cardiovascular metabolic diseases, and obesity, respectively. The market potential for these therapies is substantial [4]. Product Pipeline - The FXI siRNA is currently in Phase II clinical trials in Europe and has a collaboration agreement with CRISPR Therapeutics for joint development, sharing global rights on a 50:50 basis. This partnership includes a cash infusion of $25 million and $70 million in equity, along with potential milestone payments totaling $800 million [4][6]. - The Lp(a) siRNA is in Phase I clinical trials, showing over 95% suppression after 43 days at doses of 300mg and 600mg, with a sustained effect lasting at least 12 weeks. There is potential for a dosing regimen of once every six months or even annually [9]. - The INHBE siRNA is also part of the pipeline, with ongoing development efforts [4]. Financial Background - Since its inception, Jingyin Pharmaceutical has completed three rounds of financing, with the latest being a $47.5 million Series B2 round in May 2025, resulting in a post-money valuation of $252.5 million [6][7]. Technology Platform - The company utilizes a proprietary PEPR technology platform that employs unique chemical modification techniques, enhancing the efficacy and durability of target protein knockdown. This platform positions Jingyin Pharmaceutical favorably within the siRNA landscape [11][14]. Market Position - The emergence of small nucleic acids as a significant drug modality, alongside antibodies and small molecules, is expanding treatment options for various diseases, including genetic disorders and cardiovascular diseases. Jingyin Pharmaceutical's established siRNA technology platform and differentiated product pipeline may position it as a new benchmark in the international market for small nucleic acids [14].

Core Viewpoint - The article discusses the significant organizational changes and strategic shifts at Huaxi Biological, particularly focusing on the recent layoffs and the company's investment in Saint Nor Pharmaceutical, indicating a transition from a "traffic empire" to a "technology engine" in the biopharmaceutical sector [3][4][5]. Group 1: Layoff Turmoil - Huaxi Biological has undergone substantial layoffs and internal investigations, with teams like "Muscle Vitality" and "Mibelle" shrinking from over a hundred to just a few members, and compensation standards set at N+1 [4][6]. - CEO Zhao Yan has initiated a "clean-up" campaign, emphasizing zero tolerance for corruption and restructuring the company's power dynamics, leading to significant turnover in the executive team [4][5]. - The layoffs have created organizational instability, risking knowledge leakage and hindering product development and market responsiveness [6]. Group 2: Shift to Small Nucleic Acids - Huaxi Biological has invested approximately HKD 139 million to acquire a 9.44% stake in Saint Nor Pharmaceutical, aiming to leverage its RNA interference technology for weight loss treatments [8][9]. - Saint Nor is recognized as the first small nucleic acid drug company listed in the Asia-Pacific region, with a robust pipeline targeting various diseases, including cancer and metabolic disorders [9][10]. - The investment is seen as a strategic move to enter the innovative drug sector, with Huaxi planning to collaborate on clinical and commercial efforts for the STP705 targeted weight loss project [11]. Group 3: The Weight Loss Drug Market - The weight loss drug market is projected to grow significantly, with estimates suggesting it could reach CNY 800-1000 billion by 2030, capturing over one-third of the global market share [13][14]. - The increasing prevalence of obesity in China, with over 200 million individuals classified as obese, presents a substantial market opportunity for weight loss solutions [13]. - Regulatory support and advancements in drug technology are expected to facilitate market entry for new weight loss drugs, enhancing the competitive landscape [12][14]. Group 4: Future Market Dynamics - The article highlights the potential for the weight loss drug market to evolve into a comprehensive metabolic management ecosystem, integrating various therapeutic approaches and technologies [16]. - By 2035, advancements in drug delivery and treatment methodologies could significantly expand the market, with the potential for annual customer value to increase from CNY 10,000 to CNY 50,000 [16]. - The narrative emphasizes the need for stakeholders to view the weight loss drug market as part of a larger metabolic revolution, rather than merely a series of individual product launches [16].

Investment Rating - The report assigns a "Buy" rating for the company, marking it as the first recommendation [6][10]. Core Insights - The acquisition of Langyan Life will enhance the company's industrial layout, allowing it to achieve a "CRO + pharmaceutical industry" strategic positioning. Langyan Life specializes in the R&D, production, and sales of high-end chemical drugs and raw materials, which will synergize with the company's R&D services [6]. - The company’s self-developed STC007 injection is entering critical clinical phases, with promising results in its Phase II trials for postoperative pain and chronic kidney disease-related itching. The drug targets peripheral pain relief without central nervous system side effects, indicating a strong market potential [7]. - The company is also advancing in the small nucleic acid drug development sector, with two products in preclinical stages. One product, ABA001, is expected to enter IND status by 2026, indicating a forward-looking approach in drug development [8]. Financial Projections - The company forecasts revenues of 12.59 billion, 14.15 billion, and 16.15 billion yuan for 2025, 2026, and 2027 respectively, with corresponding EPS of 2.06, 2.41, and 2.96 yuan. The current stock price corresponds to PE ratios of 32.4, 27.7, and 22.5 for the same years [10][12]. - The projected net profit for 2025 is 231 million yuan, with a growth rate of 30% compared to the previous year. The net profit is expected to continue growing at rates of 16.9% and 22.9% in 2026 and 2027 respectively [12][13].

Group 1: Product Development and Clinical Trials - The company has a mature layout in the positive symptoms of schizophrenia, with ongoing projects NHL35700, NH300231, and NH140068 targeting negative symptoms [2][4] - NH600001 is expected to achieve a market scale of over 2 billion RMB after its launch, leveraging the advantages of etomidate while reducing adverse reactions [4] - NHL35700 is in the communication phase for its Phase III clinical trial with CDE, aiming for efficient advancement to benefit schizophrenia patients [6][8] Group 2: Market Strategy and Revenue Projections - The company has established online and offline retail divisions to expand its market presence, particularly for sleep-related products [5] - The revenue share from innovative drugs is projected to increase significantly with the upcoming launches, with a goal of one innovative drug being launched each year during the 14th Five-Year Plan [8][14] - The company anticipates a fundamental and sustainable improvement in operations starting next year with the approval of NH600001 [14] Group 3: Research and Development Focus - The company is exploring AI applications in drug development and aims to establish a system for early-stage research [12] - There is a focus on small nucleic acid drugs as a key development direction, with ongoing projects and collaborations in this area [13] - The company prioritizes unmet clinical needs in both domestic and international markets for its innovative drug projects [11] Group 4: Business Development and Licensing - The company plans to continue introducing differentiated projects from abroad and is in discussions for potential licensing-out opportunities [6][9] - The company is optimistic about the market potential for R-ketamine, which has completed Phase II trials and is preparing for Phase III [7] Group 5: Impact of Policy Changes - The company expects minimal impact from centralized procurement on its key products, with a strategic shift towards becoming an innovative drug-focused enterprise [14]

Investment Rating - The investment rating for the company is "Buy" (initial coverage) [2] Core Views - The company is focusing on innovative drug development, particularly in small nucleic acid technology and long-acting HIV treatments, which are expected to drive future growth [5][30] - The core product, Aikening (艾可宁), has shown stable sales growth and is positioned to expand its market presence, especially in the HIV treatment sector [7][26] - The company has a clear and stable shareholding structure, with a significant stock incentive plan aimed at enhancing long-term growth [16][21] Summary by Sections 1. Company Overview - Founded in 2013 and listed on the Shanghai Stock Exchange in 2020, the company has developed Aikening, a key HIV treatment, which was approved in 2018 [5][14] - The company is building a pipeline that combines innovative drugs and high-end generics, enhancing its competitive edge [5][30] 2. Financial Performance - Revenue has grown from 0.47 billion yuan in 2020 to an estimated 1.43 billion yuan in 2025, with a CAGR of approximately 29.1% [22] - The net profit attributable to shareholders is projected to improve from a loss of 329 million yuan in 2023 to a loss of 176 million yuan in 2025 [8][22] - The gross margin is expected to stabilize around 32.7% in 2025, reflecting improved operational efficiency [8][27] 3. Product Pipeline - The company is advancing its small nucleic acid drug pipeline, targeting chronic diseases with several candidates in clinical development, including treatments for IgA nephropathy and dyslipidemia [6][34] - Aikening is the first long-acting HIV drug approved globally, and the company is working on a comprehensive long-acting treatment regimen [7][30] 4. Strategic Initiatives - The company has launched a stock incentive plan to align the interests of management and shareholders, focusing on innovation and commercialization [21][19] - The research team is experienced, with a strong background in drug development, which supports the company's long-term innovation strategy [19][20]

Summary of Key Points from the Conference Call Industry Overview: Hong Kong Biotech Sector - The Hong Kong biotech sector has shown significant growth, achieving a revenue increase of approximately 10% in the first half of the year, aligning with A-share innovative drug companies [3] - The sector has achieved its first half-year profitability, with a turnaround of nearly 150%, resulting in a net profit margin of 3.5% [1][3] - The sector's sales expense ratio peaked at around 25% in 2023 but has since been optimized, focusing on core pipelines and reducing non-core projects [4] Financial Performance and Trends - The cash and cash equivalents of the sector have shown double-digit growth in the first half of the year, despite a slight decline from over 40 billion RMB in 2022 [4] - Monthly financing activities have seen double-digit growth, reversing last year's downward trend, which has strengthened the innovative drug pipeline [5] - The sector is expected to maintain a positive growth trajectory over the next 3-5 years, driven by increased business volume and active financing activities [6] Competitive Landscape - China holds a significant position in the global oncology drug innovation field, with its oncology pipeline accounting for 35% of the global total [7] - Chinese companies have improved their delivery ratios in large business development (BD) transactions, with several projects exceeding 50 million USD [8] - The Hong Kong biotech industry has seen a mix of button and direct licensing transactions, with notable large deals such as a nearly 4.7 billion USD transaction between Heber and AZ [9] Company Highlights - **HaploMed**: Achieved a revenue of 100 million USD and a net profit of 70 million USD in the first half of the year, with a long-term partnership with AZ that highlights its MICE platform advantages [2][12] - **Deqi Pharma**: Experienced a nearly tenfold increase in market value, focusing on two core pipeline platforms, with promising data from clinical trials [10] - **Overall Market Sentiment**: Companies like Rongchang and Basestone have shown strong performance post-financing, receiving positive feedback from the capital market [5] Future Outlook - The Hong Kong biotech sector is expected to continue its growth, with increased financing activities and enhanced research efficiency anticipated to contribute positively to performance [6] - The competitive advantages of Chinese innovative drug companies in the international market include a large number of oncology pipelines, enhanced delivery capabilities in large BD projects, and the ability to refresh transaction limits in emerging fields like small nucleic acid drugs [13]