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 ...

Switzerland-based global healthcare company Novartis (NVS) is gaining momentum this year, thanks to solid pipeline execution, new regulatory wins, high-value license transactions, capital return to shareholders, and positive market sentiment. Novartis stock has surged 36.3% year-to-date, outperforming the broader market. Following Sandoz’s (SDZNY) 2023 spinoff, Novartis is a pure-play innovative medicines firm focused on high-value therapeutic areas. More News from Barchart The company is scheduled to r ...

Key Takeaways Pfizer's deal with Trump cuts drug prices, boosts U.S. investment and lifts pharma sector sentiment.Investor optimism grows as major drugmakers eye similar tariff-free pricing agreements with Trump.J&J, Bayer and Novartis show strong growth momentum, making them attractive pharma investment picks.Late last month, Pfizer (PFE) announced a landmark deal with the Trump administration to cut drug prices and expand U.S. innovation and manufacturing.Under the deal, Pfizer agreed to slash prices of s ...

Industry Overview - The drug and biotech sector is currently facing multiple challenges, including potential tariffs on pharmaceutical imports, pipeline setbacks, and regulatory risks [1][2][10] - Despite these challenges, the industry's focus on innovation and positive developments in drug pipelines suggests a favorable long-term outlook [2][5] Financial Performance - The drug and biotech sector had a better-than-expected second quarter, with most large drugmakers reporting strong quarterly results and optimism for continued growth in the second half of 2025 [2] - The Zacks Large Cap Pharmaceuticals industry has collectively risen 1.1% year to date, outperforming the Zacks Medical Sector's decline of 0.5%, but underperforming the S&P 500's rise of 12.0% [15] Valuation Metrics - The industry is currently trading at a forward 12-month price-to-earnings (P/E) ratio of 14.71X, compared to the S&P 500's 22.95X and the Zacks Medical Sector's 19.36X [18] Key Players - Eli Lilly (LLY), Johnson & Johnson (JNJ), Novartis (NVS), Pfizer (PFE), and Bayer (BAYRY) are highlighted as strong candidates for investment due to their robust revenue streams and profitability [3] - Johnson & Johnson's Innovative Medicine unit saw a 2.4% sales increase in the first half of 2025, driven by key products and new drug launches [21] - Bayer's growth is fueled by key drugs like Nubeqa and Kerendia, with plans to launch new drugs in 2025 [27] - Pfizer has strengthened its oncology position with the acquisition of Seagen and is focusing on cost cuts to save $7.7 billion by the end of 2027 [32][34] - Novartis maintains strong momentum with a diverse portfolio and is solidifying its presence in gene therapy, despite facing generic competition [38] Innovation and M&A Activity - The sector is characterized by aggressive mergers and acquisitions (M&A), with large pharmaceutical companies acquiring innovative small and mid-cap biotech firms to expand their pipelines [7][8] - Recent notable M&A deals include Sanofi's acquisition of Blueprint Medicines for approximately $9.5 billion and Merck's offer to acquire Verona Pharma for around $10 billion [9] Future Outlook - The industry's focus on innovation, particularly in areas like rare diseases, oncology, and obesity, is attracting investor attention and is expected to drive growth [6][8] - Continuous investment in R&D and the adoption of new technologies such as AI and gene editing are seen as key to maintaining competitive advantage [5]

Investment Rating - The report indicates a strong interest in the small nucleic acid drug sector, particularly through acquisitions and partnerships, highlighting the potential for significant growth in this area [3][5][15]. Core Insights - Novartis is focusing on expanding its pipeline in rare disease treatments by acquiring Avidity Biosciences, a leader in the Antibody-Oligonucleotide Conjugates (AOC) field, which is valued at nearly $6 billion [3][11]. - The small nucleic acid drugs, including siRNA, ASO, and miRNA, are gaining traction due to their ability to target traditionally "undruggable" disease-causing genes, offering breakthrough treatment options for genetic, rare, and chronic diseases [4][9]. - The report emphasizes the importance of delivery technologies for small nucleic acid drugs, particularly the need for effective delivery systems to target tissues outside the liver, as 90% of disease-causing genes are expressed in non-liver tissues [8][9]. Summary by Sections Acquisition and Market Dynamics - Novartis has been actively acquiring companies to fill the pipeline gap created by upcoming patent expirations of key drugs, such as Entresto, which is projected to generate $7.822 billion in sales in 2024 [5][6]. - The report outlines several significant acquisitions in the small nucleic acid space, including Novartis's $9.7 billion acquisition of The Medicines Company and a $1.7 billion deal for Regulus Therapeutics [6][12]. Delivery Technology and Challenges - The report discusses the challenges faced by small nucleic acid drugs in terms of delivery, particularly the degradation by nucleases and the difficulty in penetrating the blood-brain barrier [7][8]. - Innovations in delivery systems, such as the FALCON platform by DTx Pharma, are highlighted as crucial for advancing the application of RNA therapies beyond liver-targeted treatments [8][10]. Competitive Landscape - The competitive landscape is characterized by major pharmaceutical companies, including Sanofi, Pfizer, and Eli Lilly, making significant investments in small nucleic acid drug technologies to capture the emerging market [6][10]. - Avidity Biosciences is noted as a key player with multiple clinical-stage AOC pipelines, including a breakthrough therapy for Duchenne Muscular Dystrophy [12][13]. Regulatory and Funding Environment - The report mentions supportive policies from the Chinese government aimed at promoting RNA and small nucleic acid drug development, including funding initiatives for innovative research [14][15]. - Recent financing activities in the small nucleic acid drug sector indicate growing investor interest, with several companies securing substantial funding to advance their research and development efforts [16].

Core Insights - Novartis will present data from 19 abstracts related to its cardiovascular portfolio at the 2025 European Society of Cardiology Congress in Madrid from August 29 to September 1, 2025 [2][3] - The company aims to showcase innovative treatments that can significantly improve cardiovascular outcomes, focusing on established treatments like Entresto and Leqvio, as well as pipeline assets such as pelacarsen and abelacimab [3][7] Presentation Highlights - Pelacarsen will be featured in multiple presentations, including its effect on reducing the need for lipoprotein apheresis in patients with elevated lipoprotein(a) and cardiovascular disease [4][5] - Leqvio's impact on patient quality of life will be highlighted in two VictORION studies, along with its efficacy as a cholesterol-lowering monotherapy [5][6] - The PARACHUTE-HF study will evaluate the efficacy and safety of Entresto in treating heart failure with reduced ejection fraction due to chronic Chagas disease [5][6] Research Focus - Novartis is dedicated to addressing significant unmet needs in cardiovascular disease through advanced science and technology, particularly in genetically driven risk factors and common heart conditions [7][10] - The company collaborates with patients, healthcare professionals, and organizations globally to enhance cardiovascular care beyond medication [8][10]

Core Insights - Alnylam Pharmaceuticals reported second-quarter 2025 earnings of $0.32 per share, significantly surpassing the Zacks Consensus Estimate of a loss of $0.03, primarily due to increased revenues from its lead drug, Amvuttra [1][11] - Total revenues for the quarter reached $773.7 million, exceeding the Zacks Consensus Estimate of $673 million, and reflecting a 17% year-over-year increase [2] - The company's net product revenues were $672.2 million, a 64% increase year-over-year, driven by strong demand for Amvuttra, Givlaari, and Oxlumo [3] Revenue Breakdown - Alnylam's net product revenues from Amvuttra amounted to $492 million, representing a 114% increase year-over-year, aided by expanded label use and patient switches from Onpattro [8][11] - Givlaari generated sales of $80.8 million, a 30% year-over-year increase, while Oxlumo recorded revenues of $46.9 million, up 15% year-over-year [9] - Net revenues from collaborators were $61.5 million, down 73% from the previous year, primarily due to a significant milestone payment received in the prior quarter [4] Financial Guidance - Alnylam raised its 2025 product revenue guidance to a range of $2.65 billion to $2.8 billion, up from the previous range of $2.05 billion to $2.25 billion [11][16] - The guidance for net revenues from collaborations and royalties remains unchanged at $650 million to $750 million [16] Research and Development - Adjusted R&D expenses increased by 11% year-over-year to $274.1 million, reflecting costs associated with new studies in partnership with Roche [13] - Adjusted SG&A expenses rose by 26% year-over-year to $261.2 million, driven by higher employee compensation and marketing investments related to Amvuttra [14] Cash Position - As of June 30, 2025, Alnylam's cash, cash equivalents, and marketable securities totaled $2.86 billion, an increase from $2.63 billion as of March 31, 2025, primarily due to net cash inflows from operating activities [15]

Group 1: Macro Economic Overview - Domestic industrial product prices are showing strong performance, with fluctuations in supply and demand impacting the market [4][5] - Construction activity remains at a seasonal low, with recent weeks showing a decline in operational rates for asphalt and cement, although there are signs of stabilization [4] - The demand side shows weakness in construction, while automotive and home appliance demand is fluctuating [4][6] Group 2: Investment Strategy - Central Huijin has significantly increased its holdings in major ETFs, exceeding 190 billion yuan, indicating strong market resilience and risk appetite [10][11] - The market is experiencing a structural shift with long-term funds playing a crucial role in stabilizing market expectations and supporting liquidity [11][12] - The focus on core assets remains essential as the market experiences rapid sector rotation, with policy-driven support expected to continue [13] Group 3: Institutional Research Trends - There has been a decline in overall institutional research activity, but interest in sectors such as computing, media, and coal has increased [15][17] - Specific companies like Ice Wheel Environment and Dongfang Tantalum are gaining attention due to their market positioning and growth potential [21] Group 4: Chemical Industry Insights - The chemical industry is facing increased competition, necessitating a "de-involution" approach to improve profitability and market conditions [48][49] - Domestic demand is expected to stabilize, but export growth may be limited due to international trade challenges [49][50] Group 5: AI and Entertainment Sector - The gaming, music, and entertainment sectors are experiencing high growth, with companies like Heartbeat and Dreamland seeing significant revenue increases [37][38] - The rise of AI applications in music and entertainment is expected to enhance user engagement and market opportunities [40] Group 6: Robotics Industry Developments - Yushutech is leading in the robotics sector, particularly in dog robots and humanoid robots, with a valuation of 12 billion yuan following recent funding rounds [42][44] - The company is focusing on self-developed core hardware and advanced control systems to enhance product performance and market competitiveness [43][44] Group 7: Pharmaceutical Sector Growth - The siRNA drug market is rapidly developing, with significant sales growth reported by companies like Novartis, indicating strong potential for domestic pharmaceutical firms [53][54] - The expansion of siRNA applications into chronic disease areas presents a promising market opportunity for future growth [54]

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]

Q2 2025 Financial Performance - Novartis delivered double-digit sales growth and core margin expansion in Q2 2025 [134] - Q2 sales reached USD 141 billion, an increase of 11% in constant currencies (cc) compared to Q2 2024 [14] - Core operating income for Q2 2025 was USD 59 billion, a 21% increase in constant currencies (cc) compared to Q2 2024 [14] - Core margin improved to 422%, a 340 basis points increase [14] - Free cash flow for H1 2025 increased by 46% to USD 97 billion [104] FY 2025 Guidance and Capital Allocation - FY 2025 core operating income guidance upgraded to low-teens growth from low double-digit [15] - A new share buyback program of up to USD 10 billion was initiated, to be completed by the end of 2027 [108] - The company assumes Entresto US generic entry in mid-2025 for forecasting purposes [112] Key Products Performance - Kisqali grew by 64% cc in Q2, achieving TRx leadership in mBC [23] - Kesimpta grew by 33% cc in Q2, driven by strong demand growth [31] - Pluvicto grew by 30% cc in Q2, with promising uptake since pre-taxane indication approval in the US [36] - Leqvio grew by 61% cc in Q2, on track for blockbuster status in 2025 [45] - Scemblix grew by 79% cc in Q2, with strong momentum in early lines and global leadership in 3L+ CML [52]