注：如提及个股仅供参考，不代表投资建议。指数/基金短期涨跌幅及历史表现仅供分析参考，不 预示未来表现。市场观点随市场环境变化而变动，不构成任何投资建议或承诺。文中提及指数仅供参 考，不构成任何投资建议，也不构成对基金业绩的预测和保证。如需购买相关基金产品，请选择与风险 等级相匹配的产品。基金有风险，投资需谨慎。 华福证券指出，2025年小核酸行业迎来首个爆发周期，商业化进展和新平台验证均超出市场预期。 Alnylam公司治疗ATTR的大单品AMVUTTRA今年有望成为首个销售额突破20亿美元的小核酸药物； Ionis治疗sHTG的大单品在9月读出阳性数据后股价大涨35%，其2026年关键3期数据和商业化进展值得 关注。Arrowhead依托领先的肝外Trim平台，重点布局减肥和CNS项目，并再次将授权项目给诺华，11 月迎来首个治疗FCS商业化产品Plozasiran，想象空间巨大。小核酸药物商业化加速带动相关原料和外包 研发需求持续增长。当前医药板块情绪较低，但临近12月有ASH会议数据发布、新版医保目录及首版商 业健康险创新药目录发布，从景气度角度继续看好创新药+CXO&产业链+创新医疗器械。 疫苗ETF ...

华福证券指出，2025年小核酸行业迎来首个爆发周期，商业化进展和新平台验证超出市场预期。 Alnylam公司治疗ATTR的大单品AMVUTTRA销售额有望突破20亿美金，Ionis治疗sHTG的产品在关键3 期数据读出后股价大涨35%，Arrowhead依托肝外Trim平台布局减肥和CNS项目，并迎来首个商业化产 品Plozasiran。小核酸药物商业化加速带动原料和外包研发需求增长。临近12月，ASH会议数据、新版 医保目录和商业健康险创新药目录发布或成为行业催化剂。 科创创新药ETF国泰（589720）跟踪的是科创新药指数（950161），单日涨跌幅达20%，该指数从 市场中选取涉及生物医药、化学制药等领域的创新型企业证券作为指数样本，重点覆盖高研发投入、具 备核心技术优势及成长潜力的医药公司，以反映科技创新驱动的新药研发企业整体表现。行业分布上突 出生物科技与制药板块。 注：如提及个股仅供参考，不代表投资建议。指数/基金短期涨跌幅及历史表现仅供分析参考，不 预示未来表现。市场观点随市场环境变化而变动，不构成任何投资建议或承诺。文中提及指数仅供参 考，不构成任何投资建议，也不构成对基金业绩的预测和保证。如 ...

消息面，小核酸药物治疗乙肝取得突破性数据，未来小核酸药物在乙肝领域的推进值得期待。受消息面提振，单日涨跌幅20%的科创创新药 ETF（589720） 涨超2.5%。资金持续布局，连续5日迎资金净流入。 关注20cm标的科创创新药 ETF（589720），跟踪指数"924行情"以来跑赢港股创新药 展望后市，创新药行业在出海持续突破、政策红利不断释放以及中国创新药企业研发实力稳步提升的多重驱动下，正迎来广阔的发展空间，具备较好的投资 机会，可以关注相关20cm标的科创创新药 ETF国泰（589720）。 科创创新药ETF国泰（589720）聚焦科创板创新药企业，跟踪汇聚30家代表性优质公司的上证科创板创新药指数，以高成长biotech为主，产品20%涨跌幅限 制使其更贴合板块波动。 2025年小核酸迎来首个爆发周期 小核酸药物治疗乙肝取得突破性数据，在近日举办的2025年美国肝病研究协会（AASLD2025）年会上浩博医药披露了两项反义寡核苷酸（ASO）药物AHB- 137针对慢性乙型肝炎患者治疗结束后24周的2期随访数据，从现有数据来看，AHB-137有望成为潜在最佳的乙肝小核酸药物，未来小核酸药物在乙肝领域的 ...

Investment Rating - The report maintains an "Outperform" rating for the pharmaceutical sector [6]. Core Insights - The small nucleic acid drug industry is entering a significant commercialization phase in 2025, with major advancements in product launches and new platform validations expected [16]. - Alnylam's AMVUTTRA is projected to exceed $2 billion in sales, marking a significant milestone for small nucleic acid drugs [3][17]. - Ionis is anticipated to have multiple catalysts in 2026, with key products like Olezarsen showing promising results [25]. - Arrowhead has launched its first commercial product, Plozasiran, and is focusing on its innovative Trim platform for weight loss and CNS projects [35]. Summary by Sections 1. Weekly Performance of Recommended Pharmaceutical Combinations - The recommended combinations outperformed the pharmaceutical index by 2.4 percentage points but underperformed the broader market by 0.6 percentage points [12]. 2. Small Nucleic Acid Industry Update - The small nucleic acid sector is witnessing rapid commercialization and platform breakthroughs, with significant investment opportunities emerging [16]. - Alnylam's AMVUTTRA sales are expected to reach $2.475 to $2.525 billion for the year, reflecting strong market demand [23]. - Ionis's Olezarsen has shown a 72% reduction in triglycerides in patients, indicating a strong commercial outlook [17][25]. - Arrowhead's Plozasiran is now approved for treating familial chylomicronemia syndrome, marking a significant achievement for the company [17]. 3. Weekly Market Review of the Pharmaceutical Sector - The CITIC Pharmaceutical Index fell by 6.8% during the week of November 17-21, 2025, underperforming the CSI 300 Index by 3 percentage points [47]. - The pharmaceutical sector has seen a year-to-date increase of 14.7%, outperforming the CSI 300 Index by 1.5 percentage points [47]. - The top-performing stocks in the sector included Hainan Haiyao (+23.8%) and Changyao Holdings (+19.7%) [59]. 4. Market Trends and Future Outlook - The report suggests a continued focus on innovative drugs, CXO, and the pharmaceutical supply chain, especially with upcoming ASH conference data releases [4]. - The overall valuation of the pharmaceutical sector is currently at 28.5, indicating a relative premium over the broader market [54].

Core Insights - Alnylam is recognized as a leading RNAi company, leveraging Nobel Prize-winning science to develop innovative therapies [2] - The company has successfully launched 6 marketed products and has a robust clinical pipeline with over 20 programs [2] - Alnylam is focused on three main areas for future growth, with an emphasis on achieving leadership in TTR (transthyretin) therapeutics [3] Company Overview - Alnylam has built an extraordinary company over the last couple of decades, emphasizing innovation and the development of new medicines [2] - The company is committed to maintaining a productive innovation engine that will continue to deliver new therapies for decades [2] Product Launch - The launch of AMVUTTRA for cardiomyopathy has been described as a terrific start, indicating strong initial market reception [3]

Financial Data and Key Metrics Changes - The company reported an 11% growth in both portfolio receipts and royalty receipts, indicating strong business momentum [4][20] - Return on invested capital was 15.7% and return on invested equity was 22.9% for the last 12 months [5][23] - Portfolio receipts for the third quarter reached $814 million, reflecting an 11% increase [20] - Full year 2025 top line guidance was raised to between $3.2 billion and $3.25 billion, representing growth of approximately 14%-16% [8][25] Business Line Data and Key Metrics Changes - The company maintained strong returns with significant contributions from Voranigo, Tremfya, and the Cystic Fibrosis Franchise [20] - The development stage pipeline expanded to 17 therapies, with multiple pivotal readouts expected in the near future [7][14] Market Data and Key Metrics Changes - The company is actively participating in the growing market for royalties, with recent acquisitions including a royalty interest in Amgen's Imdelltra and Alnylam's AMVUTTRA [6][10] - The company has seen a significant uptick in deal activity, with a total of approximately $1.6 billion in announced value across three different disease areas [9] Company Strategy and Development Direction - The company aims to be the premier capital allocator in life sciences, focusing on innovative therapies with high patient impact [4][13] - The strategy includes a therapy area agnostic investment approach, allowing the company to invest across various disease areas [15][16] - The company is committed to creating value for shareholders through investments in innovative therapies [13][30] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the external environment, noting that increased biotech M&A activity could benefit their capital allocation strategy [38] - The company is excited about growth opportunities in China, particularly in out-licensing deals with multinationals [39] - Management emphasized the stability and consistency of their return metrics, indicating confidence in maintaining attractive returns [48][23] Other Important Information - The company has returned a record $1.5 billion to shareholders in the first nine months of the year, including share repurchases and dividends [24][25] - The company maintains financial flexibility with cash and equivalents of $939 million and access to a $1.8 billion revolver [23][24] Q&A Session Summary Question: Impact of biotech M&A and interest rates on royalty-driven deal activity - Management noted that increased M&A activity does not significantly impact their operations and that they see it as beneficial for capital needs in the sector [38][39] Question: Frequency of collaborations with large pharma - Management indicated that as the largest royalty buyer, they are actively looking at all deals and see a growing opportunity for using royalties to fund trials with both biotech and big pharma [41][42] Question: Insights on AMVUTTRA deal and competition - Management highlighted their confidence in AMVUTTRA's potential despite competition from Nucresiran, emphasizing their thorough analysis of various scenarios [51][55] Question: Implications of trial designs in the LP(a) space - Management acknowledged differences in trial designs and expressed optimism about both Amgen's and Novartis's trials, maintaining a positive outlook on their investments in this area [67][75] Question: Updates on obexelimab and its market potential - Management confirmed that the recent phase II data for obexelimab validated their investment thesis and highlighted the focus on IgG4-related disease for near-term capital needs [77][76]

Core Insights - Royalty Pharma has acquired a royalty interest in Alnylam's AMVUTTRA for $310 million from Blackstone Life Sciences, which is expected to enhance Royalty Pharma's portfolio significantly [1][3][4] - AMVUTTRA is an FDA-approved RNAi therapeutic for treating ATTR amyloidosis, a serious disease caused by misfolded proteins [2][3] - The acquisition includes a 1% royalty on worldwide net sales of AMVUTTRA, with sales projected to exceed $6 billion by 2028, following a strong sales performance of approximately $1 billion in 2024, representing a 74% year-over-year growth [3][4] Company Overview - Royalty Pharma is the largest buyer of biopharmaceutical royalties and funds innovation across the biopharmaceutical industry, collaborating with various entities from academic institutions to leading pharmaceutical companies [6] - The company has a diverse portfolio that includes royalties on over 35 commercial products, indicating a strong market presence [6] Market Context - AMVUTTRA addresses a growing market for ATTR-CM, with approximately 300,000 patients globally, of which only 20% are currently diagnosed, highlighting significant growth potential [3] - The transaction is expected to deliver returns consistent with Royalty Pharma's targets, despite potential competition from Alnylam's follow-on product, nucresiran [4]

NEW YORK and CAMBRIDGE, Mass., Nov. 04, 2025 (GLOBE NEWSWIRE) -- Royalty Pharma plc (Nasdaq: RPRX) today announced that it has acquired a royalty interest in Alnylam’s AMVUTTRA from funds managed by Blackstone Life Sciences (“Blackstone”) for $310 million. The royalty interest being sold stems from Blackstone’s 2020 financing collaboration with Alnylam, in which Blackstone invested to support AMVUTTRA’s pivotal Phase 3 HELIOS-B trial. AMVUTTRA is an FDA-approved RNAi therapeutic for the treatment of ATTR am ...

Financial Data and Key Metrics Changes - Alnylam Pharmaceuticals reported total net product revenues of $851 million for Q3 2025, reflecting a 103% year-over-year growth [8][27] - The TTR franchise revenues reached $724 million, representing a 135% increase year-over-year and a 33% increase compared to the previous quarter [7][10] - The company increased its total net product revenue guidance for 2025 from a range of $2.65 billion to $2.8 billion to a revised range of $2.95 billion to $3.05 billion, indicating a 10% increase at the midpoint [8][30] Business Line Data and Key Metrics Changes - The rare disease portfolio achieved $127 million in sales, up 14% year-over-year, driven by ongoing patient demand [9][10] - U.S. net sales for the TTR franchise grew 42% quarter-over-quarter and 194% year-over-year, primarily due to the AMVUTTRA ATTR CM label expansion [11][14] - International revenues grew 13% quarter-over-quarter and 46% year-over-year, with strong performance in Japan and Germany [11][12] Market Data and Key Metrics Changes - The U.S. TTR franchise generated $543 million in net product revenues, reflecting a significant increase in patient demand [14][16] - The company noted that the international performance is benefiting from increased awareness and diagnosis in the hereditary ATTR polyneuropathy market [13] Company Strategy and Development Direction - Alnylam focuses on three core pillars: TTR leadership, growth through innovation, and strong financial performance [5][6] - The company is advancing its pipeline with new Phase 3 trials for zilebesiran and ALN-APP, targeting significant market opportunities [8][20] - The strategy includes expanding the AMVUTTRA ATTR CM launch globally, with expectations for further launches in 2026 [16][30] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term opportunity for AMVUTTRA, highlighting substantial growth potential in the TTR category [16][17] - The company is optimistic about the ongoing demand and adoption of its therapies, particularly in the U.S. market [10][14] - Management acknowledged the competitive landscape but emphasized their strong positioning and execution capabilities [11][13] Other Important Information - The gross margin on product sales was reported at 77%, down from 80% in the same quarter last year, primarily due to increased royalties on AMVUTTRA [27] - The company ended the quarter with cash, cash equivalents, and marketable securities of $2.7 billion [29] Q&A Session Summary Question: Momentum of AMVUTTRA in first line versus switch population - Management noted that patient demand for AMVUTTRA has doubled, with broad adoption across various settings, indicating strong momentum [34][35] Question: Confidence in continued patient additions - Management highlighted the strong launch and raised guidance twice, indicating confidence in sustained momentum despite a decline in ONPATTRO [38][40] Question: Payer dynamics and feedback - Management reported no significant payer headwinds and positive feedback regarding the value of AMVUTTRA, with ongoing negotiations for 2026 [45][48] Question: Inventory demand and expectations for Europe - Management explained that inventory growth was offset by increased gross to net deductions, with modest contributions expected from the ex-U.S. market in Q4 [52][56] Question: TTR franchise guidance update - Management indicated that the updated guidance reflects expectations for Q4 revenues, estimating total TTR global revenue of $850 million to $900 million [60][61] Question: Impact of reimbursement cuts on diagnostics and therapeutics - Management acknowledged the importance of reimbursement for growth and expressed confidence in managing potential changes as policies become clearer [66][68]

Financial Data and Key Metrics Changes - Total net product revenues for Q3 2025 were $851 million, representing a 103% increase year-over-year [7][28] - TTR franchise revenues reached $724 million, showing a 135% year-over-year growth, primarily driven by the AMVUTTRA ATTR CM launch in the U.S. [6][11] - The company increased its total net product revenue guidance for 2025 from a range of $2.65 billion to $2.8 billion to a revised range of $2.95 billion to $3.05 billion, reflecting a 10% increase at the midpoint [8][33] Business Line Data and Key Metrics Changes - The rare disease portfolio achieved $127 million in sales, up 14% compared to Q3 2024, driven by ongoing patient demand [10] - U.S. net sales for the TTR franchise grew 42% quarter-over-quarter and 194% year-over-year, reflecting strong adoption following the AMVUTTRA ATTR CM label expansion [12][14] - Outside the U.S., revenues grew 13% quarter-over-quarter and 46% year-over-year, indicating continued global momentum [12] Market Data and Key Metrics Changes - The U.S. TTR franchise delivered $543 million in net product revenues, representing an increase of approximately $160 million versus the prior quarter [14] - In Japan, the AMVUTTRA ATTR CM launch is progressing well, tracking in line with leading launch analogs [12] - The international performance reflects the strength of the hereditary ATTR polyneuropathy legacy business, which remains robust despite new competition [12] Company Strategy and Development Direction - The company focuses on three core pillars: TTR leadership, growth through innovation, and strong financial performance [5] - The company is committed to establishing long-term leadership in TTR and advancing its pipeline of RNAi therapeutics [5][6] - The company plans to expand its global reach with anticipated international AMVUTTRA ATTR CM launches in 2026 [17] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the AMVUTTRA ATTR CM launch and the overall growth trajectory of the TTR franchise [8][11] - The management highlighted the importance of early treatment in ATTR cardiomyopathy and the positive reception from payers and physicians [62] - The company is optimistic about the potential of RNAi therapeutics to become the standard of care in TTR amyloidosis [19] Other Important Information - The company ended the quarter with cash, cash equivalents, and marketable securities of $2.7 billion [32] - Non-GAAP R&D expenses increased by 23% to $310 million, driven by costs associated with multiple Phase 3 clinical studies [31] - The company announced the initiation of a Phase 1 trial of ALN-5288 targeting Alzheimer's disease [26] Q&A Session Summary Question: Can you discuss the momentum of AMVUTTRA in first line versus switch population? - Management noted that patient demand for AMVUTTRA has doubled, with broad adoption across various settings, indicating healthy uptake [36][38] Question: What gives you confidence in continued patient additions next year? - Management highlighted the strong launch momentum and the raised guidance as indicators of confidence in sustained growth [53] Question: Can you provide insights on payer dynamics as you approach 2026? - Management reported no significant payer headwinds and positive feedback from payers regarding the value of AMVUTTRA [60][62] Question: How should we model inventory demand for AMVUTTRA? - Management explained that inventory growth was driven by increased demand, with expectations for continued growth in the fourth quarter [66][68] Question: What is the expected pricing strategy for AMVUTTRA in Europe? - Management indicated ongoing negotiations for pricing in Europe, with expectations for a gradual decline in net price over time [90][93]