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 ...

行 业 研 究 医药生物 2025 年 11 月 23 日 医药生物 小核酸药物：三家标杆 2026 年的催化剂有哪些？ 投资要点： 强于大市（维持评级） 行 业 定 期 报 行情回顾：本周（2025 年 11 月 17 日- 2025 年 11 月 21 日）中信医药 指数上跌 6.8%，跑输沪深 300 指数 3.0pct，在中信一级行业分类中排名第 25 位；2025 年初至今中信医药生物板块指数上涨 14.7%，跑赢沪深 300 指 数 1.5pct，在中信行业分类中排名第 14 位。本周涨幅前五的个股为：海南 海药（+23.8%）、长药控股（+19.7%）、江苏吴中（+15.5%）、怡和嘉业 （+13.9%）、京新药业（+10.3%） 告 小核酸行业更新：商业化持续兑现和新平台迎来突破，积极关注产业 链机会。2025 年小核酸迎来首个爆发周期，无论是产品的商业化还是小核 酸肝外新平台的进展，都超出市场预期。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 亿美金的小核酸药物。 Ionis 治疗 sHTG 的大单品在 9 月读出阳性数据后，公司股价大涨 ...

Regulatory Overview - The FDA has approved 34 novel drugs from January to October 2025, compared to 38 during the same period in 2024 [2] - Several biotech stocks are highlighted as they approach key regulatory decisions in November 2025 [2] Arrowhead Pharmaceuticals Inc. (ARWR) - ARWR's New Drug Application for Plozasiran is under FDA review, with a decision expected on November 18, 2025 [3] - Plozasiran targets familial chylomicronemia syndrome, a condition characterized by extremely high triglyceride levels, which can lead to acute pancreatitis [3] - Clinical trials showed significant reductions in triglycerides and the incidence of acute pancreatitis, potentially providing a new treatment option alongside Tryngolza, the only FDA-approved drug for this condition [4] Otsuka Holdings Co. Ltd. (OTSKF) - OTSKF is awaiting the FDA's decision on Sibeprenlimab, scheduled for November 28, 2025 [6] - Sibeprenlimab is an investigational monoclonal antibody for treating immunoglobulin A nephropathy (IgAN), a chronic kidney disease [6] - Clinical trials demonstrated a significant reduction in urine protein-to-creatinine ratio, indicating its potential as a blockbuster drug if approved [8] Bayer (BAYRY) - Bayer's investigational compound Sevabertinib is under priority review by the FDA, with a decision expected on November 28, 2025 [9] - Sevabertinib is proposed for treating advanced non-small cell lung cancer (NSCLC) with HER2 mutations, providing an additional option for previously treated patients [10] Ascendis Pharma A/S (ASND) - The FDA decision on Navepegritide, aimed at treating children with achondroplasia, is due on November 30, 2025 [11] - Achondroplasia is a rare genetic condition affecting over 250,000 people globally, leading to serious complications [12] - Navepegritide is designed for weekly subcutaneous administration, targeting the overactive FGFR3 pathway [13] Kura Oncology Inc. (KURA) - KURA is expecting FDA approval for Ziftomenib on November 30, 2025, for treating relapsed or refractory acute myeloid leukemia (AML) with NPM1 mutations [15] - Ziftomenib has shown significant efficacy in clinical trials, with a market opportunity estimated at $350 million to $400 million annually in the U.S. if approved [17]

Company Highlights - Aurinia Pharmaceuticals Inc. (AUPH) has seen solid growth for its product LUPKYNIS, with expected net product sales for 2025 ranging from $250 million to $260 million, up from $216.2 million last year [2][3] - Arrowhead Pharmaceuticals Inc. (ARWR) is preparing for its first commercial launch in 2025, with its lead drug candidate Plozasiran under FDA review, decision expected on November 18, 2025 [4][5] - Beta Bionics Inc. (BBNX) reported a 54% growth in second-quarter net sales, with expectations for full-year 2025 net sales to range between $88 million and $93 million, compared to $65.1 million last year [7][8] - Kiniksa Pharmaceuticals International plc (KNSA) anticipates net product revenue for its drug Arcalyst to be between $625 million and $640 million for 2025, up from $417 million in 2024 [10][11] - Ionis Pharmaceuticals Inc. (IONS) generated net product sales of $19 million in Q2 2025, with a supplemental new drug application expected by year-end [12][14] - Stoke Therapeutics Inc. (STOK) is developing Zorevunersen for Dravet syndrome, with a 52-week high of $24.60 reached recently, representing an 82% gain in less than two months [16][17] - Fortress Biotech Inc. (FBIO) is awaiting an FDA decision on its investigational drug CUTX-101 for Menkes disease, expected on September 30, 2025 [18][21] Stock Performance - AUPH stock increased by 168% over 1.5 years, reaching a 52-week high of $12.87 [3] - ARWR stock gained 65% in less than 9 months, hitting a 52-week high of $31.13 [5] - BBNX stock rose nearly 55% in less than 4 months, reaching a 52-week high of $26.66 [8] - KNSA stock saw a 46% increase in less than four months, touching a 52-week high of $37.34 [11] - IONS stock gained 52% in less than two months, reaching a 52-week high of $64.72 [15] - STOK stock increased by 82% in less than two months, achieving a 52-week high of $24.60 [17] - FBIO stock rose by 61% in less than a month, reaching a 52-week high of $3.97 [21]

Summary of Arrowhead Pharmaceuticals FY Conference Call Company Overview - **Company**: Arrowhead Pharmaceuticals (NasdaqGS:ARWR) - **Event**: FY Conference Call on September 10, 2025 - **Key Speakers**: Chris Anzalone (CEO), James Hamilton (CMO and Head of R&D) Core Points and Arguments Transition to Commercialization - Arrowhead Pharmaceuticals is transitioning into a commercial company with a PDUFA date for Plozasiran on November 18, 2025, indicating a significant milestone in their development pipeline [2][4] Plozasiran Overview - Plozasiran is designed to reduce ApoC3 expression, lowering triglycerides in patients with Familial Chylomicronemia Syndrome (FCS) [4] - Phase 3 study results showed an approximately 80% reduction in triglycerides from baseline and a significant decrease in the risk of acute pancreatitis [4][12] Market Opportunity in FCS - FCS is classified as an ultra-orphan disease with an estimated 1,000 genetically defined patients in the U.S. [6] - Arrowhead also targets clinically defined FCS patients, potentially expanding the market beyond the genetically defined group [7] Severe Hypertriglyceridemia (SHTG) Market - The SHTG market is significantly larger, targeting patients with triglyceride levels above 500 mg/dL [9] - Arrowhead is conducting several Phase 3 studies (Shasta-3, Shasta-4, Shasta-5, NEER-3) to evaluate Plozasiran's efficacy in this population [10][19] Competitive Landscape - Arrowhead acknowledges competition in the market but believes that having multiple companies addressing the same medical need is beneficial for patient education and treatment options [16][17] - Plozasiran is expected to show greater efficacy in triglyceride reduction compared to competitors' products [25] Zodasiran Development - Zodasiran is focused on the Homozygous Familial Hypercholesterolemia (HoFH) population, with a Phase 3 study (Yosemite) underway [28] - The drug has shown promising results in earlier studies, with expectations of similar LDL-C reductions as existing therapies [29] Obesity Programs - Arrowhead is developing two obesity treatments targeting the INHBE and ALK7 pathways, with data expected by the end of the year [32][33] - The company aims to demonstrate high-quality weight loss and muscle sparing in clinical trials [35] Bispecific PCSK9/ApoC3 Dimer - A new bispecific dimer targeting both PCSK9 and ApoC3 is set to enter clinical trials, aiming to lower both LDL cholesterol and triglycerides [41][42] CNS Platform - Arrowhead has a promising CNS platform with three near-term clinical programs targeting tau, Huntington's disease, and alpha-synuclein [44][45] - The company has partnered with Novartis for the alpha-synuclein program, while retaining control over the MAPT program [49] Financial Position and Future Outlook - Arrowhead's current cash position and partnerships are expected to sustain operations into 2028, allowing continued development of their pipeline [60] - Upcoming catalysts include the PDUFA update for Plozasiran, obesity data, and Phase 3 readouts for severe hypertriglyceridemia [63][64] Additional Important Points - The partnership with Sarepta Therapeutics has been financially beneficial, with nearly $1 billion received in the past year [55] - Arrowhead emphasizes the importance of maintaining a productive R&D organization while transitioning to commercialization [61] This summary encapsulates the key points discussed during the conference call, highlighting Arrowhead Pharmaceuticals' strategic direction, product pipeline, market opportunities, and financial outlook.

Group 1 - Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR) is considered one of the best stocks to buy for the next three months according to hedge funds, with a Buy rating and a price target of $80 set by H.C. Wainwright analyst Patrick Trucchio [1] - The recent deal with Novartis is highlighted as a strong vote of confidence, providing $200 million upfront and the potential for a $2 billion milestone, which validates Arrowhead's approach to treating CNS diseases and adds funding without share dilution [2] - Positive results from Ionis Pharmaceuticals' Olezarsen support Arrowhead's Plozasiran drug, both targeting severe hypertriglyceridemia, with Arrowhead's offering of quarterly dosing potentially attracting more patients and doctors [3]

Core Viewpoint - Arrowhead Pharmaceuticals reported significant misses in both GAAP revenue and earnings for Q3 FY2025, highlighting ongoing financial volatility despite progress in its clinical pipeline [1][5][6] Financial Performance - Q3 FY2025 GAAP revenue was $27.8 million, missing analyst estimates of $46.8 million by nearly 41% [5] - GAAP earnings per share for Q3 FY2025 were $(1.26), falling short of the $(0.95) forecast by $0.31 [1][5] - Operating loss for Q3 FY2025 was $(165.6 million), an improvement of 6% compared to $(176.1 million) in Q3 FY2024 [2][6] - Research and development expenses increased by 6.6% year-over-year to $162.4 million [2][6] - Total cash resources rose to $900.4 million, a 32.1% increase from $681.0 million in Q3 FY2024 [2][7] Company Overview and Strategy - Arrowhead Pharmaceuticals focuses on RNA interference (RNAi) therapeutics, targeting diseases with significant unmet needs using its TRiMTM platform [3][4] - The company is advancing its drug pipeline with an emphasis on late-stage development and regulatory approvals, while building strategic partnerships with larger pharmaceutical companies [4] Product Pipeline Progress - Plozasiran, Arrowhead's leading RNAi candidate, received NDA acceptance from the FDA for familial chylomicronemia syndrome, with a PDUFA target date of November 18, 2025 [8][11] - Phase 3 studies for severe hypertriglyceridemia are ongoing, with data expected in mid-2026 [8][11] - Zodasiran, targeting rare cholesterol disorders, has commenced its Phase 3 trial [12] - The obesity portfolio includes ARO-INHBE and ARO-ALK7, which are in Phase 1/2 studies [13] Strategic Partnerships and Milestones - Visirna, a subsidiary, entered a deal with Sanofi for rights to four cardiometabolic programs, resulting in immediate payments of $130 million and potential milestones of up to $265 million [9] - The company anticipates additional milestone payments from Sarepta Therapeutics, totaling up to $200 million by the end of 2025 [9] Commercial Readiness - Arrowhead is preparing for the potential launch of plozasiran by expanding its U.S. sales force and engaging with payers [10] - The company is focused on rare disease awareness and patient identification, crucial for the target population of plozasiran [10] Outlook and Guidance - Management indicated that Arrowhead is funded through 2028, focusing on regulatory approval for plozasiran and commercialization efforts [15] - Key near-term catalysts include pivotal SHTG data readout in mid-2026 and potential partnership milestones within the next twelve months [15]