诺华重注小核酸肝外递送 2025-08-20 诺华重注小核酸肝外递送 原创 来觅研究院 RimeData 来觅数据 撰稿 张梓英 2025-08-20 导读： 8 月 7 日消息，诺华拟收购目前市值近 60 亿美元的 AOC 领域领军者 Avidity Biosciences （NASDAQ：RNA），旨在通过扩充管线强化罕见病治疗领域的核心布局。据介绍，Avidity Biosciences 是一家致力于提供一类名为抗体寡核苷酸偶联药物（AOC）的新型 RNA 疗法的生物制药公司，目前旗下 共有三条处于临床阶段的 AOC 管线。 小核酸药物（寡核苷酸药物）是一类通过调控基因表达实现疾病治疗的生物大分子药，包括 siRNA、ASO、 miRNA 等，其作用机制主要基于碱基互补配对原则：siRNA 通过 RNA 干扰（RNAi）途径降解靶 mRNA， ASO 通过空间位阻或 RNase H 介导的切割抑制蛋白翻译，miRNA 则通过调控多基因网络影响细胞功 能，这类药物的核心优势在于能靶向传统"不可成药"的致病基因，在基因层面直接调控疾病相关蛋白的 表达，为遗传病、罕见病和慢性病提供了突破性治疗手段。这类药物疗 ...

Core Insights - Royalty Pharma reported a revenue of $727 million for the quarter ended June 2025, reflecting a year-over-year increase of 19.6% and surpassing the Zacks Consensus Estimate of $686.22 million by 5.94% [1] - The company's EPS for the quarter was $1.14, compared to $0.96 in the same quarter last year, resulting in an EPS surprise of 3.64% against the consensus estimate of $1.10 [1] Financial Performance Metrics - The total portfolio receipts from royalty products amounted to $672 million, slightly below the average estimate of $680.22 million [4] - Key product performances included: - Cystic fibrosis franchise: $194 million, -0.4% year-over-year, below the estimate of $206.12 million [4] - Tysabri: $56 million, -12.3% year-over-year, above the estimate of $53.9 million [4] - Imbruvica: $44 million, -10.4% year-over-year, above the estimate of $39.81 million [4] - Xtandi: $42 million, +8.8% year-over-year, below the estimate of $42.75 million [4] - Promacta: $33 million, +8.2% year-over-year, below the estimate of $39.51 million [4] - Evrysdi: $33 million, +32.4% year-over-year, above the estimate of $31.21 million [4] - Trelegy: $57 million, +17.6% year-over-year, slightly below the estimate of $57.04 million [4] - Tremfya: $37 million, +23.7% year-over-year, below the estimate of $38.47 million [4] Stock Performance - Over the past month, Royalty Pharma's shares have returned +6.8%, outperforming the Zacks S&P 500 composite's +0.5% change [3] - The stock currently holds a Zacks Rank 3 (Hold), indicating expected performance in line with the broader market in the near term [3]

Core Insights - Biogen reported strong second-quarter 2025 results, with adjusted earnings per share (EPS) of $5.47, significantly exceeding the Zacks Consensus Estimate of $3.93, and a 4% year-over-year increase in earnings [1] - Total revenues for the quarter reached $2.65 billion, up 7% year over year, surpassing the Zacks Consensus Estimate of $2.32 billion [2] Revenue Breakdown - Product sales were $1.9 billion, a slight decline of 1% year over year, while revenues from anti-CD20 therapeutic programs increased by 5% to $467 million [3] - Contract manufacturing and royalty revenues surged 124% year over year to $245 million, and Alzheimer's collaboration revenues rose to $55 million from $12 million in the previous year [4][5] Performance of Key Products - Sales of Biogen's multiple sclerosis (MS) drugs totaled $1.1 billion, down 4% year over year, primarily due to generic competition for Tecfidera and biosimilar competition for Tysabri [7] - Tecfidera sales fell nearly 23% to $193.6 million, while Vumerity sales increased by around 30% to $212.2 million [8] - Spinraza sales declined 8.5% to $392.7 million, missing estimates, while rare disease drug Skyclarys generated $130.3 million, up 5.2% sequentially [12] New Product Performance - New drugs Leqembi, Skyclarys, and Zurzuvae showed strong demand and sequential sales growth, with Leqembi generating $63 million in U.S. sales, a 20% increase [5][11] - Zurzuvae recorded $46.4 million in sales, up 68% sequentially, driven by increased demand [13] Cost and Guidance Updates - Adjusted R&D expenses decreased by 13% to $394 million, while SG&A expenses rose 7% to $579 million [16] - Biogen raised its 2025 EPS outlook to $15.50-$16.00, reflecting a stronger business outlook, and expects total revenues to be flat compared to 2024 [18] Market Reaction and Future Outlook - The positive guidance and strong sales of Leqembi boosted investor confidence, leading to a more than 5% rise in pre-market trading [20] - Despite rising competitive pressure on MS drugs, new products have the potential to drive growth, although it remains uncertain if they can offset declines in existing products [21]

Key Takeaways IONS posted Q2 EPS of $0.86 and revenue of $452M, both beating estimates and doubling year over year.Tryngolza and Wainua drove a 43% rise in commercial revenues, reaching $103M for the quarter.IONS raised its 2025 revenue outlook to $825-$850M, citing strong Tryngolza uptake and market expansion plans.Ionis Pharmaceuticals (IONS) reported second-quarter 2025 adjusted earnings per share (EPS) of 86 cents, significantly beating the Zacks Consensus Estimate of 27 cents. In the year-ago period, t ...

Core Viewpoint - Biogen is expected to report second-quarter 2025 results on July 31, with sales and earnings estimates at $2.32 billion and $3.95 per share, respectively, following a previous earnings miss of 7.4% [1][11]. Group 1: Sales Performance - Lower sales of Biogen's multiple sclerosis (MS) drugs are anticipated, likely offset by revenue growth from new products [2]. - Sales of Tecfidera and Tysabri are projected to decline due to generic and biosimilar competition, with estimates for Tecfidera at $178.0 million and Tysabri at $373.0 million [3][4]. - Vumerity's sales are expected to rise, with estimates at $70.0 million and $181.9 million [4]. - Spinraza's sales are estimated at $397.0 million and $403.0 million, with a potential reversal in favorable shipment timing impacting second-quarter results [5]. - Skyclarys is likely to see mixed dynamics in the U.S. and global markets, with growth driven more by ex-U.S. sales [6][9]. Group 2: Collaborations and New Products - Biogen's collaboration with Sage Therapeutics for Zurzuvae involves shared profits and losses, with strong patient demand expected to continue [7]. - Alzheimer's collaboration revenues, including Biogen's share from Leqembi, are expected to rise, with Leqembi's sales improving sequentially [8][10]. Group 3: Earnings Expectations - Biogen's earnings surprise history shows a mixed performance, with a four-quarter average surprise of 8.36% [11]. - The Earnings ESP for Biogen is -0.85%, indicating a lower likelihood of an earnings beat this quarter [14].

Investment Rating - The report maintains a "Positive" outlook for the pharmaceutical and biotechnology industry in China [6]. Core Insights - Small nucleic acid drugs are expected to become the third major class of drugs after small molecules and antibodies, with unique advantages such as broad targets, strong specificity, high development efficiency, and long dosing intervals [9][38]. - The commercialization of rare diseases is maturing, and the long-term advantages for chronic diseases are becoming evident, with significant sales growth for products like Spinraza and Leqivo [9][60]. - There is a notable increase in business development (BD) activities, highlighting the potential of early-stage chronic disease pipelines [9]. Summary by Sections 1. Small Nucleic Acids: Potential as a New Drug Class - Small nucleic acid drugs, including ASO, siRNA, and Aptamer, interact with mRNA to regulate gene expression, offering a new technological pathway for drug development [13]. - The global market for small nucleic acid drugs has grown from $1.04 billion in 2017 to $5.09 billion in 2024, with a CAGR of 25.5% [60]. 2. Milestones in Overseas and Domestic Markets - In the overseas market, significant developments are expected in the TTR field and cardiovascular diseases, with drugs like Vutrisiran and Pelacarsen showing promise [9]. - In China, new therapies for chronic hepatitis B and competitive advancements in cardiovascular drugs are emerging, with several companies making progress in their pipelines [9][60]. 3. Investment Recommendations and Targets - The report suggests focusing on high-quality domestic companies involved in the development of small nucleic acid drugs targeting chronic hepatitis B and cardiovascular diseases, such as HengRui Medicine, China National Pharmaceutical Group, and others [9].

Core Insights - Royalty Pharma plc has appointed Carole Ho and Elizabeth (Bess) Weatherman to its Board of Directors, enhancing its leadership team with expertise in biopharmaceuticals and finance [1][2][3] Group 1: Board Appointments - Carole Ho is the Chief Medical Officer and Head of Development at Denali Therapeutics, with 20 years of experience in biopharma, previously serving as Vice President of Clinical Development at Genentech [2][3] - Bess Weatherman has 35 years of experience as an investor in the healthcare industry and is currently a Special Limited Partner at Warburg Pincus, having joined the firm in 1988 [3] Group 2: Company Overview - Royalty Pharma, founded in 1996, is the largest buyer of biopharmaceutical royalties and a key funder of innovation in the biopharmaceutical sector, collaborating with various innovators [4] - The company has a portfolio that includes royalties on over 35 commercial products and 16 development-stage product candidates, indicating a strong position in the market [4] Group 3: Corporate Governance - The appointment of the new board members increases independent representation on the board to over 90%, reflecting Royalty Pharma's commitment to enhanced corporate governance [5]

Core Insights - Royalty Pharma will participate in the Goldman Sachs 46th Annual Global Healthcare Conference on June 10, 2025 [1] - The event will be accessible via a webcast on Royalty Pharma's website and archived for at least thirty days [1] Company Overview - Royalty Pharma, founded in 1996, is the largest buyer of biopharmaceutical royalties and a key funder of innovation in the biopharmaceutical sector [2] - The company collaborates with a range of entities, from academic institutions to leading global pharmaceutical companies, to support innovation [2] - Royalty Pharma's portfolio includes royalties from over 35 commercial products, such as Vertex's Trikafta and GSK's Trelegy, as well as 15 development-stage product candidates [2]

Core Insights - Ionis Pharmaceuticals announced positive top-line results from the phase III ESSENCE study for its drug Tryngolza, targeting moderate hypertriglyceridemia in patients at risk for atherosclerotic cardiovascular disease (ASCVD) [1][2] - The study achieved its primary endpoint, showing a statistically significant reduction in triglyceride (TG) levels, with reductions of 61% and 58% for 80 mg and 50 mg monthly doses, respectively [2] - A majority of participants in the study had their TG levels fall within the normal range (<150 mg/dL) post-treatment, and the drug met all key secondary endpoints [3] Recent Developments - Tryngolza received FDA approval for treating familial chylomicronemia syndrome (FCS), marking it as the first approved treatment for this rare genetic condition in the U.S. and Ionis' first independent commercial launch [4] - Ionis out-licensed ex-U.S. rights for Tryngolza to Sweden-based Sobi, which will handle future regulatory filings and commercialization outside the U.S. [5] - The drug is also being evaluated for severe hypertriglyceridemia (sHTG) in two late-stage studies, with data expected in Q3 2025 [6][7] Financial and Revenue Streams - Ionis has a diverse revenue stream through collaborations with major pharmaceutical companies like AstraZeneca, Biogen, GSK, and Novartis, providing funds for its development pipeline [10] - The company earns commercial revenues from royalties on Spinraza, which treats spinal muscular atrophy, and is also involved in marketing Qalsody for amyotrophic lateral sclerosis [11] - Recent approvals for drugs like Wainua and ongoing studies for other candidates could further enhance Ionis' revenue and reduce reliance on collaboration partners [12][14]

Core Viewpoint - Biogen reported first-quarter 2025 adjusted earnings per share (EPS) of $3.02, missing the Zacks Consensus Estimate of $3.32, with an 18% year-over-year decline in earnings due to a $165 million upfront payment to Stoke Therapeutics for a collaboration agreement [1][2][19] Financial Performance - Total revenues for Q1 2025 were $2.43 billion, reflecting a 6% year-over-year increase on a reported basis and an 8% increase on a constant-currency basis, surpassing the Zacks Consensus Estimate of $2.23 billion [2] - Total product sales reached $1.73 billion, up 1% year over year on a reported basis and 3% on a constant-currency basis [4] - Contract manufacturing and royalty revenues surged 61% year over year to $293 million, while Alzheimer's collaboration revenues increased to $33 million from $3 million in the prior year [5][6] Product Sales Breakdown - Multiple sclerosis (MS) revenues totaled $953 million, down 11% on a reported basis due to generic competition for Tecfidera [9] - Sales of Spinraza rose 24.2% to $423.9 million, exceeding the Zacks Consensus Estimate of $365 million [12] - New drug Qalsody for ALS recorded sales of $15.5 million, while Zurzuvae for postpartum depression generated $28 million in sales, reflecting a 21.7% sequential increase [13][14] Cost and Guidance - Adjusted R&D expenses decreased 3% year over year to $427 million, while adjusted SG&A expenses rose 1% to $572 million [16] - The company reaffirmed its total revenue guidance for 2025, expecting a mid-single-digit percentage decline in constant currency terms compared to 2024, and lowered its adjusted EPS guidance to a range of $14.50 to $15.50 [18][19] Market Performance - Year to date, Biogen's shares have declined 20.9%, compared to a 3.3% decrease in the industry [3]