证券研究报告 | 行业点评 gszqdatemark 2025 07 01 年 月 日 医药生物 【创新药大牛市】分支思路（三）中小市值管线爆款：镇痛机制趋 向多元化，关注相关药品临床进展 镇痛市场前景可期，存在市场结构性挑战与法规限制：随着慢性病患病率 增加、老龄化程度的加深，以及政府对药品监管和医保政策的逐步完善， 中国镇痛药市场前景广阔。根据 Mordor intelligence 数据，2021 年全球疼 痛管理市场价值 794 亿美元，预计到 2027 年将增长至 1207 亿美元，复 合年增长率为 7.39%。目前主流镇痛药物为阿片类药物与非甾体解热镇痛 药，阿片类药物（如吗啡、芬太尼等）在术后镇痛领域具有强大效果，但 由于滥用和成瘾问题，阿片类药物在医院使用过程中受到严格的政策限 制，部分重度疼痛患者得不到有效的镇痛治疗。非甾体抗炎药（NSAIDs） 应用广泛，但由于其对肾脏、胃肠道和心血管系统的副作用，长期使用也 存在一定的风险。 镇痛机制趋向多元化，有效规避成瘾风险：随着镇痛靶点创新与技术突破， 非阿片类药物的市场份额有望逐渐扩大。 KOR 激动剂是一种外周选择性阿片受体激动剂，可减少中枢副 ...

Core Viewpoint - Vertex Pharmaceuticals has experienced an 8.9% decline in stock price over the past three months, attributed to soft first-quarter results, slower uptake of new drugs, and geopolitical tensions affecting economic growth [1][2][10]. Group 1: Financial Performance - Vertex's CF product sales continue to grow, driven by demand for Trikafta/Kaftrio among younger age groups, although there are concerns about a slight slowdown in sales growth [4]. - The company expects CF sales to grow approximately 8% in 2025, supported by the launch of new products and continued demand for existing therapies [10][24]. - The Zacks Consensus Estimate for 2025 earnings has increased from $17.69 to $17.82 per share over the past 60 days, indicating positive analyst sentiment [22]. Group 2: Product Pipeline and Approvals - Vertex has gained approval for two new products: Journavx, a non-opioid pain medicine, and Alyftrek, a new CF treatment, with expectations for sales to improve in the second half of 2025 [5][10]. - Casgevy, a CRISPR-based therapy for sickle cell disease and beta-thalassemia, is anticipated to ramp up revenues as more patients are treated in approved regions [8][11]. - Vertex's pipeline includes several mid- to late-stage programs targeting various diseases, with four programs in pivotal development and potential regulatory filings expected next year [12][13]. Group 3: Market Position and Competition - Vertex maintains a dominant position in the CF market with minimal competition, which is expected to support continued sales growth despite recent challenges [24]. - The company is addressing concerns about its reliance on the CF franchise for growth by diversifying its product offerings with new therapies [24].

Group 1: Market Trends - Current popular themes on Wall Street include artificial intelligence, weight management medicines, and the impact of tariffs on equities and the economy [1] - The market is expected to continue delivering competitive returns over the next decade despite changing trends [1] Group 2: Shopify - Shopify has outperformed the market since its IPO 10 years ago, maintaining a strong position in the fast-growing e-commerce sector with over 12% market share in the U.S. by gross merchandise volume as of the end of 2024 [4][5] - The company's competitive edge is driven by high switching costs and network effects, making it difficult for merchants to leave the platform once they have invested time and resources [6] - Shopify's app store enhances its attractiveness to merchants, as it offers thousands of customization options [7] - The e-commerce market is expected to grow, providing Shopify with a larger consumer base, although over 80% of retail transactions still occur offline in the U.S. [8] - Despite not being profitable currently, Shopify has made significant operational changes that are expected to lead to consistent profitability in the coming years [9] - The stock is considered a buy due to its long-term growth prospects, despite potential short-term volatility [10] Group 3: Vertex Pharmaceuticals - Vertex Pharmaceuticals has faced challenges over the past year, including clinical setbacks and competition from illegal copies of its medicines in Russia, leading to lower-than-expected sales [11] - The company remains a leader in cystic fibrosis treatments, with its products targeting the underlying causes of the disease [13] - Vertex's new product, Journavx, has potential to generate significant revenue, estimated at $2.9 billion by 2030, due to high unmet needs in non-opioid pain treatments [14] - The company plans to expand its product lineup, including a potential type 1 diabetes treatment, and has a strong pipeline of early- and late-stage programs [15] - Vertex is expected to deliver superior returns through 2035, driven by its strong cystic fibrosis franchise and new product launches [15]

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) Goldman Sachs 46th Annual Global Healthcare Conference Call June 10, 2025 2:40 PM ET Company Participants David M. Altshuler - Executive VP & Chief Scientific Officer Reshma Kewalramani - CEO, President & Director Conference Call Participants Salveen Jaswal Richter - Goldman Sachs Group, Inc., Research Division Salveen Jaswal Richter Good afternoon, everyone. Thank you so much for joining us. I'm Salveen Richter, biotechnology at Goldman Sachs and really ple ...

Market Overview - The U.S. equity market in 2025 has been volatile due to trade tensions, macroeconomic uncertainties, and geopolitical challenges impacting investor sentiment [1] Investment Strategy - Market volatility presents opportunities to acquire fundamentally strong, high-quality stocks with robust growth prospects at attractive valuations, historically yielding significant returns for patient investors [2] Stock Analysis Microsoft - Microsoft is well-positioned to capitalize on the AI wave, with a critical role in building AI infrastructure globally and a deep partnership with OpenAI [5][6] - The company commands a 22% market share in the AI infrastructure space through its Azure cloud platform, which is expanding with new data centers in 10 countries [6] - Microsoft has a high annuity mix of 98% in recurring revenue, with commercial remaining performance obligations growing 34% year-over-year to $315 billion [7] - The company maintains a robust cash balance of $79.6 billion, allowing for aggressive AI investments while returning $9.7 billion to shareholders [7][8] Meta Platforms - Meta Platforms dominates digital advertising, generating nearly $41.4 billion in revenue by reaching 3.4 billion daily users across its social media applications [9] - AI investments have led to increased user engagement, with time spent on Facebook and Instagram rising by 7% and 6%, respectively, and ad conversion rates for Reels improving by 5% [10] - The company plans to invest $64 billion to $72 billion in fiscal 2025, leveraging its large user base for business messaging and mobile commerce [11] Amazon - Amazon's AWS leads the cloud infrastructure market with a 29% share and achieved a $117 billion annualized revenue run rate with a 40% margin [12] - The e-commerce segment is strengthening through improved logistics and automation, while advertising revenue reached $13.9 billion in the first quarter [13] - Amazon's AI capabilities are rapidly growing, with the AI business already at a "multibillion-dollar annual run rate" and expanding at triple-digit year-over-year percentages [14][15] Vertex Pharmaceuticals - Vertex Pharmaceuticals leads the cystic fibrosis market, generating over $10 billion in annual revenue from its CF franchise, primarily driven by the drug Trikafta [16][17] - The recently approved CF drug Alyftrek shows better efficacy and convenience, potentially expanding market penetration [17] - Vertex is financially stable with $11.4 billion in cash, allowing for significant investment in growth initiatives, including a strong pipeline of late-stage programs [19]

大摩注意到，Vertex Pharmaceuticals(VRTX.US)的药物Journavx(一种口服 Nav1.8 抑制剂)最近已获美国 食品药品监督管理局(FDA)批准用于急性疼痛——首个获得FDA批准针对 NaV1.8 的止痛药物，并且针 对糖尿病周围神经病变(慢性疼痛)的三期临床试验正在进行中，目前仍在招募患者。此外，Vertex正在 开发第二代 Nav1.8 抑制剂(VX-993)，预计将于 2025 年下半年公布急性疼痛的初步临床数据。 根据礼来公司的公告，SiteOne还在推进针对 Nav1.7、Nav1.8 以及其他离子通道的小分子抑制剂的研 发，用于治疗疼痛、咳嗽以及其他涉及外周神经系统过度兴奋的病症。该公司还在推进其他新型候选药 物的研发，这些药物对特定离子通道亚型具有精准的选择性，用于治疗其他感觉神经高反应性疾病，如 慢性咳嗽和慢性眼表疼痛。 花旗认为，此次收购进一步证实了钠通道抑制在非阿片类止痛治疗方案中的有效性，并且进一步增强了 花旗对这个正在缓慢但稳步发展的市场的信心，同时也增强了对Vertex的 Journavx的信心(2030 年预计 销售额为 16 亿美元)。的确，STC ...

Core Viewpoint - Eli Lilly (LLY) has entered into a definitive agreement to acquire SiteOne Therapeutics for $1 billion, aiming to enhance its neuroscience pipeline and develop non-opioid pain management solutions [1][2]. Group 1: Acquisition Details - The acquisition is valued at $1 billion, which includes upfront and milestone payments [4]. - SiteOne's lead asset, STC-004, is a phase II-ready drug that acts as a Nav1.8 inhibitor, designed to block pain signal transmission [2][3]. - The deal will also incorporate other investigational drugs targeting pain, cough, and conditions related to peripheral nervous system hyperexcitability [3]. Group 2: Strategic Fit and Benefits - The acquisition aligns with Eli Lilly's strategy to diversify its non-opioid pain pipeline, which already includes multiple candidates in mid-stage studies [8]. - The mechanism of STC-004 has been validated by the FDA's approval of Vertex Pharmaceuticals' Journavx, the first non-opioid oral pain signal inhibitor approved in over 20 years [9][10]. - SiteOne benefits from the acquisition as it lacks the commercial infrastructure to bring drugs to market, an area where Eli Lilly is well-established [10]. Group 3: Company Background - SiteOne Therapeutics, founded in 2010, has attracted interest from major pharmaceutical companies for its non-opioid pipeline and recently closed a $100 million funding round led by Novo Holdings [5]. - SiteOne has a partnership with Vertex Pharmaceuticals to advance NaV1.7 inhibitors for pain treatment [5]. Group 4: Stock Performance - Eli Lilly's stock performance is in line with the industry for the year-to-date period [6].

Believe it or not, but we're not far away from being halfway through the year. Time flies when you're having fun -- and when you're not having fun, too. The stock market could remain highly volatile for months to come. That doesn't mean you can't find great stocks to buy, though. Here are three top growth stocks to buy in the second half of 2025. Could the Trump administration's tariffs hurt Amazon (AMZN -0.87%)? Sure. However, the more I've thought about it, the more confident I am that the company's busin ...

Market Overview - The recent market correction appears significant on a year-to-date chart but is expected to look less daunting over a decade, as bull markets typically last longer than downturns, leading to strong returns for most stocks over the long term [1] E-commerce Industry - E-commerce is projected to continue its growth, with only 16.2% of retail transactions in the U.S. being online as of the first quarter, indicating substantial room for growth [5] - Shopify is positioned to benefit from this trend as it enables merchants to create online storefronts, which is essential for success in the digital age [4][6] Shopify - Shopify has established itself as an innovator in the e-commerce market, offering a comprehensive suite of services including marketing, payment processing, and inventory management, which allows business owners to focus on other aspects of their operations [6] - The company has a competitive advantage due to high switching costs, which is crucial for long-term performance, although it is not consistently profitable and faces stiff competition [7] - Shopify has captured over 12% of the U.S. e-commerce market by gross merchandise volume and is expected to perform well in the next decade, especially after strategic changes that have brought it closer to profitability [8][9] Vertex Pharmaceuticals - Vertex Pharmaceuticals is a leading biotech company that has faced challenges, including disappointing phase 2 results for a drug and poor first-quarter results, but its long-term outlook remains attractive [10][11] - The company has a strong position in the cystic fibrosis market, being the only provider of medicines that address the underlying causes of the disease, which continues to drive top-line growth [11] - Vertex is expanding its product lineup with recent approvals for new medicines and has several other drugs in development, including a potential approval for a type 1 diabetes program by next year [14] - Despite recent performance issues, Vertex is considered a strong buy at current levels, with expectations for significant returns through 2035 [15]

Industry Overview - The bear market caused by the Federal Reserve's monetary tightening ended in 2023, but the biotech sector continues to lag behind the broader market, with the SDPR S&P Biotech ETF trading at levels similar to 2017 [1] - Despite the skepticism surrounding biotech stocks post-COVID-19 pandemic, there are signs of potential recovery in 2025, driven by companies making progress on new drugs and innovative therapies [2] Company Highlights Vertex Pharmaceuticals - Vertex Pharmaceuticals has been a leader in developing cystic fibrosis treatments, including Kalydeco and Trikafta, and has recently gained FDA approval for Journavx, a novel non-opioid pain medication [6][7] - Despite a recent 15% stock decline and an earnings miss, Vertex is still rated as a Moderate Buy with an average price target of $515, indicating potential upside [8] Regeneron Pharmaceuticals - Regeneron Pharmaceuticals, known for its blockbuster drug Eyelea, reported over $14 billion in sales last year and has expanded its portfolio with drugs like Dupixent and Kevzara [9][10] - Although Regeneron's stock has dropped over 45% in the past year, analysts maintain a consensus Moderate Buy rating with an average price target of $892, suggesting significant upside potential [12] Akero Therapeutics - Akero Therapeutics focuses on liver disease treatments, particularly efruxifermin for metabolic dysfunction-associated steatohepatitis (MASH), currently in Phase 3 trials [13][14] - The stock saw a significant increase in January following positive Phase 2 trial results, and analysts unanimously rate it as a Buy, with a consensus price target of $76, indicating over 90% upside potential [14]