安全性问题频出，Sarepta的AAV基因疗法Elevidys因患者死亡事件被FDA勒令暂停销售，Capsida Biotherapeutics公司的AAV相关临床试验也因儿童患者死 亡被迫中止。 加之"天价"问题，商业化局面迟迟难以打开，近一年来，AAV基因治疗领域退潮信号愈发强烈：今年2月，辉瑞宣布终止最后一个AAV项目Beqvez；5月， 福泰制药明确表示不再将AAV用作基因治疗项目的递送机制；9月底，渤健更是宣布直接清空了全部AAV管线。 昔日炙手可热的AAV赛道，突然间弥漫着撤退、危险的气息，这与几年前的盛况形成了鲜明对比。 作为曾经的明星载体，AAV成功助推了一批划时代的基因疗法问世，也吸引着无数biotech与大药企的押注。 然而，随着临床深入与商业落地加速，AAV的隐患无处遁形：高剂量毒性、免疫应答失控、天价成本……AAV载体一步步滑落神坛，甚至被大药企纷 纷"嫌弃"、抛弃。 困境，远比想象中更残酷。从被争相追逐的"黄金工具"，到被质疑甚至放弃的递送系统，AAV载体未来何去何从？ 三大硬伤 基因治疗草莽初创之际，其他递送系统尚存在诸多不成熟因素，AAV以其特性——结构稳定、表达持久、较低的免疫 ...

Core Insights - BioMarin Pharmaceutical (BMRN) reported Q2 2025 adjusted EPS of $1.44, exceeding the Zacks Consensus Estimate of $1.03, with a 50% year-over-year increase driven by higher product sales and lower operating expenses [1][9] - Total revenues reached $825.4 million, reflecting a 16% year-over-year increase, surpassing the Zacks Consensus Estimate of $766.2 million [1][9] Revenue Breakdown - Product revenues totaled $813 million, a 16% year-over-year increase, primarily due to higher sales from Voxzogo, Palynziq, Vimizim, and Aldurazyme, partially offset by lower Kuvan sales [2] - Voxzogo generated $221 million in sales, up 20% year over year, exceeding the Zacks Consensus Estimate of $219 million [3] - Enzyme Therapies sales rose 15% year over year to $555 million, driven by increased patient demand and large government orders [4] - Palynziq injection sales increased 20% year over year to $106 million, surpassing both the Zacks Consensus Estimate and internal model estimates [5] - Vimizim sales rose 21% year over year to $215 million, beating both the Zacks Consensus Estimate and internal model estimates [6] - Aldurazyme sales totaled $56 million, up 44% year over year, attributed to favorable order fulfillment timing [6] Financial Guidance - BioMarin revised its 2025 revenue forecast to $3.13-$3.20 billion, reflecting an 11% year-over-year increase at the midpoint [11] - Adjusted EPS guidance was raised to $4.40-$4.55, indicating a 27% growth over the previous year at the midpoint [14] - The company expects Voxzogo sales to be between $900-$935 million, with higher revenues anticipated in the second half of the year [12] Pipeline Developments - The acquisition of Inozyme added BMN 401, an investigational enzyme replacement therapy for rare disorders, with interim results expected in early 2026 [18][19] - BioMarin is advancing its CANOPY clinical program for Voxzogo, targeting additional indications with data expected in 2026 [22] - BMN 333, a long-acting formulation of CNP, is set to enter a phase II/III study in 2026, aiming for a potential launch in 2030 [23] - The company plans to file for expanded use of Palynziq in adolescents based on positive late-stage study results [21]

Core Insights - BioMarin Pharmaceutical reported first-quarter 2025 adjusted earnings per share of $1.13, exceeding the Zacks Consensus Estimate of 94 cents, with a year-over-year earnings increase of 59% driven by higher product sales and lower operating expenses [1][2] - Total revenues reached $745 million, reflecting a 15% year-over-year increase on a reported basis and a 17% increase on a constant-currency basis, surpassing the Zacks Consensus Estimate of $737 million [2] Revenue Breakdown - Product revenues amounted to $734.6 million, a 15% increase year over year, primarily due to higher sales from Voxzogo, Palynziq, and Aldurazyme, partially offset by lower Kuvan sales [4] - Voxzogo generated $214 million in sales, a 40% year-over-year increase, although it slightly missed the Zacks Consensus Estimate of $215 million [5] - Enzyme Therapies sales rose 8% year over year to $484 million, driven by increased patient demand and large government orders [7] - Palynziq injection sales totaled $93 million, up 22% year over year, while Vimizim sales declined 3% to $188 million [8] Other Revenue Insights - Aldurazyme sales reached $49 million, a 40% increase year over year, attributed to favorable order fulfillment timing with Sanofi [9] - New gene therapy Roctavian generated $11 million in sales compared to $1 million in the previous year, reflecting a strategic focus on the U.S., Germany, and Italy [10] Financial Guidance - BioMarin expects total revenues for the full year to be in the range of $3.1-$3.2 billion, indicating a 10% year-over-year increase at the midpoint, with Voxzogo projected to contribute significantly [11] - Adjusted earnings per share are anticipated to be between $4.20-$4.40, suggesting a 22% growth over the previous year [12] Pipeline Developments - BioMarin completed enrollment in a pivotal program for Voxzogo in patients with hypochondroplasia, with top-line data expected next year [13] - The company is advancing Voxzogo in clinical studies for multiple short-stature conditions, with ongoing enrollment [14] - BMN 333, a long-acting formulation of CNP, is in a first-in-human study, with initial data expected by the end of this year [15]

Industry Overview - The American trade structure is undergoing a significant transformation due to the Trump administration's new tariff policy, which has complex implications for the healthcare industry [1][3] - Pharmaceuticals are exempt from the new tariffs, protecting over $200 billion in annual U.S. pharmaceutical imports from immediate cost increases, although concerns remain about potential future tariffs on foreign-made drugs [3][4] - Medical devices and healthcare supplies are significantly affected by the tariffs, leading to warnings from industry associations about job losses, delayed innovation, and increased healthcare costs [4] Economic Impact - The new tariffs introduce macroeconomic volatility, with Goldman Sachs raising the odds of a U.S. recession from 20% to 45% due to trade-related uncertainties [5] - Retaliation from trade partners, particularly the EU and China, could further disrupt access to essential materials and technologies [5] Investment Opportunities - In the current volatile environment, investors are advised to focus on large-cap U.S.-based healthcare stocks, which are better positioned to absorb market shocks and maintain profitability [2][6] - Universal Health Services (UHS) is expected to see net revenue growth of 7.6% year-over-year in 2025, driven by expansions in licensed bed capacity and positive impacts from Medicaid programs [7] - BioMarin Pharmaceutical is benefiting from strong demand for its dwarfism drug Voxzogo and has promising developments in gene therapy, with a projected earnings growth of 22.2% for 2025 [9][11] - Abbott is unlocking new growth opportunities through its product pipeline, with a focus on next-generation diagnostics and biosimilars, expecting earnings growth of 10.3% in 2025 [12][13]

Core Insights - The article emphasizes the growing popularity of value investing as investors seek strategies that assess a stock's inherent potential amidst market volatility [1][2] - Warren Buffett's perspective on understanding a stock's "intrinsic value" is highlighted as a means to mitigate common pitfalls in value investing [2] - The article discusses four stocks that have recently performed well under a value investment strategy: Molson Coors Beverage, BioMarin Pharma, Devon Energy, and Jazz Pharmaceuticals [3] Value Investment Strategy - Value investing is gaining traction, but it can lead to "value traps" if investors do not fully understand the strategy [4] - Key metrics for identifying value stocks include dividend yield, P/E ratio, and P/B ratio, which help determine if a stock is undervalued [4] Importance of Earnings Growth - Investors are advised to focus on a stock's earnings growth potential over the next 12 to 24 months to avoid value traps [5] - The PEG ratio, which considers both price/earnings and earnings growth rate, is presented as a valuable metric for assessing intrinsic value [6] Screening Criteria for Value Stocks - Effective screening criteria for identifying promising value stocks include: - PEG Ratio less than industry median - P/E Ratio less than industry median - Zacks Rank 1 or 2 - Market Capitalization greater than $1 billion - Average 20-Day Volume greater than 50,000 - Percentage Change in F1 Earnings Estimate Revisions greater than 5% - Value Score of A or B [7] Featured Stocks - **Molson Coors Beverage (TAP)**: Holds a Zacks Rank 1, Value Score of A, and a five-year expected growth rate of 6.3% [8][9] - **BioMarin Pharma (BMRN)**: Has a Zacks Rank 2, Value Score of B, and a long-term historical growth rate of 77.6% [9][10] - **Devon Energy (DVN)**: Carries a Zacks Rank 2, Value Score of A, and a five-year historical growth rate of 40.1% [10][11] - **Jazz Pharmaceuticals (JAZZ)**: Holds a Value Score of A, Zacks Rank 1, and an expected long-term earnings growth rate of 7.4% [11][12]