Financial Data and Key Metrics Changes - BioMarin reported an 11% year-over-year increase in total revenue, with cash and investments reaching approximately $2 billion at the end of Q3 2025 [5][6][15] - The company raised its full-year 2025 total revenue guidance to a range of $3.15 billion, reflecting strong performance and operational efficiencies [12][15] - Non-GAAP diluted earnings per share guidance was updated to between $3.50 and $3.60, with an operating margin guidance of 26% to 27% [15][17] Business Line Data and Key Metrics Changes - The Enzyme Therapies business unit experienced 8% year-to-date growth, with Palynziq and VoxoGo each increasing by over 20% year-to-date [12][19] - VoxoGo revenue is expected to reach between $900 million and $935 million for the full year 2025, with a 24% increase year-to-date compared to 2024 [6][12][20] - The Skeletal Conditions business unit, driven by FOXOGO, is projected to generate over $900 million in revenue for 2025, representing a 25% growth at the midpoint of guidance [6][19] Market Data and Key Metrics Changes - Approximately 75% of year-to-date VoxoGo revenue was generated outside the U.S., indicating strong international demand [20][21] - VoxoGo is now available in 55 countries, with significant growth in patient numbers quarter-over-quarter [20][21] Company Strategy and Development Direction - BioMarin is focusing on strategic investments in the Enzyme Therapies and Skeletal Conditions business units while pursuing options to divest ROCTAVIAN [9][10] - The company aims to expand its global footprint and deepen penetration in existing markets, particularly for VoxoGo and upcoming indications [20][21] - Future growth is anticipated through new product launches and business development opportunities, with a focus on advancing internal pipelines [11][15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving record commercial results for the full year, despite challenges from potential competition [5][11] - The company is monitoring various market dynamics and has developed multiple scenarios for future revenue, particularly concerning VoxoGo competition [17][42] - Management emphasized the importance of maintaining value-creating activities while pursuing operational efficiencies [75] Other Important Information - A charge of $221 million for acquired in-process research and development related to the Enizyme Pharma acquisition impacted Q3 results [14] - The company is preparing for pivotal data readouts and potential new approvals in the coming quarters, particularly for hypochondroplasia and BMN 333 [36][37] Q&A Session Summary Question: Can you elaborate on the 2027 guidance and the changes since it was initially issued? - Management indicated that various factors, including potential VoxoGo competition and the acquisition of Inozyme, influenced the revised outlook, leading to a range of estimates rather than a specific forecast [39][42] Question: Why were VoxoGo sales down quarter-over-quarter? - Management noted that the decline was due to timing shifts in larger orders and reaffirmed the total revenue range for VoxoGo for the year [48][50] Question: What is the company's strategy for business development? - Management highlighted a focus on early-stage collaborations and Phase III assets, emphasizing that business development remains a high priority [52][54] Question: What are the expectations for the upcoming BMN 333 data? - Management stated that they are looking for significant increases in free CNP levels to demonstrate clinical superiority over VoxoGo [64][66] Question: Can you clarify the 2027 cash flow targets? - Management confirmed that the cash flow targets are tied to revenue scenarios and emphasized ongoing optimization initiatives [79][80]

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Third Quarter 2025 Earnings October 27, 2025 Forward-Looking Statements This presentation and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: future financial performance, including the expectations of Total Revenues, Non-GAAP Operating Margin percentage, Non-GAAP Diluted EPS and Operating Cash Flow for, in certain instances, the full-year 2025 and future pe ...

Exhibit 99.1 Contact: Investors: Media: Traci McCarty Marni Kottle BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc. (415) 455-7558 (650) 374-2803 BioMarin Reports Third Quarter 2025 Results and Provides Corporate Update Raises Full-year 2025 Total Revenues Guidance at the Midpoint; Reaffirms VOXZOGO Full-year Outlook 2025 Year-to-date Total Revenues Increased 11% Y/Y Led by More Than 20% Revenue Growth for PALYNZIQ and VOXZOGO BioMarin Focuses Commercial Portfolio; Pursuing Options to Divest ROCTAV ...

Accessibility StatementSkip Navigation Raises Full-year 2025 Total Revenues Guidance at the Midpoint; Reaffirms VOXZOGO Full-year Outlook 2025 Year-to-date Total Revenues Increased 11% Y/Y Led by More Than 20% Revenue Growth for PALYNZIQ and VOXZOGO BioMarin Focuses Commercial Portfolio; Pursuing Options to Divest ROCTAVIAN Conference Call and Webcast Scheduled Today at 4:30 p.m. ET SAN RAFAEL, Calif., Oct. 27, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial result ...

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the best healthcare stocks with the highest upside. In a report released on October 14, Cantor Fitzgerald analyst Olivia Brayer reiterated a Buy rating for BioMarin Pharmaceutical Inc. (NASDAQ:BMRN). However, the analyst slashed the firm’s price target on the stock from $90 to $80. BioMarin Pharmaceutical Inc. (BMRN): Among Small-Cap Healthcare Stocks Hedge Funds is Buying J.P. Morgan analyst Jessica Fye also maintained a Buy rating on BioMarin Pharmac ...

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the best biotech stocks with high potential. BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) reported total revenues of $825 million in fiscal Q2 2025, up 16% year-over-year and up 17% year-over-year at constant currency. GAAP diluted earnings per share (EPS) for the quarter reached $1.23. BioMarin Pharmaceutical Inc. (BMRN): Among Small-Cap Healthcare Stocks Hedge Funds is Buying In a report released on September 17, Christopher Raymond from Raymond James ...

Group 1: Company Overview - BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is a biotechnology company focused on providing solutions for serious and rare diseases and medical conditions, established in 1996 and headquartered in San Rafael, California [4] Group 2: Investment Interest - Hedge funds are showing strong interest in BioMarin, with Tealwood Asset Management Inc. increasing its holdings by 37.4% in the second quarter, acquiring an additional 5,874 shares for a total investment of $1,185,000 [1] Group 3: Future Outlook - The future of BioMarin is characterized by three key aspects: valuation, growth, and pipeline [2] - The company generates only 34% of its revenues from the U.S. healthcare system, which is currently under scrutiny for its high costs, indicating a relatively stable position for BioMarin [2] - BioMarin's pipeline includes significant candidates like Palynziq, which targets phenylketonuria (PKU) with an estimated global prevalence of 1 in 10,000 to 15,000 people, and is projected to have a compound annual growth rate (CAGR) of 9.38% from 2025 to 2030 [3]

Core Insights - Artificial intelligence (AI) is identified as the greatest investment opportunity of the current era, with a strong emphasis on the urgent need for energy to support its growth [1][2][3] - A specific company is highlighted as a key player in the AI energy sector, owning critical energy infrastructure assets that are essential for meeting the increasing energy demands of AI technologies [3][7][8] Investment Opportunity - Wall Street is investing heavily in AI, but there is a looming energy crisis as AI technologies require vast amounts of electricity, comparable to the consumption of small cities [2][3] - The company in focus is positioned to benefit from the surge in demand for electricity driven by AI data centers, making it a potentially lucrative investment [3][8] Company Profile - The company owns significant nuclear energy infrastructure assets and is capable of executing large-scale engineering, procurement, and construction projects across various energy sectors [7][8] - It is noted for being debt-free and having a substantial cash reserve, which is nearly one-third of its market capitalization, providing financial stability and growth potential [8][10] Market Position - The company is described as a "toll booth" operator in the AI energy boom, collecting fees from energy exports and benefiting from the onshoring trend driven by tariffs [5][6] - It has an equity stake in another AI-related company, offering investors indirect exposure to multiple growth engines in the AI sector [9][10] Future Outlook - The article emphasizes the importance of investing in AI and energy infrastructure as the future of technology and economic growth, with a call to action for investors to seize the opportunity before significant price increases occur [11][12][13]

Core Insights - The biotechnology sector is increasingly recognized for its dynamic growth and potential for high returns, particularly in gene therapy and precision medicine [2][3] - Small- and mid-cap biotech companies are leading biopharmaceutical innovation, contributing to two-thirds of the industry's R&D pipeline [2] Company Highlights - **BioMarin Pharmaceutical Inc. (NASDAQ:BMRN)** - Hedge fund holdings increased to 58, with Tealwood Asset Management expanding its position by 37.4%, now owning shares worth $1,185,000 [7] - The company's future is characterized by valuation, growth, and a strong pipeline, with only 34% of revenues derived from the U.S. healthcare market [8] - Key product Palynziq has a significant addressable market, with an estimated 9.38% CAGR from 2025 to 2030 for the drug [9][10] - **Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY)** - Hedge fund holdings also at 58, with Wealth Enhancement Advisory Services reducing its stake by 64.5%, now holding shares worth $1,699,000 [11] - The company is expected to achieve over $1 billion in quarterly revenue by year-end and more than $1 billion in adjusted earnings in the upcoming year [12] - Alnylam's stock has outperformed the market by nearly 50% over the past year, driven by the successful launch of Amvuttra in TTR-CM [13][14]