Financial Data and Key Metrics Changes - BioMarin reported a revenue growth of 15% in Q1 2025, totaling $745 million compared to the same period last year [14] - Non-GAAP earnings per share reached $1.13, representing a 59% year-over-year increase, indicating significant profitability expansion [6][20] - Non-GAAP operating margin expanded to 35.7%, an increase of 11.9 percentage points year over year, driven by strong revenue performance and a reset in spending [18][20] - Positive operating cash flow was $174 million, a 271% increase over Q1 2024, supporting future growth investments [22] Business Line Data and Key Metrics Changes - VOXZOGO's global revenue reached $214 million, a 40% increase year over year, continuing its strong growth trajectory since approval [15] - Revenue from the Enzyme Therapies business unit grew 8% year over year to $484 million, with Palynziq contributing a 22% increase compared to Q1 2024 [15][27] - The company expects VOXZOGO full-year revenues to be between $900 million and $950 million, representing a 26% growth at the midpoint [16] Market Data and Key Metrics Changes - Approximately two-thirds of BioMarin's total revenues originate from outside the United States, providing insulation from U.S. macroeconomic conditions [9][10] - The company is focused on expanding access to VOXZOGO in more than 60 countries by 2027, with current access in 49 countries [24][26] Company Strategy and Development Direction - BioMarin is implementing a strategic transformation aimed at enhancing growth and profitability, with a focus on innovation and expansion [8][13] - The company is making progress on its innovation strategy, including pivotal studies for new products like Voxogo and Palynziq [12][28] - BioMarin is exploring business development opportunities aligned with its strengths in genetically defined conditions and clinical stage assets [45] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in navigating uncertainties in the pharmaceutical sector, citing the company's strong fundamentals and global revenue base [9][10] - The company anticipates continued strong performance and cash generation, supporting its growth agenda for 2025 and beyond [22][13] Other Important Information - BioMarin's R&D expenses were $147 million in Q1 2025, lower than the same quarter in 2024, reflecting a reprioritization strategy [17] - SG&A expenses decreased to $183 million year over year, attributed to cost transformation initiatives [18] Q&A Session Summary Question: Will Q2 revenue for VOXZOGO decline? - Management indicated that while revenue growth is expected to be stronger in the second half of the year, Q2 may show a slight decline due to global order dynamics [36][37] Question: How could U.S. tariffs impact financials? - Management stated that current guidance includes modest impacts from existing tariffs, but they are evaluating potential future scenarios and mitigation strategies [39][40] Question: What strategies are being implemented to drive further adoption of VOXZOGO? - The company is focusing on increasing awareness and expanding the prescriber base, particularly among pediatric endocrinologists, to drive adoption across all age groups [55][56] Question: What is the timeline for BMN 333 and its potential indications? - Management anticipates data from ongoing studies in 2027, with plans for pivotal studies based on the outcomes of BMN 333 [84] Question: How does the U.S. versus ex-U.S. sales for VOXZOGO compare? - Approximately 75% of VOXZOGO revenues come from outside the U.S., with expectations for this split to fluctuate as the brand matures [88]

Financial Performance - Total revenues for Q1 2025 reached $745 million, a 15% year-over-year increase[13] - VOXZOGO revenues experienced substantial growth, increasing by 40% year-over-year to $214 million[14] - Enzyme Therapies revenues grew by 8% year-over-year, driven by PALYNZIQ's 22% growth[14, 17] - Non-GAAP Diluted EPS increased by 59% year-over-year, reaching $1.13[14] - GAAP Diluted Earnings per Share (EPS) showed significant improvement, increasing by 107% to $0.95[14] - Non-GAAP Operating Margin percentage was 35.7%, an increase of 11.9 percentage points year-over-year[14] Guidance and Pipeline - Full-year 2025 guidance reaffirmed for Total Revenues ($3.1 billion to $3.2 billion), Non-GAAP Operating Margin (32% to 33%), and Non-GAAP Diluted EPS ($4.20 to $4.40)[20] - Initial data for BMN 351 for DMD is expected in the second half of 2025[17] - Pivotal study with VOXZOGO in hypochondroplasia fully enrolled in April 2025, with potential launch in 2027[17] - Positive pivotal topline results for PALYNZIQ in adolescents ages 12-17 shared in April, supporting planned submissions to regulatory authorities in the U.S and EU in 2H'25[17]

Exhibit 99.1 Contact: Investors: Media: Traci McCarty Marni Kottle BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc. (415) 455-7558 (650) 374-2803 BioMarin Reports First Quarter 2025 Results and Reaffirms Full-year Guidance First Quarter 2025 Total Revenues of $745 million (+15% Y/Y and +17% at Constant Currency Y/Y) First Quarter 2025 Business Highlights Innovation 2 First Quarter 2025 GAAP Diluted Earnings Per Share (EPS) of $0.95 (+107% Y/Y) First Quarter 2025 Non-GAAP Diluted EPS of $1.13 (+59% ...

SAN RAFAEL, Calif., April 17, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Alexander Hardy, President and Chief Executive Officer of BioMarin, will host a conference call and webcast on Thursday, May 1, 2025, at 4:30 p.m. ET to discuss first quarter 2025 financial results and provide a general business update.Dial-in Number U.S. / Canada Dial-in Number: 888-596-4144International Dial-in Number: 646-968-2525Conference Call ID: 4327591U.S. / Canada Replay Dial-in Numbe ...

Market Overview - The stock market has experienced historic volatility, with the S&P 500 losing 10.5% in just two trading days, marking its fifth-largest two-day decline in 75 years [2] - President Trump's temporary reversal of tariff policies led to significant single-session gains for major indices on April 9, with the Dow Jones increasing by 2,963 points, S&P 500 by 474 points, and Nasdaq Composite by 1,857 points, all record highs [3] Investment Opportunities - Despite the market being historically pricey, price dislocations have emerged, allowing for the purchase of undervalued stocks [4] Pfizer - Pfizer is viewed as mispriced, with concerns over potential tariffs on pharmaceuticals being considered overblown; the company was nearing an 8% dividend yield [5] - Sales from COVID-19 therapies dropped from over $56 billion in 2022 to approximately $11 billion last year, yet Pfizer's net sales increased by 52% to $63.6 billion from 2020 to 2024 [6][7] - The acquisition of Seagen is expected to enhance Pfizer's growth, with a forward P/E ratio just above 7 being attractive [8] Sirius XM Holdings - Sirius XM is identified as having a price dislocation, with its unique position as the only licensed satellite-radio operator providing it with pricing power [9][10] - The company generates 76% of its net sales from subscriptions, making its cash flow more predictable during economic downturns; shares were trading at a forward P/E of 6, the lowest since going public [12] Intel - Intel's shares are trading below book value, presenting a buying opportunity despite challenges from competitors [13] - The company remains a dominant player in CPUs for laptops and desktops, and new CEO Lip-Bu Tan is expected to improve operations and margins [15] - Intel's potential in AI remains, despite current struggles in the market [16] Fastly - Fastly is recognized for its content delivery network and security solutions, with a compelling long-term outlook despite recent growth disappointments [18][19] - The company has a net retention rate of 102% and a consistent annual revenue retention rate above 99%, indicating customer loyalty [20] - Shares are trading below book value and at a low sales multiple, making it an attractive investment [21] BioMarin Pharmaceutical - BioMarin focuses on orphan-disease therapies, which leads to predictable demand and strong pricing power [23] - The company reported $2.85 billion in sales last year, an 18% increase, with its top-selling drug Voxzogo contributing significantly [24] - BioMarin's valuation is at historic lows, with a forecasted P/E of just over 10 for 2026 [25] PubMatic - PubMatic is positioned well in the digital advertising space, focusing on connected TV and mobile, with a significant growth segment [28] - The company has developed its own cloud infrastructure, which is expected to enhance margins as it scales [29] - With a strong cash position and positive operating cash flow for a decade, shares were valued at 10 times forecast EPS, presenting a clear price dislocation [30]

Core Viewpoint - BioMarin Pharmaceutical (BMRN) is positioned well to potentially beat earnings estimates in its upcoming quarterly report, supported by a strong history of exceeding expectations [1]. Group 1: Earnings Performance - BioMarin has a solid track record of surpassing earnings estimates, with an average surprise of 21.35% over the last two quarters [2]. - In the most recent quarter, BioMarin reported earnings of $0.92 per share, exceeding the expected $0.73 per share by 26.03% [3]. - For the previous quarter, the company reported $0.91 per share against an expectation of $0.78 per share, resulting in a surprise of 16.67% [3]. Group 2: Earnings Estimates and Predictions - Recent changes in earnings estimates for BioMarin have been favorable, with a positive Zacks Earnings ESP (Expected Surprise Prediction) indicating a strong likelihood of an earnings beat [6]. - The combination of a positive Earnings ESP and a Zacks Rank 2 (Buy) suggests that another earnings beat is likely [9]. - BioMarin currently has an Earnings ESP of +6.51%, reflecting increased analyst optimism regarding the company's earnings prospects [9]. Group 3: Importance of Earnings ESP - Stocks with a positive Earnings ESP and a Zacks Rank of 3 (Hold) or better have a nearly 70% chance of producing a positive surprise [7]. - The Zacks Earnings ESP compares the Most Accurate Estimate to the Zacks Consensus Estimate, with the Most Accurate Estimate being more reflective of recent analyst revisions [8]. - It is crucial to check a company's Earnings ESP before quarterly releases to enhance the likelihood of successful investment decisions [10].

Company Overview - BioMarin Pharmaceutical (BMRN) shares increased by 6.3% to $59.43, following a significant trading volume, contrasting with a 20.7% loss over the past four weeks [1] - The rise in share price was influenced by President Trump's announcement of a 90-day pause on tariffs against non-retaliating countries [1] Earnings Expectations - BioMarin is projected to report quarterly earnings of $0.95 per share, reflecting a year-over-year increase of 33.8% [2] - Expected revenues for the upcoming quarter are $738.75 million, which is a 13.9% increase compared to the same quarter last year [2] Earnings Estimate Revisions - The consensus EPS estimate for BioMarin has been slightly revised higher in the last 30 days, indicating a positive trend that typically correlates with stock price appreciation [3] - The stock currently holds a Zacks Rank of 2 (Buy), suggesting favorable market sentiment [3] Industry Comparison - BioMarin operates within the Zacks Medical - Biomedical and Genetics industry, where another company, Merus N.V. (MRUS), saw a 13.4% increase in its stock price, closing at $39.56 [3] - Merus' consensus EPS estimate has decreased by 3.7% to -$1.17, representing a significant year-over-year decline of 98.3% [4]

Core Viewpoint - BioMarin Pharmaceutical (BMRN) has received a Zacks Rank 2 (Buy) upgrade, indicating a positive outlook based on rising earnings estimates, which significantly influence stock prices [1][3]. Earnings Estimates and Stock Performance - The Zacks rating system emphasizes the importance of earnings estimate revisions, which are strongly correlated with stock price movements, particularly due to institutional investors' reliance on these estimates for valuation [4][6]. - BioMarin is projected to earn $4.30 per share for the fiscal year ending December 2025, reflecting a year-over-year increase of 22.2% [8]. Analyst Sentiment and Market Position - Over the past three months, the Zacks Consensus Estimate for BioMarin has increased by 9.6%, indicating a positive trend in analyst sentiment [8]. - The upgrade to Zacks Rank 2 places BioMarin in the top 20% of Zacks-covered stocks, suggesting potential for market-beating returns in the near term [10].

Core Insights - BioMarin Pharmaceutical Inc. announced positive results from its phase 3 Pegasus trial for its drug Palynziq (pegvaliase) aimed at treating patients with phenylketonuria (PKU) [2] Company Overview - BioMarin Pharmaceutical Inc. is focused on developing therapies for rare genetic diseases, with Palynziq being a significant product in its pipeline [2] Industry Context - The success of the Pegasus trial may enhance BioMarin's position in the biotech sector, particularly in the treatment of PKU, which is a rare metabolic disorder [2]

Core Insights - BioMarin Pharmaceutical Inc. announced that the Phase 3 PEGASUS trial for PALYNZIQ met its primary efficacy endpoint, showing a statistically significant reduction in blood Phe levels in adolescents aged 12-17 with phenylketonuria (PKU) compared to diet alone [1][2][3] - The company plans to submit detailed results to global health authorities later this year to request a label expansion for PALYNZIQ to include adolescents [2][3] Company Overview - BioMarin has been a pioneer in developing treatments for PKU for over two decades, with PALYNZIQ being the first and only enzyme therapy approved for adults with PKU [3][9] - The company aims to continue its innovation and research pipeline to provide meaningful options for patients transitioning to adult living [3] Study Details - The PEGASUS trial is a Phase 3 multi-center open-label randomized controlled study involving 55 adolescents aged 12-17 with PKU, focusing on the safety and efficacy of PALYNZIQ compared to diet alone [4][5] - The study consists of two parts: a primary treatment phase lasting 73 weeks and an extension phase of up to 80 weeks for monitoring [5] Product Information - PALYNZIQ works by substituting the deficient PAH enzyme in PKU with a PEGylated version to break down Phe, and it is administered with a dosing regimen designed for tolerability [6][12] - The product is currently approved for adults in the U.S. and for individuals aged 15 and older in Japan, among other regions, for those with uncontrolled blood Phe levels greater than 600 micromol/L [7][12] Patient Management - The management of PKU typically involves a strict Phe-restricted diet, which can be challenging for patients, especially during adolescence and adulthood [11] - BioMarin emphasizes the importance of monitoring blood Phe levels and dietary intake for effective management of PKU [20]