Core Viewpoint - BioMarin Pharmaceutical Inc. has announced the acquisition of Inozyme Pharma, Inc. for $4.00 per share, totaling approximately $270 million, which is expected to enhance BioMarin's enzyme therapies portfolio and provide a potential first-in-class treatment for ENPP1 Deficiency [1][2][3] Acquisition Details - The acquisition is an all-cash transaction, unanimously approved by both companies' Boards of Directors, and is anticipated to close in Q3 2025, pending regulatory approval and other customary conditions [1][7][8] - BioMarin will commence a cash tender offer for all outstanding shares of Inozyme common stock at a price of $4.00 per share, with Inozyme's Board recommending that stockholders tender their shares [7][8] Product Information - INZ-701, the late-stage enzyme replacement therapy being developed for ENPP1 Deficiency, is expected to have pivotal data readout in early 2026, with potential regulatory approval in 2027 [2][4] - ENPP1 Deficiency is a rare genetic condition that leads to serious health complications, including increased cardiovascular mortality risk and severe rickets [6] Clinical Development - INZ-701 is being developed for patients of all ages, with ongoing studies for infants and plans for supportive studies for adolescents and adults [4][5] - A Phase 1/2 study in adults showed a favorable safety profile for INZ-701, with improvements in key health indicators [5] Financial Guidance - BioMarin reaffirmed its full-year 2025 financial guidance and plans to achieve a 40% Non-GAAP Operating Margin in 2026, excluding the impact of the acquisition [9]

Core Insights - New data indicates that treatment with VOXZOGO significantly improves tibial bowing in children with achondroplasia, addressing a common source of pain and functional impairment [1][2] - A large observational study revealed higher rates of comorbidities, surgeries, and doctor visits in individuals with hypochondroplasia compared to those without [4] Group 1: VOXZOGO Treatment Efficacy - VOXZOGO treatment resulted in a significant reduction in tibial bowing in children compared to placebo, with sustained improvements observed over several years [2] - Continuous and early treatment with VOXZOGO is associated with sustained increases in annualized growth velocity (AGV), potentially leading to a height increase of 21.7 cm in girls and 26.4 cm in boys when treated from 6 months until final adult height [3] Group 2: Hypochondroplasia Insights - Research on hypochondroplasia indicates that individuals experience significantly higher rates of comorbidities and increased healthcare utilization, including more frequent doctor visits and surgeries [4][5] - Early diagnosis of hypochondroplasia is emphasized as critical for timely intervention and management [5] Group 3: Ongoing Research and Development - BioMarin is advancing its CANOPY clinical program for VOXZOGO, with ongoing studies in various conditions including hypochondroplasia and Turner syndrome, and plans to share topline data in 2026 [7] - VOXZOGO is currently approved in multiple regions for increasing linear growth in children with achondroplasia, with ongoing evaluations to confirm clinical benefits [15][19]

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]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 ______________________________________ Form 10-Q ______________________________________ (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 Or☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to . Commission File Number: 000-26727 ________________________ ...

BioMarin Pharmaceutical (BMRN) came out with quarterly earnings of $1.13 per share, beating the Zacks Consensus Estimate of $0.94 per share. This compares to earnings of $0.71 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 20.21%. A quarter ago, it was expected that this rare disease biopharmaceutical would post earnings of $0.73 per share when it actually produced earnings of $0.92, delivering a surprise of 26.03%.Over the l ...

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 [12] - Non-GAAP earnings per share reached $1.13, representing a 59% year-over-year increase, indicating significant profitability expansion [5][19] - Non-GAAP operating margin expanded to 35.7%, an increase of 11.9 percentage points year-over-year [15] 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 [13] - 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 [13][22] - Full-year revenue expectations for Voxzogo are between $900 million and $950 million, representing a 26% growth at the midpoint [14] 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 [7][9] - The company is analyzing potential exposure to pharmaceutical tariffs and believes its global revenue base offers protection against such impacts [8][9] Company Strategy and Development Direction - BioMarin is focused on implementing changes to its strategy and operating model announced in September 2024, aiming for record full-year performance in 2025 [6][11] - The company is advancing its innovation strategy, with pivotal studies for Voxzogo and plans for new product applications in the second half of 2025 [10][24] - BioMarin aims to leverage its established global capabilities to support growth and innovation, particularly in rare disease treatments [11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in BioMarin's ability to navigate market uncertainties due to its focus on rare conditions and strong cash generation [6][7] - The company anticipates continued strong performance and innovation in the coming quarters, supported by its strategic transformation [11][19] Other Important Information - Non-GAAP R&D expenses were $147 million, lower than the same quarter in 2024, reflecting a reprioritization of R&D efforts [14] - Non-GAAP SG&A expenses decreased to $183 million due to cost transformation initiatives [15] Q&A Session Summary Question: Will Q2 revenue for Voxzogo decline? - Management indicated that while Q1 showed growth, Q2 revenue may appear flat due to global order dynamics, with expectations for stronger growth in the second half of the year [35][36] Question: How could potential U.S. tariffs impact financials? - Management stated that current guidance includes modest impacts from existing tariffs, but they are modeling various scenarios for potential future tariffs [36][37] 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? - BMN 333 is in a Phase I study, with plans for pivotal studies in 2027, and the company is evaluating its future role in additional indications [84] Question: How have U.S. versus ex-U.S. sales evolved for Voxzogo? - Approximately 75% of Voxzogo revenues come from outside the U.S., and this split is expected to fluctuate as the brand matures [88]

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 ...