44th Annual J.P. Morgan Healthcare Conference Alexander Hardy Chief Executive Officer Greg Friberg Chief R&D Officer BioMarin Pharmaceutical Inc. January 12, 2026 These risks and uncertainties include, without limitations, risks relating to the completion of BioMarin's normal quarterly and annual accounting and financial statement closing procedures for the quarter and the year ended December 31, 2025; the timing of orders for commercial products; BioMarin's ability to meet product demand; risks and uncerta ...

Core Insights - BioMarin Pharmaceutical Inc. is identified as a stock with exponential growth potential heading into 2026, driven by strong performance in its Enzyme Therapies and Skeletal Conditions business units [1] - The company reported a 4% increase in total revenues for Q3 2025, amounting to $776 million, supported by over 20% revenue growth from PALYNZIQ and VOXZOGO [3] Financial Performance - Total revenues for Q3 2025 reached $776 million, reflecting a 4% increase compared to Q3 2024 [3] - Revenue growth was significantly attributed to new patient initiations for VOXZOGO and PALYNZIQ across all regions, indicating a robust demand for these therapies [3] Strategic Focus - The company is concentrating on its Enzyme Therapies and Skeletal Conditions as key components of its growth strategy, while also exploring new business development opportunities [2] - BioMarin is considering divesting ROCTAVIAN to streamline its portfolio and focus on strategic priorities [2]

Core Insights - BioMarin Pharmaceutical reported $776.13 million in revenue for Q3 2025, a 4.1% year-over-year increase, but fell short of the Zacks Consensus Estimate of $784.4 million, resulting in a surprise of -1.05% [1] - The company’s EPS for the quarter was $0.12, significantly lower than the $0.91 reported a year ago, but exceeded the consensus estimate of -$0.15, resulting in an EPS surprise of +180% [1] Revenue Breakdown - ALDURAZYME generated $54 million, surpassing the average estimate of $45.06 million from 11 analysts [4] - KUVAN reported $24 million, exceeding the average estimate of $21.1 million, but showed a year-over-year decline of -14.3% [4] - NAGLAZYME achieved $122 million, slightly below the estimated $123.14 million, reflecting a -7.6% change year-over-year [4] - VIMIZIM brought in $183 million, which was lower than the average estimate of $192.42 million, but represented a +2.8% year-over-year increase [4] - Royalty and other revenues totaled $15.32 million, exceeding the average estimate of $11.31 million, marking a +29% year-over-year increase [4] - PALYNZIQ generated $109 million, surpassing the average estimate of $103.1 million, with a year-over-year increase of +19.8% [4] - VOXZOGO reported $218 million, slightly below the average estimate of $233.96 million, but showed a +14.7% year-over-year increase [4] - ROCTAVIAN's revenue was $3 million, significantly lower than the estimated $9.39 million, reflecting a -57.1% year-over-year decline [4] - Total net product revenues were $760.81 million, below the average estimate of $778.49 million, but represented a +3.7% year-over-year increase [4] - BRINEURA achieved $48 million, exceeding the average estimate of $43.28 million, with a year-over-year increase of +29.7% [4] Stock Performance - BioMarin's shares returned +1.2% over the past month, underperforming the Zacks S&P 500 composite's +2.5% change [3] - The stock currently holds a Zacks Rank 4 (Sell), indicating potential underperformance relative to the broader market in the near term [3]

Financial Data and Key Metrics Changes - BioMarin reported an 11% year-over-year increase in total revenues, leading to an updated full-year 2025 total revenue guidance of $3.15 billion, reflecting double-digit growth [5][11][14] - The company’s cash and investments balance reached approximately $2 billion at the end of Q3 2025, with operating cash flow of $369 million for the quarter and $728 million year-to-date [5][14] - Non-GAAP diluted earnings per share guidance was updated to between $3.50 and $3.60, with non-GAAP operating margin guidance revised to between 26% and 27% [14][15] Business Line Data and Key Metrics Changes - The enzyme therapies business unit achieved 8% year-to-date growth, with PALYNZIQ driving over 20% growth [11][17] - VOXZOGO, the treatment for achondroplasia, is expected to generate over $900 million in revenue for 2025, representing a 25% growth at the midpoint of guidance [7][11] - VOXZOGO revenue increased by 24% year-to-date compared to 2024, with a 15% growth in Q3 alone [11][18] Market Data and Key Metrics Changes - Approximately 75% of year-to-date VOXZOGO revenue was generated outside the U.S., indicating strong international demand [19][20] - The company is expanding its global footprint, with VOXZOGO now available in 55 countries, and anticipates significant growth opportunities as it penetrates new markets [18][19] Company Strategy and Development Direction - BioMarin is focusing on strategic investments in enzyme therapies and skeletal conditions, while divesting from non-core assets like ROCTAVIAN [9][10] - The company is preparing for the launch of VOXZOGO's second indication for hypochondroplasia, with pivotal data expected in the first half of 2026 [8][21] - BioMarin aims to enhance its business development strategy by pursuing early-stage collaborations and phase III assets to drive growth [43][44] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving record commercial results for the full year, despite competitive pressures and market dynamics [5][10] - The company acknowledged the uncertainty surrounding potential competition for VOXZOGO, leading to a range of revenue scenarios for 2027 [15][33] - Management emphasized the importance of maintaining strong patient adherence and expanding the prescriber base to sustain growth [20][69] Other Important Information - BioMarin announced a $221 million charge for acquired in-process research and development related to the Enzyme Pharma acquisition, impacting both GAAP and non-GAAP financials [12][13] - The company is advancing its pipeline with multiple data readouts and regulatory milestones expected in the coming quarters [30][31] Q&A Session Summary Question: Can you elaborate on the 2027 guidance and the changes since it was initially issued? - Management indicated that the original $4 billion 2027 outlook was reassessed due to potential VOXZOGO competition and other market dynamics, leading to a range of estimates rather than a specific forecast [33][34][35] Question: Why were VOXZOGO sales down quarter-over-quarter? - Management attributed the decline to timing shifts in larger orders and reaffirmed the total VOXZOGO revenue range for the year, emphasizing steady patient additions across all markets [39][41][42] Question: What is the strategy for business development given the focus on VOXZOGO? - Management highlighted the importance of business development as a priority, with ongoing efforts to pursue deals that could enhance the company's portfolio [43][44] Question: What are the expectations for the upcoming BMN-333 data? - Management indicated that they are looking for significant increases in exposure levels compared to VOXZOGO to demonstrate clinical superiority [53][54] Question: Can you provide insights on the long-term growth targets and cash flow expectations? - Management confirmed that the target for 40% non-GAAP operating margins remains, but acknowledged that it is dependent on top-line performance [59][60]

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]

Financial Performance - Year-to-date 2025 total revenues increased by 11% year-over-year, driven by over 20% year-over-year revenue growth for PALYNZIQ and VOXZOGO[11] - The company raised full-year 2025 total revenues guidance to between $3150 million and $3200 million, representing 11% fiscal year 2025 growth year-over-year[13] - VOXZOGO's fiscal year 2025 revenue outlook is reaffirmed to be between $900 million and $935 million[13] - Q3 2025 GAAP operating margin was negative 60%, a decrease of 213 percentage points year-over-year, while non-GAAP operating margin was 28%, a decrease of 249 percentage points year-over-year[21] - Q3 2025 GAAP diluted EPS was negative $016, a decrease of 129%, while non-GAAP diluted EPS was $012, a decrease of 87%[21] - Operating cash flow for Q3 2025 was $369 million, an increase of 66%[21] - Full-year 2025 non-GAAP operating margin guidance updated to 26% - 27% and non-GAAP diluted EPS guidance updated to $350 - $360[24] Product Performance - VOXZOGO Q3 year-to-date revenue increased 24% year-over-year[20] - Enzyme Therapies Q3 year-to-date revenue increased 8% year-over-year[20] - VOXZOGO Q3 2025 revenue was $218 million, a 15% increase year-over-year, and Q3 2025 year-to-date revenue was $654 million, a 24% increase year-over-year[33] Research and Development - The company is preparing for VOXZOGO in hypochondroplasia (HCH) launch, with pivotal data readout expected in the first half of 2026[38]

Core Insights - BioMarin Pharmaceutical Inc. raised its full-year 2025 total revenues guidance at the midpoint, reflecting strong demand for its therapies, particularly VOXZOGO and PALYNZIQ, which saw over 20% revenue growth [1][6][11] - The company reported a GAAP net loss of $31 million for Q3 2025, a significant decline from a net income of $106 million in Q3 2024, primarily due to a $221 million in-process research and development charge related to the acquisition of Inozyme Pharma [6][7][11] - BioMarin plans to divest ROCTAVIAN while continuing to evaluate out-licensing options for the gene therapy, aligning with its strategic focus on core business units [2][6][11] Financial Performance - Total revenues for Q3 2025 were $776 million, a 4% increase from $746 million in Q3 2024, driven by strong growth in VOXZOGO and PALYNZIQ [6][7] - Year-to-date total revenues increased by 11% to $2.347 billion compared to $2.107 billion in the same period last year [7][11] - Non-GAAP income for Q3 2025 decreased to $22 million from $178 million in Q3 2024, reflecting the impact of the IPR&D charge [6][7] Product Performance - VOXZOGO revenue for Q3 2025 was $218 million, up 15% year-over-year, with year-to-date revenue increasing 24% to $654 million [7][11] - PALYNZIQ achieved a 20% year-over-year growth in Q3 2025, with total enzyme therapies revenue growing 8% year-to-date [6][7] - The company is advancing its pipeline with several upcoming pivotal data readouts and potential product launches, including BMN 333 and BMN 401 [6][11] Strategic Focus - BioMarin is concentrating on its Enzyme Therapies and Skeletal Conditions business units, which are central to its growth strategy [2][6] - The company aims to expand VOXZOGO access in over 60 countries by 2027, with significant uptake in markets outside the U.S. [6][11] - The decision to divest ROCTAVIAN is part of a broader strategy to streamline its portfolio and focus on high-potential therapies [2][6]

Core Thesis - BioMarin Pharmaceutical Inc. is positioned as a leader in the rare disease space with a strong pipeline and revenue growth potential despite recent stock underperformance [1][5]. Financial Performance - BioMarin reported total revenues of $813 million for Q2 2025, reflecting a 16% year-over-year increase, driven by a 20% growth in the U.S. and international expansion [2][3]. - Five of its eight core products achieved double-digit revenue growth, with VOXZOGO and VIMIZIM contributing over half of total revenue [3]. - Operating income increased by 130% year-over-year, with margins expanding significantly to 62.3% for operating income and 29.1% for net income [4]. Market Position and Growth Potential - The company has a robust clinical and preclinical pipeline, with potential launches from products like BMN-333, BMN-401, BMN-351, and BMN-349 expected in 2026-2027 [3]. - Analyst consensus suggests a potential upside of 33-65% for the stock, supported by a recent $270 million acquisition of Inozyme Pharma [5]. Balance Sheet Strength - BioMarin's balance sheet has strengthened, with cash up 25%, total debt down 45%, a debt-to-equity ratio of 0.1, and a current ratio of 5.56x [4].

Core Insights - BioMarin Pharmaceutical reported revenue of $825.41 million for the quarter ended June 2025, reflecting a year-over-year increase of 15.9% [1] - The company's EPS was $1.44, up from $0.96 in the same quarter last year, indicating a significant improvement in profitability [1] - The reported revenue exceeded the Zacks Consensus Estimate of $766.23 million by 7.72%, and the EPS surpassed the consensus estimate of $1.03 by 39.81% [1] Revenue Breakdown - Net product revenues totaled $812.98 million, exceeding the average estimate of $755.45 million by analysts, marking a 15.8% increase year-over-year [4] - Royalty and other revenues reached $12.43 million, surpassing the estimated $10.77 million, representing a 25.5% increase compared to the previous year [4] - Specific product revenue highlights include: - NAGLAZYME: $129 million, slightly below the estimate of $120.87 million, a decrease of 2.3% year-over-year [4] - VIMIZIM: $215 million, exceeding the estimate of $186.74 million, a 20.8% increase [4] - PALYNZIQ: $106 million, above the estimate of $100.63 million, a 20.1% increase [4] - VOXZOGO: $221 million, slightly above the estimate of $219.28 million, a 20.2% increase [4] - ROCTAVIAN: $9 million, below the estimate of $12.52 million, a 21.6% increase [4] - KUVAN: $27 million, exceeding the estimate of $21.49 million, a decrease of 5.6% [4] - ALDURAZYME: $56 million, surpassing the estimate of $43.18 million, a significant increase of 45.1% [4] - BRINEURA: $49 million, above the estimate of $45.89 million, an 8.2% increase [4] Stock Performance - BioMarin's shares have returned +1.1% over the past month, outperforming the Zacks S&P 500 composite's +0.6% change [3] - The stock currently holds a Zacks Rank 3 (Hold), suggesting it may perform in line with the broader market in the near term [3]

Financial Performance - Total Revenues for Q2 2025 reached $825 million, a 16% year-over-year increase[17, 22] - VOXZOGO revenue in Q2 2025 was $221 million, representing a 20% year-over-year growth[17, 22] - Enzyme Therapies revenue in Q2 2025 was $555 million, a 15% year-over-year increase[17, 22] - Non-GAAP Operating Margin for Q2 2025 was 39.9%, an increase of 8.7 percentage points year-over-year[25] - Non-GAAP Diluted EPS for Q2 2025 was $1.44, a 50% increase year-over-year[17, 30] - Operating Cash Flow for Q2 2025 was $185 million, a 55% increase year-over-year[30] - The company increased FY'25 Total Revenues guidance to $3,125 million to $3,200 million[24, 34] - The company increased FY'25 Non-GAAP Diluted EPS guidance to $4.40 to $4.55[33, 34] Product and Pipeline Updates - VOXZOGO's contribution to full-year 2025 Total Revenues is expected to be in the range of $900 million to $935 million[24, 34] - BMN 333 Phase 1 data showed >3x AUC PK free CNP levels vs other long-acting CNP[17, 45] - Submissions for PALYNZIQ age eligibility expansion for adolescents (12-17 y/o) in the U S and Europe are planned for 2H 2025[3, 48, 51, 56]