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 Viewpoint - BioMarin Pharmaceutical Inc. is considered a cheap healthcare stock with potential for investment heading into 2026, despite recent downgrades in price targets by analysts [1][3]. Group 1: Analyst Ratings and Price Targets - Leerink reduced the price target on BioMarin to $60 from $82 and downgraded it to Market Perform from Outperform on December 3 [1]. - Bernstein also lowered the price target to $88 from $95, maintaining a Market Perform rating, noting that the company's quarterly performance met revenue expectations but missed EPS targets [3]. - The majority of analysts assign a 'Buy' or equivalent rating to BioMarin, with target prices ranging from $55 to $122, and a median price target of $91 suggesting an upside potential of about 70% [4]. Group 2: Company Actions and Market Position - New leadership's actions, including workforce cuts and discontinuation of the Roctavian program, are seen as strengthening BioMarin's long-term position [2]. - Rising competitive pressure in the achondroplasia market and a significant gap in the company's pipeline may necessitate mergers and acquisitions, creating uncertainty about the effectiveness of current measures to boost stock performance [2]. Group 3: Company Overview - BioMarin Pharmaceutical Inc. is a California-based biotechnology company focused on developing and marketing therapies for serious rare diseases, with core offerings including VIMIZIM, VOXZOGO, NAGLAZYME, and PALYNZIQ [4].

Group 1 - BioMarin Pharmaceutical Inc. reported fiscal Q3 2025 total revenues of $776 million, reflecting a 4% growth year-over-year, driven by strong performance in VOXZOGO and PALYNZIQ due to new patient initiations [1] - Year-to-date total revenues increased by 11% year-over-year, with PALYNZIQ and VOXZOGO showing over 20% revenue growth [2] - J.P. Morgan analyst Jessica Fye reiterated a Buy rating on BioMarin, indicating that the company's underlying business is undervalued [2] Group 2 - The company has a strong growth potential and a solid market position, supported by considerable capital for business development and a robust product pipeline [3] - BioMarin develops therapies for serious and life-threatening medical conditions and rare diseases, with key products in its pipeline including Valoctocogene roxaparvovec, Vosoritide, and BMN 307 [3]

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 Inc reported weaker-than-expected earnings for Q3, with earnings of $0.12 per share, missing the consensus estimate of $0.37 per share, and quarterly sales of $776.133 million, below the expected $780.629 million [1] Financial Performance - The company lowered its FY2025 adjusted EPS guidance from $4.40-$4.55 to $3.50-$3.60, while raising its sales guidance from $3.125 billion-$3.200 billion to $3.150 billion-$3.200 billion [2] - The CEO highlighted over 20% revenue growth from PALYNZIQ and VOXZOGO as key contributors to the business units [2] Stock Performance - Following the earnings announcement, BioMarin shares gained 4.3% to $54.95 [3] Analyst Ratings and Price Targets - Morgan Stanley maintained an Overweight rating but lowered the price target from $104 to $98 [5] - HC Wainwright & Co. maintained a Neutral rating and cut the price target from $60 to $55 [5] - Barclays also maintained an Overweight rating while lowering the price target from $86 to $80 [5]

Core Insights - BioMarin Pharmaceutical Inc reported weaker-than-expected earnings for Q3, with earnings of 12 cents per share, missing the analyst consensus estimate of 37 cents per share [1] - Quarterly sales were reported at $776.133 million, also missing the analyst consensus estimate of $780.629 million [1] Financial Guidance - The company lowered its FY2025 adjusted EPS guidance from $4.40-$4.55 to $3.50-$3.60, while raising its sales guidance from $3.125 billion-$3.200 billion to $3.150 billion-$3.200 billion [2] - The CEO highlighted over 20% revenue growth from PALYNZIQ and VOXZOGO as key contributors to the business units [2] Stock Performance - Following the earnings announcement, BioMarin Pharmaceutical shares gained 4.3%, reaching $54.95 [3] - Analysts adjusted their price targets for the stock after the earnings report [3] Analyst Ratings - Morgan Stanley maintained an Overweight rating but lowered the price target from $104 to $98 [5] - HC Wainwright & Co. maintained a Neutral rating and cut the price target from $60 to $55 [5] - Barclays also maintained an Overweight rating, lowering the price target from $86 to $80 [5]

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 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]