BioMarin Pharmaceutical (NasdaqGS:BMRN) Q4 2025 Earnings call February 23, 2026 04:30 PM ET Company ParticipantsAlexander Hardy - CEOBrian Mueller - CFOCristin Hubbard - Chief Commercial OfficerGena Wang - Managing Director of Biotech Equity ResearchGreg Friberg - Chief Research and Development OfficerJason Gerberry - Managing DirectorMohit Bansal - Managing Director and Co-Head of Therapeutics ResearchPaul Matteis - Managing DirectorTraci McCarty - Head of Investor RelationsConference Call ParticipantsChri ...

BioMarin Pharmaceutical (NasdaqGS:BMRN) Q4 2025 Earnings call February 23, 2026 04:30 PM ET Speaker10Thank you for standing by. My name is Kate, and I will be your conference operator today. At this time, I would like to welcome everyone to the BioMarin Pharmaceutical fourth quarter and full year 2025 conference call. All lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question and answer session. If you would like to ask a question during this ti ...

Core Insights - BioMarin Pharmaceutical is targeting a preliminary revenue of $3.2 billion for 2025, with Voxzogo expected to contribute $920 million, reflecting a strong performance and a projected 27% year-over-year growth in Q4 [3][6] - The company is focusing on expanding Voxzogo's market presence and enhancing its enzyme therapies portfolio, alongside the strategic acquisition of Amicus, valued at $4.8 billion, which is expected to diversify and accelerate revenue growth [7][8] Revenue and Growth Projections - BioMarin anticipates Voxzogo's growth in achondroplasia will be supported by new country launches and deeper market penetration, particularly emphasizing early treatment decisions for infants [2][6] - The acquisition of Amicus is expected to be accretive within 12 months post-close and substantially accretive starting in 2027, with confidence in outperforming previous growth expectations for enzyme therapies [8][7] Strategic Focus and Pipeline Development - The company's strategy is built on three pillars: innovation, growth, and a value commitment, prioritizing pipeline programs with transformative potential, including BMN 333 and BMN 351 [5][6] - BioMarin plans to accelerate revenue through geographic expansion of Voxzogo and potential label expansions for other therapies, including Palynziq [4][6] Clinical and Regulatory Catalysts - BioMarin is preparing to file for full approval of Voxzogo with the FDA, leveraging extensive safety data and additional efficacy endpoints [11] - Upcoming regulatory actions include an FDA PDUFA date for Palynziq's adolescent label expansion and significant data readouts for BMN 351 and BMN 333 [12][10][13] Competitive Dynamics and Intellectual Property Strategy - The company is committed to defending its intellectual property for Voxzogo, including seeking orphan drug exclusivity and engaging in ongoing litigation to protect its market position [15][16] - BioMarin views BMN 333 as a potential long-term game changer for achondroplasia, based on its promising exposure profile [16]

BioMarin Pharmaceutical FY Conference Summary Company Overview - **Company**: BioMarin Pharmaceutical (NasdaqGS: BMRN) - **Event**: FY Conference held on January 12, 2026 - **CEO**: Alexander Hardy Key Industry Insights - BioMarin is a leader in genetically defined conditions with a strong track record of innovation, having six first-in-disease treatments and many best-in-disease treatments [6][7] - The company operates in 80 countries and has significant R&D capabilities and in-house manufacturing [6][7] Core Financial Highlights - Preliminary revenue for 2025 is projected at **$3.2 billion**, with a **15% CAGR** over the last two years [9][21] - Voxzogo, a treatment for achondroplasia, is expected to generate **$920 million** in revenue for 2025, reflecting a **27% year-over-year growth** [19][21] Strategic Pillars 1. **Innovation**: Focus on transformative potential in the pipeline, with key assets like 333 and 351 showing promise [8][12] 2. **Growth**: Significant growth rates driven by enzyme therapies and the expansion of Voxzogo into new indications and countries [9][10] 3. **Value Commitment**: Cost transformation efforts have identified **$500 million** in cost reductions, enhancing profitability and cash flow [10] Amicus Acquisition - BioMarin announced the acquisition of Amicus for an equity value of **$4.8 billion**, which is expected to enhance revenue growth and diversify the revenue base [14][15] - Key products from Amicus include: - **Galafold** for Fabry disease, currently available in **40 countries**, with plans to expand to BioMarin's **80-country footprint** [16][17] - **Pombiliti** for Pompe disease, currently reimbursed in **15 countries**, with potential for significant growth [18] Pipeline and R&D Updates - Upcoming catalysts include: - Filing for full approval of Voxzogo in achondroplasia [12][34] - Two phase 3 data readouts and label expansions expected within the year [12][24] - BMN 351 for Duchenne muscular dystrophy showing promising early results [24][25] - BMN 333 for achondroplasia demonstrating over **13-fold** increase in free CNP exposure [28] Market Position and Competition - BioMarin is focused on defending its market position for Voxzogo against potential competitors by leveraging its established safety profile and efficacy data [49][50] - The company has petitioned for orphan drug exclusivity to delay the approval of competing products for **three more years** [49] Future Outlook - BioMarin aims for sustained double-digit CAGR growth into the 2030s, supported by the integration of Amicus products and ongoing pipeline developments [22][37] - The company is optimistic about its ability to reach more patients and enhance treatment options through its expanded portfolio [15][51] Additional Notes - The adherence rate for Voxzogo is approximately **90%**, indicating strong patient retention [45] - The company emphasizes the importance of early diagnosis and treatment in achondroplasia, which is expected to drive future growth [39][40]

Group 1 - BioMarin Pharmaceutical Inc. is recognized as one of the best biotech stocks to buy according to Wall Street analysts, with a focus on developing therapies for severe, life-threatening rare genetic diseases [1][4] - The company is currently implementing a $500 million cost transformation program, which is about two-thirds complete, aiming for a 40% non-GAAP operating margin in the next year, an increase from 19% in 2023 [1] - BioMarin is concentrating on genetically defined conditions with few competitors, highlighting the global rollout of Voxogo in 55 countries and the growth of Palynziq at over 20% annually [2] Group 2 - Analysts at Leerink Partners downgraded BioMarin's stock to Market Perform from Outperform and reduced the price target to $60 from $82, while acknowledging the company's restructuring efforts [3] - The restructuring includes labor force reduction, pipeline reorganization, and the discontinuation of the Roctavian program, alongside the acquisition of Inozyme, which is expected to drive future growth [3] - The company is focused on innovation and speeding up the development of its pipeline to create value for shareholders, despite facing regulatory and competitive pressures [2][3]

Summary of BioMarin Pharmaceutical Conference Call Company Overview - **Company**: BioMarin Pharmaceutical (NasdaqGS: BMRN) - **Event**: 2025 Conference on November 18, 2025 Key Points Company Strategy and Financials - BioMarin's CEO highlighted a two-year milestone, reflecting on significant changes and a focus on execution [2] - The company has reorganized its strategy around three pillars: growth from the existing portfolio, innovation through its pipeline, and value commitment to enhance profitability and cash flow [2] - A cost transformation initiative has identified $500 million in potential savings, which has been directed towards improving cash flow and strengthening the balance sheet [3][4] Pipeline Developments - Upcoming pipeline readouts include: - **BMN 351** for Duchenne muscular dystrophy, with top-line results expected by the end of 2025 [3] - **Voxogo** for hypochondroplasia, with Phase 3 data readout anticipated early next year [3] - **Palynziq** for adolescents, with an FDA action date set for February 28, 2026, and current growth exceeding 20% [4] Business Development (BD) Environment - The company has a robust BD function aimed at supplementing internal innovation, focusing on genetically defined conditions with relatively low competitive pressure [6][9] - BioMarin is strategically positioned to leverage its geographic footprint, with 68% of patients for achondroplasia located outside the U.S. and Europe [10] Market Dynamics and Competition - The company believes that Voxogo will continue to grow, with significant opportunities in both achondroplasia and hypochondroplasia markets [19] - Concerns about competition are acknowledged, but the company maintains confidence in Voxogo's market position and patient retention due to established safety and efficacy [26][28] Regulatory and Approval Considerations - Discussions around the potential for full approval of Voxogo are ongoing, with emphasis on presenting comprehensive data to regulators [34] - The company is also engaged in litigation regarding intellectual property, which may impact market competition [38] Hypochondroplasia Insights - The addressable market for hypochondroplasia is estimated at 14,000 patients, with a focus on creating awareness and understanding of the disease [41] - The study for hypochondroplasia is powered to look for an average growth height velocity of 1.5 cm per year, with expectations based on previous data showing higher growth rates [44] Conclusion - BioMarin is positioned for growth through strategic investments, a strong pipeline, and a focus on genetically defined conditions, while navigating competitive and regulatory landscapes [2][4][19][34]

Core Viewpoint - BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is recognized as a promising growth stock with a maintained Buy rating and a price target of $104 by analysts [1][2]. Group 1: Market Position and Growth Potential - The company has a strong market position and significant growth potential, particularly through the international expansion of its product Voxzogo, which has demonstrated revenue growth [2]. - Management is expected to continue expanding Voxzogo into additional countries, which is identified as a key driver for the company's growth [2]. Group 2: Pipeline and Future Catalysts - BioMarin has a promising pipeline, with BMN 351 and BMN 333 highlighted as potential catalysts for future growth [3]. - The company's ability to maintain its leadership in skeletal dysplasias, despite challenges, further strengthens its market position [3]. Group 3: Company Overview - BioMarin Pharmaceutical Inc. is an international biopharmaceutical company focused on developing and commercializing targeted therapies for rare genetic conditions [4].

Core Insights - BioMarin Pharmaceutical (BMRN) reported Q2 2025 adjusted EPS of $1.44, exceeding the Zacks Consensus Estimate of $1.03, with a 50% year-over-year increase driven by higher product sales and lower operating expenses [1][9] - Total revenues reached $825.4 million, reflecting a 16% year-over-year increase, surpassing the Zacks Consensus Estimate of $766.2 million [1][9] Revenue Breakdown - Product revenues totaled $813 million, a 16% year-over-year increase, primarily due to higher sales from Voxzogo, Palynziq, Vimizim, and Aldurazyme, partially offset by lower Kuvan sales [2] - Voxzogo generated $221 million in sales, up 20% year over year, exceeding the Zacks Consensus Estimate of $219 million [3] - Enzyme Therapies sales rose 15% year over year to $555 million, driven by increased patient demand and large government orders [4] - Palynziq injection sales increased 20% year over year to $106 million, surpassing both the Zacks Consensus Estimate and internal model estimates [5] - Vimizim sales rose 21% year over year to $215 million, beating both the Zacks Consensus Estimate and internal model estimates [6] - Aldurazyme sales totaled $56 million, up 44% year over year, attributed to favorable order fulfillment timing [6] Financial Guidance - BioMarin revised its 2025 revenue forecast to $3.13-$3.20 billion, reflecting an 11% year-over-year increase at the midpoint [11] - Adjusted EPS guidance was raised to $4.40-$4.55, indicating a 27% growth over the previous year at the midpoint [14] - The company expects Voxzogo sales to be between $900-$935 million, with higher revenues anticipated in the second half of the year [12] Pipeline Developments - The acquisition of Inozyme added BMN 401, an investigational enzyme replacement therapy for rare disorders, with interim results expected in early 2026 [18][19] - BioMarin is advancing its CANOPY clinical program for Voxzogo, targeting additional indications with data expected in 2026 [22] - BMN 333, a long-acting formulation of CNP, is set to enter a phase II/III study in 2026, aiming for a potential launch in 2030 [23] - The company plans to file for expanded use of Palynziq in adolescents based on positive late-stage study results [21]

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]