Key Takeaways BioMarin reported Q4 EPS of 46 cents, beating estimates while revenues rose 17% Y/Y to $875M.Voxzogo sales jumped 31% to $273M in Q4, while BioMarin's enzyme therapies sales rose 13%.BioMarin will withdraw Roctavian after failing to find a buyer, recording a $119.2M charge in Q4.BioMarin Pharmaceutical (BMRN) reported fourth-quarter 2025 adjusted earnings per share of 46 cents, beating the Zacks Consensus Estimate of 25 cents. However, earnings declined 50% year over year, largely due to a $11 ...

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 Viewpoint - BioMarin Pharmaceutical is expected to report its Q4 and full-year 2025 results soon, with potential strong performance from its rare-disease drug Voxzogo, but there are also concerns regarding competition and revenue guidance [1][2][3][10]. Financial Performance - BioMarin's CFO indicated that Voxzogo revenue is projected to reach its highest level in Q4, contributing to an optimistic outlook for the quarter [2]. - The company raised the lower end of its 2025 revenue guidance to $3.15 billion, reflecting double-digit year-over-year growth expectations [3]. Upcoming Catalysts - The FDA has set a PDUFA date of February 28, 2026, for Palynziq's approval for adolescents with phenylketonuria, with potential EU approval also expected in the first half of the year [4]. - BioMarin plans to announce results from two Phase 3 clinical studies in the first half of 2026 and intends to file for full FDA approval of Voxzogo for achondroplasia [5]. Market Data - BioMarin's current market capitalization is $11 billion, with a gross margin of 80.64% [7]. - The stock is currently trading at $56.51, with a day's range of $55.30 to $56.72 [6][7]. Investment Considerations - There are reasons to be cautious, including reliance on large contracted ex-U.S. orders for Voxzogo, which if not fulfilled, could lead to missing Q4 estimates [8]. - Long-term uncertainty exists regarding Voxzogo's market position due to potential competition, which could impact future revenue projections [9]. - The acquisition of Amicus Therapeutics is seen as a potential growth booster, but waiting to invest may be prudent given current uncertainties [10].

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]

Core Insights - BioMarin Pharmaceutical (BMRN) has entered a definitive agreement to acquire Amicus Therapeutics (FOLD) for $14.50 per share, totaling $4.8 billion, with the deal expected to close in Q2 2026 [1][6] - The acquisition will enhance BioMarin's portfolio with Amicus' marketed therapies, Galafold and Pombiliti-Opfolda, which generated $449 million in sales in the first nine months of 2025, reflecting a 19% year-over-year growth [2][6] - BioMarin will also gain exclusive U.S. rights to the late-stage drug DMX-200, aimed at treating focal segmental glomerulosclerosis, a rare kidney disease [4][6] Acquisition Details - The acquisition is financed through BioMarin's existing cash and $3.7 billion in non-convertible debt [1] - The deal is expected to be accretive to BioMarin's bottom line within the first 12 months post-closure [8] - BioMarin's strategic fit includes expanding its enzyme therapies portfolio, which already consists of five first-in-disease therapies [7] Market Reaction - Following the acquisition announcement, shares of Amicus Therapeutics rose by 30%, while BioMarin's shares increased by 18% [5] - Year-to-date performance shows BioMarin's shares have decreased by 7%, whereas Amicus shares have increased by nearly 51% [5] Strategic Context - This acquisition aligns with BioMarin's strategy of pursuing partnerships and acquisitions, with an estimated $4-$5 billion available for future growth initiatives [9] - This marks BioMarin's second targeted acquisition in 2023, following the acquisition of Inozyme Pharma for $270 million [10]

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 Insights - BioMarin Pharmaceutical (BMRN) reported Q3 2025 adjusted EPS of 12 cents, surpassing the Zacks Consensus Estimate of a loss of 15 cents, but reflecting an 87% year-over-year decline due to IPR&D charges of $221 million related to the acquisition of Inozyme Pharma [1][7][14] - Total revenues reached $776 million, a 4% increase year-over-year, but fell short of the Zacks Consensus Estimate of $784 million [1][14] Revenue Breakdown - Product revenues amounted to nearly $761 million, up 4% year-over-year, driven by Voxzogo and Palynziq, although partially offset by lower sales of Aldurazyme and Kuvan [4][10] - Voxzogo sales were $218 million, a 15% increase year-over-year, primarily due to new patient initiations outside the U.S., despite some unfavorable order timing [5][8] - Palynziq injection sales totaled $109 million, up 20% year-over-year, exceeding both the Zacks Consensus Estimate and internal model estimates [10] - Enzyme Therapies sales rose 1% year-over-year to $516 million, with Vimizim sales increasing 3% to $183 million, although it missed estimates [9][10] Financial Guidance and Outlook - BioMarin revised its full-year revenue guidance to $3.15-$3.20 billion, reflecting an increase of $25 million from previous estimates, indicating over 11% growth year-over-year at the midpoint [14][15] - Adjusted EPS guidance was lowered to $3.50-$3.60, down from $4.40-$4.55, implying a 1% growth over last year's level at the midpoint [16] - The company plans to divest Roctavian to focus on strategic growth and partnerships, while maintaining marketing efforts until a final decision is made [17][18] Pipeline Developments - BioMarin is advancing its CANOPY clinical program for Voxzogo, targeting additional indications, with data expected in the first half of 2026 [20] - The company is also pursuing multiple partnership and acquisition opportunities, with an estimated $4-$5 billion available for future growth initiatives [17] - Initial proof-of-concept data for BMN 351, an oral therapeutic for liver disease, is expected by the end of this year [23]