BioMarin Pharmaceutical Conference Call Summary Company Overview - **Company**: BioMarin Pharmaceutical (NasdaqGS: BMRN) - **Date**: March 09, 2026 - **Key Speakers**: Brian Mueller (CFO), Cristin Hubbard (CCO) Key Points Industry and Company Performance - BioMarin reported a **13% revenue growth** in 2025, with the Skeletal Conditions unit (including VOXZOGO) growing by **26%** and Enzyme Therapies by **9%** [4][5] - The company anticipates **high single-digit growth** for both Skeletal Conditions and Enzyme Therapies in 2026 [4] Strategic Growth Pillars - BioMarin's corporate strategy is built on three pillars: **growth, innovation, and value commitment** [4] - The company is preparing for the **Amicus acquisition** in Q2 2026, which is expected to enhance its portfolio with two high-growth products in rare genetic conditions [5] Product Pipeline and Innovations - Recent approval of **PALYNZIQ** for adolescents and upcoming **Phase 3 data readouts** for VOXZOGO in hypochondroplasia and BMN 401 for ENPP1 deficiency [5][6] - Ongoing development of **BMN 351** for Duchenne muscular dystrophy and **BMN 333**, a long-acting CNP for achondroplasia [6] Market Dynamics and Patient Retention - BioMarin is focused on patient retention for VOXZOGO, emphasizing that **efficacy and safety** are key factors influencing caregivers' decisions to switch therapies [10][11] - Approximately **two-thirds** of KOLs and caregivers believe that switching therapies is unlikely if patients are doing well on current treatments [11] Achondroplasia Market Insights - The company is working to build a broader health narrative around achondroplasia beyond just growth, focusing on overall health benefits [12][14] - BioMarin has a long history with VOXZOGO, having started its development program over **15 years ago**, and is now filing for full approval based on long-term data [20] Hypochondroplasia Opportunity - The Phase 3 trial for hypochondroplasia is expected to yield data in the **first half of 2026**, with strong enrollment indicating market excitement [25][26] - The company is focusing on **disease awareness** and improving the diagnosis journey for hypochondroplasia patients [28][30] Amicus Acquisition and Market Expansion - The acquisition of Amicus is expected to enhance BioMarin's market presence, with **Galafold** currently available in **40 countries** and BioMarin operating in **80** [38] - The integration of Amicus products is anticipated to unlock additional value through deeper market penetration and improved diagnosis efforts [39][43] Financial Projections - The Amicus acquisition may be slightly dilutive in **2026** but is expected to be accretive in the first 12 months and substantially accretive thereafter [45] - BioMarin expects **VOXZOGO revenues** in the second half of 2026 to exceed the first half, primarily due to international order timing [64][66] Manufacturing and Operational Strategy - BioMarin is recognized for its high-quality manufacturing capabilities and plans to leverage these skills for Amicus products, particularly POMP [51][53] Clinical Data and Future Expectations - Upcoming data cuts for **BMN 351** and pivotal Phase 3 data for **ENPP1 deficiency** are anticipated, with a focus on patient diagnosis and treatment initiation [54][57] Conclusion BioMarin Pharmaceutical is positioned for continued growth through strategic acquisitions, a robust product pipeline, and a commitment to enhancing patient care in rare genetic conditions. The company is focused on leveraging its manufacturing expertise and expanding its market presence while maintaining a strong emphasis on patient retention and treatment efficacy.

Fourth Quarter and Full Year 2025 Earnings Q4'25 and FY'25 Earnings Agenda: Alexander Hardy Chief Executive Officer Brian Mueller Chief Financial Officer Cristin Hubbard Chief Commercial Officer February 23, 2026 Forward-Looking Statements This presentation and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: future financial performance, including the expect ...

Core Viewpoint - BioMarin Pharmaceutical (BMRN) is expected to report a significant decline in quarterly earnings per share, while revenues are projected to increase year-over-year, indicating mixed performance expectations from analysts. Earnings Estimates - Analysts predict quarterly earnings of $0.25 per share, reflecting a decline of 72.8% compared to the same period last year [1] - There has been a downward revision of 2.3% in the consensus EPS estimate over the last 30 days [2] Revenue Forecasts - Total revenues are forecasted to be $829.66 million, representing a year-over-year increase of 11% [1] - Analysts expect 'Revenues- Net product revenues' to be $813.04 million, indicating a change of +10.5% year-over-year [4] - 'Revenues- Royalty and other revenues' are estimated at $13.32 million, reflecting a year-over-year change of +14.1% [5] Product-Specific Revenue Estimates - 'Revenues- Net Product Revenues- NAGLAZYME' is projected to reach $123.94 million, indicating a change of +12.7% from the prior-year quarter [5] - 'Revenues- Net Product Revenues- VIMIZIM' is estimated at $201.47 million, reflecting a change of +5.5% from the prior-year quarter [6] - 'Revenues- Net Product Revenues- VOXZOGO' is expected to be $262.31 million, indicating a year-over-year change of +26.1% [7] - 'Revenues- Net Product Revenues- KUVAN' is projected to reach $20.82 million, reflecting a decline of -25.7% from the year-ago quarter [7] - 'Revenues- Net Product Revenues- ALDURAZYME' is estimated at $44.95 million, indicating a change of +15.3% from the prior-year quarter [7] - 'Revenues- Net Product Revenues- BRINEURA' is forecasted to be $47.51 million, reflecting a year-over-year change of -1% [8] - 'Revenues- Net Product Revenues- ROCTAVIAN' is expected to reach $7.58 million, indicating a change of -31.1% from the prior-year quarter [8] Market Performance - Shares of BioMarin have shown returns of +8.9% over the past month, outperforming the Zacks S&P 500 composite's -0.8% change [8] - BioMarin holds a Zacks Rank 3 (Hold), suggesting it is expected to mirror overall market performance in the near future [8]

Core Insights - BioMarin Pharmaceutical Inc. is identified as a stock with significant upside potential, with price targets raised by analysts indicating a potential increase of up to 74.4% from current levels [1] - The company is focusing on maintaining double-digit revenue growth into the 2030s, supported by its 2025 revenue guidance aligning with market expectations [1] Group 1: Market Position and Growth Strategy - BioMarin is strengthening its leadership in achondroplasia through its product Voxzogo, which benefits from a broad label and strong patient adherence [2] - The company is pursuing label expansions in hypochondroplasia and other skeletal disorders, alongside regulatory efforts to protect its market position [2] Group 2: Pipeline Developments - Recent pipeline advancements include BMN-351, which shows promise in Duchenne muscular dystrophy, and BMN-333, which is progressing to Phase 2/3 development [3] - Analysts from Wedbush and Evercore ISI have reaffirmed their Buy ratings, with price targets set at $94 and $110 respectively [3] Group 3: Company Overview - BioMarin operates as a biotechnology company focused on therapies for life-threatening rare diseases, with products including VIMIZIM, VOXZOGO, NAGLAZYME, and PALYNZIQ [4]

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]