2/23/2026

speaker
Kate
Conference Operator

Thank 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 Biomarine 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 time, simply press star followed by the number one on your telephone keypad. If you would like to withdraw your question, press star one again. Thank you. I would now like to turn the call over to Tracy McCarty, Head of Investor Relations. Please go ahead.

speaker
Tracy McCarty
Head of Investor Relations

Thank you, Operator, and starting on slide two. To remind you, this non-confidential presentation contains forward-looking statements about the business prospects of Biomarin Pharmaceutical Inc., including expectations regarding Biomarin's financial performance, commercial products, and potential future products in different areas of therapeutic research and development. Results may differ materially depending on the progress of Biomarin's product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market, and developments by competitors, and those factors detailed in Biomarin's filings with the Securities and Exchange Commission, such as 10Q, 10K, and 8K reports. In addition, we will use non-GAAP financial measures as defined in Regulation G during the call today. These non-GAAP measures should not be considered in isolation from, as substitutes for, or superior to financial measures prepared in accordance with U.S. GAAP, and you can find the related reconciliations to U.S. GAAP in the earnings release and earnings presentation, both of which are now available in the investor relations section of our website. Please note that our commentary on today's call will focus on non-GAAP financial measures unless otherwise indicated. Moving to slide three and introducing Biomarin's management team joining today's call, Alexander Hardy, Chief Executive Officer, Brian Mueller, Chief Financial Officer, Kristen Hubbard, Chief Commercial Officer, and Greg Freiberg, Chief R&D Officer. I will now turn the call over to Biomarin's CEO, Alexander Hardy.

speaker
Alexander Hardy
Chief Executive Officer

Thank you, Tracy. And moving now to slide five. Thank you all for joining us today to discuss Biomarin's fourth quarter and full year 2025 results, as well as our outlook for 2026, which will be an exciting year. We are particularly proud to have accomplished our strategic goals for 2025 while achieving outstanding growth. In 2025, total revenues grew by 13% to a record $3.22 billion for the year, an operational excellence led to strong profitability and increasing cash flow. This result was fueled by a 9% increase in enzyme therapies revenue and a remarkable 26% rise in Voxogo revenues. Importantly, enzyme therapies revenue has grown at a 9% CAGR over the last five years, demonstrating the durability and reach of this $2 billion plus franchise across our 80 country footprint. With VoxOgo now in its fifth year on the global market, we're very pleased with the consistent, strong growth already achieved and have deep conviction in our ability to continue expanding should another product be approved. Biomarin's 2025 performance and 2026 outlook highlight both our deep global capabilities and the vital importance of our targeted therapies to the patients we serve. Building on this strong foundation, in 2026, we will expand our therapeutic and commercial reach with the addition of assets from two significant acquisitions announced last year. The first, Inozyme, strengthened our enzyme therapies portfolio with the addition of BMN401 for ENPP1 deficiency. This is a condition for which there is no approved targeted therapy, so we're really pleased about the possibility of launching what could be our sixth first in disease enzyme therapy should pivotal data be supportive. We look forward to sharing that update in the coming months and to filing submissions soon thereafter. Our second acquisition, Amicus, potentially adds both Gallifold for the treatment of Fabry disease and Pombility and Alfolda for Pompe disease to our commercial portfolio and is expected to close next quarter. This transaction presents a particularly compelling opportunity to further build on Amicus's success by leveraging our scale and global capabilities to serve even greater numbers of patients. In addition to the expansion of Biomarin's portfolio from acquisitions, we're particularly excited to build upon Voxogo's leadership in achondroplasia with a potential addition for treatment of hypochondroplasia. Because CNP is the master regulator of bone growth and supported by the one-year results from the investigator-sponsored study, we are excited to see and share the upcoming pivotal results with Voxergo for the treatment of hypochondriplasia in the coming months. We look forward to the possibility of adding hypochondriplasia to our global skeletal conditions treatment offerings by early next year. On top of Voxergo indication expansion, based on the very encouraging PK data recently shared, we're preparing to begin enrollment in our Phase 2-3 study for BMN 333. We believe this next generation long-acting CNP therapy has the potential to set a new standard of care and to demonstrate superiority compared to any candidates under development for achondroplasia. Those are just a few of the anticipated pipeline highlights expected this year. so you can understand our enthusiasm for what's ahead in the coming months. Turning to innovation. During the period where we are de-levering the financing associated with the announced acquisition of Amicus, we will remain actively engaged in business development activities targeting pipeline assets. This is an important component of a larger pipeline expansion plan, a plan that both accelerates our financial performance and further diversifies our portfolio to drive durable, long-term growth. In closing, we are pleased that the transformational work implemented over the last 24 months has already delivered significant results and positions us for even more revenue growth, profitability, and pipeline expansion. Prior to any contributions from the amicus assets, this year we anticipate Biomarin's enzyme therapies will deliver high single-digit growth, and Voxogo will continue along its trajectory towards blockbuster status. Following the completion of the transaction, we anticipate that integrating Gallifold and Pombility and Upfolder will enable both medicines to reach more people around the world living with Fabre and Pompei, significantly enhancing our 2026 outlook and enabling accelerated revenue growth through the 2030s. With that, I will turn the call over to Brian to provide additional financial updates. Brian.

Disclaimer

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Investor presentation