5/4/2026

speaker
Operator
Conference Operator

Good afternoon and welcome everyone to the Biomarin Pharmaceutical First Quarter 2026 conference call. Today's conference is being recorded. 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 the star key, followed by the number one on your telephone keypad. If you would like to withdraw your question, press star one again. At this time, I would like to turn the conference over to Tracy McCarty, Head of Investor Relations. Please go ahead.

speaker
Tracy McCarty
Head of Investor Relations

Thank you, operator. 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 US 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. Beginning on slide three and introducing Biomarin's management team joining today's call are 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 President and CEO, Alexander Hardy.

speaker
Alexander Hardy
President and Chief Executive Officer

Thank you, Tracy, and thank you all for joining us today. I am so pleased that we completed the amicus acquisition last week, starting a new and exciting chapter for Biomarin with the addition of two innovative therapies, Galifold for Fabry disease and Pombility and Opfolder for Pompe disease. The acquisition accelerates our anticipated year-over-year 2026 revenue growth to 20% at the midpoint of today's updated guidance. This strengthening trajectory is just the beginning of Biomarin's enhanced longer-term financial outlook supported by our larger, more diversified commercial portfolio. Since announcing our plans to acquire Amicus late last year, we have been preparing for rapid integration beginning on day one with the goal of increasing the peak potential of these newly added products. Following the recent close of the transaction, we initiated our targeted integration plan focused on leveraging Biomarin's operating scale and capabilities to drive diagnosis and treatment rates for patients with Fabry disease and late onset Pompe disease. Next quarter, we plan to share this roadmap as well as more detail on Biomarin's growth acceleration with the addition of Gallifold and Pombility and Opfolder to our commercial portfolio, and DMX-200 for FSGS in phase three to our late stage pipeline. Turning briefly to first quarter results and outlook for the remainder of this year, and starting with enzyme therapies, I am pleased with the strong interest we are seeing from the PKU community following the recent Palenzec adolescent label expansion in the United States. which Kristen will discuss in more detail. We expect enzyme therapies will deliver robust growth in 2026, further supported by the addition of Galifold and Pombility and Opfolder to the portfolio. Moving to our skeletal conditions business unit, we saw strong patient demand for Boxergo, with new patient starts increasing across all regions in the first quarter. In the US, the majority of new patient starts were from the underage two cohort. These results reflect our focused investments in increasing adoption of Oxogo, particularly among younger patients. Building on our leadership in achondroplasia, we are pleased to have submitted the SNDA full approval of Oxogo. We expect to hear the timing of our review in the coming months. I want to congratulate our regulatory team for the outstanding work that went into this comprehensive submission package. We also look forward to pivotal results for VOXOGO in hypochondriplasia and BMN401 for ENPP1 deficiency, those later in Q2. In summary, we expect 2026 to be a momentous year for Biomarin. Our immediate focus remains on the seamless and rapid integration of amicus pursuing regulatory next steps following the two upcoming pivotal data readouts. The addition of Gallifold and Pompility and Opfolder to Biomarin's portfolio of innovative medicines provides an opportunity to reach more patients around the world, creating significant value for all of our stakeholders in the near and longer term. As we enter this next chapter, I would like to express my appreciation to the employees of both Biomarin and Amicus, whose dedication forms the foundation of our shared mission to serve patients. Thank you for your attention, and I will now turn the call over to Brian to provide additional financial updates. Brian. Thank you, Alexander.

Disclaimer

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Investor presentation