8/4/2025

speaker
Operator

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'd like to turn the conference over to Tracy McCarty, Investor Relations at BioMarin. Please go ahead.

speaker
Tracy McCarty
Investor Relations, BioMarin

Tracy McCarty 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 U.S. GAAP in earnings release and earnings presentation, both of which are 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. Introducing the Biomarin management team today. On the call, we have Alexander Hardy, President and Chief Executive Officer, Brian Mueller, Executive Vice President, Chief Financial Officer. Kristen Hubbard, Executive Vice President, Chief Commercial Officer. And Greg Freiberg, Executive Vice President, Chief R&D Officer. I will now turn the call over to Biomarin's President and CEO, Alexander Hardy.

speaker
Alexander Hardy
President & Chief Executive Officer

Thank you, Tracy. And thank you all for joining us today for Biomarin's second quarter update. Now moving to slide six. we were very pleased with our Q2 performance across all aspects of the business, including strong growth, exciting progress across the pipeline, and delivery of our business development strategy. Starting with our strong growth in the second quarter, leveraging our business unit structure to drive focus and accountability, Biomarin achieved double-digit year-over-year revenue growth and significant profitability expansion. Turning to pipeline progress, I'm excited to share that BMN 333, Biomarin's non-acting therapy for children with achondroplasia, achieved our target profile. Our goal is for BMN 333 to demonstrate superiority to VoxOgo and set a new standard for the treatment of achondroplasia. We plan to advance the program to the next stage of development and expect to begin a registrational Phase 2-3 study in the first half of next year. Greg will provide additional details in a few moments. Moving to our business development strategy, we successfully completed the acquisition of Inozyme on July 1, broadening our enzyme therapies portfolio. We executed the transaction with precision and focus, moving from agreement to close in less than two months. A lead program, BMN 401, formerly known as INZ701, is a treatment for ENPP1 deficiency, a condition with high unmet need and no approved treatment options. And we look forward to the pivotal data readout from this program in the first half of 2026. Greg will provide an update on some anticipated milestones with BMN401. Moving to slide seven. our outlook for the remainder of 2025. Building on the strong momentum so far, we will continue to execute on our 2025 priorities with urgency to deliver value to our stakeholders. Looking ahead and building on the momentum, we expect continued strong growth throughout the remainder of 2025, leading us to increase our full-year guidance for total revenues, non-GAAP operating margin, and earnings per share. We are progressing our pipeline, as announced today, with BMN 333 advancing, BMN 401 pivotal data expected to read out next year, and a number of other updates anticipated over the coming quarters. And building on the InDesign acquisition, we plan to continue to augment our portfolio with strategic business development transactions to diversify our growth strategy. In conclusion, with the first half of the year now complete, I'm pleased with our progress and remain enthusiastic about our potential to deliver for patients, employees, and our shareholders through the remainder of 2025 and beyond. Thank you for your attention. I will now turn the call over to Brian to provide our financial highlights for the quarter. Brian. Thank you, Alexander.

Disclaimer

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