BioMarin Pharmaceutical Inc.

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. The 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 available in the investor relations section of our website. On the call from Biomarin Management today are Alexander Hardy, President and Chief Executive Officer, Hank Fuchs, President of Worldwide R&D, Brian Mueller, Executive Vice President, Chief Financial Officer, and Kristen Hubbard, Executive Vice President, Chief Commercial Officer. I will now turn the call over to Biomarin's President and CEO, Alexander Hardy.

Thank you, Tracy, and good afternoon, everyone. Thank you all for joining us today. We're pleased to today share our strong quarterly results and the decision on Roptavian. This quarter, we've also made significant progress towards finalizing Biomarin's evolved corporate vision and strategy. This work gives us confidence we can amplify our progress and mission to deliver more impact on patients' lives and greater value for shareholders. We look forward to sharing more about our vision and strategy for sustainable growth and accelerated profitability next month at Investor Day. Moving briefly to specific progress made in the quarter on our four strategic priorities outlined in January. Beginning with our opportunity to accelerate and maximize VoxOgo, we were pleased with continued strong demand and patient uptake all geographies, resulting in nearly 900 children beginning VoxOgo treatment in the first half of this year, the highest in VoxOgo's history. In now the third year of our commercial launch, we have seen rapid global expansion VoxOgo across all ages, and particularly from families pursuing treatment for their infants and young children to allow for the maximum potential therapeutic benefit. Outside of the US, we've seen significant penetration in key strategic markets, but still with room to grow in those markets. In the United States, our largest potential market opportunity, we saw a strong uptake in children of all ages. But note that new prescriptions for children under the age of five accounted for the majority. Going forward, we intend to leverage the momentum we're seeing in the United States and expand penetration rates across all other geographies. Even with this tremendous progress, we are still at the beginning of realizing the full opportunity with VoxOgo in achondroplasia. And we intend to build on our expanding leadership to rapidly advance across multiple new indications. In the quarter, we made significant progress advancing the development programs in hyperchondroplasia, idiopathic short stature, Noonan syndrome, Turner's syndrome, and shocks deficiency. And Hank will provide an update momentarily. The growing body of evidence supporting the safety profile, the proven mechanism of action, and the expanding data across many attributes beyond height give us a high degree of confidence in our sustainable leadership strategy across multiple growth-related conditions. The second priority to establish the Roktavian opportunity was addressed today in a separate press release. The result of our evaluation over the last couple of quarters supported our decision to enable Roktavian's ability to contribute to long-term revenue growth while balancing resource allocation consistent with BioMarid's profitability. This decision on Roktavian was based on four conclusions. One, our belief in the therapeutic profile of Roktavian and its role in Haemophilia A. Second, our understanding that a launch like this takes time. Third, signs of progress in the United States, Germany and Italy. And lastly, a revised expense profile that gives us confidence Roktavian will contribute to profitability. By focusing commercial research and manufacturing efforts, we expect to reduce the annual direct Roktavian expenses to approximately $60 million. We will begin to operationalize these reductions in Roktavian expenses by the end of this year. So the new lowered expenses are in effect beginning for the full year 2025. As a result of these changes, the company is committed to Roktavion being profitable by the end of 2025. In addition to changes in resource allocation to allow Roktavion to realize its potential, as well as to contribute to Biomarin's profitability, we will reorganize the global Roktavion support team be a dedicated unit that will focus exclusively on Roktavion's success and enable the rest of the enterprise to focus only on the rest of our portfolio. We believe that this direct accountability will enable Roktavion's maximum potential of this revised strategy. Kristen will elaborate on the incremental progress we are seeing in the three prioritized markets where we have made progress in securing reimbursement and providing access to patients. The third priority is our focus on the most promising R&D assets. We made continued progress on BMN 351, BMN 349, and BMN 333, the three prioritized programs announced last quarter. All five new indications with Voxogo also advanced and developed. And we look forward to providing an update on all these programs, as well as our internal external innovation strategy at investor day, including how it fits into our capital allocation strategy. Lastly, our fourth priority, to increase profitability faster than originally planned. Today, we announced 20% growth in total revenues and 78% growth in non-GAAP earnings per share, proof of our ability to drive growth, realize cost efficiencies, and accelerate profitability. Strength across the business resulted in today's increase in all four-year guidance items. Today's results are just the beginning of the profitability expansion we're planning over the next couple of years and into the longer term. Also at the end of July, we were very pleased to have received approval for Benura that expands access to children under the age of three years with CLN2 disease. This expansion now enables treatment for children of all ages with CLNL2 disease, regardless of whether they're symptomatic or pre-symptomatic. In this rapidly progressing neurodegenerative disease, beginning Brunera treatment as early as possible has the potential to alter the natural course of the disease. I want to extend my thanks to the families in our program and Biomarin's R&D team who worked diligently to gain this expanded approval on their behalf. In summary, we have made significant progress across the enterprise to reshape operations, to enable greater efficiencies, and focus on what we do best, create innovative and high-impact medicines for patients. We look forward to seeing you next month to share more specifics on our outlook and strategy. Thank you for your attention, and I will now turn the call over to Kristen to provide an overview on commercial highlights in the quarter.

Thank you, Alexander. I'm pleased to join you on my first quarterly results call since joining Biomarin in May. Now, I've been impressed by the breadth of commercial expertise and patient focus at Biomarin, and I'm excited about what lies ahead as we drive continued patient impact through the expansion of our innovative medicines. Turning to quarterly highlights, strength across our brands drove 20% revenue growth year over year, including notable contributions from Voxogo, Naglazyme, Allenzic, Brunura, Vemizem, and Roctavian. Moving to specific accelerators in the quarter, Voxogo revenues were up 62% to $184 million compared to the second quarter of last year. By the end of the second quarter, 3,500 children were receiving Voxogo treatment. As we told you previously, we expected to reach ample supply capacity by mid-year, and we did. We've navigated through the supply constraint of the last few quarters, enabling high market penetration rates in several strategic markets in the quarter, including strong momentum in the United States, our largest potential market. Our successful supply chain efforts allowed customers to normalize stock events in Q2, and we expect this to meet demand going forward. Now, in the U.S., confidence in Voxogo's safety and efficacy from treating physicians and families is solidifying our leadership in achondroplasia, and is underscored by our more than 25 years of experience treating children with skeletal dysplasia. We intend to build on strong demand in Q2 to drive continued market expansion in the United States over the coming quarters. Our efforts to expand our base of prescribers for families seeking treatment with Vuxota are rapidly gaining momentum. Now, outside the U.S., we've achieved significant penetration rates in all strategic markets and expect to continue to grow in those markets, albeit at a more measured pace. Voxogo is the only approved product for achondroplasia and has an extremely strong track record of safety and efficacy with more than 6,000 patient years of evidence to support it. This experience facilitated direct entry into our pivotal study in hypochondroplasia, our second indication, and will set us up for continued expansion into four additional federal indications, none of which have any competitors on the horizon. In my experience, having the opportunity to build the standard of care with a brand like Voxogo does not happen that frequently. It's a tremendous opportunity to have an impact on the lives of thousands of children across a multitude of growth-related conditions, and I look forward to helping build and sustain our leadership. Turning now to market dynamics in the quarter across our enzyme therapies, we were pleased to see combined quarter-over-quarter growth of 15% compared to Q2 last year. 47% growth of Naglazyme year-over-year was driven by the timing of a few large orders. and 18% growth in Palazinc was driven by new patient starts in the quarter, primarily in the U.S. Now, turning to our Roctavian results, we were encouraged that patients were treated in both the U.S. and Italy, driving approximately $7 million of revenue in the quarter. Alexander has outlined our updated strategy to reduce the resourcing of Roctavian and to focus on three existing commercial markets where the drug is approved and reimbursed, which are the United States, Germany, and Italy. To expand on this rationale, the decision was based on signs of progress in those markets, including the successful treatment with Ractavian at multiple U.S. hemophilia treatment centers. We believe as more and more centers gain patient treatment experience in the U.S., patient flow will become more straightforward. In Germany, we're also making progress. At our last update, we shared that subinsurers had yet to green light reimbursement for Ractavian treatment, despite there being an approved government price. And I'm pleased to share that we have finalized an agreement with one sub-insurer, opening access for some wanting treatment with Ractavian. And we continue to make good progress with a number of other sub-insurers. Now, in Italy, we're seeing encouraging signs of patient demand, and two patients received treatment there in the second quarter, advancing access and experience. As we shared in our press release today, we are prioritizing these three commercial markets with a focus on facilitating patient access at the site level. With this encouraging progress and the strategic focus on viable commercial markets, enabling a significant reduction in operating expenses related to Roktavian, we believe there's a path forward to treat more patients with severe hemophilia A while also contributing to the company's profitability. We believe that adjusting Roktavian expenses to approximately $60 million beginning in 2025 is the right level to be able to make progress in our three prioritized markets. So in conclusion, Strong commercial results in the quarter and expectations for the remainder of 2024 led to today's increase of a full year guidance. I'm excited by the prospects ahead and the opportunity to drive profound patient impact with our innovative therapies. So, thank you for your attention, and I'll now turn the call over to Hank to provide an R&D update. Hank?