BioMarin Pharmaceutical Inc.

Welcome to the BioMarin Pharmaceuticals First Quarter Investor Update Call. Hosting the conference call today from BioMarin is Tracy McCarty, Group Vice President, Investor Relations. Please go ahead, Tracy.

Thank you, Ross, and thank you all for joining us today. To remind you, this non-confidential presentation contains forward-looking statements about the business prospects of BioMarin Pharmaceuticals, 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 authority, availability of capital, future actions in the pharmaceutical market, and developments by competitors. And those factors detailed in Biomarin's filing with the Securities and Exchange Commission, such as 10Q, 10K, and 8K reports. On the call today from Biomarin's management team, our JJ Bien-Aimé, Chairman and Chief Executive Officer, Jeff Ager, Executive Vice President, Chief Commercial Officer, Hank Fuchs, President, Worldwide Research and Development, Greg Geyer, Executive Vice President, Chief Technical Officer, and Brian Mueller, Executive Vice President and Chief Financial Officer. I will now turn the call over to our Chairman and CEO, JJ Bien-Aimé.

Thank you, Tracy, and good afternoon, everyone. Thank you for joining us today. So we are very pleased with BioBrain's progress in the first quarter as more families gain access to VoxOgo as well as our other essential medicines. Our financial performance in the quarter was strong, especially in light of ongoing macroeconomic challenges across the globe. 15% growth of the top line and 15% non-GAAP income growth on the bottom line puts Biomarine firmly on track to achieving our 2023 financial goals. These results include $88 million in Voxogo revenues and strong Q1 profitability of $51 million on a gap basis. And all this from our fully-owned portfolio of commercial products. With Q1 total revenues coming in at just under $600 million, we are on the path to achieving our 2023 objectives of double-digit revenue growth and significant operating leverage, driving approximately 30% growth in bottom-line profitability in 2023, as communicated in February. We are very pleased with the continued cadence of Voxogo uptake worldwide. Voxogo is now being used in around 1,500 patients in 35 different geographies. And we have seen significant growth of Voxogo in Japan since approval there. And as a result, we are raising, again, our 2023 four-year guidance midpoint by $50 million at the midpoint level based on increasing expectations for the brand. Based on the still very limited market penetration, we believe Voxogo is on its way to become a blockbuster. Turning to Roktavion in Europe, today we have begun working directly with a single national German insurance fund, or GKV, on a final federal German price, and therefore we will not pursue any additional outcome-based agreements with sub-insurance, which are the German, most of them regional SIG funds, at this time. We believe that the highly innovative profile of Roktavion offers an attractive treatment option for those people with severe hemophilia A, interested in an efficacious alternative to chronic therapy. We believe that working directly with a primary health insurance provider in Germany, which we expect will cover Roktavian treatment with a one-time payment and without an outcome-based component, will facilitate access. We also believe the German healthcare system will recognize Roktavian value based on the transformational efficacy observed in a majority of participants across our extensive development program with Roktavian. Our initial interactions with the GKV have been positive and we anticipate a final German reimbursement price that would be representative of the full value Roktavian is delivering to patients, especially based on our recent three-year updates of our phase three trial. Over the long term, we don't expect today's updates to impact our expectations for Roktavian as we continue to hear feedback from German hematologists that they are ready to treat despite some of the challenges Balmain has experienced with German regional stick funds reimbursement in the very short term. We are pleased to share that other markets in Europe and outside of Europe are actively pursuing access to Rokavian, which Jeff will review in a moment. With the US PDUFA action date only two months away, we are optimistic, cautiously of course, for what lies ahead In the second half of the year, the commercial team is working hard to prepare for another successful product launch. And as we communicated during our Q4 conference call a couple months ago, in the United States, roughly 300 patients from the bleeding disorders community have engaged with Biomarine directly to learn more about Roktavion, which is a very positive sign. We believe 2023 is the year of Roktavion, and we are looking forward to continued progress ahead in both Europe, the U.S., and the rest of the world. So, in summary, we are very pleased with Balmarine's performance in the first quarter and our outlook for the remainder of the year. The momentum behind VARSOVA continues, driving record financial results. We are making good progress on the European launch of Roptavian, and we look forward to the outcome of the June 30th PDUFA milestone in the U.S. We are ready. Importantly, we have made the transition to an earnings growth site a unique accomplishment in our industry, and we thank you for your continued support. I will now turn the call over to Jeff to discuss the commercial business updates.