BioMarin Pharmaceutical Inc.

Welcome to the Biomarin First Quarter 2021 Financial Results Conference Call. Hosting the conference call today from Biomarin is Tracy McCarty, Vice President of Investor Relations. Please go ahead, Tracy.

Thank you, Grace. Thank you all for joining us today. 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 authority, availability of capital, future actions in the pharmaceutical market, and developments by competitors. And those factors detailed in Biomarin's filings with Securities and Exchange Commission, such as 10Q, 10K, and 8K reports. On the call remotely from Biomarin management today, are 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, Chief Financial Officer. I will now turn the call over to BioMarin's Chairman and CEO, JJ Bien-Aimé.

Thank you, Tracy, and good afternoon, everyone. We hope you and your families are well and enjoying the return to normal day-to-day activities as access to COVID-19 vaccines increases and the U.S. continues to open up. So we had a very strong start to 2021, recording $486 million of total revenues and gap net income of $17 million in the first quarter and $114 million of cash flow from operations, an important achievement. We have also reaffirmed previously released financial guidance for the full year. This takes into consideration both the typical uneven ordering patterns in some of our ex-US regions, as well as continued uncertainty outside of the US due to COVID-19. It is not by chance that Biomarine has successfully managed through the challenges caused by the global pandemic. Our business is built to weather uncertainty, given the essential nature of our products. The underlying strength of our business is quantifiable across a number of metrics. Balmain's five-year non-GAAP income compounded annual growth rate, or CAGR, of 62% far exceeds the 12% average of biotech companies with at least $1 billion of revenues in the last five years. Our five-year revenue CAGR of 16% is better than 10 of the 15 biotech companies with at least $1 billion of revenues over the last five years. With the approval of Bozoritide in European sites and the EMA marketing representing an opportunity, the new EMA market, so we're representing an opportunity that's roughly three times the size of the U.S., we are poised to deliver meaningful growth beginning in 2022. On the heels of potential Bozoritide approvals this year in the U.S. and Europe, and with the European filing of Roptavion, she's targeted for this June, we could potentially receive a second significant product approval in Europe in the second quarter of next year. Similar to the achondroplasia market opportunity in the EMA, we believe that there are three times as many severe hemophilia patients in that region as compared to North America, representing transformational growth opportunities with the zorotide and roptavian approvals and commercializations. Assuming favorable results with Roktavian at year two, we will target a biologic license application, or BLA, resubmission in the U.S. in the second quarter of 2022, followed by an expected six-month review procedure and then potential U.S. approval at the end of 2022, next year. Based on the cumulative data to date with Roktavian, with both the 60 to the 13 and the 40 to the 13 doses, We believe it is reasonable to expect durable bleeding control at year two in the phase three study. And also that's based on the first 17 patients that we communicated that reached also the two-year goalpost. So if the data are supportive to your efficacy and safety data from our expansive program, it would represent significant cost savings to global healthcare providers. It should be a very momentous 2021 and 2022. with this exciting catalyst on the horizon. Thank you all for your continued support, and I would like to turn the call over to Jeff to discuss the commercial business update. Jeff?