BioMarin Pharmaceutical Inc.

Welcome to the BioMarin fourth quarter and full year 2020 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, Nikoa, and thank you everyone 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 progress of Biomarin's product program, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical markets, 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. On the call remotely from Biomarin Management Today, our J.J. Bien-Aimé, Chairman and Chief Executive Officer, Jeff Ager, Executive Vice President, Chief Commercial Officer, Hank Fuchs, President, Worldwide Research and Development, and Brian Mueller, Executive Vice President, Chief Financial Officer. We do hope to keep this call to one hour today, so we respectfully request that you limit yourself to one question during the Q&A portion of the call. Thank you for your understanding. I'll now turn the call over to our Chairman and CEO, J.J. Bien-Aimé.

Thank you, Tracy. Good afternoon, and thank you for joining us on today's call. We delivered strong results in the fourth quarter and full year 2020, demonstrating our continued operational excellence despite challenges brought about by the global COVID-19 pandemic. Excluding KUVAN contributions, biomarine revenues grew 13% in 2020 as compared to the full year 2019 revenues. And the company generated $85 million of positive operating cash flows for the full year 2020, underscoring our resilience in a challenging environment and the significant admin needs our medicines address. Our 2021 guidance does assume that the business environment remains fluid due to the ongoing effects of the pandemic, but also reflects the underlying global demand for our commercial brands, including KuVan, again, KuVan contributions in 2021. We expect 9% growth in biomarine marketed product revenue based on the midpoint of today's full year 2021 revenue guidance. We also expect continued positive operating cash flows for the full year 2021, both important indicators of the strength of our global brand. And in a moment, Jeff will provide more details on these market dynamics. Turning to our late-stage R&D programs and upcoming milestones, we are planning for a number of key events that we expect will drive substantial value over the coming quarters. Beginning with our European regulatory update, first with vasoetide for the treatment of children with achondrophagia, we are targeting a CHMP opinion this June, followed by the European Commission decision in late summer leading to a potential commercial launch in Europe this fall. To highlight the significance of these key regulatory milestones, we anticipate that Rosaritide's revenues X-to-X over the next five years represent close to 70% of our anticipated global revenues. Rosaritide continues to advance its plan, and we are optimistic that investors will share the same degrees of enthusiasm as the first pharmacological treatment to address the underlying genetic cause of achondroplasia that we have experienced from the patient community. In Europe, with Octavian gene therapy for severe hemophilia, we are also tracking the plan as we prepare for the resubmission of our marketing application in the second quarter of this year, next quarter. So assuming we remain on anticipated timelines in Europe, this could potentially lead to a Octavian CHMP opinion in the first half of next year, with potential European launch in the third quarter of 2022. As a reminder, We believe that there are three times as many severe hemophilia patients in Europe as compared to North America. In the United States, with both Vazoritide and Octavian, we are focused on providing additional phase three data to inform FDA's reviews of these highly innovative products. We look forward to providing the FDA with the recently available Vazoritide phase three results that demonstrate a sustained growth effect for over two years in children with achondroplasia. further corroborating the resort treatment effects and the long-term durability as will be required for the product's full benefits to accrue. In addition, if few patients from Phase 1-2 are now or at near final adult height, that is substantially in excess of that which their age and gender match peers would have achieved. This is great news for the patients and the patient community. So this shows that zoetide addresses the underlying cause of achondroplasia and that has always been our goal so that the patients may benefit in ways that go beyond natural gain. We believe that results to date across our phase three and phase two programs are supportive of zoetide becoming the first pharmacological treatment for this condition. With Roktavion in the United States, we are planning to dialogue with the FDA to explore the potential submission of the DLA based on available one-year data package as planned for submission to the EMA, with provision of two-year data during the review period. We recently convened a meeting with an expert advisory council to seek community and expert advice into our overall strategy and regulatory considerations. The council provided positive feedback on the data based on the dramatic lead control demonstrated with Octavian treatment. We were pleased with the feedback and insight into the regulatory strategy and the approvability of Octavian, and we look forward to further engagement with both the FDA and the EMA. And Hank, in a few minutes, will provide more details on the feedback. Moving to our earlier stage pipeline, we have numerous programs advancing this year. Starting with BMN307, gene therapy for PKU, we are pleased to share that we are moving to the next higher dose in our Phase I-II study, advancing the third potential treatment modality for our PKU franchise. We are encouraged by the sea-lowering and safety results observed in the first 2E13 cohort in our Phase I-II study, and we are now ready to move to the next dose of 2E13, which is similar to the Roktavian dose. Based on this early data from the 2013 cohort and our prior steep dose response experience with Octavian, we are optimistic that the CC13 dose will be our optimal dose. We will share the next update on the BMN307 from the dose confirmation phase of this study once we have selected the dose for registration enabling studies. In addition to BMN307, we have four other earlier state programs advancing that span a variety of therapeutic modalities and indications, including an oligonucleotide for Duchenne muscular dystrophy, gene therapy products for hereditary angioedema, and also hypertrophic poliomyopathies, respectively, and a small molecule for a subset of a chronic renal disease. In conclusion, despite the challenges, Faced against the backdrop of the global pandemic, demand for biomedicine drove strong results in 2020, and we are expected to grow 9% in 2021, again, excluding the contribution from Kuvaan, which is facing generic competition in the U.S. We look forward to this new year and the goals that lie ahead over the coming quarter. With our foundations of brands that constitute a strong-based business, By being positive operating cash flow, enabling the advancement of our next significant product opportunities with Vazorita and Roktavion, we are well positioned for substantial growth over the coming quarters and we anticipate significant revenue growth starting in 2022. After Vazorita and Roktavion, we look forward towards our earlier stage pipeline to ensure a steady flow of new product opportunities on the medium and long-term horizons. And we'll search all these programs in a moment. And finally, I just want to add that we ended 2020 with over $1.3 billion in cash and investments. And we anticipate being operating cash so far this year. So thank you for your continued support. And I will now turn the call over to Jeff for a discussion on our commercial business. Jeff.