BioMarin Pharmaceutical Inc.

Welcome to the Biomarine Second Quarter 2021 Financial Results Conference Call. Hosting this conference call today from Biomarine is Tracy McCarthy, 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 Biomarine Pharmaceuticals, including expectations regarding Biomarine'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 program, 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 the Securities and Exchange Commission, such as 10Q, 10K, and 8K reports. On the call today from Biomarine Management are JJ Vianame, 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. We hope to keep the call today to one hour and give everyone a chance to ask a question, 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 will now turn the call over to Biomart's Chairman and CEO, JJ Bien-Aimé.

Thank you, Tracy, and good afternoon, everyone. We hope your families are enjoying the summer. So we had very strong results in the first half of this year, recording $988 million in total revenues in the first six months of the year, and half a billion dollars in total revenues in the second quarter loan. representing a 17% top-line year-over-year growth, including Kuvant, and 38% year-over-year growth in Q2, excluding Kuvant, which, as you know, has been facing generic competition in the U.S. since October of last year. These results underscore our ability to execute despite the impact of a global pandemic, and more importantly, are a testament to the value our products represent to patients. Based on this strong performance, we are pleased to be raising our total revenue and bottom line guidance for the year, and Jeff and Brian will provide more details in a moment. With the commercial business increasing steadily, we look to the next generation of bio marine products to drive transformational growth in the coming years. With our late stage products, Voxogo and Roctavian, currently under review by health authorities, the R&D organization achieved a number of key goals in the first half of the year. In this quarter, Hank and his team advanced regulatory progress with both products, setting up for four potential US and European approvals, beginning with Vox Togo in Europe in the next few weeks, hopefully by the end of next month. And actually, we are going to start treating Our first commercial box local patients in France imminently based on early access program which was approved the so called ATU in France and Jeff will give some more details in a little while. So we expect EU approval to be followed by potential approval in the US based on the current action date of November 21 and discussions with the FDA are moving forward. With Rokavian, the recent validation of the MAA and potential CSMP opinion in the first half of 2022 paves the way for a third potential measure of market approval in the next 12 months upon completion of the three-year observation from all 134 patients in our pivotal GNRI-1 study With Roptavia, this will be the basis of a potential BLA submission next year in the U.S. Should the data be supportive, as we expect, and then anticipating a fourth major market approval, if possible, potentially in late 2022. The value proposition of Roptavia increases with each additional year of bleeding control as demonstrated. As we just shared at the recent ICH meeting, from our PIVOTL-GENERATE-1 study, over 90% of the 134 patients had an annualized beating rate of zero or lower than baseline after four weeks following treatment with Roktavian, and this is just with one intravenous infusion of Roktavian. Also, an ISDH from our Phase I-II study following a single infusion of Roktavian throughout these five years, we observed a 95% reduction in mean annualized bleeding rates to less than one bleed per year compared to the bleed rates from participants previously treated with standard of care factor V8 prophylaxis. Again, we're not comparing ourselves to placebo here. We're comparing ourselves to steel of care and standard of care. So we are recognizing that it has to be determined how long rhododendron will demonstrate hemostatic efficacy. But we have reason to believe, based on the current data, that bleeding control may be sustained for at least five years and beyond, based on the current data. And while gene therapy is not curative, and it's not as likely to be lifetime therapy at this time, the value proposition Roktavion represents to patients and payers only strengthens with each year of data we are accumulating. based on the five-year bid control from our Phase 1-2 study, at least $3.25 million per patient has been spared for each patient in the clinical trial in the U.S. This is based on the fact that in the U.S., recover and factorate prophylactic therapy costs about $650,000 per year. So as you can imagine, payers will understand this kind of math very easily. So when considering value, we recently learned from a review of some insurance claims of patients taking Imlibra in the U.S. that many of them are still using recalibrated factor VIII despite being treated with Imlibra. This is based on a review of insurance claims in the year 2020. This not only highlights the ongoing infusion burden with Imlibra, but the tremendous cost of potential duotherapy. Hemolibra, which is chronic therapy and can cost over $700,000 per year for your patients, in addition to whatever factor weight the patients are taking. The bottom line is that the sustained, durable hemostatic efficacy observed following treatment with roctavion in the longest and largest gene therapy trial ever done in hemophilia positions roctavion as a very attractive treatment option to both patients and payers pursuing approval in the U.S. and in Europe. As we shared with you previously from a paper published by Coteau in 2019, the lifetime cost of factor V prophylaxis alone, and not including other aspects of the disease management and surgeries that the patients have to undergo on a regular basis. So the lifetime cost of factor V prophylaxis alone is estimated in that paper to be, and other papers, to be between $28 million and $31 million. So obviously, if we price our product between $2.5 and $3 million, we're not pricing our product as a lifetime cure. But more relevant to the value proposition for Octavian, specifically as noted in an ICER report last year, ICER determined that the value proposition for Octavian was around $2.5 million, and that was a cost-effective threshold for treatment with Octavian, and that was based only on three years of efficacy data. Now we have five years. So we are thrilled with the dramatic B-control demonstrated with Roktavion, and more than ever we believe that it will be an important treatment choice based on efficacy for patients and cost savings for payers. As our next potential significant growth drivers, Roktavion and Voxoco have been a key focus and we expect will be transformational to BioNREN upon potential approvals. but it is our next generation of early-stage pipeline products that we look forward to unveiling in more details later this year. The bottom-end value proposition reaches far beyond our strong-based business and our near-term opportunities in hemophilia and achondroplasia, and we're excited to share more details with you at our upcoming R&D Day in November of this year. So thank you all for your continuous support, and I would like to turn the call over to Jeff discuss the commercial business update in more details.