BioMarin Pharmaceutical Inc.

Welcome to the BioMarin second quarter 2020 financial results conference call. Hosting the conference call today from BioMarin is Tracy McCarthy, Vice President of Investor Relations. Please go ahead, Tracy.

Thank you, Lori, 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 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. 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, Robert Baffey, President of Global Manufacturing and Technical Operations, Hank Fuchs, President Worldwide Research and Development, and Brian Mueller, Executive Vice President and Chief Financial Officer. We hope to keep this call to one hour, 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 our Chairman and CEO, JJ Bien-Aimé.

Thank you, Tracy. Good afternoon and thank you all for joining us on today's call. We hope that you and your families have been well during these uncertain times. For BioMarin, we continue to manage the business in new ways to ensure access to our essential therapies around the world. I want to commend our employees for their extraordinary commitment to our mission of providing innovative treatments to people with rare diseases, which has been especially challenging in the COVID-19 world. Our second quarter results of $430 million in total revenues or 11% growth over last year is a testament to the importance of our therapies, our diversified product base, and far-reaching commercial food practice. Consistent with our commentary in Q1, we experienced impact from COVID-19 In the second quarter, we hope to return to normal demand patterns by the end of 2020, barring any significant deterioration of the pandemic situation before year-end. We continue to expect to achieve full year revenue and operating expenses guidance as communicated on our first quarter call. Moving now to the most anticipated milestones of the year, the advancement of our two product candidates Under regulatory review, Roctavian gene therapy for the treatment of severe Haemophilia A and Rosoritide for the treatment of achondroplasia. In June, we were pleased to share the four-year Roctavian data update at the World Federation of Haemophilia's virtual summit. Following a one-time infusion of Roctavian, all study participants who received the 2B marketed dose remain off exogenous factor VIII prophylaxis therapy and demonstrated a greater than 90% reduction in bleeding episodes four years after treatment. With our PDUFA target action date later in August, we are going to continue our policy not to comment on the status of the review of our BLA. I want to thank you for bearing with us as we look forward to potential FDA approval later this month. Stay tuned. Moving to Missouri Tide, two weeks ago we submitted the marketing authorization application to the European Medicine Agency for the treatment of children with achondroplasia. I want to thank everyone across the organizations who worked on this application under unusual and difficult circumstances due to challenges faced by COVID-19. It seems there is nothing the teams can't accomplish. Once the application is validated, We expect the NDA review to begin later this month. We are on track to submit the Rosoritide NDA to the FDA later in this quarter. If approved, Rosoritide would be the first and only medicine designated for the treatment of achondroplasia, the most common form of dwarfism in the U.S. and in the EU. These key events, combined with our goal of turning profitable on a cap pace since this year, for the first time in the company's history, has had us very busy. It is no coincidence that BioLine is at a significant inflection point in 2020. We have been growing the company for many years, honing our R&D platform, advancing our manufacturing capabilities, expanding our commercial footprint, and working with health authorities around the world to develop essential medicines for people with rare diseases. We have positioned ourselves for substantial success