Ultragenyx Pharmaceutical Inc.

Good day and thank you for standing by. Welcome to the first quarter 2021 Financial Results and Corporate Update conference call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1 on your telephone. If you require any further assistance, please press star 0. I would now like to hand the conference over to your speaker today, Joshua Higa. Please go ahead.

Thank you. Good afternoon, and welcome to the Ultragenyx Financial Results and Corporate Update Conference Call for the first quarter 2021. We have issued a press release detailing our financial results, which you can find on our website at ultragenyx.com. I am Joshua Higa, Director of Investor Relations. Joining me on this call are Emil Kakas, Chief Executive Officer and President, Camille Bedrosian, Chief Medical Officer, Eric Harris, Chief Commercial Officer, and Marty Deer, Chief Financial Officer. I would like to remind investors that this call will include forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including but not limited to the types of statements identified as forward-looking in our annual report on Form 10-K that was filed on February 12, 2021, our quarterly report on Form 10-Q that will be filed soon, and our periodic subsequent reports filed with the SEC, which will all be available in the investor section on our website. These forward-looking statements represent our views only as of the date of this call. It involves substantial risks and uncertainties, including many that are beyond our control. Please note the actual results could differ materially from those projected in any forward-looking statement. For a further description of the risks and uncertainties that could cause actual results to differ materially from those expressed in the forward-looking statements, as well as risks relating to our business, see our periodic reports filed with the SEC. I'll now turn the call over to Emil.

Thank you, Josh, and welcome to everyone on the phone. So far, 2021 has been a productive year for Ultragenyx. We've made great progress toward the initiation of four pivotal clinical studies with novel therapies for rare genetic diseases and a broad portfolio of six clinical stage programs. The clinical progress is complemented by continued advances in our three commercial franchises, marking 2021 as an important inflection point in our growth as a company. Starting with gene therapy, we made significant regulatory and operational advances toward the initiation of the DTX301, DTX401, and UX701 pivotal studies. Most recently, we completed successful regulatory discussions for DTX301, our AAV8 gene therapy for OTC deficiency as one of the last key steps before initiating that Phase III study. We've also completed all the formal regulatory discussions for the DTX401 Phase III and are working with sites to get the study started soon. Early in the year, we announced that the IND is active for our innovative Phase 1-2-3 study for UX701 and AV9 gene therapy for Wilson disease. We're currently on track to launch all three of these pivotal studies this year, with the GSD1A and Wilson studies on track to begin rolling early in the second half. In addition to the late-stage gene therapies, we are pushing ahead with UX143, the monoclonal antibody for osteogenesis imperfecta, that we recently licensed from Muriel Biopharma. We are in the planning process for the Phase 2-3 study for this program, and we are currently on track to kick off this pivotal study in pediatric patients by the end of the year. As a reminder, osteogenesis imperfecta is one of the largest groups of rare genetic bone disorders and is an excellent complement to Chris Vita with respect to the clinical and commercial capabilities needed to craft and execute a top-notch development and launch plan. Moving to GTX-102, the antisense alginucleotide program for Angelman syndrome. We are continuing to make progress towards resuming the Phase 1-2 study. Our goal for the program at this specific time is twofold. First, to move forward as quickly as possible, we start dosing at the low doses that showed activity with an individually monitored dose penetration plan to assure safety. And second, to broaden the scope of the program to allow the program to accelerate once a safe and active dose level has been determined, given the strong activity we have seen with the molecule so far. To those goals, our partner, GT Genetics, submitted an amendment to the existing approved Canadian filing to initiate the study in Canada. Genetics also submitted a new clinical trial application with a second ex-U.S. regulatory agency to open clinical sites outside of the U.S., We had an encouraging discussion with that European regulatory agency regarding GTX102, including all the detailed efficacy, safety, and nonclinical data, along with the same dosing and monitoring plan that was provided to the FDA. The European agency agreed in principle with this analysis and plan, and a full filing has been made on this basis to that agency. Based on this interaction, we are confident we can start the study outside the U.S. this year. Our discussions are continuing with the FDA on removing the clinical hold. We have submitted additional clinical information requested, and a meeting is pending with the FDA. Camille will provide a little more detail on this later in the call. We look forward to providing updates as definitive milestones are made in the ANGMAN program. Moving to our newest clinical stage program, UX053 is our mRNA candidate for GSD3, or Glycogen Stored Disease Type 3, D branch or deficiency. This is the first clinical program to come out of our collaboration with Arcturus, through which we have rights to 12 programs. We're working on two other earlier projects in the mRNA space that we will discuss as they mature. We're always evaluating additional opportunities to harness this promising technology for patients with rare genetic diseases. In the last year, mRNA has come forward as not just a theory, but a viable strategy based on the highly successful COVID vaccine program. And we are also very encouraged with what we see with this technology. The USO5-3 program has received clearance from the FDA to start the Phase 1-2 study in Glycogen Steroid Disease Type 3, or Deep Branch Deficiency. And this program will further build on our experience with gene therapy in Glycogen Steroid Disease Type 1a, or von Gierke's disease, and is expected to start later this year. Turning to our commercial portfolio, we had a good Q1 that Eric and Marty will describe further. CRISFIDA in North America continues to do well now that we are three years into launch. As COVID restrictions have eased somewhat and clinics further reopened in 2021, we have seen an increase in diagnosis of XLH patients that we expect to help drive long-term growth for CRISFIDA. We have initiated or strengthened a number of efforts to support this increased diagnosis in XLH. This includes increasing our digital and social media activities, broadening the set of physicians we meet with, and using patient education and genetic counseling to improve diagnosis across a broad family tree. In Latin America, the team continues to make good progress in establishing the value of CRISFIDA for patients with XLH, and we continue to see more and more patients winning injunctions to receive reimbursed therapy, which has started to generate meaningful revenue. Turning to OJOLVI, which has been off to a great start, We saw strong demand in the third and fourth quarter of 2020, driven by conversion of 80 clinical trial and existing compassionate use patients and the most severe of diagnosed patients. We see a solid growth rate of new starts going forward, and we're encouraged by the great positive feedback we are receiving from prescribers in the U.S. In Europe, our discussions with regulators continue, but we're also seeing strong inpatient demand for Dojolbe. Recent longer-term efficacy data and safety from 18 ATU patients in France. These are named patient treated patients. We're published in molecular genetics and metabolism. Camille will get into the details later in this call, but it's good to see our phase two data corroborated by an independent French investigating team in their patients in France. I'll now hand the call off to Eric to provide more detail on our commercial progress.