Ultragenyx Pharmaceutical Inc.

Good afternoon, and welcome to the Ultragenyx fourth quarter and full year 2023 financial results conference call. At this time, all participants are in a listen-only mode. At the end of prepared remarks, you will have an opportunity to ask questions during the Q&A portion of the call. It is now my pleasure to turn the call to Joshua Higa, Vice President of Investor Relations.

Thank you. We have issued a press release detailing our financial results, which you can find on our website at Ultragenyx.com. Joining me on this call are Emil Kakas, Chief Executive Officer and President, Eric Harris, Chief Commercial Officer, Howard Horn, Chief Financial Officer, and Eric Krambes, Chief Medical Officer. I'd like to remind everyone that during today's call, we will be making forward-looking statements. These statements are subject to certain risks and uncertainties, and our actual results may differ materially. Please refer to the risk factors discussed in our latest SEC filings. I'll now turn the call over to Emil.

Thanks, Josh, and good afternoon, everyone. In 2023, we generated significant momentum across our commercial and clinical programs that have set us up for Catalyst Bridge 2024. On the commercial front, we progressed international regulatory and reimbursement negotiations across our marketed products, continuing to add to new approvals and positive reimbursement decisions. This geographic expansion, along with growing demand in our existing markets, put us in a position to maintain our trajectory of robust year-over-year growth. On the clinical side, we released new data on our key programs in 2023 and successfully advanced enrollment of our priority programs that can accelerate value creation for our company this year and in the coming years. At our analyzed day in October, we shared exciting data from three programs that all will have additional data catalysts in 2024. On UX143 for osteogenesis imperfecta, interim phase two data showed rapid and substantial increases in bone marrow density and Z-score after just six months of treatment. This led to a 67% reduction in analyzed fracture rate with 20 of the 24 patients showing no radiographically confirmed fractures at six months. On GTX102 for Angelina Syndrome, data from the long-term extension cohorts in the Phase 1-2 study demonstrated clinically meaningful improvements across multiple neurodevelopmental domains, including cognition, receptive communication, gross motors, behavior, and sleep. These data show we can repeatedly dose GTX102 for much longer than one year with acceptable safety profiles. On UX701 for Wilson disease, the data we presented showed four or five patients in the lowest dose cohort had begun tapering their standard of care, including two who were completely off key letters and or zinc therapy. While still early for the UX701 program, we are encouraged by the data we have observed. Our momentum is continuing into 2024 with multiple clinical development updates. Last week, GTX1-2 was granted priority medicine or PRIME designation by the European Medical Authority based on the positive early clinical data from the long-term extension cohorts and the potential of GTX1-2 to address the unmet need for a treatment in this disease. The clinical data we've shared with the street, along with positive U.S. and European regulatory interactions, give us confidence that we will be able to navigate the development path for this program. For UX111 and Sanfilippo A syndrome, we released Phase III data last week from extension and newly treated patients at the World Symposium Annual Research Meeting in San Diego. The results we shared demonstrated treatment with UX111 resulted in rapid and sustained decreased levels of heparin sulfate in the cerebral spinal fluid, and the sustained reductions in CSF heparin sulfate exposure over time was correlated with improved long-term cognitive development. Our discussions on accelerated approval pathway with the FDA are ongoing, and we continue to believe there's a strong case to be made in this program. Looking ahead, we are entering 2024 in a robust financial position with $777 million on the balance sheet, including proceeds from our equity offering in the Q4 that raised $326 million. We're also continuing to focus our spend and resources on the key clinical programs that will drive value for our company, many of which will be updated in the coming months. Our Chief Medical Officer, Eric Krambez, will review more of these updates in his section. There's a lot to look forward to as we continue our efforts to lead the future of rare disease medicine. Now I'll turn the call over to our Chief Commercial Officer, Eric Harris, to provide an update on our commercial efforts last year that led to a 20% revenue growth in 2023.