Ultragenyx Pharmaceutical Inc.

Good afternoon and welcome to the Ultragenyx First Quarter 2025 Financial Results Conference Call. At this time, all participants are in a listen-only mode. At the end of the 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 over 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 ultragenics.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 the first quarter, we continued to make meaningful progress across one of the most productive commercial and development pipelines in rare diseases. Our commercial team delivered a strong quarter that puts us in position to have another year with meaningful revenue growth. Our early investments in high-performing teams have helped generate substantial revenue growth. while we commercialize our products outside of the United States. At the same time, we are preparing to launch our next set of programs in the U.S. and around the world. Our development teams have advanced our large and late-stage programs as well. For the UX143 and Osteogenesis Perfecta, patients in the phase three phase have now been enrolled for at least a year, and the process has begun to clean and lock the databases for our second interim analysis. For GTX102 and Angelman syndrome, the Phase III is enrolling efficiently at sites in the United States, Canada, Japan, Germany, Poland, and Spain, and data are expected in 2026. For DTX301 and ornithine transcarbamylase deficiency, or OTC, the Phase III study completed enrollment in the first quarter and is on track to read out data over the next year. For UX701 and Wilson disease, the study is now enrolling the fourth dose finding core that will enable dose selection and transition to the pivotal stage. At the same time, we're working on two separate BLAs, one currently under review and a second to be submitted. The GTX 401 for GSD 1A BLA submission is on track for mid-2025 after successful completing the PPQ runs, the qualification laws essentially, at our manufacturing facility in Bedford, Massachusetts. The UX111 for San Filippo syndrome BLA under review by the FDA is progressing on schedule as expected. It's not our standard practice to go into the details of regular interactions, but I think it's meaningful at this current time for investors to be aware that our interaction with the FDA on the UX111 BLA review thus far remain on track. Last month, we had our mid-cycle review meeting that occurred on the standard timeline. We also know the inspections of the manufacturing facilities and clinical sites have been scheduled according to normal cadence and are currently underway. We remain on track for the BDUFA action date of August 18. Going forward, we don't plan on giving the details of all our regulatory interactions, but we did want to share enough detail for you to remain confident as we are that the UXDLA review is progressing according to plan. With that, I'll turn it over to the rest of the team to share the details of why 2025 will be a transformational year for Ultragenyx. Eric, I'll hand it off to you to go through the commercial team's execution in the first quarter.