Ultragenyx Pharmaceutical Inc.

Good afternoon and welcome to the Ultragenyx second quarter 2024 financial results conference call. At this time, all participants are in listen only mode. At the end of the prepared remarks, you will have an opportunity to ask questions during the question and answer portion of the call. Vice President of Investor Relations, please go ahead.

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 filing. I'll now turn the call over to Emil.

Thanks, Josh, and good afternoon, everyone. We've had an incredible first half of the year and look forward to sharing more with you today. On the commercial front, our strong revenue performance puts us on a trajectory to outperform our prior projections, and so we're raising our total revenue guidance range. We're happy with what this means for access to our drugs globally, And Eric and Howard will share more on the revenue details. Within our clinical pipeline, we've meaningfully advanced our late stage program through multiple positive data readouts and successful and critically important regulatory interactions. On the data front, in addition to the positive results we shared earlier this year on both UX111 in San Filippo Syndrome and GTX102 in Angelman Syndrome, We recently announced positive phase three results from the DTX401 gene therapy for the treatment of patients with glycogen storage disease type 1A and additional long-term positive phase two results from the UX143 antibody for the treatment of patients with osteogenesis imperfecta. For GTX102 for Angelman syndrome, we announced the successful completion of an end of phase two meeting with the FDA where we aligned on phase three study design and key endpoints to be evaluated. Our teams have been working with our study sites to initiate our global phase three study by the end of this year. On UX111 for Sanfilippo syndrome type A, we also reached agreement with AHC on a path forward to seek accelerated approval. The FDA has agreed that cerebral spinal fluid heparin sulfate is a reasonable surrogate endpoint to support submission of a BLA supported by our clinical data to date. Our next step is to finalize the details of our submission with the agency in a pre-BLA meeting, and we intend to submit this BLA later this year or early next. Collectively, this puts us in a position to have multiple regulatory marketing submissions and key clinical data readouts over the next 6 to 18 months, which is extraordinary. I spent My career developing therapies in rare disease, and I can tell you I've never seen a rare disease company with the breadth and depth of opportunities we have ahead of us, nor with our ability to move all these things forward. These achievements are a direct result of our excellent execution across the company from our committed employees and our best-in-class approach to rare disease drug development. It's a very exciting time for Ultragenyx. I'll now turn the call over to our Chief Commercial Officer, Eric Harris. to provide an update on the momentum across our commercial portfolio that has led us to raise our total revenue guidance for the year.