Ultragenyx Pharmaceutical Inc.

Good afternoon and welcome to the Ultragenix second 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 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 ultragenix.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. And now I'll turn the call over to Emil.

Thanks, Josh, and good afternoon, everyone. In April 2025, we marked our 15-year anniversary as a company. Over that time, we've taken four programs all the way through approval in multiple regions and added a fifth that we are commercializing outside of the U.S. At the same time, we've also refilled the clinical pipeline and now have five Phase III clinical programs fully enrolled or at the BLA submission stage. And over the last 15 years, Ultragenyx has been the most productive rare disease company in the industry. across a broad range of modalities and therapeutic areas. Through the first half of 2025, continuous momentum with meaningful progress across our larger programs. I'll begin with UX143, citruzumab, for our fully human monoclonal antibody for the treatment of osteogenesis imperfecta. The orbit and cosmic studies are continuing to the final analyses that will occur around the end of the year. While I hope the studies might have stopped early at the interim time point last month, we remain confident in completing a successful study. We're pleased the safety profile is as expected, and that after looking at the data, the DMC recommend we continue to the final analysis. As we head to the final analysis, the continued treatment of Phase III should further strengthen bones of the treated patients. The additional six months of treatment for the treated subjects, along with the larger P-value threshold at 0.04, will help power the final assessment. We look forward to unblinding the Phase III datasets and sharing the results around the end of the year. Now, based on all the data we've seen in Phase II, we're confident UX143 will be a transformational treatment for pediatric and adult patients with osteogenesis imperfecta. The combination mechanism of building bone and reducing excess resorption at exactly the sites in their body where they need more bone will increase bone strength and reduce fractures, while at the same time improving overall bone health. In addition to reducing fractures, we were encouraged by the functional effect we were seeing on increasing physical activity ability that speaks to long-term potential for this treatment. Now, shifting now to GTX102, our antisense oligonucleotide, or ASO, for the treatment of Angelman syndrome. GTX102 received breakthrough therapy designation from the FDA in June. BTD aims to expedite the development and review of drugs that are intended to treat serious or life-threatening diseases and whose preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies. Historically, it seems like there have been relatively few breakthrough therapy designations granted in the field of neurology because it can be challenging to demonstrate a meaningful potential clinical effect on neurologic disease symptoms, but for GTICS 1 and 2, we have been able to do that. The BTD designation was obtained based on our Phase I-II study data in 74 patients with a full maternal UB3A deletion that showed consistent developmental gains with rapid sustained and continued improvements across multiple symptom domains when patients have been on therapy for up to three years. For GTX102, the magnitude and durability of our Phase I-II data indicate our ASO is meaningfully improving lives of patients who have this neurodevelopmental disorder. Last week, we also announced that GTX 1 and 2 Phase 3 study, ASPIRE, completed enrollment ahead of plan with 129 patients in seven months. The interest from investigators and patients helped drive the rapid enrollment, but I also applaud our team for their efforts to expeditiously enroll this study. We're thankful for the support from the patient communities and investigators who helped us exceed our expectations. We're now on track to read out Phase III data from this 48-week study in the second half of 2026. Now switching to our commercial progress for the first half of 2025, our commercial teams continue their trend of delivering double-digit revenue growth. The $306 million in total revenue across the first two quarters represents 20 percent growth versus the prior year, and keep us on track to deliver $640 to $670 million of total revenue this year. Prisvita is continuing to grow in line with our expectations from Royalty Revenue U.S. and Canada and Product Revenue in Latin America and Turkey. Dolcevi, Avkisa, and Metsevi also continue to contribute to our top lines. Each of these launches are progressing well. I'll now hand it off to our Chief Commercial Officer, Eric Harris, to give us some additional details on his team's recent performance.