Ultragenyx Pharmaceutical Inc.

Good afternoon and welcome to Ultragenyx first quarter 2026 financial results conference call. At this time, all participants are on 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 introduce your host, 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 Kronbez, 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. We are now in our 16th year since our founding, and this year is expected to be transformative with growing revenue and multiple new drug approvals. We're on track to well exceed $700 million in revenue from our global commercial business with a consistent track record of double-digit annual revenue growth. We have a baduka date for two gene therapies that would bring first-ever treatments to patients and families with no other disease-monifying options. Also, we'll unwind our phase three ASPIRE study, evaluating GTX102 in patients with Angelman syndrome in the second half of this year. We continue to execute global clinical trials across the largest late-stage pipeline in rare diseases. We're also manufacturing our gene therapy products in our new facility in Bedford, Massachusetts. I'll start with GTX102 for Angelman syndrome. The first patients enrolled in the Phase III Aspar study have reached their day 338 visit and have transitioned to open-label extension study. The rest of the patients will be completing the blinded portion of the study in the next few months and crossing over to open-label extension. With the Aurora study, we're expanding GTX102 treatment to other ages and genetic types of Einstein syndrome, and enrollment in that study continues to go well. We prefer to develop the first-ever treatments for diseases that have not had significant breakthroughs in the past, and so do not typically work in disease areas where there are other competitor programs at similar stages of development. In the case of Einstein syndrome, we made an exception given the size indication and the excellent work genetics had done with the scientist Scott Dindo to develop a potent ASO that worked in a large animal model rather than just mice, which often do not predict human biology. The preclinical research Scott conducted in his lab at Texas A&M is a tour de force in molecular genetics. He was able to identify and target a separate and distinct region of the Anderson's message transcript that has led to more efficient transcript knockdown and greater potency in the clinic. This was the peer science that led us to study Angevin syndrome. Later in the call, Dr. Promes will walk through the longer-term efficacy, durability, and safety data from the Phase I-II study in his section. But I want to highlight now that we have 66 patients on therapy for an average of three years and with the longest approaching five years, with continuing and improving benefits across multiple domains and with a favorable safety profile. Based on this longer-term data, we believe GTX1 and 2 can deliver clinically meaningful treatment effects and can be safely administered in chronic dosing. What we see in the Phase 1 and 2 data, we believe GTX1 and 2 is making an important difference in the lives of these patients and their families. Shifting now to our global commercial efforts, our commercial business continues to deliver. We're now generating revenue in more than 35 countries, a result of strategically investing in high-quality teams who can efficiently navigate the complex approval and reimbursing processes around the world. This reflects a disciplined country-by-country execution our teams deliver every quarter. This base business is not only generating meaningful and growing revenue, it is the engine that will power our next phase of growth. Our teams currently commercializing Dolgioli and Metsevi are poised to add DTX 401, and US 111 to their responsibilities as we look forward to approvals for these two products later this year. Our established global commercial business is bringing first-ever treatments to patients who need them, paving the path to profitability in 2027. I'll now turn the call over to our Chief Commercial Officer, Eric Harris, who will provide details on his team's efforts in the first quarter.