Ultragenyx Pharmaceutical Inc.

Good afternoon and welcome to the Ultragenyx Second Quarter 2026 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, Chief of Staff and 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 Kakkis, Chief Executive Officer and President, Howard Horn, Chief Financial Officer, Erik Harris, Chief Commercial Officer, and Erik Crombez, 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 second quarter, we continued our pattern of strong execution across the development and commercial organizations. The commercial teams delivered the highest quarterly revenue in the history of the company, which supports our reaffirmed full-year revenue guidance. As they have done in prior quarters, they continue to find new patients and expand access to Chris Vita, Bill Jolby, Evkisa, and Mepsevi around the world. The commercial and field teams are also preparing for our first two potential gene therapy launches, which are anticipated in the coming months. If approved, both these therapies will represent first-ever treatments for disease with significant unmet needs and high urgency to treat. The PDUPA date for DTX401 for GSD1A is just a few weeks away. Patients with GSD1A have to drink a flurry of corn starch every few hours around the clock, day and night, knowing that a single missed dose could lead to their death. This is a constant reminder of their disease and the severe consequence of missing a dose. In our Phase 1-2 and Phase 3 studies, patients treated with DTX401 have been able to significantly reduce the amount and frequency of corn starch doses to a pre-specified clinically meaningful degree. More importantly, this gene therapy allows patients to have a more normal glucose metabolism, staying in the normal range a larger fraction of the day. The expression of the missing G6PAs enzyme from the DTX401 vector should allow their liver to break down glycogen to produce glucose during times of fasting or metabolic stress. The ability to regulate glucose better has reduced the burden of disease and reduced the potential for rapid decline to dangerous glucose levels. Transitioning now to UX111, I've been in and around the NPS community for many years, and I've seen so many San Felipe patients have to watch helplessly as their children decline and die. San Francisco syndrome type A has an urgent need to treat, and yet there has been nothing for them. San Francisco syndrome is a horrible neurodegenerative disease, and kids are losing brain cells every day. Patients between the age of two and six years progressively lose their cognitive function. By 10 years old or so, they're often bedridden and tube fed. and in and out of the hospital for many years. They can end up non-responsive for the last five or ten years and often die as teenagers. Until now, families have had no option but to watch helplessly as their children decline and die. Based on our clinical data, treatment with UX111 reduces heparin sulfate in these patients and enables them to stabilize and retain cognitive function compared to natural history. While patients across the age range in the study showed benefits following treatment with UX-111, it's also clear that treating earlier demonstrated better results by protecting their brains before they had lost too much function. With a disease like Sample Epilepsy Syndrome, there's no greater urgency to treat, as the longer you wait, the less function you may have. Our development organization under Dr. Crombez has been working with regulatory agencies around the world on BLA submissions for both DTX401 for GSD1A and UX111 for Sanfilippo-Ain syndrome. These include preparing the applications, responding to information requests, and supporting facility inspections. This would be a lot for a single BLA. Our team is working simultaneously through the process for two BLAs that have Paducah dates a month apart. We remain confident in the work our team is doing to support these applications. Shifting to GTX1 and 2 for Angelin Syndrome, where we have the most advanced clinical ASO program. In the Phase 3 ASPIRE study, the last patient in has had their 48-week visit, and the team is in the process of cleaning and locking the database. For a global study like this, the process can take months, and we expect to unblind and share top-line results with you in September or October timeframe. With three major catalysts on the horizon, the second half of 2026 is poised to be the most significant period in our company's history. We're positioned to deliver another exciting series of potential firsts for our company, the communities we serve, and the broader field of rare disease medicine, including the first gene therapy approvals for all genetics, the first FDA approvals for two devastating and intractable rare diseases, and the first late-stage clinical data in Angelman syndrome. It's a privilege to continue to lead the future of rare disease medicine and we believe the combination of continued growth from our current products accelerated by contribution from potential upcoming launches and disciplined expense management gives us a clear path to profitability in 2027. I'll now turn the call over to our Chief Commercial Officer, Eric Harris, who will provide details on the commercial business and launch of its activities in the second quarter.