Ultragenyx Pharmaceutical Inc.

Good afternoon and welcome to the Ultragenyx Third 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 Inresa Relations.

Thank you. We've 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 filings. I'll now turn the call over to Emil.

Thanks, Josh, and good afternoon, everyone. Today, Ultragenyx is on the cusp of significant evolution and growth. We have four commercial products that have delivered consistent and substantial double-digit annual revenue growth over many years. We have two Bill 8 submissions in progress for programs that are poised to address significant medical needs for patients with ultra-rare diseases. We also have multiple late-stage clinical programs with transformative commercial potential that are approaching pivotal data readouts. We're at a defining moment for the company, and I'm pleased to report that our team is ready to maximize the opportunities ahead. We announced earlier today that we took an important step to strengthen our financial position, receiving $400 million of non-dilutive capital from OMERS through the capped sale of a portion of our CRISPR-B2 royalties. Importantly, we were able to defer the start of payments under this financing until January 2028. These funds and this timing bolster our balance sheet as we approach pivotal data readouts for our most significant commercial opportunities in osteogenesis imperfecta and ageism syndrome. Importantly, we'll continue to focus on managing our cash burn and prioritizing our investments. Shifting to clinical, We continue to see exciting momentum across our late-stage programs, beginning with GTX1 and 2, our investigational antisense oligonucleotide for Angelman syndrome. In July, we announced the pivotal 48-week ISPAR study, completed enrollment with 129 patients, and is expected to read out data in the second half of 2026. Last week, we announced the first patient had been dosed in the Phase 2-3 Aurora study, which evaluates GTX102 in additional ages and genotypes. This study, along with the fully enrolled phase three SPAR study, will generate data across the spectrum of genotypes and ages. Turning to UX143 for the treatment of osteogenesis imperfecta, the conduct of the phase three ORBIT and COSMIC study continues to go well. We hear stories from investigators who have patients in the open-label phase two about how well their patients are doing, the improvements in their bone density, and the profound effect this drug is having on their lives. Data from the Phase III studies are on track to read out around the end of the year, which to us means December or January. As we move into the final analyses, we remain confident that truzumab is a mechanism of action, its ability to make more bone in the places that need more strength, which should reduce fractures. If successful, this will lead to a transformational treatment for pediatric and adult patients with osteogenesis imperfecta. For our existing approved products, our global commercial organization continues to deliver meaningful revenue and cash flow every year. This year, they are on track to deliver total revenue between 640 and 670 million, which would be 14 to 20% growth from last year. Chris Vita is the largest product in the portfolio, and we expect revenue to continue growing in the US, Canada, Latin America, and Turkey as more and more patients initiate this important medicine. Toljovi, FKISA, Mepsevi also meaningfully contribute to our financial base and provide a steady, diversified source of revenue that's also expected to grow over time. I'll now turn the call over to Eric Harris to share more details on his team's efforts last quarter.