Ultragenyx Pharmaceutical Inc.

Good afternoon and welcome to the Ultragenyx third quarter 2023 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, Vice President of Investor Relations.

Thank you. We've issued a press release detailing our financial results, which you can find on our website at ultragenics.com. Joining me on this call are Emil Kakas, Chief Executive Officer and President, Eric Harris, Chief Commercial Officer, Howard Horn, Chief Financial Officer, Eric Krambes, Chief Medical Officer, Aaron Olson, Senior Vice President of Corporate Strategy and Finance, and Ted Huizenga, Chief Accounting Officer. I'd like to remind everyone that during today's call, we'll 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 Abel.

Thanks, Josh, and good afternoon, everyone. Before we get started, I'd like to welcome our new CFO, Howard Horn, to the call. To say he has hit the ground running is an understatement, He already has an Ultragenetics Analyst Day and a financing under his belt. We're happy to have him on board as we prepare for our Catalyst Rich 2024. It was great to see many of you at our Analyst Day a few weeks ago. We hope that the new data shared at the event, along with the positive clinical impressions of the investigators on two of our programs, showed you why we have confidence in our lead therapeutic candidates, in their potential to transform debilitating disease with limited or no treatment options. We kicked off the day by sharing new data from the ORBIT study for citruzumab in osteogenesis imperfecta. It has been prevented a few days earlier at ASBMR, the American Society for Bone Mineral Research, 2023 annual meeting. Interim data from the Phase II portion of the study demonstrated that citruzumab significantly reduced the analyzed fracture rate by 67%. after at least six months of treatment. It continued to demonstrate ongoing substantial increases in lumbar spine bone marrow density, reaching 19% in just six months in the younger patients. We also provided an update on our GTX102 program in age and syndrome. The data we covered was longer-term extension data from the dose escalation cohorts in the Phase 1-2 study. And it showed the treatment with GTX1-2 resulted in clinically meaningful improvements across multiple domains through both the loading and maintenance phase of treatment. The improvements we saw in Bayley and the age and severity assessments, formerly known as the CGI-S severity scale, were also supported by objective changes in EEG and comparisons to natural history. We also looked at the emerging first-ever development changes from three of the original U.S. patients. When these patients stopped receiving GTX1 and 2, they lost these first-ever developmental gains, but were able to gain these skills once they started treatment with new dose and regimen. Other patients also saw many first-ever development gains, which gives us confidence in the potential transformative effect for this neurodevelopmental disease. These first errors are very important to the families and give clinical meaningful to the changes we are seeing in the quantitative assessment. The third program we highlighted was our UX701 AAV gene therapy for Wilson disease. We provided data on the five patients in the first lowest dose cohort. At 5E12 GC per kilo, four of the five patients showed improvements in copper trafficking and had begun tapering off standard of care with key layers and or zinc therapy. This includes two of the earlier treated patients who are now completely off standard of care and doing well. Eric Kramiz will provide more detailed updates on these programs later in the call, but it's clear we have three large value programs all generating data that meaningfully de-risk the probabilities of their success. Now I'll turn the call over to Eric Harris to provide an update on our commercial efforts for the first quarter.