Ultragenyx Pharmaceutical Inc.

Good afternoon and welcome to the Ultragenyx first 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 and Head of Investor Relations.

Thank you. We have 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, 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 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 first part of the year, we made meaningful progress for generating data from our key clinical programs. The Phase II portion of the UX143 Pivotal Study for Osteogenesis Imperfecta is fully enrolled, and the team has begun analyzing the data in advance of the data release in mid-2023. Around that same time, we expect to begin dosing patients in the randomized placebo-controlled Phase III portion of the study. Based on the KOL feedback, we believe that Phase III should enroll swiftly and move us that much closer to a potential therapy for patients with this bone disease. Similarly, with GTX102 Phase I-II for angiogen, we have begun XUS dosing in the expansion cohorts. Outside the U.S., we've seen positive feedback on our protocol and are activating multiple sites in Australia and across Europe. This broadens the number of sites who have experience with GTX102 to support conduct of the Phase 1-2 and can participate in a future Phase 3 study. In the U.S., we've had productive discussion with the FDA and look forward to possibly expanding dosing in the U.S. On the commercial side of the business, we continue to grow revenue and make progress with expanding access to our therapies around the world. In the first quarter of 2023, CRISFIDA revenue in Latin America grew 120% to $20 million compared to $9 million the prior year. Five years into launch, there are still meaningful opportunities for significant growth. We recently began the next chapter for CRISFIDA in North America with Keogh Karen assuming primary commercialization responsibilities for the program in the U.S. and Canada last week. We will continue our joint commercial efforts in the U.S. through April 2024. And beyond that, we'll continue promoting this blockbuster therapy to medical geneticists. In the span of 10 years, Chris Vita has moved through late stage development with approvals in two rare genetic bone disorders and reached thousands of patients around the world. I want to acknowledge the work and dedication of our integrated Keohokalani and Yeltsin Genetics teams. in their commitment to the patient community and therefore their most recent efforts in planning for and achieving a smooth transition with patient experience as the top priority. I also want to briefly comment on the recent remarks made by Peter Marks proposing an Operation Warp Speed type approach to ultra-rare disease. This year marks the 40th anniversary of the Orphan Drug Act. While it has been instrumental to progress in treating rare diseases, is left ultra rare diseases behind. We have an opportunity right now to make progress across many life-threatening diseases afflicting infants and children for which there are currently no treatments, to offer hope and to pass forward to the parents and families of these children. I'm awfully supportive, very supportive of the agency providing a clear framework that makes the Accelerated Approval Pathway accessible to therapies in ultra rare genetic diseases, and I'm hopeful that the agency will work with the urgency required. Too many small companies with limited resources have had to halt promising programs. We greatly fear that we may lose the majority of these new treatments for ultra-rare diseases. I'm encouraged that the agency is showing receptivity and listening to the community. Before I turn the call over to Eric Harris, I'd like to welcome Eric Krambas to the call in his new position as Chief Medical Officer and Executive Vice President. Eric has been a driving force on our gene therapy programs and has extensive experience with rare metabolic disorders. He and Camille have worked closely together for over five years on advancing our clinical programs, and it has been a seamless transition as she steps into her new role as a full-time strategic advisor. I also want to thank Camille for her passion and dedication to both ultragenics and the rare disease community. She's been instrumental in the development of our clinical pipeline, and I'm grateful for her ongoing partnership going forward. Now I'll turn the call over to Eric Harris to provide an update on our commercial efforts for the quarter.