Ultragenyx Pharmaceutical Inc.

Good afternoon, and welcome to the Ultragenyx fourth quarter and full year 2022 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 the opportunity to ask questions during the Q&A portion of the call. It is now my pleasure to turn the call over to Josh Ahiga, Executive Director 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 Ultragenyx.com. Joining me on this call are Emil Kakas, Chief Executive Officer and President, Eric Harris, Chief Commercial Officer, Camille Bedrosian, 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. Some of you may have listened in on my presentation at the recent JPMorgan Healthcare Conference where I talked about our focus for areas for 2023. Over the last year, we completed several strategic investments aligned our teams and resources around the highest value-generating programs for the company. This year, we'll continue to focus on operational efficiency while generating data from our key clinical programs in Angerman, osteogenesis imperfecta, and our pivotal gene therapy studies. In 2022, we generated more than $350 million of global revenue in our fifth year as a commercial company. This year, we expect revenue to be in the $420 million to $450 million range, from our four products across five indications. We expect a steady revenue growth to continue as the existing products are still in relatively early phases of commercialization. At the same time, the investments made over the last few years have positioned us to drive meaningful value from large opportunity programs like GTX 102 for Angemin and UX 143 for Ausgenis and Perfecta, and we have more recently bolstered our business with additional strategic steps Specifically, we secured a license and collaboration agreement to commercialize Avkisa for clinical homozygous familial hypercholesterolemia in countries outside of the U.S., which will leverage our global commercial infrastructure and capabilities. We acquired global rise to UX111 in AAV gene therapy for the treatment of Sanfilippo syndrome from Aviona, based on the strength of the pivotal marker data and clinical data generated in the TransferA study. We also excised our option to acquire GeneTx following interim data from the Phase 1-2 of GTX102 for the treatment of ANG1 syndrome. The GeneTx acquisition gives us full control over that program. And we completed the build-out of our gene therapy manufacturing plant in Bedford, Massachusetts, which is expected to begin production this spring. This facility and our manufacturing capabilities grant us the ability to better control the timing, cost, and scale of the production of our gene therapies, establishing us as a commercial-ready gene therapy company. Within our pipeline, we made strong progress over the past year in addition to progressing our engagement program. We initiated a pivotal Phase II-III program for UX143 and OI and have completed enrollment of the Phase II portion of the study. We advanced DTX401 into a pivotal Phase III study for GSD1A, and have enrolled the last patient into the baseline screening period. We also initiated a phase three study of DTX301 in OTC where we have randomized and dosed the first patient in this study with additional patients in the baseline screening period. And we initiated and dosed patients in the pivotal seamless study of UX701 for Wilson disease. As a commercial company with growing revenue and a diverse late stage clinical pipeline, we recently evaluated ways to improve our operating efficiency. We will focus on leveraging our established global infrastructure, actively managing expenses and headcount to drive value from the commercial launches and execute on our most important clinical programs. With that, I'll turn the call over to Eric to provide more specifics on the commercial programs and their successes last year.