Ultragenyx Pharmaceutical Inc.

Welcome to the Ultragenyx First Quarter 2022 Financial Results and Corporate Update Conference Call. At this time, all participants are in a listen-only mode. After the speaker presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1 on your telephone. Please be advised that today's conference is being recorded. If you'd like to have assistance during the conference, please press star 0. I would now like to hand the conference over to your speaker today, Joshua Higa.

Good afternoon, and welcome to the Ultragenyx pharmaceutical financial results, and corporate update conference calls for the first quarter of 2022. We have issued a press release detailing our financial results, which you can find on our website at ultragenics.com. I am Joshua Higa, Director of Investor Relations. Joining me on this call are Emil Kakas, Chief Executive Officer and President, Eric Harris, Chief Commercial Officer, Marty Deer, Chief Financial Officer, and Camille Bedrosian, Chief Medical Officer. I would 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 quarter, we continued to make progress across our diverse clinical and commercial programs. In the late stage clinical pipeline, we're now enrolling patients in three registrational studies with a fourth to initiate later this year. This includes the three gene therapy programs for glycogen store disease type 1A, Wilson disease, and ornithine transcarbamylase deficiency, as well as our anti-sclerostin antibody for osteogenesis imperfecta. The GTX102 study for Angelman's syndrome is progressing well and we remain confident in the program. The results we shared in 2020 from the original five patients led us to reimagine what is possible for patients with Angelman syndrome. Given the importance of this program, we've looked at various ways to accelerate this program. And recently, we amended our agreement with genetics to allow us an additional option to acquire them at an earlier time point based on interim data. I'll let Camille share more in her section about the favorable safety profile and enrollment status across the region. We also look forward to providing a more robust interim update in mid-2022. On the commercial side, the teams continue developing and executing their plans to define more patients who could benefit from CRIS-Vita, Dojolvi, and Mepcevi. These efforts are supported by the work our clinical and regulatory teams are doing with country-specific authorities to enable greater access to these therapies. In the first quarter, we also closed on the strategic partnership with Regeneron, which pairs our expertise in global rare disease launches with the industry leader in antibody drug discovery and development. We'll leverage our global commercial medical affairs regulatory functions to bring, as Kiesa, a novel, high-potent, and approved antibody to patients where the current standard of care does not adequately reduce the LDL cholesterol in patients with HFH. Since closing the deal, we transferred the marketing authorization in Europe and have begun the process to submit reimbursement dossiers. While this review process and negotiations can take time, we will leverage the team that currently supports MEPSAV and Dojovi to respond to requests for named patient access within Europe. Feedback from the KOL community has been enthusiastic for the important role that Keith could play in the management of familial hypercholesterolemia. The PISA gives us a fourth product across five different indications that will be generating revenue for the company. This creates a diversified base of value that will help support our continued clinical execution for years to come. I'll let the team go into more detail on their progress in the quarter. Eric, can you begin?