Ultragenyx Pharmaceutical Inc. (RARE) Q4 2025 Earnings Call

Investor webinar replay

Replay transcript excerpt

Good afternoon, and welcome to the Ultragenyx fourth quarter and full year 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 Investor 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, Howard Horn, Chief Financial Officer, Eric Harris, Chief Commercial Officer, and Eric Cromdes, 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. 2026 is poised to be a significant year for the company as we reach key inflection points across multiple programs. This includes two potential approvals in MPS3A or Sanfilippo type A syndrome and glycogen storage disease type 1A, and a pivotal data readout in Angerman syndrome. These programs are excellent examples of our mission to bring important first-ever treatments to patients and families while also delivering meaningful long-term value to shareholders. Just last week, we presented updated data at the World Symposium from the UX111 for MPS3A program. The new data reflect an additional year of follow-up and continue to demonstrate sustained and significant further separation of early treated patients in multiple ...