Ultragenyx Pharmaceutical Inc at Morgan Stanley Global Healthcare Conference Summary

The following is excerpted from the question-and-answer section of the transcript.

Question: Jeff Hung - Morgan Stanley - Analyst : Great. Let's start with setrusumab. Can you just talk about what you saw on the Phase 2 portion that gives you confidence in the program?

Question: Jeff Hung - Morgan Stanley - Analyst : And you said that more detailed 14-month data will be presented at a future medical meeting. What other aspects of the data should we expect to see or look out for?

Question: Jeff Hung - Morgan Stanley - Analyst : And for the Phase 3 Orbit study, how should we be thinking about the fracture rate reduction that we've seen in the Phase 2 translating to the Phase 3 and what will be clinically meaningful?

Question: Jeff Hung - Morgan Stanley - Analyst : The setrusumab is also being explored in an open-label Phase 3 Cosmic study in pediatric patients, randomized setrusumab or bisphosphonates. Can you just remind us what the bar is in terms of an existing treatment effect achieved by bisphosphonates?

Question: Jeff Hung - Morgan Stanley - Analyst : Now there are two interim analyses plan starting by year-end or early next year. Can you just remind us of what the analysis consists of and what might be formally announced?

Question: Jeff Hung - Morgan Stanley - Analyst : Great. Let's shift to GTX-102 for Angelman Syndrome. You recently had a successful end of Phase 2 meeting. Can you provide some highlights on the data that you presented to the FDA? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 04, 2024 / 6:35PM, RARE.OQ - Ultragenyx Pharmaceutical Inc at Morgan Stanley Global Healthcare Conference

Question: Jeff Hung - Morgan Stanley - Analyst : On what aspects of the data do patients and caregivers find most meaningful?

Question: Jeff Hung - Morgan Stanley - Analyst : Okay. Now Ionis announced results recently from its program. How do you view that data? And are the differences between the two programs, more a function of the difference between LNA versus MOE chemistry?

Question: Jeff Hung - Morgan Stanley - Analyst : Now for the open label AURORA study, do you expect any differences in the way that GTX-102 functions in patients with other genotypes outside of deletion mutation? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 04, 2024 / 6:35PM, RARE.OQ - Ultragenyx Pharmaceutical Inc at Morgan Stanley Global Healthcare Conference

Question: Jeff Hung - Morgan Stanley - Analyst : Any updated thoughts on whether the loading doses will be reduced from four to 3?

Question: Jeff Hung - Morgan Stanley - Analyst : Okay. So for UX701 in Wilson disease, you plan to report data from Stage 1. What should we expect to see and how many patients will be included in that readout?

Question: Jeff Hung - Morgan Stanley - Analyst : And so what are the main considerations for dose selection? As you go higher in dose, what kinds of potential safety concerns might arise? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 04, 2024 / 6:35PM, RARE.OQ - Ultragenyx Pharmaceutical Inc at Morgan Stanley Global Healthcare Conference

Question: Jeff Hung - Morgan Stanley - Analyst : And what is your current thinking on the design of Stage II? I guess besides the placebo arm and the selected dose, are there other adjustments that you might make from Stage I?

Question: Jeff Hung - Morgan Stanley - Analyst : Great. Maybe a couple of questions on UX111. Can you just remind us of the data that you've seen and the correlations between CSF heparan sulfate and cognitive function? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 04, 2024 / 6:35PM, RARE.OQ - Ultragenyx Pharmaceutical Inc at Morgan Stanley Global Healthcare Conference

Question: Jeff Hung - Morgan Stanley - Analyst : Now on the BLA, can you just share whether you've met with the FDA yet for the pre-BLA meeting and any feedback that you received? And is there anything that outstanding that's required for the BLA filing later this year or early next year?

Question: Jeff Hung - Morgan Stanley - Analyst : Great. Maybe some questions on some of your other programs. I guess for DTX401, you've had positive top line results. What should we expect to see -- like following the positive topline results, what should we expect to see in the full 48-week data that was not revealed? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 04, 2024 / 6:35PM, RARE.OQ - Ultragenyx Pharmaceutical Inc at Morgan Stanley Global Healthcare Conference

Question: Jeff Hung - Morgan Stanley - Analyst : Great. So for Crysvita, can you just talk about the progress in Latin America? And how should we think about the geography over the next couple of years?

Question: Jeff Hung - Morgan Stanley - Analyst : Great. Maybe one last question. What excites you most about the other early-stage pipeline programs?

Question: Jeff Hung - Morgan Stanley - Analyst : Great. Thank you so much for your time, Emil.