Ultragenyx Pharmaceutical Inc at JPMorgan Healthcare Conference Transcript

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

Question: Anupam Rama - JPMorgan - Analyst : Thanks Emel. You want to introduce who's on stage?

Question: Anupam Rama - JPMorgan - Analyst : Just want to remind.

Question: Anupam Rama - JPMorgan - Analyst : I just want to remind everybody that there's kind of three ways to ask your question, right? Old school, raise your hand. I'll call on you. You can submit a question to the portal and or you can just email me and I can ask on your behalf. But I guess we all start. There's this thesis out there now that you've gone past the first interim to the second interim that somehow the probability of technical success is now different for the orbit study. Do you agree with this or would you push back on that?

Question: Anupam Rama - JPMorgan - Analyst : Oh Yeah. Question in the audience. Unidentified Participant Yes. So you know, there's a lot of concern about the scalability. No why do that? Companying rare disease. What is your idea? Or.

Question: Anupam Rama - JPMorgan - Analyst : Go ahead. Question in the audience. Unidentified Participant Can you elaborate on some of your reimbursement solutions? Explor?

Question: Anupam Rama - JPMorgan - Analyst : We have an email question here which is how are you notified by the data Monitoring committee that you, that you're moving to the second interim? I thought basically, the question is I thought you had communicated that you would only be notified if you hit the interim but not if you did not.

Question: Anupam Rama - JPMorgan - Analyst : Question from the audience. Right. Yeah. Unidentified Participant Yeah. Related to dose delivery, single injection therapies. On the medical device side, is there any innovation or additional care that you're doing to ensure dose delivery? Is there any thing that you're doing? That's proprietary. The innovation side.

Question: Anupam Rama - JPMorgan - Analyst : We have a couple of portal questions here, which is, can you remind us of what percent fracture reduction you need to see in the placebo on the place on a placebo adjusted basis to hit stats in interim analysis too? And do you plan on sharing the baseline characteristics for patients in orbit prior to interim analysis too?

Question: Anupam Rama - JPMorgan - Analyst : Questions from the audience.

Question: Anupam Rama - JPMorgan - Analyst : Maybe more of a medical you know, a market research type question from the portal which is does the location and type of fracture pattern for type one and type 20 I patients differ.

Question: Anupam Rama - JPMorgan - Analyst : What this is another sort of market research question. What do you, what is the feedback basically on bisphosphate in any treatment in O I? And how could CTU map be a paradigm shift?

Question: Anupam Rama - JPMorgan - Analyst : All right. Oh, go ahead. Great. Unidentified Participant Thank you. Asteroid had a great spine data. Can you hit with this trial with spine and maybe not as powerful on the long bones to be amphib? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. JANUARY 13, 2025 / 6:30PM, RARE.OQ - Ultragenyx Pharmaceutical Inc at JPMorgan Healthcare Conference

Question: Anupam Rama - JPMorgan - Analyst : All right guys, we're up on time. Thanks everyone.