Ultragenyx Pharmaceutical Inc Q4 2024 Earnings Call Transcript

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

Question: Tazeen Ahmad - BofA Global Research - Analyst : Hi, guys. Thanks for taking my question. So mine is going to be on OI and how we should be thinking -- I think you've talked about this many times, but Emil, if you could give some color about your confidence in the second interim read being sufficient to stop the study versus needing to go to the third interim? I know you've expressed overall confidence in the trial design and the likelihood of success. But we'd like to hear your thoughts about how you're thinking about the importance of the study being stopped at the second interim. Thanks.

Question: Salveen Ritcher - Goldman Sachs - Analyst : Good afternoon. Thanks for taking my question. Just a follow-up to the first one here. Maybe help us understand if it doesn't hit on the second interim? What would those reasons be or what are the risks to that? And then how long would we have to wait for the final analysis? Thank you.

Question: Gena Wang - Barclays - Analyst : Thank you. Sorry, I will ask another question. Given the study complete enrollment on May 1, 2024, is it fair to assume June/July timeframe, you should share the second interim update? And then how would you communicate with the investor in the case this study didn't hit a step? And in the case, the study hits steps would you need to take extra time to analyze the data and therefore share the update a little later?

Question: Gena Wang - Barclays - Analyst : Yeah. So if you hit that means like it would share with us a little bit later since you will need to take a few more weeks to analyze the data?

Question: Gena Wang - Barclays - Analyst : Okay, great. Thank you. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 13, 2025 / 10:00PM, RARE.OQ - Q4 2024 Ultragenyx Pharmaceutical Inc Earnings Call

Question: Yaron Werber - TD Cowen - Analyst : Hi, thank you. Right. So I also, shockingly, we have a 143 question. In the Orbit study, are you stratifying just remind us type 1, 3, and 4 between the two arms? And then secondly, when you look at the primary fracture rates, do you have a secondary looking fracture rates by type underlying type? Thank you.

Question: Joon Lee - Truist Securities - Analyst : Thanks for taking our questions. If the OI study goes to completion in 4Q, does that implies that the magnitude of the set may not be as great as expected? And in that case, how competitive would setrusumab being compared to bisphosphonate?

Question: Jeff Hung - Morgan Stanley - Analyst : Thanks for taking my question. I just wanted to clarify, make sure I understood correctly. You talked about how the Phase 2 data and the 0.014. But just firstly, for situation with the Orbit Phase 2 portion have hit with the second interim analysis criteria, and if not, how were the baseline fracture rates different from the Phase 2 portion? Thanks.

Question: Jeff Hung - Morgan Stanley - Analyst : Yes.

Question: Ric Miller - Cantor Fitzgerald - Analyst : Hey, everyone. This is [Ric Miller] on for Kristen. Thanks for taking our question. We're going to mix it up a little bit and ask about OTC deficiency. So just to better understand the amended protocol you talked about today. When the patients in the Phase 3 are in the initial 36-week blinded period, are they still eligible for titrating down on ammonia damages and diet control or would that just be an option in the following unblinded period? And then based on the natural history for the disease, is there any good understanding that you have based on what a meaningful reduction in ammonia looks like and how this correlates with normalizing some of the clinical effects you see? Thanks.

Question: Luca Issi - RBC Capital Markets - Analyst : Great. Thanks so much for taking our questions. I guess we'll revert back to the mean here and maybe ask a question on OI. What's the latest thinking on duration of therapy assuming that the trial is successful. We think that the FDA will cap the duration of therapy to 12 months as they have done for romosozumab in osteoporosis? Or do you think this will be a chronic dosing with no cap. So that's one. And then second, very quickly, can you just remind us why for Angelman running a sham-controlled trial is the better way to go versus a placebo-controlled trial? Thanks so much.

Question: Luca Issi - RBC Capital Markets - Analyst : That second question on Angelman.

Question: Luca Issi - RBC Capital Markets - Analyst : Super helpful. Thanks so much.

Question: Liisa Bayko - Evercore ISI - Analyst : Hi. Thanks for taking the question. A question on the variability in OI. In your work preparing for the trial and assumptions, can you talk about any sort of registry or database views you've got on variability? Is there some way to describe that? And I guess, is there a trend like if a patient has a fracture, are they more or less prone to a fracture in a certain period of time? I just would love some color around that. Thank you so much. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 13, 2025 / 10:00PM, RARE.OQ - Q4 2024 Ultragenyx Pharmaceutical Inc Earnings Call

Question: Yaron Werber - TD Cowen - Analyst : Yeah. Thanks for taking my follow-up. I just had a couple, Emil. I know the first one on 401. So given that you're fully enrolled now and the primary is at 36 weeks, and then you unblind, is there a chance that we'll get the data, the first sort of the primaries this year? And then secondly, I think I misheard, but I just want to double check. It sounds like you're saying in Cosmic, you'll do an interim analysis now midyear as well. Is that indeed -- that's going to be the first interim for that? And I think you mentioned it 12 months, but with a 0.01 alpha, but I thought that was referring to Orbit. So I just want to double check the interim analysis on Cosmic.

Question: Yaron Werber - TD Cowen - Analyst : Okay, thanks.