Ultragenyx Pharmaceutical Inc at Evercore ISI HealthCONx Conference Transcript

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

Question: Liisa Bayko - Evercore ISI - Analyst : Can you be profitable, let's say, just with the base business, if you were to do nothing else?

Question: Liisa Bayko - Evercore ISI - Analyst : Approved. I was going to get to that next. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 04, 2024 / 2:10PM, RARE.OQ - Ultragenyx Pharmaceutical Inc at Evercore ISI HealthCONx Conference

Question: Liisa Bayko - Evercore ISI - Analyst : Well, that's why there's so much excitement, I think, on the pipeline. It's OI, Wilsons, Angelman, those are kind of the big, I think, targets are just very sizable diseases with big unmet medical needs that you could possibly address Okay. So I want to turn to some of those programs. There's just so much interest in those. So for OI, walk us just through the rationale for setrusumab and OI.

Question: Liisa Bayko - Evercore ISI - Analyst : When you say much better than bisphosphonates, what are bisphosphonates doing that's different?

Question: Liisa Bayko - Evercore ISI - Analyst : We get this question a lot from kind of like the pushback to the story is like, okay, you're making more bone. But if the bone is not good quality bone because you still have the collagen problem, is that really helping the issue? Do I just have more bad bone? And even though my bone mineral density has improved, is it really stronger bone? So that -- like what's your response?

Question: Liisa Bayko - Evercore ISI - Analyst : Right. Well, there's always the question of there wasn't a placebo, so what's going in the background. So that actually brings my next question. What are you thinking about as sort of the background rate? Should we think about sort of the -- how patients did before they came into the study as the sort of what would happen on sort of placebo and a (inaudible). What about the impact of being on a trial and all those kind of like things that happen? Maybe you're more careful, maybe you're getting better care, these kinds of things.

Question: Liisa Bayko - Evercore ISI - Analyst : We've been getting a lot of questions and Josh has been fielding a lot of them. What to expect for placebo, right? Because that's the biggest unknown, because we didn't have a placebo in Phase 2. And I think we can -- we have a decent sense of drug because we saw some pretty impressive data in Phase 2. So -- and you've talked a little bit about sort of -- can you talk through, I guess, your expectations for placebo and where is that derived from?

Question: Liisa Bayko - Evercore ISI - Analyst : And you did some interesting things in the trial, you sort of changed the end, reduce the size, you actually added these sort of interim analyses. What was the impetus for that?

Question: Liisa Bayko - Evercore ISI - Analyst : I like it. Okay. Just one more point on this that I want to ask about. And we've been trying to do a lot of digging on the connection between bone mineral density and fracture rate in the OI population specifically. We know it's there for osteoporosis and other things. But what is the evidence for that in this disease? Because actually, the bone mineral density change is very compelling. And the question is, is that predictive in some way in this particular population.

Question: Liisa Bayko - Evercore ISI - Analyst : Okay. I want to hit briefly on the other two programs. We don't have a lot of time but Wilson's disease, what is the data there so far suggest to you?

Question: Liisa Bayko - Evercore ISI - Analyst : So when will we see that data?

Question: Liisa Bayko - Evercore ISI - Analyst : Okay. So maybe it's a 2026 thing or --

Question: Liisa Bayko - Evercore ISI - Analyst : Okay. And what level of -- I guess, what level of copper, I guess, control do you want to see? Or how should we think about?

Question: Liisa Bayko - Evercore ISI - Analyst : That makes sense. Okay. And then just quickly on Angelman, you're just getting kind of your Phase 3 up and running. You're focused on cognition, and we know there's one other antisense out there that's focusing on expressive communication. There's some hierarchy here. Actually, I was talking about that with Josh last couple of days.

Question: Liisa Bayko - Evercore ISI - Analyst : It's always hard because companies are always using different end points. And we always like to compare and contrast, makes it hard whenever --

Question: Liisa Bayko - Evercore ISI - Analyst : Okay. And then just on a final point, I know we're out of time, but can you just give us a view on 2025? What are the key events, catalysts that we can look forward to?

Question: Liisa Bayko - Evercore ISI - Analyst : Okay. Great. Thank you.