Ultragenyx Pharmaceutical Inc at Citi Global Healthcare Conference Summary

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

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : <_ALACRA_META_ABSTRACT>So well, maybe we can just start with maybe a question that you don't usually get from the analysts, which is why did you found the company? What was your thinking back in 2011 or whenever it was originally started? You were BioMarin. So tell us why you felt we needed another a new rare disease company, and what are you going to do differently?

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : Okay. That's a good intro. So you mentioned OI, so maybe we can start on that one since there's been a lot of questions there. The first of 2 interims is coming with end of this year or early next year. So just to give a bit of background, what's the standard of care. You have the 2 Phase III trials, Orbit and Cosmic. What are the goals of each of those? Maybe just a quick on the mechanism on anti-sclerostin and then we can talk about other things.

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : Do you have any sense of the -- on a blinded basis, what the run rate is on the fracture rate or you don't have access to that?

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : And then as far as the disclosure, my understanding is that if it hits, obviously, you'll tell us. But if it doesn't, then you just -- you don't comment. So I guess the thinking will be if you don't comment after a period of time, we'll just assume that we're on to the next one?

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : And can we just quickly go through the differences between Orbit, which is main one and then Cosmic, which is I believe you've indicated in the past that you might get the Cosmic first, but Orbit is going to basically drive the filing?

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : Okay. And then I get this question often from people that are new to the story. I'm sure you get it too. Obviously, Amgen has romosozumab and Evenity is the trade name. They have expressed no interest in OI and there's some ceding of the rights to you in some respect. Can you kind of walk through that and just explain why romosozumab is not a threat?

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : And then the blueprint for the launch, assuming you hit your endpoints in Phase III, you mentioned a lot of overlap in the prescriber base with x-linked hypophosphatemia. So can you just talk a little bit more about how that would play out?

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : Okay. Let's shift over to gene therapy then, but before we get into the detailed programs, just at a high level, how do you think about which diseases make sense for Ultragenyx to pursue. You've got some good ones here, but what are the criteria that you use to evaluate whether to go into a certain rare disease for gene therapy?

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : Yes.

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : So just -- so people understand, with the standard of care with the cornstarch, are they setting alarms in the middle of the night to wake up? Like what if they don't take it? What is the risk of dying on a given night or any night, just in general?

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : So it's more of a deterministic thing, like it will happen.

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : So you will have a pre-BLA meeting coming up. What are the goals there? And what -- I mean, the label seems pretty straightforward, but what do you want to achieve at that meeting? And what's the market look like for this product?

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : Instead, they know there's a back step?

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : I see. Okay.

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : They're not setting the alarms anymore.

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : Okay. Let's keep going because we've got 10 minutes. So Wilson's. What's the path? You've shown some nice data already. People weaning off the chelators, some fully, some partial. What's the strategy there? And is there much of a competitive dynamic, it doesn't seem like it, but apart from the chelators? 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 03, 2024 / 6:45PM, RARE.OQ - Ultragenyx Pharmaceutical Inc at Citi Global Healthcare Conference

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : Which tissues typically are those the storage tissues, the excess storage tissues and where are you seeing expression?

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : One more on gene therapy, and then we can quickly touch on Angelman. OTC, you spend less time on that. The Phase III is underway enrollment. When is the data coming because we don't really talk about that one too much?

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : Okay. And then Angelman's quickly, and then we'll do some commercial. So you picked Bayley-4 raw. Just explain why you didn't do the Bayley growth scale value, the GSV and your competitor picked one piece of that -- the one piece of that domain, the expressive communication, I believe, so you didn't do that. So just explain -- and of course, you're focusing on the deletion as the primary?

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : Okay. And then just very quickly, obviously, there's one competitor that's relevant. You have the...

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : Okay. We can discuss, but you got the IP from Texas A&M from the inventor there. So just -- it would be very interesting to just talk about quickly the strength of that IP and the reason why you think it is the superior molecule?

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : All right. Thank you so much, Emil. Appreciate it. I'll have to save the commercial discussion for the earnings call. It's going well.

Question: Yigal Nochomovitz - Citigroup Inc. - Analyst : All right. So thanks again. Appreciate it.