Ultragenyx Pharmaceutical Inc at Cantor Global Healthcare Conference Transcript

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

Question: Kristen Kluska - Cantor Fitzgerald & Co., Inc. - Analyst : Okay, but before I do that, maybe just big picture, what are you most excited for in the company over the next 12 months?

Question: Kristen Kluska - Cantor Fitzgerald & Co., Inc. - Analyst : Yes, and beyond this, the voice and work you've done for the rare disease community even outside of Ultragenyx hasn't gone unnoticed. So thank you so much for all of your support for the field in general.

Question: Kristen Kluska - Cantor Fitzgerald & Co., Inc. - Analyst : Yes. All right, let's dive into OI. So two years ago, you made a really bold statement around setrusumab. You described it as your highest-value program with the strongest data and the highest probability of success. Is that still the case today? And at that time, why were you so confident without having all of the data you've now since generated?

Question: Kristen Kluska - Cantor Fitzgerald & Co., Inc. - Analyst : Yes, I would love to spend a little bit more time on the science about how an anabolic agent really works on both sides of the bone formation equation, whereas drugs like disphosphonates tend to only work on one with limited benefit. So I understand that you don't necessarily need to fix the collagen defect, but how do we just know? I mean, it sounds so simple, right? Greater bone strength and mass should mean a better bone profile, but what specifically is going on there that makes that bone so strong that when you fall or you experience some type of trauma, you're less likely to fracture? 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 17, 2024 / 5:20PM, RARE.OQ - Ultragenyx Pharmaceutical Inc at Cantor Global Healthcare Conference

Question: Kristen Kluska - Cantor Fitzgerald & Co., Inc. - Analyst : Okay, so obviously fractures is a very critical component of this disease, but the field is really eager for any kind of treatment option that can remedy other manifestations of the disorder. So how might setrusumab have the potential to address some of the secondary clinical issues, things like pain, level of activity, mobility, wheelchair requirement, et cetera?

Question: Kristen Kluska - Cantor Fitzgerald & Co., Inc. - Analyst : Yes, I'd love to talk about pain in a little bit more detail. So I actually got to go to an OI Foundation Conference in July, and I remember eating breakfast with a table full of OI patients, and they were all saying that they got half an hour, one hour of sleep. Why? Because they couldn't sleep on the bed. They were in pain. I would describe this as a very average comfortable mattress. I don't have OI. So something to me like that was like wow, this truly impacts every single day if you can't sleep on literally a bed of cotton. So when you think about the potential to reduce pain, is it because you're not sedentary you're getting up, you're doing more things, or is it that if you're building a better bone profile and fracturing less, or is it a little bit of everything that may have that impact?

Question: Kristen Kluska - Cantor Fitzgerald & Co., Inc. - Analyst : Yes, but it really does put it into perspective, right? It's not just pain from falling. It's literally going to sleep at night and you're not comfortable. So how do these factors all correlate to their quality of life and being able to maintain -- I would think they'd want some level of independence as much as they can have.

Question: Kristen Kluska - Cantor Fitzgerald & Co., Inc. - Analyst : One other thing I heard from some doctors is that a lot of the patients end up getting small fractures in their vertebrae, and they go unnoticed because they're not -- the full bone isn't fracturing, but they complain about back pain and that's something that they already commonly deal with, right? But that can be quite dangerous because unnoticed, it gets worse. Can we talk maybe a little bit about the different types of fractures? 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 17, 2024 / 5:20PM, RARE.OQ - Ultragenyx Pharmaceutical Inc at Cantor Global Healthcare Conference Obviously, no fractures are good, but is that something that you've thought about, the ones that can end up going unnoticed and are quite dangerous?

Question: Kristen Kluska - Cantor Fitzgerald & Co., Inc. - Analyst : Sure. So putting all these metrics together, if this product is ultimately approved, there are going to be patients that want this product even if they're ones that are actively not fracturing for a lot of these other benefits we described. Is that a fair statement in your opinion?

Question: Kristen Kluska - Cantor Fitzgerald & Co., Inc. - Analyst : And when you use this term that patients aren't doing activities, are we talking about things that are more on the extreme end, like playing in the backyard, soccer, or are we even frankly talking about things as simple as walking around the house, even if you have to get from point A to point B, maybe you use a wheelchair or a walker when really you can probably technically be able to walk there on your own, you just might be afraid. 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 17, 2024 / 5:20PM, RARE.OQ - Ultragenyx Pharmaceutical Inc at Cantor Global Healthcare Conference

Question: Kristen Kluska - Cantor Fitzgerald & Co., Inc. - Analyst : Yes. Thanks for that perspective. So let's talk about the trial, the upcoming readouts. You have a few interim readouts for overwhelming efficacy only. You're going to remain blinded, but can you help frame for our audience essentially what would be the trigger point for these and how are you also thinking about expectations? The first one's a pretty high bar that you have to hit.

Question: Kristen Kluska - Cantor Fitzgerald & Co., Inc. - Analyst : Thank you for that. So the Cosmic and Orbit studies were enrolled at very similar paces. Do you have different expectations for each patient population given the early impact on bone mineral density changes we saw in different age groups?

Question: Kristen Kluska - Cantor Fitzgerald & Co., Inc. - Analyst : Okay, thanks. And a lot of investors we speak to truly do see the potential to setrusumab. But one question that keeps coming up is, how's the placebo going to perform? We think the drug is great, but when we think about all these powering assumptions, where do we get the level of comfort there? So can you walk us through your powering assumptions and then also how you came up with that assumption for the placebo performance?

Question: Kristen Kluska - Cantor Fitzgerald & Co., Inc. - Analyst : I think something that's very underappreciated is there's a wonderful comp about what the market opportunity could look like. And that own comp is your own drug, which makes it a lot better because when we think about Crysvita and that process towards approval, you had to build the specialty sales team for that. Now you're talking about a drug where there could be over 90% overlap. So how are you thinking about that opportunity and especially because things are already kind of on the ground, in place, ready to go if you got approval?

Question: Kristen Kluska - Cantor Fitzgerald & Co., Inc. - Analyst : Great. Thank you. Maybe to close, obviously again, there's a lot going on at Ultragenyx. I appreciate you allowing me to really just focus on this program. But why should somebody own the company for this opportunity alone? Forget all the other potential upside leavers. Y-o-Y alone is a good reason to own the stock now.

Question: Kristen Kluska - Cantor Fitzgerald & Co., Inc. - Analyst : Great. Well, thank you very much. Appreciate your time today. Thanks, everyone.