Ultragenyx Pharmaceutical Inc at Bank of America Global Healthcare Conference Transcript

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

Question: Dan Lundquist - Bank of America - Analyst : Maybe start. We'll kick it off with a general question about in our overview of the commercial clinical programmes as the company focuses on you know the rest of the year in early 2025.

Question: Dan Lundquist - Bank of America - Analyst : Great. So let's dig into some of the programmes. So first and foremost, let's dig into Angelman, can you provide us an overview of the GTX 102 programme, discussing the complexity of the neuro development disorder? Current unmet need for safe and efficacious treatment, the mechanism of action and then we'll dig into a little bit around the endpoints because I know there was a lot of debate around that. Before you had the data earlier this year.

Question: Dan Lundquist - Bank of America - Analyst : And what would you say having conversations with KOLs are the biggest priorities for the patient, the patient's family in terms of what they want to get from therapies that sweep communication card division, cross-border skills, et cetera?

Question: Dan Lundquist - Bank of America - Analyst : And maybe you'd referenced today present end of last year recently, has the data at AAN and maybe just discuss the kind of clinical function data that you saw, how it compared to the expectations. Just give us a little sense of both?

Question: Dan Lundquist - Bank of America - Analyst : You mentioned that you're going to be starting the pivotal Phase 3 Aspire study by year end. Can you share some of the feedback the FDA gave you regarding the proposed trial design and what is still any gating factors finish in that show?

Question: Dan Lundquist - Bank of America - Analyst : You're running a separate trial as well as the Aurora trial today driving other engine types to one larger study. Can you maybe talk us through the thought process, there have been additional separate studies.

Question: Dan Lundquist - Bank of America - Analyst : Maybe kind of shifting gears to the primary endpoint, you mentioned cognitions it's early for cognition. What would be a clinically meaningful results for that endpoint?

Question: Dan Lundquist - Bank of America - Analyst : And maybe just thinking about how [barley flour] could be dependent on patient engagement motivation, what's your expectation for the placebo response. And the reason I ask the question is there a risk that it could have a higher response and the clinically meaningful pipeline point cut off since investigators, patients, families et cetera no, but the endpoint is going to be.

Question: Dan Lundquist - Bank of America - Analyst : And on the secondary endpoints, can you help us understand the different components of the MDRI. Secondary, how you decide on what the minimally important difference with leverage sublimit?

Question: Dan Lundquist - Bank of America - Analyst : Great. And then maybe finally, can you share details on how the lower extremity weakness AE was management typical time line process resolution? I felt like in advance of that of the data, there was a lot of focus on safety and emerging from the data that was lot of focus on the robustness of the efficacy. So maybe just on that AE and just the safety profile overall.

Question: Dan Lundquist - Bank of America - Analyst : Would you say there's any specific risk factors that you've identified that might put a child on risks.

Question: Dan Lundquist - Bank of America - Analyst : We really think it's just that local irritation (inaudible) especially on that side and how it impacts we don't need to do any (inaudible).

Question: Dan Lundquist - Bank of America - Analyst : Yeah, an indication, of course, yeah (inaudible)

Question: Dan Lundquist - Bank of America - Analyst : Excellent, maybe turn the page to osteogenesis imperfecta. Can you provide an overview of the program in Allied, unmet need and space rationale for its crossing them away the most recent [14] update?

Question: Dan Lundquist - Bank of America - Analyst : What percentage of these patients are on background by phosphates.

Question: Dan Lundquist - Bank of America - Analyst : Can you just discuss any benefits you've seen on bone marrow -- bone mineral density in the expedition three through to long-term bone health for these patients.

Question: Dan Lundquist - Bank of America - Analyst : Can you walk through the current Phase 2 trial design time line for your planned interim looks at attempting to say to be stopped early?

Question: Dan Lundquist - Bank of America - Analyst : Is there any scenario where you may hit on interim for actually continue wanting to sort of generate that larger safety database, which will be very important in the real world. I know you mentioned that you intend to all the patients out, but would you at the interims just continue running through that, slowed it?

Question: Dan Lundquist - Bank of America - Analyst : All right. And maybe just given '25 is going to be a very important year for Ally thinking ahead to how commercial market could look, how much do you think you could leverage your existing Crysvita sales force infrastructure being a successful commercial company in terms of the initial launch expectations? 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 18, 2024 / 2:05PM, RARE.OQ - Ultragenyx Pharmaceutical Inc at Bank of America Global Healthcare Conference

Question: Dan Lundquist - Bank of America - Analyst : Any questions on ally that will move over to Wilson disease. Can you give us an update on your Wilson Disease program? What do you think or what should we expect to see in terms of updates in terms of second half?

Question: Dan Lundquist - Bank of America - Analyst : Can you maybe help us level set some expectations for the update? Would you be looking for specifically to guide the dose selection decision?

Question: Dan Lundquist - Bank of America - Analyst : Great any questions on Wilson's. Awesome, maybe we move to GSDIa, can you discuss how you're thinking about the DTX 401 program in GSDIa, why do you think efficacy could improve in the real world setting?

Question: Dan Lundquist - Bank of America - Analyst : Great. We will be presenting additional analyses from the Phase 3?

Question: Dan Lundquist - Bank of America - Analyst : And what's the current status of the tech transfer any updates to your expectation of when we expect to file for regulatory approval and tech transfer as far as manufacturing test?

Question: Dan Lundquist - Bank of America - Analyst : Maybe last couple on GSDIa. So how are you thinking about the pricing environment given the standard of care is dietary supplementation? And what's the value proposition of gene therapy in this disease?

Question: Dan Lundquist - Bank of America - Analyst : And in terms of patient identification, how well identified our patients in the US with this disease where an efficient treated and ourselves for surgery for launch?

Question: Dan Lundquist - Bank of America - Analyst : And maybe the last couple of minutes here we could shift focus to on Sanfilippo syndrome has to be a pre-BLA meeting with the FDA been scheduled at this point. What were the key points of discussion be at the meeting. How soon after that meeting, will you look to update us? 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 18, 2024 / 2:05PM, RARE.OQ - Ultragenyx Pharmaceutical Inc at Bank of America Global Healthcare Conference

Question: Dan Lundquist - Bank of America - Analyst : And just remind us the overall market opportunity for Sanfilippo.