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Ultragenyx Pharmaceutical Inc. (RARE) Presents at Bank of America Global Healthcare Conference 2025 Transcript

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Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Bank of America Global Healthcare Conference 2025 September 23, 2025 6:35 AM EDT

Company Participants

Eric Crombez – Chief Medical Officer & Executive VP

Conference Call Participants

Tazeen Ahmad – BofA Securities, Research Division

Presentation

Tazeen Ahmad
BofA Securities, Research Division

Good morning to everybody listening in. Welcome to the Bank of America Healthcare Conference. I am Tazeen Ahmad, one of the senior SMID biotech analyst. It is my pleasure to have with me our next presenting company, Ultragenyx. Presenting for Ultragenyx and sitting with me is Eric Crombez, who is Chief Medical Officer.

Eric, thanks for making the trip over from the — [indiscernible] not as far.

Question-and-Answer Session

Tazeen Ahmad
BofA Securities, Research Division

There’s a lot going on at Ultragenyx. There’s been a lot of questions being as I guess, two of your programs, OI and Angelman. And so not a surprise, we’ll spend most of the next 40 minutes or so talking about that.

Before I do that though, maybe since you’ve had so many conversations with folks at this point, can you just give us an overview of both the OI and AS programs, where you stand with those, and then we’ll go into specifics on each, if that’s okay.

Eric Crombez
Chief Medical Officer & Executive VP

Yes. I guess starting a little bit in order. So osteogenesis imperfecta for the OI, Angelman programs being our big value drivers and then also including the gene therapy programs.

But focusing on osteogenesis imperfecta, we obviously did not clear our second interim analysis, and we announced that earlier in the year that leads us to full study readout. We’re saying we will talk about that externally around the end of the year, and we define around end of the year at December or January. Certainly, we share

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