Ultragenyx Pharmaceutical Inc. (RARE) Citi Annual Global Healthcare Conference 2025 December 2, 2025 3:15 PM EST
Company Participants
Eric Crombez – Chief Medical Officer & Executive VP
Conference Call Participants
Yigal Nochomovitz – Citigroup Inc., Research Division
Presentation
Yigal Nochomovitz
Citigroup Inc., Research Division
We can get started. Welcome, everyone. I’m Yigal Nochomovitz, one of the biotech analysts at Citi, and welcome to our Global Healthcare Conference in Miami.
So our next session is with Ultragenyx. We have the Chief Medical Officer, Eric Crombez. Eric, welcome. Thank you for making the trip. We’ve been covering you for many years and have been a lot of progress.
Question-and-Answer Session
Yigal Nochomovitz
Citigroup Inc., Research Division
So maybe we could start out just at a high level. You obviously have a very important catalyst coming up essentially imminently with setrusumab. So before we get into the details of that, just kind of set the stage as far as what is the opportunity there? You had and are continuing to have a lot of success with Crysvita, another genetic disease. So talk about what the opportunity is in osteogenesis imperfecta and how you’re setting up for a market launch there?
Eric Crombez
Chief Medical Officer & Executive VP
Yes. Great. And thanks for having me. So osteogenesis imperfecta, it is our near-term value driver. We’ve been talking about final data readout around the end of the year, and we’ve defined that as December or January. So we’re certainly within that period now and is about as near term as it gets. So osteogenesis imperfecta, also known as brittle bone disease, and I think really illustrates the disease and the opportunity. The only currently available treatment to really stopping these patients from fracturing with very little to no trauma is bisphosphonates. Those are used off-label in the U.S. and most parts of the world.
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