Ultragenyx Pharmaceutical Inc. (RARE) TD Cowen 46th Annual Health Care Conference March 2, 2026 1:50 PM EST
Company Participants
Eric Crombez – Chief Medical Officer & Executive VP
Conference Call Participants
Yaron Werber – TD Cowen, Research Division
Presentation
Yaron Werber
TD Cowen, Research Division
Okay. Well, good afternoon, everybody, and welcome once again to the 46th Annual TD Cowen Healthcare Conference. I’m Yaron Werber from the biotech team, and it’s a great pleasure to introduce and have with us today, Eric Crombez, who’s Chief Medical Officer and EVP at Ultragenyx.
Eric, good to see you. Thanks for coming.
Eric Crombez
Chief Medical Officer & Executive VP
Thank you.
Question-and-Answer Session
Yaron Werber
TD Cowen, Research Division
So lots going on in — maybe we’ll start with Angelman syndrome. That’s going to be the next, I think, one of the big catalysts in the second half, maybe even Q3, the way we’re kind of calculating and trying to back into a more fine-tuned timing. The Aspire study is about 130 patients, 4- to 17-year-old with a deletion. That’s about 70% of patients fall into that, Randomized 1:1 versus sham. The primary endpoint is cognition based on the Bayley IV. You obviously also have a Tandem study, the Aurora study, which we’ll get into that in a second. When you’re kind of thinking about powering for a benefit, what’s considered clinically meaningful for cognition?
Eric Crombez
Chief Medical Officer & Executive VP
Yes. So I think, obviously, interconnected, but a little bit different. So I think the best way to think about clinically significant and for Angelman with our conversation with the FDA, we’ve shifted to MSD, Meaningful Score Difference. So when we’re setting that threshold, and we specifically needed to do that as part of our MDRI, which is a second primary endpoint for us and set that MSD, your clinical