Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Q4 2022 Earnings Call Transcript

Emil Kakkis: Sure. Well I’ll answer the agent question first. And then maybe, Erik, you can touch on the Crysvita and the strong Q4 question. So on Angelman, we’ve been releasing. We’ve been doing these dose titration costs, which were just relatively small for patient groups. But it’s clear we need to have something like at least 10 patients in each group to kind of give us the kind of data that we can do statistics on and at least do enough projection to understand how to power a study. So I’m looking for the announced to have maybe 20 patients or about half of what we need to enroll. That’s giving you a rough idea. And now in addition, we would have the patients we’ve already treated who will have been titrated in maintenance mode, and I would expect that would be the kind of volume of data that would allow us to accurately project what Phase III will look like, which is I think what investors ask for us is to have confident data that will allow them to see the future for the program in a Phase III program.

So that’s that. And for Crysvita Q4, I think it’s a pattern we’ve seen before, Erik. Perhaps you want to add to the pattern.

Erik Harris: Yes. We had expected a strong second half of the year as we were hampered in early 2022 with the Omicron variant. We saw continued increase in patient volumes in the offices as they opened up more and more as well as participation in live of educational meetings and programs. And I think consistently growing demand throughout the year was one of the main factors driving the strong Q4.

Emil Kakkis: So it’s a bit of a rebound out of the hampered first part of the year, but it generally has been strong in Q4.

Operator: This does conclude the question-and-answer session of today’s program. I’d like to turn the program back to Joshua Higa for any further remarks.

Joshua Higa: Thank you. This concludes today’s call. If there are additional questions, please contact us by phone or at ir@ultragenyx.com. Thank you for joining us.

Operator: Ladies and gentlemen, for your participation in today’s conference. This does conclude the program. You may now disconnect. Good day.