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

And so that will come relatively soon. And we’ll let you know as soon as we know what the answer is to that question. It doesn’t mean the program is — it just means we can go a little faster. If we can go faster, it just allows us to file earlier. We still have to set up the CMC manufacturing pieces of the program. And we’re trying to run that in a capital efficient way as possible because we do have a lot on our plate. So GTX-102, we disclosed that there was some dose and time-dependent differences. We have actually mentioned this before. When we put out information earlier, we had talked about the fact that the quantitative endpoints around the Bailey were better with the higher dose groups and with the lowest dose group at two mg in the U.S. So that was the dose-dependent piece.

And we’ve said that patients seem to improve over time, and that’s the time-dependent piece. So it’s information we’ve been seeing it’s in the main drivers are the ones we’ve already disclosed, which are — we’re talking about in the Bailey scores and language and sleep. And we’re evaluating multiple things and looking at many things, but we feel good about the pattern of response we’re seeing with both dose and time-dependent and that’s why we’re moving into expansion cohorts.

Operator: And our next question comes from the line of Joel Beatty from R W. Baird.

Joel Beatty: For revenue, are there any seasonal effects from Q4 of last year and Q1 of this year to be aware of?

Emil Kakkis: Well, there are always seasonal effects, but I think actually, I think we did well last year in terms of hitting our marks. And I’m not sure Aaron would have anything more to say. But I think you can always expect some lumpiness particularly with Latin America because the orders come in burst and off and on. And so it’s a little bit harder to predict. It’s just part of the nature of the rare disease business. But I think things are well. Erik, did you have anything else to add on lumpiness?

Erik Harris: You covered it as far as Latin America is concerned, I mean, I think it’s important to recognize that we’ve had steady growth over the last five years for Crysvita. It establishes the gold standard for treatment of XLH and we expect it to continue to grow as we’ve seen consistent increases in key performance indicators, such as patient finding, start forms, reimbursed patients and unique prescribers. So demand remains strong. That said, we do experience some seasonality in ceasing ordering patterns in Q4 and Q1, which has been consistent year-over-year, if you look back over the previous years. Q1 being a little lighter as expected as patients work through the reauthorization and changeable insurance process. So it just takes a little longer on to get on. But we remain confident in the underlying demand and which is why we reaffirmed our guidance.

Operator: And our next question comes from the line of Yaron Smith from Cowen.

Unidentified Analyst: This is Brendan on for Yaron. Sorry, if you just answer my phone call for just a second. But for Crysvita, I noticed Q4 sales really did jump up a bit. I was hoping you can maybe give us any color as to what was really underlying that just in Q4 relative to Q3. And if this is kind of some trends that you’re expecting to hold forward hold moving forward? And then I just wanted to ask on Angelman. I know there’s a lot of questions here on dosing and things like this. But can you just give us a sense of what we could expect in the next day to readout in terms of really how many patients you’re thinking to include whether they’ll include them from the expansion cohorts and maybe over how long really kind of just trying to get a sense of where the internal bar is for you all in terms of when you want to put out the data.