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

Emil Kakkis: Yes. The patients, as we said before, a resolved essentially resolved the problems they had. I think that we’re continuing to evaluating him. He’s not redosed yet. We have to wait until his we will wait until his protein levels have resolved, which we will do at intervals and measure, but we’re encouraged how that’s gone and the second question was on — yes. So we’re planning that discussion shortly, and we’ll be presenting how to approach moving ahead with expansion cohorts in the U.S. And we’re expecting a good meeting. We think we’ve gotten good responses through discussions with them lately and I feel like there’s an opportunity to move ahead. But we still need to have that formal discussion in order to open up the protocol in the U.S., which we think will be important in being able to rapidly expand the study and put us in position to get a large enough amount of data to be confident about designing a Phase III.

Operator: And our next question comes from the line of Liisa Bayko from Evercore.

Liisa Bayko: Could you run through just with the change in — to the royalty. Can you just remind us of that? And how you’re seeing kind of the net profit from that changing is the royalty somewhat equivalent to what you’d be getting from the profit split, that would be helpful.

Emil Kakkis: Yes. So to be clear, you’re talking about the switch from a profit share with our partner, Kirin to the royalty period. Is that correct? That’s what you’re talking about?

Liisa Bayko: Yes. Exactly, yes.

Emil Kakkis: Well, in general, that crossover will essentially be even. There won’t be a significant change, really. The way we structured it originally made was actually pretty well modeled in terms of predicting what our share should be. So the 29%, the top tier of the royalty will cover and expect to cover some commercial costs. We are sharing some costs with them. But at this point, if you look at the total picture of how the profit share was designed and how this is — it’s pretty flat, pretty similar. And so we feel very good that you wouldn’t see a significant step off or change in the revenue stream. I don’t know if there’s anything more, Aaron, you don’t want to say to that.

Emil Kakkis: No, that was well summarized. Thanks, Emil.

Operator: Our next question comes from the line of Anupam Rama from JPMorgan.

Anupam Rama: Two quick ones from me. Following up on Tazeen’s question on UX111 and MPS III has been meeting with the FDA been calendared? And maybe walk us through the scenarios that could play out here. And if you are able to go with an accelerated path forward for that program, is there any read-through to your broader gene therapy programs. And then question two, maybe could you expand on the statement in the press release that with GTX-102. There continues to be a dose and time-dependent clinical activity in the population. And maybe if you could provide any type of color on what type of benefit you’re seeing over what time period? That would be helpful.

Emil Kakkis: Yes. Normally, with regulatory authority discussions, we don’t precisely put out the exact timing of meetings and events that degree the meeting is set, and we’re planning to meet with them shortly. But does the point of the meeting is that the FDA had already agreed on a clinical endpoint and with a longer period of time to evaluate. However, of late, they have started showing some acceptance of heparan sulfate as that is predictive. And we believe in our public discussion with Peter Marks and in other settings as the heparan sulfate marker may be possible as the primary, which would allow us to file earlier than waiting for the clinical data. So our discussion will focus on can we use heparan sulfate of the particular high-quality assay that’s being used for purposes of filing with a commitment to complete the clinical follow-up data, of course, to verify the clinical benefit.