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

Emil Kakkis: Thanks, Tazeen. So with Sanfilippo IIIa, one of the decisions we had to make in picking up the program was whether there was a very good option around that we could do just as well. And our view was there wasn’t really. The other program in lifetime, they are actually — they’re going out of business. This product was better than that product, I think, from a standpoint of biomarker reductions in effect. There is a lentivirus approach, which I think has its own challenges, it’s fairly early on. But really, there isn’t another, I think, effective AAV gene therapy out there that’s at the stage that says. So our feeling was that this would be the lead program and had the best efficacy that I’ve seen from at least a biomarker standpoint.

So that was our decision. With regard to the population of the world, there’s probably a few thousand patients, but the — you have to think of this as a little bit more of an incident population, right? Because the best effect appears to be in kids that are under two years old, so it was a little bit like Zolgensma, they need to be young to get the best benefit would we treat older patients, perhaps, it depends on what happens in the regulatory process. I think there may be benefit in older patients, but it’s clearly the optimal benefit happens when you treat Young. So I would look at it that way. What I was saying to you about this gene therapy and unlike some of the other diseases where there are other treatments. If we add a gene therapy that’s doing what we believe we see is doing right now, we think essentially every sample patient won’t want to get treated because the other options are just none at this point in time.

And so it has the kind of devastating impact that you have with SMA, and I think the adoption rate would be very high if we can get it approved promptly. Now the other question was the manufacturing plant and capacity. The plant has two suites that can run 30 runs a year. One suite is operating right now. We will — we can operate a second suite. 30 runs year gets a lot of what we need done. I don’t know that we’ll handle 100% of the needs because it depends on which programs you’re talking about to achieve that. Our plan has always been to have some hybrids, some contract manufacturing and some of our own. And if necessary, we do have a green space a greenfield plant space next to our plant that we could double the plant further. But right now, I think 30 runs will take us a long way in the future.

And if we’re at that high-level success rate, treating that many gene therapies, we can then invest in expanding that capacity further. But it will take us a long way for what we need at the moment.

Operator: And our next question comes from the line of Jeff Hung from Morgan Stanley.

Jeff Hung: I guess for setrusumab, you talked about the two doses what kind of incremental benefit would you expect or want to see with the 40-milligram per kilogram dose over the 20? And then can you talk about the feedback that you’ve been hearing on the launch of Evkeeza in Europe?