BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) Q4 2022 Earnings Call Transcript

Jeff Ajer: And relative to your question on the U.S. centers, Jessica, there’s approaching 150 hemophilia treatment centers in the U.S. and those range from very large comprehensive care centers, you might call them to much smaller hemophilia treatment centers. So ,our intention is, as we noted in the prepared remarks, is to have a small number of the most capable and largest centers ready to go on or shortly after launch. We haven’t guided to a specific number, largely because we don’t think it’s that relevant. But we’ll be targeting a focused group of the biggest, the most capable centers to be ready to go.

Operator: Your next question comes from Chris Raymond from Piper Sandler.

Chris Raymond: Hey. Thanks guys. And if you’ll bear with me another ROCTAVIAN question — or two of them actually kind of related. So I just want to square a couple of things. JJ, at the beginning of last month, I think I heard you when you’re talking about this dynamic, you project that German commercial patients would be treated in the first quarter, if not this month, which I took as January. This March 15th date when insurers are derisked, is that a new learning for you guys? Because I don’t think I’ve really heard that date before, sort of that dynamic. And then maybe a related question, and I know you guys don’t give quarterly guidance. But I guess a clumsy way of asking is this setup would seem to maybe make pretty negligible Q1 ROCTAVIAN revenue. Is that — am I making the correct assumption there? Thanks.

JJ Bienaimé: Yes. I think we said — I remind you, I think we see in the coming weeks back in January, we didn’t say in January. This March 15th date is not a new date. We knew that this would be coming. So, we still hope to have some patients in Q1. But Brian, do you want to pick from there?

Brian Mueller: Yes. Thanks, Chris. It’s Brian. I think that you’ve interpreted correctly. Jeff described the current dynamics, we’re closing in on those first patients with the patients undergoing testing. Here, we are February 27. So 2/3 of the way through Q1. So, I think JJ is right, we’ll hope for a few patients, but not the material trend that the guidance would imply, if you will.

Operator: And our next question comes from Robyn Karnauskas from Truist Securities.

Unidentified Analyst: This is Nishant on for Robyn. Just a couple of questions, one on VOXZOGO. So with regards to launch dynamics, now that you have the drug approved in Japan and it’s for all age populations. Can you provide more color on the demand in unit population? How is it — how are people or patients are responding to it? And in terms of — I’m asking this question because I know you’re expecting expanded approval in U.S. and EU. So just to give us a clear sense of more demand in the younger patient population. And one question on pipeline. I know with the BMN 331 gene therapy, you had presented some data last time in January. So, with the highest dose, 6e13, I think the C1 level was approaching like a normal range. So, do you have any more color on the data beyond week nine? And do you expect to like dose at a higher concentration considering like for some of the patients in…

JJ Bienaimé: It’s getting too long, if I may. So why don’t we start answering that question.