BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) Q4 2023 Earnings Call Transcript

Henry Fuchs: Thanks, Kostas. I think a big part of the purpose of the early part of the year of prioritizing R&D portfolio, ties to the conversations that Brian was just having around capital allocation, Alexander was having around longer-term financial outlook. The way I think about that is that it’s a real – it’s a great thing that our company can generate cash that can be reinvested in our R&D portfolio. And we have to earn that through developing assets that have value to then reapply to generate new medicines. So we have to do that in a business context. And so as soon as we can make decisions about what’s going to go forward, we want to invest in expediting that and also things that are not going to go forward, we want to make sure that we don’t spend money on things that are not going to go forward.

So that’s very much the intention, and it’s, I think, ties to Alexander’s statement of how the progress of our process through to Investor Day could be potentially incrementally informed over the course of the year.

Operator: Our next question comes from the line of Jack Allen with Baird. Please go ahead.

Jack Allen: Hi. Thanks for taking our question and congratulations on all the progress made over the course of the quarter. I wanted to ask one on VOXZOGO. You mentioned that about 70% of the new US starts in the fourth quarter were the younger patients and the label expansion. I guess, could you provide some more context around how penetrated the older patients are? And how should we think about growth of VOXZOGO moving forward? Is it mainly expected that it will come from younger patients? Or is there still opportunity to expand uptake in the older patients in the US as well?

Henry Fuchs: Thanks for the question, Jack. In the United States, we haven’t disclosed penetration rates and we’re not going to introduce that metric overall, with reported numbers of about 2,600 patients, and you heard me quote the question to Gena earlier of an eligible patient population of 20,000, you can do the quick arithmetic on that yourself. Specifically from the United States, which is one of our largest and highest value markets, we’re really encouraged by both the continued uptake in the older children, those ages five and up from the original approval and also the new demand from the younger patients. So I think we’re pretty confident that we’ve got an opportunity for increased uptake there going forward.

Jack Allen: Okay, thanks.

Operator: Our next question comes from the line of David Lebowitz with Citigroup. Please go ahead.

David Lebowitz: Thank you, very much for taking my question. Just following up on that, with 70% of patients on VOXZOGO from new scripts being young patients. Is it weak to assume that, that number would be maintained next year? Or should that dial back as the year goes on?

Alexander Hardy: David, let me take that one on. I don’t think we have enough data to specifically say that we’re going to maintain that mix. But that’s a onetime metric intended to give you a little bit of color on how things are going in that key market since we got the younger age label expansion. But it’s an important signal, and I think it’s a good one, good and encouraging looking forward.

David Lebowitz: Thanks for taking my question.

Operator: Our final question comes from the line of Luca Issi with RBC Capital Markets. Please go head. Thanks so much.

Luca Issi: Hello, great. Thanks much. Thanks for squeezing me in. Maybe Alexander at the R&D day last year, which I appreciate was before your time, BioMarin talked quite extensively about life cycle management in valrox including testing patients with neutralizing antibodies to AAV5, patients with Factor VIII inhibitors, younger patients and so on. Given the relatively slow launch, is that still a plan? Or is there a scenario don’t invest that capital, just you prioritize operational efficiency to really make the story here, driven primarily by VOXZOGO [indiscernible] and valrox any color there, much appreciated.

Henry Fuchs: We do intend to include a review of those studies in our portfolio assessment and prioritization. And so we’ll communicate the plans for each of the additional potential ROCTAVIAN indications that we did outline at R&D day that you did just list, and as we complete our portfolio prioritization process, as well as our overall corporate strategy and so we’ll follow up then.

Alexander Hardy: And I would just add to that, that as we’ve said that R&D prioritization is looking at the potential benefit in terms of the medical impact together with the commercial potential. So we’ll be taking into mind our experience with the commercial uptake of ROCTAVIAN to inform the business case around those life cycle developments.

Luca Issi: Thank you, very much.

Operator: I would now like to turn the call over to Alexander Hardy, President and CEO of BioMarin, for closing remarks.