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

So that’s the Q4, Q1 dynamic, that plus decrease in KUVAN in Q1 is just an overall comment that Q1 is not going to be our strongest quarter, and we’re going to see most of the growth in the second half of the year.

Alexander Hardy: Thanks, Jessica. As regards to the concept of basket, one of the great benefits of having Dr. Dauber do his study is we actually have really good regulatory feedback on that question already. And in fact, you could see that his study is itself a basket of things that might be formerly known as subsets of idiopathic short stature or hypochondroplasia. So the concept of basketing has already been in front of the FDA and has already gained a little bit of acceptance. And having said that, though, as reflected in our prepared comments, we are in the stages of finalizing our game plan after our last interactions with health authorities around the world, and we’ll communicate final design decisions when those studies come online.

All that said, we’re not waiting for any of that because we’re also implementing our theater studies, the baseline run-in studies that are essential for enrolling these baskets of studies so that we can expedite the start and completion of these additional registration activities. So in sum, I think we’re trying to be as cover as we can be about getting studies underway and working with health authorities around the world to ensure that they meet the demands of regulators.

Jessica Fye: Thank you.

Operator: Our next question comes from the line of Phil Nadeau with TD Cowen. Please go ahead.

Phil Nadeau: Thanks for taking my question. A follow-up question on VOXZOGO. In the past, BioMarin had talked about converting VOXZOGO’s approval from accelerated to full and that potentially changing what would be necessary for the competition to be approved. Is there any update on the process of converting VOXZOGO’s approval to full? And at this point, would it have any impact on the regulatory requirements for the potential competition that’s in pivotal studies already? Thanks.

Henry Fuchs: Hi, Phil. No particular update on the regulatory status or strategy or read-through on competitors. But one of the things that’s really exciting about the update that we can report is that every so often we look at ongoing studies to assess the durability of VOXZOGO’s effect, and we continue to be reassured that VOXZOGO, unlike other growth-promoting types of drugs has this – been a remarkable benefit of sustaining its effect. And that’s really one of the key features of benefit of VOXZOGO patients. And I think it’s going to be an important bar for any future innovation in this space, to ensure that patients can resume a physiologically normal growth rate safely and through the entire period of their growth, starting from earliest diagnosis all the way through the closure of the growth plate, so good on VOXZOGO so far.

Phil Nadeau: Perfect. Thank you.

Operator: Our next question comes from Geoff Meacham with Bank of America. Please go ahead.

Geoff Meacham: Hey, guys. Afternoon and thanks for the question. Another one on VOXZOGO, I know it’s a centerpiece of growth and the major pipeline investment for the year and beyond. I wanted to ask you, what’s been the impact of your commercial investments so far in terms of the pace of new starts or broaden the prescriber base looking into the second half of last year. And then what do you think about what does success look like for 2025? I’m just trying to figure out a couple of metrics that we can kind of evaluate over the course of this year for VOXZOGO. Thank you.

Jeffrey Ajer: Hi, Geoff, its Jeff here. Let me take a shot at that. So I think we’ve had some good returns on our commercial investments to-date. You mentioned new starts, new starts were accelerated starting late last year following the label expansion, as noted in the prepared remarks. Expanding the prescriber base, particularly in the United States, to pediatric endocrinologists is a particular area of focus, both last year and into this year. We’re making good progress. And in terms of metrics, I would say we’re dialing back from specific quantitative metrics on these launches and turning more towards revenue. I might suggest that was a post-launch revenue base for VOXZOGO in particular, the best metric, I would recommend now and going forward is the revenue line.

Geoff Meacham: Great. Thank you.

Operator: Our next question comes from the line of Ellie Merle with UBS Financial. Please go ahead.

Ellie Merle: Hey, guys. Thanks for taking my question. At the ISS VOXZOGO Data Update, you mentioned will be coming in May. What should we expect from that in terms of the patient numbers and what you’re looking to see? And then just as you look across the many different short stature conditions, how are you thinking about prioritization across the indications and which you’d move forward versus not? And any threshold in terms of efficacy?

Henry Fuchs: Okay. As regards to Dr. Dauber’s ISS, the anticipated update, this is his study, and so it’s very much under his determination as to what he’s going to update. My understanding is that he’s enrolled more hypochondroplasia patients than other indications. But I think he be reporting unless he felt that there was useful information to be reported. I think the things to be looking for really are corroboration of what, I think, most scientists fundamentally believe, which is that CNP as a master regulator of bone growth is going to work across a wide variety of indications. And so as we had seen with the hypochondroplasia program, so it’s actually sort of the first half of the cohort at the 6 month mark that enabled FDA to gain confidence that we could go directly into Phase 3 and that’s predicated on a lot of this biology.

And therefore, I think the important outcome of the ISS presentation at PES is going to be are we seeing basically the same effect that we expect to see in the different types of stature disorders. And as regard to your second question, I think the prioritization process has been described, really boils down to fairly conventional metrics around time and cost to proof-of-concept or to global registration, value that gets created for patients and for shareholders. With the idea of by setting a high bar for something like proof-of-concept, then you really only advance your winners. And by focusing on those, we hope that will expedite development and bring faster and higher value to patients and shareholders of the undertaking.

Operator: Our next question comes from the line of Joseph Schwartz with Leerink Partners. Please go ahead.