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

Joseph Schwartz: Great. Thank you very much. And welcome, Alexander. I think I heard you say that the outcomes of the strategic and operating review committee could be communicated incrementally as well as at the Investor Day in the third quarter. So I was wondering if you have a sense of which updates might emerge sooner and which updates could require more time to formulate?

Alexander Hardy: Thanks, Joseph. Thanks very much for the question. The SORC is proceeding extremely well. We’ve met frequently and we’re making progress on the financial guidance, long-term financial guidance and the capital allocation strategy. We also have a lot of other work underway, as you get a sense from the cost optimization, the acceleration of VOXOGO, even faster R&D prioritization, et cetera, et cetera. And right now, we’re figuring out what is the best timing to update the external world on our progress on these points. Obviously, the benefit of the Investor Day is, allows us to put this all together with a strategic context of where we’re going to take BioMarin into the future. At the same time, we’re keen to share the information we can — as soon as we can on the progress we’re making.

So, as I said in my prepared remarks, please stand by for more information. The latest possible day will be Investor Day for all of those information. If it makes sense, we’ll communicate other information sooner.

Operator: Our next question comes from the line of Robyn Karnauskas with Truist Securities. Please go ahead.

Unidentified Analyst: Hi. This is Nishant. I’m on for Robyn. One on VOXZOGO. So I know you have spoken on competitors’ oral treatment for achondroplasia. Can you give us any color anecdotally about how patients on VOXZOGO or prescribers think about the orals [ph] Is there room for both if it gets approved? Thank you.

Henry Fuchs: Yes, we’ve shared robust and highly significant growth data from VOXZOGO in our clinical trials. And as I just referenced, the accumulating data on height gain is really quite positive and informative. And we see more of that at ACMG, demonstrating these long-term positive effects on growth, importantly, also on improvements beyond height. For example, improvements in the physical domains of quality of life types of scores. And so, we’re really focused on us building our leadership position in the genetic statural conditions where we see an incredible potential for VOXZOGO, again, to normalize growth to sustain that normalization of growth through the entire growing period and having the greatest possible impact in patients’ lives.

Unidentified Analyst: Thank you.

Operator: Our next question comes from the line of Mohit Bansal with Wells Fargo. Please go ahead.

Mohit Bansal: Hey. Thank you very much for taking my question and congrats to Alexander, and welcome. Maybe one question for you. In general, what is your general thought on gene therapy as a therapeutic modality? I’m asking because a significant portion of your pipeline is investing in gene therapy and would love to understand how you think about gene therapy in general for rare diseases? And what are the puts and takes there? Thank you.

Alexander Hardy: Thank you very much for your question. As I referenced a little bit, but let me go in a little bit more detail. We’ve got a very broad and exciting pipeline, across multiple modalities, including gene therapies. Gene therapy is offered obviously significant potential benefits for patients, transformative benefits. We have an expertise and ability to develop, manufacture and now commercialize gene therapies in multiple geographies. That is a unique set of capabilities that BioMarin has. As we look at our pipeline, as Hank was talking about, we’re going to be assessing at a program-by-program basis. What is the transformative potential of the program, together with the commercial potential? At the same time, we’re also looking at the overall strategy of BioMarin.

What are we really, really good at? And where we’re confident we’re going to have a sustainable competitive advantage going forward. And that will include asking and answering the question about our exposure to gene therapy, which will and form also the prioritization of our portfolio. So, more to come on this. Please hold your question. We’ll have a clearer idea of this, and we’ll share it at our Investor Day later in 2024.

Mohit Bansal: Got it. Thank you.

Operator: Our next question comes from Gena Wang with Barclays. Please go ahead.

Gena Wang: Thank you. I have two very quick questions. The first one is regarding VOXZOGO. In your press release, you said you already treated 2,613 achondroplasia patients across 41 active markets. What is the total market opportunity in terms of achondroplasia numbers for these 41 active markets? And also, what is the pricing dynamics there? And then second, very quickly regarding ROCTAVIAN. I think the last earnings was mentioned that six patients were ready to be dosed in 4Q 2023. Should we expect the remaining five patients to be dosed in first quarter 2024?

Jeffrey Ajer: Hi, Gena, it’s Jeff here. I’m going to take that first question on VOXZOGO patient numbers. So essentially, those 41 markets represent kind of the core of the commercial footprint that we usually use to quote eligible patient numbers and market potential. And for that group of patients, that would now total about 20,000 patients eligible, and that’s essentially matching the lower age limit in the different labels, which are zero, four months or six months depending on where you are in the world. And in terms of the ROCTAVIAN question, back to Alexander.

Alexander Hardy: Thanks, Gena. Yeah, let me take your second question. We’re going to provide details on Q1 including product revenues at our conference call in April, H1. We’re tracking progress on site readiness at hemophilia treatment centers with payers, completed positive CDxs tests. But clearly, these metrics so far have not yet correlated directly to patient uptake. So we’re no longer going to be providing specific updates on these metrics. Once again, just to reiterate, we’re going to let the actual experience with actual commercial patient infusions form our future expectations and we’ll report at the end of the quarter about our progress this year with infusions and revenue.

Operator: Our next question comes from the line of Paul Matteis with Stifel. Please go ahead.