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

Unidentified Analyst: Hi. This is Julian on for Paul. Thanks so much for taking our question. I guess just in terms of thinking about capital allocation moving forward. Obviously, you’re reviewing the pipeline. But could you just provide a little bit of color on, do you anticipate reinvesting in the pipeline, perhaps after cooling programs that don’t meet that high bar that you speak of for advancement? Or are you sort of more focusing on growing the commercial business for VOXZOGO? I guess just any color you can provide on future capital allocation for 2024 and beyond, would be super helpful. Thank you.

Brian Mueller: Yeah. Thanks for the question. This is Brian. We’ll ask you to wait a bit longer for the outcomes of our strategic review to give you details on our capital allocation strategy. I mean, we can say that internal innovation, given our track record and value created in the current business is going to continue to be a priority. But we also recognize that BioMarin not only carries a substantial amount of total cash and investments today, but we are now cash flow positive. So we are committed to developing a capital allocation strategy that’s in the interest of maximizing shareholder value. That work is in process, and we will look forward very much to sharing it with you, but I’ll just ask you to stay tuned until we finish the work.

Unidentified Analyst: Thanks.

Operator: Our next question comes from the line of Vikram Purohit with Morgan Stanley. Please go ahead.

Vikram Purohit: Hi, good afternoon. Thanks for taking the question. We had a follow-up question on ROCTAVIAN, a bit of a broader one. So your release mentions and you also just mentioned a couple of different areas of work that you’re going through to help progress commercialization here, from patient education to site readiness. But I was wondering now that you’ve had a few months of experience with the process for ROCTAVIAN, what do you see as the one or two specific factors or drivers of uptake that you think you have the most near-term leverage within and control over that could support the trajectory here in 2024?

Jeffrey Ajer: Hi, Vikram, this is Jeff Ajer. I’ll talk a little bit about that. What we’ve said is that we’ve seen very nice signals of patient demand, for example, in the United States, much of last year, we talked about the buildup of a patient funnel in the strategic market and priority market of Germany where we had gotten good uptake of CDx testing. We’ve also talked about the importance of site readiness and having reimbursement approvals that kind of clear that important reimbursement gate. As Alexander noted, we haven’t seen a direct correlation. So we haven’t been able to say, hey, this or that or are these leading indicators are good predictive indicators of patient infusion and revenue uptakes. And so I think the idea from here is instead of trying to better tune that model we’re going to let the actual revenue do the speaking for ROCTAVIAN.

Operator: Our next question comes from the line of Tim Lugo with William Blair. Please go ahead.

Tim Lugo: Thanks for the questions. You introduced operating margin in your guidance and you have a nice step-up year-over-year, what are the initial impressions on whether BioMarin will be able to continue to expand operating margins and eventually reach north of 40%, like some of the other rare disease-focused companies?

Brian Mueller: Yeah. Thanks a lot, Tim. I appreciate the question. It’s a great one. So again, we are pleased to be able to report and deliver over the last few years kind of year-on-year operating margin improvement. We’ve been talking about it for a while, and it is – we’re pleased to be able to actually deliver upon that. I’ll give a two part answer to your question. On one hand, we know there’s a lot of opportunity with the type of revenue growth that BioMarin has, not to mention the outcomes of the strategic work that we’ve talked about a lot today, and potential impact on revenue into the future. We also recognize that we’ve got operating model and cost efficiency opportunities. We built this global infrastructure that now we’re leveraging to support the strong launch of VOXZOGO for example, in a very deliberate and organic way over the years.

We’ve reached a lot of scale. We’ve got world-class capabilities across manufacturing, R&D, commercial, business support. But we’ve been focused on growing and scaling for years. And what that means is now we have a real opportunity to optimize and optimize the business model and make the cost structure more efficient. That’s the work we’re doing together with SORC. And by the way, the entire leadership team and enterprise, these are enterprise-wide initiatives that the company is really rallying behind. And the second part of my question is, for that reason, we’ll have to ask you to stay tuned. But by all means, you can picture the work that we’re doing to design what the appropriate and deliverable financial targets for long-term guidance purposes will be for the company.

And we’ll be sure to not just share those numerically, but the rationale, the strategy and our plans to deliver upon them. So that’s just going to take a bit more time. Thank you, though, for the question.

Tim Lugo: I understood. Thank you.

Operator: Our next question comes from the line of Olivia Brayer with Cantor Fitzgerald. Please go ahead.

Olivia Brayer: Hey. Good afternoon guys. Thank you for the question. Wanted to ask about life cycle management for your VOXZOGO franchise, obviously, you have a lot of expansion opportunities within that product, but could we see any efforts to maybe better optimize the dosing schedule or maybe any other avenues to keep that product as competitive as possible? Thank you.

Henry Fuchs: Yeah, thanks. That’s a good question. And yes, in fact, we do plan to undertake some further dose optimization. Right now, our thinking is to try to do that in parallel with registration-enabling trials. But again, details to be communicated when study designs are final, after regulatory authority alignment.

Operator: Our next question comes from the line of Kostas Biliouris with BMO Capital Markets. Please go ahead.

Kostas Biliouris: Hello, everyone. Congrats on the progress. Thanks for taking our question. Given that your R&D day is planned for the third quarter of 2024, which is about a year after Alex took over, and likely, this is when you will potentially share pipeline prioritization details. I’m wondering whether you will continue to progress all pipeline programs, full steam until this third quarter, or you are going to pause the progression of some of the programs until you decide on their fate? Thank you.