BioMarin Pharmaceutical Inc at Jefferies Global Healthcare Conference Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Akash Tewari - Jefferies - Analyst : Thank you. And actually, I'm going to start look into the guide, we'll get to the products. But I wanted to talk a bit about the unique BD environment we're seeing right now. I've had two companies where there was a public offer and then there was a topping bid, and it is annoying for me to deal with. But nonetheless, you don't see that typically. I think the question I have for your team is there is this kind of dynamic of people who have cash, and you have about $2 billion in terms of flexibility. But then you might be competing against a mega cap company where they might have a limitless balance sheet seemingly in terms of getting an acquisition done. How does that concern apply to your because your new Head of BD has come in. You guys have topped up external acquisitions. Do you feel like it has been more difficult for you to identify assets that you find compelling and pay a price that makes sense? Or actually that's not the case.

Question: Akash Tewari - Jefferies - Analyst : Understood. And to me, you're kind of thinking about like peak sales and risk adjustment. I can't help but think, Alexander. You're thinking, give me a Palynziq, Voxogo like opportunity that fits into this business unit approach, right, genetic to sort of skeletal muscles, skeletal conditions and then enzyme replacement. It seems like that's the kind of sweet spot and then either pre-commercial or just launch? I mean, is that the right framework we should be thinking about in terms of external BD.

Question: Akash Tewari - Jefferies - Analyst : Maybe just a point on that because I remember last year, there was this question of would you do -- you have your long-term guide, would you do a deal that would impair that? And I think your answer was like, look, in general, we want to keep our long-term approach. We think that's really important. If there's an asset that we think is super compelling, we're going to have to go for it. But I think lost in that kind of nuances and especially in what you just said, it's about acquisitions that are earnings accretive, right? Talk to me about the importance of if you are in-licensing assets, how does that change the long-term financial profile of the company? And how coherent is that with your long-term guide as well?

Question: Akash Tewari - Jefferies - Analyst : Okay. Understood. Now maybe just kind of stepping back. And as a part of that kind of revised tweet guidance, I think there's a bit of confusion about Voxogo's trajectory, both into next year and 2027. And this -- I'm just kind of thinking out loud, would you agree -- if I were just to say, I'm very confident, a, Voxogo is going to grow in terms of just net patient adds year-over-year in a manner that's quite similar to 2025. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 18, 2025 / 10:30AM, BMRN.OQ - BioMarin Pharmaceutical Inc at Jefferies Global Healthcare Conference And then number two, in 2027, there are scenarios that we can easily imagine that patient adds in achondroplasia or sorry, hypochondroplasia more than offset any competition you face in achondroplasia. How crazy is that framework for you?

Question: Akash Tewari - Jefferies - Analyst : Understood. And actually, I'm just thinking about this, there seems to be kind of a -- what I find kind of amusing maybe a little sad is, you have other CEOs that are entering in the space, and they are convinced that there's going to be this kind of explosion for their programs. And it almost implies not only is Voxogo's share just going to go to zero in a way that I just don't think maybe makes logical sense. But there's also just that there's just patients out there that we're not seeing, right? From my perspective, there seems to be kind of consistent patient adds we see in achondroplasia. And in a lot of your markets, you're going from kind of a prevalence to an incident type of framework. So you've done a good job talking about 70% of your patients are ex-US. But if you were to tell me, like, roughly speaking, how much of the markets you're going in an incident market where you have a zero to two label versus a prevalence market where there's still kind of room to expand. Help us understand that.

Question: Akash Tewari - Jefferies - Analyst : So I'm going to dig into that, and I think it's quite important as we get into the Ascendas approval. And that decision is coming up soon. Look, again, I have a horse in this race, but I can't just help but think. It's hard for me -- like I look at the multidiscipline review for Voxogo, there are age match analyses that were done. There are ways that the FDA cuts the data and some of those analyses end up reflecting what's in the label. And we're in this kind of, I think, it's a real scenario where if it's 0.3 or it's 1.5 -- 1.3 versus 1.5, there's a perceived meaningful difference in terms of impact to our franchise. Do you feel like there are scenarios where the Ascendis label could show efficacy that looks numerically worse than Voxogo on an H-matched basis. Then Greg, I'd love if you could comment on this in terms of switch. What is the label that you anticipate them getting given that they did run a different clinical trial?

Question: Akash Tewari - Jefferies - Analyst : Okay. So just to hit on that. We got into this question of full approval for Voxogo and final adult height and whenever -- when does that end up getting reached, could you potentially make an argument to the FDA that we shouldn't just look at final adult height when you're thinking about that decision. But maybe more holistically, looking at some of these other kind of secondary biomarker-driven endpoints as a part of a full approval decision? And could you be having that decision with -- that discussion with regulators right now?

Question: Akash Tewari - Jefferies - Analyst : Okay. Understood. Maybe just lastly, there is a regulatory and IP discussion to be had here. You guys have seen my research. I'm personally a little skeptical that you had a citizen's petition that perhaps a citizen's petition would be able to block a competitor product from getting on to the market. And to us, we dealt with this with Jazz and Avadel where you had a once nightly and that still got approved. But I think one of the things that maybe doesn't get caught in that discussion was there are two separate decisions that get made by the FDA, right? There is a separate division that makes a decision on granting both exclusivity and designation. And then there's a separate part of the FDA that actually grants approval. I think if we look back at the Avadel scenario, there was a delay in outright approval as you waited for the actual ODE decision. Is that an angle we should be thinking about here with the citizen position. It's not just as binary as launch or not, but maybe potentially a delay.

Question: Akash Tewari - Jefferies - Analyst : Understood. Now maybe going into hypochondroplasia, can you talk about the size of that population relative to achondroplasia because there are degrees of severity with hypochondroplasia. And then number two, the Dr. Dauber data so far, I mean, it looks just as good in hypochondroplasia as it does in achondroplasia. Can you give us directionally your powering assumptions for that trial and what you expect on average growth, high velocity? And then maybe lastly, what investors love to know is there warehousing? Have you identified patients? Could this be a bolus launch because it's the same doctor. So how immediate is that commercial opportunity for you, let's say, if you get approval in 2027?

Question: Akash Tewari - Jefferies - Analyst : We're out of time. I'm going to (inaudible) one more question, and I think it's quite important, Greg, can you just comment on how to do an apples-to-apples comparison with your DMD corrector data versus the DyniVIDiTY data sets, I think it's quite important.

Question: Akash Tewari - Jefferies - Analyst : Got it. Thank you so much. I appreciate it. Thanks, everyone, for joining us. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 18, 2025 / 10:30AM, BMRN.OQ - BioMarin Pharmaceutical Inc at Jefferies Global Healthcare Conference