BioMarin Pharmaceutical Inc at Wells Fargo Healthcare Conference Summary

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

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : <_ALACRA_META_ABSTRACT>All right, thank you very much for joining us today. My name is Mohit Bansal. I'm one of the Biotech Pharma analysts here at Wells Fargo, and this is my first session of the morning. And I'm very happy to have from BioMarin, Greg Freiberg, with us here. Greg is the EVP and Chief Research and Development Officer at BioMarin. Thank you very much for being here. Gregory Friberg - BioMarin Pharmaceutical Inc - Executive Vice President, Chief Research & Development Officer Yeah, thanks for the opportunity to join you.

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : It has been a year, right? So yeah, I mean, I think BioMarin from the science point of view is a great company. I mean, so many rare disease drugs you have developed. So can you talk a little bit about your experience in the last one year? And what investors should look forward to from the R&D engine side of the company?

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Great. Thank you very much for this overview. One question I want to ask is that, in the last 18 months, the company has gone through a lot of, rationalization of R&D in terms of what makes sense, what doesn't make sense, and you were part of some of those processes, and some were in place before you joined as well. So can you walk us through like what -- when you look at prioritizing an asset and deprioritizing, what do you really do?

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Got it. So I mean, on that -- I mean, something that can really be helpful for the patients. I mean, 333, the recent data you generated, it wasn't healthy volunteers. But at the same time, can you talk a little bit about the significance of that? Also like I mean you have VOXZOGO, but then there is a longer acting CNP competition coming so in the context of how does fee could be in four to five years? What is the -- what is the significance of these data?

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Got it. So let's just -- I want to talk a little bit about the AUC aspect of it. Do we know? So when you say 3x of AUC, it is compared to the long-acting CNP, not the VOXZOGO, right?

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Got it. So did you see higher AUC for the longer-acting CNP, the competitor CNP versus VOXZOGO?

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Got it. So this is the other question, is that these patients have at least the trial, you enroll about 4 centimeters of growth velocity by year on the baseline. The baseline, and these drugs can actually get to 1.5 centimeters extra per year. How much more you can extract out of this mechanism? Could it be like 2, 2.5 centimeters? Like what is the normal, 6, 6.5?

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Got it. And I want to bring in the question about the growth hormones as well. So I mean, I think competitor shared some data in combination with growth hormones. Basically, the thought process, the new growth hormones, the way they work, they actually give you a nice growth initially, right? And then -- and that's -- like you can see the difference, while VOXZOGO and all, I mean, because of the mechanism is like over a slope of time. Do you see a reason to use a CNP in combination with growth hormones or?

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Makes sense actually.

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Of course. Just talk a little bit about the plans for registrational study. I mean, you are planning to start it in next year.

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : For 333. You are talking about there could be a superior profile to VOXZOGO, but again...

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : That's the ambition. So how do you design a trial like that? And do you go for CNP-naive patients? You go for experienced patients? Because I mean, by that time, there will be a lot of patients already on CNP, right? So like I say, it reminds me of Alexion, right, back in the day, they were trying to develop (inaudible) 100%, right? So how do you think about this?

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : And regulatory landscape seems to have changed as well around one year now, right? I mean, so people -- FDA is probably looking for one year at this point rather than two years, or?

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst :

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Great. So how about we talk a little bit about like moving away from 333 now and talk about the other new assets, the DMD assets at this point? So I think you are trying to share some data by end of this year. So can you talk a little bit about what to expect there? What data will be shared?

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Awesome. So this will be a 6-month data. The goal is to get to 10% by 1 year. So this -- you should not expect 10% here?

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : 3% to 6% is what you are looking for. And Dyne has showed 3.3% at the same timeframe, roughly?

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Got it.

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Got it. And for Stride Velocity, we should not expect anything at six months in terms of...

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Better than six-minute walk as well?

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Got it. Very helpful. So can you help remind us what did [10%] show at that time from...

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Got it. Very helpful. And then what about the safety side of it, right? I mean, it should have the ASO level safety or...

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Got it. That's very helpful. Maybe last few minutes. So quickly, if I want to talk about how should we think about the hypochondroplasia data when they come out? What is the bar for success? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 04, 2025 / 12:45PM, BMRN.OQ - BioMarin Pharmaceutical Inc at Wells Fargo Healthcare Conference

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Awesome. So a similar topic for BMN 5401, like we'll have data for the top line next year. So I mean what should we expect there?

Question: Mohit Bansal - Wells Fargo Securities LLC - Analyst : Awesome. On that high note, thank you very much for joining us. You have a lot going on.