BioMarin Pharmaceutical Inc Q2 2025 Earnings Call Summary

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

Question: Cory Kasimov - Evercore Inc - Analyst : Good afternoon, guys. Thanks for taking the question and great to see the continued execution and the preliminary 333 data. On the achondroplasia front, I'd be interested in your thoughts on the evolving competitive landscape here, with the recent data showing a long-acting CNP in combination with growth hormone and how you think that could impact the market down the road. Thank you. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. AUGUST 04, 2025 / 8:30PM, BMRN.OQ - Q2 2025 BioMarin Pharmaceutical Inc Earnings Call

Question: Cory Kasimov - Evercore Inc - Analyst : That's helpful. Thank you, Greg.

Question: Salveen Richter - Goldman Sachs Group Inc - Analyst : Thanks for taking our question. This is [Tommy] on for Salveen. Just on the VOXZOGO guidance, which was slightly adjusted, could you lay out the contributions of the various factors if you think about ex-US order timing, demand, and your US expansion initiatives? Thank you.

Question: Akash Tewari - Jefferies LLC - Analyst : Hey, thanks so much. Can you just give us a little more detail about the design of that 333 superiority trial? Because when I look at the VOXZOGO Phase 3, you had about 55 patients per arm. You're 90% power to get a 1.75% average growth velocity delta. I feel like if you have the same powering per arm and you're going for superiority, you're probably looking at like a 0.8 to 1 centimeter improvement between 333 and VOXZOGO for that trial to hit on superiority. Is that the right way to think about efficacy? And like what gives you the confidence you can do that? And then maybe number two, can you talk about the potential to pursue maybe beyond weekly dosing with the profile that you showed in healthy volunteers? Thank you.

Question: Shawn Lemon - Morgan Stanley - Analyst : Good afternoon, everyone, and thank you for taking my question. Hope everyone's well. Just thinking a little forward to your $4 billion revenue aspirations in 2027, I think it is. Just wondering how important other indications for VOXZOGO are to that and what sort of contribution do you think they might make. And, even going beyond hypochondroplasia, indications such as Noonan and Turner, when might we expect those? Thank you.

Question: Gena Wang - Barclays Services Corp - Analyst : Thank you for taking my questions. I also will ask about the 333 program. So Greg, you said that now you already dosed the six cohorts. What are your thoughts when you're going to the patient population? Is the aim to maintain area under the curve over 3 times, any upper limit you will be looking for regarding the safety? And then, if you can remind us, the VOXZOGO low dose, high dose, what was the area under the curve comparison? We do understand the CMAX could be very different. But when you look at the area under the curve, what was the difference between the low dose and the high dose of VOXZOGO?

Question: Jasmine Fels - UBS AG - Analyst : Hey, this is Jasmine on for Elle. Thanks so much for taking your question. Just another one on 333, trying to understand what the 3 times greater AUC reading means. From your preclinical work, can you give any more color on what this level of CNP exposure translates to in terms of growth? And then secondly, given the data that you've now seen, how does this impact your thinking on prioritizing 333 versus VOXZOGO for other growth disorders going forward? Thanks.

Question: Jason Gerberry - Bofa Merrill Lynch Asset Holdings Inc - Analyst : Hey guys. Thanks for taking my question. Wanted to follow up on the HCH opportunity and the commentary about getting to the patient earlier. I guess we observed with ACH or achondroplasia that getting the below five indication was really key commercially in a number of geographies. And with HCH, at least in the prior Phase 2 work, I think the average age was about six years of age or enrollment criteria is three years. So I just wonder, given it's a milder disease, the motivation to treat and sort of the risk, I guess from a patient perspective, that they're going to come on later in life and may not get the sort of lifetime benefit that they see in HCH. So just wondering if you can maybe expound upon some of those efforts and how you see that evolving. Thanks.

Question: Ellen Horste, Ph.D. - Cowen and Company LLC - Analyst : Thanks for hearing my question and congrats on the exciting quarter. Regarding BMN 401, I'm wondering what success looks like for the ENERGY 3 trial, including potential functional endpoints that might be required for approval outside the US. And then if you can remind us how many patients have been identified so far and what's involved with identifying the 2,000 to 2,500 prevalence you estimate in your territory. Thanks.

Question: Allison Bratzel - Piper Jaffray Inc - Analyst : Good afternoon and thanks for taking the question. Another one on BMN 333. Just a clarification. And I'm sorry if I missed it, but can you help us better understand what exactly is gating to initiation of the dose ranging portion of the registration trial? And I think you've received FDA alignment for the Phase 2/3 plans, but could you just talk to your confidence in achieving regulatory alignment ex-US and timing there? Thank you.