BioMarin Pharmaceutical Inc Q3 2025 Earnings Call Transcript

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

Question: Philip Nadeau - Cowen and Company LLC - Analyst : Hi, thanks for taking our question. Our question is on the 2027 guidance. Can you talk a little bit more about the scenarios you see? And maybe more specifically, why are you rescinding the guidance now? What has changed over the last year since it was initially issued? Thank you.

Question: Philip Nadeau - Cowen and Company LLC - Analyst : And maybe just a follow-up. I think when you issued the guidance a year ago, you were -- you suggested that competition aspect into the guidance. Do you now have a different appreciation or a different concern about how that share that competition can take, specifically specifically against VOXZOGO. Thank you.

Question: Salveen Richter - Goldman Sachs Group Inc - Analyst : Good afternoon. Thanks for taking my questions. Can you speak to why VOXZOGO sales were down quarter-over-quarter? And then also -- just help us understand here the business development strategy, just given that there seems to be so much of a focus on VOXZOGO and how that plays out, but you're kind of stuck from just given all these various dynamics competitively, when can we start to see that business development lever or levers kind of emerge to really add to your portfolio? Thank you.

Question: Cory Kasimov - Evercore Inc - Analyst : Hey, good afternoon, guys. Thanks for taking my question. I guess I have a follow-up on two questions that were asked. First of all, Brian, I appreciate the commentary you made on the previously issued '27 guidance. I think the way you framed it is helpful. However, I'm curious if you have any qualitative commentary, you could also share in your prior long-term mid-2030s guidance with regard both to the opportunity for VOXZOGO as well as the anticipated CAGR for the Enzyme Therapies business? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. OCTOBER 27, 2025 / 8:30PM, BMRN.OQ - Q3 2025 BioMarin Pharmaceutical Inc Earnings Call And then a follow-up for Alexander's capital deployment commentary. Given the pretty big cash balance and good operating cash flow generation you alluded to, have you given much consideration to share buybacks? Or are you really just holding that capital for other uses like BDs you were talking about? Thank you.

Question: Joseph Schwartz - Leerink Partners LLC - Analyst : Great. Thanks very much. For the upcoming BMN 333 PK data, what specific exposure levels would give you confidence that you can achieve clinical superiority over VOXZOGO? And as you move into a head-to-head superior trial against VOXZOGO, what is the minimum annualized growth philosophy delta over VOXZOGO that you believe could be required to demonstrate clear clinical superiority, drive patient switching and reestablish a standard of care in the face of potential competition?

Question: Jessica Fye - JPMorgan Chase & Co - Analyst : Hey guys, good afternoon. Thanks for taking my question. I had a couple on the guidance and a couple on the pipeline. On the guidance, I don't have FactSet. What is the 2027 FactSet consensus, excluding Roctavian just we know what that lower bound is? And then the second one on the guidance -- maybe just asking about the other two elements of the longer-term targets that I don't think Cory mentioned. Should we still expect 40% non-GAAP operating margin starting in '26 that could expand to the low to mid-40s and the greater than $1.25 billion of CFO starting in 2027? Or were those sort of top line dependent and more in question now? And then the two on the pipeline. For 351, my understanding is look at six-month biopsy data for the 6-milligram cohort. What should we expect for the 9-milligram cohort? And second one, the pipeline is for hypochondroplasia, can you remind us of the powering for that trial? And is that sig, sufficient in your mind? Or is there some minimum delta on efficacy you want to see to meet your target product profile?

Question: Christopher Raymond - Raymond James - Analyst : Hey, thanks, for taking the question. Just two actually for me. Brian, I heard what you said about '27 not being guidance, just to sort of range of outcomes. But so I won't say it this way. But -- is it safe to say you're more concerned about TransCon CNP than infigratinib? And I guess it is -- when you talk about that FactSet number being sort of the low end, is it your assumption that Ascendis gets first cycle approval with just one year's worth of data when their PDUFA date comes next month? Or does that even -- does that factor into your thinking? I know it's a little bit removed from 2027, but just kind of are you getting that granular in your thinking? And then maybe an M&A question, Alexander. You guys; talk about wanting assets that are under resourced and could be better served by the BioMarin infrastructure. For the Enzyme asset 401, can you maybe talk about how you have leveraged and approved upon Enzyme's efforts in terms of patient identification, outreach, other things that you've done to make that asset better? Thanks.

Question: Sean Laaman - Morgan Stanley - Analyst : Hi, Alex and team. Hope everyone's well and thanks for taking my questions. Just get your latest thoughts on orphan drug exclusivity, kids under five and what you think about the potential switching to a competitor is the first one. And the second one, if I'm getting a narrative for without business development, BioMarin, a capital accumulator. Just to get your thoughts on what you think your balance sheet capacity is and what you see as an efficient balance sheet structure?

Question: Sean Laaman - Morgan Stanley - Analyst : Sure. It was.