BioMarin Pharmaceutical Inc Q1 2025 Earnings Call Summary

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

Question: Philip Nadeau - TD Cowen - Analyst : Two financial ones. First on VOXZOGO, you've mentioned a couple of times about H2 being larger than H1 and also the revenue in the course through Q2 being relatively similar. I'm just curious whether -- does that mean a decline in Q2 revenue? It does look like revenue grew a bit from Q4 to Q1. So if revenue is approximately intermediate between those two, maybe there'll be a down quarter, Q2? And then second financial question on exposure to tariffs. I think your guidance as to the current tariff situation is very clear. Could you give us some sense of how your financials could be impacted should the US impose a 25% tariff on EU pharmaceuticals? And then -- maybe should that situation be retaliated against with a 25% tariff from the EU on the US?

Question: Tommie Reerink - Goldman Sachs - Analyst : Congrats on all the progress. This is Tommie on for Salveen. Just wondering if we can get an update on your interest and maybe timing for BD after all of the meetings that you had in January? And when we think about the data that you'll give us for BMN 333 this year, can you maybe characterize the level of detail we'll get before you kind of present the full data at the Congress next year?

Question: Jessica Fye - JPMorgan - Analyst : So BioMarin continues to execute across the business. However, the market seems worried about long-term competition for VOXZOGO. Can you talk about how you think you can best address the market's perception of VOXZOGO's long-term value, particularly beyond 2030?

Question: Jessica Fye - JPMorgan - Analyst : Should we expect the same endpoint for 333 in a pivotal trial as for VOXZOGO?

Question: Paul Matteis - Stifel Nicolaus and Company, Incorporated - Analyst : I had one for Brian and one clinical question. Brian, I wanted to just clarify as it relates to VOXZOGO over the past couple of quarters and what you're seeing going into 2Q, has the net price on a global basis of the drug changed? Just curious if their price has gone down a bit as either diversified into other markets and your expectation there? And then as it relates to the 333 pivotal, that was super interesting to kind of get the color on a comparative design. Are you looking to actually show superiority? Is it going to be powerful noninferiority? I mean maybe it's too early to say, but given your conviction that higher exposure may drive greater efficacy, what do you see the opportunity to show there?

Question: Gena Wang - Barclays - Analyst : I have two sets of questions. First one is regarding also the VOXZOGO franchise. So maybe the 333, can you help us understand because you have a healthy volunteer data and mainly is the PK data, how do you define that will be the top line data you shared with us, that will be good enough that defined as a supportive to start your registration trial? And will you only identify one dose for your registration trial? And then a related question, I know there is a competitor running [CNP] plus growth hormone studies. Any thoughts there? We know historically, growth hormone didn't work. So maybe any thoughts there regarding CNP plus growth human study? And the third part, very quickly on the manufacturing side for VOXZOGO, do you have a manufacturing sites both in the US and the ex-US?

Question: Kostas Biliouris - BMO Capital Markets - Analyst : Congrats on the progress. Maybe one question for Greg on BMN 333. There is a recent presentation by Children's National Hospital at ESPE that shows that there is no really meaningful correlation between VOXZOGO PK and growth outcomes in hypochondroplasia. So given this data, I wonder to what extent will we be able to draw conclusions about BMN 333 outcomes based on the PK data that we will see in 2025?

Question: Olivia Brayer - Cantor Fitzgerald - Analyst : Greg, just wanted to clarify a comment you made earlier. Will we not see any level of Phase II data from other short stature indications until 2027? Or could we actually start to see some initial clinical characterization and maybe select patient groups, just to help derisk some of those new VOXZOGO indications? And then on the P&L, you guys are on track for over 20% EPS growth this year. Any comments on what kind of earnings power you think you could have as we get into the next few years?

Question: Vikram Purohit - Morgan Stanley - Analyst : Thanks for fitting me in. We just had two quick clarification questions on BMN 333. Apologies if these topics were discussed and we missed it, but just to make sure we understand. So for the pivotal program plan to initiate in 2026, would the bar for approval necessarily be superior efficacy versus VOXZOGO through the comparative effectiveness study that you're planning to run? And then secondly, could you talk a bit about your plans to seek approval and define a plan forward ex US as well?