BioMarin Pharmaceutical Inc Q4 2024 Earnings Call Summary

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

Question: Philip Nadeau - TD Cowen - Analyst : Good afternoon. Thanks for taking our question. We went to zero in on the VOXZOGO guidance. We're firmly expected to be $200 million year over year. That's healthy, but it is decelerating. Can you talk a bit more on where you expect that growth to come from, and in particular which territories and where they're currently reimbursed or new reimbursement, it will be coming this year and maybe more generally on the pushes and pulls, on the guidance what could cause performance to be better and what could cause it to fall short. Thanks.

Question: Jessica Fye - JPMorgan Chase & Co. - Analyst : Hey guys, good afternoon. Thanks for taking my question. I was wondering if you could spend a little more time talking about your priorities for business development. For example, should we think of BioMarin as more focused on bringing in pipeline assets versus commercial assets to leverage your global footprint and what's your appetite for clinical risk. And maybe just a quick follow up anything you would call out quarter-over-quarter or year-over-year for the first quarter, in the enzyme business like any international ordering patterns to think about there. Thank you.

Question: Cory Kasimov - Evercore ISI - Analyst : Hey, good afternoon, guys. Thanks for taking the question, Greg, maybe another one for you on the DMD program. I believe you've mentioned that steady state dystrophin levels and treated patients are attained at about the 1 year mark. So when you mentioned this 10% goal, is that your expectation for the week 25 biopsies or should they be somewhere below that? Thank you.

Question: Chris Raymond - Piper Sandler & Co. - Analyst : Thanks. Two questions just on the ERT business, you guys have, I think for some time now talked about being able to select, sort of the demand higher on harmonizing diagnostic protocols, I think across all geographies. I know this print was helped along by some government buy-ins, and I heard you, Brian, on your commentary on '25 being sort of back and loaded in terms of growth, but Any sort of color you can give us to the mix of, contribution from this diagnostic harmonization, is this something that's, where are we, I guess, in terms of maybe which ending in terms of being able to affect that that change. And then on 351 data timing, maybe splitting hairs a bit, but the press release says data second half, the slides say mid-'25. Is this, are you indicating maybe you'll be top lining the data in mid-'25 and then presenting the full data at a conference later, any sort of color that would be great. Thanks.

Question: Gena Wang - Barcalys PLC - Analyst : Thank you for taking my questions. I will ask one regarding the IT, for your CMT franchise. I know you mentioned that you already, started processing Europe. Maybe if you can provide some update there and also your strategy in the US. And then the second question is regarding the 349 AATD, you can complete that and also start mad. In the last year, maybe show us what you are looking for, regarding the data update.

Question: Akash Tewari - Jefferies Financial Group, Inc. - Analyst : Hey, thanks so much. Maybe just on BD. I mean, if you think about [Infograib], there is, this kind of potential of an oral therapy that has similar, if not a better efficacy than Voxogo. And I think for a lot of investors, that's a big reason why they have difficulty modeling the terminal value on Voxogo. There are companies like Relay, which, have assets that have the same mechanism as [Infragraib], and I think they're looking for strategic options. What's the appetite for BioMarin to kind of have their own infragratina-like approach in achondroplasia? Is your team very confident that CNP is the only way forward, or is there a potential that, you could be looking to in license one of these assets? Thank you.

Question: Eliana Merle - UBS Group AG - Analyst : Hey guys, thanks so much for taking the question. So for VOXZOGO , do you expect the mix of US and ex US revenues to remain stable, or do you see the contribution from ex US increasing over '25 and '26? And then in the US, I guess what's the proportion of eligible patients with achondroplasia who are not currently treated? And I guess for those patients, why do you think this is? Is it that the patients aren't, currently under care by the right prescribers, or do you expect less penetration in the older patients? I guess what do you see sort of longer term as the potential for uptake and penetration into this population? Thanks.

Question: Kostas Biliouris - BMO Capital Markets - Analyst : Hi everyone, thanks for taking our question and congrats on the strong numbers. Great to see that three-fourth of VOXZOGO sales coming from ex-US. Maybe one question follow up on Akash's question on BB in the in the (technical difficulty) liver and the lung manifestation of the disease, and these approaches already have clinical validation, early clinical validation. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 19, 2025 / 9:30PM, BMRN.OQ - Q4 2024 BioMarin Pharmaceutical Inc Earnings Call Given your interest in that space, would you consider different modalities to complement your pipeline modalities, or you prefer to go only with one modality in that disease? Thank you.

Question: Kostas Biliouris - BMO Capital Markets - Analyst : I was asking about RNA editing approaches in alpha ones inefficiency and whether you would have interest in, such a process to complement your approach in your pipeline.

Question: Olivia Brayer - Cantor Fitzgerald & Co. - Analyst : Hi, good afternoon. Thank you for the question. How are you guys thinking about the level of growth that we might see from the enzyme business over these next couple of years? I know you've talked about a high single digit keger over the next 10 or so years, but what about for '25, '26, '27? And then any comments around what the margin expansion could look like for that enzyme franchise? Just especially considering, patients getting older and some of these medicines are weight-based. Thank you.

Question: Alex Hammond - Wolfe Research, LLC - Analyst : Thanks for squeezing me in. Just quickly on VOXZOGO , can you provide any color on the expected degree of switch market dynamics following potential competitor launches? And just to follow up on that as well, can you kind of dimensionalize how those dynamics may differ across geographies as well as patient age groups?