BioMarin Pharmaceutical Inc. Q4 FY2025 earnings call

• 2025 total revenues grew 13% to record $3.22B, driven by 9% enzyme therapies growth and 26% Voxzogo growth. Enzyme therapies have 9% CAGR over 5 years. • Acquisitions: Inozyme strengthens enzyme therapies portfolio with BMN 401 for ENPP1 deficiency; Amicus acquisition expected Q2 2026 adds Galafold, Pombiliti, Opfolda to commercial portfolio. • Voxzogo potential indication expansion for hypochondroplasia, with pivotal results expected soon, and Phase II/III study of BMN 333 to begin enrollment. • Enzyme therapies in 2025 had year-over-year growth across all products, Palynziq outperformed with 22% growth. 2026 Palynziq expected growth supported by adolescent-label expansion. • Voxzogo 2025 had 26% growth, ~75% revenue outside US, growth strategy focuses on new starts globally, deep penetration in existing and new markets. • R&D: Preparing to submit full approval package for Voxzogo in achondroplasia, BMN 333 Phase II/III study design with 90% power to detect 50% growth increase vs Voxzogo, Phase III data readouts expected for Voxzogo in hypochondroplasia and BMN 401 in ENPP1 deficiency, Palynziq sBLA action date Feb 28, BMN 351 Duchenne muscular dystrophy data to be presented at MDA meeting.

In 2025, total revenues grew 13% to $3.22 billion. Enzyme therapies revenue grew 9%, with Palynziq up 22% and Vimizim up 7%. Voxzogo revenues rose 26%, with ~73% of its $927 million revenue in 2025 generated outside the US. Fourth quarter 2025 total revenues were $875 million, 17% y-o-y growth, with Voxzogo up 31% and enzyme therapies up 13%. Roctavian withdrawal led to ~$240 million special items in Q4. Full year 2025 non-GAAP diluted EPS was $3.15, underlying business EPS grew ~34%, and operating cash flow was $828 million, 45% y-o-y increase. 2026 guidance: enzyme therapies revenue $2.225 - $2.275B, Voxzogo revenue $975M - $1.025B, total revenues $3.325 - $3.425B, non-GAAP diluted EPS $4.95 - $5.15, underlying organic operating margin ~40% before Amicus, slightly below 40% after Amicus close. Q1 2026 expected lowest revenue quarter, second half 2026 and Q4 expected higher revenue due to international order timing.

• 2026 enzyme therapies revenue expected $2.225B - $2.275B, Voxzogo revenue $975M - $1.025B. • Total 2026 revenues expected $3.325B - $3.425B, excluding Amicus contributions. • 2026 non-GAAP diluted EPS $4.95 - $5.15. • Underlying organic operating margin ~40% before Amicus, slightly below 40% after Amicus close. • Q1 2026 expected lowest revenue quarter, second half 2026 and Q4 expected higher revenue due to international order timing. • Guidance reflects preclose integration costs and interest expense related to Amicus transaction. • Confidence in underlying business profitability growth outpacing top line growth.

Q: Congrats on all the progress. Just one point just -- and the dynamic in the achondroplasia market now that we have data for the oral medicine also out there, so how do you see this market evolving with the availability of weekly as well as oral at some point? And then the last part of the question is basically, if you do deliver 2.25 centimeters or above growth velocity, above -- over and above placebo with BMN 333. Do you think injections could be the first-line therapy there before orals?

A: Thank you, Mohit. This is Greg Friberg. I think I'll take the first half of your question, and then I'll hand it off to Cristin. With regard to the recently released data for the FGFR3 inhibitor, our take there, of course, was that this was generally comparable to other CNP class effects at 1 year. Of course, this is 1-year data. We'll need to see both durability as well as safety data over time. And the read through, of course, in terms of how we believe Voxzogo can deliver value here is that Voxzogo not just having one year of data has a tremendous amount of supporting evidence behind it. We talk about the 10,000 patient years of safety. We also, of course, have a deep a wealth of information with regard to evidence beyond height, whether it's foramen magnum, physical function, tibial bowing, quality of life, all of this data, again, creates a real confidence that, that data is something that patients can rely on. With regard to 333 and the effect size, we've stated again what the powering assumptions would be, and we do believe that the 2.25 centimeters of growth not only are differentiating on a clinical level, but we have some preliminary market research that suggests as well that, that would be important. It's important not to get too caught up in the growth being the outcome that we care most about because the growth is a surrogate for the health and wellness of these patients. Again, going back to Voxzogo, we do believe that increase in growth would, in all likelihood, again, lead to a best-in-disease profile that patients, payers and physicians with value. So with regard to the market dynamics, I'm going to hand it off to Cristin.

Q: I'm hearing你 guys on the switcher dynamic and the comment that if patients are doing well, there will be a limited motivation to switch, but we were kind of surprised to learn one of your competitors is anticipating about half of their patients in their early achondroplasia trials will be Voxzogo experienced. And I think their commentary is that there actually is a decent amount of parents and patients out there that are interested in switching to something. Maybe just talk about what you're seeing more broadly. Is there a demographic or some attribute other than just performance that might drive this decision or desire to switch? And then if I can maybe ask a question on 351. I'm kind of struck by -- this drug is mentioned in your commentary, but I'm just looking at the dystrophin data, it looks pretty differentiated, if not superior to what's out there. Maybe just talk a little bit about your plan to communicate this. I know there's data, the full data is going to be at the MDA conference next month, but just talk about your plan to communicate the waypoints on that drug, please?

A: Thanks so much, Chris, and this is Greg Friberg, again. Let me take your second question first. I would agree with you. The data is quite encouraging from BMN 351. The 5% absolute dystrophin measure, again, which if you look at our PKPD model predicts roughly 10% at steady state, that is an unprecedented number for the exon 51 skip amenable patients. Now what we also know from the program is that data monitoring committee has allowed us, again, to go to a higher dose level and complete the study. So we see quite a bit of value to continuing the experiment for a variety of reasons. Number one, of course, the 12-milligram per kilogram level, we'll see whether we can see something superior to that number, knowing that within all these patients, there's some degree of variability. Number two, we'll obviously get to see more chronic safety data as well. And this is something, again, that in this field, of course, where benefit burden is key is something that we absolutely want to continue to follow. And finally, we'll get our first look at some functional data as well as we treat these patients for longer and look at, for example, the Stride Velocity 95C data. We expect that this next tranche of data will have it before the end of the year. And in the meantime, we will be presenting our complete 6- and 9-milligram per kilogram data at the Duchenne Muscular Dystrophy Association meeting in March, just a couple of weeks away. So we're sitting tight. And again, encouraged by what we've seen. We want to see more data there for this weekly IV administered antisense oligonucleotide. With regard to Voxzogo, your question is a nuanced one. It's one of whether or not, again, patients, physicians, their caretakers, whether they can have the confidence in the data package for the medicine that they're given in many cases, to very, very young infants. And of course, with Voxzogo, we have a wealth of data. We're looking forward to present that in our full evidence package that we'll be presenting for full approval to the FDA in the coming months. And I think I've gone through some of the package there, that goes through, again, some of those data points. With that, though, from the commercial perspective, I want to give Cristin, a chance to share her perspective.

Q: Another question on Voxzogo, but on hypochondroplasia. Can you help us frame the upcoming results? What magnitude of growth velocity increase do you think would be meaningful in hypochondroplasia patients? And can you talk about the dynamics of that market? Would you expect uptake as quick as in achondroplasia? Or are there other factors which could make it less rapid?

A: Thanks, Phil. This is Greg again. I'm going to take it for stab, and again, I hand it to Cristin at that point. With regard to the study, again, we're very excited that we'll be turning the card over in the first half of this year. The study is designed to measure an effect size roughly equivalent to what Voxzogo delivers for achondroplasia. That being said, that's a fairly conservative assumption. We know that Dr. Dauber's data, for example, is showing slightly larger, more like a 1.8-centimeter growth of AGV in the hypochondroplasia patients. And it's our goal and intent also to follow through, as I mentioned previously, not just measuring AGV, but also looking at measures of health and wellness for these patients. So we believe we've designed the right study. Now it's a question of reaching the endpoint and turning it over, and we're very excited to see the data. Cristin?

Q: This is Siyue Wang on for Akash. One on Voxzogo again, just following on the infigratinib data, where does this fall in your scenario analysis for your 2027 revenue outlook? And additionally, can you talk about the importance of the full approval for Voxzogo with the final adult height and how you expect that to impact patient preference?

A: This is Brian. I'll take the first part of the question on '27 and any impact from infigratinib data. We don't have new updates on '27 at this time. I will say in response to your question, that the assumptions we made in the more competition impacted scenarios when we did update our views on that range, this is in line, we assumed two competitors and comparable data to Voxzogo.

Q: We just wanted to drill a little bit more into the factors assumed in your Voxzogo guidance, especially as it relates to new starts and potential switching in the context of upcoming competition. And also for 333, kind of your confidence in establishing as the preferred option for new patients from switching given that the long-acting competitor will have a bit of a head start here.

A: Tommie, it's Brian. I'll start with the first part of your question. Thank you. In terms of the Voxzogo guidance for '26, first of all, just to note, we're really pleased to have grown the product 26% in '25. Its fourth year on the market. And with respect to the range, the $975 million to $1.025 billion really just reflects a handful of scenarios. On the lower end of that range, you can assume a stronger competitive impact from the first competitor potentially coming to market in '27. I'll also share that we're being guarded on a couple of routine market access renegotiations in '26. These are normal course of business. Again, the product is fifth year on the market. Those will resolve this year, but we're being measured on those. And then at the high end of the range, it would just assume a lesser impact from those couple of swing factors in the guidance.

Q: Just what's your latest expected timing for the data readouts from the Phase II CANOPY basket study for Voxzogo? And second, just to ask a bit more on the Voxzogo guidance. Could you comment specifically on what the guidance reflects or how you're thinking about U.S. growth versus ex-U.S. growth over the course of the year? And then if you could just elaborate on that last comment that you're being guarded on a few routine market access conversations and sort of the implications for that for Voxzogo?

A: And this is Greg. Maybe I'll knock down the CANOPY question first, if that's okay. We're anticipating that we're on track for data in the 2027 time frame, as we've stated previously. No updates in that regard. And that refers to IFS, and the Noonan, Turner, SHOX program. Our CANOPY study, of course, also includes hypochondroplasia, which we're excited to turn the card over in the first half of this year.

Q: So one of the levers you highlighted for longer-term growth when you announced the Amicus acquisition was some initiatives to identify patients who might have Fabry and Pompe that wasn't diagnosed yet. I think Amicus had some pilot programs to do this. And in a few centers -- and I was just wondering if BioMarin had the opportunity to see the results of these pilot programs and if they are along the lines of what you expect to roll out. Can you just talk a little bit about your initiatives there?

A: Thanks for the question, Joe, it's Cristin. So you are indeed exactly what you just said that there are -- they've been running pilots. There's different models. And just to reiterate that we are in the process now of really diving between sign to close, really understanding what's going on, what makes sense, given what we already have in our company versus going on over there. And at the end of the day, we are running as two independent businesses at this point in time. And so they're continuing their work on any of the pilots that they have ongoing.

Q: I have a few follow-ups on Voxzogo. First is just maybe can you talk about level of confidence in actually hitting the numbers that you laid out this year? Just in light of a weaker first quarter due to those ordering patterns that you highlighted, Brian. Anything more you can tell us on those ordering patterns, and why they might be so heavily weighted to the fourth quarter this year. I'm just trying to understand how having a new entrant on the market could impact some of those patterns and just cadence of quarterly Voxzogo revenue this year? And then a quick follow-up on hypochondroplasia. When do you think we could realistically see that launch and uptake in those patients? And any comments at this point just around how quickly you could start to see added sales from hypochondroplasia patients especially as you think about navigating some of the competitive headwinds in the achondroplasia market next year.

A: Thanks, Olivia, this is Brian. I'll start with a little more color on the quarter. I appreciate the question. Yes, so a few brief things there. First of all, we've always experienced these kind of bolus orders and the potential volatility of quarterly revenue. And by the way, we've typically seen step-down from Q4 to Q1. We do -- I think we -- especially after this year, we definitely observed some Q4 buying globally. It isn't always inventory levels. It could be, in some cases, of single national payers kind of just making their way through their budgets. There's a number of factors. But we've seen this in Q4, it was -- seen this before for Q4 was exacerbated here in '25.

Q: Just on the Phase III powering in the 2.25 centimeter per year. Is that a threshold effect when you're doing your Phase IIs ranging, if you're learning that perhaps that may be too ambitious of a target, would you just kind of adjust your statistical analysis plan. Is it -- I think in your earlier comments, not to get too hung up, I think, on that 2.25 number. So if it looks like你're on a trajectory for, say, 2 or 2.15, is it still worth moving forward in the Phase III with BMN 333. So I just wanted to clarify if that's the threshold effect in a go no-go threshold in the dose-ranging work?

A: Yes. Thanks for the question, Jason. And that 2.25 really comes from the sample size that we've laid out, that's 60 versus 60. There's some standard deviation, some alpha numbers behind that as well. But we wanted to share with you again what our ambition was, what we thought not only statistically significant but clinically meaningful. We will have a chance to look at our Phase II data. It's not a straightforward black-or-white situation. We're doing actually a Bayesian approach where we'll be looking at the totality of the data. And setting a certain threshold for confidence to move forward. Rather than get into the details there, I will just answer your question directly and say we would have the opportunity to potentially modify the protocol thereafter.