Earnings call transcript: BioMarin beats Q1 2025 forecasts, stock rises after hours

BioMarin Pharmaceutical Inc. (BMRN) reported robust financial results for the first quarter of 2025, surpassing Wall Street’s expectations with both its earnings and revenue figures. The company posted earnings per share (EPS) of $1.13, significantly beating the forecasted $0.70. Revenue for the quarter reached $745 million, exceeding the anticipated $741.02 million. Following these results, BioMarin’s stock saw a rise of 0.8% in after-hours trading, closing at $63.21. According to InvestingPro analysis, the company maintains a "GREAT" financial health score of 3.52, demonstrating strong operational fundamentals.

Want deeper insights? InvestingPro subscribers have access to 7 additional expert tips about BioMarin’s financial position and growth potential.

Key Takeaways

• BioMarin’s Q1 2025 EPS of $1.13 outperformed the forecast by 61%.
• Revenue grew 15% year-over-year, reaching $745 million.
• After-hours trading reflected a 0.8% increase in stock price.
• VOXZOGO’s global revenue surged by 40% year-over-year.
• Operating cash flow increased by 271% from the previous year.

Company Performance

BioMarin demonstrated strong financial health in Q1 2025, with a notable 15% increase in total revenue compared to the same period last year. The company’s growth was driven by its rare disease treatment portfolio, particularly VOXZOGO, which saw a significant rise in global revenue. With an impressive gross profit margin of 79.67% and a five-year revenue CAGR of 11%, BioMarin’s strategic focus on genetically defined conditions and its limited exposure to Medicare have provided a buffer against broader macroeconomic challenges.

Financial Highlights

• Revenue: $745 million, up 15% year-over-year
• Earnings per share: $1.13, up 59% year-over-year
• Operating margin: 35.7%, expanded by 11.9 percentage points
• Operating cash flow: $174 million, up 271% from Q1 2024

Earnings vs. Forecast

BioMarin’s Q1 2025 earnings exceeded analyst expectations, with EPS of $1.13 compared to the forecasted $0.70, marking a 61% surprise. Revenue also surpassed projections, coming in at $745 million versus the expected $741.02 million. This performance indicates a strong start to the year, continuing the company’s trend of outperforming market expectations.

Market Reaction

Following the earnings announcement, BioMarin’s stock experienced a modest increase in after-hours trading, rising by 0.8% to $63.21. This movement contrasts with the stock’s decline of 1.54% during regular trading hours, reflecting renewed investor confidence in the company’s growth trajectory. The stock remains below its 52-week high of $94.85, with analyst targets ranging from $65 to $126, suggesting potential upside. InvestingPro’s Fair Value analysis indicates the stock is currently undervalued.

Outlook & Guidance

BioMarin remains optimistic about its future prospects, projecting full-year VOXZOGO revenue between $900 million and $950 million, representing a 26% growth. The company aims to achieve a $4 billion revenue target by 2027, with a focus on expanding its operating margin to 40% and beyond. These goals are supported by ongoing product innovation and strategic business development initiatives.

Executive Commentary

"We are well positioned to manage through ongoing market uncertainty based on the global demand for our innovative medicines," said Alexander Hardy, CEO of BioMarin. Greg Fryberg, Chief R&D Officer, added, "Our goal with BMN 333 isn’t just a more convenient version of CNP, it’s to actually increase the free CNP exposure that we can reach in patients."

Risks and Challenges

• Potential pharmaceutical tariff impacts could affect international revenue.
• Market saturation in key regions may slow growth.
• Regulatory hurdles for new product approvals could delay launches.
• Exchange rate fluctuations pose a risk to overseas earnings.
• Competitive pressures in the rare disease treatment market remain high.

Q&A

During the earnings call, analysts inquired about the potential impacts of pharmaceutical tariffs and the dynamics driving VOXZOGO’s revenue growth. Executives also detailed their development plans for BMN 333 and other pipeline assets, emphasizing the importance of safety in their Duchenne muscular dystrophy program.

Full transcript - Biomarin Pharmaceutical Inc (BMRN) Q1 2025:

Conference Operator: Good day, everyone, and welcome to the BioMarin Pharmaceutical First Quarter twenty twenty five Conference Call. Just a reminder that this conference is being recorded. I would now like to hand things over to Ms. Tracy McCarty. Please go ahead, ma’am.

Tracy McCarty, Investor Relations, BioMarin Pharmaceutical: Thank you, operator. To remind you, this non confidential presentation contains forward looking statements about the business prospects of BioMarin Pharmaceutical Inc, including expectations regarding BioMarin’s financial performance, commercial products, and potential future products in different areas of therapeutic research and development. Results may differ materially depending on the progress of BioMarin’s product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market and developments by competitors and those factors detailed in BioMarin’s filings with the Securities and Exchange Commission such as 10 Q, 10 ks and eight ks reports. In addition, we will use non GAAP financial measures as defined in Regulation G during the call today. These non GAAP measures should not be considered in isolation from, as substitutes for, or superior to financial measures prepared in accordance with U.

S. GAAP, and you can find the related reconciliations to U. S. GAAP in the earnings press release and earnings presentation, both of which are available in the Investor Relations section of our website. Please note that our commentary on today’s call will focus on non GAAP financial measures, unless otherwise indicated.

On the call from BioMarin management today are Alexander Hardy, President and Chief Executive Officer Brian Mueller, Executive Vice President, Chief Financial Officer Kristen Hubbard, Executive Vice President, Chief Commercial Officer and Greg Fryberg, Executive Vice President, Chief R and D Officer. I will now turn the call over to BioMarin’s President and CEO, Alexander Hardy.

Alexander Hardy, President and Chief Executive Officer, BioMarin Pharmaceutical: Thank you, Tracy, and good afternoon, everyone. Thank you for joining us today for our first quarter twenty twenty five results update. We are pleased to have delivered strong financial performance and revenue growth of 15% in the quarter. On the bottom line, non GAAP earnings per share of $1.13 represented a 59% year over year increase, reflecting significant profitability expansion, nearly four times the rate of top line growth. This performance highlights the fundamental value of our transformative medicines and the strength of our business, both of which are key attributes that distinguish BioMarin.

These results pave the way for record full year performance in 2025 and boost cash generation for reinvestment in innovation and expansion. Last September, we announced changes to BioMarin’s strategy and operating model. Since that time, we have been focused on implementing these changes, while at the same time delivering strong growth and profitability. Looking ahead, we expect to see the continued implementation and benefits of this transformation deliver even stronger results and innovation in the coming quarters and beyond. Turning briefly to external dynamics impacting the sector, we believe BioMarin is well positioned to manage through uncertainty across a number of areas.

As the preeminent innovator of medicines that treat rare conditions, BioMarin is less exposed to macroeconomic conditions impacting other sectors. Because so many of our medicines are treatments for children, we have limited exposure to policies impacting Medicare, which covers roughly only two percent of our U. S. Patient population. In addition, BioMarin’s core fundamentals also provide a level of insulation from external unknowns, increasing Jack cash generation, our extensive global revenue base with approximately two thirds of our total revenues originating from outside The United States, along with our established global commercial, regulatory and manufacturing capabilities position us well in an evolving dynamic market environment.

Moving to the specific uncertainties around potential pharmaceutical tariffs, we are analyzing potential exposure and mitigation tactics under multiple scenarios. As BioMarin’s medicines address relatively small patient populations around the world, we manufacture products that address these rare indications in single locations either in The U. S. Or Europe. Based on our current understanding, we believe that the global scope of our business model with approximately two thirds of total revenues generated from outside of The United States and a significant level of U.

S.-based manufacturing will provide some protection from potential tariff impacts should innovative medicines for rare conditions be targeted. Important to note, Thiamarin has immaterial exposure to U. S. Tariffs for China, Mexico and Canada across its global supply chain operations and product sales. Turning now to potential new products and expansions.

We’re making good progress on our innovation strategy. I am pleased to share that we recently completed enrollment in our pivotal Phase III study with Voxogo in children with hypochondroplasia, paving the way for a potential launch in 2027. With Palynziq, based on the positive pivotal data we announced in April, we are on track to submit applications in the second half of this year in The United States and Europe for the treatment of adolescents. In addition, we expect to share early clinical results later this year from both BMN three fifty one or Duchenne muscular dystrophy and BMN three thirty three, a long acting CMP, two candidates that may provide highly differentiated treatment options for the conditions they target. Finally, we expect to fortify our internal innovation with business development.

As we feel that BioMarin’s established global capabilities, coupled with the external environment create opportunities that will support our growth agenda. In summary, we believe we are well positioned to manage through ongoing market uncertainty based on the global demand for our innovative medicines. Through BioMarin’s strategic transformation implemented over the last year, we have set ourselves up to deliver significant value creation for all our stakeholders in 2025 and beyond. Thank you for your attention. I will now turn the call over to Brian to provide our financial highlights for the quarter.

Brian Mueller, Executive Vice President, Chief Financial Officer, BioMarin Pharmaceutical: Thank you, Alexander. Please refer to today’s press release for detailed first quarter twenty twenty five results, including reconciliations of GAAP to non GAAP financial measures. All 2025 results will be available in our upcoming Form 10 Q, which we expect to file in the coming days. We are pleased with BioMarin’s strong results across the business in the first quarter of twenty twenty five. First quarter ’20 ’20 ’5 total revenues rose 15% to $745,000,000 compared to the same period last year.

VOXZOGO’s demand fueled growth with global revenue reaching $214,000,000 a 40% increase year over year and continuing its strong growth trajectory since approval four years ago. Revenue from the Enzyme Therapies business unit grew 8% year over year to $484,000,000 with substantial contributions from Palynziq, which grew 22% compared to the first quarter of twenty twenty four. As I mentioned in February, we expect total revenues to be higher in the second half of this year compared to the first half due to anticipated order timing dynamics across the portfolio. These dynamics apply to VoxoGo as well with the product now available in 49 countries. While a significant volume of new patients have started VoxoGo therapy in recent quarters through today, we note that quarterly VoxoGo revenues looking back to Q4 twenty twenty four and anticipated trends through Q2 twenty twenty five have revenues slightly disconnected from patient growth and appearing relatively similar quarter to quarter.

Importantly, we continue to expect Voxelgo full year revenues to be between 900,000,000 and $950,000,000 which at the midpoint represents 26% full year growth. Moving to expenses. In the first quarter of twenty twenty five, non GAAP R and D expense was $147,000,000 and lower than the same quarter in 2024. Operating trends in the first quarter of this year were consistent with those in the fourth quarter of last year, reflecting the impact of BioMarin’s R and D reprioritization. Looking ahead in 2025, we plan to increase spend on our pipeline priorities and the VOXZOGO new indication.

In the first quarter, non GAAP SG and A expense of $183,000,000 decreased year over year, also due to the impact of cost transformation initiative, including reduced activities related to the company’s focused Rocktavian strategy announced last year. While we observed reduced expenses due to these dynamics in the quarter and noting that Q1 is typically a lower spend quarter for BioMarin, Throughout the remainder of 2025, we expect SG and A expense to increase as we invest in the strategic priorities of the newly formed Skeletal Conditions and Enzyme Therapies business units. Moving to profitability. Non GAAP operating margin reached 35.7% for the first quarter of twenty twenty five, an expansion of 11.9 percentage points year over year. These results were driven by strong revenue performance and a spending reset as we prepare to invest more aggressively across R and D and SG and A in the coming quarters.

For R and D, this will include global applications for Palynziq adolescent approval, expansion of our BMN three fifty one and BMN three thirty three studies, as well as our clinical programs advancing multiple new indications with VoxoGo,

: just to name a few.

Brian Mueller, Executive Vice President, Chief Financial Officer, BioMarin Pharmaceutical: For SG and A, this will include investments in VoxoGo and enzyme therapy global commercial expansion activities and commercial launch preparations for hypochondroplasia and Palynziq in adolescence. Over the coming quarters, increased spending on R and D and SG and A is expected to result in slightly lower non GAAP operating margin in Q2 through Q4 twenty twenty five. However, we expect that quarterly non GAAP operating margins will balance out over the course of the year to our full year non GAAP operating margin guidance of between 32% to 33% that we reaffirmed today. Strong non GAAP operating margin flowed through to the bottom line, resulting in first quarter non GAAP diluted earnings per share of $1.13 an increase of 59% year over year. Consistent with prior quarters, non GAAP diluted earnings per share grew at a rate higher than revenue grew, reflecting Vomarin’s focus on operational efficiency and our value commitment to shareholders.

Following my comments on the timing of both revenue and expenses in 2025, non GAAP earnings per share is not expected to be a smooth progression from quarter to quarter. As implied with the strong Q1 result and our reaffirmed 2025 non GAAP earnings per share guidance, earnings per share could decrease in certain quarters compared to this first quarter, but accumulate to the expected double digit growth for the full year. Please note that while this reiteration of guidance reflects the impact of the currently enacted U. S. Tariffs, it does not reflect the impact of potential future pharmaceutical tariffs.

BioMarin’s increasing profitability continues to generate cash with $174,000,000 of positive operating cash flow in the first quarter of twenty twenty five, a 271% increase over Q1 twenty twenty four. This trend is expected to continue and provides the opportunity to execute on our top capital allocation priority of investing in innovation and future growth to positively impact our patients, employees and shareholders. In conclusion, we are pleased to begin 2025 with another strong financial performance. It gives us confidence that BioMarin will continue to execute on all of the elements of our revitalized strategy. I will now hand the call over to Kristen, who will speak to our commercial performance.

Kristen Hubbard, Executive Vice President, Chief Commercial Officer, BioMarin Pharmaceutical: Thank you, Brian. The team’s focus on commercial execution led to another quarter of strong growth across the business. Starting with Voxsogo for the treatment of achondroplasia, we were very pleased to have delivered 40% year over year revenue growth with strong contributions from across the globe. As of the end of the first quarter, children across 49 countries had access to VOXOVO treatment, and we are making strong progress toward our goal of opening access in more than 60 countries by 2027. In The United States, we are focused on implementing initiatives targeting new patient uptake, which we expect will drive continued expansion beginning in the second half of the year.

Commercialization efforts include increasing field personnel to broaden the prescriber base and implementing activities to further drive the adoption of VUXOVA treatment. Outside of The U. S, we saw continued strength across all markets. Taken together, we expect these U. S.

And OUS dynamics to result in higher Voxsogo revenue in the second half of twenty twenty five compared to the first half of twenty twenty five. As our global presence treating achondroplasia expands with strong momentum contributed from recently published international guidelines, which recommend diagnosis and early treatment with OXOGO, we are well positioned to enter the hypochondriplasia market in 2027, pending support of Phase III data next year. We are focusing on our global launch preparations and will keep you apprised as progress unfolds. Moving to our enzyme therapies, we are pleased with this business unit’s performance in the first quarter, where total enzyme therapy revenues increased 8% year over year. Starting with specific dynamics in the quarter, we saw continued strong growth with Palynziq increasing 22% and Aldurazyme up 40% year over year.

Vimzim, our second largest revenue contributor, was uneven compared to the prior quarter, typical of ordering dynamics with our mature global brands. Now the strength of Palynziq is based on the opportunity for potent and durable fee reduction to international guideline levels. The unrestricted diet potential that Palynziq offers is an important product characteristic that we think will bring tremendous value to the adolescent PKU population should we get approval next year. We are pleased with the growth of the enzyme therapies products in the first quarter and expect full year contributions from this important business unit to remain robust. In summary, the team executed well in the first quarter, and we look forward to continued strong performance throughout the remainder of 2025.

Thank you for your attention, and Greg will now provide an update on R and D. Greg?

Greg Fryberg, Executive Vice President, Chief R&D Officer, BioMarin Pharmaceutical: Thank you, Kristen. As Alexander mentioned, we recently completed enrollment in our pivotal study for VOXOGO in hypochondroplasia. We will be sharing the fifty two week results next year and will be seeking an approval for 2027 should the data be supportive. Also new today, we are pleased to share that we have reached an agreement with the FDA on an overall clinical development plan for BMN three thirty three. Assuming the Phase I data are supportive, we plan to initiate a registration enabling study in 2026, supporting our previously stated ambition for 02/1930 approval date.

We will continue to engage with regulators around the globe to refine our plans in the coming months. In the ongoing Phase I study in healthy volunteers, we’ve enrolled multiple cohorts and look forward to sharing initial pharmacokinetic data in the second half of the year with detailed data to be presented at a scientific venue in the first half of twenty twenty six. We’re also pleased with the numerous data updates across the portfolio so far this year. In March, at the American College of Medical Genetics and Genomics meeting, we shared data demonstrating favorable safety and strong adherence in real world clinical practice with Voxsogo in children under three with achondroplasia. No treatment related adverse events or dose interruptions were reported among these sixty three children followed for up to two years.

The study’s safety results, including an infant as young as one month old, add to the growing body of evidence supporting early treatment initiation with Voxogo, consistent with international treatment guidelines published in the journal Nature Review Endocrinology earlier this year. Also at ACMG, Palynziq data demonstrating the importance of sustained Phe reduction and the positive impact this can have on health related quality of life outcomes were presented. Most notably, mood and attention scores for patients achieving sustained Phe levels less than or equal to 120 micromolar per liter were significantly better than those at higher levels, suggesting that sustained Phe in the normal range is both achievable and may provide additional benefits for adults living with PKU. These significant and sustained reductions in blood Phe levels, coupled with the opportunity for an unrestricted diet are unique characteristics of Palynziq, the only enzyme substitute therapy for the treatment of PKU. These benefits demonstrated in adults with PKU give us continued confidence in the treatment profile of Palynziq, including for adolescents, as supported by the positive pivotal data in the twelve to seventeen year old group as announced in April.

We plan to submit for regulatory approvals in both The United States and Europe in the second half of the year. Turning to BMN three fifty one in Duchenne muscular dystrophy, we plan to publicly present our first clinical update in the second half of this year, including twenty five week muscle biopsy data for four patients treated at the six milligram per kilogram dose cohort. We have done extensive PKPD modeling based on preclinical data to determine what value at twenty five weeks will predict muscle dystrophin levels of 10% or higher at steady state, which we anticipate to occur out

Brian Mueller, Executive Vice President, Chief Financial Officer, BioMarin Pharmaceutical: at fifty two weeks or later.

Greg Fryberg, Executive Vice President, Chief R&D Officer, BioMarin Pharmaceutical: The model takes into account the tissue half lives for several chemical species, including for BMN three fifty one, for the SCIP product production and for generation of dystrophin protein. These values were informed by our experience with BMN three fifty one in preclinical mouse and nonhuman primate studies as well as from prior human experience with other phosphorothioate oligonucleotides. The model tells us that levels at twenty five weeks can be expected to rise two to three times higher by the time steady state is reached. We also want to measure how these patients feel and function. To do so, we have included several functional measures in this study, though we anticipate such measures will be most informative once steady state is reached.

The next update on this program will be shared at a scientific congress in the second half of this year. Thank you for your attention today. We look forward to keeping you apprised of our progress as we continue to advance our high impact candidates through the pipeline. We will now open the call to your questions. Operator?

Conference Operator: Thank you, sir. We’ll go first to Phil Nadeau, TD Cowen.

Phil Nadeau, Analyst, TD Cowen: Hi, good afternoon. Thanks for taking our questions. Two financial ones. First on Voxelgo, you mentioned a couple of times about H2 being larger than H1 and also the revenue in the course through Q2 being relatively similar. We’re 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 in the intermediate between those two, maybe there’d 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 U.

S. 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 U. S? Thanks.

Brian Mueller, Executive Vice President, Chief Financial Officer, BioMarin Pharmaceutical: Thanks, Phil. This is Brian. I’ll take both of those. Appreciate the questions. First on Voxago, I think you heard accurately in terms of the trends we’re observing coming out of Q4 last year here in Q1 and then into the first half of this year.

We had previously commented that we expected most of the VOXOGO revenue growth to come in the second half of the year. And first, what I’d point to is what we’re observing are global order dynamics, which are different from what shows up in the enzyme therapy business from time to time at the quarters would be a handful of those large bolus orders from select international customers that can either accumulate to, an anomalous quarter or be missing from a quarter. It’s less that and more just the dynamics of BoxOGO being available in now 49 markets. And it doesn’t take much for some of the purchasing patterns between, again, the end of twenty twenty four, Q3, Q4 into Q1, Q2 for those to look flattish. But importantly at the same time we are steadily increasing patients from quarter to quarter.

So it’s just an accumulation of dynamics across all of those markets. And then we see it flip in the second half of the year when we expect revenue to increase more substantially. So that’s the key SOGO matter. And on tariffs, you heard accurately, because we have immaterial exposure in our business to China, Mexico and Canada, we do not have a material impact. And our reaffirmed guidance today includes any modest or immaterial impacts from tariffs in those regions.

In terms of potential future pharmaceutical tariffs, we are modeling all scenarios and evaluating all of the potential mitigating tactics. We’re going to hold off on speaking to specifics, including quantifying any of those scenarios until we have more clarity and announcements and certainty in terms of what pharmaceutical tariffs could look like. But can assure you that we’re not just looking at all scenarios but all potential levers. Many of those are in the same categories that you’re hearing some of the larger biopharma’s discuss on their Q1 calls. That includes global supply chain strategies, which also for us includes our global manufacturing network, managing inventory levels, our global intellectual property rights, and then lastly, monitoring potential global tax reforms, you know, in connection with or in response to, the overall tariffs.

So we’ve got a dynamic global business. Our products are sourced from both The U. S. And outside of The U. S.

I’ll share that we are a net exporter of products in The U. S. Due to our significant California based manufacturing site. And then just a reminder as well that from an overall exposure to U. S.

Sales imports, most of our sales are outside of The U. S. Roughly one third of our total sales are U. S. Sales and two thirds outside of The U.

S. So those are potentially insulating factors should we see pharmaceutical tariffs. So we’re going to hold off on speculating on any details at this time. Appreciate

Phil Nadeau, Analyst, TD Cowen: enough. Thanks for taking our questions.

Conference Operator: The next question is from Salveen Richter, Goldman Sachs.

Tommy, Analyst, Goldman Sachs: Thanks for taking our questions and congrats on all the progress. This is Tommy on for Salveen. Just wondering if we can get an update on your interest in 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 three thirty three this year, Can you maybe characterize the level of detail we’ll get before you kind of present the full data at Congress next year? Thank you.

Alexander Hardy, President and Chief Executive Officer, BioMarin Pharmaceutical: Thanks very much, Tommy. I’ll handle the question on BD and then hand over to Greg. So, yes, we’re continuing to be very excited about the potential for BD for BioMarin based on our strong capabilities and also obviously the state of the capital markets. We’ve been approaching it very methodically. We’ve got a great team and capabilities in place.

We’ve got a very clear strategy, which we outlined and communicated to the outside world last year, and we’ve been looking systematically at opportunities. And just to reiterate, what we’re looking at is things which are completely lined up with what Myanmarin is really good. So genetically defined conditions, we’re looking at clinical stage assets in particular as well as obviously earlier assets, Boris done that. And particularly highlighting ones that align with our business unit structure where we really think we have a strength and a right to win. So yes, everything is very much on track.

We continue to focus on doing at least one business development deal this year. We’re looking forward to providing updates as and when that makes sense. And obviously, the market continues to develop, it only creates more value from our perspective and makes those deals even more attractive from an investor standpoint. And with that, I’ll hand over to Greg.

Greg Fryberg, Executive Vice President, Chief R&D Officer, BioMarin Pharmaceutical: Thanks, Alexander. With regard to BMN three thirty three, just as a quick reminder, we have an ongoing Phase I study. It is in healthy volunteers, not in patients. So the primary purpose of this study, of course, will be to look at single dose safety, but also the PK profile. That is the data.

Again, we’re actually marching right through the study very efficiently. We’re several cohorts in at this point. And we anticipate before the end of the year that we’ll be able to provide a top line update with regard to whether or not that data is supportive of us moving forward. The more detailed PK data and totality of the Phase I, we anticipate in the first half of next year, we’ll be releasing that publicly.

Conference Operator: The next question today will come from Joseph Schwartz, Leerink Partners.

Jenny, Analyst, Leerink Partners: Hi. This is Jenny on for Joe. Thank you for taking our question. I think for previously you guys have mentioned that Voxelco has penetrated just under 20% of the market in Achondroplasia as of the second quarter last year. Can you talk a little bit more about what strategies you’re implementing to drive further adoption among patients?

And is this something that you’re going to have to reach out to physicians? And what are the main areas of possible improvement here and how you see this trending over time? Thanks.

Kristen Hubbard, Executive Vice President, Chief Commercial Officer, BioMarin Pharmaceutical: Yes. Thank you so much for the question, Jenny. This is Kristen Hubbard. So great question about the penetration of Voxsogo. We have several strategies that we are in the process of implementing that I think will certainly help us to further drive growth.

And in particular, when I think about The U. S. Market, which is our largest single market opportunity, what we’re really focused on is driving adoption across all age groups. Now as we’ve previously reported, we’ve had quite an uptick in numbers in the zero to four population. And just to remind you that we just got that label expansion at the end of twenty twenty three.

So our new patient starts have been very strong in the zero to four age group, in which case this is very well aligned with the consensus guidelines that we saw come out earlier this year, which recommends early diagnosis and early treatment with Ophsogo. So we have strategies and tactics in place targeting that age group, starting at infancy. And then of course we don’t want to forget about the importance of slightly older patients, so in the five plus age group. And that’s where we’re definitely still targeting. There’s still opportunity there in The U.

S. Continue to target those patients. I will say that our strategies in particular are really in and around driving awareness of the medicine, of the fact that there’s a treatment option available. And so we’re looking at investing in initiatives that will help us there. This will in large part help us to expand the prescriber base.

And so what we’re looking at is really making sure that we’re expanding the prescriber base. And this is true across various specialties. But where we see the most growth in terms of prescribers is in the pediatric endocrinologist, which is very in line with our strategies to expand there. We’re also investing in increased field personnel, again, to target prescribers to ensure that we’re driving awareness and adoption therein. And then when we think outside of The U.

S, where we’re looking at is continuing to look for those kind of access pathways, awareness pathways, but also importantly, we’ll continue to open new markets, which will expand further penetration.

Conference Operator: And then

Tracy McCarty, Investor Relations, BioMarin Pharmaceutical0: I should say oh, I

Kristen Hubbard, Executive Vice President, Chief Commercial Officer, BioMarin Pharmaceutical: was gonna say, and lastly, I think that all of this has really been enhanced in our ability to do this, to execute in a focused manner because of the business unit structure that we put in place. This has been a really exceptional way for us to really drive into the market insights leading to strategy and execution in a really focused and agile way.

Tracy McCarty, Investor Relations, BioMarin Pharmaceutical1: Thank you. Next question.

Conference Operator: Thank you. And up next is Akash Tewari, Jefferies.

Tracy McCarty, Investor Relations, BioMarin Pharmaceutical1: Hi. This is Zaki on for Akash. Thanks so much for taking the question. Just on the DMD Corrector three fifty one, when we looked at the recently published mouse data, it looks like there’s some mild liver signals in those mouse models at the higher exposures and some inflammation in the liver and muscle in those mice. So given those safety signals, how are you thinking about your ability to dose chronically?

And is there any data that you’re collecting, that we can see in the upcoming readout that could potentially make us more comfortable, on safety? Thank you.

Greg Fryberg, Executive Vice President, Chief R&D Officer, BioMarin Pharmaceutical: Yeah. Thanks for the question. This is Greg Fryberg. These, observations are class effects for the ASO, categories. And so, you know, from that standpoint, we’re not surprised to see at high dose levels in some of our toxicologic, studies that we’re seeing some of these end organ toxicities.

We’re watching them very closely. And in fact, we selected a chemistry type, this phosphorylthiolate, instead of, you know, other available, chemistry classes because we do believe that this is a case where it’s all about opening that therapeutic window. Again, there’s two ways to address the therapeutic window. One is to limit toxicity. The other way is to, you know, drive the potency of the molecule.

And we’ve engineered this molecule to try to really take advantage of, like we’ve talked about previously, this novel skip site and some other chemical factors, again, that get the drug where we want it to be. Those are the open questions, I think, for almost any molecule in this space, and we’re watching patients very closely for a variety of toxicities. We will be presenting our data from our six milligram per kilogram cohort in the second half of this year. And you can expect in addition to dystrophin assays on muscle biopsies that we would present the totality of our safety data that’s available at that time.

Conference Operator: The next question today is from Jessica Fye, JPMorgan.

Tracy McCarty, Investor Relations, BioMarin Pharmaceutical: Hey guys, good afternoon. Thanks for taking my question. So BioMarin continues to execute across the business. However, the market seems worried about long term competition for VoxoGo. Can you talk about how you think you can best address the market’s perception of VoxoGo’s long term value, particularly beyond 02/1930?

Thank you.

Kristen Hubbard, Executive Vice President, Chief Commercial Officer, BioMarin Pharmaceutical: I’ll start on the commercial execution here in the near term. And if you want to talk about some of the lifecycle management, think that that would be probably an IFAB. But thanks for the question, Jess. So I certainly recognize where you’re coming from in terms of the market perception. And what I can really say is that with Foxogo, where we’re really focused in terms of our strategies and where I think that this is going to be something that will be quite powerful for us is really focused in and around this notion of a start and stay treatment paradigm.

And really getting patients to start, and again, reminding everybody that our label starts at infancy, which I think is a unique differentiator here in the near term, is really based on our ability to help families and prescribers feel comfortable with the growing body of evidence, which includes 6,000 patient years of efficacy and safety data. We’ve got a tremendous and I know, Greg, you can speak to some of this growing body of evidence on the overall health benefits that go far beyond height, which I think is really important to treaters and families alike. We’ve got this broad label in terms of starting at infancy, which I think aligns nicely with the consensus guidelines. And importantly, we have a growing footprint in countries outside the world, reminding everybody that only ten percent of the total addressable patient population is in The United States. And we have an incredible commercial footprint that goes into many, many countries.

As you guys know, we’re now 49 countries we have Voxelvo access in and we’re looking to build that into greater than 60 countries by 2027. So we see the growth trajectory as something that we can maintain. And as you know, we’ve given full year guidance to a little over 25% at the midpoint for the year and we’ll continue with a 25% CAGR out into the future. But Greg, I don’t know if you maybe should speak to some of the lifecycle options we have as well.

Greg Fryberg, Executive Vice President, Chief R&D Officer, BioMarin Pharmaceutical: Yes. Thanks, Kristen. And from an R and D perspective, we are going to continue to try to leverage, again, that 6,000 patient year, it’s about 4,000 individual patients and growing experience, to actually put the data out there with regard to how we’re improving the health and wellness of these patients with achondroplasia. We get caught up in, I think, AGV numbers, sometimes, but we have to remember that the burden of disease that we really want to address, are things, that go well beyond that. And so data with regard to craniofacial volume, foramen magnum size, body proportionality, quality of life data, and of course, bone strength and bone integrity, these are the kind of data that we have already put out, but we’re also gonna continue to grow and put out.

What patients and their families want is they want a safe and dependable drug. And we think that that’s exactly what Voxogo is. It’s a targeted therapy that’s tailored for these patients with achondroplasia, and again, that long term experience, we’re going to continue to publish that. We’re also developing the molecule and other stature related disorders, And, I think we’ve spoken about that previously, but we think that there’s an opportunity here, to continue to develop the agent. We also, of course, are invested in our next generation agent, which is BMN three thirty three.

Our goal with BMN three thirty three isn’t just a more convenient version of CNP, it’s to actually increase the free CNP exposure that we can reach in the patients. And so that recent agreement with the FDA moving forward into what a pivotal plan might look like, we see that as an incremental positive, and we’re very much looking forward to the PK data that we’ll see later this year. So, again, a lot of confidence that Voxoggo is a safe and effective therapy.

Tracy McCarty, Investor Relations, BioMarin Pharmaceutical: Should we expect the same endpoint for three thirty three in a pivotal trial as for Voxoggo?

Greg Fryberg, Executive Vice President, Chief R&D Officer, BioMarin Pharmaceutical: So we’re envisioning that we will do a comparative effectiveness study, versus VoxoGo, and we’ll be looking at similar endpoints to what has been studied in previous examples.

Tracy McCarty, Investor Relations, BioMarin Pharmaceutical: Thank you.

Conference Operator: The next question comes from Paul Mathies.

Tracy McCarty, Investor Relations, BioMarin Pharmaceutical2: Hey, thanks so much for taking my questions. I had one for Brian and one clinical question. Brian, I wanted to just clarify as it relates to Boxogo over the past couple of quarters, I know what you’re seeing going into 2Q. Has the net price on a global basis of the drug change? Just curious if the price has gone down bit as you’ve diversified into other markets and your expectation there.

And then as it relates to the $3.33 pivotal, that was super interesting to kinda give the color on a comparative design. Are you looking to actually show superiority? Is it gonna be powerful non inferiority?

: I mean, maybe it’s maybe it’s

Tracy McCarty, Investor Relations, BioMarin Pharmaceutical2: too early to say, but given your conviction that higher exposure may drive greater efficacy, what do you see the opportunity to show there? Thank you.

Greg Fryberg, Executive Vice President, Chief R&D Officer, BioMarin Pharmaceutical: Yes. This is Greg. Maybe I’ll take the three thirty three question because it’s a quick answer. Yes, we are looking for superiority in growth dynamics from a higher exposure of CMP.

Brian Mueller, Executive Vice President, Chief Financial Officer, BioMarin Pharmaceutical: Greg. Thanks, Paul, for the question. Mine is likewise. A quick answer because, and it is a great question, when you talk about demand increasing and not seeing the same correlation in revenue for these couple of quarters, we are not observing not seeing any pricing dynamics. It’s truly just the lack of correlation market by market that just happens to show up here in Q1 and possibly in Q2.

No price factoring in.

Tracy McCarty, Investor Relations, BioMarin Pharmaceutical2: Makes sense. Thanks very much.

Conference Operator: The next question is from Ellie Merle, UBS.

Jenny, Analyst, Leerink Partners: Hi, this is Jasmine on for Ellie. Thanks so much for taking our question. So, what’s the latest on timing for in other growth disorders like ISS and shocks? And then how quickly do you think that you could move three thirty three here? So would you want to wait for Avaxxogo clinical success here before studying three thirty three in these indications or is that something you’d want to do sooner?

Thank you.

Greg Fryberg, Executive Vice President, Chief R&D Officer, BioMarin Pharmaceutical: Thank you for the question. This is Greg Freiberg again. With regard to the Newnans Turner and Schox basket that we have as well as idiopathic short stature, both of those protocols are open for enrollment and are continuing to progress. We anticipate that we will have data from those programs that would enable us to begin pivotal studies in 2027. So we remain on track for those goals.

With regard to the role that three thirty three would play in additional indications, just as a reminder, we still are working through with three thirty three once we’ve moved beyond healthy volunteers. There will be both dose ranging as well as the effectiveness studies to come thereafter. That’s the agreement, again, that we referred to with the FDA. Long way of saying that these reveals of information are actually gonna line up quite nicely so that we can make decisions, when we have the complete, profile for 333 to determine what the future plans would be. Right now, the discussions with three thirty three that we’ve been talking about have been achondroplasia related.

Conference Operator: Thank you. Next question is from Cory Kasimov, Evercore ISI.

Tracy McCarty, Investor Relations, BioMarin Pharmaceutical3: Hi, thanks for taking our question. This is Adi on for Cory. Wanted to ask you if you can share how The U. S. Versus ex U.

S. Sales have evolved in the first quarter for Voxsogo? And what trends do you expect to see going forward into the rest of the year?

Conference Operator: Yes. Thank you so much

Kristen Hubbard, Executive Vice President, Chief Commercial Officer, BioMarin Pharmaceutical: for the question. This is Kristen. And as you know, last quarter, we gave specifics on precisely that, which is The U. S. Versus outside U.

S. Revenues for Voxelgo. And we did that strictly so we could clarify the significant contributions that come from outside The U. S. And given that the ex U.

S. Weight of the revenues is approximately 75% total of Vox revenues, We do expect this split to fluctuate just a little bit quarter over quarter, and that goes back to what Brian had mentioned about ordering patterns becoming a little bit more bumpy as we turn into a more mature brand, which is very commensurate with other products in our portfolio. But overall, we expect the split today to be roughly around 75%. And in the long term, what we expect is that it will mirror that of our other products in our portfolio, which is where you have about onethree of the revenues coming from The U. S.

And twothree coming from outside The U.

Tracy McCarty, Investor Relations, BioMarin Pharmaceutical3: Okay. Thank you. I’ll jump back in the queue for rest of the questions.

Conference Operator: We’ll go next to Gina Wang, Barclays.

Tracy McCarty, Investor Relations, BioMarin Pharmaceutical0: Thank you. I have two sets of questions. First one is regarding also the VoxoGo franchise. So maybe the $3.33, can you help us understand because you have a healthy volunteer data and mainly is a PK data. How do you define that would be the top line data you share with us that will be good enough that define as a supportive to start your registration trial?

And will you only identify one dose for your registration trial? And then related question, know there is a competitor running CMT plus gross hormone studies. Any thoughts there? We know historically, gross hormone didn’t work. So maybe any thoughts there regarding CMP plus gross hormone study?

And the third part, quickly on the manufacturing sites for VOXZOGO. Do you have a manufacturing site both in The U. S. And the Ex U. S?

Greg Fryberg, Executive Vice President, Chief R&D Officer, BioMarin Pharmaceutical: So, thank you, Gina. First question with regard to three thirty three, you’re correct. We will have, PK data of all the species. So we’ll have not only BMN three thirty three, but also free CMP levels. Our goal for that study is to see multifold increases in sustained exposure levels for free CMP, and that data will then unlock what comes next.

That next study, while we call it, you know, one protocol, which it is, it is a multipart protocol that involves both a dose ranging portion as well as a, you know, a comparative effectiveness portion. And so in that regard, the data that we’ll be revealing, later this year will be the ungating to move into those patients with achondroplasia to do that dose ranging study, to pick that final dose, again, that will be in the comparative effectiveness side. On the growth hormone side, I think you’ve you’ve actually framed it quite nicely. We would not be, surprised at all to see that if you combine growth hormone with CNP and achondroplasia patients that you would see an increase in AGV at six or twelve months, maybe even out at two years. But historically, the challenge for growth hormone hasn’t been about those short term benefits.

It’s been about whether they translate into meaningful increases in final adult height. And as you mentioned, they’ve been transient. That’s been the challenge. This is, of course, why growth hormone isn’t actually routinely recommended by expert guidelines or regulators around the globe except for some unique regions. We also should remember that growth hormone, really in this discussion of height, is really not the most important measure that we should be talking about.

This health and wellness of the patients is, of course, the most important factor. You know, we haven’t seen that with growth hormone, at least to this date, that the kind of evidence beyond height, could be achieved in patients with achondroplasia. On the contrary, growth hormone really has added problems for patients. There’s been acceleration of bone age. We’ve worsening proportionality at different times.

Of course, I mean, you just look at the label, there’s cardiovascular and metabolic disturbances. And then one unique factor that’s been brought up by some of the treating physicians, things like tonsillar hypertrophy that don’t seem like they’d be a big deal in most children, actually, in children with airway obstruction, can be a very, challenging problem. So, you know, clearly, these need to be watched very closely with regard to growth hormone. What we’re very pleased about with Voxsogo is remember, these kids aren’t even growth hormone deficient. But what Voxsogo brings is a targeted therapy.

We think, again, we’ve demonstrated the effects on all those areas beyond height. We talked about craniofacial volume and others in a previous answer, and all while maintaining bone health. That’s what we think these patients need. That’s what they want, a safe and dependable targeted therapy for their disease.

Brian Mueller, Executive Vice President, Chief Financial Officer, BioMarin Pharmaceutical: Thanks, Greg. Hi, Gina. This is Brian. I’ll take your question on Voxelgo manufacturing. Voxelgo drug substance is sourced from The United States.

Tracy McCarty, Investor Relations, BioMarin Pharmaceutical0: Thank you.

Conference Operator: Up next, we’ll take a question from Costas Valoris, BMO Capital Markets.

: Thanks for taking our question and congrats on the progress. Maybe one question for Greg on PMN-three 33. There is a recent presentation by Children’s National Hospital at ESP that shows that there is no really meaningful correlation between VOXOGO PK and growth outcomes in hypochondroplasia. So given this data, I wonder to what extent will we be able to draw conclusions about BMN three thirty three outcomes based on the PK data that we will see in 2025? Thank you.

Greg Fryberg, Executive Vice President, Chief R&D Officer, BioMarin Pharmaceutical: Yes. Thanks for your question. I think when we talk about Voxogon PK, we also have to recognize the challenges when a drug has about a thirty to sixty minute half life of actually getting the complete profile. You know, trough p k becomes very well, trough p k, if if you’re going to measure a very low point, is easy. But but measuring that full profile, can can be a challenge.

There is a fundamental difference between the p k profile of VoxoGo and what we expect to see in BMN three three three. Of course, rather than a sawtooth pattern, we expect to see a much more of a continuous release. And fundamentally, different, you know, approaches with the pulsatile versus the continuous coverage make it difficult to measure, I I think, between the two and compare the PK profiles. But what we can do is we can look at other long acting CNP, both in preclinical models and the preclinical outcomes, and we can ask the question about sustained levels of CNP. That’s what we’ve modeled, from our synovalgus monkey models, and we believe that, again, if we can recapitulate what we’ve seen in those models against another long acting, you know, more continuous coverage and activation of n p r two, we think that, again, by seeing multifold increases above what’s been demonstrated so far, that opens the opportunity to test the hypothesis is more better.

Hopefully, answered your question.

Alexander Hardy, President and Chief Executive Officer, BioMarin Pharmaceutical: Thank you.

Conference Operator: Mohit Bansal from Wells Fargo is up next. Hi. This is Trina on for Mohit. Thanks for taking our question. Wanted to ask a question about BMN three fifty one.

In terms of the methyl dystrophin data expected in the second half, is that 10% dystrophin levels the only goal that should inform the future course of action for this molecule, or would you be looking at a bar for other parameters as well? Thank you.

Greg Fryberg, Executive Vice President, Chief R&D Officer, BioMarin Pharmaceutical: Thanks for the question. What we will have once patients have reached the six month time point is we, of course, will have a totality of safety data and and PK on a variety of species. And, you know, that includes not only the drug, but the muscle content of the drug because we’re doing muscle biopsies. We’ll also be able to look in the muscle as a skip product and the dystrophin. So all of those factors will be there.

We have actually released, you know, an illustrative example that was connected to our release today of a model that we put forward. And that model shows us that what we’re gonna see at the twenty five, twenty six week time point are dystrophin levels that, when predicted out at steady state, are gonna rise two to threefold. And so they’ll give us a line of sight whether that 10% at steady state will be achievable. We are also measuring in the study functional outcomes, which I think is the real question you’re asking. The challenge, of course, with functional outcomes is, you need to wait till, of course, you reach steady state of the drug.

We’re looking at North Star. We’re looking at six minute walk test. We’re looking at stride velocity 95C, all of those measures. We do anticipate that those will take longer than this initial first look. I also want to remind the audience that this is the first data from our six milligram per kilogram cohort.

We also have completely enrolled our nine milligram per kilogram cohort. Again, as in a relay race, that’s trailing behind in terms of time. But our goal here is to identify a molecule that not only has potent, dystrophin, induction, but also, can of course be administered safely in a chronic fashion.

Conference Operator: Our next question is from Olivia Brayer Cantor Fitzgerald.

Tracy McCarty, Investor Relations, BioMarin Pharmaceutical: Hi, good afternoon. Thank you for the question. Greg, just wanted to clarify a comment you made earlier. Will we not see any level of Phase two data from other short stature indications until 2027? Or could we actually start to see some initial clinical characterization maybe select patient groups just to help derisk some of those new Voxsogo indications?

And then on the P and 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?

Greg Fryberg, Executive Vice President, Chief R&D Officer, BioMarin Pharmaceutical: So why don’t I take a quick stab just to clarify? With regard to the short stature conditions, obviously, I mean, the Doctor. Dauber study, with Voxsogo, continues, and we do expect that that data will be updated, over time. With regard to our own programs, what we’re publicly commenting on is that we would have, information to be able to initiate our phase three study in 2027. It’s possible that any time proximal to that, depending again on speed of enrollment and so forth, that we may have data.

But we’re not committing at least right now beyond the 2027 initiation of a phase three. But clearly, we would need that data not only in house, but we would be publicly presenting that data before we would initiate those Phase III studies.

Brian Mueller, Executive Vice President, Chief Financial Officer, BioMarin Pharmaceutical: Hi, Olivia, this is Brian on your profitability question. Thanks for that. Yeah, we are pleased with this strong start to the year, on the bottom line with the $1.13 of earnings per share growth. Again, we are expecting while revenues are planning to increase in the second half of the year, we are also planning to increase investments in both R and D and SG and A over the course of the year. But by all means, we are confident in the full year EPS guidance with this strong start to the year.

And we do believe that will continue. Might in terms of earnings power, I would point you to our long term guidance that we remain confident in, at least today, excluding any potential impact of pharmaceutical tariffs. So whether it be the goal of 4,000,000,000 of revenue by 2027 or our 40% and growing non GAAP operating margin target next year, We didn’t give long term earnings per share guidance, but you can do the math along the trajectory of our OpEx line item goals over time and see that we are expecting significant earnings per share growth over the next several years and this year being 22% at the midpoint of our guidance is the trajectory that we’re talking about.

Tracy McCarty, Investor Relations, BioMarin Pharmaceutical: Okay. Thanks guys. Helpful.

Conference Operator: We have reached the end of the Q and A session. Our final question today will come from Vikram Purohit, Morgan Stanley.

Tracy McCarty, Investor Relations, BioMarin Pharmaceutical4: Hi. Good afternoon. Thanks for fitting me in. We just had two quick clarification questions on BMN three thirty three. 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 VOXAGO 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 U. S. As well? Thank you.

Greg Fryberg, Executive Vice President, Chief R&D Officer, BioMarin Pharmaceutical: Yeah. Thanks for the question. Just to clarify, the announcement today, again, was that we had reached agreement with the FDA, and we’re continuing to work with regulators around the globe. The study, that we refer to that would initiate in 2026 is a combined phase twothree study. And in that regard, we are doing dose ranging as well as the final comparative effectiveness versus VOXZOGO, and we would be looking for a superior outcome with regard to the primary endpoint in that study versus the control arm.

Conference Operator: And everyone at this time, there are no further questions. I’d like to hand the conference back to the CEO for any additional or closing remarks.

Alexander Hardy, President and Chief Executive Officer, BioMarin Pharmaceutical: Thank you, operator, and thank you all for joining us today. As we described, our first quarter performance highlights the fundamental value of our transformative medicines and the strength of our business, paving the way for record performance in full year 2025. In the face of external dynamics that are creating uncertainty across a number of areas, we think we are well positioned. We remain laser focused on what we can control, investing in the expansion of internal and external innovation, leveraging our extensive global revenue base and our commercialization capabilities, and most importantly, continuing to deliver increasing value creation for all of our stakeholders in 2025 and beyond. Thank you for your attention.

Look forward to speaking with you soon.

Conference Operator: And once again, everyone, that does conclude today’s conference. We would like to thank you all for your participation today. You may now disconnect.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.