Q1 2025 Biomarin Pharmaceutical Inc Earnings Call
Please stand by.
Speaker Change: Good day everyone and welcome to the Biomarin Pharmaceutical First Quarter 2025 conference call. Just a reminder that this conference is being recorded. I would now like to hand things over to Ms. Traci McCarty. Please go ahead, ma'am.
Traci Mccarty: NICU Operator, to remind you this non-confidential presentation contains forward-looking statements about the business prospects of Biomarin Pharmaceutical Inc.
Traci Mccarty: including expectations regarding Biomarin's financial performance, commercial products and potential future products in different areas of therapeutic research and development.
Traci Mccarty: Results may differ materially depending on the progress of Biomarin's product programs, actions of regulatory authorities. Dr. Prasimov, Dr. Prasimov, Dr. Prasimov,
Traci Mccarty: Availability of Capital, Future Actions of the Pharmaceutical Market, and Developments by Competitors, and those factors detailed in Biomarin's filings with the Securities and Exchange Commission, such as 10Q, 10K, and 8K Reports.
Traci Mccarty: In addition, we will use non-GAAP financial measures as to find in regulation G during the call today.
Traci Mccarty: These non-GAAP measures should not be considered in isolation from as substitutes for or superior to financial measures prepared in accordance with US gap and you can find the related reconciliation to US gap in the earnings press release and earnings presentation.
Traci Mccarty: Both of which are available in the Investor Relations section of our website.
Traci Mccarty: Please note that our commentary on today's call will focus on non-GAF financial measures, unless otherwise indicated.
Traci Mccarty: On the call from Biomarin Management Today, our 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 Friberg, Executive Vice President, Chief R&D Officer
Traci Mccarty: I will now turn the call over to Biomarin's President and CEO , Alexander Hardy.
Traci Mccarty: Thank you Traci and good afternoon everyone. Thank you for joining us today for our first quarter 2025 results update. We are pleased to have delivered strong financial performance and revenue growth of 15% in the quarter.
Traci Mccarty: 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.
Traci Mccarty: 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.
Traci Mccarty: Last September , we announced changes to Biomarin 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. Thank you very much.
Traci Mccarty: 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.
Traci Mccarty: Turning briefly to external dynamics, impacting the sector, we believe Biomarin is well positioned to manage through uncertainty across a number of areas.
Traci Mccarty: As the preeminent innovator of medicines that treat rare conditions, Biomarin is less exposed to macroeconomic conditions impacting other sectors.
Traci Mccarty: Because so many of our medicines are treatments for children, we have limited exposure to policies impacting Medicare which covers roughly only 2% of our U.S. patient population.
Traci Mccarty: In addition, Biomarin's core fundamentals also provide a level of insulation from external unknowns.
Traci Mccarty: Increasing Jack Kas' generation, our extensive global revenue base with approximately two-thirds of our total revenues originating from outside the United States. [inaudible]
Traci Mccarty: Along with our established global commercial regulatory and manufacturing capabilities, position as well in an evolving and dynamic market environment .
Traci Mccarty: Moving to the specific uncertainties around potential pharmaceutical tariffs we are analysing potential exposure and mitigation tactics under multiple scenarios.
either in the US or Europe . [inaudible]
Traci Mccarty: 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 US-based manufacturing will provide some protection.
Traci Mccarty: from potential tariff impacts should innovative mentions for rare conditions be targeted. Important to note, Biomarin has immaterial exposure to U.S. tariffs for China, Mexico and Canada across its global supply chain operations and product sales.
Traci Mccarty: Turning now to potential new products and expansions, we're making good progress on our innovation strategy.
Traci Mccarty: I am pleased to share that we recently completed enrollment in our pivotal phase three study with Fox Ogo in children with hyperconduplesia, Paving the way for a potential launch in 2027.
Traci Mccarty: with Palandzik 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 adolescence. [inaudible]
Traci Mccarty: In addition, we expect to share early clinical results later this year from both B.M.N. 351 for Dushan Muscular Dystrophy and B.M.N. 333, along Acting CNP. Two kind of this.
Traci Mccarty: that may provide highly differentiated treatment options for the conditions they target. Finally, we expect to fortify our internal innovation with business development. Thank you very much.
Traci Mccarty: In summary, we believe we are well positioned to manage through ongoing market uncertainty based on the global demand for our innovative medicines.
Traci Mccarty: Through Biomarin's strategic transformation implemented over the last year, we have set ourselves up to deliver significant value creation for all arch-stakeholders in 2025 and beyond.
Traci Mccarty: Thank you for your attention. I will now turn the call over to Brian to provide our financial highlights for the quarter.
Brian: Thank you, Alexander. Please refer to today's press release for detailed first quarter 2025 results, including reconciliation of gap to not GAAP financial measures.
Brian: All 2025 results will be available in our upcoming Form 10Q which we expect to file in the coming days.
Brian: We are pleased with Biomarin's strong results across the business in the first quarter of 2025.
Brian: First quarter, 2025 total revenues rose 15% to $745 million compared to the same period last year.
Brian: Boxo goes demand field growth with global revenue reaching $214 million, a 40% increase year over year, and continuing its strong growth trajectory since approval four years ago [inaudible]
Brian: Revenue from the Enzyme Therapies Business Unit grew 8% year-over-year to $484 million, with substantial contributions from Palin Zeke, which grew 22% compared to the first quarter of 2024.
Brian: 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. Thank you.
Brian: These dynamics apply to Voxogo as well, with the product now available in 49 countries.
Brian: While a significant volume of new patients have started voxogal therapy in recent quarters through today,
Brian: We note that quarter-leave oxygone revenues looking back to Q4 2024 and anticipated trends through Q2 2025 have revenues slightly disconnected from patient growth and appearing relatively similar quarter to quarter. [inaudible]
Brian: Importantly, we continue to expect box-ovo full-year revenues to be between $900 and $950 million, which at the midpoint represents 26% full-year growth. [inaudible]
Brian: Moving to expenses in the first quarter of 2025, non-GAAP R&D expense was $147 million and lower than the same quarter in 2024
Brian: Operating trends in the first quarter of this year were consistent with those in the fourth quarter of last year, reflecting the impact of biomorins R&D reprioritization.
Brian: Looking ahead in 2025, we find the increase R&D spend on our pipeline priorities and the box will go new indication
Brian: In the first quarter, non-GAAP SGNA expensive $183 million decreased year-over-year, also due to the impact of cost transformation initiative.
Brian: including reduced activities related to the company's focused rock TV and strategy announced last year. Well, we observed reduced expenses due to these dynamics in the quarter and noting that Q1 is typically a lower-spend quarter for Biomarin.
Brian: Throughout the remainder of 2025, we expect S.G. and A. expense to increase as we invest in the strategic priorities of the newly formed skeletal conditions and enzyme therapy's business units. [inaudible]
Brian: Moving to profitability, Nogap Operating Margin reached 35.7% for their first quarter of 2025, an expansion of 11.9% to 0.0% over year.
Brian: These results were driven by strong revenue performance and a spending reset as we prepare to invest more aggressively across R&D and SGNA in the coming quarters.
Brian: For R&D, this will include global applications for balancing adolescent approval, expansion of our BMM-351 and BMM-333 studies, as well as our clinical programs advancing multiple new indications with FOX-OGO, just to name a few.
Brian: For SG&A, this will include investments in Vaxilgo and Enzyme Therapy Global Commercial Expansion Activities, and commercial launch preparations for Hypochandroplasia and Palenzykin Adolescence.
Brian: Over the coming quarters, increased spending on R&D and SGNA is expected to result in slightly lower non-GAAP operating margin in Q2 through Q4 2025.
Brian: 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.
Brian: Strong non-GAAP operating margins 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 growth.
Brian: Reflecting Biomarin's focus on operational efficiency and our value commitment to shareholders.
Brian: 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
Brian: As implied, with the strong Q1 result in 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.
Biomarin's increasing profitability continues to generate cash.
Brian: with $174 million of positive operating cash flow in the first quarter of 2025. Thank you, bye.
Brian: A 271% increase over Q1 2024. 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.
Brian: 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.
Brian: I will now hand the call over to Kristen. We will speak to our commercial performance. Kristen?
Cristin: Thank you, Brian . The team's focus on commercial execution led to another quarter of strong growth across the business.
Cristin: Starting with Voxogo for the treatment of Acondroplasia, we were very pleased to have delivered 40% year-over-year revenue growth with strong contributions from across the globe.
Cristin: As of the end of the first quarter, children across 49 countries have access to Voxodo treatment and we are making strong progress toward our goal of opening access in more than 60 countries by 2027.
Cristin: In the United States, we're focused on implementing initiatives targeting new patient uptake, which we expect will drive continued expansion beginning in the second half of the year.
Cristin: Commercialization efforts include increasing field personnel to broaden the prescriber base and implementing activities to further drive the adoption of oxother treatment.
Cristin: Outside of the U.S., we saw continued strength across all markets. Taken together, we expect these U.S. and O.U.S. dynamics to result in higher bucks over revenue in the second half of 2025 compared to the first half of 2025.
Cristin: As our global presence treating acondriplasia expands, with strong momentum contributed from recently published international guidelines.
which recommend diagnosis and early treatment with OXOGO.
Cristin: We are well positioned to enter the hypo-contraplasia market in 2027, pending supportive phase 3 data next year. We are focusing on our global launch preparations and will keep you apprised as progress unfolds.
Cristin: 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.
Cristin: Starting with specific dynamics in the quarter, we saw continued strong growth with Palinzik, increasing 22%, and Alderazyme, up 40% year-over-year. Vinzim, our second largest revenue contributor, was uneven compared to the prior quarter, typical of ordering dynamics with our mature global brands.
Cristin: Now the strength of Palomziq is based on the opportunity for potent and durable fee reduction to international
Cristin: The unrestricted diet potential that Palin Zeke offers is an important product characteristic that we think will bring tremendous value to the adolescent P.K.U. population. Should we get approval next year?
Cristin: 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.
Cristin: 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&D. Greg?
Greg: Thank you, Cristin. As Alexander mentioned, we recently completed enrollment in our pivotal study for Voxoga when hypocondropesia [inaudible]
Greg: We will be sharing the 52-week results next year and we will be seeking an approval for 2027 should the data be supportive.
Greg: Also new today, we are pleased to share that we have reached an agreement with the FDA on the overall clinical development plan for BMN 3333
Greg: Assuming the phase one data are supportive, we plan to initiate a registration enabling study in 2026 supporting our previously stated ambition for 2030 approval date.
Greg: We will continue to engage with regulators around the globe to refine our plans in the coming months.
Greg: In the ongoing phase one study in healthy volunteers, we've enrolled multiple cohorts and looked 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 2026.
Greg: We're also pleased with the numerous data updates across the portfolio so far this year.
Greg: 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 Voxogo in children under three with Acondriplasia
Greg: No, treatment-related adverse events or dose interactions were reported among the 63 children followed for up to two years. [inaudible]
Greg: The study's safety results, including an instance 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 andocrinology, earlier this year.
Greg: Also at ACMG, Palinzik Data, demonstrating the importance of sustained fee reduction and the positive impact this can have on health-related quality of life outcomes were presented.
Greg: Most notably, mood and attention scores for patients achieving sustained C levels, less than or equal to 120 micromolar per liter were significantly better than those at higher levels, suggesting that sustained C in the normal range is it both achievable and may provide additional benefits for adults living with PKU.
Greg: These significant and sustained reductions in blood fee levels, coupled with the opportunity for an unrestricted diet, are unique characteristics of power and seek, the only enzyme substitute therapy for the treatment of PKU.
Greg: These benefits demonstrated in adults with PKU give us continued confidence in the Treatment Profile of Paloanzeak, including for adolescents, as supported by the positive pivotal data in the 12 to 17 year old group as announced in April .
Greg: We have done extensive PK-PD modeling based on pre-clinical data to determine what value at 25 weeks will predict muscle-distrofen levels of 10% or higher at steady state, which we anticipate to occur out at 52 weeks or later.
Greg: These values were informed by our experience with BMN351 in pre-clinical mouse and non-human primate studies, as well as from prior human experience with other phosphorythiawate organucleotides
Greg: The model tells us that levels at 25 weeks can be expected to rise two to three times higher by the time steady state is reached.
Greg: We also want to measure how these patients feel in 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.
Greg: The next update on this program will be shared at a scientific congress in the second half of this year.
Greg: 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? Thank you.
Speaker Change: Thank you, sir, and everyone just a reminder that it is star one if you have a question. We'll go first to Phil Nadeau, TD Cowan.
Phil Nadeau: 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...
Q2 being relatively similar. We're just curious whether
Phil Nadeau: 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 be a down quarter of Q2. And then second, the finance question on exposure to tariffs. I think your guidance as to the current tariff situations are very clear. Thank you very much.
Phil Nadeau: 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...
Phil Nadeau: Maybe should that situation be retaliated against with a 25% tariff from the EU on the US? Thanks [inaudible]
Brian: Thanks Phil, this is Brian , I'll take all of those, appreciate the questions.
Brian: First on Voxogo, I think you heard accurately in terms of the trends for observing, coming out of Q4 last year here in Q1 and then into the first half of this year. Thank you very much.
Brian: You know, we had previously commented that we expected most of the voxelgo revenue growth to come in the second half of the year. And the first slide point too is what we're observing are
Global Order Dynamics [inaudible]
Eric Foner
which are different from...
Speaker Change: and it doesn't take much for some of the purchasing patterns between, again, the end of 24, Q3, Q4 into Q1, Q2, for those to look flatish, but importantly, at the same time we are steadily increasing patience from quarter to quarter. [inaudible]
Speaker Change: 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.
Speaker Change: So that's the key so-go matter. And on tariffs, you heard accurately because we have immaterial exposure in our business to China, Mexico, Canada.
Speaker Change: We do not have a material impact in our reaffirmed guidance today includes any modest or immaterial impacts from from tariffs in those regions in terms of potential future pharmaceutical tariffs.
Speaker Change: We are modeling all scenarios and evaluating all of the potential mitigating tactics.
Speaker Change: We're gonna hold off on speaking to specifics including quantifying any of those scenarios . . . .
until we have more clarity and announcements and...
Speaker Change: Many of those are in the same categories that you're hearing some of the larger biopharmas discuss on their Q1 calls.
Speaker Change: that includes Global Supply Chain Strategies, which also for us includes our Global Manufacturing Network.
Speaker Change: and then lastly, monitoring potential global tax reforms, you know, in connection with or in response to the overall tariffs. So, you know, we've got a dynamic global business
Um...
Speaker Change: Our products are sourced from both the U.S. and outside the U.S. [inaudible]
Speaker Change: and just a reminder as well that from an overall exposure to U.S. sales imports...
Speaker Change: 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, you know, insulating factors should we see pharmaceutical tariffs, but we're going to hold off on speculating on any details at this time.
Fair enough, thanks for taking our questions.
Speaker Change: And when we think about the data that you'll give us for BMM333 this year, can you maybe characterize the level of detail we'll get before you kind of present the full data to Congress next year? Thank you.
Speaker Change: Thanks very much Tommy, I'll handle the question on BD and then hand over to Greg.
Speaker Change: So, we're continuing to be very excited about the potential for...
B.D.
for Biomarin, based on our strong capabilities. [inaudible]
Speaker Change: 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. We've been looking systematically at opportunities. Thank you very much.
Speaker Change: And, you know, just to reiterate what we're looking at is things which are lined up with what Biomarin is really good. So genetically defines conditions.
Speaker Change: We're looking at clinical stage assets in particular as well as obviously earlier assets, or has 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 [inaudible]
Speaker Change: So yeah, everything is very much on track. We continue to focus on doing at least one business development deal this year.
Speaker Change: We're looking forward to providing updates as when that makes sense and obviously as the market continues to develop, you know, it only creates more value from our perspective and makes those deals even more attractive from a investor standpoint. [inaudible]
And with that, I'll hand over to Greg. Thank you.
Greg: Thanks, Alexander. With regard to the MN333, just as a quick reminder, we have an ongoing phase one 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.
Greg: 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 one, we anticipate in the first half of next year, we'll be releasing that publicly. Thank you very much.
Greg: The next question today will come from Joseph Schwartz, Filiourink Partners First.
Speaker Change: Hi, this is Denise on for Joe. Thank you for taking your question. I think for previously you guys have mentioned that Voxogo has penetrated just under 20% of the market in the Contraplasia as is the second quarter last year.
Speaker Change: 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 turning over time? Thanks.
Speaker Change: Now, as we've previously reported, we've had quite an uptick in numbers in the 0-4 population, and just to remind you that we just got that label expansion at the end of 2023. So our new patient starts have been very strong in the 0-4 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 off-sogo.
Speaker Change: 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 5 plus age group, and that's where we're definitely still targeting, there's still opportunity there in the US. [inaudible]
to continue to target those patients.
Speaker Change: 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. [inaudible]
Speaker Change: But where we see the most growth in terms of prescribers is in the pediatric endocrinologist.
Speaker Change: which is very in line with our strategies to expand there.
Speaker Change: We're also investing in increased field personnel, again, to target prescribers.
Speaker Change: to ensure that we're that we're driving awareness and adoption therein. And then when we think outside of the US 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. Thank you very much.
Speaker Change: And then I should say, I'm going to 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 and a really focused and agile way. [inaudible]
Unknown Speaker: Thank you, next wish. Thank you, and up next is the cost to worry, Geoffrey's [inaudible]
Unknown Speaker: Hi, this is Lucky on for Akash. Thanks so much for taking the question. Just on the DMD corrector.
Unknown Speaker: 351, when we looked at the recently published mouse data, it looks like there's some mile liver signals in those mouse models at the higher exposure and some inflammation in the liver and muscle in those mice.
Unknown Speaker: 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.
Yeah, thanks for the question. This is Greg Friberg. These-
Unknown Speaker: Observations are class effects for the ASO categories, and so 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 phosphofioate, instead of other available chemistry classes. [inaudible]
because we do believe...
Unknown Speaker: That this is a case where it's all about opening that therapeutic window.
Unknown Speaker: Again, there's two ways to address the therapeutic window. One is to limit toxicity. The other way is to 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, they get the drug where we want it to be. [inaudible]
Unknown Speaker: Those are the open questions, I think for almost any molecule in the space and we're watching patients very closely for a variety of toxins.
Unknown Speaker: Studies, 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 a dystrophin assays on muscle biopsies that we would present the totality of our safety data that's available at that time.
The next question today is from Jessica Fye, JP Morgan. [inaudible]
Hey, guys, good afternoon. Thanks for taking my question.
Speaker Change: So Biomarin continues to execute across the business. However, the market seems worried about long-term competition for Vox OGO.
Speaker Change: Can you talk about how you think you can best address the market's perception of Voxogos long-term value, particularly beyond 2030? Thank you [inaudible]
Speaker Change: I'll start on the commercial execution here in the nearest room, and if you want to talk about some of the life cycle management, I think it's that [inaudible]
me.
Speaker Change: Probably a nice app, but thanks for the question, Geoff. So I certainly recognize where you're coming from in terms of the market perception. And what I can really say is that with Fox Sogo, 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 stage treatment paradigm. We're really focused in and around this notion of a start and stage treatment paradigm. We're really focused in and around this notion of a start and stage treatment paradigm.
Speaker Change: 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. It's really based on our ability to help families and prescribers feel comfortable with the growing body of evidence, which includes 6000 plus
Patient years of efficacy and safety data.
Speaker Change: We've got H from Mendis, 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 [inaudible]
Speaker Change: And importantly, we have a growing footprint in countries outside the world reminding everybody that only 10% of the total addressable patient population is in the United States [inaudible]
Speaker Change: And we have an incredible commercial footprint that goes into many, many countries, as you guys know, we're now 49 countries. We have Voxogo access and we're looking to build that integrator in 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, you know, a little over 25% at the midpoint for the year. And we'll continue with the 25% Kager out into the future. But Greg, I don't know if you maybe should speak to some of the life cycle options. We have a-
of these patients where they contemplate. [inaudible]
Speaker Change: Body Proportionality, Quality of Life Data, and of course bone strength and bone integrity. These are the kind of data that we will, we have already put out, but we're also going to continue to grow and put out what patients. [inaudible]
Speaker Change: and their family's want is they want a safe and dependable drug and we think that that's exactly what Voxogo is. It's a targeted therapy for that's tailored for these patients that they conjure pleasure and again that long-term experience. [inaudible]
Speaker Change: 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, you know, continue to develop the agent. We also, of course, are invested in our next generation agent.
Speaker Change: 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. We're very much looking forward to the PK data that we'll see later this year.
Speaker Change: So, again, a lot of confidence that Voxogo is a safe and effective therapy.
Speaker Change: Should we expect the same and point for 333 in a pivotal trial as for Vox Ova? No.
Speaker Change: So we're envisioning that we will do a comparative effectiveness study, a versus Voxogo, and we'll be looking at similar endpoints to what has been studied in previous, previous.
examples.
Thank you.
The next question comes from Paul Matthews.
Paul Matthews: Hey, thanks so much for taking my questions. I had one from Brian and one clinical question. Brian , I want you to clarify as it relates to the box. So, over the past couple quarters, and then what you're seeing going into 2Q, has the net price in a global basis of the drug chain, just curious if the price has gone down a bit as you diversified into other markets and your expectation there. And then as it relates to this 333-tip, that was super interesting to hear the Colorado comparative design. Are you looking to actually show superiority? Good morning.
Speaker Change: Is it going to be power for non-inferiority? I mean, maybe it's too early to say, but you know, given your conviction that higher exposure may drop greater efficacy, what do you see the opportunity to show there? Thank you.
Speaker Change: Yeah, this is Greg, maybe I'll take the 3-3-3 question because of the quick answer, yes, we are looking for superiority in growth dynamics from a higher exposure of CNP.
Speaker Change: Greg, thanks Paul for the question. Mine is likewise a quick answer because and it's a great question when you talk about you know demand increasing and and not seeing the same correlation and revenue for these couple quarters. Thank you very much.
Speaker Change: We are not observing 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, no price factoring in. [inaudible]
Make sense, thanks very much.
The next question is from Ellie Merle, UBS. [inaudible]
Speaker Change: Hi, this is Jasmine on for Ellie, thanks so much for taking our question. [inaudible]
Speaker Change: So, what's the latest on timing for a VoxoGo and other growth disorders like ISS and shocks? And then how quickly do you think that you could move three-to-three here? So, would you want to wait for VoxoGo clinical success here before studying three-to-three in these indications? Or is that something you'd want to do sooner? Thank you.
Greg Friberg: Thank you for the question. This is Greg Friberg again. With regard to the new nuns' turner and shocks basket that we have as well as idiopathic short stature for the new nuns' turner.
Greg Friberg: 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 333 would play in additional indications. Thank you very much.
Greg Friberg: But just as a reminder, we still are working through with three through three, once we've moved beyond healthy volunteers, there will be both dose ranging as well as the effectiveness studies to come thereafter. And that's the agreement again that we referred to with the FDA. Long way of saying that these...
Greg Friberg: Reveals of information are actually going to line up quite nicely so that we can make decisions when we have the complete profile for 3-3-3 to determine what the future plans would be. Right now, the discussions with 3-3-3 that we've been talking about have been a kind of pleasure related. Thank you very much.
Thank you!
The next question is from Corey Casimo, Evercore ISI.
Speaker Change: Hi, thanks for taking our question. This is Adi on For Cory. I wanted to ask you if you can share how the US versus XUS sales have evolved in the first quarter for Vox Sogo and what trend do you expect to see going forward into the rest of the year? Thank you very much.
Speaker Change: Yeah, thank you so much for the question. This is Kristen. And as you know, last quarter, we gave specifics on precisely that, which is the US versus outside US revenues for Voxogo. And we did that strictly so we could clarify the significant contributions that come from the outside of the US. And given that the ex-US weight of the revenues is approximately 75% total of Vox revenues, [inaudible]
Speaker Change: 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.
Speaker Change: 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 one third of the revenues coming from the US and Tuesdays coming from outside to you. [inaudible]
Speaker Change: Perfect, thank you, I'll jump back in the queue for a rest of the question [inaudible]
We'll go next to Gena Wang Barclays.
Thank you, I have a two. [inaudible]
Speaker Change: Sets of questions, first one is regarding also the Voxogal franchise. Thank you guys.
So maybe the 333, can you? [inaudible]
Speaker Change: Help us understand because you have a healthy volunteer data, and the mainly is a PT data. How do you define, you know, that would be the top line data you share with us, that would be good enough.
that defined as a supportive. [inaudible]
to start your registration trial. And I will you...
Only identify one dose for your registration trial.
Speaker Change: And then related questions I know there is a competitive running...
Speaker Change: CMT Plus, Gross Hormons Studies, any thoughts there? We know historically Gross Hormons didn't work. [inaudible]
Speaker Change: So maybe any thoughts there regarding CMP plus girls' home and study?
Speaker Change: and the third pub very quickly on the manufacturing site, Phil Boxogo. Do you have a manufacturing site both in the US and the XUS?
Speaker Change: So thank you, Gina, first question with regard to 333, you're correct. Thank you very much.
Speaker Change: We will have PK data of all the species, so we'll have not only BMN333, but also FreeCNP levels [inaudible]
Speaker Change: Our goal for that study is to see multiple increases in sustained exposure levels.
ReCNP, 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 multi-part protocol that involves both a dose ranging portion as well as a, you know, a comparative effectiveness portion. Thank you.
Speaker Change: 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 acondroplasia, to do that dose ranging study, to pick that final dose, again, that will be in the comparative of that dupe this side.
Speaker Change: On the growth hormone side, I think 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 Acondruplasia patients, that you would see an increase in AGV at 6 or 12 months, maybe even out at 2 years. But historically the challenge for growth hormone hasn't been about those short-term benefits. [inaudible]
Speaker Change: 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. [inaudible]
Speaker Change: 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 and 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 acondriplasia. On the contrary growth hormone really has added problems for patients.
Speaker Change: And then, you know, one unique factor that's been brought up by some of the treating physicians, you know, things like consular 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 Voxogo is, you know, remember, these kids aren't even growth hormone deficient. [inaudible]
Speaker Change: Thanks, Greg. Hi, Gena. This is Brian . I'll take your question on voxelgo manufacturing. Voxelgo drug substance is sourced from the United States.
Speaker Change: Thank you. Up next we'll take a question from Kostas Biliouris, BMO Capital Markets.
Costa Spilloris: Thanks for taking our questions and congrats on the progress. Maybe one question.
For Gregon Piemensri, Fri, Fri, Fri.
Costa Spilloris: There is a recent presentation by Children's National Hospital at ESPE, that shows that there is no really meaningful correlation between Voxogopique and growth outcomes in Hypochondroplasia.
Speaker Change: So given these data, I wanted to what extent will we be able to draw conclusions about BMN three-finity outcomes based on the PK data that we will see in 2025? Thank you.
Speaker Change: Thanks for your question. I think when we talk about Voxogo PK, we also have to recognize the challenges when a drug has about a 30 to 60 minute half-life of actually getting the complete profile. You know, trough PK becomes very challenge. Well, trough PK if you're going to measure a very low point is easy, but measuring that full profile can be a challenge. There is a... [inaudible] You know, trough PK, you know, trough PK,
Speaker Change: Fundamental difference between the PK profile of Voxogo and what we expect to see in BMN333, of course, rather than a sawtooth pattern we expect to see a much more of a continuous release. Thank you.
Speaker Change: Fundamentally different approaches with the pulsatile versus the continuous coverage make it difficult to measure, I think, between the two and compare the P.K. Profiles, but what we can do is we can look at other long-acting CNP, both in pre-clinical models and the pre-clinical outcomes. And we can ask the question about sustained levels of CNP. That's what we've modeled from our cinemogus monkey models. And we believe that again, if we can recapitulate [inaudible]
What we've seen in those models...
Speaker Change: and more continuous coverage and activation of NPR-2. 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 that answered your question. Thank you.
Mohit Bansal from Wells Fargo is up next.
Speaker Change: Hi, this is Serena on for Mohit. Thanks for taking our question. I wanted to ask the question about BMN35351. In terms of the lots of distribution data, expect in the second half, is that?
Speaker Change: 10% district and 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.
Speaker Change: Thanks for the question. What we will have, once patients have reached the six month time point, as we of course will have a totality of safety data and PK on a variety of species.
Speaker Change: 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.
Speaker Change: 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've put forward. And that model shows us that what we're going to see at the 25, 26-week time point [inaudible]
Speaker Change: You need to wait till, of course, to 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, I've been a relay race. That's trail.
Unknown Executive, Brian Mueller
Our next question is from Olivia Brayer, Cancer Fitzgerald
Speaker Change: Or could we actually start to see some initial clinical characterization and maybe select patient groups just to help de-risk some of those new voxogo indications?
Speaker Change: 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?
Speaker Change: So what do I take a quick stab, just to clarify, you know, with regard to the short stature conditions, obviously, I mean the Dr. Dobber study with VoxoGo continues and we do expect that that data will be updated over time.
with regard to our own programs.
Speaker Change: 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 three steps.
bodies.
And hi, Olivia, it's Brian on your...
Speaker Change: The profitability question. Thanks for that. Yeah, we are pleased with this strong start to the year.
$1.13 of Williams per share of growth.
Again, we are expecting while revenues are planning to...
Speaker Change: Increase in the second half of the year, we are also planning to increase investments in both R&D and S-GNA over the course of the year
Speaker Change: But by all means, we are confident in the four year EPS guidance with the
this impact
Speaker Change: So whether it be the goal of $4 billion of revenue by 2027,
Speaker Change: 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, you know, OpEx line item goals over time and see that we are expecting. We are expecting.
Speaker Change: Significant earnings per share of 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.
Okay, thanks guys, helpful.
Speaker Change: We have reached the end of the Q&A session. Our final question today will come from Vikram Purohit, Morgan Stanley .
Vikram Purohit: Thank you, good afternoon, thanks for fitting me in. We just had two quick clarification questions on BMM-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.
Vikram Purohit: with the bar for approval, necessarily B, 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, X, U, S as well? Thank you. Thank you.
Vikram Purohit: Thanks for the question. Just to clarify, the announcement today, again, was that we had reached agreement with the FDA and were continuing to work with regulators around the globe.
Vikram Purohit: The study that we refer to that would initiate in 2026. [inaudible]
Vikram Purohit: is a combined phase two, three study. And in that regard, we are doing dose ranging as well as the final comparative effectiveness versus Voxogo. And we would be looking for a superior outcome with regard to the primary endpoint in that study versus the controller.
Speaker Change: 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. Thank you very much.
Speaker Change: in the face of external dynamics that are creating uncertainty are some number of areas we think we are well positioned. Thank you very much.
Speaker Change: and our commercialization capabilities and most importantly continuing to deliver increasing value creation all of our stakeholders in 2025 and beyond.
Speaker Change: Thank you for your attention, look forward to speaking with you soon.
Speaker Change: 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. Next.