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Operator: Good afternoon, and welcome, everyone, to the BioMarin Pharmaceutical Second Quarter 26 Conference Call. Today's conference is being recorded. All lines have been placed on mute to prevent any background noise. After the speakers' remarks, there will be a question-and-answer session. If you would like to ask a question during this time, simply press the star key, followed by the number 1 on your telephone keypad. If you would like to withdraw your question, press star 1 again. Please. At this time, I would like to turn the conference over to Traci McCarty, Head of Investor Relations.
Traci McCarty: Thank you, operator, and thank you all for joining us today. 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 therapy 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 8-K 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 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. Moving to slide 3 and introducing BioMarin's management team joining today's call, Alexander Hardy, Chief Executive Officer Cristin Hubbard, chief commercial officer Gregory Friberg, Chief R&D Officer and Brian R. Mueller, Chief Financial Officer. I will now turn the call over to Alexander to provide our quarterly highlights. Alexander?
Alexander Hardy: Thank you, Traci, and thank you all for joining us today. Starting on slide 5, BioMarin delivered a standout second quarter combining strong growth to nearly $1 billion in revenue with the successful close and integration of Amicus while delivering on milestones that strengthen our leadership in genetic conditions. Our strong performance demonstrates both the value creation of our portfolio and the continued execution of our commercial organization, executing at scale integrating meaningfully accretive assets and continuing to innovate. Enabling us to bring important medicines to people living with rare diseases as we enter an exciting new phase of growth. Second quarter highlights start with 20% year over year total revenue growth. Accelerated by a more diversified portfolio and setting up an even stronger second half of 26. To be fueled by full third and fourth quarter Galafold and Pombility and Opfolda contributions. And sustained demand across our other products Turning to Voxzogo. Double digit revenue growth in both The US and international markets led us to increase Voxzogo's full year guidance putting it on a path to become BioMarin's first $1 billion product. In its first quarter, with a US competitor, the continued revenue growth demonstrates our ability to defend Voxzogo's leadership position. Building on this momentum, we are pleased to share that we have submitted our sNDA for the approval of Voxzogo to treat hypochondroplasia based on strong pivotal data shared during the quarter. We will provide an update on the submission as part of our third quarter results. Turning to Amicus. As anticipated, when we announced the acquisition, this deal demonstrates that BioMarin leverage our scale and capabilities to rapidly integrate high growth assets to maximize value creation. Today, we provide estimated peak revenue for Galafold of $1.4 billion and for Pombility and Opfolda of $1.2 billion. Together, these innovative therapies combined with significant cost synergies, expected to reach approximately $220 million annual run rate in 2028 are anticipated to drive substantial EPS accretion. And operating cash flow. Most importantly, we look forward to bringing Galafold and Pombility and Opfolda to more patients with Fabry and Pompe disease worldwide. Briefly on our pipeline, which continues to build momentum, We recently added BMN 28 formerly DMX 2 hundred, an exciting new late stage pipeline opportunity resulting from the Amicus acquisition that Gregory will expand upon in a moment. I am also impressed by the speed at which we submitted the sNDA for Voxzogo for the treatment of hypochondroplasia. The speed of our submission reflects the benefits of our investments in AI capabilities, and sets the new standard for how BioMarin will execute going forward. Together, these results tell a clear story BioMarin is executing a scale raising guidance, outperforming on the Amicus integration, with substantial combined peak revenue potential ahead. And advancing pivotal pipeline data towards Voxzogo's next indication. As we enter the second half of 26, BioMarin is stronger, more diversified and better positioned than ever to lead in rare disease. And deliver for patients worldwide. Now on slide 6. The addition of Amicus transforms BioMarin's growth trajectory through the mid-2030s. With a combined peak revenue potential of $2.6 billion from Galafold and Pombility and Opfolda and significant cost synergies layering in along the way, we expect meaningful non GAAP EPS accretion. Expanding operating margins, and stronger operating cash flow. Hiring our next phase of growth. Moving now to slide 7. And starting with Galafold. A growing product in a growing market. From a $522 million base in 2025 we project peak revenue of $1.4 billion by the mid 20 thirties. Supported by 2 complementary growth drivers, First, we see significant opportunity to expand diagnosis and treatment. Leveraging BioMarin's proven diagnostic capabilities our goal is to more than double the number of US patients treated with Galafold. We plan to do this through scaling AI enabled patient identification initiatives expanded genetic testing, newborn screening, and family cascade screening, helping more amenable patients access treatment earlier. Second, we see meaningful opportunity to expand market penetration globally. With Galafold already established in 40 countries, we intend to deepen penetration within existing markets, while selectively expanding into new geographies leveraging BioMarin's global commercial infrastructure to accelerate access and broaden reach. Together, these drivers are expected to support an approximately 10% CAGR from 2027 to 2032. Turning to Slide 8. Pombility and Opfolda. Is at an earlier stage in its commercial journey compared to Galafold. We estimate $1.2 billion in peak revenue by the mid- to late 2030s growing at a greater than or equal to 20% CAGR from 2027 to 2032. We expect gross to be driven primarily by increased patient switching and global market expansion complemented by continued improvements in diagnosis and treatment rates. We believe switching will be supported by growing awareness among health care providers and patients of the expanded body of real world evidence demonstrating the benefits of Prombility and Upfolder on disease outcomes. At the same time, we plan to leverage our diagnostic capabilities to identify and support treatment of additional eligible patients across our global footprint. These growth drivers are expected to be further strengthened by planned expansion into more than 20 additional markets over time. Turning to Slide 9. We are pleased with the significant cost synergies identified which we expect to contribute to substantial EPS accretion beginning next year. We anticipate approximately $220 million of synergies to be fully realized in 2028. Representing a roughly 50% reduction from Amicus' 2025 non GAAP operating expenses of $432 million These synergies are weighted towards G&A which makes up more than 70% of the total with the remainder coming primarily from R&D. As planned, we retained Amicus' commercialization team to ensure patient continuity and supplement our global capabilities. These synergies combined with peak revenue align with the value creation we anticipated when we announced the deal last year. And demonstrate our ability to successfully integrate large, accretive assets that strengthen our growth profile. Turning to Slide 10. By accelerating our financial profile with the addition of accretive assets that benefit from our proven global expertise, serving patients with genetic conditions, The Amicus acquisition sets the stage for our next phase of growth. With peak targets of $1.4 billion for Galafold $1.2 billion for Pombility and Opfolda. These revenues combined with $220 million in anticipated annual cost synergies fully realized in 2028, support substantial expected non GAAP diluted EPS accretion beginning in 2027 and a significant increase in operating cash flow. Galafold and Pombility and Opfolda combined are expected to reach over 60% non GAAP operating margin by 2030, At the same time, we believe our rapid integration and growth plans will enable us to deleverage approximately 1 year sooner than initially communicated. This quarter reinforces what sets BioMarin apart. We are the leading rare disease company operating at scale, with a proven integration capability to maximize the value of high growth assets We look forward to updating on our progress scaling Galafold and Pommability in our folder as we enter the next exciting phase of BioMarin's growth. I will now turn the call over to Cristin for commercial update. Cristin?
Cristin Hubbard: Thank you, Alexander. The second quarter demonstrated the strength and growing diversity of our commercial portfolio. Now turning to Slide 12. Galafold and Pombility and Opfolda are off to a strong start. As we move quickly to integrate following the April close. On a pro form a basis, second quarter revenue for Galafold grew approximately 10% year-over-year, and Pombility and Opfolda grew over 65%. Galafold delivered broad based patient growth across both established and newer markets. This was driven largely by increased diagnosis and patient identification including continued success with family cascade screening and expanding newborn screening programs alongside ongoing gains in reimbursed access. For Pombility and Upholda, continue to add patients both in The United States and in more recently launched geographies, supported by its differentiated clinical profile as we help more physicians better identify disease progression on prior therapies. Importantly, both brands maintained commercial momentum while we rapidly integrated. A testament to the focus our combined team has kept on patience and execution. These are the levers Alexander described, and the results to date reinforce our confidence the long term opportunity for both medicines. Turning to Slide 13 and the broader metabolic conditions business unit, formerly known as enzyme therapies. With the addition of the Amicus medicines, we have changed the name of business into the Metabolic Conditions business unit to better capture the breadth of our portfolio. Total metabolic conditions revenue was $695 million and grew 25% year over year, inclusive of Galafold and Pombility and Opfolda, And the number of patients on therapy grew across every 1 of our marketed metabolic conditions brands, both year over year and sequentially. Palynziq revenue grew 27% year over year on continued patient demand while also benefiting from order timing in The US during the quarter. We were pleased to have recently received European approval to broaden the Palynziq label to adolescents ages 12 and older with PKU. In The US, we have had a strong start in the adolescent age group. And the team is energized to have the opportunity to serve this population more broadly. Across the rest of the portfolio, revenue in any given quarter reflects the timing of large orders. In the second quarter, order timing was a headwind for Vimizim following a strong first quarter, while it was a slight tailwind for Naglazyme ahead of an expected lighter third quarter. Because of these dynamic shifts between quarters, our full year 14. Voxzogo delivered 14% year over year revenue growth in Q2, driven by double digit growth in The U. S. And OUS markets. The number of children treated with Voxzogo grew more than 20% year over year globally, and approximately 3 quarters of Voxzogo revenue was generated outside of The US. Notably, even in the first quarter facing a US competitor, the number of children in The US treated with Voxzogo increased year over year. We also saw continued traction in the under-2 age group, represented more than half of the new US patient starts in the quarter, reinforcing our position as the only approved treatment for children 2 and younger. As expected, we did see switching to the competitor products since it was approved in February of this year. Since the competitor's launch, approximately 90% of The US children treated with Voxzogo remained on therapy as of the end of July based on information available to us. That reflects the continued confidence physicians and families place in Voxzogo's evidence base. Internationally, momentum remains strong across both established and newer markets. We expect both patient additions and order timing to drive higher total Voxzogo revenue in the second half of 26 compared to the first half. We are confident in Voxzogo's durability, built on a growing evidence base and the experience of the thousands of children treated to date and their caregivers. That durability is further reinforced by our exclusive ability to treat patients from birth worldwide. Our ambition is to remain the leader in skeletal conditions through competitor launches in the near term. Supported by Voxzogo's anticipated launch for achondroplasia in 2027 and the potential of BMN 3 should data be supportive. Stepping back, Voxzogo was on track to become BioMarin's first $1 billion product. Galafold and Pombility and Opfolda are on the path to join it. Each carrying peak revenue potential well above $1 billion and Vimizim is expected to also reach the $1 billion mark over time. Together, they are a clear sign that we can take innovative genetically targeted medicines and expand their reach worldwide. With that, I will turn it over to Gregory.
Gregory Friberg: Thank you, Cristin. The second quarter was a productive period for our pipeline with meaningful progress across our portfolio. Turning to Slide 16. You can see that we have had a lot of positive news over the last few months, I am particularly pleased to highlight our very recent submission of the supplemental NDA for Voxogo for the treatment of hypochondroplasia. By implementing parallel work processing, aided by technological advancements, we were able to shrink the time from database lock to filing down to just 79 days. Easily within the top quartile for modern industry benchmarks. The full phase 3 data will be presented at SP as a late breaking oral presentation in September. As Kristen noted, we are also very pleased that adolescents in both The US and Europe will now have access to Palynziq, following the label expansion in both regions earlier this year. Moving now to slide 17 and BMN 28, formerly known as DMX-200, a late stage addition to our pipeline resulting from the Amicus acquisition. The 20 is a first in class oral CCR2 inhibitor, which it accomplishes through blockade of receptor heterodimerization. It is currently in phase 3 development for focal segmental glomerulosclerosis, or FSGS, This is an asset for which we hold exclusive US commercialization rights in our partnered with Dimerix. Who remains responsible for operationalizing the phase 3 study. If the data are supportive, this could provide a new mechanism of action for the treatment of FSGS. FSGS is a progressive kidney disease that leads to proteinuria and declining kidney function over time. There are an estimated 30 thousand addressable patients in The United States. Only 1 therapy is approved as of today, and its label is somewhat narrow, excluding patients with nephrotic syndrome. Durable stabilization of kidney function remains a significant unmet need. BMN 28 targets an orthogonal mechanism to the vascular targeting agents with the potential to treat a broad FSG population. It has shown a favorable safety and tolerability profile to date, The FDA has agreed that proteinuria is an appropriate endpoint for approval in our Phase III ACTION III trial, and we expect Phase III data in 2028. If the data are supportive, e m n 28 represents an attractive new pipeline with upside in a large area of unmet need. With that, will turn the call over to Brian. Brian?
Brian R. Mueller: Thank you, Gregory. Please refer to today's press release for detailed second quarter 26 results including reconciliations of GAAP to non GAAP financial measures Which will also be available in our upcoming Form 10 Q. Turning to slide 19. We were pleased that second quarter revenue reached nearly $1 billion, representing 20% top line growth year over year. Second quarter non GAAP operating margins was 36.4%, with non GAAP diluted earnings per share of $1.20 Non GAAP R&D and SG&A expenses each increased year over year. Reflecting the operating expenses of the acquired Amicus business, together with continued investment in our pipeline and commercial execution. On a GAAP basis, second quarter SG&A results also included approximately $84 million of transaction and integration related charges associated with the acquisition. These charges are excluded from our non GAAP results. Below the operating income line, interest expense increased year over year due to the acquisition debt financing, and interest income decreased as we liquidated investments to fund the acquisition. These items, along with the higher operating expenses, contributed to the year over year decrease in non GAAP diluted earnings per share. I want to spend a moment on our interest expense and interest income to make sure your expectations are aligned with ours. Based on current interest rates, interest expense associated with the acquisition debt financing, is estimated at approximately $200 million on an annualized basis. approximately $50 million per quarter. With the term loans and senior notes scheduled to mature after 2030. Importantly, this interest expense is included in our non GAAP results and therefore reduces non GAAP diluted earnings per share. In addition, due to lower cash and investment balances, following the close of the acquisition, we expect interest income to decrease year over year in the near term. Turning to slide 20, and our updated full year 2026 guidance. On the strength of our first half performance, and our expectations for the balance of the year, We are raising our full year total revenues, Voxzogo revenue, and non GAAP diluted earnings per share guidance. As you can see, our guidance updates today reflect double digit growth from the midpoint. And our strong trajectory leading into the second half of 26. Briefly on phasing, we expect third quarter revenue to be slightly higher than the second quarter. Reflecting a full quarter of Amicus revenue contribution and continued patient growth across our brand. Similar to prior years, we expect the fourth quarter to be our strongest quarter of the year, with a significant step up versus Q3 and representing well over 50% of our second half revenue outlook. Primarily due to ordering dynamics in select markets. Our non GAAP diluted earnings per share the third quarter will reflect a full quarter of Amicus operating expenses while benefits from cost synergies are expected to become more meaningful in the fourth quarter. of synergies, Combined with the anticipated revenue phasing and realization we expect third quarter non GAAP earnings per share to be slightly higher than Q2 and fourth quarter non GAAP earnings per share to be significantly higher representing the highest quarterly earnings per share of the year. In summary, second quarter reflected strong execution disciplined investment and continued progress integrating Amicus. While some integration activities will continue into next year, the integration is well underway and on track with the majority of enabling decisions made and operating plans in place. We are impressed by and appreciative of the focus and efforts of both our BioMarin colleagues and all of our Amicus colleagues since the close of the acquisition. We are looking forward to the second half of this year where we remain focused on delivering our updated 2026 outlook while building towards the longer term revenue and earnings potential that Alexander outlined in his remarks. Thank you for your attention. We will now open the call to your questions. Operator?
Operator: Thank you. We will now begin the question-and-answer session. You have dialed in and would like to ask a question, please press 1 on your telephone keypad to raise your hand and join the queue. 1 again. We will go to our first question from Christopher Raymond at Raymond James.
Christopher Raymond: Thanks for taking my question. I got 2 actually. First, on Amicus, you guys gave a lot of metric here on synergies with this integration. Just looking at 28 synergies, they are look really impressive. I was just wondering if maybe you could maybe give a few waypoints as we get through 27 and tell us the specific steps you are taking to get to these numbers. And maybe what is behind the delivery of the 2.5 leverage a year early. Thanks. And then I have a Voxzogo question.
Brian R. Mueller: Hi, Christopher. it is Brian. I will take that. Thanks. So yeah, just to start, we know we communicated, a lot of metrics today. We do believe that this Amicus integration framework demonstrates the comprehensive value creation from this acquisition. We quantified synergies at this approximately 50% level. that is going to drive significant accretion powered by the, the revenue growth as well. Not only validates our hypothesis at the time of the transaction, but exceeded our expectations at the time And I would also like to emphasize that these decisions are made. We are now executing on an end to end integration plan to bring the Amicus business onto the BioMarin platform. And as I noted there in the prepared remarks, it took a lot of work, on both sides, and it is going very well. This accretion also translates to cash flow. As you noted, we pulled forward our leverage target by approximately 1 year. And all together, we are not only delivering on the potential of this transaction, but exceeding it And specific to your question in terms of waypoints, we shared that we expected the transaction to be modestly dilutive in calendar 2026. I will share that is still the case, but it is honestly close to breakeven. Still expecting it to be accretive in the first calendar year. But I would point you to the accretion that begins next year and then 2028 being the first full year because we are still integrating next year. 2028 will be the first full year where we are realizing all of the benefits from the synergies and integration. And in terms of a waypoint, I might guide you to saying that next year we are expecting half to slightly more than half of the synergies to be realized.
Christopher Raymond: Great. And maybe on Voxago, just hearing Cristin's commentary around 90% of Voxzogo patients remaining on therapy, I think you mentioned post the UV well launch. Gave us some numbers today. I think they said, 770 patients are enrolled to start therapy, and I think that is 2 thirds of those are actually paid. Maybe just doing some math on the switchers, and based on your commentary, can you maybe give us a sense of the dynamic in terms of your competition for new patients?
Operator: Thanks very much for your question, Christopher.
Alexander Hardy: This is Alexander. Yeah. I think this is this is really important to sort of unpack the numbers and make sure there is no misunderstandings here with the various data points communicated by both companies. I mean, clearly, their number was based on a total patient enrollment number. That includes naive patients, Voxergo discontinuations whenever those may have happened. And, of course, switches from Voxergo. And, of course, the latter 1, the switch rate is, of course, the 1 that is relevant for us. So according to our data, and we have good visibility as you would expect in The United States in terms of patient numbers and ongoing treatment. We have seen approximately 10% of patients switch That translates to less than 100 patient switches. In the approximately 6 months since they were being approved. That translates, of course, to a very small impact on almost $4 billion in revenue. This year. So, you know, taking all things into account based on the strong growth that we are seeing and projecting, with 20% of increase in patients globally on Voxergo in the quarter. We feel comfortable increasing Voxzogo revenue guidance for the year for over $1 billion. I think zooming out as well, if I could comment, I think bigger picture. Their update on the launch and the pace reflects how hard The US market is in chondroplasia. When you have geographically dispersed patients, you have low visit frequency, Your care split between general pediatricians and specialists, and this all impacts the opportunity and the pace of switches and starts And, of course, as you know, for us, you know, new patient starts at dominated by the 0-to-2 patient populations. The guidelines say diagnose treat as early as possible after birth, and as you know, know, that remain we remain the only product the less-than-2 indication and we expect to remain so. For a good amount of time. So I hope that helps, gives you a little bit of perspective on the data and what we are seeing and what we are looking forward to in the remainder of the year. Yep. Thank you.
Operator: We will go next to Cory Kasimov at Evercore ISI.
Cory Kasimov: Hey, good afternoon, guys, and thanks for taking my question. So I also wanna ask something on the heels of the competitive update this morning. And I am wondering how you think about the combination of weekly CNP analog plus growth hormone eventually slotting into the treatment algorithm? And is there anything that is stopping you from or physicians from using Voxzogo and or BMN 3 in the future with growth hormone? Thank you.
Gregory Friberg: Thanks, Cory. This is Gregory Friberg. I think I will tackle that 1. You know, looking at that data, I think first and foremost, we have to recognize that the COACH study is a small study. Think it is about 21 patients single arm split into 2, 2 cohorts. So we have to be careful in over interpreting it, particularly when we slice the data. 6 month intervals. Now that being said, I think the question with the growth hormone combination today is the same as it has been from the start. We know growth hormone alone can cause, increases in AGD, but they are temporary. And, actually, they do not result in major increases in achondroplasia and increases in final adult height. So the question remains, what are we learning from the dataset? With this data point, I would say just the eyeball test. Tells us that it looks like the effects of growth hormone adding on to C and appear to be waning. I do not know why that would be in the naive patients more than the, you know, the add on to people who are already on CNP. it is a small dataset. But the question then becomes, is this the beginning of a longer term trend? it is an unanswerable question. I think the question that our endocrinologists care most about, which they are a sophisticated group, they have worked with growth hormone for a long time, is will this ultimately lead to the health and wellness and, by extension, the final adult height of patients? The concern always is that growth hormone may close growth plates And that is not something that is in a short study of 8 month duration, something that you can really get a read on. So I think today, it is incremental data. We are certainly seeing that the growth spurts might be declining. You see the slopes increases, and, you know, that is as compared to the achieved I am sorry, the approach study where you do not see that kind of a shift out at 2 years. Those happy to see that they are following up on the 2 year data there as well. And, again, I think there are unanswered questions that time will tell. Of course, we are watching this very closely. We are gonna follow the data. We are gonna make evidence based decision. From a biologic standpoint, there is nothing unique about TransCon CNP. When it comes to taking a CNP agent and, combining it with growth hormone. it is too early to tell, and we are looking forward to seeing more data, you know, out in the order of 3 years plus. Very helpful. Thanks, Gregory.
Operator: We will take our next question from Jessica Fye at JPMorgan.
Jessica Fye: I was curious if you could speak to whether those synergies associated with the Amicus deal will fall to the bottom line or whether you expect those to be reinvested in the business?
Brian R. Mueller: Hey, Jessica. it is Brian. Thanks. Great question. We do expect those synergies to drop to the bottom line. However, to your point, there is also reinvestment. So part of the strategy is to accelerate the growth potential of Galafold and Pombility and Opfolda, which will require some incremental investment. I will share with you that compared to the synergy numbers we shared today, is a modest portion of that. But more importantly, and this is why we spoke to synergies on a gross basis, any of that reinvestment fits within the existing structure of our P and L. it is part of normal metabolic condition sales and marketing going forward. So, we do not consider it an offset to the synergies itself, which will live on And again, if you chose to calculate net synergies with the investments, it is very modest. Thank you.
Operator: Our next question comes from Salveen at Goldman Sachs.
Tommie Reerink: This is Tommie on for Salveen. Curious if you could provide more detail. You spoke to the mechanisms behind, you know, driving increased diagnosis and switching for Pompility and for Galafold. How do these efforts differ in, or strategy differ in The US versus ex US? And a follow-up, what is your appetite for, future BD, and what stage or type, if so? Thank you.
Cristin Hubbard: Yeah, I will take that first part of the question, Tommie. Thank you so much. And I hope you could hear it in the prepared remarks, but I will say it again. We are absolutely delighted about what we have both the opportunity and quite the responsibility to do for both the Fabry and Pompe communities. And the more we have been able to dig into it since the close, we have really unearthed what I think are some meaningful levers that we can pull to drive the growth and therefore target the peak revenues of $1.4 billion for Galafold and $1.2 billion for Pompe excuse me, for Pombility and Opfolda in the future. Now looking specifically, and I know you asked the question kind of differentially across The US and Ex US, in large part, the overall lever or the levers are very similar. While they may get executed at the country level slightly differently, the areas that we are going to really put our investment into are quite similar and they fit very well within the BioMarin's capabilities that we have built over the decades that we have been doing this. For Galafold, this really is going to be about diagnosis. We recognize that in the medical population or in the Fabry community at large, there really is still very limited diagnosis, especially for those with late onset and or the female patients. So we plan to really target those communities trying to drive broader diagnosis and importantly, really starting to close the gap between kind of diagnosis and treatment. So that we can show that kind of physicians that treating earlier and even in milder sets or milder conditions is really important. On the Pombility and Opfolda side, this really is about accelerating switches. And this is true again in both The US as well as outside The US. And here our focus is going to be on the waning or the clinically declining patients that are on a current therapy where we believe that we can really kind of continue to show what disease progression could look like and if patients are not meeting those targets, how to ensure that they are advocating for treatment. So those are the areas we are really going to be focused.
Gregory Friberg: And I know that the next question oh, Gregory, do you have something to add? I would just add, I mean, the medical affairs standpoint, particularly for Fabry where we know, you know, I would say generously, maybe only 40% of the patients are actually diagnosed with the condition. Just to give you some granularity there, electronic health record work to again shorten the time between diagnosis and again symptom when the symptoms arrive, family cascade testing, and cost reclassification of variants. These are things that we have done previously in other settings. There are also, some great work that our former Amicus colleagues had begun, and we have the opportunity to scale that a bit larger. Just to give you a data point, there is over almost 50 different diagnostic, activities and programs going on around the globe right now. And so is a very local phenomenon. We think that we can put more firepower and technology behind some of those assets.
Cristin Hubbard: These are things that we can start right away in the countries where Galafold and Pombility and Opfolda are already commercialized, but what is also really important to note is that we plan on geographically expanding these products. So in over 10 countries relative to where we are today with Galafold and more than 20 countries for Pombility and Opfolda and which will also help to drive that growth.
Alexander Hardy: Over to you, Alexander. Tommie? Yes. This is Alexander. I will answer to your BD question. So if you as you heard from Cristin and from Gregory, you know, the integration and acceleration of Galafold and Pombility and Opfolda you know, strengthen our growth outlook for BioMarin. So, you know, we are now more diversified in growing commercial portfolio. Our focus is now more shifting to expanding our clinical stage pipeline. Of course, we are gonna continue doing know, research collaborations as we have always done. I think you just saw you would have seen the announcement recently of the collaboration with in. But as we delever, you can expect us to do deals to expand out our clinical stage programs over the next 12 to 18 months.
Operator: We will move to our next question from Philip Nadeau at TD Cowen.
Philip Nadeau: Good afternoon. Thanks for taking our questions. 2 from us. First, on the upcoming ITC case. We expect a decision by the end of August. We are curious to get your most recent thoughts on that case. And in particular, any thoughts you have on a on a the possibility of a settlement? And then second, just to follow-up on the diagnosis points that you just made. I think years ago, we heard from Genzyme that they thought they were gonna penetrate more quickly the late onset and female patient population. So you have talked about what you can do, but why have not those patients been diagnosed so far? It does seem like others have had efforts. Where are those efforts falling short? Thanks.
Operator: Thanks for your question, Philip.
Alexander Hardy: This is Alexander. I will I will take the opportunity to clarify some of the some of the timelines and the facts around the ITC case But as you can probably expect, I am not gonna get into our legal strategy or speculate on the outcomes. So on the 20 first, of this month, the 20 first of August the administrative law judge will deliver their initial determination Within weeks following that, initial determination, the commission decides whether they are gonna review that initial determination. The final decision is expected on the December 21 of this year. that is either affirming or reversing all or proportion of that initial determination. And then either party has 60 days to lobby the president He has a presidential review period, which goes through February the 20 first of next year. But I would just highlight that if a if a exclusion order is determined in that in that decision. By the commission, then it is effective during that period. So that is sort of a what you can expect from a timeline perspective. And we are waiting that data to 20 first, which is coming shortly. I would also highlight that, you know, upon the completion of the ITC process, we would expect to enforce our patents in federal district court where, of course, monetary damages are available.
Gregory Friberg: And, Philip, I am I am gonna tag this is Gregory. I am tackle your second question on the diagnostic points, if that is alright. Perfect. You know, with regard to why there has not been more, more progress, in the field, I think it is a pretty simple answer in that this is a very elusive disease. it is 1 where patients can have a very heterogeneous presentation, something like 7 different organ systems that can, you know, be affected presenting in a variety of clinics And true with all rare diseases, it is this elusiveness of diagnosis, the 7 to 10 years to actually make their way to knowing what is causing their symptoms. And Fabry is really a case study in that. that is why we are trying to focus on where we think we can have the most impact. And, again, we highlighted a couple of them. But, obviously, this is not just about edging educating, you know, community physicians to be on the lookout. Using technology, using electronic health records, using testing and training approaches to try to identify flags earlier Again, it is not just about patient finding. About shortening that time to diagnosis. I would say that on top of that, you know, 1 of the very, fruitful endeavors that we have been involved with in other genetic conditions is family cascade testing. You find 1 person in the family who is affected by this Again, you do the boots on the ground work to find the other patients that could be affected. Finding them early is the name of the game with Fabry, but it is been elusive up till now. I think it reflects the, the wiliness and, unfortunately, the heterogeneity of this disease. Very helpful. Thank you.
Operator: Next, we will go to Eliana Merle at Barclays.
Eliana Merle: So I guess, of those 10% of patients in The U. S. Who switched from Voxzogo by the end of July, I guess what trends or characteristics are you noticing in those patients versus, say, patients that are more likely to stay on Boxogo? And I guess what degree of switching in The U. S. Is baked into the guidance for this year? And then a second part of a question on VOXOGO. You mentioned that over 50 percent of U. S. New starts were ages 2 and under. Maybe just how should we think about, I guess, the annual incidence of new starts in under age 2, or maybe just the size of The U. S. Incident market? For that age group and how you are thinking about this as a growth going forward. Thanks.
Cristin Hubbard: Yeah. Thank you for the thank you very much for the question. So onto the first question around the 10% that have switched, I mean primarily what we are hearing is injection fatigue or wanting to try a weekly therapy. But what I think is more important is looking at the 90% who we retain. And what we are finding there is that really it is about not only the surround sound services we have around these patients and their families, namely with our clinical coordinators, how we are in there talking to the family and really building out that trusted relationship that is so important in this community. But also importantly reminding them of the evidence base. That we have, the safety, the efficacy and something that quite frankly no competitor can catch up to. That is something that we find is continued to be very compelling and I expect that to be true in the future. Now with regard to the incidence of the 0-to-2 population we estimate that there is about 150 births in The US a year with infants with achondroplasia and so our intention is to very much target treatment as early as possible. As you know, the consensus guidelines certainly state that this is the most efficacious and beneficial for them to be treated early. But also what we are finding is that by targeting new specialties such as maternal fetal medicine and really getting out there early to help them understand possibly when in utero or even right at birth the attributes of treating early that is what we are out there doing and we find that to be quite a success. Thanks, Cristin.
Brian R. Mueller: Hi, Eliana. it is Brian. I will take your guidance question. Absolutely. We appreciate the interest in switch assumptions, especially given the competitor update today and our competition metric as well. As noted, in February, when we initially gave guidance, we do expect switching and we shared what we are observing today. Our guidance did include a switch assumption. But not going to quantify that at this time nor comment on expectations at this time. Thank you.
Operator: Next, we will move to Mohit Bansal at Wells Fargo.
Mohit Bansal: Great. Thank you very much for taking my questions. So Christian, regarding the 10% patients who have switched, based on market research your market research, where do you expect this to settle in The US market? And the related question is, how different or similar is ex US market in terms of in terms of your how entrenched you are versus how challenging it could be for the competitor to come in and take share from you. Thank you.
Cristin Hubbard: And, Mohit, if I could maybe could you repeat that second question just so I make sure I answered? I did not quite understand the question, the second So the question is, like, how the ex-U.S. market is similar or different versus The US market? In terms of setup and structure where it could be challenging or easy, for a competitor to take share versus The US market. So just trying to understand the structure of The US versus Ex US market for achondroplasia patients there.
Mohit Bansal: Thank you.
Cristin Hubbard: Very good. Okay. Thank you very much for the question. Now, of course, so the first question as to when do we expect what do we expect going forward? As Brian shared, we are not necessarily gonna share our expectations because the truth of the matter is we need to continue to monitor this and closely watch if this levels out, if this is a bolus or if this is steady state, this is something that we need to very much monitor at this stage in time. I do think that what you would find most likely is that the segment that is most apt to switch first and foremost, as I said, are those that either have injection fatigue, are looking for the convenience of a product that Voxzogo has a very similar efficacy profile to it. However, they might want a weekly shot. So those are kind of what we are seeing out there, but I would not be able to comment at this juncture in terms of how this is going to go in the future. Now looking at it relative to the ex-US I would say the biggest difference and I know we talked about this before in The US as you have a much more segmented market, much more geographically dispersed You have more specialties involved. And we see that certainly as I guess, a component in the competitive dynamics here. What we expect ex-U.S., we have not seen there is not been any approvals or any product in other countries at this point in time. But what we expect ex US might, you know, I am not going to speak to it necessarily but do not think that the dynamics are going to be wholly different. Ex US but that is something again that we will have to remain vigilant on and continue to see.
Operator: Moving next, we will go to Akash Tewari at Jefferies.
Akash Tewari: This is Phoebe on for Akash. Another 1 on Voxelgo. Can you talk about what market work you have done so far for hypochondriplasia and whether you expect any bolus at initial approval, and if you have already identified a certain number of hypochondroplasia patient patients. Thank you.
Cristin Hubbard: Yeah. Thank you very much for the question. I suppose Gregory and I might want to take this 1 on together. I think in terms of the hypochondroplasia market, what we have said very clearly is that we expect a global total patient population of around 14 thousand. But the work now is really getting in there in the countries and identifying those patients as early as possible so that by the time a assuming we are able to get a regulatory approval by the time we get there we can launch immediately and cover as many of those patients who are amenable to treatment as possible. And so we have already talked a little bit about some of the global initiatives we have been working on to include improving diagnosis, We talked about some of the targeted genetic reclassification work we are doing. As well as a lot of the physician and caregiver awareness that we are doing. Most recently, we have launched tactics that are really around having multiple kind of digital and media campaigns really primarily targeted in The US. And that is about shaping the marketplace. And what we want to do is make sure that we are including HCP directed disease education content as well as caregiver and patient awareness programming that really again is about making sure that we are getting as many patients diagnosed as possible and then importantly shortening that path from the time that they are diagnosed to the time that they are willing to treat. Over to you, Gregory.
Gregory Friberg: Yeah, thanks. And just to go back well, we only turned the card over 2 months ago. We are really pleased by the data that we saw in phase 3. Again, the AGD exceeded our expectations. We hit statistical significance on the height variables as well as arm span And we are looking forward to presenting the subsets and additional safety data and so forth at SP in September. I would say with regard to patient finding, you can, be rest assured that we are working hard to bring what we think could be a potentially safe and effective therapy to hypochondroplasia patients We are doing testing work. Again, we have metrics looking at not only testing rates but testing yield. We are certainly preparing to know what we think, again, the age of diagnosis is and so forth, and that is work that is ongoing right now. We see that age going downwards, which again is a good sign that the classic challenge here that these patients are not making their way to the right specialist is something that we have intervened with. At another time, I would be happy to talk about other, implementation science work we are doing. Again, to try to prepare the field in, you know, in a pre approval appropriate way to again make sure that the science is following and that we will be able to, again, reach the most number of patients as possible.
Operator: We will go next to On BD, Paul Matteis at Stifel.
Paul Matteis: what is next in terms of the scope of the types of things that BioMarin's looking at? And, Alexander, when you take a step back now and look at the revenue base the profitability profile you have, what is the optimal number of I guess, like, phase 1, 2, 3 assets in a pipeline of BioMarin size? Thank you.
Alexander Hardy: Thanks very much for the question. So, you know, we are looking obviously at We see ourselves as the leader in the space of genetic conditions. We have strong business units as you heard now. We call it metabolic conditions, skeletal conditions. So, you know, those are areas we are looking to supplement the many products we have in those in those spaces that the 9 products in our portfolio. But we are also interested in genetic conditions. Where it is a good fit with our capability, our expertise in genetics for example, our regulatory expertise, our manufacturing expertise. Our commercialization. So we are not getting specific at this point about, you know, what those additional therapy areas within the umbrella of genetic conditions I think you probably have a sense of sorts of sorts of types of diseases which really leverage that capability. And I think when you look at the Amicus acquisition, it dropped just perfectly into that metabolic conditions business unit you can see it is really the capabilities that we have that we can leverage that allows us to really say that, you know, the peak sales potential of these products is greater than they were before. We think there is a really great opportunity for us to do that. With other programs. But bringing them in the clinical stages. So expect more progress in the next 12 to 18 months. We are looking for, you know, a nice steady flow of products at all stages of development. We were excited, and I think, you know, it is worth just highlighting We are excited to announce this quarter. We actually put more of a focus and really dug in on the DMX 200 asset, which we call them now. DMN 28. And we are really excited to with that asset as we dug in to have a phase 3 asset in the renal space. So this hopefully gives you a sense of kind of what we are thinking about from a BD standpoint in a general perspective. But, you know, we are excited about growth prospects and the opportunity for cash flow generation and the optionality this gives us to further strengthen our pipeline.
Operator: We will take our next question from Sean Laaman at Morgan Stanley.
Sean Laaman: I guess if you look at the $2.6 billion in Pombility and Galafold guidance, for mid-2030. So like how much of that is like, market acceleration versus what BioMarin's adding to the pie? And you know, since you have been able to, you know, get the business under your hood, what have you learned down that front that gives you good confidence you might not have learned before. And then if I can slip 1 in on BMN 33, you know, how would you characterize the rate of enrollment in that study? And when might we see the next signpost? Thank you.
Cristin Hubbard: Yeah. So thanks for the question, Sean, around kind of how much of the of the contribution there is related to perhaps what we can do differently. I would say that when you look at the contribution in the build and we really did do a bottoms up looking kind of country by country about what we could do. So we were not playing around with the prevalence numbers or changing anything about the disease characteristics per se. This really was about when we put this onto the BioMarin platform, what could we do differently and how does that look country by country? So I would say the biggest kind of contributor as you would expect on the Galafold side was opening up the diagnosis. Certainly treatment rate plays a role there, but really the biggest contributor there was around opening up the diagnosis rates. On the Prombility and Upholda, that really was the biggest contributor definitely how you know, around switches and how quickly we could get switch rate to move on that. And so the question becomes, how are we able to do this And so as I mentioned we looked at this country by country and really do feel confident about how we can click these into either existing countries where they are already opened up in those markets or importantly have already set in motion what is going to be the regulatory as well as the reimbursement pathway moving into those specific countries. So I would say that is the biggest thing. And again, this was not about changing prevalence numbers. Or tweaking with the funnel. And that way, this really was about building on our own capabilities. Yeah.
Gregory Friberg: And thanks, Sean, for the interest in 3. We, of course, are incredibly excited. We have, active enrollment going on in multiple time zones, multiple countries around the world. Again, we are looking for naive patients. We are entering the steep part of the enrollment curve. I do not expect that we will give an update until we are completed enrollment in those 40 patients for the phase 2 portion, but we wanna just reiterate that, again, our expectation is in 2027, we are gonna answer this question. I know that there is been debate out there of whether or not, again, the free CNP will translate into more AGV. We have a strong conviction that it is an absolutely valid hypothesis. Happy to drill into that with others. In more detail. Now is the time building on the phase 1 data that we saw when we know we can increase exposure of free CMP, we know that pulls into pharmacodynamics in the plasma, cyclic GMP. Now is the question to look at growth. And that will be a question that we answer in the next calendar year. Wonderful.
Operator: We will go next to Alexandria Janet Hammond at Wolfe Research.
Alexandria Janet Hammond: Just 2 from us. So first, on the guidance bump does that have less to do about switching or more about growth? And is that growth more US or o US? And then on the BMN-333 as well, how does the Aspen studies operational seamless design give you levers to pull the timeline forward from the phase 3 perspective. Thank you.
Brian R. Mueller: Hey, Alexandria. Thanks. This is Brian. Appreciate the question on the Voxzogo guidance raise. First and foremost, pleased with the strong performance in Q2 and our confidence in the outlook for the second half of the year to be able to raise the guidance and get back to the blockbuster status at the bottom end of the guidance. I will note that remember previously 1 of the variables that I pointed out at the beginning of the year when we guided was a couple of, international price negotiations that were in process. I will share that 1 of those closed successfully. With a good outcome. And the other had some initial setbacks, but we are continuing with the process. But there was some upside there to some of the contingency that was in the range. So that was a bit behind it. Then the rest of it was growth and performance I will just say that it was both US and global, adding new patients, growing revenue, confidence in 2026. Thanks.
Gregory Friberg: Yeah. And thank you for the question again on 3. The operationally seamless phase 3 design really gets most of it or provides most of its benefit through recruitment acceleration and site startup. Not every country can start at the same time. They have different requirements with regard to regulatory approvals and so forth. This allows us to, under the, the umbrella of 1 protocol, work with the same IRBs, work with the same sites, have a parking lot of patients identified, and really I think the most impressive, benefits will come with the phase 3 recruitment. The phase 2, again, is up and going, and we are off to the races.
Operator: And this concludes our Q and A session. I will now turn the conference back over to BioMarin's CEO, Alexander Hardy, for closing remarks.
Alexander Hardy: Thank you, operator, and thank you all for joining us today. The standout quarter across the business, 20% top line growth, rapid close and integration of Amicus, advancing pivotal data with Voxzogo's second indication, hypochondroplasia. Strong demand for our innovative products led us to increase guidance today including full year total revenues Voxzogo, now at the low end of $1 billion non GAAP earnings per share. As we enter the second half of 26, BioMarin is stronger, more diversified, better positioned than ever to lead in rare disease. Deliver for patients worldwide. Thank you for your continued support, Look forward to speaking to you soon.
Operator: And this concludes today's conference call. Thank you for your participation. You may now disconnect.
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