Amicus Therapeutics Inc (FOLD) 2025 Q3 法說會逐字稿

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Third Quarter 2025 Financial Results Conference Call and webcast. (Operator Instructions) As a reminder, this conference call is being recorded.

I would now like to turn the conference over to your host, Mr. Andrew Faughnan, Vice President of Investor Relations. You may begin.

Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics' Third Quarter 2025 Financial Results and Corporate Highlights. Leading today's call, we have Bradley Campbell, President and Chief Executive Officer; Sebastien Martel, Chief Business Officer; Dr. Jeff Castelli, Chief Development Officer; and Simon Harford, Chief Financial Officer. Joining for Q&A, we have Ellen Rosenberg, Chief Legal Officer.

As referenced on slide 2 of the presentation, I would like to remind you that we will be making forward-looking statements on today's call. I encourage you to read the disclaimers in our slide presentation, the press release we issued this morning and the disclosures in our SEC filings, which are all available on the IR portion of our corporate website. Forward-looking statements are subject to substantial risks and uncertainties, speak only as of the call's original date, and we undertake no obligation to update or revise any of the statements. Additionally, you are cautioned not to place undue reliance on any forward-looking statements.

At this time, it's my pleasure to turn the call over to Bradley Campbell, President and Chief Executive Officer. Bradley?

Great. Thank you, Andrew, and welcome, everyone, to our third quarter conference call. I'm very pleased to report another great quarter for Amicus, highlighted by strong revenue growth, GAAP profitability and continued confidence in our positive outlook going forward. Let me go through a few highlights before I turn it over to the team to go through in more detail. First, we delivered another quarter of double-digit revenue growth in our Fabry and Pompe core business, a trend we expect to sustain into the years ahead.

Second, we remain firmly confident in our growth trajectory as we approach year-end. Galafold delivered 13% year-over-year patient growth this quarter, driven by record demand and robust new patient starts. For Pombiliti and Opfolda, Q3 represented another strong quarter marked by significant momentum in both our established and newly launched markets, underpinned by growing commercial demand and increasing new patient starts. In fact, this was the strongest quarter ever for new commercial demand for both Galafold and Pombiliti and Opfolda. Both products are on track to meet current consensus sales estimates for the full year.

Third, we continue to emphasize the growing body of evidence and differentiation of Pombiliti and Opfolda through scientific publications and congress presentations. In fact, recently at ICIEM in September, we shared new four-year data from the PROPEL ongoing extension study, demonstrating stability or improvement in key endpoints of muscle function and strength in ERT-experienced patients, which Jeff will review in more detail later in the call, along with some exciting new real-world evidence supporting Pombiliti and Opfolda.

Fourth, we reaffirm our confidence that our two commercial products, each with blockbuster potential, are on track to deliver combined sales of $1 billion in 2028. Galafold's strong growth trajectory supported by improving diagnostic rates and patient access, coupled with meaningful contribution we expect from Pombiliti and Opfolda to our long-term performance reinforces our confidence in this milestone.

Fifth, alongside our partners at Dimerix, we continue to advance the development of DMX-200, a first-in-class therapy in late-stage Phase 3 development for FSGS, a rare life-threatening kidney disease. The ACTION3 pivotal study is over 90% enrolled and remains on track to complete enrollment by the end of the year. And our work with DMX-200 and the potential to address the considerable unmet need in FSGS represents an important and growing part of the Amicus story.

And finally, as we continue to maintain our financial discipline, we are pleased to deliver GAAP profitability and a growing cash position in the third quarter and remain very confident in achieving GAAP net income for the second half of the year. Altogether, we are proud of our achievements this quarter and believe Amicus is well positioned to continue to create significant shareholder value while fulfilling our mission for patients in the years ahead.

With that, let me now hand the call over to Sebastien to review the commercial business in more detail. Sebastien?

Thank you, Bradley, and good morning to everyone. So let's start with Galafold on slide 5. You see that revenue reached $138.3 million, up 12% at constant exchange rates and up 15% in reported terms. The underlying growth of this product remains very positive and is driven by the number of new patient starts globally. Year-over-year, the underlying growth in patient demand increased by 13%.

We ended the quarter with approximately 69% of the global market share of treated Fabry patients with amenable mutations. Galafold is clearly positioned as the treatment of choice for amenable patients among prescribers, and there are still many more patients eligible for our therapy.

Turning to slide 6. Our leading market continue to be the biggest driver of the strong patient demand for Galafold. We saw record high demand in Q3, as Bradley just mentioned. And on a year-to-date basis, new patient starts have reached their highest level since launch. The global mix of patients on Galafold today is about 65% naive and 35% switch.

This compares with 60% and 40%, respectively, in 2024. So clearly, we're seeing stronger uptake in naive populations. And while we continue to achieve high market shares in countries where we've been approved the longest, there's still plenty of opportunity to switch patients over to Galafold and to keep growing the market as we penetrate the diagnosed, untreated and newly diagnosed segments. Given the sustained growth in patient demand and our projection of a record level of new patient starts this year, we remain highly confident in our full year 2025 growth guidance for Galafold, trending in line with current full year consensus estimates.

Key drivers behind the robust demand for Galafold, which we expect to continue well beyond 2025 are: first, finding new patients and penetrating into the diagnosed and treated population, including shortening the pathway to diagnosis; second, expanding Galafold into new markets and extending the label; third, driving Galafold's share of treated amenable patients. We're actually seeing that in most mature markets, we can reach 85%, 90% share, so we know that there's the potential to reach those levels globally. And fourth, sustaining compliance and adherence rates above 90% so that patients who go on Galafold predominantly stay on Galafold.

On slide 5, you can see the significant unmet need in Fabry disease today. So over 12,000 people receive Fabry treatment worldwide, while about 6,000 diagnosed patients remain untreated. The literature suggests actual prevalence may exceed 100,000 patients, indicating meaningfully larger undiagnosed populations and substantial market opportunity for Galafold. We're highly confident that a small molecule is a compelling treatment option for the untreated and undiagnosed populations as indicated by the increase in naive new patient starts. Late onset Fabry makes up a growing percentage of the newly diagnosed and treated population, which is enriched for amenability to Galafold.

On slide 8, we just provide a great example of our ongoing efforts to enhance Fabry disease awareness and support improved diagnosis. The diagnosis of Fabry disease is unfortunately often delayed for up to a decade or more due to the rarity of the disease, high variability of presentation and symptoms that are quite often nonspecific and resemble those of other diseases. So the new Finding Fabry campaign in the US aims to help health care professionals recognize these diverse symptoms and prevent misdiagnosis. So to wrap up on our Fabry section, with excellent momentum, the sizable untreated population and our strong IP protection, Galafold has a long runway well into the next decade and a clear path to surpassing $1 billion in revenue.

Turning on now to Pompe disease on slide 10. We outline our global launch progress with Pombiliti and Opfolda. Third quarter revenue reached $30.7 million, up 42% at constant exchange rates and up 45% in reporting terms. Year-to-date, Pombiliti and Opfolda has grown 59% at CER and 61% in reported revenue.

The majority of sales came from our initial five launch countries, the US, UK, Germany, Spain, Austria, although as I'll highlight in a moment, we've actually secured reimbursement in additional 10 countries thus far in 2025 with four new markets since the end of Q2, namely Japan, Belgium, Ireland and Luxembourg. For the quarter, the US represented approximately 43% of revenue, while ex-US represented 57% of sales. Q3 showed strong sales growth and a high level of patient demand. We continue to see patients switching proportionately based on market share as well as a broadening and deepening of prescriptions with more sites coming online and multiple new prescriptions from physicians.

We're also seeing a growing number of patients exploring alternative treatment options and actually advocating for their own decisions to switch, especially in the US. In 2025, we have observed many positive lead indicators that support the growing launch momentum. Globally, the number of avalglucosidase alfa patients that have switched to PomOp have actually doubled in the first nine months of 2025 as compared to the entirety of 2024. Similarly, naive prescriptions ex-US have also doubled in the first nine months as compared to the total of 2024.

Given these indicators, we're reiterating our full year 2025 revenue growth guidance for Pombiliti and Opfolda of 50% to 65% at constant exchange rates, trending in line with the current full year consensus sales estimates. We expect Pombiliti and Opfolda to be a major contributor to multiyear growth for Amicus based on key growth drivers, namely: first, continuing to increase the number of net new patients; second, increasing the depth and breadth of prescribers; third, launching in up to 10 new countries in 2025; fourth, differentiating our therapy through evidence generation and real-world evidence; and five, maintaining over 90% compliance and adherence rates.

Moving to slide 11, looking at the geographic expansion of Pombiliti and Opfolda. As I mentioned, today, we're now reimbursed in 15 countries and continue to observe strong execution in the newer launch markets. Notably, five of the countries launched during the second quarter generated revenue in Q3, Switzerland, Italy, the Czech Republic, Portugal and the Netherlands. As mentioned, we recently reached pricing and reimbursement agreements in Japan, Belgium, Ireland and Luxembourg. We also continue our work to secure broad access to patients through the EU.

A little more color on Japan as it's a bit of a unique market. At the end of Q3, we saw the first commercial patients in Japan. Majority of patients in Japan have actually been on Nexviazyme for two to three years, and that represents a strong market opportunity for us. I hope that commercial overview provides a strong sense of the continued execution and growth in Galafold and the building momentum in the launch of Pombiliti and Opfolda.

With that, I'll now hand the call over to Jeff to highlight the work we do to further differentiate Pombiliti and Opfolda. Jeff?

Thank you, Sebastien, and good morning, everyone. Moving on to slide 12. We highlight a few examples of our rapidly expanding and diverse body of evidence supporting the differentiation of Pombiliti and Opfolda in Pompe disease across clinical trials, mechanistic studies, real-world data and case studies. In September, we presented new four-year data from the PROPEL open-label extension. This new analysis presented at the International Congress of Inborn Errors of Metabolism showed patients within the ERT experience group demonstrated durable improvements or stability on measures of muscle function, muscle strength and biomarkers out to four years.

On slide 13 and also presented at ICIEM, we'd like to highlight a presentation by an independent group in the UK that followed 28 patients switched from Myozyme to PomOp, which was an n of 13 or Myozyme to Nexviazyme, which was an n of 15. And that analysis reported improvements in motor function for the patients that switched from Myozyme to Pombiliti Opfolda. We expect this body of evidence to continue expanding over time, strengthening the case for Pombiliti and Opfolda as a compelling treatment option in Pompe disease.

Now moving to slide 15 and DMX-200. As previously announced, we took a major step forward in our strategy to strengthen our portfolio through a successful US licensing agreement with Dimerix to commercialize DMX-200, a first-in-class treatment in late-stage development for FSGS, a rare and potentially fatal kidney disease. With blockbuster market potential, we remain highly encouraged by the data seen to date and believe this asset brings immediate strategic value to Amicus today and will create value for patients and shareholders moving forward.

Moving on to slide 16. We are impressed by the strong momentum Dimerix has built and the growing body of evidence supporting this transformative potential of DMX-200. The pivotal Phase 3 ACTION trial -- sorry, ACTION3 trial is progressing well with more than 90% of patients now enrolled and remains on track for full enrollment by year-end. This study is robustly designed and strongly powered with several successful interim analyses already completed. Importantly, there is FDA alignment on proteinuria as the primary endpoint for approval.

In October, Dimerix provided an update on PARASOL data analysis that showed a consistent result in line with the prior analyses, supporting, again, proteinuria as an endpoint for standard approval. We anticipate requesting an additional meeting with the FDA in the first quarter to further discuss the next interim analysis from the ACTION3 study and the next steps for DMX-200 development.

With that, let me now hand the call over to Simon to review our financial results and outlook. Simon?

Thank you, Jeff. Our financial summary begins on slide 18 with our income statement for the third quarter ending September 30, 2025. For Q3, we achieved total revenue of $169.1 million, which is a 19% increase over the same period in 2024. At constant exchange rates, revenue grew 17%. The global geographic breakdown of total revenue in the quarter consisted of $98.8 million or 58% of revenue generated outside the United States and the remaining $70.3 million or 42% coming from within the US.

Cost of goods sold as a percentage of net sales was 12% for Q3 compared to 9% in the same period last year. The total GAAP operating expenses increased to $115.3 million for the third quarter of 2025 as compared to $106.6 million in the third quarter of 2024, an increase of 8%. On a non-GAAP basis, total operating expenses increased to $95.4 million for the third quarter of 2025 as compared to $82.6 million in the third quarter of 2024, an increase of 15%. We define non-GAAP operating expense as research and development and SG&A expenses, excluding stock-based compensation expense, loss on impairment of assets, changes in fair value of contingent consideration, restructuring charges and finally, depreciation and amortization.

On a GAAP basis, net income in the third quarter 2025 was $17.3 million or $0.06 per share compared to a net loss of $6.7 million or $0.02 per share for the third quarter of 2024. This was the first quarter of 2025 that Amicus delivered positive GAAP net income, consistent with our guidance to have positive GAAP net income during the second half of 2025. While we are pleased with the positive Q3 GAAP net income results, let me remind you that in the early stages of turning profitable, GAAP profitability may not be linear quarter-to-quarter. We do, however, anticipate having positive GAAP net income for the second half of 2025.

In Q3 2025, non-GAAP net income was $54.2 million or $0.18 per share compared to non-GAAP net income of $30.8 million or $0.10 per share in the third quarter of last year. Cash, cash equivalents and marketable securities were $263.8 million as of September 30, 2025, compared to $249.9 million as of December 31, 2024. This is a $32.8 million increase during the third quarter versus the prior quarter. So we are importantly generating cash also this quarter.

On slide 19, we are reiterating our full year financial guidance for 2025 as follows: total revenue growth of 15% to 22%. Galafold revenue growth of 10% to 15%. Pombiliti and Opfolda revenue growth of 50% to 65%, all growth rates are at constant exchange rates. Based on our performance for the nine months and our clearer line of sight for the fourth quarter, including the anticipated FX impact, we are confident that total and product revenues for the full year are trending in line with full year consensus numbers, which you can find via the Investors portion of our website.

Gross margin is expected to be in the mid-80s, which we define as 83% to 87% approximately and will likely be at the top end of that range. As a reminder, 2025 is a hybrid year for Pombiliti Opfolda COGS as we have worked through previously expensed inventory during the first three quarters of 2025. As a result, Pombiliti Opfolda COGS will be expensed through Q4 '25. Non-GAAP operating expense guidance remains $380 million to $400 million. However, we anticipate being at the high end of the guidance range. And finally, we anticipate positive GAAP net income for the second half of 2025.

And with that, let me turn the call back over to Bradley for our closing remarks.

Great. Thank you, Simon, Jeff, Sebastien. As we come to the end of our presentation, let me just remind you of our strategic priorities for the year. And in closing, I just want to reiterate how encouraged we are by the growing impact of our therapies plus the very promising Phase 3 asset that we've added to our pipeline for FSGS. Our expertise in rare diseases and proven track record of commercial execution support our ongoing commitment to our mission and sustaining long-term growth in 2025 and beyond. I'm confident we can continue to develop and deliver transformative treatments and create enduring value for patients and shareholders alike.

With that, operator, we can now open the call to questions.

(Operator Instructions) Joe Schwartz, Leerink Partners.

Congrats on the strong performance. Now that we're into the second full year of the Pom-Op launch, I was wondering if you could talk a little bit about the overall reception to both the label for Pom-Op, especially in the US and the real-world evidence that you seem to be layering in now and how that's driving prescription patterns and whether the conversations vary across treatment centers? Do any certain types of physicians or patients seem to appreciate one set of data more than others? What are you finding is encouraging the most patients to switch to Pom-Op nowadays?

Joe, thanks a lot for the question. I like the multipart question there, but we'll do our best to get to all of those pieces. I heard some labeling questions as well as real-world evidence and how that's influencing or impacting the prescriber base. And then finally, is there a particular data set that is more compelling or not. So from a label perspective, I think it's largely been well received, although clearly, in the United States, we hope to continue to look for ways to expand that label, in particular, down to pediatric patients with late-onset Pompe disease and infantile onset patients as well.

As Jeff highlighted, we have ongoing studies there. So -- and those should support label expansion globally. So I think we continue to look for ways to expand that population, and we should start seeing the benefit of that sometime next year.

In terms of real-world evidence, for sure, that is a growing and important part of the conversation with physicians. I can tell you, I've had the privilege of attending a number of launch meetings around the world. And as that body of evidence grows, Jeff highlighted one, we've talked about some case studies previously, we will continue to support those publications. That will only be more and more supportive, we think, of the use of Pombiliti and Opfolda. So please look out for continued highlights there in the medical congresses throughout this year and into next year.

And then maybe, Jeff, from your perspective, just talk a little bit about some of the different types of data that we think are so important for physicians and maybe also about quickly the importance of finding the right endpoints for patients as well as physicians.

Yes. Thanks, Brad, and thanks, Joe, for the question. From our -- like the four-year data we just reported at ICIEM, I think the long-term data is of particular interest really across the populations. That's always been one of the challenges with early Pompe treatments is sort of a lack of the durability. And we've been quite pleased and physicians and stakeholders have been quite pleased with the durability we've seen across trials.

In terms of the real-world data, the switch experience was something in our trials that was very strong, and we continue to see that in various real-world settings. What is lacking, of course, is indirect comparisons of PomOp and Nexviazyme, and we were very pleased to see one of the most robust indirect comparisons come out at ICIEM, which we highlighted in the slides from a large group in the UK that switched from either Myozyme to PomOp or Myozyme to Nex.

And lastly, I would just say, individual case studies are always very important. You can always learn a ton even from individual patients who might have a unique background. We've seen some really interesting case studies of people on 4 times the dose of Myozyme and not doing well and switching to PomOp and having quite good experiences. And of course, pediatrics is important. We've had some really compelling pediatric case studies.

We continue to really invest in our pediatric trials and look forward to actually expanding the labeling here in the US, hopefully mid next year for adolescent patients. And in terms of endpoints, of course, it's always critical what do you look in trials versus in following patients in the real world. And usually, you don't do quite the same comparisons, and we're really helping work with key stakeholders about what is the right sort of cadence of monitoring patients, what are the key endpoints to look at, especially as physicians try to make decisions on switching.

Dennis Ding, Jefferies.

We have two on Pompe, if we may. Number one, congrats on the progress, but talk about the US new patient starts in Q3 as they continue to go up relative to some of the commentary you made earlier this year for April and May?

And then number two, as we think about 2026, consensus seems to expect a big inflection in Pompe revenue. Can you talk about things that you can actively do to accelerate that revenue trajectory relative to 2025? And expanding your sales force is something you're considering, particularly in the US?

Thanks, Dennis, for the questions. I think you were talking about kind of further color on progress in the US and our other markets in Q3 and then also kind of looking forward to the future, how can we continue to drive that building momentum. So yes, Q3, as I said on the call, was the largest ever net commercial demand for Pombiliti and Opfolda, which is really exciting. And that -- the US was a huge contributor to that growth. We are seeing significant increases in breadth and depth of prescriptions in the United States. We've actually improved our time to reimbursement and everyday helps. So that -- I think that helps with the margins as well. And we're seeing very strong adherence and compliance rates as well. So really excited about the US, but that's not just here in the States, as Sebastien highlighted on the call, all of our launch markets, we think, are progressing really well.

As we head into next year, of course, it's a little bit too soon to give guidance, but I think the momentum we're building in Q4 gives us confidence to see continued momentum going into 2026. What do we do to continue to support that momentum growth? Part of it for sure is just experience, and I think Jeff's point around that real-world evidence. I've literally been in meetings where some of those data points are presented for the first time and the excitement as people are seeing new data and new populations I think, is a key part of seeing that translate then into wanting to use the product in a new or different way. So I think that's part of it for sure.

As Sebastien mentioned, we have a whole host of new countries contributing to the demand. This quarter, those new markets like Japan, as an example, will be modest in contribution from a revenue perspective. But of course, going into next year, the countries that we're launching in Q4 will have a significant contribution. Another one that we didn't highlight on the call, but is definitely going on in the background is the experience in the Netherlands. We talked before about the expectation there that we'll see 70% of patients switch over to late -- or excuse me, switch over to PomOp. That will continue to be a significant contributor as well. We've made great progress over Q3 and into Q4 in switching those patients.

So I hope that gives you some flavor for the places that we're focused. But again, I think as more than anything, as more and more people have experience, as more and more real-world evidence comes out, that fuels the momentum that we're seeing.

Anupam Rama, J.P. Morgan.

Single question, single part from me. For Galafold, in particular, you talked about the strongest patient adds since launch on a year-to-date basis. Can you expand a little bit on if that's coming from core countries? Or is that being more driven by emerging countries?

Yes. Thanks, Anupam. Sebastien, do you want to take that one?

Yes. So Anupam, as I mentioned, the key countries continue to grow, and this is, for the most part, driven by naive patients being diagnosed and us having over time established Galafold in those markets now as the standard of care for newly diagnosed naive patients with amenable mutations. We continue to see some degree of switches in those markets where we've launched in the more recent past, if you want. But as I highlighted, there is a significant underlying growth potential simply because we continue to see that Fabry is unfortunately really underdiagnosed. And so we've talked in the past about the fact that today, the number of diagnosed patients has far exceeded what we had projected right before we launched. And we continue to see, again, demand that is stronger than what we anticipated right before launch.

Maxwell Skor, Morgan Stanley.

Just one on DMX-200. Is there a defined threshold for MCP-1 levels or other inflammatory markers that would make patients particularly good candidates for DMX-200? And any thoughts on FILSPARI, the FDA no longer requiring an advisory committee meeting for FILSPARI.

Maybe I'll take the second one, and then, Jeff, you can talk about the MCP-1 levels, which we do believe are correlated with the potential for that product. So it's a great question. As it relates to the AdCom for FILSPARI, what I'll just say is we are eagerly anticipating the progress there. We're hopeful for them, and we think it's a good sign, in fact, that they are no longer requiring an AdCom, and we're wishing them well. As it relates to the relationship of MCP-1 and proteinuria, Jeff, maybe you can respond there.

Yes. Thanks for the question. So briefly, MCP-1 is the monocyte chemoattractant protein. That is the chemokine that binds the CCL2 receptor and leads to the monocyte-driven inflammation. So DMX-200 is interrupting that signaling.

In Phase 2, we did see a nice effect on MCP-1 levels from DMX-200. And we actually saw the patients with the highest MCP-1 and the highest proteinuria showed the most robust responses on proteinuria in that Phase 2.

For the Phase 3 study, we did not have any sort of entry criteria in MCP-1. We are measuring MCP-1 throughout the study, and we do intend to sort of analyze the results based on different MCP-1 levels. We do anticipate, again, like we saw in the Phase 2 that people with higher MCP-1 are likely to have more inflammation and therefore, are likely to show even more of an impact of DMX-200. So we certainly will continue to learn more, and that is one of the key biomarker endpoints in the Phase 3.

Ritu Baral, TD Cowen.

This is Joshua Fleishman on the line for Ritu. Congrats on the quarter. So what impact may BIOSECURE 2.0 have on the usability of Ireland plant PomOp product in the US? And how has physician feedback changed over the last quarter on the competitive dynamic in Pompe? Are the goalposts changing from when docs feel comfortable to switch patients to PomOp?

Thanks, Joshua, for the questions. We had one on BIOSECURE 2.0 and one on the continued sentiment around experience with Pombiliti Opfolda. So on the first one with BIOSECURE, look, we've continued to believe that we do think highlighting the importance of US biotech manufacturing is critical for our economy going forward. However, the move to Dundalk and you might have seen on the call, the approval now from Europe of that facility, and we're eagerly anticipating the approval of the United States as well, I think, is a great way to ensure that we have supply coming from a friend-shored location in Ireland.

We are confident that we will be able to maintain security of supply and at least the way that BIOSECURE has been evolving from last year into this year, I think, gives us even more confidence that we'll have a stable supply coming out of Ireland for the long time to come.

And so we're not concerned about the evolution of that legislation. As it relates to the experience, I think everybody, I think, is eagerly anticipating or eagerly asking those questions and is exactly the right question. You may remember that when we launched, we said that part of our goal is to continue to provide experience and evidence to the physician and patient population so they can understand the opportunity with Pombiliti Opfolda. And as that evidence mounts, we are convinced that we will demonstrate that Pombiliti Opfolda is the right therapy for patients. And so I think I would encourage folks to continue to look at the posters and presentations as well as the publications that have come out.

I think that evidence will only grow. And as Jeff said, part of it also is with now the availability of multiple therapies, what should physicians use in a real-world setting to understand that better. And I think we're helping the community answer that question as well. Yes, it's going to be mobility and breathing, but I think there'll be a number of other subtle -- more subtle endpoints or more patient-driven endpoints that will drive that as well. And so we'll continue to highlight those as we're able to support the community in developing them.

Kristen Kluska, Cantor Fitzgerald.

This is Rick Miller on for Kristen. Just one for us on Pompe. How should we be thinking about when you could potentially receive infantile onset Pompe disease label expansion? And will this be solely contingent on the ROSELLA, trial?

Great question. Maybe I'll frame, but then, Jeff, you can answer the specifics. So clearly, the biggest unmet need, the most fragile population within Pompe disease is the infantile onset Pompe patients, and it's critical for us to be able to serve those patients. Jeff can talk about the timing there.

I would say, though, the largest portion of remaining patients who don't have access to PomOp today are the pediatric late-onset Pompe patients, so 12 to 17 and then 1 to 12. And so those are also a priority for us, and we've made great progress there. And in fact, those would probably become earlier label expansion opportunities than infantile onset. So maybe, Jeff, just remind us the rough timing on the pediatric late onset Pompe and then to the specific question from Rick, the infantile onset timing.

Yes. Thanks for the questions. So in terms of the first cohort of the 12- to 17-year-old late-onset patients as adolescent patients, we anticipate having a submission shortly and would look to an expansion of the label sort of mid next year. That will be the first pediatric expansion. We're completing enrollment in that younger LOPD group below the age of 12. So with enrollment completing here probably by the end of the year, you're looking at a year or so of follow-up and then time for submission. So a couple of years probably until that group gets added to the label.

And then similarly for IOPD, we have two cohorts of patients, those that are switch patients and those that are naive patients. We're making great progress on the switch patient enrollment. That's nearly completed, starting to make good progress in the naives and again, that will be a year-plus study and the time for submission. So that would be probably even coming shortly after that younger LOPD group. But as Brad said, with the newborn screening in the US and how much more patients are followed earlier, in particular, LOPD, we're very excited to hopefully have an updated labeling into that adolescent group mid next year.

Salveen Richter, Goldman Sachs.

As we look to 2026 here with the switches from Nexviazyme expected in the US and more ex-US countries coming along, how should we think about the commercial trajectory of PomOp versus what we've seen this year? And I guess as you think forward to kind of a steady state, how are you thinking about market share for this asset?

Yes. Thanks, Salveen. As I said earlier, we're very confident in the growth as we come to the end of the year here, expect continued momentum into next year. A little early to give specifics on guidance, but we are confident it will be a strong growth year next year as well. In terms of market share, look, -- what we've seen in countries like the UK as an example, where we were available through that EAMS program for a number of years prior to launch, we're now getting to market shares in the 40%-plus after, call it, three or four years on the market, probably four years in the market there. Our strong belief is that we can establish this product as the leading product for treating Pompe patients. And so for me, that means over a 50% market share at peak.

And I think if you look at where the market is headed from a growth perspective, that's how we get to $1 billion plus at peak potential. So we're very confident in the end, this will be the leading prescribed product for people living with Pompe disease. We're still on that journey, of course, but we're starting to see signs that we can get to those shares after a period of time. So more to come.

That was our last question. This does conclude today's conference call. We hope you have a great day, and you may now disconnect.

Great. Thank you all.