Amicus Therapeutics, Inc.

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Second Quarter 2025 Financial Results Conference Call and Webcast. At this time, all participants are in a listen-only mode. Later, we will conduct a -and-answer session, and instructions will follow at that time. As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Mr. Andrew Fonan, Vice President of Investor Relations. You may begin.

Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics Second Quarter 2025 Financial Results and Corporate Highlights. Leading today's call, we have Bradley Campbell, President and Chief Executive Officer, Sebastian Martel, Chief Business Officer, Dr. Jeff Costelli, Chief Development Officer, and Simon Harford, Chief Financial Officer. Joining for Q&A is Ellen Rosenberg, Chief Legal Officer. As referenced on slide two 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 is 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 second quarter conference call. I'm very pleased today to highlight our strong quarter and positive outlook for Amicus for the remainder of the year. First, we've delivered yet another quarter of strong double-digit revenue growth on our business in Pompeii and Fabri disease. This is our 17th consecutive quarter with double-digit sales growth at CER, and we see that trend continuing for years to come. Second, we remain highly confident in our growth trajectory that you'll see throughout the remainder of the year. Gallifold delivered 13% -over-year patient growth this quarter and is on track to achieve the highest number of global patient starts this year. For Pombiliti and Opfolda, Q2 marked the strongest quarter for commercial demand since our long launch, with significant momentum both in the United States and the five new launch countries in Europe, as well as the existing launch countries there as well. Third, we're steadily building the body of evidence, highlighting the differentiation of Pombiliti and Opfolda and Pompeii disease, including a recent publication in Muscle and Nerve, which demonstrates the benefits for switching ERT-experienced patients to Pombiliti and Opfolda. Fourth, we are reiterating our confidence that these two products will reach a combined sales of $1 billion by the end of 2028. We continue to believe that Gallifold is uniquely positioned to further its reach as diagnostic rates and patient access continues to improve, offering a substantial runway for sustainable growth, and Pombiliti and Opfolda is becoming an increasingly meaningful contributor to our long-term performance. As a reminder, each of these products have a $1 billion plus in peak sales potential. Fifth, we continue to advance our strategic partnership with Dimerix and DMX200, a -in-class compound in late-stage Phase III development for a rare and life-threatening kidney disease. The Action 3 pivotal study remains on track for full enrollment by the end of the year, marking a key milestone in this important program. DMX200 and our opportunity to meet the significant unmet needs that exist for people living with FSGS is an important new part of the Amicus story, and we look forward to telling you more over the course of the year. And finally, as we continue to maintain our financial discipline, we reiterate that we are on track to achieve gap profitability in the second half of this year. Altogether, we're pleased with our accomplishments this quarter and believe Amicus is in a very strong position to generate meaningful value for our shareholders and deliver on our mission for patients in 2025 and in the years ahead. With that, let me now hand the call over to Sebastien to go through some more detail. Sebastien?

Thank you, Bradley, and good morning, everybody. So, let's start with Gatapult on slide five. You see that revenue reached $128.9 million this quarter, 12% at constant exchange rates and up 16% in reported terms. The underlying growth of this product remains very positive and is driven by the number of new patient starts globally. We ended the quarter with more than 69% of the global market share for treated FABRI patients with amedible mutations. Gatapult is clearly positioned as the treatment of choice amongst prescribers, and there's still many more potential patients eligible for our therapy. Turning to slide six, our leading markets continue to be the biggest driver of strong patient demand for Gatapult. The U.S. actually contributed significantly to growth, reaching more than a thousand PRS since launch, a major milestone. When we look at the global mix of patients on Gatapult today, which is about 65% naive and 35% switch, we're now 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 Gatapult and to keep growing the market as we penetrate the diagnosed, untreated, and newly diagnosed segments. With underlying growth in patient demand at 13% this quarter, and our projection of a record level of new patient stocks this year, we remain highly confident in our full year 2025 growth guidance for Gatapult. The key drivers behind the robust demand for Gatapult, which we expect to continue well beyond 2025, are the following. First, finding new patients and penetrating into the diagnosed, untreated population, including shortening the pathway to diagnosis. Second, expanding Gatapult into new markets and extend the label. Third, driving Gatapult's share of treated and amenable patients. We're actually seeing in our most mature market that 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 Gatapult predominantly stay on Gatapult. On slide seven, we highlight the significant unmet need in Fabri disease today. Over 12,000 people receive Fabri treatment worldwide, while 6,000 diagnosed patients remain untreated. Literature suggests actual prevalence makes it over 100,000 individuals, indicating a meaningfully larger under-diagnosed population than originally believed, and substantial market opportunity for Gatapult. We're highly confident that a small molecule is a compelling treatment option for the untreated and undiagnosed populations, as indicated by the record high growth in naive new patient stocks. We're just only scratching the surface with Gatapult today. And as disease awareness and enhanced diagnostic initiatives further shape the Fabri market, we're confident in long-term potential for these medicines, which we think continues to be underappreciated. With excellent momentum, the sizable untreated population, and our strong IP protection, Gatapult has a long runway well into the next decade and a clear path to surpassing $1 billion in revenue. Turning now to Pompidities on slide nine, we outline our global launch progress with Pompidities and Opfolda. The second quarter revenue reached $25.8 million, up 58% at constant exchange rates. The majority of sales came from our initial five launch countries, the US, UK, Germany, Spain, and Austria. Although, as I'll highlight in a moment, we launched into five new markets in Q2 alone. The US represented approximately 42% of revenue, while ex-US represented 58% of revenue. Q2 showed strong sales growth, as well as record levels of patient demand. We continue to see patients switching proportionally based on market shares, as well as the broadening and deepening of prescriptions, with more sites coming online and multiple new prescriptions from divisions. Given these indicators, we are reiterating our full year 2025 revenue growth guidance for Pompidities and Opfolda of 50% to 65% growth at constant exchange rates. Our guidance implies a healthy exit rate heading into the next year, and we'll remain highly confident in the 2025 and long-term outlook for this therapy. We expect Pompidities and Opfolda to be a major contributor to multi-year growth for amicus based on key growth drivers, namely continuing to increase the number of new patients, increasing the depth and breadth of prescribers, launching in new countries including up to 10 this year alone, differentiating our therapy through evidence generation and real-world evidence, and last, maintaining 90% plus compliance and adherence rates. Moving to slide 10, looking at the geographic expansion of Pompidities and Opfolda. In the second quarter, we recorded revenue in 11 countries. So, six countries had their first patient start during the first half of 2025. Italy, Switzerland, Portugal, the Czech Republic, Sweden, and the Netherlands. We're very pleased that Pompidities and Opfolda was selected as a preferred treatment for adults with LOPD in the Netherlands. It's an important toolkit. All patients groups go through one site, and the site is actively transitioning patients. It will be a key driver for us in the second half of this year. We estimate well over 100 patients and intend to take up to 70% of this population. This will then become the largest cohort in any single center worldwide, and definitely a rich source of data on Pompidities and Opfolda. We also recently received regulatory approval in Japan, and are excited to have a label indicated for people ages 15 and older. We're also continuing our work to secure broad patient access throughout the EU. I hope that the commercial overview provides a strong sense of the continued economic execution and growth in Gallifold, and the building momentum in the launch of Pompidities and Opfolda. With that, I will now hand the call over to Jeff to highlight the work we do to further differentiate Pompidities and Opfolda.

Jeff? Thank you, Sebastian, and good morning, everyone. Starting on slide 11, we highlight a few examples of our rapidly expanding and diverse body of evidence supporting the differentiation of Pompidities and Opfolda in Pompe disease. And specifically on slide 12, we summarize two recent case studies recently presented at the ACMG 2025 Annual Meeting, supporting the experiences of individuals switching from Nexvizime to Pompidities and Opfolda. These case studies, along with the growing body of real-world evidence, continue to show that improvement is possible for many patients when switching to Pompidities and Opfolda. We believe our ongoing efforts to grow this body of evidence will ultimately drive wider adoption of Pompidities and Opfolda. Moving to slide 14, as previously announced, we took a major step forward in our strategy to strengthen our portfolio through a very successful U.S. licensing agreement with Dimerics to commercialize DMX 200, a -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 and will create value for patients and for shareholders. Moving to slide 15, we think it's important to highlight the very differentiated and very compelling mechanism of action of DMX 200, which continues to resonate well with physicians in the FSGS research community. There are currently no FDA-approved therapies for FSGS. Standard of care includes nonspecific therapies such as corticosteroids, calcium urine inhibitors, and angiotensin receptor blockers, none of which adequately address the monocyte-driven inflammatory aspect of FSGS. DMX 200 is an oral small molecule taken in combination with ARBs that specifically target this monocyte-driven inflammatory component of FSGS by inhibiting signaling from the angiotensin 1 receptor and chemokine receptor type 2 hetermy that is formed in damaged kidney cells. It delivers a kidney-selective anti-inflammatory effect directly targeting this key unaddressed driver of disease, in particular in patients which are many of them in FSGS with persistent proteinuria and active inflammation. Preclinical and Phase II studies support this mechanism of action and demonstrate impacts on proteinuria with a well-tolerated safety profile to date, with no evidence importantly of the MCP1 rebound effects observed with traditional CCR2 inhibitors. Moving on to slide 16, we are very impressed by the strong momentum Dimerix has built and growing body of evidence supporting the transformative potential of DMX 200. The pivotal Phase III action trial is progressing really well, with more than 75% 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 completed to date. Importantly, there is FDA alignment on proteinuria as the primary endpoint for approval, and taken together with all these facts, we believe DMX 200 is positioned to truly be a meaningful advancement for FSGS patients. Following additional analysis and coordination with the Parasol Consortium over the coming months, we anticipate requesting an additional meeting with the FDA to discuss the next interim assessment of efficacy from the Action III study and next steps for DMX 200. 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 second quarter ending June 30, 2025. For Q2, we achieved total revenue of $154.7 million, which is a 22% increase over the period in 2024. At constant exchange rates, revenue grew 18%. The global geographic breakdown of total revenue in the quarter consisted of $90.4 million or 58% of revenue generated outside the United States and the remaining $64.3 million or 42% coming from the U.S. Cost of goods sold as a percentage of net sales was 10% for Q2 as compared to 9% in the same period last year. Total gap operating expenses increased to $148.9 million for the second quarter of 2025 as compared to $100.4 million in the second quarter of 2024, an increase of 48%. It is important to remember that Q2 operating expenses included the upfront payment of $30 million for the U.S. licensing rights to DMX 200. On a non-gap basis, total operating expenses increased to $127.8 million for the second quarter as compared to $82.1 million in the second quarter of 2024, an increase of 56%. We define non-gap 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 depreciation and amortization. On a gap basis, net loss in the second quarter of 2025 was $24.4 million or 8 cents per share compared to a net loss of $15.7 million or 5 cents per share for the second quarter of 2024. Excluding the $30 million upfront payment related to DMX 200 agreement, we would have delivered positive gap net income for the quarter. In Q2 2025, non-gap net income was $1.9 million or 1 cent per share compared to non-gap net income of $18.5 million or 6 cents per share in the second quarter of 2024. Cash, cash equivalents, and marketable securities were $231 million as of June 30, 2025, compared to $250 million as of December 31, 2024. On slide 19, we are reiterating our full year financial guidance for 2025 as follows. Total revenue growth of 15 to 22 percent, Garland Fold revenue growth of 10 to 15 percent, Pombility and Otfolder revenue growth of 50 to 65 percent. All of these growth rates are at constant exchange rates. Gross margin is expected to be in the mid-80s. Non-gap operating expense guidance remains at $380 to $400 million. And we anticipate positive gap net income during the second half of 2025. As mentioned earlier this year, 2025 will be a hybrid year for Pombility and Otfolder inventory during the first half of the year. As a result, we expect our gross margin to be in the mid-80s for 2025 as we begin to recognize Pombility, Otfolder, COGS through the P&L in the second half of the year. And with that, let me turn the call back over to Bradley for our closing comments.

Great. Thank you, Simon, Jeff, and Sebastian. As we come to the end of our presentation, here's just a quick reminder of our strategic priorities for the year. And in closing, I want to reiterate how encouraged we are by the growing demand for our therapies and the very promising Phase III asset that we've added to our pipeline. We see a clear path to sustained growth in 2025 and beyond, and we've demonstrated that we have the portfolio and capabilities to deliver that at a highly attractive growth trajectory. Amicus continues to represent a very differentiated company in biotech and rare disease with now 17 successive quarters of double-digit revenue growth, a de-risk portfolio in growing categories, and an efficient and highly effective organization that is laser-focused on delivering for patients with rare diseases. I have full confidence that we will continue to advance transformative treatments and create lasting value for patients and shareholders alike. With that, operator, we can now open the call to questions.

Ladies and gentlemen, if you have a question, please press star 1-1 on your touchtone telephone. At this time, we request that you only ask one question. If you have an additional question, please enter back into the queue. Thank you. Please stand by while we compile the Q&A roster.

Our first question comes from the line of Anupam

Rama

of JPMorgan.

Your line is now open.

Hi, guys. This is Priyanka Anupam. Congrats on the quarter. So looking at the real-world evidence, what clinical assessments really resonate with physicians and KOLs and patients switched from -via-zyme to palm-ops? And are there differences between the U.S. and .U.S.? Thanks.

Thanks, Priyanka. I'll turn it over to Jeff in a minute to provide some detail, but I think the really important thing here is now that we have multiple treatments, this is exactly the question that I think we're helping to drive in the scientific community. And as we develop more evidence and as we demonstrate the effects of Pompiliti-Apfolda, I think that will continue to be an important part of the story. But Jeff, maybe talk kind of how that's evolved somewhat with these new therapies and what the physicians and patients are looking at.

Yeah, thanks for the question. You know, what physicians are looking for when switching from lumizyme to palm-op are not that different from what they're looking for when they're switching from -via-zyme to palm-op. I mean, the majority of patients that are switching here early in the launch tend to be those that are on -via-zyme, have either were naive and went on -via-zyme or switched from lumizyme and are not having the outcome that they had hoped when they went on -via-zyme. And they're looking for either stability of the declining function or improvements in things where there had been stability previously. And typically as shown, you know, on the slide in the presentation, they look at things like biomarkers, muscle strength, and then things like -minute-long FEC. As well, of course, is just quality of life. How is the patient doing -to-day and activities of daily living? And what was really exciting from the two case studies highlighted here in the presentation as well as what we're hearing more broadly is that similar to what we saw in trials and so far in some of the different studies ongoing, those patients switching from -via-zyme seem to also be having, you know, on average or, you know, in many cases a very positive experience on that switch. But it really is not that different switching from -via-zyme than switching from lumizyme. So, I think it's a pretty similar process.

Thanks, Jeff. And then I don't think to your question, Priyanka, I do not think there's really much difference between the U.S. and other geographies.

One moment for our next question.

Our next question comes from the line of Joe Schwartz of Lirinc. Your line is now open.

Great. Thanks very much and congrats on a strong quarter. So, my one question, I guess, will be on with tariffs and MFN remaining a topic of discussion now. I was wondering if you could just update us on your additional manufacturing facility for palm op in Ireland. When could that come online and does that get you to where you think you need to be to the extent anyone can forecast the future in this regard? And do you think that could supply all of the palm op that you forecast you'll need? And can you just remind us how much drug you've stockpiled in the U.S.? Thanks.

Thank you very much, Joe. A few questions there. Maybe I'll kind of go in reverse order. So, we brought all of the material into the United States for palm bilia folder that we needed for commercial use this year and clinical use as well. And that is what led us to say that there is no material impact of tariffs on our P&L this year. And any forecast that we've been able to do going forward even at relatively conservative levels is very manageable within our P&L based on the new global supply strategy. As relates to Ireland, from a capacity perspective, yes, especially as we look toward the second generation manufacturing process, which will evolve over the next kind of five years, that could supply the global demand that we forecast. However, it's very likely that we may have a secondary site just from a good strategic perspective. Right now, that site is China, which could serve Europe and -U.S. markets, but there is a lot of other things that are happening there. And I would point you to the announcement we had in Q1, which was for the very first time bringing drug product manufacturing to the United States with a collaboration with Sharp. We may continue to evolve that as time goes on, depending on the political landscape. But I think we've been very prudent and very forward looking to have a diverse supply chain. And the last question in terms of when those things will come on board. For Ireland, we believe that that material will enter the commercial supply chain toward the back half of this year in Europe and then sometime next year in the United States, which is exactly what we forecasted. So we think we're in really good shape. We've been able to navigate all the kind of headwinds and challenges that are out there, and we expect a very robust optimized supply chain going forward.

Very helpful. Thanks for the insight. Yeah. Thanks,

Joe.

One moment for our next question. Our next question comes from the line of Maxwell Skor of Morgan Stanley. Your line is now open.

Great. Thank you for taking my question and congratulations on the quarter. So now that you've read the brief submitted by Urubindo for summary judgment, do you still feel confident in your IP position and the potential for a settlement? Thank you.

Yeah. Thanks, Max. Just as a reminder, we've said that we remain highly confident in the strength of our IP and the long-term opportunity to support February patients in the many years to come. Of course, the settlement with TEVA reinforces our confidence in the strength of our case against any remaining litigants, including Urubindo, and overall the strength, breadth, and depth of our IP estate. And because we're still in litigation, we can't comment further on the details there, but I would just say we remain highly confident that our IP is long and is supported. And we would remind everybody just statistically the vast majority of these cases ultimately leads to settlement, in particular when one party reaches a settlement first. So hopefully that's helpful and I look forward to providing further updates as time goes on.

Great. Thank you.

One moment for our next question. Our next question comes in line of Ritu Borrow of TD Cohen. Your line is now open.

Hi, Brad. This is Joshua Fleischman on the line for Ritu. One multi-part question. How are timelines progressing for the new U.S. manufacturing process and what do you think its impact could be on COGS? How do you view additional pipeline expansion and what would your priorities be? And what should we expect for POM Ops launch in the next few months? And what are the next 12 months? As more -the-asign patients approach the important two-year mark for treatment reevaluation? And how is the current reimbursement situation? Thank you.

So I see you've adopted Ritu's approach to one question with multiple parts. We'll do our best to answer all of them. Thank you, Joshua. Maybe, Sebastian, do you want to speak to the U.S. drug product manufacturing facility and the general timelines there? We haven't given real specifics, but just a flavor for sort of how that will evolve and then we'll take the next few as well.

Yes. Thanks, Brad. So you saw that as we announced in Q1, we signed an agreement with Sharp Sterol to bring the POMBILITY drug product manufacturing to the U.S. So it's essentially on-shoring DP manufacturing for POMBILITY. And so we'll be working through our PBQs in the next few quarters. We haven't shared specific timelines yet on what that site might be up and running. In the meantime, you've heard from Brande the progress we've made on the DS side of things from the Dundalk Island site. So very excited with the progress we're making here with both EMA and FDA. And then in parallel, we have another site for DP in Germany, Leverkusen, where we're also making great progress and are actually just started. PPQ runs as we speak. So very advanced in our overall manufacturing strategy for POMBILITY.

Thanks, Sebastian. Just to hit a couple of the other points you made there, I'll start with the maybe quickest one first. So reimbursement continues to go really well in all of our geographies. You've seen we have oftentimes been first or fastest in multiple markets like the first ever approval from NICE prior to MHRA approval, fastest to getting to reimbursement in a number of markets, getting lead position in a number of markets like the Netherlands, and I think that just reflects our approach to maximizing access and delivering value for all stakeholders. We'll continue to do that. To your point about NexVizime switches, as we've said previously, a significant portion of the United States as an example of the NexVizime community have come to that sort of two-year switch point and that obviously will continue to grow over time. I think it was important, Sebastian's highlight, that we are switching sort of relative to market share. So in the U.S. where the majority of patients are on NexVizime, a majority of our new patients are coming from NexVizime as well. And then your last question on pipeline expansion, we're really excited about DMX 200 and about telling that story in more detail over the course of the year. From a BD perspective, we still think there are opportunities to leverage our infrastructure and capabilities globally and would continue to be focused on late-stage de-risk assets, near commercial assets, similar to DMX 200. Hopefully that was helpful. I think I caught them all, so thanks, Joshua, for the questions. Thank you so much.

One moment for our next question. Our next question comes from the line of Eliana Merle of UBS. Your line is now open.

Hi, this is Tejasan for LE. Congrats on the quarter. Could you guys give a little bit more color on how starts are going in -U.S. markets? I know you mentioned the Netherlands, Sweden. So any color there would be great. Thank you.

Sebastian, you want to just give a few highlights on some of the exciting things we're seeing in some of the different markets. There's lots we could tell, but maybe Spain would be a good one to highlight. I don't know, Germany, the UK, etc.

Yeah, yeah. So we continue to see strengths from the first market we launched. So as Brad mentioned, the UK and Germany. Interestingly, in the UK, when you take into account the EAMS program and the fact that POMOT was essentially available for physicians almost two years prior to launch, we've been in that market for about three and a half years, and now our market share is reaching 35%. So we see that market with a lot of enthusiasm as to what we could achieve in other markets as well. Germany remains strong. Spain, as you said, Brad, this was an interesting situation where we actually launched -on-neck with Nixiazine. We're seeing, again, making significant inroads from a market share standpoint in that market. We've got smaller markets like Sweden, where POMOT has actually the drug to be on if you have LOPD in Sweden, and so we have a disproportionate market share in the Swedish market as a result of that. We're launching in Italy as we speak. I did say that this year alone we have now six new countries we launched, and we anticipate another four for the remaining of the year. That would include our Japanese launch in the second half. So lots of room to continue to grow POMBILITY and our fall down. Great. Thanks, Sebastian.

Thanks for the question.

One moment for our next question. Our next question comes from the line of Kristin Kluska of Cancer Fitzgerald. Your line is now open.

Hi. Good morning and congrats on the nice revenue beat. So for POMOP, with 40% of the patient pool treated on Nixiazine reaching that two years this year, curious now that you have more data behind your hands, what's making patients switch right at two years versus earlier versus perhaps later on? Is it their total time diagnosed with POMP, are there any specific drivers that again would make someone switch earlier, later, or right at that two year mark? Thank you.

Yeah, thanks, Kristin. I think embedded in your question is the reality, which is it's not like at two years everybody switches. It is a continuum. I think the earlier switches have been people who were clearly declining on regardless of what therapy they're on, Luma-Zymon or Nixiazine, and I think that will continue to happen if it's an obvious decline. I think physicians and patients are looking for something new and different. And that probably skews the initial patient population also to a more severe patient population. Sometimes that can be an older patient population. But the exciting thing is we're the only product in our -to-head study that showed an improvement, and I think that's, as Jeff said, improvement is possible with this product. I think that's really the promise of what we can offer. Over time though, I think two things can happen. The first is what Jeff talked about earlier, which is I think the physician community now, I don't think, I know, is asking themselves what do we need to look at, how closely do we need to look at some of these measures to find a more subtle early predictor of decline? And so I think that's a very important conversation that's happening right now. I think the holy grail in what our ambition is, and we kind of saw this with Gallifold, is instead of waiting for decline, eventually we believe we can establish palm op as the best therapy out there, and then you'll see a proactive switch. The other dynamic that's kind of flowing through all of that is also where patients sort of raise their hand and say, hey, I'm not feeling well or I want to try something new, and that's been an exciting part of the story as well. So hopefully that gives you a flavor of some of the dynamics that you're getting at in your question.

And Brad, the only other thing I'd add is just looking at the long-term data that we've seen from NexBiozyme, from Lumizyme, for example, what you see on that kind of average response across parameters, like six-minute walk if you see, is that after one year, two years, you generally see on average a continued slow decline. So you would expect just thinking about people not doing well, there's going to be more patients not doing well after three years of Nex than after two, or after four years of Nex versus three, so that will continue to add people if you're just looking at those not doing well. We'd expect there to be a kind of continued growth of that over time.

Thank you. One moment for our next question.

Our next question comes from the line of Dennis Ding of Jeffries. Your line is now open.

Hi, good morning. Thanks for taking our questions. Two for me. On FSGS, can you go into a little bit more detail on the regulatory alignment you have with the FDA on three-year protein area and how much of that is actually written in stone per se? I'm just curious about the impact to that alignment if the TRIVIR outcome doesn't support two-year traditional approval. And then on number two, a question on Pompeii. I appreciate that revenue growth does look second half weighted, but I'm curious on what you hope to be the exit break going into 2026 and on a continued acceleration in 2026. And I guess what additional new countries you plan to launch in 2026. Thanks.

Great. Thank you for the questions. Maybe we'll go kind of in reverse order. So for Pompeii, exactly right, we'll see a continued benefit of that acceleration in the second half. And I think even with all the kind of new launch countries that Sebastian highlighted, I think even a further impact in Q4 as an example, which is also pretty typical anyway to what we've seen with Gallifold is Q4 tends to be a strong quarter. As we go to next year, TBD as relates to run rate going into next year, but it's exactly the question to ask. We'll know more as we go forward here. I would just say that we do think next year will be a higher absolute revenue growth than this year. We'll have a lot more color to say on that going forward. In terms of FSGS, I'll start, but then I'll have Jeff take over that question. As part of our diligence and as a prerequisite of the deal, we had to see the FDA minutes from their type C meeting. And we were very pleased with the feedback that FDA had given to Dimerics, in particular that proteinuria could serve as the primary endpoint for that study. That was really important for us to see and I think an exciting development for the community. But Jeff, you know, speak a little bit to what we've said publicly around what that may mean for the primary and then how we see Trivier's ad com as relates to our program.

Yeah, thanks, Brad. So as Brad said, the feedback from FDA for the Dimerics program is quite clear in terms of suitability of proteinuria at two years as a primary endpoint with just supportive data from GFRs as secondary. That can be measured as a percent change as responder thresholds and meeting certain thresholds of proteinuria and we expect that we'll do all of those. As it relates to the ad com, look, it's not surprising there's an ad com for Fulspare, sort of the first product going through with proteinuria as a potential primary. They had a complicated phase three where the technical GFR endpoint was missed and now they're looking at proteinuria as sort of an alternative way for approval. So I think, NET, that'll be a great conversation to have at that ad com around proteinuria. I think there's a number of really important similarities and differences between Fulspare and DMX 200 that sort of you have to think about around the ad com. So clearly similarities are they're both targeting very similar FSGS population sort of primary genetic FSGS with significant proteinuria and they're both planning to use proteinuria as a primary endpoint. Other than that, there's a lot of differences. MOAs are very different. They target different underlying pathologies, sort of the hemodynamic side of things versus the inflammatory side with DMX 200. DMX 200 is going to have a prospectively defined proteinuria endpoint for phase three and ultimately the data on proteinuria and importantly on GFR might be different for the two products. So we view a positive ad com reinforces proteinuria as a suitable endpoint, in particular when it's prospectively defined. And we would not view a Fulspare approval as a downside at all if anything that would help sort of start to really prep the FSGS community for new treatments. Mechanistically we think they're very differentiated products and would work best in different types of patients and ultimately could be synergistic together. In a negative ad com, similarly, we think will inform us about how advisors, FDA are viewing proteinuria GFR, will help us position our data. A negative outcome could be due to specifics around that kind of complex Fulspare data set and not necessarily read through to DMX 200 and ultimately could even position us as first to market if that did not work for Fulspare. So we view the ad com as sort of a positive for us. We're really looking to be informed by it, but ultimately we would hope that it's a positive ad com and Fulspare will get approved for FSGS patients and then we can quickly behind it have DMX 200 addressing a different aspect.

And sorry, one last question. Dennis, you also asked about additional countries for next year. Australia, Canada are two big ones. Over time we'll continue as we did with Gallifold going into Asia Pacific, into Latham, into more European countries. So there's still quite a bit of geographic expansion for Pompeii as well. But thanks so much for the questions. Perfect.

Thanks guys.

One moment for our next question. Our next question comes from the line of Salvin Richter of Goldman Sachs. Your line is now open.

Hey, this is Mark on for Salvin. Thanks so much for taking our question. So on DMX 200, so when can we expect the Phase 3 data and also what is the bar for success here and what would be clinically relevant in FSGS?

Jeff, do you want to take those? Just a reminder of the timelines and then the clinical meaningfulness and kind of what we think the success bar would be.

Yeah, thanks for the question. So as we said, enrollment is going extremely well in the Phase 3 study. We're on track for last patient in, which is 286 adult patients by the end of the year. That would mean last patient out two years, which are the end of 2027. So that would be the timeline with the top line full two year data. What was the second part of that question?

It was clinically relevant and the bar for success. So

clinically relevant. So one thing we're really excited about is from a powering perspective with the 286 patients two years, the study is powered to show small changes in protein area, percent changes less than 10% between groups or responder thresholds less than 10%. Then it comes down to clinical meaningfulness. You know, frankly, you could make an argument that any improvement in protein is clinically meaningful. I think one of the better ways to show that is through responder analyses. It's well established. You can get patients below certain thresholds like below three grams per gram or below 1.5 or below 0.7 that those really improve the outcomes on progression and end-stage renal disease. So I think looking at those responder thresholds will be important at a kind of patient level.

All right. Thank you. One moment for

our next question. Our next question comes from the line of Shuan Hui. Your line is

now open. Shuan, your line is now open.

I don't see any further questions. This concludes today's conference call. Have a great day.