Amicus Therapeutics Inc (FOLD) 2025 Q2 法說會逐字稿

Good morning, ladies and gentlemen. Welcome to the Amicus Therapeutics' second-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' second-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 is 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 is my pleasure to turn the call over to Bradley Campbell, President and Chief Executive Officer. Bradley?

Great. Thank you, Andrew. 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 core business in Pompe and Fabry disease. This is our 17th consecutive quarter with double-digit sales growth at CER. 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. Galafold delivered 13% year-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 (inaudible) demand since our 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 in Pompe 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 Galafold 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 in DMX-200, a first-in-class compound in late-stage Phase III development for a rare and life-threatening kidney disease. The ACTION3 pivotal study remains on track for full enrollment by the end of the year, marking a key milestone in this important program.

DMX-200 and our opportunity to meet the significant unmet need that exists for people living with FSGS is an important new part of the Amicus story. 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 GAAP 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. Good morning, everybody.

Let's start with Galafold on slide 5. You see that revenue reached $128.9 million this quarter, up 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 Fabry patients with amenable mutations. Galafold is clearly positioned as the treatment of choice amongst prescribers. There's still many more potential patients eligible for our therapy.

Turning to slide 6, our leading markets continue to be the biggest driver of strong patient demand for Galafold. The US actually contributed significantly to growth, reaching more than a 1,000 PRS since launch, a major milestone. When we look at the global mix of patients on Galafold today, which is about 65% naïve and 35% switch, we're now seeing stronger uptake in naïve populations.

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.

With underlying growth in patient demand at 13% this quarter 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.

The key drivers behind the robust demand for Galafold, 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 Galafold into new markets and extend the label.

Third, driving Galafold's share of treated 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 Galafold predominantly stay on Galafold.

On slide 7, we highlight the significant unmet need in Fabry disease today. Over 12,000 people receive Fabry treatment worldwide, while 6,000 diagnosed patients remain untreated. Literature suggests actual prevalence makes it over 100,000 individuals, indicating a meaningfully larger underdiagnosed population than originally believed 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 record high growth in naïve new patient starts. We're just only scratching the surface with Galafold today. And as disease awareness and enhanced diagnostics initiatives further shape the Fabry market, we're confident in the 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, Galafold has a long runway well into the next decade and a clear path to surpassing $1 billion in revenue.

Turning now to Pompe disease on slide 9, we outline our global launch progress with Pombiliti 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 a broadening and deepening of prescriptions with more sites coming online and multiple new prescriptions from physicians.

Given these indicators, we are reiterating our full-year 2025 revenue growth guidance for Pombiliti and Opfolda of 50% to 65% growth at constant exchange rates. Our guidance implies a healthy exit rate heading into the next year. We remain highly confident in the 2025 and long-term outlook for this therapy. We expect Pombiliti 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 net-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 Pombiliti and Opfolda, in the second quarter, we recorded revenue in 11 countries. 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 Pombiliti and Opfolda was selected as a preferred treatment for adults with LOPD in the Netherlands. It's an important market. All patients go to 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 Pombiliti 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 execution and growth in Galafold and the building momentum in the launch of Pombiliti and Opfolda.

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

Thank you, Sebastien. 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 Pombiliti 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 Nexviazyme to Pombiliti 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 Pombiliti and Opfolda. We believe our ongoing efforts to grow this body of evidence will ultimately drive wider adoption of Pombiliti 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 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 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 non-specific therapies such as corticosteroids, calcineurin 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 in chemokine receptor type 2 [heterodimer] 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.

Pre-clinical 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 MCP-1 rebound effects observed with traditional CCR2 inhibitors.

Moving on to slide 16, we are very impressed by the strong momentum Dimerix has built and the 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 in 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 ACTION3 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 same 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 US.

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 GAAP operating expenses increased to $148.9 million for the second quarter of 2025 as compared to $100.4 million in the second quarter of '24, an increase of 48%. It is important to remember that Q2 operating expenses included the up-front payment of $30 million for the US licensing rights to DMX-200. On a non-GAAP 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-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 depreciation and amortization.

On a GAAP basis, net loss in the second quarter of 2025 was $24.4 million or $0.08 per share compared to a net loss of $15.7 million or $0.05 per share for the second quarter of 2024. Excluding the $30 million up-front payment related to DMX-200 agreement, we would have delivered positive GAAP net income for the quarter. In Q2 2025, non-GAAP net income was $1.9 million or $0.01 per share as compared to non-GAAP net income of $18.5 million or $0.06 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, '24.

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 of these growth rates are at constant exchange rates.

Gross margin is expected to be in the mid-80%s. Non-GAAP operating expense guidance remains at $380 million to $400 million. And we anticipate positive GAAP net income during the second half of 2025.

As mentioned earlier this year, 2025 will be a hybrid year for Pombiliti and Opfolda COGS, as we have worked through the previously expensed or zero cost inventory during the first half of the year. As a result, we expect our gross margin to be in the mid-80%s for 2025 as we begin to recognize Pombiliti-Opfolda COGS through the P&L in the second half of the year.

With that, let me turn the call back over to Bradley for our closing comments.

Great. Thank you, Simon, Jeff, and Sebastien.

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. 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-risked 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.

(Operator Instructions)

Anupam Rama, J.P. Morgan.

This is Priyanka, on for Anupam. Congrats on the quarter.

Looking at the real-world evidence, what clinical assessments really resonate with physicians and KOLs and patients switch from Nexviazyme to Pom-Op? Are there differences between the US and OUS?

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.

As we develop more evidence and as we demonstrate the effects of Pombiliti-Opfolda, I think that will continue to be an important part of the story. But Jeff, maybe talk how that's evolved somewhat with these new therapies and what the physicians and patients are looking at.

What physicians are looking for when switching from Lumizyme to Pom-Op are not that different from what they're looking for when they're switching from Nexviazyme to Pom-Op. The majority of patients that are switching here early in the launch tend to be those that are on Nexviazyme -- either were naïve and went on Nexviazyme or switched from Lumizyme and are not having the outcome that they had hoped when they went on Nexviazyme. They're looking for either stability of the declining function or improvements in things where there had been stability previously.

Typically, as shown on the slide in the presentation, they look at things like biomarkers, muscle strength, and then things like six-minute walk FVC; as well, of course, as just quality of life, how is the patient doing day to day, and active as a daily living.

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 Nexviazyme seem to also be having -- on average or in many cases -- a very positive experience on that switch.

But it really is not that different switching from Nex than switching from Lumizyme, 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 US and other geographies.

Joe Schwartz, Leerink.

Congrats on a strong quarter.

My one question will be 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 Pom-Op in Ireland. When could that come online? Does that get you to where you think you need to be to the extent anyone can forecast the future in this regard? Do you think that could supply all of the Pom-Op that you forecast you'll need? Can you just remind us how much drug you've stockpiled in the US?

Joe, a few questions there. Maybe, I'll go in reverse order.

We brought all of the material into the United States for Pombiliti-Opfolda that we needed for commercial use this year and clinical use, as well. That is what led us to say that there is no material impact of tariffs on our P&L this year. 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 it relates to Ireland, from a capacity perspective, yes -- especially as we look towards the second-generation manufacturing process, which will evolve over the next 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 ex-US markets, but there might be other opportunities there. 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.

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 towards 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 headwinds and challenges that are out there. And we expect a very robust, optimized supply chain going forward.

Maxwell Skor, Morgan Stanley.

Congratulations on the quarter.

Now that you've read the brief submitted by Aurobindo for summary judgment, do you still feel confident in your IP position and the potential for a settlement?

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 Fabry 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 Aurobindo; and overall, the strength, breadth, and depth of our IP estate.

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.

We would remind everybody just statistically, the vast majority of these cases ultimately lead to settlement, in particular, when one party reaches a settlement first. So hopefully, that's helpful. Look forward to providing further updates as time goes on.

Ritu Baral, TD Cowen.

Brad, this is Joshua Fleishman, on the line for Ritu. One multi-part question.

How are timelines progressing for the new US manufacturing process? What do you think its impact could be on COGS? How do you view additional pipeline expansion? What would your priorities be? What should we expect for Pom-Op's launch in the next 12 months, as more Nexviazyme patients approach the important two-year mark for treatment re-evaluation? How is the current reimbursement situation?

I see you've adopted Ritu's approach to one question with multiple parts. We'll do our best to answer all of them.

Maybe, Sebastien, do you want to speak to the US drug product manufacturing facility and the general timelines there? We haven't given real specifics but just a flavor for how that will evolve. And then, we'll take the next few as well.

Yes. Thanks, Brad.

You saw that as we announced in Q1, we signed an agreement with Sharp Sterile to bring the Pombiliti drug product manufacturing to the US. So it's essentially onshoring DP manufacturing for Pombiliti. And so we'll be working through our PPQs in the next few quarters.

We haven't shared specific timelines yet on when that site might be up and running. In the meantime, you've heard from the progress we've made on the DS side of things from the Dundalk island site. So very excited to 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 actually have just started PPQ runs, as we speak. So very advanced in our overall manufacturing strategy for Pombiliti.

Thanks, Sebastien.

Just to hit a couple of the other points you made there -- I'll start with the, maybe, quickest one first. 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, which Sebastien highlighted.

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 Nexviazyme switches, yeah, as we've said previously, a significant portion in the United States, as an example, of the Nexviazyme community have come to that two-year switch point. That obviously will continue to grow over time.

I think it was important, Sebastien, to highlight that we are switching relative to market share. So in the US, where the majority of patients are on Nexviazyme, a majority of our new patients are coming from Nexviazyme 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 derisked assets, near-commercial assets similar to DMX-200.

Hopefully, that was helpful. I think I caught them all.

Eliana Merle, UBS.

This is [Tejas], on for Eli. Congrats on the quarter.

Just, could you guys give a little bit more color on how starts are going in ex-US markets? I know you mentioned the Netherlands, Sweden. So any color there would be great.

Sebastien, do 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, UK, et cetera.

Yeah. We continue to see strength from the first market we launched -- as Brad mentioned, the UK and Germany. Interestingly, in the UK, when you take into account the EAMS program and the fact that Pom-Op was essentially available for physicians almost two years prior to launch -- so 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've said, Brad, was an interesting situation where we actually launched neck and neck with Nexviazyme. And we're seeing -- again, making significant inroads from a market share standpoint in that market.

We've got smaller markets like Sweden, where Pom-Op is actually (technical difficulty) as 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 in 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 Pombiliti and Opfolda.

Kristen Kluska, Cantor Fitzgerald.

Congrats on the nice revenue beat.

For Pom-Op, with 40% of the patient pool treated on Nexviazyme 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 Pompe or are there any specific drivers that, again, would make someone switch earlier, later, or right at that two-year mark?

Kristen, 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, regardless of what therapy they're on, Lumizyme or Nexviazyme. 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 head-to-head study that showed an improvement. And I think that, 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 and what our ambition is -- and we saw this with Galafold -- is instead of waiting for decline, eventually, we believe we can establish Pom-Op as the best therapy out there and then, you'll see a proactive switch.

The other dynamic that's flowing through all of that is also where patients 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.

Brad, the only other thing I'd add is just looking at the long-term data that we've seen from Nexviazyme, from Lumizyme, for example, what you see on that average response across parameters like six-minute walk, FVC 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 and 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.

Dennis Ding, Jefferies.

Two for me.

On FSGS, can you go into a little bit more detail on the regulatory alignment you have with the FDA on your proteinuria and how much of that is actually written in stone, per se? Just curious about the impact to that alignment if the Travere AdCom doesn't support two-year traditional approval.

And then, on number two, a question on Pompe. I appreciate that revenue growth does look second-half weighted but curious on where you hope to be the exit rate going into 2026 and on a continued acceleration in 2026? What additional new countries you plan to launch in 2026?

Maybe, we'll go in reverse order.

For Pompe, exactly right, we'll see a continued benefit of that acceleration in the second half. And I think even with all the new launch countries that Sebastien highlighted, I think even a further impact in Q4, as an example; which is also pretty typical anyway to what we've seen with Galafold, Q4 tends to be a strong quarter.

As we go to next year, TBD as it 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. We were very pleased with the feedback that FDA had given to Dimerix; 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, speak a little bit to what we've said publicly around what that may mean for the primary and then, how we see Travere's AdCom as it relates to our program.

Yeah. Thanks, Brad.

As Brad said, the feedback from FDA for the Dimerix program is quite clear in terms of suitability of proteinuria at two years as a primary endpoint with just supportive data from GFR as a secondary. That can be measured as a percent change as responder thresholds and meeting certain thresholds of proteinuria. We expect that we'll do all of those.

As it relates to the AdCom, look, it's not surprising there's an AdCom for FILSPARI. It's the first product going through with proteinuria as a potential primary. They had a complicated Phase III where the technical GFR endpoint was missed. And now, they're looking at proteinuria as an alternative way for approval. So I think net-net, that will be a great conversation to have at that AdCom around proteinuria.

I think there's a number of really important similarities and differences between FILSPARI and DMX-200 that you have to think about around the AdCom. So clearly, similarities are they're both targeting very similar FSGS population, primary genetic FSGS with significant proteinuria. They're both planning to use proteinuria as a primary endpoint.

Other than that, there's a lot of differences. The MOAs are very different. They target different underlying pathology, 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 III. And ultimately, the data on proteinuria and importantly, on GFR might be different for the two products.

So we view a positive AdCom reinforces proteinuria as a suitable endpoint; in particular, when it's prospectively defined. And we would not view a FILSPARI approval as a downside at all. If anything, that would help 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.

And a negative AdCom, similarly, we think will inform us about how advisers, FDA are viewing proteinuria and GFR; will help us position our data. A negative outcome could be due to specifics around that complex FILSPARI dataset and not necessarily a readthrough to DMX-200 and ultimately, could even position us as first to market, if that did not work for FILSPARI.

So we view the AdCom as a positive-positive for us. We're really looking to be informed by it. But ultimately, we would hope that it's a positive AdCom and FILSPARI will get approved for FSGS patients; and then, we can quickly, behind it, have DMX-200 addressing a different aspect.

Sorry, one last (technical difficulty), 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 Galafold going into Asia-Pacific, into LatAm, into more European countries.

So there's still quite a bit of geographic expansion for Pompe, as well.

Salveen Richter, Goldman Sachs.

This is Mark, on for Salveen.

On DMX-200, when can we expect the Phase III data? And also, what is the bar for success here? 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 what we think the success bar would be.

Yeah. As we said, enrollment is going extremely well in the Phase III 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 would be end of 2027. So that would be the time line of the top-line full two-year data.

And so what was the second part of that question?

What was clinically relevant and the bar for success?

Clinically relevant. 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 proteinuria, percent changes less than 10% between groups or responder thresholds less than 10%.

So then it comes down to clinical meaningfulness. 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 analysis. It's well established.

If you can get patients below certain thresholds, like below 3 grams per gram or below 1.5 or below 0.7, that those really improve the outcomes on progression to end-stage renal disease, I think looking at those responder thresholds will be important at a patient level.

[Xuan Hui], (inaudible).

(technical difficulty)

At this time, I don't see any further questions.

This concludes today's conference call. Have a great day.