Amicus Therapeutics at Morgan Stanley Conference: Strategic Growth and Expansion

On Tuesday, 09 September 2025, Amicus Therapeutics (NASDAQ:FOLD) presented at the Morgan Stanley 23rd Annual Global Healthcare Conference. The company outlined its strategic plans for growth, focusing on strong Q2 financial results and ambitious projections for its key products, Galafold and Pombility/Upholda. While the outlook is positive, with expectations of achieving GAAP profitability by 2025, Amicus also acknowledged the challenges posed by regulatory landscapes and competition.

Key Takeaways

• Amicus exceeded $150 million in sales for the first time in Q2 2024.
• Projected growth for Galafold is 10% to 15% in 2025, while Pombility/Upholda is expected to grow by 50% to 65%.
• The company is on track to achieve GAAP profitability in 2025.
• Amicus is expanding its market reach, launching in six new markets this year, with plans for further geographic expansion.
• Collaboration with Dimerix on DMX-200 for FSGS shows potential, with Phase 3 study results anticipated by early 2028.

Financial Results

• Q2 2024 saw sales exceeding $150 million, marking a significant milestone.
• Galafold and Pombility/Upholda are expected to drive substantial growth, with sales guidance reflecting a 10% to 15% increase for Galafold and a 50% to 65% increase for Pombility/Upholda in 2025.
• Amicus anticipates reaching GAAP profitability in 2025, leveraging significant operating income margin growth.

Operational Updates

• Galafold: Focused on targeting underdiagnosed Fabry patients, especially women and those with late-onset disease. The global market share of treated amenable patients is approximately 65%.
• Pombility/Upholda: Launched in six new markets this year, with plans to enter 10 additional countries by 2025. Aims to switch patients from Myozyme to Pomab in the Netherlands.
• DMX-200 (FSGS): The Phase 3 ACTION3 study is underway, with top-line data expected in early 2028. The study focuses on addressing the inflammatory component of FSGS.

Future Outlook

• Galafold: Growth driven by newborn screening and diagnosis of late-onset Fabry patients.
• Pombility/Upholda: Plans for continued acceleration into 2026, with launches in Canada, Australia, and smaller European markets.
• BD Strategy: Targets late-stage assets in rare renal and neuromuscular diseases, focusing on in-licensing deals with small upfront investments.

Q&A Highlights

• Galafold IP: Settlement with Teva supports protection through January 2037. The company remains confident in its patents amidst ongoing trials.
• DMX-200 (FSGS): Amicus is closely monitoring FDA reviews to inform its strategy.
• Regulatory Interactions: Tariff impacts are mitigated through manufacturing diversification and strategic pricing.

In conclusion, Amicus Therapeutics’ presentation at the Morgan Stanley Conference highlighted its strategic focus on growth and innovation. For a detailed review, refer to the full transcript below.

Full transcript - Morgan Stanley 23rd Annual Global Healthcare Conference:

Max Score, Biotech Analyst, Morgan Stanley: I’m Max Score, a biotech analyst with Morgan Stanley. I’m happy to be hosting Amicus Therapeutics. With us today is Sebastian Martell, chief business officer Jeff Castelli, chief development officer. But before we dive in, I just want to note for important disclosures, please see the Morgan Stanley research disclosure website at www.morganstanley.com/researchdisclosures. If you have any questions, please reach out to your Morgan Stanley sales representative.

So with that, maybe if we have anyone who’s not as familiar with the Amicus story, we’ll open up the floor with any high level takeaways to introduce the company. That’d be great.

Sebastian Martell, Chief Business Officer, Amicus Therapeutics: Thank you. Thank you so much. So look, you know, as as you know, Amicus is a bit of a unique, biotech story, with significant growth potential and, and profitability as well. So we’re just coming out of a strong set of Q2 numbers. For the first time, we reached a 100 and more than a $150,000,000 of sales.

We saw great traction for our Galafold therapy for patients with amenable mutations in Fabry disease. And we continue to see strong growth in our Pombility and Upholda combination therapy for Pompe disease. So we look to grow these two products this year at a very nice rate. For Galafold, we’ve given guidance for 10% to 15% growth in 2025 and for Pombility and Upholda, 50% to 65% growth. We just also recently in the second quarter signed an exciting in licensing deal for an asset for FSGS, rare kidney disease currently in Phase three.

So very excited also by that opportunity. And returning to 2025, where we expect to continue to build momentum and reach GAAP profitability you know, for the first time. So an interesting turning point for the company.

Max Score, Biotech Analyst, Morgan Stanley: Yes. Definitely. So maybe if we can dive a bit deeper, could you give us a snapshot of your business today and your plans to to sustain Galafold momentum and accelerate adoption through year end and and into 2026?

Sebastian Martell, Chief Business Officer, Amicus Therapeutics: Yes. So let’s start with Galafold. So we operate in a Fabry market that is, you know, a nicely growing market. It’s a sizable market for rare disease. Sales were $2,100,000,000 last year, for the whole of, Fabry.

And, you know, we’ve been on the market for now six years in The UK sorry, in The US and seven or eight years, in Europe, and we continue to grow at a at a double digit rate. The key drivers are really identification of naive patients. And so we’re seeing a sustained level of newly diagnosed Fabry patients. We’re seeing also a great number of women being treated for Fabry disease, a growing proportion of patients with late onset Fabry disease being diagnosed. So those are really driving the patient numbers.

If I think back to where we were prior to launch of Galafold, we were estimating the market to be about ten thousand probably patients diagnosed and half of them being, treated. Fast forward to 2024, and we estimate that there are 18,000 Fabry patients diagnosed, six thousand still untreated, and another twelve thousand are currently being treated. So there’s been very strong growth. We’re in a unique situation with, you know, having the only oral therapy for Fabry disease, only indicated for a subset of Fabry patients with amenable mutations. We continue to see strong growth even in the countries where we’ve been, the longest, and that is, again, really mostly driven by new patient identification.

Okay. On the do you want me to jump to

Max Score, Biotech Analyst, Morgan Stanley: Yeah. No. Palm Up would be great.

Sebastian Martell, Chief Business Officer, Amicus Therapeutics: So, PalmUp, you know, is a more, recent launch. We’re coming into, a market slightly smaller than the Fabry market. 2024 sales for Pompe was, were, 1,500,000,000.0, growing close to double digit. So again, a healthy healthy market and sizable rare market. We’ve been launching by the ’24, we were in actually five countries.

Our commitment this year was to launch in 10 additional countries by the ’2. We were already launched in six new markets. Last week, we launched in Japan. Yesterday, we got approval for pricing and reimbursement in Belgium. So we’re getting close to eight markets now.

We’ve got a couple more that we’ll hope to launch in the fourth quarter. So we’re definitely on track and and building momentum here. Growth will come from both an acceleration of switches from competing products in The US as well as our geographic expansion that I that I just mentioned.

Max Score, Biotech Analyst, Morgan Stanley: So I think, if I remember correct, in the first quarter, the team addressed a a rebate issue, an unexpected rebate issue in The UK that seems to have been dealt with, and you’ve been guiding to acceleration of palmop sales in the second half. Could you give any more clarity or expectation around what that curve looks like?

Sebastian Martell, Chief Business Officer, Amicus Therapeutics: Yeah. So, you know, we continue to believe that we’ll see stronger overall sales in the ’25 than we saw in the first half. This is driven by, you know, the the new launches that we’ve had. So back in late q two, for example, you know, we initiated, our launch in The Netherlands. We we won here, you know, a significant place where, Pompilipinopoda is now, seen as, you know, first rank treatment if you want.

So we’re gonna earn seventy percent of the Pompe patients in The Netherlands. That’s a bit of an interesting market because it’s a highly concentrated market. The disease is very prevalent in The Netherlands. The disease is managed through one main treatment center, so very concentrated. And so, you know, they’re planning to switch roughly seventy patients, you know, from Myozyme onto Pomab over the next twelve to eighteen months.

And this has, started towards the very end of Q2. We had our first, patient switched. And since then, we’ve continued to see patients move on to, onto Prombility Abfolda. And the the other part, you know, besides geographic expansion is really the dynamic that we’re seeing in The US. So you may remember that our approval into The US got a little bit delayed because of, the FDA wanted to inspect manufacturing site in, in China.

And that gave, almost a couple of years, as a head start to Nexeozyme. And so by the time, you know, we got into the market, there were a number of patients who had fairly recently switched onto NexaZyme. Now what we’ve seen from longitudinal data on NexaZyme or NexaZyme even is that over time, there’s a proportion of patients who start declining, and it seems that the two year end point is a bit of a turning point. There’s, you know, close to fifty percent of patients that start to experience some signs of decline after two years on therapy. As we entered the year, there was about a third of patients in The US who had been on Nexa time for two years.

As will exit the year, they’ll be close to sixty six percent of those patients. So a growing pool of patients that could potentially see a decline and would therefore be more prone to look for alternative therapies. So that’s what driving the the acceleration in The US.

Max Score, Biotech Analyst, Morgan Stanley: So through your market research, talking to doctors, obviously, you’re starting to see that switch dynamic play out. Is there any guidance in regards to where it would be where we can expect the the greatest inflection? Would it be second half of the year, or is it more cautious to say maybe in 2026, we’ll see

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: more of the switch patients come on?

Sebastian Martell, Chief Business Officer, Amicus Therapeutics: So we we definitely stick to the guidance that we’ll see stronger sales in the second half this year. You know, it’s a bit early to get into 2026, but, you know, we would expect as a result of both US acceleration and then further geographical expansion to to continue to see an acceleration into 2026. You know, if you think of new markets, we got approval for Canada and Australia, for example, this year, but have not launched yet because we’re in the midst of pricing reimbursement discussions. These will lead us to launch in 2026. And next year, we’ll still have, you know, three or four smaller European markets where we intend to launch as well.

So there’ll be some further new countries to come in in into play in 2026.

Max Score, Biotech Analyst, Morgan Stanley: Okay. That’s helpful. So in regards to Palm Op, where are you seeing the most friction? We can also highlight the tailwinds. You’ve talked about expansion.

But in regards to the most friction or or issues you’re trying to address currently, is it on US payer coverage, step through, side of care dynamics, switching dynamics? Any any additional color on that? No. So on

Sebastian Martell, Chief Business Officer, Amicus Therapeutics: the payer side, we’re not seeing any, specific issues. On the, just the logistics of getting from prescription to actual infusion, you know, we’ve we’ve seen since launch a a gradual improvement into those timelines and now we’ve we’ve kind of reached, what I would say is, you know, normal expected timelines, for this process. So this is now a well oiled, machine. I think we, you know, we continue to invest a good amount of our time and especially through our medical teams in, you know, helping educate physicians and and patients as well on in some way, resetting expectations, you know, what goods look like. And for too long, the expectation was that stability was okay.

So if the patient treated was stable, that was good enough. Trying to shift the mindset and make people realize that given the data we’ve generated with POM up, people can hope for improvements while on therapy. And that takes a bit of time. We’re adding and maybe, Jeff, you know, I’ll ask you to build on that, but we’re collecting data, building our real world evidence data, and and those will become also critical as we continue to differentiate, probability and uphold them.

Max Score, Biotech Analyst, Morgan Stanley: Yeah. That was my next question in regards.

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Great. Great segue. Thanks, and and good afternoon, everyone. So, yeah, as Ed mentioned, you know, our phase three data, the initial studies in PROPEL showed patients actually after being on Lumizyme for long term had improvements in motor function after switching and stability and forced vital capacity, two of the key measures in Pompe disease. Actually, we just came off a very successful ICIM meeting in Japan just in the last couple of days.

Some great data presented there showing that on motor function over four years in the extension, those initial improvements in motor function were maintained over four years. And similarly, we’ve seen up to seven year durability of effect from our phase two patients. We’re starting to see now also a lot more real world evidence, not only of patients going on a palmop having good experiences, but interestingly, you know, NexBiozym switching to palmop case studies. We’ve had some of those reported at conferences. We’re continuing to hear that that is a very positive experience for those patients, seeing things like biomarker improvement, muscle strength improvement, some functional improvements in some of those case studies.

We also, for the first time, just had presented independently from Amicus at ICM in The UK. Some of the sites that have the most experience with PAMA that were involved in the expanded access to medicines program had large pools of patients, and one of the sites put about half their patients on Palm Op and half on NexViZone. And they just reported out how those patients did across different measures, and it was very favorable for those patients going to Palm Op and especially on the motor function different measures that there was a noticeable improvement in the patients moved to palm up and and not so for the patients that went on to next. So I think additional data like that is gonna continue to highlight the differentiated profile that we believe we have with palm up and hopefully move beyond, as as Sebastian said, you know, waiting for patients to be worsening to switch to palmop and start to actually have patients switch when they’re actually stable on the treatment because they have that potential to to get improvement.

Max Score, Biotech Analyst, Morgan Stanley: And can you clarify in regards to new patient starts, let’s say, treatment naive patients, what that opportunity looks like, who’s capturing that market now, and any color around that? Yes. So

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: outside of The United States, PALMoF has a broad label, naive patients, switch patients, and we are winning more than our fair share of those naive patient starts. Generally, a small number, but we believe, and I think the physicians in those countries believe the best time to start palm up is right away to have the most time to be on that product. In The United States, we have a different label. That label is for patients not improving on their current ERT, which means naive patients typically have to go on to one of the other treatments. Physicians interpret that.

If my patient is not actually experiencing improvement, then they can consider POM off, so that would be stable or worsening patients. Currently, now it’s probably used more for those patients where there’s some worsening. I think as we continue to build on that body of evidence and really start to win over kind of the mindset of POM OPS differentiated, we’ll start to hopefully get into that stable group as well. But we’re we’re really excited about that opportunity in The US. But maybe even down the future, we’ll continue to collect data on naive patients.

Patients in our trials have done well when they started on palm up. And at some point, hopefully, we can actually work with FDA and and get the label expanded to naive patients like like it should be.

Max Score, Biotech Analyst, Morgan Stanley: So what is the gating factor there? Is it just more data, receptive FDA?

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Both.

Max Score, Biotech Analyst, Morgan Stanley: Okay. Maybe I’ll take it quick. It is an opportunity just to ask, in regards to the regulatory landscape, how have interactions gone with the FDA? Are you having any sort of regular interactions?

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Yeah. We we continue to interact with FDA. We still have ongoing pediatric studies on both Fabry and Pompe, and we’ve worked with FDA on those studies. We are getting ready to start to interact with FDA more on FSGS, which we’ll talk about in a bit. We are always looking to engage as there might be opportunities to, as I mentioned, maybe look for naive labeling for a palmop, for example.

But so far, you know, we it’s it’s not a high interaction time with FDA for us just based on we are where we are with our products. But certainly for DMX 200, I think we’ll have some very substantial meetings with the FDA probably early next year.

Max Score, Biotech Analyst, Morgan Stanley: Okay. That’s helpful. And now switching over to Galafold. Where are you focusing medical education now? Amendable mutation testing workflow, adherence support, or expansion into newer geographies?

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Yeah. So, I mean, with Galafold in our mature markets, Galafold is now standard of care in amenable patients. We have upwards eighty, ninety percent plus of share of treated amenable patients. We still are having geographic expansion in some countries. I think we’re up to about 65% global market share of treated amenable patients.

So still some significant opportunity to get that closer to 90%. Where we’re really seeing the most growth, even this far into launch, six, eight years into launch, is in new patients coming on to treatment. Fabry remains one of the most underdiagnosed genetic diseases. There’s a lot of tailwinds for diagnosis. It used to be that patients with their physicians would do everything in the world before they’d actually get a genetic test because it costs too much.

Now there’s low cost genetic tests for cardiac panels, renal panels that as soon as the physician suspects, I have a renal patient or cardiac patient or a patient I can’t figure out what’s going on, they’ll pretty quickly defer to those tests, and we can have Fabry diagnosis much more easily. So that’s one tailwind. Another big tailwind in The US is newborn screening. For Fabry, there’s eight states that are actively screening for Fabry. That’s about fifteen percent of newborns covered in those states.

And what you’re finding there is not that that newborn needs treatment right away, but you find a whole family when you diagnose that newborn because Fabry is an excellent dominant disease, and you can do family screening. I think that contributed to the twenty five percent or so growth we had last quarter year over year in The US, bless you, for Fabry. So we think there’s a lot of tailwinds to to keep growing that market. And and the last thing I’ll mention, so Galafold is only for amenable Fabry patients with certain types of mutations. Most of the patients in the in the Fabry community left to be diagnosed are later onset Fabry.

They don’t have the early onset mutations that are easier to diagnose. Most of the late onset mutations are amenable to Galafold. So all this continued growth in the future of growing diagnosis is gonna be slanted towards people amenable for Galafold. So I think we’ll continue to see, like we have seen, the Galafold growth is much higher than the overall Fabry market. Okay.

And remind me the compliance rate. It’s high. It’s greater than ninety percent? We’ve continued since launch to have over ninety percent adherence compliance, which for an oral medication administered every other day is quite remarkable, and I think speaks to the experience that the the patients are having on the product.

Max Score, Biotech Analyst, Morgan Stanley: Okay. And then I get questions around the IP IP discussion around Galafold. So could you just frame how the Teva settlement supporting protection through January 2037 should be interpreted by investors? How are you thinking about remaining legal overhangs and, potentially, there would be no challenge?

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Yeah. So we had three generics that filed for for Galafold. So Teva, Aurobindo, and Lupin. Lupin took a stay, which means they’re sort of now dependent on what happens with the other two parties. End of last year, we settled with Teva for January 2037 entrance.

Our patent estate actually over 40 patents, one’s book listed, goes into the early twenty forties. So that was a settlement with Teva where we felt like it was appropriate that we gave up some of the long term for that settlement. Orbindo is is still actively looking towards a trial. As you look, we’ve said that most cases, Orbindo, when a lead sort of party has settled, they would generally settle. The trial date is set for September 29.

We still think there is a good chance of a settlement up until that date. But that being said, if we go to trial, we’re still very confident. We still feel very confident in our our patents and we would look forward to to the outcome of of that trial. Should we be anticipating a summary judgment from

Max Score, Biotech Analyst, Morgan Stanley: the judge coming out at any point now before the twenty ninth?

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: No. That’s a great question, Max. And so so one of the things is Orbundo did file for summary judgment. So they they’ve already acknowledged the potential for infringement. So if if they are not successful, they will be infringing our patents.

They’ve acknowledged that. What they’re trying to do is look at obviousness claims to say these are sort of obvious inventions. So they they filed for summary judgment. The judge does not have to rule on that. The judge can actually say, I’m just gonna let the trial happen.

You know, one of the things that could be a factor out there that could weigh in on, you a settlement would be if there is no summary judgment, that does sort of put more pressure a little bit on Aurobindo to settle, or they go to trial where they run the expense the risk of all the expenses of the trial. And if they’re not successful, they would be dependent on waiting for the twenty forty two patents to end, whereas Teva could end in the market in 2037.

Max Score, Biotech Analyst, Morgan Stanley: So how timing around the trial, how long would generally this process take? So trial is typically it’ll it’ll be four to five

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: days starting on the twenty ninth. And then that’s the sort of oral arguments, and then the the judge can take up to six months typically after that to actually issue a ruling. Okay.

Max Score, Biotech Analyst, Morgan Stanley: Helpful. So maybe let’s switch over to DMX 200, a product of a recent business development deal that that you the team signed. Maybe if you can inter introduce this asset, the the indication you’re going after, and and catalyst that we’re we can look forward to.

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Yeah. So, I mean, we’re we’re very excited to be partnered with Dimerix on DMX 200 for FSGS, which is focal segmental glomerulosclerosis. It basically means that the glomerulus, which is the filtering unit of the kidney, has parts of its segments that are sclerotic or fibrotic. And and, basically, the kidney is in an ongoing cycle of kind of losing activity, and eventually, those patients will end up needing transplant or dialysis. And it’s a very serious disease.

No current treatments approved. About forty to eighty thousand patients in The US are estimated to

Max Score, Biotech Analyst, Morgan Stanley: have

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: FSGS. The deal that we did, Amicus has US rights. Dimerix has ex US partners. And the DMX two hundred is a CCR two inhibitor. So CCR two is one of the key receptors that leads to monocyte or macrophage driven inflammation.

So a key aspect of FSGS is the kidney gets damaged. And then to compensate for that, because the kidney needs to filter the blood, the body starts to put more pressure into the kidney to try to get the filtering to happen. That’s sort of the hemofiltration damage that can happen, and drugs like ARBs and even Filsparia that’s out there for FSGS are addressing that component. Another component is that leads to inflammation that further kind of causes fibrosis and sclerosis in the kidney that makes it work even less well, so more hemofiltration pressure. So you get in this pathogenic loop of high hyperfiltration, high inflammation.

And what we’re really looking with DMX two 100 is to break the inflammatory side of that cycle on the monocyte macrophage inflammation. So no other agent is addressing that aspect of this disease. The the product is in phase three for a large action three study targeting final enrollment by the end of the year of two hundred eighty six patients. So we’re very excited to continue to work with Dimerix to enroll that trial. That would mean a top line readout.

The study is a two year study currently as designed. We just recently got agreement with FDA that proteinuria can serve as the primary endpoint, which is a big win and was really part of the the final bit of data that we wanted to see before we executed the deal. We feel that the power is ex the the study is extremely well powered for proteinuria. So we’re gearing up for that two year top line data 2027, early twenty twenty eight, based on the proteinuria with supportive data on GFR. One upcoming event here is the you know, Filspari is another drug from Trevyr that’s in FSGS.

It approaches a different mechanistic side of the disease, more

Max Score, Biotech Analyst, Morgan Stanley: in that

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: hemodynamic hemodynamic hyperfiltration damage I talked about. They have an AdCom in November. They have a PDUFA date in January. So we’re looking forward to that AdCom to really see how FDA is is sort of or we see advisers and FDA are looking at the data package of proteinuria and GFR so we can then finalize the protocol, the SAP for our, our study.

Max Score, Biotech Analyst, Morgan Stanley: For the phase three study? Yes. Is there anything more you can tell us about the design of the proposed phase three study, or it’s really hinging on this AdCom? I mean,

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: the the study is well underway. So it is in patients with primary genetic FHTS that despite ongoing treatments, corticosteroids, angiotensin receptor blockers have high proteinuria, and the patients are sort of put on the stable ARBs, which is important, and then randomized to either DMX two hundred or placebo. As I mentioned, a two year study. Proteinuria will be the primary endpoint. We’ll sort of look from the AdCom, you know, how to sort of measure that, how to position it.

FDA said GFR needs to be a secondary endpoint and supportive. If you look at the FOLSPARI data set, you know, we interpret that as meaning GFR trending in the right direction, but not necessarily significant. Although, you know, we are looking to power the action three study for superiority in GFR as well. That’s important for Demerix and partners outside of The US. So the study is designed I would say we’re really just looking to finalize the the statistical analysis plan and sort of the how we tier the endpoints, how we measure the proteinuria.

Max Score, Biotech Analyst, Morgan Stanley: Okay. And so, hypothetically, how do you envision positioning it commercially given the lack of FDA approved therapies in this space? But there is a lot happening.

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Yeah. Well, you know, assuming Filspire is successful, they would be on the market. And let’s assume that it’s a two year study for us and and then and then an approval and a launch.

Max Score, Biotech Analyst, Morgan Stanley: We’d be about two and

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: a half, three years behind Fulspari. So I think, clearly, they’d be out there educating the market about new treatments. I think there will certainly be patients that initially don’t respond to Tulspari’s mechanism of action that could be where their issue is more of a monocyte macrophage inflammation that could then be candidates for DMX two hundred. I do think there also are ways that physicians can start to look at their patients and figure out which ones are likely to benefit from DMX two hundred, for example. There are some things you can measure, like the the levels of the MCP one, which is the chemokine that is really being kind of inhibited in in with DMX 200, other inflammatory markers that you can measure.

So I think there’ll be a, you know, a way that physicians can start to identify that these are patients where we think DMX 200 could really have a benefit. And I think at some point, you’ll see that there’s generally different populations that are responding better to one or the other, and we could actually see some combination use possibly as well. There certainly seems that logically, there could be a synergistic effect of having an ARB, an endothelin inhibition, which really is Vosparis bringing that component to the story and then and then a monocyte macrophage anti inflammatory.

Max Score, Biotech Analyst, Morgan Stanley: Okay. And just going back to The UK reimbursement dynamic one more time, this will reset next year. Correct? Is it won’t carry through. Is there any clarity on what it looks like for 2026?

Sebastian Martell, Chief Business Officer, Amicus Therapeutics: You’re talking about the For VBAG? Yes. VBAG. Yeah. So, you know, earlier this year, or very late last year, you know, we finally got, what the rate was gonna be for 2025, and it was a a higher level than what was expected last year.

It was running at about a 15% rebate. And this year, this was ultimately set at 22.9%. So ABPI, the the industry body in The UK, has been having discussions and negotiations with The UK Government. Our understanding is that those have stalled for now, and so we’re still under the same scheme. I think for, you know, financial modeling purposes, you know, I I would expect to essentially assume a rate very similar, to the current one, maybe very slightly, higher than, than the twenty two point nine, but, you know, I don’t know, between 01% ish.

And so what that means is that the year over year impact, you know, should, for now, you know, be expected to be limited or minor.

Max Score, Biotech Analyst, Morgan Stanley: Minor. Okay. So just touching on BD potentially. I know we just talked about the MX 200. But on future BD, what are some of the priorities that define a fit for Amicus?

Payer comparability, geographic overlap, current assets, center based care, time to cash, development stage. How are you thinking about future BD potential?

Sebastian Martell, Chief Business Officer, Amicus Therapeutics: Yeah. Look. You know, we we really think that the the merits in licensing that we executed earlier this year was the right deal at the right time and at the right price for us. As you know, we’ve made, some important commitment with regards to profitability, and it is becoming gap profitable in the second half of this year and and, you know, hopefully thereafter. Now interestingly, if you look over the next five years, we’re gonna have, you know, continued significant growth in the top line.

We don’t expect our OpEx, you know, to grow at a at a high rate, probably, you know, low to mid single digit, rates. So that actually means that we we offer over the next five years some fairly significant operating leverage. So we’re gonna see, you know, our profitability and and, operating income margin grow in a significant way. This will, over time, give us space to take on more, r and d spend. And, you know, I would I would say that our focus is more on late stage assets.

You know, we continue to look for opportunities for, you know, things that could be adjacent to the therapeutic areas we’re in. So rare renal diseases, rare neuromuscular diseases, but with a very strong focus on ongoing phase three or even post phase three readouts, products and registration. Ideally, we like, you know, in licensing, where, you know, you put, relatively small upfront at risk and and then pay for success, and look for things that could be fairly accretive to, to both top line and and bottom line. So looking for ways to further accelerate the growth profile that, we offer with, the current setup.

Max Score, Biotech Analyst, Morgan Stanley: Okay. Great. So we’ve been asking some of our our companies some survey questions. I think it’s appropriate now to ask this question regarding China’s rise in biotech innovation. How are you thinking about your competitive position here, and will it at all influence your r and d or BD strategy?

Sebastian Martell, Chief Business Officer, Amicus Therapeutics: So we’re we’re kind of agnostic as to, you know, where the the the potential licensing opportunities may may come up. You know, the Dimerix transaction was with an Australian company with whom we had built relationship for the last two years. So there’s an awful lot going on with US biotech companies as well. We nurture relationship with a number of partners across, you know, different countries in the world, including including Chinese companies as well. You know, our framework is very much around, you know, some specific therapeutic areas.

We pay a lot of interest on the unmet need, on, the probability of success of, you know, of a phase three trial. We look at the, you know, the manufacturing aspects as well. They they, they play a big role also. There’s nothing wrong with small molecules. And yeah, so we keep an open mind about, you know, where innovation may come from.

Max Score, Biotech Analyst, Morgan Stanley: Great. One more question in, in regards to what’s been most impactful from a regulatory side? Would you say it’s, the FDA, MFN, or tariffs?

Sebastian Martell, Chief Business Officer, Amicus Therapeutics: I’ll leave you this one. Yeah.

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: I mean, tariffs, for us, when you look at Galafold, very low cost of goods. So tariffs really don’t have much impact there. When you look at sort of government payers sorry. Sorry. So finishing off tariffs for for Pompe, obviously, a little bit more cost.

Most of that for this year is fully accounted for. Next year, we’ve said fully manageable with our p and l, what we might project for tariff costs. We have been very successfully transferring manufacturing from Wuxi, China to Wuxi, Ireland. So we feel tariffs are pretty mitigated. You know, FDA, it’s so there’s gives and takes, I think, there.

Where we are, you know, so far, Dimerix has had great interactions with FDA. So I I view that as as not really an issue right now at all. The MFN theoretically could be a a a a challenge. But, again, as you look at, you know, the political stance where that is, it’s it’s likely that rare disease could be carved out or whatever happened similar to IRA. Also, if you look kind of our government US government sales, they’re about 10% of our sales as a company, and that’s really what we most at risk is maybe having a 50% haircut in that.

So while 5% of revenue is not great, certainly, is manageable. But, again, we think it’s probably low likelihood for rare disease. So none of the above would be my answer.

Max Score, Biotech Analyst, Morgan Stanley: Yeah. That’s great. And I think we’re up on time. Thank you very much. Really appreciate it.

Thank you.

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