Amicus at UBS Conference: Strategic Expansion in Rare Diseases

On Monday, 10 November 2025, Amicus Therapeutics (NASDAQ:FOLD) presented at the UBS Global Healthcare Conference 2025. The company outlined its strategic initiatives, focusing on growth in rare diseases and financial milestones. Amicus expects profitability in the second half of the year, supported by strong cash reserves. However, challenges such as potential tariffs and ongoing patent litigation were also discussed.

Key Takeaways

• Amicus anticipates over $600 million in revenue this year, aiming for $1 billion by 2028.
• The company holds a strong cash position with over $260 million and expects profitability soon.
• Key programs include Galafold, Pombiliti Opfolda, and DMX-200, each with significant growth potential.
• Manufacturing is transitioning from China to Ireland to mitigate tariff impacts.
• Amicus is navigating intellectual property challenges with confidence in its patent portfolio.

Financial Results

• Galafold: Sales are expected to grow by 10-15%, reaching over $500 million this year.
• Pombiliti Opfolda: Anticipated sales growth of 50-65%, exceeding $100 million.
• Total Revenue: Projected to surpass $600 million this year, with a target of $1 billion by 2028.
• Cash Position: Over $260 million in cash, with $30 million generated in Q3.
• Profitability: The company is on track to turn profitable in the latter half of the year.

Operational Updates

• Galafold: Amicus aims to increase its market share from 65% to 85-90% in established markets.
• Pombiliti Opfolda: Expanded to eight new countries, aiming for a total of ten, with first-line wins in the Netherlands and Sweden.
• DMX-200: Enrollment in the Action 3 study is over 90% complete, with potential accelerated approval being explored.
• Manufacturing: Transitioning operations to Ireland to minimize tariff impacts, with European approval secured and FDA approval expected early next year.

Future Outlook

• Galafold: Continued growth driven by increased diagnosis rates and focus on VUS patients.
• Pombiliti Opfolda: Geographic expansion and real-world data support further growth, with a label expansion for adolescents expected next year.
• DMX-200: Top-line data anticipated two years post-enrollment, with potential accelerated approval based on one-year data.
• Tariffs: Managed through manufacturing shift to Ireland.
• MFN Policies: Monitoring developments, anticipating a potential rare disease carve-out.

Q&A Highlights

• Galafold IP: Results from the Aurobindo trial expected in the second half of next year.
• FSGS Market: Viewed as synergistic, with multiple agents potentially complementing treatment.
• FDA Interactions: Positive and timely responses, with no changes in endpoint views.

Readers interested in more details are encouraged to refer to the full transcript below.

Full transcript - UBS Global Healthcare Conference 2025:

David Dye, Biotech Analyst, UBS: Challenging. Okay, great.

Okay, thanks everyone for joining us. Great pleasure to have Amicus with us. My name is David Dye. I’m a biotech analyst here at UBS. It’s great pleasure to welcome Jeff Castelli, who is the Chief Development Officer with us. Jeff, welcome.

Thank you, David. It’s a pleasure to be here. Great.

Before we start, I just want to have a quick announcement just saying that anybody who cares to scan a QR code on screen, you can scan it, ask your questions and then the question will appear on my iPad and I’ll be happy to take any questions from the audience. Just let us know if you have any questions and ask away. With that out of the way, Jeff, turning to you, just talking about the Amicus story. Right. For people who are less familiar with the story, let me just give a quick overview of Amicus. What does the company do and what progress they’ve made over the past year.

Yeah, great, thanks. Amicus is a rare disease focused company. We look for first or best in class medicines, whether it’s high and mid need. We’re very unique in terms of biotech companies in the rare disease space. We actually have three programs each with a billion dollar potential opportunity and they’re at different stages. We have a strong underlying financial strength. We’re turning profitable here in the second half of the year in a good cash position, but just top line, you know, Galafold for Fabry disease is our first approved product back in 2016, 2018. We have 10%-15% growth this year for that product. Over $500 million in sales. Lots of diagnostic tailwinds and a long runway there. We’re really excited about Galafold. For our second product, Pombiliti Opfolda, we’re kind of early in the launch there. We were approved in 2023.

That product is doing 50%-65% growth this year. Going to pass $100 million in SAL there. There’s a lot of growing body of real world evidence that we’re excited about. Again, we see that as ultimately growing to a billion dollar opportunity. We’re also excited about a recent transaction we did to get rights to DMX-200 and FSGS. That is a rare kidney disease. Currently no approved treatments. We have U.S. rights. That’s another potential blockbuster opportunity with a very unique mechanism of action that’s in a phase three trial. All that on the backdrop I mentioned of finances where we’re in the past $600 million revenue this year on our way to a billion in 2028. As I mentioned, turning profitable, $260 million plus in cash, and we actually just generated over $30 million in cash in the third quarter.

A lot of exciting dynamics and a lot of on a very strong financial backbone.

That’s very, very good. Thanks for that overview. On a high level across your products, can you maybe just help us understand some of the key drivers for each and discuss your competency in meeting the guidance?

Yeah. So again, going back to Galafold, as I mentioned, really what’s driving Galafold growth is increasing patients being diagnosed and put under treatment. We have about 65% share currently of treated amenable patients and Galafold is a precision medicine only that is targeted towards a certain type of Fabry disease caused by amenable mutations. In that segment, which is about 35-50% of Fabry, we have about 65% share. In the countries where we’ve been on the market the longest, we see that we can get to 85-90% share. There is a good switch opportunity there for Galafold still. What is really driving things in the largest markets like the U.S. and in Europe and in Japan, more and more is just growth of patients being diagnosed. It still remains a very under diagnosed disease.

There’s lots of tailwinds in terms of access to gene panels in cardiology and in nephrology. There’s newborn screening happening in the U.S. in eight states that cover 15% of newborns. There’s a great opportunity for family cascade testing after you identify an index patient. All that on the backdrop of what’s really left to diagnose in Fabry is late onset Fabry, and that tends to be highly enriched for amenability to Galafold. All those tailwinds are really in the amenable part of the Fabry population. We feel like we’re a great second quarter, third quarter. We see that continued momentum on Galafold for the fourth quarter. Moving on to Pombiliti.

You know, we just came off of the strongest quarter in the third quarter of new patient ads, and that was on the back of Q2, which previously had been the strongest quarter in new ads. We see that continued momentum going into the fourth quarter. In the U.S., those new patient ads, as we see more patients that have been on Nexviazyme for two years, which we really view as one of the key populations in the U.S. as that is continuing to grow, we’re seeing that acceleration in new ads in the U.S. In the next U.S., the geographic expansion is going quite well. We’ve added eight new countries this year on the way to adding 10 total countries. Some of those are pretty interesting. One is the Netherlands, where one of the key academic centers in Europe.

There’s actually about 100 adult patients and we won first line to get 70% of those patients. We’re well under the way of transferring those patients over to Pomal. Also have recent approvals in Sweden where we have first line. In Japan, we just launched, a very interesting dynamic there. Nearly every single patient in Japan as we enter that market has already been on Nexviazyme for two years. It is a very interesting one in terms of a built up patient demand of patients looking for a new alternative. That will be a great geography, both from an opportunity as well as to learn from. As I mentioned, we see a really strong runway here going throughout the rest of the year. Very confident in our guidance there as well.

We continue to manage expenses and cash and we feel good from that aspect as well.

Yeah, this is really good. Just help us understand, you know, what are some key factors influencing the low and high end of the guidance.

Yeah, probably the biggest factor there would be just the rate of the new patient ads in the U.S. for Pomal. That is really the one that’s probably the hardest to sort of really predict. It is one that has, in terms of pricing, the biggest impact. We feel, as I mentioned, really confident in meeting the guidance and really that probably is the biggest dynamic that moves us up or down. Coming off of the strong Q2, Q3, we feel really good about the rest of the year.

Great, great. Let’s start with some of the programs here. First on Galafold. Diagnosis increasing seems to be an important driver. How are you working to increase patient identification and diagnosis at this time?

Yeah, as I mentioned, with the newborn screening that is seeding a lot of index patients within families that haven’t been diagnosed before. One thing that we’re looking to do is to make sure that physicians, families have the resources they need to do that. Family screening, cascade screening, it’s a big opportunity. Another one is as more and more patients and mutations have been identified, there’s a growing pool of people with what are called variants of unknown significance. These are Fabry mutations that it’s not clear whether they may or may not develop symptoms and need treatment. What we’re starting to see is more data is available that it does seem like a segment of those patients do need treatment. Trying to help physicians understand why, how to find those patients.

When you actually look at that opportunity of some of these common VUS, and even if you just assume 10-15% are in need of treatment, that pool of patients is almost as big as the pool of all the other amenable mutation patients combined. That is another really important initiative for us, is to help physicians and patients understand how to figure out whether you need to treat a VUS patient and when you should treat those patients.

Got it, got it. What do you think is the ultimate true size of Fabry patient population in the market?

Yeah, so it’s amazing to see the change in that market over the years. When we launched back in 2016, it was about 5,000 diagnosed treated patients and 5,000 diagnosed untreated. As you fast forward today, it’s now that 5,000 treated is now 12,000 treated, and the 5,000 diagnosed untreated is now 6,000 diagnosed untreated. So we’ve seen tremendous growth. Still a big pool of people that have been diagnosed but haven’t yet started treatment. When you look at the numbers from newborn screening, from at risk population screening, it suggests there could be 100,000 plus of those patients still. So while it’s amazing to see that we’ve now gotten up to almost 20,000, there’s still a long way to go. Got it.

There’s bigger opportunity there. Where does Galafold go from here? What are you working on and what is the cadence of growth we should be expecting?

Yeah, so as I mentioned, you know, one focus is on getting the treated amenable from the 65% to the 85-90%. We’re continuing to add some countries, but those are smaller parts of the opportunity at this point. It’s really just trying to invest in the diagnostics and making sure we can start to find those patients, treat the patients that should be treated, help to justify that and then, you know, we might touch on it. Looking at the runway for Galafold, of course we are excited where we are with the IP in the US and some of the litigations. We see, you know, a 10 plus year runway here for Galafold. We’re really excited by that.

Got it. Okay, great. Let’s switch now to Pompe launch. What are the major puts and takes you’ve seen so far in the launch as you expand globally?

Yeah. So, you know, adding countries was key for us this year. As I mentioned, we had a target of adding 10 countries. We’ve already added eight. Some of those have been very great wins for us like the Netherlands and Sweden where we got first line. Really just trying to continue to see that geographic expansion, continuing to focus on the US and that acceleration of patients that have been on Nexviazyme for two plus years. A big focus for what Amicus is investing in is continuing to look at the real world data. We’ve just started to see, you know, our four year data from our PROPEL extension that showed those improvements in motor function and muscle strength observed in the first year, been maintained for four years. Showing that improvement is possible with Pomal, especially on motor function.

Seeing some of the real world data coming from third parties. A great presentation. ICIM by an independent group compared how patients did when they went from Lumazyme to Pomal or Lumazyme to Nexviazyme and that looked like again motor function was improved for the patients going to Pomal which was very different. We are excited by that emerging data that continues to support what we believe is a great product offering with Pomal. Really now just looking towards continuing geographic expansion, continuing to execute in the US and you know, from an IP perspective that is we are early in the launch and we have a long runway there, so really just an opportunity to keep executing.

Yeah. On that front, what do you think are the sort of relative contribution of ex U.S. markets to your estimation of the Pomal global opportunity?

Yeah, so ex-U.S. currently is in the 55-60% range. I believe probably that’ll get, you know, a little bit more slanted towards the U.S. as we go. But the U.S. in terms of patient numbers and pricing will always be the dominant country for Pomal. Ex-U.S. is a really important driver of growth for us, even with the lower price just in terms of volume of patients and new countries that we’re adding. I also think as you look at some of those markets, it does give you a good sense of where we think we can go. In the U.K., that’s the country we’ve been on the market the longest. We actually have been on for three plus years in the U.K. given some of the expanded access programs.

We’re excited to see we’ve been able to get to market shares of 40%+ in the U.K. already and that gives us sort of a feel for where we hope to end up with some of the other countries like the U.S. and others as we get there over time.

Tell us more about the pricing headwinds you’re seeing so far. You know, is this a problem?

Yeah. So in terms of pricing headwinds for the U.S. we haven’t really seen anything. I mean with Fabry and Pompe, we have products, you know, that there’s already existing products and we have a pricing philosophy where we price for access and at a discount to other products or slight discount to other products that we really focus on that access. That has worked out really well for us and we commit to not raising prices more than CPI. I think that pricing approach has served us really well in the U.S. and internationally. While there are a lot of pricing pressures, I would say in the rare disease space at least we’ve been able to keep prices at a pretty good place.

Ex U.S. haven’t seen significant erosion and obviously Pomal, we’re still sort of in that launch phase, but we’re starting to see as you have that mix of European countries, high and low, we’re starting to see that that price is sort of stabilizing.

Gotcha. Okay, great. What kind of dynamics have you been seeing for Pomal switches from other therapies?

Really from early launch we’ve seen that PAM up in a given country really sort of draws proportionally from the underlying patient population. For example, in the U.S. now where 70% of the patients on the Sanofi product are on Nexviazyme and the others are on Lumazyme, we’re primarily seeing Nexviazyme switches in the U.S. In other countries where it’s more of a Lumazyme population, we’re seeing more Lumazyme patients. I will add that outside of the U.S. where we actually have a broader label in terms of naive patients being included, we actually saw doubling of naive patients this year versus last year and we continue to win a really fair share of those naive patients, which is an exciting opportunity for us as well.

Got it. What should we expect over the rest of 2025 and into 2026 for the cadence of Pompe revenues from here?

Yeah, I mean for the rest of the year with the two months left, as I mentioned, we feel really good about in terms of where we are from our guidance based on the momentum from Q2 to Q3 that we see continued to Q4. Really no surprises as we go into next year. We have not provided guidance yet for next year, but we feel that really getting as many patients on drug as possible this year obviously sets us up for a great 2026 and a lot of these countries that we have recently launched into and all the patients we have added, while they have not necessarily shown a big impact this year, really set us up for that growth next year.

Got it. Okay, great. Jeff, you mentioned about the IP. You could just tell them more about the IP for Galafold and then you know what are some of the recent settlements, you know for IP in Pomal.

Yeah. This is an area where we get a fair amount of questions, you know. As I mentioned with Galafold, we have a long runway. Our patent protection goes into the 2040s. We have over 40 Orange Book listed patents. We did have three generic filers last year and the filers were Teva, Lupin, and Aurobindo. We did settle last year with Teva for a January 2037 date. Lupin took a stay, which means that they’re bound to the outcome of the trial. In Aurobindo, we actually went all the way to trial. We just had that trial in early September. I think the way we’ve characterized that is we felt very confident going into the trial and we feel more confident coming out of the trial. We think we’re in a great position.

That being said, next steps would be kind of there’s the post trial briefings that go back and forth and that will resolve in December. Then just based on other trials we would expect it would be about a four or five month sort of time for the judge to review things. We’ve guided towards second half next year for when we’ll see the outcome of the trial.

Got it. Okay, great. What can you say about the impact of MFN and potential tariffs on the commercial business?

Yeah, I mean starting with tariffs. Depending where they end up, I guess the good news for us is for 2025 really a negligible impact. Galafold to start has a pretty low cost of goods overall. Not a whole big impact from tariffs wherever they end up. We manufacture Galafold in Europe. Pompe would be a bigger impact of tariffs this year. We’ve already expensed most of our inventory so really does not get impacted by tariffs. Next year probably would be the year where there’s the biggest impact. We’re well underway of moving manufacturing from Wuxi China to Wuxi Ireland. We do expect currently net net there will be lower tariffs in Ireland and that was part of the reason we had initiated that move as well as just geopolitical security. That transfer is underway. We have approval in Europe.

We’re looking for approval from FDA early next year. Throughout next year we’ll start to transition supply from China to Ireland, and then by 2027 it will be primarily Ireland. There would be some impact on tariffs next year, mainly driven by probability, but very manageable within the numbers in terms of financial metrics that we have. Longer term we see that as a pretty moderate and pretty mitigated impact, frankly. MSN, little harder to get a read on, I would say. We do have our former CEO John Crowley involved as the chair of BIO. CEO of BIO. Our CEO Brad Campbell is very involved in a lot of the ongoings with the agency and with the government, with the administration.

Our current view is that if something does happen out of MFN that there might be a rare disease carve out like we see in other sort of proposals and policies. If that’s the case, there could be very muted impacts of MFN on Galafold or Pombiliti. If there is an impact, most likely it would be on the Medicaid population and that currently is about 5% of Amicus sales in the Medicaid population. There would be some impact of that percentage of sales but again fairly manageable if that were to happen. Again we think a carve out for rare is probably more likely.

Got it. Let’s talk about the DMX200, maybe just a high level perspective. Jeff, can you talk about why to bring DMX200 to bring it in and FSGS?

Yeah. So we’re really excited at the collaboration we have now with Dimerix and having US rights to DMX200. Actually just got back from ASN and came here directly and there’s just a lot of buzz in the rare kidney space with some of the recent launches in IgAN and other diseases as well as I think what everyone views as a much clearer path towards approvals for these drugs with the FDA being open to proteinuria as a primary endpoint for approval. With Dimerix it was an opportunity for us to get into rare kidney. It is adjacent to Fabry, that is, you know, a nephrologist is part of the treaters in Fabry. It fit from a kind of call point perspective. There’s a huge unmet need, currently no approved treatments, much more clarity around FDA pathway.

As I mentioned, this is a very unique mechanism of action with DMX-200. It targets the monocyte-driven macrophage damage that happens in damaged tissues like the kidney and diseases like FSGS. We were very excited by the mechanism, by the unmet need. The U.S. alone is 40,000 plus patients. We see that as potentially, even with modest market shares, a blockbuster potential product that addresses a significant unmet need. All that on a backdrop that we were able to fit it in financially to our strategy, which is to take the cash that we’re now generating from our core businesses and invest that into value-generating new programs or opportunities.

This was one where for $30 million up front, with no additional payments until positive phase three data, it was something we could really fund ourselves and we think really provides access to a really exciting new medicine in an area of high unmet need that has a lot of opportunity to benefit all the stakeholders.

Yeah, you mentioned about 3,000 patients. Could you just tell us, give us a little more detail around the market for FSGS?

Yeah. FSGS, it’s 40,000 estimated patients. It’s a rare kidney disease, multiple underlying causes. There’s primary FSGS that seems to be driven by some sort of circulating factor that damages the kidney. There’s APOL1 FSGS that kind of puts the kidney at more susceptible risk of damage. Ultimately all FSGS is kind of caused by damage to podocytes or the kidney. That damage results in inflammation. It results in other parts of the kidney having to work harder and having more hemodynamic stress that leads to more damage, more inflammation, more stress. It’s this kind of underlying pathological loop that these patients get stuck in. What we’re really looking to address with DMX-200 is to break that monocyte macrophage driven inflammation in those patients. There is another product that is from Travere, Felspari, which is approved in IgAN.

They have a PDUFA date in mid January, which would be an approval based off of their data set, which is proteinuria supported by GFR. Very aligned with where we are with FDA in terms of what we’ve heard and our plans are for our program. We view it FSGS based ultimately as a lot of different mechanisms causing the disease. I think down the line you’ll see multiple agents approved and used in different types of patients or in different combinations. Really exciting to have a chance to address that monocyte macrophage driven inflammation that really is a key part of many diseases and not really addressed well by current therapies.

With Travere being a little ahead of you, how are you thinking about your commercial strategy or your development strategy to be competitive to them?

Yeah, and we really view it as less competitive and more synergistic with Travere in the space. As I mentioned, there’s a lot of different mechanisms of disease. I think you’ll see that with certain agents, some patients respond very well, but others don’t. You might see that those patients then respond better to a different therapy. I think just, you know, getting a first product approved, starting to get the market, you know, to better follow their patients, to sort of pave the way. In some cases, you know, with Galafold and Pomal, kind of coming in with another agent has actually been a great market entry for us, and sometimes it’s not a bad place to be to come into that market second. That being said, I think there’ll be multiple agents down the line approved with FSGS, and we don’t.

We view all the players in that space as actually kind of complementary, and none of them are really addressing that monocyte macrophage aspect that DMX-200 does.

It’s actually addressing the actual underlying disease.

You know, for one of the key drivers of the underlying disease. I would say it’s, it, whatever that original injury, it kind of leads to. I think you’ll see a place for corticosteroids are used sort of first line. Some patients respond, but most don’t. I think you see ARBs are used to help manage the blood pressure and kind of lower some of the pressure, and that helps some. I think you’ll see drugs like Felspari, where they add in the endothelial inhibition, being helpful in a certain segment of patients.

are complement inhibitors that could be helpful in some patients, and then that macrophage monocyte inflammation is going to be helpful in those patients where that’s a key issue, which we think is a significant fraction of patients, not all of them, but enough where we think it’ll add a lot of benefit and certainly a lot of opportunity commercially as well.

Do you think there’s a, you know, do you think actually in the target, maybe the most severe population, is there like a difference in terms of severity, you know, population that you’re able to target immediately?

Yeah. From a phase three population, you know, we’re going after patients that are either primary or genetic FSGS that, despite treatment with corticosteroids, ARBs, still have high proteinuria above 1.5 gram per gram. These are patients most at need, frankly. Unfortunately, that is a significant amount of the FSGS patients today. Those are really the ones that we’re targeting with DMX200. We think those patients, one of the reasons that many of them have not been able to have reductions in proteinuria with current treatments is that the monocyte macrophage inflammation is a big contributor. It is enriched for people we think will respond to DMX200. In a way, you are going after the more severe patients who, frankly, have the most need.

If the patient’s managed well, you know, with the standard of care, then they probably wouldn’t be looking at a new treatment anyway. As I said, unfortunately right now in FSGS it is a lot of patients who still have proteinuria.

Think about the development plan for DMX-200. Can you just help us understand more around thoughts around the clinical trial design, the timeline for potential readout and then commercialization?

Yeah, great question. So we’re really excited. I think we recently just announced that we’re past 90% towards enrollment of the Action 3 study. We’re on track to close enrollment out around the end of the year. There is a short period of ARB stabilization before they’re randomized to DMX-200 or placebo. Early next year we’d see the last patient randomized. It’s a two year trial, so top line data two years from then. Great progress on enrollment. Dimerix did just recently announce that we also completed analysis leveraging the Parasol group and data where we match towards the Action 3 baseline parameters. That data suggested, similar to what’s been seen from Parasol data with Felspari and FSGS more broadly, that changes in proteinuria at 1 and 2 years were very predictive of progression to end stage renal disease.

That data set is very helpful for us as we go in right now and we’re finalizing the two year endpoints with FDA as well as the plans to do a sample size re-estimation on the key endpoints. Our plan is to take all that knowledge once it’s done, once protocol’s finalized, SSRE is conducted. We see the outcome of the PDUFA in January for Felspari. We’d like to then go in and talk to FDA. Is there an opportunity based on the data to potentially do a SODA approval based on one year data? We certainly have that conversation. We have been very careful to guide to say our base case is two years. Hopefully the FDA will be approving the first treatment in FSGS on two year data coming up here in January.

That being said, the data that we’ve seen from Parasol does show that one year proteinuria is very predictive of outcomes. So we certainly want to have that conversation.

Got it. That’s helpful. Any changes in the FDA in terms of their view on the endpoints? Any tightening around the regulatory side of things?

No. So far FDA’s been responding on time, which is good to see. Even during the shutdown. So far they’ve been very responsive and so far we haven’t seen any change in sort of what they’ve said in terms of how they’ve acted. Obviously it would be very informative to see what happens here with Travere coming up in January. Other than that, so far we’ve seen FDA is conducting business on time and we haven’t seen any surprises.

That’s great to hear. Great. Okay then, last question. Just what are the major catalysts coming up for over the next 12 to 18 months that we should watch for?

Yeah, so I mean, starting on Galafold, you know, just continuing on the adding patients and the revenue growth, you know, driven by that diagnosis. I think big catalysts will be on the Galafold IP as we see resolution here in the first half next year. I think that is an important one to point out for Galafold, you know, for Pomal, again, it’s looking at how that fourth quarter looks, building off of a very positive Q2 and Q3. We expect to continue to present on real world evidence on Pomal. We’re excited about some of the upcoming abstracts at the world meeting coming up in February. For Pomal, we will also, you know, keep an eye on the pediatric programs. We do expect to have our first label expansion middle of next year for the adolescent LOPD group.

I think that is another catalyst for us then DMX200 obviously finishing enrollment when we announce that. Then also an update in the first half about potential accelerator approval conversations and then I think just underlying that continuing to execute on the financials.

Excellent. Thank you, Jeff. I think with that we can wrap up the fireside chat. Thanks, Jeff, for joining us.

Thank you, David. It’s a pleasure.

Take care everyone.

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