Amicus Therapeutics at Leerink Conference: Path to Profitability

On Monday, 10 March 2025, Amicus Therapeutics (NASDAQ: FOLD) presented at Leerink’s Global Healthcare Conference 2025. The company showcased its strategic focus on achieving profitability and expanding its rare disease portfolio. While celebrating successes like the strong launch of Palm Up and the steady performance of Galafold, Amicus also addressed challenges, including navigating regulatory landscapes and managing potential risks.

Key Takeaways

• Amicus aims for GAAP profitability in the second half of 2025.
• Palm Up is expanding in Europe, with significant growth expected in 2025.
• Galafold remains a key revenue driver, with a strong market share.
• The company is exploring partnerships and business development opportunities.
• Manufacturing strategies include second-source production in Ireland.

Financial Results

Amicus reported a robust performance in 2024, with Palm Up sales reaching $70 million, surpassing guidance expectations. The company achieved non-GAAP profitability and saw a 33% growth year-on-year. For 2025, Amicus projects a revenue increase for Palm Up between 65% and 85%, translating to $115 million to $130 million. The company is on track to achieve GAAP profitability later this year, with Galafold playing a pivotal role in this financial strategy.

Operational Updates

• Palm Up Launch:

  • Expanding into over 10 European countries with reimbursement processes underway.
  • Approved in Australia and Canada, with Japanese approval anticipated.
  • U.S. market sees potential patient switches from Nexviozyme to Palm Up.

• Galafold Diagnostics:

  • Partnering with OM1 and Penn Medicine to enhance diagnostics through AI.
  • Newborn screening initiatives in 5 U.S. states support early diagnosis.
  • Maintains a 65% market share among amenable patients globally.

• Manufacturing:

  • Second-source manufacturing in Ireland with Wuxi is progressing well.
  • Majority of 2025 inventory is secured within the U.S. to mitigate tariff risks.

Future Outlook

• Naive Patient Indication:

  • Amicus is compiling data to expand Palm Up’s label for naive patients in the U.S.

• European Market Expansion:

  • Revenue from Italy and The Netherlands expected to commence in Q2 2025.

• Business Development:

  • Focus on licensing commercial assets to leverage existing infrastructure.
  • Exploring expansion into Fabry, rare neurology, and metabolic diseases.

Q&A Highlights

• Palm Up Performance:

  • U.S. patient switch speed is a key variable in 2025 revenue guidance.
  • Successful uptake outside the U.S. alongside Lumizyme launches.

• Tariffs:

  • Tariffs are not anticipated to impact 2025 materially due to inventory strategies.

• Galafold Exclusivity:

  • Intellectual property settlement with Teva extends exclusivity to 2037.

For a detailed understanding, readers are encouraged to review the full conference call transcript.

Full transcript - Leerink’s Global Healthcare Conference 2025:

Joe Schwartz, Equity Research Team, Leerink Partners: Okay. Welcome everyone.

It’s my pleasure to lead this fireside chat with Amicus Therapeutics. I’m Joe Schwartz from the Equity Research Team at Leerink Partners. It’s my pleasure to be joined by Jeff Castelli, Chief Development Officer and Simon Harford, Chief Financial Officer. Thanks so much for being with us and giving us an update.

Simon Harford, Chief Financial Officer, Amicus Therapeutics: Pleasure. Thank you, Joe.

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Great to be here. Thanks to Lyric and it’s great to be back in Miami for another investor meeting. Yeah.

Joe Schwartz, Equity Research Team, Leerink Partners: So why don’t we start by having you give us a quick level set of, the company’s latest progress and key goals for the year, Jeff?

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Yes. So, obviously, this year is continuing to execute on our commercial launches for Palm Up. We’re really excited about the additional launch countries this year. We’re launching in 10 or so different European countries that were either have secured reimbursement or in the process securing reimbursement. And continuing to see the launch here for Palm Up in The U.

S. As it evolves. Galafold continues to be a great product and success story for us and we continue to see a lot of promise there. Now what we believe is a very long runway, lots of positive tailwinds for diagnostics to help grow patients. And really on that top line growth of those two programs and continuing to execute there commercially and medically, managing the bottom line, continuing to leverage that towards growing profits and then becoming actually non or full GAAP profitable second half this year.

So really it’s a commercial financial discipline story. I know as a Chief Development Officer, I’m also very interested in our pipeline and looking at potential business development opportunities to grow the business. And that is ultimately where Amicus sees ourselves as taking everything we’re doing to actually start to generate money that we can now reinvest in the business, leveraging everything we have commercially, everything we have medically, even our clinical reg expertise. We view ourselves as a great partner or our own development company again for some of our early assets as we look to ramp some of the backup that we’ve sort of strategically kind of over the last few years, we’ve really focused more on growing the top line, minimizing the bottom line. And now we’re really looking at this as a great kind of in some ways, a clean sheet of how do we invest, how we want to and how do we leverage what we have moving forward.

So a lot of excitement, for sort of the stormy seas we see a little bit out there in biotech. We feel like we’re in a really, really great place in a pretty unique space.

Joe Schwartz, Equity Research Team, Leerink Partners: Okay, great. So where are we in the Palm Op launch in Europe and The U. S? And before we start talking about all these additional markets, which I think might begin to kick in this year. This isn’t your first launch.

And as you alluded, Jeff, Galafold has got some nice dynamics to support further growth there. But sticking with Palm Op first, what phase of the launch are we in, in Europe and The U. S? How do you think about where we are in the overall launch curve?

Simon Harford, Chief Financial Officer, Amicus Therapeutics: Yes. Maybe I can comment on that. I think where we are in the launch is we still have a ways to go because we’ve clearly launched in certain key markets. Germany, The U. K, Spain has been off to an incredible launch, but we still have Italy where we recently got reimbursement sort of coming online step by step this year as we get on hospital formularies across the regions.

We have The Netherlands where we recently got reimbursement, which is an important market for us because it was the sort of the founding country of Pompe disease, so to speak. And then there are, yes, the others that you sort of referenced that will come during this year. We recently got approval also in Australia, Canada and Japan, we expect later this year. So there’s a journey that we are on to add patients in the international markets. I think as far as The U.

S. Launch is concerned, I mean, obviously, as a one market focus, yes, we’re progressing. And what we saw in Q4 was actually an acceleration in prescriptions compared to Q3. It was a little bit sort of bumpy in 2024 in terms of quarter on quarter patient numbers, primarily because of two reasons. One, you in the first half of the year particularly, you had quite a lot of clinical trial patients ready to transfer to commercial drug.

And secondly, obviously, the timing of certain launches in new countries. But we are pretty pleased with where we exited last year, 33% growth year on year as a company. And obviously, yes, PONDBIOLITY OpFolda was a key contributor to that in addition to Garifolda.

Joe Schwartz, Equity Research Team, Leerink Partners: And how has the overall reception to the products label been, particularly in The U. S, now that you’re in the second year of launch, you’ve got this unique statement or indication statement on the label, which implies that you can help patients progress or improve rather. If they’re not improving, then they should be considered as a candidate for PAW MOP. How is that reception to that aspect of the product profile going for the company?

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Yes. Thanks for the question, Joe. And just as a quick reminder, outside of The U. S. So far, we’ve seen that we have a broad label for both naive patients, experienced ERT patients.

In The U. S, we do have this somewhat unique label of for patients not improving on their current ERT. Not unexpectedly where we’ve really seen the initial uptake has been in patients that were not doing well on their current ERT and that clearly falls into a not improving. I think the real opportunity moving forward for us is continuing to remind folks of what we saw in trials where more than half the patients after switching actually showed improvements and in cases meaningful improvements. So in some cases opportunity to regain function and I think that is an aspect that is somewhat appreciated but something we’re really trying to continue to remind folks of that where stability is not necessarily the desired outcome here.

You actually can potentially offer your patients an opportunity to regain function. So I think we’re looking at that as an opportunity continuing obviously now that over forty percent of patients here in The U. S. Have now been on Nexviozyme for two years, which is the new product from Sanofi that launched a year to two years before us. And we know that that now is a point where those patients are starting to be assessed of how are they doing on Nexvionzyme if they’re progressing.

We know they’re starting to come over to Palm Op. So that’s certainly something that will continue to progress. And then obviously the thirty percent of Lumizyme patients, we can go in and keep reminding those doctors and those patients of the data that we saw.

Joe Schwartz, Equity Research Team, Leerink Partners: Great. And how large is this early adopter group, do you think?

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Yes. It’s early adopter is an interesting term here for this market. In some way that was those Lumizyme patients that had been on Lumizyme for years. And then when next Biozyme came out, they all quickly switched to next Biozyme. Sanofi was out there switching patients from their first gen product to their second gen product but no competitor.

And not surprisingly, they got 60% of patients to switch over. So in some way that was early adopters. Now for us, it’s really just a continued growth of those next Viozyme patients as now they’ve been on long enough to now be considering a new switch and those Lumizyme patients that for whatever reason didn’t switch and we think we have a great proposition to offer those patients. So it’s really never was a great early adopter pool, I guess, out there for us, particularly in The U. S.

Given the dynamics. I will say outside of The U. S. Where we are launching more contemporaneously with Lumizyme, we’re seeing really great uptake. And in that case, I think there are, sites and patients that have sort of been waiting for the next best ERT to come along.

And that’s an area where we’re doing really well and seeing great uptake.

Joe Schwartz, Equity Research Team, Leerink Partners: And how do conversations vary, depending on the treatment center or the physicians that are at these centers? Do any, is there a lot of variance between like the level of appreciation and the need to be thinking about whether a patient can do better on a new treatment option?

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Yeah. It’s a little, you know, some of the bigger centers that have lots of patients that are more academic in nature, many of them were in our studies, for example. Those sites tend to be aware of the different products and some of the data. Interestingly though, they put all their patients on either palm up through trials or on the next. It’s actually some of those second tier sites that weren’t as quick to adopt next Biozyme where there’s more opportunity for us to find those Lumazyne patients.

So overall, it’s really one message of the theme of the data that we have from our trials. The mechanism that we believe is very differentiated and our emerging long term data. So it’s the same kind of message out there, but it is a little different in terms of some sites were much more quick to switch all their patients to next or to POM UP and other sites have been sort of waiting on the sidelines.

Joe Schwartz, Equity Research Team, Leerink Partners: Okay. And, since, PumUp sales ended up coming within the higher end of the upwardly revised guidance, can you talk about any of the specific dynamics that ended up being better than you expected last year? And how should we think about how, what kinds of things you consider when you establish your guidance such as for

Simon Harford, Chief Financial Officer, Amicus Therapeutics: this year? Yes. So Joe, I mean, clearly, we were happy that in 2024, we ended up with 70,000,000 worth of vulnerability of folder sales, as you say, at the actually sort of over the top end of the guidance. A lot of that, frankly, is related to just underlying performance of speed of, frankly, patients being slightly ahead of what we were expecting at the time we set the guidance. I think as you think about 2025, really when we set guidance, we do it based on the trends that we see of what we think is reasonable.

The components though that really build towards that year on year growth are really threefold. One is obviously the rate at which we switch patients in The U. S. From Nexviozyme and Lumozyme to PONBILETI OpFOLTA. Second component is the sort of the trends in those markets where we were already launched last year, particularly the bigger ones like the Germanys, the Spains, the U.

K. Etcetera, and their continued growth. And then the third component, which is a smaller component when it comes to absolute dollar revenue, but is important for number of patients as we continue to grow into 2026, is those markets where you’ve got pricing and reimbursement during the year. What I would say to you is probably the biggest variable because we guided to 65% to 85% growth this year, which equates to roughly $115,000,000 to $130,000,000 The biggest variable between the top and the bottom end of the range is realistically the speed at which we switch patients in The U. S.

Why? Because if you take a patient, for example, that switches in March, that’s a different dollar number than someone who switches in September.

Joe Schwartz, Equity Research Team, Leerink Partners: Makes sense. And what about the naive patient population? Is there an opportunity to expand PAM UP’s indication into that setting? What’s the latest on that front?

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Yes. So outside of The U. S. In the countries where we’re launched and we have that broader label, we are seeing good uptake into naive patients. We’re compiling that data and as part of our registries, we’ve continued to collect our long term data from our trials in naive patients and in all of our patients, frankly.

So with all of that data, there certainly are opportunities to go back and interact with FDA to try to expand the label for naive. It’s sort of a matter of how much data and when, but it’s certainly something that we’re looking at, actively and is in our plans to do, but a little early to give you specific timelines. But we think that that, while not a huge opportunity per se every year, maybe there’s forty naive patients in The U. S. Going on to treatment and ultimately they do become switch patients.

But it’s not insignificant to have an opportunity to treat those patients earlier in The U. S. And I think it also has somewhat of a halo effect of not necessarily then having PAMA be viewed as sort of the, you know, really the drug that we’re keeping there for any patient who finally declines, but really looking at it to move it up earlier in the process, which is where we believe it should be is what we believe is really the most efficacious product in our minds.

Joe Schwartz, Equity Research Team, Leerink Partners: Right. That makes sense. So in Europe, you’ve where you’ve got the, the naive label, you’re going to be adding some new markets, like you alluded to. And can you talk a little bit about the timing of those new opportunities?

Simon Harford, Chief Financial Officer, Amicus Therapeutics: Yes. So really, as it relates to 2025, so I think we said at earnings time, we sort of have reimbursement now in Italy, The Netherlands as to key markets, Sweden and a number of others. The rest will come sort of throughout the year. And I would say the way to think about it is for those handful that we already have reimbursement for, you should probably start to see revenue flow through starting in the second quarter. For those that are later in the year, they’re probably more relevant to numbers of patients to get on drug for 2026 realistically.

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Okay. And all that outside of Europe where a lot of that is we have approvals, it’s more going through the reimbursement processes and approvals. We also just did recently get approval in Australia, which was a full review of both products. We’re making great progress in Canada. We expect an approval here in the first half in Japan in the second half.

It does take time on the reimbursement side for those. And actually, we will launch first in Japan, even though that is the one that kind of comes in the second half of this year because reimbursement is much faster there. And we look at Canada and Australia really as key drivers of growth for next year.

Joe Schwartz, Equity Research Team, Leerink Partners: Okay. And then, I need to ask you about the potential impact of any tariffs given you manufacture the product in China currently, although you’re working on second source manufacturing. It sounds like you’re making good progress on that front. So can you just give us your thoughts on those items? So

Simon Harford, Chief Financial Officer, Amicus Therapeutics: gosh, changes every day, so it was slightly difficult. What I can say for 2025 is the vast majority of the inventory related to product sales for The U. S. For 2025 is already in The United States. Therefore, it really doesn’t have any material impact as far as this year is concerned, at least as we see the tariff situation today.

2026 onwards, obviously, we’ll have to wait and see where we are given the variability in this discussion at the moment. But yes, we’re not anticipating anything that would sort of change course or anything from that perspective. But yes, it’s still a bit premature to comment.

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Makes sense. And in terms of our second source manufacturing in Ireland with Wuxi, we’re on track to have product from that site actually going into the commercial pipeline here in the end of this year and in Europe First.

Joe Schwartz, Equity Research Team, Leerink Partners: Great. Okay. Well, let’s shift gears a little bit. And I’d love to hear your thoughts on the company’s relatively large presence at the World Symposium lately, both on PAMAAP and then maybe this provides

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: a good segue to talk about Galafold. Galafold. Yes. So, as usual, we had a great presence at World. I think we had somewhere close to 20 abstracts, I believe, this year, across PAMAAP and Galafold and some other just Fabry Pompe disease areas broadly.

We had lots of great interactions with physicians as we normally do through our team being there through one on one conversations, through sort of ad hoc advisory boards for various work we’re doing through our symposia that we offered. From a new data perspective, we continue to focus on insights coming out of our clinical work that we did, in particular showing actually the improvements that were observed and characterizing those improvements in patients that switched from long term Lumizyme over to palm up. I think that data was very well received that we presented there showing that half of patients had a met a threshold for a meaningful improvement in either six minute walker or force level capacity in those trials. Ongoing mechanism of action data on the differentiation there and then some of our long term data, we’ll continue to see that data coming out throughout this year too on our durability of effect. I think we’ll also be from a data perspective some of the data that will be impactful.

Last thing I will add is we also continue to hear from physicians, positive anecdotal real world reports of how patients are doing. And I think that that you’ll see this year, there will be more and more independent reports coming out from sites that have significant pools of patients on the three products. And we’re hearing that physicians are interested in actually starting to report some of those kind of indirect longitudinal comparisons among products, which we think could bode well for us coming this year as well.

Joe Schwartz, Equity Research Team, Leerink Partners: Excellent. So Galafold, also had there were some interesting initiatives that, you know, are underway with Galafold. You alluded to diagnostic enhancements earlier on in the discussion. Can we switch gears and talk about where we are in terms of the launch phase for that product?

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Yes, I’ll start and Simon please chip in. So Galafold, remember we first got approved in Europe in 2016 and The U. S. In 2018. And it’s amazing just in the last two years the growth we’ve seen and really growth driven by new patient adds and improving diagnostics and from our mature markets.

We’ve seen 18% plus year over year growth the last two years. And if we look back when we launched it was maybe five thousand diagnosed treated patients in February across products and five thousand diagnosed untreated. I think last year that was over twelve thousand diagnosed treated up from the five And actually the pool of diagnosed untreated had grown from five thousand back then to six thousand. So we’re just seeing a lot of growth. And when you look at all the numbers, from newborn screening, from at risk screening, there still are probably five to ten times more people living with Fabry that don’t even know it and are not diagnosed versus those that are diagnosed.

So just a continued opportunity to find those patients and then when you find the patients offer screening for the whole family and find three to five family members on average, you know, with being an excellent linked dominant disease. So we’re investing in things as we can and AI and medical record screening. We’ve talked about the project with OM1 and Penn that we’re excited to start to see the readout. We’ve taken over 500,000 medical records from the Penn system and ranked them from one to 500,000 on risk for Fabry disease. And now we’re offering the top 100 at risk to be worked up to see if they have Fabry.

And we’ll start to see some readouts from that later this year. So just a lot of opportunities to continue to help make a difference there in the diagnostics. And in The U. S, what’s great is there’s five states doing newborn screening. And as you find the newborn in Missouri, you know, with good family screening, you can find family members that live all across the country.

So I think that is part of the growth that we’re seeing here in The U. S. Is also the seeding from newborn screening of finding infants and then they might not need treatment, but you can find the whole family through that process.

Simon Harford, Chief Financial Officer, Amicus Therapeutics: Well, and I think then you add on to that, the what, about 65% market share in amenable patients globally, yet in major markets where it’s sort of well over 80%, which says there is still growth opportunity there. I think you then combine that with the intellectual property settlement with TAVR means essentially out to 02/1937. We’ve got another twelve years of exclusivity. Obviously, we’re still working through the follow on Ourobindo discussion, but I think the Teva settlement was a good signal of how we think about things. So that creates a certain level of additional certainty.

I think the last thing I would add is particularly with Galafold, now that we’ve built the infrastructure, we’re not really adding expense related to Galafold. So that is actually the biggest single contributor to leveraging the P and L in the nearer term. PONBILITY Op FoldUp, we’re still in the launch phase. We will start to leverage that too as we get slightly further out. But the yes, that’s a real important contributor not only to revenue but also to the profitability.

Joe Schwartz, Equity Research Team, Leerink Partners: Speaking of profitability, it’s great to hear that you’re on track to have positive GAAP income this year. How do you view the company’s overall runway and path to sustainability? That’s a term that we’ve heard management use for quite a while. And so what’s the latest on that front?

Simon Harford, Chief Financial Officer, Amicus Therapeutics: So I think we’ve gone in that direction since Bradley became CEO step by step to turn, first of all, fully non GAAP profitable is what we guided to in 2024, and we achieved that this year. As you correctly say, we’ve sort of guided to becoming GAAP profitable during the second half of the year, and that’s really the next step to the pivot to sort of long term profitability. The third step eventually will then be free cash flow positive. And so I think this year is a real pivot year. And why is that profitability important?

That profitability is important primarily actually to be able to reinvest back and grow the business beyond just PONDBILITY, Opdivolder and Galafold for

Joe Schwartz, Equity Research Team, Leerink Partners: the long term. And on that front, how does the company think about BD now? You’ve had a broader pipeline in the past, some of which was developed in house, some of which was brought in externally. So when’s the right time for the company to be revisiting that kind of a strategy? And what kind of a shape do you think that would take now?

So the word I

Simon Harford, Chief Financial Officer, Amicus Therapeutics: would use for business development is thoughtful approach. What do I mean by that? What I mean by that is I think our desire is sort of, first of all, potentially to license in sort of commercial type assets where you can leverage the infrastructure, whether that be on a global basis or potentially more likely on an international basis given our broad infrastructure across sort of 40 odd markets remains to be seen. But that’s sort of the thought process because that leverage is you can typically find assets at more reasonable prices, which for us for the nearer term does play into the calculus, so to speak. But then beyond that, as the free cash flow and profitability starts to ramp up, obviously, then we will look to build on top of that to kind of start addressing the question of for the next decade what’s next discussion.

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: And I would just add, as we’re looking to sustainably leverage what we have in a thoughtful way, as Simon notes, the areas we’re looking to do that would Fabry continues to be an area of interest for us for the non amenable patients in particular. We had some internal programs that we’ve been slowly trying to optimize and we’re also keeping an eye on everything that’s out there in that space. But there’s a lot of adjacencies for Fabry and Pompe in a rare neurology, rare metabolic, rare neuromuscular. So lots of areas where we could highly leverage what we have. And also there’s the infrastructure where we could leverage that and even go into slightly new space and rare.

So we can really we’re always going to be focused on rare, but we’re really looking for ways to unlock the value that we have already in our company through our global commercial medical, commercial team, our late stage rare clinical regulatory expertise. So we think there’s a lot of ways we can take that and sort of have value add in the next area where Amicus wants to really put our time and energy to make a difference.

Joe Schwartz, Equity Research Team, Leerink Partners: That makes sense. Anything that we didn’t touch on that, we should make sure that we note before we close? I don’t think so.

Jeff Castelli, Chief Development Officer, Amicus Therapeutics: I think we covered a lot, Joe. You did a pretty good job on your questions.

Joe Schwartz, Equity Research Team, Leerink Partners: Well, there’s definitely a lot to discuss. Keep up the great work.

Simon Harford, Chief Financial Officer, Amicus Therapeutics: Thank you. Thank you. Appreciate it. Thank you, everyone.

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