Amicus at Jefferies Conference: Strategic Growth and Profitability

On Wednesday, 04 June 2025, Amicus Therapeutics (NASDAQ:FOLD) presented at the Jefferies Global Healthcare Conference 2025, outlining its strategic initiatives and financial outlook. The company is navigating a path toward profitability while expanding its market presence. Key topics included Galafold’s growth, Pombility’s market dynamics, and a strategic acquisition in nephrology.

Key Takeaways

• Amicus aims for GAAP profitability in the second half of 2025.
• Galafold’s patient growth is driven by improved diagnosis and early treatment.
• Pombility’s revenue growth is projected between 50% and 65% this year.
• Acquisition of Dimerix asset for FSGS aligns with strategic growth in nephrology.
• AI-driven diagnostics are crucial for capturing the undiagnosed Fabry market.

Financial Results

• Galafold:

- Achieved mid-teen patient growth with a strong global market share.

- Q1 patient growth was 14%, with a full-year revenue growth forecast of 10-15%.

- Settlement with Teva extends IP protection until 2037.

• Pombility (OpFolder):

- Generated $70 million in revenue last year.

- Revised full-year revenue growth guidance to $105-115 million.

- Growth driven by patient uptake in the US, Netherlands, and Italy.

• Overall Financials:

- Achieved non-GAAP profitability in 2024.

- GAAP profitability anticipated in the latter half of 2025.

Operational Updates

• Galafold:

- Focus on diagnostic initiatives with AI collaboration screening 500,000 records.

- Significant opportunity in the undiagnosed Fabry patient population.

• Pombility:

- Monitoring switching patterns from Nexviazyme, expecting potential shifts after two years.

- Positive feedback from both patients and physicians.

• FSGS (Dimerix Asset):

- $30 million upfront payment for Dimerix rights; potential US blockbuster.

- Additional $75 million in developmental milestones to approval.

- Phase 3 study underway with proteinuria as the primary endpoint.

Future Outlook

• Galafold:

- Plans to expand market through enhanced diagnosis and early treatment.

- Confident in sustaining a 10-15% growth rate.

• Pombility:

- Aims to capture majority market share by addressing switching barriers from Nexviazyme.

• Business Development:

- Seeking commercial assets in rare diseases to leverage existing infrastructure.

- Exploring opportunities outside the US, focusing on late-stage clinical assets.

Q&A Highlights

• Galafold:

- Investors may not fully appreciate the growth potential and IP protection.

- Diagnostic opportunities are significant, with many patients undiagnosed.

• Pombility:

- Switching from Nexviazyme may be slower due to established habits.

- Reimbursement issues are not a concern.

• FSGS:

- Excitement about Phase 3 data and its potential impact.

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

Full transcript - Jefferies Global Healthcare Conference 2025:

Dennis Ding, Biotech Analyst, Jefferies: Morning. Welcome to day one of the Jefferies Healthcare Conference. My name is Dennis Ding, biotech analyst here. I have the great pleasure of having Amicus Therapeutics here with us. Welcome.

Welcome. Thank you. So maybe to kick start things, talk about Amicus and kind of the journey you guys have been on over the last one or two years to get to where you are today. And maybe also give us an update as to where you are today in terms of the base business and how you think about the outlook later this year.

Simon, CFO, Amicus Therapeutics: Yes. So maybe I’ll kick off and then Jeff can comment. So really, over the last couple of years, since Bradley Campbell has been CEO, the goal has really been to do two things. One is obviously to continue to grow strongly the Galafold and Fabry franchise. And I think really the journey of the last couple of years has really shown with the patient growth that we’ve continued to see for Galafold in sort of mid teen growth rates now in the third going into the third year of that really demonstrates the sustainability of Galafold’s growth going forward, and it’s all coming from volume.

And we see that patient growth continuing going forward. I think secondly, obviously, the launch of Pombility, OpFolder, we had $70,000,000 worth of revenue last year. The first quarter of this year was growing extremely strongly year on year. We did adjust our guidance really to reflect just sort of the timing of uptake of patients within the year, but we’re seeing very positive sort of growth in prescriptions in the last couple of months. So we’re feeling pretty optimistic about the outlook.

Really, the goal was to grow the revenues from those two products while at the same time becoming a profitable biotech. And we’ve been on a journey really in the last couple of years to reduce losses, first to have sort of profitable full year non GAAP profitability, which we had in 2024, transitioning now step by step to GAAP profitability. And we’ve said that in the second half of this year, we expect to become GAAP profitable during the second half of the year, and we’ll obviously take it from thereon out. But I think that’s important because that also reflects improving cash flow over time, too. And our view is we want to grow profitability and cash flow while also doing thoughtful either tuck in or in licensing type acquisitions.

And I think Demerix, the recent deal that we announced in FSGS, clearly demonstrates that thoughtful approach to business development, something with a potential blockbuster status in The U. S. But actually, we’ve only paid $30,000,000 upfront, almost like a call option, to flip the card on that. So overall, it’s as we go forward to the future, continue obviously to expand the current portfolio but to step by step sort of leverage our infrastructure through business development in a thoughtful way that’s balanced with not sort of overstretching the balance sheet in order to move forward.

Jeff, Amicus Therapeutics: Yes. Thank you, Simon. Just to quickly add, so good morning, everyone, and thanks to Jefferies for having us. So I’ve been with Amicus for quite a while. And external market dynamics aside, as Simon just hit on all the key points, really exciting time.

Had $458,000,000 in sales last year, 15% to 22% growth, turning GAAP profitable, just got rights to a potential phase three blockbuster drug for FSGS. Long runway on our current products and a lot of new growth with new patients being diagnosed. So from that perspective, we’re extremely bullish on the future and really excited. And I know right now it’s a little bit of an uncertain time just with external dynamics, but we feel we’re in a really good position moving forward.

Dennis Ding, Biotech Analyst, Jefferies: Thank you. I think that’s a great overview. Let’s focus on Galatol for a second. I feel like it deserves some airtime because it is really the, what I would call, the engine behind Amicus, right? It’s growing double digits.

There’s still a decent amount of runway there. But what do you think investors are missing here? Because when you look at the valuation of Amicus and you try to correlate that the value of Galafold. There’s clearly a disconnect there. What are your thoughts on that?

And what is your message to investors about Galafold and the outlook?

Simon, CFO, Amicus Therapeutics: So what I would say to you is I honestly think there are a couple of key components that are being missed. First and foremost, in terms of intellectual property protection, with the settlement with Teva to February, you have essentially provided certainty for investors for the next twelve years, which is honestly longer than some products even have sort of patent protection for in a growing market. Yes, we do still have to resolve the outstanding sort of Aurobindo challenge. But the reality is in the vast majority of cases, once the lead ANDA filer has sort of settled, Others fall into line. We’ll have to wait and see.

But I think that really extends the runway and the value of this product. I think secondly, it’s really important to bear in mind in the revenue growth versus profitability discussion that once you get about $400,000,000 worth of revenue coming out of Galafold as a small molecule, pretty much everything else falls to the bottom line from a profitability standpoint because we’re not building additional sort of infrastructure to support it. It’s more driving growth through market opportunities. Thirdly, I think as you look at patient growth, we are now in the third year of mid teen ish type patient growth. Even in Q1, where we had a few one offs in the revenue line, underlying patient growth was 14%.

And our guidance for this year continues to be 10% to 15% growth. So each year that goes by is another year reinforcing the opportunities. And

Dennis Ding, Biotech Analyst, Jefferies: I’ll

Simon, CFO, Amicus Therapeutics: let Jeff maybe talk about what some of opportunities are. But then you also have market share expansion. We’re roughly 65%, seventy % market share in those markets where we are globally. But in the major markets, we’re more like 85%, ninety %. So there is still good growth opportunities in those markets as well.

And so those are the things that I think are obviously, it’s our job to get that message out there, but key things that people need to understand. And maybe you can just comment on some of the things that reinforce that growth going forward.

Jeff, Amicus Therapeutics: Yes. Thank you, Simon. You know, really think to me the main thing underappreciated with Galafold, in addition to the long runway we have, is there’s just so many patients still out there that are not diagnosed. The diagnostic opportunity to grow the market is just huge. There’s more patients not diagnosed than are diagnosed.

There’s a lot of tailwinds for diagnosis. Physicians have a better understanding of when to think about Fabry. All of the kind of cardiac or renal screening panels include Fabry on those panels when it’s so easy genetic testing. And, there’s newborn screening initiatives going on, in The US. We have eight states that are screening over 500,000 newborns every year, diagnosing index Fabry babies, and then looking at those, family members.

So just a lot of tailwinds. You know, Amicus is trying to invest in certain things like AI and how to use medical records to also try to find undiagnosed patients. And really, see just a huge opportunity to continue growing this market at significant numbers just through diagnosing new patients. The other interesting thing is if you look like when we launched in 2016, there was about ten thousand Fabry patients, about five thousand treated, five thousand diagnosed untreated. Today it’s eighteen thousand, so it’s gone from ten thousand to 18,000 diagnosed patients.

And 12,000 are treated and 6,000 diagnosed untreated. So you’re seeing, instead of fifty percent of people treated in the past, now you’re seeing two thirds of patients treated. So in addition to a lot of diagnostic growth, we’re seeing physicians start to treat patients more early. Seeing more treatment of females. Huge opportunity there as well.

And the last thing I’d say that is an additional tailwind specifically for Galafold that I think is benefiting us and why you’re seeing more growth for Galafold than other Fabry treatments, is that in the pool of patients and families left to diagnose, they tend to have late onset Fabry because they haven’t been diagnosed with more classical symptoms. And a high, high majority of late onset Fabry is amenable. So a lot of that growth that’s coming is actually enriched for amenability. Whereas traditionally, was thirty five to fifty percent. It’s probably fifty percent plus on kind of the new diagnosis.

Okay.

Dennis Ding, Biotech Analyst, Jefferies: Those are very interesting and like, you know, it does seem like there’s a lot of tailwinds in terms of diagnosis and education and earlier treatment and things like that. When do you expect those sort of dynamics to impact the Galafold from a revenue perspective? Are we talking about in ten years or in five years? Just kind of ballpark, when do we when should we expect those tailwinds to actually kick in? I I

Jeff, Amicus Therapeutics: think the tailwinds have been kicked in. If you look, as Simon said, I think we’ve seen more new patient starts in Fabry in the last few years than we’ve seen historically ever since early launch for Galafold at least. So I think that’s what’s driving the current growth. You know, what we’re seeing is not any growth in price, it’s not that much market expansion although there still is some. What we’re seeing is mature markets where we already have 85, 90 percent share of amenable patients.

We’re just seeing a lot of new diagnosis and new patients going on treatment. So I think we’re seeing it and I think that’s what’s going to continue to see that growth. You know, you see sometimes people put models together and after three or four years they put sort of one or 2% growth with population like, there is just a lot of continued potential for Fabry. I think we’re seeing it now and I think it’ll continue to fuel that growth for the foreseeable future.

Dennis Ding, Biotech Analyst, Jefferies: I think that’s very interesting because, you know, when you look at your guidance, I think 10% to 15% year over year growth, so kind of in the low teens. And then when you look at consensus numbers moving forward, it’s actually a pretty steep deceleration over time. And if you’re telling me that with all these education and tailwinds and things like that, is there a scenario where Galefold actually accelerates from some of these efforts, right? Do you guys have any comments on that in terms of

Simon, CFO, Amicus Therapeutics: So all I would say is we believe in taking these things a little step by step. But now that we’re into the third year of that strong patient growth, obviously, our comfort level with the sustainability of that growth continues to build in question.

Jeff, Amicus Therapeutics: The only point I’d say is it’s always off of a bigger base of patients. So while we’re adding more patients sort of each year than the next, as a percentage, it’s gone down a little bit. And I think that’s what you’ve seen with the Galafold growth has gone down in that teen level. But it’s actually from a just sheer number of new patients being diagnosed, going on to treatment, it’s actually been growing the last few years.

Dennis Ding, Biotech Analyst, Jefferies: Yeah, mean, I think even outside of these different initiatives, just the fact that there’s still additional room for penetration, just that on its own can contribute to that year over year increase in net patient adds, right? I totally understand that, like from a percentage basis, it would naturally decline. But once some of these initiatives kick in, why wouldn’t that, like, why shouldn’t we be talking about a low teens growth rate over the next three to five years, right? And then with the, you know, with the generic settlement and visibility into 02/1937, like why shouldn’t we think about Amicus as a Galafold business rather than I think the narrative out there right now is more around PONBILITY Upholdo?

Simon, CFO, Amicus Therapeutics: Yes. I think you have a very valid point. I also think, obviously, as the CFO, so we’re investing in some of these initiatives. One wants to see the value of that being created. And I think we all believe with the programs on AI and the algorithms with UPenn, etcetera, that those are great opportunities to expand and diagnose at faster rates.

And we all know that as an X linked disorder, there will be other family patients out there as well. So I think your question is a totally valid one, and that’s what we are striving for. Obviously, will take it step by step as we progress Can you

Dennis Ding, Biotech Analyst, Jefferies: give an update on the AI initiative with UPenn?

Jeff, Amicus Therapeutics: Yes. So this is a collaboration we initially had with a company OM1 that helps basically uses AI in medical records. And we created a kind of diagnostic fingerprint of medical records for Fabry. We were doing a pilot project with UPenn where we’ve screened over 500,000 of their medical records of their patients at Penn and ordered them from one to 500,000 in terms of risk for Fabry based on the algorithm. Penn is in the process of engaging with those top hundred at risk and then moving on now actually to the second hundred at risk and seeing if they’re willing and able to come in and get worked up for Fabry and tested.

So we’re in that patient engagement, getting screened, getting tested, and we’ll have more to report out here probably in the second half of the year on how that’s going. But assuming that we can show proof of concept this is a way to effectively find Fabry patients, we would look to potentially do additional pilots at multiple healthcare systems in The U. S. And then importantly I think ex U. S.

Because in The U. S. As I mentioned, we already have newborn screening that is seeding a lot of index patients with families that could be all around The US. So I think that’s a really strong core already. But things like the medical records can add to that, but for me it really would be exciting to do outside of The US.

And we do have some similar projects in The UK and in Canada starting to look at sort of medical records and

Dennis Ding, Biotech Analyst, Jefferies: how to use those to diagnose patients. Okay. Okay. Sounds good. Now that we are in June of Q2, I’m just curious what sort of incremental color you can give around Galafold and maybe remind us Q1 and some of the seasonal ordering pattern things that happened that may have shifted into Q2.

Just remind us of some of those dynamics now to think about Q2 Galafold revenue.

Simon, CFO, Amicus Therapeutics: Yes. So in terms of Galafold, as you know, we guided to 10% to 15% growth for the full year, which is consistent with what we’ve been saying since the beginning of the year. And that’s really on the back of the strong patient growth we were just talking about. I think in terms of sort of quarterly dynamics, yes, there were a couple of things. One was this UK rebate called VPAG that was higher than anticipated.

That will continue during the year, but at least we’re now aware of what the rate is. But bottom line is we didn’t change our annual guidance. Typically, we tend to have in absolute dollar revenue, The smallest quarter is Q1 due to reinsurance, those type of things. But what I can say to you is we have the guidance we have, and we feel fairly good about where we are.

Dennis Ding, Biotech Analyst, Jefferies: Okay. Let’s talk about Pompe and Pummability, Alpulda. Can you remind us what happened in Q1 and some of the underlying patient trends? And I know you guys also talked about April or was it April or May? April.

April being a very strong month in terms of patient adds, particularly in The U. S. So I’m

Simon, CFO, Amicus Therapeutics: just curious if you saw that trend continue in May. So in terms of Q1, we did adjust our guidance to 50% to 65% growth for the full year, which is roughly $105,000,000 to $115,000,000 Why did we do that? We did that because, frankly, while we are seeing good prescriptions that you referenced in April and May, the same thing, If you don’t catch the patients right upfront in revenue terms, then they push further out. And that is what created the sort of the revision. It was primarily due to two things, patients in The U.

S. And secondly, patients in a couple of key markets, primarily The Netherlands and Italy, where we’ve got pricing and reimbursement, but we had to go through like regional formularies in Italy. And the patients in The Netherlands are part of a tender and there’s only one center. So it’s going to take time to get them all fully on to drug. That was the major reason, but we are April was, as you referenced, a very good month, and we continue to see positive trends.

Dennis Ding, Biotech Analyst, Jefferies: Okay. What are your thoughts on or do you have any updated thoughts on the whole Nexveazyme switching? And now that we’re a couple of years into the launch, because before you guys mentioned one or two years, like I’m just wondering if there’s any kind of updated metrics based on the like real world experience in terms of the switches?

Jeff, Amicus Therapeutics: Yes. And I think a lot of that is coming from The U. S. Where Sanofi launched Nexviazyme basically a year and a half right before we launched and they managed to switch a significant part of The US market. So I think what we’ve come to understand is it really is more about two years, where physicians and patients have enough time, enough meetings with their physician, which turns out to be more annually than twice a year.

And we’re just not seeing a lot of switching before two years. And it’s not that surprising necessarily. We do know that as patients switch from Lumizyme to Nexviazyme, they do generally start to do better. But we are seeing that a significant percent of those patients, especially after two years, look like they are now looking to potentially switch. So good dynamics for us on that front and we continue to sort of learn as we go along in terms of that journey.

But we continue to hear really positive feedback from patients, from physicians on the experience with palmop. So we remain very bullish on long term. We believe in the product. We believe that this will ultimately deliver differentiated mechanism efficacy. And we do think that eventually we’ll have majority market share.

It’s just sort of the pace which that happens. In particular, in countries where they’ve recently switched to a new drug and sort of they need to go through that journey with that new drug before they’re necessarily immediately looking to switch to something else. But from a numbers perspective also, you know, last year as we entered the year, maybe ten to fifteen percent of patients in The US had been on Nexvirozyme two years or longer. As we exited last year, that was like thirty or forty percent. And now this year, started the year at thirty, forty percent, we’ll end it around sixty, seventy percent in two years.

So it’s just a much bigger pool of patients Yeah. That had been on Nexviazyme long enough to kind of have a sense how they’re doing and potentially be looking then to switch to another option.

Dennis Ding, Biotech Analyst, Jefferies: Outside of time on Nexveazyme, there any other kind of barriers or considerations that doctors are thinking through that would prevent them from switching somebody onto Palm Up?

Jeff, Amicus Therapeutics: No. I mean, it’s interesting as you speak to the physicians and patients, there’s barriers you didn’t necessarily expect, like just things like, oh, they need to come in to the office for a few infusions if they’re on home infusions. Oftentimes they might have to switch nurses that are doing the infusions and sometimes that could be a barrier. There is the fasting for two hours during the infusion because of the up flow to stabilize or once every two weeks and all very minor things you would think compared to potentially getting on a drug that could actually treat your disease in a different way. So those are, in essence what we’ve learned is there needs to be sort of a compelling initiative from the patient or physician to want to switch.

It’s not just gonna naturally happen without sort of a reason. That reason can often be the patient’s not doing well or they didn’t get the benefit they hoped to get when they had switched. So I think it’s that’s been a learning but there’s no specific barrier. The reimbursement initially I think was challenging for some of the trial patients and there still is this mindset that like the the reimbursement could somehow be challenging but we’ve we’re seeing, you know, reimbursement is not an issue from the payers. So I think it’s just combating some of that mindset of like, oh, we need to go through this switch and it’s really not that hard to switch.

And I think we’ve

Dennis Ding, Biotech Analyst, Jefferies: heard great outputs of what’s happened when those patients have switched. Okay. We have five minutes left. Let’s talk a little bit about FSGS. Can you remind us some of the details of the transactions and just the thought process and rationale behind the Dimerix asset?

Simon, CFO, Amicus Therapeutics: Yes. On a big picture level, and then Jeff can talk about the details. But in terms of business development, we had said that one of the things we wanted to do was to do step by step business development as we became more profitable to build sustainability and expand the portfolio for the And I think in the case of Dimerix, it really fit the bill both from a point of view of being totally on strategy to assuming the drug is successful in Phase three, leveraging the infrastructure that we already have built in The U. S.

You combine that then with the fact that we didn’t need to finance the upfront costs of doing this. So basically, we paid $30,000,000 to it’s like a call option to flip the card on the Phase III data that if successful, we believe has blockbuster potential in The U. S. Alone. And the remaining sort of milestones in cash flow, assuming the drug is successful, which we would then be happy to be paying out on, will match much better with our sort of cash flows going forward.

So it really felt like almost perfect is too strong a word, but a very good opportunity for us that fits exactly what we were trying to achieve as it relates to FSGS.

Jeff, Amicus Therapeutics: Yes. I mean, I would just add, we’re super excited to be partnered with Dimerix, you know, FSGS. There’s no approved treatments. There’s forty thousand plus patients in The US suffering with FSGS, five thousand new each year. We think it’s a very, differentiated mechanism of action that really targets this monocyte macrophage driven inflammation that seems to be a significant driver of, the kidney damage in a good set of the patients.

We were very excited by the phase two data that we saw. That’s a really well powered phase three study. They just got clarity from FDA or we got clarity that proteinuria can serve as the primary endpoint. So a really well powered study. So as Simon said, we thought it was a perfect fit for us.

You know, we have kidney experience and interaction with nephrologists through Fabry, so we can leverage sort of our understanding and knowledge about the endpoints, our interactions with the physicians and kind of build that base to go into FSGS. But super excited now to be engaged with that community as well and we think it’s a perfect fit for us. We would love to also have something for ex US team as well. And we’ll certainly keep looking for similar opportunities. But it checks every box think we could hope for.

Dennis Ding, Biotech Analyst, Jefferies: Right. So maybe talk a little bit more about some of those other opportunities now that you have done this deal with Vimerix. What’s next? Should we look for something similar but OUS meaning more clinical stage, like late stage but clinical? Or should we think about commercial?

That would be

Simon, CFO, Amicus Therapeutics: my comment would be business development always has an element of opportunity attached to it. So you have to be a little bit cognizant of that as to exactly what you do in what order. Suffice it, however, to say the framework within which we’re sort of working is commercial asset that would continue to leverage our infrastructure, whether, frankly, outside of The U. S. Only or on a more global basis, depending on circumstances, in the rare disease space.

It doesn’t always have to be something that’s got an identical sort of sales force or overlaps with the sales force because in rare disease, that tends to be the less expensive piece. But where we can at least leverage the infrastructure on regulatory, on internal stuff. Yes, could be, again, late stage, depending on circumstances. But nothing that is going to what I would describe as overextend the balance sheet. We’re trying to be quite thoughtful about that.

We’ve just the ink is just drying on this deal. So we’ll proceed from here as we see suitable, but that’s the sort of direction of travel.

Dennis Ding, Biotech Analyst, Jefferies: Okay. And back on the FSGS. So remind us I know it’s $30,000,000 upfront, but remind us of the developmental milestones that may need to

Simon, CFO, Amicus Therapeutics: be paid out. Yes. So there are about $75,000,000 worth of milestones to be paid to the point of approval. Then beyond that, there are sales milestones and there are sort of primarily royalties on sales in the sort of the low teens to, I think, low 20s roughly.

Dennis Ding, Biotech Analyst, Jefferies: Okay. Good. We would be happy

Simon, CFO, Amicus Therapeutics: to pay out if drug is successful. Yes.

Dennis Ding, Biotech Analyst, Jefferies: Great. Well, thank you guys so much for hanging out with us. Really excited about the outlook moving forward and all the different catalysts actually that we see in the second half this year. So good luck, and have a great day.

Simon, CFO, Amicus Therapeutics: Thank you.

Jeff, Amicus Therapeutics: Thank you.

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