Figma Shares Indicated To Open $105/$110
On Wednesday, 14 May 2025, Amicus Therapeutics (NASDAQ:FOLD) presented its strategic vision at the BofA Securities 2025 Healthcare Conference. The discussion, led by Tazeen Ahmad from Bank of America, highlighted both opportunities and challenges for the company. Key topics included the growth of their flagship product Galafold, the promising launch of Pombility Opt Folder (POMOP), and the strategic acquisition of DMX-200.
Key Takeaways
- Amicus expects Galafold sales to exceed $500 million this year, driven by new patient diagnoses.
- The launch of POMOP is accelerating in the US, with a focus on converting patients from existing therapies.
- The acquisition of DMX-200 aligns with Amicus’s strategy to leverage infrastructure and maintain financial discipline.
- The company is monitoring the impact of the "most favored nation" executive order and tariffs, which are currently seen as manageable.
Financial Results
Amicus shared optimistic financial projections for 2025:
- Galafold: Expected to surpass $500 million in sales, with a 14% patient growth in the first quarter. The company targets 10% to 15% revenue growth for the year, noting that any sales beyond $400 million contribute directly to profits.
- POMOP: Anticipates 50% to 65% growth, aiming for approximately $110 million in sales.
- DMX-200 Acquisition: Involves a $30 million upfront fee, supporting the company’s strategic expansion.
Operational Updates
Amicus is making significant strides in operational execution:
- Galafold: Recently settled with Teva to extend IP protection to 2037. Growth is supported by improved diagnostics and AI collaboration with UPenn to identify Fabry patients.
- POMOP: US launch is gaining momentum, with strong demand in April. Expansion is underway beyond key opinion leader centers to secondary centers, with approvals in Australia and Canada expected to impact in 2026.
- DMX-200: A Phase 3 asset targeting FSGS, aiming to address a market of 40,000 patients in the US.
Future Outlook
Amicus is focused on long-term growth and innovation:
- Galafold: Continues to emphasize patient diagnosis and aims for high double-digit growth in patient identification.
- POMOP: Expects an increase in patient switches from Nexviazyme and Lumizyme, with efforts to educate physicians on its benefits.
- DMX-200: Top-line Phase 3 data is anticipated in approximately two years, reinforcing its strategic value.
Q&A Highlights
During the Q&A, executives addressed several critical issues:
- Executive Order & Tariffs: The company is closely monitoring the "most favored nation" executive order and views tariffs as manageable.
- Galafold Growth: Driven by new patient diagnoses, supported by an AI pilot project at UPenn.
- POMOP Dynamics: Success hinges on patient switches from other therapies, with a focus on the US market.
- DMX-200 Strategy: The acquisition is aligned with leveraging Amicus’s existing infrastructure and expertise.
In conclusion, Amicus Therapeutics is strategically positioning itself for growth through innovation and targeted acquisitions. For a detailed account, refer to the full transcript below.
Full transcript - BofA Securities 2025 Healthcare Conference:
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: Back to the Bank of America Healthcare Conference. I’m Tazeen Ahmad, I’m one of the senior’s med biotech analysts here at the firm. It’s my pleasure to have with us our first presenting company of the morning, Amicus Therapeutics. Sitting up here with me on stage for the company are Jeff Castelli, who is Chief Development Officer, as well as Simon Harford, who is the CFO. Gentlemen, good morning.
Thanks for making the trip over from these posts.
Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Good morning, Tazeen.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: So we’re asking everybody some macro questions right at the get go to get those out of the way. It seems like it’s quite topical these days. So I’ll start with the the more recent updates, which is earlier this week, the executive order that was announced relating to most favored nation. Wanted to get your early thoughts. I know it’s still, in flux, about what you think, the end result of that could be and what it particularly means for Amicus.
Simon Harford, CFO, Amicus Therapeutics: Yeah. I mean, like a number of these executive orders, nothing is exactly clear at this point in time. What I can say from a point of view of amicus, though, is as we think about our sales breakdown, So you’ve got Galafold with roughly 60% of revenue coming from outside of US and 40% from within The US. And then within that US number, probably about roughly slightly less than 25% is Medicare and a few percentage points more for Medicaid. That potentially could be impacted by MFN if it goes down that path.
On POMOP, you’re talking more about roughly 45% of sales globally or in The US at this point in time. And probably, again, in the region of 35 to 40% of sales of Medicare or Medicaid. So you can do the math sort of on that. I think the broader question is actually I think the president’s right. It is time for Europe to step up in terms of pricing because we’ve clearly seen that prices are substantially different in the in The US.
So part of what one will be looking at over time is what are the opportunities to with this type of situation to to increase prices, and you’ve seen what’s happened with some of the big pharma companies coming out to the European Commission to talk more specifically. So we’ll see. Right now, I would call it business as usual. We’ll have to wait and see, but that gives you a feel at least of where some of the sort of exposure could be.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: Okay. And then, some related points as well. What’s your general view on tariffs?
Simon Harford, CFO, Amicus Therapeutics: Yes. So on tariffs, as far as 2025 is concerned, really, it’s a non event for us because we had inventory already in place for most of the sales for this year. As you know, we have been transitioning way before the tariff discussion from China to Ireland. We have run some worst case scenario models, assuming sort of China at a 25%, and European Union more in the 25 ish percent range. And what I can say to you is even in that worst case scenario, it’s manageable within our sort of direction of travel of of transitioning to GAAP profitability and then starting in the second half half of the year.
Does it have an impact? Of course, it would have an impact. But it’s manageable in that guidance trajectory as the way I would describe it, where we’re going. I think that the one other thing I would say is we announced that we we are doing some dry product manufacturing going forward here in The US. So there are certain steps we can take.
I think the bigger one, though, is as you go to Ireland, you know, when we did the worst case scenario, we’re in China for the next couple of years, but then we transition. So in that scenario, we obviously had a much sort of larger impact short term than longer term, but it’s all manageables, the way I think about it.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: Is there any way of, bringing everything onshore, or would that just be too difficult?
Simon Harford, CFO, Amicus Therapeutics: Realistically, there are certain timelines. So and frankly, you know, we are where we are at this stage. So, you know, just from a timing perspective, you, yeah, you can rush through certain things short term, but it’s not practical to do that for everything.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: And then lastly, where does your IP reside?
Simon Harford, CFO, Amicus Therapeutics: Our IP resides primarily here in The US.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: Okay. So there would be there would be any transfer pricing?
Simon Harford, CFO, Amicus Therapeutics: I don’t sort of see any issues on that front.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: Okay. Got it. Alright. So moving on from there, let’s talk about the day to day business of Amicus. So can you talk about the trends that you’ve been seeing for Galafold?
So this is, I guess, mature launch by your definition in some respects, just like calendar wise. In other ways, you’re still looking for pretty big uptake in patients over the next several years. So can you walk us through how that has progressed up until this point and the efforts that you’re undertaking in order to get to your targets at the end?
Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Jeff, do you want to? Yeah, mean, so we’re extremely pleased with Galafold in terms of how the original kind of early days of launch went, and now we’re sort of, I guess, mid launch, I would say. We’re really excited by the recent settlement with Teva that gets us, we believe, IP out to 2,037. You know, we’re looking at passing 500,000,000 in sales this year of Galafold. And what’s really exciting for us to see is this is now driven by mostly our largest countries, The US, Germany, UK, and driven by naive patients.
We’ve always said Fabrya is one of the most underdiagnosed rare diseases and genetic diseases, and it really is. We know when you look at newborn screening, when you look at at risk screening, there are many more people and families still living with Fabry not diagnosed than are diagnosed. And what we’re seeing is physicians have a higher suspicion of kind of when to potentially suspect Fabry. They can send for panels of renal or cardiac tests to kind of and Fabry is on those panels. We’re seeing newborn screening in The U.
S. Eight states are screening for Fabry. And when you find that newborn, there’s family screening available, and you can typically find many family members because Fabry is an excellent dominant disease. And then one thing we’re investing in where we think we could potentially help even drive things further is using AI and medical records. We have a pilot project with collaborators at UPenn, and we’ve taken 500,000 plus of their medical records in their system, run through an AI algorithm to flag kind of risk of Fabry from one to five hundred thousand, and we’re testing the top hundred people at risk for Fabry to see if they might actually have Fabry.
And just based on the prevalence rates in that five hundred thousand, we would expect there should be one hundred Fabry patients. So we’re very excited to leverage that. And if that is successful, we could then roll that out to other medical systems in The US and even more importantly, ex US.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: So based on what you’re saying, is the path going forward to just get more patients diagnosed relative to getting patients just aren’t on anything if there is such a thing?
Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Yeah, I mean, this point, there’s still very incremental geographic expansion. In some of our more recent launches, there still are some switch patients. But it’s really at this point, future growth is going to be driven by diagnosis. Galafold is now standard of care for amenable patients, so we don’t need to know where those patients are diagnosed. Anything we can do to contribute to diagnosing Fabry patients is good for the Fabry community, and it’s good for Amicus, and it’s good for our shareholders.
So we’re really focused on helping to drive diagnosis. And one nice thing I forgot to mention, Tazeen, is another tailwind for us is that traditionally amenability for Galafold was thirty to fifty percent. It kind of varies by country. And that was very enriched for classic Fabry, what was able to be diagnosed traditionally. And a lot of that pool of existing or sort of future patients that we’re going to now are highly enriched for amenability.
So it’s going to be much more like 50% plus amenable rates in that kind of coming in new diagnosis group of patients. So diagnostic tailwinds, highly enriched from amenability, Galafold is standard of care.
Simon Harford, CFO, Amicus Therapeutics: And I would just add, I think, you know, there’s so much focus on Palmylity Opt Folder at the moment with sort of uptake as the newer product, but the reality is Galafold is the biggest single driver of value for the foreseeable future. Why? Because, you know, as we saw 14% patient growth in the first quarter, our guidance, as you know, is sort of 10% to 15% revenue growth for the the the full year. Keeping on that trajectory, the we hit basically 400,000,000 of annual sales, pretty much anything beyond that, over and above that falls to the bottom line too because we’re not increasing sort of infrastructure. Yes.
There are a few continuing trials, but that, yeah, we’re clearly leveraging the p and l. So I just want to emphasize how important that is ultimately to our valuation. And as you’ve articulated, the opportunity seems really you know, there’s no reason why it shouldn’t continue, let’s put it that way.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: So if it’s the case that going forward you have to to mostly locate new patients, how should we think about, like, the pace of uptake going forward? Isn’t that going to be a bit slower than simply, you know, having the already identified patients and switching them? Or are we thinking about that incorrectly?
Jeff Castelli, Chief Development Officer, Amicus Therapeutics: So there is a large existing pool of diagnosed untreated patients. I think it’s actually seven six or seven thousand patients are diagnosed and untreated and sort of being watched to then start treatment once they’re sort of severe enough. So there’s that pool. And when new patients are diagnosed, it’s not always that they’re sort of just early with their symptoms. When you find an index patient and then you do the family screening, you often find people that are very severe progressed along the pathway and need to go on treatment right away.
So, it’s actually the growth we’ve seen in the last couple of years has been from just raw number of patient adds. It’s been higher than we’ve seen since the very early launch days. So there’s a bigger base, obviously. So you know that the annual growth But in terms of numbers of patients, it’s actually continuing to stay the same or increase from a and when you look at the numbers, we don’t really see that slowing down based on how many people are left to be diagnosed.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: Okay, so talk about what your goals are for where you think penetration will land in this population.
Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Penetration is interesting. I think you’ll continue to see many more patients diagnosed, so high double digit or not double digit growth of patient identification and diagnosis. Some of those patients will fall into the watch and wait group, some will fall into the need to treat immediately group. Those that are amenable, I think, as I mentioned, that thirty to fifty percent will continue to increase in terms of percent amenable. And of the treated amenable, we’re at 90% plus market share in the countries where we’ve been present for several years.
So it’s really just a matter of are they amenable? Do they need treatment? Are they diagnosed? And they generally go on Galafold.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: And I guess lastly, geographically speaking, where do you think more of the patients will be found? Is it here or ex US?
Simon Harford, CFO, Amicus Therapeutics: So I I think it’s primarily in the existing markets and expansion in those markets because they tend to be a lot larger from an opportunity perspective. There are, you you know, certain newer markets where we’ve entered in Asia Pacific and Latin America, but I don’t see that as the core piece of the stage of the growth. Let’s see it more than existing.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: Okay. Got it. So let’s move on to Pompe. Can you just talk about, you know, the factors currently that you’re seeing that are leading to acceleration going forward of uptake?
Simon Harford, CFO, Amicus Therapeutics: Yes. So if I start and then maybe you can comment on a bit of color to the market. But the I think as you think about the rest of the year, we guided to earnings to 50% to 65%, which puts at the midpoint of the range, I think, roughly about $110,000,000. What we have seen is quite an acceleration, since the end of the quarter. April was frankly our strongest month ever since launch in terms of demand.
So that’s those either scheduled or actually sort of going on to treatment, which was extremely positive. We actually have a new commercial head in The US because our previous head is is retiring, who brings new perspectives also to the organization. And that we’re seeing that that acceleration is coming from a broader group of centers, so not just the key opinion leader centers, but also more secondary centers, which is kind of interesting from a dynamic perspective. Too early to sort of say, you know, is that a trend, but that’s what we’ve seen more recently. I think, there’s no question that The US acceleration is the most important piece to the sort of the top end and the bottom end of the guidance just because those patients are worth more than international patients by definition.
I think as you think then about the next step, obviously, it’s important that we continue to grow step by step in the existing markets, the Germanys, the UKs, the Spains of this world, because they’re reasonable sized markets, and that we are seeing happening. Then you have, what I call the the bigger new sort of, reimbursed markets. So Italy, where we’ve seen first patients in in recent weeks, there that will be a stepwise process again because you go through a regional formulary after reimbursement. But the you know, it’s it’s a big market. The Netherlands, we won the tender, which means that there are roughly 150 patients of which we will get probably 70 odd percent in total.
The challenge in The Netherlands is they will come on more in a drip by drip process because there’s only one center to get those patients through. So realistically, we’re saying it could take twelve to eighteen months to fully transfer everyone, but it’s a great opportunity. Those in the that come later in the year with pricing and reimbursement, probably less of an impact for this year. They’re more relevant to next year. But Japan, where we haven’t yet got approval, but we anticipate by year end, they could launch pretty quickly because it’s it’s one of those markets where you don’t have to wait that long for pricing and reimbursement.
Australia and Canada, we’ve also got approval already, but realistically they will be twenty twenty six, patients.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: Okay. Can you talk about the length of time a patient stays on the Sanofi therapies before transitioning over and if timetable is changing?
Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Yeah, it’s a great question and one of the dynamics we’re paying close attention to. So, know, traditionally Lumazyme has been available for many years and, you know, most or all treated patients were on Lumazyme. And then when Sanofi launched their next generation ERT, next Biozyme, we saw in The US a pretty quick adoption. At that point, Palmop was not available. We were still going through COVID and inspection challenges.
And by the time we entered The US market, a lot of the patients had switched from Lumizyme, the majority of patients from Lumizyme to next Biozyme. Outside of The US, it’s a little bit more contemporaneous. We’re often launching at the same time or, you know, slightly behind Nexviazyme, we’re seeing, you know, slightly different dynamics that we can talk about. But The U. S.
Was interesting because we weren’t sure how long patients would, after switching from Lumazyme to Nexfiazyme, when would they be ready to switch again to a third treatment. We still believe very much in our product. We think Palm Up offers a very differentiated mechanism of action, differentiated efficacy. We thought that maybe, you know, from research, it could be six or twelve months after starting a new treatment when patients and physicians might be ready. And what we’ve really seen is it takes about two years for them to really understand how the patient has done after switching.
Going into last year, it was about ten percent of the patients had been on two years, but coming into this year, back in January, it was about forty percent. By the end of the year, it should be around sixty, seventy percent. So our pool of patients that now have been on NexVynazyme for two plus years, if they are progressing or not doing well, might be looking for a switch has increased. And that’s really where we’re starting to see, you know, as Simon mentioned, that expansion of centers and more experience. We’re actually hearing, you know, very good positive anecdotal reports of patients when they’re switching from Nexvizam to Palm Up.
We’ve recently had some case studies that reported on those experiences. So I think as you start to get more of that kind of accelerating experience with Palm Up, we’ll start to see more and more transitioning and switching for the patients.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: Okay. So just by that dynamic alone, why wouldn’t the launch trajectory for Palm Up meaningfully accelerate from here, over the next quarter or two?
Simon Harford, CFO, Amicus Therapeutics: Yeah. I think the dynamic will be how quickly, as I said, the variability in the guidance is sort of dependent on how quickly those patients. And I do think we have to be a little bit thoughtful, though. And it’s very clear in my mind that you will, over time, get an acceleration because that pool is expanding. The question is exactly how quickly does that progress because the 40% are a pool of patients now.
Some will go transition faster than others. So But
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: what are the I guess what are the drivers of that? When we talk to investors Amicus, when we talk about the Palmop launch and the big opportunity and the number of patients and the potential for really large peak sales, the first thing we hear is, well, why is it going so slowly? So maybe you can just talk to us about what doctors are telling you about what they’re looking for as the point in time where it makes sense or how patients are involved, if at all, in that decision making process.
Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Yeah. You know, for us it’s still, we believe, a matter of when, not if a majority of Pompe patients will
Simon Harford, CFO, Amicus Therapeutics: be on
Jeff Castelli, Chief Development Officer, Amicus Therapeutics: pump I mentioned, one thing we’ve learned is sort of there’s that time period of understanding how the patient is doing now that they’re on a new product. Let’s say it’s NexViazyme. They only are coming in really more every year for a major workup. We thought it was every six months. There’s been traditionally a lack of standardization of how they’re assessed.
So one of the things we’re starting to see is more interest in sort of energy being put into standardizing how patients are followed. So now that there’s multiple options, physicians will know if their patients are stable or declining. So that’s a key. So, yeah, we don’t really see I don’t know, Simon, if you have anything to add to that. Sort of lost my train of thought.
No.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: Okay. So what are some points you can talk about as it relates to how patients respond once they have transitioned over to palm up? Are there any differences that they note that can help kind of spread word-of-mouth?
Jeff Castelli, Chief Development Officer, Amicus Therapeutics: Yeah. And just coming back, you know, for the topic I was on before, after that two year period, now it’s sort of a matter of are the patients really progressing or not? And one of the things we’ve seen with our data was when patients switched from Lumizyme, have great data showing they actually have benefits, improvement was possible. We don’t really have a great sense yet of how many patients are at two years. It’s not like every single patient is now ready to switch, but there are going to be some that are ready to switch at that point.
And then at three years and at four years, we believe that will continue to accelerate. So again, in The US, it’s sort of that dynamic of we really were stuck for a little bit in that period, and now we’re sort of starting to learn about how many patients, when are they ready to switch. I think as we get broader experience, as I mentioned, you’ll start to see more experience and more comfort switching. So it’s still sort of a point where we’re trying to understand those dynamics, and we do believe it will happen. But it’s hard to say it’s going to accelerate next quarter or this quarter, but there’s a lot of positive dynamics that we’re seeing that we expect to see continued long term adoption of Palm Op.
I think also there’s sort of a in some way, there’s a lack of urgency sometimes. Like the physicians feel like, okay, I have these treatments. I can keep one in my back pocket. So there’s part of that education as well, trying to say if there’s potentially a product that could be, you know, deliver something that the other products couldn’t, why wouldn’t you want to use that earlier? This is a progressive disease.
So part of that also is us being able to educate physicians about our data, how you monitor patients, and when you should think about switching. Okay.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: And then just to wrap it up, how should we think about the dynamics of the switches in US versus ex US?
Simon Harford, CFO, Amicus Therapeutics: Yeah. I think in terms of that, what we’re seeing at the moment in The US is that switches are coming proportionally from NexViasem and from Lumazyme. Relates to the The US marketplace. As far as Europe is concerned, it’s a little bit more of an even keel is the way I would describe it just given, the timing of launches is different than what we’ve seen in The US, and we are making, frankly, what I would describe as pretty strong progress. The fact that we’re now the number one, product in Pompe disease in The Netherlands and Sweden or at least will be shortly, should I say.
I think it’s obviously, it’s partly a recognition in some of those markets from a pricing point of view, but it’s also a recognition of value of of the product, and what it brings to patients. You know, Spain, where we pretty much launched in parallel with Sanofi, Nexvirozyme, we’re doing extremely well in that market, very competitive. And I think you question you had earlier about sort of how are sort of patients reacting to being put on Pomop. You know, Jeff probably has more stories than I do, but the yeah. Overall, the general sentiment seems to be that, it’s a very strong profile with data that probably certainly in the switch patients is is sort of stronger and available for us to use, let’s put it that way.
Okay.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: Got it. So, let’s maybe move on now to your newest edition, DMX 200. So I guess maybe as at the top, can you explain what the strategy of Amicus is with these types of of acquisitions being, you know, considered going forward and why this particular asset was the right one to do at this time?
Simon Harford, CFO, Amicus Therapeutics: So maybe I’ll just start on the bigger picture and then Jeff can comment on Dymarex. But, yeah, we would have been very clear that ultimately we wanted to do some in licensing of products ultimately to leverage the infrastructure that we have. And we were also very clear that we wanted to sort of take a little bit baby steps, if I’m honest, because we were in the process, obviously, of launching Pombility off folder on top of of Galafel and trying to move to profitability as as an organization. And so this was a great opportunity for us that fit very much on strategy sort of approach, but both from a point of view of fit with the portfolio and adjacencies to Fabry and Pompe, but also from a financing perspective. What we didn’t want to do was to go out and bet the house with a huge amount of financing on something.
We wanted to more leverage the infrastructure and the ability to pay, you know, a $30,000,000 upfront fee, to have then the next milestone payment for us at the point where you flip the card on top line phase three data seemed a very good risk reward profile and was very much, in our view, disciplined in what we’d said we would do and hit the profile. So so so that’s kind of why we did strategically what we did and financially what we did so far. I think as you think about the the future, I think it’s still more about continuing to leverage infrastructure. You know, obviously, the Dimerix deal is a US focused deal, but we also have a big international infrastructure, and there are opportunities to leverage that. So that’s probably more the road we will go down is in a similar type of approach to what we’ve just done here rather than betting the house at this point in time.
That’s how we think about it because we’re also conscious we don’t want to over dilute shareholders. We want to balance that risk reward and the capital piece.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: Yeah. And how do you decide like at what stage asset makes sense to you? You’re a very strong commercial company, so how do you balance taking something that’s ready to be marketed as opposed to something that still needs a little bit of further derisking?
Simon Harford, CFO, Amicus Therapeutics: Yeah. I I mean, so this obviously was a phase three asset. We’ve talked about commercial assets. Commercial assets are probably the more likely or or something where we’ve seen top line data already and they want a partner for outside of The US is probably the nearer term, the more likely group. But if we found another Dimerix type deal, then absolutely we’d look
Jeff Castelli, Chief Development Officer, Amicus Therapeutics: And it really does leverage our late stage clinical regulatory capabilities as well. You know, with DMX200, we saw FSGS as a great kind of adjacency to Fabry disease with the nephrology connection. We saw a really strong mechanistic rationale for the approach of addressing the monocyte macrophage driven inflammation in many rare kidney diseases. And FSGS plays a very central role in many of those patients. We thought it was a very undervalued asset, a great chance for us to get access to potential blockbuster market in The U.
S. For, as Simon said, a $30,000,000 investment. There’s forty thousand patients with FSGS in The U. S. And five thousand diagnosed each year, and there’s no current FDA approved treatments.
So we see a real opportunity there to potentially really help those patients in that community and also, you know, if successful, which we think there’s a very strong probability of success here, especially with us being able now to contribute to kind of putting together that final clinical, endpoint and interacting with FDA, we’re really excited about the potential there. I’d love to have more opportunities like that, Simon, that we can bring in.
Simon Harford, CFO, Amicus Therapeutics: And, frankly, in the nearer term, given we won’t really see the top line data for probably another two years Yeah. Actually, in the interim phase, it really doesn’t affect our trajectory to GAAP profitability and free cap flow being positive during that period because we’re not spending anymore till we see the cards turn.
Tazeen Ahmad, Senior Med Biotech Analyst, Bank of America: Okay. Perfect. With that, we’re out of time. So thank you
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.