Bradley Campbell, the President and CEO of Amicus Therapeutics. Thank you so much for being here.
Thank you for having us. Thanks to Goldman for hosting the conference. Nice to see everybody here today.
Thank you. To start off with a big picture question, can you give us a snapshot of your business today and your strategy with regard to commercial execution in the second half of the year?
Sure. Yeah. At Amicus, our mission is to develop and deliver next-generation therapies for people living with rare diseases. We are grounded in our core business, Galafold, which is the only oral therapy approved for Fabry disease. We will pass $500 million in sales this year. We have 12 more years of exclusivity on our way to $1 billion in peak sales, so a very important part of our growth story. We've guided to 10%-15% growth this year for Galafold. For Pompe disease, we've recently launched our new therapy, Pombiliti and Opfolda, for the treatment of Pompe disease. We'll pass $100 million in sales this year. Our guidance is 50%-65% growth, well on our way again to $1 billion in sales. We're very excited about that, with some additional development opportunities, in particular in the pediatric population.
We have our brand new asset, DMX-200, which we licensed from Dimerix, indicated for FSGS, which is a rare kidney disease, high unmet need, really exciting new mechanism of action, and again, potential blockbuster potential. All of that is then underpinned by, I think, significant financial discipline. We're on our way for a major milestone this year of GAAP profitability in the second half. In a few short years, by the end of 2028, we expect to see $1 billion in combined sales. We'll continue to drive these exciting therapies forward in Fabry and Pompe, build to the story with FSGS and other opportunities going forward, and then continue to maintain financial discipline so we're self-sustainable and getting to profitability and positive free cash flow.
What part of Amicus' story do you think the street is not appreciating enough, given the recent stock movements?
Is it fair to say all of it?
Yeah.
Is that a fair answer to the question? No, it's a great question. I think there's a big piece of it, which I know we'll get into, which is the macro environment. That's hitting our whole sector. Sharing some stories with investors who've been in the space for a long time, sometimes memories are short, but we've been in these kinds of dark days before. From a macro perspective, I'm highly confident, and we can talk more about it, that we'll easily work our way out. I think Amicus is actually uniquely positioned to exit this period with a very strong trajectory. I do think if you just look at those core areas of our business that we talked about, Galafold as an example, clearly undervalued today.
I think a few things that maybe people either look past when they're looking more at the launch or don't fully appreciate is, look, this is a $2 billion global market today. It's growing to $3 billion over the next 10 years. Again, we're starting this year, we believe, at over $0.5 billion in global sales for Fabry disease. We've got 12 more years of exclusivity on this product. I don't think at all people are giving full credit to where Fabry is going, just based on our track record. I also think there's lots of reasons to believe that there could be even more diagnosis in the space. We can talk more about what those might be. I think just Galafold alone, if we were a single-product company with just that asset, I think we'd be a much more valuable company today.
The second piece, of course, is Pompe. I know people are laser-focused on the launch. We can talk about why we have great confidence in the launch. Again, if you just take a big step back and say, "Okay, first year of launch into what is today over a $1.5 billion market, growing again to over $2 billion over the next 10 years. We're already at $100 million, based on our guidance and sales this year, over $100 million." We're off to a great start. We can talk about why we think we'll get more traction there. Clearly, we do not get full credit for Pompe or maybe any credit for Pompe. Maybe because FSGS is new to the story and we need to do some education on why we're so excited, but there's no chance that that's baked in.
Again, if we were a single-asset company with a highly de-risked, late-phase three product for FSGS, we'd be worth a lot more than I think we would get credit for today. Look, I get the macro environment and happy to talk about why we think that we can work through those things. I think there's a lot of opportunity for investors. If you're looking in a world today with so many companies who are starved for capital, who are running out of money, have big binary events that might be zeros or might be something positive, and then you look at Amicus, which is delivering consistent growth, soon-to-be profitable growth, and now adding to the portfolio, I think there's a lot of opportunity for us to put points on the board and create value for investors.
Definitely. If we just speak to the macro bit, how would you say Amicus is positioned in the environment when you have tariff risks, IP, transfer pricing, most favored nation, all these dynamics?
All the stuff, yeah. Clearly, the world, we all hate uncertainty. And markets in particular hate uncertainty. I think part of the issue is just macro uncertainty, which impacts all of us. Specifically for Amicus, though, I do think that the potential risk is perhaps painted with a heavier brush on Amicus and our core business. But we really believe, in fact, that the actual impact of these various factors will be minimal, if anything. I think a great example of this and maybe a lesson for all of us is the Biosecure Act. As a reminder, Biosecure last year was a big deal. It was talking about moving manufacturing from China to the United States, et cetera. Huge impact for many of us who manufactured in China. And at the end of the day, the bill was never even passed. It was a total non-factor.
The eventual bill was actually quite supportive of what would have been a very orderly transition to the U.S. I think that's a great example of this perceived black swan event that we very carefully had risk mitigated around and in the end was not a factor at all. If you had been smart, you could have taken advantage of some of that dislocation. If you think about what's facing us this year, let's start with tariffs. Tariffs are, you can debate the macroeconomic benefit or cost of tariffs. As it relates to our business, what we've said very definitively is there is a non-material impact on our business this year. Why is that? Because first of all, the majority of our revenue comes from Galafold. Galafold has a tiny, tiny cost of goods. It's manufactured in Switzerland.
Very, very little impact of any kind of potential tariff coming out of Switzerland. As it relates to Pombiliti, Opfolda, we're manufactured today in China. We're moving that manufacturing to Ireland. It will enter the U.S. supply chain in the back half of next year or early 2027. Even before then, for general supply chain continuity and security, we moved all of our commercial launch material to the United States already. All of the material this year and a significant portion of what we have anticipating for next year is already in the U.S. That's not subject to tariffs. Frankly, if you take the most conservative view of tariffs and you apply that to some portion of the Pompe cost of goods going forward, the reality is it probably looks something like a 5% increase in cost of goods.
It is not by any stretch a significant material issue that we need to be worried about. Oh, by the way, we're already long down the road of reducing our cost of goods through our second-generation process. The benefits from that savings will more than outweigh any potential cost of tariffs, which, who knows if they even survive through the next term of Congress or whomever. I think tariffs, again, are overblown as it relates to how they might impact Amicus. The second one you mentioned is IP and transfer pricing. Good news is our IP sits in the United States. There is no real issue there. MFN, that's another one. Again, I'm not a trained economist.
I think we've seen a pattern of big headlines that then lead to something that's much more pragmatic or reasonable as it relates to how this administration is operating. Again, leaving aside whether we should or shouldn't be paying more or less than Europe, just thinking about how this might impact Amicus, I think the reality is, at least from what we see, what we believe, we think that there's very little legal framework to enact what the president described in his executive order or his direction to CMS. Even if it does take effect, I think it goes something like the way IRA went, which is it focuses on the most expensive drugs in the space first. Typically, rare diseases are excerpted from that.
I think there's every chance that even if something does pass like that, we would be exempted from that, just like with the IRA. The last piece is the reality is if you were to say worst-case scenario, let's just say that MFN does pass. For Medicare and Medicaid business, you're getting some sort of negotiation. If you look at our global business across Fabry and Pompe and you assume a 50% reduction to that, that's probably a 5% impact to our long-term business. Do I want that outcome? No. Do I think it's negative for innovation? 100%. I think there are lots of different ways to do that and to work on pricing and access and affordability. Even in the worst-case scenario, Amicus is actually fairly insulated from that outcome.
I think if you put all those things together, I get that there's uncertainty. I get that that creates dislocations in value. I think we're caught up in that. I think it's a real opportunity to take advantage of that dislocation as it relates to Amicus. The one piece that you didn't mention, which I think is actually an opportunity, is the FDA. We heard a lot coming into the year about what happened to the FDA. You saw some very familiar, I think, very industry-friendly or industry-neutral figures in the FDA forced to leave. The question was, "Oh gosh, what's going to happen here?" I think the reality is the new leadership has been very clear about supporting rare disease regulatory reform. I think the net-net of all of this actually may be very positive reforms from a rare disease perspective.
If you think about our new asset in FSGS, we very much could benefit from some of those changes.
That's really helpful, color. Now, speaking to your guidance, Amicus expects to achieve positive net income in the second half of 2025. What is baked into this guidance? What are your expectations around the operating expense side heading into the later half of this year and on the forward, given Amicus has done an exceptional job in managing that till now?
Sure. Thank you. Yeah. So really the key drivers of how you get to the GAAP net income, of course, we delivered our first year of non-GAAP net income positive last year, which is great. For this year, of course, you have the top-line revenue growth. Our guidance is 15%-22%. So very strong top-line growth. On the bottom line, or excuse me, on the expense line, even with the new licensing payment from Dimerix, we've guided to $380 million-$400 million in expenses. So significantly more revenue than expenses gets you to that GAAP income in the second half of this year. As it relates to go forward, if you look at just the core business right now without including DMX or any other future opportunities, what we've said is we will continue to tighten our OPEX spending.
You'll see a little bit of spend towards our ongoing registry and post-marketing commitments. You'll see a little bit of spend towards second-generation manufacturing. By and large, that will be largely flat or maybe some inflationary increases with a significant top-line growth. That will lead to naturally going from GAAP net income in sometime during the second half to a full year to EBITDA and positive free cash flow. I think one of the challenges for a lot of other companies in the space right now is, yes, they have these macro issues. Yes, there's other things going on. A lot of them need to raise capital. That's where you really get into trouble. That's where Amicus has, I think, has differentiated itself. That's kind of in the short term.
One other exciting part about the DMX transaction is super excited about the asset, excited to collaborate with Dimerix. I know we'll talk more about that whole opportunity. The way the deal was structured, we do not have to invest a single dollar into R&D until we turn over the phase III data card. It is very smartly structured in the sense of, yes, we paid the $30 million upfront for a billion-dollar potential asset. We think that is a great price to pay. We do not have to invest anything else from a development perspective at all during the transaction. Even from a commercial perspective, we do not have to make those investments. Still, we see a positive outcome. We think it is very smartly structured. That is another way to keep those expenses relatively flat where we grow the top line.
Now, maybe we pivot to Pom-Op.
Sure.
In the first quarter, Amicus lowered its guidance for FY25. That was due to multiple headwinds. Could you speak to the headwinds that contribute to this decision as well as your confidence in achieving this updated guidance?
Sure. Yeah. With Galafold, it's a much more straightforward picture. I know we'll come back to that in a minute. In a launch year, there's a number of different factors that are going on, one of which, of course, is the launch cadence. A big reason why we had a slightly lower than expected Q1 was we had a number of countries where we were nearing finalization of reimbursement and therefore launch that we believed would come into Q1 and end up slipping into Q2. The good news is, and we'll talk more about it, there was a variety of positive outcomes from taking longer to get to the reimbursement and launch. At the end of the day, you never get a chance to reset that price in Europe. They only go down from there.
Taking a few more weeks or another month to get to a better outcome is worth it, even though the pain in the quarter was what it was. That was one contributing factor. The good news is multiple of those countries have now gotten to reimbursement at launch. I'll come back to that in a minute to your point about confidence. The second piece was this VPAS thing. One thing to remember is that this has been one way or another, VPAS or some form of it in the U.K., which is a mandatory tax on all pharmaceutical goods, has happened every year since we've been commercial with Galafold. This isn't a new thing.
What was very unusual this time is that the way the process typically works is the Industry Association in the U.K., ABPI, negotiates on industry's behalf to an agreed-upon rebate rate. Typically, they'll give you guidance at the end of the year to say, "Look, it's going to come in at 10% or 15% or 7%." They finalize the negotiation the next year. You have some ability to see what's going to happen in the following year. Every year, we've done this since we launched Galafold. Every year, it's been within a couple of basis points of what they suggested it would be. This year, for whatever reason, they guided to a 12%-15%. This is all public, by the way, a 12%-15% VPAS rate.
In the end, it was 23%, so 50% higher than what they had guided us to. 23% is also, I think, the second largest ever, at least since we have been launched, VPAS rate. It was both an outsized rate in the first place, and it was 50% more than what they told us. Unfortunately for us, the U.K. is our second or third largest market for Galafold and Pompe. It just has an outsized impact. When we looked at those two things, launching a quarter late in what ended up being five or six markets and then having this haircut come off the top of your second largest market in Pompe, we just did not feel like we could catch up to that over the course of the year. I still think that 50%-65% growth is fantastic.
Again, more than $100 million in the second full year of launch. That puts us on our way to what we think is a billion-dollar-plus opportunity. I get, especially in this market, that that was people were you just get an outsized reaction when the whole world is seemingly in a negative place. What gives us confidence in going forward? A few things. First of all, when we gave the new guidance in April, we had shared that April was turning out to be one of the largest ever net new commercial starts months. We've seen that again now in May. April and May now look like they may be the two largest months so far. That's both in the United States specifically, which is really important because it's the highest-priced market.
Also now, again, you've got six new countries contributing to the launch in Europe, in particular, Netherlands, Italy, Portugal, Belgium, Czechia, and Sweden. Six new markets that are all doing really well. When we look at kind of that trajectory, when we look at the new six markets that are there and some other, and I know we're going to drill down on a couple of different factors. I'll provide more detail in a bit. Everything we see right now says that, number one, we're on track to deliver a strong second quarter. More importantly, we'll have that acceleration in the second half that we expect to see.
Then, speaking specifically to the U.S., can you speak to the launch dynamics there in terms of reimbursement as well as what you're seeing on the competitive side with Nexviazyme?
Yeah. A few important things in the U.S. Overall, I think the biggest learning in the United States was, look, unfortunately, because our PDUFA date was during COVID, we had a delay in our launch due to the FDA not wanting to go to China to do the inspection. It is what it is. It is a two-year delay. We did not think it would be as impactful as it was. What we were surprised by was in our early market research, physicians and patients said, "Hey, if I am not doing well on any therapy, whether it is Nexviazyme or Lumizyme, which are the two competitors' products, I would switch within six months or maybe a year." As it turns out, now that we are both on the market together, they had about a two-year head start.
What we've heard very definitively, and I know that the sell side has done a number of market research surveys that corroborate this, physicians need like two years before they're willing to switch again. It kind of makes sense in the following ways. First of all, really, it turns out that patients aren't coming to see their physicians more than once a year, very rarely. If you've just switched a patient, you want to wait at least a year to see them once and then probably a year to see them again. You have some sort of trend or feel for how they're doing. To be fair, that was a surprise to us. It was different from what we initially understood.
That being said, if you look at where we are now, it actually sets us up really well for this year, seeing what we're starting to see now in April and May. Why is that? First of all, when we first launched last year, there was only 10% of patients started the year. 70% of patients were on Nexviazyme, but only 10% of them had been on for two years. Fast forward to the end of last year, it was about a third of patients. By the end of this year, it's going to be about 70% of patients. The patients moving into that switch window is going to be about 70%. Much, much bigger pool of patients. What else are we seeing? We're seeing proportional switches from Nexviazyme and Lumizyme. What does that mean?
Two-thirds of all of our commercial patients started on Nexviazyme and have switched. The balance has been Myozyme. Why does that matter? One thesis, one bare thesis would be, you'll never switch a Nexviazyme patient. Those are actually lost to you. That's not true at all. In fact, we're switching. A majority of patients are coming from Nexviazyme. Why? Because that's the biggest pool of patients to switch from. We know proportionality is driving the switch. That's good. The second piece is increasing data and then real-world evidence. There's two pieces of that. The first piece is continuing to see publications and further evidence from our phase three data, looking at things like clinical meaningfulness of the effect, things like impact on secondary endpoints.
Remember, we're the only drug that has shown clinically meaningful and statistically significant benefits when people switch from Lumizyme to a new product. Those data continue to be strong and growing. The other piece, of course, is the real-world evidence. That's showing up in case studies from Lumizyme to Pom-Op, but also now case studies from Nexviazyme to Pom-Op, increasingly supporting the switch and outcomes when people move to Pom-Op. We think that evidence will continue to grow and continue to help support that. We're also seeing increasing breadth and depth. More physicians are prescribing the first time and more physicians are prescribing for the second or third or fourth time, so that's really powerful.
On the insurance side of things, we've actually gotten that down to it's less than 30 days on average for first prescription to insurance authorization, and then about two weeks to get to the infusion. All of those things are going in the right direction. What is that translating to? It is translating at least initially to this acceleration and the highest numbers of new commercial starts in the U.S. More to come, of course. You will get the benefit of that again, more weighted the second half. Everything that we want to see, all the KPIs we track, are headed in the right direction.
Great. If we speak about the ex-U.S. dynamics, including your outlook for the launch countries in the near term.
Yeah. In the original countries, so U.K., Germany, Spain, very strong uptake. In many cases, we've been on the market either at the same time or even slightly earlier than Nexviazyme. There, we're seeing kind of market shares now entering into the third year in those cases of 20%-30%, even more than 30%. By the third year, getting to 30% plus is a great track record towards what we think is ultimately a 50% plus peak share to get to the $1 billion mark. Really positive so far in those markets. Interestingly, too, we're taking proportional switches there. In that case, there's more Lumizyme patients. It's majority Lumizyme, some Nexviazyme, though, and then also naive patients. I think in many markets, we're standard of care for naive patients, which is great. Very healthy initial launch dynamics.
In the new countries, a few very interesting things to point out. First of all, for some countries, Italy, Portugal, Belgium, we think it'll look a lot like kind of the German and U.K. launches, so roughly contemporaneously with Nexviazyme, and we'll kind of be head-to-head. I think we'll see similar trends there. A couple of interesting countries to point out, though, we've talked a little bit about this, the Netherlands being the most important of them. In the Netherlands, you have a very concentrated, there's one reference center in the Netherlands. There's a handful of patients in other places, primarily one center that treats about 150 late-onset patients, largest single center in the world. This was one of the places where we took more time to negotiate to get to a better outcome.
The Netherlands Reimbursement Authority looks at efficacy and safety, of course, and then cost. They combine those two. They compared head-to-head Lumizyme, Nexviazyme, and Pombiliti-Opfolda. In the end, we were awarded first position in the Netherlands. What does that mean? They will work with the physician to switch the majority, looks like 60% or 70% of the patients in the Netherlands over to Pombiliti-Opfolda. That will be a, so that is like 100-ish patients that are scheduled to switch over to Pombiliti-Opfolda. Number one, of course, that is an important revenue opportunity. It is a whole host of patients we can treat, which is amazing. It will be the largest cohort of patients. It is from a center which is one of the top two or three centers in the world. They are really good at publishing data.
Imagine a year from now, and they've said it'll take them, because it is one center, it'll take them about 12 to 18 months to kind of go through that switch process. Imagine a year or two years from now, the influence that that body of data can have, not just on other physicians in Europe, but really around the world. That's a great example. Interestingly, Sweden, much smaller market, only 20-ish patients, but interestingly, similar outcome where we got first position through that process as well. All told, there are about 600-650 late-onset patients in Europe with Pompe disease, which is roughly double the eligible European patients from last year. Big increase in the number of patients, some really exciting specific country dynamics like the Netherlands, then really good execution in the other markets as well.
Yeah. Wonderful. Just speaking to the kind of investor feedback that we have sometimes heard is that the Pom-Op launch has been relatively moderate when compared to initial street expectations. Just on the outlook, given all these factors that are playing out and then very positive factors that are playing out for Pom-Op, is it fair to assume that in the next few months, there might be an inflection in how you see in Pom-Op sales?
Yeah. It's a great question. Again, I think the big factors that were driving that, and I think we would share in that assessment, number one was the initial delay with the FDA. It should have been launched two years earlier. Super frustrating. We lived that tale. We got there eventually. That led to the knock-on effect of this two-year switch dynamic that really nobody anticipated. Even the physicians described it differently until they were presented with it. We're working through all that. I totally agree. I think especially the second half of this year, both because of the dynamics in the U.S. and because of just the addition of more launch countries. Oh, by the way, we have others in Europe. We have Japan and Canada and Australia. There are other markets to add there as well.
Yeah, I think we will see an inflection in the ramp of revenue. Again, if we just take a big step back and just think about how is $100 million in the second full year of launch, I think it's pretty darn good. I think our job now is to execute against the new set of expectations. I think we can do that.
Sounds good. I know we could keep talking about this, but then moving to Galafold.
Yes. Yeah.
Galafold continues to maintain really strong growth momentum despite being seven years into the market already.
Yeah. Seven in the U.S. and nine next U.S. Yeah.
Wow. What are the factors that are driving this growth? What's your outlook for Galafold?
If you remember, and you guys have been part of the story for a long time, when we originally launched into the Galafold space, it was really about could we compete in switch patients because that was kind of the low-hanging fruit. They're the ones already coming in and getting reimbursed. You had to do the competitive switch. We fought long and hard and got to a place where now we are standard of care in switch patients. We are 80% or 90% market share. The vast majority of patients that come on to Galafold are the vast majority of patients who are on Galafold were switch patients originally. In that context, the market was 10,000 diagnosed patients, 5,000 treated, 5,000 diagnosed untreated. We were focused on kind of the treated market.
Fast forward to today, 80% or 90% of our patients coming on drug today are naive patients. They have not been treated before. Part of that is penetrating into the diagnosed untreated market. We're doing that for sure. Those patients do have a progressive disease. Eventually, many of them, if not most of them, will come on to treatment. They will add to the treated market, which, by the way, is more than double as well. Now it's 11,000 patients who are treated, 11,000-12,000. There are about 6,000-7,000 who are diagnosed untreated. You still have a huge pool of patients to work on there. The other piece, though, that I think was a hypothesis back then, but it's clearly bearing out, is we're just diagnosing more and more patients. What are the drivers there?
One driver of that clearly is just low-cost genetic testing. Shire, now Takeda, Genzyme, now Sanofi, and then Amicus. Now the other new players in the Fabry space are out there educating physicians that this is not just a kidney disease. It is a cardiac disease. It is a CNS disease. You have a whole host of specialties that are now more trained to look for patients. Whenever they suspect anything genetic, it is so cheap now to do a genetic test. When we first launched, genetic testing was like thousands of dollars per patient, which could be prohibitive in many markets. Now it is hundreds of dollars, if that. It is just a much lower barrier to just run a genetic test. Fabry is on the differential for a lot of those disease areas.
The second piece is that even though in the U.S., you only have a handful of states that do newborn screenings, actually 15% of the population of newborns is being screened through newborn screening. You're finding tens of patients a year through that process, just through the newborn screening. You do family screening, and there's typically three to five family members who you're also finding. That's hundreds of new patients just through those states alone that are being found through newborn screening. That's increasing the population. You're seeing more and more sophisticated diagnostic capabilities. We're supporting AI. There are some other companies that are doing that as well. I think the piece that maybe has been, again, it was hypothetical, but what we're actually seeing now is this just significant diagnosis rate that we think will continue and may even be increasing.
There was a really cool study from Emory that was published recently at a medical congress that looked at specific mutations. There's one mutation, A413T. Yeah. I got it right. Sorry. I'm looking at stuff, which is a significant portion of the population. They looked at that patient population and found that 30% on opioids, 40%. 40% of them were on opioids, okay, to control their pain, right, versus like 5%-6% of the overall population. Our very first male patient who's still on Galafold today, by the way, his biggest complaint was pain. He had been on opioids for 10 years. There was a really elegant poster that showed him weaning off of opioids when he came on to Galafold. It's a great example of where they just screened idiopathic pain and found these massive numbers of A413T patients carrying A413T mutation.
Just another example of where there have to be these big pools of uncovered patients. I really believe, back to your original question, where is this product going to go? Look, we've got 12 more years of exclusivity. We are already at $0.5 billion, we think, by the end of this year. I think this is a $1 billion+ opportunity. I do think there could be some upside as AI and other mechanisms get better and better and better at finding patients.
That's great. Okay. Last few minutes, we have to touch upon Dimerix.
Yes.
Can you walk us through the due diligence that went through selecting this particular asset for the licensing?
Yeah. Remember, we had said originally we wanted to bring in a de-risked late-stage asset that we could fit into our commercial capabilities. We were very open to regional deals, although to be fair, we thought there was probably going to be ex-U.S. deals primarily because a lot of companies in the United States are looking to leverage our ex-U.S. infrastructure. That still could be an opportunity for us. We think it will be over time. Dimerix, Australian company, had already licensed the rights in Europe and in Japan to DMX-200. Fit very well into our adjacent capabilities, rare disease, rare kidney disease, a lot of similarities with Fabry disease, significant unmet need. Over 40,000 patients suffer from FSGS. Typically leads to significant morbidity and mortality. No approved therapy. Lots of the things that we look for for the right kind of opportunity.
We looked at the diligence and said, "Okay. What does this look like?" We saw a very clear mechanism of action preclinically, so very straightforward from that perspective. We saw great phase I data, not just in FSGS, where 85% of patients had a reduction in proteinuria in the phase II, but also in multiple other diseases where they showed safety and efficacy on similar endpoints, so a broader population. We also got to take a confidential look at the patient-level data, which gave us further confirmation of the effect that we were seeing in the broad population there. They entered into a phase III, which is already underway. They were Dimerix before we did the deal.
Importantly, had done an interim analysis that showed a statistically significant benefit in the treatment arm versus the control arm for a subset of the population that had been enrolled to that point. Another de-risking event. We got a chance to look at some blinded data there too, which further supported the statistics. The last piece was you had the positive feedback from the FDA, which was a contingent to completing the deal. You saw just before we announced our deal, Dimerix announced the FDA feedback, which confirmed proteinuria as the primary endpoint with GFR as a supportive endpoint. Put all those things together, we believe high probability of success, high met need.
The last piece was we thought a very risk-reward-based deal, so relatively low upfront, $30 million to access the phase three data, which would get you to a billion-dollar-plus opportunity.
Okay. In terms of the FSGS market, we have a bunch of other companies like Apellis, Vertex, who are exploring different mechanisms for this disease. How do you think DMX-200 is differentiated over here statistically?
Yeah. So just as a reminder, this is kind of a cascade effect. You have hemodynamic impairment from an original insult to the kidney. It can be genetic, can be obesity or other comorbidities. That leads to a cascade of inflammation, monocyte macrophage activation, MCP-1 elevates, leads to proteinuria, GFR, scarring of the kidney. You enter into this feedback loop. I think very fundamentally, most of the other mechanisms are focused on either the hemodynamic aspects of the disease or some other part of the disease. DMX-200 focuses very much on the monocyte macrophage inflammation and elevated MCP-1. What we've seen in phase two is that by blocking the signaling of MCP-1, you can lead to lower proteinuria and a subset of the population that isn't addressed by some of those other mechanisms. We do think there could be synergistic applications of some of these different products.
We think we're in a very differentiated mechanism, a very important subset of the population. We think a very likely, high probability success phase three that acts specifically on that target area of the disease, which leads to a significant opportunity for patients.
That's really interesting. Just one last question on business development following Dimerix. Has your strategy changed? What's the outlook there?
I would say strategy has not changed. Like, Amicus is still kind of drying on the Dimerix deal. I would not expect anything right away. I do still think that in the backdrop of a highly leverageable ex-U.S. infrastructure, there is an opportunity to bring something into that part of the business. I think in kind of the medium or long term, like Amicus five years from now, we will be generating, again, free cash flow, profitability. I would like to see us use some of that to put towards new development opportunities in Fabry and Pompe or perhaps other disease areas. The Amicus of five years from now, significant revenue growth, profitability, more commercial products like DMX-200, and then hopefully some interesting products in the clinic as well.
Great. Thank you so much.
Thank you.
We're done with the session.
Of course. Thanks very much. Appreciate it.
Yeah. This was great having you. Thank you to everyone who's attended the session.
Thank you.