Amicus Therapeutics at Goldman Sachs Conference: Strategic Insights Unveiled

On Tuesday, 10 June 2025, Amicus Therapeutics (NASDAQ:FOLD) participated in the Goldman Sachs 46th Annual Global Healthcare Conference. The company discussed its strategic initiatives, focusing on its core products and future growth opportunities. While Amicus faces challenges in the macroeconomic environment, it remains optimistic about achieving significant milestones, including GAAP profitability and substantial sales growth.

Key Takeaways

• Amicus aims for GAAP profitability in the second half of 2025.
• The company targets $1 billion in combined sales by the end of 2028.
• Galafold is expected to generate $500 million in sales this year, with a long-term goal of $1 billion.
• Pombility/Upholda is anticipated to see 50% to 65% growth, aiming for $100 million in sales this year.
• The recent DMX200 acquisition offers potential blockbuster growth.

Financial Results

• Galafold:

- Expected growth of 10% to 15% this year.

- Sales aim to surpass $500 million, with a peak potential of $1 billion.

- Retains 12 years of market exclusivity.

• Pombility/Upholda (Pompe):

- Projected growth of 50% to 65% this year.

- Targeting $100 million in sales.

- Long-term goal of reaching $1 billion in sales.

• DMX200 (FSGS):

- Considered a significant growth opportunity with blockbuster potential.

• Overall Financial Goals:

- Achieving GAAP profitability in the latter half of 2025.

- Combined sales target of $1 billion by 2028.

- Revenue growth forecasted at 15% to 22%.

- Operating expenses expected to range from $380 million to $400 million.

Operational Updates

• Galafold:

- Holds 80% to 90% market share among switch patients.

- Market expansion with increased diagnosis rates.

• Pombility/Upholda (Pompe):

- Strong launches in six European markets.

- US insurance authorization process streamlined to under 30 days.

- Significant patient switching from Nexviazyme and Lumizyme.

• DMX200 (FSGS):

- Licensing agreement with Dimerix includes a $30 million upfront payment.

- Positive feedback from the FDA on Phase 3 progress.

Future Outlook

• Galafold:

- Confident in achieving a $1 billion sales opportunity, aided by AI diagnostic capabilities.

• Pombility/Upholda (Pompe):

- Revenue growth expected from new market launches and US dynamics.

• Tariffs:

- Minimal impact anticipated due to strategic manufacturing locations.

• FDA Reforms:

- Anticipated positive regulatory changes to benefit rare disease therapies.

• Business Development:

- Focus on acquiring late-stage assets and exploring new opportunities in rare diseases.

Q&A Highlights

• Macro Environment:

- Amicus believes risks are overstated and is well-positioned to mitigate impacts.

- Biosecure Act cited as an example of minimal impact from perceived threats.

• Competition:

- Strong switch data from competitors Nexviazyme and Lumizyme.

Readers are encouraged to refer to the full transcript for a detailed understanding of Amicus Therapeutics’ strategic plans and insights shared during the conference.

Full transcript - Goldman Sachs 46th Annual Global Healthcare Conference:

Bradley Campbell, President and CEO, Anicus Therapeutics: Bradley Campbell, the President and CEO of Anicus Therapeutics. Thank you so much for being here.

Unidentified speaker: Thank you for having us. Thanks to Goldman for hosting the conference. Nice to see everybody here today.

Bradley Campbell, President and CEO, Anicus Therapeutics: Thank you. And 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 half of the year?

Unidentified speaker: Sure. Yes. 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 $500,000,000 in sales this year.

We have twelve more years of exclusivity on our way to $1,000,000,000 in peak sales, so very important part of our growth story. We’ve guided to 10% to 15% growth this year for Galafold. For Pompe disease, we’ve recently launched our new therapy, Pombility and Upholda for the treatment of Pompe disease. We’ll pass $100,000,000 in sales this year. Our guidance is 50% to 65% growth, well on our way again to $1,000,000,000 in sales.

We’re very excited about that with some additional development opportunities in particular in the pediatric population. And then we have our brand new asset, DMX200, 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. So 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. And in a few short years, by the end of twenty twenty eight, we expect to see $1,000,000,000 in combined sales.

So 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.

Bradley Campbell, President and CEO, Anicus Therapeutics: That’s wonderful. But what part of Anakus’ story do you think The Street is not appreciating enough, given the recent stock movements of it fair

Unidentified speaker: to say all of it? Is that a fair answer to the question? So 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 and 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 macro perspective, I am 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 within 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 $2,000,000,000 global market today.

It’s growing to $3,000,000,000 over the next ten years. Again, we’re starting this year, we believe, at over $05,000,000,000 in global sales for Fabry disease. We’ve got twelve 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 and we could talk more about what those might be, but 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 piece of course is Pompe and I know people are laser focused on the launch and we can talk about why we have great confidence in the launch. But again, if you just take a big step back and say, okay, year of launch into what is today over $1,500,000,000 market, growing again to over $2,000,000,000 over the next ten years. We’re already at $100,000,000 based on our guidance and sales this year over $100,000,000 So we’re off to a great start, and we can talk about why we think we’ll get more traction there, but clearly we don’t get full credit for Pompe or maybe any credit for Pompe. And then maybe because FSGS is new to the story and we need to do some education 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 derisked late phase three product for FSGS, we’d be worth a lot more than I think we would get credit for today.

So look, I get the macro environment and happy to talk about why we think that we can work through those things, but 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, 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.

Bradley Campbell, President and CEO, Anicus Therapeutics: Definitely. And then if we just speak to the macro a 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?

Unidentified speaker: All the stuff. Yeah. Yeah. You know, Clearly the world, we all hate uncertainty and markets in particular hate uncertainty. I think part the issue is just macro uncertainty, 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, etcetera.

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. And the eventual bill was actually quite supportive of what would have been a very orderly transition to The US. So 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.

And 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. So tariffs are, you can debate the macroeconomic benefit or cost of tariffs, but as it relates to our business, what we’ve said very definitively is there is a nonmaterial impact on our business this year. Why is that? Because of all, the majority of our revenue comes from Galafold.

Galafold has a tiny, tiny cost of goods. It’s manufactured in Switzerland and so very, very little impact of any kind of potential tariff coming out of Switzerland. As it relates to Palmdale Ulfolda, we’re manufactured today in China. We’re moving that manufacturing to Ireland. It will enter The US supply chain in the back half of next year or early twenty twenty seven.

But even before then, for general supply chain continuity and security, we moved all of our commercial launch material to The United States already. So all of the material this year and a significant portion of what we have anticipating for next year is already in The US, so that’s not subject to tariffs. And 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. So it is not, by any stretch, a significant material issue that we need to be worried about. And oh, by the way, we’re already long down the road of reducing our cost of goods through our generation process, and 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.

So I think tariffs, again, are overblown as it relates to how they might impact Amicus. The one you mentioned is IP and transfer pricing. Good news is our IP sits in The United States, so there is no real issue there. MSN, that’s another one. Again, I’m not a trained economist, but 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.

And 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, 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 and typically rare diseases are excerpted from that. So 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. And then the last piece is the reality is if you were to say worst case scenario, let’s just say that MFN does pass and so for Medicare, 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. So again, do I want that outcome? No. Do I think it’s negative for innovation? 100%.

So I think there’s lots of different ways to do that and to work on pricing and access and affordability. But even in the worst case scenario, Amicus is actually fairly insulated from that outcome. So I think if you put all those things together, I get that there’s uncertainty, I get that that creates dislocations in value and 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.

So 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. And the question was, oh gosh, what’s gonna happen here? I think the reality is the new leadership has been very clear about supporting rare disease regulatory reform. And so I think the net net of all of this actually may be very positive reforms from a rare disease perspective.

And if you think about our new asset in FSGS, we very much could benefit from some of those changes.

Bradley Campbell, President and CEO, Anicus Therapeutics: That’s really helpful color. Now speaking to your guidance, so Amicus expects to achieve positive net income in the second half of ’twenty five. What is baked into this guidance? And what are your expectations around on 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 to now?

Unidentified speaker: Sure. Thank you. Yes. So really the key drivers of how you get to the GAAP net income, of course we delivered our 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% to 22%, so very strong top line growth.

On the bottom line, or excuse me, the expense line, even with the new licensing payment from Dimerix, we’ve guided to $380,000,000 to $400,000,000 in expenses. So significantly more revenue than expenses gets you to that GAAP income in the of this year. And 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 generation manufacturing.

But by and large, that will be largely flat or maybe some inflationary increases with a significant top line growth. So that will lead to naturally going from GAAP net income in sometime during the half to a full year to EBITDA and positive free cash flow. That’s what 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 and a lot of them need to raise capital. And that’s where you really get into trouble and that’s where Amicus is, I think, has differentiated itself.

So 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, and I know we’ll talk more about that whole opportunity. But the way the deal was structured, we don’t have to invest a single dollar into R and D until we turn over the phase three data card. So it’s very smartly structured in the sense of, yes, we paid the $30,000,000 upfront for a billion dollar potential asset. We think that’s a great price to pay, and we don’t have to invest anything else from a development perspective at all during the transaction, but even from a commercial perspective, we don’t have to make those investments until we see a positive outcome.

So we think it’s very smartly structured and that’s another way to keep those expenses relatively flat where we grow the top line.

Bradley Campbell, President and CEO, Anicus Therapeutics: Now maybe we pivot to warm up. Sure. In the first quarter EPS, Amicus lowered its guidance for FY ’twenty five, and that was due to multiple headwinds. Could you speak to your or the headwinds that contribute to this decision as well as your confidence in achieving this updated guidance?

Unidentified speaker: Sure. With Galafold, it’s a much more straightforward picture and I know we’ll come back to that in a minute. But in a launch year, there’s a number of different factors that are going on. One of which, of course, is the launch cadence. So 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.

Now 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 of launch. But at the end of the day, you never get a chance to reset that price in Europe. They only go down from there. And so 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. So that was one contributing factor.

And the good news is multiple of those countries have now gotten to reimbursement and launch and I’ll come back to that in a minute, to your point about confidence. The piece was this VPAG, VPAAS thing. So one thing to remember is that this has been one way or another, VPAG or VPAAS or some form of it in The UK which is a mandatory tax on all pharmaceutical goods. Has happened every year since we’ve been commercial with Galafold. So 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 UK, ABPI, negotiates on industry’s behalf to an agreed upon rebate rate. And typically they’ll give you guidance at the end of the year to say, look, it’s gonna come in at 10% or 15% or 7%. And then they finalize the negotiation the next year, but you have some ability to see what’s gonna happen in the following year. Every year we’ve done this since we launched Galafold and 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 to 15, and this is all public by the way, a twelve to 15% VPAG rate.

And in the end it was 23%. So 50% higher than what they had guided us to. 23% is also, I think it’s the largest ever, at least since we’ve been launched, VPEG rates. So it was both an outsized rate in the place and it was 50% more than what they told us. Unfortunately for us, The UK is our or largest market for Galafold and Pompe, and so it just has an outsized impact.

So when we look at those two things, launching a quarter late in what ends up being five or six markets, and then having this haircut come off the top of your largest market in Pompe, we just didn’t feel like we could catch up to that over the course of the year. I still think that 50% to 65% growth is fantastic. Again, more than 100,000,000 in the full year of launch, that puts us on our way to what we think is a billion dollar plus opportunity. But I get, especially in this market, that that was, people were, you just get an outside reaction when the whole world is seemingly in a negative place. What gives us confidence in going forward?

So a few things. 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. So April and May now look like they may be the two largest months so far. And that’s both in The United States specifically, which is really important because it’s the highest priced market.

But also now again, you’ve got six new countries contributing to the launch in Europe, in particular Netherlands, Italy, Portugal, Belgium, Czechia, and Sweden. So six new markets that are all doing really well. So when we look at kind of that trajectory, when we look at the new six markets that are there and some other I know we’re going drill down on a couple of different factors, I’ll provide more detail in a bit. But everything we see right now says that number one, we’re on track to deliver a strong second quarter and then more importantly, we’ll have that acceleration in the second half that we expect to see.

Bradley Campbell, President and CEO, Anicus Therapeutics: Then speaking specifically to The U. S, can you speak to the launch dynamics there and in terms of reimbursement as well as what you’re seeing on the competitive side with NexVizem?

Unidentified speaker: Yeah, so a few important things in The US. So 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’s a serious delay. We didn’t think it would be as impactful it was. What we were surprised by was in our early market research, physicians and patients said, hey, if I’m not doing well on any therapy, whether it’s Nexizyme or Lumizyme, are the two competitors’ products, the competitors’ two products, I would switch within six months or maybe a year.

As it turns out, now that we’re 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. of all, really it turns out that patients aren’t coming to see their physicians more than once a year, very rarely. And so if you’ve just switched to patient, you wanna wait at least a year to see them once and then probably a year to see them again so you have some sort trend or feel for how they’re doing.

So to be fair, that was a surprise to us and 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? Well, of all, when we launched last year, there was only ten percent of patients started the year. Seventy percent of patients were on NexpiZyme, but only ten percent 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 gonna be about seventy percent of patients. So the patients moving into that switch window is gonna be about seventy percent. So much, much bigger pool of patients. What else are we seeing?

Well, 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 and the balance has been Miazyme. Why does that matter? Well, one thesis, one bare thesis would be, well, you’ll never switch a Next Visme patient.

So those are actually lost to you and that’s not true at all. In fact, we’re switching, a majority of patients are coming from Next Visme. Why? Because that’s the biggest pool of patients to switch from. So we know proportionality is driving the switch, so that’s good.

The piece is increasing data and then real world evidence. There’s two pieces of that. The 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. And remember, we’re the only drug that has shown clinically meaningful and statistically significant benefits when people switch from Lumazyme to a new product.

So those data continue to be strong and growing. But the other piece of course is the real world evidence and that’s showing up in case studies from Lumizyme to Palmop, but also now case studies from Nexizyme to Palmop increasingly supporting the switch in outcomes when people move to Palmop. So we think that evidence will continue to grow and continue to help support that. We’re also seeing increasing breadth and depth. So more physicians are prescribing the time and more physicians are prescribing for the or or time.

So that’s really powerful. And then on the insurance side of things, we’ve actually gotten that down to, it’s less than thirty days on average for prescription to insurance authorization and then about two weeks to get to the infusion. So all of those things are going in the right direction. What’s that translating to? Well, it’s translating at least initially to this acceleration in the highest numbers of new commercial starts in The US.

So more to come, of course. You’ll get the benefit of that again, more weighted to the half. But everything that we wanna see, all the KPIs we track are headed in the right direction.

Bradley Campbell, President and CEO, Anicus Therapeutics: Great. And then if we speak about the ex US dynamics, including your outlook for the launch countries in the near term.

Unidentified speaker: Yeah, so in the original countries, so UK, Germany, Spain, very strong uptake. In many cases, we’ve been on the market either at the same time or even slightly earlier than Nexizyme and there we’re seeing market shares now entering into the year in those cases of 20 to 30, even more than 30%. So by the year, getting to 30 plus percent is a great track record towards what we think is ultimately a 50 plus percent peak share to get to the billion dollar mark. So really positive so far in those markets. Interestingly too, we’re taking proportional switches there, but in that case, there’s more Lumizyme patients.

So it’s majority Lumizyme, some Nexvi Zyme though, and then also naive patients. And I think in many markets, we’re standard of care for naive patients, which is great. So very healthy initial launch dynamics. In the new countries, a few very interesting things to point out. So of all, for some countries, Italy, Portugal, Belgium, we think it’ll look a lot like kind of the German and UK launches.

So roughly contemporaneously with Nexvirozyme and we’ll kind be head to head. So I think we’ll see similar trends there. A couple of interesting countries to point out that we’ve talked a little bit about this, The Netherlands being the most important of them. So in The Netherlands, 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 is one of the places where we took more time to negotiate to get to a better outcome. The Netherlands Reimbursement Association or authority looks at efficacy and safety, of course, and then cost. And they combine those two and they compared head to head to head Lumazyme, Nexizyme, and Pomodiliopfolda. In the end, we were awarded position in The Netherlands. What does that mean?

They will work with the physician to switch the majority, it looks like sixty or seventy percent of the patients in The Netherlands over to Pommiliatfolda. That will be a, so that’s like one hundred ish patients that are scheduled to switch over to Pommiliatfolda. Number one, of course, that’s an important revenue opportunity. It’s a whole host of patients we can treat, which is amazing. It’ll be the largest cohort of patients.

It’s from a center which is one of the top two or three centers in the world. They’re 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 twelve to eighteen months to kinda go through that switch process. But 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. So that’s a great example and interestingly Sweden, much smaller market, only 20 ish patients, but interestingly, similar outcome where we got positioned through that process as well.

All told, there are about six hundred to six fifty late onset patients in Europe with Pompe disease, which is roughly double the eligible European patients from last year. So big increase in the number of patients, some really exciting specific country dynamics like The Netherlands, but then really good execution in the other markets as well.

Bradley Campbell, President and CEO, Anicus Therapeutics: Wonderful. Just speaking to the kind of investor feedback that we have sometimes heard is that the Palmop 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 perm op, is it fair to assume that in the next few months there might be an inflection in how you see in form of sales?

Unidentified speaker: Yeah, it’s a great question. And 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 in the FDA. It should have been launched two years earlier. Super frustrating. We lived tail, but we got there eventually.

But that led to the knock on effect of this two year switch dynamic that really nobody anticipated. And even the physicians described it differently until they were presented with it. But we’re working through all that. So I totally agree. I think, you know, especially the half of this year, both because of the dynamics in The US and because of the the just the addition of more launch countries.

And, oh, by the way, we have others in Europe, and then we have Japan and Canada and Australia, so 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 like how is $100,000,000 in the full year of launch, I think it’s pretty darn good. And I think our job now is to execute against the new set of expectations. And I think we can do that.

Bradley Campbell, President and CEO, Anicus Therapeutics: Sounds good. I know we could keep talking about this, but then moving to Gallifold.

Unidentified speaker: Yes, yeah.

Bradley Campbell, President and CEO, Anicus Therapeutics: Gallifold continues to maintain really strong growth momentum despite being seven years into the market already.

Unidentified speaker: Yes, seven in The U. S. And nine ex U. S, yes.

Bradley Campbell, President and CEO, Anicus Therapeutics: Wow. So what are the factors that are driving this growth? And what’s your outlook for Gallifold?

Unidentified speaker: So if you remember, and you guys have been part of the story for a long time, when we originally launched into the Scalifold space, it was really about could we compete and switch patients? Because that was kind of the low hanging fruit. They were the ones already coming in and getting reimbursed, and you had to do the competitive switch. And you know, 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 were on Galafold were switch patients originally. In that context, the market was 10,000 diagnosed patients, five thousand treated, five thousand diagnosed untreated. So we were focused on the kind of the treated market. Well, fast forward to today, eighty or ninety percent of our patients coming on drug today are naive patients, so they have not been treated before. Part of that is penetrating into the diagnosed untreated market and 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 treatment. So they will add to the treated market, which by the way is more than double as well. So now it’s eleven thousand patients who are treated, eleven thousand to 12,000, and there’s about six thousand to seven thousand who are diagnosed untreated. So 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, which is we’re just diagnosing more and more patients.

So what are the drivers there? One driver 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’s a cardiac disease, it’s a CNS disease. And so you have a whole host of specialties that are now more trained to look for patients, but whenever they suspect anything genetic, it’s so cheap now to do a genetic test. When we launched, genetic testing was like thousands of dollars per patient, which could be prohibitive in many markets.

Now it’s hundreds of dollars, if that. So it’s just a much lower barrier to just run a genetic test and Fabry’s on the differential for a lot of those disease areas. The piece is that even though in The US you only have a handful of states that do newborn screenings, actually fifteen percent of the population of newborns is being screened through newborn screening. And you’re finding tens of patients a year through that process, just through the newborn screening. And then you do family screening and there’s typically three to five family members who you’re also finding.

So that’s hundreds of new patients just in those states alone that are being found through newborn screening. So that’s increasing the population. And then you’re seeing more and more sophisticated diagnostic capabilities. We’re supporting AI. There’s some other companies that are doing that as well.

So I think the piece that maybe has been, again, it was hypothetical, but 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 and there’s one mutation A four thirteen. C. Yeah, got it right, sorry, I’m looking at it, yeah. Which is a significant portion of the population.

They looked at that patient population and found that thirty percent on opioids, forty percent. Forty percent of them were on opioids, okay, to control their pain, right? Versus like five to six percent of the overall population. Our very male patient who’s still on Galafold today, by the way, his biggest complaint was pain. He had been in opioids for ten years and there was a really elegant poster that showed him weaning off of opioids when he came onto Galafold.

So it’s a great example of where they just screened idiopathic pain and found these massive numbers of A four-one-three T patients carrying A four-one-three T mutations. So just another example of where there has to be these big pools of uncovered patients. So I really believe, back to your original question, where is this product gonna go? Look, we’ve got twelve more years of exclusivity. We are already at half a billion dollars, we think, by the end of this year.

I think this is a billion dollar plus opportunity. And I do think there could be some upside as AI and other mechanisms get better and better and better at finding patients.

Bradley Campbell, President and CEO, Anicus Therapeutics: That’s great. Okay, last few minutes, we have to touch upon Dimerix.

Unidentified speaker: Yes.

Bradley Campbell, President and CEO, Anicus Therapeutics: And can you walk us through the due diligence that went through selecting this particular asset for licensing? So

Unidentified speaker: remember, we had said originally we wanted to bring in a derisked late stage asset that we could fit into our commercial capabilities. And we were very open to regional deals, although to be fair, we thought there was probably gonna be ex US deals primarily because a lot of companies in The United States are looking to leverage our ex Symphosate. 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 two hundred, fit very well into our adjacent capabilities, rare disease, rare kidney disease, a lot of similarities with Fabry disease, significant unmet need.

So over forty thousand patients suffer from FSGS typically leads to significant morbidity and mortality. No approved therapies, so lots of the things that we look for for the right kind of opportunity. And then we looked at the diligence and said, okay, what does this look like? We saw a very clear mechanism of action pre clinically, so very straightforward from that perspective. We saw great phase two data, not just in FSGS where eighty five percent of patients had a reduction in proteinuria in the phase two, 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. Then they entered into a phase three which is already underway, made Dimerix before we did the deal, and 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. So another de risking event and we got a chance to look at some blinded data there too, which further supported the statistics. And then the last piece was you had the positive feedback from the FDA, which was a contingent to completing the deal And 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. So put all those things together, we believe high probability of success, high unmet need.

And then the last piece was we thought a very, very risk reward based deal. So relatively low upfront, 30,000,000 to access the phase three data, which would get you to a billion dollar plus opportunity.

Bradley Campbell, President and CEO, Anicus Therapeutics: Okay. And then just in terms of the FSGS market, we have a bunch of other companies like Appellis, Vertex, who are exploring different mechanisms for this disease. How do you think DMX200 is differentiated over here?

Unidentified speaker: So just as a reminder, this is kind of a cascade effect. You have hemodynamic impairment from an original insult to the kidney, be genetic, can be obesity or other comorbidities. That leads to a cascade of inflammation, monocyte macrophage activation, MCP-one elevates, leads to proteinuria, GFR scarring of the kidney and then 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 is focused very much on the monocyte macrophage inflammation and elevated MCP-one.

What we’ve seen in phase two is that by blocking the signaling of MCP-one you can lead to lower proteinuria and a subset of the population that isn’t addressed by some of those other mechanisms. So we do think there could be synergistic applications of some of these different products, but we think we’re in a very differentiated mechanism, a very important subset of the population, and we think a very likely, a high probability of success phase three that acts specifically on that target area of the disease, which leads to a significant opportunity for patients.

Bradley Campbell, President and CEO, Anicus Therapeutics: That’s really interesting. And just one last question on business development following Dimerix. Has your strategy changed? What’s the outlook there?

Unidentified speaker: I would say strategy hasn’t changed. Like, Ink is still kind of drying on the Dimerix deal, so I wouldn’t expect anything right away. I do still think that in the backdrop of a highly leverageable Ex U. S. Infrastructure, there’s an opportunity to bring something into that part of the business.

And I think in kind of the medium or long term, like Amicus five years from now, we’ll 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. So the amicus of five years from now, significant revenue growth, profitability, more commercial products like DMX200, and then hopefully some interesting products in the clinic as well.

Bradley Campbell, President and CEO, Anicus Therapeutics: Great, thank you so much. Thank done with the session.

Unidentified speaker: Of course, thanks very much, appreciate it.

Bradley Campbell, President and CEO, Anicus Therapeutics: Yeah. This is great having you, and thank you to everyone who’s attended the session.

Unidentified speaker: Thank you.

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