Amicus Therapeutics, Inc. (FOLD) Earnings Call Transcript & Summary

Yes. And these patients are really easy to find.

Yes. So of course, the ones that are being treated are coming in every other week. It's a little different than in Fabry, you can have a little bit of a different dynamic because there were so many diagnosed untreated patients in Fabry that we are now kind of going out to bring into the system. In Pompe, it's a higher majority of patients are being treated who are diagnosed. So in the United States, there are about 800 patients in Europe, in the U.K., there's about 1,500 patients that are treated. So that's a big chunk of patients to be able to switch. However, we do think that the entrance of a new -- and I know this is one of the places you want to prove on, for sure, in these rare diseases, when you see a new manufacturer come in, to a therapeutic area, you find more patients. And if you go and talk to physicians, whether it's through newborn screening or through screening like muster dystrophy clinics, Pompe disease is also underdiagnosed, not as probably not quite as much as Fabry, but certainly, there are more patients to find. And I think once we have another therapy out there from another manufacturer, we're putting our efforts towards finding new patients, I think we're going to grow the market over time that way as well.

Okay. Have you talked about how big the sales force for this will be?

Yes. So actually, it's the same team effectively that we have for Fabry. We're not adding any new sales reps or account managers outside the United States. When we looked at the numbers, about 1/3 of treaters overlapped, about 50% of centers overlapped and the vast majority, 80%, 90% of cities overlapped. So we're very confident that we -- number one, we know a lot of these people already because we're already calling on for Fabry. But number 2, we have good relationships with the centers. We're already in there. So where we did add a little bit of extra resource and it was less than 12 people globally, like I said, case management, a handful there, a handful of medical affairs folks like specific Pompe medical leads, and then a handful of patient education lays on in the United States where you could be in the field and do a little bit more supporting patients in their care journey. So very, very leverageable commercial organization.

Okay. Perfect. You will obviously announce final pricing when you get the approval. But in terms of a range, what are people modeling?

Yes. So in terms of our strategy, which we've been very clear on and was very successful with Galafold, our strategy is to maximize access to therapy, right? And the reason for that is you want to get through the reimbursement process as quickly as you can get to with the reimbursement authorities and then get patients on therapy. Maximize the number of patients on therapy. So with Galafold, we deployed a parity or modest discount strategy, modest meaning 5% or less. And it's really just to make sure that the value proposition of the payers is crystal clear. You have a new therapy, you're bringing value to them, you're charging them now more for it. And so that way you go through that process much more quickly than arguing for the next $5,000 of price, right, where you're trying to maximize profit. For us, it's maximized the number of patients on therapy as quickly as possible. It's been a super successful strategy, and we'll deploy that here as well. In terms of the price band in the United States, the price of standard of care is around $650,000, $675,000 list price, there's probably 20% list to net in the U.S. And then outside the U.S., it's more like 400 to 450 in Europe, and again, probably 20% list to net. So that's about double by comparison, the average cost of Fabry treatments.

Okay. Perfect. Let's talk about Europe actually for a few minutes and where you are with the launch there.

Great. Yes. So just as a reminder, it was a couple of weeks ago now, but we did get CHMP opinion recommending approval of Opodo, which is the enzyme stabilizer. Pombility, which is the ERT, had already been approved and then authorized by the EC as well. So the last step there in terms of full approval is getting to the EC decision on the small molecule. That's roughly a 67-day time frame from the CHMP opinion. Sometimes it goes a little longer to the next meeting, but that puts you in that kind of early Q3 time frame as well. So super excited for launch there. Same thing. Launch meetings are set. The team is trained and ready to go. We'll launch in Germany first, of course, because you have that kind of free pricing period we should come back to the U.K. because it's a really important market as well. But -- so -- and as I mentioned before, we have about 20 patients now on therapy in Germany. That number could trickle upwards as well because we have an open expanded access program. So we are adding a handful of patients there as we go. And what's different, I think, in Europe versus the United States, NextViejust looking at the reported numbers, they've had a much slower uptake for, I'm sure, a variety of different reasons, but much slower uptake. So I think there, it's really going to be more sort of head-to-head for patients. And I would note, too, the label, which has been published through the CHMP process is for all adult patients with late-onset Pompe disease. So there's no restrictions on experienced or naive patients there.

Okay. So I guess with that in mind, what do you think people are going to be most pleasantly surprised by regarding the pumping lunch? What don't we know?

The opportunities to move faster, certainly converting all the trial patients, which we talked about, I think continue to see more patients on therapy as we approach launch. We still have time. The EMS program is open in the U.K., and we're -- we have more patients in the queue. So just increasing the number of already on therapy patients will be great. I think there's -- if I look at the U.K. market, as an example, rightly, look, this is going to be competitive. We've competed against Genzyme and Takata for that matter, Samuday, in Takeda for in Fabry. So don't mistake my confidence for ingrate, they are clearly a big company they're going to do anything they can. What gives us the most confidence in terms of what's going to go well here is, I would say 2 things. The first is it's anecdotal, but there was a poster from the U.K. and one of the largest enrolling EMS sites. So I think they had 10 or 12 patients. They actually asked the patients how they were doing in their initial switch from Lumizyme. And it's not something you can promote on. It was an independent publication. But if you go read that and you hear what the patients are saying and what the nurse geneticists are saying about the switch process and how they're feeling on drug, that gives us great confidence that this is going to be -- this product is going to make a real difference for patients, which is important. The second piece is in a weird way, we have this like pure experiment on what happens in a head-to-head scenario between Nexviazyme, Lumizyme and ATGA. And if you look in the U.K., there are about 200 or so late onset patients who are on therapy on Lumizyme, both ATGA, and NexVizyme were approved for EMS at the same time, which is the early access medicine scheme. It's put in place so that you can get access to these novel therapies from submission through to the approval process. And we were approved at the same time, and we have had significant uptake, significant 30-ish patients right now with more in the queue who are on ATGA, very few less than a handful of patients went on to NexVizyme. You can't promote during that period, but the only thing you can do is offer it and it's totally inherent demand by the patients and the physicians. And there, that's your sort of 15%, 20% market share already of treated patients, and we haven't even launched the drug there yet. We haven't even promoted on it. So to me, I think, yes, we're not taking Sanofi lightly, we never would. It's great for patients to have choice, that's really important. But I think when we're out there promoting and we can talk to the data directly with physicians, I think you'll see a fantastic launch.

Okay. So maybe let's talk about Fabry for a few minutes as well. So that's your more mature launch. You've been on the market actually in Europe longer than you have in the U.S. So a question that I tend to get is, given that it's been you've been launching for a few years, where is the remaining upcycling to come from, and in terms of the trajectory, have you seen that recovery from the COVID impact start to kick in?

Yes. Great question. So yes, first of all, Fabry market, in general, is growing at a significant rate and continues to do so, and that's really, I think, driven by 2 things. The first is we just keep finding more patients.

And how are you doing that?

So Sanofi and Takeda have been out there sort of shaking trees for a long time. And I have to be honest, I didn't know how much impact we would have as a smaller company and newer therapy in increasing that diagnosis rate. But we're doing some really interesting work there. We found recently, I was in Europe a few weeks ago meeting with the team. And one of the general managers is doing a study which hasn't been published yet. In one of the largest markets in Europe that found less than 50% of Fabry patients had done a family screen. This is an x-linked disease. They're typically at least 4 family members who have the disease. Family screening is part of standard medical practice and in an advanced first world super high knowledgeable Fabry treatment system, less than half of patients were doing a family screen. So that's one thing we're doing, and we're finding patients that way. We're doing -- we've talked before about some of the high-risk screening like looking in MS clinics where we found a 5% prevalence of misdiagnosed Fabry patients. We call those kind of trapped patients because they're on the wrong track. And then frankly, we're also starting to do some really interesting AI work. Again, there's a country in Europe, which hasn't been published yet, where we're using artificial intelligence in a more of a closed system in this country and finding a whole host of interesting things, including finding more Fabry patients. And then likewise, in the United States, we've actually published on this with a company called OMI. We've started to do some work there, too. And I think we can start to deploy that hopefully over the next couple of years. So continue to find more patients. We're adding to that. We're the fastest-growing Fabry treatment. And so I think that tells you that not only are we finding more patients, but they're preferencing Galafold, which is great. And in terms of -- so then where is the growth coming from? Part of it is finding new patients. And I'll tell you exactly what we said was going to play out is starting to in the mature markets in Europe, starting to be in the United States. The majority of new patient starts are actually naive to therapy. Now they might have been diagnosed already, and there's a big pool of diagnosed untreated patients. We think there's still about 6,000 diagnosed untreated patients with Fabry out there. But some of them are also newly diagnosed. So -- and we're seeing -- we just said the first quarter was the highest net new patient starts in the last 4 years. So that growth is real and it's significant. That being said, if you look just globally at our footprint, we have about a 55% share of treated amenable patients. So you still have another 45% to switch, right? And we can get to 90% market share in our markets where we're more mature. So there's a ton of switch still to do, but it's in sort of Latin America, Asia Pacific, Central Europe, where we haven't been on the market as long. Turkey is a new country that's going to come on board. This year, we're doing pricing and reimbursement. And so there is some geographic expansion as well. So between finding and bringing new patients on therapy, switching patients, and then geographic expansion, there's tons of growth here left. And the market itself, you just take a big step back, the way I just kind of try to frame it is the market right now is sort of $2.2 billion global sales where we were $330 million of that last year. expected to grow to $3 billion by the end of this decade, 1/3 to half of patients have a mental mutations. So the patients are there. The market is growing healthily. It's just a matter of executing as we have done.

Okay. And so going back to those new regions that you've talked about focusing on, including Turkey, is it safe to say that their prices would be lower than what they are in the U.S. certainly and about comparing to the big EU countries?

Yes. So in many of those markets, you're at roughly not surprisingly, kind of U.S. and Asia Pacific tend to have some of the highest prices and then you've got Europe and then you've got kind of ROW LatAm and some of the lower GDP Asia Pacific countries are at a lower band. That being said, just because of the rate of growth is still really strong in our larger higher-priced markets, our average price per patient net still hovers around $200,000, and that's been pretty consistent. So yes, there are more patients at a lower price point coming from like Brazil or another good example, and Turkey would be a good one. It's balanced out by the continuing to grow the larger markets as well.

Okay. And so as you think about that landscape, how do you think about the resources that you have devoted to Fabry versus having to now pay attention to Pompe, do you think that you might have to make any tweaks or additions to your commercial team over the next year or so?

So I'm really looking forward to having more top-line revenue so we can do even more of the kind of diagnostic initiatives in Pompe, too. Pompe in particular, newborn screening is really effective in the United States. The majority of states now have Pompe and the Rusk and there are some really interesting work that looked at finding Pompe patients and then the importance of early treatment. And so one of our strategic objectives is to -- right now, our labels will be for late-onset patients, but we'll pursue pediatric and eventually in final upset as well. And I think what you'll see over the next 5 years is the realization that you need, even if you're diagnosing a late onset mutation through newborn screen in Pompe, they should be -- this is a progressive disease, they should probably be treated sooner. And I think you'll see the evolution of that. And as we expand our label, it will be good timing there. But no, I think we'll be able to do even more to invest in both Pompe and Fabry patient-finding activities because look, they should be diagnosed, and whether they have an amenable mutation or not, then at least the physician can make a determination as to whether or not they need to be treated.

Okay. So how should we be thinking directionally about OpEx going forward?

Yes. So it's a great point. And we made in Daphne, if you heard the call yesterday, pointed this out. So our OpEx guidance for this year is $340 million to $360 million. In the first quarter, though, we did $80 million in change in OpEx. And what we are very clear to say is that we shouldn't straight-line that for the year. There are 2 pieces of, I think, color to provide there. The first is you'll have some increase around the launch. And so that should be a Q2, Q3 number. And then we also have -- remember, we're very far along now in the Ireland manufacturing process with Wuxi, we're investing in PPQ batches and engineering batches there. And so you'll see some of that come in the middle of the year as well. And so we still reiterated our guidance of $340 million to $360 million. The other interesting change you'll see is because now Pompe is moving from R&D to commercial. Some of the R&D OpEx, which is really around some of the medical organization will move over to G&A. So you'll see kind of a shift over to G&A. It's not actually reflective of a significant increase in G&A. It's a reflective of recategories at some of the R&D OpEx to G&A. OpEx.

Okay. You brought up the Ireland facility, so let's just quickly talk about that. Now that you do have Wuxi cleared, I think it was part of your plan always to have Ireland come up. What kind of timelines are we looking at to that?

Yes. So right now, the plan is that you'll have commercial supply. So you go through the PPQ process than the licensure of that facility, commercial supply entering the supply chain in the end of '24 or first half of '25. That was by design because that's when you're sort of really getting into the meat of the European launches in particular. Over time, Ireland will actually be the dominant source of manufacturing. We're actually also moving the drug product manufacturing from China, which is in the same facility as Wuxi over to Europe as well. So you'll have an end-to-end supply chain that's European-based to have a second site of supply, but you'll also be independent of China to the extent that there continues to be sort of geopolitical or other challenges there. We'll, I'm sure, continue to use Wuxi as well. But over time, you'll migrate towards a majority coming out of Ireland. But then you're -- from a dual site perspective, you're derisked and again, even up and down the supply chain, it will be European-based.

Okay. Perfect. I think we're out of time. So with that, I'll say, thank you so much for the last 30 minutes. It was always good to catch up with you in person Thanks, everybody, for joining us here.

Thanks.