Hi, everybody. Good afternoon. Welcome to day one of the Cantor Healthcare Conference. Very happy to be hosting Amicus Therapeutics. We have Bradley Campbell, the President and CEO. Thanks so much for being here today. Thank you, Kristen. Really appreciate you coming. So let's dive right in. I think a lot of people are familiar with Amicus, but we'll start with your Pompe disease asset. We're coming up on your one-year anniversary of an FDA approval. In fact, you got your approval the day after our healthcare conference last year. Timed it almost perfectly. Initially, investors were worried that you could face more threats with the gap between the approval time with Nexviazyme. Why don't you believe that's, like, the case now that we're a year out? Yeah, it's a great question, so first of all, thank you, Kristen, to you and your team. Thank you to Cantor for having us here today. We're really excited. I think Amicus is in a great place right now. We're looking forward to executing, and we'll talk a lot about, you know, the growth drivers for the company. In terms of Pompe specifically, I think especially highlighted by Sanofi getting a jump start on their launch, I think there were really two central questions that we had coming into the launch that we feel a lot more comfortable about now that we've seen a year's worth of data. First was, look, if you believe that Nexviazyme was a better product than Lumizyme, and everybody switched over to Nexviazyme, maybe they would be kind of lost forever to come on to Pombiliti Opfolda. So once you switch to Nexviazyme, you can never get those patients. And I think what we've seen, both in the United States but globally, is that we are able to switch Nexviazyme patients about proportionally to the population that exists. So in the U.S., as an example, we estimate something like 60% or two-thirds of patients have now switched to Nexviazyme, and 60% to two-thirds of our switch patients have come from Nexviazyme. So to me, that says, "No, they're not lost forever," and in fact, we were actively looking to continue to switch those patients over. I think the other question is maybe one more of timing, which was, well, maybe because you were approved third, then every patient is gonna have to kinda step through Lumizyme and then on to Nexviazyme, and then maybe onto Pombiliti Opfolda. Is that like a four-year or six-year or eight-year...? Like, how long is that gonna take? And I think we've also seen is, in fact, again, because we're taking proportional numbers, in the U.S., we are getting a minority of Lumizyme patients because they're the minority of patients that we can draw from. But outside the U.S., where Lumizyme is still the dominant, or I guess under the brand name Myozyme, there is the dominant market share, we're taking majority of our switch patients from that product. So I think if those were the two big worries that investors had and that we might have had going into the launch, I think we've seen enough data now that says, in fact, those aren't the case. And then, do we have a sense for these patients that are switching from Nexviazyme, how long they were on that therapy for before they switched? Yeah. So we did a look at. You know, once we got to a significant number of switch patients, we looked at least the patients in the United States, where it's a little bit easier to get to that level of detail. And it looks like patients were on a year or two years on Nexviazyme before they switched to Pombiliti Opfolda. You know, that's a place where we're continuing to focus, and we can talk more about kinda how you can shorten that timeframe. But at least initially, it looks like that's the case, and then a minority were on less than a year. I think that clearly makes a question about durability. I mean, with standard of care Lumizyme, we've always known because it's been a standalone for so long, right, that it's an issue. Obviously, we're still learning for the newer therapies, but even though you're only a year commercialization, you do have some longer term data. I would imagine a lot of the patients on trial are no Mm-hmm . on commercial drugs. So is there any sense you can give us about what you're seeing over durability? Do you think this represents the best-in-class drug now, based on that? Yeah. So you're right, durability, long-term data is always, I think, you know, important to physicians and, and patients, too, of course. We have published on our Phase I/ II data set. I think that was out to four years, and those data suggested that the impact we had in Phase I/ II was generally durable across the key endpoints that we measured in that study. We've shown two-year data from our PROPEL study, and again, I think we showed durability in those outcomes. We're coming up now on actually a four-year data set, so I think sometime in the next maybe 12 months or so, we might see that come out. I think that will continue to be important to physicians and patients to understand the course of the drug, but so far, it has looked like those changes we saw in the Phase I, II study initially and in the Phase III study initially have been durable. How important is that in your conversations commercially now? Oh, I, you know, I think it's critically important. I think it starts with the fact that we're the only product in the controlled Phase of a study that's shown a benefit in patients switching from standard of care- Sure and that was kind of the anchor data we saw in the Phase III study. I do think, though, the longer you see those improvements go out, you know, the more and more you'll get conviction on what the drug has the potential to do for patients. I do think, too, though, it's also real-world evidence and real-world usage. So it's one thing in a clinical study to kind of follow those patients over time, but I think physicians and patients also wanna see what happens in their clinic, in their hands, with their patients, and that's where, you know, it's not data that's gonna show up in the label, but every time a physician or a patient has a positive experience, I think that gives that physician more and more confidence to use the product again in other patients. That's where following things like depth of prescription, so prescribing again, becomes so important. We'll do everything we can, both through case studies, so kind of N- of-1, small cohorts, but also through the registry that we're doing, through the EAMS registry in the U.K. in particular, to show what happens in the real world when patients are using Pombiliti Opfolda. One thing I've been very impressed with, with Amicus for Galafold, is every January, you give us guidance. Every single year, you've either met that guidance or exceeded it, and in my humble opinion, I think one of the reasons is you know these trends inside and out, and you're continuously learning. So how important is that driver when you think about that, and this is the first year you've given us a sense for Pompe, and how important is kind of that read-through to this indication, as you know, year over year, you continue to think about where the commercial opportunity is that year? Yeah, I appreciate that, and I think it's, you know, what we've... From a philosophical perspective, we've always tried to set, you know, reasonably ambitious targets- Yeah. and then do everything we can to execute and beat those targets, and with Galafold, I think we've as you said, and I appreciate you acknowledging it. Yeah We have a great track record of delivering on what we've said we would do, and in fact, you know, consistently, recently, actually outperforming even what we thought we could do. A big part of that, of course, is the product itself. A big part of it, I think, is the quality of our team, our global commercial and support teams, medical and others, that are out there supporting the products. And then I think it's also kind of management credibility and that philosophy that we've set. So I am very confident, you know, this year and the guidance we've given with Galafold, the revised upward guidance, actually, with Galafold. I'm very confident in delivering with Pombiliti Opfolda, and my hope is that we can continue to build momentum there as well and, you know, next year and beyond, continue to outperform. So thinking about the numbers you've provided for this year, can you walk us through essentially what was baked into those thoughts, and what are some things that may be like swing factors, where there may be potential upside, but then on the other end, things that can also be of concern? Sure When you think about the numbers this year? Yeah. So we for Pombiliti Opfolda, we gave a guidance of $62 million to 67 million in global sales, and what we had said was, "Look, we needed enough for, you know, time under our belt to start to understand where the trends were taking us," and we set that guidance in after our Q1 call. I think we did a nice job with delivering $16 million in global revenue in Q2. A few things that I think are that we're watching for, the first is, in the United States, are we continuing to narrow that time from prescription to infusion? What we had said was, we started with a goal of, like, 90 days at launch, and we're hoping to get down to 30 to 45 days, and what you're seeing is those trends are getting better and better. So that's one piece which is just sort of shortening that sort of waiting period with the queue of patients. The second thing is frankly just the rate of new patient acquisition. I think you saw we announced in Q2 186 patients who were either prescribed or on treatment, and 174 patients that were on treatment at that time. I think you wanna continue to see those, you know, those patient numbers continue to grow. The next piece is launching in new markets. We did launch in Q2 in Spain and saw a really nice uptick there. Importantly, we actually launched slightly ahead of Nexviazyme there, so I think that was a great tailwind for us. And then the last one is continuing to work through the reimbursement process in Europe and bringing more countries on board. That will be less important for revenue because they'll be sort of really towards the back part of the year, but it will be important for uptake next year. The things we're watching for that would push us, you know, maybe more towards the bottom of that $62 to $67 is, I think, more than anything, getting through the summer months. You know, summer can be slow. It's an infused product, et cetera. So, you know, some people said, "Wow, you did $16 million in Q2. If you just fast-forward that rate, you should be, you know, well above your range," and you know, we still don't have a full year yet, so I would just say, give us time to perform, and I know we can do it. But I, I'm very confident in the range we set out, and again, hope we can continue to kind of execute the way we have in the past. What's the upside in terms of naive patients? And then, when I mean naive patients, I'm saying ones that are just getting diagnosed. I mean, I sound like a broken record here, but, like, one of your first slides in your deck, it just sends a powerful message. There's only 10,000 Fabry patients diagnosed a decade ago, and now you're at 17,000? That's a massive jump for a short period of time. Is this something, you know, maybe that we could see in Pompe, and maybe not even to that extent, but just in general, more patients getting diagnosed, especially with two companies with therapies Yes working together, right, to identify? Yeah, I think one, you know, clear trend you see across lysosomal disease therapies is this kind of steady growth over time. You know, you rarely see, like, the massive one or two-year uptake that you see in some therapeutic areas, but you see real durability Yeah you know, decades of durability. And why is that? It's twofold. Number one, it's because these diseases are classically significantly underdiagnosed. And number two, it's because you... You know, it's really hard to develop therapies in these areas, and you don't see a lot of competition, and so I think that generally will define the LSD markets broadly. In Fabry, as you said, it's a significantly underdiagnosed market. There may be five or even 10 times more undiagnosed patients out there, and we'll, you know, we can talk more about kind of how do we unpack that and what the impact is on Galafold. But for Pompe, for sure, diagnosis will fuel growth in that market as well. It's a $1.2 billion market today. I think if you assume even conservative growth rates, it becomes a $2 billion market, you know, in the turn of the decade and then grows from there. No real competition on the horizon, and yeah, I do think part of it is the introduction of more efficacious therapies. I think in most therapeutic classes, as more players come in, you tend to find more patients. I think broadly we're benefiting from, you know, cheaper diagnostics, more educated physicians, a greater awareness that there are more patients to find, and so I think that is also fueling the growth. So between those two things, you just have a healthy, growing market and hopefully now multiple choices for patients to see better outcomes. ... Okay, thanks. Yeah, really, really interesting stuff. And then as you're undergoing country-by-country reimbursement discussions for Pombiliti and Opfolda, what can you say about how regulators are looking at the pharmacoeconomic cases and how this is translating to pricing? Yeah. So Amicus, when we launched Galafold, and now we've put this to practice with Pombiliti Opfolda as well, our focus has been on, I think, creating a clear, compelling value proposition and then delivering that to payers. Why is that, right? It's because we believe that faster access leads to better outcomes for patients for sure, but also increases sort of the area under the curve because you're bringing patients on sooner in the process. What do I mean by that? So Galafold went through the reimbursement landscape much faster than average, if you look at sort of industry averages, and with Pombiliti Opfolda, I think we're doing even better than that. NICE, for example, and this: they put out a press release to this effect: it was the Pombiliti Opfolda was the first therapy to be approved or recommended for reimbursement by NICE prior to MHRA approval. First ever product to go or therapy to go through that process. Similarly, in Germany, you know, you sort of launch with that free pricing period. And so you set a certain price, and then typically, when you're going through the AMNOG process, which is the reimbursement process, you end up on some discount off of that price. Again, I think we may be the first or one of the very few products that actually, when we landed on a final price, it was the same price we launched at. So very clearly, the healthcare authorities are recognizing the value that we think we're bringing with the product. Now, we have multiple countries still to go through in Europe and around the world. It's getting increasingly harder and harder to go through that. Yeah ... landscape, which, you know, in this community, we all follow. But I think our track record has been, you know, incredibly positive. And again, I think it's more valuable to bring patients on sooner than it is to argue for that last dollar of profit and take longer to get there. Okay, thank you. Let's move on to Galafold. Where do you think the initial mix will end up in terms of the split between patients, naive patients switching from other drugs as more and more patients become diagnosed? Yeah. So, and you referenced it earlier. Yeah. When we first launched the product, we estimated there were 10,000 patients diagnosed globally with Fabry disease. About half of them were on treatment, about half of them were diagnosed untreated. Fast-forward to today, there are 11,000 treated patients, 6,000 diagnosed untreated. A few really interesting dynamics you know, sort of playing out through that. First and foremost, when we launched Galafold back in the day, we and you could go back and look at our slides, and we said very clearly the focus is on switch patients. That's the world we're primarily in in Pompe today. Right. It's the low-hanging fruit. They're already getting reimbursed for their therapy. All you have to do, all you have to do is convince them that your therapy is a better choice, right? So if you look at, you know, the first three or four years of the journey with Galafold, the vast majority of patients were switch patients. Over time, what we predicted was eventually that would be more like fifty-fifty, and then in the end, the majority of growth would come from diagnosed, untreated patients or naive patients, and that's kind of where we are today. If you look at the U.S., the U.K., Germany, the markets we've been in the longest, 80% of our new patient starts this past year have come from naive patients, and they're either diagnosed, untreated or newly diagnosed. So that tells me that, you know, we're clearly finding more and more patients out there, and that's gonna be a huge growth driver to come. If you look today to two numbers, the mix of switched, naive patients on Galafold is a little over 40% of our patients are switch patients, and then the remainder have been patients naive to therapy. So we're already now in that major growth Phase. Interestingly, though, we are still at about a 60% global share of treated amenable patients, which means we still have about 40% of patients on ERT that we can switch, so that still is a growth driver. And then I think in the long term, you're really talking about a market that's growing at a healthy clip. If you just apply, you know, gosh, if you take half of the current market growth rate today, this becomes a $3 billion market again at the turn of the decade. And that's how you get to, you know, how do. You know, people ask: "Well, how does this get to be a billion-dollar product opportunity?" You get there because the market grows to over $3 billion, and we capture, you know, a third to a half of the market. What's the hindrance of that six thousand patients that are not on any drugs? Are they not severe enough? Are they ineligible for Galafold and don't wanna go on another drug? What's the breakdown of that? Yeah, I think at this point, what's fascinating is that as we find more and more undiagnosed Fabry patients, a bigger majority of them actually have amenable mutations. Why is that? It's because they tend to be later-onset patients, which is why their diagnosis typically takes longer, because they don't show the classic signs and symptoms, and late-onset patients tend to be more amenable. So if you think about that six thousand patients, we actually think, you know, if when we first launched, probably a third of patients had amenable mutations, it could be a half or even more. The literature would suggest it could be even, like, 80% or 90%, but if you just say half of that, that's still three thousand patients to sort of target with Galafold. Why aren't they on therapy today? Part of it is, it is a progressive disease, and if they have just been diagnosed, they may not be severe enough. Sure ... to be, to warrant the treatment criteria that exist in a number of countries. There are some patients, in particular, if they're only eligible for ERT, who are, you know, it's oftentimes the women are caregivers. They're caring for their parents, for their children, because it's an X-linked disease. And again, if they have amenable mutation, you can see why Galafold could be a great solution for somebody who's kind of in this really intense care time. And so, you know, part of it is just timing issue, part of it is, you know, they're literally newly diagnosed. Sure. And so it's just getting to grips with having a progressive disease and should they go into therapy. But from our perspective, you know, the vast majority of those patients will progress to needing therapy, and if they have an amenable mutation or standard of care for those patients. What does that timeline typically look like to the point of, you know, maybe you're newly diagnosed, or you're not severe enough, that at some point you're gonna say, "You know what? It's time for me to go on a therapy." And also, why not? They're eligible for a drug like Galafold. It's so easy to use. The compliance is over 90%, so clearly, it's, you know, it's not inconvenient. There's no serious side effects to worry about either. Yeah. I mean, remember, a lot of these patients now are being diagnosed through family screening- Yes which means, you know, whatever, their sister or their daughter or somebody gets diagnosed in the family, and then you do a family screen, and you go back. It's X-linked, and it's usually four or five family members also have Fabry, undiagnosed Fabry. If a doctor or your, you know, relative calls you up and says, "Hey, you've got this weird disease," you can imagine there's a period of time before you're ready to- Yeah to go onto therapy. Definitely. Everywhere but the United States has pretty strict treatment guidelines around needing to have at least one major organ system involved. So proteinuria or left ventricular hypertrophy or a stroke, and so you might have pain and some morbidities associated with Fabry, but you might not meet those treatment criteria. And that's, you know, two-thirds, three-quarters of the world's Fabry patients kinda fall in that camp. In terms of rate of progression, you know, if you think about where we were in two thousand and sixteen when we launched, if you think about that five thousand diagnosed untreated, my guess is the vast majority of them have progressed now onto treatment, and then the six thousand are newly diagnosed patients. So I think probably over a three- to five-year period, you can kind of expect those patients to progress to treatment. But I do think that we will more than replace, unfortunately, and maybe it's a good thing, though, 'cause then they're eligible for treatment. I think we'll more than replace the diagnosed, untreated population if you fast-forward the next six years. When I look at Amicus as a company, you're in a much safe place relative to other companies I cover. Yeah Where we're focused on binary events and, you know, can move the needle quite a bit in either direction. Yeah. have two great drugs. They clearly have paths for growth here. Is there anything next that we should think about beyond this stage? Are you gonna ever get back in the clinic? Do you wanna build more commercial drugs? Yeah. Yeah, great question. I would say a few things. First of all, it's the first time in a long time where, you know, you don't have some existential, is there gonna be an inspection? What's that gonna look like? And what about Biosecure? Yeah. Which by the way, we saw that- Yeah pass with very little, you know, implication for us- Sure ... and our commercial medicines. You had what happens with the launch and those kinds of things. Yeah. So I think for the first time, just from a core business perspective, we have, I think, just breathing room to execute and deliver, which is what I think Amicus is best at. So I, I would argue that today there's a lot of upside to the company and to our valuation, just on the core business alone. I would agree. Thank you. That being said, look, you know, fast-forward five years, and I think Amicus has the capability to be a much broader company in the sense of, you know, more than just Galafold and Pompe from a commercial perspective, but also a pipeline again. We have kept on to some very early stage programs that we think could have utility, in particular in Fabry, to address the non-amenable mutations. We spend, you know, no dollars on that today, but our goal is in kind of one to two years, when we have our own positive free cash flow to put to bear. I think we can start to fund a pipeline again. And in the meantime, and we've talked about this pretty openly, we think we do have a global commercial infrastructure- Yeah ... that is, you know, underleveraged today, and if we can find the right commercial asset, rare disease commercial asset, to add to our portfolio, we think that could drive a lot of value as well. So are you looking more at other companies that are developing? Are you looking at academia and picking up programs that are kind of being studied by different investigator groups, both? I would say anything in Fabry and Pompe, we are all over from a sort of diligence and knowledge- Yeah perspective. That being said, I think we've committed to investors that we're not gonna put, you know, investors' dollars at risk to fund our pipeline development. Our goal is to get to- Uh-uh positive free cash flow, and again, you can see from a, you know, this year will be full year non-GAAP profitable. You can see our way to GAAP profitable, and then positive free cash flow in the not-too-distant future, so for us, it's, you know, use our own dollars to do risky, you know, pipeline bets. Sure. And that very well could, and probably should, include Fabry or Pompe assets. In the meantime, I think the best opportunity is to focus on, you know, commercial products, whether it's regional deals, things that are below the radar of big pharma, big biotech, but things that could still move the needle for us and could leverage that infrastructure that we've already built. Yeah, I think you make a great point, and I mean, I think myself, as an analyst, when I model a lot of these various disease drugs, I think about U.S. and Europe, right? Right. But then, to your point, when you think about some of these other regions, they add up- Oh, yeah and they can be pretty meaningful, and there's only so many companies that have that infrastructure. So it's, it's definitely something worth considering. Yeah. Thank you. So maybe to close, why do you think this is an optimal time to invest in the company, and anything that you think is misunderstood that you wanna emphasize a little more? Yeah, I think it's, you know, what we talked about before, which is, you know, coming into this year, there were some big unknowns. You know, what's gonna happen with the launch? Biosecure came out of, kind of, out of nowhere. Yeah. You know, Galafold, we I think put forth healthy numbers, but we've actually improved those numbers, and I think if you play things out, I would argue we're undervalued for Galafold today. If you believe that Pompe is gonna do anything meaningful, which we certainly have high conviction around, I think that's a real opportunity, and I think there was an investor who, you know, kind of described to me, especially in this market, macro kind of strangeness, and, you know, not nearly as bad as it was a year or two ago, but certainly not quite as strong as we all believe, you know, the biotech industry can be. Something like Biosecure that maybe had a 5% chance of kind of a black swan impact, you know, that just is one more thing people are focused on. I think if you look at Q2, where we were, if you look at Biosecure kind of resolving, if you look at where we think we can take Galafold, you know, I think the core business alone has a lot of upside, and I'm super bullish, and I hope you guys are, too. Great. Well, thank you very much for your time. Appreciate it. Thank you, Kristen.