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

Welcome, everyone, to the 40th Annual JPMorgan Healthcare Conference. My name is Anupam Rama. I'm one of the senior biotech analysts here at JPMorgan, joined by Cable Smith, Malcolm Kuno and Priyanka Grover from the team. Our next presenting company is Amicus Therapeutics. And presenting on behalf of this company, we have CEO, John Crowley, and incoming CEO, Bradley Campbell. [Operator Instructions] With that, John, Brad, take it away.

Great. I'll go ahead and begin Anupam. Thank you, and thanks, everybody, for joining. This is going to be a very exciting year for Amicus as we're poised to launch our second medicine in the last several years for people living with rare diseases. So we'll come to that on the first slide here, I'll just reference that Bradley, and I will be making forward-looking statements. And then on the next slide, if we fast forward to Slide 4, you'll see a snapshot of Amicus and who we are today. So again, Galafold remains the cornerstone of our success as you'll see this year, approaching $1 million a day in global revenue, importantly, ending last year with 1,750 patients on Galafold worldwide. So Galafold remains the cornerstone, the centerpiece of our portfolio and the medicine, we're very excited to launch around the world beginning in the United States this year is AT-GAA, a novel 2-component biologic small molecule for people living with Pompe disease. So those will be our 2 marketed products and again, a rich pipeline of genetic medicine technologies that we hope to advance for people living with Fabry, Pompe and many other disorders, all of that against -- we think a world-class team now with more than 500 employees working globally, again, one global Amicus. And again, with the financial strength of the company with more than $0.5 billion on the balance sheet, combined with the cash flows of both Galafold and soon AT-GAA, leading us to financial stability, a path to self-sustainability and importantly, a path to profitability in 2023. So that's a snapshot of the company today. Please fast forward, we'll talk a little bit about what we did last year. And again, as we do every year, we set out our goals at this conference for the year. So if you look back on 2021, we achieved every 1 of the 5 objectives that we set. We achieved the double-digit growth in revenue of Galafold coming in last year at $306 million in global revenue. We also reported the results of the Phase III study and have now completed and it's been accepted in both geographies, the BLA and the MAA filings for regulatory approvals for this novel biologic. We also continued to advance clinical studies, regulatory discussions. Moving forward, our genetic medicine pipeline. We worked on furthering our very importantly through gene therapies our analytical and manufacturing capabilities and through our partners' capacity to continue to build world-class technical operations, again, in the world of gene therapy in our pipeline we continue to believe that the process is the product. And again, against that backdrop, we continued to maintain a strong financial position, including the third year in a row of keeping operating expenses within our guidance and keeping them flat. So let's look forward a little bit to what we have in 2022. And very importantly, and this is a point we've been conveying especially the last couple of days. Our continued confidence in Galafold to continue to reach many, many more people living with amenable mutations with Fabry disease around the world. Again, last year, we achieved 17% revenue growth in Galafold. This year, we continue to expect double-digit growth 15% to 20%. We are guiding conservatively to revenue this year of $350 million to $365 million. And importantly, and Brad will take us through shortly why we have confidence in this drug, all the key dynamics that you look for in a medicine. You look for continued demand from patients and physicians continued ability to support the price of the medicine, importantly, continued adherence and compliance with the medicine and then also the protections that we have, the intellectual property, orphan drug and other protections that give this franchise a chance to continue to grow to reach more people with Fabry and to continue to fund the launch of AT-GAA and our pipeline. So a lot of confidence in Galafold ahead and Brad will speak more to that in a moment. Secondly, very clear, we are going to secure FDA approval, and we are going to secure a positive opinion from the CHMP in Europe for the approvals of AT-GAA. We are also accelerating all of our launch planning for AT-GAA, we have a goal this year to initiate successful and rapid launch in the United States for AT-GAA. Again, we continue to believe that AT-GAA based on its unique molecular structure, leading to a very, very strong and differentiated clinical data set that this will become the standard of care, we believe for people living with Pompe disease worldwide. We are also continuing to advance best-in-class next-generation genetic medicines. We have, for a long time, been of the belief that looking at each disease where we pursue a solution that we need a novel approach taking into account the disease biology, the regulatory framework, competition and making sure that we are always developing a best-in-class medicine. And again, all of that against this strong financial position of being able to ensure financial strength, not have a need for dilutive financing in the company and putting us on a path to profitability in 2023. So again, we come off a very, very strong year in 2021, and this will really truly be an exceptional year for us, very exciting for a company that's been developing these medicines for years to now have what we hope will be and expect to be a marketed product with the potential to be best-in-class for people living with Pompe around the world, which takes us to this slide. We are really, really focused on building out complete franchises for people living with Fabry disease and for people living with Pompe disease. These are anchored by both Galafold and AT-GAA but again, through the gene therapy programs and other technologies that we are also continuing to explore through our internal scientific efforts, looking at other technologies. We want to build a multiyear, ultimately, a multi-decade pipeline to provide the best-in-class solutions as we built such strong relationships and trust with the patient and physician communities, the payer communities around the world. So that's our goal in Fabry and Pompe. And particularly in Pompe, this will be an incredibly important step forward as we prepare to launch AT-GAA. And I'll speak more to the Pompe section after Brad takes us through more detail with Galafold. And again, you see that reflected here broadly in next-generation research programs for the company. So I have 1 further slide, and then I'll turn it over to Bradley to go into more depth on our continued confidence with Galafold. But again, when you step back and you look at Amicus today, with 1 approved product moving now from its launch phase to its growth phase, Galafold for people living with amenable mutations in Fabry disease, a medicine with a potential to affect thousands of people's lives to generate $500 million to $1 billion in peak revenue a medicine with a long exclusive period well into the next decade and medicine with 27 Orange Book-listed patents, 13 of which go into the late 2030s. So we have that medicine and you combine that with yet now a second wholly owned Amicus medicine, complete global rights at Amicus, and we have now built our infrastructure on the heels of the success of the Galafold launch to launch AT-GAA with a very minimal investment in incremental resources globally to support that launch. So when you step back and look at Amicus, soon to be 2 products each with $0.5 billion to $1 billion potential, 1 already exceeding this year more than 1/3 of $1 billion in revenue against the backdrop of a strong pipeline and a path to profitability in 2023. So again, in many, many ways, this will be an incredibly important year for Amicus. And again, the cornerstone of this success is built on Galafold, and it's also built, frankly, on my continued partnership with my long-time business partner and friend, Brad Campbell and Brad will take us through where we are with Galafold, Bradley.

Great. Thanks, John, and good afternoon, everybody. So let me continue the story that John started terms of how we're continuing to build our leadership position in Fabry disease with our Galafold business and the continued growth. So on the next slide, we just remind everybody Fabry disease is a progressive lysosomal storage disorder primarily affects the kidney, the heart, the CNS with challenging symptoms in GI, pain, et cetera. And 1 of the things that I think we know now that we've been following the Fabry market for well over a decade is that this is a significantly under-diagnosed disease. In fact, it's caused by over 1,000 known mutations. We estimate there's about 13,000 patients diagnosed with Fabry disease around the world today. And if you go to the next slide, that under-diagnosis is really what drives the continued market growth of Fabry. And so as we look out since the inception and over the last few years, this has been a highly growing market driven by finding new patients we would remind everyone that newborn screening suggests that Fabry could be ten-fold more common than what was originally described in the literature. And today, in 2021, at least, we believe that the market will exceed $1.9 billion in global sales. And if it continues on a similar growth trajectory, we think that tracks to well over $2.5 billion by the middle of this decade. I would also point out, and we'll get to some of the drivers of this growth, Galafold was actually the fastest growing medicine within the 3 approved therapies for Fabry disease. And part of that was driven by our ability to bring on diagnosed untreated patients and newly diagnosed patients on to Galafold. And we share here that of the 1,750 patients globally that are on Galafold over 800 of them actually were naive to treatment before they started. So if you go to the next slide, Andrew. Important just to take a quick snapshot of where we were at the end of the year in 2021. As John mentioned, we did deliver on our guidance despite some headwinds, in particular, in the second half of the year as Delta and Omicron variance of COVID had some stickiness to the market, sort of disrupting the point of care between new patient starts and original prescription. But even with that as a backdrop, we continue our track record of consistently delivering on our guidance. doing $306 million in global sales in 2021. As John mentioned, we now are guiding to $350 million to $365 million in worldwide revenue in 2022 and continues that 15% to 20% growth rate that we've been on for quite some time. And you can see a couple of other important points that we continue to come back to. We've grown the number of amenable mutations that are reflected in the U.S. label, the European label. We've continued our geographic expansion. We're now approved in more than 40 countries around the world with reimbursement and well over 30%. So this is a robust and growing business. And what do we track and what do we talk to you all about in terms of how we know that this continues to grow as a business. If you go to the next slide, these are all the key performance indicators that we point to. First of all, we are now at almost a 50% share of treated amenable mutations reflected in that $306 million in global sales. Of course, that means we have a significant number of patients still to grow just within the existing business. However, we do continue to expand geographically. We've talked over the course of the week around a couple of important points. First of all, our core business in Europe, EU5, U.S. and Japan continues to grow. Those are the largest growing countries. So we haven't tapped out at all, the growth of those businesses. But what we are seeing is, in fact, in Europe, for example, where we're at an 80% or 90% market share of switch patients, the growth now is driven by bringing on new patients. Whereas in Japan and the United States, we're still maybe a 50-50 new patient acquisitions versus switch and naive. But then if you look at the new markets, areas in Latin America, Brazil, Chile, Argentina, Colombia, Asia Pacific outside of Japan, Middle East in terms of the Gulf states and soon, hopefully, Turkey, where we hope to get approval later this year. Those are really areas where we haven't seen a lot of traction until last year going into next year. And those will be continued growth drivers for us for the many years to come. A couple of other important things that I'll orient you to here continue to see well over 90% compliance and adherence. So great adherence to the drug, which we think shows that patients and physicians are having a very positive experience on the drug, it's really a core part of the foundation of why we continue to grow and be successful. And then I would remind you that we do continue to expect non-linear growth quarter-to-quarter. This is a pattern we've seen since our launch. We tend to see Q4 and Q2 was the strongest quarter-on-quarter growth and Q1 and Q3 can be relatively flat. But all of that with a backdrop towards that -- achieving that 15% to 20% growth rate this year and $350 million to $365 million in annual revenue. If you want to go to the next Slide 14. The key though for us, and this is what we've really been talking about this week is $500 million is a very important milestone. It's 1 we're confident -- 1 path to achieving. But the real opportunity here is getting to that $1 billion annual sales opportunity. I talked a little bit about where the market is heading, and we know that we can get to and well past that $500 million by those 3 key growth drivers that I've talked about, continuing to penetrate existing switch patients, continuing to expand into new geographies, broadening the labels. But then in the longer term, as the market continues to grow to $2.5 billion and upwards of $3 billion, that's where we would expect to continue to see our diagnostic initiatives play out finding more patients with Fabry disease. And also, as John mentioned, that really strong IP estate that lasts well into the next part of the decade. So with that, I would just say 1 other thing, and John mentioned this, and then I'll turn it back to John to go through Pompe disease. We've built an incredibly passionate, experienced rare disease commercial and medical organization that support Galafold around the world. We are present now in 43 countries, either directly or through distributors, as it's outlined on this slide. But what's really critical here is that these are the exact same folks who will be able to help us launch AT-GAA around the world. And what we've said is very little incremental addition to this team, maybe less than a dozen FTEs globally to support the launch. So this is a highly successful, again, highly passionate team who are ready to get out there not only to continue to grow Galafold, but now with great anticipation of what we plan to be our second approval with AT-GAA. With that, I will turn it back over to John, who can walk us through the Pompe business.

Great. Thank you, Bradley. So I'll talk more about AT-GAA that we're very excited to move through its regulatory processes and approvals this year. So again, just to remind everybody on the next slide, a summary of what is Pompe disease. It is a lysosomal storage disorder that leads to a deficiency in every case of the same enzyme and a buildup of the same substrate glycogen that destroys muscles. This is a musculoskeletal disorder. It also affects, particularly in children cardiac muscle and breathing muscle. In every case, without treatment, Pompe disease will be fatal. So what we have developed on the next slide, if you look broadly at the market opportunity to help people here, this has been a market that for 15 years, has had one enzyme therapy approved. There is now in the United States, a second enzyme therapy from another competitor company. We have developed something very distinguished here. But when you look at the opportunity to help people, you're talking about thousands and thousands of people. And in addition, like many of these lysosomal disorders, similar to Fabry disease, we see many, many more patients who we believe will benefit. This is currently a more than $1 billion market opportunity from just 1 enzyme replacement product and with multiple products and new patient identification and further market expansion we see this tracking toward about $1.5 billion market by 2025 and well north of $2 billion in the years ahead. And again, we think with the data that we've generated and with the unique molecule that this has the potential AT-GAA to become the standard of care, both for patients who would switch from the existing approved standard of care as well as newly diagnosed or untreated patients. So on the next slide, just to remind everybody what is AT-GAA. With our team of protein engineers and glycobiologists, for many years developed was a novel cell line. It's a cell line that is highly glycosylated with significant amounts of naturally occurring mannose 6-phosphate. This is the carbohydrate necessary for targeting an absorption to patients' muscles. The treatment regimen here is an every other week infusion of this novel biologic combined with a chaperone or an enzyme stabilizer AT2221, so that a patient about an hour before the infusion takes the small molecule stabilizer. The infusion then begins an hour later, and it's the intentional drug-drug interaction of the small and the large molecule with a small molecule adding confirmational stability and ultimately, potency to the large molecule. And again, much of the work, we believe, being done by the carbohydrate structures on the large molecule. So differentiated molecule, a very differentiated set of preclinical data, where we understand very well the biology of disease, but we also understand the mechanism of action here. And that's translated through years of clinical work beginning in 2016. And in the next slide, we just recently had published at the end of 2021 in Lancet Neurology, the Phase III PROPEL study results. So important that this was published in a top-tier peer-reviewed journal. I'd encourage you to read the results. You'll see that it speaks to all the key endpoints that we studied in the study, both for the patient population that was untreated previously with ERT as well as about 77% of the patients in the study who were switched from the approved enzyme therapy standard of care. So you'll see it looks at 6-minute walk. it looks at forced vital capacity, it looks at key secondary endpoints as well. So we're very pleased that resource is now out there for physicians as they consider ahead the best treatment paradigm for patients. On the next slide, we'll just summarize the key data. There's an awful lot of numbers and I assure you, I'm not going to go through each of these. The key takeaway is that we study the overall population again, about 120 patients in this study, the vast majority, about 75% -- 77% of whom were switch patients from the ERT standard of care. This was a superiority study to an approved billion medicine in a rare disease. While we just missed superiority on 6-minute walk in the overall population, we achieved nominal statistical superiority on the key secondary end point that has been the basis of approval for other Pompe enzyme therapies, forced vital capacity, a measure of pulmonary function, we achieved statistical superiority in the overall population. But also importantly, when you look at those patients that, again, the substantial numbers in this study, the vast majority who also represent, we think, a substantial number of patients, the vast majority who we'll be able to address once the medicines are approved, the ERT experience or switch population. They're in prespecified analyses, we showed superiority to ERT standard of care on both 6-minute walk and on forced vital capacity. And again, when you look at what we've highlighted here, favoring the treatment group in many cases with nominal statistical significance, you see overwhelmingly in favor of AT-GAA over ERT standard of care. This was the data that we took to regulators in the second quarter of last year. It was the data that led to our filing and the FDA acceptance of the BLA with the July 29 PDUFA date. It's the same data together with everything that we've shown before in the Phase II studies that led to the filing of the MAA and the European validation of that MAA back in November. It's also what led to the early approval under the EAMS framework, the early access for a novel medicine scheme in the United Kingdom, where we received early approval for this medicine under that scheme for patients who would switch from ERT last year. So a lot of activities. We've got a very, very rich data set that will inform patients and physicians as ultimately to what the right choice for them will be once this medicine is approved. Just briefly on the regulatory status update, again, we are approaching about the midpoint of our FDA review of AT-GAA. We're very pleased with a high level of engagement from the regulators, and we continue to believe that this medicine will be approved and that it will be launched in the second half of the year for patients in the United States. And likewise, we would expect a hopefully positive opinion from the CHMP later this year, leading to launches throughout Europe in 2023. Again, to remind everybody, well more than 150 people living with Pompe disease continued to remain on AT-GAA as their only treatment. Virtually all patients who have ever switched from the ERT approved standard of care to AT-GAA have remained on AT-GAA. And again, multiple ongoing supportive studies in children as well to expand the label to everybody living with Pompe disease. Just 1 last point, and I'll turn it back to Bradley to conclude with the financial picture and conclusions on the presentation here. Again, that's all the work that we're doing in the launch preparations. Five years ago, when we were preparing to launch Galafold around the world, we had to build an entire global commercial and supportive infrastructure to do that. We're very pleased that we've built a world-class team around the globe for Amicus to deliver Galafold. It was a very, very successful launch of Galafold, and we're now able to leverage that team, that experience our decades of work in Pompe disease and those relationships. So from the team, the medical education, the already published study of the Phase III, our experience with reimbursement and access around the world and again, all the strategic planning that we're doing together with building inventory with our partners at WuXi Biologics, we think, puts us in a very, very strong position for a second rapid and successful launch for Amicus. So with that, Bradley, I'll turn it to you to conclude.

Great. Thanks, John. So I'll move fairly quickly here as I know we're eager to get to Q&A with Anupam. So first of all, continuing to transform Amicus into a premier development and commercialization company and really continue to build upon our leadership in Fabry and Pompe disease. We'll remain patient dedicated, we'll remain true to our mission as we always have but as we move forward, we'll continue to invest in commercialization of Fabry disease and also now increasingly Pompe disease. We'll continue to move forward with the co-development of Fabry and Pompe disease gene therapies and really continue to, in the future, position Amicus as a partner of choice as we look to bring in new therapies, new technologies as we establish that leadership position in the 2 core franchises. If you want to go to the next slide. So how do we get there? First of all, continue to deliver on growth of Galafold, as we said, sustained high growth in the 15% to 20% range for the foreseeable future. Number two, secured, of course, approvals of AT-GAA and then execute fantastic launches in the United States and around the world and start to then add to that double-digit growth would be increasing revenue from AT-GAA. And then finally, continue to be prudent in our expense management. And as John mentioned, achieved that self-sustainability and profitability in 2023 and beyond. If you go to the next slide, we won't spend a lot of time here. But as a reminder, we do have very differentiated approaches in gene therapy for Fabry disease and Pompe disease. We're excited to see those move forward and again, in co-development with Caritas. But I think if you go to the last slide, in addition to continuing to drive great growth and deliver value for our shareholders. The real way that we measure success is ensuring that we continue to impact the lives of patients with rare diseases around the world. We sit here today at well over 1,900 patients globally who benefit from some form of therapy from Amicus, and we're hoping in the very near future that, that becomes thousands of patients and more. So with that, thank you very much for listening, and we'll turn it over to Anupam for Q&A.

I hope you guys can hear me?

Yes, we can. Yes.

Yes. I think they disabled my video. There it is.

There you go.

I thought you were in charge Anupam , come on.

Yes. No, you're wrong there. So there's a question portal or if anybody wants to ask a question, just put it in there, and I'll -- I'm happy to ask on your behalf. The Galafold guidance of $350 million to $365 million came a little bit below Street consensus. And I was wondering what were some of the underlying assumptions in the growth to come to that consensus with the new variant, what impact did that have on your kind of outlook and how you think about giving the guidance?

Yes. Thanks, Anupam. We've had some really good conversations in the last 1.5 days, I think, to get people in a good place on where we are. Obviously, Monday was a very frustrating and confusing day. Let me just ground everybody again. When you think about is a drug launch slowing or is the growth going to come in under, you look at a number of factors. You look at the overall demand patients, patient numbers, physician demand and perspectives. You look at price, the ability to hold price, you look at compliance adherence, you look at competitive dynamics, all of those line up terrifically well for Galafold. This continues to be a strong product that's growing in the marketplace. In a marketplace itself that's already growing. We wanted to be very conservative. There are a lot of I'll ask Bradley in a moment to speak, Anupam but some of those headwinds that you're seeing the building queue of patients in more and more geographies, including in the emerging markets, we were just beginning to launch countries like Brazil and other places. So when we think about the continued growth path for this year, again, $350 million to $365 million, while coming in slightly below consensus, it's right on line with our budgeting and planning. And importantly, we will get to that important way point of $500 million in sales. We think it will come in '24 instead of '23, but we'll get there. It will be 6- to 9-months shifted, but also very importantly, you're not losing those patients. They're not going to enzyme therapy. They're not coming out of the system. They're just delayed in their ability to access Galafold. So they will become, we believe, Galafold patients. And once we see the emergence finally, from COVID, and we see health care systems adapting to this endemic. We see the queue start to clear, then we think we could accelerate even further that growth but we wanted to be conservative now, just knowing there's a lot of uncertainty. And maybe Bradley, just speak to some of the dynamics we're seeing in some of the geographies that's leading to those building queues where it's just taking patients longer to get through.

Yes. Thanks, John. And I would echo your point that that's -- I think that's the most important thing for folks to remember. This is not reflective in any way that those patients are going away or that the business is somehow fundamentally off. It is very much on we're continuing to execute, and John touched on all those trends that I won't repeat. Specifically, though, what happens with COVID now we have what 22 -- or sorry, 26 months maybe of experience here. What happens is these patients, when they come on to Galafold, when they get an initial prescription, they have to come in for -- typically for a baseline visit. And obviously, if you have a spike of COVID in a region or even specifically in a hospital, they can't get in to do that baseline visit. And so the prescriptions are written, they're ready to go, and it might take instead of days for them to start on therapy. It might take weeks for them to start on therapy. We even shared with you earlier on upon, and we'll share it again here. We had an example in the last weeks of December, where there are actually 2 separate countries that have almost a dozen prescriptions each where they were prescribed at the end of December in a normal time, they would have gotten on therapy, but Omicron had spiked in those 2 countries, and therefore, they'll come on here in January. So the demand is still there. We know the business is strong, but it's just a shift in that time point. And I think, as John mentioned, look, $500 million for us was always a wait 5 years ago, it was a way to tell everybody. Look, we know this is going to be a really important product that's not the end goal. We're going to go well past $500 million and our goal is to make sure we get upwards to that $1 billion opportunity by the end of the decade.

We've got a couple of questions in the portal here. just as a confirmation, the lower Galafold guidance isn't due to Sanofi capturing more share?

No, not at all. In fact, if you look at the growth in the market, the greatest contributor to that growth in the last year or 2 has been Galafold. So they're not going to ERT at all.

And I would just say we didn't lower our guidance. Yes, it fell a little bit below consensus. But we've -- I think we've established a great track record of meeting and beating our guidance, and we intend to do that this year as well.

Yes, that's a really important point, Brad. When you look at that $350 million to $365 million, it is intentionally conservative. It lines up with bringing us on continued double-digit growth for many, many years ahead, including that way point of $500-plus million in '24. And then ultimately, we still believe up to $1 billion in peak revenue. And when you look back over the last 4 years, the first 3 of those years, we exceeded the guidance that we put out at this conference for Galafold. Last year with Delta, with Omicron, we came in the midpoint of our guidance. So I think we've gotten very, very good at forecasting commercially and doing so pretty conservatively.

We've got another question in the e-mail portal. How does the CLN6 discontinuation and CLN3 rework affect both the timing and valuation of the Caritas transaction?

Yes. Again, to remind everybody, we announced in September that we would take our R&D portfolio, the gene therapy portfolio and spin it into a new company, Caritas Therapeutics. A couple of pieces to that. One would be the Batten's franchise, CLN6, CLN3, much of the value though coming from the combination of Amicus Technologies and capabilities in protein engineering combined with Jim Wilson and the UPenn team and their next-generation gene therapy technologies. Again, we have the rights to about 50 rare -- global exclusive rights to 50 rare diseases in that combination. So for us, CLN6 has been a very important program for us. We were really, really disappointed when we looked here very recently at the long-term outcomes in these dozen children and saw that while we had an effect in the first couple of years at delaying significantly the onset of symptoms, it seems that the disease, it was a low-dose gene therapy, but it also seems that the disease just continued that inexorable progression. So devastating for those kids and those families, it's a very, very small indication. So there was very little value, if you will, ascribed to CLN6. CLN3 is a much larger rare disease. It is the most prevalent form of genetic blindness and fatal brain disease in children. There are some learnings from CLN6 that we've already incorporated the higher dose that the 1 child in the clinic has already received several years ago, the different promoter that was built in a few years ago and the different route of administration we look for in the future. So throughout this year, we're going to continue to work on the preclinical data. We'll look at the 2- or 3-year data in those handful of children in the CLN3 program. With respect to Caritas, we have an S-4 on file with the SEC and we would expect the new company to be created by the end of the first quarter. With that said, we're obviously very mindful of this SPAC market. And in any case, Amicus has a path to profitability in 2023.

Maybe I'll squeeze in 2 quick Pompe question. So the first is what's your base case scenario for the breadth of the label, given the totality of the PROPEL results, switch label versus inclusive of naive? Is there a difference between U.S. in OUS? And then also quickly, what are the implications of sort of that November EU ruling of neoGAA that it is not a new active substance relative to Lumizyme? And how does that impact commercial dynamics in the region?

Yes. Bradley, do you want to go ahead and take that question on what we expect for the label, maybe reference what was seen with neo even studying only a naive population, and then you could address the unique dynamic in Europe and how we're seeing it.

Sure, yes. Thanks, John. So Anupam, great question. From a labeling perspective, as John said, we take good confidence from the broad indication statement that we saw with the approval of neoGAA here in the United States as good support for us being able to achieve a similar broad label. So for both switch and naive patients and of course, biologically, there's no reason to think that we would only have an impact on switch patients versus naive patients. And in fact, our data very much supports that. So we are assuming a broad label. If for some reason, we were limited to the switch data only in our label. We still think that's a very strong label. Obviously, that's where the most unmet need is in the market. There's 3,200 patients treated with standard of care today and the vast majority of the opportunity going forward is, we think, in the switch patients initially, and we could certainly augment with naive at a future date. But right now, we're assuming we'll be able to get a broad indication statement. On your second question, you're exactly right. It's a really important point, and we're watching it carefully. As you mentioned and as was announced publicly, Sanofi failed to get new active substance status for their application around [ XY dime ], which is a brand name in Europe. They appealed that and what we saw publicly again is that they failed to overturn that indication upon appeal. And similarly, we anticipate they may be in a similar situation in terms of their orphan drug exclusivity. And what all of that means put together, is that they could be in a very challenging position around pricing and reimbursement in Europe, effectively it saying that it's not a new active substance. Of course, their primary data from their study was non-inferiority. And when they go through the reimbursement process and there are other examples of this in other indications, historically, where similar -- or same manufacturers in a similar situation have been forced to discount their products in order to bring them on to the market. And so I don't want to speak for them, and we'll wait to see how that plays out. But at the very least, what it's meant is that the time between their approval and launch and the time of our anticipated approval of launch has narrowed and certainly calls into question how the reimbursement strategy is going to go.

Okay. Guys, I want to thank you guys so much for taking us through the spectrum of programs. And appreciate all the insights. I hope you guys have a great rest of the conference, and maybe end a couple of minutes. At least so we can all do some activities of daily living, drink water, eat food go to the restroom. So all right. Thanks so much.

Thanks, everybody.

Thanks Anupam, appreciate it.

Good day, take care.

Yes. Take care.