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

Good morning. I'm Salveen Richter, biotechnology analyst at Goldman Sachs, and thank you so much for joining us for Day 2 of the 41st Annual Goldman Sachs Conference -- Healthcare Conference. And with that, I just wanted to make a couple of disclosures before we start. We are required to make certain disclosures in public appearances about Goldman Sachs' relationships with companies that we discuss. The disclosures relate to investment banking relationships, compensation received or 1% or more ownership. We are prepared to read aloud disclosures for any issuer upon request. However, these disclosures are available in the most recent reports available to you as clients at our firm portals. Disclosures and updates to these disclosures are also available by ticker on the firm's public website. In addition, disclosures applicable to research with respect to companies, if any, mentioned herein are available through your investment representative. Further information on the subject companies may be obtained from Goldman Sachs Securities Private Limited. Goldman Sachs may beneficially own 1 or more of the securities. Also the views stated by non-Goldman Sachs personnel do not necessarily reflect that of Goldman Sachs. And with that, we're pleased to have Amicus with us this morning. And with us, the CEO, John Crowley. John, thanks for joining us.

Good morning, Salveen. Nice to see you.

Good morning. You too.

And to start here, maybe a big picture question. So you have made a strategic move to add gene therapy as one of your key technological modalities as you address the field of rare disease. Can you discuss the rationale behind opting for partnerships with U. Penn and Jim Wilson and discuss the selection of the specific disease areas that you're pursuing? And could we see this expanded further?

Yes. Thanks, Salveen. So this was a big, bold and important move for Amicus, frankly, and it was one we gave great thought to beginning about 3 years ago. And what we did was to look at the landscape of diseases. We started with actually about 1,000 rare diseases. Through about a 6-month exercise we narrowed that down to about 100, and then settled initially on the first 15, but we kept an eye on 50 or so of those diseases that we were most interested in. And for Amicus, it really was a natural evolution from what we had been building over the years. It built, number one, on our science, particularly our understanding of the science of rare diseases, lysosomal disorders, but specifically, our ability to engineer proteins as we've done with AT-GAA, our advanced enzyme replacement therapy or enzyme replacement therapy that's advancing now through the clinic. So again, part of that natural evolution was the core science that we were very strong with. Also then our drug development capabilities around the world as we had gotten Galafold through clinical studies approved and reimbursed in dozens of countries around the world, so the regulatory experience, ultimately, the reimbursement experience, and manufacturing as well. We had spent about 6 years with our partners at WuXi Biologics, scaling and building the processes, the process science, for one of the world's most complicated glycosylated proteins with AT-GAA. So it wasn't a move in a different direction. It was a natural evolution. And again, coming back to the gene therapies, looking at those diseases, our initial partnership was through the acquisition of a spin-out company from Nationwide Children's Hospital and Dr. Kaspar in Ohio that brought us our franchise into different Batten disease programs. Right on the heels of that, in the fall of 2018, we signed an initial collaboration with Jim Wilson. This is very important that people understand the scope and scale and depth of our partnership with Jim and U.Penn. I've known Jim for many, many years, helped him about a decade ago launch the Center for Orphan Diseases at U. Penn, so we thought for some time about ways to work together. Our initial collaboration focused on 3 diseases: Pompe, Fabry and a rare neurologic disorder similar to Rett Syndrome called CDKL5 deficiency. We signed that in October of 2018, and we set about immediately to build a gene therapy program in Pompe initially. And again, this was a great example. We brought technology to the collaboration, significant technology and capabilities. We moved all of our science to Philadelphia. In fact, just on March 5, right before the COVID crisis, we opened our 75,000 square foot global research and gene therapy Center of Excellence right on Market Street in Philadelphia, right next to Jim's labs. But the work that we did in that latter part of 2018, early part of 2019, brought us the cell line that we designed for Pompe, again an engineered cell line to produce a highly targeted form of the GAA-Pompe protein. We combined that with the advanced vector technologies and gene therapy approaches of U. Penn, saw some tremendous data that we were not only enhancing uptake of the protein, it was getting to all key muscles in the animal model, it was substantially reducing substrate, and importantly, it was getting to the CNS. So when we saw that, when Jim saw that, we realized the power of combining the Amicus and the U. Penn technologies and approaches. And at that point, we entered into -- we negotiated, entered into a collaboration in May of 2019. Some important parts of that collaboration with U. Penn: First, we got immediate rights to some of the preclinical programs at U. Penn, including the MPS III Sanfilippo programs, so those are now Amicus programs as well in advanced preclinical development. We also agreed to $10 million a year in general research funding. And this is to fund the next-generation technologies in gene therapy at U. Penn where Jim is looking at improving the targeting of the technologies with gene therapy, tropism, safety, immunogenicity, manufacturability, delivery. Amicus will have the rights through all of those inventions for about 50 designated diseases, global exclusive rights. As I -- Jim has helped lead and form another new gene therapy company in Passage, which is a tremendous new company. We were very clear with the leaders and founders of Passage to delineate the rights that would go to Passage and the rights that would go to Amicus. Amicus, again, with rights to 50 exclusive programs out of U. Penn. It includes the majority of lysosomal storage diseases, we have the rights to out of UPenn. And 12 other larger rare diseases. We've only disclosed 3 of those: myotonic dystrophy, Rett and Angelman. We would expect we'd have some ongoing research in some of the other larger rare diseases, and we'd expect to be able to present some of that data later this year. So it's a deep and strong collaboration with Jim Wilson and his team, and I couldn't be happier with where we are, a lot of work ahead.

And are you still looking to filing or disclosure of up to 2 gene therapy IND each year starting in 2021?

We are. We think that's the path that we're going to go down and then the years after that, potentially even more, with the rights that we have. The most -- we have our 2 preclinical programs in the Batten disease area that we acquired from Nationwide Children's. Those are the CLN6 and CLN3 Batten disease programs. From the U.Penn portfolio, the most advanced program heading toward the clinic is our Pompe gene therapy program. We are now in the IND-enabling studies, the tox studies. We are also working with our contract manufacturer at Thermo Fisher Brammer on the scale-up and production of that. And we expect sometime in 2021 to have that in the clinic. And later this year, we'll talk about a second program to potentially go in the clinic in 2021.

So John, when you look out the next 5 years, where do you see Amicus, like how will this company have evolved?

We still have a very big vision for Amicus. We want to continue to grow the company as an independent organization. We've built tremendous capabilities in science, and we've built tremendous capabilities now in global commercial and market access around the world. So with Galafold being the cornerstone of our success, and then we'll talk about that shortly, followed by AT-GAA, our Pompe enzyme replacement therapy in late Phase III study, and then that whole gene therapy pipeline, my vision is in the next 3 to 5 years, by year 5, that we certainly are well north of $1 billion in global revenue, that we would have 2 approved products, at least, with AT-GAA and with, of course, Galafold. And by then, hopefully, also additional approved products in the gene therapy space and up to a dozen programs in the clinic or about ready to enter the clinic and really being an R&D engine in the field of genetic medicine.

Maybe starting here with the Pompe program, AT-GAA. So you have a pivotal program ongoing, and you're looking at patients who have switched from enzyme-replacement therapies and then more naive patients. And could you walk us through when we should expect the pivotal data set, what data we're going to see? Specifically, there are other trials that are being run on the side and the time lines associated with that and then how the rolling BLA submission that's supposed to start in the second half of this year will incorporate the data as these trials read out.

Yes. We are moving full forward toward approval, we hope, by end of '21 or early '22 and to get us there, a number of those key activities. The PROPEL study is our pivotal study for approval here. And again, that was targeted to be 100 patients. We actually overenrolled the study. There was such demand, and we get good success in manufacturing with our partners at WuXi. We kept enrollment open until December of last year, closed it with just over 120 people enrolled in the study. Again, this is designed to show superiority to the approved ERT standard of care, Lumizyme. Patients are randomized 2:1, 2 patients coming onto AT-GAA for every patient going to or staying on Lumizyme. About 70% of the patients are switching from Lumizyme. 30% of the patients are treatment-naive patients. But importantly, it will drive us toward a label for full approval for all patients living with Pompe and for both switch and naive populations, which is very important. We do have other studies you referenced, the pediatric study. We've now begun enrolling children in a cohort aged 12 to age 17. We are also soon beginning the next cohort down, aged 2 to 12, and we expect at just about the same time to begin the infantile study. Those will be open-label studies. We also have an expanded access program for infants living with Pompe. We've granted expanded access now to several Pompe infants, and we share the data on one of those infants as well. So we're putting together a pretty significant program. We expect by the time the BLA is filed about a year from now, a little less than a year from now, that several hundred patients will have taken AT-GAA. And as a matter of principle at Amicus, any time a patient comes into one of our clinical studies and comes onto our drug, they remain an Amicus patient for life. So through our clinical studies, through and to approval and then post-approval to the time toward a reimbursement, they remain Amicus patients. COVID-19, thankfully, has had no impact on any of our time lines. We had a fully enrolled Phase III prior to the COVID crisis. Once the crisis broke, we -- it was a lot of effort, frankly, for about a 6-week period. We followed patient by patient, site by site. The patients were excellent. The physicians were highly motivated to make sure that the infusions went off as planned and then also all the assessments went as planned. And we reported back in May on our earnings call that through the study, more than 97% of all of those infusions have been administered. In fact, I did report in the last month or so, nobody has missed any infusions whatsoever. So we're in a really good place. We've maintained the integrity of that Phase III study. And again, we're looking at a lot of endpoints. The primary end point in PROPEL, again, superiority based on 6-minute walk test. Our key secondary is forced vital capacity. And then a whole range of pulmonary muscle strength tests, biochemical measures, patient-reported outcomes. So it will be a robust study. It is the largest, and I can assure you, the most expensive lysosomal disease study ever conducted in the field.

And John, when it comes to those end points that you mentioned, what is the clinical bar, I guess, not just clinical but competitive bar that you need to achieve on 6-minute walk test, but also forced vital capacity because we've seen in some of these prior trials that you can get approved on the secondary end point even if you don't hit your primary.

Sure. So again, with Pompe disease, it's a disease, a neuromuscular disorder. It can affect people from birth, infants with the most classic severe form, all the way through adulthood. But in every case, it's a mutation in the same gene leading to a deficiency or an absence of the same protein in a buildup of the same substrate glycogen. Absent a therapy, you will die from your Pompe disease. The goal for this program has always been to show superiority to the approved standard of care. We have never been interested in any of our diseases and certainly not in Pompe of having just a small incremental benefit. So we designed something that we thought had the potential to be highly differentiated in its structure, and importantly, lead to substantial uptake -- improved uptake into the muscle cells. We've seen that through the preclinical data. We've seen it now in more than 4 years of Phase I and Phase II studies. And that's the bar that we've set. We designed this to be a superiority study. We are very confident in our ability to achieve that. I think looking at all the measures, certainly to be superior on 6-minute walk, but also to be able to show superiority on pulmonary functions, the patient-reported outcomes. Fatigue is an important patient-reported outcome frequently presented by people with Pompe. So the bar is certainly on the key primary end point but on as many secondary and other endpoints that we're studying to show differentiation and superiority from standard of care. I think that's important for anybody investigating any program, any new technology in Pompe. That's what patients want when you ask them. Sure, they'd love something more convenient, and yes, if it was a drug that was a bit better tolerated, that would be a benefit. But more than anything, they want to get stronger. They want to live longer.

And then when you look to integrate this drug if it works and is approved into the commercial landscape here, how do you see that playing out? And can you also just speak to the competitive landscape in terms of what's in development now? I think we're waiting Sanofi Phase III data for their drug next week. So how this space might end up evolving and as you look to gene therapy down the road?

Yes. This is one of the larger lysosomal storage disorders, about 5,000 to 10,000 patients diagnosed in the world. From a commercial standpoint, Lumizyme has been a highly successful product. It's been on the market now for about 14 years, Myozyme and then Lumizyme generating about $1 billion in sales. So there's a lot of opportunity to help a lot of people here. Again, we've designed something that we hope will show that it's highly distinguished and superior from the -- not only the standard of care, but from anything else in development. Our drug, just to give you some perspective, the data that we've seen to date at 1 year, and that's important because our pivotal study is a 12-month study. On 6-minute walk, we showed a 42-meter improvement in ERT switch patients. So those patients who, generally, after several years on the standard of care ERT had been declining. We had hoped, frankly, that we would stabilize the decline. What we actually showed is that in virtually all those patients, we can reverse the decline. So with 42-meter improvements, that's very, very meaningful for those patients. And in our Cohort 3, our treatment-naive population, we showed a 63-meter mean improvement with a very tight standard deviation and improvements in 5 out of 5 patients. Those are huge improvements, so -- when you look at a range of other endpoints. So that's one distinction from what others have studied. The study population, again, too -- you referenced the Genzyme neo study. I guess we'll see that data next week. I hope for patients that they show a substantial benefit over the approved standard of care with their product, Lumizyme. But I have no idea what it will show, so we'll see. I do know for our product, we did study or are studying both switch and naive patients. The neo COMET study, I believe, is a naive study-only. And again, biologic differences going back to almost a decade ago when we set out to build this program, it was -- we were building a cell line with -- that was selected because it had high levels of mannose-6-phosphate. So the mannose-6-phosphate targeting the overall carbohydrate structure is different, and it's produced differently for our enzyme. We chose -- chose cell line with a naturally high level of mannose-6 phosphate. We chose not to post -- excuse me, post translationally modify the protein. In addition to confer added stability, we do have the patient take a chaperone about an hour before, and that's the regimen. So we think a very distinguished study that we've designed, distinguished [ from ] prior data and significant biologic differences. But ultimately, it will come down to the data in the pivotal studies. We'll see the neo data here shortly. We'll see our data in the first part of 2021. But we have a lot of confidence in what we've built.

And John, can you speak to your gene therapy approach here? So as you've mentioned, very large opportunity in Pompe, and we've seen some gene therapy companies working to get a gene therapy available for lysosomal storage disease, and it's been challenging. Maybe you could speak to the challenges here, how you think you're going to be able to get over the goal line with your construct and then what your manufacturing process entails because I think you are looking to scale up ahead of starting that trial.

So I think this is actually a great example, Salveen, of the bringing together of the best technologies from Amicus and from Jim Wilson's lab at U.Penn. So what we wanted to do is to make sure that whatever vector we chose, whatever gene therapy construct we used, that the protein that it expressed was highly targeted to muscle. We also realized that Pompe is ultimately not only a neuromuscular disorder, it's a disease of motor neurons. And we needed to treat the CNS aspects of the disease as well. So targeting to the CNS either through the protein engineering or -- and/or through the route of delivery. And that's exactly what we've built. So Amicus designed the DNA sequence, the construct for the production of a highly targeted protein, and it was that construct that we inserted into a proprietary cell -- a proprietary vector at U. Penn, and that's what went into the animal studies. So again, the focus really is on leveraging the more advanced vectors out of U.Penn and putting into those vectors a transgene engineered by Amicus to be highly targeted to muscle as well as to the CNS. We saw great data preclinically. And again, you've got to remember, for any disease, but particularly for a disease like Pompe with an AAV technology, patients are very likely to have only one shot to take a gene therapy, so they need an optimal therapy. You cannot tell a patient, well, that's -- we understand, but that will be our second-generation approach. That doesn't work. So we had to go in with our best foot forward, our best technologies. And that's exactly what we hope. And if we see in the larger animal studies now with targeting, and then ultimately, in people, that we are able to effectively target all muscles of disease, I hope that we would have a very distinguished product as well. So that's the approach that we've taken. Importantly, Salveen, by also properly targeting the protein, we could have a much lower dose than others have proposed, which could enhance the safety profile. And to your question about manufacturing, it could also mean that certain modalities of manufacturing are open to us that may not be open to others. So right now, we're working with Thermo throughout this year really to finalize what that manufacturing platform will be, whether we could stay at a reasonable scale in the iCELLis system or whether we will use one of the suspension systems that we're working in right now. So to be determined. We'll have more data on that by the end of the year, and again, hopefully in the second half of '21, be able to file the IND.

Great. And then your other gene therapy program that's actually in the clinic right now, so Batten disease. Can you just remind us what we should expect in terms of data flow from your CLN3 program? And then also -- and then just walk through what we've seen to date. And here, what we should be looking for in terms of determining efficacy?

Sure. So again, through the acquisition of the spinout company Celenex, out of Nationwide Children's Hospital, we acquired the rights to a portfolio of programs in Batten disease. Batten is kind of an umbrella for about 14 different subtypes of Batten disease. We have now in the clinic 2 of those programs. One is CLN6 Batten disease, and there are about 750 to 1,000 children diagnosed with CLN6 in the addressable markets. We have now treated 13 children with the intrathecal delivery of an AAV-based product. We presented some of that data last year. We saw for many of those children that we had stopped the progression of the disease. And we showed a profound difference from the natural history. We showed a profound difference from their untreated or even, in some cases, they have a later-treated siblings. So we've got a lot of hope and belief in that program. The focus for CLN6 this year has been on transfer of manufacturing from Nationwide to Thermo Fisher Brammer, and we've completed that tech transfer. We're now underway with the GMP manufacturing. And the other focus has been on regulatory discussions. I want -- I need to have a plan with the FDA for exactly how we're going to get this approved. It is one of the more advanced gene therapy programs, having treated its first patient several years ago and now with 13 children dosed with gene therapy. We need to treat additional children with the GMP material. How many children and for how long is still to be determined. But hopefully, we'll have that guidance here in the second half of the year, be able to treat those children beginning in the first part of 2021, and then I hope have a path toward a BLA submission for their -- from there. The second program is CLN3 Batten. This is the largest of the Battens. It's actually the largest genetic fatal brain disease in children and also the most prevalent form of genetic blindness in children. We are the only company currently in the clinic with a CLN3 Batten's program, again, about 5,000 children with the disease in the addressable markets. We've treated a handful of children first at a low dose, intrathecal AAV. We've now treated at the high dose as well. Second half of this year, we'll have some of that data on 1 or 2 years of data for those children, both safety and an initial look at efficacy. But again, similarly, we've transferred that cell line from Nationwide Children's to Brammer Thermo Fisher. That tech transfer is complete. They're now doing the GMP manufacture. Next year, we will treat more children. That will have to be a larger study. My -- our thinking now is that's about 12 to 24 patients, children. And for how long is still to be determined, whether that's a 1 or a 2-year dosing period. But again, I think that could have a profound difference that you see the results on some of those kids with CLN6, kids who by age 5 or 6 should have been in wheelchairs, should have been blind, should have not have had the ability to speak or even think, and some of them moving to kindergarten. That's a significant change. And the longer we go along here, the more and more confident we are that it's a true separation and treatment effect from the natural history. So a lot of activity in the second half of this year, but really 2021 is when we move those programs forward as fast as we absolutely can into more patients with GMP material, with a clear road map toward BLAs in both of those programs.

I'm sorry, I misspoke, I meant CLN6. But as you look...

I got CLN3 and CLN6 in, so -- and we have other CLN programs in preclinical development. And in fact, hopefully later this year, we'll be able to share some of that preclinical data in those other programs.

How do you think about the read-through from the CLN6 program to the CLN3?

Yes. It's -- we're using the same vector. We're using the same route of delivery, the same core technology that was used for the AveXis programs, that Brian used for AveXis. So I think we've got some strong faith in the technology proven in SMA now taken into Battens. And certainly, with Battens going from CLN6 to CLN3, although the proteins are different, it's the same basic pathophysiology of disease. So it would give us -- and the same symptomology as well. So because of that, we have a high degree of confidence. CLN3, the progression of the symptoms takes longer in CLN3 than CLN6. But again, in the end, these children in their childhood will die of Batten disease, and that's what we need to intervene and stop.

And given that this is a progressive disease, and ideally, you want to treat the patient as young as possible with the gene therapy, how are you matching the data set that you're studying with the natural history data set? And how robust is that in order for you to provide the regulatory agency enough color on the effect that you're seeing here?

Yes, so we showed last summer, the data that we had compared to natural history, and we matched it by age and by progression of symptoms with those children. So it was pretty well-matched, Salveen. We're now completing that natural history study. We need to get that up to about 30 or 40 patients. Similar, for instance, to what BioMarin had as a control group for Brineura and CLN2 Batten disease. So we've been working on adding to that body of data in CLN6. But what we've matched is very consistent, and I think very-well matched with our treatment group. CLN3, we're actually in a really good place. The field academics, patient foundations had done quite a bit of work because there's a much larger population and built a much more robust natural history data set. And there, I think we'll be in a very, very good place with the data -- natural history data set of about 100 patients or so here over the next year.

And where do you stand with the Fabry gene therapy program that you're working on?

Yes. That's one, again, partnered with Jim and U. Penn that again combines the Amicus protein engineering technologies with Jim's vector science and gene therapy science. We have -- we will release this in the second half of this year. All of our preclinical proof-of-concept data, we've not shown any of that to date. So that research was completely unaffected by the COVID crisis. We were able to keep the work in the Amicus labs and at Jim's labs ongoing. That data is reading out now, and we're very excited to be able to share that, and that will then give us a path toward the clinic. And again, the same thing, we want to make sure that the protein that's being expressed by the vector is the most highly targeted, safest and most appropriate. And that's, again, the differentiation from the Amicus approach from anybody else's approach in the field, focus on both the protein and the vector. And I think with the Amicus science teams and with Jim's science teams, we're bringing a lot of experience and a lot of resources to bear. So hopefully, we'll see some strong data second half of this year in Fabry gene therapy.

And then moving to Galafold here, so...

I was going to say we have a commercial product that's going -- doing quite well.

So you seem to have had pretty limited impact from the COVID-19 pandemic on the sales effort here. Could you just comment on whether that has been continuing into 2Q and how you're managing the situation in the context of these patients?

Yes. We continue to see very strong trends for Galafold. The product had a very successful launch. You saw the sales figures for last year, and Q1, we released the sales figures as well. We're getting to more patients. And importantly, when patients come onto Galafold, they stay on Galafold. So there's very high rates of adherence and compliance. We continue to see that as well. We saw it in Q1. We continue to see it to this date as well. The supply chain was completely uninterrupted by COVID-19. We did take some additional steps to push drug deeper into the supply chain actually, to the individual distribution sites in each country. But we had ample supply of the drug. We had already been very conservative in having multiple supply stations around the world. So disruption of supply was never a factor for us. And Galafold, I think, is particularly well-suited in the COVID crisis for patients who do not want to go to the hospital for infusions, or in some cases, also patients who don't want a nurse coming into their home during the COVID crisis as well to move to an oral therapy. And if you look at the overall opportunity to help people with amenable mutations in the markets where we're now launched, which are all the major markets in the world, we've seen similar continuing trends. We're in all the large markets now. A majority of patients eligible for Galafold have now taken Galafold, but we still have many, many more patients that we can move on to Galafold, both treatment-experienced, treatment-naive and to-be-diagnosed patients. So we're really confident. We continue to reiterate our guidance of $250 million to $260 million in sales, which is pretty remarkable for this drug in a competitive market space in only its second full year of launch in the major markets in Japan, the United States and in Europe, but still much, much more to go. We are also very confident that this drug is on a firm trajectory to $500 million in global annual revenue in 2023, and in the mid to late 2020s, $1 billion in peak potential.

And as you mentioned, you're in many of the major markets. But it's just recently that you've launched in some of them. So how are you thinking about the growth trajectories in the various markets?

It really is across the board. So in markets like some of the countries in Western Europe, in Germany and the United Kingdom where we're now seeing 80%, 90% of the patients who are eligible to switch, having switched, it's making sure that they continue to have a good experience, they stay on the drug, and we do continue to see that. So incrementally more treated amenable patients to continue to switch there. In the United States, we're at about 50% switch patients. So we still have another half or so of the treated amenable population to continue to move toward Galafold. In Japan, we're not quite yet at 50%. So again, in the major markets, many more treated amenable patients to continue to put on Galafold. Then there's the larger -- in many geographies, especially in the United States, the larger population of untreated but still diagnosed amenable patients. We continue to see more and more of those patients. Now the doctors and family members, given that it's an X-linked disorder, are having a good experience on Galafold. We see now many of those patients putting their hands up and saying, I'd like to go on therapy or the doctor is now recommending that they go on therapy. So many more of those treated -- I'm sorry, untreated amenable patients. And then more and more newly diagnosed patients. This continues to be recognized as one of the most misdiagnosed or underdiagnosed diseases in the world, genetic diseases. And in those cases, where there is a newly diagnosed patient with an amenable mutation, we're finding 90-plus percent of the time that doctors are choosing to put them on Galafold, not on one of the ERTs.

And John, maybe one last question here. Just going back to the manufacturing aspect associated with gene therapy. So I mean you do have a wave of programs, right, between the ones that you have in the clinic and what could be coming here. So with regard to your collaboration with Thermo Fisher Brammer, do you have dedicated suites? How are you going to deal with manufacturing them for product basis? And any plans to bring manufacturing in-house?

Yes. I've been really, really pleased with the Thermo Fisher and the Brammer team, just enormous capabilities. They're building significant capacity, Salveen. And I think Marc Casper should be very proud of the acquisition of Brammer. And their ability to build that out into the Thermo structure really leveraged the resources of Thermo Fisher. For us, we've entered into a collaboration agreement. We have dedicated reserved suites. We have priority access we've now negotiated, and we have a long-term supply agreement for multiple of our programs. We would expect to continue that long-term relationship to expand it, I would believe, in the years ahead with Thermo Fisher. In addition, though, I believe if we're going to be one of the world's leading companies in gene therapy in the world, we have to have our own capabilities and our own capacity. We have about 70 employees at Amicus who are in manufacturing, technical operations and quality. We built that team for AT-GAA in partnership with Wuxi Biologics for our enzyme replacement program. So we have a lot of internal expertise in complex biologics manufacture, even though for AT-GAA, we never operated our own facility. For gene therapy, we believe, again, that we need to have our own capacity and capabilities. So we're currently designing a clinical-scale manufacturing facility. We've also acquired land to build a commercial-stage manufacturing facility. So over the next couple of months and years, I think we'll be able to describe that more fully as well. So I think we'll be a fully integrated company on the gene therapy side as well.

Great. Well, with that, thank you so much, John. Really appreciate your time.

Great. Thanks, Salveen. Have a great day.

You too. Bye.

Take care.