Let's go ahead and get started. Welcome to the afternoon session of the Monday of the 41st annual JPMorgan Healthcare Conference. My name is Anupam Rama. I'm one of the senior biotech analysts here at JPMorgan. I'm joined by Malcolm Cuno and Priyanka Grover from the team. Our next presenting company is Amicus Therapeutics, and presenting on behalf of the company, we have CEO Bradley Campbell. Brad.
Thank you, Anupam. Good afternoon, everybody. Great to see everybody live and in living color here at JP Morgan. Thanks to Anupam and JP Morgan for inviting us here. Thanks for also setting expectations that it was gonna rain all day. I don't know if anybody's been outside. It's actually bright and sunny outside, so well done setting low expectations and meeting and being them. I will be making certain forward-looking statements, so I'll refer you to my Safe Harbor statement. Just as a reminder, Amicus Therapeutics, our passion is for patients. Our mission is to develop next generation therapies, living for people living with rare and orphan diseases. Our vision is really to be a leader in the rare disease space with global clinical and commercialization capabilities around the world.
I think we've built a special and a rare company here. It's something that's happened over more than a decade since our founding. Today we have GALAFOLD, which is our small molecule chaperone for the treatment of Fabry disease. It's a healthy and growing product. We announced this morning that we'll do 12%-17% growth in the GALAFOLD business this year in 2023. That's a constant currency. We have a second product, AT-GAA for Pompe disease, which is in front of regulators around the world. We anticipate seeing regulatory approvals and launches for that product. All of that is supported by our global commercialization infrastructure that we've built over time.
We have global clinical development capabilities. I think that puts us in a relative unique position, in the midcap biotech space with two products, with $1.5 billion-$2 billion peak revenue combined. All of that with a very healthy balance sheet and committed to generating non-GAAP profitability in the second half of this year. 2022, a tumultuous year, I think, in the markets. Certainly a lot of opportunities in the biotech space to continue to deliver value and to execute across our business. We saw a number of important milestones. We saw operational growth of GALAFOLD of 16%, which was well within our guidance.
We continue to expand our intellectual property and our patent portfolio, continuing to provide perfection, protection for GALAFOLD, to the better part of the next decade. Continue to bring data out across both our Fabry program as well as our Pompe program, and we'll talk more about that as we go on here. Growing demand now for experience with ATGA. We're in front of regulators, as I mentioned, but we have a number of expanded access programs around the world that continues to involve both investigators and a growing number of patients. That'll be an important part of the story going forward.
Of course, hitting important regulatory milestones, we'll give a lot more color today around where we are with the FDA, but also ended the year on a really positive note, saw a positive CHMP opinion for Pombiliti, which is the enzyme replacement therapy portion of our AT-GAA therapy for Pompe disease. I think that sets us up for what may be the most important year in our history as Amicus. Again, continuing to bring GALAFOLD to as many patients around the world as possible. That's the core of our business, the core of our value. Finally now on the cusp of securing regulatory approvals for Pompe disease for AT-GAA, both the FDA, EMA as well as the U.K.
Of course, that will lead to the commercial launch of our second product, which is again a relatively rare feat for homegrown biotechnology companies. We do have a pipeline both in Fabry and Pompe gene therapy, and I'll talk a little bit about that. Continue to believe we'll play a leadership role in developing new treatments for those two diseases and all of that with a backdrop of a very strong financial profile, and again, on that path to profitability. We think 2023 will be a transformative year for Amicus and eager to continue to execute across those strategic priorities. Let me turn a minute to GALAFOLD. That's our small molecule precision medicine for Fabry disease. Just as a background, a primer on the disease, most of you probably are familiar.
Fabry disease is a lysosomal storage disorder. It's a genetic disease caused by a deficiency in the GLA enzyme. That enzyme is responsible for recycling a substrate called GL-3. The important part of that is that when that substrate builds up over time, Fabry patients have significant morbidity and mortality. Primarily Fabry disease impacts kidney, heart, CNS, also life-limiting symptoms of pain, GI distress. Importantly, it is a broad and large lysosomal storage disease. Today, there are 16,000 patients diagnosed worldwide, roughly 50/50 males and females. We think it's a significantly underdiagnosed disease, and we'll talk more about that. The Fabry market today is a little under $2 billion globally. I'm sure it'll pass that important milestone of $2 billion this year.
It is a healthy and growing market, and it's really driven by, again, that underdiagnosis. It's believed to be one of the most underdiagnosed and misdiagnosed human genetic diseases. In fact, newborn screening and most of the recent high-risk population screening studies would suggest that it's potentially tenfold more prevalent than it was originally characterized. So you have a very healthy diagnosis that's fueling that market growth. Importantly, GALAFOLD for the past two years has been the fastest-growing of the Fabry treatments and also the largest contributor to that growth. In fact, we now have over 1,000 patients who were naive to treatment before they came onto GALAFOLD, and so we've grown the market by 1,000 patients since our launch. Just a snapshot of what we delivered on in 2022.
Again, $329 million, we announced that today in our press release. $329 million in global sales. Unfortunately, significant impact from foreign exchange impact. We have significant exposure to the pound, to the yen, and to the euro. Even with that, we delivered on our operational growth guidance of again 16%. We now have 45 countries globally where we are approved, and we're continuing to pursue reimbursement in those remaining markets. And again, this is a precision medicine, so we continue to expand the amenable variants that are described in the FDA label and the European label as well. That translates to about a third to a half of patients living with Fabry who have those amenable variants. How do we know the business is performing well?
We have a number of key performance indicators that we talk about. I think the most important one is the global demand for this medicine, which is now 2,000 patients globally take GALAFOLD as their only treatment for Fabry disease. There was a time many of us may remember, I was back at Genzyme when we launched Fabrazyme back in the day. I don't know if we knew that there'd ever be 2,000 patients who were diagnosed with Fabry disease, much less on treatment, much less on a small molecule chaperone GALAFOLD. A really exciting indication of the number of lives we impact, but also the underlying demand of the business. A couple of things that were really important this year in 2022.
Net new patient trends for the past three months were better than the past six months, better than the past 12 months. Seeing some momentum, I think we're finally starting to leave the COVID impact on this business behind. Importantly, too, in the United States, the best year we've seen for net new patients since before the pandemic. I think you're continuing to see this growth in our key markets around the world. We're now at a 50% market share of treated amenable patients. Good news is lots more patients to switch, but a really healthy share of patients who have amenable mutations who are treated now with GALAFOLD. A healthy mix of switch patients versus previously untreated patients.
All of that is underpinned by a over 90% compliance and adherence rate, and we've seen that ever since launch, and in fact, trending slightly positive to that. Really important that once a patient comes onto GALAFOLD, they tend to stay on GALAFOLD. In 2023, how are we gonna continue to grow the business this year and over the next decade? Again, continuing to penetrate into the remaining switch population, continuing to get uptake into the diagnosed untreated population, continuing our geo-geographic expansion, and again, supporting that compliance and adherence. Again, that's all supported also by positive reimbursement and access mechanisms around the world. Again, we do continue to build the body of evidence in support of GALAFOLD.
We've released a number of papers over the last year looking at GALAFOLD, in terms of compliance, in terms of quality of life, in terms of the benefit on Fabry-associated outcomes, in terms of the importance of early treatment. This continues to be important part of the story for this oral precision medicine, and you'll continue to see us focus on this. In fact, one of the important milestones is we'll have the first publication coming out of our own followME registry, which is our Fabry registry, and that'll be exhibited at the WORLDSymposium here in February. Really all of that is, I think, is a backdrop for where do we see this market going over time. Over the next five years, we think this becomes over a $2.5 billion global market.
I think importantly, we'll pass a milestone where we see the amenable portion of the population for the patients that we can treat with GALAFOLD. That market should increase to over $1 billion globally. Lots of opportunity for us to continue to grow this business over time. Just to give you an idea, in the markets where we've been launched the longest, so the U.K., Germany, as an example, we get to 80% or 90% market share. We really believe that we will be standard of care in those amenable population and look forward to see that backdrop of market growth as we continue to build the business over time. Switching gears now to AT-GAA, which is how we describe our two-component therapy for Pompe disease. Maybe just a primer first on Pompe disease.
Again, another lysosomal storage disorder, this one primarily affecting the neuromuscular. It is a severe fatal disease if left untreated, and it's due to a deficiency of an enzyme called GAA or acid alpha-glucosidase. Again, like Fabry, now known to be more prevalent than what was originally characterized in the literature. Looks like a live birth incidence of 1 in 28,000 or even more prevalent than that. And that's all supported by newborn screening studies in the United States and around the world that continue to find more Pompe patients. It impacts both infants and adults. It's a spectrum of severity depending on when patients are diagnosed. Again, the primary morbidity and mortality here is cardiac, and pulmonary respiratory failure. The global Pompe market, also a healthy and growing market. It's a roughly $1.2 billion market today.
That's about 3,500 - 4,000 patients who are treated. You can see on the right-hand side how this breaks up from a geographic perspective. About 40% of the market from a dollar perspective is in the United States. From a patient perspective, it's more evenly split, and then the remainder is split between Europe and rest of world. And again, we see this market growing significantly over the next five years. This should be approaching a $2 billion market. Here, AT-GAA, we believe, has the ability to treat all patients with Pompe disease, again, depending on the label, and I'll talk more about that over the next coming slides here. What's different about AT-GAA? It is the only two-component therapy in development for Pompe disease, excuse me.
Effectively, we took a naturally selected cell line with a unique glycosylation pattern, highly phosphorylated enzyme replacement therapy that's intending to solve the uptake and targeting challenges in Pompe disease. We combine that with a small molecule miglustat, which acts as a stabilizer. It keeps the enzyme properly folded while it's in the circulation. Again, the only two-component therapy in development. Just a reminder, the data we showed in our phase III studies, our PROPEL study, this was a mix of switch and naive patients, although the vast majority were in fact switch patients compared head-to-head versus standard of care. What we saw in the primary endpoint, 6-minute walk distance, we saw a numeric benefit for ATGA compared to the control, just missed statistical significance.
On the key secondary forced vital capacity, again, in the overall population, saw nominally statistically significant improvement in forced vital capacity versus the control. If you look at the larger subpopulation, the largest subpopulation, and this is really where the majority of the opportunity lies in Fabry disease, we saw nominally statistically significant improvements in six-minute walk distance, and likewise, nominally statistically significant benefit in forced vital capacity versus the control arm. We're the only program that studied in a controlled portion of a phase III study, these switch patients. Again, this is where you see the greatest unmet need in Pompe disease. Really exciting data. This is what drove the applications that we're now pursuing around the world. Just to give a quick update on where we are. We've broken this now into three key regions.
These are the three largest market opportunities for Pompe disease. The first is in Europe. As I mentioned earlier, Pombiliti, which is now the trade name for the enzyme replacement therapy component. There are two separate applications. We received a positive CHMP opinion in December. We now expect a CHMP opinion on the small molecule miglustat in the second quarter this year. That would lead us to EC decision in the first quarter for the ERT, in the second quarter for the small molecule, which would get you to approvals and launches in the second half. In the United States, as I mentioned, we are also on file with the FDA. We did receive a deferred action letter at the time of our BLA.
Just for clarity there, those can only be issued in instances where the inspection is the only reason for the cause of the deferral. In fact, the technical language is that there are no deficiencies found in the application and it otherwise satisfies the conditions for approval. For us, this is a matter of when, not if this product gets approved. The other thing that we announced at the end of last year is that we had scheduled a Type A meeting with the FDA in order to provide plans and logistics to conduct an inspection. We had that Type A meeting, very successful, very collaborative, very eager to see us get to the point of scheduling an inspection. Haven't scheduled one yet.
However, I think you've seen the situation on the ground in China continues to get better and better. A number of the roadblocks in terms of quarantines, in terms of hospitalizations, in terms of providing negative testing, those things are going away. We think that further supports the opportunity for us to get an inspection scheduled as soon as possible. We're eager to see that happen. Once that happens, we'll provide further clarity on when we expect an approval later this year. Finally, the U.K. This is something we didn't talk a lot about, but we did actually begin the AT-GAA regulatory process in the United Kingdom. That's through the recognition procedure that keyed off of the Pombiliti approval.
That process is now underway as well, and I'll talk a lot more about why that market's so important for us in a couple slides. Just a little bit of a drill down into Europe. First, it's a very sizable market opportunity. As I mentioned, more than a third of the global opportunity from a dollar perspective sits in the European Union. That's about a $450 million market from a dollar perspective. Importantly here, we saw a very strong indication statement coming out of the Europeans, both talking about the long-term enzyme replacement therapy, but also the point that this is to be used in all adults with late onset Pompe disease, so really strong label coming out of Europe. There are about 1,300 patients that we think we can address there.
Importantly too, continue to have a broad experience with KOLs throughout the continent, both through our clinical studies, but also through expanded access programs that are active now in France and in Germany, and there are other opportunities in other markets as well. I would note too that many markets around the world will actually leverage the European label as we go to pursue approvals in other markets outside of the US and Europe. The US we talked about, of course, largest market, about $500 million, a little over $500 million from a dollar perspective. Continue to grow at a robust rate, significant uptake in the use of newborn screening in Europe as it relates to Pompe disease. We continue to see this as a healthy and growing market.
As of course, I'm sure you all know, there was a second product that was launched by Sanofi, avalglucosidase alfa. Year to date, nine months in the U.S., that was doing about 30% market share from a dollar perspective versus 70% for the standard of care. And the overall treated population is about 800 patients on treatment in the United States. As we look to, again, that differentiating data in that experienced population, really eager to be able to bring AT-GAA to the European market once we see final approval from the United States. Then the last thing I'll touch on just from a market perspective is the U.K.
It is the, as I said, top three markets, if you think about the U.S., Europe, and the United Kingdom. There we've begun the recognition procedure to pursue regulatory approvals. Really excited to see a growing body of experience through the EAMS program, the Early Access to Medicines Scheme program. Dozens of patients on treatment today through this program, many centers who have experience, and now over 200 people in the United Kingdom are estimated to be treated. So if you think about us with, you know, dozens of patients now in AT-GAA, significant number of them will be on our therapy even at the time of launch. That all is part of the ongoing clinical experience that we're seeing with Pompe around the world.
The only other detail I'll add here is that now over 75 centers around the world have physicians who are using AT-GAA to treat their patients. A growing prescriber base, as we enter into regulatory approvals. Very much launch ready. We are ready to go, super eager. We have a very healthy and experienced commercial organization. The one thing that we'll add here is that we have a very synergistic ability to use this commercial organization, only anticipate adding about 12 employees globally to support the launch. Just rounding out, I'll talk a little bit about the corporate outlook going forward. First and foremost, as I mentioned, we do have a pipeline.
I know all eyes are on our commercial business with GALAFOLD, anticipated approvals of Pompe, I would orient you to our Fabry and Pompe gene therapy, which I'll talk about in a minute. The collaboration that we announced with the University of Seville looking for next generation, chaperones for Fabry disease. They're really seeing whether we can expand the number of mutations that might be eligible, improve upon some of the chaperoning properties, continuing to be leaders in the Fabry and Pompe space. As a reminder, we do have some legacy gene therapy programs, in particular in Batten disease. We have an ongoing phase one, two study in CLN3 Batten. Intriguing data there.
I think there could be utility with that, at this point, it's likely off strategy for us, and so we're thinking about how best to move that program forward in the right way. Again, do see some, I think, intriguing data there that it could be helpful for those patients. Finally, some ongoing early research and discovery work just on platform next-generation medicine approaches. A minute on Fabry and Pompe gene therapy. As we focus on a commercial business, as we're looking to turn profitable this year, we're really, I think, investing a judicious amount of capital here to continue these programs going forward. Just a reminder, we use our unique protein engineering capabilities to optimize the engineering, the stability, the targeting, the update of these transgenes.
We think very differentiated approach to what else you see in the market for other Fabry and Pompe gene therapies. We've shown really exciting preclinical data where we see significant uptake and impact on glycogen and GL-3 reduction in the two diseases, we would look to continue to invest in these programs again once we turn profitable and again, put more resources behind them, but important parts of the future of Amicus. Finally, just the key milestones that we can look forward to this year. As I said, continue to grow GALAFOLD, 12% - 17% at constant exchange rates. Starting to see publications coming out of our own Fabry disease registry. Regulatory approvals in key geographies for Pompe, followed by, of course, beginning the global commercial launch, critical milestone for us this year.
Also, we've announced that we will have our long-term data for the PROPEL study, that will be, highlighted at the WORLDSymposium. Finally, I think turning the corner, to non-GAAP profitability in the second half this year. I'll leave you with, you know, look, we believe the true measure of success for Amicus is the number of lives that we impact with our medicines. The more patients that we can put on our medicines around the world, the more value we can create for patients, we think the more value then we can create for shareholders. Thank you very much for your time, I guess we'll turn it over to, questions. Maybe the man at the team can join me here. I guess that means I get to stand, don't I?
Maybe I'll start. I'll just also note that if you'd like to send a question via the question portal, I have this fancy iPad here that I can see your question, and I'm happy to ask on your behalf. Also, there's a mic runner in here, so you know, if you wanna ask a question, just raise your hand, and we'll make sure it happens. On the non-GAAP profitability guidance, what's assumed in there for AT-GAA specifically? I guess, what keeps you up at night, Brad and Daphne, about this guidance?
The biggest contributor still will be GALAFOLD revenue, of course. That's gonna continue to be the largest part of the top line growth. We do assume, I think, modest revenue from AT-GAA this year based on the regulatory timelines that we talked about on Pombiliti. The biggest goal for us for AT-GAA from a commercial perspective this year is to convert the patients that we can in the markets where we see approval, to get approvals first, then to launch, then to convert those patients. That really sets us up for a very strong base next year. I think the other piece of the puzzle is really around expenses. Maybe Daphne, I'll ask you to add a little bit of color there. Prudent expense management has been part of our focus for the last two years, and we'll continue that going forward.
Then the last piece of the puzzle is really around the inventory, which today hits the P&L, and it's significant, and that's relates to the AT-GAA manufacturing. Once we get to approvals, that moves over to the balance sheet. You know, what keeps me up at night? The biggest thing for me, I think we have clear line of sight to a great product. We have clear line of sight to a clear to a regulatory path in Europe and in the U.K. I'm very confident that we're making great progress with the agency on scheduling that inspection. I think the conditions on the ground are getting better and better to support that. I want that thing scheduled so then we can provide clarity to everybody. To me, that's the one thing that is. That's still to be.
I think we'll really solidify that timing to profitability. I don't know, Daphne, on the expenses, maybe talk a little bit about the trajectory of expenses this year and going forward.
Sure. As Bradley mentioned, you know, there's a, there's a piece of R&D expense that will be moving to the balance sheet, so you will see a decline in total OpEx this year as 2022 as compared to 2023. Over in the, in the outer years, as we continue to invest in our pipeline, we'll see that modestly tick up. That's how we're thinking about the OpEx spend over the next few years.
Yeah. I think we've said before, that this year in particular, you'll see OpEx actually fall below even, I think, years, sort of the 2019, 2020 years. We'll give more guidance on that at our year-end call, but OpEx is an important lever for us. To the extent that we do modestly tick that up over time, revenue will far outpace the expense line.
How do we think about the SG&A line in terms of synergies between the sales force and marketing around 2 lysosomal storage disease products? Not... Maybe there's an uptick this year, but longer term.
Yeah, I can take it if you can.
Sure. I mean, just to recall, we've built out a global commercial infrastructure, which was you know, being used just for one product. Now that we have the second product, that will drive much more efficiencies and much more leverage. That's how we're thinking about the SG&A spend. There's a modest maybe increase as we launch AT-GAA, but we basically have the infrastructure set.
We did a really cool study, looking at the overlap between the Fabry treaters and the Pompe treaters, and what we found was about 30% of the treaters were actually the same between the two products, so obviously highly leverageable. More than 50% of the treating centers are the same, and we tend to hire very experienced, what we call, key account managers. In their mind, they're actually working with the center itself, not necessarily just the physician. Over 80% of the cities are the same. That allows us to really get leverage out of the existing commercial team.
I guess one thing that I think sometimes folks worry about is when you put up the broader pipeline slide and you see the words gene therapy and things like that, they think of long, expensive trials, CMC, right? That perhaps, you know, is not like longer term, or if you're just thinking about bottom-line growth, not optimal if you have two growth products, right? So help us work through that dynamic of why that type of investment is important.
Yeah. First of all, I think it's important for Amicus to maintain its leadership in Fabry and Pompe disease. Right now, we think between the chaperone and those two gene therapies, those are the best next-generation therapies that we can be investing in. Totally agree with you, that was really the pivot, the strategic pivot earlier this year away from gene therapy is these things are costing more money, at least in our hands with AAV, with the diseases we are focused on, costing more money, regulatory timelines longer than we had anticipated them being, and really not optimized, I think, for successful commercialization. That's why we really took a step back, doing some foundational work, looking at manufacturing in particular.
If you can bring the cost down, I think it's a much more reasonable expense line for us, but also value proposition for payers once you launch. Bring the cost down, optimize efficiency of delivery and transduction, looking at some foundational redosing immunogenicity kinds of issues. Once we solve some of those things, I think you can see us moving forward in a judicious way. But totally agree with you, the most important focus is execute on the top-line business, get to non-GAAP profitability, and then preserve that going forward in whatever way we think about R&D expenditure.
Question from the audience. John?
Brad, just to make sure. Right now, you still assume the FDA would physically have to go to WuXi?
We do.
Okay.
That's what we're working on with them. Again, I mean, it's literally like the nuts and bolts of the logistics of an inspection. You probably heard this language towards the end of last year. We had been working on kind of a closed-loop inspection that would allow them to operate within the Chinese government requirements around quarantine, around, you know, documentation of negative COVID status in order to travel throughout the country. Those regulations are ending.
Mm-hmm.
I think that's gonna make it even easier. We're still working carefully to provide a similar level of protection, but I think it'll be in the backdrop of a much less regulated kind of environment. That's what gives us, I think, continued optimism that we'll be able to get them there in person. There are backup strategies we continue to work on. It's possible that we can end up, you know, they can do a remote inspection or a hybrid version, especially now that their Chinese teams can also travel more freely. Right now, the focus is get them there in person, and I think they're, you know, eager to do that as well.
what do your consultants say on the timing in which, let's say they go to China, inspect, and-
Yeah.
everything's fine. How long does that take then, just to kinda go through the nuts and bolts before they can-
The actual inspection itself, typically within one calendar week. You know, it's kind of bookended by a weekend. Occasionally, they can stay longer. I think in this case, they'll probably not wanna stay more than that amount of time. Don't wanna speak for them. Once they go through that process, typically takes what we understand about 60 days to go from conclusion of the inspection to the eventual approval. That's why, you know, again, as soon as we have clarity on the inspection happening, then we can give more line of sight to when we think an approval's gonna happen.
What about for Europe? Like, where are you in terms of finishing the confirmatory analytic testing that's required here? Maybe describe what exactly EMA-
Yeah.
wanted there.
The short answer is on track, which is great. That would get us to a CHMP opinion for the small molecule in the second quarter. Maybe, Jeff, just talk a little bit about exactly what we're trying to show and... Sorry.
Sure. You know, this is related to analytical testing of the drug product. We had provided all the information that we needed for the submission. During review, some of the sort of, not guidance, but the sort of internal policies of EMA got to a point where they said they actually wanna see specific testing with a validated assay of these, confirmatory, analytics. We got the four-month, wait.
Clock stop.
Clock stop, sorry. We're on track, as Brad said, to have that. We're making great progress on those assays, and we're on track, as we said, for the second quarter to submit those final validated analytical data. Basically, just like in the U.S. where the inspection is the only thing gating that approval, we really believe for that miglustat approval, it's just this confirmatory testing. All the efficacy, safety analysis and data is all the same as what was used for the approval of Pombiliti. Again, it's a matter of when, not if, and we have much more certainty on that timing for the second quarter.
The only other color I'd add there is that sort of keys off, again, these important regulatory events. EC decision for Pombiliti expected in the first quarter. CHMP opinion for miglustat in the second quarter. That gets you to 60 days from there, EC decision. You're talking kind of right at the end of the second quarter, beginning of third quarter for final EC decision, that keys your European launch. Of course, typically Germany first. Takes about 30 days to get product in the channel. We should see revenue starting to come in in the second half of the year.
Any questions from the audience? Okay.
Your 2023 revenue guidance expected to grow about 70%.
Yes.
Is most of that, based on the variability of AT-GAA, or is there some, like, currency effect in there?
That is specific to GALAFOLD. We aren't providing any guidance, we should say.
Okay.
our Pompe revenue right now. Again, I would expect modest contribution of revenue in the second half, but that 12%-17% is just GALAFOLD alone.
That 5% variability, is that due to...
I would just say that's a reasonable bandwidth to say what we can typically execute within.
That's fair.
Yeah. Any final questions? Yep, in the back.
Could you describe your understanding of the durability of the product of AT-GAA and how you view the world data kind of shaping that impression for doctors?
Variability in terms of efficacy.
Durability.
Oh, durability. Thank you. Yeah. Actually, Jeff, maybe talk to the long-term data of the product.
We actually presented data at the World Muscle Society from our phase I/II studies, where we treated both ERT naive and ERT-experienced patients. It was out to four years, and we've actually seen really good durability. In both groups, we saw improvements in motor function that was maintained out to four years. For pulmonary function, we saw naive patients have improvement that was maintained to four years. For the switch patients, their pulmonary function remained stable for four years. From durability, we're really excited what we've seen from the phase I/II studies. As Brad mentioned, we will see the first two-year data now from the PROPEL extension at the World Muscle Meeting coming up next month.
I think that continuing to build that body of evidence will be really important for physicians, for patients, to think about how this product can change the treatment landscape in Pompe.
Okay. Any final questions? All right. Thanks, everyone.
Thanks, guys. Appreciate it.