Thanks, Ron. Okay, well, good morning, everyone. I believe this is the first Fireside Chat of the day. So thanks for, thanks for joining this session. My name is Dae Gon Ha, one of the Biotech Analysts at Stifel, and joining me for the next half hour, we've got Amicus Therapeutics. Here with me is CEO Brad Campbell. So Brad, thanks very much for joining us for the next half hour. I'll, as I always do, I'll turn it to you for a brief intro of Amicus-
Yeah
For those that are unfamiliar, which is hard to believe, but please give us a debrief, and then we'll dive right into Q&A.
Great. Thanks, Dae Gon Ha. Thanks for having us. Thanks, Stifel, for the opportunity to speak here today. So yeah, Amicus Therapeutics, we're focused on developing next-generation therapies for people living with rare diseases. And for the first time now, we've done, I think, what is pretty rare in this space as an independent, mid-cap biotech company, which is we've launched our second drug, second therapy, I should say, Pombiliti + Opfolda for Pompe disease, which is fantastic. We're now launched in three countries around the world, four technically in Austria, so the United States, U.K., and Germany, the three largest markets, and launch is going really well. I know we'll dive into more detail there, but that's a product that I think significantly changes the opportunity to treat these patients living with Pompe disease, and we think that could be a billion-dollar-plus market opportunity.
Also will fuel significant revenue growth, in addition to Galafold, which, of course, is, I would say, the cornerstone of our valuation today. Galafold, a small molecule treatment for Fabry disease. Exciting thing there is, it's a precision medicine for people living with amenable mutations, but it is a growing product. It's growing faster than the other two products that are available today, and it's on the backdrop of a market and a patient population that continues to grow as well. So we think that also has a potential billion-dollar product opportunity. And again, this quarter, we actually surpassed $100 million in sales for the first time in a quarter, which is a great milestone for us.
Also, raised our guidance to 16%-18% growth, year-on-year growth, so it's a healthy and robust and growing product, and one that we think is making a big difference in the lives of patients. And then all of that is in the backdrop of continuing to evolve our financial strength. For the first time, this quarter, we'll be non-GAAP profitable, and I think that's a positive signal to investors that we are now headed to a path towards independence and being able to fund our own operations. And I think that's an important sort of shift after a company that's been around for a long time, to your point, and you know, largely been an R&D company historically, and now I think very much a commercial and growth company going forward.
Mm-hmm. So the market condition is now such that every launch story becomes very heavily scrutinized-
Mm
... by investors. And so even though you've done this with Galafold, I think people kinda see you as another launch story. So AT-GAA comes into focus. Before we dive in, can you just remind us the, the label that you got from the FDA, how that compares to the EMA approval label? And then we can kind of continue the conversation.
Sure. So just remember, the approvals were based on the largest ever controlled study in Pompe disease. It was a mix of switch patients, so patients who were on enzyme replacement therapy previously, and patients who were naive to therapy, but the vast majority of the patients were switch patients. And as a reminder, really the most important data that came out of that study was an improvement in six-minute walk in the patients who switched from standard of care. That's never been shown before, and I think the most powerful data set, and also stability in forced vital capacity. Those are the two registration endpoints that regulators typically look for. In the overall population, we just missed statistical significance on six-minute walk. We were statistically significant on forced vital capacity, but it was a secondary endpoint.
Then in the naive population, effectively, the control arm and Pombiliti + Opfolda or AT-GAA performed about the same. So both saw improvements in six-minute walk, but no differentiation. In Europe, they looked at the totality of the data, and we're effectively granted the label for late-onset Pompe disease patients. The most important thing there is you had those key data sets, the switch data, showing the improvement in six-minute walk and the stability in forced vital capacity. In the United States, I guess they took a slightly more strict approach, so the label in the United States is for adults with late-onset Pompe disease who are not currently improving on standard of care, and—excuse me—on enzyme replacement therapy.
I think that's a really important point that we'll come back to. Based on the enrollment criteria for adults, there's a minimum weight of 40 kg, and that was a question that I think came out when we announced the label. But the reality is, that's reflective of the weight of an adult patient, and so people under that weight are typically children. We are pursuing a pediatric indication as well, and so we'll be able to sweep in those patients at some point. But for now, that's the label. But I know we'll dive in more details, in particular, on that not improving point, which I think is really the strategic strength of the label for us.
As it pertains to the launch itself, I think we also need to take into consideration the EAP patients, as well as the clinical trial participants, which you stated as your number one goal by the end of the year. Can you just frame for us, what are those numbers across these three geographies or four geographies that you now have the approval on?
Yeah. So, as a reminder, we had our ongoing clinical study, so the PROPEL study, which then after, after the study read out, the vast majority of patients stayed in that study. And then we also had an increasing number of expanded access patients through a variety of different mechanisms. In the U.K., it was called EAMS, or the Early Access to Medicines Scheme. In Germany, it was called CUP, and I don't remember what that stands for, but that was the expanded access program there. All told, in Germany, we had 20 patients leading into the approval between the clinical trial and the expanded access. We've now converted 100% of those patients, so they're now on commercial drug. In the U.K., we had about 40-45 patients between EAMS and clinical trial-...
We now have 86% of those patients who are converted. All of the EAMS patients are converted, and we've now scheduled all of the clinical trial patients. It's a little bit more complicated because they have to come in for an end of study visit, but we'll convert all those patients by the end of the year, which is fantastic. And then in the United States, again, we had about 40 patients who were eligible for who are adults, frankly, and eligible for Pombiliti + Opfolda. We now have two-thirds of those patients who have been scheduled or sorry, who have prescriptions written and now are in the process of scheduling them. And again, the goal is to convert all of them.
So if you add all that math up together, we should have about 100 patients, conversion patients on therapy by the end of the year. And then, of course, now we're focused on bringing on new commercial patients, and what we shared on the quarterly call was we're starting to see patients in each of those markets come onto therapy, and importantly, from both of the approved enzyme replacement therapies.
Mm-hmm. The on the U.S. side, so you're already kind of receiving these start forms from these patients. What's been the experience like? Granted, you have the Galafold experience, so that should be a good bedrock to build your commercial strategy around. Any meaningful difference between what you went through in terms of getting the Galafold access in sort of the earlier days of that launch versus what you're seeing with AT-GAA? Mind you, still early days.
Sure. Yeah, I think probably the biggest difference was with Galafold, we felt like we were sort of building the plane as we were flying it. We had hired everybody within a few months of launch. We were brand new to being a commercial company, and we were fairly new to the Fabry treaters. So we were still building relationships. We had to build our hub, which is the patient support services. So very new, and there was that component of amenability that we had to educate the community around, and so that was a bit of a different, you know, different scenario. That being said, of course, the launch went really well. But I think here, you know, we're in a much stronger position. So first of all, significant overlap in treating physicians.
So about a third of the treating physicians are the same between Fabry and Pompe, and I think we're just talking about one in at Penn, of course, who is a geneticist who treats both Fabry and Pompe patients as an example. So that's about a third. About half of the physicians are in the same center, and so we have very experienced sales folks and medical affairs folks who are out there educating these physicians. And so that makes it much more efficient. You know, in the rare disease space, you know, you're probably seeing a physician maybe once a month on a regular basis, and so when you go to a major center, if you can actually call on two potential prescribers, it's a great opportunity, and then the vast majority of cities are the same.
It's like over 80%. So you have this highly leverageable, very experienced sales team. We also have a market access and patient services team who are very experienced. So the hub that we've built to support Fabry, called AMICUS ASSIST , is now supporting these Pompe patients. And so we've done a great job already in getting the patients through the reimbursement process, and we've already had multiple patients who have been approved for reimbursement through the hub services. So that's great. And then the last thing I'll say is that, you know, you don't have the same amenability question. So really any patient who's on treatment, and the vast majority of patients are on treatment, are eligible to, as long as they're adults, they're eligible to come onto the therapy.
I think that it makes it—you know, you're not sort of hunting for a smaller portion of the population. It's really anybody who's being treated today-
Mm-hmm
... is eligible for the drug.
Just to clarify, when you just mentioned a couple of seconds ago, some are already reimbursed. Just to clarify, that's EU, not necessarily in the U.S. as well?
No, U.S. as well.
Okay.
Yeah.
So maybe focus a little bit more on the U.S. side. I think you gave the number, I think, was it like $550,000 for a 40 kg patient, body weight, on the approval call, the price, the direct price?
Yep.
What kind of discussions are you having? Is there a lot of pushback? And in terms of the start form conversion to a paid or I guess, paying patient, what's that duration like? I mean, the general is four to six weeks.
Yep.
Are you seeing that? Are you seeing on the shorter end, given your experience, what's, what's that dynamic like?
Yeah. So a couple questions in there. First of all, on pricing, and one thing I should just clarify as I was sort of thinking about my previous answer, just as a reminder, it's all patients who are not improving on therapy are eligible for the Pombiliti + Opfolda. But, so to your point about market access, so pricing, it's actually about $650,000 is the implied cost for a 70 kg adult. So that's roughly... and we've said that we have a pricing philosophy, which is parity or modest discount.
The reason why we price it that way, and this is gonna get to your second question in terms of going through the insurance process, is because our goal is to maximize the number of patients who have access to the therapy. What we found is that by taking price off the table, by making it either equivalent cost or even modestly cheaper, you just go through that insurance process faster. It doesn't, you know, the classic sort of discounting strategy to drive market share doesn't really work in the rare diseases. This is purely to speed through the process, whether it's the centralized process in Europe or whether it's the payer process here in the United States. So to your last question around how does that process look in the U.S.?
So number one, we've already had patients go through the reimbursement process and have authorization from payers in the U.S., and that's been, you know, a handful of weeks. You're right, kind of steady state Galafold right now, looks like about four weeks typically, to go from prescription to authorization from the payers. And then in the early days, it's usually sort of 60 days- 90 days. And so that's why we've said, you know, our goal is to transition all of the clinical trial patients to commercial drug within 90 days, because that partially accounts for that process. But so far, we've already had approvals, and there is no pushback. As long as it's on label and as long as it's parity or discount, and it doesn't cost them more effectively, there's no pushback from payers.
Great, great. I think it's a great strategy, so congrats on that.
Thank you.
We'll look forward to the four Q-
Exactly.
- JPM-
Yes
... announcement. What's the dialogue with the physicians and patients like? Obviously, physicians who have the clinical trial experience, you don't need any more hand-holding. But I think one of the other sort of nuances of this AT-GAA or Pombiliti + Opfolda is you need to administer the oral drug, as well as-
Yeah
- the infusion within a certain, time window. How has that education been like? What's the early feedback from physicians that have previously not had AT-GAA exposure?
Yeah. You know, it's a few things. So first of all, I'll take this as a chance to say that the most important data in the label, of course, is the six-minute walk and forced vital capacity. We're the only company that has head-to-head data in switch patients, showing that you can take a patient who is presumably declining on enzyme replacement therapy and come to our drug, and you can see an improvement. So that's hugely differentiated data. At the end of the day, it's gonna be the efficacy that really drives the utilization of this drug. That being said, we specifically negotiated for that not improving on ERT language, because that's really what gives you a chance, to your point, about having that conversation with the physician and really driving them to think about...
So this is. You know, remember, we've had a an approved therapy for, you know, over 15 years, right? So physicians never had a choice. They never really had to think about whether the patient was responding well or not, primarily because there was no other choice. And I think for sure, the existing, you know, therapy, as we've seen in the studies, has some benefits, so it's certainly better than no treatment, in our opinion. But now you can ask them, "So is that patient really improving?" And what's fascinating is, we've started to have these conversations and, you know. So they may initially think, "Oh, they're stable, they're fine." But then you ask them, "But are they improving?" And then they look below the surface, of course, there's a six-minute walk and forced vital capacity, right?
But those are measures that take maybe longer to, to, show an impact. But then when they start to sort of peel back the onion and look a little bit more closely, what they see is they might look like they're stable, you know, at a high level, but then they start to ask them, "How are you feeling? How's the fatigue? How's the pain?" Et cetera. And I think it's been a fascinating education process with these physicians to go back and actually interrogate their patients. And I think what they're finding, which is not surprising based on what we see in the data, is that actually it's about 75% of patients are not improving on the drug.
You know, you have this great opportunity now to really change the way that physicians expect from what they're gonna get out of a therapy.
Mm-hmm.
Right? And if you can, if you can demonstrate that you're actually improving those patients, that's kind of the, the holy grail, right?
Mm-hmm.
So that's where it changes from, "Oh, if they're declining, I'll try something new," to, "Boy, you know what? I need to use this in all my patients." And that's the process that we're going through, which I think is fantastic. To your question about the small molecule, honestly, these patients typically are taking concomitant medications, so NSAIDs or antihistamines to control the reactions to the infusions. And so giving them a small molecule an hour before their infusion seems to be just sort of plugged right into their normal standard of care. So that, that really hasn't been an issue. What's important about the small molecule, of course, is it acts as a stabilizer for, for the enzyme replacement therapy. That's another differentiation that we could talk to around the mechanism, and that's starting to really gain traction as well.
That number one, the way that we've designed and selected our enzyme replacement therapy for naturally occurring high amounts of mannose-6-phosphate, and then you have the small molecule stabilizer, is very different from the way that Nexviazyme is designed, which chemically conjugates the mannose-6-phosphate onto the enzyme. And we're hoping to show over time, through some publications, that there are, there are significant differences between that approach, and we think that ours will, will translate to better outcomes in the end.
Got it. You had been pretty aggressive in various scientific congresses, like the World Symposium, to convey you know your message to physicians and KOLs. Now that you have the drug approved, how are you thinking strategically which sites to go after, sort of like the lowest hanging fruit, before going into sort of the broader depths of the patient communities?
Yeah. So in the first 30 days, we've either visited or scheduled visits with all the top KOLs in the centers, in the countries where we've been approved. The U.K. is very concentrated. There's only six centers that treat late-onset Pompe patients. Germany and the U.S. are more similar in the sense that it's kind of the classic. You have maybe, you know, 25 thought leaders in Germany, and maybe it's more like 50 thought leaders in the United States, and they really drive a lot of the treatment decisions, even if the patients are being treated at home through home infusion or whether they're in a, you know, they're going to an infusion clinic. There's still a pretty concentrated number of physicians.
The vast majority of those physicians have already had experience with Pombiliti + Opfolda, so for them, it's a matter of really working with them to transition their clinical trial patients, and that's really important. Our. You know, I think we've really been able to demonstrate that Amicus is a partner in this process. We don't let, patients go on drug holidays when they're transitioning over. So, you know, the sort of rude way to do it is to say: Okay, we're not gonna supply you any more clinical drugs, so if you want them to stay on drug, you better schedule a, you know, commercial infusion. And that's obviously not fair to the patients, and that's not, you know, that's not how Amicus operates.
Our goal is to make sure that everything goes smoothly, that the patients can transition as they need to, that they can do their, their end of study visit appropriately. So that, that's gone really well. There are, however, a handful of physicians out there in that, those top tiers who, who didn't participate in our trial for whatever reason, or maybe they didn't enroll a patient. And so with them, it's very much now, for the first time, you know, it's been such a process to get approved here, but for the first time, you can go and actually educate them. You can have the rep go in and talk to them, and I think that's been a really powerful process for us. And you know, I think...
As we and as the sort of the investor community has gone out there and talked to physicians, like, "What do you think about this drug? What do you think about this drug?" You have to remember, it's, it was really in a vacuum. It was really Sanofi out there educating on their all the benefits that they want to talk about of their medicine, and we were absent. You know, you can't do pre-approval promotion. So now we're finally out in the field. We're able to talk to them directly. We're able to be much more vocal at medical congresses under, you know, under the approval language.
I think what you'll start to see is a much higher level of education of the physician community around what are the differences, and, and you know, what we think, what are the benefits of Pombiliti and Opfolda.
When we think about the treatment switch, whether it's on Lumizyme or Nexviazyme before AT-GAA, and I'm using AT-GAA, Pombiliti + Opfolda-
Fine, yeah.
sort of interchangeably. One's easier to say.
Yeah.
So, I think you're already seeing both Lumizyme users as well as Nexviazyme users kind of roll over into AT-GAA.
Yep.
I know it's early days, but what are you seeing? I mean, clearly, longitudinal study has somewhat well established how the progression of disease looks like-
Right
... on patients with Lumizyme. Nexviazyme, it's still a work in progress.
Yeah.
So what kind of characteristics are you seeing with the Nexviazyme switchers versus Lumizyme switchers? Duration of therapy before switching-
Yeah
... the characteristics, and so forth.
So maybe I'll use that as a chance to sort of talk about the market's, you know, overview in the two different scenarios, in Europe and the U.K. versus the United States. So in the United States, Sanofi launched Nexviazyme over 2 years ago now. And so, you know, what we've heard from a number of physicians is, "Look, I need some time after I switch a patient before I understand kind of what's going on with that new patient or that new product." And we've heard kind of roughly 6 months-12 months is sort of the period of time that they feel like. And that's not surprising because that's really how frequently they see Pompe patients, is once every 6 months-12 months.
So one of the things, one of the questions we got, which is natural, is are, you know, how sticky are these patients gonna be, right? And in the United States, I think the last numbers that Sanofi reported is it's over 50% of patients now are on Nexviazyme, but that means the majority of them have been on for over a year. And so what we had, you know, hypothesized coming into the launch is that we'd get patients switching from both products, and that's exactly what's happened. It's still early days, but it's about 50/50 from each product. And I think that's, you know, maybe natural because that's the distribution of patients, right? And to your question about why are we seeing switchers, I think it's the same, the same.
It's either the physician feels like the patient is not doing well, or the patient feels like they're not doing well. And it's-- what's interesting, and I think, he's not here today, but Mike Caveney, who's the president of our U.S. business, shared this anecdote on, on the call, which is the first patient who-- the first new commercial patient who was prescribed, Pombiliti + Opfolda, her brother was in our clinical study.
Mm-hmm.
She observed how well he did. She was actually on Nexviazyme, and she told the physician, "Now that it's approved, I want to be on the medicine he was on." And so actually, they called us, and that's been a phenomenon, too, that I think has been... you know, we hope to see, but it's been really positive as well, is we're actually getting physicians calling us, saying, "Hey, we want you to come in and talk to us about this now that it's approved." That's very unusual to have that, and I think it reflects the experience that's been happening. So I think it's very much a similar story, which is the physician or the patient feels like they're declining on their medicine. The only difference in Europe is that there are fewer patients who have switched.
They launched later in Europe, both Germany and the U.K.. And so, you know, it's more like, 15%-20% of patients are on Nexviazyme in Europe across all the markets. But there, too, we're still seeing people come off of, off of Nexviazyme and Lumizyme. And I think at the end of the day, our job is to encourage physicians to again, go back and really ask themselves, "Are these patients improving?" If they're not, they should be on Pombiliti + Opfolda. And that, to me, if you could change the expectation for what the outcomes are gonna be on this medicine, I think that's where you're really gonna see us take the lion's share.
One clarification. On the U.S. side, you said it's almost a 50/50 split between Myozyme and Nexviazyme users.
Yeah.
What about in the EU so far? Is that also distribution similar?
Similar, except you also have some naive patients as well.
Right. So I wanted to ask about the naive patients. What's that journey like? 'Cause obviously, you have a broader label in the-
Yep
... in the EU, but do they just kind of see the data and say, "This is obviously the best one, therefore, I'm gonna go on this?" Or did it ever come across their mind that they wanna go through the whole step of, Lumizyme has the longest experience-
Right. Sure
... let's go through that?
Yeah, it's an interesting question. I think, you know, as a reminder, first of all, the vast majority of the opportunity is switch patients. So you know, there are only about 150 new treated patients every year. So that's, it's an important segment, but eventually, kind of colloquially, we say every patient is eventually a switch patient. So you know, in, while we, you know, we think of Europe as a broader label, and I guess technically it is, the reality is the vast majority of the opportunity is in switching patients. And it's an interesting point.
You know, there are some therapeutic classes where, you know, the, you know, you save the best for last, or you kind of step through, and it's, it's sort of a very typical, "I'm gonna try this first, and then I'm gonna go on to this one. That stops working, and then I'm going to this one, that stops working." And I don't think you're gonna see the, that here. I think, first of all, there's no reason to believe that, that starting on a medicine that, you know, we've compared favorably to is better for patients, and in fact, they're going to progress, and that progression, you know, it-- some of that progression is gonna be irreversible. And so I think physicians and patients are gonna wanna go on the therapy that they, they think has the best potential outcomes for patients.
And so I think now, where it's available for naive patients, that's what you're seeing. You're seeing a preferential choice for Pombiliti + Opfolda. Again, it's early days, so we'll have to see how that plays out, but I don't think you're gonna have that kind of classic mentality where I'm gonna start on the old therapy first, and then I'm gonna go on the next therapy, and then I'm gonna end on AT-GAA.
Yeah, seems a little challenging.
Yeah.
Any questions from the audience before we switch to Galafold? Okay, your cornerstone.
Hey, yeah.
Galafold, $100 million, for the first time-
Yeah
... so congrats on that.
Thank you.
Where are you continuing to see the growth here? I know you're talking about geographic expansion on the-
Yeah
... third quarter earnings call. There were some additional geographies you're setting in guidance terms-
Yeah
... if you will. But patient identification, I mean, people have always thought this is kind of a saturated, well-defined market. You're implementing AI tools and-
Yeah
... trying to find more patients that were previously misdiagnosed. Where is the critical growth avenue that you're seeing right now?
Yeah, I think, you know, so to your point, so we still have about 15% of the global geographical, you know, footprint where we're still launching into. So we talked about Taiwan on the call, where we just got approval and reimbursement. We talked about Hong Kong and New Zealand, so there are. But those are smaller markets. Turkey's another one where we have approval. We're working through the reimbursement process. So those are smaller, but still important, so 15%, that's still an opportunity, and we'll do that over time. We're also, we're about 60% market share of treated amenable patients. So that means, you know, you still have about 40% to switch, and we can get to 80% or 90% peak share in the markets where we've been launched the longest. So that's a, that's an opportunity.
But honestly, the biggest opportunity is bringing on either diagnosed, untreated patients, and I'll share some statistics there in a minute, or newly diagnosed patients. So what's remarkable is when we started this journey back in 2016, we had estimated there were about 10,000 diagnosed Fabry patients globally. About 5,000 of them were treated, and about 5,000 were diagnosed and untreated. Today, there are 11,000 treated patients, so the treated population has more than doubled, and the diagnosed population now is about 17,000. So there's 6,000 more patients who are diagnosed untreated, and we're working on those patients. And in our more mature markets, the U.K., Europe, we have far more naive patients coming on therapy than we do switch patients because we've started to saturate that market.
So that's one important segment, is that diagnosed, untreated segment, and those patients, if you think about it, they're late-onset patients, right? Because most of the classic males have already been diagnosed, and late-onset patients have a much higher percentage of amenable mutations because they tend to have missense mutations, which is a slightly, you know, slower progressing form of the disease. So that's a hugely... It's a big opportunity for us to, to, to go after, and I think that's part of why we're the fastest growing, treatment in the, in the Fabry space. The other important point is the diagnosed, or sorry, the undiagnosed population. What's remarkable here is you're still seeing significant numbers of Fabry patients who are trapped in what we call kind of a wrong track, diagnosis.
So they're diagnosed with anything from genetic cardiomyopathy to MS to irritable bowel syndrome, and, you know, they look like something else, and so physicians don't really know how to find them. We're starting to use a whole host of tools, including AI-driven diagnostic algorithms. We've piloted it once in the U.K.. We're now piloting it in the Penn health care system, and the goal, of course, is to develop a diagnostic algorithm that allows physicians to then look through their records and find patients who are highly likely to have Fabry disease.
One interesting anecdote there, in the U.K., one of our key treating centers, who is, you know, it's a woman who's been fantastic, and she did this study of all of the U.K. and found that 80% of the patients treated with Fabry disease in the U.K. came from the top two deciles of socioeconomic status, which is remarkable, which talks a lot about, number one, what we know, which is the, you know, challenges of providing healthcare across the socioeconomic deciles, and the access to healthcare in particular for people who are of low socioeconomic status. But it also tells you there's got to be a significant number of patients in the U.K. who are not diagnosed, right?
Mm-hmm.
And so that, to me, was, like, a great example of two things that you could really make a difference with. Number one, you could provide better access to healthcare across the population in the U.K., but number two, you've got to be missing a big chunk of patients, right? Even in a country which, you know, is so forward and so developed as it relates to treating rare disease. So to me, I think AI and this diagnostic process is gonna continue to play a big role, and what it's gonna mean is this market's gonna continue to grow and grow and grow simply by finding new patients.
Mm-hmm. Speaking of growth, earlier this year, I think you set out the fiscal year guidance as 12%-18% growth, constant exchange rates, and then you repeatedly kinda narrow that guidance to the most recent one, which is now 16%-18%.
Yeah.
When we think about next year, given sort of the initiatives that you have, is this mid to high teens, double digits?... what we should be looking for next year? I know you're probably gonna give us an updated number at JPM, but just give us some sense of how much confidence you have going forward-
Yeah
with the Galafold business.
So, and just as a reminder, it started as 12%-17%, and then it was-
Okay, thank you.
14-18, and now it's 16-18.
Yeah.
So we raised it, the top end of the range, and now we're sort of bringing up the bottom end. So I think first of all, this year has been fantastic growth for Galafold. We've beat expectations every quarter, raised guidance, which is great. So I think, you know, for a product that's been on the market since 2016, just to see this really strong, steady growth, I think is unusual and again, reflective of all the kind of overall dynamics that we talked about. I won't give guidance yet, but I would say that for sure, this is a double-digit growth story for, you know, we think, you know, the foreseeable future. Certainly in the teens, you know, where it comes out, the base is getting bigger. So, you know, you're growing on a growing base.
But give us, you know... We'll wait to give that-
Couple months
... that color for a couple of months. But I would say that again, we are very confident it's gonna continue to grow in the teens for the foreseeable future.
Mm-hmm.
And then you add on Pombiliti + Opfolda, and, you know, which is gonna contribute significantly, not just to the absolute revenue number, but also the growth. And I think this is an exciting story, for years to come.
Forgot to ask, so should we expect an update or even a guidance, if you will, for AT-GAA and at JPM?
We won't. I think it takes about four quarters or so to really get a sense for the launch trajectory. And so we won't give revenue guidance at JPM. We launched it about the same time, actually, with Galafold, and it wasn't until the following year, full year, that we gave revenue guidance. What we will do is, obviously, we'll report what revenue there was. We'll also talk about patient numbers. We'll talk about some of those other metrics in terms of who's switching and you know, qualitatively give a sense for where we're getting those patients. And kind of the key themes that we're hearing.
So I think we'll give people enough sense for how the launch is going and make sure that people have reasonable expectations, but I don't think we'll be ready to guide on revenue yet.
Okay. Cash question is always popping up for these fireside chats.
Yeah.
But given you have the non-GAAP profitability goal by year-end-
Right
... maybe I'll shift it a little bit. What's the trajectory there after, right?
Yeah.
So non-GAAP profitability, how should we think about GAAP profitability and-
Yeah
your continued growth?
Well, I think the first point is we were non-GAAP profitable for 1 quarter this year. So I think the first step there is to be, you know, non-GAAP profitable for the year, let's say. But we're very close to having, you know, GAAP profitability and then importantly, free cash flow. And our goal right now actually is that we do not need to access the equity capital markets to fund our operations. We've talked a little bit about maybe business development and long term and, you know, never say never. But from an... I think that's really what investors want to see us. They want to see us be independent from an operational perspective, and I think that's, you know, that's where we are.
That was part of the financing too, that we did with Blackstone, that combination of refinancing the Hayfin debt and that little bit of equity that came in. I think that was really an important way of just pushing out those principal payments-
Mm-hmm
... and making the debt a little bit cheaper as well.
Mm-hmm. Okay. Well, with that, Brad, thanks very much.
Great. Thanks, Dae Gon Ha.