Bradley Campbell: One place that you might look, Sarah, as we did, we have talked about this before, there was an independent poster that was published at the World Congress that talked about individual, of course, case studies, but it was the center itself reporting on individual patients’ response to therapy and how the switch process went. And I’ll say that’s a piece that, is never going to show up in a data. It’s not going to be something that a physician kind of promotes on, of course, but we’ve heard over and over again that the experience team we have, both the sales reps themselves, the MSLs and medical affairs team in the United States, our patient hub, which helps people work through the reimbursement process. I think we’ve really established ourselves as trusted and credible partners.
And that really makes a difference too, right? I mean, these are life-threatening diseases. These are big decisions for people to make. And I think Amicus has established ourselves as really trusted partners in this field. And I think that goes a long way towards physicians and patients wanting to take a different path and trying to pump the lead out and pull the hook.
Operator: Thank you. Our next question comes from the line of Kristen Kluska of Cantor Fitzgerald. Please go ahead, Kristen.
Kristen Kluska: Hi. Good morning, everybody. You’ve obviously reported on a number of endpoints, but I’m curious in a real-world setting, what are the biggest drivers and what feedback they’re looking to get from patients to determine whether or not their current therapies are suitable for them?
Bradley Campbell: Yeah, it’s a great question, Kristen. Thank you. And you’re right. So clearly the primary registration endpoint was a six-minute walk and four-slide capacity. And those are still very important from a physician perspective. But Jeff, maybe talk about the importance of those other endpoints and how patients might be feeling when they transition and how important that can be as well.
Jeff Castelli: Yeah. Thanks, Kristen, for the question. So as Brad said, there’s a number of different measures that physicians and patients use to sort of assess how they’re doing. Some of those do include when they come in every six months, they do get their sort of battery of more standard assessments. Many of those are similar to the trial assessments, like a six-minute walk or an FBC. But it is, there’s definitely variability across physicians on sort of what measures they use. A big part of it is also just sort of their questions to the patient about how are you doing in your daily life on doing different tasks, walking up stairs, are you having falls, etc. And then also, some of the physicians will look at bio-marks. So it’s really that totality of sort of assessments of how patients are doing.
You know, there could be opportunities within those six months, more rigorous kind of assessments to also have check-ins with their physicians. But it’s really sort of that combination of parameters that are looked at and importantly, as we look at the U.S. and sort of the not improving aspect of the label. That is, not specific to any end point or parameter. It’s more sort of left to the judgment of the physician, the patient, to sort of holistically assess if that patient is actually not improving or not.
Operator: Thank you. Please stand by for our next question. Our next question comes from the line of Dae Gon Ha of Stifel.
Dae Gon Ha: Hey, good morning, guys. Thanks for taking my question. One question on AT-GAA, Pombiliti and Opfolda. The remark you made about the naive patient experience in Europe, I’m pretty curious, what are you seeing there? What was the sort of cadence of events that led to that naive patient coming on therapy? And obviously, how do you see this kind of panning out going forward? Is this going to be a spread of the word and kind of ballooning from there to go after the naive segment? And then second for Jeff, as you think about the next gen, obviously, the non-gap profitability is well intact. But how are you thinking about the next gen molecules? We’ve seen some disappointments on the gene therapy front for Fabry and Pompei, and obviously, next gen chaperone. Just kind of curious how you’re thinking about sort of the patient segment you’re going to go after. Thanks so much.
Bradley Campbell: Thanks, Dae Gon Ha, for the questions. Yeah, so the naive segment in Europe, of course, it’s early days, but we are pleased to already be seeing newly diagnosed patients or diagnosed untreated patients come on to therapy. That’s a really important segment. Pompei is not quite as under-diagnosed as Fabry disease, but if you look at all the literature, as we’ve done newborn screening, as we’ve done high risk population screening, and as other manufacturers have focused on those segments as well, you’re still seeing a healthy diagnosis rate. And so we continue to expect there to be newly diagnosed patients fueling the market for many years to come. And of course, just as we’ve done in Fabry, we’ll invest in those as well.
And I think, look, if you look at the literature, if you look at some of the work that Dr. Kishnanti and others are starting to do, it is clear that number one, patients are slipping through the cracks and are being diagnosed later in life, well after they’ve exhibited symptoms that should warrant a treatment. But we’re also looking much more closely at patients who maybe historically would have been seen as quote unquote stable, who are newly diagnosed. And if you look closely, this is a progressive genetic disease. And I think there’s a growing body of evidence and support from physicians to treat those patients earlier. So I think that will continue to happen throughout the Pompeii community. Jeff, do you want to talk a little bit about kind of how we’re thinking about the chaperone?
And I think that the top-line that I’ll provide is that we’ll talk more about our pipeline as we go into next year. As you said, Dagon, it is a judicious investment at this point. We’re focused on profitability and using a little bit of resources to keep those programs going. But the main focus is Galafold and Pompilia-Folda. But do you want to talk a little bit about how we’re thinking about the chaperone program? Jeff?
Jeff Castelli: Yeah, thanks, Brad. And I’ll start, Dae Gon Ha, with the gene therapy programs briefly. So there we have seen some challenges in the field for some of the gene therapy programs, continued challenges around durability and some of the safety. And we know that manufacturing is a challenge in terms of costs. And regulators are wanting to see in some cases that the gene therapy is bringing some aspect of superiority to sort of justify some of the inherent risks of gene therapy. So I think you’re going to see people focusing more on sort of the patient segments that are not doing well in standard of care, where you might be able to best show some aspects of kind of benefit over approved therapies. Given that and our judicious investment in the pipeline, we think our transgene is the best in class, more potent and well-type.
But we’re really looking at trying to solve some of the challenges around delivery and safety so that when we are able to more rapidly bring those forward, what we’re bringing forward is something that really could deliver on that benefit over existing treatments. And in terms of the next-gen chaperone, really they’re still early days, but we’re trying to see if we can expand sort of the number of amenable mutations, as well as potentially improve upon, Galifold in terms of efficacy. It’s at a high bar, so, challenging to kind of improve upon the response of amenable mutations, but we think that would be great down the line, in the future, when potentially Galifold may go generic to have something that could be, incrementally better. Importantly, to expand amenable mutations.
You know, we know it’s clear that there’s a preference for oral, administration of therapies where possible. So, if we could expand that list of amenable mutations, we think that would be a big impact.
Operator: Thank you. Our next question comes from the line of Gil Blum of Needham & Company.
Gil Blum: Hey, good morning, everyone. And let me add my congrats to the progress. I’ve been kind of looking more forward here to 2024, and, currently, the majority of revenues are being recognized outside of the US. You think this dynamic may change now that you’ve, launched in Pompeii? Thank you.
