All righty, let's, let's get started. Welcome everyone, to the 42nd annual J.P. Morgan Healthcare Conference. My name's Anupam Rama. I'm one of the senior biotech analysts here at J.P. Morgan. I'm joined by my squad, Priyanka Grover, Lora Hall, and Malcolm Kuno. Our next presenting company is Sarepta, and presenting on behalf of the company, we have CEO Doug Ingram. Doug?
Thank you. Thank you, Anupam, and thank you all, both in the room and on the Internet, for joining us today. Appreciate that. We will be making some forward-looking statements, so please look to our public filings for the various risks and uncertainties that come whenever one makes predictions about the future. There's a lot one can say about Sarepta. We set for ourselves very big goals, always with the patient as our North Star. We lead from our perspective. We challenge convention when necessary, we execute. What I'd like to do in the short time we have together today is three things: First, I'm gonna talk a bit about the progress we've made over the last seven years to get to the beginning of 2024. And then I wanna talk about all of the milestones that are happening in 2024.
Then, if we are successful in 2024, I do wanna touch on where we might go from there. But to do that, we have to start back in 2017 and talk a bit about the strategy that we created in 2017, and it was an ambitious one. Our goal in 2017 was to be one of the leaders in the use of genetic medicine to treat rare disease, and in addition to our expertise in RNA, to become one of the world's leaders in the use of gene therapy, to bring a better life to patients. To understand how brazen, in a sense, that ambition was, you need to understand where we were as we entered 2017.
To remind you, when we entered 2017, we had one approved therapy, and we had about $5 million in sales as we entered 2017. We had a modest amount of cash on our balance sheet and a fairly short runway. We had a very modest pipeline. We had about 200 employees in the company, and while there was no doubt, this small cadre of mission-driven zealots at Sarepta, we had enormous gaps in expertise that we would need to figure out to make our vision a reality. Then finally, with respect to gene therapy, there were enormous aspects of gene therapy we knew next to nothing about. But we had some things going for us.
In addition to our science, the things that made us confident that we were gonna be successful, first and foremost, is the fact, and it's real for us, that we are a very mission-driven company, that we have a motivating mission to help patients that drives us and gets us out of bed every day. Second, we had a well-articulated strategy from 2017 that would guide us along the way. We had the will and the ability to hire like-minded professionals who would focus both on great science and on patients, and we had this very unique Sarepta culture. It is a culture where, when necessary, challenges tradition, where necessary, challenges convention, and gets things done. And to remind you, we got a lot done over the last seven years.
As we track into 2024, I will remind you that we have four approved therapies today. From that $5 million as we entered in 2017, we have grown our revenue over the last seven years at a compounded annual growth rate of about 115%. We ended 2023 with well over $1 billion in revenue, and we'll talk about that. We have a deep pipeline. We are, on a non-GAAP basis, profitable today, and we will be cash flow positive very soon. I'm very proud to say we have 1,500 or so of what I believe to be some of the most passionate, dedicated, expert professionals in all of biotech, about whom and with whom I am extraordinarily proud to work.
And then, most importantly, we've done a lot of good for a lot of patients over the last seven years, and we have an opportunity to do a lot more in the future. If we talk about patients, we have to start with what is core to our mission right now, which is bringing a better life to patients with Duchenne Muscular Dystrophy. So I'll apologies for those folks who know Duchenne well. Let me talk about it very briefly. Duchenne Muscular Dystrophy is an X-linked recessive disorder that results in children with Duchenne missing this shock-absorbing protein in their muscles called dystrophin. And while the course of this disease might vary, the results never vary. As a result of missing this shock absorber in their muscles, every time these boys move, they're damaged, and damaged in an irreparable way. That means they're gonna end up in a wheelchair.
It means they're gonna struggle to move their upper limbs. It means they're then gonna struggle even to breathe, and then they are gonna die, and they're gonna die young. In fact, over the great majority of history, families had little reason for any hope. A common refrain when a physician diagnosed a family was to tell their parents to go home and love your child, because over the next 15-20 years, this disease is going to steal your child from you, and there's nothing we can do about it. Today, the world's very different. We have an enormous amount of potential hope today. The dialogue that can occur between physicians and patients is very different today, and I'm really proud of the role that Sarepta has played in changing that dynamic and that future for kids.
So talking about that, in 2023, you'll know all four of our therapies are dedicated to bringing a better life to patients with Duchenne muscular dystrophy. Our team did, in my view, a brilliant job of serving this community, and if we boil that service down to numbers, as we must, here are the numbers. In the fourth quarter of 2023 for our PMOs, EXONDYS, VYONDYS, and AMONDYS, we did $234 million, which means our PMO revenue last year stood at $945 million against the guidance of $925 million for the year.
You will also know that in June of last year, we launched the first gene therapy for Duchenne muscular dystrophy, ELEVIDYS, and in the fourth quarter, we did $131 million, which brings our full year number from June till the end of the year at over $200 million, which far exceeds analyst consensus. So if you pull all of that together from the $5 million we had as we entered 2017, we did almost $1.15 billion in revenue across our four therapies last year, which represented about 30%-36% growth over the prior year. As exciting as 2023 will be, and I'll talk a bit about this in a moment, 2024 offers the opportunity to be even more exciting.
Now, with that said, I will tell you, we can't provide you with guidance yet on 2024, and the simple reason for that is both the ELEVIDYS revenue, as well as the PMO revenue, will be informed by the breadth of the eventual ELEVIDYS label and the timing of that label. But nevertheless, it is quite clear that 2024 is an enormously important year for us, and we're really excited about it. As you may recall, last year, in May of last year, we won an advisory committee on ELEVIDYS, and then in June of last year, we launched. We got approval in June, and we launched ELEVIDYS to treat Duchenne boys who were four and five years old. When we got that approval, we had two big goals.
The first goal was to do a brilliant job serving this community and launching this first gene therapy for Duchenne muscular dystrophy. I would hope you'll agree with me that given the numbers that I just showed you, we are in the process of doing exactly that. The second goal we had was to move as rapidly as possible to remove the age and ambulation restrictions that currently exist in that label so that more boys and young men can benefit from ELEVIDYS. If we're successful in 2024, and we'll talk about this, we have an opportunity to get to that second goal, not in years, but in months this year, if we're successful. The reason for that is in the fall of last year, our confirmatory trial, EMBARK, read out.
Shortly thereafter, in December of last year, we submitted our BLA supplement, asking the agency to remove what we see as unnecessary age and ambulation restrictions from the ELEVIDYS label. So what I'm going to do right now is briefly discuss the top line for EMBARK. I do this a little cautiously because I'm gonna do a far less expert job of this than our head of R&D, Louise Rodino-Klapac, but you'll forgive me for that. The first thing to recall, of course, is that we didn't hit statistical significance on our primary endpoint, which is NSAA or North Star Ambulatory Assessment. But what is also quite clear is that that did not result from the performance of ELEVIDYS, which did exactly what we had hoped it would do, which is arresting decline and stabilizing the muscle in boys that have Duchenne muscular dystrophy and stopping the decline.
It had everything to do with the fact that the tool was not sufficiently sensitive to detect the decline in the untreated patients, particularly in the six to seven year-olds. Now, you might say to yourself... In fact, let's be very clear. What NSAA would have suggested is in those six to seven year-old boys, they were doing just fine. They weren't declining at all. But that isn't true. That's an artifact of the tool. These kids were beginning what ultimately becomes the ferocious decline associated with Duchenne Muscular Dystrophy. And for those who might think that I'm speculating or guessing at that, the good news is that we're not. All of our key secondaries, which were much more sensitive, time-tested, showed exactly what was going on, which is ELEVIDYS was stabilizing the boys on therapy, but the kids that were not on therapy began ferociously declining.
In fact, with respect to time to rise, if you were on ELEVIDYS, it reduced the odds of early loss of ambulation in those children by about 90%. So let's look at the entire forest plot. So what we're looking at right here to orient you is a forest plot of our primary endpoint, are all of our key secondary endpoints, and all of our timed secondaries. That vertical line that you can see in the left, everything to the right of that favors therapy, meaning the therapy's working and helping kids. If there were measures that were to the left, that would suggest the therapy may not be working. And as you can see with your own eyes, the therapy is clearly working. Everything is obviously significantly to the right of that line. In fact, on our key secondaries, we strongly hit statistical significance.
On forced decline, which is the other timed measure, strongly associated with early loss of ambulation, we strongly hit statistical significance. We hit statistical significance on the wearable, something called SVC95C. So if you look at this with your own eyes, you can quite clearly see in this forest plot that ELEVIDYS is doing an enormous amount of good. Now, the good news as well is we can do the math on what you're seeing clearly with your eyes, because we had a pre-specified global statistical analysis, and that does exactly what you're doing. It looks at all of these measures together, it corrects for any multiplicities, and asks whether the therapy is benefiting kids or not. And the p-value on that global statistical analysis was literally 0.0044.
Beyond that, you'll recall last year, one of the alleged open questions was whether, consistent with its mechanism of action, ELEVIDYS would benefit muscle in children across ages, or whether in some way that we can't fully explain by science, the protein would stop working when a kid blew out the candles on his sixth birthday. Well, here's the answer to that. These are all the pre-specified age-related results across all of the measures, and you can see the therapy's working equally well across ages, just as one would expect. From our perspective, the summary conclusion is pretty straightforward. First thing is, ELEVIDYS is arresting decline in boys with Duchenne muscular dystrophy and stabilizing their muscle. I do want to remind you, that is the most ambitious treatment goal for a therapy like this.
It is working equally well across ages as its mechanism of action would have suggested. Its safety and tolerability has been stable and laudable across many studies, across ages, and across ambulatory status. So what we've asked for then is that the FDA remove the age and ambulation restrictions associated with ELEVIDYS. And to remind you, what we're asking for is not unprecedented. In fact, every single other therapy ever approved in history for Duchenne Muscular Dystrophy doesn't have these sorts of limitations, so that's what we're asking the FDA for. Now, as I said before, and as many of you probably already know, we submitted our BLA supplement in December 2021 of last year. There are two things that we've asked for.
The first, as you know, is we asked to remove the age and ambulation restrictions from the label. Then second of all, in light of the fact that EMBARK, which is our confirmatory trial, is now complete, and from our perspective, has confirmed the benefits of ELEVIDYS, we've asked the agency to transform this approval from an Accelerated Approval to a traditional approval. We, we submitted on December 21. Let's talk a bit about timing. So, pursuant to the regulations, the FDA has 2 months to review that file before it accepts it. And then, assuming we got an expedited review, that would be a six-month review, which means that our PDUFA date could be out as far as August of this year. However, with that said, let me remind you of two things.
The first thing is that while this BLA supplement is monumentally important to families living with Duchenne, it is relatively straightforward. It is certainly more straightforward in many ways than our original BLA, the majority of which dealt with very technical CMC and manufacturing issues. And of course, the second thing we have to all recall and remember is that families with Duchenne Muscular Dystrophy are waiting, and they are, on a daily basis, being harmed as they wait. So we are hopeful that we can do this much sooner than the official regulatory timelines. Now, if we're successful with ELEVIDYS and its expansion this year, it's gonna be a monumentally important moment, certainly for the Duchenne community and even for Sarepta and for our investors, and in my opinion, for the entire field of gene therapy.
But we still have other things to do in 2024, so I'll briefly talk about some of the other things we're doing this year. The first thing, of course, is we have four approved therapies. We're gonna continue to serve the community with all four of our therapies, and we're, you know, in the process already of doing that. We have a really important readout on our next-generation chronic therapy, the PPMO, SRP-5051. That'll read out this quarter. We'll release that information to everybody, and then we'll talk to the agency thereafter. We're advancing the entire field of gene therapy this year through suspension manufacturing, which we're aggressively working on, through this novel new capsid, about which we have an enormous amount of excitement, and about multiple lines of inquiry to knock down pre-existing neutralizing antibodies and allow gene therapy- more gene therapy patients to benefit.
Beyond that, as you know, we have a very deep pipeline. We have about 40-plus programs in our pipeline. Time doesn't permit me to go into detail on all of it. There's a lot we're doing this year, but I'm gonna touch on one program in particular. It's within our Limb-Girdle portfolio. Limb-Girdle muscular dystrophy is a collection of muscular dystrophies that are, like Duchenne, degenerative and often result in life-limiting or life-ending disease in patients. Our lead program is SRP-9003, and it treats Limb-Girdle type 2E, and that's an ultra-rare disease. I'm happy to tell you that our pivotal trial for 9003 has already been initiated. In fact, we'll be screening patients for our pivotal trial this year, and that's extraordinarily important.
It is extraordinarily important for families living with limb-girdle type 2E, and it's important for limb-girdle more generally, but in my view, it's far more important even than that. With respect to ultra-rare diseases, it's Sarepta's perspective that we have to find viable ways to build programs that can serve ultra-rare disease. It is not acceptable from our perspective that science can stand ready to benefit patients, but that we can't find a way to get there administratively or viably. And I think the approach we're taking here could be a pathfinder for that. So what is our pivotal trial? First, we're not using a placebo-controlled trial. We're using external controls. Second of all, we're properly sizing this trial in light of the ultra-rare nature of this disease.
We're using lack of the native protein as the primary endpoint in the trial, and then we've got multiple inquiries into the impact of SRP-9003 on the pathophysiology of Limb-girdle type 2E that will form the confirmatory evidence for this. If we're successful here, it'll be enormously important for 2E patients and for Limb-girdle, but I think it could be a blueprint for the way we approach improving the lives of patients with ultra-rare disease, with sophisticated therapies like gene therapy and cell therapy and the like. All of which is to say, if we're successful, 2024 is going to be an extraordinarily monumental year for all of us. In fact, if you go back to that vision we created back in 2017, we will have largely fulfilled the vision that we set out to fulfill back in 2017.
Which will ask us, you know, obviously, an important question, which is: What do we do next? What happens if we're successful in 2024? One possibility is we're going to have years of very robust growth. We could take a victory lap, right? We could rest on our laurels, but we're not gonna do that. We have to come up with a grander vision for ourselves. And the reason we believe we have to do that is that, from our perspective, with little more than great science and a lot of grit, Sarepta has built an organization and team that knows how to serve patients and do great science. And I think we owe it to the community and, frankly, to society more generally, to make the most of this. So this is our grand next goal.
Our grand next goal, if we are successful in 2024, is by 2030, through a combination of our internal pipeline, but also through external moves and business development and the like, and bringing other technologies on, to become the next global big biotech, measured not in incremental growth, but in multiples of growth, and measured not in incremental benefit to multiples of benefit. If you looked at that, you might say that seems excessively ambitious. I would argue, however, given all of the tools available to us today, that it is no more ambitious than the ambition we had in 2017 to get where we can get in 2024. In fact, there's a real argument to be had that the only thing that stands in the way of our ability to realize this vision is lack of imagination.
And a lot has been said about Sarepta over the years. I don't think anyone has ever said that we lack imagination. So we have a lot to do, a lot to execute in 2024, a lot to imagine for 2030. Let's end with the words of Buddy Cassidy, a leader in Duchenne. If you don't know who Buddy Cassidy is, Google search him, because you should know who he is. He's a tremendous human being. And what he said is: "There is more to do, but now let's pause and bask in the glow of our achievement." We'll pause for a second, and then we got to get to work, because we have a ton to do in 2024, and I'm really looking forward to updating you along the way. Thank you.
Thank you, Doug. I just want to remind the audience, there's three ways to ask a question, right? So old school, raise your hand, I'll call on you. Two, there's an iPad where you can submit a question portal.
Hey, guys, how are you? There's some pressure there.
Yeah. Oh, you can email me. You can email me. All right, so we actually do have a couple questions in the question portal. I guess the first is: Can you provide some color on how timelines for the efficacy supplement that you filed compares to the PMR that you filed, and should we hear about both filings at the same time, and do they go to the same group at the FDA?
Yeah. So the short answer is that you should hear about them at the same time. We've submitted a BLA supplement that, that had both issues in it. So just to remind you, and I said this in the presentation, we have both, a request to change the label to remove the, the limitations associated with age and ambulation, and we also have a request to transform accelerated approval to traditional approval. And so the goal is to have these things decided together.
Okay. And then, when are you expecting the full data presentation and/or a publication of EMBARK?
We'll be publishing this in, sort of, in the future.
Okay. And then,
We're working on a publication as we speak.
Yep. And, are you expecting priority review and an ad com for the update, the regulatory review for ELEVIDYS?
You asked me to... I got a bet. I got a bet with some folks on this issue. On priority review, yeah, we are expecting expedited review and, as to the ad com, no.
Questions from the audience.
By the way, for those who wonder, we had a bet whether I could answer just no. No, I think I lost the bet.
Questions from the audience. Yes?
I guess, despite what happens with the label, and the FDA, it seems like docs want it, patients want it.
Mm-hmm.
Do payers want to pay for this with, you know, the kind of, question of efficacy with this quote, "significance in EMBARK?" Like, is there any payer friction here?
First, I'm gonna ignore that you said that questionable efficacy. With that said, I'm gonna turn it to Dallin. You can comment on this.
Yeah, we're... Thanks for the question. We're generally pleased with the reaction of the payers, most of the payers, and many of the big plans have already issued policies covering to label. The payers have come a long way in the last seven years understanding this disease, and, and, and they, they do understand the treatment goals. They, they understand this data. We're working very closely with them, and, so it has been, a nd, and as you saw from the results today, the team has done an incredible job engaging with the payers. And as you said,
. yourself, the Kay Wells really have really understand the disease, and they themselves have helped the payers understand it and are working closely.
I think a lot of payers deserve, you know, a lot of credit for what's going on. Some of them, the larger payers, and most interestingly, issued policies to cover to label fairly rapidly. And we're in an interesting place right now because we have this very age-restricted label right now. You could envision a world in which, a bottom line-oriented payer might try to find ways to game it, so a kid ends up over six years old and doesn't get therapy. And I'm really pleased to say we have not seen that right now. Payers are actually doing the right thing. So, it's... And I give a ton of credit to the team.
They're doing a brilliant job working with payers and getting to understand the data and the need for this therapy, and I give a ton of credit for payers for doing the right thing.
Questions from the audience? Another question in the portal: Are you planning to change your production process for ELEVIDYS from CDMO and doing something in-house? And how long will that process take, and will it require an FDA bridging study?
Well, there's a bunch there. So generally speaking, we use this hybrid model, where we own a lot of the expertise around the manufacture of our products, process development, analytical development, assays, that stuff. And then we work with partners. In the case of ELEVIDYS right now, we work with Catalent. We've been very pleased with our partnership with Catalent. We always think about whether we would do some part of manufacturing in-house, but it won't take the place of this hybrid model more generally. The real interesting issue from a manufacturing perspective, longer term, is moving to suspension, and we're working on that right now. In fact, we're in engineering runs at 500 liters right now for suspension.
We wanna move as fast as possible in suspension, really to support a lot of the ex-U.S. growth that can come in the sort of the next waves of ex-U.S.
Questions from the audience? Maybe a clarification, Doug, on timelines. Question in the portal. You noted that the PDUFA in the scenario that you posed, would be in August?
Mm-hmm.
Does that assume a priority review? I think there's a view that maybe the PDUFA should have been in June.
Well, remember, we have to—there's two—they have two months to accept this, so it really depends on when they accept the, the submission. So we submit, then they accept it, then it's filed, and then you get the six-month review.
Got it. Questions from the audience?
But I do wanna be clear, you know, those are the regulatory timelines. You know, we are very hopeful that we can move more expeditiously than that.
Questions from the audience? Yep.
I'm curious on your thoughts on the next steps for the PPMO after the disclosure readout, optimism or general thoughts regarding that?
Well, let me say we're, you know, we're very excited about the concept of the PPMO. The PPMO, conceptually, just so people know, so we have this chronic therapy, the PMOs. The limitation on the PMOs, as great as they are, is that they're not wildly bioavailable. They don't get into the cells brilliantly, and what we've used with the PPMO is a proprietary peptide to draw far more in animal models. And early readouts of our studies have suggested that we'll get a lot more PMO at the right place, and then more exon skipping, and then more dystrophin. So we're, you know, very excited about that. In addition to that, it'd be monthly dosing as opposed to weekly dosing. So the next steps are really straightforward.
We need to get the data, collate the data, look at the data, to release the data. And then the very next thing we do, and it'll take some months, is we need to meet with the FDA. So I, you know... Again, as you know, I think with respect to our PMOs, there's a very well-understood pathway for accelerated approval based on dystrophin production. We need to meet with the agency, show them the data, and confirm their view that that pathway applies for the PPMO as well, and that's what we'll do later this year.
Yeah, maybe just one thing to add to that, though, is obviously the data that we've seen from ELEVIDYS has tremendous efficacy. And so the landscape has to evolve with that. And so, while PPMO, if we're able to achieve 5%-10% dystrophin, is certainly very significant from a scientific perspective, would be a big breakthrough. We also have to think about the landscape in terms of access and reimbursement. And I think what we've seen from the SMA market is you have not seen a ton of combination use, and therefore, you know, we're gonna have to see. Now, obviously, we're one company who could potentially have both products, but so far, we haven't seen a ton of combination use between the two, and so we have to take that into consideration.
Obviously, because ELEVIDYS has hit a very, very high bar from an efficacy perspective, I think it is more challenging for other approaches that don't produce as much dystrophin.
Questions from the audience? Yeah, over here. All the way over.
I had a quick question in regards to a potential expansion of the label. If the label is expanded, are there enough goods and products to continue to meet the initial, increase in demand?
We're planning for that, yes. Yes. We've been, we're planning for that, and frankly, we've been planning for that since 2018. So we're, we're ready to serve the community with an expanded label.
Over here. Oh, here. One second. Let's get you a mic here.
... Okay, so what do you think of the competition situation with the CRISPR gene editing doing the exon skipping for DMD? So just like Vertex or some other gene editing company.
Yeah, I mean, that's interesting. So just for everyone's – the question was particularly relating to CRISPR-Cas9 and gene editing. We think it's a really interesting concept. We actually have a program. We have a Gene Editing Innovation Center in Durham, North Carolina. We don't think we're there yet, and we don't think anyone else is there yet, and I think Vertex actually is retooling as well. So is there an opportunity in Duchenne Muscular Dystrophy for gene editing? Yeah, possibly. Would it be better than gene therapy today? I don't think that we know the answer to that, and I don't think there's an immense thesis that would say it could be, but it's an interesting technology. It's just not mature yet, from our perspective.
Questions, other questions from the audience? We've got another one in the portal, which is: When thinking about the ongoing review of ELEVIDYS, is it the same group of reviewers that is reviewing this time around, basically?
Yes.
Yeah. Okay.
Maybe, maybe just one quick follow-up to that. You know, there is a big distinct change, though, in terms of the leadership at OTAT, with Nicole Verdun now taking the helm, and we've certainly seen her speak and echo the words of Peter Marks when she's giving presentations, and so we feel confident that he has more support to effectuate and realize his vision.
Yeah, for sure.
All right, maybe the first question from me. So, we've seen a couple headlines over the last couple of days related to REGENXBIO and your ongoing IP situation there.
Yeah.
Maybe you could comment on that.
Yeah, I'm going to comment very briefly on it. Just for those who may not know, REGENXBIO had filed a lawsuit against Sarepta, claiming that a particular patent they had covered our product. As we had said repeatedly from the beginning, we were confident that we did not infringe any valid and extant patents for REGENXBIO or anyone else, and the court agreed with us a couple of days ago and granted summary judgment, dismissing that case. So we're all I'll say to that is, you know, we're very pleased with the result, and we think it is a win for innovation.
Questions from the audience?
Doug, what do you believe are the most rational arguments for label expansion in four to seven year-olds, ambulatory patients, or a non-restrictive label?
Sure, and you know I'm going to end up with non-restrictive label, right?
Yes.
That's, that's our goal. So let's go through the theoretical choices. Someone, some might say, "Well, if you're going to expand it, why don't you just expand it to the studied group, four to seven year-olds?" Let me start there. I don't think that would be a rational answer for a host of reasons. One, that just simply isn't the way development goes. You're not required to study fives and fives and fives and sixs and sevens and sevens and sevens and eights, and then we'd have to study nines and 10s and 11s to 12s. And, you know, our grandchildren will pick this up, and they'll be doing these studies for us. That isn't the way development works. We had a confirmatory trial.
The confirmatory trial was EMBARK. The goal of EMBARK was to answer the question about whether this therapy would benefit children and whether the signals supported the conclusion that it worked equally well across ages. And so I think practically stopping at four to sevens wouldn't be consistent with the approach, with the confirmatory trial or good science. Then we go to the next level is the ambulatory, non-ambulatory, right? So, you know, what about stopping at the ambulatory and not giving us an approval for the non-ambulatory? I would argue that from a mechanism of action perspective, there is simply no good reason for that. And that's because this is a... You know, what this therapy does is quite straightforward.
I mean, it's complicated, but you can describe it in a straightforward manner, which is it is a shock absorber that protects your muscles when you move. So as long as you have muscle, it should be protective and benefiting these kids. The mere fact that a kid would be in a wheelchair shouldn't stop that therapy from working. In fact, kind of seems like a particularly cruel answer if a kid can't get out of a wheelchair to not let him get this therapy. Now, there's another argument to be had, if I can be more nuanced. So I don't think that stopping at ambulatory would be consistent with the science. That's certainly our perspective. Then there's another question, which is, okay, what about traditional versus accelerated, right?
Now, I can argue that logically, we should get a traditional approval on everyone. There is a way to give us an Accelerated Approval for non-ambulatory and use our ongoing study called ENVISION as the confirmatory trial. So I think that's where maybe the debate could lie with the agency. Now, do we get approval for the non-ambulatory, yes or no? And then the real question is, do you get traditional approval or Accelerated Approval? And that wouldn't surprise me that that could be a discussion topic with the FDA.
Any final questions from the audience? All right. Doug and team, thank you so much.
Thank you very much. Thanks for having us.