Good afternoon and thank you for standing by. Welcome to the Sarepta Therapeutics Second Quarter 2024 Financial Results Conference Call. At this time, all participants are in a listen-only mode. After the speakers' presentation, there will be a question-and-answer session. [Operator Instructions] Please be advised that today's conference is being recorded. I would now like to turn the conference over to your speaker for today, Mary Jenkins, Associate Director, Investor Relations and Corporate Communications. Please go ahead.

Thank you, Lisa, and thank you all for joining today's call. Earlier this afternoon, we released our financial results for the second quarter 2024. The press release is available on our website at sarepta.com, and our 10-Q was filed with the Securities and Exchange Commission this afternoon. Joining us on the call today are Doug Ingram; Ian Estepan; Dallan Murray; and Dr. Louise Rodino-Klapac. After our formal remarks, we'll open the call for Q&A. I'd like to note that, during this call, we will be making a number of forward-looking statements. Please take a moment to review our slide on the webcast, which contains our forward-looking statements. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Actual results could materially differ from these forward-looking statements, and any such risks can materially and adversely affect the business, the results of operations, and trading prices for Sarepta's common stock. For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent annual quarterly report on Form 10-Q filed with the SEC, as well as the company's other SEC filings. The company does not undertake any obligation to publicly update its forward-looking statement, including any financial projections provided today based on subsequent events or circumstances. And now I'll turn the call over to our President and CEO, Doug Ingram, who will provide an overview of our recent progress. Doug?

Thank you, Mary. Good afternoon, everyone, and thank you for joining Sarepta Therapeutics second quarter 2024 financial results conference call. I joined Sarepta a little over seven years ago for one reason. I believe that Sarepta had the greatest hope of developing medicines that could improve the lives of a majority of Duchenne patients, and I wanted to be part of that effort. We've had many accomplishments over the last seven years, successfully launching four life-changing therapies and through an obsessive attention to detail, making all of those launches very successful. We have grown revenue at about 150% CAGR from the beginning of 2017 through the end of 2023. We've built a deep pipeline. We've established ourselves as the leaders in RNA and gene therapy for rare disease. And we have become profitable. And as of this of this quarter, we are cash flow positive. But it was only in the second quarter year that we that we can finally say have reached that objective that brought me and so many of compatriots to Sarepta, because it was in June of 2024 that we finally obtained a broad approval to make ELEVIDYS available to over 80% of patients living with and dying from Duchenne muscular dystrophy in the United States. As you are aware, in June of this year, the FDA granted traditional approval for all ambulant patients for and older and consistent with every other Duchenne approval in history, also granted approval for the treatment of non-ambulatory patients on an accelerated basis. Also in June, our partner, Roche, announced that the European Medicines Agency had accepted the ELEVIDYS submission for review. Roche also has obtained six ex US approvals so far and is making good progress serving those communities. So here we are, launching the largest gene therapy yet…

Thank you, Doug, and good afternoon. As Doug outlined, we generated roughly $361 million in net product revenues from our Duchenne franchise in the second quarter of 2024. Q2 is the last quarter in which we will have executed on the limited four to five year old label for ELEVIDYS, given that we received a broad label on June 20. As we had expected, the net product revenue for ELEVIDYS was essentially flat when compared to each of the two prior quarters. Of note, a few patients rescheduled doses at the end of the second quarter which resulted in those infusions being completed early in the third quarter. Net product revenues for ELEVIDYS remain nearly $122 million for the quarter. Net product revenue for the PMO franchise in Q2 was roughly $239 million, which represented modest growth over Q1. Going forward, the team remains committed to supporting those existing PMO patients who have been remarkably compliant with their weekly PMO regimen for years now. And while we expect some cannibalization by elevates in US in the coming quarters, ex-US PMO growth will help to mitigate this cannibalization. Now turning to the ELEVIDYS launch. We're pleased with the launch progress date and are on track to realize the opportunity in front of us. To put the current situation into perspective, almost the entire Duchenne population became eligible for ELEVIDYS essentially overnight. What we're seeing right now is the key neuromuscular centers reacting to unprecedented demand from entirety of their Duchenne patient populations. The treating sites are rapidly working through and prioritizing patient demand. We're confident in their ability to manage this, given the fact that these are the same centers who navigated all of the recent Duchenne and SMA launches, including Zolgensma. Your uptake assumptions should reflect the patient journey to…

Thanks, Dallan, and good afternoon. I'll begin my remarks with the approval of the ELEVIDYS label expansion and then provide an update on our pipeline. Following decades of research and development and after a thorough scientific review that critically considered all of the available evidence across multiple studies, the Office of Therapeutic Products of the FDA approved expansion of the ELEVIDYS label based on the following: the clinical benefits of ELEVIDYS in ambulatory patients was confirmed by our double-blind, placebo-controlled study embark and related study. And based on these data, traditional approval was granted to all ambulatory Duchenne patients, aged four and older. The mechanism of action of ELEVIDYS is universal, regardless of disease state as long as muscle is present. As a result, the ELEVIDYS dystrophin expressed by our therapy in nonambulatory patients is reasonably likely to clinical benefit in this population. As a result, accelerated approval or AA has been granted for the treatment of nonambulatory patients, ages 4 and older. The accelerated approval for nonambulatory patients includes a post-marketing commitment to confirm clinical benefit, which we addressed via our nonambulatory and late ambulatory study 303, also known as ENVISION. As a reminder, ENVISION is a global randomized, double-blind, placebo-controlled 2-part study, evaluating the safety and efficacy of SRP-9001 gene therapy in nonambulatory and older ambulatory individuals with Duchenne. ENVISION is progressing well with US enrollment complete and the remaining recruitment occurring ex-US. Enrollment is expected to be completed in 2025 and with our last patient last visit expected in 2027, following an 18-month placebo-controlled period. We also continued to advance clinical studies that monitor long-term follow-up with ELEVIDYS. Our long-term follow-up studies include ENDEAVOR and Expedition. ENDEAVOR is a Phase IV observational study that will follow individuals treated with ELEVIDYS for up to 10 years. One-third of…

Thanks so much, Louise. Good afternoon, everyone. Before I review our financial results, I want to discuss quickly just three topics. First, I just want to add some color on our approach to revenue guidance. We plan to refine the guidance as we get further into the launch, but I think what we've provided today will help everyone model the ELEVIDYS launch curve more appropriately. The guidance likely isn't surprising anyone as the trajectory is consistent with our previous CMO launches and the guidance also provides some visibility of our expectations and how we expect ELEVIDYS will impact our PMO franchise. It's apparent that we expect modest cannibalization of the PMOs over the upcoming year? So all-in-all, the broad takeaway from our guidance is that the ELEVIDYS launch will put us in a very, very strong financial position. In fact, as Doug mentioned, we were actually cash flow positive for the first time in our history. We expect to achieve sustainable cash flow positivity in the middle of next year. So with strong revenue growth reaching almost at the end of the decade, we've had several questions on how we plan to utilize our cash. So I want to briefly talk about how we're thinking about deploying capital in our BD strategy. And before I went into it, I think it often gets overlooked that we have a proven track record of successful business development. In fact, we wouldn't be here today if we had an identified evidence as a differentiated approach to treating DMD and acquired it. We'll continue to leverage our capabilities in both neuromuscular and rare disease space, and this will remain a core area of focus of ours. We have the ability to use propensity match controls to better evaluate efficacy early in studies, which can…

Thank you very much. Again, Lisa, let's open the lines for Q&A.

[Operator Instructions] And our first question today will be coming from Tazeen Ahmad of Bank of America Securities. Your line is open.

Hi, guys. Good afternoon. Thanks for taking my questions. Maybe I just want to get an understanding of what the actual bottleneck here is because based on everything that you said and based on work that seemingly a lot of folks have done, demand seems to be quite high from patients and physicians. You talked about start forms and you're pleased with the number of start forms. But what is the issue with the timing of getting the patient journey from prescription to actual drug in hand. Has that changed from the time that you got approved for the four to five year roles, when did it start lengthening to what you're saying, what is it three to six months that it's going to take or --

Three to five months

3 to 5 months, when did that start? Is that the key bottleneck here? Thanks.

Thank you very much for your question, Tazeen. The short answer is, there really is no bottleneck at all. Now as I think we said in the last earnings call, it's clearly the case that with the four to five year olds, we were all in -- I mean all, not just us, physicians, families and the payers, we're all in kind of a crisis mode, prioritizing kids that were about to age out of the label, and we're able to do it more rapidly than is normal. But the normal process is about three to five months. And I mean normal that's not atypical for these sorts of therapies, but it's very typical for EXONDYS, VYONDYS, AMONDYS and now ELEVIDYS, ELEVIDYS has some additional requirements, including, for instance, the requirement that one test for and is negative for neutralizing antibodies. So to be very clear, there is no bottleneck here. We're doing brilliantly. That start forms are great. Patient and physician demand is great. Manufacturing is great. Everything going very, very well. Payer interactions are really, really encouraging. I think Dallan mentioned, we've been having conversations for quite a long time, but just recently, we've had conversations with payers that cover some 225, almost 0.25 billion lives in the United States. And that's enormous. And they've been very productive, and we've already seen pull through good policies. Now a lot of -- somebody of these policies is going to take some time. So just we're very clear, it takes about three to five months from start form to infusion. We're getting an enormous number of start forms. We're getting a crazy number of assay kit requirements. We had to literally go into our own mini crisis mode to make sure we could satisfy all of the assays that were…

Thank you. [Operator Instructions] And our next question will be coming from Anupam Rama of JMP. Your line is open.

Hey guys. Thanks for taking the question. Also, it's JPM. The -- thanks for providing that 2025 net product guidance. And I think you said multiple times, addressing the prevalent population kind of into the 2030-2031 time frame. Is that -- should we be thinking about more linear growth over that time? Or how should we be thinking about, say, like a time to peak scenario over the course of the year.

Yes. I mean we've given obviously -- thank you very much for your question, Anupam. We've given obviously more granular longer-term guidance that is typical of us, but we wanted to really reflect accurately what we're seeing in this launch. I'm not going to get into a time more granularity on the long-term forecast other than to say, peak year sales will occur in the back half of this -- significantly in the back half of this decade. And as I said before in the script, we'll be treating the prevalent population through the entire decade, and we'll be moving the incident population in the 2030s.

And our next question will be coming from Gena Wang of Barclays. Your line is open.

Thank you. I will also ask one more question regarding the revenue. So Doug, did I hear you correctly, 3Q, you still expect modest growth even despite you will take 3 to 5 months for staff warm to infusion. So that was the first question, just to confirm. And then the guidance, sorry, I will ask again in guidance, $2.9 billion to $3.1 billion. But when we look at, that's in line with first order analytics of a $3 billion 2025 consensus, and they do have the numbers the sell-side consensus number for ELEVIDYS for 2025 is $2 billion. So do you think that, that number is in line with your assumption? And what are the early launch data to support your assumption?

Yes. Thank you very much for your question, Gena. So first on Q3, relative to what I would characterize as the explosive growth that we're going to have as the start forms turn into infusions. In third quarter, I've said, we'll have modest growth, but it's still 30% quarter over the immediate preceding quarter. So it will be very nice robust growth. But remember, we just got approved at the end of Q2, and then it does take about three to five months. So it will be really kind of back-end loaded in the quarter. But we'll see, broadly speaking, about 30% growth, then we'll double it in Q4. And then on 2025, I think we are largely in line with where the external world is, although that wasn't our goal. I think it's a happy accident that we happen to be lined up. And to the question of sort of what are we seeing -- it is early days. So what are we seeing early days that gives us confidence. There's a lot. Just so we're very clear -- again, no one has more experience in launching Duchenne therapies than Sarepta Therapeutics. Our force therapy in some weird ways, it's our fifth therapy because we launched this very narrow label on ELEVIDYS and now we're launching with a broader label -- so a couple of things. We know Epi [ph] extraordinarily well. We know Epi down to the state level. So we're very confident on the Epi here. We have a lot of confidence around the launch curves itself. And what we're envisioning from the launch curve right now is exactly what we envisioned we would see in all of our modeling, and it is consistent with, again, the launch of EXONDYS in august and VYONDYS. And we have a lot of granular data. Just remember, we have down to patient-level data on start forms and the like. We know all of the sites. We just have an enormous amount of visibility. It's still early days. So to Ian's very good point. We will update our views on things as we track to the end of the year, into early next year. But obviously, early days, we felt a pretty significant amount of confidence to provide people with this overall shape of the launch and discuss 2025, which for us is a bit unusual. We wouldn't provide this kind of level of information is surely other than we wanted to really map out what we're seeing right now and feel like we're being transparent with folks. So we feel very good about this launch right now.

Thank you. Our next question today will be coming from Joseph Schwartz of Leerink. Your line is open.

Hi, all. This is Will on for Joe. Thanks for taking our question and congrats on the progress this quarter. So one from us. For many years, we've seen the company successfully execute on a mission to help boys at DMD. However, this is something that may not be fully appreciated by some of the community. As it appears that the company will continue to invest in next-generation therapies and support these patients. Why do you think there is a disconnect with some of the community? And what are you doing to actively address it, especially with competition or potential competition on the horizon? Thank you.

I think anybody who's been watching us carefully over the last seven to eight years, we'll know that we've been wholly committed to bringing a better life to the Duchenne community. And we've at the risk of being a bit of modest, we've been pretty brilliant in our ability to do that for therapies later, all of very successful launches and I believe, very transformative, meaningful dystrophin restorative therapies, which we now can reach well over 80% of the Duchenne population. And we're working on actually making it even greater. We are successful with our attempts to knock down pre-existing neutralizing antibody, and we have a lot of conviction that we will be. We're already dosing in one of those two studies. The other one will come very soon. We'll be getting to much higher even than 80% of the Duchenne community. And the good news is that I think that those who are informed and have been watching us get this. I think to be honest, the great bulk of the patient community fully understands and is cheering us along. I can tell you, we all stay very close with the community and the community understands that and that's why in addition to cheering us along, people have been watching the data and are very excited and hopeful they can get on the therapy as soon as possible. Same answer with the physician community, I would say to those who are curious, just talked to physicians who have actually dosed this therapy across the spectrum of Duchenne and ask them what their experiences have been. I think you'll hear that they're very excited about that. So I think the right-minded people are getting it. And I think that there's a lot of enthusiasm for this therapy as well, there should be and it creates for us an enormous responsibility to get this therapy out to these kids. And this team is doing everything they can to do that. And the great news is that, there's no team better than this team to do it. So I don't want to give Dallan and his team too big ahead, but the fact is that this team is brilliant at serving this community, and it will not change with the ELEVIDYS. We're off to a great start.

Thank you. Our next question will be coming from Brian Abrahams of RBC Capital.

Hey, good afternoon, guys. Thanks for taking my question. And really appreciate all the granularity here on the launch dynamics. You did mention that there were some wait times at centers as they navigate demand. And so it sounds like capacity is somewhat of a gating factor here. Can you give us a sense of what some of the limitations here are at the center level any additional infrastructure or processes that these sites need to put into place to administer ELEVIDYS and monitor patients? And where you think that could expand over time? What's embedded in your guidance terms of capacity expansion? Thanks.

Okay. I'm going to turn this question over to Dallan, but let me say a few things in advance. First, we don't have a fundamental capacity issue. I want to be very clear about that. We have about 75 sites in the US. 75% of which have already dosed patients. The probably the number one metric to the success of therapy is, is the experience with it and how well it's working. But with that said, then the experience with the actual infusion is really important. So having such a significant number of sites, I believe, more than any other gene therapy ever launched with and having 75% of them already having those patients is significant. And I think one of the things we're seeing early days is there are definitely some sites that in the absence of more information are just worried about the onslaught and trying to manage the amount of demand that's coming in because there are some 12,000 or so patients in the United States with Duchenne muscular dystrophy. 80% or more of them can be treated with this therapy. They've got to prioritize and things that issue through and they're pondering it. But with that, Dallan, if you want to provide more granularity than I have on this topic, please.

Yeah. Thanks, Doug. And Brian, thanks for the question. And as Doug said, we've got more than enough capacity. We've got more than enough capacity today to accommodate even the peak years of sales, Brian, but really what is happening out there is, as I said in the -- in my remarks that essentially overnight, the vast majority of patients became eligible. So each individual center just have to work through all of their patients. They've got all of their patients reaching out and figure out how they're going to approach who they're going to prioritize who they're going to dose first. And this is to be expected and everything is really exactly as we have forecast anticipated and expected. And we're doing everything we can to support these centers. What makes it -- what should provide a lot of confidence is, these are the centers that are at the forefront of precision genetic medicine. They were the ones that figured out Spinraza. They figured out Zolgensma. They figured out our previous narrow label, and they're just in the process of figuring this out. And these early stages are just an adjustment period as they ramp up their ability to serve the whole patient population.

Thank you. Our next question today will be coming from Salveen Richter of Goldman Sachs. Your line is open.

Good afternoon. Thanks for taking my question. Just going back here to this guidance. It seems like you have the infusion centers, but there's some kind of bottleneck or lever you're using with regard to kind of how you're determining 2025 guidance. So is -- if the gating factor here, as you -- given the demand, is it number of patients that can be treated at an infusion center at over a time period? Or is it the manufacturing supply in the context of the demand? And then help us understand, how you're thinking about cannibalization of the PMO side of the business in your '25 guidance? Thank you.

So again, I would say that -- first of all, thank you for your question, Salveen. I would say I think it would be a mischaracterization to ENVISION that there is some significant bottleneck. The shape of the curve is related to a number of things. It's not related to demand, patient demand, physician demand is going to be very, very significant. But then there's process -- the process of getting through the infusions, payer interaction and the like, all defines the shape of the revenue curve over time, and we're very pleased with where we are. And we don't have a problem to solve. We will, if all goes well, and it is going exactly that well, means we're going to do $2.9 billion to $3.1 billion next year, which means a very significant number of patients are going to significantly benefit from our therapies. And I would remind everyone that is years away from peak year sales, and we'll be treating the prevalent population over the entire course of this decade into the early 2030s. And then on cannibalization, there will be cannibalization, if for no other reason, there is a kind of competition for start forms for patients as well. But in this year and into next year, we see that cannibalization is quite modest. Thank you again for your question.

Thank you. And our next question will be coming from Ritu Baral of TD Cowen. Your line is open.

Good afternoon guys. Thanks for taking the question. Doug, you've given different sort of aspects of guidance going forward, including the fact that you guys think that peak is going to be towards the end of the decade. You've previously given us a rough figure of about $4 billion peak for ELEVIDYS. And this was a few years ago and the centers were all not all up and running and stuff like that. But do you still see that as the general peak in your internal assumptions? And I wanted to also ask about the sort of levers between the top of your guidance and the bottom of your guidance. It's a really, really tight guidance. So I must say I have to start for that will track all the way out through some element of 2025, can you talk to the volume of start forms and whether -- what the levers are for the top and the bottom and how far out those start forms will map your revenues, enhance start forms?

So a couple of thoughts. On your first question, yes, some years ago, I provided long-term guidance. There is -- we have no basis for amending that guidance at all. We feel very comfortable about the prior guidance that we provided. So there's no need to update that. It is -- does appear still to be very accurate. And on the number one thing, we have a lot of starts forms. They don't have all the starts forms yet, of course. That's not the way it works. But remember, we have more than anything else, great experience. Like we know what level of start forms correlate to what, we know what the launch curve should look like we have a ton of algorithmic ways to look at this launch and map our expectations versus the current launch. And the short answer is based on all of our experience and knowledge and algos, we are doing very well. As I've said, the early signals exceed our optimistic view on this launch. So we're -- I'll just say again, we are comfortable with the guidance that we're providing right now. And to your point, it is -- we're getting to the relatively big companies. So while it's tight. I mean there's still $100 million on each side. But to your point, it's getting to be pretty tight, but we feel very comfortable about providing that level of guidance.

Thank you. And our next question will be coming from Mike Ulz of Morgan Stanley. Your line is open.

Hey, guys. Thanks for taking the question. Maybe just a quick follow-up on the PMO cannibalization. You're expecting modest cannibalization in 2025. But just curious how you're thinking about that longer-term? Should we still be thinking some modest impact? Or should that ramp up as sales start to ramp up? Thanks.

Well, it's -- so first of all, thank you for your question, Mike. It's an interesting issue. We have always modeled some robust -- eventually some robust cannibalization of the PMO franchise what we've seen so far has been fairly been very modest. And what we're modeling into next year is modest. It might be more significant in years beyond that, but there's also some reason to believe that our views have been a little bit conservative on how much cannibalization there will be, or I should say, aggressive on the amount of cannibalization. We may have been a bit -- our own internal malls may be a bit aggressive on the amount of cannibalization. But what I would say right now is our model suggests that the cannibalization is very modest now. It will remain pretty modest next year, and it might be more significant in the years after that.

Thank you. And our next question will be coming from Brian Skorney of Baird. Your line is open.

Good afternoon, guys. My question is on the cannibalization of PMO as well. And just trying to contextualize when you say early modest cannibalization, why isn't it just a straight-up equation based on the prevalence of the exon amenable patients and the penetration that you have into PMO. Like I would sort of calculate that somewhere between eight and 10 ELEVIDYS patients should result in one PMO patient cannibalization assuming that person would have to be forced by the insurance company to choose to take ELEVIDYS and come off the PMO. Is that not the case? Are you seeing patients who are getting commercially treated with ELEVIDYS and maintaining their PMO post- ELEVIDYS?

Dallan, do you want to touch on this? .

Yes, it's a good question. One of the reasons it's not a -- you can't map it out that way, is that right now, there's a significant percentage of our PMO business that doesn't come from the U.S. In terms of commercially right now, are there patients that have been dosed with gene therapies that have gotten PMOs. We haven't seen that to-date, but we've seen that in the SMA space. And I think we're aware of some patients that are -- that are trying to get access that have dosed with ELEVIDYS before, but we haven't seen that to-date.

So, noting that, that's the problem. Dallan touched on one of the confounders to your analysis, Brian, which is that ex-U.S. sales exist and have been relatively robust. So, your math is seen with the closed system and we don't have a close system, we actually have an open system with ex-THANK YOU PMO sales.

Thank you. Our next question is coming from Uy Ear of Mizuho. Your line is open.

Thanks for taking our questions. So, I guess maybe just going back to the start form. You said that since the approval in 2023, you've been receiving forms from all these sites and physicians. Just wondering if there were any forms that will be filled in patients who aged out before they can get treatment. And could you maybe also sort of define what you mean by patients who are declining? Are these older patients? Or does it ages doesn't matter at all? Thanks.

I want to apologize. Dallan, if you understand -- I didn't quite understand the question, apologies.

Yes. No, I think understood it, and please let me know if I did. In terms of the last part of your question, the where the doctors seem to be prioritizing, no two sites or doing it exactly the same way. But they -- as we've seen with our PMO launches, patients who are in declining ambulatory phase. So, that's kind of in the 9 to 11 year old age ranges, those patients were predominantly prioritizing the previous three broad label exon skipping launches, and we've seen the same thing here. And what was the first part of your question again?

I think you may have been asking if we -- any patients that would have otherwise aged out.

Aged out. Yes, the age, Yes, I was excited about that one because the team did such a great job in execution. There were very few patients that did age out with the -- in the initial narrow label. There were a few. And of course, team never gives up on any patient. And we will -- those patients that had aged out in the prior label will now be eligible again. So, we will do everything we can to support anybody who did age out and comes back into the into the system.

And then finally, what Dallan is suggesting on the prioritizing the rate declining -- and simply, that's just -- it's just a mathematical fact. You're seeing patients start function as early as four years into their old 30s. And then you see kind of a peak right now in the -- right in that kind of 10, 11, 9 age range. And that's what we've seen with the PMOs as well. But the good news is we're seeing a robust start forms across that entire group, which gives us a lot of confidence in where we're heading as an organization.

Thank you. Our next question today will be coming from Robert Finke of Guggenheim. Your line is open.

Hey, thank you for taking our question. This is Robert on for Debjit. From our side, a couple of questions here. Do you anticipate manufacturing capacity will be a gating factor at any point between now and peak sales?

So the short answer is we don't envision that manufacturing is going to be the bottleneck for the next few years. And by the time that we would see it as being a rate limiter will be hopefully in suspension by that point, and then we shouldn't see any limitation. So we're very good where we are right now.

Thank you. Our next question is from David Hoang of Citigroup. Your line is open.

Hi, there. Thanks for all the great updates, and taking my question. I just wanted to ask if you've got any feedback from your centers about post-administration monitoring of these patients. There's obviously a number of labs that need to be followed for a period of time for safety. And I just wanted to ask whether value consideration and sort of the speed of how fast patients can be done and then ultimately, overall, the shape of the launch curve?

Yeah. Thank you very much for that question, and it's very insightful. A lot of times, you think about the infusion at the last moment and that -- that sort of the cadence of infusion describes the amount of patients that can be dosed. But of course, if 1 is going to do this in a thoughtful way, it's important that there's a lot of very good follow-up, and there is. So that amount of infusions you can do relate not just the amount of infusion room infusion appointments you can have, but good follow-up, and we're very, very supportive of great follow-up. So that is a part of the entire process -- and to the best of my knowledge, we're seeing a very stable safety profile as well based on that monitoring, but Louise or -- Louise in particular, that on getting any of that wrong revenue.

Thank you. Our next question will be coming from Kristen Kluska of Cantor Fitzgerald. Your line is open.

Hi, everyone. All the best with the expanded launch here. I wanted to ask, if your 2025 numbers assume anything related to a European approval? Is that baked in here? And then I understand that this process takes a few months' time. But what's the key driver behind reaching peak sales in a few years versus, say, the 2020 time frame? Thank you.

Yeah. So our guidance does not assume -- first I want to be very clear. We'll leave it to Roche to talk about their ex US approvals. And as everybody knows, EMA has already accepted their submission for review and that should result in an action in 2025. But the numbers that we're giving here right now don't presume that issue either way, just really relating to our US sales. And the short answer on the revenue curve is, of course, there is a revenue curve that occurs and it is a function of everything from the process to the site, the monitoring and site infusions and payer interactions and the like. And so as I've said, we believe right now that we're going to do $2.9 billion to $3.1 billion next year across all our therapies. We will continue to treat the prevalent population over the course of this entire decade. Peak year sales will be some years after 2025, and we feel very good about where we are and the good work we're doing for patients.

Thank you. Our next question will be coming from Biren Amin of Piper Sandler. Your line is open.

Thanks for taking my guys. You commented on the number of infusing sites over the last few quarters with the number of infusing sites increasing in Q2 from Q1 by about 13 to 15 sites according to our calculation. So how do we think about patient treated per site and the consistency of the volume of patients treated per site?

Well, so let's first -- I'll turn this over to Dallan, and he can chat a bit about that. But just so we're very clear, we are very comfortable with the number of sites that we see. Now what you may see over time is a general increase in some sites. That is not part of some grand proactive strategy, but really relates more than anything else, but being responsive to requests from site. So, if an appropriate site asked to be a site, I want to go through the educational process and to be validated to be a site we would certainly consider that. But as it sits here right now, we are very comfortable. And I will remind everyone in advance of this of this launch, we had an aspiration to be 50 sites with a goal of maybe even someday getting to 70 sites, all of which would be much higher than any other launch on with gene therapy. And so sitting at 75 sites, we feel very, very good about it. But if you want to provide any other metrics down on kind of productivity by side of the life. Feel free.

Yes. No, thank you for the question. And I don't know where those numbers came from because our site -- the number of sites has been very consistent. We have a rightsized model and we've been hovering right around 75%. Now that said, just as Doug said, our model is flexible. So we can bring new sites on, get them up to speed and bring them on very rapidly. And one of the things with broad label is the possibility that we'll be bringing on some adult neuromuscular sites to accommodate older patients as well. So, as Doug alluded, we have more than enough capacity today, but importantly, to serve patients. We have built flexibility into our model, so that as needed to support patients, we can bring on new sites very, very rapidly. The team is very responsive.

Thank you. And our next question will be coming from Gavin Clark-Gartner of Evercore. Your line is open.

Hey guys. Thanks for taking the question. So I fully appreciate the longer conversion cycle as we're looking at the back half of '24 and into 2025. But you've also referenced your approved PMOs as analog for adoption. Those themselves are the largest ramps within the first 1 to 2 years after approval when we're talking about new patient adds. And that's basically how all other rare disease launches go with very high unmet need. So I guess like even with a few months of the lag process, why would peak sales not be at some point in 2026?

Because it won't be. We have good modeling. We know exactly -- we have a very good view of where we're heading with capacities and the process and the like, and we're confident that it won't be to your sales in 2026. Another thing you have to remember -- and by the way, I think the people are trying to solve for whether varies symbolically, there's not we're very pleased with this approach. One has to remember that there's a one-time therapy that we have to be -- we have to prioritize being responsible and we want to make sure that all of these sites are in a good place where they're not prioritizing, getting as many patients as possible dosed without regard to safety and follow-up and the like. And we've done a really good job of that. The sites are doing a very good job of that, all of which means we're going to be very successful this year. We're going to have good modest growth in the Q3. We have a very, very robust growth in Q4, doubling in Q4, we're going to do $2.9 billion, $3.1 billion across our therapies in 2025. And then as it relates to 3Q sales, it will be in the back half of this decade.

And Doug, can I just jump in? I think you've been alluding to this, but Gavin, your question is a good one, and I think Doug has been alluding to the fact we have visibility at these sites at a very granular level. We haven't mapped out by payer coverage by patient. We know exactly who's lined up, how they're going to be lined up. We map this out in a very, very granular detail. And this is why we have a very high level of confidence in our forecast assumptions. So I hope that added level of detail helps a bit.

Thank you, Dallan.

Thank you. And our next question will be coming from Kostas Biliouris of BMO Capital Markets. Your line is open.

This is Dale on for Kostas. Thank you for taking our question. So our question is on the LGMD gene therapy portfolio. Given that half of your LGMD gene therapy portfolio uses the same vector and promoter -- and actually, all of them are using the same vector as ELEVIDYS. So how are you thinking about potentially leveraging FDA platform designation platform to accelerate the development of the LGMD gene therapies and other early pipeline assets. And also if you can comment on the potential impact from the inclusion ELEVIDYS effect on the time function test in the label on patients and physician uptick payer negotiations and potential future competition? Thank you.

I'll turn the LGMD question over to Louise and our approach to accelerating the LGMD development.

Yes. Thanks for the question. I think you're exactly right in terms of using the ELEVIDYS experience to help accelerate the LGMD platform. We've been using the same rh74 vector for these programs and just to cite the sarcoglycanopathy, similarities between them. We've received Fast Track Designation, as I mentioned in my remarks, and pulling on every lever possible with FDA to make sure that we're moving in the fastest speed possible. \ We've been very happy with the interactions so far, and our 9003 pivotal trial, as noted is our open-label trial with in terms of looking at this ultra-rare population and being thoughtful about the way that we develop these with LGMD2D and QC coming along. So certainly, we're leveraging everything possible and our safety experience is ELEVIDYS for the LGMD portfolio.

Before we move on Dallan, did you have something else to add?

I think Dale asked about the time function test, the data that's in our label and how those may be impacting the payer conversations. And Dale, it's a great -- really, really great question because that is really what the team is focused on with the payers is that incredible new data that's in our label. And there has been -- as Doug outlined in his comments, I did a little bit in mine, the engagement, the interest from the payers to understand that data. And so that's a big part of that engagement with the payers right now going through that new data that supports a broader label.

Just to remind everyone that not only did we hit all of those not only statistically significantly. The importance of that can't be overstated. I would remind people that time to rise, the benefit we've seen on time to rise is correlated with over a 90% decrease in the risk of early loss of ambulation. So, I mean these are not only important metrics, but they correlate importantly, the very -- things that matter a lot to patients.

Thank you. And our next question will be coming from Tim Lugo of William Blair. Your line is open.

Hey team. This is John on for Tim. Thanks so much for taking our question. Maybe a follow-up on the last one. Beyond the internal pipeline, with your healthy balance sheet and your expanded launch in front of you. Just wondering if you can give us any updated thoughts on how you're thinking about potentially in-licensing new programs?

Sure. I will turn this to Ian. Ian?

Hey thanks for the questions. This is what I discussed in our prepared remarks, I mean as an organization that's been developing therapies and successfully manufacturing them and all the way to commercialization, it gives us a wide amount of substrate to evaluate -- and so we're going to be just looking at ones that fit very well with our existing capabilities and being able to leverage that and being able to deliver to patients quickly. So, -- we've been really fiscally responsible at the transactions we've done in the in the past. Like I said prepared remarks, we're going to have a lot more resources to deploy, but we're still going to use that same approach. There are a lot of interesting therapies that are being developed. But before maybe this week, the valuations were incredibly high, and we're not going to chase opportunities. We're going to look for ways to build value from a development perspective for patients, but also for investors. So, we're very keen on looking at valuation and making sure that it makes sense based on the market opportunities.

Thank you. And our next question will be coming from Gil Blum of Needham & Company. Your line is open..

Hi everyone and thanks for squeezing us in. Maybe a last question, I'm trying to understand the potential bottle next year. But does on the really just boil down to how many treating physicians you have at each center? And if you can provide any thoughts on how that metric works? Thanks.

So, again, I'm going to frustrate you by answering this question again the same way, which is we don't have bottlenecks. We have a launch curve. We're doing very, very well. We have a significant number of sites, a significant number of treating physicians at sites, a lot of enthusiasm. We have more than enough sites, more than enough positions, extraordinary demand from patients and those physicians, great interactions with payers, a very strong manufacturing approach right now. We feel very good about that from a capacity and supply perspective and a great distribution channel. As a result of that, we're going to have a really successful back half of this year, and we're going to have a 2025 that will mean that we're going to do revenue across our four approved therapies of some $2.9 billion $3.1 billion. We are profitable today. We were cash flow positive this quarter. We will be in the next couple of quarters, very consistently cash flow positive on a go-forward basis. We're in a very different place than the vast majority of biotech today. We have a very strong, very sustainable business, all of which is focused first and foremost on bringing a better life to these patients. We're going to bring a better life to a lot of patients over the next many years this decade. And secondarily, but also importantly, rewarding those investors who have stuck with us and have committed themselves to this mission. So we're in great shape, and not to be overly defensive, but we don't have a bottleneck that we need to solve for. We feel very good about where we are.

This concludes today's Q&A session. I would like to turn the call over to Doug for closing remarks. Please go ahead.

Thank you very much. Thank you all for your questions. Your incite questions this evening, and thank you my team for those great answers. Very excited about where we're going for the rest of this year. We have a lot of work to do this year. ELEVIDYS launch is going well. Our continuing service of our PMOs are going very well. We understand the enormous responsibility we have to these patients to ensure that the greatest number of these patients can benefit from our therapies, and we're going to make the ELEVIDYS launch brilliant. I look forward to updating you across this year on launch, performance and with the team update you on our pipeline advancement as well. And with that, have a lovely evening, and we look forward to talking you soon. Thanks.

This does conclude today's conference call. You may all disconnect.