Great. Good morning, everyone. It's my pleasure to introduce the Sarepta team. With us, we have Doug Ingram, CEO; Ian Estepan, President and COO; Ryan Wong, CFO; and Louise Rodino-Klapac, President of R&D. Thank you for joining us. Maybe we'll start here, Doug, with a question for you. You've announced your retirement as CEO by the end of the year. How are you thinking about working through the transition and the overall succession plan? You can ask these folks if I've been winding down recently. I think they're surprised my intensity level's been going up. Right now, we're not transitioning anything. We got a lot to do as an organization. I did announce that by the end of this year, I will retire. There's an active process ongoing. When the Board makes its final decision on that, then I will obviously do everything in my power to ensure that that individual is maximally successful, both for the company and its investors, and for patients as well. I stand ready to do whatever they want for as long as that successor would like to ensure that we're successful, and I'm very confident that we will be. Great. You have laid out your priorities for 2026, notably stabilizing ELEVIDYS, advancing the pipeline, and strengthening the financial setup. To start here, can you lay out the progress towards these priorities and what the key milestones for the company will be over the next 12-18 months? Sure. Right. To your very good point, the strategy at Sarepta is quite straightforward. Let us start first with our four approved therapies. We have three PMOs, as you know, and our gene therapy, ELEVIDYS. The goal there is to maximize that opportunity, really first and foremost, for the patients who benefit from that therapy. Even in our base case, we're going to do quite fine. We would like to exceed that base case because that means even more children and young men with Duchenne muscular dystrophy are going to live better, freer lives because of it. We're doing very well there. We'll come back and talk about that. Let me just talk about each pillar. That really leads to the state of play in the company from a financial perspective. We're in a really strong financial perspective. Just consider that we're cash flow positive. We were a little over $800 million, I believe. What was it at the last quarter? Maybe we were $750 million. Seven something, we'll be back to $900 million+. Yeah. We'll be significantly over $900 million. We're cash flow positive, we're profitable, usually on a GAAP and a non-GAAP basis. That will continue to be the case over the course of the intervening years, even as we fully invest in our pipeline. Just consider our sales forecast. We've said that we will do somewhere between $1.2 billion-$1.4 billion. Even if you assumed we were at the very bottom of our guidance, our cost structure is somewhere in the hunt of a little over $800 million. We're just a comfortably cash flow positive organization that isn't, thank goodness, given where our stock price is today, which if I can editorialize, is surrealistically goofy. In one sense, other than employee morale, it really doesn't matter because we are not slaves to the equity markets to get our stuff done. Of course, where are we going in the future? We have a really exciting pipeline. Our siRNA pipeline is exciting. We have five therapies that are in the clinic dosing patients right now. We have a few more that are heading to the clinic soon. We have six research programs on top of that with our really exciting during the time. Now let's talk about success in each one, and I'll do it real quickly, and then we can actually get to Q&A, so I'm not just- Okay. I don't need this from y'all. They were taking bets on whether any of them would have a chance to talk today. I'll be brief. From a sales perspective, the delta between $1.2 billion and $1.4 billion is really entirely ELEVIDYS and ELEVIDYS success. We've always said, for the course of this year, you should really be modeling toward the lower part of the $1.2 billion-$1.4 billion for the simple reason that the actions that we're taking and the activities we're doing are going to be successful, but this is a very long cycle of process. It's going to take some time. We've done great there. We've more than doubled our sales force. They're out and being very productive, both from a reach perspective and a call perspective. That's going well. Our promotional campaigns themselves are really powerful right now. We have a contract sales force in the field, and we have a group of what we call the patient education liaisons, whose goal it is to really educate patients and make sure they're prepared to have thoughtful discussions with their physician about the journey, particularly for ELEVIDYS. That's going well. Tons of green shoots of success there. I could give you interesting stats on that. That is going well. We're doing great financially. From an siRNA perspective, you saw that with DM1 and FSHD, we had very encouraging results from our SAD study that we announced a couple of months ago. We're really excited about getting a look at the next set of data, which is going to be even more robust. It'll be multi-ascending dose. It'll be more patients and more biopsies and the like, and that'll happen in the second half of this year. We've already started dosing our Huntington's program, which we're excited about. IPF is dosing. SCO2 is dosing as well. We've made a lot of progress across that portfolio as well. Can you touch on the leadership changes at the FDA and whether there's any risk to you from that dynamic? As we sit here today, we see no risk at all from changes at the FDA. For two reasons. First of all, let's think about where we are with ELEVIDYS. We had a bunch of risk last year. People for abandoning our stock in the summer of last year. We had a really strange interaction with the FDA where they were pressuring this therapy off the market, notwithstanding the fact that we were able to get that therapy back on the market within five business days. I would suggest it would be very difficult for someone to replicate that success. It was understandable that people were worried about that relationship. From there, things just got very good for us. We negotiated the label, we negotiated a label update with the FDA that got out there to the physicians. We negotiated and got their blessings on Cohort 8, which is our pretreatment with sirolimus, which will be the pathway back for the non-ambulatory population. There really aren't any open extent significant risks in the ELEVIDYS. Even with the change, it doesn't matter. On the PMO side, that really didn't exist as an organic risk anyways. I think that what we're seeing so far in the changes at the FDA have been very positive. I think the acting commissioner, I have not interacted with him directly myself, but really universally, people are saying very good things about him. He comes from the food division, so he may not be steeped in the drug development process, but he understands how to run large organizations. He's apparently very rational and very reasonable, and everybody that's interacted with him so far has come away saying he's a good, steady hand at the FDA. I think the same thing has been said about the CDER side as well and that change, and I don't know much about the CBER side. I think there's a lot of hope for a stable FDA, and that's what we need. I have been a big proponent for many years over the idea of really trying to modernize the FDA and get more thoughtful and mirror the FDA today with the current state of science, and certainly in genetic medicine and rare disease. What we really need more than anything else right now at the FDA is a stable and predictable regulation. We, for one, are very excited. An important point. It didn't seem like the markets really fully discounted both the risks that I think were happening previously, and now I think a very strong tailwind now because of that exact point that you're making in terms of predictability. I think they're making dramatic improvements in trying to make sure that goalposts don't get moved, reviewing applications that previously had gone against what the agency had communicated previously, and now actually factoring that in and hopefully, we'll see in the next few months what happens with some of those applications that are getting re-reviewed. I think that stability and the ability to be able to predict what's going to happen should be really important for the investment community. I think it's a much better backdrop. Okay. In the end, let's be very clear, this is what we want as an organization. We want the FDA to apply the standards that exist at the FDA and to review therapies and the totality of the evidence, and to support those therapies where it makes sense to, but not to support therapies where it doesn't. We're not looking for some cowboy country. We want an organization that applies standard and rigor, and I think that there is some hope for that, given some of the new leadership at the FDA, at least on an interim basis. That standards part is incredibly important, because if you don't have a standard, then payers start weighing in and other factors start contributing to whether a drug gets utilized or not. Making sure that the standards are met, and that the safe and effective therapies are going to patients is just critically important. Yeah. Starting with ELEVIDYS here, you've spoken about the information gap regarding ELEVIDYS and your initiative to drive penetration in the ambulatory patients. What are priorities for the expanded field team, and what leading indicators are you seeing with physician engagement that suggests a return to growth? The number one overarching thing was to rebalance the discussion, if there was even a discussion. Remember, in 2025, we had two surprising and unfortunate fatalities associated with non-ambulatory, more advanced patients. We spent the entire year focusing only on that, not talking about the benefits and the risks of the therapy and contextualizing those risks, because you can really I would remind you, it's horrible for those families, but two out of nearly 1,400, I mean, this is less than 0.12%. That's the big thing, to get the promotional material in the right place where we can have smart, thoughtful discussions about that. That's what they're doing right now. That's what the main sales force is doing. We have a contract sales force that's really trying to expand that reach into places around the country that are not the top centers, but have opportunities for referral, have opportunities for neuromuscular physicians to get educated that maybe have never been educated before so they can have thoughtful discussions, and that's the big focus. There's lots of interesting, what Patrick Moss, our Head of Commercial, likes to call the green shoots of success, quite apart from the numbers of people in the field. You'll see something like 50% of start forms are coming from sites where a sales rep had visited that site within the last 60 days. You're seeing a significant percentage of sites, I think it's over 30%. A site that had either paused back last year or had never dosed a patient starting to send in start forms. Without getting into too much detail, because I don't know if it violates HIPAA, there's one site that has enormous opportunity but has been very slow, and they've actually put in multiple start forms over the course of the last couple of weeks. That doesn't show up immediately in sales. Remember, it's a six-month process to go from start form to infusion, and it can take even longer with the kinds of appointments you need to have and testing you need to have, antibody testing. It's a great sign for the future and a great sign for 2027. With regard to the total revenue guidance, you spoke to the $1.2 billion-$1.4 billion. We estimate about $300 million-$500 million of it coming from ELEVIDYS based on historical performance of PMOs. What are the factors that influence whether or not you fall in this range? It's all ELEVIDYS, and it's all the impact of our external activities, right? There's still more to do. For instance, we have this, what I believe to be just absolutely knock-out three-year data on ELEVIDYS, and if you look at the three-year data and you're not thinking about getting your kid dosed, you just don't understand what you're looking at. That actually has to get into the promotional material. That's not even yet in the promotional material. When we do adverts and we share it with physicians, the impact for there is just obvious. We need to get that done. It's all that, and it's just the delta there is just that moving this up with such a long cycle therapy is, to use an absolutely worn-out bromide, is turning a tanker ship, so it's going to take some time. I'd suggest a lot of the activity we're doing right now is going to redound to the benefit of 2027, not 2026. What does the FDA need to see from Cohort 8, is that right? The sirolimus study to include ambulatory patients in the label, what could the timeline look post the top-line data here in the second half? I'll turn to Louise, who can talk about the study itself, then we can chat about what we'd expect to see. Cohort 8 of ENDEAVOR is an open-label study in non-ambulatory. This is about the non-ambulatory population. By definition for the study, we're looking for a 50% reduction in the rates of ALI. That's what the FDA will be officially looking for, as will we. It's open label, we have the ability to look at the data throughout. Based on the mechanism of action, all the tremendous real-world evidence we have with physicians using it in the field, we would predict that we would certainly achieve that. We'd go back to the FDA after the end of the year, talk to them about bringing the non-ambulatory patients back to the label, and what the mechanism would be to do so. It could. If I'm not mistaken, I think success in the protocol is a reduction in the risk of ALI. Remember ALI, if you reduce ALI, you're going to dramatically reduce even the theoretical risk of ALF, which is the ultimate thing we would worry about. I think it's by 50%, if I'm not mistaken. That would be the goal. We'll see how this goes, we'll get to watch this data over the course of the year. We do have data right now. We're thinking about the context within which we discuss it. We have something called ENDURE, where we get to collect data on patients in a more formalized fashion as they roll out of the study and as they're on commercial therapy, then on commercial therapy as well. There have been somewhere in the hunt of 11 or so to 14 patients that have been prophylactically dosed with sirolimus. I don't want to suggest that this is the standard we have to meet. I don't want to create too high a standard for us. So far, in all of those patients, there has not been any evidence of an increase in liver enzymes if one pretreats with sirolimus. It gives us a lot of conviction that this approach we're taking is going to be a reasonable one to get non-ambulatory patients back on the label. For those who may wonder why we care so much about this, look, if you want to be just dollars and cents, you'd say the non-ambulatory patient population represents 50% of Duchenne. It is, in fact, an enormous opportunity. There's a more important issue for us than that, and that is everybody abandons the non-ambulatory patient. Everyone always has abandoned the non-ambulatory population. Really, nobody has ever treated these folks or even cared to treat these folks. It's going to get worse. If we don't win with this, if we can't safely dose these kids and bring them a better life, I posit no one will ever do it. Everybody's going to walk away from it. Every current therapy in development right now is ignoring the heck out of these people. The good news is there's a nice synergy there. It's going to be great for investors if we're successful. It's going to be great financially, but it's going to really be great for people that desperately need help and shouldn't be left behind simply because they're in a wheelchair. I think it's important, we've seen about 20%-25% of sites dosing the ambulant patients with sirolimus also. Obviously, this is something that's not in our label, so we can't promote to it. This is how the field is evolving based on the experience that we're seeing, which I think is important to the investment community also, because if you're able to just limit the risk. In the ambulant patients, I think that's obviously incredibly important from a patient perspective, but also from an investment perspective, if you're able to cut that risk. Hopefully, if the data continues to emerge and is very supportive, you continue to see that adoption. Your partner, Roche, announced a new global phase III trial here. Maybe talk about the read-through from that to your commercialization plan, but also why Roche has taken this viewpoint to invest more in the program. Well, I think a couple things. Well, first, they have to do another study. They've taken scientific advice. With EMA and CHMP, they realized that is the pathway. I would say the why they're doing it and investing is because they've seen the data that we've seen, and I think that our colleagues over at Roche are as confident as we are about the way that this therapy is changing the lives of patients. They've done quite well in ex-US. That gives them a really on-the-ground opportunity to see these patients up close and personal, and I think they realize the life-changing benefits of this therapy, and I give them kudos for understanding that and appreciating that. The study itself won't have any read-through to us. It's a placebo-controlled blinded study. It'll take some time to read out. It seems like they've been fairly thoughtful in the approach they've taken. They've learned a lot from the work that we've done, so I'm confident in their study and its outcome, and it'll just give additional evidence in support of this great therapy. Maybe transitioning over to the Arrowhead platform here, or your partnered or your acquired Arrowhead platform. Regarding the siRNA programs, can you lay out how your constructs differ from the others here, such as Avidity and Dyne, and how you see those differences play out in the early data in FSHD, but also DM1? Good. I didn't know I want to do it. You do it. Focusing on DM1 and FSHD, which are our two advanced programs. We use a targeting ligand called alpha-v beta-6, and that's really the differentiator for these two programs, which are delivered IV. Really that gives us the ability, and why we did the deal in the first place, to be most efficient in muscle, getting into muscle, driving that siRNA in there. We know that siRNA itself is more potent in terms of the effects within the cell. You need 25x-50x more molecules of an ASO versus an siRNA to have the same effect within the cell. We also have a large safety margin, 10X. What we've seen from our SAD data, and now going into our MAD data, is the ability to continue to dose up, which is important for getting maximal knockdown and then downstream biological effects within the cell. I'll stop there. You can add anything. Well, I would agree with you. As you move to higher and multiple doses, how are you thinking about receptor saturation or potential for dose-limiting toxicities? Pre-clinically, we've not seen any evidence of receptor saturation. Again, this is where the alpha-v beta-6 comes in. We don't have the same problems with the TFR that others have because of the use of that receptor for iron homeostasis, we're not seeing things like anemia, like others are seeing. That really gives us the opportunity to continue to dose up, and we haven't seen that saturation that others may have. Our NOEL is significantly above where we're actually looking to dose, I'm feel confident about it. How much proof of concept do you think that initial data represented? Can you frame how to think about translation to the next data release and how the overall profile could ultimately land? I think part of what I'm also getting at is did you show a selective group of patients here that we can take that overall view yet? Yeah. Selective sounds like cherry-picking. We didn't cherry-pick. Let me be very clear. We're really excited about the data, and we'll talk about the limitations of it. We're very excited about that data, and the reason we were so particularly excited about this early read is that it was very confirming of what one sees in the preclinical data. That's exactly what we had hoped we would see, which is the ability to dose escalate when others can't, the ability to use the integrin receptor to drive up significant muscle concentration, even at equivalent doses, and then the ability to continue to drive it up, and then the resulting knockdown that we saw in the early signs. There was really nothing in that early data that didn't completely confirm what we saw in the preclinical models. We have to be thoughtful about that. It's early data. It's just a single ascending dose. It gave us a ton of confidence, and now we need to see the MAD data. We need to see it in multiple doses, over time, in a larger cohort of patients over other dose ranges. We'll have that in the second half of this year. If that looks great, we are really heading toward the very strong possibility that we will be the best- in- class, both for DM1 and FSHD. I would say we need that. I wouldn't over-interpret the first one other than to say, oh, it's really comforting that it was exactly what the three clinical models predicted. With that second, I guess the six-month update in the second half, could you just frame what you'll show in terms of patients at each dose, biomarkers, early functional data, and what you see as relevant benchmarks? Sure. We expect to have, as you mentioned, six months of data in both FSHD and DM1, and the timing of that, we'll see. At our higher doses, as Doug mentioned, this is the MAD data. Again, we'll be looking for safety, target engagement, PK, PD. In terms of FSHD, we're targeting circulating biomarkers. Early evidence of function, six months is not a long time in FSHD, but we'll be looking at that. In DM1, we'll be looking at DMPK knockdowns, CASQ2 splicing indices, vHOT, which we can see at an earlier time point. We're excited to see this data. As Doug mentioned, having multiple doses at higher concentration, I think we're looking forward to seeing. EVOLVE, good with this data release. You've talked about starting a phase III post, maybe talk about those timelines. Let's be realistic about what we can and can't see with these heterogeneous diseases. People shouldn't over-index on some functional endpoint that relies upon degeneration. We need to be realistic. These are slowly degenerating diseases, and I think we're all so excited about seeing something as soon as possible that you want to look at something at six months that would actually take a couple years to see in the placebo arm, I think. Just to be thoughtful. That's the beauty of vHOT. A lot of complaints about vHOT. vHOT may not be a great model for predicting other declines in a short period of time. It is clearly a functional manifestation of a benefit or lack thereof, and it can be done very soon because it's not a degenerative issue. Myotonia is not degenerative in nature. Really, if you're going to focus on the future, what should you really care about? You should care about safety. You should care about the ability to dose escalate with safety. You should care about muscle concentration and knockdown and downstream splice correction. That's the big one. Then going beyond that are maybe other biomarkers that'll be fascinating. Then there's vHOT, and then I would say the rest of it's exploratory, if that makes sense. I think with respect to We're going to move as fast as possible. That's the short answer. We're going to move as fast as possible. I don't think that we have the timelines in front of us today because we've got to do more work. We heard just the other day that, I think our colleagues said it, if I'm not mistaken. No, Dyne were suggesting that they are going to go for an accelerated approval, at least in DM1. I think it's a very interesting idea, and I think it is certainly not an irrational thing for them to be thinking about with these patients. We're going to take on all of that thinking, and we're going to fashion for ourselves the fastest possible approach. That will be our goal. We need to get through some more of this. We are clearly going to start, as an example, we're going to start pivotal trials next year as soon as possible after we get the readout this year. What the exact pathway is to approval, is it the end of those pivotals? Is it an interim accelerated approval on a well-confirmed biomarker? Those are things we'll have to figure out, and then we'll have to figure out in concert with our regulatory colleagues at the FDA. Just one thing to follow up, which Doug alluded to it, but just to say it more directly. Knockdown is, to Doug's point, the most important thing to see because function will follow. You shouldn't have this toxic protein or toxic mRNA in either one, in DM1 or FSHD in your body. If you're able to knock that down most significantly, that is eventually going to lead to the best functional outcomes. Whether you're able to see it in a heterogeneous disease in a very short period of time, that's challenging. However, knowing that the best knockdown is going to lead to the best function. Not to be personal, I have family members that have DM1. If I see in the multi-ascending dose confirmation of what I see in the singular ascending dose, I'm not sitting around waiting for two years of functional data so I can confirm what is obvious from using Occam's razor and good scientific analysis. I'm sure that's where other people would be, and I would hope that's where our FDA colleagues would be. That's why I frankly think Dyne's thinking on it is innovative, and I give them kudos for that. Can you just speak to how you intend to leverage your teams and infrastructure from the PMOs in ELEVIDYS for a potential launch here? We're going to have to do a lot more. We've got a great team. There's tons of parts of our team. We are neuromuscular experts as an organization. There's going to be a ton of leverage there from a marketing perspective, promotional perspective, sales force perspective, MSL perspective, our PELs. There's going to be a ton of opportunity. It would also be a mistake to be arrogant and imagine that there's no adaptation needed or no augmentation needed. We're going to work those issues through as well, so that we are as prepared to launch in DM1 and FSHD and the unique elements of those different diseases as we were in DMD. For your PMO franchise, speak to your confidence in the applications in the context of the confirmatory studies, but also how you strategically are thinking about the runway here in the context of competitors. Yeah. I think first of all. As you all know, we've got sNDAs that have been submitted on AMONDYS and VYONDYS to transition them to traditional approval. We are at a minimum confident, based on our own analysis, that these therapies will continue to benefit patients long into the future. Just to remind you, we had signals in our confirmatory trial. We have great real-world evidence. That's the beauty of having these therapies on the market from an accelerated approval perspective for so many years. You get to actually see, not in some stilted short-term experiment, but over a long term, what's really happening here? They are significantly benefiting patients, keeping them out of a wheelchair and ambulatory and off a vent and out of emergency rooms and out of the hospital, and a great mortality benefit across all these modalities. That's EXONDYS and VYONDYS and AMONDYS. We think the data for it is really powerful. I would remind you, patients love them. Some of these patients have been on for over 10 years, and they fight to stay on them. We have a well over 95% compliance rate on those. Physicians also love them, and they're safe as heck. They have a really laudable safety profile over these many, many years. It would be patently irrational to deny these families, I can't believe a rational FDA would even be thinking along those lines. Now, with respect to competition, the primary competitor that we have will be Dyne. Dyne will have a therapy that will compete with EXONDYS next year. They're going to seek accelerated approval. In that context, I think that's rational for them to do that. I think we'll be prepared for that competition. Not focusing on them and focusing on us, we have a lot of things to say about our EXONDYS therapy. Families have been on it for a long time. We have a 10-year safety record. We have great patient services. We know how to get these kids on therapy, keep them on this therapy. We do in-home infusions, so we make life easy for them on the therapy. Of course, Dyne will have its arguments, and it'll be a very credible, I think, competitor as well. Great. Well, with that, thank you so much. Thank you. We made forward-looking statements, so please just look at our SEC filings for the risks associated with that. Sorry to end on such a..
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