All right. Good morning, everyone, and thanks for joining us at the Morgan Stanley Global Healthcare Conference. I am Mike Ulz, one of the biotech analysts here, and it is my pleasure to introduce the team from Sarepta Therapeutics. To my left, Michael Severino, CEO, and to his left is James Richardson, the CMO. Just a reminder, the format for today is a fireside chat, but if anyone in the audience has a question, please raise your hand and we can try and address it in our discussion. Before we get started, I just need to read a quick disclaimer. For important disclosures, please see the Morgan Stanley Research Disclosure website at www.morganstanley.com/researchdisclosures. If you have any questions, please reach out to your Morgan Stanley sales representative. With that, I will turn it over to Michael to make some introductory comments, and then we can hop into Q&A. All right. Well, thank you, Mike. It is a pleasure to be here with you all today. James and I are happy to tell you a bit about what is going on at Sarepta. I have just recently joined Sarepta. I have been here going on 2 months now, but it has been a very exciting and very rewarding 2 months. I think Sarepta has a story that is very unusual, and I think we can actually say unique, even though that word is often overused in our industry, because it is a company that has a cadre of marketed products that are serving patients today and have been doing so for many years, that offer us a number of benefits. First of all, they offer patient benefits, which is what is most important in our industry, but they also generate revenue that gives us a very strong and stable financial position and a very strong balance sheet. We can use that strong financial position to drive a very innovative and very promising pipeline that has the potential to expand our therapeutic footprint to drive growth, we believe, in the near term and long term. I am sure we will talk about that pipeline and everything that we are doing, for example, in the siRNA space. As I said, this is a setup that you do not find in our industry. As someone who has spent more than 25 years discovering, developing, and commercializing therapeutics, when I looked at this opportunity, I was compelled by the interplay between those marketed products and a pipeline that I think holds tremendous scientific progress and can make a real difference for patients. I look forward to speaking to you all a little bit more about that as our session continues today. Yeah. Great. Thanks for that introduction, Michael. I guess just, as you mentioned, you've only been there for about 2 months, and maybe just talk us through your strategy near term, what's the focus, and then maybe just how that might evolve longer term. Yeah. So near term, there are a number of important areas of focus. First of all, we want to continue to support and drive the business for our marketed products. As I mentioned, we have a cadre of marketed products, ELEVIDYS, people obviously are very familiar with, and our PMO franchise, both very important to the company. We recognized that there was some disruption, particularly on the ELEVIDYS side in 2025. Whenever you have a disruption caused by safety events like those that were disclosed in 2025, attention shifts very considerably towards safety, towards risk. Yeah. It's important in our industry and our business and for patients to focus on benefit risk. ELEVIDYS clearly demonstrates a strong benefit for patients. You see that in the initial registrational trials. You see it in the long-term studies, and we released 3-year data earlier this year that I think strongly makes that case for long-term benefit. So getting that conversation shifted back towards benefit risk is absolutely essential because we need to keep in mind that unfortunately, the condition that we're treating with ELEVIDYS is progressive, debilitating, and unfortunately life-shortening. Yeah. And so one needs to look at benefit risk and the benefit that is delivered. We focused on recentering that conversation. We have expanded our commercial footprint. We have expanded our medical support for the product, and I think we are in that process of recentering, stabilizing, and continuing to support that business. Driving that business as well as driving our PMO franchise is important to that longer-term strategy that I described. But equally importantly, continuing to drive the pipeline is going to be absolutely essential. We have been serving DMD patients for a number of years, and we want to continue to serve DMD patients in the near and long term. But we want to expand our footprint, as I have said, and we have a very promising siRNA pipeline. I dug in deeply to the data behind our siRNA pipeline before I decided to join. I think it is truly differentiated. I think the delivery technology that we have available to us drives very high muscle concentrations for programs where that is relevant and also gives us the opportunity to penetrate deep brain nuclei in the CNS, for example, in our Huntington's program. We have extremely potent constructs that can knock down genes effectively. In our space, where target validation is not an issue, in other words, we know what causes these diseases, the ability to deliver a therapeutic to the cell type of interest, to do it with high efficiency, to do it with good safety profile, and to efficiently, in the case of the conditions we are talking about, knock down the gene very efficiently, that is what is crucial to driving long-term success. Those sorts of data translate into the clinic and translate into late-stage development with a much higher probability than most areas of drug discovery and development. So continuing to focus on that pipeline, driving it forward, driving through the near-term readouts, which we have coming later on this year and beyond as we move those programs we believe into registrational studies, is going to be a core focus If you look at those two elements of the strategy, supporting the ongoing business and driving the pipeline, we believe those can create a significant amount of value. Yep. Everybody believes that they are undervalued, but I think we can make a strong case based on our cash flows, based on our pipeline, that there is tremendous untapped potential in this company. Those two areas of focus are what will unlock that in the near term and then give us the license in the longer term to continue to think about how we grow and expand our business. Yeah. That's what we're focused on right now. Yep. Makes sense. Maybe we could focus to start a little bit more on the pipeline because there's some near-term updates as you mentioned. Maybe just if we start with SRP-1001, that's your FSHD program. Maybe talk about some of the early data you've shared this year and what it means for the program. Yeah, certainly. Earlier this year, we shared data from the single ascending dose portion of the phase I trial. The trial has two components, the single ascending dose and the multiple ascending dose components of the trial. Earlier this year, we shared single-dose data, and we saw good PK, not only systemic PK, but delivery to muscle. We saw dose proportional increases in muscle concentration. As we go up in dose in a proportional way, we get more of the therapeutic into the muscle, which is not always the case. There can often be a point of diminishing return as one escalates systemic concentrations. We saw a very good safety profile. We saw no dose-limiting toxicity. For the FSHD program, we saw very good early evidence of knockdown of the gene of interest, DUX4 knockdown in the biopsy samples. That's critical because that's what drives that condition. We're going to continue to extend on those data. We're going to have more data from that study coming later this year. We're going to have data from the multiple ascending dose portion. We'll have data from higher doses as well, both this year and even higher doses next year as that study continues. That's a program that we're very excited about. Yep. You mentioned the DUX4 knockdown. Can you maybe put that in the context of maybe what some competitors have shown? Certainly. James, do you want to take that question? Yeah, absolutely. What we showed were the DUX4 gene regulated panels. They can be expressed in a number of different ways. But we looked at both the panel used by Avidity, so the same gene panel, and then the same gene panel used previously by Fulcrum. And we have seen in excess of 90% correction within the pooled single ascending dose data versus around 40%-50% seen with the Avidity program. I think that is super exciting. I think the other thing just to highlight from a PD perspective in the second half of this year is that there is an underlying challenge in FSHD with the stochastic nature of expression that makes muscle biopsy a little more challenging from a PD perspective. So we are looking forward to showing some circulating data around DUX4 knockdown as well. That is just another compartment that balances out some of that stochastic nature you see in the muscle. When you share updated data later this year, can you maybe comment on what endpoints, what cohorts, et cetera, you might share and what do you think the focus should be? Can you say that, Mike? Yeah, you can go ahead and take that, James. Yeah. Okay. We are going to see six months out up to the 8 milligram per kilogram dose for FSHD. There is one higher dose from that in clinic that we will not be showing by this release just because of timing. I think the important thing is just to repeat what we saw at a higher dose level the first half of this year. Again, just continuing on that hypothesis that Mike laid out that you can safely dose escalate. We know that is not something that has been shown across different programs in this space. There have been dose-limiting toxicities, but we continue to see this differentiating muscle concentration. From a PD perspective, as I have said, we will look again at the DUX4 gene panels. I think as exciting new data, we will be circulating biomarkers of DUX4 correction. Yeah. And maybe just one thing to add, completely agree with everything that James has said. This is a condition where we know that more is better with respect to knockdown because DUX4 is a gene that should not be expressed outside of development. So in a normal adult or even child after the period of development, you shouldn't have DUX4 expression. And the expression of that gene is by definition, therefore, abnormal. And the DUX4 regulated genes that are a consequence of that abnormal expression are what cause the pathophysiology, the morbidity, the long-term consequences of FSHD. So if we can knock that down harder- Yeah we're quite confident that we're going to see that translate into very strong clinical results. And that's one of the things that I was referring to in my opening remarks when I say target validation is very solid in this space. And the ability to predict from either preclinical data or early clinical data what one is likely to see in later stage studies is much more solid in this area than it is in most of drug discovery and development. So it sounds like with multi-dose and higher doses, you might be able to drive that very high knockdown even higher. Is that a potential outcome here? 90-plus is very high. And so you get to the very desirable point where there's just not a lot of dynamic range left. Yep. But the ability to knock down very substantially, we believe in the long term, and to maintain that knockdown over time, we believe in the long term will translate into improved outcomes for patients. Can you talk about some of those functional or outcome measures, and when might we see data on those endpoints? Yeah, we can certainly talk about them, and I'll let James Richardson give a little bit more detail in a second. The one thing that I would say is that we will look at functional endpoints in our early-stage studies, both in FSHD and DM1. I would really caution folks who look at the data not to over-index on those functional measures early on in a drug discovery and development program because you have trials that just simply aren't large enough and aren't designed to show a difference on functional endpoints. What these studies are designed to do is to look at the chain of events from PK, systemic PK, to muscle exposure, to target engagement, and biochemical knockdown of these targets, and what is the consequence of that knockdown. When one looks at functional endpoints too early, you can essentially fool yourself by making decisions based on the variability. So we'll look at those endpoints, but I would caution folks not to over-index on them until we get into larger, later-stage trials. But James, you might want to talk about some of the endpoints that we will be looking at both now and in the future as we move forward. Absolutely. This is an evolving field, endpoint generation validation in FSHD. What I would say, as a company, we are extremely experienced in neuromuscular development and in working with neuromuscular outcomes. I feel confident that we're going to. I guess what our focus really is right now is what are the best outcomes we can get for our phase III development. I am very confident that we have the expertise in-house to use these data and use natural history data to make that decision. To be specific on that question, the endpoints we're looking at are things like reachable workspace. I think that was obviously a challenging outcome in the Fulcrum phase III. Certainly we'll give it its due diligence. I am not sure whether that's something that we're going to be persisting with long term. Then measures of strength and measures of mobility, chiefly, so Timed Up and Go, 10-meter walk/run. I think what's going to be really interesting from these data is, as Mike Severino said, not so much that we're going to really be picking out signals from noise, but we will get an idea of how responsive these endpoints are Yeah for use in a larger, more homogenous trial population. No, makes sense. Maybe we can shift gears now to SRP-1003. That's your DM1 program. Maybe just talk about, you shared some early data this year. Maybe highlight some of that and what you learned there. Yeah, certainly. Earlier this year as well, we shared data from the single ascending dose portion of that study. That study, while there are subtle differences, conceptually has a similar design to what I described before, which is there's an integrated single ascending dose and multiple ascending dose portion. Earlier this year, we showed data that had many of the same themes that we talked about in FSHD: good PK, good relationship between systemic PK and muscle concentration, the ability to see dose proportional increases in muscle exposure with a good safety profile, so with no dose-limiting toxicities identified. We showed very early evidence of target engagement. We will have the multiple ascending dose portion of that study reading out later this year as well. That will continue to extend those observations and give us the ability to extend observations around target engagement, target knockdown, and the consequences of target knockdown, the PD consequences of target knockdown. Yep. Maybe you can talk about what's a good outcome on some of those endpoints, knockdown, for example, other endpoints that are important there. Yeah. Well, principle for DMPK, the gene that is responsible for myotonic dystrophy, for DM1, again, we're in a situation where more knockdown is better. Yeah. The exact specifics of how these genes are regulated are a little bit different, but still that basic theme that more knockdown is better, I think is quite clear and shown in the data. We want to be able to drive knockdown that clearly differentiates us from others. We will have more data later on this year, as I said. As I mentioned, we have even higher dose cohorts coming early next year. Making that PK/PD correlation, showing DMPK knockdown and the correlation to splicing correction. Yep. Because what the abnormal form of DMPK does is it alters the machinery that regulates splicing and gives a detrimental and sort of a fetal splicing profile in the setting where that should not be the case. That is what is responsible for the downstream consequences in DM1. Showing that chain of events will be an important part of the data that will be coming later on this year. Showing that PK/PD relationship will be important. Yeah. That is important also as we have an eye towards even higher doses that can come early next year. Yeah. It is going to be an important data set for us. Yep, makes sense. There are a lot of companies developing treatments for DM1, and Novartis actually had shared some data recently from their phase III study missed the primary endpoint, which was VHOT. Maybe just comment your thoughts there, how it impacts your thinking relative to your program, thoughts around VHOT, et cetera. Certainly. I will start, and then I will turn it over to James to provide some additional detail and color. Obviously, we are paying close attention to the space, and we are well aware of the top-line outcome of the Novartis study. One important caveat is there were not a lot of data yet, and that is typical in this space. What we know is that that study failed to hit its primary endpoint. There was also a comment made that there were favorable indicators of movement in secondary endpoints. The details of that will have to come out at a later time, probably in a scientific meeting is the way these things would typically proceed. We will learn a lot more. When we think about that outcome for that program and its potential implications for our program, we think about a couple of things. The first is, what degree of knockdown were they able to achieve? I certainly do not know within that specific study what they were able to achieve, but if you look at their earlier data, you would estimate that they are somewhere between about 30% and 40%, which is the range of knockdown that they have shown. As I mentioned, this is a condition where, with respect to knockdown, more is better. The first thing that we would look at is can we drive higher levels of knockdown with whatever dose it is that we ultimately carry forward into later-stage development, whether it is one of the two doses that we will update on later on this year, or whether it is an even higher dose that we can update on early next year. I think there is a very real opportunity for something that can deliver a greater degree of knockdown to have a larger benefit and to be able to show that benefit in late-stage studies. Because the one thing I can tell you the Novartis result doesn't do is it doesn't shake our confidence in target validation. Yeah. In so many areas of drug discovery and development, you can't say that. If you're working in immuno-oncology or some other area and there's a major failure, the first question that comes to mind is this even the right target? That's not a question that we worry about in these conditions because we know that the DMPK mutations are causal. So the question then becomes can you knock down the abnormal form to a sufficient extent? Can you do it consistently? Can you do it with a good safety profile? We're going to be focused on that PK/PD and picking a dose for later-stage trials that we believe will be truly differentiated with respect to the ability to achieve knockdown. After that, then one has to think about questions regarding study design. So was VHOT, video hand opening time, was that the right endpoint? I'm not saying it was or it wasn't, but we'll learn more when those data are released more completely. Are there other features of study design, stratification? So what was the nature of the baseline dystonia that existed in those patients? Does one need to control for that in ways that are different than was done in the Novartis trial? Those are all things that we'll learn as the data come forward. Those are all things that we can build into our program. But ultimately, we think the opportunity is very clearly there. In particular, it's very clearly there for a differentiated program that can drive maximal or near maximal levels of knockdown. Yep. James, I don't know if you want to talk more about the specifics of study design and some of those endpoints. Yeah, I think you said a lot of it really, Mike, in that clearly there's very little doubt in the fundamental biology of DM1, which then leaves the speculation on Novartis around the drug. Is it study design? From a study design perspective, I think it's quite likely that they paid a pioneer's penalty here. We did it enough times in DMD. You're using outcomes often for the first time in a trial of treatment, and you learn a lot from those studies. I think that is a distinct advantage for companies like Sarepta Therapeutics who are not yet locked down in our development plans. We continue doing a lot of work in this space. We have, as I said, a very experienced in-house team in the neuromuscular space. We have well-networked in the KOL space and have plentiful natural history data, our own in-house clinical data, all of which to build on whatever we get to understand from the Novartis study to make sure, to Mike's point, that we are picking the right primary outcome for our study and the right population to show a change in that primary outcome. I think it's too early to throw out VHOT as an endpoint. It has a lot of appeal. It clearly does show drug response on a very subjective nature. You hear patients, you hear your PIs, patients know their myotonia changes. How it responds in a larger study over a longer time period is what I think we're learning at the moment, what Novartis have probably learned in this piece. I think we need to make a decision now, what is our best primary endpoint based on all of these data and our expertise and the population in which to drive a change in that. But as said, I am for one happy to be in a position a little behind so we can learn on some of the others' experiences. Yep. Makes sense. Maybe last sort of pipeline question before we move on. Just any updates around the timing of the MAD data readouts for DM1 or FSHD, and whether those readouts will be together or separated? As we've said, we expect data readouts in the second half of this year. Yep. We're in the second half of the year. Yep. In the coming months, we'll read out both of those studies. They're independent programs. They each have their own considerations, and they each have their own unique value propositions. Our expectation is we would release each as it is ready. I think they will likely be separate data releases. We want to make sure that folks are able to look at the data and absorb those data and understand what they mean for each program individually. They also have their own unique time frames. Yep. With respect to the FSHD program, as James mentioned, we're going to be focusing on a number of circulating biomarkers, including some novel biomarkers, and there's assay validation work that we're doing to make sure that we can bring forward a very comprehensive and high-quality data set. That program may be shifted a bit back in time within that window. later on this year, but likely to be the second of the two. Okay. I think the DM1 program is likely to be the first. The exact timing is not yet determined because that's based on the progress in those programs, but we're well on track to release data from both studies in the back half of this year, as we've said. Okay. Yeah, great. We're all looking forward to those data. Maybe shifting to your commercial program, you talked a little bit about the history over the past couple of years and some of the challenges, but if we start with ELEVIDYS and maybe talk about some of the dynamics you're seeing. It sounded like you're getting to some stabilization and maybe some positive dynamics there as well. Maybe if you could talk about those. The key for ELEVIDYS is moving back to a balanced conversation around benefit-risk, as I described. A number of factors have enabled us to shift that conversation back to that balanced conversation of benefit-risk. One is just time from the events. It takes time for folks to absorb new data. There's sort of a natural arc of people's understanding of new data, like the safety data that were released last year, and an arc of their ability to sort of integrate that, together with a revolve thinking about the benefit-risk of the program. We've also released new data, long-term data, the 3-year data as I described, which helps reinforce that long-term benefit for both prescribing physicians and ultimately families, patients, and their caregivers who are making these decisions. We've put a number of initiatives in place to expand our field force, to give them the new data that are available to facilitate those conversations. Those conversations are going well. We've dramatically increased the number of physician discussions that we've had. Subjectively, those have been very balanced, with focus not only on safety but also on benefit. Those benefit-risk conversations are improving and I think becoming more centered in their focus. With that, we're seeing stabilization of demand and early positive signs, as we said on our last earnings call. Okay. Overall, I would say that's going well. We would view it as a build off of this base. Yeah. Sort of a gradual build, so we're not expecting any dramatic inflection point in any particular moment in time, whether it is later on this year or early next year. But we do see signs of stabilization and forward momentum. Yep. And that growth that you are seeing or sort of expect over time, you said, I do not want to put words in your mouth, but some growth here, and then does it sort of stabilize at some point in the future, and that becomes just the recurring revenue stream that is stable at a certain point in the future, or? We would see it as a gradual- Okay build over time- Okay as I said. That's with respect to the currently indicated population. Yeah. Right. Nothing that I've described contemplates yet- Yeah whether we return to the non-ambulatory population, and we can talk about that- Yep in a second. But with respect to the currently indicated population, we would see that gradual build over time. We certainly think there's durability in this franchise. We know that there is unmet medical need. We know that there are patients who can benefit from therapy. And so we would see long-term stabilization. We're not in a position today to give long-term guidance. Yep We do see that positive momentum, and we see ELEVIDYS being a very substantial contributor in the long term as well. Yep. As we think about what to expect the remainder of this year, should we be thinking flattish, or how should we think about the progression there? Well, as we said on our last earnings call, the first half of the year benefited from a large number of patients that were already in queue from 2025. Obviously, there were safety disruptions in 2025, and some patients dropped out, but many did not. In particular, in the short term, there may have been a delay in making treatment decisions as those data became absorbed by the community. So that bowl of patients worked its way through the first half of the year, and results in the first half of the year benefited from those patients. What we're going to see in the second half of the year is demand that was generated earlier in 2026, at a period in time before we had our commercial initiatives in place, before we had centered that benefit-risk conversation. We are expecting that to be modestly lower than the first half, but stabilizing over time. Again, we are seeing that long build off of that base. What I would caution is that there is about a 6-month journey from enrollment form to treatment, on average. The effects that we are seeing on enrollment forms, the positive momentum that we are starting to see in the marketplace, it is going to take time for that to translate into revenues. That is more something that we will see in 2027 than in the back half of this year. Great. You talked about label expansion into the non-ambulatory patients with ENDEAVOR data later this year. Maybe talk a little bit about that and your confidence in being able to expand the label. The non-ambulatory population is not currently in our label. We have a study ongoing, we have Cohort 8 of the ENDEAVOR study, which is looking at the impact of sirolimus on hepatic safety in that population. That will be an important component of future discussions around labeling for that non-ambulatory population. The hypothesis in that Cohort 8 is that sirolimus can be effective in reducing the incidence of acute liver injury in the non-ambulatory population. The specific hypothesis we built into that study is a 50% reduction in the incidence of acute liver injury, which would be quite meaningful. When you broadly decrease the incidence of acute liver injury, what you are also going to do, although it is hard to study in a clinical trial, is you are going to reduce the likelihood of those severe cases, which are the ones that we are really worried about. We are essentially looking at the base of the pyramid to determine whether we can improve hepatic safety, and that should, based on everything we know about drug discovery and development, reduce the likelihood of those more severe cases. That study is underway, so we do not have data yet. It is enrolling as we speak. We expect to have it completely enrolled this year, with 12-week data in the first quarter of next year. Those will be important data for us and important data for us to discuss with the FDA around the potential to return to the non-ambulatory population. Those data will form the centerpiece of those conversations. As we have said, we will reach out to the agency, our plan is to do that in the first quarter of next year, with data in hand to have those conversations. Those discussions will really define what the path is to a return to the non-ambulatory population. Makes sense. Maybe we can, last 2 minutes here, just the PMO business. Looks like you've got some nice stability there, but you may be facing competition next year. So maybe just talk about some of the near-term dynamics and how you think things might evolve next year. It is a durable franchise. These are products that have been on the market for a number of years. They've delivered clear patient benefit over that time period. We have extensive real-world evidence, which we've published in a number of settings, which demonstrates that. Which shows, for example, prolonged time to ambulation, decreased rate of pulmonary decline, cardiac decline, even overall survival in a real-world setting based on that published evidence. Those are things that matter to patients and to their physicians. We feel good about the benefit that those have brought long-term. We also have supported those products extensively in the marketplace in terms of establishing reimbursement pathways, helping patients navigate a very complicated reimbursement landscape. Other patient support systems, home infusion, all of those things matter as well and are contributors to that 90-plus percent adherence that we've demonstrated in the real world. We think that all matters to patients. There are programs that are moving forward in this space. In 2027, Dyne will have a PDUFA date. We welcome drug discovery and development in this space as innovators. We understand that over time, we will move into a competitive marketplace.. We think we're well positioned to compete in that marketplace. For example, all of those things that I mentioned, the long-term experience, the data that have been published, the reimbursement pathways, the patient support systems, the home infusion, which is critical for these patients to maintain them on therapy in a way that is not disruptive to their lives or their caregivers' lives. We think all of those things matter and will add stickiness to this franchise. Yeah. We think we're well positioned to compete in that longer-term marketplace. Yeah. Okay, great. Looks like we're just about out of time. Michael and James, thanks so much. Really appreciate your time today. It's been a pleasure. Thank you very much.