Good afternoon, and welcome to the Sarepta Therapeutics' ELEVIDYS FDA accelerated approval call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question-and-answer session. To ask a question during the session, you'll need to press star one one on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star one one again. As a reminder, today's program is being recorded. At this time, I'll turn the call over to Doug Ingram, President and CEO of Sarepta Therapeutics. Please go ahead. Thank you, Victor, and thank you all for joining us for this call to discuss the approval and launch of the first and only approved gene therapy for Duchenne muscular dystrophy. Very next slide. I will be making some forward-looking statements today. Please refer to our public filings for the various risks and uncertainties that exist with statements and predictions about the future. Next slide. As you know, earlier today, the FDA approved our gene therapy, SRP-9001, to delandistrogene moxeparvovec-rokl for the treatment of Duchenne patients. The division has initially approved the therapy for Duchenne patients aged 4 through 5 years, with our ongoing phase III trial, EMBARK, acting as the confirmatory study for the approval, and if the study meets its objectives for label expansion. I will discuss later our plans to move rapidly to expand the label to treat the majority of Duchenne patients. Before I move on, I am excited we can now move out of stealth mode and announce the brand name for SRP-9001. Let me officially introduce you to ELEVIDYS, which is how I will be referring to this therapy for the remainder of today's presentation. Next slide. As the first gene therapy of its kind, the approval of ELEVIDYS is a historical milestone in the translation of genetic science to genetic medicine. It is also a monumental moment for those patients who will have access with this approval, and an important step toward our paramount and near-term goal of providing a potentially transformative therapy to the vast majority of patients living with Duchenne. Next slide, please. To contextualize the importance of ELEVIDYS, let's briefly review this ferociously degenerative disease, Duchenne. Duchenne is caused by a mutation on the gene that codes for the structural protein dystrophin. Dystrophin acts as a shock absorber that protects our muscles as we move. Boys with Duchenne do not possess this dystrophin shock absorber. Thus, they damage themselves literally every time they contract their muscles. Every hour of every day, they irreparably lose muscle and are relentlessly losing milestones. Duchenne damages them from birth and beyond. Typically, they begin to struggle in their very early years. By 8 to 11, they will struggle to walk. By their early teens, they are typically consigned to a power wheelchair. In their mid-teens, they are struggling to use their upper limbs, struggling to breathe, and perhaps on a part-time or even a full-time ventilator. Then, often by their late teens to mid-20s, this disease will kill them. This is what we're up against, a relentless, ferociously degenerative and lethal disease. ELEVIDYS is a gene therapy that works by delivering the muscle around the body, a gene cassette, that codes for a shortened but functional form of the shock-absorbing dystrophin that these boys and young men are missing. The goal is to halt the otherwise irreversible muscle damage caused by Duchenne. That is why this approval is so important for the boys who are permitted access to it today. It is why we must move as rapidly as science and our regulators will permit to expand this label and make ELEVIDYS available to all those whose muscles are being irreparably ravaged by this disease every single hour of every day. Next slide, please. With our approval in hand, we are well prepared to launch ELEVIDYS and make it successful. Sarepta is the undisputed leader in serving the Duchenne community. We have previously launched three Duchenne therapies, which, without a single price increase, have collectively achieved about a 40% compounded annual growth rate since 2016. We have the knowledge, the expertise, and operational acumen to launch and make ELEVIDYS a success, first with patients covered by this approval, and soon after, should EMBARK meet its objectives with the vast majority of Duchenne patients. Next slide. I am, of course, confident that there is no team more capable than the Sarepta team to make this launch a success. Given the evidence set for ELEVIDYS, we expect considerable demand, both from patients and their caregivers and from physicians. With that said, I do want to set some expectations about the initial launch and its ramp. First, of course, we have been preparing for a label that would serve all ambulatory patients. Our age-limited label will make the initial launch more measured than we had planned for. Second, it is important to consider the patient journey, the similarities to our oligonucleotides, but also some of the things that make gene therapy unique. Next slide. In addition to all of the early work that must be done with payers to get medical policies in place, reimbursement codes established and the like, the typical patient journey for one of our oligonucleotide therapies goes from diagnosis, to consult, to prescription, to payer authorization, infusion coordination, infusion reimbursement, and eventually to an ongoing reauthorization process. Next slide, please. For gene therapy, one has nearly all of these steps, excepting reauthorization, but then we must add to this the unique aspects for a one-time gene therapy, including initially the unique aspects of the distribution model, then referral to a gene therapy infusion site, screening for neutralizing antibodies, site procurement, and post-therapy monitoring. You will recall that for EXONDYS 51, for example, it took about 3-4 months before we began to see a significant launch ramp, and then we had a very substantial ramp in treatment and in revenue. This will be the same for ELEVIDYS, but it will be attenuated, first by the size of the population and then by another few months for the additional gene therapy steps that must be executed. We will update the investment community on launch progress on upcoming earnings calls. We do not intend to provide sales guidance for ELEVIDYS at this point, given we are at the beginning of a nearly one-of-a-kind launch. To set expectations, as we have done with our prior therapies, we intend to reference net sales as the external metric for performance rather than things like patient numbers and the like. As relates to our currently approved therapies, the launch of ELEVIDYS should have some cannibalizing impact on sales. However, we anticipate it will be modest in 2023. We remain comfortable guiding to $925 million for full year 2023 net product revenue from our three approved oligonucleotide therapies. Next slide. I have said many times that the cost of the healthcare system for ELEVIDYS will be much lower than the value that ELEVIDYS will bring to patients living with Duchenne muscular dystrophy. Let us review that now. Sponsors' pricing of their therapies in relation to a cost-effectiveness model can sometimes be opaque. We have gone much further than most, not merely by referencing a cost-effectiveness analysis, but by publishing the ELEVIDYS cost-effectiveness analysis in a highly respected peer-reviewed journal. On May 26th of this year, the Journal of Market Access and Health Policy published the article "Assessing the Value of Dystrophin moxeparvovec, SRP-9001 Gene Therapy in Patients with Duchenne Muscular Dystrophy in the United States," a detailed cost-effectiveness model for SRP-9001, now, as you know, known as ELEVIDYS. The published cost-effectiveness analysis employed an established pharmacoeconomic model to project long-term health outcomes by extrapolating clinical trial data, leveraging clinical and health economic expertise, and employing peer-reviewed scientific literature to assess the value of ELEVIDYS. The evaluation predicts that compared to corticosteroids alone, treatment with ELEVIDYS will add 26.4 undiscounted life years and 10 discounted life years gained to individuals with Duchenne. The publication concludes that ELEVIDYS is cost-effectiveness at a price, depending upon various assumptions, of anywhere between $5 million and as much as $13 million. Next slide. To avoid any debate that might delay access, the cost-effectiveness model used is a more traditional approach that looks only at the cost to the healthcare system without all of the adjustments necessary to capture the full value of a therapy like ELEVIDYS to society. On this slide, you will see reference to the broader value to society that is not captured in traditional cost-effectiveness tools. For instance, the model doesn't capture the lost income for a Duchenne patient, which over the life of a patient is approximately $1.91 million. It doesn't account for the impact on the lives and livelihoods of caregivers. It doesn't account for the risk-based investments required to develop a novel therapy like ELEVIDYS. It doesn't account for the impact an approval will have on the innovation ecosystem and the future benefits that will derive from compounding innovation. Next slide. All of which is to say that using a relatively conservative approach, ELEVIDYS would be cost effective in a range of around $5 million-$13 million. As we have said, our approach is to ensure that the cost to the healthcare system is less than the potential benefits of ELEVIDYS. We have set the wholesale acquisition cost or gross price of ELEVIDYS at $3.2 million. Remember, this is the gross price, not the actual price. Nearly all infusions will be subject to a statutory discount for Medicaid or 340B discounts. With distribution and other discounts, you should be modeling a gross to net adjustment in the mid-20% range. Looking at the payer budget impact and applying the gross ELEVIDYS price of $3.2 million for the approved Duchenne population, the cost per member per month would be about $0.25 at launch and at about $0.10 at steady state, or even lower when statutory discounts are applied. The per member per month costs for other therapies, particularly specialty therapies, can reach beyond $6, much higher than the estimated ELEVIDYS budget impact to payers. In short, we have achieved our goal of ensuring that the burden on the healthcare system is significantly lower than the potential benefits of ELEVIDYS, and we have done this very transparently. Next slide. Based on statutory and regulatory authority, precedent approvals, the outcome of our advisory committee, and the mechanism of action of ELEVIDYS, we had submitted and had been preparing for a label without age restrictions. The approval of ELEVIDYS, however, is initially indicated for 4 through 5-year-old patients. This is an enormously important moment for those boys who can now be treated, and it is an important milestone for the rest of the community, but our work is by no means done. We will make this launch a success. We will serve the patients available for treatment now, and at the same time, we will work rapidly to broaden the label to cover the majority of patients. The FDA has indicated to us that in addition to confirming the results of the initial BLA approval, if the readout of our next trial, EMBARK, meets its objectives, the agency intends to entertain a non-age-restricted expansion of this label. We have also been assured that this will be done with maximal speed by the FDA. Our goal is this: The last patient visit for our confirmatory trial, EMBARK, is in September of this year. As soon as we have the top line available, we will move quickly to supply it to the division, even before we have submitted our BLA supplement, so the division has an opportunity to begin its review immediately. Our goal is to submit a BLA supplement as soon as possible post-EMBARK and to expand the label to remove any restrictions or restrictions on the basis of ambulatory assessment, consistent with all past Duchenne therapy approvals. On the topic of non-ambulatory patients, I am happy to announce that we have commenced our non-ambulatory study, ENVISION, or Study 303, and we are dosing now. This is important as it will provide us with additional safety and expression data to support the expansion of the label when EMBARK reads out, and it does support ex-US approvals as well. Next slide. Now, even with an expanded label, we would still be excluding those 13.9% of patients who will screen out for preexisting antibodies. In addition to ENVISION, we will shortly commence multiple trials to explore the clearance of preexisting antibodies. Our goal, if successful, and if the evidence supports it, is to expand the label to treat as much as 95% of the Duchenne population. Next slide. The approval of ELEVIDYS, the first gene therapy for Duchenne muscular dystrophy, marks an important milestone in our journey to serve the broad Duchenne community with transformative therapies. Many have played a crucial role in getting to this point, and we feel a deep sense of gratitude for all of their contribution. It starts with Doctors Louise Rodino-Klapac and Jerry Mendell and their teams at Nationwide Children's Hospital, who started designing, testing, redesigning, testing, and optimizing what eventually became ELEVIDYS literally 20 or so years ago. It includes all of our external advisors and our clinical investigators and their teams. It includes the unique cross-functional team of Sarepta professionals. This milestone speaks reams to the Sarepta culture, a fearless, tenacious, patient-driven, science-focused tribe of professionals who persist even in the face of resistance and have overcome countless obstacles to get to this moment. I would like to end with a very special thank you to the Duchenne community and to the courageous families who chose to participate in our studies that support this approval. Their dedication benefits the science of Duchenne and has brought hope of a brighter, longer, richer future to the broader Duchenne community. While our approval today is a monumentally important one, our work goes on. We will continue to fight for every patient with Duchenne muscular dystrophy in the U.S. and around the world, we will do so with the urgency that this fight requires. Regardless of our obstacles, we will, as we say often, drag tomorrow into today for Duchenne patients. With that, Victor, let's open the line for Q&A. Thank you. As a reminder, to ask a question, please press star one one on your telephone and wait for your name to be announced. To withdraw your question, please press star one one again. Please limit yourself to 1 question. Please stand by while we compile the Q&A roster. 1 moment for our first question. Our first question comes from the line of Tazeen Ahmad from Bank of America. Your line is open. Hi, guys. Good afternoon, congrats on this initial launch. My 1 question for you is, just trying to understand how long you think it'll take before insurance companies start to reimburse? I think that's probably the number 1 inbound question I've been getting. Just given that EMBARK is so close to reading out, why would payers start to pay now versus wanting to wait to see what the results of that pivotal study show? Thanks. You know, thank you very much for your question, Cristina, and thank you for your statement of congratulations. We've had very good conversations with payers. In fact, we've had conversations with payers covering more than 200 million lives already, and I think those conversations are going quite well. I think the work that we've done to support the pricing of this therapy is potentially a blueprint for others. I think most conversations are going well. We're very confident about reimbursement, and I don't think it would be appropriate for patients to wait. In fact, I think there's a compelling need, given the age restrictions in the initial label, to get patients, you know, dosed as soon as possible, so they don't age out of the label. With that said, I do wanna make clear, really unrelated to payers', you know, appetite for reimbursement, that there is going to be a process that we have to go through to get policies finalized and agreements finalized and the like. You know, I think there will be, before we see a significant ramp, you know, you ought to be looking at a few months. As relates specifically to the, you know, payer attitudes, I think we've done an enormous amount of work over really the last few years to put ourselves in a position to have very constructive discussions with payers. So far, the dialogue that we've had has been very positive. Dallan, is there anything I missed in that? No, not at all. The dialogue has been positive. We've been engaging with commercial and Medicaid payers to ensure they're prepared for this launch. Our objective is to align these payer policies with the FDA indication. We achieve this by engaging with payers to provide the clinical evidence supporting the treatment. It's going well, and we'll continue to drive to support patient access as rapidly as possible. Just to clarify, you don't expect to need to wait for EMBARK to get reimbursed? That will not be the rate limiter. There are a lot of, you know, procedural issues we need to go through and policies in place. There's a natural order to these things. I don't imagine that we're gonna get significant resistance with the idea that we ought to be waiting for EMBARK to put a kid on the therapy. I think that... Frankly, you know, Cristina, we've never had a hint of that in our discussions with payers, and it wouldn't be appropriate. These kids need therapy today. One of the things I said in my prepared remarks was that, as we all know, with respect to Duchenne muscular dystrophy, you know, time is muscle, as we've heard others say. We've got to get these kids dosed in this therapy, the families as fast as we can. First, with the labeled indication and then as fast as we can, post-EMBARK, broadening this label so that other patients can benefit from ELEVIDYS. Thank you. One moment for our next question. Our next question will come from the line of Brian Abrahams from RBC Capital Markets. Your line is open. Hi there. Good afternoon. Congratulations, and thanks for taking my question. Based on this label and your most recent regulatory discussions, I'm wondering, what do you believe you'll need to show in EMBARK, particularly in the 6 to 7-year-old patients there, in order to get a broad age, agnostic label? Does the trial just need to hit statistical significance or and/or show strong trends overall? Do you need to show a specific type of trend within the 6 to 7-year-old population, statistical significance in that age group? Thanks very much. Yeah, thank you very much for that question, Brian. First, let me say, I think the standard that the FDA uses to look at this data is the totality of the evidence. I think, first and foremost, once EMBARK reads out, the standard for the FDA is to look at the totality of evidence to determine if it's confirmed the therapy and, you know, is it appropriate to expand the label to get rid of the age limitation that hasn't been present, by the way, in any other approved Duchenne therapy. With that said, I'll be clear about our expectations in the study itself, which is a slightly different question about what the standard for approval is. The standard for approval is clearly the totality of evidence. What do we expect out of the trial? We are very confident that EMBARK is powered well. We powered EMBARK to show a statistically significant and clinically meaningful benefit on our primary endpoint functionally, which is NSAA. We made some assumptions around the powering at the time that EMBARK was studied, and we've done additional studies since then and have additional insight. That additional insight actually makes us even more confident in our conviction around the outcome of EMBARK. While the broad standard for the review is totality of evidence, we're very excited for the results of EMBARK, and we have powered that study to see statistical significance on NSAA. Thank you. One moment for our next question. Our next question will come from the line of Colin Bristow from UBS. Your line is open. Hey, good afternoon, and, you know, a big congratulations on such a landmark approval. I, first question is, what is your expectation for the average time it's gonna take for a patient to receive SRP-9001 after it's prescribed? I saw the patient journey. I'm not sure if I heard that specific timeline. Also, as we think about sort of exon skippers, what's your expectation around their use and reimbursement in patients who've received SRP-9001? Thanks. Yeah, let me answer the second question first, and then I'm gonna turn it over to Dallan for the first. As relates to post-infusion, continuing use of an exon skipping therapy, like EXONDYS or VYONDYS and AMONDYS 45, I think the working assumption right now is there's gonna be significant cannibalization of that therapy. I think that, you know, as we've said many times, folks ought to model that. As it relates to 2023 in particular, I think that the cannibalization will be quite modest. Therefore, I think, you know, before our previous guidance has been greater than $925 million, while there will be some modest cannibalization, we're still very comfortable with the guidance that is at $925 million. It's a slight change in guidance, but we are comfortable with that. Now, with respect to the length of time, Dallan, there's two answers to this question. One is just getting up and running and all of the policy-related issues that have to occur before the infusions occur and the ramp occurs. I think the specific question that Colin is asking is, assuming steady state, you know, post that those early days, what's the typical time between a start form and an infusion, if we have a view on that? Thanks, Doug. As you say, it'll be months to get the policies in place, and it'll be case by case until that is the case. As we get to steady state, we'll be looking to, especially because of the narrow age range, we will be looking to get that time as short as possible. It's very early days in the launch now to really lean in on an exact time. We'll keep you updated as we go. You know, you can look to the PMO launches as initial guidance, but obviously, in this narrower patient population, we're gonna have to try to find ways to get it faster. Thank you. One moment for our next question. Our next question comes from the line of Gena Wang from Barclays. Your line is open. Thank you. Also congrats on the approval. I will also ask one question, one more question regarding the payer discussion. Doug, you lay out the patient journey. When we look through, I think at the beginning part, most of these have been done. Mainly we'll be wondering, you know, regarding the payer part, would that be pay for performance? Would that, you know, would that be any possibility for pay for performance? If that's the case, would that be delaying the patient receiving the drug due to the more complex discussion with the payers? Yeah, thank you very much for that question, Gena, and thank you for your statement of congratulations. Duchenne, and particularly with a, with a narrower population and a narrower label, and considering that Duchenne is a heterogeneous, degenerative disease, looking at some of the recent outcome-based agreements, isn't something that's likely or possible for ELEVIDYS. I think the payers, particularly the sophisticated payers, who, you know, through our engagement over the last six years, who know Duchenne well, I think generally understand that and expect that. We have some innovative concepts that we're working on with payers. They would be different than an outcome-based approach, but in any event, none of that will, in any way, delay or slow down a child's ability to get a therapy. I think it's gonna be generally straightforward, with the exception that you've got to get policies in place and contracts finalized. The good news is we haven't been sitting back waiting for this approval, and then we're gonna jump into action. We started this process. I mean, I'm not exaggerating when I think we started our discussions with payers in literally mid-2018. Of course, as we've moved through this journey, as clinical development has progressed and as data has come in, those discussions have become more and more concrete. I think very recently, Dallan's team has had conversations with payers that I think, you know, Dalen will correct me if I've got these numbers wrong, but I think it's over 220 million lives in the United States, covered by the payers that we've had discussions with. All of that has to translate into some more work and some policies in place, but I think we're in very good, we're in a very good place to complete those discussions and get kids on therapy. To Dallas' point, while we keep speaking, you know, cautiously about the early days of the launch, I do want to be clear, we've got to move rapidly, because one of the things that we need to assure doesn't happen as often as it might otherwise happen, is that kids who are amenable to getting this therapy and are within the indication, you know, age out of this indication and are denied therapy because of the labeled indication itself. The team's gonna work really hard and as fast as possible with that. Again, you know, I mean, I will say this at the risk of being immodest, there's no team better prepared to work with payers and to bring this therapy to Duchenne patients than this team. I think this team, Dallas' team, our medical affairs organization, our distribution folks, have proven time and time again over the last 6 years that they know how to work with payers, get access, and deliver the therapies to kids that need it. Thank you. One moment for our next question. Our next question will come from the line of Judah Frommer from Credit Suisse. Your line is open. Hi. Congratulations as well, and thanks for taking the question. Another one on kind of broad timelines here. You mentioned helping FDA move as quickly as possible, once you have EMBARK data, do you have a sense for how quickly you might be able to achieve label expansion, post BLA supplement submission with positive EMBARK data, and then whether you'd have a similar multi-month delay between getting that expanded label and administering drug to those older patients? Yeah. The first answer is, the good news is that EMBARK last patient, last visit, will be in September of this year. Our goal, our aspiration, and I think it's a very, you know, it's a reasonable one, is that we would have this label expanded in the first half of next year, assuming that EMBARK reads out positively, as we anticipate that it will, given all of our analyses. In the, you know, we think certainly in the first half of next year, we can expand the label. As I've said in my prepared remarks, our goal is to expand it, to have no age limitation, and the FDA has confirmed that that is an appropriate goal and to have no reference to ambulatory status. You know, the good news is, from a precedent perspective, none of the Duchenne therapies that have been approved to date, to the best of my knowledge, have had those kinds of restrictions, so I think it's very consistent with precedent. As it relates to the next wave of launch, when we have a broader label, you know, I'm sure we'll try to dampen down expectations at that moment, but the fact is, a lot of the work we're doing right now will inure to the benefit, not only of the initial launch with our four to five-year-old boys, but to the broader patient population as well. The policies and all of that work is gonna benefit both of these launches, and so we should be able to move even faster with our broader label, assuming that it all works out, we get that early next year. Thank you. One moment for our next question. Our next question comes from the line of Gil Blum from Needham. Your line is open. Good afternoon, everyone, and allow me to also add my congratulations. Maybe a bit of a hypothetical. Do you expect any pushback from payers on longer-term reimbursement if patients who are receiving the gene therapy will require further treatment, let's say, with the next exon-skipping drug? Thank you. Well, I think that, you know, I don't think that is going to be a significant discussion at launch because I think the working assumption is that children that have opportunity to get ELEVIDYS are gonna result in significant cannibalization of PMOs. I don't think that that's gonna be any kind of pushback on the use of ELEVIDYS and the reimbursement and access for ELEVIDYS. I think that the idea of a combination use of ELEVIDYS and then followed up with a PMO, I think would require an enormous amount of discussion with payers. That's why, you know, as I've said in earlier, I think, you know, you ought to plan for over time, significant cannibalization of PMO use with ELEVIDYS. Again, I know I'm repeating myself, but just to make very clear from a 2023 perspective, there will be some modest cannibalization with the success of ELEVIDYS, but it will be modest. As I've said, our guidance for the year is $925 million, and we're very comfortable with that right now. That, just to be completely transparent so everyone understands, that's for our three approved oligonucleotide therapies alone. That's EXONDYS, AMONDYS, and VYONDYS 53. That does not include any revenue, net sales associated with ELEVIDYS. Thank you. One moment for our next question. Our next question comes from the line of Neena Bitritto-Garg from Citi. Your line is open. ... Hey, guys. Thank you for taking my question. Congrats on the update. I just wanted to clarify some of the commentary on the potential for label expansion, and just confirm that it sounds like the ENVISION study would be needed for expansion into non-ambulatory patients. In ambulatory patients, I just wanted to clarify again what the bar for kind of that expansion would be, whether or not you do need to see success in both age subgroups. I guess a separate question: If you see, let's say, statistical significance just in the overall analysis, but not in the four to five-year-old subgroup, have you had a discussion with FDA on what that would imply about the label? Thanks. The goal of EMBARK, and its study, it's the placebo-controlled trial, as you know, about 125 patients, one-to-one placebo control, is to confirm the mechanism of action of ELEVIDYS, you know, which would then be applicable across ages. It's been powered to show that across the ages that are being studied, which is 4 through 7 years old. Once that mechanism of action has been established, it's pretty straightforward that we ought to get a broad label that has no age-bound restrictions in it, and that's what we expect. That has been the subject of our discussions with the FDA, I think that's consistent with our discussions with the FDA. As it relates, to go back to the, your first part of your question, as it relates to ENVISION and the need for ENVISION to support the nonambulatory part, I want to be very clear about that. What is valuable about ENVISION for expansion for nonambulatory is to get even more... We already have good exposure, for nonambulatory and older patients out of our prior studies. ENVISION gives us even more of that, and we'll take a cut of that to confirm in even more patients the expression and safety associated with ELEVIDYS in the older and nonambulatory patients. That's what we need. I don't want people to envision, to imagine that we would anticipate having to wait for the conclusion of ENVISION to get to nonambulatory patients. That is not at all our assumption. ENVISION serves a very important, it serves a number of important goals, one of which is to get from that subset of patients sufficient additional evidence to comfortably expand the label to older and nonambulatory patients, and we would have that in time to have that label expansion with our BLA supplement post-EMBARK. The other significant value out of ENVISION as well, is that it supports the label for XUS as well. Thank you. One moment for our next question. Our next question comes from the line of Ritu Baral from TD Cowen. Your line is open. Hi, guys. Let me add my congratulations on the approval. Just to rewind a bit, Doug, I just want to make sure I know how the pricing works. The label mentioned that ELEVIDYS is going to be provided in kit form, with a number of vials per patient, depending on patient weight. The $3.2 million growth that you mentioned, is that the kit price, regardless of the number of vials, or will this essentially be priced per vial? If it is a kit, by the way, if it is a kit, are we looking at a different kit pricing upon potential age expansion and the dynamics around that? Thanks. Well, Ritu, first of all, thank you for your congratulations, and thank you for this question because interestingly, we've made the presumption that folks would know this, and it does give us an opportunity. The pricing for ELEVIDYS is a single price per kit. It is not gonna vary by numbers of vials. So as additional vials may be required for, you know, heavier patients, the price would remain at $3.2 million gross, and then, of course, subject to 340B and Medicaid discounts and other distribution-related discounts that are in the, as I said, in the mid-20% range. As it stands today, we don't anticipate modifying that as the label expands. Got it. Thank you. Thank you very much. Thank you. One moment for our next question. Our next question will come from the line of Michael Ulz from Morgan Stanley. Your line is open. Hey, guys. Thanks for taking the question, and congratulations on the approval as well. Just a quick question from us in terms of the patient population here, given the narrower label, just how should we think about that initial addressable patient number in those ambulatory patients, 4-5 years old? Thanks. Thank you very much for that, Mike. You know, one of the things I said, and do want to be cautious because we are going to avoid discussing patient numbers because we don't want to use that as the metric for success. We're really going to use 1 metric. It's the one we've been using for the last 6+, 6.5 years, which is net sales themselves. I think you can get guided by the fact that, broadly speaking, there are about 400 new Duchenne patients in the U.S. this year. There are some epi literature that would suggest it might be a tad higher than that, but I think for our purposes, we use 400. That should guide you a bit on the, you know, potential addressable patient population. Thank you. One moment for our next question. Our next question will come from the line of Salveen Richter from Goldman Sachs. Your line is open. Yeah, thank you very much, and congratulations. When I look at the last three gene therapy launches, it's taken, you know, at least six months from approval to first patient getting infused here. Should we kind of think of that as really an expectation as we think about your launch? Are there any infrastructure or logistical aspects we should be aware of here? Just a second question, does EMBARK put the current accelerated approval for four to five-year-olds at risk if it were not to be successful? Well, I'll answer the second question first, and we're gonna be successful with EMBARK, and we're not particularly concerned about that. I'll be honest with you, we're very excited about that. As relates to sort of, I'd say two things that are sort of in tension with one another in answering your first part of your question. The one thing I would say is that there is, it's going to take some time to get, you know, policies in place, and, you know, codes and all of that. It's gonna take a bit of time, and that's why really before you see any real significant ramp, you ought to, you know, you ought to anticipate it's gonna be some months to really see a significant ramp. From an infrastructure perspective, there are a lot of logistical issues, but we have been working hard on them, and I will tell you that Dalen and his team have an obsessive attention to detail on these issues, so I think we're in good shape. The second thing I would say, again, at the, you know, significant risk of being immodest, is that generally speaking, I probably wouldn't use precedent approvals as a metric for how we're going to do. I think there's gonna be an enormous amount of demand, and I think, you know, obsessive attention to detail by our team to get this therapy to patients. We're already getting... I can tell you, since we've had this approval just a few hours ago, the amount of attention and focus and, you know, pressure from not just the patient community, but the physician community, has been significant. Frankly, even some positive statements from the payer community. I think, you know, I think we're gonna do quite well, but I don't wanna start creating the impression that it's not gonna take some time to really see a ramp. It's gonna take some time. Thank you. One moment for our next question. Our next question will come from the line of Anupam Rama from JP Morgan. Your line is open. Hey, guys. Thanks so much for taking the question, and congrats on the approval. Maybe following up on the last question, where are you in terms of, like, site activation and sites being ready to administer SRP-9001, and obviously work through all of those logistical things that you guys have been talking about on the call? Thanks so much. Yeah, I'm gonna turn this over to Dalen. Let me say a couple things. We've been working on site activation for a long time now. Probably, you know, a lot of planning before about a year ago, and then really working into working with these sites to get them up and running. There are a few things that you have to wait to do until you get the approval. Now that we have it, those last few things will get done. Just to remind you, to kind of contextualize what the sort of site activation and site readiness, remember a couple things. One, know that the vast majority of sites that will be infusing ELEVIDYS have significant experience because of Zolgensma, so we really do benefit from the prior approval of Zolgensma. Know with respect to Duchenne muscular dystrophy, about 50 total sites already cover 80% of the Duchenne population. Finally, remember that, you know, we had, until very recently, been planning for a very broad label, and all of our site activation and site work was on that basis. We, you know, we're in a particularly good place right now. With that, Dalen, do you want to kind of provide any of the numbers behind any of that? Yeah. Thank you, Doug. Yeah, those 50 sites and the 80%, covering 80% of the population are key ones. Anupam, thanks for the question. In the last several months, we've actively engaged with well over 100 HCPs across this site network and who do intend to dose patients with ELEVIDYS. We have, as we sit here today, post PDUFA site activation and label education meetings set up with nearly all of those 50 sites. You know, we're gonna be training the team on the final label tonight and tomorrow. We already have 5 of those site activation visits Friday. We'll already be doing this on Friday. We have another several scheduled on Monday. The team is, as Doug said, from the moment that they got the green light, have started moving forward. Thank you. One moment for our next question. Our next question will come from the line of Kristen Kluska from Cantor Fitzgerald. Your line is open. Hi, everyone. Congratulations to the entire Sarepta team. Can you remind us what other additional endpoints and color you might share with the agency after you have the data later this year? Would you also plan to continue sharing some of the longer-term data you've been collecting from the earlier conducted trials? Yeah, as it relates to the second question, of course, you know, we're always looking for appropriate medical meetings to share additional information and updates. I assume as it relates to the first question, you're really asking, what other endpoints are we looking at besides our primary endpoint? If such to do with a time test and the like, Louise, you know, You want to sort of make a few comments on some of the other endpoints that we have? Sure. I think the question was will we share with FDA, and certainly we would share all of the complete data set, including all of the secondary and exploratory, which would be the time test expression, et cetera. Yes, all of that data will be shared on an ongoing basis with the FDA. Oh, I'm sure. Yeah, of course, we're, you know, we have both an obligation and, you know, to ourselves and to the agency to share everything with them as we get it, and we certainly do all of that. We have an interesting one. We have an interesting exploratory endpoint. While I don't think it's gonna form the basis for label expansion on its own, I think it's really interesting. We've got with respect to EMBARK, some novel concepts like wearables and the like. All of that will be interesting data that we'll have over time. Thank you. One moment for our next question. Our next question will come from the line of Tim Lugo from William Blair. Your line is open. Thanks for the question, and congratulations. It's obviously a great day for the community. Can you expand a bit on your strategy for those patients with preexisting antibodies? I think there might be two strategies that you're exploring, and maybe CureDuchenne has a strategy as well. I'd just love to know when we could hear some progress for these patients. Our goal... We have two different approaches. No, let me say, I, the great news is I think a lot of folks are looking at ways to knock down preexisting antibodies. I think a lot of folks are looking at ways to empower redosing as well. I'm excited about the, all of that. We are also very excited about it as well, and there's two approaches that we are gonna take with the goal of starting both of these studies this year. One is in partnership with a company called Hansa in Europe, the use of Imlifidase to cleave preexisting antibodies to empower redosing. The second is to use apheresis to explore the ability to clear antibodies and then allow for redosing. We have really very encouraging preclinical data that gives us some hope around that. So we're gonna get going on that because this is an extraordinarily important issue. You know, about 13.9% of children with Duchenne muscular dystrophy will screen out of our therapy because of the preexisting antibodies, which that's our best estimate today. While that is a, you know, very low percentage relative to other serotypes and other AAV capsids, it's still too high. In fact, I think I've said this before, I've had parents, more than one parent say to me that the second worst day in their life was hearing that their child screened out of the ability to get ELEVIDYS. The first being, of course, the original diagnosis. We're gonna get on that. We're gonna start those trials this year, and then if they're successful, we're gonna move as fast as we can to get that in the labels and to do so safely so that patients that are NAB positive can get this therapy. Of course, our goal with all of this, if we're ultimately successful, is to extend this therapy to as many as, you know, 95% of kids with Duchenne, and we want to do that as fast as we can. Thank you. One moment for our next question. Our next question will come from the line of Eka Gigari from Oppenheimer. Your line is open. Hello, this is Eka, on for Hartaj Singh today, and thank you for taking our question, and congratulations to Sarepta team, as well as the Duchenne community for this initial milestone. We have a question on your competition. Can you just talk about your confidence level in ELEVIDYS launch in the context of the competitors pivotal trial in Duchenne that is going to be reading out in late 2023 or early 2024, at the time when you are going to be in the middle of the launch and also potentially looking at broadening the addressable patient population? Thank you. Yeah, I'm gonna try not to get excessively competitive, so let me just say we are very confident, if you don't. We're enormously confident. Our. We have a very, very significant competitor right now, and that is Duchenne muscular dystrophy. While we're enormously excited about this launch, we need to move as fast as possible to confirm things through EMBARK and get this therapy to more kids before more damage is done. That's our razor-focused issue right now. To be honest, you know, competition, you know, commercial competition is an important one because it first of all, it's great for patients. It fuels us to move fast. As it sits right now, the biggest issues in front of us right now are not other organizations, but really is, you know, a fight against time, trying to get this therapy to kids with Duchenne muscular dystrophy before more damage is done. In the broadest of sense, let me just say, we're confident. Thank you. One moment for our next question. Our next question will come from the line of Brian Skorney from Baird. Your line is open. Hey, good afternoon, everyone. Congrats. Thanks for taking my question. I know I've pushed on this a few times, offline, but wanted to maybe push again online around the powering assumptions for EMBARK. I know the panel, Chris Mullen, talked about specifics around the assumptions, but I think that actually created more confusion than color. You said before that EMBARK was designed on what you saw on Study 102. Can you give us more specifics here? Like, if EMBARK exactly replicates the 1.7 treatment mean improvement from baseline and the 0.9 point placebo mean improvement from baseline with a standard deviation of 2.7, would that result in a statistically significant effect in EMBARK? Or can you detail exactly what your assumptions are around standard deviation on these mean changes? Yeah, and that was. Sorry about that, Brian. Didn't mean to interrupt. you know, I will say, notwithstanding the fact that we were required to discuss it at the advisory committee, we've been pretty careful for competitive reasons to avoid going into a lot of detail about our powering assumptions. One of the things I will say, while the information provided at the advisory committee was accurate at the time that we did our powering assumptions, the updated data gives us additional confidence, and it actually makes it even higher powered. We could have changed those assumptions and still had a very powerful, a very well-powered study, you know, including everything from you know, standard deviation and the like. I think we feel we're in great shape right now. We felt good about the powering. We picked what was really the highest number at the time that we could choose without, you know, it being at risk of overpowering the study. It's only improved since then. Of the various numbers we could improve, we picked 120. We've actually over-enrolled to about 125 patients right now. We feel like we're in great shape, and I think the study has performed very well, at least from an enrollment perspective. The last patient, last visit will be in September, then we'll rapidly unblind that and have that data out, both to the FDA. We're gonna give that to the FDA as soon as we have it QC'd. We're gonna give it to the FDA. Our goal is to give it to the FDA before we even are able to provide a compiled BLA supplement, so that to the extent the FDA has the appetite for it and a commitment to patients, they'll start that review as soon as possible. Then, we'll submit the BLA supplement, and then, you know, hopefully, as early as possible next year, we'll expand the label. That's sort of where we are right now. We haven't given a lot more detail on powering assumptions beyond that. Is there anything else you'd like to say on that, Louise, or I hit the high points? You captured it well. Thank you. One moment for our next question. Our next question will come from the line of Joseph Schwartz, from Leerink Partners. Your line is open. Thanks very much. Hats off to everyone at Sarepta. Doug, this is cause for applause. I was wondering, oftentimes we see trials not result in a clear win or loss, but something in between with divergent results in different subgroups. I was wondering, what happens if EMBARK isn't a clear win or there's, you know, different results in the two different subgroups? Is your statistical analysis plan set up in a manner or pre-specified in a manner that is strategic at all, and you can analyze the data in a manner that maximizes the probability that EMBARK fulfills its objectives with the FDA? Yeah. Thank you for your comments, too, and your congratulations. That really means a lot to the team. This has been a lot of hard work and tenacity by a lot of folks, so I really do appreciate that. I mean, again, let me say, I mean, we're very confident in the powering of our study and the outcome. Obviously, in the end, you know, the goal is to look at the totality of evidence. We'll have to see what that looks like when we see it. I mean, I do think we've been strategic in the way we've powered this study. We, you know, as you know, one of the things that, you know, there are numbers of different ways that you go into statistical analysis plans. I think we have managed our statistics in a way I think that maximizes the potential for success, and that is by powering the study to see a statistically significant and clinically meaningful effect across the entire patient population. You know, if we started dividing that up in smaller segments, you know, you might lose power. We've got as our primary endpoint, statistical significance across the, you know, this entire patient population. With, when I say entire patient population, I do want to remind you that it is a very, it is a much narrower patient population than the patients we're going to serve. This isn't as if we've taken patients between the age of 4 and 12, and we're looking across that group. This is a, you know, fairly tight range of, four through seven-year-olds, and so, we feel very good about it. Thank you. One moment for our next question. Our next question will come from the line of Danielle Brill from Raymond James. Your line is open. Hi, guys. Good afternoon. Allow me to extend my congrats as well, for getting SRP-9001 across the finish line. I just wanted to clarify what proportion of the population has mutations in exons B and 9. Thank you. Yeah, thank you for that. Something I should comment on. As you know, we had at the commencement of study of EMBARK, what we've always called 301 before that, we had conservatively excluded patients, you know, any patient that had mutations in 1 through 17. We've done another updated study to narrow those mutations into where the issues that appear of real risk, and that's, as you know, contraindicated in patients that have any mutation that covers either 8 or 9 or 8 and 9. Which is, you know, it's unfortunate that any patients get left behind, but that's probably 5% or so of patients at most. It is a fairly small percentage of patients that are subject to that contraindication. One moment for our next question. Our next question comes line of Gavin Clark-Gartner from Evercore ISI. Your line is open. Hey, congrats on the approval, and thanks for taking my one question. For your approved HMO products, these have broad labels, whereas many of the large payer policies today, they have restrictions based on age and ambulation in the clinical trials. I was just hoping you could describe your historical ability to secure access to these products outside of the published prior authorization criteria. Yeah. Let me divide this up into two answers because I want to be very clear. I don't want to be misread here. There's one question about whether you know, the extent to which you have a narrower payer policy than the approved label. I'll answer that. That's, I think, the question you're asking, and I'll answer that question. There's another concept, which is the extent to which you can get patients, you know, treatment to patients that are outside of the label. Let me answer that second first. I think this is the question you're asking, I want to be very clear about it. We will not promote or market our therapy outside the label, nor do I think that that's going to be a significant issue in any event, because I think payers are going to restrict themselves to the labeled indication itself. One should envision that that's the label we have is going to be the label that we're going to, you know, market and promote and serve, and that should be the. Your assumption should be that group. Let me answer a different question, which is, to the extent that a payer had a restriction that was more restrictive than the label itself, what has the success rate then of this team in working with those payers to get therapy to kids that are within the label, but outside of the payer policy? The answer is extraordinarily well. They've done very, very well. As an example, you'll recall when EXONDYS was approved back in late 2016, given what was, it turns out, with the benefit of hindsight, an enormously unnecessary amount of controversy with that approval, there were large payers, Anthem being one, that had issued a policy that restricted the use of EXONDYS at all in their patients. By the end of 2017, I think all of the patients that were amenable to that therapy were on therapy, and that was through working with Anthem, notwithstanding their policy and then, you know, working medical affairs group to medical affairs group to show them the data and justify it. This team, I will say, is well prepared to work with payers to ensure that, to the fullest extent permitted by the label and appropriate kids with Duchenne muscular dystrophy are going to have access to ELEVIDYS. Initially, that means kids in the 4 to 5-year-old range. Then as we expand the label, and if we're successful in doing that, it will mean the broader patient population. This team knows how to work with payers. Thank you. One moment for our last question. Our last question comes from the line of Uy Ear from Mizuho Securities. Your line is open. Hi, everyone, thanks for squeezing me in. I'd like to add my congratulations. Could you elaborate on the timeline for the nonambulatory potential approval? I guess it, if it gets approved, I thought I heard Doug mention that you're going to submit the data from the ENVISION with the sBLA along with the EMBARK data. Is that correct? Thanks. Yeah, thank you for your question, and thank you for your congratulations. yes, so what we're gonna do is we're gonna look to a subset of the ENVISION patients, for both safety and expression. We'll add that to what we already have from patients that we've dosed in other studies that are older and nonambulatory, and that would support, from our perspective, the expansion of this label beyond the four to five-year-olds, to all patients without restriction on age and without restriction on ambulatory status. That would all occur at the same time. Our goal is the EMBARK will read out, you know, hopefully shortly after the last patient, last visit, sometime in the, toward the back half of the year. We'll submit a BLA, very early in 2024, then hopefully very shortly thereafter, we would have a broad label that would be unrestricted both on ambulatory status and age. I will note once again that that would be consistent with every other approval for Duchenne muscular dystrophy. We have a lot of conviction around that. Thank you for your question. Thank you. I will now like to turn the conference back to Doug Ingram for any closing remarks. Oh, thank you very much. Thank you all for your questions this evening and for spending time with us. I just wanna say once again, thank you for the community, for supporting us. Thank you for the broader team, both externally and internally, at Sarepta, that have been razor-focused on getting to this moment. At this time, this has been an enormous amount of work. I'm just enormously fortunate to have such an extraordinary ecosystem. We're also extraordinarily fortunate that the science has broken the right way for what I believe to be an unbelievably important therapy for the Duchenne community, ELEVIDYS. With that said, I do wanna be very clear about something, that I think, you know, we are going to celebrate this moment because this is an enormous milestone for Sarepta, for the Duchenne community, and for the patients that get access to this therapy now. However, we have a lot of work to do. We are not gonna you know, stand on our laurels. We are not gonna slow down. We have a ton to do right now. We're gonna make this launch a success. We're gonna serve the patients that are in front of us today and bring them a better life. We are going to work to get this label expanded with the success of EMBARK, having last patient, last vision in September. We're gonna expand this label as soon as we are able to, and when the science supports that, so that we can bring this therapy to patients first in the United States and then around the world, who have Duchenne muscular dystrophy. I look forward to updating you all across the course of this year as we continue to execute. We will, as I said at the beginning, I'll say at the end, we're gonna fight for patients with the ferocity and the urgency that this fight requires. Thank you all very much for all of your support. With that, this concludes today's conference call. Thank you for participating. You may now disconnect. Everyone, have a great day.