Good afternoon, ladies and gentlemen, and welcome to the Sarepta Therapeutics First Quarter 2022 Earnings Call. As a reminder, today's program is being recorded. At this time, I'll turn the call over to Mary Jenkins, Senior Manager, Investor Relations. Please go ahead. Thank you, Chloe, and thank you all for joining today's call. Earlier today, we released our financial results for the first quarter of 2022. The press release is available on our website at sarepta.com, and our 10-Q was filed with the Securities and Exchange Commission earlier this afternoon. Joining us today on the call are Doug Ingram, Ian Estepan, Dallan Murray, and Dr. Louise Rodino-Klapac. After our formal remarks, we'll open the call for Q&A. I'd like to note that during this call, we will be making a number of forward-looking statements. Please take a moment to review our slide on the webcast, which contains our forward-looking statements. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Actual results could materially differ from these forward-looking statements, and any such risks can materially and adversely affect the business. The results of operations and trading prices for Sarepta's common stock. For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent quarterly report on Form 10-Q filed with the SEC, as well as the company's other SEC filings. The company does not undertake any obligation to publicly update its forward-looking statements, including any financial projections provided today based on subsequent events or circumstances. Now I'll turn the call over to our President and CEO, Doug Ingram, who will provide an overview of our recent progress. Doug? Thank you, Mary. Good afternoon, everyone, and thank you all for joining Sarepta Therapeutics First Quarter 2022 Investor Conference Call. As you will have seen from our press release issued earlier today, Sarepta enjoyed yet another straight quarter of strong revenue growth for our three approved therapies. EXONDYS 51, VYONDYS 53, and AMONDYS 45. Achieving total revenues of $210.8 million and net product revenues of nearly $190 million. Our net product revenue performance represents an impressive 50% growth over the same quarter last year. Led by AMONDYS 45, each of our three therapies contributed to our outsized growth over the same quarter prior year. While I have said it many times, it does indeed bear repeating. We have achieved over five years of sustained growth with a compounded annual growth rate of nearly 40%. We've done that without a single price increase and by pricing all of our therapies at par. Said another way, our consistently strong performance comes from serving the patient community, and it reflects the value that these therapies bring to individuals living with Duchenne. Based on our performance to date, we can confirm our prior guidance of net product revenue of over $800 million for full year 2022, translating into product revenue growth of over 30% over 2021. Our Chief Commercial Officer, Dallan Murray, will provide additional color on our performance in a moment, and our CFO, Ian Estepan, will provide an update on our financials. In the first quarter, we continued to advance our large pipeline of potentially life-enhancing therapies focused on rare genetic diseases across our three platforms. As you know, that's RNA, gene therapy, and gene editing as well. As you know, we are now in pivotal trials for our lead programs in both our gene therapy and our RNA platform. With respect to our gene therapy platform, we continue to initiate sites globally and to dose EMBARK, our 120-patient pivotal trial for SRP-9001. SRP-9001 is Sarepta's gene therapy, largely agnostic to mutation, to treat Duchenne by delivering a truncated but functional version of dystrophin. EMBARK is well-powered and informed by the wealth of positive expression, safety, and functional evidence that has come from previously dosing over 80 patients across three studies. I, as I've said often, you should assume that the success in EMBARK, a well-powered phase III pivotal study, will be our path to a successful launch of SRP-9001 in the United States and around the world. As I've also confirmed in the past, we are exploring with the FDA the possibility of a more expedited BLA filing for SRP-9001. Anticipating your question, we will provide an update once those discussions are completed and will not provide additional color until that time. Again, for now at least, one should assume that EMBARK, which we'll read out next year, is our pathway to approval. As we continue enrollment of EMBARK, we are also leveraging our world-leading gene therapy platform to advance our six LGMD candidates, all of which share the same AAVrh74 vector and many of the same promoter as SRP-9001. On the RNA side, we are enrolling and dosing in MOMENTUM Part B, our pivotal trial for SRP-5051, our next-generation RNA-based PPMO for the treatment of Duchenne patients amenable to skipping exon 51. We are particularly excited to move MOMENTUM forward, given the results of Part A. In MOMENTUM Part A, we were encouraged to see 18 times greater exon skipping and eight times greater dystrophin over eteplirsen, and we saw it in half the time and at one-fifth the drug exposure. If confirmed in MOMENTUM Part B, SRP-5051 could be a profound improvement over the current standard of care. At the same time, we are progressing the preclinical work to additional PPMOs to treat a much greater percentage of the Duchenne population than we currently treat. Our Head of R&D and Chief Scientific Officer, Dr. Louise Rodino-Klapac, will provide additional color on the performance of our R&D pipeline and readouts in the first quarter in a moment. Now, I recognize that for many biotechs and for those that invest in them, the last 15 months or so have been challenging, and some biotechs have particularly struggled in response to external environment. We feel no schadenfreude for that, but Sarepta remains, on the other hand, in a very strong position despite the external environment. We continue to enjoy strong revenue growth, well on our way to eventually achieving that vaunted $1 billion in yearly revenue milestone. We entered into 2022 with over $2.1 billion on our balance sheet, and even as we have aggressively invested in our pipeline, we exited the first quarter with over $2 billion in cash. Our pipeline continues to perform, and we are in pivotal trials with potentially transformative therapies in both our RNA and our gene therapy platforms. We have one of the strongest and most operationally excellent teams in all of genetic medicine. With our success, we're not standing still, but we are growing. In addition to the expansion of our Genetic Therapies Center of Excellence in Eastern Ohio, we are building out a new 288,000 sq ft facility in Bedford, Massachusetts, where we will centralize our efforts, including RNA research and process development function, translational biology, gene therapy process development and quality control, and we're building early phase, GMP gene therapy manufacturing capacity that will help accelerate our future programs. We continue to grow and add best-in-class talent as well. In 2022, we will add another 40% to our employee base, the bulk of whom will be in research and development, regulatory affairs, clinical development, and technical operations. With that, let me turn the call over to our Head of R&D and our Chief Scientific Officer, Dr. Louise Rodino-Klapac, for an update on our research and development activities. Louise? Thanks, Doug. We are pleased with the continued progress and momentum across all of our R&D programs. I'll begin today with our late-stage clinical programs. For SRP-9001, our gene therapy in development for the treatment of Duchenne, the data presented to date continues to give us confidence in the therapy's potential to alter the trajectory of the disease, with an emphasis on improving function, quality of life, and preventing premature and early death. Driving this transformative effort is the underlying strength of our construct, combined with our deep understanding of Duchenne neuromuscular disease and our science. We have demonstrated consistency of the therapy over three trials, Study 101, Study 102, and Study 103, having dosed 85 patients across these studies. Based on these results, we would expect the treatment benefit to continue to increase over time due to the progressive nature of Duchenne. Across these studies, we have seen sustained functional improvements as compared to natural history, with the longest now in year four of follow-up. Notably, the safety profile has remained consistent across different patient ages and weights. We continue to generate data from Studies 101, 102, and 103, including two-year data from Part one of Study 102 and one-year data from Study 103. As we have mentioned on our fourth quarter call, we plan to perform an integrated analysis of the one-year data from Studies 101, 102, and 103 for all patients who received a target dose. Our plan is to share the totality of these data with regulators and present all of these results at a medical meeting later this year. In parallel, enrollment in EMBARK, or Study 301, our 120-patient global, multicenter, double-blind, placebo-controlled phase III trial evaluating commercially representative SRP-9001 material in patients with Duchenne between the ages of 4-7, remains on track. We expect to have the trial fully enrolled in the middle of the year. Now continuing with our gene therapy franchise. For SRP-9003 and our other LGMD programs, we continue to make good progress with respect to building our manufacturing process, including assay development and validation. When we're ready to test commercially represented material in a clinical trial, we will discuss the study design with OCTGT. Moving now to our RNA-based platform. For our next-generation PPMO program, SRP-5051, in development to treat individuals with Duchenne amenable to exon 51 skipping, enrollment continues on pace for Part B of the MOMENTUM study. To remind you, MOMENTUM Part B is a multi-arm global ascending dose study of SRP-5051 infused monthly, assessing dystrophin protein level in skeletal muscle following SRP-5051 treatment. As we've guided previously, we anticipate Part B to be fully enrolled in the second half of 2022. If the trial is successful, we anticipate Part B will serve as a pivotal study for SRP-5051, and we plan to seek accelerated approval. Now turning to some updates from the 2022 Muscular Dystrophy Association Annual Clinical & Scientific Conference that took place in March. We shared data from across our pipeline at the conference, which included three podium and 10 poster presentations. Notably, for the SRP-9003 program, our lead gene therapy in development for the treatment of Limb-Girdle Muscular Dystrophy Type 2E or LGMD2E, we reported new data from the two dose cohorts in study SRP-9003-101. We also shared the full results from EXPLORE DMD, a study to assess the rate of pre-existing antibodies to the AAVrh74 vector in individuals with Duchenne. For study SRP-9003-101, we presented three-year functional and two-year biopsy data from cohort one, the low dose cohort, and two-year functional and biopsy data from cohort two, the high dose cohort. From a safety standpoint, since gene therapy is only dosed one time, the adverse events that are typically observed during the first few months after dosing. Therefore, as expected, we did not observe any new safety, significant drug safety events at year three. These results continue to reinforce the favorable safety profile of therapies that use the AAVrh74 vector, which also has read through to other gene therapies administered with this vector. In terms of the durability of the therapy, the results show that the persistence of the SRP-9003 vector in transduced muscle continues to drive meaningful levels of beta-dystroglycan protein expression over time, leading to sustained improvements in functional outcomes. We are very pleased with these results because of the potential read-through to our portfolio of five other LGMD programs, which all share the same AAVrh74 vector. These data are also important for the read-through to our ongoing SRP-9001 program for Duchenne because the SRP-9003 program uses the same AAVrh74 vector, an MHCK7 promoter, and design and approach as SRP-9001. As we add to the growing body of clinical evidence for these programs, supportive, consistent safety and durability data for one program has read through to the platform. Also presented at MDA were data from EXPLORE DMD, a study assessing the rate of pre-existing antibodies to the AAVrh74 vector in individuals with Duchenne. The final results demonstrate that the majority of patients screened, 86.1%, were seronegative, meaning they have a titer of less than one to 400 for anti-AAVrh74 total binding antibodies. This low seroprevalence of antibodies against AAVrh74 supports the broad applicability of AAVrh74-based gene therapy to patients with Duchenne. The findings of this study are important for several reasons. Because pre-existing antibodies against AAV vectors can hamper the therapeutic efficacy and potentially pose a safety concern, it's imperative that we have a reliable way to pre-screen individuals before they receive gene therapy. Additionally, our goal is to treat all individuals with Duchenne. The ability to better understand the population with pre-existing antibodies allows us to target our research and development efforts to find novel ways to knock down pre-existing antibodies so that these individuals could be eligible to receive therapy in the future. Further, our comprehensive approach of measuring total binding antibodies may help improve the safety and efficacy of AAV-based gene transfer therapy. The results of the seroprevalence study have also been observed in the trial, including for the EMBARK study for SRP-9001. We believe our observed low screen-out rate will allow more patients to be eligible to receive SRP-9001 compared to other gene therapies. As I conclude today, I'd like to thank my Sarepta colleagues who remain grounded in our mission to translate the very best science into the very best treatments for patients in the shortest time possible. Thanks as well to our partners in science, clinical trial investigators, and the patient community for their dedication. I will now turn the call to Dallan on our commercial activities. Dallan? Thank you, Louise, and good afternoon, everyone. Driven by continued demand for our three RNA-based PMO therapies, I'm pleased to report that the team delivered approximately $190 million in net product revenue for the first quarter of 2022. This performance represents more than 50% growth or greater than $60 million in net revenue growth over the approximately $125 million in net revenue for the first quarter of 2021. For the first quarter of 2022 compared to the first quarter of last year, we delivered nearly 10% growth for EXONDYS 51 and more than 50% growth for VYONDYS 53, while maintaining our market leadership position in the exon 53 amenable population. The performance of AMONDYS 45 continued unabated in the first quarter of 2022. With 22 quarters of consecutive quarter-over-quarter growth, it's tempting to gloss over the day-to-day challenges the team has overcome to deliver on our objectives. I'll take a moment to elaborate. Firstly, as we've come to expect through our deep experience in serving the Duchenne community and consistent with our historical experience, insurance changes at the beginning of each year impacted our revenue across all three therapies. Second, we saw competition for newly diagnosed patients in the 4- to 7-year-old range from our own EMBARK trial for SRP-9001. We planned for this issue and factored it in to our 2022 guidance. This competition disproportionately impacts EXONDYS 51 and VYONDYS 53 due to our penetration in these populations and subsequent reliance on these patients for new starts. Lastly, in addition to facing competition from the EMBARK trial, we are also actively enrolling exon 51 amenable patients in our MISSION study and part B of the MOMENTUM study to support the approval of our PPMO candidate, SRP-5051. Our performance for the first quarter is even more impressive in light of the fact that all of this competition is from our own R&D group. We expect the pressures from clinical trial enrollment will persist for the remainder of the year, but we remain comfortable with our 2022 net product revenue guidance of over $800 million. I'll now outline individual net product revenues for the first quarter for each of our three approved RNA-based PMO therapies. Beginning with EXONDYS 51, which totaled roughly $117 million, representing approximately 9% growth over the first quarter of 2021. You'll notice the revenue dipped slightly compared to the fourth quarter of 2021, but this is expected and primarily driven by the insurance changes at the beginning of each year. By the end of the first quarter of 2022, the team had worked through this challenge, and we are now exactly where we forecasted we would be at this point in the year. The expectation for the remainder of the year is a return to the modest growth trajectory for EXONDYS 51. For VYONDYS 53, revenue totaled approximately $28 million, representing roughly 60% growth versus the first quarter of 2021. As we've noted previously, the smaller revenue for VYONDYS 53 when compared to AMONDYS 45 is primarily driven by a larger than expected exon 45 amenable population compared to the exon 53 population. This is also consistent with what we've seen in clinical trials. In general, we're pleased with the performance of VYONDYS 53 and particularly our continued leadership position within the exon 53 amenable population. For AMONDYS 45, revenue totaled nearly $44 million, representing greater than 25% in sequential growth over the fourth quarter of 2021. The team continues to execute in the launch phase of AMONDYS 45, driving both patient identification and also working hard to gain access for the patients who have already submitted start forms. We expect the growth of AMONDYS 45 to continue in the coming quarters. I'm proud of this successful start to 2022 and of our dedicated and highly motivated team who work every day to execute on our mission and support the nearly 30% of patients who are amenable to one of our three approved RNA-based PMO therapies. As a fully integrated biotechnology company focused on Duchenne and other unmet needs in precision medicine, we look forward to working closely with our R&D colleagues as they discover and develop therapies such as SRP-9001 for an even larger proportion of the Duchenne population, as well as advancing our deep portfolio of therapies in gene therapy, RNA, and gene editing. Now I'll turn the call over to Ian Estepan for an update on our financials. Ian. Thanks, Dallan. Good afternoon, everyone. This afternoon's financial results press release provided details for the first quarter of 2022 on a non-GAAP basis as well as a GAAP basis. Please refer to the press release available on Sarepta's website for a full reconciliation of GAAP to non-GAAP financial results. For the three months ended March 31, 2022, the company recorded revenues of $210.8 million, which consists of net product revenues and collaboration revenues, compared to revenue of $146.9 million for the same period of 2021, an increase of $63.9 million. Net product revenue for the first quarter of 2022 from our PMO exon skipping franchise was $188.8 million compared to $124.9 million for the same period of 2021. For the first quarter of 2022, individual net product sales were $117.1 million for EXONDYS 51, $43.6 million for AMONDYS 45, and $28.1 million for VYONDYS 53. The increase in net product revenue primarily reflects higher demand for our products and a full quarter of AMONDYS 45 sales during the three months ended March 31, 2022, given its commercial launch in February 2021. As noted earlier in the call, we are reiterating our 2022 total revenue guidance of greater than $880 million for our net product revenue. Guidance for our RNA franchise was greater than $800 million. In each of the quarters ended March 31, 2022 and 2021, we recognized $22 million of collaboration revenue, which relates to our collaboration arrangement with Roche. The reimbursable co-development costs under the Roche agreement totaled $17.7 million for the first quarter of 2022, compared to $13.4 million for the same period of 2021. On a GAAP basis, we reported a net loss of $105.1 million and $167.3 million, or $1.20 and $2.10 per basic and diluted share for the first quarter of 2022 and 2021 respectively. We reported a non-GAAP net loss of $48.6 million or $0.56 per basic and diluted share in the first quarter of 2022, compared to a non-GAAP net loss of $114.5 million or $1.44 per basic and diluted share in the first quarter of 2021. In the first quarter of 2022, we recorded approximately $31.4 million in cost of sales, compared to $22.3 million in the same period of 2021. The increase in cost of sales is primarily due to increasing demand for our products, partially offset by write-off of certain batches of our products not meeting the quality specifications for the three months ended March 31, 2021, with no similar activity for the three months ended March 31, 2022. On a GAAP basis, we recorded $194.3 million and $195.1 million in R&D expenses for the first quarter of 2022 and 2021 respectively, a year-over-year decrease of $800,000. This decrease is primarily due to decreases in sponsored research with academic institutions and decreases in upfront and milestone expenses during the first quarter of 2022 compared to the first quarter of 2021. On a non-GAAP basis, R&D expenses were $173.2 million for the first quarter of 2022, compared to $177.5 million for the same period of 2021, a decrease of $4.3 million. Now turning to SG&A. On a GAAP basis, we recorded approximately $71.8 million and $71.1 million of expenses for the first quarters of 2022 and 2021, respectively, an increase of $700,000. The increase was primarily driven by an increase in professional service expenses. On a non-GAAP basis, the SG&A expenses were $53.2 million for the first quarter of 2022, compared to $51.5 million for the same period of 2021, an increase of $1.7 million. On a GAAP basis, we recorded $17.3 million in other expenses net for the first quarter of 2022, compared to $15.5 million in other expenses net for the same period of 2021. The increase primarily reflects an increase in our mark-to-market adjustment on our strategic investments during the three months ended March 31, 2022, compared to the same period of 2021. We had approximately $2 billion in cash equivalents, and restricted cash and investments as of March 31, 2022. Based on our current assumptions, we believe our balance sheet provides us runway beyond the readout of Study 301 and into 2024. With that, I'll turn the call back to Doug to start the Q&A. Doug? Thank you very much, Ian. Chloe, let's open the line for questions. Absolutely. At this time, if you would like to ask a question, please press the star and one on your touchtone phone. You may withdraw your question at any time by pressing the pound key. Once again, for your questions today, that is star and one. We'll take our first question from Colin with UBS. Please go ahead. Hey, good afternoon and, congrats on all the progress. Doug, maybe you could just give us a quick update on, you know, when you expect to meet with FDA regarding the path forward and filing strategy for 9001. It seems that hasn't happened yet. Then, just quickly on your competitor, Pfizer, they're about to initiate trials in the U.S., so just wonder your updated thinking on how you view them as a competitor from a timing and, I guess, clinical profile perspective. Thanks. Yeah. Colin, thank you very much for both of your questions. I actually particularly appreciate the first one because it gives me the opportunity to answer this for everyone. The short answer is that, as I said before, I said in the prepared remarks, and I'm gonna be consistent about this, you know, we have said that we are going to talk to the FDA. We will provide an update once we have concluded those discussions. I would prefer not to provide additional color until we have all of those discussions completed and we are in a position to provide an update. I promise you all we will provide an update. In the interim, as I've said many times, the EMBARK is a very well-powered placebo-controlled study. We're very excited about it. It's recruiting and dosing, and we're getting additional sites up and running, literally on a weekly basis. That will enroll around the middle of this year, and then we will have a readout next year. That ought to be the assumption of our pathway to approval both in the United States and around the world, unless and until we hear something different. As it relates to Pfizer, yes, we did see that Pfizer announced they would recommence its study that had been placed on clinical hold for safety reasons previously. It doesn't affect us, is the short answer. You know, we're focused on our program. We're focused on enrolling EMBARK. We're very, very excited about EMBARK. EMBARK's protocol enjoys a very straightforward, you know, entrance criteria and the like. It is almost everywhere in the world, an outpatient protocol which will benefit not only the enrollment of EMBARK, but will certainly benefit the label for EMBARK as well. It also, I'm very happy to say, enjoys a relatively very low screen out rate, which, if you know, the screen out rate for SRP-9001 and AAVrh74 is less than 15%. That does not appear to be the case with other constructs. We're very focused on our program, very focused on continuing EMBARK, and I think that, you know, we see as our primary competitor Duchenne muscular dystrophy, and we take that very seriously. We're gonna get EMBARK fully enrolled, and then we're gonna get that read out. We have a high conviction on the success of that therapy, and then we're gonna get that therapy to kids in the United States and around the world with the success of EMBARK. Thank you very much for that, Colin. As a reminder, that is star and one. We also do ask that you limit yourself to one question. We'll move next to Brian with RBC Capital Markets. Please go ahead. Hi, good afternoon, and thanks for taking my questions. I think there's maybe a little bit of confusion around phase three timelines. I was hoping you could clarify. It sounds like nine-zero-zero-one enrollment is on track for mid this year. When one considers screening and data analysis, should we be expecting top-line data release around mid-2023 or closer to year-end? Then maybe on a similar note, on the registrational path for nine-zero-zero-one, I recognize you aren't in a position to comment on the specifics of ongoing FDA dialogue, but just taking a step back, should we be thinking about this as accelerated approval or full approval, or are there any middle ground expedited scenarios we should contemplate, such as maybe a rolling BLA with existing data and integrated one-year analysis that you could update at the end of the submission with the EMBARK data when that becomes available? Thanks. EMBARK is a 52-week study, so if we're fully enrolled around the middle of this year, that means we'll have data by around the middle of next year. Of course, then we'll have to ensure that we get it collated and quality controlled and all of the data, including the secondary endpoints and biomarkers done. I'm sure it'll be in the second half of next year, and then our goal is to file as soon thereafter as possible, and our goal is to file in 2023. With respect to, again, I'm gonna be careful. I'm not gonna provide updates until we have something to update on. EMBARK is... The goal of EMBARK is a full approval, just so we're very clear about that, not looking to EMBARK, for instance, as an accelerated approval. We intend to have dialogue with the division at the FDA about the potential for a faster approach. The most obvious faster approach would be the use of the accelerated approval pathway to bring this therapy to patients. We certainly think the data justifies that dialogue. We have three studies of data. We have exceptional expression. We have a very good and very superior safety profile. We have very consistent functional benefits across all of the studies. I think that dialogue makes a ton of sense for us, but we'll provide an update on the outcome of those discussions when they are completed. We'll move next to Judah with Credit Suisse. Please go ahead. Hi. Thanks for taking the question. Just wanna on kind of timelines for Study 103 data readout. I think you've said that FDA would get that 1-year data before kind of it's shared publicly. Should we expect that we'll get 1-year 103 data with the FDA conversation update, or could that be reported before that potentially? We will. We're at a medical meeting this year, Louise and team are sort of mapping out right now. We will provide an update on SRP-9001. It will include some data from 103. It'll include 2-year data for 102 part 2, some additional very interesting information, including an integrated analysis across our studies, and that'll occur this year. We don't have a time to share yet with it, but the team's working on that. We'll move next to Gena with Barclays. Please go ahead. Hi, this is Sheldon on for Gena. Thanks for taking our question. Maybe just two quick ones. One is on the approval pathway for nine-zero-zero-one outside of U.S., for example, in Europe. Do you have any feedback from EMA on the approval requirements? Is it only pathway for full approval, or is there any other possibility for expedited process? Another question is on the related regulatory and the sales milestone that you can receive from Roche. I remember the total amount is $1.7 billion. Could you give us some color on the breakdown of regulatory versus commercial milestones there? Thanks. Sure. I'm gonna leave the second question to Ian, who will answer consistent with what we've said historically in the past. With respect to Europe, I just wanna be respectful of our very good partner, Roche, and not step in front of them. I will note just simply noting at the theoretical level that there, of course, is the opportunity for getting the sort of traditional approval in Europe. There is a more expedited pathway. It's different than the United States, but there is a conditional approval pathway that exists in Europe. I'm sure we'll be thinking creatively together about which of those pathways makes the most sense for the patients that we serve. Ian, with that, you might wanna touch briefly on milestones. Yeah, as it relates to the milestones, we haven't broken out the exact details between the split between commercial or regulatory. The question we often get is in terms of timing, and that is gonna be, you know, you know, very late in either the regulatory framework or obviously post-launch. These are back-end loaded milestone payments. We'll take our next question from Brian with Baird. Please go ahead. Hey, good afternoon. Thank you for taking my question. Just going on to the Pfizer's recent announcement, I know that they're moving back into the clinic in the U.S. Moving into the clinic sounds like there's some restrictions, notably the seven-day hospitalization period post-dosing. Was just wondering, can you remind us of any sort of protocol requirements, SRP-9001 has for post-dosing? Is there an overnight observation period or any sort of mandatory post-dosing observation period? Thanks. Yeah. Other than in Japan, everything is outpatient. Physicians obviously have the discretion if they wanna keep a child overnight if they want, but it's outpatient dosing everywhere but Japan. Very different than the Pfizer protocol. You know, I'm sure it's informed by the experience that we've both had with these therapies and the safety profile of development therapies. Our next question comes from Gil with Needham & Company. Your line is open. Hey, good afternoon, everyone, and congrats on the progress in this quarter. Just a quick one from us. Assuming Pfizer is gonna be opening sites in the U.S., doesn't that exacerbate the competition for patients that usually go on EXONDYS? If you have any commentary around that? Thank you. I think the greatest competition for EXONDYS is Sarepta. I think as Dallan noted for you earlier, EXONDYS in the United States competes with EMBARK, it competes with Mission, and I'm trying to remember. There's a third one. Dallan, you'll have to help me with it. I'm losing train of thought. Momentum. MOMENTUM, of course, for PPMO 5051. Notwithstanding all of that, and that existed, of course, tracking into this year, EXONDYS grew fairly substantially versus the first quarter of last year versus the first quarter of this year, and our guidance for the year has that in it. We're not particularly concerned about it. Then on the competition for patients for our relevant gene therapies, without being excessively snarky, we are not at all concerned about the demand that we have for SRP-9001 and EMBARK. We'll move next to Ritu with Cowen. Please go ahead. Hi, guys. This is Anvita for Ritu. Two quick questions from us. What updates are you presenting at the ASGCT this month and maybe PPMD also in June? When should we expect top-line data from the MOMENTUM study? Thank you. Louise, do you wanna take that? Yeah. At the ASGCT, we are presenting preclinical data on cardiac outcomes for 9001. We'll be doing follow-up presentations for our clinical programs consistent with what's done previously. We don't have final plan outlines for PPMD. We'll move next to Hartaj with Oppenheimer & Co. Please go ahead. Great. Thank you. Thanks for the question. Just focusing in on PPMO, you know, part of your study actually had boys, most of them who were over the age of eight, you know, averaging 11, 13, 10 years. I think you just mentioned that you're seeing about, you know, 80-85% of patients, you know, without that are still negative for AAVrh74. Could you talk about your PPMO opportunity, excuse me, in the context of older boys, also XLA, and then maybe boys that might not, you know, get gene therapy. I'm sure as well. Thank you. Yeah. Hartaj, thanks for that question. I mean, one of the answers to that question right now is that we don't know with certainty what the impact will be on the PMO and PPMO from the launch of gene therapy. I think one of the things I've said in a prior earnings call was that making what I think is a relatively conservative assumption that there's going to be a pretty significant amount of cannibalization of the RNA technology PMO and maybe to a lesser extent PPMO from gene therapy. To your very good point, I think there's reason to believe that's excessively conservative. First, let's start with the fact that there will be about 15%, hopefully a little bit less than that, really slightly under 14% our sterile prevalence study tells us, that will screen out. We have solved the issue of knocking down pre-existing neutralizing antibodies. We'll screen out for gene therapy. There may be older children that have an opportunity to get on an RNA technology before they're able to get on a gene therapy, and that opportunity will exist as well. I think there will be geographic areas that I have, you know, it's, I've worked in countries outside the United States. I've launched products outside the United States. The vagaries of the various systems outside of the United States are different region to region and sometimes not perfectly aligned with what you might think is perfect economic sense, but they have to do with budgetary issues and yearly budgets versus longer budgets. I think there is a real opportunity geographically as well to offer some patients the PPMO or PMO as well. I think there is a real potential even if there's not co-dosing for a significant amount of the use of the PMO and when the PPMO is approved, the PPMO, and that's why we're going so strenuously forward with the PPMOs, just to let you know. We will just about double the number of patients that have a PMO available to them with the PPMOs that we're working on pre-clinically right now. All of that is before another thesis, which is the concept that we might be able to provide even greater longer-term benefit to patients through the use of a one-time therapy, SRP-9001, and then a chronic therapy thereafter. There are issues associated with that. First, we need the science to support that conclusion, and then we've got to do the pharmacoeconomic work to justify that concept. I think there's a lot of potential promise with our PPMOs, and that's why we're so excited about it. With that said, I'm gonna turn it over to L.R.K. with a piece of this answer as well, if I'm not mistaken. I was actually I missed the second half of the previous question. It was about the readout for MOMENTUM. The MOMENTUM study Part B is expected to complete enrollment at the end of the year. It's a 28-week endpoint, so we expect to present the top-line data in 2023. Sorry for the missed. SVB Securities, please go ahead. Hi. Thanks very much. I was wondering if you can speak to the steroid treatment regimens being used in EMBARK and how balanced they are between the treatment and placebo arms. Are there any key differences? You know, are you doing anything to enhance the interpretability of the data and the contribution from SRP-9001 in these patients when you report the data? Thanks, Joe. I'll give the broad stroke answer, and then, Louise, if there's any nuance that I missed, you tell me. The answer is they're identical across both arms, placebo-controlled trial. All kids are on stable steroids before they enter the trial, and then they both get the same regimen of steroids, including the same regimen of pre-dosing steroids as well. Louise, if there's any nuance that I've missed, please let me know. No, that's accurate. Exactly the same. We'll go next to Tim with William Blair. Please go ahead. Oh, thanks for the question. I promise I won't ask an FDA question, but maybe one on the base business. You mentioned that the exon 45 amenable population is larger than maybe originally expected. Can you dig into that a bit? Maybe how many patients do you expect a few years ago? What's your current thinking? Is this something that's also kind of an ex-U.S. phenomenon or just U.S.? Well, it's definitely whatever this phenomenon that we're seeing, and then, Dallan, you can take it from where I am, broad stroke. Whatever we're seeing is definitely not a U.S.-specific issue because we've seen it in the U.S., we've seen it in Europe as well. How is that the case? It's because, you know, why would we have seen it in Europe? Because we have EXONDYS in Europe. One of the things we noticed fairly early on, and it caught us off, surprised us, was that enrollment for exon 45 went very fast relative to what we would have expected from our experience with VYONDYS, which began to give us the view that there is perhaps a difference in the epi versus what we thought. Of course, we've launched the commercial therapy, and the uptake has been tremendous. I do wanna say, you know, I do really wanna say significantly to our field-based force, our commercial colleagues, and our medical affairs colleagues, that a lot of that has to do with execution. We have gotten better and better and better. Just let me linger on that for a second to make the point. We now have cumulative $2 billion in sales. We've already achieved that as an organization. We achieved the second $2 billion in significantly less than half the time of the first $1 billion. I think a lot of that has to do. Some of that, of course, has to be approvals. Some of it has significantly to do with execution, the team really knows how to serve these patient communities. None of it, by the way, has to do with price increases because we don't do price increases. We certainly have not yet since the inception. If we look across it, we don't know with certainty yet what the prevalence rate is, the epi is between these two. I think there are some studies. There was a study, if I'm not mistaken, Dallan, in Canada, that would have put it. It was inconsistent with what the common belief is, but it put, you know, the exon 45 maybe up, you know, even above 10%. We, we're not standing behind that yet. We don't know. Dallan, perhaps you have more insight into this than I'm providing. Yeah. No, just exactly what Doug has said, and thank you for the question, by the way. I think historically, all of the epidemiology that we were looking to showed that the two populations, the exon 53 amenable population and the 45 amenable population, were the exact same size. As Doug explained, the first hint that we got that this wasn't the case was in our clinical development program. What we saw in the clinical development program is exactly what we see playing out right now in the commercial market. That was, you know, our assumptions were based on the older initial epidemiology. As Doug said, there are some new publications that are more in line with what we saw in the clinical development space and what we're seeing in the commercial market. We're continuing to look at this as we go. There's nothing obvious in terms of differences regionally. As Doug said, in the clinical development program, that enrollment was a global trial where we saw the same phenomenon as what we're seeing in this U.S. market. Again, thanks for the question. Once more for your questions, that's star and one. We'll move to Kristen with Cantor Fitzgerald. Please go ahead. Hi. Good afternoon, everybody. Thanks for taking the question. Wanted to ask one around longer term plans of the company. If you see this cannibalization around gene therapy and are able to solve the problem for the 15% or so of patients with preexisting neutralizing antibodies, given the success you've seen, would you consider utilizing the PPMO platform for other potential indications or areas beyond DMD? We absolutely would, and we're exploring that pre-clinically right now, but perhaps Louise is gonna provide a bit more color on that. Thanks for the question. We're extremely excited about the opportunity for PPMO, both in additional exons in Duchenne, but then also other, indications to include muscles, but also include other, targets based on the PPMO's ability, to penetrate other tissues. More to come on that, but we're certainly very excited about the opportunity, for multiple indications for PPMO. We'll take our next question from Yanan Zhu with Wells Fargo Securities. Please go ahead. Hi. Thank you very much for taking the question. My question is on the LGMD program. Sorry if I missed if you had provided an update, but what about the next step and also the timeline? Are you still working on the CMC issues? Do you have a plan to move the 2E program forward or do you think, given the prevalence, that a basket study will make more sense and you're trying to align the FDA on that plan, please? Thank you. First of all, we are definitely exploring the concept of a basket study, which can be very interesting for us or at least a basket approach for all of the sarcoglycans. That isn't delaying our activity with respect to LGMD2E and SRP-9003. With respect to SRP-9003, the rate-limiting step is CMC. I wanna be very clear about what kind of CMC. It's not really the process development at all. In fact, we've manufactured therapy, so it's not about that, and we've really benefited from all of the work we've done with SRP-9001. It's the analytical work and getting all of the assays together. One would assume that you could have sort of generic assays across multiple programs. That such is not the case. There are a number of different assays that require them to be bespoke for each particular therapy. The good news is we know exactly how to do it. We've done it with SRP-9001 to great effect. We don't have to guess about that. The process, it takes some time. It's a combination of design, but it's also testing and empiricism and then testing. It's moving along nicely, not ever as fast as I would want, but I don't think there's anybody at Sarepta that thinks that I'm patient about anything. It's gonna, it's just gonna take a little bit of time, but we're on track. We just have to get the assays complete. Louise, have I misstated anything? No, that's accurate. I just will add that we are actively enrolling our natural history study called JOURNEY for LGMD, and so that will certainly support us in moving ahead for our next phase of the trials in terms of patient identification and also understanding the natural history, which will inform our design. That's enrolling well. I'll take our next question from Lekinkumar Patel with Berenberg Capital. Please go ahead. Hi, this is Amantica on for Z. I just wanted to ask a quick question. Can you just provide a bit more color about your statement that you made about the cost of sales where you said write off of certain batches of the company's products are not meeting its quality specifications? Sure. I'll turn that to Ian. Ian? Yeah. That was just related to, and this is, to be clear, related to our PMO franchise, that there were a few batches, you know, last year that didn't meet our standards, and therefore we wrote them off, but nothing to. It wasn't significant at all and didn't have any major impact. We saw none of that. It just happens that some runs don't meet your qualifications. From time to time it happens, obviously, we didn't see any this quarter. Once more for your questions, that is star and one. We'll move next to Gavin with Evercore ISI. Please go ahead. Hey, thanks for taking the question. Just wondering what the status of the ESSENCE confirmatory trial enrollment was. If I look at the clinical track, you think that enrollment should be done soon. Thanks. We should be fully enrolled by the end of this year. Once more, that is star and one. Move back to Matthew, Morgan Stanley. Please go ahead. Hi. Thanks for taking our questions. This is Weijing on line for Matthew. We are wondering whether you already have any regulatory discussion with FDA about the LGMD program. Oh, the LGMD program. Yeah, I'm sorry. Go ahead. Apologies. Yeah. If so, what's the minimum requirement you are expecting from the regulators? Apologies for having interrupted you. Yeah, we haven't had, you know, actual meetings with them. We've had written responses from OTAT. It was, if I'm not mistaken, last year. We haven't updated it. We're going to have one comprehensive discussion with them once the CMC issues are in a position where we can have a discussion with them both about the clinical design as well as and show them the CMC and get their blessing on the manufacturing. We had a general discussion with them in writing last year in which they, and then independently, interestingly enough, Europe as well, confirmed the potential for the use of beta-sarcoglycan as a surrogate endpoint. We obviously have additional discussions that we need to have and a, you know, more concrete discussion about the actual development program. Certainly conceptually there was an acknowledgement of that possibility. Of course it makes brilliant sense to remind everyone with respect to SRP-9003, that is a gene therapy that treats a very rare disease, and it's a disease that's characterized, well characterized by the fact that it is patients that are degenerating and dying as a result of the lack of a structural protein called beta-sarcoglycan. Given the size of that gene, SRP-9003 is able to deliver a construct that codes for the full length wild type beta-sarcoglycan properly localized. We're seeing very, very good expression. We're seeing the kinds of expression that we see with SRP-9001. We're seeing the kinds of safety that we see with SRP-9001. Clearly, AAVrh74 is performing very well. The idea of using a more expedited pathway and using surrogate endpoints makes a ton of sense. Now we just have to get our stuff with the division and get their concurrence on the design of that next trial, then we'll get going on it. Hopefully get going on that and the other sarcoglycans as well. It does appear there are no further questions at this time. I would now like to turn it back to Doug for any closing remarks. Well, thank you all very much for joining us this evening and for your questions. We appreciate all of them. We're very excited about the progress that we've made. We're very excited about our ability to serve the community that we serve and to continue to advance our research pipeline as we advance our development programs, including completing our pivotal trial enrollment for EMBARK and completing our pivotal trial for MOMENTUM as well. I look forward on behalf of all of the team at Sarepta to provide additional updates as we gain additional progress over the course of 2022. Thank you all and have a lovely evening. This does conclude today's program. Thank you for your participation. You may disconnect at any time and have a wonderful evening.