Good day, ladies and gentlemen, and welcome to the Sarepta Therapeutics Second Quarter 2020 Earnings Call. [Operator Instructions] As a reminder, today’s program is being recorded. At this time, I’ll turn the call over to Mary Jenkins, Manager, Investor Relations. Please go ahead.

Thank you, operator and thank you all for joining today’s call. Earlier today, we released our financial results for the second quarter 2020. The press release is available on our website at sarepta.com, and our 10-Q was filed with the SEC earlier this afternoon. Joining us on the call today are Doug Ingram, Bo Cumbo, Ian Estepan, Dr. Gilmore O’Neill and Dr. Louise Rodino-Klapac. After our formal remarks, we’ll open up 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 slides 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 risk can materially and adversely affect the business, the results of operations and trading prices for Sarepta’s common stock. For a detailed description of applicable risks and uncertainties, we encourage you to review the company’s most recent annual report on Form 10-K and most recent quarterly report on Q-10 filed with the Securities and Exchange Commission 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. With that, let me turn the call over to our CEO, Doug Ingram, who will provide an overview of our recent progress. Doug?

Thank you, Mary. Good afternoon and thank you all for joining us for Sarepta Therapeutics second quarter 2020 investor conference call. As I have mentioned in the past, on March 13 of this year, all but the small contingent of our workforce transitioned it to a virtual work environment. Notwithstanding this unusual approach, we are not merely remaining productive, but have arguably become even more efficient and effective as we advanced our multi program, multi-platform genetic medicine ambitions. As you will hear this afternoon, we have continued to serve the patient community with our approved therapies, have already achieved or remain on track or many planned 2020 milestones expanded our three-pronged approach to building our enduring gene therapy engine. And as we have generated additional evidence in 2020, continued to build confidence in our unique approach to gene therapy is proving successful and highly differentiated. With that, let us review our performance. As we continue to serve our patient community, while endeavoring to keep them safe in this pandemic, I am pleased to report that our second quarter net sales stand at $111.3 million, an 18% increase over the same quarter last year. As I mentioned in our last quarterly conference call, we withdrew our 2020 guidance in light of the potential impact of COVID-19 on clinic visits. As you can see, that impact has thus far been modest. Given the uncertainties of the external environment, we are not ready yet to set full year guidance. However, consistent with what we have seen thus far in 2020, we anticipate that any continuing impact from COVID-19 will remain modest. Moving to our development programs, let me begin by commenting on our RNA franchise. In the second quarter, we completed our NDA submission for casimersen. Our RNA therapy built on our PMO…

Thank you, Doug. Good afternoon, everyone. Our experienced teams at Sarepta are continuing to work through the headwinds of the COVID-19 pandemic. I’m pleased to report that our product revenue for the second quarter of 2020 totaled $111.3 million. We’ve overcome some unique obstacles created by the pandemic and are extremely proud of the teams and their accomplishments and the face of these challenges. While we’re still navigating and responding to the strain the pandemic has placed on the healthcare systems. The modifications we’ve made to our commercial execution strategy have allowed eligible patients to start and stay on therapy in this unprecedented time. Our strong relationships with partners have played a key role in the ability to start new patients, keep others on therapy. This is a testament to our team’s expertise and commitment to our mission to serve the Duchenne community. We continue to provide an uninterrupted supply of therapies to our patients and mitigate major treatment disruptions through a unified effort with our manufacturers, distributors, especially pharmacies, healthcare providers and payers. Many of our patients are choosing not to delay or stop therapy. And we believe this is partly due to the fact that the majority of patients on EXONDYS 51 and VYONDYS 53 are receiving weekly infusions in their homes. We are currently working closely with healthcare providers and specialty pharmacies to transition additional patients to weekly home infusions. Since many clinics and hospitals still have restrictions in place. We have thoughtfully deployed measures to minimize the risk of transmitting COVID-19, which includes making personal protective equipment available to all our patients who have requested supplies. This will help ensure that patients have access to protective equipment, when nurses’ administer their weekly infusions at their homes. Patient safety remains our top priority, and we will continue…

Thanks Bo. Good afternoon, everyone. This afternoon’s press release provided details for the second quarter of 2020 on a non-GAAP basis, as well as a GAAP basis. The press release is available on Sarepta’s website. Please refer to our press release for a full reconciliation of GAAP to non-GAAP financial results. Net product revenue for the second quarter of 2020 from our products, EXONDYS 51 and VYONDYS 53 was $111.3 million compared to $94.7 million for EXONDYS 51 alone for the same period of 2019. The increase primarily reflects higher demand for our products. In the quarter ended June 30, 2020, we recognize $26 million of collaboration revenue, which primarily relates to our collaboration arrangement with the Roche. The co-development costs under the Roche agreement totaled $8.9 million for the second quarter and are included as a reduction to our R&D expenses. On a GAAP basis, we reported a net loss of $150.8 million and $276.4 million or $1.93 and $3.74 per basic and diluted share for the second quarter of 2020 and 2019 respectively. We reported a non-GAAP net loss of $117.9 million or $1.51 per basic and diluted share in the second quarter of 2020, compared to a non-GAAP net loss of $61.2 million, or $0.83 per basic and diluted share in the second quarter of 2019. In the second quarter of 2020, we recorded approximately $13.3 million in cost of sales compared to $15.9 million in the same period of 2019. The decrease was primarily due to write-offs of certain batches of EXONDYS 51, not meeting our quality specifications for the three months ended June 30, 2019 with no similar activity for the three months ended June 30, 2020. On a GAAP basis, we’ve recorded $188.5 million and $113.3 million in R&D expenses for the second quarter…

Thank you very much, Dr. O’Neill. And Crystal with that, let’s open the call for questions.

Thank you. [Operator Instructions] And our first question comes from Tazeen Ahmad from Bank of America. Your line is open.

Okay, good afternoon. Thanks so much for taking my question. Doug, I just wanted to get a little bit more color as it relates to the FDA discussion this quarter. Can you talk about some of the more important items that you’ll need to get alignment on from the agency? And also do you plan on asking them about the plan to pass the non-ambulatory or older patients as well? Thanks.

Thanks a lot for that Tazeen. So let’s review, one of the things, we had said earlier this year that we would have GMP material on hand by July of this year. And the good news is that we did. So we’ve got two things to do. As you know, we’ve got a – get our next study using commercial process material up and running and getting patients dosed. And we’re going to do that in the second half of this year and the team outstanding COVID-19, and the distractions that occur, and some of the challenges occur with COVID-19 have done really a remarkable job keeping on pace and moving to get that study started. But before we can do that, we have to have our meeting with the agency. And I said in my prepared remarks, we will be meeting with the agency this quarter and there really are just two things that there are significant things, but there are two things that we need to get alignment and concurrence on with the agency. The first, of course, is our CMC approach itself for the commercial material to use the commercial material in the next study. We’re very pleased at how these GMP runs have come out, very, very confident in the approach that we’re taking from our perspective. There are no significant quality attributes that are different from the clinical material in a manner that would be predicted to affect the therapy in anyway functionally or efficacy wise or safety wise. So that’s one significant concur as we need. And of course, the second one is to gain concurrence on the next study. Certainly, in connection with that, we will have conversations with the agency about our non-ambulatory study as well. It is one of our goals to commence the non-ambulatory study as soon thereafter as it’s possible, perhaps even close to being concurrently with the next study, the main study that we’ll be using if we’re successful for the approval of the therapy in the United States.

Thank you. Our next question comes from Salveen Richter from Goldman Sachs. Your line is open.

Good afternoon. Thanks for taking my question. It’s successful, how do you see the PPMO platform fitting into the treatment paradigm versus PMO in gene therapy in DMD and what are your plans for non-DMD approach on the forward?

Yes. That’s a great question. I’m going to – I’ll answer that briefly and then I’ll turn this over to Dr. O’Neill. Could he maybe talk about some of the areas we could take this RNA technology. So first and foremost, the excitement of the PPMO, if it is successful is for an even more profound impact from an RNA perspective, with respect to Duchenne muscular dystrophy. Now, of course, the question will immediately arise thereafter, what is the – how will gene therapy and RNA co-exist? In the short answer to that is, we don’t yet know. There will certainly be even with a transformative gene therapy. There will be a place for a profound RNA for Duchenne muscular dystrophy in children who for instance, are screened out for gene therapy in places where gene therapy is not available or not yet available. So there will already be a significant place for it. And if one considers just that preexisting neutralizing antibodies alone, that is with respect to our capsid right now about 15% or so. And that population that would be screened out until we’ve been able to address that issue scientifically that is larger than the 10% population, but of course, with a significantly more impactful RNA platform. And they very well be the case that there is a role for a combination, both of gene therapy and of RNA. So first we need to see what the PPMO will look like. And then we are doing work even as we speak right now from a non-clinical perspective to test the hypothesis about whether a profound gene therapy followed either in the near term or in the longer term with a profound RNA would be synergistically beneficial to patients. But beyond that, of course, if one of…

One final thing, I will say, moving back to DMD, just – and I think people know this, but I’ll remind you. We’re currently using 5051 exon 51 PPMO as our proof-of-concept. And of course, later this year, we’ll have some of the PK/PD tissue dosing safety and exon skipping. Importantly, exon skipping for 5051 as our proof-of-concept, we have already built beyond 5051 constructs with the peptide conjugated to them for exon that could treat already about 50% of the Duchenne community with a path as Dr. O’Neill mentioned in his opening remarks, with a path to get to a significant percentage beyond that perhaps another 35% beyond that with a slightly more exotic platform approach. And so – and we will start moving as rapidly as we can, assuming of course that the results are support that when we look at them later this year.

Thank you. Our question comes from Anupam Rama from JPMorgan. Your line is open.

Hey, guys. Thanks so much for taking the question. I got a quick one here on we’ve gotten a lot of inbounds on 9001 safety recently, and I think some of it may revolve around some comment from Dr. Mendell, maybe at ASGCT that 9001 will be associated with some sort of mild compliment activation. Any context you can provide here and just maybe remind us of how frequently did the DSMB for Study 2. Thanks so much.

Sure. I’m going to turn this question over to Louise, who can answer this. But I will say upfront, just so we’re absolutely clear. That was a misunderstanding. There is absolutely no signal that we’ve seen as compliment associated with 9001 at all, but I think Dr. Rodino-Klapac can provide the context around that misunderstanding.

Sure. Yes. Dr. Mendell was speaking about his broad experience in gene therapy and not includes other vectors like AV9. As Doug mentioned, we presented our data in both different medical meetings, as well as investor calls on LGMD2E, as well as our terminology paper. And just to reiterate, we’ve not seen any evidence of compliment activation in our study. And Study 1 continues on as planned no interruptions.

I think people were reading through comments. He was making about other programs. This has been seen in a number of other programs, primarily associated with AV9. He wasn’t speaking about AAVrh74, we’ve seen nothing relating to compliment there. And just to remind you, and of course, I know Dr. O’Neill is a proper scientist. We have to measure ourselves by the evidence on hand, but the good news for us is that with respect to rh74 across both Duchenne muscular dystrophy and Limb-Girdle, we have now dosed, as you heard in my opening remarks over 35 children, and the Study 1 and 2 continues to move a pace. So I think the evidence on our broad safety profile, I think is only getting bolstered over the course of the last 24 months and really significantly this year.

Thank you. Our next question comes from Brian Skorney from Baird. Your line is open. [Technical Difficulty] Pardon me, sir. We’re not able to hear you. And then we’ll move on to our next caller Matthew Harrison with Morgan Stanley.

Hi, this is Max Skor on for Matthew Harrison. Just a quick question, based on the recent agreements you signed, how are you thinking about integrating these new technologies into your pipeline? Thank you.

We are – thanks a lot for that question, Max. So to just remind everybody, I think you heard in my opening remarks, we’ve done a number of really interesting transactions over the last quarter to evolve the science and gene therapy, as there’s – we’ve got Dyno, which is artificial intelligence and machine learning for making better CapsidMap. We’ve got Codiak, which is an entirely different approach using exosomes for delivery device, that you can use an RNA gene therapy, gene editing. And then I think it was sort of more near term perhaps, but really impactful is both Hansa with imlifidase, and Selecta with ImmTOR. Hansa’s technology would ablate preexisting neutralizing antibodies so that you could essentially empower – at a minimum empower the people that would otherwise have preexisting neutralizing antibodies that they would have got environmentally have them in frames for the ability to get gene therapy. Of course, Selecta, with this concept of co-administering their ImmTOR technology with a gene therapy so that you could essentially trick the body into believing that the gene therapy that it’s receiving and the candidates receiving itself and not for it, you don’t build up neutralizing antibodies, which could empower re-dosing even multiple doses every time. The short answer on your question, however, is we’ve just entered into these agreements. These tools have gone into the armamentarium that Dr. Rodino-Klapac and her team have to advance this. And we’re looking carefully at the timelines and how we can move these as fast as possible forward. As you can imagine, as a mission-driven organization, it is our goal to move these technologies as fast as possible, and improve gene therapy as much as possible. More importantly, bring the largest number of patients into frame for gene therapy. So we don’t have timelines right now, but we’re moving as fast as we can on that. And I suspect we’ll have updates early next year on that.

Thank you. My next question comes from Martin Auster from Credit Suisse. Your line is open.

Hi everyone. This is Mark on for Marty. Thanks for taking my question. So my question relates to the upcoming readout for SRP-5051. I know that you frame the program relative to exon does in terms of dystrophin expression. I’m curious, what is the level of exon skipping that exon does achieves in humans? And could you speak to the magnitude of improvement in exon skipping, you’re hoping to see SRP-5051 as part of this initial clinical trial update? Thank you.

Sure. Dr. O’Neill, you might want to take this and talk a little bit about what we’ll be looking for in the second half and perhaps the way we would look at to compare it to what we might be seeing with eteplirsen, golodirsen and casimersen? Gilmore O’Neill: Yes. So I think the key thing is that we will be looking, first of all, it’s very important to understand that we are using digital drug PCR and we will actually be looking of that for PMO and PPMO. It’s important to actually distinguish that from the older quantitative RTP service being used in the past. And we will actually be looking at data from both PMO historical samples, and then looking at our ddPCR in our 5051 program. I think the other point that wants to make is that, or restate is that we have actually with our non-clinical data both in vitro and cell line, as well as in animal model. As seeing a robust correlation between dd digital drop PCR and dystrophin levels. And what we’ve actually seen there is a ratio of between two to three. Obviously, we have to validate that in human, but what we’ve seen in our experimentation to date has been how should I say gives us confidence with regards to both the robustness of the correlation and the ratios and what we hope to see in our human studies.

Thank you. Next question comes from Alethia Young from Cantor Fitzgerald. Your line is open.

Hey guys, thanks for taking my question and congrats on the quarter. I just wanted to talk a little bit about kind of hitting the GMP pre-material and the compatibility. It seems like that’s a big deal for you guys and want to talk about what that means going forward for this and other platforms of your? And then just on Selecta and that ImmTOR, I’m always just interested in Dr. Klapac perspective on like, how to think about re-dosing and how this technology in hypothetically might be important in that. Thank you.

Okay. Before I turn it over to Louise and talk about Selecta at all. So thank you, Alicia. I agree with you, it a big deal. I mean it, and it’s not a one quarter effort. The fact that we have GMP material on hand right now is the result of an enormous amount of work by a significant number of professionals over a long period of time. We started this really at ideation stage a couple of years ago that we were going to build out this gene therapy engine. And we knew at that time that we need to build out manufacturing, not simply for capacity, but also expertise, process development, analytical development, et cetera. And you’ve sort of lived with us as we’ve worked through that process, optimized or the process of optimizing process development, getting all of our analytical development done this, 24 assays involved here, all are complete, even qualified or validated, and if the case is required for each of those. And then, of course, we’ve done numerous runs and having GMP material on hand right now is enormously important. And it will – now I will also say, of course, we also have to meet with the agency. We’re going to do that this quarter. We have to get them do concur in our view the material, et cetera, but we feel very good about where we are and the results that we’ve seen. And to your very good question, it’s going to pay significant dividends on our platform more broadly, as I said, and I think in my prepared remarks, this concept of a gene therapy engine isn’t simply a narrative. It is a platform that is built on three significant pillars, all of which are required to build an enduring gene…

Yes. Thank you for that question. So we’re excited about both Hansa and Selecta. Hansa gives us an opportunity to ablate antibodies in patients that are positive for AV antibodies. So that helps us potentially get to those 15% or so patients that have pre-existing antibodies and also gives us the opportunity to potentially for re-dosing down the road. Selecta, we’re particularly excited about because it has the potential to prevent those antibodies from forming in the first place. So and with the first dose of gene therapy patients could be co-administer this, is strives to potentially prevent those antibodies from forming with the potential to re-dose down the road. So we just entered into these agreements. And then there’s additional non-clinical studies to be able to prove that these will be effective and safe. But we’re definitely excited about the potential there. We want to make sure that we have every, every tool to reach all patients and that’s our goal. So we’re just we may never need it. But it’s better to be looking at it proactively. And that’s what we’re doing. So thank you.

Thank you. Our next question comes from Tyler Van Buren from Piper Sandler. Your line is open.

Hey, good afternoon. I had Just a quick follow-up question on the MOMENTUM trial. So you mentioned that you started at 4 mgs per kg and are up now to 20 mgs per kg, but if I’m not mistaken, you mentioned you could also potentially go higher than that. So based upon the animal model data, can you just define potentially a ceiling or a dose ceiling that you observed and what that was based on? I’m assuming it was some sort of safety or toxicity.

Yes. Thank you for that question. So the short answer is that we’re going to continue – so first understand the following. We are already above what we had – we would have considered success at least from our preclinical models. Now, please, don’t over read that we’ve got to get the data and we’ve got to see the biopsies and we’ve got to do the work. And so I don’t want to oversell this. But I will say, if you look from a preclinical prediction perspective, we’re already beyond that point where we would have said, we are very excited and this is great. We are going to continue to push that dose. We know what we’re looking for and what it is showing. And in preclinical models, that will be the dose limiting signal that will help us choose the dose. Our goal of course, is to achieve – reasonably achieved the highest dose that is safe for these kids. And so we’re going to continue to dose up until we get to a place where we think we’ve seen a signal that tells us that we’re at the right margin between safety and efficacy. And certainly we’re going to go to 30 mgs per kg this year. So you won’t have a doubt on 30 mgs per kg. It won’t come in time, but we’ll have the 20 mgs per kg, which will be – they’ll be certainly insightful for us. Is there anything there that I missed, Dr. O’Neill? Gilmore O’Neill: No, you nailed this. The target organ is the kidney and that’s what we’re monitoring closely. And it’s been good so far.

Thank you. Our next question comes from Brian Abrahams from RBC Capital Markets. Your line is open.

Thanks for taking my question and congrats on all the progress. With the full one year data for 9001 now published. I’m curious how this longer-term data guides you with respect to conduct patient selection, endpoint selection for your next studies. Just when we look across some of the positive results, but maybe a little bit of variability measure to measure and CK and function across the NSA components as well as the learnings may be there from the cardiac MRI. Thanks.

I would say, broadly one of the values of Study 101 is that it allows us real time insight as we build out – as we build out Study 102 and then as we look forward to our commercial process trial as well. So we already had a significant amount of experience with 101 by the time we commenced Study 102. So it already informed our thinking and it informed the powering of that study, as well as the selection of the primary functional endpoint being NSAA. We’ve had an opportunity to continue to monitor these children as you saw, of course. And then we have the one year data that reported out in JAMA Neurology. And I will tell you that it is confirming of the approach that we took with respect to Study 102, the way we’ve chosen NSA, and frankly, the way we have powered that study. So we feel very good about where we are and I think 101 has been very helpful in helping us power that study and give us confidence that we’re on the right track.

Thank you. Our next question comes from Gena Wang from Barclays. Your line is open.

Thank you for taking my questions. I have one question regarding PPMO has three parts. So the first is just follow-up with the earlier question. Could you remind us the percentage of exon-skipping for X down the 51 in early animal data? And the second question is for the non-muscle non-DMD program. What peptide you have in mind for the PPMO program? And lastly, do you see antibody oligo-based therapy as threat to your PPMO franchise?

Yes, fine. Okay. So first of all, to the last question I’ll answer now, and we’re excited that other folks are looking at researching ways to bring better last to Duchenne muscular dystrophy. But we certainly feel that – our programs are the most advanced and the most hopeful. So we’re really focused on our own programs. Going back to your other two questions, and I will turn this over to Dr. O’Neill, who can give you some perspective. But broadly speaking – and I’ll let Dr. O’Neill touch on this with more expertise than me. So, first of all, know that the percentage of exon-skipping you might look to in the historically with respect to eteplirsen, it won’t be meaningful here because the approach to looking at exon-skipping is different. We’re using digital drop PCR now, in the past we used a different form. I think it was rPCR. But Dr. O’Neill will remind us of that. And so it’s a bit apples to oranges. The good news, just so we know is that when we announced the results the 20 mgs on the PPMO, we will have data using the same exon-skipping technology for eteplirsen, so there will be an ability to see what we might’ve seen with eteplirsen versus what we will see with 5051. And then I believe I’ll let Dr. O’Neill confirm it, that we would be using the same proprietary peptide that we’re currently using, that we put a lot of work into. But Dr. O’Neill, you can correct me if I’ve said anything incorrect there. Gilmore O’Neill: No, that’s correct. And I think Gena, you asked – just to restate, you have two questions about peptide and the PMO skipping. I think I could do one better with regards to the ddPCR in the animal, and that we did actually look at it at 24 weeks, actually over several week period, but at 24 weeks in the PROMOVI in humans – in patients we saw about 0.6% skipping that exosome, but it’s very important to emphasize that’s digital drop PCR, it’s not the quantitative PCR that was used in prior descriptions. And that I think it’s disclosed in our MDA poster. So the beauty of the ddPCR is very tight and actually allows you then to compare across patients and very importantly, and more importantly to protein expression downstream. And then you asked other question about peptide. We’ve not disclosed the nature of the self entered peptide. But I think Doug and I have already highlighted in this earnings call and in others that we continue to – we’re never complacent. We’re never satisfied with the status quo and we continue to invest in exploring and optimizing possibly better peptides. And obviously our Codiak collaboration that we announced is also a part of an overall approach to both improve tissue penetration and frankly specific our target or tissue specific targeted.

Thank you. Our next question comes from Joel Beatty from Citi. Your line is open.

Hi. This is Shawn Egan calling in for Joel. Thanks for the updates today and for taking my questions. Maybe could you talk a little bit about the package you plan to go to the FDA with in regards to the path forward for limb-girdle muscular dystrophies in general? I guess, from the fact that these are mostly monogenic conditions, what other factors help make the case for an accelerated path to approval? And what else should we be kind of considering?

Yes. Thank you for that. Great question. So I think I said in my opening remarks, we have two significant things to do over the course of this year. One is to complete the GMP runs, which is a combination of two things. Just so we’re clear, it’s not simply the runs themselves, but it’s of course all the assay work. We can leverage a lot of what we’ve done. I mean, an enormous amount of what we’ve done with 9001. But there are the spoke assays for each particular program. So we’ve got to complete that work as well. And the second thing to your good question is, we need to engage in a dialogue with the agency about the appropriate regulatory and development pathway forward. And one of the things we said is that we will – we’ve already started – we are commencing a dialogue there and we intend to have a meeting before the end of this year on that issue as well. And to your point, why would we – we are, I think we’re unabashed about our view at least and we’ll see where we end up that it would be in the best interest of patients and also appropriate from a development and regulatory perspective to have a rapid and accelerated approach to the development of not only limb-girdle 2E but also at a minimum, the other cervical eye cancer, perhaps beyond that. There really are a number of characteristics of this disease and this therapy that we at least believe justify that. To your point, these are well-characterized monogenic diseases. We know what is causing these diseases, and it’s the lack of a structural protein that is the sole and exclusive reason that these children are degenerating and are living shortened lives.…

Thank you. Our next question comes from Vincent Chen from Bernstein. Your line is open.

Thanks for taking the question and congrats on the progress. Over the last few months, in addition to, I guess, your updated one year data for your DMD gene therapy program, like I said, we’ve also seen some updates from your competitor or peer Pfizer’s micro dystrophin gene therapy program. And clearly they’re different programs, but I was wondering whether you have a sense, do you think that their findings could provide some insights into the likely effect size with micro dystrophin gene therapy? And if so, how has this affected how you think about what is the likely effect size with a micro dystrophin gene therapy and your thinking around the powering of Study 102?

Well, let me say this, I’m going to do my best to talk about our programs and not about others. I will say that to the extent that others see some functional signals, even with very modest expression, that of course only gives us additional confidence in what we have seen both pre-clinically and given the robust expression that we’re able to achieve with our current constructs the functional results that we ought to see. And that frankly, although they are small numbers, what we appear to be seeing already is, we’ve talked about – we Study 101 in the first four kids, every child, they’re not stabilized, they’re improving on every functional endpoint with respect to those kids. And then you go over to limb-girdle, very similar, there’s an enormous amount of read through. And of course you see the same thing and you get great expression. And it appears to be tolerable and safe and we’re getting even at a low dose. In one year with low dose of LGMD, can we construct SRP-9003, you’ve seen a significant benefit on every functional endpoint there. And then we’ve got great expression with respect to the higher dose 2E using super coil PCR. So we’re excited there. What I would say, this is – what I’m going to try to do is avoid talking about other programs, because as I was, especially with someone earlier today. For those of you on the call that may play chess, there’s a famous strategy in chest, it says you’re supposed to play the board, don’t play the opponent and that’s what we’re going to do. And frankly, we’re excited about the way the words wide out, laying out right now. Our gene therapy engine and the approach that we take with these constructs…

Thank you. Our next question comes from Joseph Schwartz from SVB Leerink. Your line is open.

Thanks very much. So given the NSAA is comprised of 17 domains and they’re scored from zero, 1 and 2. I was wondering if there are certain patterns in the patients that have been treated with 9001 that make you confident that the results will replicated in the Phase 3 or different time course observations. Any sorts of characterizations you can share that make you particularly optimistic.

Yes. I can turn this over to Louise. I’ll make an introductory statement about it. I mean, what – look, first of all, of course there is always risk in over analyzing small data sets. This is for children with respect to 9001 and so far three children with respect to 9003 that we’ve seen functional results regarding. But with that said, you asked a very good question, which is, so you’re seeing these significant improvements across this composite score of NSAA, which is built on lots of individual tasks. It could be the case that what we’re seeing in aggregate is one function that is significantly improving versus all of the other functions. And then you can worry about what that might mean. The thing that gives us a lot of – it excites us and gives us confidence both in DMD with 101 as well as with currently with the low dose of limb-girdle, because that’s where we’ve seen the functional results so far is that every kid is benefiting on every functional endpoint. And if you look at the composite, they’re benefiting across all of the functions in that composite. It is really, they are generally improving functionally both on time tests on NSAA and on the individual components of NSAA, which is certainly confirmatory. But with that, Dr. Louise, you might have some more nuance news than I’ve just given.

I think you captured it well. I think what’s important is that across the components of the NSAA, as well as the other supportive measures, like time to rise as well as all of the time tests, we see supportive data coming out of our 101 study that led to the power end our Study 101. And so we feel confident based on those results across those measures as well as our other studies, like our limb-girdle study that we will – it’ll be successful.

Thank you. Our next question comes from Difei Yang from Mizuho Securities. Your line is open.

Hi. Good afternoon and thanks for taking my question. I have a quick one with regard to the 9001 program registrational pathway. So to what extent do you expect the interim analysis of a Study 301 will be part of that package?

It is certainly our goal that if they’re successful with 102 and with interim analysis of Study 301, that the interim analysis of 301 would play significant role in the registration of the trial. One of the things I said, if all goes well and we start the study in the second half of this year, then we should have in the first quarter, a significant data set on the functional aspects as well as the safety and tolerability aspects were 9001 to treat Duchenne muscular dystrophy. Across both Study 102 and an interim analysis of 301, we would have functional results showing in a placebo-controlled manner that the therapy provides functional benefits to these children and then it’s method is working and it’s benefiting them, we would have a very significant data set on safety and tolerability across the two studies. And then of course, with biopsies and that’s one of the things that we get here, but in some programs you don’t get, like for instance Zolgensma in that program, there are no biopsies. But here we have the opportunity to get biopsy. So that means in the first quarter of next year, we could have it be an interim analysis, not only all of the CMC work that we’ve already done showing that in all of the material ways that how operate in the same way as the clinical material. But also we would have actual empirical in vivo biopsy data, showing the expression levels as well as safety and tolerability. And on that basis, we would certainly intend to approach and discuss with the agents, the opportunity to submit for a BLA. But that will be a discussion that we’ll have with data in hand in the first quarter of 2029.

Thank you. And I am showing no further questions from the phone lines at this time. I’d now like to turn the conference back over to Doug Ingram for any closing remarks.

Thank you very much. Thank you all for participating this evening and thanks for the questions. We really appreciated the opportunity to answer them. I’ll repeat – apologies. I will repeat what I said in my opening remarks, which is simply we have a lot to do as an organization. We’ve had a lot of success. We’re very excited about the progress we’ve made to date on our gene therapy engine. We’re very excited about the opportunity to continue to serve our Duchenne muscular dystrophy community with our two approved therapies. And hopefully if approved three therapies by the first quarter of next year and we’re very excited about the potential for our next generation RNA technology PPMO to deliver even more profound impact both in Duchenne muscular dystrophy and beyond Duchenne muscular dystrophy, the other areas where steering blocking technology could bring therapeutic benefit for patients. We have a lot to do. So as much as we are excited about the progress we’ve made to date, it would be a mistake for us to develop hubris or complacency. And hope you would agree with me that the team at Sarepta does not intend to do that. This is a team that’s very focused. I’m really proud of our team. I’m very proud of our partners. I’m very proud of our clinical investigators who have continued to serve these patients during what has been for everyone, a trying time over the course of 2020. We’ll stay on mission and I look forward to providing additional updates as we progress across 2020. With that have a wonderful evening and the rest of the week.

Ladies and gentlemen, thank you for participating in today’s conference. This does conclude the program. You may all disconnect. Everyone have a wonderful day.