Good afternoon, ladies and gentlemen, and welcome to the Sarepta Therapeutics Third Quarter 2020 Earnings Call. At this time all participants’ lines are in a listen-only mode. After the speakers’ presentation, there will be a question-and-answer session. [Operator Instructions] 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, Catherine, and thank you all for joining today’s call. Earlier today, we released our financial results for the third quarter 2020. The press release is available on our website at sarepta.com, and our 10-Q was filed with the Securities & Exchange Commission earlier this afternoon. Joining us on the call today are Doug Ingram, 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 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 annual report on Form 10-K and 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. 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, everyone. And thank you for joining Sarepta Therapeutics third quarter 2020 investor conference call. Tonight, I’m very proud to share with you the progress we have made this quarter, including our performance and serving the Duchenne community with EXONDYS 51 and VYONDYS 53, our progress with respect to our RNA platform and the strides we’ve made in building out our enduring gene therapy engine. But, I’m going to take things a bit out of order this evening. I’m going to commence to this call by discussing an important milestone for Sarepta and for the Duchenne patient community. As you will have read in our press release today, having worked with the FDA, we now anticipate dosing a trial with commercial process material this year. Let me provide some background and context. As you will know, SRP-9001 is our gene therapy, and the goal is to treat Duchenne muscular dystrophy by safely delivering the skeletal, diaphragm and cardiac muscle, a gene that robustly codes for a truncated functional form of the structural protein dystrophin that we call micro-dystrophin. Children with DMD make essentially no dystrophin, which results in rapid degeneration and inevitable death, and the goal is to arrest this degeneration by giving them back a properly localized, functional form of that structural protein. After many years of design and preclinical testing by doctors, Jerry Mendell and Louise Rodino-Klapac, we commenced and completed a small four-patient proof-of-concept study, Study 101, that was positive and very encouraging. The therapy was well-tolerated; the gene was indeed robustly delivered to the target muscles, about 3.3 genome copies per nucleus. The expression of the protein approached nearly normal levels, and on all functional measures the children continued to improve in ways that cannot be explained by natural history. Based on…

Thanks, Doug. Good afternoon, everyone. This afternoon’s press release provided details for the third 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. Now, starting with our net product revenue for the third quarter of 2020 from our products EXONDYS 51 and VYONDYS 53, was $121.4 million, compared to $99 million for EXONDYS 51 alone for the same period of 2019. The increase primarily reflects higher demand for our products. In the quarter ended September 30, 2020, we recognized $22.5 million of collaboration revenue, which relates to our collaboration arrangement with Roche. The co-development costs under the Roche agreement totaled $16.9 million for the third quarter and are included as a reduction for our operating expenses. On a GAAP basis, we reported a net loss of $196.5 million and $126.3 million or $2.50 and $1.70 per basic and diluted share for the third quarter of 2020 and 2019, respectively. We reported a non-GAAP net loss of $111.5 million or $1.42 per basic and diluted share in the third quarter of 2020, compared to a non-GAAP net loss of $84.4 million or $1.14 per basic and diluted share in the third quarter of 2019. In the third quarter of 2020, we recorded approximately $15 million in cost of sales, compared to $13 million in the same period of 2019. The increase is primarily due to increasing demand for our products. On a GAAP basis, we recorded $190.4 million and $133.9 million in R&D expenses for the third quarter of 2020 and 2019, respectively, which is a year-over-year increase of $56.5 million. This increase is primarily related to a $42.5 million increase in manufacturing…

Thank you very much, Dr. O’Neill. Catherine, let’s open the lines for Q&A.

Thank you. [Operator Instructions] And our first question comes from Brian Skorney with Baird.

Hi. Thank you. This is Jack dialing in for Brian. So, just one question really from us. What do you expect to see from the 10-person clinical trial you’re expecting to initiate? And will the data be strong enough to show its effect compared to the data that you have in Study 102? We know there’s significant variability within the first four patients of data looking at the Phase 1 trials for the DMD product. And we’re just wondering what the lower and upper bound of confidence are with respect to the dystrophin measurements that you’re looking at for this new planned clinical trial? Thank you.

So, first, let me answer the latter part of the question first. 10 patients is very sufficiently powered to obtain the data that we’re interested in obtaining. Let me explain what this is about. So, let’s go back. Remember, for some time, we had been thinking about a larger placebo-controlled trial using commercial material, and we’ll still do that at some point as well. There was a number of reasons for that study, which is called Study 301, of course. One is global purposes. The other is additional confirmatory data. Remember, we’re going to have Study 102 that’s going to read out at the beginning of next year. So we’re already going to have results from Study 102, both on safety and function of the construct. But the other thing that we would get out of Study 301 is larger study and acutely the thing that we are most interested in to get as quickly as possible is validation of that commercial material. This is the same construct, but generated using a different process and a different manufacturing process, as you know, and I saw those units. And so, what we had planned with respect to 301 is to get what we really are most interested in right now. We would do an interim analysis of a subset of those patients around the same subset is what we’re actually going to do with 103 early next year. So, we would start that study, but we would actually get the real data that we’re curious -- we’re most interested in right now, which is the validation of our commercial material, both on expression and on safety early next year. And then, of course, in the summer, as we thought this through, we watch the external environment, we thought about the risks…

Our next question comes from Tazeen Ahmad with Bank of America. Your line is open.

Doug, just to stay on topic with Study 103. So I just want to make sure I understand this. You were not doing a specific potency assay, but you are using Study 103 in lieu of a specific potency assay. Just want to confirm that. And then, secondly, do you still have to do Study 301? Is it still part of your assumption that you will need to submit study 2 plus now Study 103 and some data from 301, or is it your plan to go talk to FDA to see if you can apply, assuming that the study 2 and 103 show what you want those to show and not necessarily have to do 301?

Thank you for the questions. Let me clarify something that I might have created to confuse you about. Now Study -- we have come to an understanding with the agency on the assay approach to Study 103. As you may recall, as we said at the time that we had the Type C meeting, we had an approach that we thought was appropriate with respect to the potency release assay for Study 103. The division had questions and disagreed with the approach that we were taking. We didn’t have clarity on precisely what that -- those issues were. And if we had gone through a formal process, it could literally have taken 2 to 6 months to ultimately have enough clarity to know where to go. By the end of September, we had an informal meeting with the agency, we understood much better what their issues were, and we were able to quickly propose essentially a new potency approach that they found acceptable. We then gathered the data for that. That’s already done and completed. And so, we have the potency approach now for Study 103, and that allows us of course to start Study 103. With respect to Study 301. So -- and Study 103 will get us exactly what we wanted out of an interim analysis from Study 301. So, really, it is a very thoughtful approach to a pandemic, to gather the information through Study 103 and get the most salient information in the near-term when we get 102 read out, which is that in patients and in biopsies we could show that as we would predict from all of our CMC work, the material and Study 103 from a commercial material perspective will perform in a similar way, both from micro-dystrophin expression and also from a…

And our next question comes from Alethia Young with Cantor Fitzgerald. Your line is open.

This is Emma on for Alethia. So, given the agreement here with the FDA on Study 103 is kind of a leaner approach for getting the most important information for 9001 in the midst of COVID. Is there -- do you expect kind of similar flexibility for 003 in LGMD to support a streamlined pivotal program?

We won’t know that until we have conversations with them, obviously. So, I don’t want to make any presumptions. One of the things that we said is that we need to do a couple of things with respect to -- this is for limb-girdle, I assume you’re saying 9003. We have to do it to get the GMP material completed and released. We’re still working on that. We’ve actually got the process development done, and we’re running a process development run. We have a lot of analytical work to do as well. Obviously, the work that we’ve done recently with the agency is going to be very helpful to 9003. And then, with that in hand, we want to sit down with the agency and talk about the next trial and hopefully, what we would like to believe will be our pivotal trial for 9003. But, we’ll give an update on that early next year once we have that framed out. Obviously, as we’ve seen, given the pandemic in [indiscernible] the agency is the opportunities to have discussions with the agency are formal, and we’ve got to be very thoughtful about that. So, I want to make sure we have the right kind of information in hand when we have those discussions. And then, we can give a better view on what the pathway forward is for 9003.

Our next question comes from Anupam Rama with J.P. Morgan.

I’m sorry. I’m just a little bit confused. So, for Study 103, looking at safety and some of the assay comparability work, right, and I think that’s at 12 weeks, and you called that part one. So, are there additional parts of the study where you’ll be looking at more functional type of measures? And as the FDA indicated any sort of interest in seeing that before you have some of the regulatory discussions, right?

Yes. It’s a great question. No. Look, this is an open label study. The goal of SRP -- goal of Study 103 is to get essentially exactly what we would have gotten out of an interim analysis for 301. It is essentially precisely the same thing. The reason I mentioned that is part one is, of course in these studies, you’ll have longer term safety follow-up. And so, in a sense, the study maintained even after you get the primary readout from it. It’s the same with all of our studies. It’s the same with frankly Study 102. We’ll have Study 102, we’ll have the readout from a primary analysis, one year analysis that will be -- with the kids crossed over, et cetera, and we’ll look at the results, and we’ll see if -- as we have hypothesized, we’ll see a statistically significant and meaningful improvement in NSA versus placebo. And then, of course, that study will continue. Those kids will continue to be monitored from a safety perspective. So, that’s what we mean when we say part 1, part 2, but the real goal of Study 103 is essentially to get in a sort of a more straightforward, leaner way, the information that we were looking to get out of an interim analysis of Study 301, which was the previously proposed placebo-controlled trial.

Our next question comes from Gena Wang with Barclays.

So, maybe Doug, just wondering, what is the criteria for approval. Will Study 102 plus Study 103, would that be sufficient? And also, did the FDA already signed off the potency assay? And do you still need to do the release assay in order to get approval? And in the case you have to run Study 301, do you need to run another potency assay for Study 301?

So, the FDA signed off on our approach to the release assay for Study 103. I can’t tell you what the requirements will be precisely for an approval because as I think we’ve said a number of times in the past, talked about it, we’re not going to broach that issue and have those discussions with the agency until we have the data in hand. Let’s get through Study 102, let’s see how compelling the data looks from 102, let’s get through our commercial validation study, let’s see how compelling the micro-dystrophin expression and safety looks there, and then we’ll have that dialogue in telling the micro-dystrophin expression and safety looks there, and then we’ll have that dialogue with the agency and figure out what the appropriate pathway forward is. As you can imagine, we feel a great sense of duty and obligation to move as fast as possible. So, you would envision that we are going to certainly propose the leanest approach forward. But, that discussion will happen once we have the data available to us. As it relates to the release out there -- and this is the release out there. I want to be very clear, if I’m not making it clear. We haven’t approached the potency release assay. We’ve had the blessing of the agency on that approach. We have the data for that available and that’s -- so that’s why we get to start the study, and that’s why we get to move forward. And we’ve been able to essentially unpause what was a pause part of this program. That approach could certainly -- when we get around doing additional studies, 301 is only one of the other ones, we want to do a non-ambulant study as well. We could obviously very likely use the same potency release approach or we could use an updated one. As we’ve said many times in the past, one of the reasons we were so confident that -- we weren’t confident on the timing at the time of when we would be able to get this issue resolved with the agency. We were always confident that we would get it resolved. And one of the reasons for that is that we have a number of assays that could be potency assay. So, we’ll use the most optimized one at the time that we start additional studies, but certainly, one of the possibilities is to use the potency assay that we’re using for Study 103 because we’ve obtained the concurrence from the agency on that one right now.

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

I was wondering, have the sites that you were going to use for 301, led -- do they lead you to believe that it was going to be challenging to execute the trial? And, how do you feel about their ability to collect and evaluate data from 102 in this arm?

So, the short answer to that question would be, no. Sites were not coming back to us and saying that this will be challenging. But, to be honest, there is an extraordinarily high motivation from sites to participate in our studies for SRP-9001. It is certainly I think people are very excited about the potential for what this could mean for children with Duchenne muscular dystrophy. I think of situations where even in the raging portions of the pandemic, sites were -- even when sites were currently shut down, they were suggesting they could start immediately. So, the truth is, we had to make an independent assessment of this issue. And it really was -- this discussion really comes down to this. We want to start 301, we certainly do. We have a global ambition. It will be very important globally. And it will be good in the U.S. as well. I don’t think it’s just global. But, the thing that we really acutely need is that right after we have Study 102 results, we need to have results from a commercial material validation study that shows that in patients that we’re getting the same kinds of micro-dystrophin expression, and we have the same safety. We have a lot of CMC work done that tells us that should be the case, and we feel very good about it, but we need to confirm that. And as we thought about that, and we’ve thought about this in the summer time frankly, we realized that we could do this as an interim look in a larger study or we could simply adapt ourselves to the environment we’re in and really create a lean study that gets that information to validate the commercial material from an expression and safety perspective directly. Let’s just…

Thank you. And our next question comes from Colin Bristow with UBS.

So, first one, I understand the agency hasn’t fully blessed the faster market, but in terms of the willingness to accept this 8 to 10 patients is sufficient evidence of comparability between the commercial and clinical product. Have they signed off on that? And then, just quickly on the timing of Phase 3 initiation or 301, it seems like 103 is going to ring fence this commercial to clinical products question. And just, I guess, why not initiate the 301, either in parallel or as soon as you can get the results of the potency assay issue ironed out?

Yes, a couple of things. One, I want to be very clear about this. We have not had discussions with the agency about the sufficiency of evidence to support a BLA. We’ve been absolutely on purpose. And I think, I said long in advance of the Type C meeting that we explicitly don’t want to have those discussions until we have data available. I don’t want -- with very busy agency, I don’t want to have those kinds of discussions, but at theoretic level I want to have it with data. And then, with respect to 103, 103 was designed originally because of the pandemic to get us the information we really need in the leanest way, without risk to the program and more importantly, without placing risk on children, including placebo kids, who would be having to go into doctors’ offices with the potential of being on placebo. So, there is a number of reasons why -- we talked to the agency informally. We talked about the commencement of Study 103. The reason we are -- we have proposed to the agency actually that we would consider a commencement of Study 301, once we had 103 in hand, and they concurred in that that concept. There’s a lot of reasons for that. One, it’s the leanest way to get to the information we’re most interested in right now, and it allows us to focus on the information that we want most acutely, which is the validation of the commercial material, which will marry up with the results of Study 102 and then have discussions with the agency. Two, it reduces the risk to the program and to the patients along the way. And then, three, of course, we might learn stuff from 103 that could inform the way we designed 301. And so, it made sense to us to really focus on Study 103. That’s what we focused on in our informal discussion with the agency. And that’s what we got the blessing, the comments.

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

Hey, guys. Thanks for taking my questions. So, could you clarify, is FDA sign-off on the actual potency data that you’ve generated using the agreed upon approach dating for the start of Study 103 or for ultimate approval? And how do you view, I guess, the difference in potential risk, based on your discussions and any regulatory precedent, to seeking approval using open-label rather than placebo-controlled safety and expression data from commercial scale material? Thanks.

A couple of things. We have the data in hand. So, we feel very confident that we’re going to commence the study. We don’t -- we don’t believe that we [indiscernible] gating items other than just getting it executed to commence Study 103, from our viewpoint, given what we’re looking at for the commercial validation study, the fact that it’s open label versus placebo really isn’t significantly relevant. We’re looking at expression. Obviously, you can’t placebo -- you can’t get us a placebo effect on expression. And we’re looking at safety as well. So we don’t think that’s going to be a significant delta. But, we haven’t had the conversations about the pathway to approve. We’ll have those discussions at the appropriate time of the agency with data and that we’ll know when we know it.

Our next question comes from Ritu Baral with Cowen. Your line is open.

Doug, help me understand this. What assurance or I guess general messaging you have from FDA that the 10 patients that you’re thinking about the 103 is going to be large enough to capture the potential variance of the commercial product and the safety of the commercial product, just given what we’ve seen with the size of compound and complement in some patients and not others, what do you have in writing from the Type C meeting on the end of 10? And also, now that you have, it sounds like you have the potency assay settled, what you’re going to use, having it validated, do you have all the clinical product you need to potentially run 301? I want to make sure that you’re not going from 70 to 10 because of a lack of validated product?

Yes. That’s a really good question. Thank you for asking that. We do not have a material shortage problem. So, thank you. I hadn’t even considered that someone might have imagined that would be the case. We have done many GMP runs. We have sufficient material to start Study 103, 301 et cetera, non-ambulatory studies as well. So, none of this relates to the unavailability of material. So, thank you actually for giving me an opportunity to say that. I haven’t considered that someone might have worried about that. The issues -- the decision about the size of the study is us. It really isn’t the FDA. The division doesn’t essentially bless it or confirm that they agree with the analysis that we’re doing, and they haven’t, and we wouldn’t have asked them to do that because really, it’s -- that is our decision. I mean, the risk around the size of the study is ours. I will note, however, that the size of this study is essentially exactly the size of what we would have envisioned in an interim analysis for Study 301. So, this is essentially the same thing we were going to do with Study 301 in the form of an interim analysis on a subset of patients. We were kind of looking at the 8% to 12% range, and that’s where we are now at 10 patients in this study. And that relies on our own evaluation. And we are confident about the size of the study, and we’re confident about the fact that we’ll get insight and validation around commercial material, both expression and safety out of it. You could touch on something, I just -- you are well aware of this, but I’ll say it yet again. So, we’re absolutely clear. We have -- I know that other programs have seen very troubling issues, for instance, completing [indiscernible] likely never seen those issues. And there’s no reason to believe that we would see anything like that in -- out of our commercial process materially here. And so, we’re very confident about that. But, that’s never been an -- that’s appeared with any of our rh74-driven programs.

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

A couple for me, largely following up and just clarifying some things that were asked before. First is really just to clarify, is there specific data you’re waiting for from Study 103 that gates the start of Study 301, or in a non-COVID world, could you go ahead and start Study 301, if you wanted to since the FDA sort of aligned on the potency assay? And then, I have a follow-up after that.

Yes. Thank you. I mean, we proposed Study 103 because of the COVID issues. And frankly, to be honest. It’s only -- we proposed this in the summer, it’s only become more compelling recently. It does appear, unfortunately, certainly in the United States, and probably around the world right now that the second wave is upon us. So, it only becomes more compelling to us. When we met with the agency in our formal discussions at the end of September, we proposed and they concurred in the concept of the Study 103 and we actually proposed that we would start 301 after we had data from 103 and they concurred that. So, that’s the decision that we made in light of where we are. And we think it makes ton of sense. And there might be something that we learn out of 103 that could actually inform 301. But,, we’re very focused on getting the information that we’re most interested in right now, which is validating our commercial material, both in expression and safety in children with Duchenne muscular dystrophy, and having that information as soon after the readout of Study 102 and hopefully as close in time as is possible, so that we have together, at the same time, essentially three things. We have -- from a placebo-controlled trial, we have the compelling evidence that the therapy is providing significant benefits to these kids, and believe for right, statistically significant and clinically meaningfully. And then we have safety data. And then, of course, with respect to the commercial material, we have commercial validation that we’re getting the kinds of expression we would expect out of the commercial material. We’re seeing the safety signs out of the commercial material that we will see out of the Study 102, at least, what we’ve seen certainly so far. So, we’re really focused on that. And then, we’ll have a discussion with the agency, and we’ll talk about the pathway to getting this therapy to these kids who are, as we all know, waiting, and time is not on their side. So, we’ll certainly meet with the agency as soon as we have that information on our books.

[Operator Instructions] Our next question comes from Tyler Van Buren with Piper Sandler. Your line is open.

I had a question on the MOMENTUM trial readout by year-end. I was just hoping you could refine expectations there. I think, you mentioned specifically, you’re going to compare digital drop PCR, the 12-week PPMO data to 24-week PMO data. So, is the ultimate goal at this initial readout to see skipping that is higher with the PPMO product at the early time point, or are you hoping to see several magnitudes higher of skipping, could you put a finer point on that? And are you dose-escalating because maybe the skipping is not where you would like to see it yet, and you think by getting to 30 or 40 you’ll be able to achieve that ultimate goal, even though it appears that you’re seeing great safety?

So, I would say, first of all, don’t read much into -- don’t read anything into my comments about what we’ve seen because we haven’t -- the reason we haven’t released the 20 mgs per kg data is that as we don’t have the data in hand right now. So, I’m going to turn this over to Dr. O’Neil to provide some color on what we’re going to look at between the two. I’d say a couple of things. It would be nice to see some additional exon skipping, even though it’s in a different time point, between the PMO and the PPMO at 20 mgs per kg. But the reason that we’re dose-escalating is because it has always been our goal. The goal is to get to the highest possible dose we can get to before we see a dose-limiting toxicity, so that we know that we are creating the most exon skipping and dystrophin possible. And we’ve seen in the animal models that if you push the dose high enough and you can do so safely that at least -- if the animal models bear out, you will see a very substantial -- eventually, very substantial increase. Where that exactly is, it’s correlating between humans and animals is always difficult, is it [indiscernible] dosing is it late base dosing, is this a hybrid between the two. So, we don’t know yet, but we’re very confident in the mechanism of action. And so, I will say, one more thing and then I’m going to turn it over to Dr. O’Neil to give more data-driven color on this. This is all about safety for us. We’re really -- what we’re curious is one of the safety signals, the 20 mgs per kg, what’s the change we can go to 30…

Our next question comes from Danielle Brill with Raymond James.

This is Anil [ph] on for Danielle Brill. Thank you for taking our question. Staying on the topic of PPMO. Good question in terms of dystrophin expression. As you’re dose-escalating, are you collecting the dystrophin expression data? And do you have any idea when you might be able to share that? Thank you.

We’re going to have an update on the 20 mgs per kg at 12 weeks. Dr. O’Neill, you’re going to correct me if I misdating anything. It’s 12 weeks with the PPMO. We’re actually going to compare it ironically to the PMO at 24 weeks. But, we’re going to have an update on exon skipping and tissue exposure at 12 weeks. We’re not looking for dystrophin right now for the simple reason that it is very early. You can get some lead of exon skipping in an early stage, but one of the things we know with certainty is that dystrophin builds over time, and 12 weeks is very early to start looking for dystrophin. We’ve seen with our PMOS and EXONDYS as an example, we’ve seen a significant difference in the amount of dystrophin that was produced, literally at four years versus one year so, nothing will be that long, but 12 weeks is a bit early to look at dystrophin. With that, Dr. O’Neill, do you want to add any additional color on that? Dr. Gilmore O’Neill: I can confirm that what Doug has said is entirely accurate. And the only thing I will remind you of is that we have demonstrated non-clinically that there is a proportionality between exon skipping and dystrophin expression downstream. But, as Doug has said, 12 weeks data is very early for dystrophin expression. And as we have learned in the past with our PMO, dystrophin does accumulate over time. And so, that is the reason that from a proof-of-concept and a -- proof-of-concept point of view, we are focusing on, first and foremost, tissue distribution or tissue concentration as well as exon skipping at 12 weeks.

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

This is Sonia [ph] on for Salveen. Where do you stand with longer term strategy on DMD with next-generation approaches using genomic medicine?

Dr. O’Neill -- first of all, I’ll say this, we are, of course, in addition to our gene therapies, in addition to -- both our PMOs right now and our next-generation PPMO, which we’ve just touched upon, and in addition to gene editing, and I should tell you, we have a gene editing innovation center in Durham, North Carolina, under Dr. Charlie Gersbach, that’s looking at a number of different things, including the potential for gene editing and Duchenne. I think that’s a bit out -- a bit off in the distance. There are a number of other ideas that we’re looking at as well that I don’t think I come to fruition yet. But if you have any additional thoughts on that, Dr. O’Neill, let me know. Dr. Gilmore O’Neill: I think you said it nicely. The key things are that we actually have a nice portfolio of platform approaches with gene delivery, gene editing and our antisense And I think that choice -- certainly, when I think about gene delivery pending gene editing, further the revolution, and our antisense enables patients to make optimal choices we hope in the future. And obviously, they complement one another. And I think, as we’ve said before, you remember, we are even looking at the possibility of combinations of these therapies in the context of optimizing therapies for patients with Duchenne. And I will actually also highlight what Doug said earlier is that obviously, the other thing we want to do with our portfolio for Duchenne patients is ensure that we develop the data for patients at various stages of their disease, just to make sure we can maximize benefits for as many people as possible. Thank you.

Thank you. Our next question comes from Joel Beatty with Citi.

This is Sean Egan calling in for Joel. Circling back to 9001, it seems like not much has changed today in regards to what you guys hope to expect to eventually file that that being a positive 102 study and positive bridging results. But maybe can you confirm that there’s nothing new from the FDA discussions, which suggest that a positive 102 and positive bridging study alone would not support approval? And also, has blinded 102 data helped inform the design and scale of Study 103 in any way?

I’d say, Study 101 really has informed both 102 and 103. And again, just lingering for a moment, I want to make sure I’m very clear about the fact that we haven’t had no discussions with the agency, just solicit their views on what the data would be necessary for, for instance, the BLA side, I don’t want to mislead. But, I do agree with you that we are in the same position with our approach today as we would have been -- as we were thinking about Study 301, which was an interim analysis. Probably the one difference -- so two differences, one, if you roll the clock back of course, we were under the impression that the potency assay approach that we had as we tracked to the September meeting was appropriate, and that has caused us a couple of months delay as we sorted that out, got new data that difference. But, I must say, in fairness, and I really want to give tons of kudos, both to the teams and the willingness of OTAT, to talk to us, we were able to resolve those issues in advance of 103, as fast as I think it is possible to do. I think, in light of how busy that agency is and in light of pandemic and the ability to actually get up informal meeting with the division so soon after our Type C meeting and to come to a resolution, literally by the end of September, I think has said a lot about the team and its willingness to execute and its execution ability and it also said something about the division’s willingness to have conversations with us. And I really appreciate that. I think, the one additional thing that I’d say between where we are and…

Our next question comes from Hartaj Singh with Oppenheimer.

This is Jackie [ph] on for Hartaj. Thanks for squeezing us in. Maybe just a clarification question. Since you noted in your earlier prepared remarks that you’re going to pursue additional studies in older and non-ambulant boys, could you just clarify whether those will be separate from Study 301 or cohorts added to that study? Thank you.

There would be a separate study on non-ambulatory patients, at least that’s the way we envision it today.

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

So, just asking a clarifying historical question. So, back in the days, before all this discussion on potency test started, when the original plan was to go to the FDA with study -- with results from 102 plus interim readout on 301, did agency -- did the FDA ever confirm that will be enough to you form the basis for BLA submission, or was it at the point just enough to start the conversation about potential submission?

It’s always been our strategy, I think we’ve communicated this often that we will have those discussions with the division once we have data in hand, and that until we have that in hand, we won’t have discussions about the level of evidence necessary for a BLA. So the simple reason that I think it’s asking a lot of the agency in the middle of all how busy they are, the pandemic and the like to have theoretical discussions. We’ll get 102 executed. We’ll get the results of 102. We’ll get the material from our commercial validation study. And then, we’ll talk to the agency about the path forward with initial studies or a BLA or the like. I think, everyone knows what our goals are in that regard. But, we’ve been very consistent from that perspective.

Our next question comes from Matthew Harrison with Morgan Stanley.

This is Max Skor on for Matthew Harrison. Just a quick question. Is Study 103 gating at all for initiating discussions with the agency regarding the limb-girdle 2E trial with commercial material? More specifically, is there any read-through from one potency assay there?

I don’t think there’s any gating -- there’s no gating item between Study 103 and the discussions with the agency about limb-girdle 2E. No. I think that there’s no doubt that the discussions we’ve had with the agency about the potency assay approach will inform the approach we take with limb-girdle 2E. So we’re in much better shape as a result of those discussions. We can apply those -- that learning to the limb-girdle 2E to which without a doubt, the real gating item to have discussions with the agency limb-girdle 2E is getting the GMP material released. And that requires us to continue to do analytical work, and work that is well on the way, but not completed.

Our next question comes from Martin Auster with Credit Suisse.

Hi. This is Matt Terwelp on for Marty. Thank you for taking the questions. I just wanted to confirm the Study 103 is a single center study? Thanks a lot.

I believe it is, but I’m going to let Dr. O’Neill confirm that for us. Dr. Gilmore O’Neill: The final number of sites will actually be determined as we execute the study. We have actually built in redundancy because as Doug had said, one of the things we did and the reason that we actually conceived of the 103 Study was to actually handle the -- or deal with the consequences of the ongoing pandemic. And so, we have actually built in flexibility, so that we are able to enroll and execute the study as quickly as possible.

Thank you. There are no further questions in the queue. I’d like to turn the call back to management for any closing remarks.

Thank you all very much for joining us this evening. I will say, I’m very pleased with the team for continuing to execute during this pandemic, both in the performance of our proof therapies as well as our development programs. As it relates specifically to SRP-9001 is of course, the top of mind for Sarepta because it is such an important program for Duchenne muscular dystrophy boys that are waiting. We did have a setback in September as one could expect to have it when you’re ambitious. We had hoped in September that we would have a blessing to commence Study 103 in September. But, I am very pleased to say that we executed consistent with our culture. We moved rapidly. We were able to get an informal meeting with the agency at the end of September. We were able to come to a meeting of the minds on the commencement of Study 103 and the potency release assay associated with Study 103. We could not have, I think, envisioned a faster resolution of this issue, so that we can commence with little delay the next commercial validation study in an extraordinarily important program. And so, I’ll continue to update people across the course of the year as we execute. I want to give a lot of thanks to OTAT certainly for their willingness to engage with us and help us find the pathway forward. I want to thank the Sarepta team for their willingness to continue to execute fiercely across the organization and serve these patients. And I of course, want to thank the patients, both those who participate in our trials as well as those patients across all of our disease states, both Duchenne muscular dystrophy and currently limb-girdle muscular dystrophy for their commitment to the programs but also for staying with us as we continue to execute these programs. We have a single-minded goal as an organization. Our success will come from our ability to intervene in the lines of patients who are suffering from these rare diseases in far, far too often with respect to Duchenne muscular dystrophy and these limb-girdles being stolen from their families by a shorter life because of these diseases. Again, we will work like mad to move as fast as possible. And I do believe that Study 103, which we proposed and was accepted by the agency is going to be an enormously important step forward in our ability to accelerate the therapy to patients that are waiting as soon as the science cooperates. With that, have a lovely evening. And we will continue to update across the course of the year and into next year. Thank you.

Ladies and gentlemen, this concludes today’s conference call. Thank you for participating. You may now disconnect. Everyone, have a great day.