Welcome to the Q3 2014 Sarepta Therapeutics Incorporated Earnings Conference Call. My name is Paulet and I will be your operator for today's call. (Operator Instructions). Please note that this conference is being recorded. I will now turn the call over to Sarah Bard. Sarah, you may begin.

Thank you, operator; and thank you, all, for joining today's call. Earlier today, we released our financial results for the third quarter of 2014. The press release is available on our website at www.sarepta.com and our 10-Q was filed this morning. Joining me on the call today are Chris Garabedian, Sarepta's Chief Executive Officer; Sandy Mahatme, Sarepta's Chief Financial Officer and Ed Kaye, Sarepta's Chief Medical Officer. I would like to note that during this call, we will make a number of statements that are forward-looking, including but not limited to statements about our plans to work with the FDA to achieve an acceptable NDA submission and filing for eteplirsen, including our plans and ability to comply with FDA requirements and requests for data, information and analyses. Our plans and ability to include such information in an eteplirsen NDA submission and the projected timing for the same. The potential and timeframes for an NDA filing by the FDA and regulatory approval for eteplirsen; the potential for eteplirsen to qualify for accelerated approval through pathways in the EU; potential for further development of our Ebola product candidate; availability of drug product and number of treatment courses we could potentially make and the expectation that any Ebola development efforts would not impact efforts in our DMD program. Our release relating to the potential safety, use, clinical benefit of and market for our product candidates and platform technology and communications with regulatory authorities, government entities and international bodies relating to the same; exploring development opportunities with various third parties and updated guidance on expected non-GAAP loss from operations. These forward-looking statements involve risks and uncertainties, any of which are beyond Sarepta's control. Actual results could materially differ from these forward-looking statements as any such risks can materially and adversely affect the business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties we face, you're encouraged to review the company's most recent annual and quarterly reports and other official corporate documents filed with the Securities and Exchange Commission. With that, let me turn the call over to Chris Garabedian, Sarepta's Chief Executive Officer. Chris?

Thank you, Sarah and good morning, everyone. Today, I would like to provide an update on our recent communications with the FDA regarding an NDA submission for eteplirsen, our lead product for the treatment of Duchenne Muscular Dystrophy or DMD along with an update from Dr. Ed Kaye, our Chief Medical Officer on the status of our broader DMD clinical program and the various studies that are underway in process or in discussion. Our corporate update will be followed by an update of our latest financials by Sandy Mahatme, our Chief Financial Officer. First, I'd like to address our recent communications with the FDA related to our pre-NDA guidance and meeting minutes that was outlined in our October 27th press release and conference call as well as a subsequent posting by the FDA on their website on October 30. We've received a tremendous number of questions from investors, the patient community and even congressional staff regarding these communications and I'd like to make a few comments in response to these inquiries with the hope that we can focus on the events that are ahead of us and allow us at Sarepta to work constructively with the FDA to ultimately achieve an acceptable NDA filing for eteplirsen. In particular, I would like to address recent disclosures related to our FDA discussions, comments related to the dystrophin message and data set and the specific guidance related to our NDA submission. Since the public disclosure of our 48 week clinical data from study 201, 202 in October of 2012 and the subsequent initiation of discussions with the FDA that have continued over these two years to determine the feasibility of an accelerated approval of eteplirsen. We’ve understood the intense interest among investors and the patient community in our discussions with the FDA and…

Thank you, Chris. Good morning, everyone. I will now provide an overview of all of the activities related to our DMD clinical program and the progress we've made since our last update. During the past several months, the teams at Sarepta, our CROs and the individual respective sites have been working very hard to initiate four new clinical trials. These trials include study 301, our open-label eteplirsen confirmatory study in ambulant patients greater than seven years of age, study 204, our open-label eteplirsen study in non-ambulatory DMD patients or those who are more progressed and don't meet the minimum walking distance on the 6-minute walk test for study 301. Study 203, our open-label eteplirsen study in younger DMD patients between the ages of 4 to 6 years and finally, our study testing the exon 53 skipping drug, SRP-4053 consisting of a two-part study, part one being a blinded, placebo-controlled, dose titration phase followed by a larger open-label Part II extension study. As most of you know, there are many time-consuming aspects involved in the study startup. The sites need to be identified and qualified prior to the initiation of the process. Investigators, physical therapists, health authority and IRB approvals, informed essence and consents, contracts, budgets, all need to be established for each site. IRBs will frequently ask for clarification of the protocol and specific language for the essence and consents need to be adjusted for each institution. An investigator meeting and site initiation visit are required prior to screening the first patient. Also, prior to collecting efficacy data, the physical therapist at each site must be certified. Working with our CROs, we’ve assembled a very experienced Sarepta team of clinical operations experts, physicians, statisticians and data managers who have previously worked at many of the Massachusetts based biotech and large…

Thanks, Ed. But first related to our DMD program, I'd like to restate that we have final drug product of the drug that we need to begin dosing all of the patients across these clinical studies and our manufacturing runs have produced quality drug product that have met the release specifications discussed with the FDA for our eteplirsen studies. As I mentioned on our regulatory update call last week, we have had our pre-NDA CMC meeting and received our meeting minutes with no significant concerns raised related to what we discussed, related to our CMC section for the NDA. Turning to Europe, we’re tentatively scheduled to have a meeting with the EMA and waiting for a confirmed date from them on the pathway to discuss the pathway for eteplirsen in Europe and the possibility of a conditional approval based on our existing and emerging dataset. Before turning the call over to our CFO, Sandy Mahatme for a financial update, I'd like to address the many increase we received related to the status of our Ebola drug AVI-7537 and activities related to the ongoing outbreak as well as provide an update on our pandemic influenza program along with other research efforts, all of which underscore the versatility of our PMO chemistry platform. First, I would like to touch on the ongoing Ebola outbreak as we've received a significant number of inquiries and interest over the past few months from investors, analysts, government agencies, the media and most recently several hospitals and treatment centers across the country regarding the status of our Ebola program, our drug supply and our ability to potentially help. The Ebola crisis has estimated to have already affected more than 13,000 people and resulted in over 4800 deaths reported so far this year. We’ve continued to keep government…

Thanks, Chris. Good morning, everyone. This morning's press release provided details for the third quarter of 2014 in both an adjusted or non-GAAP basis as well as a GAAP basis. The press release is available on the SEC and company websites. The non-GAAP results we will discuss on this call provide a more accurate picture of our ongoing operations and the impact of operations on our cash balance and they exclude the impact from the valuation of our outstanding warrants and stock compensation expense. I would like to point out that all warrants have been exercised or have expired as of the end of the third quarter of 2014. Please refer to our press release for a full reconciliation of GAAP to non-GAAP. In the third quarter of 2014, we reported an adjusted or non-GAAP net loss of $28.8 million or $0.70 per share compared to non-GAAP net loss of $21.3 million or $0.63 per share in the third quarter of 2013. The incremental loss is primarily the result of an increase of $4.5 million in R&D and G&A expenses due to corporate growth and a decrease of $3.1 million in revenue from government contracts. Revenue for the third quarter of 2014 was $1.1 million down from $4.2 million in the third quarter of 2013. The $3.1 million decrease was primarily due to lower revenue from our various government contracts. The government contract under which the Marburg drug candidate was being developed expired in July of 2014 and we’re currently evaluating options to continue advancing our Marburg drug candidate and other infectious disease research and development efforts. Adjusted research and development expenses were $20.2 million for the third quarter of 2014 compared to $19.9 million in the third quarter of 2013, an increase of $0.3 million. Adjusted general and administrative…

Thanks, Sandy. Operator, we're happy to open the call to questions.

(Operator Instructions). And our first question comes from Tim Lugo from William Blair. Please go ahead.

When you present the fourth biopsy results, are you going to also be presenting these alternative methods which were discussed by Kauffman between yourselves and the FDA?

Well, with the fourth biopsy, we would intend to do multiple dystrophin measures. I think western blot will definitely be part of that, immunofluorescence will be part of that. Obviously we want to have a portion of it to be consistent with what was done previously for comparison, reasons to the existing data set, but also to be able to correlate other measures of dystrophin, obviously the method that you mentioned is another way to quantify. And so, we will be determining what other exploratory measures beyond the standard measures we will consider. And so we’re discussing all of that internally before we actually finalize that protocol.

And I think just to be clear, I think some of the newer methods are indeed that of new or they haven't been validated. We're looking at everything. I think the (indiscernible) and has to be taken out. So it's a slightly different way of doing in western, but in essence what we're trying to do is find the best way to measure the protein. And we're in the process of doing that and obviously working with a lot of different people to make sure that we're all in agreement that we have the best methodology.

I know there were questions around independent assessment of the dystrophin images, you mention also -- I think what I don’t quite understand is, you also mentioned in the press release independent assessment of the 6-minute walk results as well as maybe some of our clinical pulmonary function data you'll be taking on at week 168, can you walk me through how that will be independently assessed?

No, Tim, that's not accurate. The reassessment was specific to our dystrophin analysis and specific to the dystrophin-positive fibers. They linked the need for a 168-week clinical data in that passage that was outlined in our press release and the only context further that they wanted was for us to attempt to get all of the 6-minute walk data from the natural history cohorts and you know we’re attempting to do that.

And our next question comes from Jeremiah Shepard from Credit Suisse. Please go ahead.

First off, is how much data from the week 168 analysis do you plan on releasing now that you have this updated guidance in the FDA and if so when and where might you do that?

Yes, we're not ready to provide guidance on that at this time. Part of this I explained on the last call is first we need to ensure that when we're going to get that 168-week data, we did provide flexibility to extend that 168 timeframe through when that fourth biopsy would be collected, we thought it was more important to prioritize, scheduling that fourth biopsy and this is a big burden on the families and so what we wanted to do was give the site flexibility to not demand two separate visits all of these boys and families are flying in from around the country for both the clinical visit and the biopsy. And so some of them can't find the time to do that and so we gave them flexibility. So we're not even sure when we'll have that final data set. We will determine over the coming months if when, what venue that 168-week data would be presented.

Okay. And based on the recent FDA commentary on that they made about the dystrophin sustaining, can this commentary be included in a new methodology that you want to carry out for the week 168 analysis, or is there any additional and separate validation work needed to confirm whatever commentary that are asking for -- I mean any additional procedures they're asking for?

Well, Jeremiah I think you're referring to fourth biopsy that isn't necessarily directly linked to the 168-week data but would be approximate to that timeframe. Look, we want to continue to enhance and improve the quantification and the capture of dystrophin in these muscles and so I outlined earlier, all of the different ways that we're going to take assessments of dystrophin on these fourth muscle biopsies and again we think it just will add to the overall evidence that we provided to-date using various methods of our drugs mechanism of action in production of dystrophin. So again we continue the dialogue with the FDA as we have been over the last year and a half as it relates to dystrophin methodologies quantification, it's an evolving field, of course, we want to stay on the leading edge of that but also make sure we rely on validated measures and proven ways of capturing this. Again none of these methods are perfect right, these are imperfect measurements but we believe as a collective and in totality and if the level of dystrophin are significant that, that's the most important thing that our drug is producing dystrophin in the muscle and that forms the basis or the foundation of the support of the clinical outcomes that we're seeing.

And Jeremiah I think one of the -- at least in my experience, it's not unusual for the FDA to have a lot of questions about a new circuit endpoint or a biomarker. And so there is a great deal of effort, and it should allow them to understand and remember we're working with some of the most experienced investigators in the world who have been doing this type of analysis for decades. So for the FDA to try to understand it very quickly -- they have a lot of questions. And so this is not unexpected in a lot of things that they are asking are fairly customary like asking more than one reader to do the analysis. So I think what we're trying to do is educate everyone so that the FDA can understand what's done and what is really the state of the art. And I think we're making some progress and I think having the collaborators that we're working with goes a long way in allowing that to happen.

Okay and just one last question, in terms of the MRI results that the FDA was asking for in the last communication, you previously mentioned that 11 or 12 boys in Phase IIb study had received an MRI at some point previously. How longer were these exams completed? And can you also comment if you know this like in terms of like how much the muscle biology might change in that period?

Yes. So Jeremiah, look the FDA requested this MRI data that we tried to capture it for the first time in this recent communication, we will attempt to do that. Again this was not a study that was conducted by Sarepta nor data that we own internally. And I think it is a promising exploratory measure, I think we don't know enough about what to expect or what we would see related to a treatment effect. I think it's an incredibly potential strong natural history dataset as it relates to MRI, but this is going on for a couple of years now. Ed, do you want to elaborate?

Yes. So, this is a large multi-centered trial and Chris (indiscernible) from University of Florida and (indiscernible) have been the major investigators on this. And so, what they were -- the attempt was is to understand what happens in muscle on MRI over time. So we participate, we allow the participation of some of our boys in this and it's really ongoing, they are collecting data. So they get MRIs every year in a yearly update. And so, all of this data has just really become available and we're looking at it. No one has ever looked at change in MRI to any treatment and so, this is obviously very exploratory. We think it could be very interesting, but we're in the process we've asked for more data to be able to compare and that's ongoing and we should have the data to be able to submit to the NDA.

I mean one thing that we thought was interesting. Ed and the team here was able to go down and look at some of the eteplirsen boys images. And one finding that was pretty market was that the two non-ambulant boys in our study look significantly different in terms of their muscle mass relative to the other boys who are still walking. So it's supported and seem to validate why these two boys had lost ambulation and why they may have been too far gone to have had an impact on their walk test.

Our next question comes from Brian Klein from Stifel. Please go ahead.

So on a Confirmatory Phase III study, can you just give us your expectations for when you would complete enrollment in that study?

Yes. So I think as we mentioned, there is a lot of work that has to be done in preparation. So we're now really seeing the fruition -- it's fruition of all of the work. So we have, as I mentioned, we have four sites initiated and another four to be starting soon and patients will be starting dosing in this month. So I think, based on our projections obviously it's always challenging, but I think what we're starting to see is, there has been a lot of interest from the community and from patients and patients are being lined up. So as soon as we have those centers online, obviously then we can make a better projection is how long it will take to enroll.

Yes. And again with the eteplirsen open label study, we do expect to enroll as many as we can as quickly as possible. And it will afford us to look at analysis potentially if we have 40, then we have a longer experience. We could do those types of analysis versus the untreated cohort. So I think our goal of 60 in the primary analysis up to 80 if we include those that would be outside of the primary analysis, we will provide appropriate updates as we understand when we could achieve those specific targets.

Okay. As I am looking on clinicaltrials.gov, it suggests that the primary evaluation would be completed by May 2016, which would essentially imply that you could complete enrollment by May 2015 to allow yourself some time to analyze data. Is that a fairtime frame for us to think about for enrollment in the study?

I wouldn't base our expectations around that. I would say that would be an optimistic target for enrollment. But again, it could be that we're comfortable with a number of patients that we can optimally get in that timeframe to do a final analysis that meets that timeframe. But again, we're not providing guidance on that at this point and would not try to interpret what that guidance might be relative to that date on the clintrials.gov website.

And then, just one final question in terms of the trial for the elderly or the older DMD patients, can you give us a sense of what your expectations might be in terms of a clinical benefit with eteplirsen treatment? Thank you.

Yes. And again, in these patients who don't qualify for 301 or are non-ambulatory, the name, clinical outcome measure is really looking at pulmonary function. We're looking at a lot of exploratory endpoints in regards to upper extremity. So I think given the small number of patients, this is really -- we would really want to make sure that in this population, it's safe and obviously, we will be looking at, let's say, the clinical outcome measures as we go along. So I think right now, we don't have any specific expectations.

Yes. The older, non-ambulant is not intended for an efficacy endpoint. Again, it's a smaller sample size. It's an open-label study; it's a more heterogeneous population. As we already mentioned that they're going to have some who might be walking but more advanced in their progression, some that are non-ambulant, some that are further along with more compromised pulmonary function. So we just wanted to ensure that there was safety. The other thing I would mention is that we have some of the best clinical data in the non-ambulatory population as it relates to a disease-modifying therapy or dystrophin-producing technologies and that the two boys in our study have now been followed on drug assessing clinical outcomes for over 2.5 years now in that non-ambulant state. So again, we believe we're already getting some informative clinical outcomes on those two non-ambulant boys in our current study.

Our next question comes from Ritu Baral from Cowen. Please go ahead. Ritu Baral - Cowen & Company: To restart with the manufacturing status, you mentioned that you have supply to start the trial; do you feel that you're in a good position to supply pretty much the entire trial right now and are you on track with previous expectations that you would be able to supply a significant portion of the community by end of the year, next year?

Yes. So, Ritu, look, we're very comfortable with our manufacturing capability and the reproducibility we have seen to-date that gives us a high level of confidence in our ability to continue to provide drug supply for our trials and to prepare ultimately for commercial approval. With the recent guidance and the delay in our NDA submission, we're going to be looking hard at our gating of when we would need what drug supply. So again, we're also trying to make sure that we hold back because it's one of the most expensive cost drivers we have as a company and to make sure that we’re always focused on what drug we need, give ourselves some margin of error, but not to overproduce a drug because it is an expensive endeavor for us. So I think right now, we’re on track. We don't have any concerns about providing drug supply for all of these studies on an ongoing basis and at the time that we have a better sense of the commercial launch of eteplirsen, we believe we have sufficient time to prepare for that full commercial demand. Simply put, right now while there is no guarantees, we do not believe manufacturing capacity or capability is a gating item to our success with our Duchenne program. Ritu Baral - Cowen & Company: And what is the shelf life of the current clinical drug product right now? How long could you keep it around?

Look, we’ve different levels of stability on our drug substance. Usually, you have a shorter amount of time on your drug product that is in vials. We have obviously longer-term stability on the small-scale batches we've had. We're accumulating more and more stability on our mid-scale batches. We're not providing any specific on all of those batches and what level of time, but what I would say is that we've described the stability that we have and that we would have at the time of an NDA for the FDA and there were no issues that were raised that would prohibit us from moving forward within NDA, having said that with this delay we want to ensure that any updated stability data we have would be included at the time and also the demand for this drug in the market at this point would not preclude us from meeting a shelf life of many years, but by the time we needed that we believe we will have sufficient stability. We also can draw on our other products that we have been producing under GMP conditions for clinical trials, you know, West Nile virus was one of the first trials we did as a company, more than a decade ago. And we recently did some HPLC testing on that within the last couple of years and it had very good stability. So there is nothing that suggests that we're going to have a stability problem with this drug product moving forward, but we continue to accumulate that data as we go. Ritu Baral - Cowen & Company: And a last question, given your comments on the path forward in the EU in the patent situation there, what do you think the path forward would be in at one point -- what point is the intellectual property situation even considered by the regulatory authorities there?

Well, the first the thing we've been saying all along is that -- the first thing we need to do as it relates to Europe is determine what is needed to get the drug approved. And I've said previously that if we were handed a free and full license for eteplirsen in Europe tomorrow it would not change our current activities because we still need to find out if the existing data set is sufficient for approval, what additional data would be needed, how would they perceive a conditional approval of eteplirsen, if we were to get that guidance even under the most aggressive timeframe where we might have an MAA for conditional approval submitted next year. We're still looking at a long approval timeline and ultimately reimbursement approvals throughout Europe to get eteplirsen utilized and there is a lot of time to determine the appropriate pathway and freedom to operate for eteplirsen in Europe. So again the regulators, their job is to look at the data set efficacy and safety and determine the approvability based on that, it's the European patent courts of which we have filed appeals and that's in the queue that would be determining if the opposition on 51 is overturned.

Our next question comes from Brian Skorney from Robert W. Baird. Please go ahead.

So I guess one of questions I’ve is speaking with some of the parents prior to the most recent set of FDA communications or sort of this assertion that they would agree to fourth part biopsy under certain conditions from the FDA, notably that the FDA would sort of at least intend on acting on the results. And I'm just wondering have you talked to the parents subsequent to the most recent round of communications with the FDA and do you think there is any risk that you won't be able to get a family as to agree to the fourth biopsy now?

Brian, we're not trying to go direct to the families in our studies, we’re trying to state publicly, why it's important? We're trying to convey to the site while we believe it's important and to make sure that when Jerry Mendell and the staff at Nationwide understand our reasons for wanting to pursue the fourth biopsy. What we've stated previously is that while absolutely the FDA is important as a customer and to ensure that we do this fourth biopsy with as best we can, the methodologies and the quantification that would be satisfactory to the FDA, they are not our only customer who would be interested in this. Remember, if we were to submit the fourth biopsy data into an NDA, even if the FDA presented concerns or questions about the fourth biopsy data to an advisory committee panel, we would make our best argument to an advisory committee panel and we believe that could potentially be a compelling data set to get an independent advisory panel comfortable with the drugs activity and the supportive data of the clinical outcomes that we're seeing. On top of that, we would hope to see that as part of a package insert or something that we could discuss with payers, okay. In addition to that, we think that's something that's important that clinicians who are going to be thinking about using this product would find important that three years after a drug initiation we're still finding evidence of dystrophin in the muscle biopsy. So absolutely, the FDA assessment of the fourth biopsy is important. They are not the only customer we're thinking about in capturing these fourth biopsies.

And our next question comes from Steve Brozak from WBB Securities. Please go ahead.

Since most of the questions have been asked and answered, I actually do have one question, specifically the parents, what kind of feedback are you getting from the parents that are affected right now? I'm kind of curious, because obviously these people have been waiting on for quite a while and what in essence are you getting from them and the physicians?

I mean when we provide these communications, we first and foremost hear from those who have been following Sarepta and investors who are interested in our communications and clarity around our communications. And so in the two days following our communications that was a priority, the analysts that you -- all of you on this call also want clarity, so you can provide your reports. There is media interest in clarifying this to the biotech industry. We obviously have staff that try to reach out to clarify for the advocacy organizations and the patients who inquire about what this means, the timelines, etcetera. But again, our first priority is to make sure that we are clear in the guidance we're receiving from the FDA, clear in our intention for how we're going to pursue the approval of eteplirsen and then to the extent that the community needs clarity on our communications, we provide that, okay. All along, of course, we've stated, I think the FDA would agree with this, is that nobody wants to hold up an effective therapy from patients, okay. And I mentioned on the regulatory update call, it really doesn't matter if there are parents that are convinced that this drug would be useful and safe for their boys, it doesn't matter that physicians would like this drug to use in their patients, what matters is the FDA is comfortable with the dataset and that they believe it meets their requirements and guidelines to approve this drug and that is our first priority and of course, we’ve the patient in mind when we're trying to move as quickly as we can. We’re disappointed too that there are any delays in our clinical trials because of all of the things that Ed described are essential to having a good quality program. But again, we provide our communications and the patients and the community is going to respond as they may and there may be different responses from the patient community. The other important thing to keep in mind is the FDA themselves have opened their doors and communicate directly to the parents. So it's not as though the community is getting all of their information from Sarepta. The FDA is providing direct communication to advocacy organizations and parents directly. So that's the best way I know how to answer the question you're posing.

One last follow-up on that, everything you've told us obviously during this conference call and everything you've relayed in printed communications basically says that you effectively are set up to start producing product in the quantities that will be needed into the future. Can I take that message as something that you're comfortable with?

Yes, well, you know we've been saying for many months now that we would be prepared to satisfy the full commercial demand in the market, assuming the previous timeline of mid-2015. Now however, with that timeline moving out, right, we also want to make sure that we're going to be producing the drug at the appropriate time that we can provide it to the broader community upon a commercial approval and so, again with all of the caveats and risk factors outlined in our SEC filings and associate with any company, trying to manufacture large quantities of drugs. With all of that, there is nothing that we see where we sit today that would be a barrier to providing that grade [ph]. But ultimately until you produce those levels of drug, you don't know, but we're confident based on the reproducibility of the runs we've had to-date, the consistency of the quality of the drug product. Again experienced, the more experience you get with doing these runs, the more confident you are that it's working and that we understand if there is any tweaks along the way that need to be done that we're aware of that. But to-date, we've had very good results and we're confident in our manufacturing moving forward.

Our next question comes from Christopher Marai from Oppenheimer. Please go ahead.

On first, maybe we could just touch upon the biopsy data from the potential fourth biopsy. I was just wondering under what kind of different analysis are you thinking to look at these. Specifically are you looking to have a control group, either healthy normals for untreated patients? Has the FDA provided any guidance with respect to that?

Look, we’re going to be working with the FDA first and foremost to share. We've shared and outlined and we’re trying to work on the specific protocol. But we have communicated all along that we intend to not just look for one specific measure or one specific methodology of dystrophin quantification, but multiple measures. And what we hope is that, the multiple measures that we can show that dystrophin is present in quantities that are larger than what would be expected from Duchenne, controls or banked samples or again the data that we’ve generated from this specific population at baseline in previous biopsies. Again, we're trying to build the totality of the data to show dystrophin. But again, there has been a lot of attention appropriately on dystrophin. But I want to remind everybody that we have a very, what we believe compelling clinical dataset across this population that we presented, Jerry Mendell presented at World Muscle across 6-minute walk test pulmonary function and again over this, 3-year timeframe that we've reported to-date, we think they're behaving very different than what natural history would suggest. The FDA is urging, we're trying to get more detailed matched natural history data and we're pursuing that, but based on all the literature, I think there is a compelling case to be made clinically and that's not even factoring in what the families are saying or happening with these boys who are in the eteplirsen trial and that becomes a very compelling component as well. As we start new trials, we’re going to get new safety data, we think with across all of our eteplirsen studies, we will have more than a 100 eteplirsen patients targeted for drugs. So we’re going to get all of these questions answered over time and for the fourth biopsy we just want to try to get as much additional dystrophin by a variety of measures as possible.

Okay. And then another question, just regarding the EU potential conditional approval or approval path, just reminds us is the EMA on board with your confirmatory trial design for eteplirsen? Did you consult with them regarding that? And then additionally, have you discussed the potential for that master protocols follow-on candidates with EMA?

No. This will be our first guidance meeting with the EMA; we've been saying that all along Chris. So again, they've given us some tentative dates. We're trying to confirm those dates that would happen before the end of the year and all of these issues will be discussed. And again, we believe that the broader clinical program that we have for eteplirsen should be sufficient for the EMA. Recall, they have supported or exon-53 study, which is an open label experience study looking at biopsies, looking at clinical outcomes after the short titration placebo controlled phase. We know looking at our competitor exon-skipping drug that their second drug in I believe is an exon-44 drug, the EMA supported an open label study there. So there is a lot of evidence out there that supports that the EMA may be even more flexible than what the FDA has been.

And then one last question here on patient screening, I'm just wondering if you could comment a little bit more on where that screening is relative to your expectation and if you don't have enough data yet on that, you know what sort of your anticipated enrollment curve, do you expect Ebola patients to enroll right at beginning or the majority of this sort of enrolled towards the end of that enrollment?

Yes, Chris, we're not going to provide that guidance at this time. A lot of this is obviously -- we've been working with the various sites. The sites have been working on identifying patients, we obviously don't know how many of them will qualify, how many may drop out by inclusion-exclusion criteria. So there is a lot of factors that control for that and once we’ve a better idea after we get more sites up and running after we look at what -- how many patients are making it through the inclusion-exclusion criteria, that's when we can provide better clarity on that. Ed, do you want to add anything?

Yes, I think, Chris, you know what we would like to do is we -- you make kind of an arbitrary projection of how many patients you can enroll and then we have to match that with -- depending how many sites are up and running unless they are -- the average number of patients that are enrolling per site. So usually we don't like to make projections this early in the course and so we have some data to extrapolate. So we should have that later.

Okay. And so that the numbers on the anticipated patient's enrollment per site, you might provide that later, is that correct or you could you provide that now, if I'm understanding it correctly--?

Yes. Once we have better information and we're confident with the type of guidance we can provide on enrollment we will, that's not something we're prepared to do right now.

Got it. So one last really one question and do you anticipate that you will share data from the confirmatory trial given that it's an open label trial? Thanks.

Yes, Chris again we will provide guidance on any further updates and data from our current study, from our ongoing studies are up and running again. So stay tuned for -- look we haven't even dosed our first patient yet and so we know that there is some time before we can see a clinical outcome effect that would be different than natural history. And we want a large enough sample whether we wait for full enrollment, our target enrollment or whether we look at our interim analysis, we’ve not determined that yet. So, again as we get more information we'll provide the appropriate guidance.

And our next question comes from Robyn Karnauskas from Deutsche Bank. Please go ahead.

Tis is Evan on for Robyn, thanks for taking my question. And can you just remind us why you opted not to do a rolling review despite the FDA seemingly suggesting that this type of review could be appropriate in the recent statement from last week?

Yes, so I think thanks for the question because I think this needs some clarity, we understand what a rolling NDA is and any discussions we've had with the FDA about a rolling NDA has been clear and again we're not taking a rolling NDA off the table. What we've been talking about previously from our previous FDA discussions was not a rolling NDA, but the idea of additional data submitted after an NDA is submitted or filed and when we talk about an NDA, we mean the full and final NDA not a section of the NDA. And I think everybody understands that a rolling NDA in the first section that submitted does not change the clock, okay or the PDUFA timeline, okay. So the clock starts and the assessment of whether an NDA is fileable or not would start when all of those NDA modules are in. With all of the additional data that we will be compiling and the additional stability data described for our CMC section, we’ve at least four sections, okay, that would be waiting for additional data for submission. There may be one section that we could submit prior to but we want to look at it in its totality before we make that decision about a rolling NDA. So I just wanted to clear that we understand what a rolling NDA is, we understand the optics of looking like we’re getting one of the sections in. But the main argument that is often used with a rolling NDA is, hey, you could give the FDA a head start, they can start looking at data before you have your full NDA in. Now, there is some debate whether they have the resource to actually put against a section of an NDA before they even…

And one just follow-up, in regard to the -- I guess discrepancies in information regarding what's needed for this dystrophin analysis and their advice in the April or May site visit, they said that they've been giving consistent advice to you all along, I guess what's the disconnect here?

Yes, look, we had our prepared remarks, I gave an extensive overview of all of our communications, we stand by our public communications and our press releases and our disclosures related to all of the FDA correspondence. And again, we would really like to move forward regardless of what might be perceived as disconnects or inconsistencies, go back and look at all of our communications, we've been consistent, clear and stand by our public communications and we're not going to engage in further trying to tease out how people perceive the differences of what the FDA has said versus our communications.

Our last question comes from Kumar Raja from Citi. Please go ahead.

This is Kumar in for Yaron, thank you for taking my question. So in U.S., Sarepta and Prosensa have a patent dispute relating to freedom to operate exon-51 and also there is a question about the first company getting approved might block the other under orphan drug exclusivity. Can you comment on both of this, especially given that Prosensa is going to file for approval before Sarepta does?

Yes. So look, we've already talked about the patent opposition in Europe and where that stands and we filed our appeals related to the European Patent Opposition and Appeal. In the U.S., you're probably referring to the interference. Again, we've had all those disclosures out there and so that interference process we've described in previous calls and how that plays out. Our current patents remain valid and it does not impact our efforts to pursue an NDA filing and a drug approval. And ultimately, in terms of orphan drug exclusivity, we believe that these drugs are not the same. They have a different chemistry, they have a different sequence, they have a different dataset and so we think by all of the different measures, a different chemical entity that the drug would not -- any first approval would not preclude another drug from getting to market.

Great. Thank you.

Okay. Well, thanks, everybody, for your interest in Sarepta. Again, we've tried to be very clear with respect to our recent communications with the FDA and our path forward. We're focused on that. As Ed described, we're really focused on getting more boys on eteplirsen in our clinical studies and also focusing on the follow-on exons and we've got a lot going on here and I appreciate your interest in Sarepta and stay tuned till the next call. Thank you.