Welcome to the Second Quarter 2014 Sarepta Therapeutics Incorporated Earnings Conference Call. My name is Allen, and I will be your operator for today’s call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session. Please note that this conference is being recorded. I will now turn the call over to [Hannah Reswood]. Ms. Reswood, you may begin.

Thank you, Operator, and thank you all for joining today’s call. Earlier today, we released our financial results for the second 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 the timing of and submission of information and data we are planning to provide to the FDA in support of our planned NDA filing, including results from our ongoing Phase IIb expansion study, potential biopsies and the timing of the same, and ongoing safety data collected. Our beliefs relating to the used clinical benefit and importance of eteplirsen safety profile for treatment decision by healthcare professionals and families, our assessments relating to remaining on track to submit an NDA for eteplirsen for the treatment of the Duchenne Muscular Dystrophy by the end of the year, our planned meetings with the FDA relating to CMC and the NDA filing and clinical trials we are currently planning, and our optimism that the FDA will be flexible with trial designs and the use of a master protocol, our plans to submit an IND for exon 53 skipping candidates, SRP-4053 later this year. The potential portion of the DMD population that we believe can be treated with our product candidates targeting exon 51, 45 and 53. Our plans for meeting with the EMA later this year to discuss feasibility, a conditional approval based on the existing eteplirsen data set and expectations on receiving feedback, our plans to complete live scale batches…

Thank you, [Hannah] (ph). Welcome everyone. Today we will provide a comprehensive overview of our broader DMD, Duchenne Muscular Dystrophy program, including an update on the clinical, regulatory and manufacturing activities for eteplirsen and the latest on our follow-on exon skipping drugs for DMD. We will also discuss our infectious disease programs including recent activity related to the Ebola outbreak and our discovery research activities as we prepare to build the pipeline based on our proprietary PMO chemistry. To begin our update on our DMD program, let me first provide a brief summary of our recent 144-week data, which we announced on July 10th. The topline data we released from our ongoing Phase II extension study, Study 202 was a summary of six-minute walk test and pulmonary function outcomes and the safety profile for eteplirsen through 144 weeks. We continue to be encouraged by the performance of the 12 boys in our study, all of whom were showing unexpected outcomes in the six-minute walk test and/or pulmonary function test compared to the natural history of this disease. These data were especially encouraging given the average age of these patients and our selection criteria, which excluded boys who were less progress on the six-minute walk baseline. It’s important to note that at the time they entered into this study, all of the boys would be expected to have suffered significant loss of muscle and muscle fibrosis, and we are anticipated to decline during the course of the study based on the natural history of DMD. The 10 boys in our modified intent to treat analysis, which included all patients who were still ambulate and could perform the six-minute walk test remained ambulant through 144 weeks and decline more slowly than would be expected compared to natural history studies and compared to…

Thank you, Chris. We have been very busy over the last few months working on the essential regulatory and clinical activities related to eteplirsen and our follow-on exon skipping drugs for DMD. First, let me describe our activities as they relate to our eteplirsen confirmatory study in ambulatory DMD patients who have genotypes that are amenable to exon 51 skipping. We have a final protocol that was reviewed by the FDA in early June. Shortly thereafter, we submitted the protocol and informed consent to the sites that we selected for potential participation. Contracts were sent sites for review in early July and we conducted an investigator meeting with staff from 36 of the sites in mid-July for a two day review of all of the aspect of the study to ensure that there is broad awareness and understanding of the study protocol and that the trial can be conducted efficiently and with consistent high quality. Right now, the final protocol is making its way through either local, institutional IRBs or central IRBs depending on each site’s standard operating procedures. We have reached out to 39 sites for potential participation with as many as 14 regional hub sites, which will conduct clinical outcome measures, including the six-minute walk test and pulmonary function test. In addition, we have plan for two of these sties to conduct a surgical biopsies. We have adopted this approach through hub spoke model to ensure that the best quality data is captured across train sites with a higher level of consistency, while attempting to minimize the burden on families by having many more infusion sites for dosing on a weekly basis. Clinical outcome measures will be obtained every 12 weeks for the first year. We’ve also completed our protocol for the eteplirsen study in the limited ambulation…

Thanks Ed. Before turning the call over to our CFO, Sandy Mahatme, for financial update, I'd like to address the many inquiries we’ve received related to our ebola drug AVI-7257 and activities related to the recent outbreak as well as provide some insights on our programs to treat Marburg hemorrhagic fever virus and pandemic influenza along with the research update, all of which underscore the versatility of our PMO chemistry platform. To be clear, these programs are all in early stages of development and in no way impact our efforts to get eteplirsen or other DMD candidates to the boys who need them. First I’d like to touch on the ongoing Ebola outbreak as we proceed a significant amount of inquiries and interest over the past week from investors, analysts, government agencies and the media regarding the status of our Ebola program and ability to potentially help. We have offered to lend assistance to fighting the Ebola crisis that is estimated to have already infected more than 1700 people and resulted in over 900 deaths so far this year. The confirmed reports regarding the use of a “experimental” drug as an emergency treatment for two American citizens who became infected while providing humanitarian aid and medical assistance in Africa have put a spotlight on the handful of companies and organizations that have developed Ebola vaccines or antivirals which include Sarepta. Over the past few weeks, we have been in contact with government officials at HHS, the CDC, FDA, BARDA, Department of Defense, State Department and the White House as well as NGOs or non-governmental organizations, including the World Health Organization about our willingness to help based on our existing inventory of drug. By way of background, we received Department of Defense funding from July 2010 through October 2012 for an…

Thanks, Chris. Good morning everyone. This morning's press release provided details for the second quarter of 2014 in both, an adjusted or non-GAAP basis as well as the 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. Please refer to our press release for full reconciliation of GAAP to non-GAAP. In the second quarter of 2014, we reported an adjusted or non-GAAP net loss of $24.5 million, or $0.61 per share, compared to a non-GAAP net loss of $14.6 million, or $0.46 per share in the second quarter of 2013. The incremental loss is primarily the result of an increase of $9.9 million, due to increase R&D and G&A expenses. Revenue for the second quarter of 2014 was $2.6 million, down from $3 million in the second quarter of 2013. The $400,000 decrease was primarily due to decreases in revenue from our various government contracts. The government contract when they reached the Marburg drug candidate was being developed, expired in July of 2014 and the company is currently evaluating options to continue advancing as Marburg candidate and other infectious disease research and development efforts. Adjusted research and development expenses were $18.3 million for the second quarter of 2014 compared to $12.2 million in the second quarter of 2013, an increase of $6.1 million. Adjusted general and administrative expenses were $9 million for the second quarter of 2014 compared to $5.3 million in the second quarter of 2013, an increase of $3.7 million. Our cash, cash equivalents and short-term investments balance was $284.2 million…

Thanks, Sandy. As you can see, we’re quite busy at the company across clinical, regulatory, manufacturing and pre-commercial activities, all with a clear focus and priority to execute on our DMD program and so the boys who need them. With that operator, we’d like to open up the call to questions.

Thank you. (Operator Instructions) Our first question is from Tanya Joseph with Bank of America Merrill Lynch. Please go ahead.

Hi. Good morning. I’m wondering if you could provide us with any incremental details on the proposed study design for the follow-on exon confirmatory study. And in addition to that, is the timing of this proposed sort of I’m going to call master protocols study consistent with the open-label historical controls study, is that fair for us to assume? Thank you.

Yeah. Thanks, Tonya. So, I’ll let Ed speak to the timing at a moment. The study design was not finalized yet but it’s going to be very similar to the other studies you’ve seen, which looks at the 6-minute walk test as the primary endpoint, capture of dystrophin at various time points 24 weeks, 48 weeks. It will be a placebo-controlled study unlike our historically controlled study. And again, we believe as you go beyond the eteplirsen-amenable patients, the numbers that you'll need to have an adequately powered study becomes more challenging. And we believe that the way this technology behaves is that the pharmacokinetics and safety, we’ve seen today across three drugs in preclinical studies are almost identical. And we expect a standard dose and these drugs to behave the same way. And so, we think that combining and pulling these two next drugs to enhance the statistical power of our analysis would be beneficial. Of course, we could always look secondarily at the individual drug performance to see if there is any differences across them. But this is what we just need to work out the final details. But it will look similar to other study, we’ve done in the DMD space. However the timing is a little delay to our eteplirsen study.

And I think it’s important to remember that what we’re trying to do is really to expedite this program. And so we're looking at the long-term picture and how can we do this as quickly as possible. And obviously, we’re trying to maximize all the study. So by combining two drugs together, it does give us an advantage. We obviously have to have discussions with the FDA, if we’re in the process of doing that. I think, we’ve had a very good collaboration with the FDA to try to be innovative and come up with innovative trial designs. So we’re looking at this at the beginning of the year. But I think we’re maintaining our timelines pretty well.

Thank you. So just to confirm, is that the beginning of the year apply to the open label study as well?

No. The open label is going to be initiated earlier than that.

Yeah. The open label study were very well advanced on that. So it will be very close to screening patients and that’s well on its way. So that will be definitely at third quarter this year.

That’s for September, October, got it. Thank you very much.

The next question is from Tim Lugo with William Blair. Please go ahead.

Thanks for taking my question. Maybe I miss this in the opening comments but have there been an update on looking for an additional biopsy from current patients. I believe previously it had been mentioned that this could occur by year end?

Yes. So we have -- we talked with the sites and they have sent out a request of how many patients in there. As of last night, 8 of the 12 patients had said they would be interested in the biopsy. So we’re pursuing that. We've completed an amendment for that protocol yesterday. And we’re adjusting the process of trying to arrange all the details to get that done. So that we can -- again, this is just another way of trying to get the FDA comfortable that we have dystrophin. We are very interested on the long-term production of dystrophin and so this is a unique opportunity for us to really understand what’s going on with dystrophin. So yes, it’s well on its way.

And this is being done in coordination with the FDA. I believe that was maybe discussed earlier the methods and I guess, collection protocol for the fourth biopsy.

Yes. We’ve had a number of discussions with the FDA. We submitted the protocol as we want to make sure they’re comfortable with the process and so far things again here have been going well.

Great. Thanks.

The next question is from Brian Skorney with Robert Baird. Please go ahead.

Good morning, guys. Thanks for taking my questions. I guess, sort of just on the couples on confirmatory study, sounds like the protocol has been approved by the FDA? Is there a predetermined threshold for success in the study? Is there a timeline for one primary endpoint would be read out? Any sort of color on exactly what the FDA believe you need to have or what your expectation for success, this study would be helpful?

Yeah. Brian, we’ve share this previously and its consistent with the April guidance letter we’ve received from the FDA. The FDA did not put specific guidance around the time frame nor around the specific endpoint that would be compared to historical controls. Essentially it was enrolled this group of patients and we will follow them on an open label basis and whether that is determined at a year or 18 months or two years, whether that’s going to be comparing 6-minute walk or pulmonary function or a constellation of outcomes or other outcomes to historical controls. That remains open and at the FDAs discretion as we continue to have dialogue around this open label study. However, we as the company decided to put in a untreated cohort into the study with a specific endpoint 6-minute walk, that will be evaluated in a specific time point 48 weeks. But to be clear the FDA is not holding us to that outcome to determine if the drug is showing utility or not. We believe it’s appropriate to do because we think we can show something. But the FDA again is not going to hold us. If we fail to show the 6-minute walk benefit compared to our untreated cohort at 48 weeks, we will simply continue to follow these patients on an open-label basis, until we have sufficient evident to show they’re behaving differently and if the FDA agrees then historical controls. Again, this would be to confirm the benefit should -- eteplirsen get an accelerated approval next year.

As also we have, of course, secondary endpoint of the pulmonary functions, so we’ll be looking at that very carefully. And that's obviously a new endpoint that as we’ve discovered these boys have been stable over now three years. And we will be obtaining biopsy. So I think there’s a lot of information that we will have that we can present to the FDA. And I think the FDA is really looking at what is the totality of the data and so we are going to have biopsies. We’ll have pulmonary function. We’ll have 6-minute walk test data. And hopefully, that will make them comfortable that this is having a therapeutic effect.

Got you. And then just on the USPTO interference proceeding required in the quarter, can you give me color on what sort of claims potential was giving seniority on the interference with these just broad anti-transfer for DMD claims or are there any oligo-specific or [animal links] (ph) that are found to be interference? Any sort of detail on that would be helpful.

We’re not going to comment on the details of the interference but to be -- but the USPTO not in action initiated by percents. And to be clear, the interference declaration doesn't impact any of our development or submission plans. Our pattern claims are issued and remain valid for both the interference proceeding and subsequent appeals. And again, Prosensa’s patent claims are pending and will not issue into a patent that can be enforced unless it prevails at the conclusion of both the proceeding and subsequent appeals. And then there’s other steps beyond that. They have to convince the USPTO that its applications are patentable. So again, we just simply disclose that this interference was declared by the USPTO and will provide information that’s relevant as we understand it as time goes on.

Okay. Thanks, guys.

Thanks.

The next question is from Christopher Marai with Oppenheimer. Please go ahead.

Hi. Good morning, guys. Thanks for taking my question. First, just with respect to some changes at the management level, I was wondering who's now managing the preclinical program given the CSO departure? And remember going back in interactions, maybe four years ago, Dr. Peter O'Hanley was quite capable on that respect and I just wonder if he is still with the firm and also how much involvement he has with respect to the preclinical sections of the NDA? And then also is there any loss of continuity with respect to the NDA and FDA discussion, following the CSO departure? Thanks.

Great. Thanks, Chris. Actually, we have a good team of senior leaders in charge of research and preclinical at the VP, Senior Director and Director levels. Yes. So Dr. Peter O'Hanley was previously in-charge of our preclinical program. He was essentially the architect of all of our preclinical studies was the point person with the FDA on all of our preclinical and dystrophin discussions through the April guidance. Every meeting we had at that point was managed by O'Hanley. And again, our former CSO did not have any involvement through that guidance. So what we did was we had. Dr. O'Hanley, reassume all of those -- the oversight of all the preclinical. He had hired that team previously. He is back in-charge leading that team and involved in some other activities with the company across medical affairs and other communications. Related to the path moving forward, related to our NDA, again, most of that worked. The study reports, the sections of the NDA were managed, facilitate basically, put together by Pete O'Hanley and his team and they will continue to review and prepare. And frankly most of that section has been completed and is going through formal final review. So, this is why we communicated very clearly. We don't anticipate any interruption, lack of continuity across our plan with FDA and with our path toward NDA submission later this year that resulted from our CSO departure.

Great. Thanks. And then with respect to the confirmatory trial for eteplirsen, you mentioned 39 sites. I was wondering, are this all DMD centers of excellence? How are you training them? Obviously 6-minute walk test endpoint is important and we know difficulties in getting consistency across that endpoint in prior trials. Then additionally, what type of inclusion criteria beyond age and baseline 6-minute walk test are you actually including for that confirmatory trial, for instance the Gowers’ maneuver percent predicted. Thanks.

Yeah. Thanks, Chris. Obviously, we’re very aware with some of the challenges that have been found in other trials in DMD, and so that was the whole focus that we had as far as for our hub-and-spoke model. So we’ve contacted 14 centers of excellence and these are located throughout the United States. And specifically they have great deal of expertise in the 6-minute walk test. We’ve also screened all of these sites. We have a group of expert physical therapist that went to the sites, made sure that they had the ability and they had backup for the physical therapist. They could perform it in the same way, also for pulmonary function. And we’re trying to control all of these as much as possible, using the same equipment for the pulmonary function, made sure that everyone is trained. We have two centers of excellence from the biopsies. Again, as you recall quite well that had been a problem in other studies where the quality of the biopsies were not very good. It will be processed at one site. We have a computerized reading. So a lot of work has been done. So really make sure that all of the sites are trained because it's challenging for families, they have to travel a long distance for weekly infusions and we certainly appreciate the difficulty of this. We have included a number of sites throughout the country closer to where families are located, so that can reduce the burden. But I think, having these centers of excellence that do it -- do all the clinical outcome measures that really helps to reduce it. And I think, we’re obviously -- we’re looking in a very similar to what we’ve learned from the phase IIb and the population is going to be similar than we’ve revised it slightly. But again, trying to just get the most homogeneous population and I think we've been able to do that.

Great. Thanks for taking my questions, guys.

The next question is from Debjit Chattopadhyay with ROTH Capital Partners. Please go ahead.

Hey, good morning, guys and thank you for taking my questions. Question regarding before the biopsy, do you think the timing of the biopsy is going to be dependent on the FDA accepting the prior biopsies in terms of the way they evaluated and conducted before you go on with the fourth biopsy. And then a question regarding the European strategy, you mentioned, you are in discussions or will be in discussions with the EMA. How does that affect the patent position of Prosensa versus yours? Thank you so much.

Debjit, let me answer your second question first and then Ed can comment on the biopsy, fourth biopsy. So look, as it relates to our patent -- freedom to operate issues in Europe, obviously, what we need to first speak to the regulators, even understand what’s feasible about potential approval of eteplirsen in Europe and that’s what we intend to do. Obviously, once we have a line of site and clarity on whether or not eteplirsen could be approved on the existing data set, then obviously we would look to figure out how to resolve those issues in a variety of ways. And so our first goal though is, look, let me put it another way, if we were handed a free license tomorrow in Europe for eteplirsen, it would not change the activities of the company as it relates to talking to the EMA, finding out what it would take to get eteplirsen approved before we proceed with if they needed additional study in Europe with our confirmatory studies in the U.S. suffice. What is the timing that we would potentially be launching eteplirsen, potentially? So again, we’re continuing these activities and at the appropriate time, we expect, we would resolve to not see this drug barred from Europe. I’ll let Ed speak to the fourth biopsy.

Yeah. So the fourth biopsy is going ahead. I mean, this is very valuable information and clearly the FDA is very interested in stand dystrophin and how this could be used as a biomarker. So we’ve had a discussion about improved ways of looking at in measuring dystrophin, getting them comfortable. It would be done in another laboratory. So this is going ahead and we’re also looking for the panel. Remember that we not only have to convince the FDA but our expectations, of course, would be in advisory committee panel. And these physicians also are very interested in dystrophin. So we really want to make sure that we can demonstrate in multiple laboratories which show dystrophin on that. It’s at a reasonable production and then we have long-term expressions. So this is going ahead.

One more question. In terms of the confirmatory trial design, supposing, something goes wrong with the NDA, accelerated NDA filing and FDA’s rejection of that. How does that change your strategy for the confirmatory trial? Being that it such an open ended trial with no clear guidance as to the endpoints and timing from the FDA.

Yeah. Debjit, this is exactly why we took our own initiative to put in a harder endpoint and a untreated cohort because that gives us an insurance policy to have a study design that in any event that this is not approved next year that we have a good case to be made for a treatment effect versus again a population that natural history suggest behave similarly as our eteplirsen amenable patients. And also one that would have the same inclusion criteria that we would use for the eteplirsen treated patients. So that’s why we did what we did in the event that you described eteplirsen isn’t approved next year. I’d say, in terms of timing. I’m going to have to limit one question for the remainder -- the remaining. We still have a queue. So appreciate your question, Debjit.

The next question is from Chad Messer with Needham & Company. Please go ahead. Chad Messer - Needham & Company: Great. Thanks for taking my question. I was wondering if you could sort of update on your latest thinking about possible ways you could be approved. Obviously, the door is open here for dystrophin to be used as a surrogate endpoint. It sounds like you got a little bit more work to do to convince the FDA there, but that’s certainly possible? In the past, you’ve discussed that six-minute walk was also a potential path to approval, just wondering if you still view that is the case and if there is any sort differences in your eyes or any effect of being approved one or the other would have.

Yeah. Look -- I would just say the guidance that we received in April. April was very clear. We outlined that in the press release and a conference call and they laid out the two pathways that they could approve eteplirsen under the Accelerated Approval pathway. As you mentioned, one was in intermediate clinical endpoint from our current Study 201, 202, ongoing extensions study, not related on any new efficacy data from subsequent studies that will be ongoing or -- and/or dystrophin as a surrogate endpoint on the existing data set upon a more detailed review and as Ed described, we are getting a fourth biopsy. So, we believe those -- that guidance is still intact. We don’t believe that is impacted at all in fact our 144-week date and now we have almost three years of data, these boys are still ambulant, I just described earlier in the prepared remarks, why we think this is supportive of an efficacy claim. And Ed, do you want to comment further, we could be approved on either pathway.

Yeah. And I think it is a -- this is a clear interest in the six-minute walk test and it certainly has been use as a primary endpoint. And I think not only the FDA but the EMA is certainly comfortable with this is as an endpoint. And also remember this has been a great deal of interest in the pulmonary function. It’s been considered for other neuromuscular diseases as an endpoint and the FDA has even mentioned this as a potential clinical outcome measure. So I think we have a lot of different ways to demonstrate that this is working. I look at this is, multiple pathways that we could use to try to get this. So, I think, there is a lot of flexibility which is unusual for a program at this stage.

The next question is from Steve Brozak with WBB Securities. Please go ahead.

Hey. Good morning and thank you for this update. A lot of questions have been asked and answered, so I will just go down and cover one. You have taken great pains to talk about in this call and other calls, the patients critically interested? You just spent a good deal of time talking about the manufacturing and the regulatory? Obviously, you’ve tied everything up a very well? But the question I have is, are there any other areas that we should be focusing on in terms of a transition from, obviously, clinical development to what can -- what is the logical next step which is commercial? What are the other things that you might want to start to talk about and that we should start to monitor and I’ll have one follow-up after that?

Okay. Yeah. Look, obviously, as we prepare for an NDA submission, we have to also prepare for success and that means that if we submitted NDA by the end of the year. And we have a filing accepted by, let say, February of next year and if we get priority review that would put a PDUFA date at August of next year. So we are looking at a year from now, the possibility that we could be launching eteplirsen or getting approval in the U.S market. And so, we’ve been building out a commercial and outward facing medical affairs function over the last year. And we hired some very senior level talent who has experience launching products, experience in the rare disease field, experience in preparing reimbursement, access distribution. And we are just not by talking publicly about a lot of these efforts. But as we get closer to that NDA filings, we will be providing updates how we are preparing for the commercial launch of eteplirsen in the U.S. So, yeah, look, you see, our guidance has increased this year and this is consist with the companies that are preparing for success to get ready launch a commercial product and start generating revenue in the near-term. So, yeah, there is a lot going on the company across all the different functional areas including our pre-commercial activities.

And the last part on the manufacturing, again, I appreciate the deliberation that you took in terms of manufacturing? What are -- how temperamental is this or how much of the mast treat are you comfortable with on a manufacturing side? Obviously, that’s one of the major things that you spent time on this morning? Is there anything else we should look at it as far as that goes?

No. I mean, I think, for us the mid-scale production and the quality and the success of that mid-scale production. As I mentioned, eight batches now where we have the consistent quality and stability to able to use this drug in our clinical programs. That was the big hurdle and I think, we -- in a pinch, right. We could expand that mid-scale production and be able to prepare to launch commercially off of that capability. So these we believe derisk the supply issue significantly. However, it’s not stopping us from scaling up to the next level and now we have more know-how, more experience and applying those learning’s to the large-scale, we believe, gives us the good chance to succeed at a large scale production. And at that point that should provide the sufficient capacity not just for eteplirsen but then to easily adapt that to our follow-on DMD drugs. Obviously, we have been producing our PMO plus chemistry for the infection diseases programs. So there is additional know-how there. So we’re confident in the manufacturing. Look, when, going back a year or two ago we were concerned about an earlier approval and having enough supply. We don’t have that concern as we sit here today to have enough drug supply upon a commercial launch if that were to occur a year from now.

Great. I look forward to the next update and congratulations on everything you said today. Thank you.

Thanks, Steve.

The next question is from Brian Klein with Stifel. Please go ahead.

Great. Thank you for taking my question. Just in terms enrolment of the confirmatory study? Can you provide us what your expectations are there and will the full enrollment be completed before any potential Accelerated Approval? Thank you.

Sure. Thank you for the question. Obviously, we have a lot of work that was done prior to initial of the study, so we have been contacting sites and have done surveys as far as the number of patients that are available, we have our skip NMD, people have been signing up. And so, we’ve a lot of information. Clearly, we have suggestions from the sites. We don’t have exact numbers, because it just started. But what I can say is there has been an enormous amount of enthusiasm. And also the fact that, on very short notice we able to get 36 out of 39 sites coming to Boston, eager to anticipate. And what has been said, the investigators spoke to me directly and said, they have never had so much enquiry from patients on any of the study that they have done. So I think there is a lot of interest in this and hopefully that will translate into a fairly quick accelerated inclusion of patients into the study.

Brian to answer your question, I mean, in the event that we get Accelerated Approval, we believe that all of the patients that would qualified for our clinical studies who are interested in getting access to eteplirsen to a study earlier then they would otherwise get access to the drug under an approval, a year from now, that enrolment will have kind of picked out and but, obviously, whatever the enrollment is at the time we got approval. We understand that that could quickly shift to folks, just wanting to get on the drug post-approval. So if you recall from the April guidance, one of the criteria was to make sure we were enrolling both the eteplirsen open label confirmatory study and our follow-on exon confirmatory study placebo control at the time they approved the drug and we believe that will be easily met at a time we would potentially get approval next year.

Great. Thank you.

Thanks.

The next question is from Robyn Karnauskas with Deutsche Bank. Please go ahead.

Hi, guys. Thanks. Just wanted to take a big picture of that question, we have been focus on eteplirsen? I know large are waiting for like the next product, you mentioned, that you are looking about (indiscernible) the clinic? Can you help us understand, what is your constraint for moving forward in the clinical with other non-DMD products, first, timing and cost, since you don’t know whether you’re getting accelerated approval yet and then how far can you develop those program before you have to finance, if you don’t get approval early? Thanks.

Yeah. So there is two answers to that question, the follow-on DMD drugs are moving forward nicely. So those are the backups if you will for eteplirsen. And we -- one comment on that is since eteplirsen was the first product that we brought into development, we didn’t necessarily do all of the detailed optimization work to identify the most efficient and effective exons skipping sequence. We have better sequences to target 51 but more importantly all of the follow-on exons appear to have at least as good exon skipping efficiency and we would expect that to translate at least as good of efficacy as we see with eteplirsen. And so that gives us confidence in the follow-on exon. We also show safety and pharmacokinetics that are similar. And so that’s something that we’re confident that it would behave similarly as eteplirsen on a safety standpoint. Regarding the other application, obviously we will need to start with preclinical program. This takes 18 months to 2 years once we identify a lead preclinical candidate. Our test is two-fold, one we have to have chemistry, a new chemistry that can be applied, that we believe has a therapeutic index that is safe. We believe we have some of those chemistries depending on the disease that we’re going after, the cells that we’re targeting. And then we also have to have activity related to that chemistry and whatever the sequences that’s targeting its specific gene target that gives us confident to move forward with that lead candidate. And this is what I’ve said all along is we will announce once we have that dataset, right, that we can identify for a specific target against the specific disease. But what I provided in this scripted comments were a variety of areas that we were investigating and looking into and again stay tuned for data that would lead to that benefit.

Okay. Thanks.

The next question is from Joe Schwartz with Leerink. Please go ahead.

Hey, guys. Thanks for taking our questions. This is Paul on for Joe. Just a quick question on the EU exon-53 trial and its re-through is for the 53, 45 term in U.S? I was looking at the post you presented recently. And it shows that placebo-controlled portion of the study was 12-weeks long, which -- and we were wondering kind of what the logic is underlying that given that it takes longer, theoretically speaking, to make dystrophin and show a comparative benefit. And then given that how long you would think you would run the placebo-control portion of the 45, 53 trials before going into some sort of open-label phase or discontinuing placebo dosing? Thanks very much.

Sure, for the exon-53, I think it’s important to note that that this study has been in preparation for almost two years because it is part of a very larger grand. So it was -- it really is a dose-titration phase for the first 12-weeks in which we go up to the 30 milligrams per kilograms. And this was just fast way to accelerate it in the EU. That’s placebo control then we add another 12 patients, everybody goes on drugs. So it will be similar outcome measures with 6-minute walk test, pulmonary function and will be up also getting, obviously kind of experimental outcome measures, such as MRI. So it’s -- but it will be the actual -- it will be a one-year study on an open-label getting the basically all of the efficacy measures. And in regards to the study of the 45, 53 studies in the U.S., we are still under discussion of that that with the FDA. So we don’t have any final guidance on that.

Okay. Thanks.

Our next question is from Yaron Werber with Citi. Please go ahead.

Hi. This is [Kumar] again for Yaron. Thank you for taking my question. So far the mid scale supply, how long will it last for. And once you speak to the large scale batch of next year, do you need to do a bridging study?

Yeah. No, so look, again this is a synthetic molecule. So, more akin to a small molecule drug synthesis than a biologic. So we believe, classic comparability testing was suffice to show that regardless of scale, if you use the same process that the stop in the vial is comparable, right, in terms of active drug, purity profile and stability. So the stability of the production runs to-date, we have very good and long stability on our API. And we typically won’t go to fill and finish until we are closer to the need because again we have good stability in vials but just not as long term as we do with API. So we API is there and obviously, we would do a testing once we move to vials. And this was part of the discussions with FDA of creating that drug supply chain for our clinical program and eventually commercial scale. So we don’t anticipate, there will be bridging studies per se other than showing the comparability batch-to-batch, lots of lot and from small scale, mid scale to large scale. Again as I said earlier, we’re very confident in the supply chain from the data we have today.

Okay. Great, thank you. And also for the PND and PMC meeting later this year, what are your expectations from those meeting?

Look, these are classic meetings that take place to make sure, we check the boxes we highlight based on the guidelines for NDA formatting, what’s expected. We just highlight and clarify exactly what’s going to be in it. So we have a time to make any final comments adjustments before we actually hit send and put the submission in. Of course this is not a guarantee that they will accept the filling, but it does increase at least the confidence that we can clarify any issues around those final touches on the NDA. Ed, did you want to comment further about?

No. I think this is really if you’re within the similar specs, it should be fine. So this is not biologic and we’ve made that difficult transition for small scale to mid scale. It’s much easier to go from mid to large scale. So far we’re not anticipating any issues.

Yeah. And on the clinical and preclinical sections, it’s just the matter of saying, here is what we have, here is what you‘re going to see in the NDA. Few question related to that but again we look at these meeting as relatively perfunctory as we prepare for that NDA. We kind of know what we need to do with the NDA. It’s just a great opportunity to have a touchpoint with the FDA before we hit that send button.

Okay. Great, thank you so much.

We have no further question at this time. I’d like to turn the call back over to Chris Garabedian for closing remarks.

Thank you, Operator. We appreciate your interest in Sarepta Therapeutics and we remain committed to advancing our DMD program with urgency and with the commitment to getting our drugs through the regulatory approval pathways as rapidly as possible. We look forward to speaking to all of you on the next conference call. Thank you very much.