Welcome to the Third Quarter 2013 Sarepta Therapeutics Earnings Call. My name is Dawn and I will be the 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 Erin Cox. You may begin.

Thank you, Dawn, and thank you for joining today’s call. Earlier today, we released our financial results for the third quarter of 2013. 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, our President and CEO, Sandy Mahatme, our Chief Financial Officer, and Ed Kaye, our Chief Medical Officer. I would like to note that during this call, we will make a number of statements that are forward-looking including statements about the development and clinical status of Sarepta's product candidates and the potential efficacy, safety and clinical results from ongoing or future studies involving product candidates, the potential and timing for regulatory review and approval of Sarepta's product candidates, the potential for an accelerated approval by the FDA for eteplirsen, the potential for dystrophin as a surrogate to predict clinical benefits, the clinical significance of our six minute walks data to date in amount and type of data and other requirements that will be necessary for the FDA's regulatory determinations, the impact of manufacturing and development activities on NDA submission timelines, the potential pricing and market opportunities for our product candidates, our ability to manufacture candidates, our ability to protect our intellectual property rights, future financial performance including revenues, expenses and financings, sufficiency of our cash reserves, potential funding from the government and other sources and collaboration and partnering opportunities. 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 recently filed 10-Q 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 President and Chief Executive Officer. Chris?

Thank you, Erin. Good morning everyone and thank you for joining us today for our quarterly financial and corporate update for the third quarter of 2013. We also issued another press release this morning providing an update on our FDA discussions related to the eteplirsen clinical program and I will be addressing this topic on the call and during Q&A as well. Last Friday, at 4 pm Eastern Time, we had a meeting with the FDA that was intended to focus on finalizing our confirmatory study design with eteplirsen. We were very pleased with the FDA's involvement at this meeting and that they extended our allotted meeting time from 60 to 90 minutes and there were about 25 participants from the FDA including Janet Woodcock, the Director of CDER; John Jenkins, the Director of Office of New Drugs; Bob Temple, the Deputy Director of Clinical Science for CDER; and Ellis Unger the Director of Drug Evaluation I, along with Eric Bastings and Ron Farkas the Acting Director and Clinical Team Leader for the Division of Neurology Products. So, we were very thankful to have the FDA's active involvement in this program on the eve of a holiday weekend and believe it highlights the importance of this program within this agency. While the express purpose of the meeting was to finalize our confirmatory trial design. The agency provided unsolicited comments related to a change in their thinking on the regulatory status of eteplirsen, our clinical data and our clinical development plans as a result of new data that they have considered and interpreted since our last meeting in July. Specifically, they indicated that since the last meeting with Sarepta and based on the recent failed Phase III trial of drisapersen, GSK and Prosensa’s exon skipping drug candidate for DMD as well…

Thanks Chris and good morning everyone. As you know we recently presented 96 week data from our long term Phase IIb extension study of eteplirsen at the World Muscle Society Congress last month. We continue to be very encouraged by the results of this study in which eteplirsen treated patients are valuable on the six minute walk test have demonstrated generally stable walking ability for nearly two years. Based on the natural history and our clinical experience we would not expect to see stabilization in boys at this stage. In the study the boys are between 11 and 13 years old and while the remaining three are 9 years of age. The 96 week data showed less than a 5% decline from baseline in walking ability for the continuous eteplirsen treated patients are valuable on the six-minute walk test. In addition patients in the placebo delay treatment cohort demonstrated stabilization in walking ability from week 36 through 96, the period from which meaningful levels of dystrophin would likely produce with the decline of about 18.5 meters over this timeframe. At 96 weeks, the safety profile for eteplirsen continue to be favorable with no clinically significant treatment related adverse events nearly to two years. There were no discontinuations, withdrawals or hospitalizations and all patients continue to seek treatment. Since our last update there was one serious adverse event involving a late fracture that was assessed as treatment unrelated. This occurred in one of the boys who went non-ambulatory early in the study, because he was excluded from the modified intend to treat population, the injury did not have an impact on the six-minute walk test results that I just reviewed. Thankfully, we understand he is recovering and doing well. In addition to the presentation of these data to World Muscle Society…

Thanks Ed. I’d like to also highlight that we are continuing our scale-up manufacturing activities and we had a productive CMC meeting with the FDA, where they demonstrated the flexibility we were hoping for us, stability data, comparability and process validation. And we do not expect the FDA to be a barrier in providing drug to patients in our confirmatory clinical trial. While the FDA’s decision to reconsider the potential for an NDA filing in 2014, will require us to rethink the need for scaling up for commercial demand. We have not altered our plans at this time to prepare for large scale production in the hopes that the agency may reconsider being open to an early NDA filing on our existing dataset. Now I will turn the call over to Sandy to review our third quarter financials.

Thanks Chris. Good morning everyone. This morning’s press release provided details for the third quarter of 2013 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 for our ongoing operations and the impact of operations on our cash balance. Beginning in the first quarter of 2013, we have presented non-GAAP results primarily due to the significant non-cash impact, the [devaluation] of our outstanding warrants has on our net results. These results also exclude the impact of stock-based compensation and our relocation to Cambridge, we just substantially complete. Please refer to our press release for a full reconciliation of GAAP to non-GAAP. In the third quarter of 2013, we reported an adjusted or a non-GAAP operating loss of $21.3 million or $0.63 per share compared with an operating loss of $6.1 million or $0.27 per share in the third quarter of 2012. The incremental operating loss is a result of $3.4 million decrease in contract revenue and an $11.8 million increase in operating expenses. Revenue for the third quarter of 2013 was $4.2 million, down from $7.6 million in the third quarter of 2012. The decrease was largely due to the August 2012 stop-work-order and subsequent termination of the Ebola portion of the Ebola Marburg US government contract. Adjusted research and development expenses were $19.9 million for the third quarter of 2013 compared to $10.6 million in the third quarter of 2012, which is an increase of $9.3 million. General and administrative expenses as adjusted in the third quarter of 2013 were $5.7 million compared to $3.1 million in the third quarter of 2012, which is an increase of $2.6 million. During the third quarter, we completed our previously announced at the market equity offering raising a $125 million in proceeds by issuing approximately $3.4 million shares. All shares under the offering were sold during the third quarter of this year. We ended the third quarter of the year with cash, cash equivalents and invested cash of $281.4 million as compared to a $187.7 million at December 31, 2012. Turning to our full year 2013 guidance, we expect our net operating loss for the year to be in the range of $80 million to $90 million. Our operating expenses for the third quarter were higher than the second quarter of 2013 as we have begun to gradually make investments in manufacturing capacity. With that I’d like to turn the call back over to Chris.

Thanks Sandy. Operator, we can open up the call for questions.

Thank you. We will now begin the question-and-answer session. (Operator Instructions) Our first question comes from Brian Skorney from Robert W. Baird. Please go ahead.

Hey, good morning guys. Thanks for taking the question and sorry to hear about the disappointing notification from the FDA. I guess Chris, it sounds like a lot of what you’re saying you’re still thinking about a potential for an early filing. Did the FDA leave any opening to reconsider this decision on the acceptability of an early filing or give us some color around what you think the path forward here is and how hard and fast the FDA is about saying no to an early NDA?

Yeah. So, thanks Brian. With the FDA it’s an ongoing dialog. And these culminate in meetings and pre-meeting comments and meeting minutes. And the conversation can evolve. When we received the pre-meeting comments which typically we get a day before the actual meeting, often times the meeting allows for a good conversation and dialog, in fact it’s intended to discuss what’s in the pre-meeting comment. So we again had 90 minutes in the meeting and we used that time to really describe how differentiated our product is from drisapersen. Both the chemistry, for example we highlighted how we have data that shows our chemistry has multi-fold better exon skipping that our sequence versus their sequence all things being controlled has multi-fold activity. We talked about the higher dose we used in our studies, five to eight-fold higher dose in our clinical study. And we shared contrasting data on RTPCR, dystrophin as best as we could do apples-to-apples side-by-side. We hope that that does influence their conclusions from drawing extrapolations from the failed drisapersen trial. We will have to see if the continued dialog we have with them is enough to have them reconsider being open to an NDA filing, I mean our teams have been working diligently over the last few months since we had that initial guidance from the FDA that they would be open to an NDA filing. So we will continue compiling those documents and NDA modules in a hope that we can convince them over the next weeks to months to reconsider. So I think we believe the door is open for them to reconsider this, but at this time what we have disclosed is all we can provide as an update at this moment. But we do believe, just like the FDA kind of change their position from July till now, we think as we are able to describe more of the data that they may change their position yet again.

Got you. And then do you know has the FDA received and reviewed the GSK, Prosensa data in any formal manner, I mean what level of analysis have they done that have sort of driven this decision and the comparison between the two?

Yeah. It’s a good question because I think their conclusions clearly were driven in large part by the sales study. They did mention that they’ve seen some dystrophin data that was not in the public domain. We obviously had access to what was in the public domain, we discussed that, shared it, try to put context around it. And we told them that whatever additional dystrophin data they may be looking at, we’d love to have a dialog with them about it so that we can put it in the right context vis-à-vis our data. And what we understand is the right way to quantify the dystrophin. When we had a discussion around the clinical data, they did indicate in the meeting and in comments after the meeting that they have requested the data from GSK, as of last Friday they did not have that data in-house, but they hope to receive that data sometime in the near future. And then they would start analyzing that data is how they described it to determine if there is more insight that can be cleaned in how our drug is performing in our study, but they have not yet done that analysis nor as of Friday had they received that data.

Thank you. Our next question comes from Liisa Bayko from JMP Securities. Please go ahead.

Thanks for taking the question, sorry about the news on this. Can you just maybe talk about, Chris you mentioned that you had used a 90 minute talk about chemistry and safety and sort of some differentiated features there, did you feel like that resonated with the agency, can you just maybe talk about their response?

We hope so. There were slides that we presented and we obviously provide those for them to look at following the meeting. It's hard to say. I would say generally, the FDA likes to consider the discussion in a meeting and reflect that in the meeting minutes. Now we always try to get feedback immediately after having a dialog presenting data, but it seem they prefer to reserve the judgment and reflect that in the meeting minutes. I also mentioned and it was in the press release that we did request a follow-up meeting to discuss confirmatory study, that's another opportunity to answer any questions they may have had from our Friday meeting and that could be reflected in minutes as well. So again, it's an ongoing dialog, but it's unclear how persuasive that was and we probably won't have insight into that until the minutes come out.

Okay, great. Thank you. And then as you look now to your next meeting, can you just remind us when do you anticipate that sort of meeting to discuss more details about Phase III will be?

Yeah. Well, we said this month we don't give specific dates of when those meetings take place, but I will say that we requested more time with the agency to discuss these very important issues and with a sense of urgency and they are responding and meeting that urgency and getting time on the calendar. I mentioned there were approximately 25 participants from the FDA, you can understand scheduling. It can be difficult across all those important people. So, ultimately though they have been very responsive and we're pleased with their willingness to give us more time this month.

Thank you. Our next question comes from Robin Karnauskas from Deutsche Bank. Please go ahead.

Thanks for taking my question. So actually I have two if I can. So I guess as regarding the design of the trial, it sounds like placebo-controlled is out, do you have suggestions for them to use things that they will agree to, and what kind of patient sub-populations do you think are on the table? And then second question is, do you think that if you continue to push for solid approval that that could damage your relationship with the FDA, I know the FDA. I appreciate that the patients really need this drug, but I am also -- I am concerned that the FDA might be turned off by pushing for something that they said no to already?

Yeah. Well, so, Robin, first I would say, let me answer your last question first. So they said no recently, but they weren’t saying no as of July, right. They were opened to an NDA filing. So that gave us encouragement and that we started plans to pursue that. So all we're doing and all we can do is respond to the FDA messages and comments. So there was a change in that belief and perspective since the July meeting. So we don’t feel that we've been very thoughtful and considered all along the way. In fact, we indicated that we would simply initially only talk to them about dystrophin as a surrogate and we've raised that earlier this year and we had ongoing dialogue in which we've shared publicly about all the steps along the way of how that conversation has evolved. So we’ve never pushed something that they have been very clear about. Obviously today’s communication is recent and we’re digesting their perspective. At the same time, we believe it’s appropriate for us to have a response because we think that some of the conclusions that they have come to related to dystrophin or comparisons to drisapersen, the natural history are not complete and we want to be in a position to be able to have that full conversation with them. So regarding the trial design, you may have missed both, but you said placebo-controlled is out. To the contrary, they are restating their desire to have a placebo-controlled study. We have been clear with them along the way the challenge with a placebo-controlled study. As you are aware, we don’t at this moment have the freedom to operate in Europe to entertain sites in Europe. So we are looking at U.S. North American sites. It’s a limited…

Thank you. Our next question comes from Ritu Baral from Canaccord. Please go ahead.

Thanks. Just to piggyback off of Robin’s question a bit, what other endpoints would you guys consider, I mean FDA doesn’t seem to like biochemicals endpoints like dystrophin just like six-minute walk because of the effort based nature, like a lot of like functional endpoints. What are you left with in DMD that's been in anyway should perform decently characterize?

Yeah, we see that, that's a great question, because it's why we picked the six-minute walk test, it's why every company that has been doing DMD trials has focused on the six-minute walk test. The FDA has used this as a basis for approval as a primary or co-primary end point in other neuromuscular conditions. Obviously, it's well known in the PAH population. But the DMD studies that have been done with PTC Atalauren, the previous study was with six-minute walk, their new Phase III study is being done with six-minute walk. The drisapersen trials have all been with six-minute walk and it was a primary clinical outcome measure that we followed. So that we believe is the most validated, reliable and predictive end point. And so we're disappointed that it's being called in the question and we don't believe there are better validated endpoints in the DMD population. Obviously, pulmonary function is something we've been capturing, anything beyond that has not been very well characterized. And although they did not provide a reference for exactly the natural history data they were citing, we believe it came from Craig McDonald's most recent publication, of which even the conclusion of his own paper was that his findings confirm the clinical meaningfulness of the six-minute walk test as the most accepted primary clinical endpoint in ambulatory DMD trial. So we've really tried to follow those before us and what's in the literature. So it's hard to say and obviously we have to begin those conversations with the FDA about what they think might be acceptable other endpoints in these other subpopulation.

And could you give us an idea of how timelines might be affected by inclusion of the people biopsy and/or without I mean just sort of bracketing, would you expect that would add in a six months issue enrollment time, 12 months just the general idea?

Yeah, we've done some feasibility surveys, we've identified. We've talked to a lot of sites. We also have a little bit of information on the drisapersen U.S. study that they’ve enrolled 53 patients that were pretty much the same patients we would be looking for and as you know that can be confounding where you have safety exposure to these patients as well as potential. And so were any dystrophin being produced, we need to understand what they had a baseline and also the idea of suggesting those drisapersen patients who already received biopsies, even if they were on placebo to suggest them again to two more biopsies and the chance of getting a placebo. We understand the challenges with that even from an IRB perspective. So we know there are in fact many patients that would be available for the selected ambulatory endpoint we're looking for and we believe we would have been challenged with let’s say a six months or nine months enrollment of the 60 patients we have proposed. Now to find a twice as many patients, it would not just take twice as long, it could be impossible to find that many patients and it could take three times as long or more, it’s hard to say based on the feasibility. We thought we were going to have a hard time finding 60 patients. So the requirement or seeming inflexibility of allowing alternative designs that don’t require a placebo-controlled in the exon 51 amenable population really calls in a question exactly how we would accelerate the enrollment. Now the FDA, again as I mentioned said they understand that that may raise feasibility issues of enrollment and that’s why they have introduced the concept of new endpoints, sub-population, they even introduced the concept of combinations of endpoints to be combined mathematically. Again, it’s not clear these are just ideas that are on paper right now that have not been discussed and they have been left fairly open-ended at this point. So I think we had to disclose what we did today and we expect continued dialogue with the FDA on this confirmatory study design as we get more clarity and more confirmation of that and the potential timing of the start of that study and timing of enrollment based on the number of subjects we will provide that.

Thank you. Our next question comes from Tim Lugo from William Blair. Please go ahead.

Thanks for taking the questions. I guess given the FDA seemingly questions around the six-minute walk test, is there a 170 week data that you think would change their mind? Is there a threshold where the Phase IIb patients stability you think would push them to may be change the current stands. And can you remind if there was the additional biopsy done for any of the Phase IIb patients than was in any of that data presented at this meeting?

Yeah. So first on your first question. We believe we already have a level of evidence through 96 weeks of which none of the natural history that they sight has followed patients through 96 weeks that was only based on 48 weeks. And when you look at the literature almost every other natural history study shows a decline in similar population or exon deletion population in natural history. And the only two year longitude that was published this past year by Elena Mazzone and Eugenio Mercuri that shows a 33% decline or over a 100 meters loss over a two year timeframe. So we will be getting -- so in the third year of the study we moved from every 12 weeks to every 24 weeks of six minute walk. So we will have 120 weeks datapoint to provide the FDA and again if that shows continued stability that would be beyond anything that has been characterized in most of the natural history data that is available. So it’s hard to predict if any of that would produce a different perspective, but we thought they were already there based on our July meeting where they again were open to an NDA and were going to weigh in on dystrophin after filing and we talked about approval on the six minute walk test and they say, yeah they do have the flexibility based on the clinical data. So, Ed you want to comment on why we believe our 96 week data is differentiated from the natural history.

Thanks Chris. I think if we look at the natural history and based on our population, again remember these boys are older than had been studied in the dystrophin study. And the natural decline is way out of expected. The other, I think strong aspect of the data is if you look at the placebo treated boys, they did start to decline, they had a significant decline. And then when the dystrophin would be expected to be present based on the biopsies, we could see that they stabilize. So, I think it's very unexpected data and I think the longer we have -- I think at the end of the day, I think the science need to strict for itself. And at some point, the data will be strong enough and I think we really have to present it and realize that we've been sitting on this data for two years, analyzing that and I think we need to give the FDA an opportunity to really look at the strength of the data and the totality of the data to let them see, what we're seeing in the data.

Yeah. And let me just reemphasize a point that Ed just raised. The comments from the FDA were focused on the patients who were above 350, okay. And again we dispute that it’s a broader natural history data particularly in this amenable population shows no decline. But we did have the four placebo patients who started above 350, they declined precipitously to below 350. So when they started at week 36, before we had confirmed dystrophin, they were below 350 and we were able to stabilize them once we produce dystrophin. And they have been stable now for over 60 weeks. So we even have within our study a sample that refutes the claim of the greater than 350 argument. So Tim your other question was about the other biopsy data from the drisapersen study is that correct? Tim? Okay. In terms of the data they shared, not revealed exactly what data they had captured from the drisapersen that was driving their decisions around the notion that their drug was producing dystrophin and failed nonetheless. So operator, next question.

We have Chris Marai online from Wedbush Securities. Please go ahead.

Hi good morning Chris. Sorry to hear the news from the FDA. A quick question regarding your discussions on safety and the safety dataset, had the FDA indicated anything regarding that to you as a concern given the limited number of patients that you've exposed to eteplirsen to-date?

No. We've obviously provided our safety updates to the FDA. We've reported publicly the safety through 96 weeks and that did not come up in this discussion and we've had the long-term animal tox, all of those clinical study reports have been provided to the FDA previously. But more importantly we think safety should be considered vis-à-vis the drisapersen data, because we understand the toxicity profile of drisapersen, require them to terminate dosing from all of their studies, because they could no longer put a drug that could cause toxicities with no clear benefit. We believe that we don’t have that issue to-date and that that should have been considered before making the decision to say the NDA is premature, but we will continue to have a dialog as Ed described, we hope to have the opportunity to share more of that, but Ed do you want to comment on our views about safety and the risk benefits.

Okay great. And then how does World Muscle spent a lot of time with documents down there. You’ve highlighted that there have been some analysis looking at percent predicted decline in some of these boys with respect to your Phase II data. Have you shared any of that data with the FDA at this time or have you shared any other potential data that you have on strength measures or do you have any data on strength measures and alike and has that been shared with the FDA at this time point?

Yeah. So obviously we did not have predicted as an outcome measure or a baseline criteria, that’s a derived measure that they had all of the data that they needed to calculate percent predicted, but we’ve provided all of the data you describe for them to have on their own percent predicted, but all of the other endpoints we said previously they requested patient level data or all the clinical endpoints; if you recall we gave them a white paper on all of our clinical outcomes as well as an overview of the natural history on those outcomes and that we provided a 96 week update on all of those outcomes as well. So they have all of the data that they need. The other thing about the reference, again they didn’t cite Craig McDonald or his papers by name or as a reference, but we know where that 350 meter data came from as they described it. And it’s important to note that to highlight that natural history data that was based on the atalauren patient population’s placebo arm where atalauren is stop code on nonsense mutations. There were three of those genotypes in that study. Eugenio Mercuri presented our data at World Muscle in a symposium that showed that the stop code on nonsense mutations have a milder phenotype than the exon deletions, particularly exon deletions of exon 51, 53 and 45. We know that they have characterize that study is having Becker Muscular Dystrophy patients in that database, but that was done on kind of clinical manifestations and not on any kind of genotypic basis. So we think all of the other natural history that is much more relevant is in the exon deletion or broader DMD population of which we’ve described, the number of studies that show the decline in the over 7 year old population. We also think that the drisapersen same study is very informative. This is exactly the exon 51 amenable population that we are enrolling and including in our studies. So that is probably the most informative. And so there if you look at the greater than 7 year old population, they declined 76 to 83 meters, 83 meters in the placebo arm, 76 meters in the drisapersen treated arm. So we think that there were a lot more six-minute walk data that they could have and should have referred to, to characterize our data. Now in fairness, the FDA said they would like to analyze the GSK data once they receive it to understand if they can draw the same conclusions that we have. But they had not yet done that, when we spoke to them on Friday.

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

Hi, thanks for taking my question. So none of the FDA has seemingly called into question the six-minute walk time as an appropriate endpoint, how can we gain comfort about the validity of any of the other potential endpoints that you’ve suggested including things like composite endpoints or other measures of muscle function?

Yeah. Well, we've been struggling with that as well. This is why we’ve put so much emphasis on the six-minute walk test. There seems to be broad consensus across the industry, there seems to be president at the FDA. And so to introduce new and less validated endpoints, we have to understand what the FDA is thinking about this and have that dialog with them. Again we've proposed to them what we thought was a actionable reliable study design in which we were going to have a kind of a match natural history expectation to compare against a untreated cohort to compare against. They can do matched controlled with the drisapersen arm as another third comparison. And so all of those we felt were sufficient in a rare disease like this that is difficult to enroll in which we don’t have validated endpoints beyond what most would say is validated in the six-minute walk test. So again, this is why we've requested further discussions with them in a timely manner. And we look forward to those discussions too. And also as Ed described, to share our view on this and why we proposed what we did, but it’s unclear to us exactly how we would look at other endpoints and how we will power those studies in subpopulations on endpoints that are not as well characterized as the six-minute walk.

Great, thanks. And then just real briefly, did the FDA comment at all about your proposal to do staggered biopsies in different patient groups? Thanks.

Thanks. No, they did not comment on the staggered nature. Again the questions about dystrophin have increased. And we’d like to continue that dialog with them because we think many of those concerns are unfounded. And so part of the request is that before we do any further biopsies including the original fourth biopsy that they had requested in the last meeting in July, they are saying we need to understand better the assay and get comfort around validation of an assay before we consider dystrophin further. And so this is complicated because we really don’t think we could feasibly start a study and capture a biopsy sample surgically from the patients without knowing how we are going to measure it or what we are going to do with those muscle biopsies. One proposal was that we could figure out later just get the biopsy samples and we’ll figure out and we appreciate the FDA thinking creatively about this, but we think that would be hard to get IRB approval to subject not only DMD boys, but placebo patients to two surgical biopsies before we even knew what we’d be doing with those biopsies. So look, this is an ongoing dialog, we are trying provide the material information that we need to in a timely manner, but this will, we’ll have continued discussions and I expect continued clarity on the confirmatory study as we get it.

Thank you. Our next question comes from Ed Tenthoff from Piper Jaffray. Please go ahead.

Great, thank you very much. And very disappointed for the Duchenne community on the setback today from the FDA, seems like a real reversal on their position. Actually just housekeeping question if I may, how many shares are outstanding as of right now including the 3.4? And can you be a little bit more précised in terms of what the exact number of shares issued under the ATM is and the average price?

Yeah. So we have about 37.6 million fully diluted shares. We issued 3.4 million shares and the average price under the ATM was about 37.3 million I think, 37.30 million.

Okay. Thank you.

Thank you. Our next question comes from Edward Nash from Cowen and Company. Please go ahead.

Ed?

Mr. Nash, are you there? Okay, I am going on to next person. We have Bill Tanner on line from FBR Capital. Please go ahead.

Thanks for taking the question. Chris, I appreciate obviously you expeditiously wanting to get the drug to the market or into the clinic I guess or commercialized, but I am wondering about the commitment to the second quarter 2014 start given that it seems like there is a lot of discussion yet to be had as it relates to the design of it and it seem like you probably have one shot to get this right. So I am just kind of curious the feasibility of that or being able to do that and actually getting to an answer in a relatively timely fashion? I have follow-up please.

Yeah. Bill, so that’s why we said it’s moved to at least to the second quarter. We think the reason we put second quarter as reasonable is that we do believe the FDA understands the urgency here and wants to come to a resolution on the confirmatory study as quickly as possible. So to that end, again I just want to clarify, they proposed a lot of different ideas and I don’t want this to be perceived that the FDA, they are trying to offer alternatives to just relying on the six-minute walk. Now they indicate that they would still accept six-minute walk in a appropriately powered study, but highlight they understand that the feasibility of enrollment could be hampered because of their requirement of a placebo control. So this is where, we don't believe, we can easily or at all potentially find enough patients to do the placebo control that they want in a timely manner or may not be able to fully recruit that many patients to power. So what they are doing is saying let's talk then alright, because we want to come to an agreement on a confirmatory study. So we'll have more clarity on that and we think that they're willing to work quickly with us to nail down the details of that confirmatory study. And we think and hope that we can still start dosing in the second quarter. Now this is, anybody in the industry knows this is not an easy task, this is in months planning with our CRO, with identification sites in crafting a protocol, we actually had two protocols that we had developed; one with the placebo, one without with the expectation that what would happen if they didn't understand the feasibility of enrolling a placebo patient. So this would introduce the idea of potentially a whole new protocol and that takes some time to develop. So we think second quarter is still reasonable based on the responsiveness that we’ve seen from the FDA so far.

Okay. And then am I say it was a pretty impressive roaster of attendees that you've listed at the beginning of your prepared remarks and so I guess one interpretation would be that it’s somewhat of a show of unity as it relates to agency’s opinion. So I am just kind of curious and maybe it’s an unfair question, how convinced you think or how strong you think the case can be made for the FDA to reconsider, I mean I understand that there was one or two of the division directors is one thing, but it certainly seems like again it was a fairly impressive roaster of attendees?

Well, how we’re going to responded to that Bill I don’t have real insight into the mechanism and inter-workings of the FDA. I will say we obviously know that the Division of Neurology Products is the one really reviewing all of our data and answering our questions and they’re the ones who put together this update for us. But we believe that the involvement and dimension of the involvement of the hierarchy is important in that at least they’re aware they’re paying attention to this and we think it’s a good thing that they also are hearing Sarepta’s arguments, right. And our disagreements and disputes on some of the conclusions they’re drawing that led them to reconsider or call the NDA filing premature. So we don’t know if this will influence all of those that were in the room when we meet with them next or when the minutes come out or in subsequent discussions. So we are just appreciative that they are paying attentions to this program, they understand the urgency and that they are involved, I don’t know if there is full alignment and agreement within the agency and among all those people I named that will bear out over time.

Thank you. We have Yaron Werber from Citi online with the question. Please go ahead.

Thanks for taking my question. I definitely appreciate it. So I mean I wanted to kind of follow up on two things. The first one is just again to follow up on Bill’s question on the second quarter of next year, I mean one of the things that I imagine, obviously you want to do and it’s critically important for patients is to make sure you maximize the chance for success for the next study. I am just -- the FDA kind of noted they have obviously growing concerns about your assay or your quantification method and you noted that they are -- the biggest challenge the drug seems to have activity, the biggest challenge is how do you show it. So would you consider you will probably need to validate some of these new endpoints wouldn’t that be the case? Would it make sense to do a Phase II first? And then I have a follow up just to maximize the chances when you run a Phase II you can really powered correctly? And then I have a follow-up if you don’t mind.

Sure, Yaron, look one of the reasons we pursued an early approval based on what we believe very encouraging and compelling data both biochemically, clinically and from a safety standpoint was because of the urgency of this disease, okay. That’s why we are all in this industry right is to try to get drugs approved to treat patients and we believe we have a drug that can benefit and help patients, okay. So we are trying to work as expeditiously as possible, we want to be smart, we want to be creative, we want to be thorough, but we believe we can do all of those things as we progress toward a confirmatory clinical trial. If you look back with the history of drug approvals or orphan drug approvals many of them were approved on single studies, some of them are open label studies. There is a wonderful white paper written by Frank Sasinowski who was Chairman of NORD, National Organization for Rare Disease at the time. That highlighted the flexibility of the FDA and their understanding of the urgency. We know that Congress have legislation with the [deja] saying that the FDA should utilize their capacity to approve drug under accelerated approval surrogate markers for rate diseases like Duchenne PPMD had a nice paper highlighting the Duchenne really fits into the [deja] intent of the type of rate disease and what the FDA should show that flexibility and look at accelerate approval. So we don’t believe we are doing anything that isn’t supported and highlighted by precedence, by legislation and so we think a confirmatory study at most is what would be required for approval of this drug.

Okay. Thank you for that. And then just a question, so in terms of manufacturing, just give us a little bit of sense where are you and are you going to take the midscale into the next study or what should [they lay the start]?

I am sorry, was there follow-up there?

And how much capacity would you have?

All right, so we mentioned previously the midscale is what we have been focused on to generate a drug supply for the clinical program. We highlighted that we had a very productive meeting with the CMC division with the FDA and believe that the FDA will not be rate limiting and there is no guarantees of course, but that we believe that they have not posed any barriers for us to start dosing that confirmatory study. We had been planning for large scale production in the event that we submitted an NDA to have commercial scale and commercial supply come offline by the end of next year. We'll have to evaluate as we continue our discussions with the FDA, whether we will have to put that on hold or whether we'll continue that pursuit, but we don't have to make that decision at this very moment because the real production runs for the large scale begin in earnest next year for the large scale batches. So that's where we stand on the manufacturing and our efforts next year.

Thank you. We have Joseph Schwartz online from Leerink. Please go ahead.

Thanks very much. Sorry to hear the news. I was wondering since the FDA's expressed a desire to explore additional subgroups or subpopulations and endpoints. How reasonable do you think it is to go straight into a Phase III versus even what you were saying before about there might be difficulties finding enough patients for that? And the FDA has said that they might be open to mathematically pulling across different studies. Why not do some more Phase II work in order to qualify these additional subpopulations endpoints and just make sure that a big investment in Phase III is likely to succeed?

Yeah. So they didn't suggest the mathematical combination of cross-studies. They were saying you could potentially enroll patients of various status, younger patients, older patients, non-ambulatory patients that might require us to look a different endpoints than the 6-minute walk test and that maybe there is a way to combine these endpoints. Again we think that’s challenge because these are generally invalidated endpoints and how do mathematically combine them and then power study, but we can have that discussion with the FDA. So look if you followed both GSK, Prosensa’s communications around their studies as well as PTC’s communications around their previous failed study with Atalauren, they all talked about the difficult of powering studies for 6-minute walk benefit, right. And those were large studies, those were 180 patients studies in size and they were still complaining about the inability to power, a study we believe we have an active drug that can produce a 6-minute walk benefit that does not required that larger the study. And so we think it’s the most prudent to do the right size study, well powered on a validated endpoint and again that's what we've tried to propose to the agency and we will continue that discussion.

And then you talked about or the FDA suggested that you should have an adequately validated, quantitative assay for dystrophin, what are your activities there and how much is that a weight limiting factor to start the next study versus can you take the biopsies and then figure out exactly how to analyze them at a future point?

Yeah, we need to have that dialogue with them. Again we -- Ed why don’t you describe there is emerging data on dystrophin analysis and the right methodologies and Ed was recently in London, he was talking to some investigators in DMD and this is unpublished data, but this is an example of what's emerging. Ed, do you want to describe what you learned?

Yeah, certainly. It’s a head-on opportunity to talk about. Again I think in the academic community, people have been concerned about what’s the reliability of dystrophin-positive fibers. And so in order to try to consolidate that and try to learn more information, a group of investigators, which included nation-wide children, which did our 201/202 study, (inaudible) who also did our intramuscular study in our Study 28 and including and also Prosensa. So they got together, sent out samples and what they were able to demonstrate that in fact, if you use the same protocol very similar to what we've been doing, you can have very reliable data in regards to immunofluorescent for the dystrophin-positive fibers. The most next important was the intensity. And so I think we will obviously share this data once it becomes publish with the FDA to demonstrate that I think that it is -- this is not a very challenging assay, if it’s done in a consistent way.

Thank you.

We only have time for couple more questions. So one each from the next in the queue.

We have Debjit Chattopadhyay from Emerging Growth. Please go ahead.

Sorry to hear the news Chris. In terms of the confirmatory study if they do accept for the 6-minute walk test as a valid clinical endpoint, do you think you can get away with the 48-week endpoints or do you need to go to 96 weeks? And the stability data being suggested for 356 meters cut off, how do we know there were no Becker patients in that study population? Thank you.

Yeah. So related to the 48-week data, they did comment on this, I think based on the failed studies of the other dystrophin producing technologies. And they mentioned and I’ll just read, a duration of 48 weeks may not be long enough to identify efficacy in a slowly progressive disease such as DMD. We believe our drug is active enough to show a benefit over 48 weeks. If you can imagine that we showed in our ambulatory population, the modified intent to treat a robust p-value after only 24 weeks of treatment and before dystrophin was present at 36 weeks, we believe that if you followed a cohort out of 48 weeks without treatment versus 48 weeks on treatment that we would be able to power a study sufficiently. And again so we believe 48 weeks is sufficient. The other benefit of the open-label design that we initially provided was that they could continue to follow those patients indefinitely against the untreated cohort or compared to natural history studies. So we think 48 weeks is sufficient with our drug to show a benefit. And Debjit, did you ask if how do we know if there weren’t more Becker patients in the Atalauren natural history analysis? Okay, sorry. Yeah we don’t know again exactly the criteria that use to define Becker, but we just think it’s not the most amenable patient population to compare our drug and the exon deletion population, exon 51 deletion in particular against the three genotypes that had stopped put on (inaudible). I think we have time for one more question operator.

We have Kimberly online from Janney Capital. Please go ahead.

Thanks for taking the questions. I apologize if I missed this. What are your plans for the rest of the pipeline and what are the implications you think of the results from the FDA on with regards to the rest of your pipeline and clinical endpoints and study designs? Thank you.

Yeah. Kim it’s a great question, because our entire program was largely predicated on dystrophin as an acceptable surrogate biomarker. And so we were going to do that with the eteplirsen studies, because we can enroll enough patients to show that clinical benefit correlated to the dystrophin that we are capturing. Again with the FDA calling in the question dystrophin as a biomarker, we don’t believe that is something that can be affirmed decision, because it would render us enable to get other drugs approved for the rare exon because we would not be able to power studies on 6-minute walker these other endpoints, I just described how difficult it is to enroll patient with eteplirsen amenable exon deletions. So this is why we need to work with the FDA closely to come to an agreement and assessment of how to capture dystrophin in a way that they believe is validated, so that we can have streamlined approvals of the other exon. So it’s difficult to say where I sit today of what those development tasks look like for the follow-on exon and how the FDA will approach the potential regulatory approval of those other exon.

Thank you. I will now turn the call back to Chris Garabedian for closing remarks.

Okay. Thank you, Operator. Ultimately well today represented a disappointing setback in the eteplirsen path toward approval. We believe strongly that this product will serve a significant need for DMD patients and are committed to working with the FDA to get it approved in a timely manner as possible. We continue to develop our follow-on exon through pre-clinical towards clinical development. We want the DMD patients and families and broader DMD community to know that we appreciate their efforts and the support that we've seen from them in highlighting the rapid progressive and irreversible nature of this disease and the urgent need for promising treatments as it keeps us focused on our goal of getting drugs approve to treat every DMD patient, who can benefit from our exon skipping technology. Thank you everyone for listening in to our quarterly call and we appreciate your interest in Sarepta.

Thank you. Ladies and gentlemen, this concludes third quarter 2013 Sarepta Therapeutics earnings call. Thank you for participating. You may now disconnect.