Welcome to the Q4 2013 and Full-Year Sarepta Therapeutics Incorporated Earnings Conference 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. Erin, you may begin.

Thank you, Dawn, and thank you all for joining today’s call. Earlier today, we released our financial results for the fourth quarter and full-year of 2013. The press release is available on our website at www.sarepta.com and our 10-K will be filed on or before March 3rd. Joining me on the call today are Chris Garabedian, our Chief Executive Officer, Sandy Mahatme, our Chief Financial Officer, and Ed Kaye, our Chief Medical Officer. I’d like to note that during this call, we will be making 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 Sarepta’s product candidates, the potential and timing for obtaining clarity on the pivotal trial design, finalizing the protocol and dosing of patients with eteplirsen, regulatory meetings, submissions, filing and review and approval of Sarepta's product candidate. The potential for an expedited approval by the FDA for eteplirsen, the potential for dystrophin as a surrogate marker, the amount and type of data that will be necessary for the FDA's regulatory determinations, our manufacturing plan, 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 successfully, our plans to expand our research efforts and capabilities and strategic investments to grow our business and advance our product 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 annual and quarterly reports and other official corporate documents filed with the Securities and Exchange Commission. With that, let me turn the call over to Chris Garabedian, Sarepta's Chief Executive Officer. Chris?

Thank you, Erin. Good morning everyone and thank you for joining us today for our financial and corporate update for the fourth quarter and full-year of 2013. The last 12 months were foundational for Sarepta as we continue to build the business dedicated to delivering innovative medicines to patients. First and foremost, we made important progress with eteplirsen, our lead clinical program for Duchenne muscular dystrophy. Last month we announced updated data from our long-term Phase IIb expansion study that show general stability on measures of walking and pulmonary function after a 120-weeks of follow-up. Combined with a favorable safety profile and biochemical outcome showing an increase in dystrophin protein, we believe the long-term clinical data for eteplirsen are the most compelling generated in DMD to-date. Throughout 2013, we shared the eteplirsen data with the scientific community at a number of important medical congresses and in a peer-reviewed publication in the Annals of Neurology. We also strengthened our manufacturing capability. We have now completed several mid-scale production batches and had moved three of those batches through fill and finish and are in the process of completing the analytical testing and comparability analysis which will be -- which we will be submitting to the FDA in the coming month for potential drug release. Based on our progress to-date and provided the analytical testing is successful, we remain on track to supply drug for the upcoming eteplirsen clinical study. In addition, we have a plan in place to further scale up a global network of suppliers should the FDA reconsider its position on a potential filing for early approval of eteplirsen or otherwise approve eteplirsen through an expedited regulatory pathway. Our government supported infectious disease programs continued to progress as well. Earlier this month, we announced the new data from a Phase…

Thank you, Chris, and good morning, everyone. As you all know we recently disclosed 120-week data from our long-term Phase IIb extension study of eteplirsen. This data included updated 6-minute walk test results as well as new data and pulmonary function test. Over more than two years the data showed general stability on measures of walking ability and pulmonary function in a population that we’d expect to decline. Eteplirsen safety profile also continues to be favorable with no reported clinically significant treatment-related adverse events. From baseline to week 20 there was about a 14 meter decline in walking ability for continuous eteplirsen treated patients evaluable on the 6-minute walk test, representing less than 5% decline in these patients. In addition, the placebo delayed treatment cohort demonstrated stabilization in walking ability from week 36 through week 120, the periods on which meaningful levels of dystrophin were likely produced, with a decline of about 10 meters over this timeframe. At week 120, there was a statistically significant 65 meter difference between the continuous eteplirsen and placebo-treated cohorts. And when we look at the data longitudinally it show a generally consistent 50 meter to 70 meter separation between the two cohorts from week 36 to week 120, again suggesting stabilization across the study population evaluable on the 6-minute walk test. For context, natural history data published last year by Mazzone and colleagues showed a mean decline of about 125 meters over two years in patients older than seven years of age. This is consistent with other natural history studies by Goemans et al. Additionally, GSK had previously reported an 83 meter decline after 48 weeks in the same exon 51 amenable population who were over seven years of age in their drisapersen Phase 3 study and further declined in the second year of…

Thanks, Ed. I just wanted to provide a brief update on our other exon skipping drugs for DMD. We are on track for filing at least two additional INDs later this year for exons 45 and 53. Our IND enabling studies are on track and are demonstrating a preclinical safety profile that appears similar to that of eteplirsen. Specifically, the genotox and safety pharmacology studies were all negative for any drugs related finding. [Mouse tox] [ph] and ADME studies are ongoing, but most strikingly the results from the 12-week non-human primate repeat dose toxicity study showed no adverse effect up to the top dose tested of 320mg/kg. We’ve also initiated the exon 53 preclinical tox package and so far again the limited data we have back from those studies indicate a profile similar to that of eteplirsen. It's a final report on exons 45 and 53 showed similar findings as we obtained with eteplirsen. It provides a good base of data to argue that we have sequence independent safety, a key consideration if we are able to pursue the class approval strategy to gain approval on the exon skipping drug that treat the rare exon deletion genotypes. Once we are in the clinic with these drugs, we hope to demonstrate dystrophin production of a similar range of eteplirsen at a standard dose, so that we can provide sequence dependent activity and efficacy. As we previously reported, we also brought three additional drug candidates into sequence lead optimization. Building on the clinical success with eteplirsen, we expanded our DMD pipeline and began sequence optimization work for three new drug candidates of targeting exons 52, 55 and exon 8 to add to the other drug candidates targeting exons 45, 53, 50 and 44. In all, we now have eight product candidates for DMD in various stages of preclinical and clinical development that have the potential to treat about half of the DMD population amendable to exon skipping if approved. Now, I’d like to turn the call over to Sandy Mahatme, our Chief Financial Officer for an update on our financials for the 2013 year-end.

Thank you, Chris. Good morning, everyone. This morning’s press release provided details for the fourth quarter and full-year for 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 that the valuation of our outstanding warrants has on our net results. These results also exclude the impact of our stock-based compensation and our relocation to Cambridge. Please refer to our press release for a full reconciliation of GAAP to non-GAAP. In the fourth quarter of 2013, we reported an adjusted or non-GAAP net loss of $29.1 million or $0.77 per share compared with a non-GAAP net loss of $8.9 million or $0.34 per share in the fourth quarter of 2012. The incremental net loss is a result of a $4.7 million decrease in contract revenue and a $15.5 million increase in non-GAAP operating expenses. Revenue for the fourth quarter of 2013 was $2.6 million, down from $7.3 million in the fourth quarter of 2012. The decrease was primarily due to the August 2012 stop-work-order and subsequent termination of the Ebola portion of the Ebola Marburg U.S government contract due to a lack of available U.S government funding as well as timing of activities associate with the Marburg portion of the contract. These decreases were partially offset by revenue from our European Union Brand supports development of an exon 53 skipping therapeutic. Adjusted research and development expenses were $23.6 million for the fourth quarter of 2013 compared to $12.4 million…

Thanks, Sandy. Before we open the call up for questions, I want to close by highlighting some of our key upcoming milestones. As I mentioned earlier, we plan to work with the FDA to obtain clarity on the eteplirsen confirmatory clinical study design later this quarter. As we work through that process, we have taken steps to select and prepare clinical sites as well as prepare our internal teams and our external CRO to conduct the study. We are also taking steps to rapidly advance our follow-on exon skipping candidates. Later this quarter, we plan to have a pre-IND meeting with the FDA for our exon 53 skipping trial and we plan to initiate dosing in a clinical study of this candidate in Europe in the second half of 2014. Finally, we plan to submit at least two INDs to the FDA for follow-on exon skipping drug candidates before year-end. In closing, we're working hard to address the inherent challenges associated with advancing an innovative technology in a complex rare disease. We are optimistic about our path forward and are hopeful that our discussions with the FDA will lead to greater clarity on our path forward for eteplirsen. We strongly believe in eteplirsen and its potential to benefit patients with DMD and are also excited about the opportunity to extend the clinical success we’ve had with eteplirsen to other genetic subtypes in DMD. With that operator we can now open the call for questions.

Thank you. (Operator Instructions) Our first question comes from Ritu Baral from Canaccord Genuity. Please go ahead.

Thanks for taking the question guys. Chris, a little more clarity on the information that FDA is requesting or you guys or has requested of you guys. Does it include non exon 51 6-minute walk data as well as information on pulmonary function. And based on the nature of the requests for data that you’ve gotten, where do you think the next meeting will -- what sort of endpoints you think the next meeting should focus on. Where do you think they’re going with the requests?

Yes, so we’ve mentioned they have had several requests at various time points over the last couple of months, and we tried to provide all of that data in the most expeditious timeframe possible. A lot of that has focused on dystrophin. Again just more detail we’ve provided them a lot, but this time the source data that is behind a lot of the summary data and specific patient level data that they had. They’ve have also, as we mentioned requested additional clinical data, that we provided them through a 120 weeks. So, on the pulmonary function they have all of our 120 week pulmonary function data and data on other clinical endpoints through that timeframe. They have also requested information as I mentioned on natural history studies and to ask us to attempt to get the detailed patient level data that supports some of those publications that were published by Elena Mazzone, Natalie Goemans and the like. So that is data that is outside of the exon 51 amenable population but would be consistent with exon deletions that decline at a comparable rate. And so, again that is to presumably get a better understanding of the natural history and our data in that context. And of course we know that they’ve communicated they’ve had the percent of GSK dataset in-house to be able to look at that specific exon 51 amenable population and how those boys fared over a year or longer. Regarding the next meeting with the FDA we just mentioned that we expect that to take place in March. We’ve not gotten a confirmed date, but we expect to get a confirmed date in March soon. And again we expect the bulk of that will focus on our confirmatory study design. But at this time I don’t have any further insights on the emphasis and focus of that other than getting alignment on a feasible and agreeable study design.

Got it. Were you meeting at those CMC meeting before you start dosing for the next trial?

No, we don’t believe so. We will have to submit the analytical testing and comparability data on our fill and finish, and then after a review period approximately 30 days we will know if that drug is able to be used in our clinical study.

Got it. And the last question, you mentioned one of the things you’re looking at right now, business development for the pipeline. Could you elaborate on that a little bit, are you referring to out-licensing of current earlier stage candidates or in-licensing of new assets. How are you looking at BD right now?

Yes, we’re not ready to disclose any details. What we have mentioned previously is that we’re very excited about our platform technology and our new morpholino chemistry and the application of those. We have been doing a lot of academic collaborations as data comes in that is compelling to convince us to move forward with new targets in new disease areas. We will communicate at the appropriate time that, that data supports it. We are open to licensing out our technology for really any applications, we think we have built an internal expertise in the rare neuromuscular space, so we think whatever data emerges to support other neuromuscular applications; we have to decide whether we can pursue that on our own or whether it's more appropriate with a collaborator and a partner. But I’d say, everything and anything is on the table as it relates to our platform technology, and stay tuned we know there’s interest out there from companies in our technology and so, and we know that the RNA space has seen a lot of types of deals done recently. And so that’s not lost on us and we look forward to engaging in discussions with the potential collaborators.

Thank you. Our next question comes from Chad Messer from Needham & Company. Please go ahead. Chad Messer - Needham & Company: Great. Thanks for taking my question. Is it possible for you to discuss what you think some of the potential issues that you need to workout with the FDA regarding the confirmatory trial, what you anticipate the discussion to be around specifically in terms of trial design?

Chad, I think what we’ve said previously, I think is the only thing that we can say currently which is, we have highlighted the challenges and feasibility of a placebo controlled trial to be able to actually produce a treatment benefit on the 6-minute walk test. Obviously going back to the November communications, they were open to considering other populations, other less known and a less validated endpoint for consideration. But we believe that the 6-minute walk is the most well characterized and most reliable of those endpoints and so we’d like to stick with that. And we believe that there are other controls that they can employ to understand if eteplirsen is producing a treatment effect in an open label arm. That would be the abundance of natural history data specifically with exon deletion patients that they have been published in the last year or so from multiple sites with a large number of patients. So that’s one control that they can determine what would be the natural rate of decline with a similar population. We know they have the GSK datasets which is the exact genotypic amenable population that we would be enrolling. So, we already have the benefit of a year of a controlled -- placebo controlled clinical trial following the same patients that we would be enrolling in our study. And then the third control would be this untreated exon deletion non-eteplirsen amenable population that we would follow prospectively to see how they would decline controlling for similar inclusion criteria and the natural history data does support that for most of these exon deletions that we would include due decline at a similar rate, exon 53, exon 45, exon 50 as an example. So, again those are the main discussion points that we look forward to getting alignment and agreement on with the FDA. So again it's hard to give more insight or perspective on that as this point. Chad Messer - Needham & Company: Okay, thanks. That’s much appreciated.

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

Great, thank you. Can you give us a quick update on the European strategy and if you expect you’ll be able to enroll European patients in the upcoming confirmatory study?

Well the European study that we described earlier was for exon 53 which we have good freedom to operate and we have been collaborating and we have a grant from the EU to help us through their early clinical development. So that’s where we have been working with several key investigators in Europe, and we have been having advisory meetings in Europe for the exon 53 drug and program. So that, again we’re not ready to disclose any further details at this time, but obviously we’re looking as we are in the eteplirsen in the U.S. application to have the most expeditious regulatory path possible. For eteplirsen in Europe we hope to engage the EMA in discussion of what it would take to gain approval in Europe with eteplirsen even though we currently have challenges with freedom to operate with eteplirsen in Europe. We just want to understand what would they be looking for if we were able to enroll patients in a clinical study in Europe. What endpoints would they be looking for, what kind of trial design is acceptable for the EMA. So we hope to engage in those discussions later this year on eteplirsen, but we do have confidence that our exon 53 program, we could arrive at a nice pathway forward for that drug in which we do have freedom to operate.

Great. Any comments on potential collaboration or negotiation with Prosensa in order to gain that freedom to operate in Europe?

Not at this time. Again we have filed an appeal to oppose the patent ruling on the November 2011 patent opposition in which we did not prevail on eteplirsen or exon 51. And obviously we want to get better clarity on what do we need to do in Europe and could we for example submit an application as an MAA in Europe on the Phase II data. That would be something that we want to understand better before we really thought about other alternatives to our current patent opposition appeal to get freedom to operate for eteplirsen in Europe.

Thank you. Our next question comes from Chris Marai from Wedbush Securities. Please go ahead.

Good morning, thanks for taking my questions. First with respect to exon 53 and the new studies you’re going to be running there; we want to look at the study design specifically would they allow for you to start enrolling beyond a sort of Phase I to full Phase I design into to be a pivotal study. And the with respect to your cash utilization guidance, what percentage of that if you could kind of break it out for us would be allocated to producing products for that trials typically, but also I guess your plans for the confirmatory trial. Thanks.

Yes, so Chris on exon 53 I mean all I would say at this point whenever you disclose the details, is we obviously have an eye towards expediting a regulatory approval of the exon 53 program, and so the quicker that we can get to a standard dose of which we believe 30mg would be an appropriate, safe and efficacious dose for the exon 53 program as well. And obviously ideally with more patients then we had the opportunity to dose in our Phase IIb study for eteplirsen in the U.S. And so those things would be putting us in a more favorable position vis-à-vis eteplirsen to have a more concrete dialogue about a path forward. Obviously we do expect to look at 6-minute walk test. We do expect to follow these patients for at least 48 weeks. We do expect to capture dystrophin, that’s the extent of what we’re willing to say about the exon 53 trial design at this point. Regarding the spend and the drug supply, obviously ramping up drug supply is a significant expenditure and we’re not at full commercial scale yet, so we haven't realized all of the cost efficiencies that we see in a commercial scale. And it's a significant part of our confirmatory trial cost and obviously if we start to ramp up for any future studies with other exons and or commercial supply for eteplirsen then that would be a significant investment. We have given guidance that assumes that we’re moving forward with the current plans for this year with the confirmatory study design. And as I mentioned ready to move to large commercial scale if we were to ever get a signal from the FDA on an earlier NDA pathway. Again that’s all we’re willing to say at this point on the drug cost.

Okay, and then with respect to the amount of drug that it has available, would you be able to dose say a 100 to 110 patients so the FDA request that similar trial design to those that we have seen in the past? Thanks.

Yes, what we said is that our mid-scale production capability will be sufficient to supply drug for the broader clinical trial needs for the organization. That includes our clinical study with eteplirsen including a size that you named there and we would just adjust accordingly if we needed more patients than what we had been planning on of about 60 patients treated. It includes the exon 53 ramp up for clinical studies in Europe. It includes the preclinical studies that I have been describing on exon 53, exon 45. So again more broadly our clinical trial needs and we will adjust change to a various exon if that’s needed but our mid-scale production capability should not put us in a position to be constrained in terms of drug supply for our broader clinical and pre-clinical program needs.

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

Thanks for taking my question. Can you talk about the hurdle rate you have internally for a potential partnership, what you’re looking for in a partner. We’ve obviously seen some large deals recently. I wonder if you’re hoping a partner maybe could bring expertise to the table as well as obviously some capital. And if it's, if your discussions center around people, the traditional orphan players or people outside that orphan arena?

I think if we look at what we’ve been able to generate with our technology over the last couple of years, we think it's very impressive. So not only the DMD data, but again our infectious disease dataset which is emerging with a proprietary good patent protection conjugated morpholino with safety up to 16mg/kg over 14 days. We have other advance chemistries that we’ve been working on over the last couple of years of which we can start to describe with potential partners. And obviously we think the dose window that we’re working with here in the therapeutic window and safety margin it's sufficient to get into multiple sell types and to go after various disease areas even beyond the neuromuscular rare genetic disease phase. So, understanding all of that, and now with a new CFO in place in our creed who has a lot of credibility, history and background in working with a lot of the RNA chemistry, now that he is digging in deep into what we’ve been able to generate, he’s very good at communicating the utility and application of RNA technology and soon morpholino chemistry specifically. And we are open to a variety of structures across a variety of therapeutic areas. And obviously we have seen a lot of deals in the RNA space, notably Alnylam did a nice deal where they had a equity component with a step up premium to that equity, that’s something we could consider as well. So, again we are, we have several companies that have expressed interest and we look forward to meeting with those companies and talking about all the potential applications and what type of deal structure to work with them. Obviously on DND we have the broader program there and as we have said we are comfortable with our path forward, in the U.S. we don’t feel that there’s any special expertise that were lacking to do that, but we’ve always said that for a ex-U.S. or even ex-U.S. Europe partnership we’re open to talking about all the other exons and obviously we have much stronger IP protection, freedom to operate beyond eteplirsen around the world for these other drug candidates.

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

Great. Thanks guys and thanks for the update. I want to dig a little bit deeper with respect to spend and I appreciate the comments that you’re giving. And I guess I am going to ask it two ways, so should we consider 4Q kind of what the spent is going to look like going forward. And the second component of that is what still remains to be paid from the government from the Marburg contract, should we be thinking something comparable to all of 2013 or something comparable to the fourth quarter of 2013?

Ted we gave guidance and generally speaking, you see a slow steady ramp up quarter-to-quarter, example you don’t give that detailed guidance at this point. So, again we gave the range that we said will be required to accomplish all of our goals this year. Regarding the Marburg, because we are in the process of determining what the extension of that first stage of the Marburg program of which we had the contract approved, we will find out this year what that next phase looks like. If that will be funded to what degree that will be funded and what potential revenues are going to be. That’s why we weren’t able to give guidance at this point on potential revenues related to that. And frankly as it relates to our spent and our operating loss projections it would have minimal impact because again as you know any expenditures are usually reimbursed and that is what dictates the revenue, so again that’s the extent that we were ready to talk about our future Marburg guy extension.

So just to be sure I understand that, there’s no government revenue’s in that guidance?

We have not given revenue guidance but -- and that’s because it's hard to predict what maybe extended or not. But we are still -- have an active program on Marburg in any expenditures that were incurring on that we do get reimbursed and creates revenue in calendar year 2014.

Okay, let me ask -- I am sorry, I am just going to ask one more question; is there dollar still to be paid under the Marburg contract?

Yes. We will be looking revenue against Marburg in 2014.

Okay, fair enough.

We have not given guidance because we can’t say what that’s going to look for the rest of the year.

Great. Looking forward to additional updates on eteplirsen and the rest of the pipeline. Thanks.

Thanks a lot, Ted.

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

Hi, this is Drew Prigodich on for Liisa Bayko. I had few questions; the first is I was wondering if you could give a comment on Billy Dunn the new Acting Director of the FDA’s neuro division and early interactions with him and his level of interest and experience with DMD. I was also wondering on the eteplirsen trial design are you still considering at all the possibility of enrolling patients in Europe for that confirmatory trial or is that something you’re looking to avoid doing given the current landscape, and the last question is, just on other exons, we talked a little bit about it, but I’m hoping you can give us a little bit more detail on how you see the landscape of other exons progressing and moving towards a potential classed approval down the road? Thanks.

All right. So, regarding the change of Acting Director to Billy Dunn and people like to speculate a lot. We really don’t want to add to any speculation with just commentary. Obviously he’s been a part of the division he’s in a better position than somebody who would be outside the division to understand this program the dataset and so we’re pleased that there’s an Acting Director that is from within the division. Regarding the EU patients again we don’t have the freedom to operate we’d like in Europe and so that challenges us with enrolling patients in a clinical study in which we don’t have a pathway to commercialize that drug. Having said that, we know that most of those European patients that would qualify for a study were exposed to drisapersen and so that brings complexities in and off itself. Number two, we know that Prosensa has communicated that they may look to begin re-dosing those patients. Number three, we haven't talked to the EMA about what are the requirements they are going to need in Europe for European patients to be enrolled in a study that may look like, have a different design than what our regulatory requirements are in the U.S. for the FDA. And as we discussed before, as you start to expand to additional sites particularly in different geographic territories and particularly when we’re trying to get dystrophin, biopsy samples, it adds complexity, variability and the need to power a study with more patients goes up, the more we expand to other sites outside of the U.S. So, these are all things that really complicate or make it less of an issue of our specific dialogue in discussions with the FDA. So, in regarding the other exons and the path forward, we have shared previously a pathway that we believe has the intention to show sequence independent, safety pharmacokinetics at a standard dose and sequence dependent dystrophin production and efficacy. And as that data continued to emerge to prove those things we think we’re in a better position to have a dialogue with the regulators both in the U.S. and Europe to consider an accelerated pathway for the follow-on exons and ultimately a class approval where it's infeasible to do clinical powered studies against the rarest exons beyond the first few exon skipping candidates. So with that if I can ask the other analysts to hold their questions to one key question as we still have several on the queue. Thank you.

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

Thanks. This is Mohit for Robyn, thanks for taking my question. So, I have a question regarding the study. So like, assuming that you’ll get the clarity by the end of this quarter or earlier next quarter and then you’ll submit a protocol, I mean how soon we can see a patient first patient being dosed under study. And then I mean is there a possibility of looking, including patient outside of Europe and U.S. because for instance that did include patients from Latin America and Asia. Thanks.

So, your last question I just spoke to which is the additional recruiting that would be required to go to different countries and other sites and the variability that went into, and the control that we would need around the surgical biopsies, the dystrophin analysis and the variability that we would expect on 6-minute walk, doesn’t necessarily make the job any easier in terms of powering a study and the feasibility of finding the patients by expanding to these other countries. And so then we also have to overlay that with freedom to operate an IP. Regarding your other question in terms of, am sorry what was the -- the dosing, we did say on the call and the scripted comment that it's unlikely that we’re going to be able to have first patient dosed by the end of the second quarter, given we still have a meeting in March that we hope to confirm with the FDA to discuss those details. And then obviously we have to finalize the protocol. We have to submit to the FDA. Usually it's about 30 days for them to review and make sure that there is no issue’s that they would raise concerns about the study design. Then we have to submit to IRB, then we have to screen patients, then we have to dose. We also have to parallel submit the analytical testing comparability data to the FDA to get drug released. So there is a lot of moving parts that doesn’t happen. We get a protocol and then two weeks later we can dose. So that’s why we think it's unlikely to get that first patient dosed by the end of the second quarter. But if we resolved the study design by the end of this quarter we think hopefully, shortly thereafter we can have first patient dosed.

Thank you. Our next question comes from Debjit Chattopadhyay from Emerging Growth Equities. Please go ahead.

Hi, Chris, thanks for taking the question. You just mentioned about or talked about the variability. We have heard from top leaders and you’ve seen that in the Prosensa trial, 6-minute walk distance tests are administered very differently depending upon where you are. So, how do you standardize that given that, that’s most likely going to be a primary endpoint?

Yes, Ed do you want to address that?

Yes, we have actually put a great deal of effort on considering the 6-minute walk test. So to reduce the variability, we would have a small group of expert physical therapists who have a lot of experience in a 6-minute walk test and we have set a whole guideline of training and reproducibility that will follow them and throughout the study. So we believe that we can reduce a lot of those variability and we’re also reducing the number of types so that not everyone is impacted during the 6-minute walk test. We have regional centers of excellence that will be doing it. So I think these should reduce a lot of the variability in the testing.

Yes, this is where it's good to follow other companies who treaded this ground, so we can be more informed and thoughtful about controlling and containing that variability and have more consistency of that clinical measurement.

Thank you. Our next question comes from Bill Tanner from FBR Capital. Please go ahead.

Yes, thanks for taking the question. Chris, just on the manufacturing I am curious how confident you are that the analytics of the mid-scale batches will be satisfactory, trying to separate what might be the complexities of this emphasis versus just operator error. And then why haven't you gotten the minutes from the meetings last year seems curious and does that kind of leave you in the dark maybe a little bit just to how the FDA is viewing things?

Yes, so regarding analytical testing we’re confident right that we’re going to have drug that’s useful and acceptable to the FDA for dosing our confirmatory study. And so again we can’t provide more data until we have that at this point, but again we believe with confidence so we can start dosing but we have to just kind of go through that process and submit that data and get FDA’s clearance on that. Regarding the lack of minutes look again the FDA is under no mandates to provide minutes. We mentioned back in November when they gave us the second meeting in November that it was unlikely that we were going to get individual minutes for each meeting because it was an ongoing dialogue. Well, I think through December and through the external meetings that they have had in December and in January that were put on or earlier this month, they got more information about DMD from experts. And so we think that precipitated more inquiries about our dataset, the source data, and so I think they’re still studying and analyzing and I would not read into or speculate what that mean other than the FDA is still doing some work on this before they provide us more definitive direction and guidance. That's all we know at this point. I think it's kind of a false exercise to try to speculate what that would mean.

Thank you. Our next question comes from the Joe Schwartz from Leerink. Please go ahead.

Hey, thanks for taking my question. It’s Paul on for Joe. I'm just wondering what your updated thoughts are on the types of patients that you’d hope to enroll in a pivotal trial both with respect to age and baseline 6-minute walk? And then also it is now the compelling respiratory data you presented could enable you to enroll older patients and trying to show a benefit there, even if there are – they’re able to do 6-minute walk? Thanks.

Yes. So what we have said for a long time now that based on all the natural history data, based on all the clinical studies that had been done in DMD with other dystrophin producing technologies that -- you really need to stick with the over seven population that in almost every natural history and study you see increases on 6-minute walk in boys that are less than seven. So the variability is much higher and it becomes very confounding to interpret any type of treatment benefit that would be derived in any patients less than seven. I think dystrophin production is another matter. You can measure dystrophin less than seven, when it comes to clinical outcomes particularly 6 minute walk, we think it's very difficult to try to glean a treatment effect on that clinical outcome. In terms of 6-minute walk, just like we tried to achieve with the Phase IIb where we did not want boys who were too far gone, who are in the precipice of that loosing ambulation, but likewise, we did not want patients who were too healthy. So unlike a lot of the clinical studies that have been conducted and including natural history studies like the Craig McDonald where the conclusion was drawn at boys over 350 have general stability, they included boys who were over 400 meters, over 450 meters, many had over 500 meters. We believe and agree that those kids are too healthy. If you're over 450, let's say where you would see general stability or may be even an increase if you have different phenotypes, than DMD included in that. So again, we expect over seven years and to some restriction requirement of the 6-minute walk test. Regarding the non-ambulant pulmonary function, obviously we’re encouraged by our pulmonary function data and we're looking into figuring out if that is something that we could look at in older population, but we do think the ambulatory is also important to get that pulmonary function data. Ed, do you want to mention anymore on the pulmonary?

Yes, I think one of the things that has become quite clear is that the pulmonary function especially is a MIP and the MEP percent predicted begin to decline probably after seven years of age and those were steady decline all the way through the teen years. And so I think we just need to collect more data, we are collecting more natural history, we are looking at a lot of the new pieces of information that are coming out and I think we'll have a better idea of how to use this data in the clinical trial. But I think it is going to be an important parameter that we are going to need to follow and it also does allow us to look at the non-ambulatory population.

Thank you. Our next question comes from Yaron Werber from Citi. Please go ahead.

Great. Thanks for taking my question. Chris, just trying to follow-up and understand a little bit, I mean, I’m trying to get a sense here. Do you really think accelerated approval is really on the table or is FDA just trying to collect as much natural history to really allow for new studies? And then secondly, you just -- all the other companies have had to generate randomized data with a control using exon 51. So I'm just trying to get a sense how realistic is it to have a multi-exon control? Thank you. I'm just trying to get a level of the consistency FDA has to play with.

Sure, right. So first regarding the any commentary on a change in their position on accelerated approval or early NDA. As I mentioned, the FDA feedback and guidance has not changed since we last communicated their November pre-meeting comments. We can't speculate on any change in their position at this point or read into these request for data. And again, we’ve been focused from the beginning of that November on clarifying the trial design and the confirmatory study design to move forward with eteplirsen. So regarding the -- again, non-exon 51 population as a control, first, I'll just remind you that this was one of three controls that we highlighted. Natural history and the GSK clinical trial placebo arm, which is in possession at the FDA, but all of the natural history data and in fact including a presentation by Eugenio Mercuri, who has one of the largest and longitudinal databases, suggests that other factors are a bigger influence over the course of a DMD patient than the exon deletion. So for example, age, if you look at all these other exon deletions you see increases in patients below seven and decreases in all patients over seven. Those decreases are generally consistent regardless if its exon 51, exon 53, exon 45 and that any variability which there always is variability is -- we believe, driven by a lot of other factors, individual patient factors, phenotypic considerations et cetera and not the exon deletion. And just a reminder, we had five different genotypes within our eteplirsen study and there was no difference in dystrophin production. And again, the stability and clinical outcomes and decline of the placebo arm were very similar. So that’s what gives us confidence, but again the FDA is considering that and studying that. So thanks for your question, Yaron.

Thank you. Our last question comes from Kim Lee from Janney Capital. Please go ahead.

Good morning. Thanks for taking the question. Just a quick question on your requested meeting with the FDA in March, how likely you think it is that you guys will be discussing the child design for the confirmatory study versus could the FDA want to talk about something else maybe all of the data that you’ve collected? Thanks.

It’s hard to say I think, we go into these meetings with our thoughts of what guidance we need. But the FDA is working hard to understand our dataset, to understand DMD. So for example, the December meeting in which we were hoping and expecting a detailed discussion on the confirmatory design, was largely spent on interpreting and translating our clinical dataset and data that we had provided to them previously. So we used a lot of time answering questions, clarifying points around our clinical dataset and almost no time or essentially no time to discuss the confirmatory studies that I talked. Its hard to say, but we know that they’ve been working hard and we hope to have a more comprehensive definitive perspective and again -- but we’ve been focused on trying to get clarity on that, on the trial design.

Thank you. We have no further questions at this time. I'll now turn the call back to Chris Garabedian for closing remarks.

Yes, thank you. So again, thanks everybody for your interest in Sarepta. We are very hopeful that we'll get clarity from the FDA in the coming weeks and hope to confirm that March meeting very soon with them and at that point we’ll be on our way and we’ll be able to give a better guidance on what that trial design looks like, the number of patients, when we can dose first patient. So we again, hope to resolve that and provide clarity to all of you once we have more definitive read from the FDA. So thank you and we’ll talk to you on the next call.

Thank you. Ladies and gentlemen, this concludes today's conference. Thank you for participating. You may now disconnect.