Sarah Bard - IR Chris Garabedian - CEO Sandy Mahatme - CFO Ed Kaye - Chief Medical Officer.

Tim Lugo - William Blair Jeremiah Shepard - Credit Suisse Brian Klein - Stifel Ritu Baral - Cowen & Company Brian Skorney - Robert W. Baird Steve Brozak - WBB Securities Christopher Marai - Oppenheimer Kumar Raja - Citi.

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

Thank you, operator; and thank you, all, for joining today's call. Earlier today, we released our financial results for the third quarter of 2014. The press release is available on our website at www.sarepta.com and our 10-Q was filed this morning.

Joining me on the call today are Chris Garabedian, Sarepta's Chief Executive Officer; Sandy Mahatme, Sarepta's Chief Financial Officer and Ed Kaye, Sarepta's Chief Medical Officer.

I would like to note that during this call, we will make a number of statements that are forward-looking, including but not limited to statements about our plans to work with the FDA to achieve an acceptable NDA submission and filing for eteplirsen, including our plans and ability to comply with FDA requirements and requests for data, information and analyses.

Our plans and ability to include such information in an eteplirsen NDA submission and the projected timing for the same.

The potential and timeframes for an NDA filing by the FDA and regulatory approval for eteplirsen; the potential for eteplirsen to qualify for accelerated approval through pathways in the EU; potential for further development of our Ebola product candidate; availability of drug product and number of treatment courses we could potentially make and the expectation that any Ebola development efforts would not impact efforts in our DMD program.

Our release relating to the potential safety, use, clinical benefit of and market for our product candidates and platform technology and communications with regulatory authorities, government entities and international bodies relating to the same; exploring development opportunities with various third parties and updated guidance on expected non-GAAP loss from operations.

These forward-looking statements involve risks and uncertainties, any of which are beyond Sarepta's control. Actual results could materially differ from these forward-looking statements as any such risks can materially and adversely affect the business, results of operations and the trading price of Sarepta's common stock.

For a detailed description of risks and uncertainties we face, you're encouraged to review the company's most recent annual and quarterly reports and other official corporate documents filed with the Securities and Exchange Commission. With that, let me turn the call over to Chris Garabedian, Sarepta's Chief Executive Officer.

Chris?.

Thank you, Sarah and good morning, everyone. Today, I would like to provide an update on our recent communications with the FDA regarding an NDA submission for eteplirsen, our lead product for the treatment of Duchenne Muscular Dystrophy or DMD along with an update from Dr.

Ed Kaye, our Chief Medical Officer on the status of our broader DMD clinical program and the various studies that are underway in process or in discussion. Our corporate update will be followed by an update of our latest financials by Sandy Mahatme, our Chief Financial Officer.

First, I'd like to address our recent communications with the FDA related to our pre-NDA guidance and meeting minutes that was outlined in our October 27th press release and conference call as well as a subsequent posting by the FDA on their website on October 30.

We've received a tremendous number of questions from investors, the patient community and even congressional staff regarding these communications and I'd like to make a few comments in response to these inquiries with the hope that we can focus on the events that are ahead of us and allow us at Sarepta to work constructively with the FDA to ultimately achieve an acceptable NDA filing for eteplirsen.

In particular, I would like to address recent disclosures related to our FDA discussions, comments related to the dystrophin message and data set and the specific guidance related to our NDA submission.

Since the public disclosure of our 48 week clinical data from study 201, 202 in October of 2012 and the subsequent initiation of discussions with the FDA that have continued over these two years to determine the feasibility of an accelerated approval of eteplirsen.

We’ve understood the intense interest among investors and the patient community in our discussions with the FDA and the FDA's guidance to the company.

To this end, we have sought to be more transparent and detailed in our updates related to our FDA interactions than is typical, for example, we’ve chosen to provide specific excerpts from the FDA communications to make abundantly clear the exact language we relied upon in our communications to investors, patients and others Sarepta stakeholders regarding the FDA's position on considering eteplirsen for a potential FDA approval under the accelerated approval pathway.

Updates of these types have been provided by us beginning in April of last year in connection with meeting minutes from a March 2013 guidance meeting with the agency up to our most recent update on October 27 last week.

We understand the importance of a productive relationship with the agency, the challenges they face with limited resources and the need to satisfy their requests as they evaluate first-in-class technologies and endpoints.

It is for that reason in each of these communications we discussed and acknowledged the FDA's request for additional data or information in order to support a successful filing consistent with the FDA's open letter last week.

For example, our April release specifically quoted the FDA's April guidance letter that "With additional safety and efficacy data and NDA should be filable." And included excerpts outlining a number of suggestions we discussed with the FDA for additional information or data that could be used to supplement what was then the existing data set.

In support of this ongoing dialogue reaching back to December 2013, we have responded to 18 different FDA requests which encompassed approximately three dozen specific requests for information.

In addition, in an effort to work with the FDA to obtain clarity regarding their expectations, we held discussions with the FDA that preceded and in part form the basis of our April announcement.

As part of those clarifying discussions, we described in detail the type of information we can provide the timeframes in which it could be provided and confirmed that it would be acceptable to submit data, whether it be additional safety or additional clinical data after the NDA was submitted by us or even after an NDA filing by the FDA during the review.

Acknowledging the ongoing dialogueue with the FDA regarding their needs for additional data to be provided in connection within NDA which we have described consistently in regulatory releases going back to March 2013, the most recent October meeting minutes introduced more detailed requirements and requests that had been previously discussed.

It is these new more specific requirements and requests that have impacted our previously disclosed timelines, for example the meeting minutes expanded our prior commitments to provide the limited safety data we anticipated would be available at the end of this year and to update that data during the review process by introducing for the first time a specific number of patients and a specific duration of safety that should be included at the time of an NDA submission, in this case, a minimum duration of three months in 12 to 24 new patients.

In addition, the FDA communicated in the meeting minutes a new requirement that an independent reassessment of our dystrophin positive fibers analysis and a 168 week clinical data from study 202 be included in our NDA submission rather than at a later time point such as prior to filing or during review, an evolution in the previously discussed flexibility for data submission based on "the market disparities" resulting from a site visit to nationwide hospital in May 2014.

Despite our ongoing belief in dystrophin as a surrogate endpoint and in nationwide as a site, we have long felt it important to communicate the FDA's reservations regarding dystrophin and have done so repeatedly since March of 2013.

For example, in our April 2014 release, we cited FDA's skepticism about the persuasiveness of the dystrophin data and included excerpts that made clear, they were still uncertain whether the existing data would be persuasive enough to serve as a surrogate endpoint and that additional work was necessary.

As part of that April guidance and the ongoing dialogueue regarding additional efficacy information, the FDA highlighted ways to allay these reservations including how a fourth biopsy could enhance the argument for dystrophin as a surrogate for accelerated approval and further suggested a collaboration with Sarepta that included their visit in May to the nationwide site to understand and evaluate the dystrophin quantification protocols administered as part of study 201, 202 as well as others dialogue including a conference call orseveral conference calls this summer outlining several methods of dystrophin quantification which included one where Francesco Muntoni provided an overview of his recent publication in The Annals of Neurology showing the reproducibility of immunohistochemistry and western blot methods across multiple independent labs and participation by Eric Kauffman, and a researcher from his lab presenting an alternative method dystrophin quantification.

While we were aware of the FDA's visit to Nationwide Children's Hospital in May and helped facilitate it, no specific FDA concerns in connection with the site visit were communicated prior to the October meeting minutes.

Indeed no return summary of the site visit was received by either Nationwide or Sarepta prior to the meeting minutes and no 483's were issued. In July 2014 we received a request for a reassessment of our primary dystrophin endpoint by independent pathologists, along with several other request for information related to our dystrophin analysis.

However, this was not presented as a requirement for an NDA submission nor was it described as resulting from concerns uncovered as part of the Nationwide site visit.

Rather the request for a reassessment of dystrophin-positive fibers by independent labs was one of the 18 different occasions and approximately three dozen data and information request received by Sarepta by the FDA over the past year.

In an effort to address this request, we have been working with the FDA to agree on a reassessment protocol and received initial feedback last week after our press release and conference call with specific questions and concerns that we will use as we work toward a final protocol over the coming weeks.

To be clear, we stand by the quality of the work that was conducted by Nationwide Children's in Columbus, Ohio and believe they are one of the most experienced clinical trial sites in Duchenne and a neuromuscular diseases in the world.

We continue to feel privileged to have them as part of our past, current and future studies as we consider them one of the best sites available to conduct high quality DMD clinical studies.

In summary, as stated in the FDA letter last week in our press prior releases, we have had ongoing discussions with the agency regarding potential additional safety and efficacy data, but it is the more specific requirements and requests introduced in October that have resulted in our updated submission timelines.

While we thought it important to clarify these recent communications, we’re now focused on moving forward with the FDA to further the regulatory process for eteplirsen. As part of this ongoing dialogue, we now have detailed request from the agency as to what they would require and expect to see in an NDA submission to enable an acceptable NDA filing.

Armed with this information we will work towards fulfilling these requests over the coming months with the goal of a mid-year NDA submission, as the FDA has requested we will incorporate safety data in newly exposed eteplirsen patients,.

160-week clinical data, dystrophin reassessment data from independent labs and any additional data we’re able to obtain from independent groups related to natural history and MRI in DMD patients into our NDA.

Additionally, we were still pursuing a fourth biopsy to provide eteplirsen's long-term effect on dystrophin levels in the muscle and the ability to provide multiple assessments of dystrophin including immunofluorescence RT-PCR and quantitative measures of protein such as western blot.

We believe this additional dystrophin data if positive will be important as we prepare for a potential advisory committee panel during eteplirsen NDA review and will be beneficial information to have in a future package insert to inform clinicians who might be interested in eteplirsen further DMD patients following the potential approval of the drug.

We look forward to the ongoing dialogue with the FDA with an aim of completing this important step in the regulatory process.

While the regulatory discussions and activities related to our eteplirsen NDA are very important, we've been equally focused on getting additional DMD boys on eteplirsen and our follow-on exon skipping drugs across our broader DMD clinical program. We continue to advance clinical studies with eteplirsen and our follow on exon skipping drugs for DMD.

We have 2 of 3 new eteplirsen studies with open sites enrolling patients with dosing to begin this month in our confirmatory study and ambulatory patients and our study in advance stage patients which includes patients who are non-ambulant. We have also initiated our exon 53 study in Europe and will begin screening patients later this month.

We are meeting with the FDA to discuss our randomized placebo-controlled master protocol for exon's 45 and 53 and hope to have feedback by year's end on a final protocol.

Let me add that while we have experienced some delays in ramping up our multiple clinical studies compared to our expectations we had in April and May, we’ve tried to ensure the optimal conduct of these studies in generating the highest quality data.

Our dosing in the eteplirsen studies are all beginning within a couple of months from the originally anticipated aggressive timelines as we’ve tried to balance getting drug to additional patients as soon as possible without compromising the quality of the trial and data that is collected.

With that, I'll turn the call over to Ed Kaye, who will provide a detailed update on our DMD clinical programs..

Thank you, Chris. Good morning, everyone. I will now provide an overview of all of the activities related to our DMD clinical program and the progress we've made since our last update. During the past several months, the teams at Sarepta, our CROs and the individual respective sites have been working very hard to initiate four new clinical trials.

These trials include study 301, our open-label eteplirsen confirmatory study in ambulant patients greater than seven years of age, study 204, our open-label eteplirsen study in non-ambulatory DMD patients or those who are more progressed and don't meet the minimum walking distance on the 6-minute walk test for study 301.

Study 203, our open-label eteplirsen study in younger DMD patients between the ages of 4 to 6 years and finally, our study testing the exon 53 skipping drug, SRP-4053 consisting of a two-part study, part one being a blinded, placebo-controlled, dose titration phase followed by a larger open-label Part II extension study.

As most of you know, there are many time-consuming aspects involved in the study startup. The sites need to be identified and qualified prior to the initiation of the process. Investigators, physical therapists, health authority and IRB approvals, informed essence and consents, contracts, budgets, all need to be established for each site.

IRBs will frequently ask for clarification of the protocol and specific language for the essence and consents need to be adjusted for each institution. An investigator meeting and site initiation visit are required prior to screening the first patient. Also, prior to collecting efficacy data, the physical therapist at each site must be certified.

Working with our CROs, we’ve assembled a very experienced Sarepta team of clinical operations experts, physicians, statisticians and data managers who have previously worked at many of the Massachusetts based biotech and large pharmaceutical companies.

We also have dedicated contract specialist and a travel coordinator who are focused exclusively on these clinical trials.

The confirmatory study 301 is projected to consist of 39 sites, 14 of which will be regional efficacy centers, two are designated as biopsy centers and 25 sites will be performing infusions and collecting safety and laboratory data. We’ve four sites already initiated and four study initiation visits planned for next week.

Patient screening began last month and we are scheduled to begin dosing the first patient during the third week of this month. Study 204, which may include up to 10 sites, has one site initiated and three patients undergoing screening this week.

Dosing for the first patient enrolled in this study is anticipated to occur next week and another site initiation is planned within a few weeks. Study 203 involves younger patients and is right behind the other studies with regard to operational activity.

Up to 10 sites could be chosen for this study and we’re in the process of contracting for the various centers. Site initiation and screening is expected to commence by the end of this year or early next year.

Finally, the exon-53 study in Europe is well on its way with Investigator Meeting occurring a few weeks ago and we already have two sites initiated. Site selection has been considered a priority for these studies and we have some of the most experienced neuromuscular centers in the country and in Europe.

We’re fortunate to be able to continue to include Nationwide Children's Hospital as a major participant. Nationwide Children's Hospital and their Neuromuscular Research Center under the direction of Dr. Jerry Mendell is one of the most experienced clinical research centers with decades of experience in clinical trials in DMD and other diseases.

They have pioneered research in DMD, spinal muscular atrophy and other diseases and developed novel techniques for various drug and gene therapy approaches. The Neuromuscular Laboratory at Nationwide has performed dystrophin analysis for diagnostic and clinical trials and serves as a national center for diagnostic muscle biopsies.

We are also very pleased to have the Neuromuscular Group at the University of Iowa which has extensive experience in clinical trials, research and muscle biopsy. Both of these institutions are designated by the NIH as Paul Wellstone Centers for Neuromuscular Research.

The regional centers that collect the efficacy data are all experienced and highly recognized research and clinical care centers in DMD and have exceeded the stringent requirements we have set forth in order to become a designated research center.

In summary, we have been very busy with getting several new studies up and running and are excited to begin dosing with new patients with eteplirsen imminently. We are also pleased to be working with dozens of more clinical sites in the U.S.

with many more investigators that will gain experience with eteplirsen along with leading sites in Europe that will be the first to evaluate our exon-53 drug candidate, SRP-4053, our second exon-skipping drug to be dosed in DMD patients.

Finally, in addition to posting clinical trial information and enrolling in sites like clinicaltrials.gov, we also provide information to patients interested in our clinical trials in our patient information website www.skipahead.com.

Now I'll turn over to Chris to speak about our other programs in infectious disease and our discovery research efforts..

Thanks, Ed. But first related to our DMD program, I'd like to restate that we have final drug product of the drug that we need to begin dosing all of the patients across these clinical studies and our manufacturing runs have produced quality drug product that have met the release specifications discussed with the FDA for our eteplirsen studies.

As I mentioned on our regulatory update call last week, we have had our pre-NDA CMC meeting and received our meeting minutes with no significant concerns raised related to what we discussed, related to our CMC section for the NDA.

Turning to Europe, we’re tentatively scheduled to have a meeting with the EMA and waiting for a confirmed date from them on the pathway to discuss the pathway for eteplirsen in Europe and the possibility of a conditional approval based on our existing and emerging dataset.

Before turning the call over to our CFO, Sandy Mahatme for a financial update, I'd like to address the many increase we received related to the status of our Ebola drug AVI-7537 and activities related to the ongoing outbreak as well as provide an update on our pandemic influenza program along with other research efforts, all of which underscore the versatility of our PMO chemistry platform.

First, I would like to touch on the ongoing Ebola outbreak as we've received a significant number of inquiries and interest over the past few months from investors, analysts, government agencies, the media and most recently several hospitals and treatment centers across the country regarding the status of our Ebola program, our drug supply and our ability to potentially help.

The Ebola crisis has estimated to have already affected more than 13,000 people and resulted in over 4800 deaths reported so far this year. We’ve continued to keep government officials informed on the progress we've made with respect to product availability.

We recently completed, fill and finish of the available drug product in vials and ready to go and have the potential to supply drug for over 20 treatment courses depending on several factors and assumptions regarding dose duration and weight.

We could also potentially make enough drug products for over 250 more treatment courses using subunits remaining from the contract we previously had with the Department of Defense more than originally reported as we identified supply of additional manufacturing components to bring our raw drug materials to allow more final drug product.

To be clear these activities would not interfere, de-prioritize nor impact our plans to get eteplirsen and our other DMD candidates to the boys who need them. Given the number of companies who have come forward recently highlighting their respective drug activity and experience against Ebola virus.

I would like to add as a reminder that we receive DoD funding from July 2010 through October 2012 for an Ebola Therapeutic Advanced Development Program leveraging our proprietary PMO chemistry platform.

During that time we generated some compelling primate and early clinical data in collaboration with USAMRIID, BSL-4 lab at Fort Detrick our lead Ebola compound AVI-7537 that leads us in the Department of Defense to believe in its potential to fight the Ebola virus. FDA designated AVI-7537 as an orphan drug and granted it fast track status.

However, the program was unfortunately halted in 2012 due to funding cuts, despite the suspension of this program two years ago, we believe we’ve one of the most robust animal efficacy in early clinical data sets of the handful of Ebola therapeutic candidates in development, specifically our extensive experience with hemorrhagic fever viruses spans across Ebola and Marburg a similar Ebola like virus and we've studied over 200 primates in qualified BSL-4 lab settings and across both indications over 150 primates have been treated with either our Ebola or Marburg, PMO plus candidates demonstrating between 60% to 100% survival compared with 0% survival in all the untreated animals consistently.

AVI-7537 has also been shown to be safe and well tolerated in a completed Phase I single-ascending dose study that featured AVI-7537 in combination use with another compound that was later shown not to contribute to efficacy and subsequently dropped from the program.

AVI-7288 Sarepta's Marburg candidate, which again uses the same PMO plus chemical backbone as our Ebola drug candidate has been further tested in a multiple ascending dose clinical trial with 30 healthy human volunteers and has proven say up to 16 mg/kg per day for 14 days with no serious adverse events and no observed maximum tolerated dose.

The existing clinical and pre-clinical data for these PMO plus compounds, including nearly identical pharmacokinetic profiles and drug-like characteristics strongly indicate sequence independent safety such that a similar safety profile can be expected across compounds.

In addition, recent results from an NIH conducted Phase 1 study of AVI-7100 Sarepta's lead influenza drug candidate which also utilizes the PMO plus technology found no dose-dependent adverse events or toxicity and further support safety of our PMO plus compounds irrespective of the target.

Therefore, we believe the safety and tolerability data obtained from the AVI-7288 multiple ascending dose study can be extrapolated to both inform and support the use of AVI-7537 and its safety in an emergency situation.

Based on our non-human primate studies in Marburg and Ebola we believe a dose of 16 mg/kg per day for 14 days may provide protection in an infected or exposed patient with Ebola and based on our safety results with Marburg we expect our Ebola drug to be safe for administration at this dose.

Given the dire circumstances and expected outcome for any person who becomes infected. It seems the risk-benefit ratio for this application should be more than justifiable for administration of our drug.

We’re in discussions with international bodies, including the International Severe Acute Respiratory and Emerging Infection Consortium, the ISARIC; the World Health Organization, Medecins Sans Frontieres or Doctors Without Borders, the Wellcome Trust, among others as well as the U.S.

government along with what we understand to be a small handful of other companies with Ebola drug candidates regarding clinical trials that are expected to be stood up in the near term to explore safety and efficacy of Ebola therapeutics in patients infected with Ebola.

We’re providing information and data to these entities to help as they prioritize their efforts. We are also exploring opportunities to continue advancing the development as funding of AVI-7537, including the generation of additional safety, efficacy data and support for manufacturing scale-up.

Given reports that the Ebola virus is mutating rapidly as it spreads, I think it is important to note that AVI-7537 targets a highly conserved sequence noted as VP24 and that the mutations to-date do not appear to impact AVI-7537's targeted region and so we would not expect efficacy of our drug to be compromised whatsoever.

I mentioned earlier our influenza drug, AVI-7100, has completed a Phase I, single-ascending dose safety study in healthy volunteers and I just want to expand on that for a moment. To-date, we’ve dosed up to 8 mg/kg with no safety issues. This is the third drug with no safety issues with our PMOplus chemistry.

The Data Safety Monitoring Board recommended proceeding to the multiple dose portion of this study, which will examine a dose of 8 mg/kg per day for seven days. We expect dosing to be initiated by the end of the year and to complete the study in the first half of 2015.

As a reminder, this study is being funded and being conducted by the NIH's NIAID division.

Despite challenges associated with developing drugs with an unpredictable commercial market and the reliance that this creates on government support and the potential for stockpiling, we do believe there is value in continuing to advance our infectious disease pipeline in the interest of public health and national security.

We’re encouraged by the recent news with the White House pledging billions of dollars in commitment to the current Ebola crisis and we’re committed to exploring the potential to partner with various agencies and seek to establish public-private partnerships that can advance a rapid response capability for our nation.

The ongoing Ebola outbreak is a reminder that versatile technology like ours, that can address Ebola, but also be adapted to newly identified infectious disease threats, needs to be developed before the next threat evolves into a nation-changing or global health cross crisis.

Lastly, I would like to mention that we are excited about the potential of our platform technology and have been collaborating with several academic institutions on evaluating our proprietary chemistries against novel targets and disease models beyond DMD and the current viral targets I just mentioned.

We've made some advances in identifying some potential new targets and applications for our proprietary PMO technology; and while we’re preparing to provide an update on some of these activities next week at the Credit Suisse Investment Conference on Tuesday, November 11th as we’re committed to building a pipeline beyond our promising DMD portfolio and our current infectious disease viral pathogens.

At this time, I would like to turn the call over to Sandy Mahatme, our CFO to provide an update on our third quarter financials.

Sandy?.

Thanks, Chris. Good morning, everyone. This morning's press release provided details for the third quarter of 2014 in both an adjusted or non-GAAP basis as well as a GAAP basis. The press release is available on the SEC and company websites.

The non-GAAP results we will discuss on this call provide a more accurate picture of our ongoing operations and the impact of operations on our cash balance and they exclude the impact from the valuation of our outstanding warrants and stock compensation expense.

I would like to point out that all warrants have been exercised or have expired as of the end of the third quarter of 2014. Please refer to our press release for a full reconciliation of GAAP to non-GAAP.

In the third quarter of 2014, we reported an adjusted or non-GAAP net loss of $28.8 million or $0.70 per share compared to non-GAAP net loss of $21.3 million or $0.63 per share in the third quarter of 2013.

The incremental loss is primarily the result of an increase of $4.5 million in R&D and G&A expenses due to corporate growth and a decrease of $3.1 million in revenue from government contracts. Revenue for the third quarter of 2014 was $1.1 million down from $4.2 million in the third quarter of 2013.

The $3.1 million decrease was primarily due to lower revenue from our various government contracts.

The government contract under which the Marburg drug candidate was being developed expired in July of 2014 and we’re currently evaluating options to continue advancing our Marburg drug candidate and other infectious disease research and development efforts.

Adjusted research and development expenses were $20.2 million for the third quarter of 2014 compared to $19.9 million in the third quarter of 2013, an increase of $0.3 million.

Adjusted general and administrative expenses were $9.9 million for the third quarter of 2014 compared to $5.7 million in the third quarter of 2013, an increase of $4.2 million.

The balance of our cash, cash equivalents and investments was $240.7 million as of September 30, 2014 compared to $264.9 million as of December 31, 2013, a decrease of $24.2 million.

The decrease was primarily driven by the use of cash to fund our ongoing operations during the first nine months of 2014, offset by the net proceeds received from our public offering in April of 2014.

Turning to our 2014 full year guidance, we continue to maintain a strong balance sheet and expect to end the year with more than $200 million of cash, cash equivalents and investments.

In light of the recent FDA guidance for eteplirsen NDA, we have reduced our expenditures for the remainder of the year and we now expect non-GAAP loss from operations to range from $110 million to $120 million versus our previous guidance of $135 million to $145 million.

We expect that our current financial position and prudent expense management should support the execution of a strategic business plan as we continue our inventory build and prepare for the potential commercialization for eteplirsen while also working to advance our pipeline in DMD and infectious diseases.

With that I would like to turn the call back over to Chris..

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

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

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

Well, with the fourth biopsy, we would intend to do multiple dystrophin measures. I think western blot will definitely be part of that, immunofluorescence will be part of that.

Obviously we want to have a portion of it to be consistent with what was done previously for comparison, reasons to the existing data set, but also to be able to correlate other measures of dystrophin, obviously the method that you mentioned is another way to quantify.

And so, we will be determining what other exploratory measures beyond the standard measures we will consider. And so we’re discussing all of that internally before we actually finalize that protocol..

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

And we're in the process of doing that and obviously working with a lot of different people to make sure that we're all in agreement that we have the best methodology..

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

No, Tim, that's not accurate. The reassessment was specific to our dystrophin analysis and specific to the dystrophin-positive fibers.

They linked the need for a 168-week clinical data in that passage that was outlined in our press release and the only context further that they wanted was for us to attempt to get all of the 6-minute walk data from the natural history cohorts and you know we’re attempting to do that..

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

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

Yes, we're not ready to provide guidance on that at this time.

Part of this I explained on the last call is first we need to ensure that when we're going to get that 168-week data, we did provide flexibility to extend that 168 timeframe through when that fourth biopsy would be collected, we thought it was more important to prioritize, scheduling that fourth biopsy and this is a big burden on the families and so what we wanted to do was give the site flexibility to not demand two separate visits all of these boys and families are flying in from around the country for both the clinical visit and the biopsy.

And so some of them can't find the time to do that and so we gave them flexibility. So we're not even sure when we'll have that final data set. We will determine over the coming months if when, what venue that 168-week data would be presented..

Okay.

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

Well, Jeremiah I think you're referring to fourth biopsy that isn't necessarily directly linked to the 168-week data but would be approximate to that timeframe.

Look, we want to continue to enhance and improve the quantification and the capture of dystrophin in these muscles and so I outlined earlier, all of the different ways that we're going to take assessments of dystrophin on these fourth muscle biopsies and again we think it just will add to the overall evidence that we provided to-date using various methods of our drugs mechanism of action in production of dystrophin.

So again we continue the dialogue with the FDA as we have been over the last year and a half as it relates to dystrophin methodologies quantification, it's an evolving field, of course, we want to stay on the leading edge of that but also make sure we rely on validated measures and proven ways of capturing this.

Again none of these methods are perfect right, these are imperfect measurements but we believe as a collective and in totality and if the level of dystrophin are significant that, that's the most important thing that our drug is producing dystrophin in the muscle and that forms the basis or the foundation of the support of the clinical outcomes that we're seeing..

And Jeremiah I think one of the -- at least in my experience, it's not unusual for the FDA to have a lot of questions about a new circuit endpoint or a biomarker.

And so there is a great deal of effort, and it should allow them to understand and remember we're working with some of the most experienced investigators in the world who have been doing this type of analysis for decades. So for the FDA to try to understand it very quickly -- they have a lot of questions.

And so this is not unexpected in a lot of things that they are asking are fairly customary like asking more than one reader to do the analysis. So I think what we're trying to do is educate everyone so that the FDA can understand what's done and what is really the state of the art.

And I think we're making some progress and I think having the collaborators that we're working with goes a long way in allowing that to happen..

Okay and just one last question, in terms of the MRI results that the FDA was asking for in the last communication, you previously mentioned that 11 or 12 boys in Phase IIb study had received an MRI at some point previously.

How longer were these exams completed? And can you also comment if you know this like in terms of like how much the muscle biology might change in that period?.

Yes. So Jeremiah, look the FDA requested this MRI data that we tried to capture it for the first time in this recent communication, we will attempt to do that. Again this was not a study that was conducted by Sarepta nor data that we own internally.

And I think it is a promising exploratory measure, I think we don't know enough about what to expect or what we would see related to a treatment effect. I think it's an incredibly potential strong natural history dataset as it relates to MRI, but this is going on for a couple of years now.

Ed, do you want to elaborate?.

Yes. So, this is a large multi-centered trial and Chris (indiscernible) from University of Florida and (indiscernible) have been the major investigators on this. And so, what they were -- the attempt was is to understand what happens in muscle on MRI over time.

So we participate, we allow the participation of some of our boys in this and it's really ongoing, they are collecting data. So they get MRIs every year in a yearly update. And so, all of this data has just really become available and we're looking at it.

No one has ever looked at change in MRI to any treatment and so, this is obviously very exploratory. We think it could be very interesting, but we're in the process we've asked for more data to be able to compare and that's ongoing and we should have the data to be able to submit to the NDA..

I mean one thing that we thought was interesting. Ed and the team here was able to go down and look at some of the eteplirsen boys images. And one finding that was pretty market was that the two non-ambulant boys in our study look significantly different in terms of their muscle mass relative to the other boys who are still walking.

So it's supported and seem to validate why these two boys had lost ambulation and why they may have been too far gone to have had an impact on their walk test. .

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

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

Yes. So I think as we mentioned, there is a lot of work that has to be done in preparation. So we're now really seeing the fruition -- it's fruition of all of the work. So we have, as I mentioned, we have four sites initiated and another four to be starting soon and patients will be starting dosing in this month.

So I think, based on our projections obviously it's always challenging, but I think what we're starting to see is, there has been a lot of interest from the community and from patients and patients are being lined up. So as soon as we have those centers online, obviously then we can make a better projection is how long it will take to enroll..

Yes. And again with the eteplirsen open label study, we do expect to enroll as many as we can as quickly as possible. And it will afford us to look at analysis potentially if we have 40, then we have a longer experience. We could do those types of analysis versus the untreated cohort.

So I think our goal of 60 in the primary analysis up to 80 if we include those that would be outside of the primary analysis, we will provide appropriate updates as we understand when we could achieve those specific targets..

Okay. As I am looking on clinicaltrials.gov, it suggests that the primary evaluation would be completed by May 2016, which would essentially imply that you could complete enrollment by May 2015 to allow yourself some time to analyze data.

Is that a fairtime frame for us to think about for enrollment in the study?.

I wouldn't base our expectations around that. I would say that would be an optimistic target for enrollment. But again, it could be that we're comfortable with a number of patients that we can optimally get in that timeframe to do a final analysis that meets that timeframe.

But again, we're not providing guidance on that at this point and would not try to interpret what that guidance might be relative to that date on the clintrials.gov website..

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

Yes. And again, in these patients who don't qualify for 301 or are non-ambulatory, the name, clinical outcome measure is really looking at pulmonary function. We're looking at a lot of exploratory endpoints in regards to upper extremity.

So I think given the small number of patients, this is really -- we would really want to make sure that in this population, it's safe and obviously, we will be looking at, let's say, the clinical outcome measures as we go along. So I think right now, we don't have any specific expectations..

Yes. The older, non-ambulant is not intended for an efficacy endpoint. Again, it's a smaller sample size. It's an open-label study; it's a more heterogeneous population.

As we already mentioned that they're going to have some who might be walking but more advanced in their progression, some that are non-ambulant, some that are further along with more compromised pulmonary function. So we just wanted to ensure that there was safety.

The other thing I would mention is that we have some of the best clinical data in the non-ambulatory population as it relates to a disease-modifying therapy or dystrophin-producing technologies and that the two boys in our study have now been followed on drug assessing clinical outcomes for over 2.5 years now in that non-ambulant state.

So again, we believe we're already getting some informative clinical outcomes on those two non-ambulant boys in our current study..

Our next question comes from Ritu Baral from Cowen. Please go ahead..

To restart with the manufacturing status, you mentioned that you have supply to start the trial; do you feel that you're in a good position to supply pretty much the entire trial right now and are you on track with previous expectations that you would be able to supply a significant portion of the community by end of the year, next year?.

Yes. So, Ritu, look, we're very comfortable with our manufacturing capability and the reproducibility we have seen to-date that gives us a high level of confidence in our ability to continue to provide drug supply for our trials and to prepare ultimately for commercial approval.

With the recent guidance and the delay in our NDA submission, we're going to be looking hard at our gating of when we would need what drug supply.

So again, we're also trying to make sure that we hold back because it's one of the most expensive cost drivers we have as a company and to make sure that we’re always focused on what drug we need, give ourselves some margin of error, but not to overproduce a drug because it is an expensive endeavor for us. So I think right now, we’re on track.

We don't have any concerns about providing drug supply for all of these studies on an ongoing basis and at the time that we have a better sense of the commercial launch of eteplirsen, we believe we have sufficient time to prepare for that full commercial demand.

Simply put, right now while there is no guarantees, we do not believe manufacturing capacity or capability is a gating item to our success with our Duchenne program..

And what is the shelf life of the current clinical drug product right now? How long could you keep it around?.

Look, we’ve different levels of stability on our drug substance. Usually, you have a shorter amount of time on your drug product that is in vials. We have obviously longer-term stability on the small-scale batches we've had. We're accumulating more and more stability on our mid-scale batches.

We're not providing any specific on all of those batches and what level of time, but what I would say is that we've described the stability that we have and that we would have at the time of an NDA for the FDA and there were no issues that were raised that would prohibit us from moving forward within NDA, having said that with this delay we want to ensure that any updated stability data we have would be included at the time and also the demand for this drug in the market at this point would not preclude us from meeting a shelf life of many years, but by the time we needed that we believe we will have sufficient stability.

We also can draw on our other products that we have been producing under GMP conditions for clinical trials, you know, West Nile virus was one of the first trials we did as a company, more than a decade ago. And we recently did some HPLC testing on that within the last couple of years and it had very good stability.

So there is nothing that suggests that we're going to have a stability problem with this drug product moving forward, but we continue to accumulate that data as we go..

And a last question, given your comments on the path forward in the EU in the patent situation there, what do you think the path forward would be in at one point -- what point is the intellectual property situation even considered by the regulatory authorities there?.

Well, the first the thing we've been saying all along is that -- the first thing we need to do as it relates to Europe is determine what is needed to get the drug approved.

And I've said previously that if we were handed a free and full license for eteplirsen in Europe tomorrow it would not change our current activities because we still need to find out if the existing data set is sufficient for approval, what additional data would be needed, how would they perceive a conditional approval of eteplirsen, if we were to get that guidance even under the most aggressive timeframe where we might have an MAA for conditional approval submitted next year.

We're still looking at a long approval timeline and ultimately reimbursement approvals throughout Europe to get eteplirsen utilized and there is a lot of time to determine the appropriate pathway and freedom to operate for eteplirsen in Europe.

So again the regulators, their job is to look at the data set efficacy and safety and determine the approvability based on that, it's the European patent courts of which we have filed appeals and that's in the queue that would be determining if the opposition on 51 is overturned..

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

So I guess one of questions I’ve is speaking with some of the parents prior to the most recent set of FDA communications or sort of this assertion that they would agree to fourth part biopsy under certain conditions from the FDA, notably that the FDA would sort of at least intend on acting on the results.

And I'm just wondering have you talked to the parents subsequent to the most recent round of communications with the FDA and do you think there is any risk that you won't be able to get a family as to agree to the fourth biopsy now?.

Brian, we're not trying to go direct to the families in our studies, we’re trying to state publicly, why it's important? We're trying to convey to the site while we believe it's important and to make sure that when Jerry Mendell and the staff at Nationwide understand our reasons for wanting to pursue the fourth biopsy.

What we've stated previously is that while absolutely the FDA is important as a customer and to ensure that we do this fourth biopsy with as best we can, the methodologies and the quantification that would be satisfactory to the FDA, they are not our only customer who would be interested in this.

Remember, if we were to submit the fourth biopsy data into an NDA, even if the FDA presented concerns or questions about the fourth biopsy data to an advisory committee panel, we would make our best argument to an advisory committee panel and we believe that could potentially be a compelling data set to get an independent advisory panel comfortable with the drugs activity and the supportive data of the clinical outcomes that we're seeing.

On top of that, we would hope to see that as part of a package insert or something that we could discuss with payers, okay.

In addition to that, we think that's something that's important that clinicians who are going to be thinking about using this product would find important that three years after a drug initiation we're still finding evidence of dystrophin in the muscle biopsy. So absolutely, the FDA assessment of the fourth biopsy is important.

They are not the only customer we're thinking about in capturing these fourth biopsies..

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

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

I mean when we provide these communications, we first and foremost hear from those who have been following Sarepta and investors who are interested in our communications and clarity around our communications.

And so in the two days following our communications that was a priority, the analysts that you -- all of you on this call also want clarity, so you can provide your reports. There is media interest in clarifying this to the biotech industry.

We obviously have staff that try to reach out to clarify for the advocacy organizations and the patients who inquire about what this means, the timelines, etcetera.

But again, our first priority is to make sure that we are clear in the guidance we're receiving from the FDA, clear in our intention for how we're going to pursue the approval of eteplirsen and then to the extent that the community needs clarity on our communications, we provide that, okay.

All along, of course, we've stated, I think the FDA would agree with this, is that nobody wants to hold up an effective therapy from patients, okay.

And I mentioned on the regulatory update call, it really doesn't matter if there are parents that are convinced that this drug would be useful and safe for their boys, it doesn't matter that physicians would like this drug to use in their patients, what matters is the FDA is comfortable with the dataset and that they believe it meets their requirements and guidelines to approve this drug and that is our first priority and of course, we’ve the patient in mind when we're trying to move as quickly as we can.

We’re disappointed too that there are any delays in our clinical trials because of all of the things that Ed described are essential to having a good quality program. But again, we provide our communications and the patients and the community is going to respond as they may and there may be different responses from the patient community.

The other important thing to keep in mind is the FDA themselves have opened their doors and communicate directly to the parents. So it's not as though the community is getting all of their information from Sarepta. The FDA is providing direct communication to advocacy organizations and parents directly.

So that's the best way I know how to answer the question you're posing..

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

Can I take that message as something that you're comfortable with?.

Yes, well, you know we've been saying for many months now that we would be prepared to satisfy the full commercial demand in the market, assuming the previous timeline of mid-2015.

Now however, with that timeline moving out, right, we also want to make sure that we're going to be producing the drug at the appropriate time that we can provide it to the broader community upon a commercial approval and so, again with all of the caveats and risk factors outlined in our SEC filings and associate with any company, trying to manufacture large quantities of drugs.

With all of that, there is nothing that we see where we sit today that would be a barrier to providing that grade [ph]. But ultimately until you produce those levels of drug, you don't know, but we're confident based on the reproducibility of the runs we've had to-date, the consistency of the quality of the drug product.

Again experienced, the more experience you get with doing these runs, the more confident you are that it's working and that we understand if there is any tweaks along the way that need to be done that we're aware of that. But to-date, we've had very good results and we're confident in our manufacturing moving forward..

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

On first, maybe we could just touch upon the biopsy data from the potential fourth biopsy. I was just wondering under what kind of different analysis are you thinking to look at these.

Specifically are you looking to have a control group, either healthy normals for untreated patients? Has the FDA provided any guidance with respect to that?.

Look, we’re going to be working with the FDA first and foremost to share. We've shared and outlined and we’re trying to work on the specific protocol. But we have communicated all along that we intend to not just look for one specific measure or one specific methodology of dystrophin quantification, but multiple measures.

And what we hope is that, the multiple measures that we can show that dystrophin is present in quantities that are larger than what would be expected from Duchenne, controls or banked samples or again the data that we’ve generated from this specific population at baseline in previous biopsies.

Again, we're trying to build the totality of the data to show dystrophin. But again, there has been a lot of attention appropriately on dystrophin.

But I want to remind everybody that we have a very, what we believe compelling clinical dataset across this population that we presented, Jerry Mendell presented at World Muscle across 6-minute walk test pulmonary function and again over this, 3-year timeframe that we've reported to-date, we think they're behaving very different than what natural history would suggest.

The FDA is urging, we're trying to get more detailed matched natural history data and we're pursuing that, but based on all the literature, I think there is a compelling case to be made clinically and that's not even factoring in what the families are saying or happening with these boys who are in the eteplirsen trial and that becomes a very compelling component as well.

As we start new trials, we’re going to get new safety data, we think with across all of our eteplirsen studies, we will have more than a 100 eteplirsen patients targeted for drugs.

So we’re going to get all of these questions answered over time and for the fourth biopsy we just want to try to get as much additional dystrophin by a variety of measures as possible..

Okay.

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

No. This will be our first guidance meeting with the EMA; we've been saying that all along Chris. So again, they've given us some tentative dates. We're trying to confirm those dates that would happen before the end of the year and all of these issues will be discussed.

And again, we believe that the broader clinical program that we have for eteplirsen should be sufficient for the EMA. Recall, they have supported or exon-53 study, which is an open label experience study looking at biopsies, looking at clinical outcomes after the short titration placebo controlled phase.

We know looking at our competitor exon-skipping drug that their second drug in I believe is an exon-44 drug, the EMA supported an open label study there. So there is a lot of evidence out there that supports that the EMA may be even more flexible than what the FDA has been..

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

Yes, Chris, we're not going to provide that guidance at this time. A lot of this is obviously -- we've been working with the various sites. The sites have been working on identifying patients, we obviously don't know how many of them will qualify, how many may drop out by inclusion-exclusion criteria.

So there is a lot of factors that control for that and once we’ve a better idea after we get more sites up and running after we look at what -- how many patients are making it through the inclusion-exclusion criteria, that's when we can provide better clarity on that.

Ed, do you want to add anything?.

Yes, I think, Chris, you know what we would like to do is we -- you make kind of an arbitrary projection of how many patients you can enroll and then we have to match that with -- depending how many sites are up and running unless they are -- the average number of patients that are enrolling per site.

So usually we don't like to make projections this early in the course and so we have some data to extrapolate. So we should have that later..

Okay.

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

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

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

Yes, Chris again we will provide guidance on any further updates and data from our current study, from our ongoing studies are up and running again. So stay tuned for -- look we haven't even dosed our first patient yet and so we know that there is some time before we can see a clinical outcome effect that would be different than natural history.

And we want a large enough sample whether we wait for full enrollment, our target enrollment or whether we look at our interim analysis, we’ve not determined that yet. So, again as we get more information we'll provide the appropriate guidance..

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

Tis is Evan on for Robyn, thanks for taking my question.

And can you just remind us why you opted not to do a rolling review despite the FDA seemingly suggesting that this type of review could be appropriate in the recent statement from last week?.

Yes, so I think thanks for the question because I think this needs some clarity, we understand what a rolling NDA is and any discussions we've had with the FDA about a rolling NDA has been clear and again we're not taking a rolling NDA off the table.

What we've been talking about previously from our previous FDA discussions was not a rolling NDA, but the idea of additional data submitted after an NDA is submitted or filed and when we talk about an NDA, we mean the full and final NDA not a section of the NDA.

And I think everybody understands that a rolling NDA in the first section that submitted does not change the clock, okay or the PDUFA timeline, okay. So the clock starts and the assessment of whether an NDA is fileable or not would start when all of those NDA modules are in.

With all of the additional data that we will be compiling and the additional stability data described for our CMC section, we’ve at least four sections, okay, that would be waiting for additional data for submission.

There may be one section that we could submit prior to but we want to look at it in its totality before we make that decision about a rolling NDA. So I just wanted to clear that we understand what a rolling NDA is, we understand the optics of looking like we’re getting one of the sections in.

But the main argument that is often used with a rolling NDA is, hey, you could give the FDA a head start, they can start looking at data before you have your full NDA in.

Now, there is some debate whether they have the resource to actually put against a section of an NDA before they even have all of the sections in, but I go further to say we've provided as I've outlined a significant amount of our existing dataset, a large portion of what would be in an NDA and the FDA has continued to ask for additional data.

So I mean I would go as far as to say, this is probably one of most extensive datasets proportionally that they've received on a program prior to an NDA.

So I think in terms of the last year and half of reviewing white papers we've provided, raw data, we provided the raw images of the dystrophin earlier this year, before the April guidance, the idea that the FDA doesn't have any of our data in-house to start reviewing is a misnomer.

So again, we're not taking a rolling NDA off the table, but our main focus is completing the essential elements of an NDA to start the clock because the community and the investors I would say are more interested in when are you going to get a filing accepted? When is an adcom going to happen? And when can the drug be approved? And that is not driven by the first submission as part of an NDA..

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

Yes, look, we had our prepared remarks, I gave an extensive overview of all of our communications, we stand by our public communications and our press releases and our disclosures related to all of the FDA correspondence.

And again, we would really like to move forward regardless of what might be perceived as disconnects or inconsistencies, go back and look at all of our communications, we've been consistent, clear and stand by our public communications and we're not going to engage in further trying to tease out how people perceive the differences of what the FDA has said versus our communications..

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

This is Kumar in for Yaron, thank you for taking my question. So in U.S., Sarepta and Prosensa have a patent dispute relating to freedom to operate exon-51 and also there is a question about the first company getting approved might block the other under orphan drug exclusivity.

Can you comment on both of this, especially given that Prosensa is going to file for approval before Sarepta does?.

Yes. So look, we've already talked about the patent opposition in Europe and where that stands and we filed our appeals related to the European Patent Opposition and Appeal. In the U.S., you're probably referring to the interference.

Again, we've had all those disclosures out there and so that interference process we've described in previous calls and how that plays out. Our current patents remain valid and it does not impact our efforts to pursue an NDA filing and a drug approval. And ultimately, in terms of orphan drug exclusivity, we believe that these drugs are not the same.

They have a different chemistry, they have a different sequence, they have a different dataset and so we think by all of the different measures, a different chemical entity that the drug would not -- any first approval would not preclude another drug from getting to market..

Great. Thank you..

Okay. Well, thanks, everybody, for your interest in Sarepta. Again, we've tried to be very clear with respect to our recent communications with the FDA and our path forward. We're focused on that.

As Ed described, we're really focused on getting more boys on eteplirsen in our clinical studies and also focusing on the follow-on exons and we've got a lot going on here and I appreciate your interest in Sarepta and stay tuned till the next call. Thank you..

Thank you ladies and gentleman. This concludes today's conference. Thank you for participating. You may now disconnect..