Sarepta Therapeutics, Inc. (NASDAQ:SRPT)
Regulatory Update Conference Call
April 21, 2014 08:00 AM ET
Erin Cox - Manager, IR
Chris Garabedian - President and CEO
Ed Kaye - CMO
Ritu Baral - Canaccord Genuity
Brian Skorney - Robert W. Baird & Co.
Mohit Bansal - Deutsche Bank
Liisa Bayko - JMP Securities
Ted Tenthoff - Piper Jaffray
Brian Klein - Stifel Nicolaus
Chris Marai - Wedbush Securities
Tim Lugo - William Blair
Bill Tanner - FBR Capital
Joseph Schwartz - Leerink Partners
Yaron Werber - Citi
Kim Lee - Janney Capital Markets
Steve Brozak - WBB Securities
Welcome to the Sarepta Therapeutics Incorporated to give regulatory update Conference Call. My name is Paulette and I will be your operator for today’s call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session. Please note that this conference is being recorded.
I will now turn the call over to Erin Cox. Erin, you may begin.
Thank you, Paulette, and thank you all for joining today’s call. Earlier today, we released a press release announcing a regulatory update for our product candidate eteplirsen, and this press release is available on our web site at www.sarepta.com. Joining me on the call today are Chris Garabedian, our Chief Executive Officer, and Ed Kaye, our Chief Medical Officer.
I would like to note that during this call, we will make a number of statements that are forward-looking including but not limited to the statements about the timing of a new drug application submission for eteplirsen in the treatment of Duchenne muscular dystrophy, a substantial filing and acceptance of an NDA for eteplirsen by the FDA, the timing entitled plan submission of additional data analysis and other information to the FDA necessary for the FDA's new regulatory determinations, the preparations for timing and design, and our ability to initiate additional studies for eteplirsen and other follow-on exon. The development timing of IND filings for exon 45 and exon 53 candidates, our ability to produce drug supply for the planned additional study, and potential commercialization and the potential in timing of accelerated approval for eteplirsen.
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 SEC.
With that, let me turn the call over to Chris Garabedian, Sarepta's Chief Executive Officer. Chris?
Thank you, Erin. I’m very pleased to provide an update on our Duchenne Muscular Dystrophy program in the path forward for eteplirsen, our drug for DMD patients with genetic mutations amenable to exon 51 skipping, as well as an update on the development of our follow-on DMD drugs to address other genetic sub-groups in DMD.
Since our last FDA guidance update more than five months ago in November of last year, we have had four meetings with the FDA and provided additional information and data related to our eteplirsen program. As a combination of all of these interactions, we have now received their guidance letter that serves as the final meeting minutes for all of the interactions that have taken place since early November.
I’m very happy to say that we now have clear direction from the FDA to help move our DMD program forward. Based on this guidance, we plan to treat more patients with eteplirsen and a broader population of patients in clinical trials beginning this year. And with additional data and analysis, pursue an NDA filing that we will plan to submit by the end of this year for a potential early approval of eteplirsen sometime in 2015. Overall, the FDA’s guidance has several major implications for Sarepta.
First, we now understand how the FDA suggests that we enhance our existing dataset over the short-term to increase their confidence in our data and increase the likelihood of an acceptable NDA filing and a potentially favorable review for an accelerated approval of eteplirsen. While we continue to believe that our current dataset is strong enough on its own to be considered for an NDA filing, we believe the additional data that we will collect over the next six to eight months, if positive, will provide the FDA with supporting evidence of eteplirsen safety and efficacy that should result in an acceptable NDA for filing.
To this end, we will work diligently to compile these additional data to allow us to submit an NDA by the end of this year. Based on this timeline and continued positive data, we believe the FDA could make a determination regarding an accelerated approval if they deem our NDA submission acceptable for filing as early as the second half of 2015.
Second, we reached an agreement with the FDA on an open label confirmatory clinical study design, and we plan to take steps to initiate this study as soon as possible. In addition, we reached an agreement on additional studies of eteplirsen in a broader patient population including DMD boys younger than seven years of age and older boys who are more advanced in their disease progression or those that are non-ambulant that is no longer able to walk.
Third, we have initial guidance from the FDA on the clinical development of our follow-on exon skipping drug candidates which will enable us to accelerate these programs to address more patients with DMD who have genetic mutations that are not amendable to eteplirsen treatment. As we have previously described, eteplirsen only treats about 13% of DMD patients, but our technology has the potential to treat more than five times as many patients, and we have already identified seven additional drug candidates that have the potential to treat almost half of all DMD patients.
So receiving guidance on a potential path for our follow-on exon skipping drug is quite encouraging. The FDA’s guidance was very detailed and included specific comments related to a potential accelerated approval pathway for eteplirsen and additional clinical development requirements to advance our overall DMD program. So, I will provide an overview of this guidance and Ed Kaye, our Chief Medical Officer will outline our clinical development plans as a result of this FDA guidance. With regard to an NDA for eteplirsen, the FDA stated that "with additional data to support the efficacy and safety of eteplirsen for the treatment of DMD, an NDA should be fileable". In a moment, we will talk about the types of additional efficacy and safety data that we will be generating and compiling in the remainder of the year that we believe will strengthen an NDA filing and that will add to our existing dataset to enable a potentially more favorable review for accelerated approval.
First let me outline the two approaches to accelerated approval described in the FDA’s guidance letter . Either approach has the potential for an eteplirsen approval prior to completion of a confirmatory study. The FDA would decide which of these two specific ways and approaches would best apply during the NDA review process if they were to support a decision to file the NDA and approve eteplirsen under the accelerated approval pathway. The first approach would be to grant eteplirsen an accelerated approval on the clinical data from our Study 201, 202 Study based on the six minute walk test results as an intermediate clinical endpoint. The second approach would be to grant eteplirsen an accelerated approval on the dystrophin biomarker as a surrogate endpoint and the agency acknowledged that they have discussed the possibility of using a number of modalities to quantify dystrophin and how one or more of these approaches might be used as a surrogate endpoint or endpoints to support accelerated approval.
Both of these applications of the accelerated approval pathway are outlined in the FDA guidelines for accelerated approval and were clarified and enhanced as part of the 2012 FDASIA legislation and in subsequent interpretations of this legislation. Under both approaches, the guidance letter described the FDA’s reservation that the existing dataset may not be sufficient to support an NDA filing or be compelling enough for a favorable review. However, they provided a number of possible ways that supplemental data could enhance the filing and the prospects for a favorable review under the two approaches to an accelerated approval pathway prior to the completion of a confirmatory study. Now, I would like to outline some of the additional data that we expect to have over the next year that could be incorporated into an NDA filing by year-end or provided shortly thereafter as part of an NDA review.
Let me start with additional data that could support the six minute walk test as an intermediate clinical endpoint to support accelerated approval. As you know, we are continuing to generate data from our eteplirsen Phase IIb extension study what we referred to as Study 202 and we will be accumulating additional data and perspectives that could be included in an NDA later this year. In particular, we will have a 144 week data supporting longer term clinical outcomes in safety in the second half of this year, if positive, could supplement currently available six minute walk data and/or pulmonary function test or other outcomes over a longer duration in a group of boys that will be over 12 years on average across the study. Furthermore, we will have 168-week outcomes data that could also be submitted as part of an NDA review and provided shortly after an NDA filing at the end of this year.
With respect to dystrophin as a surrogate endpoint to support accelerated approval, the FDA indicated that it will collaborate with Sarepta to conduct a detailed review of the current dystrophin biomarker data from our Phase II study. If the results of the review were deemed adequate, it may support accelerated approval. To this end, over the coming months, the FDA will be meeting with a pathologist that supervise the dystrophin quantification to better understand the blinded review, the detailed methodologies, and the controlled conditions in which the various dystrophin measures were conducted.
Secondly, the FDA also suggested that a positive fourth biopsy from the existing Phase II patients could serve to enhance the current dataset by reinforcing dystrophin as a surrogate marker to support accelerated approval. We will be exploring this possibility and if feasible, work with our investigator side to pursue biopsies later this year.
Finally in support of both approaches to a potential accelerated approval, we plan to begin enrolling more patients on eteplirsen beginning in the third quarter of this year. And we will be capturing safety data on these patients which we would include in an NDA by the end of this year. While we believe our current datasets stands on its own and should be enough for an acceptable NDA filing and a positive review, we believe that incorporating any one of these measures that I described, would strengthen the argument for accelerated approval of eteplirsen and by addressing FDA’s questions about the current dataset, we will have the potential to make the FDA more comfortable with an NDA filing and a possible early approval of eteplirsen. But we are hopeful that we will have supported data on a number of these measures or potentially all of them to include with the NDA filing later this year.
Importantly, the FDA has expressed flexibility in accepting supplemental data after an NDA filing if additional data becomes available in the NDA review period. This could include additional safety data from additional patients or longer exposures in patients from our eteplirsen confirmatory study and on-going program which may include safety in patients younger than seven years of age and patients who are older and are more advanced in their disease progression or in a non-ambulatory state. Additionally, we will likely have longer term clinical outcomes in safety from our Phase IIb study after an NDA filing that could be filed prior to an advisory panel and during the review process such as 168-week data as I described earlier in early 2015 shortly after an NDA submission.
This longer term safety and clinical outcomes data if positive could demonstrate the safety of Eteplirsen beyond three years and provide more than three year data on key clinical outcome measures like the six-minute walk test and pulmonary function test like maximum inspiratory pressures and maximum expiratory pressures.
In summary, we are pleased the FDA provided a clear path forward for filing a NDA for eteplirsen under an accelerated approval pathway, and in particular, we appreciate the flexibility that the FDA has outlined in applying two different approaches to a potential accelerated approval pathway to enable an early approval of eteplirsen as well as their suggestions as to a number of ways that Sarepta can add supplemental data to strengthen our existing dataset in the near term in order to address their concerns and increase a likelihood of an acceptable NDA filing and favorable NDA review.
The second major area that was addressed in the FDA guidance letter was clarity regarding an eteplirsen confirmatory study. We are happy to report that we have agreed on the most significant aspects of a path forward for a confirmatory eteplirsen clinical trial. We wanted to ensure that an eteplirsen confirmatory study was feasible and could provide meaningful clinical data on the safety and efficacy of Eteplirsen while at the same time providing a study that was enrollable study and ideally could allow the FDA to consider an early approval pathway for eteplirsen and not compromised our ability to complete the study. We have agreed on a confirmatory trial design and clinical program that would be possible even in the event of an accelerated approval of eteplirsen and would be well underway at the time of a potential commercial approval of eteplirsen even if that occurred as early as mid-2015.
We also came to an agreement with the FDA to pursue studies in a broader population of DMD patients to collect additional safety and biomarker data and we intend to conduct additional clinical trials that will enroll eteplirsen amenable patients that are younger patient population, that is those who are less than seven years of age as well as patients who are older and/or more advanced than their disease who are non-ambulant. This eteplirsen clinical development guideline was outlined as one of two ways to provide confirmatory evidence of eteplirsen’s efficacy in the event of an accelerated approval of eteplirsen in order to provide an eventual full or standard approval. Specifically, the agency stated that they “envision two approaches for confirmatory trials” which include an open label eteplirsen study in which they described that “a historically-controlled trial might be acceptable to confirm clinical benefit following accelerated approval.”
They also feel that this study would add substantially to the safety database, highlighting that the current extent of patient exposure to eteplirsen is insufficient to support a full or standard approval of eteplirsen. The FDA also offered a second approach to confirm evidence of eteplirsen’s efficacy in the event of an accelerated approval and recommended that “a randomized placebo control trial of another PMO with a similar mechanism of action directed at a different exon” such a study could include could include our follow-up exon’s skipping drugs that are in the latest stage development. SRP-4053 and/or SRP-4045 drugs that treat patients amenable to genotypes who are amenable to skipping of exons 53 and 45 respectively and the FDA would view the results of such study confirmatory if there was a “demonstration of a correlation between dystrophin production and a definitive clinical benefit on six-minute walk or another measure.”
The FDA went further to state that they “urged Sarepta to initiate both of these trials as soon as possible.” And added that these “confirmatory studies should be underway at the time of approval.” We will be moving as rapidly as possible to initiate these trials and have both of the confirmatory studies and additional eteplirsen experience studies underway by the end of the year and per the FDA suggestion, would be well underway at the time of eteplirsen’s approval even if they want to receive an accelerated approval by mid-2015.
The approach that the FDA has taken in offering us two ways to confirm the results of eteplirsen’s Phase IIb results in the events of an accelerated approval serves the purpose of not only providing more than one way to obtain a full and standard approval of eteplirsen, but may serve to accelerate the approval of follow-on exon by urging us to quickly move into a randomized placebo controlled study that will be designed to show a clinical benefit and may serve to establish the correlation of dystrophin with clinical outcomes to be used as a surrogate marker for efficacy across the platform for use in the rare exon skipping targets. This will be important as we think about how we can accelerate the development and approval of follow on DMD drug candidates to treat all of the boys who might benefit from our platform technology and increase the likelihood of what we refer to as a class approval.
To this end I’d like to turn the call over to Ed Kaye, our Chief Medical Officer, who will outline our plans for the clinical development of Eteplirsen and our initial plans for a follow on DMD drug study for patients with genotypes amenable to other exon targets that we have in development. Ed?
Thank you, Chris. And first I want to reiterate that we are very pleased that we have received specific guidance on the clinical development path forward for eteplirsen and our follow on exon skipping drugs for DMD. While we have yet not finalized all of the specific details for each of the studies that we plan to initiate between now and the end of the year, I will be sharing some of the important elements of these studies such as the population that will be studied, the end point and the timing of the potential study start.
First let’s start with our eteplirsen confirmatory clinical study, over the last few months, we at Sarepta have been preparing for a variety of scenarios on this clinical study as we wanted to be ready to move quickly upon receiving FDA’s guidance on the path forward. As a result, we are ready to move forward with our confirmatory study with eteplirsen. This study will enroll a patient population with Duchenne muscular dystrophy, who have genotypes that are amenable to an exon 51 skipping drug and are still ambulant and able to walk at least 300 meters on the six minute walk test.
We expect to enroll 60 to 80 patients between the ages of seven and 16 years and the study will be powered on a predefined clinical outcome measure with these boys who can walk between 300 and 450 meters but we will allow boys who can walk over 450 meters to be enrolled and receive treatment even though they will be included in the primary analysis. We will also be collecting pre and post treatment biopsies to test discipline in these patients to better understand the correlation of dystrophin and clinical benefit. We will allow patients who are currently or have previously been in drisapersen studies including drisapersen exposed patients although we will likely require a washout period of at least 24 weeks.
We will also have an arm of DMD patients with similar inclusion exclusion criteria who are not amenable to exon 51 skipping and could not benefit from eteplirsen but who will serve as an untreated control arm. While the FDA indicated that this arm is not necessary to meet the requirements for the confirmatory study and may present some challenges with interpretation of the eteplirsen treatment effect, they did not object to our including this arm if we found it advantageous to our overall DMD program. We believe that the majority of DMD patients with exon deletions decline at a similar rate when adjusting for age and base line function or walking ability and therefore believe we will gain valuable information to see how these untreated boys progress versus those similarly massed eteplirsen treated boys.
We are working out the details of these specific genotypes that will be go out as part of this untreated cohort and hope to have details on this in the coming weeks. Since we are close to finalizing this protocol and have begun to identify potential sites that could enroll patients in this study, we expect to submit a protocol to the FDA at selected IRBs in the coming weeks. We have already begun the process of submitting stability data and comparability data to obtain drug release for use in this study and we will be planning to kick off the study initiation by the end of this quarter with patient dosing to begin in the third quarter.
As Chris mentioned, the FDA encouraged us to obtain safety data with eteplirsen in a broader population of DMD patients including younger patients and patients with more advanced disease. With this guidance from the FDA, we have begun to work on preparing to conduct these two additional studies and I’d like to describe these clinical trials in more detail. We intend to proceed with these two studies in parallel so we can start enrolling patients later this year. The first study that will broaden the population of DMD boys exposed to eteplirsen will be in younger ambulatory DMD patients who are amenable to eteplirsen and between the ages of four and six years. We expect to enroll at least 20 patients in this study. The primary purpose of this study is to obtain safety information on eteplirsen in this younger age group.
In addition, we will be obtaining pre and post treatment biopsies in these patients as this will help us demonstrate that eteplirsen is able to restore dystrophin on a consistent basis regardless of the age or stage of disease. This will also add important data in establishing dystrophin as an acceptable biomarker for the broader use of exon-skipping and eteplirsen across a spectrum of DMD boys.
Like our confirmatory eteplirsen study, we will allow patients who are currently or have previously been into eteplirsen studies, including eteplirsen exposed patients, although we would require these patients to have a no exposure to the drug in the last 24 weeks.
While our confirmatory eteplirsen study will still require a stable dose of steroids prior to enrolling, this study will allow either boys or on a stable dose of steroids for at least 24 weeks, or those who have not been treated with steroids or are steroid naïve.
The second study to evaluate eteplirsen in a broader population will enroll patients who are more advanced in their disease progression and who are generally older in age. This study will evaluate the DMD patients who are amenable to eteplirsen and cannot walk a minimum of 300 meters on a six minute walk test or are non-ambulant. We expect to enroll at least 20 boys in this study. The primary purpose of the study is to obtain safety information on eteplirsen disorder in more advance population and we will determining the additional qualification for inclusion in the study, so we can get the most useful exploratory information on eteplirsen affect in this advanced stage of the disease.
Like the confirmatory eteplirsen study and the study in younger patients, we will be obtaining pre and post treatment biopsies in these patients and this will help us demonstrate that eteplirsen is able to restore dystrophin in the more advanced stages of the disease. This will also add important data in establishing dystrophin as an acceptable biomarker to the broader use of exon-skipping in eteplirsen across the spectrum of DMD patients.
Since the older and more advanced patient population represents such a wide spectrum of the disease, we will be determining the appropriate inclusion and exclusion criteria in over the coming weeks. As you can see we will be very busy advancing the development of the eteplirsen over the rest of this year and we expect to be dosing patients in our confirmatory ambulatory study with pre-defined endpoints by the third quarter and expect to be dosing the first patients in our younger and older more advanced patients later this year.
In addition to gaining clear guidance on our eteplirsen clinical program, the FDA also urged us to move quickly into our follow-on exon-skipping drug as soon as possible and proposed a randomized placebo control study to demonstrate a correlation between discipline production and a definitive clinical benefit on six minute walk or another measure.
While the FDA indicated that this study could provide confirmatory evidence of eteplirsen’s clinical benefit under an accelerated approval scenario, we will also see that the study can serve and to enable faster approval strategy for follow-on exons if we were to show comparable safety discipline in clinical outcomes consistent with our eteplirsen studies.
These two products are closest to -- the two products that are closest to IND submissions are SRP 4045 and 4053 both of which have completed pre-clinical toxicology packages and we expect INDs to be submitted on both of these products by the third quarter. We would expect to initially enroll patients with genotypes amenable to exon-skipping of exon 45 and/or exon 53. The
pharmacokinetic and safety profiles of both of these follow-on exon-skipping drugs, are remarkably similar to eteplirsen and while there are no assurances, we expect them to behave similarly in patients at the same dose as it relates to safety, efficacy and discipline production as eteplirsen.
As per the FDAs guidance letter to initiate enrollment on the studies as soon as possible, we expect to move forward with our plans for IND submissions, begin plans to produce drug supply for these next two follow-on DMD drugs and will begin preparing a protocol that can be ready for patient screening and initial enrolment by the end of the year. We will be working on the design of the study over the coming weeks and we’ll communicate more details about this protocol once we have determined the appropriate inclusion criteria and end points.
Additionally while we will be enrolling an untreated cohort of DMD patients as part of our confirmatory ambulatory study, we will also be initiating a national history study that can be used to identify patients that can serve as prospectively mass controls to our other eteplirsen studies in younger and more advanced patients and this national history study can also serve to identify patients who might qualify for other DMD treatment studies as we advance our pipeline in to our follow-on exon-skipping drugs and those drug in our pipeline for less prevalent genotypes.
As a reminder, we currently have seven additional exon-skipping drugs besides eteplirsen in various stages of research in pre-clinical development and these include drugs for genotypes amenable to exon-skipping as exon 45, 53, 50, 44, 55, 52 and 8, with eteplirsen these eight drugs comprise about half of the DMD patients.
Lastly, it’s important to understand that there is a lot of work that goes on into a clinical study before a patient can be dosed. This includes finalization of a protocol and the appropriate review and feedback from the FDA in various IRBs, the identification and training of clinical trial sites and investigators, the contracting of a clinical research organization to assist, and monitor the research quality of the study, the preparation of the drug supply and the requisite approvals on drug release before labeling and shipping to the various sites for dosing, and screening and baseline the evaluation of patients which may include scheduling of pre-treatment surgical biopsy.
However, please note that we understand the urgency to begin these trials as soon as possible and we will work diligently to get these up and running while ensuring that the studies meet the rigid regulatory standards with the conduct of the trials and the collection of important study data.
With that, I will like to turn the call back over to Chris for an update on manufacturing.
Thank you, Ed. I just want to provide a brief update on our progress with manufacturing and our efforts to ensure drug supply for all of the clinical studies that Ed and I have described and the preparation for commercial supply if we were to gain an FDA approval of eteplirsen in the U.S. in 2015. As we have previously reported, we have been preparing the completion of our stability and comparability data from our midscale batches and I’m happy to report that we have now submitted these data to the FDA and believe that they meet the requirements for drug release.
Assuming the FDA agrees that the drug is acceptable for release, we have prepared sufficient supply of eteplirsen to meet all of the clinical trial needs I described over the next year. Additionally, we will continue with our plans to prepare for large-scale production of eteplirsen in order to meet the commercial supply needs in the event that an NDA filing is accepted and if it were to receive a favorable review and approval of eteplirsen in the U.S. in 2015.
While we still believe our existing dataset with eteplirsen is strong enough to support an NDA filing and is worthy of a review for a potential accelerated approval based on the combination of the safety profile, the consistent evidence of dystrophin production across all patients in the study and the strong clinical outcomes and general stability of over more than two years on the six minute walk test and pulmonary function measures, we also understand that the FDA wants a greater degree of assurance and some additional data to help address their concerns and questions and to bolster their confidence in the strength of the data. Along with a desire to get more patients and a broader set of patients exposed to eteplirsen to ensure that it is safe for approval in all patients who might benefit from the drug.
And finally, to have the assurance, that we begin the confirmatory studies for both eteplirsen and our follow on exon skipping drugs for other DMD genotypes. We believe that the balance of all of these factors lead us to the decision that an acceptable NDA filing and a potentially successful NDA review will be much stronger if we submit an application at the end of this year after we receive some of the additional data that I’ve outlined.
Before we open the call for questions, I want to acknowledge the strong voice of the patient advocacy community and their efforts in recent months. The DMD community expressed their frustration to us and to the FDA in wanting to see progress and moving the drug development process along more rapidly. And they voiced their frustration at the lack in clarity about the possibility of an accelerated approval pathway for eteplirsen.
We understand that this process has required all of you in the DMD community to have tremendous patience and resolve to wait for progress and we also understand that it took tremendous courage to stand up and have your voice heard in demanding action. You have helped us better understand the urgency you live with every day, in doing everything in your power to try to intervene and halt this relentless, progressive and irreversible disease. And in doing so you’ve enabled or maybe demanded that the FDA and Sarepta arrive at a possible path forward that gives us the chance to get eteplirsen to all of the DMD patients that can benefit.
We have read all of your emails to us, watched all of the news clips, followed the broad support across the country and around the world, and among the DMD patient advocacy community in supporting a White House petition ensuring that the FDASIA legislation was applied as it was intended. And we learned how individual parents along with research and clinical experts in the field of Duchenne muscular dystrophy, independently sort out and held meetings with the FDA in the recent months.
We believe that these efforts have helped to clarify many critical questions about this disease and helped put our own eteplirsen data in the appropriate context but most of all by demanding that the patient voice deserved to see at the table in explaining the tremendous unmet need in a disease like Duchenne and that there are situations like this that warrant flexibility in the clinical development and drug approval process.
With that, operator, let’s please open the call for questions.
Thank you. (Operator Instructions) And our first question comes from Ritu Baral from Canaccord Genuity. Please go ahead.
Ritu Baral - Canaccord Genuity
Good morning guys. Thanks for taking the question and all the clarity on what’s been happening the last few months. Chris and Ed, did you guys say, did I miss it, what the treatment period of the new open label study will be?
Yes, we issued a -- they basically did not provide a hard timeframe of follow-up, and so while we believe that we can show stability like we showed in the Phase IIb as early as 48 weeks, I think the FDA is likely acknowledging that if we need to follow these open-label patients beyond 48 weeks that that would be acceptable, understand that this design was with an understanding and expectation that the drug might be approved under the accelerated approval pathway and therefore gives us a little more latitude in how long we can follow these patients to generate additional data.
Ritu Baral - Canaccord Genuity
I see. As far as what you are thinking now is the biopsy point baseline and 48 weeks is what you are thinking now?
Actually, we are anticipating that we may have staggered biopsies, so patients would receive pre-treatment but we may be looking at the time course of dystrophin over 24, 48 weeks, but this is not part of the detailed guidance the FDA provided, this would be Sarepta’s choice on what will be the most meaningful and what could create the most compelling understanding and demonstration of the dystrophin production from eteplirsen. So, at this time, we don’t have more details but we will be working those out in the coming weeks.
And our next question comes from Brian Skorney from Robert Baird. Please go ahead.
Brian Skorney - Robert W. Baird & Co.
I guess, couple of quick questions, starting off just on the request on the fourth biopsy, I know the FDA has brought this up before, and can you just refresh our memories as to what the feedback you received from the parents was in the prior request? And what exactly is the FDA looking for here? Are they just looking for a confirmation using the assays that you’ve already used or are they specifically requesting certain assays?
No, this is Ed. What we are looking at is, they would really like just confirmation of the dystrophin. It gives an opportunity at a later time point and just allows another look at the dystrophin data with the current technologies, and as we stated they will be working with the pathologists who have reviewed the data. They want to get comfortable on how it’s done, and I think this just gives more information about the utility of dystrophin.
Brian Skorney - Robert W. Baird & Co.
And do you have a sense that the parents will be down for this?
Yes, Brian let me add, so when this request came through last year prior to November, we were challenged with getting kind of support of the fourth biopsy because there wasn’t a clear understanding of why this was important and how it was going to enable a higher likelihood of early access and approval of eteplirsen. I think now it’s clear that the FDA providing guidance that this could really support and enhance the dystrophin dataset. And so, we have not gone out to our investigators side, it’s a reinitiating contact. Obviously, this is brand new, but we intend to and we hope that we get support from the boys in our study to get that fourth biopsy. If we were to capture that later this year, we think we can turn that around and include it as part of an NDA filing which again would provide -- assuming it’s positive, we believe would be a further assurance of our dystrophin dataset.
Having said that, the FDA also is collaborating with us to work with the investigator pathologist who oversaw the existing dystrophin analysis we’ve created. And they highlighted that they may have their confidence increase with our current dystrophin after that collaborated with you. So, again, they are giving us a lot of different ways to bolster the strength of our dataset, and this is just one of those and we will pursue it.
And our next question comes from Robyn Karnauskas from Deutsche Bank. Please go ahead.
Mohit Bansal - Deutsche Bank
Thanks. This is Mohit Bansal for Robyn. Thanks for taking my question and thanks for providing this update as well. So, I have two questions, first of all is, you have mentioned couple of things you need to submit from now to NDA filing. So, can you please help us understand which is the most important gating factor if you don’t have to submit all of them? And then I have a follow-up. Thanks.
That’s not how it was described in terms of some kind of gating. We don’t think any of these individually is critical. We think it’s an accumulation of additional data, each one just adds to the demonstration of efficacy, safety of the drug. The way to think about it is almost like a sliding scale, right. We could submit our NDA now on the existing dataset but the FDA has highlighted questions and concerns and they are not as comfortable with just the existing dataset. We hear them and while we could submit an NDA now, we believe that we are going to be in a much better position if we just wait for some of these additional pieces of data. At any time, we think it will just strengthen that package, and we think the amount of data that’s coming across over the next six to eight months leads us to the timing that an NDA at the end of the year is going to put us in the best position not only for an acceptable NDA filing, but for a favorable review for accelerated approval. Obviously, the data has to come through, we expect it will, and so there is no individual piece of data that we see as make or break the chances , it’s an accumulation of an already strong dataset in our mine to increase the chance of a favorable review.
Mohit Bansal - Deutsche Bank
Got it and then on your confirmatory study, have you already got a sign off from FDA on price design in terms of the size of the trial and endpoint or due you intend to do in future?
Yeah, now we already have the protocol almost completed. We were anticipating and preparing for this non-placebo-controlled trial. So we’re very close to having all of the details on this and they stated that they were comfortable with our current design.
And our next question comes from Liisa Bayko from JMP Securities. Please go ahead.
Liisa Bayko - JMP Securities
Hi, there. First of all congratulation, this is a great step forward and great for everyone I think. I wanted to just clarify, so the data coming this year that would help strengthen the packages the 144-week data hopefully by the time you’ve submitted during that review period you’ve also got the 168 and then it’s the fourth biopsy, is there anything else I missing?
Well, the safety data from the early experience of our confirmatory study, the FDA did highlight that could be in enhancing the chances of the positive review in an acceptable filing. Obviously, the interim period will also be focused on the collaborative effort that I described. So this is an important element although it’s not generating new data it could serve to reinforce the acceptability of the existing dystrophin dataset. We’ve already started to put the appropriate people from our investigator side in touch with the FDA and there will be meetings ensuing over the coming weeks, and after the FDA finalizes that review that could provide another kind of bolstering of the confidence that the FDA might have in our current dystrophin dataset.
Liisa Bayko - JMP Securities
And then for the confirmatory study, can you may be described geography how many sites locations as just U.S. and then the patients that are the 68 patients that you’re targeting for enrollment, is that, are those only the ones who meet the criteria or that subset. I know you’ve said that boys who can walk greater than 450 meters would not be included in primary analysis but would be which I think of a great idea would be included for safety purposes and just to see how it performs, are those included in the 60-day or 68 just the core people who meet the criteria?
Liisa the 68 to 80 would be the entire population including the boys who are watching greater than 450. We want to just focus on the primary analysis but again we’re trying to open the study up to really treat as many patients as possible and that’s the reason for doing the younger and the older boy another studies also, and we would be focusing on the U.S. potentially some Canadian sites and about 35 sites in total is our estimate.
And then next question comes from Ted Tenthoff from Piper Jaffray. Please go ahead.
Ted Tenthoff - Piper Jaffray
Great, thank, and my congrats as well, there is really kind coming day for patients and their families and I appreciate you guys hard work to continue push this treatment along for these boys. Firstly a quick repeat if you would, did you say that the primary endpoints will be 48 weeks or how long you think it will be for follow up?
So, first of all the primary clinical endpoint is really appropriate in the confirmatory study, this is the ambulatory boys that Ed described would be the 300 to 450 base line six-minute walk. We are including an untreated cohort in which it would be powered to show a benefit on six-minute walk over this untreated cohort. Now, we described how the FDA did not require that untreated cohort but we believe we can show evidence of that affected 48 weeks. Having said that, the FDA think that they are comfortable just looking on how that Eteplirsen treated arm performs versus what would be expected from the natural history or in a historically-controlled well matched population. And as I described earlier that doesn’t necessarily have a timeframe that could be extended out beyond 48 weeks. So, I guess the best way to think about this is, we are preparing for multiple shots on goal to have a victory here. And so the untreated cohort is 48 weeks on 6-minute walk is one but the FDA is providing us flexibility to look at that treated cohort even beyond 48 weeks. So, again we are trying to do both of those things.
Ted Tenthoff - Piper Jaffray
That’s actually helpful clarification. And then if I may, I have a question on sort of the follow-on compounds and sort of what the process is there, so I appreciate that we will be filing INDs for 53 and 45 in the third quarter. Would those move straight into a placebo-controlled confirmatory study or would you have to do Phase I first, tell us a little bit more about how that process would go and when you think you would actually have data from those study first patients?
Yes, so Ted we are still working out the detail, this was initial guidance but based on the guidance letter and the communications we had surrounding this follow-on randomized placebo-controlled study, we believe the FDA recognizes and understands that this chemistry behave similarly. I mentioned and Ed also reiterated that we have conducted the preclinical toxicology. They have seen some of that data, it will be provided in the full IND submission and again we are seeing these other drugs behave very remarkably similar to eteplirsen and show good safety up to 320 mg/kg in the primate models. So, given that there is a good therapeutic window here and that the safety margin seem significant, we believe that the FDA’s comfort in moving into a well powered randomized placebo-controlled trial that as they have suggested they would like to see the correlation of clinical outcomes to dystrophin. We believe that we need to get to that 30 mgs/kg and we would hope that we can move as rapidly to that, what we believe is a therapeutic and effective dose quickly. So, we will figure out if there is any exposure data that would be needed prior to getting to that 30 mgs/kgs, but let me describe what we are doing in our European study which is supported by an E grant for exon 53.
We have a short period of time that we dose escalate to get to 30 mg/kg. We could potentially have some of that data by the end of the year as well to show that we can get to 30 mg/kg rapidly with another exon-skipping drug. And so the plan would be that when this study is completed that we will have hopefully a compelling clinical dataset, a dystrophin dataset and a safety dataset that could potentially be used to submit an application on those follow-on drug. It’s early days in that but in terms of speculating the soonest that we would have data, I think the earliest we have dystrophin data is after 24 weeks of treatment after that’s fully enrolled. And so again, it’s early to speculate on that. Ed do you have any additional comments?
No, I think this really is somewhat unprecedented and the amount of flexibility that we have been given and I think if you look at we have lot of discussions with the regulatory authorities in Europe and they were very comfortable with our exon 53 program which is a dose titration of the 12 weeks to get up to 30 milligrams and then expanding the cohort to include a much larger group at 30 milligrams. And certainly that’s something that we could foresee happening with the other programs. So, again I think what’s been very useful is the agency has looked at the chemistry and saw a lot of the similarities and because of our experience with eteplirsen that gives us much more flexibility when we look at the follow-on exons.
And our next question comes from Brian Klein from Stifel. Please go ahead.
Brian Klein - Stifel Nicolaus
Hi guys. Thanks for taking my question and congrats on the news. I know it’s still early days and you haven’t even started a trial yet but can you give us your best estimate as to how quickly you might enroll the confirmatory study?
Look, we have already been contacting a lot of sites and doing feasibility for the population that would be enrolled in the confirmatory study. We imagine many of those same sites may be patients who qualify for these other protocols that we described in younger patients and older patients. I think most importantly and I think the very encouraging part of the guidance for us was the openness to the open label design. This means that we can start collecting information safety and efficacy data on patients that enroll very quickly and if we don’t enroll 60 patients rapidly then we can continue to accumulate additional patients to be enrolled. So, we think that this gives us the utmost flexibility and that there is no hard timeline to close complete enrollment. If we find 45 patients right away we’re going to get great valuable information, if we find 55, if we find 65 or 70, it’s less critical as it would be if we were enrolling placebo controlled trial to complete enrolment before we could even look at data. So, again we think that’s less important but over the coming weeks we’ll have a better idea of how many patients we can enroll quickly and when we could potential get to that goal of 60 to 80.
Yes. And the other thing is on products remember is the ability for us to enroll previous drisapersen patients. Also opens up the pool of patients and clearly many of those boys are interested in participating in another trail especially if it’s a non-placebo controlled trial. So I think that hopefully will help us with our enrolment.
Brian Klein - Stifel Nicolaus
Great, and then along those lines given some patients will have prior drisapersen therapy, would their natural history in terms of disease course be altered in some way from what you would expect from a non-treated patient and do you have to separate those patients in terms of your analysis?
Well, we will be looking at it very well in our Cisco analysis plan to look specifically on that. But based on the data that has been submitted so far, there really wasn’t a difference in drisapersen treated to the natural history, so I don’t think this is going to be a major thing. And the only concern that we had and we want to make sure that we look at these patients very carefully that we just want to make sure that there are no carry on safety issues that could be present and then that’s the only thing we’re really worried about.
And our next question comes from Chris Marai from Wedbush Securities. Please go ahead.
Chris Marai - Wedbush Securities
Good morning guys. Thanks for taking my questions, and congratulations. I was wondering with respect to the dosing in the confirmatory trial, could you say what dose you’d be looking at 30 mgs per kg or 50 mgs per kg?
Yes, we would be looking at 30 and based on all the data that we have so far we haven’t been able to see a difference between 50 and 30. So, our plan is to continue on the 30 mgs per kg as the highest dose.
Chris Marai - Wedbush Securities
Okay, great. And then with respect to the follow on exon studies and looking at the idea about demonstrating correlation between dystrophin and then six minute walk test. Has the FDA provided any guidance with respect to numerical type of correlation they’re looking for or are they looking for the type of correlation you seen with your 12 patient study to-date?
Chris this was initial guidance and I think the right way to look at it is that I think it was a recognition by the FDA that we did a placebo controlled study with Eteplirsen granted it was only over 24 weeks we’ve had follow update of now beyond two year. There is a lot that we know about Eteplirsen and if a placebo controlled trial were required with Eteplirsen that would delay the potential for early approval of Eteplirsen by several years. And so I think the guidance was trying to balance the desire to not stand in the way of a potential early approval if the data is there and the NDA gets accepted in it positive review ensues, while at the same time trying to take advantage of an opportunity to better understand our technology more broadly and this could be done with the follow-on exons where we could enroll a placebo controlled study on these follow-on exons.
There is no alternatives right now and we think we could get to a rapid understanding of the both safety, clinical utility and dystrophin production in follow on exons. And so again the way to look at it is that as it if it were a primary pivotal study we’re going to learn a lot and we’re it’s too premature in early stage to I think on the FDA part and our part to suggest what that correlation might look like.
And our next question comes from Tim Lugo from William Blair. Please go ahead.
Tim Lugo - William Blair
Thanks for taking my question, and I’d like to add my congratulations as well. So, obviously have flexible regulatory process. I just want is there a reason not all parents would be amenable to a fourth biopsy and if not all of them do allow their children have a fourth biopsy I guess how does that, does that change anything in your view?
I’m afraid, we don’t Tim we think that again we think that our dystrophin dataset is very strong. And Tim basically the FDA has agreed to collaborate with us and the site to make sure that they fully understand the methodology how the blindly review is conducted the controlled settings and standards that were in place. And we think that that could bolster the way of our existing dataset. The fourth biopsy is we think a good option to bolster even further our understanding of dystrophin and hopefully address any lingering questions or concerns that the FDA has regarding our current dystrophin data set. So we think it’s the right thing to understand this technology, understand the time course of dystrophin there’s a lot that can be learnt with the biopsy that’s been taken, three plus years, after initiation of the drug. So again, we don’t think we needed necessarily but we think it could help a great deal to increasing the chances in the likelihood that this would be a favorable review and gone in our accelerated approval.
And our next question comes from Bill Tanner from FBR Capital Markets. Please go ahead.
Bill Tanner - FBR Capital
Thank you for taking the questions. Chris, just a couple, one, you did mentioned with more 6-minute walk test and you anticipate getting over the balance of the year if it’s positive that would support the filing and so I guess, positive as it is kind of in the eye of the beholder, what would you be expecting? What would you think would be positive? Would it be kind of a continuation of what you’re seeing thus far then have a follow-up?
Well, yeah, I mean I think a continuation of what we have seen as far it would be very positive, I mean we’re looking, at almost three years of data that’s coming up and this boys on average will be over 12 years on average. The fact that, you know these 10 boys from the time dystrophin was produced have stayed on their feet over this period of time. We think is remarkable. So again, we’re not creating healthy boys, right, we’re not expecting to -- we’re not restoring the full link dystrophin we’re converting essentially these 10 boys into better muscular dystrophy. And we are just continue to be encouraged by this stability. So it’s hard to stay and handicapped exactly, what would be great versus good but we think this type stability and keeping these boys under feet is what we hope to see and we’ll know so enough in the second half of this year.
Bill Tanner - FBR Capital
Okay. And then secondly, just kind of curious why the FDA is viewing the 6-minute walk as stated in intermediate clinical endpoint because it certainly seems like that would be more than just intermediate clinical endpoint. But then we kind of get back to the notion that there needs to be some correlation, so kind of help me understand that little bit?
Yeah. Well, again I think this is just a signal that the FDA is applying flexibility in their guidelines under accelerate approval and the guidelines do describe the idea of this intermediate clinical endpoint that kind of predict, kind of a future clinical benefit. And this does not mean that 6-minute walks cannot be shown to demonstrate and prove that clinical benefit and so this is again is just highlighting the flexibility that the FDA had in saying we not only can approve eteplirsen on a surrogate marker like dystrophin, but we also have the flexibility to look at the clinical data and clinical outcome and describe that as an intermediate clinical endpoint that would be further validated in confirmatory studies.
No. I think, that’s the way to look at it, Chris, I think it really is, it is utilizing all of the data legislature that gives them more tool. So because it’s a small data set, this 6-minute walk as data could be consider in term -- intermediate data set. But then our confirmatory if we show again that very similar 6-minute walk as that certainly could be used for a full approval.
And the next question comes from Joseph Schwartz from Leerink Partners. Please go ahead.
Joseph Schwartz - Leerink Partners
Great. Good morning. Thanks very much. I wanted to ask a question each of the two potential pathways to accelerate their approval. And it looks like the FDA said that based on the core tier in your press release that they had significant concerns on the ability to drug, there were conclusion based on the existing data in those areas that interpretation that they want to gain some clarity into? And then also on the dystrophin quantification it seems they actually said that they remain skeptical about that data? So I am wondering what are they issues that you think that will ultimately come up in the approve in the potential panel? And review on the primary and well, the primary and also the secondary endpoint, it be dystrophin and then the 6-minute walk, do you think that they are going to lean towards including patients that lose ambulation for example because they are included in being natural history data? And then they are looking for other methodologies of quantifying dystrophin productions such as western blot things like that? Thanks.
Yeah. So, Joe they did provide us some perspective on the areas of questions particularly around the intermediate clinical endpoint 6-minute walk. And so just to give you a sense of the types of things that they highlighted, yes, they identified that our most persuasive data was in the modified intent to treat and that you know excluding the two boys who went non-ambulant early you know is not an appropriate intent to treat analysis. So they highlighted that, we’ve acknowledged that from the beginning and have been very clear about the intent to treat. Having said that we think our pulmonary function over the extension timeframe is in the intent to treat population and includes the two non-ambulant twins. They highlighted for example the open label nature of our extension study and that suggesting that the walk test can be strongly influenced by motivation and coaching and that open label trials are susceptible to bias on the part of investigators or patients or parents, so these are things that are inherent in open label studies and so I think that coupled with the small sample size right, is leading them to say, well if you can show this type of activity in a larger cohort, right, which the confirmatory study would do in the same open label format that’s the type of thing that they’ve highlighted as concern. And of course the 144th and 168th week will go further to be able to compare this population versus what you would expect in the natural history, so the further out you go, the easier it is to see that these boys are behaving differently than the natural history. And so they want to make sure that this population can be controlled to historical, natural history in the right way and this is why we’ve said on earlier call that we’ve requested the raw natural history data from the recent publication to see if those can be sent to the FDA, this will allow them to look more specifically at a more matched historically controlled population and compare them to our current study and we think that’s another way that they can get comfortable on the existing data set. As it relates to dystrophin, we talk a lot about this, there’s a lot of different opinions about dystrophin and the different ways to quantify it and the different ways to interpret the various assays and so we remain steadfast that we think we did a very controlled methodology that produced very robust dystrophin data set and we appreciate the FDA is saying okay let us work with you to better understand it. So maybe they can get closer to our point of view on how we did things, our belief in the current data set and then the fourth biopsy if it’s feasible just gives us another chance to work with them and do it in a way that we can all agree on will be persuasive and important. So again these issues and questions and concerns are not new, this has been going on throughout our discussions with the FDA, for the first time I think we have clarity around where those questions actually are and how we can work towards addressing them over the coming months both to interpret the existing data as well as generating new data.
And our next question comes from Yaron Werber from Citi, please go ahead.
Yaron Werber - Citi
Great, thanks for taking my question Chris, I have a few questions or so. So I just wanted to maybe follow-on on the last question relating to the fourth biopsy and the need to potentially some level correlation, does the FDA, I’m trying to get a sense, I mean the FDA obviously can suggest but they can’t mandate, how important is this fourth biopsy because it sounds like it’s fairly tentative sort of from your angle I can imagine being a parent I wouldn’t necessarily want my kids getting a fourth biopsy, how important is that and I have a follow on too.
As Chris had mentioned previously in the call, again this is only piece of data and information and I think one of the advantages of this recent information from the FDA is there’s a lot of flexibility, so maybe they could get comfortable with it, is this, with the current data set by looking at how we obtain the dystrophin (Ph) and measured it, so I think overall it’s not that critical but it’s one of the many paths that we can use for clinical and from a biopsy perspective and dystrophin expression to get them comfortable with the data set.
Yaron Werber - Citi
And it does mention, and this wasn’t discussed in the call that there is going to be an FDA ad com, what do you think that ad com is going to discuss because you can imagine the FDA’s view, the FDA is going to highlight all the areas of concern, where to fix the endpoints, a lack of correlation in dystrophin, what do think is going to be discussed and then do envision that other companies at this point one would imagine are probably going to be filing also, are all companies going to be subject to the same panel or not.
Yaron, I mean I can’t comment on what other companies may or may not do and what that means, related to the advisory com, we’re not surprised at all that they would highlight that this would go to a public advisory panel. Look, it’s clear this program has captured a lot of attention. The patient community is very engaged and involved. Congress is trying to see if the déjà is been interpreted and lived up to. So there is a lot of people with eyes on this program and this decision this password approval. So it’s not a surprise at all that they would let us know that advisory public advisory panel is likely in this. And we actually are happy about that because, it’s been difficult to have these negotiations with the FDA over the last year, because they are proprietary discussions. And we think that everybody will benefit from hearing our arguments and hearing us address and interpret our data in the right way and be able to address any FDA questions or concerns in a public forum. And obviously the patient voice has been heard more loudly but that’s an important piece to understand the risk benefit in a public area.
But more importantly the advisory panel gives us another opportunity to prepare additional data even after NDA submission and the FDA has told us that they have flexibility in accepting additional data after the submission and in time for an advisory panel meeting. So we think they’re giving us an opportunity to put our best foot forward in a public way in an advisory panel and gives them the opportunity to for lack of a better word make it easier for them to say yes if the data is strong enough and they want to approve the drug.
Our next question comes from Kim Lee from Janney Capital. Please go ahead.
Kim Lee - Janney Capital Markets
Good morning and thanks for the update. Just a clarification on what does the FDA want to see as far as dystrophin goes to make them comfortable, is there a correlation and kind of what’s been their feedback on what they want to see as far dystrophin goes? Thanks.
Kim as we described I think we did a very controlled methodology using what were the state of the art practices, using immune florescence, dystrophin fibers and dystrophin intensity, we did RT-PCR, we would do all of this western blot and a full analysis and we were able to show dystrophin across the various unit type. I think this is a new field and really the first time that a drug technology has demonstrated this level of dystrophin using these methods. So I think the FDA wants to get comfortable that they understand how to interpret this and understand the methodologies more fully. And I think we will both arrive at a more specific point of view on what to expect in terms of dystrophin production, how to best quantify it. The various ways that we can triangulate that across the different methods and we appreciate the FDA is saying we want to collaborate with production, how to best quantify it. The various ways that we can triangulate that across the different methods and we appreciate the FDA is saying we want to collaborate with you Sarepta and with the site that we’ll be conducting this to interpret our existing data and to help us moving forward.
So again it’s pretty mature to suggest exactly the type of co-relation that they would be looking for, I think the data that we generate will help us better interpret it.
Our next question comes from Steve Brozak from WBB Securities. Please go ahead.
Steve Brozak - WBB Securities
Good morning and congratulations like everyone else but you had mentioned something a couple question back and actually picking my curiosity, as far as an advisory panel is concerned these are obviously made up of clinicians that have a day to day understanding of what takes place. And the community is pretty small to begin with on the clinician side, so having dealt with the FDA repeatedly and having dealt with the clinicians. What kind of feedback are you getting from the clinicians as far as what has just taken place in the significant amount of back and forth with the FDA. Can you start with that and I got one follow up please.
Well first we know and became aware that there have been a couple meetings among the FDA with experts in Duchenne. One of them took place in December and another one took place in early February. They really got exposed to a broad swath of researchers and clinicians who are expert in Duchenne and could help them understand not only the expected natural history of this disease but also how to best interpret the data that we’ve generated to date. Independent of that, we as a company attend the medical conferences we are asked to share our latest data and provide that to many researchers and clinicians in the field so they can better understand this technology in the emerging data set and we have received very positive favorable responses from the DND researchers and clinicians that we talk to and how they interpret this relative to what would be expected in the natural history and what they seem to data with other attempts to produce dystrophin or to have a clinical benefit. But as you described you talked to a lot of these…
Yeah I think over the last several months we have had an opportunity to talk to a number of people. And the one positive result of this as we present the full data set and get people comfortable with the amount of work and data that has been done. People and the experts and the community I think have been very supportive. And this has been very useful for us. And I think the two aspects that really have come out is the natural history as what we’re seeing in our outcome as far as 6-minute walk test and the pulmonary data are really not what you would expect from an untreated boy. And that has been reiterated over and over again.
So I think the community is getting much more comfortable with our data set as we give them more information and provide them with the full outcome measures that we have obtained.
I will now turn the call back over to Chris Garabedian for closing remarks.
Yeah thank you operator. Again we’re pleased that we have received this guidance letter from the FDA. It provides us all of the information we need to be able to move our program forward not only with the eteplirsen but on the follow on exons importantly. And we have a clear set of guidance to prepare our NDA for submission later this year. We’re really excited about this news and we think we’ll be in good shape to submit our NDA by the end of the year, which would potentially give us an opportunity to see the filing excepted in early 2015. A panel that would take place in the first half of 2015 for a potential of the drug approval by mid ‘15 or later.
So we really appreciate this and thank you for your interest in Sarepta.
Thank you ladies and gentlemen, this concludes today’s conference. Thank you for participating, you may now disconnect.
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