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Sarepta Therapeutics, Inc. (NASDAQ:SRPT)

Deutsche Bank Healthcare Conference Call

May 07, 2014 08:40 a.m. ET

Executives

Chris Garabedian – President and Chief Executive Officer

Analysts

Robyn Karnauskas – Deutsche Bank

Robyn Karnauskas

Good afternoon, my name is Robyn Karnauskas, I am one of the biotechnology analysts at Deutsche Bank, and it’s my pleasure this morning to have with us Sarepta Therapeutics. With us, we have the CEO, Chris Garabedian. Thank you, Chris, very much, I appreciate it.

Chris Garabedian

Thanks Robyn. It's great to be here.

Robyn Karnauskas

So I guess the big picture, for those of you who don't know Sarepta who are listening in is they are developing a DMD drug, and there has been some controversy around the development strategy for that drug, and recently they got clarity from the FDA as far as the path forward, which I think is really positive for the company and should be the main focus of investors in my view. But, maybe talk a little bit about, I guess, I will start off with what change between November and April that lead to the recent FDA’s guidance for developing the drug, and why do you think an NDA is no longer premature?

Chris Garabedian

Yes, thanks Robyn. The guidance we received a couple weeks ago, and then we had a conference call on was really what we were waiting for. It had been a long year. We had many interactions going back over a year now with the FDA, but really it started to become more critical in the November communications. At that time, they had questions about our clinical program. They indicated that an NDA was premature on the existing data set, and there was a lot that transpired between that early November timeframe and ultimately when we got the April communications.

So, in that five-and-a-half months or so, we had four meetings with the FDA. We had multiple requests for additional data, and this was on top of a lot of detailed data we gave them already, and some of that was source material to -- that supported our dystrophin analysis, but they also did their own work so to speak on the DMD market. They talked to expert panels in December and February, and they had met with further patient advocates. There was a lot going on in the DMD community, where there was a petition and congressional involvement around, is FDA living up to FDASIA. So, there was a lot going on in that five-and-a-half months, and it culminated in this letter that they indicated represents the minutes for all of the four meetings that we had from November to April, and it really provided the most clarity that we have received to date.

Okay, and so that's really why their communication changed from November to April, and I wouldn't say that they said, “Oh! we were wrong and we didn’t mean what we said in November,” but it was an evolution. It was “okay, while we said that the current data set was premature, we believe an NDA -- their words NDA should be fileable with additional safety and efficacy,” but when they described all the different ways that we could supplement that additional safety and efficacy, and I would also add perspectives on the current data set, they really looked – they gave us almost a sliding scale of saying, “there are many ways that you can bolster the current data set.” I am paraphrasing here, but they didn’t say “we are not telling you can't submit an NDA tomorrow on the existing data set, we are not saying it wouldn't be fileable on the current data set, but we are telling you that we have raised enough concerns on the existing data set that you would bolster your case for an NDA filing and potentially a favorable review if you allow us to do a more detailed review of your dystrophin methodology, if you maybe consider getting a fourth biopsy, if you add exposure data from the new patients that are enrolled in the confirmatory study, if you supplement it with the 144-week data, and by the way whenever you submit that NDA, we don't always do this but we are opened to receiving data after the NDA is submitted”, and so that's when we said we can get 168-week data early next year.

We can get additional safety exposures early next year, and what we chose to do as a company is we said, “what's the best position we can be in to not only have the strongest NDA submission but also going to a public advisory panel with the strongest arguments, so an independent panel of experts can see the additional clinical data, the additional safety exposure data, potentially the additional dystrophin data.” So, that's why we chose to highlight the NDA would be submitted at the end of this year.

So, there was lot that transpired, a lot that changed over that intervening timeframe, and we finally ended up with the kind of guidance that we were looking for in terms of clarity around the NDA.

Robyn Karnauskas

That's helpful. I think where people get stuck is they feel like the FDA offered up all these options for accelerated approval, but when they look at if you were to execute on those options, that maybe they wouldn't hold up, like just to give a simple example, the modified Intent-to-Treat, well, they don't like the modified Intent-to-Treat, but if you include those patients, then it's not stat sig, so then every time you go down the line with, okay the FDA said you could do this, but if you do it, then like it may not prove anything, that's where people are stuck, so can you maybe help us on these points like what do think is your strongest point that you can support for the FDA to believe you in an accelerated filing?.

Chris Garabedian

First of all, I think you could talk to anybody in, any CEO in the biotech industry, and they would say I don't think there exists an FDA guidance document that doesn’t provide some counterbalancing views, right, of something that would be positive, right. It's their job to not guaranteeing anything. It's their job to highlight what questions and concerns, and I don't think there are many examples of an advisory panel where the FDA isn’t out there presenting in a public forum their concerns and highlighting why, maybe we should be cautious about approving a drug, but at the end of the day, it's the totality of the data, and it's the risk/benefit of the disease, and it's making the right decision of, do we know enough, right, to approve this, and in this case of accelerated approval, the burden is a little bit different, it’s reasonably likely, right, and so what they basically told us, and this is in the press release and using their words is, they said “we can see the possibility of an accelerated approval in two different ways, the first is through dystrophin as a surrogate marker,” and they basically said that upon a more detailed review of which we have already begun that process with them in over the next several months, we expect them to have met with the site that did the work, they already know the methodology, we’ve provided them a lot of details of how we did the analysis, but they haven’t actually gone there and talked to the pathologist, talked to the technician, talked to the reviewers, and looked at the lab and saw how they read the images in full resolution, etc.

So, upon more detailed review, they said, “we could envision approving it under accelerated approval on dystrophin, including the existing data set without any additional dystrophin data.

They also said you might want to explore the feasibility knowing it may not be possible, but explore the feasibility of a fourth biopsy, so we are doing that and will know in the coming weeks how many parents may agree to participate in that and what the timing of that potentially would be later this year, but the hope is if we can get a fourth biopsy that we could submit that as part of the NDA later this year.

The second pathway that they outlined was what they referred to as an intermediate clinical endpoint, so this is not a surrogate marker that we (inaudible) predict, but it's an intermediate clinical endpoint, and they explicitly state we could see an accelerated approval on the intermediate endpoint of six-minute walk just from our 201/202 Study, so that's the existing 10 boys that are being followed on six-minute walk.

Now, that doesn’t erase all of the questions they would have about how is your ITT analysis modified, look the boys who are in a wheelchair, they are not going to – we don't expect them to get up and walk and start contributing, so the FDA knows that, they wouldn't have given us the guidance about six-minute walk being acceptable --.

Robyn Karnauskas

Can I just interrupt and ask you about that, though, like if you included, though, it is not stat sig, so the FDA needed stat sig trial??

Chris Garabedian

No, we don't believe they do, of course we do have the statistical significance on the modified Intent-to-Treat. We had that from week 32, okay, when it was still a blinded study even after the patients rolled over under open label. We know that they often say if the clinical outcome is meaningful, okay, that the statistical significance isn’t necessarily or the lack of it isn’t necessarily a deal breaker.

Conversely, a lot of times, they don't like overpowered studies where you might see -- you could power a study, let's say, and show a five-meter benefit that's not clinically relevant, and they might not approve it because it's not a clinically meaningful endpoint. So, I think they look at the totality of the data, and we believe that this intermediate clinical endpoint that FDASIA reinforced in 2012 as, please consider these intermediate clinical endpoints as another alternative to surrogates on the basis of accelerated approval.

Robyn Karnauskas

But really what you are saying is by intermediate clinical endpoint, it's really just six-minute walk without having the stat sig, and if there was a real end point, it would be a stat sig six-minute walk?

Chris Garabedian

That's one way to look at it, that's one way to look at it. The other thing that we would highlight and emphasize, although this wasn’t mentioned in the guidance, is the pulmonary function. We do have an Intent-to-Treat analysis that has shown really remarkable stability over this two-and-a-half-year time frame. So, we would hope that they would see the meaningfulness of our pulmonary function that complements the modified Intent-to-Treat of six-minute walk.

Robyn Karnauskas

But when they talked about like the intermediate endpoint, they were really -- they specifically were talking about six-minute walk, they didn’t bring up the pulmonary function. So, you guys are bringing that up as another option for the intermediate.

Chris Garabedian

Well, we would put our best foot forward, and we think that is a reasonable endpoint that is well characterized, that has been validated as an endpoint in the basis of other approvals, and so we don't think that omission was intentional. We just -- that's something we have only recently shared is the 120-week data on pulmonary function, so we think that could bolster the confidence in the six-minute walk data that we have.

Robyn Karnauskas

Okay, I am sorry to interrupt.

Chris Garabedian

No, please.

Robyn Karnauskas

So you were going to – you said dystrophin intermediate clinical endpoint, what other options as far as for accelerated approval, are those the main ones?

Chris Garabedian

Those were the two that they have outlined.

Robyn Karnauskas

Can we talk more a little bit about dystrophin, because then I am going to get to some other questions people ask me. They are still confused about like, okay so what is that really the FDA need, you mentioned that maybe they need to fully understand how you do the dystrophin analysis and maybe they need a fourth biopsy. Do they need to be convinced of the correlation that there actually is one or do they just need to be convinced that it was done properly? Can you sort of give a little bit more description as to what you think the FDA might really be going after there?

Chris Garabedian

Yes. It's hard to know exactly, right, but what we can try to glean from their communications and our interactions with them is that they have their data, okay, so they have the same data set that we know was derived from the analysis, and I think it's about getting a comfort with how the analysis was conducted, so there is obviously a lot of moving parts when you do this, and we took great pains to control for these to make sure there was a blinded review, to make sure there was consistency in the methodology across those three biopsy time points, to make sure the conditions were the right conditions, in other words it has to be that you can't look at a biopsy slide in a room like this on an overhead, right, and try to see which fibers are positive and which are negative. You have to almost be in a dark room type condition and really keep the amplifications consistent, these are high-resolution images, and you have to do it on a consistent basis across every single one and not knowing what you are looking at, right, and so is it a 30 mgs, is it 50 mgs, is it delayed treatment, is it placebo, is it a pretreatment, all of these were done in controlled conditions. So, I think they want to understand and talk to the people who actually did this and make sure that they are comfortable that it was done the right way. I can tell you, I talked to a lot of the members of the lab there, and I think they are world-class in terms of how they conduct this.

Jerry Mendell’s site at Nationwide in Columbus, he is probably the most experienced clinical trialist in Duchenne, he does multiple studies. The reason that we selected his site was because we could do everything in the four walls. He has a world-class physical therapy team. He has got special, some of the leading cardiologists in Duchenne, he has got a leading immunologist in Duchenne, and so this is a world-class organization, and we think when the FDA actually speaks to these folks, they will agree with them.

Robyn Karnauskas

Okay. So it's really more about how it's being done rather than like this correlation in your view.

Chris Garabedian

Yes.

Robyn Karnauskas

Okay. Then big picture, so we are thinking about accelerated approval, you know I guess I can't ask you what the probability you would place on it, but are you more confident or less confident than you were before, because a lot has happened, say from the very beginning from the first time you filed and said we are going to go for accelerated approval, where do you think you are now?

Chris Garabedian

You are right, it is hard to handicap what the FDA will ultimately do.

Robyn Karnauskas

Be careful because people are just going to put that number --.

Chris Garabedian

I know I am not going to give the number, that’s why I’am going to give the numbers. So but here is what I would say, we at Sarepta who have looked at this data from day one with a critical eye have believed that there is a real effect here biochemically, clinically, and we are very pleased with the safety profile. So, from the beginning when we submitted that first briefing document on the 48-week data back in February 2013, we were confident that this might warrant being looked at for an accelerated approval, okay. We have only seen our confidence increase, okay, as we have gotten more and more updates on the clinical and safety outcome parameters. Our confidence in our data set has increased even further now that we’ve spend the last six months really talking to a lot of experts in the field. These are experts in natural history, they are experts in dystrophin, they are experts in pulmonary function, they are just practicing clinicians who see a lot of Duchenne patients, and the more we share our data set with them and get their feedback, what do they think, that gives us even more confidence that our view on the data set is the right one that this drug is working.

So our confidence level, I would say, has increased overtime, as the data has held up, and I would say given the recent FDA communications, I am confident that there is more of an understanding on the FDA's part of this disease, right, of how we conducted the study, of the expert opinions, they have talked to some of the experts directly themselves. And so, I think overall, my confidence level has increased over that period, over the last year because of additional data, additional perspectives from experts.

Robyn Karnauskas

And confidence not just in the data, but in accelerated approval?

Chris Garabedian

Well, I think it goes hand in hand. The accelerated approval, it has to be on the basis of how strong the data set is, and I also don't want to underestimate the patient voice in this whole process, okay. They have weighed in, they have demanded a seat at the table to say this is our disease, we are living with this every day, and we are seeing the data with our own eyes, right, and we have tried to be transparent as a company in the level of detail that we have shared so that not only clinicians but patients can also see what is our data saying, and they are saying this is good enough for us, this is safe enough and the clinical signals and the biochemical signals are enough.

So they have demanded that seat at the table, they have requested frankly more meetings with the FDA across the advocacy organizations than we have, so they’ve probably had in total more hours in front of the FDA and the FDA hierarchy than we have, and I can't underestimate the role and impact that the patient community has had in the FDA's view on how to treat a disease and treat a data set when they consider an accelerated approval.

Robyn Karnauskas

Okay, and then the last question, and then we will go to the (inaudible) and then the broader options for Sarepta, so this is an investor perception question, again they keep asking why are we still focused on accelerated approval? Why don't you set the bar really low and say, look we don't know, so that they kind of get some of the volatility out of the stock. Maybe you can answer that little bit.

Chris Garabedian

Well, actually, I think a lot of the volatility was driven by two things. A lot of uncertainty and lack of clarity from interpreting the FDA guidance and what does it really mean. This is always a hard game anyway, but it's been particularly hard with this particular program. So, I think that uncertainty and clarity has been taken away, and the other thing is that it really is hard to separate, and I think the FDA recognized this too, the path toward accelerated approval and what the company needs to do to confirm the results that we have seen, and how to move forward with our program, and the reason I say that is that clearly if the FDA mandated a placebo controlled study with eteplirsen to confirm the results, then there is no pathway or possibility of an early approval for several years, and the community recognized that. I think the FDA recognized that. So they had to really assess and weigh in on the possibility of accelerated approval because it would impact our ability to do a study or complete a study.

So, I think their net outcome was, we are going to give you a pathway that allows an open label study that you can continue indefinitely even in the event that we approve your drug next year, so they gave us that pathway and we couldn’t get that clear guidance and understand if accelerated approval was possible until they told us that. But what they did was something very novel and creative, which was they said, okay, but we are still not going to lower our standards of what we would need in terms of confirmation, so although we are going to ask you to do this larger, broader population study with the eteplirsen open label and will let you follow them indefinitely and will decide at the point that we think they are different enough from natural history, that could be at a year, it could be at 18 months, it could be at two years or beyond; it could be on six-minute walk, it could be on other measures, but they are saying go ahead and do that study and get more exposure and experience with eteplirsen, but here is something that we think is even more rigorous, and we are willing to consider not and/but/or a confirmatory evidence on your follow-on exons in a placebo controlled, well-powered study, and if you can prove with the same technology and you know it's the same drug components that we use, the same manufacturing process, the same dose that we would be enrolling in a confirmatory study, if you prove that, then we could use that as confirmatory evidence if we approve eteplirsen under accelerated approval.

Robyn Karnauskas

So if they approve under accelerated approval, you could go, but you are still doing another trial in exon 51, so then what would that trial be, and why do you need the other trial, this is where it gets very confusing to people.

Chris Garabedian

Yes, well…

Robyn Karnauskas

And you should put out a chart which explains like the different --.

Chris Garabedian

Well, it shouldn't be confusing because essentially what they are saying is that we are giving you two possibilities to confirm the results that we have seen in the event we approve it early under accelerated approval. We are telling you two different ways, now, to be safe, we are not letting you choose which one of those, we want you to do both, but we are only asking for one of those to confirm the results.

Robyn Karnauskas

What if one fails and the other one works?

Chris Garabedian

Well, I will tell you one thing that they provided in terms of color. They said if the follow-on placebo controlled study, because I think they are thinking that if it's – if there is a placebo control, we may actually get to an answer sooner, right, versus the control that we could find that in 48 weeks. They may not believe that we can be definitive proof at 48 weeks in eteplirsen, so they actually suggested that we can talk about terminating the eteplirsen study if you have evidence of your follow-on exon study to show confirmation of an accelerated approval of eteplirsen.

Now, we wouldn't do that. We would say we want to follow these patients, we want to continue them indefinitely, because that's valuable long-term data, but they are suggesting if you prove it on one, we don't need it. If we prove it on the natural history component, great, that supports eteplirsen as well. So, I think they are showing very impressive flexibility to do the right thing for this program and for the patients in Duchenne. So, we see it as a positive, but they are holding us to say they want both of those studies to be enrolling patients at the time we approve the drug.

So that's why we are rushing very hard to get both of those up and running by the end of the year, and we think that they will be well under enrolling if we were to get approval my mid next year.

Robyn Karnauskas

Okay. Now walk us through the time lines, like for example, are we going to get color, any color on a fourth biopsy? When would we get that, would you provide that? Walk us through the little miniature milestones leading up to potentially a panel like so we can understand the time lines for this and maybe even clarity on what path you may ultimately take?

Chris Garabedian

Well, so what we stated on the call a few weeks ago is that at the end of the year what we would hope to have as part of an NDA submission is we would have the 144-week data by that time. We haven’t determined when we are going to announce that specifically, but we would expect that to be presented at World Muscle later this year, so that's the 144-week data. The early exposures of the eteplirsen confirmatory study, we said we expect to start dosing in the third quarter.

We won’t have a lot of patients for a long duration of safety, but whatever we can have when we kind of cut that data set, we will submit as part of the NDA, but remember the FDA said we can also supplement additional safety after the NDA goes in, so that may go in after a submission in the early part of next year.

Robyn Karnauskas

So basically we could just have additional in first quarter 2015 safety data, would you press release set?

Chris Garabedian

It's not clear. We’d probably include that as part of our general update, but we haven’t determined, because it's a – we don't know how quickly enrollment will go, we don't know how many patients we will have or how long the duration is, but whatever we have we would like to summarize that and submit it as part of the NDA by the end of this year. The fourth biopsy, we are looking at the feasibility of that. We are encouraging it. We think that this could be invaluable to not only reinforce our dystrophin data set, but here we are approaching three years of treatment, they were dosing in August of 2011, so this would be August of 2014. It would be nice to know, and I am sure the FDA would like to know what level of dystrophin do we have after three years of treatment. Is it more, is it the same, and so that's something that we would hope to have to submit to an NDA by the end of this year as well. It's not clear when that would happen, but hopefully sometime in the third quarter.

Robyn Karnauskas

Would you let us know how many people are opting in for that, like maybe on the next quarter call and when will we give clarity on that?

Chris Garabedian

Yes, I think we would have to make sure that we knew definitively, but yes I mean, I am not sure when we would do that or how we would do that, but as you know we have always tried to be transparent with our program moving forward, so we will figure out what details are important for investors to know -- to understand what data we will be generating, but we don't have that information as I said here today. The other piece of information is the detailed review of our dystrophin methodology. As I mentioned, we expect this to happen over the coming months. It’s not clear if that’s going to result in any kind of follow-up communication, it just may be an increased comfort with the FDA, it may be informal communication, but that's something that we will have a better idea of how that interaction has gone with the site, and again I don't have information of when that might occur at this point, but we expect over the next several months that that interaction between the site and the FDA will have occurred.

Robyn Karnauskas

So it sounds like near-term on the fourth biopsy color and potential color on dystrophin methodology would be there, and for the end of the year more clinical data.

Chris Garabedian

Yes, and I think maybe World Muscle is in October, again there may be a communication prior to World Muscle, but again we don't want to compromise in the podium presentation that we would hope to get at World Muscle.

Robyn Karnauskas

And you are going to let us analysts go this year --?

Chris Garabedian

It's not up to me.

Robyn Karnauskas

I would like to ask the audience if there are any question, because I want to focus on the platform. Does anyone have any questions?

Okay great. Okay. So, another key thing and especially for my thesis as well as for other investors is understanding this is the platform technology, and DMD is a very challenging disease, but you can actually maybe apply it to other diseases easier. And then the question is how do you finance that, so when do we get to see the next data point of like the (inaudible) platform which could de-risk your - de-risk the stock, could de-risk the program?

Chris Garabedian

Sure. So, first you said -- and how we are going to finance that, obviously we just completed $100 million offering in the recent weeks, and so that has really bolstered our balance sheet. We have an earnings call tomorrow. So ultimately, we have just moved in recently to a new facility in Cambridge that has 15,000 square foot of new lab space.

We hired a new Chief Scientific Officer, Art Krieg, earlier this year. We still have lab space open and working in Corvallis, Oregon, and we have really picked up our collaboration with academia over the last year-and-a-half, and so as we get data from these collaborations, we will share it when there is something compelling where we can get excited about preclinical development of the next target, the next disease area.

So, look, we are as anxious and excited to reveal the promise of this technology as anybody, but we are not going to just put out a bunch targets out there until we have, we believe, proof of concept that is differentiated and that is compelling enough for people to believe that this could be something that becomes a clinical candidate and something that’s going to have an impact on patients and the market.

So but we are getting to work. We are building out a broader team of research through Art Krieg’s leadership, and stay tuned, but I can't speculate on what that will be, how compelling that it is going to be, but when we have it, we will be ready to share it.

Robyn Karnauskas

Will it be this year?

Chris Garabedian

We hope.

Robyn Karnauskas

Would you host an Analyst Day around that to better understand the technology?

Chris Garabedian

Yes, it's a great question. We have talked commonly about -- we think it's probably time to start engaging analysts and investors more specifically about our platform technology and where it can go, and we want to see if we can have additional data to start sharing some of this, and so we expect that to be in the later part of the year. Now, we have got a lot of stuff going on, and it's busy, but we are working to figure out what the right timing of that is and stay tuned for that.

Robyn Karnauskas

So as far as financing, say you identify a target and you have some proof of concept, do you have enough cash to take that into Phase II program? It could directly go to Phase III, I assume. So how do you think about developing the next generation budgeting not versus the DMD program?

Chris Garabedian

Well, look, we are very hopeful for this technology and a lot of technologies have focused disproportionately on liver generated targets, okay. We think our modified and conjugated chemistry can go after other cell types that broaden the potential application in other disease areas, and we aren’t going to have the bandwidth and the ability to prioritize, I mean, the DMD pipeline alone is going to be keeping us busy for the next several years. We think the neuromuscular markets, we can navigate well, maybe some other rare genetic disease targets, but if you think about immunology and inflammatory disease or you think about hematology/oncology or you think about cardiovascular, metabolic, or more broad CNS targets or infectious disease, we have good proof of concept in infectious disease. We are not expecting to have five or six new therapeutic areas that we are going to try to do de-novo development on, and that's where we are opening the door to strategic partners to help us with new targets and new disease areas, and as you know, it's hard to speculate on that, but we think we finally have good proof of concept, we are ready to…

Robyn Karnauskas

Do you have it yet or -- do you have the proof of concept already for these?

Chris Garabedian

We have, no have good proof of concept on…

Robyn Karnauskas

For your drug?

Chris Garabedian

…what has been published.

Robyn Karnauskas

And so these guys need proof of concept in these individual areas to partner?

Chris Garabedian

Well, I think they need to have confidence in the technology platform. The beauty of RNA therapeutics is that in some ways it turns drug development on its head. We know the target, we know we can screen against the target very efficiently whether its translucent suppression or altering splicing, so it's the vehicle, it's the chemistry that has to get into the right cell type. Once you get into that right cell type, call it T-cell or whatever you are aiming for, then we are opened to whatever the partner wants to look at from a target, and that proof of concept can go fairly rapidly to say this can knock down this target, right or this can alter splicing in the right way, and so ultimately that’s something we could do in collaboration with a partner.

Robyn Karnauskas

Because we look other collaborations that have been transformational for a company, you would get a lot more validation, I think, if you had like Genzyme has done this (inaudible), you are really the only one in like the public space that has not had a partnership yet, so but what we have seen I think when those partnerships have happened is there has been some proof of concept data like some in their indications (inaudible).

Chris Garabedian

Look, we think we have enough to get partners excited, and yes it's not lost on me that the type of deal that Sanofi Genzyme did with Alnylam, it was very interesting. It reinforced that companies are ready to invest in early research and platform deals, and there was an equity component there, right, that was able to bring in additional cash with the right strategic partner, and we think that we have best-in-class chemistry and can go in all the places other RNA companies are going, short of any blocking IP you can look at any RNA therapeutic company pipeline, and for the most part our technology can go there as well whether it's microRNA, whether it is siRNA, whether it’s antisense. So, I think, as we convey that and we have a great CSO who is helping us translate this who has a long history in RNA therapeutics who has interacted with almost anybody who is a who’s who in the RNA space, and I think he has a lot of credibility, and so that was the important piece for us to fill in so that people have the confidence and the belief that this technology is real or we wouldn't have attracted the likes of in Art Krieg.

Robyn Karnauskas

And that would be groundbreaking for the company’s valuation, I think investors would be happy. Any last question? I guess only one and you guys give this to me, and I get this all the time, so I should ask Sarepta’s global strategy, update on global strategy to bring the drug to patients outside of the United States, what's the latest update?

Chris Garabedian

Yes, so what we have been saying all along was we have focused on the FDA, we focused on US, and now that we have that guidance, we have that clarity, and we know what it's going to take to get eteplirsen approved in the U.S., we have always said that's when we are going to shift our focus to the EMA and that's what we are doing. We’re already planning to dose our next exon, skipping drug exon 53 in the fall in Europe, and so we expect that to get into a confirmatory or a pivotal study, you know, shortly after a dose escalation phase for that, and that's working with the leading opinion leaders in Europe. We are also looking for scientific advice on eteplirsen in Europe, so we are going to be talking with EMA about much like we have with the FDA, what is it going to take to consider eteplirsen approval in the EU and is this current data set something that they could apply under their similar type of accelerated approval that they would have in Europe, but more importantly, the follow-on exons besides exon-53, we would expect this placebo-controlled study we are doing with exon-45 and exon-53 we want to make sure that we have that design so that we could potentially add global sites to that so that when those data read out that we can use that for registrational approval in other territories including Europe.

So, I think this is the year that we are really shifting and moving into a more global footprint and to make sure that we start to think more broadly about not just eteplirsen, but our broader DMD program globally.

Speaker

Could that be discussed at the Analyst Day in the fall?

Chris Garabedian

It actually could.

Robyn Karnauskas

Okay. Thank you very much.

Chris Garabedian

Alright. Thank you.

Question-and-Answer Session

[No formal Q&A session for this event]

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Source: Sarepta Therapeutics' (SRPT) CEO Chris Garabedian at Deutsche Bank Healthcare Conference (Transcript)
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