Good day, ladies and gentlemen, welcome to the Sarepta update call. At this time, all participants are in a listen-only mode. Later, we will conduct a Q&A session, instructions will follow at that time. If anyone should require assistance during the conference, please press star then zero on your touch-tone telephone to speak with an operator. As a reminder, this conference call is being recorded. I will now like to turn the conference over to Mr. Ian Estepan, Vice President, Chief of Staff and Corporate Affairs. Sir, you may begin.
Thanks, Brian. Good afternoon. Thank you for joining us on our micro-dystrophin update call. I'm joined today by Doug Ingram, our Chief Executive Officer, Ryan H. Wong, our Chief Financial Officer, Dr. Gilmore O'Neill, our Chief Medical Officer, and Dr. Louise Rodino-Klapac, our Vice President of Gene Therapy. I'd like to note that during this call, we'll be making a number of forward-looking statements. Please take a moment to review our slide on the webcast, which contains our forward-looking statements. These forward-looking statements involve risks and uncertainties, any of which are beyond Sarepta's control. Actual results can materially differ from these forward-looking statements, as any and such risks can materially and adversely affect the business, results of operations, and trading price of Sarepta's common stock.
For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent quarterly report on Form 10-Q filed with the Securities and Exchange Commission, as well as the company's other SEC filings. The company does not undertake any obligation to publicly update its forward-looking statements based on subsequent events or circumstances. With that, I'd like to turn the call over to Doug to provide an update. Doug?
Thank you. Good afternoon, everyone, thank you for participating in this call with us. As announced in our press release this afternoon, Nationwide Children's Hospital received a letter from the Food and Drug Administration on July 24, 2018, yesterday, stating that the phase I/IIa Duchenne muscular dystrophy micro-dystrophin trial has been placed on clinical hold. My goal in this call is to place that clinical hold in context and provide ours and Nationwide Children's plan to expeditiously satisfy the requirements to come off clinical hold with the goal of not delaying our clinical program. Let me briefly at first describe the reason for the clinical hold. In connection with routine quality assurance testing on a lot of the gene therapy material, trace amounts of a DNA fragment were detected.
This trace fragment came from a lot of research-grade plasmid material supplied by a third-party manufacturer for use in the gene therapy material. Plasmids are engineered segments of DNA essential to the delivery of gene therapy. That tested lot has not been used in patients. A prior lot of gene therapy material also used the same lot of third-party plasmid, and that lot was used in dosing four patients. Some important facts about prior dosing. First, there have been no safety issues observed with this material. Second, biopsies from the patients have been tested, and the fragment is not present. This is consistent with preliminary testing in animal models that indicate that the fragment is not capable of protein expression, shows no toxicity, and very quickly is cleared by the body if present.
As it relates to the clinical hold itself and upcoming dosing, it is important to understand that we know the source of the problem, we have been given clear guidance on how to address the issue, and we have a plan for our clinical development program to remain on track. Nationwide Children's and Sarepta are rapidly developing a corrective action plan for submission to the FDA that will include GMP-sourced plasmid for the program that should avoid issues such as this in the future. Subject to the FDA's acceptance of our corrective action plan, Sarepta does not anticipate any material delay in dosing patients as originally planned by year-end 2018. As I have said, this hold results from the presence of a trace amount of DNA fragment in research-grade third-party supplied plasmid. It has been standard practice for academic institutions to use research-grade plasmid material for early-stage clinical programs.
Indeed, this grade of material was previously cleared for use. However, as reflected in recent FDA guidance on gene therapy and rare disease, the FDA has encouraged sponsors to design first in-patient studies as potential pivotal trials, and it is not at all surprising that as gene therapy evolves, manufacturing standards and expectations will also evolve. As a leader in gene therapy, Nationwide Children's and Sarepta both embrace these evolving standards and will together move rapidly to implement them and to satisfy the FDA with the target of coming off clinical hold in time to commence dosing later this year.
Separately, as the agency in its recent guidance has shown a willingness to consider studies as pivotal trials under appropriate circumstances, we will request a meeting with the FDA to discuss the micro-dystrophin program and to gain alignment on the requirements for the Phase I/IIb clinical trial to serve as an actual registration study. With that, I would open it up the call for Q&A. Operator?
Of course. Ladies and gentlemen, if you have a question at this time, please press star followed by the one key on your touch-tone telephone. If your question has been answered or you wish to be removed from the queue, press pound. Once again, for questions, that's star and one. Our first question comes from the line of Ross Weinreb from Goldman Sachs. Sir, your line is now open
Hi, it's actually Salveen Richter on the line from Goldman. Just wanted to follow up with regard to your action plan. When will you actually provide that plan to the FDA? How does that impact at all your meeting that's planned to discuss the pivotal Cohort C study? I have a follow-up post that.
Yeah. The short answer is we're moving very rapidly to satisfy the complete response to the clinical hold. While I don't want to promise exact dates, we believe that we can get this done. We're well on our way within the next month.
Great. Then you've talked about, you've treated four patients so far. With patient five, is it your plan, notwithstanding whatever's happening here, that you were going to treat that patient in the Cohort C arm or Cohort C study?
Yeah. One of the things we had talked about separately from this issue is the fact that as we've talked through, both with Jerry Mendell as well as Dr. Rodino-Klapac, I think the proof of concept on gene therapy in Cohort B has been established. We were already contemplating how we take Cohort C, this 24-patient study, and we look to that as our focus. Of course, subsequent to the FDA's recent guidance, it became obvious to us that there is a real opportunity to dialogue with the FDA about how to ensure that we've designed a trial that could potentially, if successful, act as an actual registration trial for our gene therapy program.
Our goal, independent of this issue, was before dosing a Cohort C or another patient, was actually to call for a meeting with the FDA and have a meeting where we get aligned with the division on the appropriate approach to rapidly bring this therapy to the community. Our goal here then is first and foremost, to provide a full response, a complete response to the division so that we can get off clinical hold. We will separately call for a meeting with the FDA to discuss the clinical program. Our goal is to get aligned rapidly, literally in the next few months, on the pathway forward to bring this therapy to the community. Hopefully, we're in a position that before the end of this year, we're actually dosing patients on what, if successful, could be a pivotal trial for our gene therapy program.
Great, maybe just one last question. Do you know where you're sourcing the GMP-sourced plasmid from? Or which company you're going to get it provided from?
We have good ideas of where we're going to go for our third-party supplier, we're very confident that there's very good GMP-sourced plasmid available to us, we're already in that process now.
Perfect. Thank you.
Our next question comes to the line of Joseph Schwartz from Leerink Partners. Your line's now open.
Hi, guys. Thanks for the call. This is actually [Degan] dialing in for Joe. I guess just looking at the cohort, I just want to clarify, in terms of the phase I/IIa study, the Cohort B, you mentioned that the four patients received a lot that was prior to, but that didn't contain any material there. With regards to the overall Cohort B plan, which was supposed to enroll six patients, I just wanted to get some clarity here. Did you finish dosing all six patients? Were any of them subjected to the lot or from the material that this supplier provided, whether contaminated or not contaminated? I have a follow-up.
Yeah. Let me answer a couple. First, we've dosed four of the patients in Cohort B. We haven't dosed all of the six patients, and we are going to, frankly, independent even of this, focus on the concept of a registration trial. As you know, we have a Cohort C, which we are going to adapt with the alignment from the FDA after meeting into what might very well be a pivotal trial if we get agreement with the FDA, and that's our focus right now. The second thing I do want to make clear about, if I haven't been already clear, is that the clinical hold that we received was the result of release testing for a lot that has not been used on patients. That lot contained a plasmid lot that was research grade, and that plasmid lot contained this DNA fragment.
The prior lot also contained the same lot of that plasmid. The potential for a plasmid fragment to have existed with those patients exists, but their biopsies have been looked at, and there is no plasmid found in any of the biopsies. That is not surprising, even if that prior lot had this research-grade plasmid that had a DNA fragment in it, because we've done animal testing. With respect to our animal testing, even if an animal has a DNA fragment in it, that DNA fragment doesn't express, is not toxic, and very rapidly is cleared from the body. It's not at all surprising to us that these patients wouldn't be expressing any kind of protein or even have the presence of the DNA fragment after a very short period of time, if they ever had it at all.
Great. Thanks for that, Doug. Just to follow up, I guess the first one is, given the ongoing trial phase I/IIa, just wanted to get your take on, in addition to the year-end 2018 goal that you've reiterated of dosing patients, assuming all the meetings go well, can we still expect an update on the phase I/IIa in the fourth quarter? Do you still stand by that at all?
Yeah.
The other question is, with regards to this supplier, are you aware of the supplier providing material not only to you guys, but for any other gene therapy companies out there that's currently running clinical trials?
Well, I would certainly assume that. Obviously, Nationwide uses very reputable suppliers, and I want to be very clear that this was research-grade plasmid. The suppliers that are used by Nationwide and the suppliers that we embrace also have GMP-sourced material as well, we're very confident about the sourcing of GMP-grade material. That will be our goal. We are going to, in connection with the corrective action plan with the FDA, commit along with Nationwide to the use of only GMP-grade plasmid material. As it relates to that, I do want to stress again, so that there's no misunderstanding, that the use of research-grade plasmid in connection with early-phase human clinical trials in gene therapy is not at all uncommon for academic institutions. In fact, the very research-grade level of plasmid here has been cleared by the division in the past.
This is an evolving standard, and we are going to embrace that evolution as well as Nationwide is going to embrace that evolution.
Next question.
Our next question comes from the line of Matthew Harrison from Morgan Stanley. Your line is now open.
Hello, this is David Lebowitz in for Matthew. What's the GMPs plasmid, and what types of changes, I guess, are going to have to occur in the manufacturing process?
Obviously we're in the process of completing the CAPA, the corrective action plan and preventative action. Obviously I don't want to oversimplify this, but as it relates to the use of GMP plasmid versus research-grade, it is literally as simple as ordering GMP-sourced plasmid as opposed to research-grade plasmid. That is the fundamental change that will occur in the manufacturing, and obviously that is very executable. The third-party suppliers that we use for plasmid material have ample ability to make GMP-sourced material as well as to make research-grade. Those differences include things like isolated suites, single-use material, cleaning, validation, and the like, and we're going to focus on that going forward.
As far as the FDA meeting, is there any speculation on the timing of that and when we might get an update as to the outcome?
I think I'm going to be over-promising if I give a date. We're going to get ourselves well-prepared. First, we're going to take this in order. First, we're going to ensure that we give the division a complete response and a thoughtful, complete response on the clinical hold. We resolve that to the satisfaction of the division, answer any additional questions they have with the goal of rapidly getting off the clinical hold as soon as is reasonably possible in cooperation with the division. Then we're going to focus on the meeting with the FDA. It is unquestionably our goal to have that by the fall of this year so that we can have a really good line of sight in what the pivotal trial would look like.
Assuming that the division agrees with us, we should be focusing on a pivotal trial, and then the goal is to dose by the end of this year. If we do this right, and I don't want to overpromise, but if we do this right, then we can resolve this clinical hold, improve the program, and we haven't actually slowed down the program at the same time. There's a lot of work to do in all of that, and we're obviously working on it even as we speak.
Thanks for taking my questions.
Thank you.
Our next question comes from the line of Ritu Baral from Cowen. Your line is now open.
Hey, guys. Thanks for taking the question. Doug, was there anything in particular about this DNA fragment, its sequence that was of particular concern? I know you mentioned it was rapidly cleared, it wasn't incorporated. Was there anything in the sequence that was particularly problematic, or was it a zero-tolerance approach by FDA?
No, there's nothing particular in the sequence that was itself troubling. It was just the very existence of having a DNA fragment. Just so we're very clear, Nationwide's routine release process identified the fragment, and then we communicated to the agency about that, and then subsequently received the letter yesterday. There was nothing in particular about the fragment other than its existence.
Was it part of the plasmid, or was it completely foreign?
It was foreign to the plasmid that we were using.
Okay. Are you still considering the same third-party supplier for your GMP product, or are you going to go to a different supplier?
Yes. It's certainly very possible that we would use the same supplier. There's nothing about the supplier that itself is particularly concerning. The good news in all of this, so we're clear, is that we can identify the source of the issue. The source of the issue is the use of a research-grade plasmid versus the use of a GMP-sourced plasmid. The solution, and I don't want to oversimplify this. There's more we need to do. We obviously need to audit to ensure the GMP-sourced material is in fact GMP-sourced and the like. The fundamental solve to the issue is ensuring that we're using GMP-sourced material, and I think we're working with very reputable suppliers that can assure that for us.
On the research product versus the GMP product, what characterizes that production? Is it additional purification steps, additional quantification of impurities, et cetera?
I'm going to get rapidly well over my skis if I get into too much detail. In the broadest of strokes, GMP source material relies upon segregated suites, cleaning validation, single-use equipment to ensure that there's extraordinarily low risk of any potential independent DNA fragment in the material.
Got it. Thanks for taking all the questions.
Thank you so much.
Our next question comes from the line of Tim Lugo from William Blair. Your line is now open.
Hi, thanks for taking the questions, guys. Myles Minter for Tim Lugo. Just a question about the plans for Cohort C, the potential pivotal one to begin at the end of the year. Are all of those patients going to be dosed from the same lot of AAVrh74 micro-dystrophin, or is it too much to do that with? If it is different, what sort of inter-lot quality control do you need to show the FDA for them to get comfortable with that?
Hold on. Let me go to Louise. Yeah, that's fair. I'm going to give a half an answer if I do, so why don't I send that over to Dr. Louise Rodino-Klapac, who can frame that answer for you.
Sure. The intention is to use one lot to dose all patients. In the event that we would have to use more than one lot, we have bridging studies in place, potency studies, to ensure adequate comparability between lots.
Okay. Thanks. Just a follow-up on that. Your end product, I understand that's going to come from your collaboration with Brammer Bio. Before a potential approval, we even get in those talks, do they want to see data from that particular lot being made in collaboration before they make a decision?
Yes. Obviously, this is all going to depend upon the conversations we have with the FDA and our meeting with the FDA. It is certainly our working assumption right now that the agency would expect us to, along this pathway, have a cohort where patients are dosed on and bridged over to the commercial supply, and that's going to be the proposal that we're going to make.
Okay, beautiful. Thanks very much for the question.
Thank you very much.
Our next question comes from the line of Yun Zhong from Janney. Your line is now open.
Hi. Thank you very much for taking the question. Just want to confirm, if you don't mind. Even if the clinical hold were to be resolved immediately, just to assume, but you're still not going to dose any additional patient until you have got clarity from the FDA on the potential registration trial. Is that correct?
That is correct. Our plan, independent of the clinical hold issue, is to meet with the FDA and talk through the FDA and get the FDA's alignment around the program as a potential pivotal program. The reason for that, the evolution on that thinking comes from the recent guidance from the FDA. As you may recall, a couple of weeks ago, the agency issued a collection of draft guidances on gene therapy, and one of the guidances is on the use of gene therapy in rare disease. It was really very helpful to us. One of the things that is in that guidance, and I'm paraphrasing essentially, is that at first, of course, with respect to gene therapy, one shouldn't be thinking about dosing in healthy volunteers, and we would have expected that anyways.
One ought to be looking to these gene therapies, even from the first study, as potentially registration trials and design them in that way. That's an extraordinary opportunity, very innovative and forward-thinking, but it also tells us that rather than guessing, we ought to come up with our best program and then share that with the division and work with the division in the hope that they agree with us that we have the right pathway. Independent of the hold, we would be calling for a meeting with the FDA and talking through this with the FDA before we dosed.
Okay. Are there any additional details that you are able to share in terms of what you would like to propose to the FDA when you meet with the agency?
We're working on it. We've already shared what we have right now, which is what we call Cohort C. The base case for us is a 24-patient study, 12 and 12, one-to-one randomization between placebo and active. That's the sort of a going-in proposition. We're continuing to refine our thinking around that in a number of regards, one of which is to ensure that we have a pathway that is robust but also as rapid as possible to the extent that there's positive data both from a safety and efficacy perspective. Also really thinking through that program, not only in the United States but beyond the United States. We've got a lot of thinking to do. Our goal is not to serve the community in the United States, but to serve the United States and the rest of the world.
There are as many as 70,000 patients around the world who have Duchenne muscular dystrophy. To also ensure that we've built a program that serves the broadest population of patients with Duchenne muscular dystrophy, and that's age, that's weight, that's exon amendability, and the like. We've got some thinking to do as we plan for our meeting with the agency.
Any potential that the study doesn't have to be a placebo-controlled study?
We will certainly discuss those issues with the agency. We've had some discussions internally. The question is what the right answer is, not just in the U.S. but around the world. We've got some things to work through. Our base case right now is a placebo-controlled trial, one-to-one placebo to active.
Okay, great. Thank you.
Thank you so much.
Our next question comes from the line of Hartaj Singh from Oppenheimer. Your line is now open.
Great. Thank you. Just a quick question. As we await sort of the updates, Doug, from the meetings. The February document from FDA guidance on DMD had also asked about, for example, immune response with AAV, a special interest, and to dystrophin and to other muscle components. Then they'd also asked for specific testing of mutations. Just any thoughts. From the patients that you have now, do you think that you'd be able to address a lot of these issues by the time you get to that fall meeting with the FDA in order to move forward with the pivotal? Again, thank you.
I think broadly speaking, yes. Broadly speaking, we'll have a lot of insight from the first four patients that we've dosed.
Great. Thanks, Doug.
Thank you so much, Hartaj.
Our next question comes from the line of Anupam Rama from JPMorgan. Your line is now open.
Hey, guys. Thanks so much for taking the question. A lot of our questions have been answered here, but maybe, Doug, a little bit more clarity on, I know you guys are working rapidly to file your submission to the FDA. Has the FDA given you any guidance on their review timelines? Is there any precedent that we can be thinking about here? Thanks so much.
Yeah. Thank you. Once we have submitted a complete response to their clinical hold, their review time is 30 days by regulation. The first assumption is we will rapidly complete it. We want to be robust and complete, and they've given us very good, direct guidance on the things we need to respond to. We're not operating in the dark here. We've got a good roadmap for getting that done, and we're doing a lot of work. We've already kicked it off, and we've got a good roadmap for that. Then once we have a complete response, and they agree with us we have a complete response, they'll have a 30-day turnaround time.
Great. Thanks for taking our question.
Thank you.
Our next question comes from the line of Salveen Richter from Instinet. Your line is now open.
Hi. Thanks for taking the question. Doug, I was wondering, what's sort of the rate limiting step to using commercially sourced product in the phase III, is there a possibility here to proceed in that trial with commercially sourced products rather than that from Nationwide? Thank you.
Obviously, there's a timing and a bridging process. We've got to do a number of steps to bridge from a clinical supply to commercial supply. We're deep in the process of that right now, but first, there's obviously the process of tech transferring over to our suppliers. As you know, we have a relationship with Brammer Bio that we're very excited about. Then we've got to adapt the manufacturing process that is sufficient to be commercially scalable. We are being very thoughtful. We've certainly learned from others before us that have been looking at this, we're not going to make enormous changes. We're trying to hew as close as possible to many of the processes that are already existent at Nationwide so that we don't have enormous risks on the bridging side of things.
From there, we've got to scale up, we're going to have to make commercial products. I think the most reasonable approach that gets us moving as fast as possible is to use clinical supply from Nationwide Children's Hospital as we planned, to complete our process for commercial supply, to complete a bridge. I think that is the best chance of getting to the community as fast as possible and as possible.
Okay. When you think about the number of patients you'll need to dose and the potential for supply from Nationwide, is their capacity sufficient to be able to enable the number of patients you envision in the pivotal study, or will that need to be supplemented by a product from a commercial supplier? Thank you.
Yeah. Thank you. The answer is, of course, subject to release testing and the like. I don't want to promise in advance. The short answer is yes, we can comfortably supply what we believe to be a pivotal study, if the FDA agrees with us, from our Nationwide Children's Hospital relationship and the slots we have at their GMP facility. As you know, if we do end up with a 24-patient study, and that is all subject to further discussions with the FDA, we would have 12 patients on placebo, 12 patients on active. What we would envision is that the crossover at one year, all of the placebo patients will, of course, receive active therapy, and it's very likely by then that would be commercial supply.
Okay. Then just with respect to the supply from Nationwide, is it possible that ability to dose patients in the phase III would be rate limiting and I guess subsequent patient dosing? For instance, dose the first 2 with available supply and then proceed following capacity constraints to dose the following 2, et cetera. Is that going to be a rate limiting step? Thank you. That's my last question.
I really apologize. We cut out here, I couldn't quite understand the question. I know it's something about the staging of dosing, I wasn't able to get that. Apologies for that.
Yeah. My question, Doug, was is there a capacity constraint from the Nationwide supply that could slow dosing of patients in your phase III trial?
Oh. No, we're doing a lot of-.
It's the first two. Yeah.
Yeah.
You have to
So the sh-
wait for availability of product. Yeah, that kind of thing.
Yeah, no. We're doing a lot of good long-range planning with Nationwide Children's Hospital to put us in a position where that isn't the case. There's a lot of planning that has to go into it. The Nationwide Children's Hospital GMP facility requires that we get slots, and we have slots and the like, and we do a lot of work in that. We've planned it out so that assuming that things go the right way, assuming that the release testing works the right way, et cetera, that we shouldn't have a problem where we're dosing a couple and then waiting, and then dosing a couple. We're going to go to the agency with a view on how the rapidity of dosing and the periodicity of dosing across the two cohorts.
If we get what we would like, and the manufacturing works the right way and the release testing works the right way, then we should be able to dose at that level, and we won't have to sort of start and stop.
Okay, great. Thank you.
Thank you very much.
Our next question comes from the line of Jonathan Wolleben from JMP Securities. Your line is now open.
Hi there. Just a separate question. I know I've been bugging you about this, but I'm wondering if you could give us any visibility on when the next presentation will be of the patients that have already been dosed.
Oh, yes. I apologize. That question was asked earlier, and I failed to respond to it. First, I want to be very clear. Any additional data from the patients that have been dosed, both the fourth patient, whose biopsy has not been reviewed yet, and additional data on the three patients that were previously discussed at R&D Day, will be at a scientific meeting. Jerry Mendell will choose the appropriate meeting. I could certainly envision an update before the end of the year, but really, I think Jerry Mendell is going to be the one making the decision about the appropriate meeting. It's certainly very possible that an update could occur before the end of the year.
Thanks a lot.
Thank you so much.
Our next question comes from the line of Tim Chiang from BTIG. Your line is now open.
All right. Thanks. Doug, does the clinical hold impact the timing of when you might start Cohort A in the open label trial? I know that was something.
you had talked about at the R&D Day.
At the R&D Day. We had repurposed. By the R&D Day, we had actually repurposed Cohort A.
Just for those on the line, historically, there was a Cohort A and a Cohort B of different ages, four to seven, then younger children, I think three months to three. Actually, we had already, as we were attempting to just really focus on the concept of registration trial, we repurposed the Cohort A, created a Cohort C of 24 patients. We were already focusing on that area. Then with the guidance, our goal is to get to the agency, really ensure that we have a robust plan that the agency is aligned with as a potential registration trial, then dose there. Actually, we've already, by the R&D Day, had evolved away from the idea of dosing a Cohort A.
Okay. Basically, you're focusing on the DMD patients aged between four and seven years of age. Is that right?
Yeah. The concept is, Dr. Mendell did a really nice job of explaining this at the PPMD conference in Scottsdale. The goal, of course, is for the main study, we've got to pick a group that we can actually have a good chance of seeing intragroup differences for. We can't pick all ages because this is a degenerative disease, and of course, different stages have different declines, different areas. The goal in the main study is four to seven. At least that's what we're currently anticipating, subject to discussions with the FDA. On the path to approval, we will dose older patients, we will dose younger patients, and there are some exons that have been excluded, some of the earlier exons that have been excluded in the main study. Out of abundance of caution, we will dose some of those exons as well.
All with the goal that by the time we get to the approval point, we have not only a robust study that's been intelligently designed to have the greatest chance of not getting a false negative because we've broadened the population of the main study too much, but with a broad label that allows us, to the fullest extent possible, subject to the science, of course, to fully serve the community.
Okay, great. Thanks, Doug.
Thank you so much.
Our next question comes from the line of Gena Wang from Barclays. Your line is now open.
Thank you for taking my questions. Maybe I just follow up. First question regarding the Cohort C potential registration trial. Any thoughts on approvable endpoint? Doug, do you think biomarker data will be sufficient, or additional functional data will be required as primary endpoint?
That, it's a question that we are doing a lot of analysis on right now. I think more than anything, that's gonna be a discussion that we're gonna have to have with the FDA before we know. There are a lot of really interesting possibilities, and I think it's going to come down to talking to the FDA. We have biomarkers. We have expression levels in numbers of different ways. We have really interesting biomarkers, as I think everyone knows. It was at R&D Day, we had really interesting preliminary data on CK levels. Of course, we're going to be looking at a panoply of functional data as well. We really need to talk through the FDA, what they would expect. We also need to do something else.
We need to make sure that we're thoughtful, again, beyond the FDA, out to Europe, and to South America, and to Asia, and Africa, and Middle East, and the like, and really be thoughtful about a program that's sufficiently robust that it's fit for purpose around the world. While at the same time, not overburdening ourselves in a way that slows the program down because, as we all know, the community is waiting.
Okay. Another question is regarding the manufacturing. Again, just wondering how many patients you can dose with one lot of a GMP production from Nationwide Children's Hospital.
I'm going to turn that over to Dr. Rodino-Klapac, who I'm sure can answer that more robustly than I can answer it.
We have planned the GMP production to be able to service the number of patients in the planned Cohort C trial. It's expandable to be able to meet those demands.
Just wanted to make sure I understand correct. One lot of production, is that enough for six patients or four patients?
It's enough for the Cohort C trial as planned.
Okay, one lot is enough for 24 patients.
Well, it wouldn't be 24, just so we're clear, because remember.
12 patients. Yeah. Okay.
Placebo patients, yeah.
Okay, basically one lot is sufficient for 12 patients.
That's correct.
Okay. Doc, if you can provide some update regarding the Brammer Bio. Are you planning, I would assume at some stage you will use the commercial product with the collaboration with Brammer Bio, right? Just wondering.
Yeah
at what stage you will start to use their product and then do the comparison in terms of the dosing commercial versus GMP grade, even though it's different commercial and clinical grade.
Excellent. I can't give a precise date. I can tell you we're working diligently with Brammer on the tech transfer and the build-up. The study will commence, and the initial patients will all be dosed on clinical supply from Nationwide Children's Hospital. We will be able to dose with commercial supply next year. There's an opportunity. As I've mentioned, there will be, all subject to additional discussions with the FDA, a number of different potential cohorts that we're going to have along the way to the approval of the therapy, and we can dose one of those cohorts with commercial products. Certainly, we have at one year, we'll have a crossover where all of the placebo patients will have the opportunity to get on gene therapy and off of placebo, and the commercial supply will be available for that.
Our next question comes from the line of Joseph Schwartz from Leerink Partners. Your line is now open.
Hey, Doc. Thanks for taking the follow-up. Just switching gears a little bit. The other program, limb-girdle. The first program is scheduled to initiate sometime this quarter. Just wondering if today's announcement has any impact to that program at all, and if that will be a wait-and-see approach or if that's on cruise control mode at this point. Thanks.
These are early days. I don't want to overstate things. I can tell you the clinical hold is very specific to our program with micro-dystrophin, and we don't have any lots associated with the limb-girdle program that have been out of spec in any way.
Awesome. Thanks for taking the follow-up.
Thank you so much.
I am closing now for the questions. I will now like to turn the call back to Doug Ingram for any closing remarks.
Well, thank you again, all of you, for joining us on the call this evening. We look forward to your participation in our second quarter earnings call scheduled for Wednesday, August 8, 2018, at 4:30 P.M. Eastern Time. Have a good evening.
Ladies and gentlemen, thank you for your participation in today's conference. This concludes today's program, you may all disconnect. Everyone, have a great day.