Good day, ladies and gentlemen, and welcome to the Sarepta Update Call. At this time, all participants are in a listen only mode. Later, we will conduct a Q and A session and instructions will follow at that time. As a reminder, this conference call is being recorded. I would now like to turn the conference over to Mr.
Ian Estefan, 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 Sandy Mahatnay, our Chief Financial Officer Doctor. Gilmore O'Neill, our Chief Medical Officer and Doctor.
Luis Rodino Klapac, our Vice President of Gene Therapy. I'd like to note that during this call, we will 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 could 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. And with that, I'd like to turn the call over to Doug to provide an update. Doug?
Thank you. Good afternoon, everyone, and 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 1a2a Duchenne muscular dystrophy microdystrophin trial has been placed on clinical hold. My goal in this call is to place that clinical hold in context and provide ours in 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 for patients.
So some important facts about prior dosing. First, there have been no safety issues observed with this material. 2nd, 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 plasmids 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 disease, the FDA has encouraged sponsors to design 1st 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 IIIa clinical trial to serve as an actual registration study.
And with that, I would open it up the call for Q and A. So operator?
And 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? And how does that impact at all your meeting that's planned to discuss the pivotal Cohort C study? And I have a follow-up post that.
Yes. So the short answer is we're moving very rapidly to satisfy the complete response to the clinical hold. Well, 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.
And then you've talked about you've treated 4 patients so far with patient 5, 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?
Yes. So one of the things we had talked about separately from this issue is the fact that we've as we've talked through both with Doctor. Mendel as well as Doctor. Rodino Klapac, I think the proof of concept on gene therapy in Cohort B has been established. So we were already contemplating how we take Cohort C, this 24 patient study, and we look to that as our focus.
And then of course subsequent to the FDA's recent guidance, it became obvious to us 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. So 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. So our goal here then is 1st and foremost to provide a full response, a complete response to the division so that we can get off clinical hold. Then we will separately call for a meeting with the FDA to discuss the clinical program. Then our goal is to get aligned rapidly literally in the next few months on the pathway forward to bring this therapy to the community.
And then 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 a gene therapy program.
Great. And maybe just one last question. Do you know where you're sourcing the GMP source 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 3rd party supplier and we're very confident that there is very good GMP sourced plasmid available to us and we're already in that process now.
Perfect. Thank you.
And our next question comes from the line of Joseph Schwartz from New York Partners. Your line is
now open. Hi, guys. Thanks for the call. This is actually Dae Gon dialing in for Joe. So I guess just looking at the cohort, I just want to clarify, in terms of the Phase IIIa study, the Cohort B, you mentioned that the 4 patients received a lot that was prior to, but that didn't contain any material there.
So with regards to the overall Cohort B plan, which was supposed to enroll 6 patients, I just wanted to get some clarity Did you finish dosing all 6 patients? And were any of them subjected to the lot or from the material that this supplier provided whether contaminated or not contaminated? And I have a follow-up.
Yes. So let me answer a couple of them. So first, we dosed 4 of the patients in Cohort B. So we haven't dosed all of the 6 patients. And we are going to, frankly, independent even of this, focus on the concept of 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.
So the potential for a plasmid fragment to have existed with those patients exists, but they've been their biopsies have been looked at and there is no plasmid found in any of the biopsies. And 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. And 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. So 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. So just two follow-up. I guess the first one is given the ongoing, the trial Phase 1, 2a, 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 IIIa in the Q4?
Do you still stand by that at all? And then the other question is with regards to this supplier, 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. We 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. And so we're very confident about the sourcing of GMP grade material.
And that will be our goal. So we are going to, in connection with FDA, commit along with nationwide to the use of only GMP grade plasma material. But 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 and gene therapy is not at all uncommon for academic institutions. And in fact, the very research grade level of plasmid here has been cleared by the division in the past. So 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?
And our next question comes from the line of Matthew Harrison from Morgan Stanley. Your line is open.
Hello, this is Dave Leibowitz in for Matthew. What's the GMPS plasmid and how what types of changes I guess are going to have to occur in the manufacturing process?
So obviously, we're in the process of completing the kappa, the corrective action plan and preventative action. So 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, very executable. And the suppliers the 3rd price suppliers that we use for plasmid material have ample ability to make GMP source material as well as to make research grade. And 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.
And 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 get ourselves we're going to take this in order. 1st, we're going to ensure that we give the division a complete response and a thoughtful complete response on the clinical hold. So 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.
And 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. So if we do this right and there's a bit I don't want to over promise, 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, but there's a lot of work to do in all of that. We're obviously working on it even as we speak.
Thanks for taking my questions.
Thank you.
And our next question comes from the line of Ritabh Bora 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. But was there anything in the sequence that was particularly problematic or was it a zero tolerance approach by FDA?
No, there was 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. So there was nothing in particular about the fragment than its existence.
Was it part of the plasmid or was it completely foreign?
It was foreign to the plasmid that we're using.
Okay. And are you still considering this same third party supplier for your GMP product or are you going to go to a different supplier?
Yes. Yes. It's certainly very possible that we would use the same supplier. There's nothing about the supplier that itself is particularly concerning. So that's 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 source plasmid. And so 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 source material is in fact GMP sourced and the like. But the fundamental solve to the issue is ensuring that we're using GMP GMP source material. And I think we're working with very reputable suppliers that can ensure that for us.
On the research product versus the GMP product, are there what characterizes that production? Is it additional purification steps, additional quantification of impurities, etcetera?
I'm going to get rapidly well over my skis if I get into too much detail. But 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 open.
Hi, thanks for taking the question. Scott, it's Miles Muncher on for Tim Lugo. Just a question about the plans for Cohort 2, the potential pivotal ones begin at the end of the year. All of those patients going to be dosed from the same lot of rh74 micro dystrophin? Or is it too much to do that with?
And if it is different, what sort of interlot quality control do you need to show the FDA for them to get comfortable with that?
Yes, that's fair. Let me I'm going to give half an answer if I do. So why don't I send it over to Doctor. Louise Rodino Klapac who can frame that answer for
it. 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. And just a follow-up on that. Your end product, I understand that's going to come from your collaboration with Brammer Bio. So 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. So 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 questions.
Thank you very much.
Our next question comes from the line of Yoon Song from Janney. Your line is now open.
Hi. Thank you very much for taking the question. So, just want to confirm, you don't mind, even if the clinical hope were to be resolved immediately, just 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. That is correct. We are 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. And the reason for that, the evolution on that thinking comes from the recent guidance from the FDA. So as you may recall, 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.
And it was really very helpful to us. And one of the things that is in that guidance and I'm paraphrasing essentially is that, that first of course with respect to gene therapy, one shouldn't be about dosing in healthy volunteers and we would have expected that anyways. Then one ought to be looking to these gene therapies even from the first study as potentially registration trials and design them in that way. And 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 the hope that they agree with us that we have the right pathway.
So independent of the whole, we would be calling for a meeting with the FDA and talking through this with the FDA before we dose.
Okay. And so 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. So the base case for us is a 24 patient study, 1212, 1 to 1 randomization between placebo and active. And that's the sort of our going in proposition, but 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. And 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 the chain muscular dystrophy. And to also ensure that we've built a program that serves the broadest population of patients with Duchenne muscular dystrophy and that goes that's age, that's weight, that's exon amendability and the like. So 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.
So we've got a lot of we've got some things to work through. But our base case right now is a placebo controlled trial 1 to 1 placebo to active.
Okay, great. Thank you.
Thank you so much.
And our next question comes from the line of Hartaj Singh from Oppenheimer. Your line is now open.
Great. Thank you. Just quick question. As we wait sort of the updates, Doug, from the meetings, the February document from FDA and the guidance on DMD had also asked about, for example, immune responses, A's of special interest and dystrophin and to other muscle components. And then they'd also ask 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 mean, 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, Tim.
Thank you so much, Rich.
And our next question comes from the line of Amupam 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, but has the FDA given you any guidance on their review timelines? Or is there any precedent that we can be thinking about here? Thanks so much.
Yes. Thank you. So once we have submitted a full complete response to their clinical hold, then their review time is 30 days by regulation. So 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.
So we're not operating in the good road map for that. And then once we have a good roadmap for that. And 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.
And our next question comes from the line of Christopher Marai from Instinet.
Hi, thanks for taking the question. Doug, I was wondering what sort of the rate limiting step to using commercially sourced product in the Phase 3? And is there a possibility here to proceed in that trial with commercially sourced product rather than that from nationwide?
Obviously, there's a timing and a bridging process. So we've got to do a number of steps to bridge from clinical supply to commercial supply. We're in that we're deep in the process of that right now. But there's first, there's obviously the process of tech transferring over to our suppliers. As you know, we have a relationship with Brammer Biosciences that we're very excited about.
Then we've got to adapt the process, 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, so that we're not going to make enormous changes. We're trying to hue as close as possible to many of the processes that are already exist into Nationwide so that we don't have enormous risks on the bridging side of things. And then from there, we've got to scale up and then we're going to have to make commercial products.
So 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 and then to complete our process for commercial supply and then to complete a bridge. I think that is the best chance of getting to the community as fast as possible and as robust as possible.
Okay. And then when you think about the number of patients you'll need to dose and the potential for supply from nationwide, is there 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 products from a commercial buyer? Thank you.
Yes. Thank you. So the answer is, of course, subject to release supply, our what we believe to be a pivotal supply our what we believe to be a pivotal study, if the FDA agrees with us from our Nationwide Children's Hospital relationship in there and the slots we have with at their GMP facility. And then at the as you know, if we do end up with a 24 patient study, and that is all subject to further discussions with the FDA, then we would have 12 patients on placebo, 12 patients on active. What we would envision is that the crossover at 1 year, all of the placebo patients will of course
Okay. And then just with respect to the supply from Nationwide, is it possible that ability to patients in the Phase 3 would be rate limiting and I guess subsequent patient dosing? So for instance, the first two was available to buy and then proceed following capacity constraints to those following 2, etcetera, etcetera. Is that going to be a great one? Thank you.
That's my last question.
I really apologize. We cut out here. So I couldn't quite understand the question. I know it's something about the staging of dosing, but I wasn't able to get apologies for that.
Yes. My question, Doug, was, is there a capacity constraint from a nationwide supply that could slow dosing of patients in your Phase III trial?
No, we're doing a lot of So it's
referred to.
And then you
have to wait for availability of products. Yes.
I know 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. So there's a lot of plan 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. But we planned it out so that assuming that things go the right way, assuming that the release testing works the right way, etcetera, that we shouldn't have a problem where we're dosing a couple and then waiting and then dosing a couple. So we're going to go to the agency with a view on how the rapidity of dosing and the periodicity of dosing across the 2 cohorts.
And if we get what we would like, then we'll be able to 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 Lisa Velko 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?
Yes. And I apologize. That question was asked earlier and I failed to respond to it. So first, I want to be very clear. This is we're going to the net any additional data from the patients that have been dosed, both the 4th patient, which whose biopsy has not been reviewed yet And additional data on the 3 patients that were previously discussed at R and D Day will be at a scientific meeting and Doctor.
Mendell will choose the appropriate meeting. I couldn't certainly envision an update before the end of the year, but really we I think Doctor. Mendel is going to be the one making the decision about the appropriate meeting, but 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.
Hi, 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 that you talked about at the R and D day.
So we had repurposed. So we had already by the R and D Day, we had actually repurposed Cohort A. So just for those on the line, what historically there was a Cohort A and a Cohort B of different ages, 4 to 7 and then younger children, I think 3 months to 4 to 3. But actually, we had already as we were attempting to really focus on the concept of registration trial, we repurposed the Cohort A, created a Cohort C of 24 patients. So we are already focusing on that area.
And then with the guidance, our goal is to get to the agency, really ensure that we have a robust plan at the agency is aligned with as a potential registration trial and then dose there. So actually we're already we've already by the R and D, they had evolved away from the idea of dosing a Cohort A.
Okay. So basically you're focusing on the DMD patients aged between 4 7 years of age, is that right?
Yes. So and the concept there is and Doctor. Mendel did a really nice job of explaining this at the PPMD Conference in Scottsdale. So the goal, of course, is we for the main study, we've got to pick a group that we can actually have a good chance of seeing intra group differences for. And so we can't pick a broad enough we can't pick all ages because this is a degenerative disease and of course different stages have different declines in different areas.
So the goal in the main study is 4 to 7, at least that's what we're currently anticipating subject to discussions with the FDA. We will 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 and 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 fully 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 Gina 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? And Doug, do you think a biomarker data will be sufficient or additional functional data will be required as primary endpoint?
So that it's a question that we're doing a lot of analysis on right now. And I think more than anything, that's going to be a discussion that we're going to 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 talk to the FDA. We have biomarkers, we have expression levels, we have expression levels in numbers in different ways. We have really interesting biomarkers as I think everyone knows it was at R and D Day.
We had really interesting preliminary data on CK levels. And then of course, we're going to be looking at a panoply of functional data as well. And we really need to talk through the FDA what they would expect. And then 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 like and really be thoughtful about a program that's sufficiently robust and 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. And then another question is regarding the manufacturing again. Just wondering how many patients you can dose with 1 lot of GMP production from Nationwide Children's Hospital?
I'm going to turn that over to Doctor. 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.
Sorry, just so one lot, just wanted to make sure I understand correct. So one lot of production, is that enough for 6 patient or 4 patient?
It's enough for the Cohort C trial as planned.
Okay. So one lot is enough for 24 patient?
It wouldn't be 24 just so we're clear because remember
12 patient. Yes. Yes. Yes. Yes.
Yes.
Yes. Yes. Yes. Yes.
Update regarding the Braemobile, are you planning I would assume at some stage you will use the commercial product with the collaboration with Brammer Bio, right? Just wondering like 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. So I can't give a precise date. I can tell you we're working diligently with Brammer on the tech transfer and the buildup. 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.
So 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 product. And certainly we have at 1 year, we'll have a crossover where all of the placebo patients will have the opportunity to get on gene therapy off of placebo and the commercial supply will be available for that.
And our next question comes from line of Joseph Schwartz from Leerink Partners. Your line is now open.
Hey, Doug. Thanks for taking the follow-up. Just switching gears a little bit. So 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.
Well, these are early days, so I don't want to overstate things. I can tell you the clinical hold is very specific to our program with micro dystrophin. We don't have any lot 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.
And I'm currently seeing no further questions. I would 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 Q2 earnings call scheduled for Wednesday, August 8, 2018 at 4:30 pm Eastern Time. Have a good evening.
Ladies and gentlemen, thank you for your participation in today's conference. This concludes today's program and you may all disconnect. Everyone have a great day.