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Sarepta Therapeutics, Inc. (SRPT) Presents at JPMorgan Healthcare Broker Conference Call - (Transcript)

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About: Sarepta Therapeutics, Inc. (SRPT)
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Sarepta Therapeutics, Inc. (NASDAQ:SRPT) JPMorgan Healthcare Conference Call January 13, 2019 12:30 PM ET

Company Participants

Douglas Ingram - President, CEO & Director

Gilmore O'Neill - EVP, R&D and Chief Medical Officer

Louise Rodino-Klapac - SVP, Gene Therapy

Conference Call Participants

Anupam Rama - JPMorgan Chase & Co.

Anupam Rama

Okay. We'll go ahead and get started. Welcome, everyone, to the 2020 JP Morgan Healthcare Conference. My name is Anupam Rama. I'm one of the senior biotech analysts here at JP Morgan. I'm joined by Tessa Romero and Matt Bannon from the team. Our next presenting company is Sarepta, and presenting on behalf of the company is CEO, Doug Ingram. Doug?

Douglas Ingram

Thank you. Thank you, Anupam. And to those in the room and also on the web, thank you for joining Sarepta this morning. Before I begin, let me say that I'll be making some forward-looking statements. Here is a laundry list of the various risks and uncertainties you may want to consider whenever one has the temerity to try to predict the future.

And with that, let's begin. So with the great advances that have occurred in genetic medicine, in 2018, Sarepta envisioned a revolution, and we built a strategy with the goal of being among the most significant leaders in that revolution. And that strategy was based on some fairly distinct pillars, the first of which, of course, is our 2 platforms, RNA and gene therapy. We have a strategy that assumed that with our RNA therapies, we can advance constructs to treat greater and greater percentages of the Duchenne patient population and to use the accelerated approval pathway to do so rapidly. And our gene therapy engine assumed -- gene therapy platform and engine assumed that we can bring constructs through rapidly to treat first prevalent populations and then incident populations, bringing a better life, if we're successful, to thousands upon thousands of patients with rare disease.

It's also a strategy that relies on the concept of building infrastructure. If one is going to be an enduring genetic medicine company, we are going to need infrastructure and in particular, building talent, including our gene therapy center of excellence, and I'll talk more about that today. It's a strategy that's built around this concept of a hybrid manufacturing model, one that assumed that we could build the expertise in the intellectual aspects, process development, analytical development while at the same time, building more gene therapy manufacturing capacity than at least in 2018, existed in all of the industry. And all of this was founded on our mission. It is a mission to use the science of genetic medicine to bring a better and longer life to rare disease patients who, at least, historically, over the long arc of human history, have had little bit degeneration and early death until at least now.

In 2019, we went about executing, bringing our strategy into sharper focus and working to make this revolution real. So let's review the progress that we've made to date and the implications that, that may have in 2020 and beyond. Back in 2018, we already had a very significant pipeline. We had 25 programs, 11 of which we were driving ourselves, but 14 of those programs, we were driving through alliances with the best and brightest in genetic medicine outside of Sarepta. And this was the formation of what we call our incubation strategy, which would allow us to both drive our future pipeline through the best and the brightest, accelerate our expertise, but allow us ourselves to remain razor focused on execution, ensuring that we're meeting our milestones and driving our vision.

As we stand here today, we have what some may think is a shocking 20 -- 42 total programs, 20 of which we're driving ourselves, 6 of which we're driving in gene therapy. But we now have 22 programs with our outside alliances and genetic medicine partners and experts outside of the company. So we've really built out this concept of our incubation model.

We've also been building the tools to ensure that we remain leaders into the future, and we drive the science of genetic medicine. That includes, for instance, the human-derived capsids that we're working on with Dr. Guangping Gao as well as access to the rationally designed capsids that could have better tropism as well as in immuno-abating aspects with StrideBio. And I'm very excited to announce that we're deepening our relationship with Dr. Charlie Gersbach at Duke University and getting even deeper into the research around gene editing as a research platform. In fact, we are going to build, this very year, a gene editing center of excellence in Durham, North Carolina. We've also entered into a relationship with a gene editing pioneer, that's Amy Wagers at Harvard, who has a very specific approach to gene editing with respect to stem cells or satellite cells, and we're excited about that as well.

But we've done more than simply build a pipeline and build tools, we have also been advancing our pipeline through clinical development. Let me begin first with SRP-9001, that is our gene therapy to treat Duchenne muscular dystrophy. Duchenne muscular dystrophy is a very devastating neuromuscular disease that results from a mutation on the gene that couture this structural protein called dystrophin. Dystrophin is a sort of shock absorber in our muscles. It protects our muscles when we move them, so they don't degenerate. Children, mostly boys because it's X-linked recessive, who have Duchenne muscular dystrophy, have no dystrophin. And as a result of having no dystrophin, they will 100% of the time degenerate, and unfortunately, 100% of the time, this disease will kill them usually at a very young age. The goal of 9001 is to address this issue through gene therapy by inserting into the muscles of these children with Duchenne muscular dystrophy, a gene that codes for a highly truncated version, but functional version of dystrophin. And we have a construct that is unique to Sarepta in numerous ways, and I think explains the reason we're seeing the kinds of results that we're already receiving.

Back in 2018, we announced the expression and safety results from our first 4 patient cohort with respect to SRP-9001. The results are here, we were seeing a very impressive and unparalleled expression and the therapy was also well tolerated. In 2019, we came back with the 9-month functional data for SRP-9001, I've just shown it here. Let me make it straightforward. All 4 boys on every single functional endpoint was not degenerating, was not stable, but was actually improving and improving in ways that natural history could not have explained. And based on those results, we are moving mad to advance this therapy. The first thing we're doing is moving on Study-102. Study-102 is our placebo-controlled trial using clinical material from Nationwide Children's Hospital. It's a 40-patient trial. I'm very pleased to announce that we have now dosed all of the patients, in fact, we've dosed 41 patients in that trial. More than that, we're dosing patients on crossover as well. This is a 48-week placebo trial. We have dosed a significant number of patients on crossover. So in whole, we dosed significantly more than 40 patients in this study. Study continues to progress well. It's progressing uninterrupted, and we should have results from that study in very early 2021.

We are also moving very significantly on what we call Study 301. Study 301 is our trial, placebo-controlled as well with commercial supply. As it relates to that, we've already been in dialogue with the agency and taking advice to design that trial. We've made great progress on manufacturing. I'll discuss that in a little bit, and we're on track to start that trial by around the middle of 2020. So things are going quite well there.

So things are going well with our gene therapy for micro-dystrophin, let's move on to limb-girdle. Limb-girdle is an umbrella disease that covers a number of sub diseases that are very similar to Duchenne muscular dystrophy. They are neuromuscular in nature. They are very often the result of the -- a missing structural protein. And they always cause degeneration and far too often, unfortunately, they cause the early demise of patients that have limb-girdle. We have a total of 6 programs in limb-girdle. If we are successful, we could treat and bring a better life to over 70% of adults and children with limb-girdle muscular dystrophy.

Our lead program is SRP-9003. That treats a limb-girdle called 2E. 2E is a disease that results from a lack of a structural protein called beta-sarcoglycan. The construct here is very similar to SRP-9003 for micro-dystrophin. It is the same vector. It is the same promoter. But here, the transgene is different. Importantly, the transgene here codes for the actual unaltered native protein, the very protein the lack of which is causing degeneration and an early death of these patients. So we're very excited to move this therapy forward as fast as possible.

At the beginning of last year, we announced the results, expression and safety, of our 3-patient first cohort in the low-dose version. And as you will see here, even at a dose that was 1/4 of the dose of our micro-dystrophin therapy, we got very good expression results and the therapy was well tolerated. We came back at the end of the year -- I can't go backwards, I'll just stay, you saw that earlier, we came back at the end of the year, we announced the 9-month functional data for our limb-girdle 2E, and the same answer holds there that holds with our micro-dystrophin program. Every child at every single functional endpoint over the 9-month period actually improved in ways that couldn't be explained by natural history.

What do we have to do going forward with our limb-girdle programs? The first thing we're doing is we're dosing one additional cohort of 3 patients with the goal of making a dose decision in early the next quarter or second quarter of this year. We've dosed already 2 of those patients. The third patient will actually be dosed this very week. And so we should be on track to have results by the early second quarter, and we'll have a dose decision in early second quarter of next year.

One thing I should note here is you'll see that both of the children that we've dosed already are on the larger side, 30 kilograms and 40 kilograms. And at least with respect to that 40-kilogram child, one can say that this dose itself is 4x higher than the prior dose and this child is 40 kilograms. This is almost certainly the greatest dose of gene therapy that any patient in gene therapy has received to date and the therapy appears to be well tolerated. So things are going well there.

We have 2 additional things to do with limb-girdle 2E this year. The first is to define our development and regulatory pathway. We have already started that dialogue with the agency, and we have more work to do there. And the second thing is to -- and this is very important, is to finish the assay development and process development work with respect to limb-girdle 2E, so that we're in a position to commence that trial, and we're working on that right now, and we should be complete with that by the fourth quarter of 2020. And of course, the work we're doing with 2E endures to the benefit not only of the 2E program, but for all of our limb-girdle programs, of which we have 6, that could, if successful, treat 70% or more of patients with limb-girdle muscular dystrophy.

Let's move now to MPS IIIA. MPS IIIA, also known as Sanfilippo disease, is a horrible neurological condition, it is also a lysosomal storage disease. It steals from children their cognition, it then steals their movement and 100% of the time, it will steal their life at a very young age. We have access to Lysogene's gene therapy through our relationship with Lysogene. And the goal here is to replace that enzyme that's missing that's causing the disease by direct infusions into the white matter of the brain. We have a 20-patient Phase II/III study ongoing. It's going quite well. We've already dosed 15 of the 20 patients, and our goal is to have all patients enrolled and dosed by the middle of this year. So we're making great progress on our gene therapy engine.

Let's move on to our RNA platform. Our RNA platform is based on this PMO chemistry, which is itself very clever. Our PMO chemistry allows us to literally edit at the pre-messenger and messenger RNA level, to bring the pre-messenger RNA back into frame and to upregulate proteins that would otherwise be missing but necessary as a result of genetic disease. We've made great progress in 2019. In 2016, EXONDYS was approved that treats about 13% of children with Duchenne muscular dystrophy. In August of last year, we had anticipated an approval, but instead received, to our disappointment and surprise, a complete response letter for our VYONDYS, our therapy that could treat another 8% of children. And while that was deeply disappointing, it at least gave us an opportunity to show, both to the agency and to the rest of the world, who we are, and that's an evidence-based company that knows how to execute. And we did just that. We commenced the formal data-driven appeal. We were able to win that appeal, that resulted in the fastest reversal of a CRL in that manner and FDA history. And that means that at the end of 2019, VYONDYS was approved. We were able to launch VYONDYS that very week. And as a result of that, we were able to increase the number of patients with Duchenne muscular dystrophy that might benefit from our therapy by some 60% over EXONDYS alone.

With respect to EXONDYS, we're making great progress. At the beginning of last year, you will recall, our guidance was $365 million to $375 million in revenue. We were able to increase that guidance over the course of the year. You will have seen this morning that we've announced that our fourth quarter revenue stands at over $100 million. That means that for our third full year of -- from launch, we are growing at $381 million or about 26% over the prior year. So we continue to serve the Duchenne muscular dystrophy community with EXONDYS.

I am very pleased now to announce the following: which is, last Friday, we commenced our rolling submission for the approval of casimersen, which, if successful, will treat another 8% or so of the Duchenne muscular dystrophy community. It is our goal to obtain that approval in 2020. If we do that, we will more than double the number of patients between VYONDYS and casimersen that could benefit from our PMO versus EXONDYS alone. And it also means we will be among that very rare club of biotechs that have 3 or more internally developed and FDA-approved therapies benefiting patients.

So let's move on to our platform, our next-generation platform for RNA, and that is our peptide conjugated PMO, otherwise known as our PMO -- our PPMO. Our PMO itself has a limitation. It's neutrally charged, it gets into cells only passively. The peptide-conjugated PMO intends to resolve that and to solve that by using the peptide, positively charged, it's a sort of carrier, to drag into the cells more of therapy. And in animal models, that's exactly what happens. The PPMO directs far more therapy into the muscle cells, it creates more exon skipping, it creates more dystrophin, and therefore, it creates greater benefit. We started last year our multi-ascending dose trial, and we should have both safety insight and dosing insight by the middle of this year. That's extremely important because we have already built candidates that could treat over 50% of Duchenne muscular dystrophy in the event that this proof-of-concept bears out.

We've continued to build infrastructure over the last year, which is important for our enduring model. We had about 500 employees at the beginning of 2019. We stand at about 800 employees today, but far more important than the number of employees itself is the fact that we're building talent where it matters to drive our strategy. We -- that's clinical development, manufacturing, techops, quality, research and the like. And we built this gene therapy center of excellence in Columbus. I can't begin to tell you how excited I am about this. Already we are prolific with that gene therapy center of excellence. We're already building constructs, testing constructs, advancing the science, and we'll be expanding on that gene therapy center of excellence in 2020. We've also built out the intellectual hub for our hybrid manufacturing strategy, both in Burlington, Massachusetts, where we have an AD and PD facility as well with our capabilities in Columbus, Ohio.

And we've made great progress from a manufacturing perspective as well over the course of 2019. In 2019, we deepened our relationship both with Thermo Fisher and with Catalent, Paragon. We completed with Thermo Fisher our single-use site in Lexington, Massachusetts for micro-dystrophin, and we built even more capacity than that with Catalent, Paragon. We've made great progress from an assay development and process development perspective now. Certainly, we still have more to do there, both in assay development and process development, but I do have 2 very important key takeaways. The first is, you should know, that we already have at scale data confirming that we are getting to commercially viable yields, which is extraordinarily important. And we are already, even as we speak, in the middle of our engineering runs to support Study 301 and its commencement at or around the middle of 2020. So things are going very well there.

I mentioned earlier, and I'll mention again, this concept of alliance is extraordinarily important. This is a big part of our ability to both stay focused on execution today, but build our pipeline for the future so that we can remain an enduring genetic medicine company. I am proud of the fact that in a very short period of time, we have become the partner of choice in -- among gene therapy and gene -- and genetic medicine companies generally around the world, and we've more than doubled the number of alliances over the last year or so. And at the very end of last year, we announced what is very likely the most impactful alliance that we've had in the history of Sarepta, and that, of course, is our relationship with Roche to bring SRP-9001, our micro-dystrophin construct, to patients living with and dying from Duchenne muscular dystrophy around the world. This is the largest ex-U.S. single candidate license of its kind in pharmaceutical history. And as impressive as that is and as validating as that is, it completely understates the point of this alliance. This alliance is much more important than that because if we're successful with SRP-9001, our alliance with Roche means that we will be able to treat vastly more Duchenne muscular dystrophy patients and vastly faster than Sarepta could ever have done on its own. And for those who wonder whether that's meaningful, consider this, every single day, every hour of every day, around the world, every hour, a child with Duchenne muscular dystrophy dies. And at the end of that 24-hour period, unrelenting period, you still have about 12 or 15 boys that died that you didn't account for. So the ability to move fast around the world is extraordinarily important to us.

And beyond that, when this alliance closes, and that will occur this quarter, we will have available to us at Sarepta over $2 billion that will allow us to invest in our programs, drive our vision and to make that revolution real. And speaking about growing and driving with the best and the brightest and alliances, I am delighted to announce that Dr. John Martin has agreed to join our already impressive Board at Sarepta Therapeutics and to provide us insight and guidance as we take our leadership role. I'm particularly proud of the fact that to the best of my knowledge, this is the first public company Board, unaffiliated with Gilead, that Dr. Martin has decided to join. I know I'm probably telling you all something that you're all already well aware of, but Dr. Martin is among the most impressive and singularly successful executives in all of biopharmaceuticals. He drove Gilead from basically a startup to one of the most impactful and meaningful healthcare companies around the globe. And along the way, both with his HIV focus and their hep C focus, they cured and saved innumerable lives. I am thrilled about the addition of John Martin and what it might mean to Sarepta's strategy and execution.

We've made a lot of progress over the last few years. Nevertheless, without going into detail on the slide, I can tell you that 2020 will be our busiest, and if we're successful, our most transformative year in the long story of Sarepta. This is Connor. Connor is one of the first 4 boys who was treated with SRP-9001, our gene therapy for Duchenne muscular dystrophy. This is Jamison. Jamison was one of the first 3 children who was treated with SRP-9003, our gene therapy intended to treat limb-girdle type 2E. This little girl Lulu, she has MPS IIIA, and she was treated with the Lysogene gene therapy for MPS IIIA. And this young man right here is Billy. Billy has Duchenne muscular dystrophy. Billy has also been on EXONDYS for 8 years. And by complete coincidence last year, our Board of Directors was meeting live the very evening that Billy was accepting his diploma from high school. And so we on the board had an opportunity to do something that we were very honored about, which is, via a live feed, we were able to watch as Billy, at the time 18 years old, with Duchenne muscular dystrophy, walked up on stage and accept his diploma. This is what the revolution looks like. Thank you.

Anupam Rama

Let's get this started. This is the Sarepta breakout session. Doug, if you want to just introduce up here on the panel with you to get started?

Douglas Ingram

Sure. Thank you all for joining us. To my far right is our Head of Research and Development, Dr. Gilmore O' Neill; to my immediately right is our Head of Gene Therapy, Dr. Louise Rodino-Klapac. She likes to be called LRK, by the way; to my left is our CFO, Sandy Mahatme; and standing over there trying to blend into the woodwork is Bo Cumbo, who's our Chief Commercial Officer.

Question-and-Answer Session

Q - Anupam Rama

Doug. Maybe talk a little bit about Study 2, the enrollment dynamics there. I think there was a hope that you might get some data this year. Was there something that happened in December in terms of enrollment dynamics that pushed it out to early 2021?

Douglas Ingram

No. I think the short answer is we're doing really well from an enrollment perspective. The fact is, if you think about it, we'll get to the -- we'll -- the study will be technically rolling over at the end of its 48-week period before the end of 2020, but we'll have to lock the data, scrub the data. So the truth is we won't get a true readout on that until early 2021. So this is not a delay, it's just the reality about the fact that this study is going to lock at the very end of the year and then read out right at the beginning of the following year. As you probably saw, actually, things are going quite well. We overperformed. We ended up enrolling, across Perry Shieh and Dr. Mendell's site, 41, not 40 patients. And more important than that -- well, equally important is that we're dosing crossover patients as well. So we've dosed a significant number of patients, more than the -- certainly, more than the 40 already. And it's going well and progressing well and certainly progressing uninterrupted. So things are really on track with respect to 102.

Anupam Rama

How many patients have you dosed crossover in lieu of those rhesus?

Douglas Ingram

We haven't -- first of all, we haven't said how many, and frankly, I'll be wrong because we probably did one today, but significant, a significant number of crossovers are rolling over right now. So we're certainly north of -- significantly north of 40 patients dosed so far in the trial.

Anupam Rama

I think you've talked about previously, a potential publication of the Phase I/II data. Is that going to -- is that still on track for this year, potentially? Will it have any new like longitudinal updates? Or is it something going to be a publication of data to be kind of [indiscernible].

Douglas Ingram

It's a 12-month data. So we will be updating it and it will occur this year. I don't know, Louise, if you want to comment on that?

Louise Rodino-Klapac

Yes, that should be coming out in the -- early in this year. And it will be the 12-month data, so not all of the data has been seen before.

Douglas Ingram

Got it.

Anupam Rama

Maybe you can help us think a little bit about the 2Q update in limb-girdle. Like I think you mentioned up there, maybe even size and scope that the patients will be getting, what should we be focused on in the higher dose cohort?

Douglas Ingram

Sure. And just for the web, I'll repeat the question. Broadly speaking, Anupam asked, what kind of update will we get in the second quarter as it relates to limb-girdle 2E. So it will be a 3-patient cohort. The -- as you may recall, the last cohort that we had was at 5x E^13. This is a dose that's 4x higher than that, 2x E^14. And the update we'll have there will obviously be expression, and it won't be function because it will be too early for function, but it will be expression and safety. And then I think on that basis, we can very likely make some dosing decisions between the 2. The good news is on that, we're already at a great place. As you saw on protein positive fibers, at the 5x E^13 dose, we're seeing over 51% versus normal. This is the native protein. So we feel like we're in good shape, and now let's just do one higher dose and then decide between the 2 which is the best answer for these children.

Anupam Rama

Any questions from the audience?

Unidentified Analyst

[Indiscernible] from you from the context of that company coming [indiscernible].

Douglas Ingram

Yes, I'll touch on it briefly. And Dr. O'Neill, if you want to touch on it. The broad strokes is we'll -- remember the background on the peptide conjugated PMO. So in animal models, this peptide has worked brilliantly. And it's -- it undoubtedly, in animal models, drags far more of the therapy into the muscles, and it creates more exon skipping. And in fact, you can get as much as an order of magnitude more dystrophin production if you had the right doses. So mechanically, there's little reason to believe that wouldn't do something similar in human beings. The real question is, can we get to the right dose and can we do that safely? So while that may seem like a soft answer, it's going to be a really important proof-of-concept by the middle of this year. The multi-ascending dose and the single-ascending dose studies are both going quite well. But we'll get the true insight on both the dosing levels and the safety by the middle of this year. And if you want to expand?

Gilmore O'Neill

Yes. Gilmore here from R&D. We will be looking in Q2 at safety and exposure and potentially some expression data and skipping data in tissues later in Q2 -- or rather, later this year in Q2. And we'll actually have a very robust data set, we believe, to make decisions about dosing going forward.

Anupam Rama

Can you just remind us the number of patients who follow up and the dose that you're at right now?

Gilmore O'Neill

So we haven't -- have we disclosed the dose levels that we're at?

Douglas Ingram

I don't think we have yet.

Gilmore O'Neill

No. So we are actually aggressively pushing dosing. So I won't go into more details than that, but we will actually give more details in Q2. But we are actually using -- I don't think we've actually disclosed the size of our cohorts either. But we are actually using a pretty standard dose escalation cohort design or serial cohort design. But I think what is important is that we will and are looking at tissues, muscle tissue specifically, and that will actually give us a very good data set to determine what doses we should be using to move forward. We actually are using -- as you know, our other patients who are participating in the multi-ascending dose both have early and later disease. So we will actually also have a sense of how the drug performs in tissues at different stages of the disease.

Douglas Ingram

I just noticed I should have introduced Ian Estepan as well. He was lurking behind me. Ian Estepan is not only our Head of IR, he's my Chief of Staff. Apologies for that.

Anupam Rama

Doug, on stage, you highlighted that the rolling submission for casimersen only started. What specific gating factor [indiscernible]?

Douglas Ingram

Yes. So the -- it's a rolling submission simply because we have some additional CMC and spec work that has to be done. It's all very much technical in nature. That doesn't affect -- just so we're clear, that doesn't have anything to do with safety or efficacy or approvability. We didn't want to wait for that to get done and what and then submit. We wanted to start a rolling submission. So we've got to get that work done. That will get done in the near future. I haven't announced exactly when yet because I don't want to overpromise on that, but our goal certainly is to get all of that work done and get the -- a PDUFA date and, hopefully, if all things work well, get approved by the end of this year.

Anupam Rama

A question from the audience.

Unidentified Analyst

Can you talk about the [indiscernible]?

Douglas Ingram

I apologize. I didn't -- did you get -- I didn't get the question.

Anupam Rama

[Indiscernible] what was driving the first CRL and then the quick reversal?

Douglas Ingram

Oh, it was -- I mean to be quite direct, it was precisely what we said along the way, which is the CRL itself was based on two purported safety signals that were not seen in humans, but -- where one was theoretical ports, which was purely theoretical, and one was an extrapolation from some animal data. They didn't appear to us on the face of it to be the kinds of things that would warrant the lack of an approval. So we commenced an appeal. I'm going to say a couple of things about that appeal. I've said it before. Let's be very clear about this. We assiduously followed the guidance. There was no stepping. We didn't step across the line or there was no political influence. This was purely -- we read the guidance on what the formal appeal process looks like. We gathered the data that supported our view. We submitted that appeal. That appeal was heard by Dr. Peter Stein in the Office of New Drugs, which is where those appeals are heard. There was a hearing. Our own Dr. Gilmore O'Neill represented us in that appeal and that data presentation. At the end of that process, Dr. Stein, in a very detailed opinion, agreed with our perspective on the safety signals and on the approvability of the therapy, and on that basis, we received approval.

I should also say one additional thing. I'm very proud of the fact that this team executed well and was able to receive a reversal of a CRL in very quick time. But it would be unfair of me not to mention the great work that the Food and Drug Administration did as well, particularly the neuro division. The neurology division did a brilliant job of moving on a -- in an objective way, but very fast to get this done. From the appeal, we had to fully resubmit an NDA and that NDA had to be reviewed in full by the neuro division as well. And I really think that they deserve and the patient community deserve -- should give them a big kudos for their willingness to move fast to get this therapy that's going to be important for patients approved.

Unidentified Analyst

Describe the primary [indiscernible] analysis for [indiscernible] under analysis.

Douglas Ingram

Well, and so the question was what -- how many -- what are the patients in the primary arm. With respect to 102, I believe, at the 48-week mark, it will be -- it'll either be '20 or '21 because we have 41 patients now enrolled and dosed in the study, and that will occur at the 48-week period. So that should occur before the end of this year. And then, of course, it will take us time to lock it down, scrub the data and have the results, and that's why it'll be early 2021.

Unidentified Analyst

Do you have biopsy data for [indiscernible]?

Douglas Ingram

Yes.

Gilmore O'Neill

Yes, we are collecting biopsies as part of the protocol.

Douglas Ingram

And the question there for the web was whether we would have biopsy data for the PPMO, the peptide conjugated PMO.

Unidentified Analyst

Doug, on stage, you mentioned enrollment completion. Are you thinking in the first half or midyear for MPS IIIB? What would be the time line to date within [indiscernible] one, proof-of-concept [indiscernible].

Douglas Ingram

I'm going to -- so the question, for the web, again, is as it relates to MPS IIIA. Yes, IIIA. This is -- what we are tracking right now in dosed 15 patients, and our goal is to dose all 20 patients before the middle of this year. And then, Louise, do you want to touch on the current analysis?

Louise Rodino-Klapac

[Indiscernible] Dr. O'Neill?

Gilmore O'Neill

So just everyone on the Webex knows what the question was, just around the design and what the output is. So obviously, it's an open-label study. What we're actually looking at are evidence of clinical improvement. And so because of that, we can actually do a number of cuts of the data, look at the analyses over time. And that's how the study is designed.

Anupam Rama

Questions from the audience?

Unidentified Analyst

So on the manufacturing side, do you guys -- is there any plans to try to take scale on what you have with margin?

Douglas Ingram

Yes. So very good question. So the question is whether -- we have spoken on the stage and before this about the fact that this Roche alliance will provide us with an opportunity to expand the number of patients that could be treated with and if it's successful, benefiting from the SRP-9001, our gene therapy. And of course, the question then becomes, but you didn't build your capacity with that breadth in mind. And that's true. So in the near to midterm, we have good capacity. As I've said before, we have a full single-use site in Lexington, Massachusetts completely built out. And we have significantly more actually built from a capacity's perspective at Paragon. But we are going to have to sit with our partner, Roche, think about places around the world that we haven't even envisioned on our own to be able to get to it in a reasonable period of time.

I'll just throw one obvious one out, which is China. China has somewhere between 55,000 and 65,000 patients who are living with and then 100% of the time, unfortunately, dying from Duchenne. We've got to look at that work. And so we got to really think that through with them. If -- as we think that through, if that requires us to increase capacity over time, and I suspect it will, then we'll just simply do that. And the real rate limiter with respect to gene therapy manufacturing is assay development and process development work. The actual ability to scale beyond that is something we can certainly do.

Anupam Rama

Other questions from the audience?

Unidentified Analyst

I know it's super early in the VYONDYS launch, but maybe any initial sort of market color on what makes it relative to EXONDYS had a similar platform?

Douglas Ingram

Bo doesn't want to commit himself. It's too early. Let me say the following with respect to VYONDYS. The work that Bo and his team have done over time and the work that a number of really innovative patient advocacy groups have done over time to make sure that patients are getting genotypes and the like is going to inure to the benefit of VYONDYS and the like. Nevertheless, this is a process. And it is, whether we like or not, often a slower process than we would like from start form, to initiation, to working with access and reimbursement. So as much as we will get a significant benefit from all the great work they're doing, it's going to take some time to really start significantly ramping that up. It's probably a 4 to 6-month process often from start form to initiation. Did I get that generally right, Bo? The web says -- Bo says yes to the web.

Unidentified Analyst

I think one of the key questions that, as we think about -- especially, too, is how do you think about how the placebo [indiscernible] or 48 weeks. And given I think these patients are allowed to be on stable steroids. We know in this [indiscernible] may some benefit over 12 months. So how do we think about that? I mean [indiscernible].

Douglas Ingram

Yes. Well, a couple of thoughts. One, if I'm not mistaken, every child was on stable steroids. Was it how many Bo, at least?

Louise Rodino-Klapac

For at least 3 months.

Douglas Ingram

For at least 3 months, often much longer than that in advance of this therapy. So every patient, placebo and otherwise was already on stable steroids. That is not surprising because that is typical of Duchenne muscular dystrophy. These children tend to be standard of care to get on steroids. We have both literature, but we also have our own patient-level data that helps inform our views on what should happen with this patient population. You may recall last year, our n was about 20 -- was actually exactly 24 patients. We wanted to make sure that we were properly powered. We got -- we obtained more material. As you know, in the middle of 2019, we increased the n to 40 patients. We're now at 41 patients, about 40 patients officially. And as a result of that, at the time that we were doing the powering calculation, that put us at or over 90% powering. So we feel very confident about where we are with respect to the study and it's powering as we move forward across the year, certainly without -- at the risk of being immodest, there is no company that exists that knows more about Duchenne muscular dystrophy and natural history regarding it -- who're going to do Duchenne muscular dystrophy than Sarepta Therapeutics.

Anupam Rama

So any final questions?

Unidentified Analyst

So top line Phase II [indiscernible], '21 Monday -- 2021 out there.

Douglas Ingram

I'm not going to -- I refuse to commit to that. But we're doing well. We've got a lot to do. We will have a readout on 102 early next year. We want to start 301 in the back hand -- back half of this year. We've got a ton to do, and things are going really well now. And I want to give a big kudos to the Sarepta team, who have really executed across 2019, and they'll do the same in 2020. And thank you all for your support.

Anupam Rama

Thank you.