Sarepta Therapeutics, Inc. (SRPT) Earnings Call Transcript & Summary

Hi, everyone, and welcome to the 2021 Credit Suisse Healthcare Conference. We're thrilled to have Sarepta with us. Doug Ingram, CEO, will be conducting a fireside chat with myself. I'm Judah Frommer. I cover SMid-cap biotech here at CS. And I'll just let the audience know, if you do have any questions for Doug, you can certainly e-mail them to me, [email protected].

And with that, why don't we jump right into DMD, which is going to be the topic of a conversation here. So Doug, maybe can you first just frame the current therapeutic landscape for DMD, where your marketed therapies fit and the degree of unmet need that still exists?

Sure. First of all, thank you for having us. I'm here along with Ian Estepan, our CFO. So there is an enormous unmet need in Duchenne muscular dystrophy and a real opportunity not only to do good for society, but in so doing to do well by investors. I mean consider it, where we are. I think it is fairly undisputed that Sarepta is the leader in the use of therapies to treat children with Duchenne muscular dystrophy. As we sit here today, we have 3 therapies that are approved, and they treat only about 29% of kids with Duchenne muscular dystrophy and almost all of them in the United States. And they give these kids a better life, and they slow the progress of this degenerative disease. And as a result of that, we have just had our earnings call, we shared our 20th straight quarter of really robust growth. We had 40% compounded annual growth rate since I've joined Sarepta. We'll do about $605 million to $615 million. So now let's think about what the need is here, right? The need is enormous. First, we need even more profound therapies. Our PMOs are the -- without a doubt, right now, the gold standard in the treatment of Duchenne muscular dystrophy. They slow degeneration down. But we need even more profound treatments for these kids. We need to treat a greater percentage of kids. 29% isn't enough. And we need to go around the world. It can't be primarily a U.S. opportunity. The great thing about where we stand today, Sarepta, and then we can talk about the broader industry if you like, but as it relates to Sarepta, we have 2 development-stage platforms. And in those platforms, we are in pivotal trials right now enrolling patients. We've got EMBARK, which is our gene therapy, pivotal trial for SRP-9001 to treat Duchenne muscular dystrophy. And we've got MOMENTUM, which is our pivotal trial for exon 51, amenable Duchenne kids using this next-generation potentially profound improvement in our RNA technology, SRP-5051. And then we've got 40 additional programs. We can keep doing this. And we've got over $2 billion of cash to do it along with our revenue. So we're in a -- we've never been in a better place as an organization to do good.

Perfect. And I appreciate you using trial names as opposed to numbers. Maybe just a couple of more questions to set the table here. How should we think about the potential for micro-dystrophin compared to human dystrophin to kind of affect disease progression? And can you remind us of the micro-dystrophin construct and the history of its development within Sarepta?

Yes. And so I'll -- let me start with the end statement, which is in the -- at least in the work we've seen so far, both in animal models, but also in children, we'll talk about, I'm sure, some of those studies. We're very confident that our micro-dystrophin construct is going to act like a shock absorber just as dystrophin does protecting these muscles. What is it? So first of all, this all comes from the fact that -- why do we use micro-dystrophin? Why are we not inserting a gene for dystrophin? And the short answer is that dystrophin is too big. It's too large. The carrying capacity of AAV does not accommodate it. But a brilliant scientist, who is now our Head of Gene Therapy and our Chief Scientific Officer, Dr. Louise Rodino-Klapac and her mentor, Dr. Jerry Mendell, relying upon some natural history that existed some years ago, more than 15 years ago, started working on an edited version of dystrophin that would keep the protective structural -- the shock absorbing nature of the resulting protein, but could reduce the size that could fit into a capsid. Did a host of preclinical work on it, optimized that construct, which I can talk about in a second. And then we've moved into a number of different trials. In fact, we're on our fourth trial with respect to this therapy. And the difference -- we -- there are others that have been working in this area. Ours is unique to us. First, it comes from the vector. The vector is the delivery mechanism, the virus. In this case, it's something called rh74. We alone use it very different, both from a tropism perspective and a safety perspective, we can talk about it. The gene construct itself was designed both through some -- informed by the research, but then informed by the empirical work of Louise. And then we've got a unique promoter that turns the gene on. So we have a very differentiated therapy that we think, if confirmed in our next trial, could be a profound therapy for kids with Duchenne muscular dystrophy. And what's exciting about it is that unlike our RNA therapy, it is agnostic to mutation.

Got it. And before we get into 9001, maybe just can you highlight for us expectation around correlation of dystrophin expression relative to normalized function, right? The question is basically, is higher better or is there some threshold level that you'd like to get to?

Certainly, there's a -- you want to get to a certain threshold of dystrophin so that you're confident that you're going to get a functional benefit. But we are way beyond that now with our therapy. In our most recent studies, we're seeing well over 50% of normal, which is just a tremendous amount of dystrophin. And the vast majority of the fibers of these children are protected. But with that said, more is better. We're very confident that more is better. It's better both from function, but it's also better for long-term durability. So if we can get a significant number of genome copies per nucleus into the right place, we can make a ton of dystrophin, then we're very confident, given the slow turnover of muscle, that we'll have great long-term durability. And the good news is we're well over 3 genome copies per nucleus in our most recent look at the amount of gene that we're able to deliver into the muscle cell.

Okay. Perfect. And moving into the gene therapy program. I think I've heard you do this within a couple of minutes before. But can you just help us with a brief history of kind of the Phase I and II studies, kind of how you got to Phase III if we talk about 101, 102 and 103? And then we can maybe get a little deeper after that?

Yes. Yes. You're going to -- I'm going to take the challenge and try to do this briefly. So there are so many studies we've now done. So the first one, 101 is a proof-of-concept study, 4 kids. And the great thing about that is that the 1-year mark in these 4 kids, we had an R&D Day back in the middle of 2018, and we saw this -- a tremendous response from these kids. They had great expression. The safety profile was very stable. We had some elevated liver enzymes that responded to steroids. But otherwise, these kids did really well. And then they had enormous numbers of genome copies per nucleus, fabulous expression, fabulous localization, et cetera. And then you look out at 1 year, and those kids were doing just brilliantly at 1 year. Now we just announced some results at 3 years for those kids, and these kids are getting older. And remember, this is a degenerative disease. And the difference between where these kids are today and where they ought to be from a natural history perspective was stark. On a 34-point scale, which is called the NSAA scale, there was a 9-point difference with a p-value -- post-tox, but a p-value of 0.0001. And if I showed you videos, it's heart-breaking to see a 1 point difference in some of these particular measures. These kids were 9 points better than natural history. So that was exciting. Then we go to the next study. The next study is called 102. 102 was a 41-patient -- is a 41-patient, double-blind, placebo-controlled trial. We had the first part of that trial that reported out in January. We saw very good expression. We'll get better expression in the future than what we saw in early 102 because we had an issue with our clinical supplier and their titering. And then we had about 60% of the kids have lower than the target dose. That will not replicate itself. In the 4- to 5-year-olds in the Part 1, they were properly age-matched and properly baseline-matched. And we saw a really strong statistically significant benefit, clinically meaningful. Those kids were with a p-value of, I think, 0.017, were doing brilliant compared to the placebo group. The 6- to 7-year-olds, there was an enormous problem. They were completely wrongly matched against their placebo cohorts where all the treated kids were severe and all of the kids on placebo were much milder. When you actually take those kids -- and we just showed this maybe a month ago, when you take those kids and you age-match and baseline-match them against natural history, they're doing brilliant with a really strong p-value. They're significantly outperforming natural history. And then we get over to 103. 103 is really important. 103 is our own commercial supply, our own process development, analytical development. It's the material we're going to use for -- we are using for our pivotal trial. It's the material we're going to launch this therapy with and supply it to the world. And just we've seen just fantastic results. First, in our first 11 cohort group of that Study 103, we saw fantastic expression. We had 3.8 or so genome copies per nucleus, which is an enormous number of genome copies per nucleus on average. We saw western blots of about 54%. The vast majorities of the fibers were protected. And then we just looked at the 6-month results, 6 months, really short period of time. And in those -- that 103 group, those kids were already against their own baselines, already improved by 3 points at 6 months when natural history would have said they'd already be in decline. So that's the background to what we're about to start, what we're actually working on right now. We're enrolling kids right now, which is our EMBARK study, and that's going to be our pivotal trial. And that's a 120-patient study, that's worldwide, including the United States.

Perfect. That's a great brief history. But first, just taking a step back, and you touched on it for a minute there, but back to the capsid, right? Your decision to use AAVrh74, how does it differentiate versus AAV9, which is being used in other trials? And maybe specifically on the safety side of things.

Yes. rh74 is just showing itself to be markedly different than other AAV9s. And the other AAV9s are showing themselves to be very similar to one another in a number of regards. The most significant issue with a number of capsids, including AAV9, probably the lead in this is the inducement of complement-mediated immune response. There's complement-mediated aHUS that has resulted in very, very serious adverse events. And in fact, there have been a number of kidney failures as a result of this in a number of kids. Even this year, in a couple of different programs, children have had kidney failures and have had to actually go on dialysis as a result of that. I'm very pleased to say that we just do not see that with rh74. We never saw it in preclinical work. We started dosing kids. In the early days, we never got a hit of a clinical complement activation. I think the world sort of imagined maybe we were just getting lucky. We've now dosed 77 children. We've dosed the oldest children, I believe, that anyone has ever dosed in Duchenne muscular dystrophy with a full-body infusion, maybe any gene therapy full-body infusion. We've dosed the largest kids of anyone. We've dosed 81-kilogram kids and late teens. I think 19 is maybe currently our oldest kid. And we just do not see any complement -- clinical complement manifestation or aHUS or kidney failure. And we're getting extraordinary tropism with rh74. So I think Louise Rodino-Klapac, along with her partner at Nationwide, Jerry Mendell, deserve an enormous amount of kudos for the work they did to choose rh74 at a time when everybody was choosing AAV9 back before they really understood that AAV9 just may not be fit for purpose for these full-body infusions.

Got it. And coming back to the studies, maybe let's start with Part 2 of Study 102. What are you going to be looking for there? How could a delay for the placebo first cohort potentially impact comparing efficacy between the 2 cohorts there?

Yes. So the -- well, so the good news is we won't be comparing the cohorts because not only are they on different journeys, one group will be on the therapy for 2 years, one group will be on the therapy for 1 year with a 1-year run-in. And there'll be different ages along the way because they're along a different journey. So -- but what we will be comparing them against, including against themselves of course, is against a well-matched, baseline-matched, age-matched natural history cohort that will be all walked down before that study is unblinded. I want to be clear, this has all been prespecified. That's an unblinded study. So early next year, we should have some really interesting information out of Study 102 where you'll get about half the kids who've been on therapy for 2 years against the natural history cohort. And then the other half of the kids, we'll have been watching them for a year on just a placebo, although they've been unaware of it, of course, it's blinded. And then another year where we'll get to -- we'll have watched them on the therapy and then compared them against an age-matched, baseline-matched natural history cohort. So I think we're going to get a lot of really interesting insight out of that study early next year. We're very excited about it.

Got it. And kind of same question for Study 103. What's the next milestone to look for there?

Well, we'll have some -- I think, next year, we'll have -- 103 is an interesting study, and it's an ongoing study in a number of regards. It started out as an 11-patient cohort study. But we've really explored the age ranges and weight ranges using 103. As I've said before, we've dosed children -- using 103, we've expanded. We're over 30 children already in 103. We've gotten a really interesting experience with older kids. And good news that we're getting a very similar safety profile across these kids, in the older kids and the heavier kids as well. We've dosed, as I said, 81 kilograms is I think the highest we've dosed right now, which is a very, very large patient in Duchenne, and things went very well. And we're actually dosing very young kids now as well. So we're using 103 to explore that. Likely sometime next year, we'll have some additional data and some functional readouts from 103. Some of those cohorts will start hitting their 1-year mark, and we'll have that data to look at as well that will help provide additional insight as we execute EMBARK, which is our pivotal trial.

Okay. Great. And speaking of EMBARK, how should we think about the comparability of results within EMBARK once we get them to kind of the prior studies you've conducted with 9001? And how has the design of EMBARK been informed by your prior studies?

Yes. Well, a lot of -- a number of interesting things. One aspect of EMBARK -- EMBARK, yes, I'm very confident about EMBARK. I'm confident about its powering. It is a 120-patient, double-blind placebo 1:1 trial. So it's well powered from our perspective, and it is well informed by all of the studies that came before it. We have really -- we have a very tight manufacturing process for our commercial supply. And that means that our titering is spot on. So these kids will all get exactly the right dose. So I think from an expression perspective, we're very confident these kids, based on everything we've seen, are going to have really robust expression. I will predict at least that the vast majority of their fibers will be protected by dystrophin. And therefore, I think they're going to do very well in this trial. There were a number of things that helped us inform the study, including 102. We've really tried to -- one of the things you want to make sure -- we want to make sure we met with EMBARK. So not only do we have a 120-patient study, we put very hard floors and ceilings in that study. We've reduced variability by putting in a limitation on rise time. So you can't have a rise time that's slower than 5 seconds. Not because the therapy won't work on kids that rise slower than 5 seconds, but to try to reduce the heterogeneity in the population to make sure that there's 2 things that one has to do. One, you have to have a therapy that works. And of course, you can imagine, I am completely confident on that. But you have to also be able to show empirically that it works through a study. So we're narrowing the standard deviation to make sure that, that is obvious in a short period of time, a 52-week period of time. And then another thing that we've been informed of from 103 is we had always had some concern about these certain mutations around the early exons of the gene and whether those mutations might leave the kid not having had a tolerizing experience with any background dystrophin and then that kid could have an immune response to the actual gene construct and protein itself. We did see one of those cases in 103. And so we have some additional exclusions in EMBARK to -- for the purpose of EMBARK. But in the end, by the time we get to commercialize, we'll do some additional work, and I'm quite confident that the number of mutations that would be excluded will be very, very small, a small fraction of the actual kids that have Duchenne muscular dystrophy.

Okay. That's helpful. And with the next one, I'll kind of tie in an investor question that's come in as well. But what's the strategy for addressing older versus younger patients? And kind of the investor question is, could you file early based on crossover patient data and subgroup data from the Phase III in 4- to 5-year-old? And any color from regulators on that?

Yes. So let's start with the first question. So our goal, when we launch this therapy, is to have a therapy that does not have a limitation on age. And the non-ambulatory patients need this as desperately as anyone. In fact, time is ticking for those children. And so long as they have -- I am convinced, so long as they have skeletal muscle, diaphragm muscle, cardiac muscle, this therapy will help them in extending their life. So we've got to get the therapy to them. And we're doing a lot to ensure that we do. We have already -- as I said, in Study 103, we're already exploring age ranges as broad as we can to ensure that we have the safety and expression-related information. We'll also have a separate study just on non-ambulatory patients as well along this pathway. So it is our goal, and we believe, based on at least our history with -- at least with CEDR and Neuro, that we will get a broad label ultimately. And that has to be the goal for these patients, both in the United States and around the world. On -- now to the second part of your question, which is really this question, can you use a cut of 102 and all the other data and sort of the collection of totality of evidence you have on the therapy to seek an earlier approval, presumably on an accelerated approval basis from the FDA before the pivotal trial reads out? First, let me say I'm going to repeat what I've said to everyone constantly, which is everyone should assume that the pathway to approval was our pivotal trial, and that's EMBARK. What I will say is that if the data from Study 102 Part 2, in addition to all of the other data that we have paints the compelling story, and I believe it already does with respect to this therapy, then certainly we'll have conversations with the FDA next year about whether there is a faster pathway to approval for at least a subset of children with Duchenne muscular dystrophy, while we continue to execute EMBARK. But there are 2 issues there. Of course, the totality of evidence has to support it. And the division has to support it. And we have never had those conversations with the division. So again, I would really strongly emphasize to people, the most exciting thing about the 102 results will be the additional conviction they will give us as we're executing EMBARK, which is our pivotal trial. I really think from an investor perspective, that ought to be the thesis upon which I invest.

Got it. Okay. That's really helpful. And before we move off of gene therapy, I thought we could just touch on your efforts in limb-girdle muscular dystrophy, specifically LGMD2E. And how does your work in Duchenne inform the work you're doing there?

Yes. One of the great things about the limb-girdle programs and Duchenne and our gene therapy approach generally is that it is a platform. People talk often about platforms. This really is a platform. I mean if you looked at them -- if I showed you a schematic, you would see how similar they are. It is the same -- precisely the same capsid, rh74. It is the same promoter, muscle-specific promoter. MHCK7 is a really robust promoter. And then the gene constructs themselves, of course, differ. With LGMD2E being interesting because it is a small enough gene, and therefore, it's packageable inside an AAV. So it's the unaltered native gene coding for the unaltered native protein, but otherwise very similar. As a result of that, all of our work is informed across platform. So for instance, the safety profile that we see with SRP-9001 with DMD, we see the same safety profile for limb-girdle Type 2E. Of course, we've never seen clinical complement manifestation. It is generally well tolerated in the same way that we've seen. And these kids are larger. The LGMD2E kids are larger. We've gotten great results so far with the 2 cohorts that we've dosed with clinical supply. We've seen fantastic expression across both our low and high dose cohort, and we've seen great functional results across both of those cohorts. So now the goal has to be to get to a pivotal trial for limb-girdle Type 2E. We -- our goal is to do that next year. But the big rate limiter for us right now is CMC. So we've got to complete the process development and analytical development work for 2E, well informed by what we've already done with 9001. But we still have work to do there. And then we want to align on a pathway forward. Our view on limb-girdle Type 2E is pretty explicitly we think that this is a perfect opportunity for an accelerated approval pathway. And we want to do that for 2E, but we also want to do it for the other sarcoglycans. So one of the things that we're considering while we're working on the CMC is whether there's a way to accelerate some of the work in the other sarcoglycan limb-girdles so that we can get all of those therapies moving at about the same pace and then launched to the community at about the same time, which I think would be the most efficient way to serve the greatest number of patients.

Okay. That's really helpful. And I think we'll switch over to the PPMO platform. We'll start with 5051. And can you maybe contextualize 5051 and the data you've seen to date relative to what you've seen from EXONDYS? Maybe that's a good way to frame it.

Yes. So just to remind everybody, what's wonderful about our PMOs, before we get to the PPMOs, because it will explain why the PPMO could be such a game changer. The great thing about our PMOs, first, they're precise. When they get to the right place, they do their job very well. They edit messenger RNA, put it back in frame and make dystrophin. And they do it very reliable -- very, very reliable. And they've got a really wonderful safety margin associated with them. The downside or the limitation, I should say, on the PMOs is they're neutrally charged molecules. So they don't get across the cell wall very easily, and they clear the body in about 4 hours. So if you really want to get a profound increase in dystrophin, you've got to find a way to get that PMO across the cell wall and into the nucleus in much greater abundance. This positively charged peptide, our animal models told us, would do just that. It interacts with these negatively charged proteoglycans on the surface of the muscle cell, is dragged into the cell and then releases the PMO, which traffics to the nucleus and does its exon skipping. So what have we seen? Short answer against the EXONDYS very simply, at the 30 mg per kg cohort, which is the cohort that we're bringing forward, the dose we're bringing forward into the pivotal trial, we saw about 18x greater exon skipping. We saw almost an order of magnitude more dystrophin production. And we saw it in half the time and at 20% of the dose exposure of eteplirsen, EXONDYS, which means that against the current gold standard, this thing, if it all goes well and if we see in the pivotal trial what we've seen in the early signals and it's safe, this is a profound game-changing improvement in the treatment of Duchenne muscular dystrophy. And that's why we're getting really excited about moving -- we're going to move a bunch more PPMOs forward, getting far more kids in frame with this therapy as we're seeing confidence from SRP-5051, which is our PPMO for exon 51.

Okay. Got it. And speaking of the trial, what did you learn from Part A in terms of informing -- or -- and can you walk us through the design of Part B and how that's differentiated?

Yes. So Part B is -- so first of all, Part B will be for an accelerated approval. And so as I was very, very cautious about talking about accelerated approval over with gene therapy, I'm in a different place with RNA therapy. And there's a lot of reason for that. Most of it comes down to precedented history. So we have a very -- we've had many conversations over many years with the Neuro division about the use of dystrophin as a surrogate endpoint for accelerated approvals. And of course, we have lots of success there. We have EXONDYS and VYONDYS and AMONDYS, all have been approved on that basis. So we're very confident about our accelerated approval pathway. We will list -- in broad strokes then, the -- there'll be -- we're going to look at dystrophin production at 28 weeks. 28 weeks is very early to look for dystrophin production with PMOs. But we have learned from our first part of MOMENTUM that this peptide-contributed PMO is so potentially powerful that even at 28 weeks, we should see a very robust amount of dystrophin made, and it will build over time from there. So we're thinking about ways in which we can check for dystrophin later to really see how much dystrophin that will be over the long run. And then probably need a year's worth of safety data. And then we will seek an accelerated approval on the basis of dystrophin production based on Part B of MOMENTUM. And then it's about approximately 30 new kids and then 30 kids that will roll over from the prior MOMENTUM study and single ascending dose study before that into this, they'll be invited in as well. So that's kind of the broad strokes for the study itself.

Got it. Okay. And how are you thinking about kind of the bar for efficacy, right? If you're going after accelerated approval, how do you think about exon skipping percentage as a proxy for eventual dystrophin expression?

Well, exon skipping is a brilliant marker for dystrophin production, but we're going to be looking for dystrophin production. So -- and the FDA is going to want to see the actual dystrophin production itself, and that will be the biomarker that we'll use. The surrogate endpoint will be actually dystrophin itself. The official bar, at least historically, for dystrophin is really just at EXONDYS or better, but that's not our target product profile. Our target product profile is seeing something in the range of what we've seen in Part A of MOMENTUM. And as I've said, what we saw -- so what we've seen in the Part A is something that is -- it's about 8x right now, 8x more dystrophin production. And that's in half the time. So if we're right about this and if MOMENTUM goes well, we should be comfortably above 5%. And actually, our modeling says, if you look out at a year, will be significantly above 10% dystrophin reduction. The literature would tell you that that is a profound -- not just a profound improvement over EXONDYS, but that will be a profound intervention in lives of children with Duchenne muscular dystrophy, at least predicted by the literature and what we've seen so far. So that -- so there's -- the standard for approval is lower than our actual target product profile, which is far more profound.

Okay. Got it. And you talked about maybe pushing the PPMO platform into other mutations. So how should we think about the progress there and where you're headed next?

Well, the good news is we're -- I think one of the reasons we went out and raised some money some weeks ago, we raised it at a time when people would say, "You didn't have to raise money. Why did you raise money at $1.6 billion on the balance sheet." And one of the reasons for that is to make sure that being conservative that we're fully funded to fully take advantage of the pipeline we have. Probably the 2 most exciting opportunities that are empowered by that are these. One is these PPMOs. So we already have constructs built behind 5051. We want to start advancing those therapies through the preclinical tox work and then start getting them to patients as soon as possible. And we're doing some of that work even as we speak right now. At least theoretically, the PPMO can treat as many as 80% or more of children with Duchenne, pretty easily over 50%. Then we have to come to a more exotic approach to get from 50% to 80% just because of the size of the population. But it's also why I think it will give us a big opportunity ex U.S. So that's one big use of our money to start driving the preclinical tox work and other work to start getting those therapies moving. And then the other use of our proceeds just to go back to gene therapy is to get these -- the rest of these limb-girdles moving as fast as possible as well.

Okay. That's helpful. And in an ideal scenario where you do have PPMOs and gene therapies on the market for DMD, right, where does each fit? Does each have a place?

I think that each will have a quest. I think conservatively, a lot of folks, and we've done a lot of this modeling as well, have assumed that with a really profound gene therapy, perhaps it will greatly cannibalize the existing RNA therapy. And I think that there is a thesis along those lines. But I think there's also a real possibility that both of these therapies live together for a host of reasons. One, just because there will be places where -- first of all, there'll be places where gene therapy won't be usable, but the RNA therapy will. As an example, about 14% or so of children that are -- would otherwise be amenable to our therapy will screen out because they'll have preexisting neutralizing antibodies or binding antibodies, and those kids then could have an RNA therapy. There will be places around the world with the vagaries of health care systems that will make one available, perhaps not both available. So there'll be an opportunity to try to fill in, in those health care systems. And then I think there's a real possibility, although we're doing the work on it right now, where there may be a combination value of a gene therapy plus an ongoing chronic therapy that might create the greatest benefit to kids over the long haul. And if we are right in our hope, not kids, but adults and middle-aged people and the like because we look to the day when we stop talking about Duchenne muscular dystrophy kids and teens and young adults and we're talking about Duchenne muscular dystrophy middle-aged people and elderly people. And that is really honestly ambitiously our goal behind all of this. So I think there is a real chance that there's going to be a profound place for both a profound next-generation RNA therapy like our PPMO, along with our gene therapy 9001.

Got it. And a couple of investor questions are coming in here. So one, you touched on, Doug, just in terms of the timing for the capital raise. Was it to kind of accelerate other programs as opposed to necessarily some indication of confidence in your next readouts.

It's definitely not the latter, let me be very clear about that. And I will say, what did people -- I had a lot of calls that day. And of course, the question is why now, why not later? Now I will say, not to be a little snarky, but I've raised money a number of times at Sarepta. I've never had a single time we've raised money when people didn't say why now, why not later. Because I think people don't like dilution. Nor do I. I'm an investor. I want to be very clear. I'm as much an investor in this company as I am the CEO of the company. But we wanted to raise money when we didn't have to, that is our biggest issue. We have an enormous pipeline. We have a 40-program pipeline. And we are not willing to simply just take the risk of spending and then some day waking up and realizing it's time now to raise some money and feel like we're raising money with a gun against our head. So yes, we didn't have to raise right now, but we had a number of really important programs that we wanted to drive. And we kicked up the investment in them -- in those programs immediately after we raised. And I mentioned them just now to you. A significant number of our PPMOs, we want to get going from a manufacturing perspective, a preclinical perspective, a tox perspective. That's, of course, expensive, and we want to move that forward. And then on the limb-girdle side, the manufacturing for gene therapies is significant, the process development, analytical development. QA, QC is enormous. And we want to get all of those programs moving as fast as possible. And so that's the reason that we raise. The good news is we're sitting here right now at over $2 billion on the balance sheet. We're in pivotal trials on our 2 late-stage development platforms. I have an enormous amount of conviction about what the readouts are going to look like over the coming few months, and we're just in great shape as a result of that. And although as a sort of a biotech R&D company, people don't ask enough about it, we also have extraordinarily good revenue right now that's going to help fund our pipeline as well.

Okay. That's -- I think that covers that. And then the next one is just on gene therapy for CMT. Any updates on that program?

We're really -- we're very excited about our gene therapy for CMT. It could be really a profound therapy. We, along with our partner over at Nationwide Children's Hospital, Dr. Zarife Sahenk, the brilliant scientist who developed this therapy, both remain somewhat frustrated by the fact that we just don't have -- we don't have the material right now to start the study. We've contracted with Nationwide for -- and their spin-off for that material, and it just hasn't been released yet. And that's been our big issue. It's all been about waiting for the material to commence a trial. We're looking now, and of course, we have the resources now with, as I've said, over $2 billion on the balance sheet. We're looking now and exploring whether rather than having a clinical supply from a third party, we simply bring this in-house, we do our own process development, analytical development and we get moving. But it has been delayed, and it's been delayed because of manufacturing.

Got it. Okay. That's a great update. And I think we're just out of time there. So we certainly appreciate all the color and commentary. It's been really helpful. And thanks again for Sarepta's participation in the conference.

Thank you very much. Thanks for having us.

Absolutely. All right. Take care, everyone.