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44th Annual J.P. Morgan Healthcare Conference

Jan 12, 2026

Anupam Rama
Senior Biotech Analyst, JPMorgan

Welcome, everyone, to the 44th Annual JPMorgan Healthcare Conference. My name is Anupam Rama. I'm one of the Senior Biotech Analysts here at JPMorgan. I'm joined by my squad; Joyce Zhou, Priyanka Grover, and Rathi Pillai. Our next presenting company is Sarepta Therapeutics, and presenting on behalf of the company, we have CEO Doug Ingram. Doug?

Doug Ingram
CEO, Sarepta Therapeutics

Thank you very much, Anupam. You know, a Duchenne expert once said to me, and I quote, "By definition, Duchenne boys do not ride bicycles." Well, this boy here, Max, riding this bicycle in this picture, has Duchenne, and this explains why we are so passionate about everything that we do. Thank you all for joining us today. I'll be making some forward-looking statements. Please review our various public filings for the risks and uncertainties that come when making predictions about the future. We're entering 2026 on a strong financial footing with an enormous amount of opportunity in front of us. That's what we're going to talk about. Today, we're going to talk about three things. First, we'll talk about the significant untapped opportunity with our four approved therapies. We'll then move to the outsized opportunity that comes with our next-generation siRNA pipeline.

Finally, we're going to talk about that financial footing and the fact that we're in great financial shape, which will allow us to realize our ambitions and fully see the opportunities that are in front of us. Before we do all of that, let's talk briefly about Q4, full year, 2025 results. For the full year, as you can see, our total net product revenue was $1.86 billion. Elevidys posted just under $900 million, $899 million for the year. That grew at 9% over the prior year, even in the face of all of the distractions that occurred in 2025. And the PMOs posted $966 million, flat to the prior year, even though there was some modest cannibalization from Elevidys. If we look at the fourth quarter, you can see our total net product revenue was $370 million, exceeding expectations.

The PMOs stood at $259 million, and you'll see that Elevidys posted $110 million. Now, you'll also know that as we tracked to the end of the year, we also tracked into one of the most severe flu seasons in recent history. We had been tracking to flat as expectation would have had us, but in December, there were six infusions that, for safety reasons, had to be delayed into 2026. That obviously reduced the number, but we're quite confident all of those infusions will take place. We ended the year with $954 million in cash and cash equivalents, and that was about $89 million of additional cash over the prior sequential quarter. We have a number of important initiatives. I'm going to talk about at least one of them today to continue the success of our four approved therapies.

You'll also know that previously we had set a floor, yearly floor for Elevidys, of $500 million. I can confirm to you once again that we have a $500 million floor, and I can also tell you our intention to significantly grow off that floor and exceed it. I am, nevertheless, going to stop short of giving more detailed guidance today until we track into the year and we see the impact of some of these initiatives. Talking about initiatives, let's talk first about Elevidys. Elevidys is a tremendous therapy that has already brought a better life to over 1,100 boys and young men, and yet 80% of the addressable ambulatory-only population remains to be treated. That is an enormous opportunity.

We also have a significant opportunity as an organization that comes from the fact that in 2025, for very good reason, we spent the bulk of our time talking about safety, and it's now time to balance that and start also talking about the absolute wealth of evidence supporting the efficacy of Elevidys and the disease-slowing nature of Elevidys, and we're going to do exactly that. We have a really well-developed plan to do that. We have significantly increased the size of our sales force for better reach. We are augmenting that with a muscular promotional campaign to support it, and we have really interesting initiatives with the patient community to accurately and thoughtfully communicate with them. As it relates to the non-ambulatory patient population, you'll know we're not dosing them right now.

Our pathway back to dosing them would be the success of Endeavor Cohort 8, which is our trial for the pretreatment with sirolimus in the non-ambulatory patient population. We're executing that trial now, and you can expect those results at the very back end of this year. So the absolutely primary initiative commercially for Elevidys in 2026 is to broadly communicate the wealth of evidence on the disease-slowing nature of Elevidys. For instance, in 2026, we're going to spend a lot of time talking about the results from Embark Part 1, where Elevidys hit statistical significance on each of its secondary endpoints, its key secondary endpoints. Now, you'll have seen in other programs recently, others have been using as a metric of disease slowing, they use percentages.

And I think, upon reflection, that is a very thoughtful way of trying to contextualize the impact of a therapy rather than what we often do, frankly, which is give raw numbers, which mean very little to either patients or the physicians they treat. So we're doing that. And using that metric, Elevidys slowed disease progression on each of rise from floor, four-stair climb, and 10-meter walk run in each case by greater than 100%. And the way it was able to achieve 100% is because those boys who had the benefit of being on Elevidys were able to actually improve on those measures, where those boys in the placebo group that didn't have access to Elevidys, on average, started the inevitable decline on those measures that you would expect from Duchenne muscular dystrophy.

Likewise, we're going to spend a lot of time in 2026 talking about the absolutely fantastic results from Embark Part 2, which is the second part of our pivotal trial. In that part of our trial, Elevidys hit every single pre-specified primary and secondary endpoint, and using that same prior metric, Elevidys slowed disease progression on each of NSAA, on rise from floor, on 10-meter walk run by anywhere from 76% to over 100% in instances where boys were actually able to improve when they were expected to have declined. We're going to spend a lot of time in 2026 talking about the wealth of data that comes from our muscle MRI data and muscle health, and I'm going to show you that data in a moment.

And if one wonders what can happen to a boy on Elevidys over the long term, I would ask you to look no further than the boys that were treated back in 2018, because the boys that were treated in 2018, at the end of that study, which, by the way, was a five-year study, were still 7.5 points on NSAA above their own baselines when they started that study. Let me show you two additional pieces of information that are going to play a big role in our initiative this year. Now, this first data is fabulous. This is our crossover data.

It gives us a unique opportunity where these boys were blinded over our entire two-year period, and we had an opportunity to see what happens to treated boys when they're crossed over to a placebo and likewise what happens to a placebo boy when he's crossed over into treatment. Here's what we got. Look at the top line, the dark line. Those are the treated boys. So on day zero, those boys were given an infusion of Elevidys, and they were also, by the way, given a modest increase in steroids per protocol. You could see they got a lot of benefit, and then they maintained that benefit all the way through 52 weeks. Then at 52 weeks, they were given a placebo infusion.

They were also, by the way, given that same increase in steroids that they were given on day zero, and let's see what happened to those boys. What you can see happen is this: they continued to maintain all of the benefit they had previously received. They didn't receive any more benefit, as you would not have expected, since they got a placebo. And by the way, you don't see any steroid effect here, so we can just take that concept, that speculation, completely off the table. Now, let's look at what happens to the placebo boys, so the placebo boys at day zero, those boys were given a placebo infusion. They didn't get Elevidys. And you can see throughout the entire year at every measure, they lagged behind those boys that had been treated.

And then at 52 weeks, that is that vertical line, they were given a treatment of Elevidys. What happened? They rocketed up. Why did they rocket up? Occam's Razor, because Elevidys finally gave them the dystrophin necessary to protect their muscles and slow the course of this disease. Let me show you something else. This is the muscle MRI data between the placebo boys and the treated boys in our study. Now, you need to know something about Duchenne. By the time a boy with Duchenne enters his mid-teens, he has largely had his muscle destroyed and replaced by fat and fibrotic tissue. And yet, when Elevidys' treatment happens, in a short two-year period, you see a significant reduction in fat fraction compared to placebo, a strong indication of much better muscle health. There's inflammation data as well.

I'm not showing it here, but it is the same, very similar data, and it's also showing significant increase in health on treated boys versus non-treated boys. My point of all this, when I think about this initiative and I think about all this data, is that we are going to spend our time in 2026 ensuring that this is appreciated, not by some lucky few, but by all treating physicians, referring physicians, families, and patients who can benefit from knowing all this, and that's going to be a big part of 2026 for us. Let's move on to the PMOs. You know with the PMOs, we have three approved therapies.

We have Exondys 51, Vyondys 53, and Amondys 45. Those therapies have been benefiting boys in some cases for over a decade. They've been on the market for a very long time, and there's some things to know about them.

The first thing to know is that we've dosed nearly 2,000 boys with these PMOs. There's a lot of experience here. The second thing to know is that the safety profile of these therapies over this decade has just been absolutely stellar. It's been a stellar, stable safety record for over a decade. The third thing to know about these therapies is, notwithstanding the protocol, which is a weekly protocol, these families love this therapy and are fiercely committed to it. As proof of that, week after week, month after month, year after year, the compliance rates for these therapies are well over 90% over that entire decade period. And the final thing to know about these therapies is now, having been on the market for a very long time and gathering a ton of evidence, there is no doubt these therapies are slowing the progression of disease.

Because they've been on the market for so long, there is an absolute wealth of published real-world evidence on the effect and the impact of these PMOs, and that the results are both consistent and they're diverse across organ groups as well. Look at this data. The PMOs increased survival by nearly five and a half years. All right? They delay loss of ambulation from anywhere three to four years. They reduce by years, delay by years, the need for nighttime ventilation. They slow pulmonary decline. There's a 50%-90% reduction in assisted ventilation. And look at this next one, a 78% reduction in the risk of reaching an LVEF of less than 55%. That means a slowing of cardiac decline that's directly correlated to mortality. And there's a 30% reduction in visits and hospital visits.

If you look at our recently released Essence results, you see the same thing. They are supportive of our real-world evidence. I'm not going to go over them again. We had an entire call on that, but I'll give you this one snippet. If you correct for the impact of the COVID pandemic on the study, the kids on therapy slowed progression of disease by 30% versus the placebo kids. We're going to take all of this information. We've already asked for the meeting. We'll have a meeting with the FDA by the end of this quarter, and one of the goals of that is to talk about the pathway from accelerated approval to traditional approval. Now, let's talk a bit about our pipeline. As you know, we're very, very excited about our next-generation siRNA pipeline. You can see it here. These are our clinical and preclinical candidates.

They don't include our research programs. The numbers on the far right here are U.S. prevalence numbers. So you can see an enormous opportunity. To contextualize this opportunity, in the U.S., the prevalence for Duchenne is somewhere between perhaps 10,000 and 12,000. So there's an enormous amount of opportunity here. And this understates the actual opportunity because these are U.S.-only numbers. We have global rights. So if you really want to understand the impact that we can have with these therapies, you're going to need to significantly multiply all the numbers on this page, all of which is to simply say that we have a real opportunity to do good by patients and ultimately, by doing that, to do well by our investors. As you know, all of our programs are founded on Arrowhead's TRiM platform.

We did a lot of diligence before we entered into this partnership with Arrowhead, and we were impressed by three things most broadly. The first was the TRiM platform's superior tissue-targeting ability. I'm going to talk a bit about that in a moment. We were very impressed with Arrowhead's expertise in building next-generation siRNA, and we were very impressed by safety, particularly as it relates to our latest programs, which are the muscle programs, and I'll talk a bit about that in a second. As it relates to muscle, this is the approach we're taking. We're using an Integrin-targeting motif coupled with siRNA. The reason that Integrin is chosen is because, versus other approaches that are being used, the Integrin receptor approach appears to result in much more significant muscle concentration, which could result in significantly greater efficacy and knockdown.

As I said before, we're very pleased by the safety profile of the Integrin receptor approach. In preclinical models for both DM1 and FSHD, we see very high NOELs, which, if they translate to patients, means we have a real opportunity potentially to be able to dose-escalate to get optimal efficacy in ways that other programs have not been able to do because they've been beset by dose-limiting toxicities. We're also very pleased with the siRNA approach versus other approaches that some other people are taking. That's simply because siRNA has proven itself over and over again to be a very, very potent modality. In fact, it takes something like 50 times more ASO to match the potency of siRNA.

It's for all of those reasons, and of course, it's all going to have to bear out in human clinical data, but it's for those reasons that we have hope that both our FSHD and DM1 program can be not only fantastic therapies, but potentially best-in-class therapies. Now, that's the muscle approach. We're taking a different approach with respect to the CNS because here we're attempting to get across the blood-brain barrier. And to do that, we're marrying a TFR1 FAB with siRNA. Now, there's a lot of reason to believe that using the transferrin receptor holds a lot of potential for crossing the blood-brain barrier. But what's also very clear from the preclinical data is the way you go about that is crucial to whether you're going to be at all successful. The first thing is the construct itself. You have to have the right construct.

On the far left, you'll see a divalent-binding TFR1 MAB. You can see here it locks into that receptor extremely tightly. Our preclinical data says that it will not cross the blood-brain barrier. Instead, it will just induce receptor degradation and recycling. We don't use that. We use a monovalent-binding TFR1 FAB, which our data says should have a receptor transcytosis and then delivery across the blood-brain barrier. Now, that's one of two issues. That's not the entire issue. So the construct's important. The second thing we believe is absolutely crucial is route of administration, and that's why all of our programs, particularly our FSHD and DM1 program, are formulated for subcutaneous delivery. And the reason for that is because preclinically, what we've seen is if you dose subcutaneously, you stay below the transferrin receptor saturation point, and you get constant and robust delivery across the blood-brain barrier.

But as you can see on the far right slide, and this is the actual data that we have preclinically, if you use an IV approach, you get almost immediate saturation of the transferrin receptor, which should greatly reduce the probability of getting across the blood-brain barrier, if not entirely restricting the ability to get across the blood-brain barrier. So we're very excited about the approach, and I'll give you at least a piece of data that tells us that we might be on the right track with this. And this is our Huntington's program, and this is the way we're going about Huntington's.

So these first two images on the left make one simple point together, and they simply say this: that trying to reach the deep brain, which is where you must be if you're going to make a difference in something like Huntington's disease, and if you're going to try to get there through an intrathecal injection, you're very, very likely not going to be successful. You just don't get a lot of therapy to the right place through an intrathecal injection. On the other hand, if you could cross the blood-brain barrier, you're going to have robust coverage exactly where you need it in the deep brain, and that's exactly what we're seeing. The far right is our data on 1005, which is our Huntington's disease program in the non-human primate.

Here we're seeing greater than 75% knockdown in exactly those parts of the deep brain necessary for changing the course of Huntington's disease. This is some of the greatest knockdown anyone's ever seen in a non-human primate model for Huntington's disease. We're obviously very excited about this program, but to conclude, we're very excited about this entire pipeline that we have access to right now. Let's talk a bit about where we are from a financial perspective. We're in a really strong financial place. We took a couple of important steps last year to ensure that we're on a strong financial footing. In the middle of last year, you'll recall we did a difficult but important restructuring of the company. We retained the expertise to continue to execute, and importantly, we prioritized the highest value program at the same time.

The second thing we did last year over the course of a few steps is restructuring all of our convertible debt or most of our convertible debt so that as we sit here today, right now, we have no significant debt overhang throughout this entire decade. I can tell you that I can give you some broad numbers. I'm going to be careful because our CFO, Ian, wants you to know these are all very tentative. We're still working through the numbers. But broadly, for this year, we will have profit. Again, I want to be very careful. It's tentative. Our non-GAAP profit, if you exclude the Arrowhead transactions and the payments to Arrowhead, would have been just about $400 million. That would be the second year in a row of positive profit.

And we would end the cash, again, absent the Arrowhead transaction, with an additional $330 million or so of cash. As we look forward into the year and then into the coming years of this decade, we anticipate being cash flow positive throughout. We anticipate growing our cash balance even as we fully invest both in our marketed therapies and in this exciting pipeline that we have. And then finally, on that issue, just remember we have an untapped $600 million revolver. And so we have a lot of strategic opportunity and optionality in front of us if we find something that we think would be in the best interest of patients and also our shareholders. So in a strong, strong financial position, we have a lot going on in 2026. There are a lot of milestones. There's a lot of readouts.

I am not doing full service to it with what I'm going to talk about today, but I just wanted to touch on a few of the things that we're doing. I've mentioned already before that we're executing Endeavor Cohort 8. That would be, if successful, our pathway back to treating the non-ambulatory patient population, and just at the very end of this year, we'll have those results. I would remind you that the non-ambulatory population is about 50% of all Duchenne. We are going to meet with the agency at the end of this quarter, talk about Vyondys and Amondys, and of course, we'll be talking about what that pathway might look like to transition those programs over time to traditional approval. Finally, we have a lot going on in the siRNA pipeline. I'll just talk about a few.

By around the end of this quarter, we'll have the biomarker data. We'll have the safety data, probably some other interesting data and evidence on 1001, which is our treatment for FSHD. We also, around the same time, we'll have the same sort of data for DM1. So those are two extraordinarily important programs for us and for the patient community. And then, well, we've already initiated the Huntington's program, and we'll be dosing patients in our Huntington's program in the first half of this year. So a lot of things to look forward to over the course of this year. Finally, let me reflect for just a moment on the year 2025.

When we entered 2025, given all of the successes that we had had leading up to 2025, given the fact we had four absolutely fabulous therapies that were already approved, we thought 2025 was going to be an easy year. Well, it wasn't. It was obviously not only a challenging year, but a few times there were absolutely heartbreaking moments in 2025. But this team that works for me never lost sight of their mission. They never failed to execute, and as a result, thousands of boys live better lives because of them. As we track into 2026, we're tracking into 2026 on a very strong financial footing. We have an enormous amount of opportunity in front of us, both with our approved therapies and also with our next-generation siRNA pipeline. And I look forward to talking to you all as we progress across the year. Thank you.

Anupam Rama
Senior Biotech Analyst, JPMorgan

Thank you, Doug. I'll ask the first couple of questions, but there will be an opportunity for the audience to ask questions as well. Doug, you talked about the challenges of 2025. As you sit here today, could you talk a little bit about where you are with the Elevidys in terms of the patient community and reinstilling confidence in the patient community after what was a difficult 2025?

Doug Ingram
CEO, Sarepta Therapeutics

Yeah. I mean, one of the biggest parts of that is really rebalancing. I keep saying doing a better job, and every time I say doing a better job, people like Patrick recoil because, of course, they were doing a great job in many ways in 2025. But in 2025, because of the issues we were dealing with, we spent all of our time talking about safety. And that was the right thing to do and the necessary thing to do. But one needs both sides of the equation. You really need to understand the efficacy of this therapy, and the efficacy of this therapy was getting lost sometimes in those discussions. There is an absolute avalanche of data on Elevidys. It is greater than almost any other program you can think about.

If you don't agree with me, just cast your mind to other programs that are exciting in the gene therapy world right now and ask yourself if they have as much evidence to support them as Elevidys does. So that initiative itself needs to play a role. Now, we can't ignore talking about the safety issues and making sure people understand where things are from a safety perspective. It's not as if we can just ignore that, nor would we want to. We're in good shape with that. First, let's remember what happened last year. There were two fatalities. They were liver failures associated with older boys who were non-ambulatory. You have to contextualize all that. We've dosed over 1,100 patients, okay? And so you have to sort of look at the numerator and denominator, but nevertheless, a very difficult situation.

The good news is a lot has been going on to take what is already a good safety profile and continue to improve it into a great safety profile. For instance, both in clinical practice and in our label, there's enhanced monitoring that's going to happen on a go-forward basis. One of the great things about that enhanced monitoring is it's not very additionally restrictive because really it's just more labs, more important thoughtful labs rather than more instances of monitoring time. That's going to be very, very helpful to make sure that physicians have good insight along the way. The second thing to know is that in the label and in practice, there is more aggressive reaction to labs and the like. We have that in our label. Physicians independently have been looking at that in their practices.

That's going to play a role in increasing safety. There's another thing that we know. It's something that we can't promote to, but we can monitor and see what's going on, and as it sits here right now, about 25% of sites already are beginning to use sirolimus, and that seems to be growing, and while I don't want to get out ahead of our skis, we still have Endeavor Cohort 8 to complete before we are fully confident on the use of sirolimus. There is some data out there. Dr. Soslow presented data on a small cohort of patients that he dosed prophylactically with sirolimus, and it looks very good, very promising, so there's been a lot of things to enhance what is already versus other serious gene therapies, a relatively safe profile.

And I think you marry that up with efficacy, and we do a really good job of talking to people about that, and I think we motivate folks.

Anupam Rama
Senior Biotech Analyst, JPMorgan

Questions from the audience?

Hey, Doug. A patient advocate that has consulted with you met with RFK in December, posted on social media, said that Kennedy committed to not, I'll just read the quote, "Senator Kennedy committed that young men and boys like my son will not lose access to approved exon-skipping therapies." She was talking about Exondys, Vyondys. Have you heard that from RFK? Have you gotten that indication from anyone at the FDA?

Doug Ingram
CEO, Sarepta Therapeutics

I haven't had a—no, the answer for me is no. I haven't had direct communication from RFK or the FDA on the topic. I did see the same post that you saw. First thing, understand the background of that, which I think is very positive as well. And I think HHS deserves credit in this situation, which is that HHS and RFK directly appear to be very close to the Duchenne patient community themselves, and as a result of which announced a little while ago, I think before the end of the year, if I'm not mistaken, that Duchenne newborn screening would be added to what's called the RUSP, the Federal Registry, and then we'll roll that out across the states, which is an absolutely wonderful thing for them to have done that speaks to the people caring about patients.

So it does not at all surprise me that RFK would say that because it would be quite illogical to be going out over your skis to get newborn screening in place simply to take away the very therapies that could benefit patients with that newborn screening. And again, I would think that's a brilliant answer. The patient community loves these therapies. The real-world evidence, in particular, is clear that they're bringing a better life to them. They have an extraordinary safety profile. It would be an unusual thing to want to mess around with that from our perspective. So I was thrilled to see that post. I wasn't exceptionally surprised by it, but I was thrilled to see the willingness of RFK to show leadership in the patient community.

Thank you.

Maybe following up on that question on the uniform screening panel, what type of impact could this have on clinical considerations and the space overall?

I think it's going to be. I think it's really valuable, first of all. And it says a lot. And if you're wondering, for anyone who's uninformed, it is hard to get on the RUSP. It is one of the most insanely bureaucratic approach processes that can exist, and not a lot of therapies have gotten on RUSP. So it is a big deal that Duchenne muscular dystrophy has gotten on the board. So in the long run, it's going to be extraordinarily valuable for patients because we know that if you intervene early, you're going to do a lot more good. All of the therapies, starting with ours and any other therapy, frankly, including to the best of my knowledge, any therapy in the mind of a scientist today does not work by reducing damage already done, but instead stops further damage. So it is a race against time.

And if you can get newborn screening in place and you can intervene early, you can stop the damage. These kids in utero are being damaged. If you did a muscle biopsy on a boy at birth, while you would not know he had Duchenne, you wouldn't be showing it yet. You would see it in his muscles. You would see it in the muscles. The muscles are already showing damage.

Anupam Rama
Senior Biotech Analyst, JPMorgan

Speaking of.

Doug Ingram
CEO, Sarepta Therapeutics

Oh, sorry. So it's going to be - I got excited. I should have - I told you I need to allow you to.

You need space for your hands.

So now, let me say it's not going to be overnight, okay? There's a couple of things to know about newborn screening. So the first thing to know about newborn screening is that it has to be implemented state by state. So there is time administratively that goes into getting the newborn screening implemented on a state-by-state basis. We have a team working on that, a wonderful team that's been working on that for a long time. The second thing to know is that we can dose boys four and over right now. That's where we stop. So for us to be able to go out and really talk and communicate and promote to the under-four-year-olds, we need to get our label below four. Good news is we've got great data on the boys below four years old.

The safety looks wonderful, as you'd expect, in this age range, and the expression is simply off the charts. So we hope to, at some point in the not-too-distant future, talk to the agency about getting the age limit lowered so that we can benefit these kids with the newborn screening initiative.

Anupam Rama
Senior Biotech Analyst, JPMorgan

Maybe questions from the audience?

Oh, yeah, one.

Thank you. Thinking back to Sarepta's discussions with the agency and considering how ambulatory and non-ambulatory patients are different phenotypically, obviously, what's the FDA's stance now in terms of the flexibility of outcomes to expand therapy to the non-ambulatory kids? And then how to best think about combination therapies as development progresses in DMD, especially along the lines of cardiomyopathy and neuromuscular function?

Doug Ingram
CEO, Sarepta Therapeutics

Okay. On the issue of non-ambulatory, so we're not dosing now because of these two ALF cases. We have a lot of hope that the prophylactic use of sirolimus could greatly reduce the incidence even of ALI, which means it would probably greatly reduce any theoretical risk of another ALF. That's our pathway back to having discussions with the agency about getting these kids back on the therapy. It really isn't an issue of efficacy or the like. It's an issue of ensuring that there's the right risk-benefit there, and that's going to come out of the success, if it happens, of our Endeavor Cohort 8. On the issue of combination therapies, I mean, I think that Duchenne is a very, very difficult disease, even with the greatest therapies, and I am biased. I think Elevidys is the greatest therapy that exists, certainly the greatest approved therapy out there.

They're not cures. They do great things, but they're not cures. And I think there's a lot of room for combination therapies to address many elements of what is a complex and difficult disease. So I think combination therapies is actually quite a brilliant idea.

Maybe final question from me here, just on the corporate finance side, just thinking about the levers to meeting your debt obligations later in the decade, and actually, on the top line, how do we think about the key revenue contributors to consider?

Yes. Thanks for the question. So for the rest of the decade, obviously, we've taken action to remove any debt overhang. And so conservatively, even with our DMD franchise, we feel we're in a great position to fund our investments and to meet those obligations. On top of that, if we're successful with our clinical programs, we do expect to have siRNA revenue at the end of this decade. But again, with our near-term viability now removed, we're in a great position to fund our initiatives and move our strategy forward.

I should say one other thing that I hate talking about, but Ryan loves talking about, which is we've done a stress test. We've done a lot of different stress tests, including even removing the PMOs. What you would find is that while that would be painful, be horrible for patients, and we'd have to tighten our belt, you'd still be able to address your debt. Did I get that wrong, Ryan?

No, that's correct.

I'm confident that's not an issue, but over my gritted teeth, they did that analysis.

Anupam Rama
Senior Biotech Analyst, JPMorgan

Thank you, Doug and team.

Thanks.

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