Conference Transcript - SRPT 2026-06-08

Speaker 5

Great. Good morning, everyone. It's my pleasure to introduce the Sarepta team. With us, we have Doug Ingram, CEO, Ian Estepan, President and COO, Ryan Wong, CFO, and Louise Rudino-Kleipak, President of R&D. Thank you for joining us. Maybe we'll start here, Doug, with a question for you. You've announced your retirement as CEO by the end of the year. How are you thinking about working through the transition and the overall succession plan?

Doug Ingram, CEO

You can ask these folks if I've been winding down recently. I think they're surprised my intensity level's been going up. You know, right now we're not transitioning anything. We've got a lot to do as an organization. I did announce that by the end of this year I will retire. There's an active process ongoing. When the board makes its final decision on that, then I will obviously do everything in my power to ensure that that individual is maximally successful, both for the company and its investors and for patients as well. So I stand ready to do whatever they want for as long as the successor would like to ensure that we're successful, and I'm very confident that we will be.

Speaker 5

You have laid out your priorities for 2026, notably stabilizing Elevitus, advancing the pipeline and strengthening the financial setup. To start here, can you lay out the progress towards these priorities and what the key milestones for the company will be over the next 12 to 18 months? Sure. Right.

Doug Ingram, CEO

To your very good point, the strategy at Sarepta is quite straightforward. Let us start first with our four approved therapies. We have three PMOs, as you know, and our gene therapy, Elevitus. The goal there is to maximize that opportunity, really first and foremost, for the patients who benefit from that therapy because even in our base case, we're going to do quite fine. But we would like to exceed that base case because that means even more children and young men with Duchenne Musco District who are going to live better, freer lives because of it. And we're doing very well there. We'll come back and talk about that. Let me just talk about each pillar. That really leads to the state of play in the company from a financial perspective. We're in a really strong financial perspective. Just consider that we're cash flow positive. We were a little over $800 million, I believe. At the last quarter, maybe we were $750 million.

Francesca Nolan, Head of Investor Relations

$750 million will be $900 plus.

Doug Ingram, CEO

Yeah, so we'll be significantly over $900. So we're cash flow positive. We're profitable, usually on a gap and a non-gap basis. That will continue to be the case over the course of the intervening years, even as we fully invest in our pipeline. Just consider our sales forecast. We've said that we will do somewhere between $1.2 and $1.4 billion. Even if you assumed we were at the very bottom of our guidance, our cost structure is somewhere in the hunt of a little over $800 million. So we're just a comfortably cash flow positive organization that isn't, thank goodness, given where our stock price is today, which, if I can editorialize, is surrealistically goofy. In one sense, other than employee morale, it really doesn't matter because we are not slaves to the equity markets to get our stuff done. And then, of course, where are we going in the future? We have a really exciting pipeline. Our SIRNA pipeline is exciting. We have five therapies that are in the clinic dosing patients right now. We have a few more that are heading to the clinic soon. We have six research programs on top of that with our really exciting period of time. Now let's talk about success in each one, and I'll do it real quickly, and then we can actually get to Q&A. So I'm not just, I don't get this, you know. They were taking bets on whether any of them would have a chance to talk today. I'll be brief. From a sales perspective, the delta between 1.2 and 1.4 is really entirely a levitus, an levitus success. We've always said, for the course of this year, you should really be modeling toward the lower part of the 1.2 to 1.4 for the simple reason that the actions that we're taking and the activities we're doing are going to be successful, but this is a very long cycle of process. It's going to take some time. We've done great there. We've more than doubled our sales force throughout and being very productive, both from a reach perspective and a call perspective. That's going well. our promotional campaigns themselves are really powerful right now. We have a contract sales force in the field, and we have a group of what we call the patient education liaisons, whose goal it is to really educate patients and make sure they're prepared to have thoughtful discussions with their physician about the journey for, particularly for Elevitus. That's going well. Tons of green shoots of success there. I could give you interesting stats on that. That is going well. We're doing great financially. And from an SIRNA perspective, you saw that with DM1 and FSHD, we had very encouraging results from our SAD study that we announced a couple of months ago. We're really excited about getting a look at the next set of data, which is going to be even more robust. It'll be multi-ascending dose. It'll be more patients and more myopsies and the like. And that'll happen in the second half of this year. We've already started dosing our Huntington's program, which we're excited about. IPF is dosing. SCOT2 is dosing as well. So we've made a lot of progress across that portfolio as well.

Speaker 5

Can you touch on the leadership changes at the FDA and whether there's any risk to you from that dynamic?

Doug Ingram, CEO

As we say today, we see no risk at all from changes at the FDA for two reasons. First of all, let's think about where we are with a levinist. We had a bunch of risk last year for abandoning our stock in the summer of last year. We had a really strange interaction with the FDA where they were pressuring this therapy off the market. Notwithstanding the fact that we were able to get that therapy back on the market within five business days, I would suggest it would be very difficult for someone to replicate that success. it was understandable that people were worried about that relationship. But from there, things just got very good for us. We negotiated the label, we negotiated a label update with the FDA. That got out there to the physicians. We negotiated and got their blessings on cohort eight, which is our pretreatment with sirolimus, which will be the pathway back for the non-ambulatory population. So there really aren't any open, extant, significant risks in the 11th. So even with a change, it doesn't matter. On the PMO side, that really didn't exist as an organic risk anyways. And, you know, I think that what we're seeing so far in the changes at the FDA have been very positive. I think the acting commissioner, I have not interacted with him directly myself, but really universally people are saying very good things about him. He comes from the food division, so he may not be steeped in the drug development process, but he understands how to run large organizations. He's apparently very rational and very reasonable, and everybody that's interacted with him so far has come away saying he's a good, steady hand at the FDA. I think the same thing has been said about the CEDAR side as well and that change, and I don't know much about the CEPA side. So I think there's a lot of hope for a stable FDA, and that's what we need. You know, I have been a big proponent for many years over the idea of, you know, really trying to modernize the FDA and get more thoughtful and mirror the FDA today with the current state of science and certainly in genetic medicine and rare disease. What we really need more than anything else right now at the FDA is a stable and predictable,

Speaker 1

I'm very excited.

Francesca Nolan, Head of Investor Relations

It didn't seem like the markets really fully discounted both the risks that I think were happening previously and now I think a very strong tailwind now because of that exact point that you're making in terms of predictability. I think they're making dramatic improvements in trying to make sure that goalposts don't get moved, reviewing applications that previously had gone against what the agency had communicated previously and now actually factoring that in. And hopefully we'll see in the next few months what happens with some of those applications that are getting reviewed. But I think that stability and the ability to be able to predict what's going to happen should be really important for the investment community. So I think it's a much better backdrop.

Doug Ingram, CEO

In the end, let's be very clear, this is what we want as an organization. We want the FDA to apply the standards that exist at the FDA and to review therapies in the totality of the evidence and to support those therapies where it makes sense to, but not to support therapies where it doesn't. So we're not looking for some cowboy country. We want an organization that applies standard and rigor, And I think there is some hope for that, given some of the new leadership at the FDA, at least on an interim basis.

Francesca Nolan, Head of Investor Relations

And that standards part is incredibly important, because if you don't have a standard, then payers start weighing in and other factors start contributing to whether a drug gets utilized or not. And so making sure that the standards are met and that the safe and effective therapies are going to patients is just critically important.

Speaker 5

Starting with Elevitus here, you've spoken about the information gap regarding Elevitus and your initiative to drive penetration in the ambulatory patients. What are priorities for the expanded field team, and what leading indicators are you seeing with physician engagement that suggests a return to growth?

Doug Ingram, CEO

So the number one overarching thing was to rebalance the discussion, if there wasn't even a discussion. Remember, in 2025, we had two surprising and unfortunate fatalities associated with non-ambulatory, more advanced patients, and we spent the entire year focusing only on that, not talking about the benefits and the risks of the therapy, and contextualizing those risks, because it could really, I would remind you, it's horrible for those families, But two out of nearly 1,400, I mean, it's 0.12% of a percent. And so that's the big thing, to get the promotional material in the right place where we can have smart, thoughtful discussions about that. That's what they're doing right now. That's what the main sales force is doing. And then we have a contract sales force that's really trying to expand that reach into places around the country that are not the top centers but have opportunities for referral, have opportunities for neuromuscular physicians to get educated that maybe have never been educated before so they can have thoughtful discussions. And that's the big focus there. There's lots of interesting what Patrick Moss, our head of commercial, likes to call the green sheets of success, quite apart from the numbers of people in the field. you'll see something like 50% of start forms are coming from sites where a sales rep had visited that site within the last 60 days. You're seeing a significant percentage of sites, I think it's over 30% of sites that had either paused back last year or had never dosed a patient starting to send in start forms without getting into too much. violated, but there's one site that has enormous opportunity, but it's been very slow, and they've actually put in multiple start forms over the course of the last couple of weeks. So that doesn't show up immediately in sales. Remember, it's a six-month process to go from start form to infusion, and it can take even longer with the kinds of appointments you need to have and testing you need to have and antibody testing, but it's a great sign for the future.

Speaker 5

With regard to the total revenue guidance you spoke to, the 1.2 to 1.4, we estimate about 300 to 500 of it coming from Elevitus based on historical performance of PMOs. What are the factors that influence whether or not you fall in this range?

Doug Ingram, CEO

It's all Elevitus, and it's all the impact of our – and there's still more to do. So, for instance, you know, we have this, we knock out three-year data on Elevitus. And if you look at the three-year data and you're not thinking about getting your kid dosed, you just don't understand what you're looking at. That actually has to get into the promotional material. That's not even yet in the promotional material. When we do ad boards and we share it with physicians, the impact for there is just obvious. So we need to get that done. But it's all bad. And it's just that the delta there is just that, you know, moving this up with such a long-cycle therapy is, you know, to use an absolutely worn-out bromide is turning a tinkership, so it's going to take some time. So I'd suggest a lot of the activity we're doing right now is going to go down to the benefit of 2027, not 2027.

Speaker 5

And what does the FDA need to see from Cohort 8, is that right, the Ceruloma study to include ambulatory patients in the label, and what could the timeline look post the top-line data here in the second half?

Doug Ingram, CEO

I'll turn to Luisa who can talk about the study itself.

Louise Rodino-Klapac, Analyst — Other

So Cohort Aid of Endeavor is an open-label study in non-ambulatory. So this is about the non-ambulatory population. And so by definition for the study, we're looking for a 50% reduction in the rates of ALI. That's what the FDA will be officially looking for, as will we. It's open-label, so we have the ability to look at the data throughout. And based on the mechanism of action, all the tremendous real-world evidence we have with physicians using it in the field, we would predict that we would certainly achieve that. So we'd go back to the FDA after the end of the year, talk to them about bringing the patients back from the non-ambulatory patients back to the label and what the mechanism would be to do so.

Doug Ingram, CEO

It could. I think the, if I'm not mistaken, I think success in the protocol is a reduction in the risk of ALI. Remember, ALI, if you reduce ALI, you're going to dramatically reduce even the theoretical risk of ALF, which is the ultimate thing we would worry about. I think it's by 50%, if I'm not mistaken, that would be the goal. Now, we'll see how this goes, and we'll get to watch this data over the course of the year. We do have data right now. We're thinking about, we discuss it. We have something called Endure, where we get to collect data on patients in a more formalized fashion as they roll out of the study and as they're on commercial therapy, and they're on commercial therapy as well. And there have been somewhere in the hunt of 11 or so to 14 patients that have been prophylactically dosed with serotonin. Now, I don't want to suggest that this is the standard we have to meet. I don't want to create too high a standard for us, but so far in all of those patients, there has not been any, an increase in liver enzymes. So it gives us, it's going to be, and for those who may wonder why we care so much about this, I mean, look, if you want to be, you know, just dollars and cents, you'd say the non-ambulatory patient population represents 50% of Duchenne. it is in fact an enormous opportunity but there's a more important issue for us than that and that is everybody abandons the non-ambulatory patient everyone always is abandoned really nobody has ever and it's going to get worse if we don't win with this if we can't safely dose i posit no one will ever do it everybody's going to walk away from every current therapy and development right now is ignoring the heck out of these people so you know so the good news is there's a nice synergy there. It's going to be great for investors if we're successful. It's going to be great financially, but it's going to really be great for people that desperately will be left behind simply because they're in a wheelchair.

Francesca Nolan, Head of Investor Relations

And I think it's important we've seen about 20 to 25 percent of sites dosing the ambulant patients with serolimus also. And obviously this is something that's not on our label, so we can't promote to it, but this is how the field is evolving based on the experience that we're seeing, which I think is important to the investment community also, because if you're able to just limit the risk in the ambulant patients, I think that's obviously incredibly important from a patient perspective, but also from an investment perspective, if you're able to cut that risk. So hopefully if the data continues to emerge and is very supportive, you continue to see that adoption.

Speaker 5

And your partner, Roche, announced a new global Phase III trial here. Maybe talk about the read-through from that to your commercialization plan, but also why Roche has taken this viewpoint to kind of invest more in the program.

Doug Ingram, CEO

Well, I think a couple of things. Well, first, they have to do another study. They have taken scientific advice. So with EMA and CHMP, they realize that that is the pathway. And I would say the why they're doing it and investing is because they've seen the data that we've seen. And I think that our colleagues over at Roche are as confident as we are about the way that this therapy is changing the lives of patients. They've done quite well at the XUS. That gives them a really on-the-ground opportunity to see these patients up close and personal. And I think they realize the life-changing benefits of this therapy. and I give them kudos for understanding that and appreciating that. The study itself won't have any read-through to us. It's a placebo-controlled blinded study. It'll take some time to read out. It seems like they've been fairly thoughtful in the approach they've taken. They've learned a lot from the work that we've done, so I'm confident in their study and its outcome. It'll just give additional evidence in support of this great therapy.

Speaker 5

Maybe transitioning over to the Arrowhead platform here or your partnered or your acquired Airhead platform. Regarding the CERNA programs, can you lay out how your constructs differ from the others here, such as Davidity and DINE, and how you see those differences play out in the early data in FSHD, but also DM1?

Doug Ingram, CEO

You know I want to do it.

Louise Rodino-Klapac, Analyst — Other

You do it. So focusing on DM1 and FSHD, which are two advanced programs. So we use a targeting ligand called AlphaV Beta 6, and that's really the differentiator for these two programs, which are delivered IV. And really that gives us the ability, and why we did the deal in the first place, to be most efficient in muscle, getting into muscle, driving that siRNA in there. We know that siRNA itself is more potent in terms of the effect within the cell. You need 25 to 50 times more molecules of an ASO versus an siRNA to have the same effect within the cell. We also have a large safety margin, so 10X. And so what we've seen from our MAD data, from our SAD data, and now going into our MAD data is the ability to continue to dose up, which is important for getting maximal knockdown and then downstream biological effects within the cell. So stop there if you want to add. Well, I disagree with you.

Speaker 5

As you move to higher and multiple doses, how are you thinking about receptor saturation or potential for dose-limiting toxicities?

Louise Rodino-Klapac, Analyst — Other

So preclinically, we've not seen any evidence of receptor saturation. And again, this is where the alpha-V beta-6 comes in. We don't have the same problems with the TFR that others have because of the use of that receptor for iron homeostasis. So we're not seeing things like anemia like others are seeing. So that really gives us the opportunity to continue to do stuff, and we haven't seen that saturation that others may have.

Doug Ingram, CEO

Our annual is significantly above where we're actually looking at.

Speaker 5

How much proof of concept do you think that initial data represented and can you frame how to think about translation to the next data release and how the overall profile could ultimately land? And I think part of what I'm also getting at is did you show a selective group of patients here that we can take that overall view?

Doug Ingram, CEO

Yeah, selective sounds like cherry-picking. We didn't cherry-pick. Let me be very clear. We were really excited about the data, and we'll talk about the limitations of it. We're very excited about that data, and the reason we were so particularly excited about this early read is that it was very confirming of what one sees in the preclinical data. That's exactly what we had hoped we would see, which is the ability to dose escalate when others can't, the ability to use the integrative receptor to drive up significant muscle concentration, even at equivalent doses, and then the ability to continue to drive it up, and then the resulting knockdown that we saw in the early side. So there was really nothing in that early data that didn't completely confirm what we saw in the preclinical models. Now, you know, we have to be thoughtful about that. It's early data. It's just a single ascending dose. So it gave us a ton of confidence, and now we need to see the MAP data. We need to see it in multiple doses, over time, in a larger cohort of patients, over other dose ranges. We'll have that in the second half of this year. Now, if that looks great, then we are really heading toward a very strong possibility that we will be there. But I would say we need that. You know, I wouldn't overinterpret the first one other than to say, oh, you know, it's really comforting that it was exactly what the time was.

Speaker 5

And with that second, I guess, the six-month update in the second half, could you just frame what you'll show in terms of patients at each dose, biomarkers, early functional data, and what you see as relevant benchmarks?

Louise Rodino-Klapac, Analyst — Other

Sure. So we expect to have, as you mentioned, six-month data in both FSHD and GM1, and the timing of that we'll see. So at our higher stosis, as Doug mentioned, this is the MAD data. So, again, we'll be looking for safety, target engagement, PK, PD. In terms of FSHD, we'll be looking for the test scores, target genes, circulating biomarkers, early evidence of function, six months, not a long time in FSHD, but we'll be looking at that. In DM1, then we'll be looking at TNPK knockdowns, CASD22 splicing indices, and then B-HOT, which we can't see at an earlier time point. So we're excited to see this data. As I mentioned, having multiple doses at higher concentration, I think we're looking forward to seeing.

Speaker 5

And if all's good with this data release, you've talked about starting at these three posts. Maybe talk about those timelines.

Doug Ingram, CEO

Yeah, I mean, let's be realistic about what we can and can't see with these heterogeneous diseases. People shouldn't over-index on some functional endpoint that relies upon degeneration. We need to be realistic. These are slowly degenerative diseases, and I think we're all so excited about seeing something as soon as possible that you want to look at something in six months that would actually take a couple years to see in the placebo on lifting groups. So just to be thoughtful. That's the beauty of VHOT. A lot of complaints about VHOT. VHOT may not be a great model for predicting other declines in a short period of time, but it is clearly a functional manifestation of a benefit or lack thereof and it can be done very soon because it's not a degenerative issue, right? Myotani is not degenerative in nature. So let's just, you know, so really if you're going to focus on the future, what should you really care about? You should care about safety. You should care about the ability to dose escalate with safety. You should care about muscle concentration and knockdown and downstream splice correction. Like those, that's the big one. And then going beyond that, there may be other biomarkers that will be fascinating. Then there's VHOT. And then I would say, yeah, the rest of it's explored. I think with respect to what, we're going to move as fast as possible. Now, that's the short answer. We're going to move as fast as possible. I don't think that we, one of us today, because we've got to do more work. We heard just the other day that we're suggesting that they are going to go for an accelerated approval. And I think it's a very interesting idea. And I think it is certainly not an irrational. old. So we're going to take on all of that thinking and we're going to fashion for ourselves the fastest possible approach. That will be our goal. But we need to get through some more of this. We are clearly going to start, as an example, we're going to start pivotal trial. And then what the exact pathway is to approval. Is it the end of those pivotals? Is it an interim accelerated approval on a well-confirmed biomarker? Those are things we'll have to figure out. And then we'll to figure out in concert with our regulatory company.

Francesca Nolan, Head of Investor Relations

So just one thing to follow up, which Doug alluded to it, but just to say it more directly, knockdown is, to Doug's point, the most important thing to see because function will follow. You shouldn't have this toxic protein or toxic mRNA in either one, in DM1 or FSHD, in your body. So if you're able to knock that down most significantly, that is eventually going to lead to the best functional outcomes. So whether you're able to see it in a heterogeneous disease in a very short period of time, that's challenging. However, knowing that the best knockdown is going to lead you.

Doug Ingram, CEO

If I see in the multi-ascending dose confirmation of what I see in the single ascending dose, I'm not sitting here. Confer what is obvious from good scientific analysis. And I'm sure that's where other people would be, and I would hope that's where FDA colleagues would be. That's why I currently think dying is. Can you just speak to how you intend to leverage your teams

Speaker 5

and infrastructure from the PMOs and Elevitas for a potential launch here?

Doug Ingram, CEO

I don't think we've – I think we're going to have to do a lot more. We've got a great team, and there's tons of parts of our team. We are neuromuscular experts as an organization, so there's going to be a ton of leverage there from a marketing perspective, a promotional perspective, Salesforce perspective, MSL perspective, Our PELs, there's going to be a ton of opportunity, but it would also be a mistake to be arrogant and imagine that there's no adaptation needed or no augmentation needed. We are as prepared to launch in VM1 and FSHT and the unique elements of those different diseases.

Speaker 5

For your PMO franchise, speak to your confidence in the applications for the, you know, in the context of the confirmatory studies, but also how you strategically are thinking about the runway here in the context of competitors.

Doug Ingram, CEO

Yeah. I think, first of all, as you all know, we've got SNDAs that have been filed, submitted, Mondas and Biondas, to transition them to traditional approval. We are, at a minimum, confident, based on our own analysis, that these, just to remind you, we had signals in our confirmatory trial. We have great real-world evidence. That's the beauty of having these their accelerated approval perspective for so many years, you get to actually see, not in some stilted, you know, short-term experiment, but over a long-term, what's really happening here, and they are significantly benefiting patients, keeping them out of a wheelchair and ambulatory and off the vent and out of emergency rooms and out of the hospital and a great mortality benefit across all these modalities, you know, that's exondus and my on this and I'm on this, so we think the data for it's really powerful. I would remind you, patients love them. We've been, you know, some of these patients have been on for over 10 years, and they fight to stay on them. We have a well over 95% compliance rate on those physicians also love them, and they're safe as hell. I mean, they have a really laudable year, so it would be patented to deny these. With respect to competition, the primary competitor that we have, I will have a therapy that will compete with Exondis next year. They're going to seek accelerated approval, and in that context, I think that's rational for them to do that. And I think we'll be prepared for that competition. We have not focusing on them, but focusing on us. We have a lot of things to say about our Exondis therapy path. Families have been on it for a long time. We have a 10-year safety record. We have great patient services. We know how to get these kids on therapy, keep them on this therapy. We do in-home infusions, so we make life easy for them on the therapy. And then, of course, you know, DINE will have its arguments, and it will be a very credible, I think, competitor as well.

Speaker 5

Well, with that, thank you so much.

Francesca Nolan, Head of Investor Relations

Thank you. And we made forward-looking statements, so please just look at our SEC followings for this associated with that. Sorry to end on such a...