Sarepta at BofA Conference: Navigating Challenges and Growth

On Wednesday, 14 May 2025, Sarepta Therapeutics (NASDAQ:SRPT) presented at the BofA Securities 2025 Healthcare Conference. CEO Doug Ingram shared insights into the company’s mixed Q1 results and future plans. Despite missing expectations and lowering guidance, Sarepta showcased robust growth and a strong financial position amidst industry challenges.

Key Takeaways

• Sarepta reported Q1 net product revenue of $612 million, a 70% increase year-over-year.
• Revenue guidance was adjusted due to safety events and operational delays.
• The company anticipates $2.3 billion to $2.6 billion in revenue for the year.
• Sarepta plans a BLA submission for LGMD 2003 later this year, with potential approval in 2026.
• CEO Doug Ingram expressed confidence in Sarepta’s long-term prospects and financial health.

Financial Results

• Q1 Net Product Revenue: $612 million, a 70% increase from the previous year
• Elevidys Revenue: Approximately $375 million, marking a 180% growth
• PMO Revenue: Around $235 million, a 5% growth
• GAAP Operating Income: Approximately $2.75 million, reflecting 70% sequential growth
• Non-GAAP Operating Income: $335 million, representing 45% of sales
• Yearly Revenue Guidance: Between $2.3 billion and $2.6 billion, with projected profitability and positive cash flow

Operational Updates

• No observed delays in gene therapies or changes in FDA perspectives post-Peter Marks’ departure
• Ongoing enrollment for the post-marketing study of EXONDYS
• Challenges include a fatal safety event with Elevidys and prolonged cycle times impacting revenue recognition
• Focus on secondary and tertiary sites to improve patient access

Future Outlook

• Revised revenue guidance due to safety and operational challenges
• Expected revenue increase in Q3 and Q4
• Planned BLA submission for LGMD 2003 later this year, with possible approval in the first half of 2026
• Opportunities include managing Elevidys safety impacts, shortening cycle times, and expanding site capacity

Q&A Highlights

• Executive Order Impact: Minimal expected impact on Sarepta
• Manufacturing: All products are U.S.-based, minimizing tariff exposure
• FDA Interactions: Positive and ongoing discussions
• ESSENCE Readout: Anticipated in early next year, with study completion in the latter half of this year

Readers are encouraged to refer to the full transcript for more detailed information.

Full transcript - BofA Securities 2025 Healthcare Conference:

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: And here at the Bank of America Healthcare Conference, I’m Tazeen Ahmad, I’m one of the senior SMIT Biotech Analysts at the firm. It’s my pleasure to have our next presenting company with us, Sarepta Therapeutics. Here for Sarepta this morning is CEO Doug Ingram. Doug.

Doug Ingram, CEO, Sarepta Therapeutics: Thank you.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: Good morning.

Doug Ingram, CEO, Sarepta Therapeutics: Thanks. Thank you very much for having us.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: For making the trip over.

Doug Ingram, CEO, Sarepta Therapeutics: Thank you.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: What’s been going on lately?

Doug Ingram, CEO, Sarepta Therapeutics: It’s been pretty quiet. So a couple of things. First, I have to say my IR folks are giving me a dirty look. I’ll be making forward looking statements today, so please look to our public filings for that. Look, before we get off, there’s going be a lot of interesting Q and A.

We’ll talk about guidance and consensus and the like, and I don’t want to, you know, hide from that. But I do want to contextualize it a bit and talk a bit about our performance in the first quarter, putting to one side the fact that we did not meet our own expectations and we lowered guidance. Nevertheless, I would like to remind us that we did about $612,000,000 in net product revenue for the first quarter, and that is 70% growth over the prior quarter, prior year. Elevatus, while we missed our expectations, it still grew at about $375,000,000 It grew over the prior quarters, you know, same quarter prior year at about 180 percent. Even our PMOs continued to grow even in the face ofElevitas grew at about 5%.

I think they did about $235,000,000 if I’m not mistaken. And more important than that, this is missed in all of this. You know, our GAAP operating income stood at about $2.75 or so million dollars for the quarter. That was growth over the immediately sequential quarter of about 70%. And on a non GAAP basis, we grew at something like $335,000,000 which I would remind you is about 45% of sales, which is pretty strong financial performance.

And the reason I mention all of this is that we’re in a very difficult place as a biotech ecosystem right now. Let’s be very direct about it. The market is nearly uninvestable right now. It’s in chaos right now, and that’s a real problem, and we’re going to need to find our way out of that. And if you think about it right now, I think it’s about 30% of all public biotechs today are trading below their enterprise.

Their enterprise value is below their cash. Another 32% or 35% are trading at about $100,000,000 or less. So 60%, sixty five % of all biotech is trading somewhere between $100,000,000 enterprise value and literally less than zero from an enterprise value. And I got bad news, there’s not a drug that I know of in the world that can get developed and approved in The United States alone for $100,000,000 which means unless these markets open back up and people can start accessing the markets, of which well over 90%, think it’s probably upward of 98% of all biotechs need to get to the equity markets to continue to fuel. That means an enormous number of companies are going to go under, and they’re going to take with them programs.

Some of which will fail, but some of which would have really added significantly to healthcare in The United States and brought a better life to patients. That’s a problem, and there’s no Chaddon for it in that from my perspective. But I do want to contextualize that we are, while we missed guidance and I wish we hadn’t and while we changed our guidance and I wish we hadn’t, we are in a different place than other biotechs right now. I would remind you even with our guidance going down for the year, that’s still $2,300,000,000 to $2,600,000,000 this year. We are going to be profitable this year and cash flow positive this year.

So, we have a really strong financial position to weather these difficult times. We have a really exciting pipeline. Hopefully, we’ll have some time to talk about that, both in the gene therapy side and gene editing, but also in the siRNA side. And we have the ability to fund that pipeline without being slaves to the public markets or having to do equity raises or the like. We can do it from our own resources.

And finally, I should note, I think we have some of the best and most dedicated folks in the industry that know how to execute and drive forward. And I have a very low attrition rate, so we have a lot of great people doing a lot of great things. So just to contextualize it, but I’m sure the next thing we need to talk about is guidance.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: Well, we will talk about that, but I just wanted to open with some more macro related questions because every company I cover, not just Sarepta, is now having to think about, like, broader stroke items. So let’s start off with the most recent update, which was the executive order that was announced on Monday talking about most favored nation. Wow. Right? So can you, Doug, give us your interpretation of what that ultimately means and how Sarepta might be impacted by that?

Doug Ingram, CEO, Sarepta Therapeutics: Well, so it’s obviously very new, and I think, you know, probably brighter minds than than mine have looked carefully at it and will have more views on it. I think there’s still some ambiguity in what that’s going to mean. I don’t think it has a significant impact on Sarepta even extended, for instance, and I don’t think it currently does. I think there was a theory it was going to extend to Medicare. It really wouldn’t impact us.

Our direct sales are primarily in The United States today, and we have very little Medicare as part of ours. We have a lot of Medicaid, but very little Medicare. So, we’ll all be watching to figure out how that unfolds and what it means for the industry and for patients in The United States, but I don’t think it’s going to have a significant impact on Sarepta, at least on near term.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: Okay. And then secondly, for manufacturing, can you just remind us where your products are made?

Doug Ingram, CEO, Sarepta Therapeutics: Yeah. So all of our products are made in The United States. So obviously, considering the overhang and some of the ambiguity that’s associated with tariffs, we’re still doing analytics on that. We do get some material outside of The United States, but it will have a very modest impact on this company. It’ll be a small percentage impact even on cost of goods.

So it probably wouldn’t be anything that anyone external to Allergan or to Sarepta would even say. You mentioned that company earlier. So Sarepta would even notice. So we’re fairly immune, which is interesting because I think most of biotech is in a favored position with respect to these tariffs. You know, one of the things if I can just sort of semi rant for a second.

One of the things that frustrates me about the biotech industry being in such a difficult place today is that we should be generally immune from these tariff issues. That, you know, we’re a uniquely US driven enterprise. Biotech really came out of The US. Most of the really innovative companies are in The US. Most manufacturing occurs in The US.

So we should be a flight to value in the midst of chaos, and here we are. So we’ve got to solve that issue as an industry.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: Okay. We’ll work on that next. How about, I guess the last question is, how are you thinking about FDA interactions? There’s sort of multi asset interactions that you have with them. Let’s just talk about your pipeline interactions.

So limb girdle, for example. Just talk to us about the you know, what kind of interactions you have been having for the last two months. Yeah. And if anything has changed there.

Doug Ingram, CEO, Sarepta Therapeutics: So since the departure of Peter Marks, that would be really the moment one would wonder, things have gone exactly as we would have hoped that they would go. As everyone knows, the Office of Therapeutic Products in CBER, which is, you know, primarily responsible for cell and gene therapy, has been for some time now headed up by Doctor. Verdun. Doctor. Verdun is a very capable and thoughtful leader and remains at the FDA, and our interactions have been great.

So, we have not seen any delays so far in any of our programs. We have not seen any shift or change in perspective. In fact, they have reconfirmed with us their perspective on our limb girdles. We have a very innovative approach to our limb girdles. Let me start with 09/2003.

So, first of all, we’ve had a pre BLA meeting with the division. The division has confirmed their perspective on that pathway. To remind everyone with respect of all of our sarcoglycans, there’s three of those limb girdles. They all have very innovative approaches. All of them code for their gene therapies for ultra rare diseases.

They code for the native protein unaltered, the absence of which is the sole and exclusive cause of the disease, the degeneration, and ultimately often the demise of the patients who have one of these sarcoglycanopathies. So, it’s a kind of extraordinarily high probability of bringing a better life to patients so long as you can get good expression of this native protein properly localized. And they have confirmed that that’s the approach that we’re taking. We have single arm studies for all of those three. We have fairly small studies in consideration of the size of the patient population, and the primary endpoint for accelerated approval is expression of the native protein.

As relates to 09/2003 as an example, we now have the results of the 09/2003 program. I’m not going to give you the details on those results. Doctor. Luisa Rignon Klepek would be very unhappy with me as she wants to provide that information at an upcoming medical meeting at the appropriate time. But the good news is strongly positive.

We were strongly positive on the primary endpoint which was expression very, very strong statistically significant increase in beta sarcoglycan, which is fantastic. We hit the primary bard. No new safety signals, all very consistent with what we have seen in prior cohorts and is what we’ve seen more generally across this rh75 platform that we have. So, you know, it’s all a go right now. We’re going to submit a BLA for approval later this year.

That means if successful and we have a lot of conviction around this, we’ll have an approval in the first half of next year, and then we’ll roll that forward. So in ’twenty six, we’ll have an approval for a limb girdle. In ’twenty seven, we’ll have an approval for the next limb girdle. And in ’twenty eight, we’ll have the approval for the next limb girdle. That’s where we are from an LGMD perspective.

So all of which kind of to answer your broader question, we are continuing to have a very productive discussions with the agency. And we have not seen either a so far, we have not seen any shift in their approach to cell and gene therapy, and we have not seen so far any situation where just as a result of staffing or the like, they’ve missed deadlines. So far very good.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: Has Sarepta had recent interactions?

Doug Ingram, CEO, Sarepta Therapeutics: Yeah, over the last couple of months we have.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: Okay, Good. So let’s move on to the PMO franchise. So for those, they’re approved under accelerated. Right. And, you know, there’s been some questions of late just given the new people in the administration that that might be responsible at FDA, namely CBER, about, you know, what’s required to turn an accelerated approval into a permanent approval.

Any interactions with the agency on that particular question as it relates to PMOs?

Doug Ingram, CEO, Sarepta Therapeutics: So two things. The no, not yet. We’re in the middle of so first of all, just to remind everybody, we have two we have three PMOs. Two of those PMOs are subject to sort of traditional post marketing confirmatory trial, a trial called ESSENCE. The other program is EXONDYS.

EXONDYS actually doesn’t have a post marketing confirmatory trial. It only has a dose ranging trial. So there’s no trial, the goal of which is to disconfirm or confirm

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: So should we assume that that’s a permanent approval?

Doug Ingram, CEO, Sarepta Therapeutics: Well, we have to assume that when we get to the So the question for the FDA, we actually proposed a confirmatory trial and the FDA rejected it. This is years ago. This is before my time actually, I think it was in late twenty sixteen. FDA instead wanted to ask a different question, is you’re distributing a thirty mgkg version Exondis. Would it be more effective at one hundred mgkg or even up to two hundred mgkg?

So, in the midst of that. So, the real result of that study is either you confirm that thirty mgkg is the right dose or you have a higher dose, one hundred or, you know, maybe in the most extreme case, two hundred mgkg. And of course, we’ll let the data drive us in the direction of that. And then with ESSENCE, of course, it is a traditional confirmatory trial, but we haven’t had any new interactions. The individual that I assume you’re referring to at the FDA is Doctor.

Vinay Prasad. He is the center director for CBER. So he’s not over at CBER. I don’t think we have a new leader for CBER yet.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: So is it your expectation that everything that you understood from the agency before will still hold as it relates to the requirements that you need to show for PMO?

Doug Ingram, CEO, Sarepta Therapeutics: It is. And because it’s not only the expectations from the agency, but also the regulations themselves are fairly clear. For an accelerated approval, what one does is, you know, you’ve got some postmark typically you have a post marketing commitment, usually a post marketing confirmatory trial. You are required to complete that trial. And then at the end of that trial, you look at the totality of the evidence associated with the therapy and then ask the question, is there any reason to believe that this therapy is there any evidence that the therapy is safe and efficacious, or is there a strong evidence based on the totality of evidence that it’s not?

And then it’s on that basis. So it is a totality of evidence look. And so at the end of ESSENCE, we’ll look at that. We’ll also look at all of the real world data. So one of the nice things about having had these therapies in the community for some time now is that we have the ability to do very thoughtful post marketing analysis ourselves and do a long term real world evidence type of studies.

And that’s what’s really required. Because remember, with respect to our PMOs, we are not arresting the decline in patients who have Duchenne. We’re slowing the decline given the amount of dystrophin we’re making. And so to see those differences requires a lot of time. You’re not going to see them in six months.

You know, we saw that recently with Viltebsa who had a very short study and was unsuccessful there and had to do yet another post marketing trial. And it wasn’t that surprising to us, not because I think in the long run Valtepso won’t work, but because with these long degenerative diseases, you really need to watch patients over a long period of time. We’ve had the opportunity to do that particularly with EXONDYS. And what we’ve seen with EXONDYS is that you look out over six years, it is keeping kids out of a wheelchair By years, it’s keeping them off of a ventilator. By years, it’s greatly reducing emergency room visits and contractures, and the hazard ratio suggests that we are extending their lives by years.

And so, we’ll take all of that information plus whatever else we glean, for instance, from ESSENCE, and then we’ll, you know, have a thoughtful discussion with the agency about where we are and whether we can translate these accelerated approvals to traditional approvals.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: Okay. And just remind us when does ESSENCE readout?

Doug Ingram, CEO, Sarepta Therapeutics: I think we’ve said early next year.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: Okay. And then the last part But

Doug Ingram, CEO, Sarepta Therapeutics: it should be completed at the back half of this year.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: Something going So the last part of the question is on Elevitus and on the accelerated portion of non ambulant patients. Any queries or interactions with the agency on that?

Doug Ingram, CEO, Sarepta Therapeutics: No. No. I mean, you know, one thing I will remind us all that an accelerated approval and a traditional approval are both approvals. This is not like in Europe where an accelerated approval is a conditional approval. That’s a wrong way to look at it.

They both have satisfied the obligation for substantial evidence to justify them. The difference between a traditional approval and an accelerated approval is you have a post marketing commitment with your accelerated approval that is a confirmatory typically. Know, Axonis is a little different. But very, very typically you have a post marketing confirmatory trial obligation. And in this case, do.

We have a trial that’s ongoing for that. And when that trial reads out, which should be sometime in 2027, we’ll have the discussion about the ability to convert that accelerated approval to a traditional approval, to what we have with all ambulatory children.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: Okay, got it. So now let’s move on to the guidance question.

Doug Ingram, CEO, Sarepta Therapeutics: Okay.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: Take a take a sip of your coffee, and then we can talk about it. Alright. Alright. So let’s be forward looking as opposed to looking back. Talk to us about the factors that you’ve taken into account with your revised guidance and why you’re confident in that.

Doug Ingram, CEO, Sarepta Therapeutics: Yeah. Okay. There’s two parts of this. I’ll be happy to talk about Q1 if you’d like. We were short of what our own expectations were in the first quarter by, I don’t know, 15 kids or something like that, maybe a little more than that.

And there’s a lot of reasons for that, some of which are not replicable, but some of which were insightful for the long term guidance. But I’ll just talk about our guidance over the course of this year. There’s three things that informed our decision to reduce our guidance for the rest of the year. And I will say I’ve been doing this for a very, very long time. This is the first time in all my entire career that I’ve ever reduced guidance.

So I do want everyone to know I didn’t do it hastily. I did it after a lot of analysis. So there really are three things. One is, as you would know, late in the first quarter, there was a really unfortunate safety event for a young man on Alevitus who passed away with a liver injury subsequent to being dosed with a levodus. And that required in the first quarter, and it’s going to require for some time forward us getting out there and really contextualizing and giving the information and giving the context both to families so that they’re well armed to have good conversations and physicians.

One of the errors that we can make, and I think, you know, I would make it personally as well, and I think some of our analysts would make it as well, if we go out and talk to the people that we normally talk to, all of the big thought leaders who are really well informed about gene therapy and AAV and the like in the context of this fatal disease, they would say this does not affect our practice at all. That’s what I heard at MDA. I was actually at the FDA conference at the time that this came out, and that’s what I heard from every physician. But the issue is that we’ve got to get beyond them. We’ve got to get out around the country.

We’ve to get to secondary sites and tertiary sites and referring sites, we’ve to get to the broader patient community. So, that is going to have an impact. It had an impact in the first quarter, not at all surprisingly. If you hear about this issue and you’re going to get, you know, either going to infuse someone as a physician or be infused, you know, five days later, you’re probably going to want to pause and figure out what was that about, make sure that nothing new, nothing’s changed in your metric. And that’s going continue to occur I suspect through the second quarter just because of the cycle time.

And then we’ll get on the other side of that as we get good information out. So that’s kind of the one big issue. The other learning from the first quarter was that, you know, we’ve always said that there’s a long cycle time from start form to infusion. It’s about four to six weeks longer than we had assumed. Now, I want to be very clear.

This is not ultimately an access issue because the fact is that all of the kids so far, whether they’ve had to go seamlessly through the process or even go through the appeal process, which often happens, they’ve gotten on therapy. So where our success rate today is one hundred percent with Alebenist. It’s more in the mid-ninety percentile with the PMO. So if you asked me, what what would I anticipate five years from now, I bet it’s kind of in the mid-90s. But today it’s one hundred percent.

So, it’s not about whether kids are getting on therapy, it’s the length of time it takes to get on therapy. And there’s a bunch of reasons for that. There’s a single case agreement concept that takes some four, could even take six weeks. There’s more screening with a gene therapy than there are. There are multiple more appointments.

There’s the antibody test. It just takes longer. You know, it might be the case. If you want to be a Pollyanna, like I sometimes am accused of being, you might assume that there were things that happened in the first quarter that made it particularly long and maybe it won’t be as long going forward. But our assumption for the rest of the year is that cycle time is going to be the stable cycle time for the rest of this year.

That has a pretty significant impact on revenue and revenue recognition. And then the third thing is we’re looking at it, is a little bit tertiary to the other two, but is important is when we really think about getting information to physicians, we’ve done such a good job with the big thought leaders that focusing in that area isn’t going to help kids this year because those folks, if you went out and talked to most of the big sites, you know all the names, Tazeen, they’re going to tell you I’m booked for a year. So if you want to bring a start form to me right now, you’ll get maybe dosed in May of twenty six. You’re not getting dosed this year. So we really need to think from a call perspective, getting to the secondary sites, all well trained.

So it’s not an issue of quality and just really focusing on how we get more productivity and new start forms there. So those three things together, the first two probably most significantly drive our guidance for the year.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: Okay. Thanks for that color. So on that third point, that is something that Sarepta effectively can control. So what are you doing exactly to get those, you know, other sites up and running? I don’t want to say capacity constraints, you do need to, for a child to wait a year, their symptoms would meaningfully progress during that time.

So, you know, how do you kind of balance needing to make sure that everything is taken, you know, in the proper steps with the time urgency to get these patients on therapy?

Doug Ingram, CEO, Sarepta Therapeutics: Well, a couple things. First, let’s be very clear. When we’re talking about these other sites, these are not like, you know, lower quality, you know, sites. They are well trained. We don’t allow someone to get a Levenus unless they’ve been well trained.

They’re great sites. So, there’s no issue from a quality perspective there. And to your very good point, why am I so passionate about this as an issue? You should see our data right now. For those who don’t know, if you’ve seen our crossover data and you’ve seen the MRI data on muscle, I mean, I think we’ve probably buried the lead on this muscle MRI data too much.

One year even at two year, one year and two years for muscle MRI, it’s stark. In just fifty two weeks, if you’re on this therapy versus not on this therapy, it’s the difference from having a lot more muscle. You’ll lose a lot of muscle if you’re not on it. You’ll have a lot more inflammation. You’ll have a lot more fat and fibrotic tissue infiltration.

So to your very good point, there’s no waiting. Like any informed patient that saw the data and any informed physician that saw the data would realize that I got to find a way to get these kids dosed. You know, maybe I can wait two or three months, but I can’t wait twelve months. It would be travesty. So, it’s very much in our control.

It’s just a matter of focus. We just need to focus hard on those areas that have the opportunity to dose kids this year.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: So, do you define as focus? What is your sales force? Paul,

Doug Ingram, CEO, Sarepta Therapeutics: Salesforce, MSLs, just giving them all the support they need. Finance, our finance part of our commercial organization. One of the interesting things with the gene therapy is that you have to actually be in there in the finance department because you’ve got credit issues and the like that you have to work with. So, there’s a multidisciplinary approach that we take with our sites. And as we think about it, we just need to make sure that we continue to provide appropriate support to our big thought leaders, but really focus a lot of time and attention to education, efficacy education, safety education, you know, process related education and the like.

It’s very complicated. I’ve said this many times before. At an individual site to dose a single kid, it takes something like 22, 20 three people across the whole internal site ecosystem to get that kid dosed. So, it’s just in a matter of focus. So, to me, that’s very much in our hands.

The safety information and getting that information, that’s very much in our hands. And whether we can shorten the cycle times remains to be seen whether we can actually do that or whether we’re stable.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: Okay. So on the question of safety, for the parents who decided to hold off their appointments after they found out about that unfortunate patient death, is there a way that Sarepta is able to track those families and have a sense of if they have decided to go ahead and reschedule their son for the treatment?

Doug Ingram, CEO, Sarepta Therapeutics: Yeah, I mean I don’t have the information, but we have some visibility into those issues, and so we’re tracking that and the like. And I’m very confident. The issue is one of the things you would imagine is that if you postponed and then you immediately could get a new appointment in a month, everything would be great. There’s a couple different problems with that. The first issue is that if you get out of the system, it may take months to get back in the system.

It may take a brand new start form because you may need a new authorization. Your authorization may be stale. In fact, more often than not is stale. You probably need a new antibody test. You’re going go through a whole new screening cycle.

So, that complicates. And then another thing that is interesting and complicates it is, you know, in our first quarter, I didn’t mention this, we had this administrative issue in LA where there was kind of a pure administrative dust up on single case agreements between the local Medi Cal provider in the site or payer in the site, and it was just an administrative issue. It caused nine kids to get kicked. Got solved. Got solved in March, but again, get solving in March means your kid’s not going to get dosed in March.

Because those kids were all at top sites, if those kids get slotted in the next quarter, other kids get pushed back. So, it’s just, you know, it just rolls forward, which is another reason we really need to focus on getting to some of the sites that have the opportunity to dose kids today.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: On average, how many kids can a site dose per day?

Doug Ingram, CEO, Sarepta Therapeutics: It’s per, well, per week. Mean, it’s-

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: In a day, how many? Is it only one child?

Doug Ingram, CEO, Sarepta Therapeutics: Yeah. I mean, it depends on the site. So, it’s very, like I can give you big numbers, but it’s very, very variable. It’s variable. Like once a week would be, you know, would be the kind of thing that a top site might do.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: How many of those?

Doug Ingram, CEO, Sarepta Therapeutics: Because remember, and you say, oh, we can fix that. Like find a way for them to have more infusion rooms or find a way to have them more nurses. Or you would imagine there’s some easy fix, but there isn’t because it’s really complicated. Like, you know, you got credit issues. Like, you know, the finance folks are going be like, there’s only so much credit risk I’m going to take with these payers in any one quarter.

So you bump up against those kinds of issues. So that’s kind of defines the cadence of all of this.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: So I’m assuming that all of these are new observations to the company relative to what you thought last year. So I guess what was the disconnect? Was it that you were confident that you had enough sites or that the demand is just outpacing what you thought it would be at this point?

Doug Ingram, CEO, Sarepta Therapeutics: I think that there was I think number one, let’s go through each of them probably. I think that

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: We’ll do it in thirty seconds.

Doug Ingram, CEO, Sarepta Therapeutics: I’ll do it quickly. I’ll do it quickly. Can very One on the safety issue was new. By the way, not a new safety signal for AAV mediated gene therapy. Let me be clear, every AAV mediated gene therapy has some risk of liver injury.

Real quick, this was a shocker to us because we dosed over 800 kids. We’ve never had anything like this before. There is, I pause it without, you know, yet substantial evidence. I pause that there is something unique about this case that was very different than the other cases. We’re continuing to explore it.

We’ll have the autopsy results hopefully in the next month or two. Let’s hope that we can get to a place where we can understand what was unique about it because we’ve dosed over 800 kits. And on whole, can tell you that 11 is for a full body infusion is almost certainly the safest gene therapy yet approved. But the safety event was new. The cycle times was new to us because in the fourth quarter we were seeing it lower.

That may very well been because we were just approved and people were prioritizing their easiest cases. You know? And as you get to more difficult cases where you have to go through multiple rounds with a payer, you just extend a bit. So, that was just new to us. And on the division, think that’s just a Q1 learning.

Perhaps we could have been more forward thinking about it and learned it earlier. But I think those first two were the greater impacts.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: Okay. So just to wrap it up, how should we be thinking about You talked about 2Q. How should we be thinking about the back half of the year? So you’ve given us a range for numbers. Should we account for any seasonality in 4Q, for example, because of the holidays?

Doug Ingram, CEO, Sarepta Therapeutics: So, you know, what we see, we’re not going to know fully on seasonality. We’re going live with this for some We do have some belief that there is going to be a pickup in the summer season because, you know, this is a very burdensome concept to be away from. You have to do a lot of things to have the therapy and the monitoring. And so, I do think that some families have prioritized the summer and said we may see a pickup there. Against that in the second quarter, we’re just going to have this read through of the safety event and having to get that information out and get that done.

But what we’re envisioning, you’ll see in our guidance, is that we’re going to be down a little bit in the second quarter, still the hangover from all the things we talked about in Q1. And then we’ll start to see a pickup in the Q3, Q4 to get to our guidance.

Tazeen Ahmad, Senior SMIT Biotech Analyst, Bank of America: Okay. Got it. So we’re out of time for this morning. Sallan, thank you for making the trip out here. And thanks everybody for sitting with us with this fireside chat.

We’ll talk soon.

Doug Ingram, CEO, Sarepta Therapeutics: Thank you all very much.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.