Scholar Rock Holding Corp (SRRK) September 10, 2026 Conference Transcript

It's great to be here with you.

Maybe just, you know, for those on the webcast that are maybe not as familiar with the story, just give us a quick, like, two-minute, and then we'll get right into some of the questions.

Yeah, so quickly, we're in an exciting moment in time for the company, 15 years since we founded the organization, and now really, you know, three weeks away from our PDUFA date with the FDA for for a monoclonal antibody epitogramab, which is the first and only myostatin inhibitor that's ever been proven to deliver statistically significant and clinically meaningful benefits in a phase three trial, despite the fact that nearly all major farmer and biotech companies have tried to drug myostatin since it was discovered at Johns Hopkins in 1997 as the body's natural negative regulator for growing muscle. So obviously if one could, you know, harness the potential of safely inhibiting myostatin and effectively inhibiting myostatin, you could restore and deliver muscle growth, muscle strength for a whole host of diseases. And we, of course, are focused on a set of muscular atrophies and muscular dystrophies. The first, of course, is spinal muscular atrophy, where 35,000 patients globally are receiving at least one SMN targeted therapy. And in our seven-year development program where we took no shortcuts, our phase three trial was 188 patients and we delivered a statistically meaningful benefit in the highest bar, the Hammersmith motor function scale for returning motor function improvement in those patients. And so on file at the FDA and ready to launch, Jeff, we'll talk about that with our time together this morning. The action date for the FDA is September 30th. Looking to resubmit our MAA in Europe because we've recently withdrawn, all tied to the compliance issues at our third-party fill-finish manufacturing facility. And then, of course, we were really excited to announce that we have an agreement with PMDA to file our JNDA by the end of this year, needing no additional clinical trials in a local Japanese population. And this really advances our ambition to reach these 35,000 patients and upwards to 50 countries around the world as VCAS and I and the team have done at other companies. And so we're super excited about that and look forward to digging in with Jeff this morning.

Yeah. So let's talk about the the you know catalan facility and kind of the the the background there so that that is you know now you're talking about preceding the bla right through the through the alternative uh facility um what you know maybe what gives you the the confidence that you know you're there's no remaining kind of hurdles uh just give us kind of an update on the on the manufacturing Absolutely.

Look, Jeff, you know, Jeff is appropriately so asking probably the question on everyone's mind, which is if we had a PDUFA date last year of September 22nd, 2025, all signs read positive for an approval. We were discussing the label, you know, essentially a day or two before our PDUFA date. And then we received a CRL for the sole approvability issue being a general site inspection at our third-party Phil Finish facility, Catalan, Indiana, owned by Novo Nordisk. And that same facility now has, you know, just had another poor inspection in April of 2026. that was a re-inspection that was supposed to clear the facility. Why are we confident that being back on file with the FDA with an action date in just 20 days, why are we confident that we're heading toward an approval? And that's, it really dates back to the following. At the same time that we were working with Novo Nordisk and the FDA for them to remediate their facility and improve their GMP practices, we were also standing up an alternate fill-finish facility that was in good standing with both U.S. and European regulators. We actually announced on November 14th, which was our third quarter earnings call of 2025, that we were under a contract with an alternate fill finish facility and that we had secured commercial manufacturing at that facility in the first half of 2026. So we did that very, very quickly. We rolled out tech transfer in breakneck speed. And on December, early December, first week in December, we actually requested a type C meeting with the FDA to talk about the progress at our alternate or second fill finish facility. That led to a March 3rd type C meeting where really what was on the table was the progress that we had made at this second fill finish facility. And Jeff, it was during that type C meeting with all the leaders of the FDA, OMQ, and OPQ who oversee facilities and product quality that they said, look, this is an important drug for an important patient population. Our suggestion is that we actually have a horse race. Resubmit your BLA with both Catalan, Indiana, and your alternate fill-finish facility. Whichever one enables approval fastest stays in the BLA. Whichever one would not, you remove from the BLA with no timeline hit, no major amendment, and away we go. So that was March 3rd of this year. On March 31st of this year, we announced that we had resubmitted our BLA just the day before, and that we had resubmitted it in complete alignment with the FDA with both fill-finished facilities. Now, look, when we heard inspectors showed up at Catalan, Indiana on April 13th, so just like a couple of weeks after we resubmitted our BLA, you might imagine with all the attention that Novo Nordisk was putting on remediating this plant, we thought that would have obviously been the fastest path to an approval. we would have dropped our alternate site, and then we would have resubmitted that alternate site as an SBLA after approval. We now all know that that inspection from April 13th to April 24th didn't go so well. The Form 483 had multiple observations that all read like the poor inspection in 2025. There was definitely improvements at the plan, but not enough to get the FDA through it, downgrading that facility. And so it kind of started to read for us like the most expeditious path was going to be this alternate facility. Importantly, we have now viled more commercial epitogram at this second facility than we ever did at Catalan, Indiana. And vials from this second facility are now awaiting labeling and packaging at our third-party provider on the day of approval. And so we are in a very good position from a launch perspective. Now, specifically to Jeff's question, was it a poor inspection and we removed cattle in Indiana and, you know, we're kind of flying blind on the second facility? The answer to that question is no. In fact, what gives us great confidence is that we're in ongoing dialogue with the FDA. Based upon the OAI classification of the April re-inspection, we actually said to the FDA, we think it's time to remove cattle in Indiana. The FDA said, wait a minute, let us just complete our review of your alternate facility, and then we'll actually walk you through the process to remove cattle in Indiana. And of course, in early August, we did announce by press release that we had removed cattle in Indiana from the BLA under FDA guidance. And that was following their review. I don't want to say that the FDA has ever completely done, but they definitely had a checkpoint in the way. So things are reading positive from both the CMC review and then the clinical review. There just isn't very much to do since the label was essentially finalized during the last cycle. And we remain very confident that we're going to be receiving an approval here in September.

Well, let me do one more and then to draw away an interview for a few. So just when you talk Talk about the alternate facility. Is the capacity similar? Talk about the ability to scale and maybe the early stages of the launch.

It's an important point. This is not a boutique or opaque fill-finish facility. It's a substantial facility with a track record globally. They've had European positive inspections as well as U.S. regulator positive inspections over the long run and recently. One of the things, Jeff, that we thought was important when we identified this facility was that they would have multiple lines that were actually ideal, where they had lines that were qualified for our vial configuration. Now, why was that important? That was important for speed, because if you change anything about your packaging, that would definitely not enable us to add a second vialer as quickly as we did. So we validated multiple lines with our engineering and PPQ runs. We've scaled up manufacturing there, which, of course, again, it's bill finished. So it's, you know, they're taking our drug substance and they're violating it. Their work has been extraordinary so far. As I noted, they're in good standing with U.S. and European regulators. They're on file with a number of Japanese applications as well. And over the long run, Vicas and I will always look for redundancy in our supply chain. But for now, we feel really good with this fill finish facility. One other important note I would like to make, when we disclosed that we had removed catalanin from our BLA, it was only as a commercial supplier of the drug. Their data remains in our BLA. We continue to use the drug. No one's ever claimed the epitogromab that's been viled there is not a high-quality product. In fact, that's the product that we continue to dose our long-term clinical trial patients now for upwards to seven-plus years. So technically, some of the Catalan, Indiana data is in our BLA. They are just removed as a commercial supplier of epitogromab for now.

Great. Yeah, thank you so much for giving us an overview of some of what's been going on behind the scenes. You touched upon that the labeling was pretty much completed during the last cycle of review back in 2025. So maybe help remind us, some of you who are listening, about some of the discussions that had happened and maybe expectations for, you know, ambulatory versus non-ambulatory or maybe a broad label that could happen.

Yeah, these are excellent questions. And I guess I would first start because we've worked, you know, while we've been disappointed with the delay of a first and only muscle-directed, muscle-targeted treatment for SMA. We have been very grateful to the FDA for their collaboration during these these these past few months since the CRL. And so it'd be maybe inappropriate for me to comment specifically on the label. But what I have said in the past is we were pleased with where the label was at the last exchange that we had with FDA, which was September 18th of 2025. So that was just, you know, two business days before the PDUFA date. We had historically seen that the FDA, as it relates to SMA, has usually seen SMA as a single disease rather than a disease where they're chopping it up by, you know, age group, copy numbers, subtypes, or ambulatory status. Like if you just look at the other drugs that they have approved, they've generally not done that. Obviously, the gene therapy was in a very young patient population. So our feeling is that, you know, obviously the under two-year-old population will not be unlabeled because we just hadn't studied that in our clinical trials. It was really two and older. We do believe that very much in our label discussions, we were very much talking about SMA more broadly, and the benefits of SAPPHIRE that our phase three SAPPHIRE trial were able to demonstrate. And so we were pleased, just to give you guys a sense of the exchange, when you submit a BLA, you submit with your draft label. I'm on record as saying we were never, even in the first red line from the FDA, we were never in a different stratosphere from the FDA on what the label should look like. We were always kind of seeing things very similarly. And so it was very collaborative in the last cycle. They sent us the latest red line on, it was like 10 a.m. on Thursday, September 18th, and why we thought we were gonna get approved in that cycle. Remember, this is before Catalan, Indiana was classified as OAI and before they got a warning letter. So here we are in deep label discussions. They send us the label the morning of September 18th. They wanted it the morning of September 19th, which was Friday. The Padufa was Monday, the 22nd. We sent back comments to that. We had sandpaper out. We definitely didn't have major, you know, we didn't have major tools, chainsaws, you know, axes out at all. We were sandpapering some of their comments. We sent it back in the evening of September 18th. What they asked for in this cycle, in this re-review of the application, is for us to go back to the label we sent to them on the evening of the 18th. That was the starting point for this cycle. So that's why we were, I've also, I'm also on record as saying, even if they took none of our comments that we sent back on the 18th, we were in a win scenario for the SMA community. We were in a spot where we would have been able to serve a meaningful proportion of the SMA community that could benefit from epitogramab therapy. And, you know, hopefully we'll be able to share in a lot of detail the label in just a few weeks.

Awesome. Yeah, that's great to hear. So maybe let's then shift to what could happen after Epicromad launches, and we're all very interested in hearing what the branded name will be. Maybe just, you know, as the initial patients start to flow through, you know, maybe tell us about your expectations for, you know, age, you know, two years above, or, you know, what adult status, ambulatory status, background estimate, then targeted therapy, maybe the prior treatment history, just trying to get a sense of where you're thinking of the flow of patients coming in and what they may look like.

It's an excellent question. So where do we think the demand comes from? Is it the younger patients who perhaps, you know, doctors' ambition is to provide as much, you know, normal musculature before atrophy sets in? Is it adult patients who maybe have missed some of the benefits of the SMN-targeted therapies because they had a lot of motor neuron deterioration even in 2016 when Spinraza was first approved, when the first drug was approved? And it's a little bit of all. It really depends on the patient, the family, and the doctors. And maybe I can walk you through that. So as a reminder, we took patients that on average had been on either Spinraza or Avrisdi for about five years in our phase three trial. Those patients were randomized to either receive nothing at all, placebo or epitogramab. And what we demonstrated in that trial is that those patients that were on Evrizdi and Spinraza over the 12-month treatment period actually were losing motor function. And that's actually pretty consistent with some of the long-term data that even those companies have shared of their registration trials. that patients do better initially when they get more smn protein they gain motor function they plateau and then they can actually you know start actually losing motor function again and what we demonstrated in the sapphire trial is patients went from a loss of motor function to a gain of motor function and in fact what we believe will be the recommended dose the 10 milligram per kilogram dose there was a 2.2 point difference in the hammersmith motor function scale which is the gold standard and highest bar to hit, even if RISD did not hit that prospectively in their phase three trial, and we had a statistically significant benefit there. And then we had a 3.8 times greater likelihood of a three-point improvement or more when patients on Spinraza and RISD were randomized to epitogramab versus not. And this is, again, a pretty broad patient population. So the Topaz study, we did have children as early as two years old in that study. They've done exceptionally well. So what we're hearing is the following, 90 to 95% of patients claim today with SMA that their number one need is muscle strength and more motor function. So nearly the entire community is saying we need more muscle. 75% of neurologists are now saying that we believe the best way to treat SMA patients is by treating both the motor neuron with SMN protein and the muscle by eliminating myostatin, because it's those two things that actually make up the motor unit, which actually is what provides patients motor function. And why would anyone want to have the body's natural negative regulator for muscle growth floating around their body if they had SMA. So we think the dynamics are really solid for demand for the drug at approval. We'll get into, I'm sure, reimbursement and access here as well. But I think the way I would answer your question is I think it depends on the patient, their patient demand for the drug and the physician as to whether or not there will be, you know, more children or more adults. I think it will be a pretty balanced mix across the board, dependent upon the physician, the patient, and the family. But we just know there is very strong support to access epitogramab once it is approved, because each and every patient feels like they can benefit from more motor function driven by, you know, muscle strength and muscle growth.

Just to follow up on that, what are the strategies, I guess, to maybe maximize awareness? How would you characterize kind of among the experts and the SMN community, you know, the awareness for EpidigraMap today? But then do you need to get in guidelines? Do you need to have pre-discussions, you know, on reimbursement with payers? Like what types of things can you do, you know, prior to formal approval to kind of help help manage that?

Great question, Jeff. It's, in fact, one of the benefits of the delay we've had, you know, over this past year is we did build a U.S. commercial organization and U.S. medical organization that was ready to launch last September. Fairly small, didn't really burn us from an expense perspective. In fact, Jeff, to your point on awareness, it's actually been quite helpful to map out the patient journey at all the SMN. There's 140 SMA treatment centers in the U.S. It actually helped us to educate the community that the innovation around motor neuron health has been incredible from Spinraza, Evriz, Diesel, Gensma, incredible. but that the principal organ clinically affected for patients with SMA is the muscle that's actually in the disease and so that education that motor function is a sum of motor unit strength not just motor neuron not just muscle but the combination of those two has really been helpful and so we've seen surveys done in 2025 versus 2026 that show you know last year at this time about a quarter of KOLs said they would call all of their patients and offer them epitogramab at approval. That number a year later, that percentage has jumped to like 42, 43%. So just with the awareness factor, and I think, you know, patients thought epitogramab was coming last September. I think that delay has created even more, you know, interest in accessing the therapy. And that's why we think the landscape is pretty strong from the outset on demand. Obviously, as a, you know, a once monthly infusion, you know, we'll have to be thoughtful about pricing reimbursement and access to treatment, which always can be a little bit of a bumpy road when you're introducing a new drug. And certainly we can speak about that as well. But I think the demand dynamics and the awareness of this therapy, and I think the patient advocacy groups like Cure SMA have been wonderful about getting the message out that this is a first and only muscle-targeted therapy to likely be approved by regulators and available for patients here in 2026.

Maybe helpful just to talk through like maybe your market research on the different modalities in SMA and initially, obviously, start off with just Spinraza and then you've layered on multiple, um, therapies. So how has the sort of from mono to doublets, you know, is that, is that still, is that still reasonable, uh, going forward? You probably won't have a triplet, you know, type of approach. Um, but I don't know, maybe, uh, maybe that's, that's not a crazy scenario.

Yeah. I mean, we're sort of breaking down the treatment of SMA in three errors. There's the pre-2016 era, which was, you know, really one of a disease of high mortality, early mortality. When you would go to a patient meeting, you would hear all the ventilators in the background. There was just, you know, nothing to address motor neuron or muscle health. You know, one of the only therapies patients had was like physical therapy, and then supportive care like vent support. Then the needed innovation was ushered in by Spinraza first, and then of course followed by Zolgensma and Evrizdi. And again, all focused on SMN protein production to try to preserve whatever motor neuron function, you know, one could have. And at the same time, as patients have been living longer, they now want to live better. They really want to be able to do more. They want more self-care, and they view that as being driven by, again, muscle strength and motor function, which is why more than 90% are saying that is their number one need, and that's consistently coming out of Cure SMA surveys. And so I think what we've done well, Jeff, is to differentiate that we are a completely different modality that is not in competition with the SMN-targeted therapies, but we are truly addressing the other half of the motor unit, which is the muscle. And again, the way we designed our clinical trial showed that if you did nothing at all, this is what your outcome will be from a motor function perspective. And then if you take your background SMN targeted therapy and epitogramab is also administered to patients, you have, again, an improvement in motor function. And by the way, in our long-term data from our phase two trial, we see that patients plateau, but never really are giving up their gains that they had. And we think that is just such an important element of the outcome. I think it's also why, by the way, we're at 95% compliance, 250 patients in our phase two, phase three clinical trials, those patients remain on therapy upwards to seven years later. And so back to your point, Jeff, I think like where we come from is no matter what the physician and patient want for SMN protein production is fine for us. That's actually great for us. We just think they're only going to have one choice to address the muscle component of the disease, and that is going to be epitachromab. And so we get super excited about that. One other element I just want to underscore to Jeff's point about how much more the patients want. Even though there is no well-controlled clinical trials using more than one of these SMN-targeted therapies at the same time, about a third of patients in the U.S. are either sequentially or concurrently getting two or more of these treatments. They might be getting Zolgensma as a one-time gene therapy and then followed up with Ivrisdi or Inspinraza. Some are getting two or more of these therapies, all because physicians and patients do want more motor function. And we think that bodes well for us. As to whether or not there'll be triplet therapy for some of these folks on two therapies or not, I don't know. But I do know that that is certainly underscoring the need for more innovation. And so what we see, right, first error was no innovation. Last 10 years have been the error of SMN-targeted therapies ushered in a massive error of innovation for patients with SMA. Now, 2026 and beyond ushers in the muscle-targeted error of innovation for the SMA community, and we're excited to be really at the forefront of bringing that forward.

Great. So maybe now let's pivot over to the payer side of things. I know you've kind of touched on some of the challenges that maybe some of the bumps in the road that can be part of launching a rare disease without a J code implemented immediately. So maybe talk a little bit about expectations for the initial launch, maybe some of the authorization criteria that patients and physicians may have to go through, and maybe what your expectations are for some of the greater friction points the first six months to the year.

Yeah, thank you for raising it, because we do think this is a sophisticated community with great levels of persistence and everything in their life. They've obviously all already had experience with payers to obtain their SMN targeted therapy. And yet we do expect with any new rare disease therapy, there will be some speed bumps along the way. We think that while we think demand will be high and while we have actually developed a patient assistance program known as Scholarock Support. Every patient and family will be assigned a Scholarock sort of care coordinator to help usher them through the reimbursement process. There are a number of things that we think could delay access to treatment. I don't think it will be a stop sign, but I think it may delay, you know, in the form of a speed bump. One, as you noted, is a miscellaneous J-code. This is a once-monthly or every four-week infusion, and we'll have a miscellaneous J-code. Let's assume we get approved in September. It would at least be Q4 and Q1 that we have a miscellaneous J-code, and then starting next April, we would have a drug-specific J-code. That really does help with reimbursement and timely reimbursement. You mentioned medical policies. Let's assume from your earlier question that our label is more broad than our phase three clinical trial enrollment criteria. We do know, and you all know this from all the companies that you cover, oftentimes an initial medical policy may be more narrow than even the FDA label. That could prevent initially some patients gaining access to treatment. But again, we think that's a matter of when and not if, because it's a very difficult thing for payers to stand by. We do think with 140 SMA treatment centers of excellence, some of those centers are going to have formulary status issues that they'll need to walk themselves through. And they'll also have to think about the logistics of integrating a once 28-day infusion into their practice? Do the patients get it at the hospital? Do they get it at the physician office? Do they get the medicine at an infusion center? And frankly, as we've also mentioned, we've got a 10,000 nurse home infusion network, and we'd like patients to be able to get it at home if that's at all possible. So I do think we will see a high conversion rate in a short amount of conversion time, probably a year after approval, but we will definitely have to be playing a little bit of whack-a-mole in the first year along with the SMA community to make sure that every patient who can benefit from epitogramab will have access to epitogramab.

I just have a few minutes left, but I wanted to ask about the global opportunity. Maybe give us a status update on European, Japanese, or OUS filings.

So in August, we announced three things related to our global regulatory update. The first is that under FDA guidance, as I've already mentioned, we removed Catalan, Indiana from our BLA and in the PDUFA date remained September 30th because there was some concern that removing that might have created a delay in PDUFA action date. That was not the case. That's number one. And number two, based on the fact, see, we never had the equivalent of a CRL in Europe. So as a result, we had received the benefit from European regulators of multiple clock stops waiting for the inspection outcome of Catalan, Indiana, which was the only fill-finished facility in our MAA. And then when it came down in April of this year, I'm sorry, of August of this year, just last month, everyone was on vacation in Europe. there was really nothing they could do about adding the second viler. And so it was their advice to us to withdraw the MAA. That would be the way to remove Catalina and add the second viler and resubmit the MAA. We are ready to resubmit whenever they tell us to. As you guys know, in Europe, there's a specific day each month to resubmit. We're just waiting to get a go ahead on, you know, is that this month? Is that next month? And hopefully that will be an expeditious review, but I really have no guidance on timeline yet. But once they get back from vacation and give us that guidance, we'll be letting the world know, probably in the form of we've now resubmitted our MAA. So there's a little bit of a pushback in Europe. We have built the team in Germany. They are ready to go. But what we announced also last month, Jeff, with a little bit of a pushback in Europe is we think it more than makes up for a little bit of a delay in Europe is the acceleration in Japan. Because if PMDA said we had to do a Japanese clinical trial, that would have been multiple years in the making. But they looked at the merits of our global clinical trial program, the importance of epitogram for the SMA community, and they agreed that we could file our JNDA by the end of this year. And again, we'll be able to advise when we submit, when it's accepted, in what we think the review timeline will be. But as you know, as 120 million population country, that more than makes up for a little bit of delay in Europe where we would have only initially launched in Germany an 80 million population country. So we're super gratified for that. As I think all of you know, there are no other muscle-targeted therapies even in clinical development in SMA. So our ambition to reach patients in 50 countries around the world, Europe, U.S., and Japan gets us to close to 30 We think we're well on our way to doing that without any meaningful competition over the next five to ten years. We think this is really our market to develop and grow and protect with a significant moat for many years to come.

Well, with that, David Vakas, thank you very much. Great to be here with you, Jeff. Thank you.