Conference Transcript - SRRK 2026-06-03

Amy Lee, Analyst — Jefferies

Guess we can get started. Thanks to everyone for attending day one of our Jeffries New York Healthcare Conference. I'm Amy Lee. I'm a biotech analyst at Jeffries, and I have the pleasure of welcoming David Halal, the CEO of Scholarock. I'll turn it over to him for some opening remarks.

David Hallal, CEO

Thanks, Amy. It's great to be here at Jeffries once again. We are extraordinarily excited at Scholarock today as 2026 sets up to be a transformative year for the company. We are excited that this will be our transition year into a commercial stage biotech company as we are currently on file with the FDA for epitogramab for the treatment of children and adults living with SMA with a PDUFA date of September 30th. We are also on file with the European Medicines Agency. Our MAA was filed last year, and we are looking forward to meaningful progress there. We remain on track and are expecting a decision from CHMP around and mid this year. And then importantly, you know, we see the opportunity to reach patients in upwards to 50 countries around the world, and we look forward to sharing more of our plans and how we will be submitting regulatory applications in jurisdictions outside of the U.S. and Europe as well. But most importantly, we're ready to go with the introduction of epitogromab and the launch in the U.S. and Europe for children and adults living with SMA. As an organization, we see this as a multibillion-dollar-a-year opportunity to serve these patients with no meaningful competition for the next five to ten years. There really has not been an approach that has worked on a muscle-targeted therapy for patients with SMA. And in addition to that, we are very excited about our pipeline in a product strategy with our Phase II FSHD study, which will commence near midyear. And we also have an extremely strong cash position, as we announced at the end of Q1, $480 million on the balance sheet. So really looking forward to 2026 being that transition year for the company.

Amy Lee, Analyst — Jefferies

Well, let's start off with manufacturing. I'm sure that's probably top of mind for everyone.

David Hallal, CEO

Why would you want to talk about manufacturing, Amy? I promise. I have no idea. You're throwing me such a curveball. I have no idea.

Amy Lee, Analyst — Jefferies

So I think the one thing that you've always emphasized is you had two different paths, right, to approval. And you said that when you resubmitted with the FDA, you have optionality. You don't want to rely solely on Catalan. You have a backup fill finish, which you accelerated, I would say, in pretty record time. So let's start with the Catalan site. There is a post-inspection Form 83, Form 443 that came out. It listed eight observations. Some of the mammalian hair is still there. And then it talked about some of these process changes. What is kind of feedback from, you know, internally, as well as your consultants on kind of the likelihood of this getting resolved within a 90-day window? And then feedback from Novo as well.

David Hallal, CEO

Yeah, so there's a lot there. And maybe if I could for the audience kind of walk back the story a little bit and we'll come right back to the recent re-inspection by the FDA of Catalan, Indiana, owned and operated by Novo Nordisk. So just to level set, as everybody knows, we had an original PDUFA date with our BLA submission of September 22, 2025 under priority review, and that is really based on the transformative potential of epitogramab as the world's first and only muscle-targeted therapy for patients living with SMA. As we disclosed in August of 2025, there was a general site inspection of Catalan, Indiana, not related to epitogramab, that resulted in a Form 483 with multiple observations. We know this is a site prior to Novo owning the site, that it had some difficult inspections prior to 2025, and so we disclosed that in August. On our PDUFA date, we were expecting an approval. Remember, that facility was still classified as VAI on the day of our PDUFA date, and we were surprised to receive a CRL from the FDA, which highlighted that the sole approvability issue for epitogramab was really that general site inspection and the state of GMP compliance in the facility. And what the FDA said in that CRL, which has been published to the public, is that when that facility was coming back into compliance, that is when we would be able to resubmit our BLA. So since that time, we've had constructive type A meetings with the FDA, along with Novo Nordisk and the patient community to talk about the progress at the facility. We know the facility did go under a warning letter and an OAI designation, and we were sort of working with Novo to make sure that they, in an expedited fashion, could deliver on their remediation plan for a re-inspection and an eventual reclassification of the facility. That's really been a lot of activity since September of 25 to where we are today now in June of 26. At the same time, as Amy knows quite well, we had our type A meeting on November 12th of 2025, and then we had our earnings call on November 14th of 2025, where we shed light on the constructive and collaborative type A meeting that we had with the FDA. importantly at that time we did also announce that we did get under contract with a second fill finish facility and we had gained commercial capacity in the first half of 2026 so from a long time ago while we've been working with Novo Nordisk we've been working expeditiously on making sure that when we did resubmit the BLA we may have an opportunity to strengthen this BLA versus the last BLA by having two fill-finish facilities in that. So what has happened subsequently is in March of 2026, in complete alignment with the FDA, we resubmitted our BLA with both Catalan, Indiana, and a second fill-finish facility. So that was with the encouragement of the FDA to make sure that we had two independent paths to an approval. Now, how would we get there with those two independent paths? Well, for Catalan, Indiana, it would be a re-inspection and site reclassification downgraded from OAI into, let's just say, a VAI category. But we did submit that BLA before the re-inspection because the FDA acknowledged improvement of the facility through all of their meetings with Novo Nordisk and their site visit that took place in the first quarter of 2026. At the same time through our ongoing work with our second fill finish facility the FDA said let's resubmit your BLA with both Catalan, Indiana with the reinspection really being a review issue let's also submit the BLA with the second fill finish facility in whichever one enables us to approve epitogramab for SMA fastest will be the path forward. And the other one could then be moved to an SBLA status. And so that's just want to lay the groundwork for kind of how we got here. As many of you know, the FDA issued a warning letter to Catalan, Indiana, in November of 2025. they then um novo nordisk responded to that warning letter by mid-december 2025 and the fda reached out to novo nordisk right before the holidays in december of 2025 and scheduled a meeting to talk about the remediation plan what has happened subsequently is through all of that dialogue between novo and the fda and novo saying that they would be re-inspection ready early in 2026. Novo stopped operations at the facility just to focus on their remediation plan and then they resumed those manufacturing activities at the end of February. The FDA then re-inspected the facility. I think that re-inspection commenced on April 13th. So just you know, probably six weeks after routine manufacturing activities had commenced at Novo Nordisk. And of course, we, along with Novo, I think we're disappointed that these eight observations were cited in the Form 483. What we know has happened now subsequently is that Novo has responded to the Form 483 in the 15 business days that they had. And now, really, we and Novo need to allow the FDA to do their work, to look at those responses, understand what commitments Novo Nordisk is making, and within this 90-day period, which kind of takes you into late July, the FDA will make a decision. Do they maintain the OAI classification, which would largely prevent epitogramab from being approved at Catalan, Indiana, if that were to happen, Or do they downgrade the facility to a VAI, which we do not believe is not possible, given the improvements that have taken place at Novo? At the same time, in July, all of the drug that we have viled and is ready to go from our second fill-finish facility is also going to be releasable upon approval. And that is why what we like to say is really the elegance of our BLA resubmission, which went in in late March, is that there is two independent paths to an approval for epitogramab. And actually, I would throw in a third. The first is, the first independent path is Catalan, Indiana. Well, there are two independent paths. It's either Catalan, Indiana, or the second fill-finish facility. A third option is that all around the July time point, everything is getting resolved, and there is the potential of the drug being approved with two fill-finish facilities. Taking a step back from all of that, we are very confident that we are launching this drug at any time between now and September 30th, and we are eagerly awaiting the FDA's decision. We are now under file. We are having dialogue with the FDA today, and I think we continue to be working collaboratively and constructively with them for the fastest path to an approval to bringing epitogramab to the U.S. market as quickly as possible.

Amy Lee, Analyst — Jefferies

Awesome, David. Thank you so much. That was incredibly helpful. Maybe just actually last question on the Catalan, Indiana, because I remember when we spoke, you said the timing of the batches is important, right? If the timing of the batches was before the shutdown of the site versus after, there could be more leniency by the FDA. Can you just tell us, one, what was the language that stood out to you in the Form 483? And then, number two, in terms of your base case, do you think the FDA will re-inspect the re-inspected Catalan site, or do you think they have all they need?

David Hallal, CEO

No, these are really good questions. I think for anybody reading the Form 483, you'd be disappointed that some of the same sort of observations seem to be persistent from the prior inspection. At the same time, as is noted, two of the inspectors in the April 2026 re-inspection were the same inspectors from last summer. If one looks at the original inspection report from last summer, they clearly call out repeat observations. They bold those. They don't really call it out this time. I don't know whether or not that's relevant. It's just a fact. It's not bolded out that these are repeat observations. But to read them, anybody would read them and say some of these things seem to be persistent with particulate matter, fulsome investigations, and, of course, as you noted, mammalian here. The responses that Novo puts in are not empty. They're not baseless. What Novo might say is they believe one of the root causes for the mammalian here are some of the supplies that come into the facility like the stoppers or the caps for the vials. And they've introduced like a washing step for those caps because as a CDMO, they cannot swap out the caps or the stoppers without an approval to do that because you can't change any packaging of any FDA-approved product. But by changing out a lot of them and by coming up with like a washing or sterilization step, like they would claim that the mammalian here has been reduced by 85% in the facility. So not zero, but meaningful improvement. That's what we mean by like to the naked eye, there has been meaningful improvement at the facility. And I think importantly what NOVO would also claim is that, yes, there were still some batches with mammalian here, but they were also caught at the facility and not released into the marketplace. They would claim that what was found in the inspection report was that those happened, but they also were not released in the market. So that's where I mean that one could look at this and say, I'm disappointed in the observations, there is improvement here, and there is, I don't mean, no pun intended, there is improvement here at the facility, and I think it really does now come down to the FDA's view on the responses and any dialogue that they have with Novo Nordisk, and we'll look forward to the FDA doing their work. And between now and late July, we should have a clearer answer on that. I don't really foresee this as, within that window, another inspection of the facility, as you asked. I think it's just going to come down to dialogue, review of the responses, and an FDA determination. And it always comes from headquarters in these situations, and that's what will happen.

Amy Lee, Analyst — Jefferies

That was super clear. And then just moving on to your second field finish, I think a lot of the questions that I've been getting is, How are you able to, you know, be two to three years behind Regeneron and almost accelerate your timelines to almost be like neck and neck on, you know, right? So can you tell us about the work that you've done and also, like, the FDA allowing you to take some of the, you know, data already generated at Catalan and you don't have to read? Like, can you kind of tell us the plan there?

David Hallal, CEO

Yeah. Maybe the first point that I'll make for the audience is we have several hundred patients. And SMA is a rare disease. We've been dosing patients living with SMA now since 2018, 2019 from our Phase II Topaz study. And we also had an early access program on top of our open label extension trial. And so between here and Europe, we have hundreds of patients that remain on the drug. all of those patients continue to get every four weeks epitogramab safely from the drug that has been viled at Catalan, Indiana. And I think it's also important to note Catalan, Indiana remains operational from a commercial perspective. So everything already approved at Catalan, Indiana continues to get viled and released into the marketplace on an ongoing basis. So one, I just wanted to start there because I think that that's important. Now, how did we do this so quickly. At the time that the OAI classification came down from FDA to Catalan, Indiana, it was around Columbus Day weekend 2025, so October of 25, several weeks after our CRL, and really from that point to November 14th, we had boiled the ocean. We had evaluated every fill finish facility that we possibly could. We looked at those that had a clean inspection history with the U.S. and European regulators. We looked at those that had multiple lines that were validated for our current vial configuration, because to do it fast, you can't change anything about your packaging. We also wanted one that had dozens of products that were commercially approved and that they were certified not only in the U.S., but this will very much be a global product. So we wanted a facility that could service the U.S., Europe, and Japan. And we did want one U.S.-based. We still thought that was important to be, you know, sort of U.S.-based. So we were able to identify one with a great management team, a great technical team, and one that wanted to take on this project with us with a sense of urgency and really prioritize epitogramab in that facility. So tech transfer started almost immediately. We did reserve capacity in the first half of 2026 for engineering runs and PPQ runs, so we really accelerated that. We did those not just on one line but on two lines because if you're validating a new fill finish facility the elegance of not just waiting for one line to open up every time you need product but to have multiple lines that was important to us as well and between that second fill finish facility and our team I'm just so proud of the work that's been done all of the epitogramab that we need to launch in the U.S. and Europe has been viled independently at that second fill finish facility there is no more work that needs to be done though we will keep filling there. And we are in a wonderful spot for the FDA to review the data from that epitogramab that has been now viled under multiple runs at that facility. And with the drug having a minimum amount of stability from that facility, but leveraging the multiple years of stability, and that's why I really went into our patients continue to get epitogramab that has been viled at Catalan on an ongoing basis, that stability data in agreement with the FDA is being leveraged so that we do not have short-dated drug at approval should we launch only with the second fill-finish facility. And then I think, Amy, it's important to note again, prior to the re-inspection that commenced on April 13th with eight observations on the Form 483, we had so many robust discussions directly with the FDA dating to our type C meeting on March 3rd, 2026, the meeting minutes from that type C meeting, and other emails that really showed that we were in complete alignment and agreement with the FDA on the strategy to file with two fill-finish facilities. We gained agreement on everything that would be necessary from the second fill-finish facility in advance of even knowing if an inspection would happen fast, if an inspection would be successful or not. All of this was prospectively done, as the FDA said, to essentially have a horse race as to which facility could get validated or cleared fast in the Catalan-Indiana case, cleared, in the second fill-finish facility, validated and qualified for epitogramab the fastest. And that would be the strategy that would enable the SMA community to finally get the first ever muscle-directed therapy for SMA. And we do feel like we have been in complete lockstep with the FDA on this strategy and feel really good about where we are today.

Amy Lee, Analyst — Jefferies

Awesome. Just to put a finer point on the FDA alignment, when you had your type C meeting, was the Office of Inspections also there, you know, like when they were kind of reviewing the data that you're generating in the second fill finish? And then another question, in terms of the – because you've emphasized you want to try to keep processes as similar to Catalan as possible in order for this, you know, data that you're bringing over to be translatable, right? what exactly is being consistent is it you know the process because you said it's a simple mixing or is the equipment the same is the vial and the stopper the same yeah a couple of things

David Hallal, CEO

that can make a tech transfer and qualifying a facility for fill finish a longer term process one would be stability yes two would be if you change the vial at all let's just say the line wasn't qualified for the vial that you're using and you change out the vial because then you couldn't use any stability. You couldn't leverage the years of stability. And by the way, this is an extremely stable molecule, Lepidogromab is. So we don't, we never, we've just never seen anything not be stable. Like we're going to run up to what is considered to probably be the longest you could ever have a drug like stable for, which is usually like in a five year, 60 month kind of period of time. Like it is never not look stable. So that's why we're so confident in that. The other thing that can take a long time, and I've made this point from the beginning, is if there are a lot of products where the release testing is done at the fill-finish facility. So if you also had to tech transfer in the release assay, and then you needed to validate the release assay, that can be timely. We've always said proudly that our release test happens outside of Catalin, Indiana. We have a third-party freestanding release testing site so that actually also enabled us to do this really fast because we were not tech transferring in a release assay and then having to validate that at another site and so from that standpoint in our alignment with the FDA it was literally they told us the amount of you know sort of engineering runs PPQ runs that would be required what they would be able to leverage from Cattle in Indiana, and again, we were able to do it so quickly that it put us in a wonderful position here that between now and September 30th, we would be in a position to be approved even in an independent way with only the second fill finish facility and all the work that's been done there.

Amy Lee, Analyst — Jefferies

Okay, awesome. And then the first part of the question, sorry, I'm asking multi-parts, was the Office of Inspections present? Yeah.

David Hallal, CEO

We have always taken an approach for, I think you're asking specifically about the type C meeting. I know OMQ and OPQ, which is, I mean, that's clearly, that's the group that classifies facilities. They send the inspectors out to different facilities, and OPQ does all of the work in reviewing new data. So we had everything covered from Catalan, Indiana, to the second fill finish facility, and there have been multiple meetings that we've been a part of, and I think while they get directed by OMQ, OPQ, I think there's always been general attendance by the Office of Inspections as well. So we've always wanted to have unity there, and of course at our Type A meeting we've indicated not only did we have all of the leaders from those shops in attendance, But we also had the neuro-review division because, of course, the neuro-review division wants to approve the product, but they also need that clearance and that checkbox from OMQ, OPQ. And we feel like we're well on our way to finally making that happen here.

Amy Lee, Analyst — Jefferies

Awesome. Amazing. Incredibly helpful color. Europe, you know, you've mentioned that the current application references the Catalan slide, but there could, you know, they are in understanding of the second fill finish. Can you kind of walk us through what the range of outcomes can be? And, you know, are there precedents where you could potentially add in the second fill finish during an ongoing review process in Europe?

David Hallal, CEO

Yeah, so Amy's referencing an important point. We never had the equivalent of a CRL in Europe. So the MAA has always been active, and it's always included Catalan, Indiana only. There is mutual recognition between EMA and FDA. and EMA has been following this very closely. But, you know, to one, just state a fact, the MAA today only has cattle in Indiana as part of it and I think the EMA is hopeful and they're aware of the inspection and the inspection report, but I think they're hopeful of resolution with cattle in Indiana. By the way, there's other products for Europe that are being held up for this too, so I think that's on EMA's minds. Our RAP, our co-RAP, and the project leader in Europe, they understand everything that's happening with the second fill-finish facility, though we are not actively on file with the second fill-finish facility. Our own work has indicated that there has been some precedents where a second fill-finish facility can be added to an active MAA. I don't know how many times that's been done, but it has been done. And I think that they've also indicated to us the importance of our drug as a first and only muscle-targeted therapy and sort of not wanting to reject our MAA, not wanting us to withdraw the MAA, but wanting to see some clarity with cattle in Indiana. So what I would just say to you all is that we still stand in our position that around mid-year, we are expecting a CHMP decision. Should, and we're continuing to have dialogue with MAA on how that would happen, should we need to come off of that timeline for any reason, you guys will be the first to know because that will be a disclosable event to us. And that would either be because we have learned that the facility is not getting reclassified and we might need a little bit more time for the second fill finish facility or the FDA hasn't even reclassified. They're still taking more time to make a classification decision. So we'll maintain close ties and communication with the European Medicines Agency. I do think it's important to note that while we are expecting EMA approval, when you do get an approval for the European Medicines Agency, the only country you can launch in is Germany. The other countries get staged over a couple of years because you have to go through country-by-country reimbursement processes. So I'd like the audience to not say, oh, my God, that's 400 million citizens of Europe that are being withheld access to epitogramab. It's still going to take a couple of years in Europe. Typically, you launch in Europe over a multi-year process. Germany, though, is right after approval. So it's that 80 million population country of Germany that we have in mind. Our German team is built, is trained, is ready to go. But should we have a delay there, we will certainly keep everybody apprised. I think given the Form 483 with eight observations, that's a possibility. And we'll just have to let the FDA do their work and see how then that impacts our timing for CHMP. And then usually two months after a CHMP opinion is when the European approval comes in. One other thing I would note for our audience, CHMP agendas get published. usually the day before CHMP. So none of this is going to be done behind the veil or curtain of secrecy. This is going to be out for public consumption for everybody to see, and we'll keep you guys apprised with everything that we know there. Awesome. I know I said I wasn't going to make it all about manufacturing. We do have a commercial drug to launch eventually. Maybe I can close with the 37 seconds that we have. Look, I think there's a couple of things. I get all of the questions. I get some of the tension, but the one thing is for sure is that epitogramab is positioned to be a blockbuster rare disease medicine. There's 37,000 patients in the world that are receiving either Spinraza or Evrizdi alone or in combination, and epitogramab is the only muscle-targeted therapy that allows physicians and patients for the first time to address not not only the motor neuron component of SMA, but also the muscle component of SMA. And we think we're well positioned to serve a great number of these patients over time and build an extremely valuable business. And that is why we say with confidence as we come to the end of our time together that we believe the opportunity for epitogramab and SMA alone provides us an opportunity to create strong and steady growth through the end of this decade and well into the next with a multi-billion dollar a year opportunity and we look forward to talking to Amy maybe in London about much more than manufacturing as we get to the other side of this and start serving patients living with SMA.

Amy Lee, Analyst — Jefferies

Awesome well thank you so much David thank you so much everyone.