Here at the Bank of America Healthcare Conference, we're kicking off our very first session of the very first day. It's my pleasure to have with me, Scholar Rock as our first presenting company. Sitting up here on stage with me is David Hallal, who is, of course, Chief Executive Officer and Chairman. David, good morning.
Great to be with you, Tazeen.
Thanks for making the trip over from the East Coast. Maybe let's just do a quick overview of the company. Just give us a little bit of an overview of the focus.
Yeah
of what you're trying to do, and, how you might be differentiated from other companies trying to do at least similar indications.
Thanks Tazeen. Yeah, we're super proud at Scholar Rock to have succeeded where, you know, 20, 30 years of failure preceded us, and that is that, you know, in 1987 when myostatin was discovered, it was certainly something that just about every biotech or pharma company, mostly the big pharma companies, had tried to target and block. What myostatin is, of course, is the body's natural negative regulator of growing muscle. The thought was if one could safely and effectively inhibit myostatin, one could then, you know, release muscle growth in the body that could be applied to a set of diseases. Certainly we're focused on rare neuromuscular.
We had an idea when we started the company at Scholar Rock, which was, if we could do it slightly differently, we may be able to really harness this, this value off of our platform. That is mature myostatin looks like other things in the body. When you look to inhibit that, you actually have off-target effects. We focus on pro and latent myostatin, we actually inhibit it in a chemical form that is very, very unique. We were gratified in late 2024 to have the first ever phase III clinical trial success with statistically significant clinically meaningful benefit in patients, children, and adults with spinal muscular atrophy. Now we are poised to launch the first myostatin inhibitor in the history of our industry.
Why that differentiates Scholar Rock is there really is no meaningful competition in front of us. Even most recently, other big pharmas had failed to target myostatin successfully. What that really positions us to do is, starting with spinal muscular atrophy and then a set of other rare and devastating neuromuscular disorders, we can play out this unique capability that we have as the world leaders in myostatin biology to hopefully address a set of devastating disorders that affect children and adults, again, as I noted, starting with spinal muscular atrophy. What that means for us really is an opportunity to establish ourselves as a leader in serving patients with rare disease, with our core focus being what we know well and do well, which today it is inhibiting myostatin.
Yeah, maybe let's dig a little deeper. You mentioned that, you know, other and bigger companies have tried to go down this path but have had some roadblocks. What do you think in particular differentiates your program that's allowed you to get to this stage where you're basically now just trying to complete a resubmission?
Right. Thank you Tazeen. Ultimately what differentiates us is this idea that we had that myostatin unfortunately is homologous to other proteins in the body. When all of these efforts to target either mature myostatin or the myostatin receptor, the trap programs have just led to suboptimal efficacy and/or safety, and they've resulted in terminations of clinical programs, most recently the one from Roche in SMA and FSHD. From our standpoint, we had this very unique way of approaching both pro and latent myostatin. That gave us exquisite selectivity. As a result, we think we had a molecule that we could move into any patients, and today we'll get into this. We're even treating now toddlers and infants with our drug.
It's exquisitely selective and very safe. That's one, is starting with a molecule that truly is differentiated off of our platform. I think secondarily, we chose well. We chose the right indication. We actually did a robust phase I, phase II, phase III clinical trial program. We were informed every step of the way on the right dose, the right interval, the right patient population to study in our pivotal trial. As a result, I think it has set us up to demonstrate this, you know, incredible opportunity to transform the lives of children and adults living with spinal muscular atrophy, and then sets up with a series of genetic and acquired myopathies and neuropathies. It gives us a set of different diseases to apply our success in SMA to over the years to come.
Okay. Thanks for that. We'll talk about the resubmission in a second, but since we're on the topic of the drug itself, it's also differentiated in the sense that you're not trying to have patients swap out of whatever other drug that they might be taking. Maybe talk to us about the benefits of that, both for the patient and commercially speaking?
You know, I'm really thankful you mentioned that Tazeen because I often hear that, while there have been innovations brought to the SMA community over the last decade, and these are very, very important therapies that have brought massive innovation to a community that desperately needed it, all of those new therapies focused on motor neuron survival and motor neuron health. Certainly Biogen ushered that in and Ionis with SPINRAZA. Over the last 10 years we've seen this innovation come in, really, Spinal Muscular Atrophy, the principal organ affected by the disease is the muscle. When one is trying to improve the lives of patients living with Spinal Muscular Atrophy, two things have to happen. One needs motor neuron health, the other is they need muscle health.
One entire side of the motor unit, which is comprised of the motor neuron and the muscle, there's been no innovation. Our idea was if we could address the muscle component associated with this disease, we could make a meaningful difference for patients. I think one thing is really clear, and our phase III SAPPHIRE trial really underscored this. If you're treating a patient, child or adult, with SMA, two things you should want to do. One is, you should want as much SMN protein as possible to address motor neuron health, and you should probably try to eliminate myostatin, which is the body's natural negative regulator for muscle growth. If you can do those two things biologically plausibly, you can provide the best outcome for patients from the perspective of motor function.
We're really excited to be, we think now just a few months away, any time now, from ushering in the next wave of innovation for patients living with spinal muscular atrophy, which of course is the first and only muscle-targeted therapy, that has been successful in a phase III trial, and that's apitegromab.
Yeah. Okay. Now let's go back to the business at hand, which is the resubmission process.
Yeah.
Right? How are you feeling about that? Where are you in that process? You've talked about this now at length on multiple earnings calls, but we're here now, and is it your expectation that by year-end you will have a commercially available drug?
First of all, I want to thank you for your patience Tazeen, 'cause I think you initiated coverage, like, right before some of all of this began with our fill-finish facility and ultimately the delay that we have had. Maybe just walking back the audience to where we were, we had this incredible phase III data that we presented in Q4 of 2024. We submitted our BLA in January of 2025, and the FDA, given the high level of innovation of the first ever muscle-targeted therapy that was studied for patients living with SMA, granted us a priority review with an action date of September 22nd of 2025. We were poised to launch the drug in Q4 of last year.
Of course, we disclosed on August sixth that there was a general site inspection at our fill-finish facility, Catalent, Indiana, which is owned and operated now by Novo Nordisk. This is a plant that has had, you know, some dust-ups before on inspections that were impacting other therapies, including around the time of the pandemic, SPIKEVAX from Moderna. We had thought and hoped that their Novo Nordisk's response to those observations would get us through to the other side by September of 2022. That was not the case. We received a CRL solely based upon the state of compliance at our fill-finish facility. That followed an Official Action Indicated classification of the facility in a Warning Letter, which usually can lead to a prolonged, you know, path to remediation of that plant.
All of that happened in Q4. At the same time, we had a really productive, constructive, and collaborative in-person Type A meeting with the FDA in November of 2025. Recall where we were at that time. It was the longest federal government shutdown in the history of this country, and yet we were granted an in-person meeting with the FDA. All the leaders of the FDA were there. Novo Nordisk came in. The leaders from Cure SMA were there as well. One thing that came out of that meeting is that we believe there was a shared understanding of the unmet need that still existed for the SMA community, despite the level of innovation targeting the motor neuron, and that there was a shared sense of urgency to try to get this resolved for apitegromab.
They've been working, the FDA, with Novo Nordisk pretty aggressively, which has led to a re-inspection of the facility just recently. At the same time, you know, we looked ourselves in the eyes, we looked at the SMA community, and we said, "We've got to be better on this application than we were on the last one, and we should actually have an alternative to Catalent, Indiana." We worked very, very hard on a second fill-finish facility.
Really in close collaboration with the FDA from November of 2025, which was our Type A meeting, to our Type C meeting in March of 2026, we aligned with the FDA that we should resubmit our BLA with not only Catalent, Indiana, but also a second fill-finish facility, one that we have, I think, beat speed records to stand up that facility, and we are going to have commercial drug that will be releasable as early as early Q3 from that second fill-finish facility. We were very pleased last week on our Q1 call to let you all know that our BLA has now been accepted by the FDA with not only Catalent, Indiana, but two fill-finish facilities.
The re-inspection has taken place now at Catalent, Indiana, the FDA, by their own guidelines, has 90 days to evaluate that inspection and determine if they can reclassify that facility. Within that same 90 days, we're going to have drug ready to go from the second fill-finish facility. Overall, we were granted a Class 2 resubmission, with an action date of September 30th. What we have been guiding is that we see with either or both of these facilities a path to approval, by Q3, we're very excited about that. I can assure everybody that we will have plenty of apitegromab launch supply for Keith and team, should one or both of those facilities be approved, within our application.
Okay. Thanks for all of that color. There's a lot of moving parts between now and September. You know, questions that we tend to get are there experiences where FDA can, or you can point to where FDA has had a similar scenario where there was the potential of one or two facilities that they would need to look at in order to get to an approval date?
There's a few, if I may, that are also within this window of what's been happening with us. You guys may have noticed beyond Eylea HD and Eylea HD pre-filled syringe, Regeneron had a cancer therapy in October that they actually had two fill-finish facilities as part of their application, Catalent Indiana, and I think they had one through their relationship with Sanofi. When it became very clear in October of 2025 that Catalent Indiana was not gonna be reclassified in time, they dropped Catalent Indiana without any timeline hit, and they were approved with the other facility. Within the contours of our Type C meeting in March of 2025 with FDA, we did talk about that this can, in essence, be a horse race.
What helps us get approved the fastest because of this high unmet need for the SMA community and the shared sense of urgency for apitegromab to be launched for children and adults living with SMA? Should one look like it was outpacing the other, we could actually move one to an sBLA, but let's get this drug approved. Now, what has happened is this 90-day window post re-inspection and how fast we've been moving at the second fill-finish facility, these lines are starting to sort of overlap with one another. We'll just remain in close contact with the FDA on this progress. Look, I would love nothing more than something fast and with both.
At this point, our commitment is to the SMA community, and we'll take either one at this moment in time so we can, you know, get to the children and adults that desperately want this innovative therapy, apitegromab, to be added to their current treatment regimen.
Okay. Just to clarify one thing you said, if within that 90 days FDA does come back for whatever reason, they might have additional questions, do you believe that the time remaining would allow for that second facility to meet their standards and get approved by your September PDUFA?
It's an excellent point. I noted last Thursday, but I haven't noted here, and I think it's important to say this to our audience, is that all of the apitegromab that is required for review and approval from the second fill-finish facility has been filed. It's like there isn't really anything in front of us. The data the FDA will have from the product that has been filed, and like I said, the drug is all ready to be released upon approval as early as several months before the September 30th PDUFA date. You start to see a lot of July. There's a July with the commercial drug that's available from the second fill-finish.
There's a July, mid to end of July, that is that back end of the 90-day window for the FDA to deliberate on whether or not to reclassify the facility. All of those things may be aligned. Let's just say the FDA woke up and said, "We want a clear Catalent Indiana," we would move the second fill-finish facility into an sBLA, and we would launch with Catalent Indiana. Vice versa. We're not gonna give up on Catalent Indiana. If they say, "Look, you've made meaningful improvement, but a Warning Letter is serious business. We wanna keep you here for a little bit of time.
We may not require another inspection, but see how you work through, you know, your remaining issues," if that were to be the case, then there definitely would be the case where you could see Catalent, Indiana becoming an sBLA and us approving with the second fill-finish facility. Again, we're hopeful that it's both, let's see how the days progress. I wanna be, you know, publicly thankful to the FDA 'cause they really have been extremely flexible with us in terms of understanding the different paths to approval as quickly as possible.
Okay. Great. Understanding that the most important thing is to get the drug available for patients as soon as possible, longer term, do you have a preference for which facility you think would be your primary facility?
I do, it's largely business model related. Our second fill-finish facility, which I should note is a U.S.-based facility, it's in great standing with all regulators. They have a long-term and recent positive inspection history, in fact, some of their most recent therapies that have been approved, the FDA waived the PLI, which is actually interesting as well. As I noted, they have about three dozen approved products. That is their business model. As you may know, I think it's worth describing, Catalent Indiana was owned by Catalent, which is now owned by Novo Holdings. This is a little confusing, let me clarify that.
When Novo Nordisk needed more fill-finish capacity, after Catalent was acquired by Novo Holdings, Novo Holdings then sold three fill-finish facilities to Novo Nordisk, and they are now part of the internal operations of Novo Nordisk, which really has no interest in being a third-party CDMO. We know, and we have known, that they're gonna exit the CDMO business, so we would be migrating our operations from there anyway. I think overall our ambition would be long-term to have both 'cause, you know, having redundancy is always a good thing from a supply chain perspective. It is that second fill-finish that looks like the long-term supplier, and we'll be thoughtful about this incredible commercial demand that I expect from Keith, and how much redundancy we do wanna have in the supply chain over time. Hopefully, that's helpful.
Yeah, that is. Part of the reason I ask is because I wanted to understand the role that the Indiana facility is gonna play for your European application.
Tazeen is raising a really important point that hopefully I can clarify for our audience here in Las Vegas as well as tuning in remotely. Unlike the FDA and EMA, we have not had any delay and/or, you know, the equivalent of a CRL. Okay, we're still on file at EMA, and in fact, the European regulators have been really flexible also, and we like the state of where we are with our MAA in Europe. The difference is because we haven't had a rejection, our application includes Catalent, Indiana only today.
Now, they're obviously, because they have a mutual recognition program with the FDA, they're waiting for that facility to be cleared, and then we would imagine we get on the agenda for CHMP, and then we're approved, and we launch in Europe starting in Germany in the second half of this year. That is our guidance. We remain firm on that guidance. EMA is well aware of the second facility. The second facility is in very good standing with EMA. We're talking to them about the same elegance of what we have, you know, with FDA, you know, and wanting to make sure that we have that same level of optionality.
Today, the EMA only has Catalent, Indiana in the application, and we are discussing with the European regulators, the nature of the second fill-finish facility, all the good work that's been done, the agreement with the FDA, and how that could work to provide us that same belt and suspenders approach to an approval in Europe that we currently have in the U.S.
Okay. Now let's talk about the commercial opportunity. We know Keith Woods from his previous life. As you mentioned, you've set up a good team to do the launch, but maybe walk us through what you think the initial uptake is going to look like. Included in that would be questions around negotiating with payers. Presumably, you've already had some early discussions.
Yep
about that. Maybe talk about range of pricing, if you could.
Sure. One of the things I think Keith and I would say and have said is we would not have wanted a delay for even 24 hours. Given our own time that, you know, we've taken to be with the community, be with patients, be with families, be with parents, be with the patient advocacy groups. They, they were clamoring for this therapy given the strength of our phase III data. If one were to kind of make lemonade out of this, how when we brought Keith in at the end of April, and, of course, he's done incredible work with us at Alexion and, of course, his best work yet was at argenx.
He really didn't have time to make all of the, I think, execution and planning decisions that he would have liked to have made before September 22nd of 2025. How do you use that extra time to actually make sure you got the team, you have the plan, and you have everything in place to support every one of the stakeholders, from a patient, healthcare provider, and payer perspective. We've been using that time wisely. I think as a result, Keith has spent a lot of time on the access programs, has spent a lot of time on the getting beyond just national payers into regional payers, and making sure that they understand, you know, you know, what apitegromab can offer to them.
As a result, I think we're more well-positioned for the commercial launch today, you know, in these next, you know, weeks and months than we were back then. What do we expect? You know, sometimes when something is ready for an approval, like we were all thinking the review was reading positive for an approval in September of 2025 and it doesn't get approved, the demand for it just increases. I think what we've seen is almost 100% of the SMA community, I think Keith, as you quoted, 95% of SMA patients say their number one need is more muscle strength, and motor function, tied to their irreversible, muscular atrophy that they're currently facing and muscle wasting. Almost all patients want to address their muscle component of their disease.
Nearly 3/4 of all neurologists acknowledge that dual modality is the way to go for the SMA community. Treat the motor neuron as well as treat the muscle. That's a really nice setup for demand. I think as you aptly state, Tazeen, there is that payer side. There's usually like a little bit of time before you get to that steady state of conversion time and conversion rate. You know, will they want to limit, you know, an approval for reimbursement to the phase III criteria or the label that we have? We believe it's not a matter of if, it's when the payers are paying according to the label that the FDA grants us. There's a miscellaneous J code. There are formularies to work with. There are infusion schedules.
This is an every 4-week infusion, as you all know, where 95% of our patients that have been in our long-term extension trial remain on treatment. We think that says a lot about the value proposition. I think the demand will be there. I think the, I'll get to pricing in a second. I think it will take a little bit of time before we get to sort of that steady state of conversion time and conversion rate that makes this a little bit more predictable. One thing's for sure, the SMA community does not take no for an answer. They've been through this before when they've been waiting for their SMN-targeted therapy.
I think we're going to between physicians and patients and payers, and of course, with Scholar Rock Supports, which is our access program that we've set up to walk every individual patient through the reimbursement process. We think the setup is quite well for us. Finally, regarding pricing. I've been asked about this quite a bit, and while it's a little premature to talk about the specific price, what I would say is we always think about a couple of things. One, the rarity of SMA. There's only a few thousand patients in the U.S. living with SMA that have received an SMN targeted therapy, about 7,000 of those, and another 28,000 around the world. That's number one. Number two, the severity of the disease without our therapy.
Despite this high level of innovation of these SMN targeted therapies, patients eventually plateau and start to progress or revert to the progressive form of SMA, where they're losing motor function year on year. We saw that in our phase III trial. We see that in their long-term extension trials. Yet with apitegromab, what we saw was a move from a loss of motor function to a return of a gain of motor function for the entire population of patients, and nearly a 4x greater likelihood of patients having a 3-point improvement in the highest bar that you could have, which is the Hammersmith Functional Motor Scale in SMA. We think the value proposition is there. I think we can price proudly. I think we'll be reimbursed, you know, at that price.
I think it's very much reflective of the value that we're going to be providing. I do think the community and the patient size is not so large that the budget impact will be significantly great. Demand will front run the access side, but I think overall we're really excited about the long-term prospects to serve these 35,000 patients globally for many years to come.
What do you think the level of awareness is among patients? This is a small community, but a very educated community. Obviously you don't start detailing until you get approved.
Yeah.
Based on where you are, you know, should we expect a bolus of demand upon approval?
Demand is probably going to be measured in that enrollment in Scholar Rock Supports, which is going to be some combination of and it has to happen this way, right? The patient has to want the therapy, and the physician has got to want to prescribe the therapy, and then they enroll in Scholar Rock Supports, and that will be that first indicator. Keith is still determining exactly what launch metrics he might share. Obviously, revenue at the end of the day is what you all care about. Look, I think demand will front run revenue. That is just the way it is usually with these rare disease launches, especially when, you know, it is an IV infusion and it is a miscellaneous J code.
I do think that The awareness is high, as I was mentioning, and I really can't wait to have a trade name or a brand name for the drug because unfortunately, the SMA community cannot say apitegromab. Actually, half the people at Scholar Rock cannot say apitegromab. Nobody knows how to pronounce it. What they say is they just want Scholar Rock. They call our drug Scholar Rock. They can't wait for Scholar Rock to be approved, and I think that's really cute. The adults call it the juice, which is altogether a different thing. I think the awareness of this therapy is quite high. I think the delay has actually even heightened the awareness, and the leadership at Cure SMA has kept us front and center.
We'll again have a very, very strong presence at the Cure SMA annual conference in Orlando in June. We'll be as well represented as any company, and Keith, as you know, and Vikas, we just had a great presence at SMA Europe also as we get ready to launch outside of the U.S. It is there for sure, we think we're just the right team and the right company and it's the right product. This is something that I think the SMA community is very much looking forward to.
Okay. We'll keep saying apitegromab until people can get it right.
Yeah, you and I are gonna keep saying apitegromab.
Repeat it. Of course it's gonna take a little bit of time to record actual revenue, but looking at other rare disease launches as a comp, new patient start forms, whatever information you're gonna be providing in the early portion, I think people are wanting to know. It sounds like from what you're saying that the demand is going to be there, that you will see the translation pretty quickly from approval to, you know, doctors wanting to get their patients on it. I don't wanna put words in your mouth, but am I interpreting that correctly?
I think there's reasonable demand on day one. I think it is just the combination of sort of like conversion rate and conversion time from the payer side, and we'll be patient 'cause it is, it's gonna be a matter of when and not if.
Right
The product gets reimbursed and I think the setup is quite nice for us. But I would just advise folks to not get crazy about what the revenue curve looks like 'cause these things take time to do it right. I think that we're well set up to do it the right way over time for the, for the community.
Okay. Last question really quickly. Cash needs balance sheet strength.
We were very thankful in our call last week to announce that our cash balance was $480 million at the close of Q1. That was fortified by a pull down on our debt facility of $100 million in Q1, as well as leveraging our ATM program for another $98 million. I think the combination of the $480 million, the clarity now on an accepted BLA with two fill-finish facilities, the ability to pull down another $150 million off the debt facility at approval, and of course we do have a priority review voucher, which we intend to monetize at approval as well. Sets us up very, very well.
I know we didn't get to the pipeline, not only to support the commercial launch for apitegromab in the U.S. and Europe, but also to keep fueling the pipeline that Akshay is very focused on with FSHD, with the OPAL study in infants and toddlers, with SubQ apitegromab, and with SRK-439, which is our highly innovative, high potency, low volume, high affinity myostatin inhibitor, which is in phase I, and we'll have some data on that later this year also. We wanna continue to build what we believe is a very special rare disease company. Our superpower is myostatin biology. We think we're well-positioned for growth through the end of this decade and well into the next, and I look forward to working with Tazeen and the Bank of America team and keeping you all apprised on our progress.
Okay. With that, we're out of time. Thank you David.
Thanks Tazeen.
Thanks everybody.