Hello, and welcome to Scholar Rock's BLA resubmission call. At this time, all participants are in a listen-only mode. Following the presentation, we will conduct a question-and-answer session. To ask the question during the session, you will need to press star one one on your telephone. You would then hear an automated message advising your hand is raised. To withdraw your question, please press star one one again. We ask that you limit yourself to one question and one follow-up. Please be advised that today's conference is being recorded. I would now like to turn the conference over to Scholar Rock. Please go ahead.
Good morning. I'm Laura Ekas, Vice President of Investor Relations at Scholar Rock. With me today are David Hallal, Chairman and Chief Executive Officer, Akshay Vaishnaw, President of R&D, Keith Woods, Chief Operating Officer, and Vikas Sinha, Chief Financial Officer. During today's call, David and Akshay will provide remarks. We will then open the call for Q&A with all four members of the management team. Before we begin, I'd like to remind you that during this call, we will be making various statements about Scholar Rock's expectations, plans, and prospects that constitute forward-looking statements for the purposes of the Safe Harbor Provisions under the Private Securities Litigation Reform Act of 1995. Any forward-looking statements that are made only today and should not be relied upon as representing our views as of any future date.
I encourage you to go to the Investors and Media section of our website for our most up-to-date SEC statements and filings. With that, I'd like to turn the call over to David. David.
Thank you, Laura, and thanks to everyone for joining the call today. I am pleased to announce that we have sub-resubmitted our Biologics License Application to the FDA for Apitegromab for the treatment of children and adults living with spinal muscular atrophy. As a reminder, the sole approvability issue for Apitegromab noted in the complete response letter that we received on our priority review PDUFA date was related to observations identified at a routine general site inspection of the Catalent Indiana fill-finish facility, which is owned and operated by Novo Nordisk. Our BLA resubmission is an important step forward in our mission to deliver Apitegromab to the SMA community as quickly as possible. Patients with SMA continue to experience muscle wasting and functional decline despite ongoing chronic treatment with SMN-targeted therapies.
Apitegromab has the potential to be the first and only muscle-targeted treatment to improve motor function in SMA by addressing the principal organs clinically affected by the disease. We continue to work urgently to bring this transformative therapy to children and adults in need. As we have noted, since our constructive and collaborative in-person Type A meeting last quarter, which included Cure SMA and Novo Nordisk, we have been gratified by the agency's shared understanding of the high unmet need in the SMA community and the shared sense of urgency to bring Apitegromab through the final step in the U.S. regulatory process. Since that Type A meeting, we have made steady and rapid progress. This has enabled us to resubmit our BLA in complete alignment with the agency to include Catalent Indiana and Scholar Rock's additional fill-finish facility, reflecting the meaningful progress at both fill-finish facilities.
Let me first address the rationale for resubmitting the Apitegromab BLA prior to FDA reinspection of Catalent Indiana. This decision was made in alignment with the FDA and reflects the significant progress that has been made by Novo Nordisk at the Indiana facility. As we discussed during our Q4 call, the FDA and Novo had a constructive meeting in early Q1, which was followed by an FDA site visit. During both the Q1 meeting and the site visit, no additional corrective actions were requested by the FDA to Novo's remediation plan. FDA also stated to Novo that it intends to conduct a site reinspection following routine manufacturing activities, which resumed in late February. Based on this progress, we were pleased to align with the FDA to resubmit our BLA prior to reinspection. We continue to anticipate a reinspection in the coming period.
I would like to now turn to the decision to include our second fill-finish facility in the BLA resubmission. As we discussed on our Q4 earnings call, I continue to be very pleased that progress with our second fill-finish facility is moving quickly to strengthen our supply chain and support future growing demand across our planned global commercial footprint. On March 3rd, we engaged with the FDA in a positive Type C meeting to discuss our progress towards qualifying this second facility. Based on our accelerated fill-finish timelines for Apitegromab commercial supply, we aligned with the FDA to include the second fill-finish facility in our BLA resubmission. Our approach provides a significant optionality with two independent paths to Apitegromab FDA approval.
Importantly, it reflects both our confidence in an FDA re-inspection of Catalent Indiana in the coming period and our accelerated timelines to release commercial Apitegromab from the second fill-finish facility. We anticipate a review period of up to six months from the date of resubmission and a PDUFA date in late September. Akshay will share more details on the BLA resubmission and discuss the flexibility and optionality we have with two fill-finish facilities included in the application. We remain committed on behalf of patients, and we are grateful that important progress continues to be made. The sustained cadence of activity over these past few months reflects the efforts by all parties to bring Apitegromab to children and adults living with SMA. We expect this momentum to continue. Commercially, we are ready now.
Our team continues to operate with urgency as we prepare for the U.S. launch of Apitegromab. Scholar Rock remains focused and disciplined, advancing the critical activities and capabilities required to deliver a seamless launch and support patients from day one. In addition to the U.S., we look forward to serving children and adults with SMA in Europe. The review of our MAA is progressing very well, and we expect a mid-2026 decision from the European Medicines Agency. We continue to build momentum with launch readiness activities and anticipate a launch in the second half of the year, beginning with Germany. Akshay will share more details on the continued progress of our Apitegromab MAA shortly. We know it is not a matter of if, but when Apitegromab will be approved for children and adults with SMA in the U.S. and Europe.
We continue to be emboldened by the commitment we have made to the more than 35,000 patients globally living with SMA who have received an SMN-targeted therapy. With that, I'll now turn the call over to Akshay. Akshay?
Thanks, David, and good morning, everybody. As David noted, we remain focused on bringing Apitegromab to children and adults with SMA as rapidly as possible. To that end, I'm delighted that based on alignment with FDA, we have now resubmitted our BLA with Catalent Indiana and a second U.S.-based fill-finish facility. The updates to the BLA for resubmission were limited in scope and primarily composed of a standard safety update from patients enrolled in the ongoing Apitegromab clinical studies and the early access program. The resubmission document also included the draft labeling from our last round of interactions with the FDA. Regarding the path forward, we anticipate an FDA decision on acceptance within 30 days of this filing and up to a six-month review period. Based on Novo Nordisk's progress, we're confident in Catalent Indiana's readiness for a re-inspection. The steps required are as follows.
First, an unannounced FDA re-inspection of the Indiana facility will occur in the coming period. Next, following conclusion of that re-inspection, any Form 483 observations will be issued. Parenthetically, I note that it is typical for the FDA to issue a Form 483 following inspection, and this would not necessarily limit FDA's ability to favorably reclassify the status of the Indiana facility. Finally, subsequent to the re-inspection, Novo would have 15 business days to respond to any potential observations, following which FDA would review the responses and reclassify Indiana as appropriate. While the length of the process I've just outlined can vary, we remain encouraged by the rate of progress and the level of FDA engagement. With both Catalent Indiana and the second fill-finish facility included in the BLA resubmission, importantly, we now have significant optionality with two independent paths to FDA approval.
We've been guided by the agency that whichever fill-finish facility leads to the most rapid approval will remain in the BLA while the other could be removed. If there were a case where one facility was removed, we would subsequently submit an sBLA for the other facility shortly after the Apitegromab approval to strengthen our supply chain and support future commercial demand. Importantly, we anticipate having plenty of commercial Apitegromab filled and released from our second facility a few months prior to an anticipated PDUFA action date. With the collaborative and urgent approach that the FDA has shown since the Type A meeting last November, we anticipate the decision by no later than the end of September 2026. Turning now to Europe, our MAA for Apitegromab for treatment of children and adults with SMA continues to progress well through EMA review.
As we mentioned during our Q4 call, approval in Europe also requires FDA clearance of the Catalent Indiana facility. Based on our discussions with EMA, they're aware of the progress at Indiana and are comfortable with the review timeline that accounts for the FDA's clearance of that site. We continue to be very pleased with how the review is progressing and believe we're on track for a mid-2026 decision in Europe. With that, I'll turn the call back over to David. David?
Thanks, Akshay. In closing, we remain focused on bringing Apitegromab to children and adults living with SMA. We are working expeditiously to deliver on our ambition that globally, any patient with SMA who can benefit from Apitegromab should have access to Apitegromab. This is indeed what we know well and what we do well, and we are confident in the significant opportunity that we have to serve patients with SMA, which provides a strong foundation for growth through the end of this decade and well into the next. I would like to thank the FDA, Novo Nordisk, and our second fill-finish facility for their continued high level of engagement. I'd also like to thank the Scholar Rock team, who show up every day with relentless focus. Importantly, I am grateful to Cure SMA and the global SMA community for their continued support.
We look forward to updating you on our continued progress. With that, we'll now open the line for questions. Operator?
Thank you. Ladies, and gentlemen, as a reminder to ask the question, please press star one one on your telephone, then wait for your name to be announced. To withdraw your question, please press star one one again. We ask that you limit yourself to one question and one follow-up. Please stand by while we compile the Q&A roster. Our first question comes from the line of Mani Foroohar with Leerink Partners . Your line is open.
Hey, guys. Thanks for taking the question. Congrats. Great day for you guys and most importantly for patients. A quick one on approval timelines. You laid out a path to, presumably PDUFA date that assumes kind of a usual six months, external review process. Given how much you've disclosed and how often we and others have talked about the amount of the review that has already occurred, some label discussions, review of the data packet under prior submission, how should we think about that timing? Is that? Should we think of that as an outside date, mid-range of expectations? How should we think about it?
Yeah. Thanks, Mani. I'll start, and then Akshay will jump in. I think we were very clear that we view getting back on file with the anticipated acceptance of our resubmission as being accepted under Class 2 for the following reasons: The reinspection has not yet happened at Catalent, Indiana, and of course, you know, the second fill-finish facility requires new data for the FDA to review. Importantly, as Akshay laid out, we are gonna have commercial drug release from that second facility several months before an anticipated, you know, sort of Class 2 review period of up to six months. But that's how Akshay and I are looking at this, is up to six months.
Hopefully, the FDA's engagement, you know, really since our highly collaborative, in-person Type A meeting last quarter, is sort of a predictor that we're gonna continue to work very collaboratively and urgently together. Akshay.
Yeah. You know, just on that last point, I think we'd be very gratified and grateful to the FDA for their collaboration and how much they've tried to help us with the best path forward. I'm sure during this review period, they'll do the same. You know, as you said, September is the standard six-month or so timeline, but I'm sure everyone, including us, FDA and all parties, will do their best to get this drug approved into patients as soon as possible. Let's go from there.
Mani, just to cap it off, you are absolutely right. The resubmission is a lighter document, right? I mean, Akshay can comment on really what is in the resubmission. This is really anchored to clearing one of these two fill-finish facilities more than anything else. Akshay.
Yeah. I mean, you know, there are these two significant options now available to us that the FDA is going to help with during their review. I'm sure they will work as expeditiously as possible to enable getting this drug to patients based on all the interactions we've had and the quality of the supportive dialogue we've had. You know, it could happen before September. I'm sure everyone's trying to because all parties appreciate the urgency of getting this drug to SMA patients. At the same time, we have to let FDA do their work.
That's helpful. A quick follow-up, which I guess maybe is more of the cost question or a commercial one. As you've had time to engage with the market, with this somewhat more protracted than usual review process, given the regulatory catalyst, how should we think about pricing in terms of where we should think about potential price ranges that you'd launch at? I know you can't give us an exact number, but where we should look in terms of precedents, et cetera. While I know this is certainly not the type of indication that draws a lot of pricing scrutiny, to what extent does the evolution of the U.S. versus OUS pricing discussion, MFN, et cetera, influence how you think about that?
Yeah, it's a great question, Mani. I'll start. This is David. First and foremost, as we noted, a big part of wanting to have, you know, multiple fill-finish facilities is our anticipated 50-country operating platform, so that we can reach the 35,000 patients that have received at least one SMN-targeted therapy. We wanna have, you know, we really wanna have a very robust supply chain. We also feel like, you know, with the things that are happening with MFN, it really our global plans are unchanged in any way. We see very little impact to Scholar Rock in the global launch of Apitegromab.
As it relates to pricing, it's still a little premature, but I will say that we continue to be anchored by a couple of principles as we think about establishing a price for Apitegromab. One is the rarity of the disease in which, you know, there's somewhere between 5,000 and 7,000 patients in the U.S. that have received at least one SMN-targeted therapy and about 28,000 outside of the U.S. So it's quite a rare disease. Secondly, is the severity of the disease, and as Akshay and I have noted, you know, multiple times, despite the use of ongoing chronic very important SMN-targeted therapies, patients tend to, over time, revert to the progressing form of SMA, where there's a loss of motor function.
Lastly, the compelling clinical data that we've been able to deliver through our robust clinical development program over these last seven, eight years, where we were able to show that patients had a return or gain of motor function as opposed to a loss of motor function. We think those things combined are very important considerations as we move forward and establish a price. That, again, as I've noted, we see very little impact on MFN to us, and we are really looking forward to reaching patients in upwards to 50 countries around the world over time.
Great. Thanks again, guys.
Thanks, Mani.
Thank you. Our next question comes from the line of Tess Romero with JP Morgan. Your line is open.
Hey, David and team. Congrats on all the progress here, and thanks for taking our question. First one from us is, procedurally, at this Type C meeting, did the FDA align with you on submitting the BLA before the reinspection because your second fill-finish facility was BLA ready, or was it also because of progress at Catalent? Second question, just as a matter of housekeeping, the facilities are not tiered in your filing, right? They are just both included. Thank you.
Yeah. They are both included in the resubmission. Akshay should take on. I think your question is a really good one. The resubmission, including both fill-finish facilities, was on the merit of the progress in each one of the fill-finish facilities. I think that is a really important point, and I'm glad you asked the question. For a closer look at that, I'll turn it over to Akshay.
Yeah, a closer look, but I would also say, Tess, that, you know, we have to respect the process here. The FDA always considers each sponsor and each file in the context of what's contained there, the unmet need, the quality of the data, and the progress to date. You know, in our situation, they looked at the two sites we're working with, and they've guided us to this resubmission, which we're grateful for. You know, everyone will work hard now to bring one or both to fruition and get this drug to patients. You know, I'm not going to go into the exact back and forth at the various meetings we've had.
We're just grateful that there have been, you know, multiple meetings and that there's been good alignment and, you know, very good guidance to get this drug to patients as efficient as possible.
We do think it was helpful, as we noted on our Q4 call and again here today, you know, the interactions that FDA has had with Novo, both in terms of the early Q1 meeting and the site visit. All is helpful for the FDA to assess the progress being made by Novo on their remediation plan.
Thank you.
Thank you. Our next question comes from the line of Michael Yee with UBS. Your line is open.
Hey, guys. This is Kyle Yang for Michael Yee. Thanks for taking our questions. Just help us understand the scenarios there. Hypothetically, if the Catalent reinspection is not successful, do you expect to still able to get approval with the second facility? What other boxes do you have to check, you know, with the second facility to ensure this can get approved by the potential September PDUFA date? Just help us understand the cadence of events if the reinspection is not successful. Thanks.
Yeah. No, thank you. I think Akshay and I tried to illuminate in our prepared remarks the beauty of filing with all the progress that's been made at both fill-finish facilities. The beauty of filing with both really provides us some wonderful flexibility and optionality, and I'll have Akshay make some comments on that.
Yeah. I think this filing has been enabled by outstanding work, by Novo Nordisk, by our second fill-finish facility. Obviously, all our colleagues here at Scholar Rock have been working with those two facilities. Everyone in that triumvirate has worked as expeditiously as possible to make this filing happen. Most of all, it's all happened with complete transparency with FDA and their guidance. I think we're looking forward, hopefully, to a successful reinspection of the Catalent Indiana facility based on all the progress they've made, and they've reported to us and the agency. We're optimistic about that. Of course, the FDA has to do an unannounced inspection and we have to let them do that work, but we do feel good about Catalent Indiana, and that they're on the right track.
That is the first goal, to get Catalent Indiana facility back online, with the FDA's blessing and get this drug approved within the time frames we've already discussed. Now, the beauty of it is that, based on the guidance by the FDA, we've worked hard on a second fill-finish facility, and it's great the progress we've made to date has also allowed inclusion of that in this resubmission. Again, with the agency's blessing. I'm not gonna sort of outline exactly where we are in that process, but I think most of you are familiar that bringing a second facility involves engineering runs, PPQ runs, and all the rest of it. We've made outstanding progress on all of that stuff. There's more work to do and more data to file.
Again, we're very pleased that there are two significant options here for Apitegromab for patients, and the agency will be endeavoring to get to the finish line with one or the other, or both.
Thank you, Akshay. I would just highlight, as Akshay noted earlier in the call, we think it's really important with those accelerated timelines that we will have, commercial Apitegromab ready for release from that second facility several months before an anticipated, late September action date.
Thank you. Our next question comes from the line of Amy Li with Jefferies. Your line is open.
Hey, thanks so much for taking my question, and congrats on the progress. Just quickly on the September PDUFA date, and then the 30-day to acceptance. What visibility have you had gotten from the FDA on the re-inspection timing? Would you need to get re-inspected before the FDA accepts the BLA? Or could that happen afterwards? I have a quick follow-up on the second facility.
Sure. Yeah, no, I think the way to look at it, right, is, first of all, everything that we've described today, as Akshay and I have noted, has been in complete alignment with the FDA, through you know, really high-quality interactions with the FDA. The resubmission of the BLA, which is inclusive of both facilities, is based upon the progress of each one of those facilities. Recognizing that we are now resubmitting prior to re-inspection, which again, was agreed upon with the FDA, the way to look at it is that that re-inspection would not need to have happened for the acceptance of our resubmission. The way to think about it is it would be a review issue as part of our resubmission. The outcome of that inspection would be, you know, the key review issue, if you will.
I think that's the right way to look at it.
Okay. Thanks so much. In terms of the second fill phase, fill-finish facility, during your Type C meeting with the FDA, have they been able to review some of the initial data from PPQ runs, comparability, bridging data? I know, you know, we discussed that, you know, they're allowing kind of an accelerated pathway because you're able to take away some of the data you generated from Catalent. Just wanted to see from a September, like when this site will be ready, if there will need to be inspections and if that site will kind of fulfill the September PDUFA, just in case anything happens to Catalent.
Yeah, I'll start and then Akshay can jump in. Yeah, the FDA in that Type C meeting is well aware of all of our timelines, all of the progress. Everything is actually just laid out from tech transfer, engineering runs, PPQ runs, release of product, when all of the data would, you know, they would have access to, leveraging data from the other filler. All of that was obviously discussed and agreed upon with the agency. I would just note that if there is a, you know, a PAI required, it would all be contemplated within the framework of a review timeline that we've sort of anticipated would provide a late September PDUFA date. Akshay?
Yeah. I think just to add one thing to what David said, which is that the second fill-finish facility is a U.S.-based facility and is in good standing with the FDA. There have been multiple inspections before now, so we have a high degree of confidence in the site. Yeah, they're executing on that sort of list of work that David just mentioned. FDA is well aware of where we are on that path. In alignment with all parties, we've now resubmitted the BLA.
Excellent. Thank you.
Thanks, Amy.
Thank you. Our next question comes from the line of Tazeen Ahmad with Bank of America. Your line is open.
Hi, good morning. Thanks for taking my question. I have one on the label. Based on discussions that you've had with payers so far, is there specific language that you think is gonna be necessitated in the label in order to allow for broad-based coverage? 'Cause this is a rare disease, and I'm just trying to understand if there is something outside of simply getting an approval from the FDA that might enhance and, you know, make more efficient the process to get covered. Thanks.
Yeah. I'll start it and Keith may wanna jump in. Tazeen, as we've described it, you know, over these past few months, Akshay and myself, the company felt really good about, you know, where we last left it. With the agency in our label discussions, which were just a few days before the PDUFA, and it's called out in the CRL that is redacted that the FDA released to the public that they would like us to just go back to that label in our resubmission, which we have done. We felt, you know, really good about where we are, but we have been very disciplined not to comment too specifically, given the fact that we want the agency to do their work.
When we talk about the label, we wanna actually have an approval, a letter from the FDA. Nonetheless, I think what you're highlighting is, you know, given some precedents at the FDA and the way they've looked at SMA as one disease as opposed to, you know, sort of segmenting it out, we generally felt like we were in a good spot to be able to serve a meaningful number of patients with SMA. I would just say that Keith and his team have used this time very, very well to engage with key national, key regional payers and begin to establish alignment on just sort of how would one think about a label that might be broader than that of a phase III pivotal trial criteria.
I think that work will continue to be ongoing. As I've noted, and I'm very thankful to Keith and the team that they are ready to launch now. I think that's reflective of a lot of work that's been done with the physician and healthcare provider community, the patient community, as well as the payer community. We are ready to go. Keith, anything you would add?
Yeah, I guess, Tazeen, the only thing I would add is when you bring the payers into it in our meetings with them. Although, you know, we do not discuss an indication statement because it wouldn't be appropriate to as we go through the discussions with the FDA, what we have done is talked about example policies that could potentially benefit patients that patients that can benefit from Apitegromab can have access. Our goal is to have policies that are put in place that are not aligned with the inclusion, exclusion criteria of our phase III, but more aligned that would give a broader sense of utilization for the community. It's been well received in our discussions to this point. Thanks.
Thank you. Please stand by for our next question. Our next question comes from the line of Marc Frahm with TD Cowen. Your line is open.
Hi. Thanks for taking my questions, and congrats on the update today. Maybe just thinking through the kind of timelines, you know, as you've kind of spoken to a bit earlier on the call and certainly in prior venues. You know, there is some hope that certainly if the inspection goes well, the Catalent Indiana site might be able to support an approval, you know, or a rapid approval on a resubmission and well ahead of the PDUFA. Would the secondary fill-finish be ready from a stability and everything else perspective if that would happen? Or would you possibly, you know, in the kind of very good scenario with Catalent need to kind of pull the secondary facility and ultimately resubmit it?
Kind of vice versa, if things go, you know, maybe not as well as expected in the inspection, you know, do you think you need that absolute full PDUFA window to have that second facility, you know, the product really ready from a stability and all the different follow-up assays that have to happen after these engineering runs? I'll fire a follow-up.
No, thanks, Marc. Look, I think for sure one takeaway from this call is that the timelines have been getting closer together at both facilities, largely due to, you know, how much we've accelerated timelines at our second fill-finish facility, and noting that, you know, we have now resubmitted our BLA here in Q1 inclusive of that second facility and commercial product being released from that second facility several months before an anticipated late September PDUFA. So all of that, you know, bodes well for us. Obviously, when you're releasing product, you know, from that facility, the FDA would have all of the data from that second facility that they would need. I think that Akshay will cover this, you know, once again.
I think what's elegant about today's announcement is that with meaningful progress at both fill-finish facilities, it offers tremendous optionality and flexibility for us. I think it allows us and our partners and of course the agency, which we're very gratified to them, for their ongoing engagement on this, it allows them you know really the opportunity to continue to work in an expedited fashion to meet the needs of the SMA community. Akshay.
Yeah. Marc, I think both sites have made tremendous progress. The second fill-finish facility, you know, the sequence of engineering runs, PPQ runs, the stability program, the progress that's been made towards those aspects and also the work that is yet to be done on some of those, that's all being discussed internally with our second fill-finish facility with the FDA. That body of work is well underway. We feel that within this six-month time frame, you know, one or both sites will be ready. We're very confident with the BLA resubmission that we're enabling this drug as best as we can, and then allow the FDA to do its work now and get us to the finish line by September, hopefully sooner, if at all possible.
Either site could work out.
Then Marc, to your point, right? I mean, if the timelines are a little bit more distant apart. I think Akshay highlighted the, it's very nice to have some optionality to drop one, move forward with an approval, and then shortly thereafter resubmit an sBLA, which was always contemplated in our planning as well. We think that this announcement today is wonderful news more than anything else for the SMA community, but we have a significant amount of optionality moving forward from here.
Okay. That's all. Very helpful. Just if you end up having to go down that path of the secondary facility becoming the primary and kind of sole approval, do these early runs have enough, you know, are you building enough inventory and capacity through these early runs to fully supply the commercial market? Or is there a scenario where if the launch goes well, you know, where if demand is good, that maybe there's a period where you kind of have to meter access to the drug just to build up the kind of supply chain?
Yeah, Marc, that's a great question, and I'm happy that you asked it so we can clearly state that we will have plenty of commercial Apitegromab to launch with several months before a late September PDUFA date, so that Keith and the commercial team would be unencumbered in meeting the needs of the SMA community at approval. We're very grateful to all of the parties, including our second fill-finish facility and our internal team that has been accelerating the timelines on a daily basis, for us to be able to say that. This is not, certainly not us trying to get cute with an approval, but there'd be limited supply. There will be more than enough supply to meet the needs of the marketplace with a commercial Apitegromab from the second facility alone.
It is our intention that both facilities, not a matter of if, but when, they are both approved one way or the other through this current resubmission or one gets dropped, we get approved, and then an sBLA so that all drug would be in the marketplace at some point in time. Certainly we're in a good spot from an inventory perspective, and that's been contemplated in all of our timelines with the second fill-finish facility.
Great. Thank you, and congrats.
Thank you.
Our next question comes from the line of Geoff Meacham with Citigroup. Your line is open.
Hey, guys. This is Jarwei on for Geoff. Want to add our congrats on the progress with the regulatory agencies. Maybe just real quick on the EMA for the mid-2026 expectations for the timeline. I guess, you know, you previously said that the European agency is comfortable with the current timeline as is with progress being made at Catalent. But I guess, if things were to shift to the second fill-finish facility, you know, whether it's just delays at FDA with Catalent or maybe CRL again, could you maybe expand on how that might affect the current expectations for mid-2026? Thanks.
Yeah. I think it's an important question, and it's one that we and under Akshay's leadership and the team and EMA, this has obviously been very public, since our disappointing news, you know, late last year. One that has been out in the open and discussed as we've all continued to be very pleased with the progress we're making with our MAA, and the ability to eventually serve children and adults living with SMA in Europe as well as the U.S. Akshay can comment on your question specifically, should we rely on the second fill-finish facility?
Yeah. Good question, and the first thing I want to say is that we have to remember the tremendous progress made by Novo at the Catalent facility. You know, we're looking forward to hopefully a successful reinspection in the coming period, and that will enable all parties to help us get to the finish line, FDA, EMA, and everybody. That's the first thing to say. I think the second thing is, as we've emphasized, about the guidance we've had from the FDA, we've also had very good guidance all along and understanding with the CHMP of the EMA during this review process. I think should it come to, you know, a necessity to switch to the second fill-finish facility, I'm sure we'll be able to come to an understanding.
We'll cross that bridge when we come to it. You know, we'll obviously keep everybody informed. So far, excellent meeting of minds on all sides. Looking forward to getting this Catalent facility through their inspection with Novo's help, obviously, and the FDA review, and then we'll go from there.
Thanks, Akshay. I think the questions have been very helpful today because they do illuminate that this resubmission with both fill-finish facilities is evident of meaningful progress that the FDA has seen at both fill-finish facilities, including the progress that Novo has made at the Indiana site.
Thank you.
Thanks, guys.
Please stand by for our next question. Our next question comes from the line of Kripa Devarakonda with Truist Securities. Your line is open.
Guys, thank you so much for taking my question and let me also add my congratulations on the regulatory progress. A couple of questions for me. One, with better visibility into approval timelines, do you expect any incremental cash burn between now and approval? Second one, you know, a competitor made the decision to discontinue their myostatin program in SMA. Akshay, I would love to get your thoughts on what this means for the anti-myostatin, especially in these rare neuromuscular diseases. Keith, maybe you can comment on does this change your pricing strategy in any way because there's lesser competition now? Thank you.
Yeah, Kripa, I'll start, then Vikas on the balance sheet, and then Keith can kind of come back to any implications with really the final potential competitor discontinuing their SMA program and announcing that just in the last couple of weeks and any implications for us. You know, I would just note that, you know, for a long time here, in the coming few months I'll be starting my 10th year at the company, we have just been really excited about the unique way that we're able to inhibit difficult to inhibit proteins or growth factors in the body. We've chosen, you know, myostatin as our first meaningful target to inhibit.
As you know, there's been 20, 30 years of failure in the pharmaceutical and biotech space to try to, you know, drug effectively this very elusive target. We think over these last couple of years, it's really borne out that we are the world leaders in myostatin biology with Apitegromab, with subcutaneous Apitegromab, and with SRK-439. We feel like we have quite a unique sort of capability here. Our aim is to not only transform the lives of children and adults living with SMA, but Akshay has very meaningful plans across a range of rare, severe, and debilitating neuromuscular disorders that we look forward to keeping you updated on. Related to the cash burn, Vikas, with some visibility, I'll have you comment, and then Keith, you can talk about pricing.
Hey, Kripa. Your question was, would we accelerate some expenses? You know, most of the expenses that are already in the numbers are headcount related and that our team is already fully ready to launch as quickly as possible. The launch costs are right now gated for us to go forward. We'll look at defining the timing of some of them, but it's not gonna be really material from where we are. We have a good cash balance, and we have, you know, the PRV is gonna come on the approval, so and $150 million more that we could get at the time of approval from our lenders. It gives us a very, very good position right now. So small amount might move up and down, but nothing material to worry about.
Yeah. Kripa. Then in regard to pricing and what this means for us, you know, as we've shared before, we've been doing a lot of work on pricing, not only for U.S., but also outside of U.S. and taking several things into consideration. The bottom line is our meetings that we've had with payers and actually with governments, it is clearly known that there still exists a substantial unmet medical need in SMA. You know, as David referenced before, you know, the majority of these patients will plateau and then begin to regress. And ultimately, our data being the first and only myostatin to have clinically significant and statistically significant results in a registrational trial, you know, allow us to turn a loss of motor function into a gain of motor function.
That is not being lost on the payers that we are speaking to and on the governments. I guess the last thing that I would mention is just we just believe that the total impact to any single payer or government is probably not very large because, you know, we are looking at a rare disease as a whole. You know, we will continue to do this. Trust me, I really look forward to the launch call, and we'll get really specific on pricing.
Then finally, Kripa, I would just say on behalf of everyone here at Scholar Rock, we do not take this responsibility lightly that we are the only company that has successfully developed a therapy that targets myostatin safely and effectively through phase III.
That we are really poised for many years to come to be the first and only company to launch a muscle-targeted therapy to patients in need. This responsibility sits with us every single day to do it in a very focused to be good stewards of a community that we ourselves look up to every day for strength and ambition and optimism. We look forward to holding that mantle with a great deal of pride.
Thank you so much, and congrats again, guys.
Thank you.
Our next question comes from the line of Etzer Daro ut with Barclays. Your line is open.
Hi, this is Luke on for Etzer. Thanks for taking my call. So with the timeline shared, did FDA give any inclination or do you have any idea as to which facility may reach that initial first? How does selection of a facility like in this stage in this way affect your commercialization plans in the future? Do you kinda have like a favorite child in this fight? You know, if it ends up being that Catalent isn't able to, you know, if they get CRL'd again. I know you mentioned to a previous question that you have enough for the commercial launch from the second fill-finish, but will you need to, you know, acquire more capacity from them to maintain a further launch?
Yeah, thank you. I'll address the question. I think as we noted today, we were gratified to be in alignment with the FDA to resubmit our BLA with both Catalent Indiana and our second fill-finish facility. We were able to do that based upon meaningful progress that's taken place at both sites. Novo Nordisk has been working diligently on their remediation plan and their readiness for reinspection in the coming period. Certainly we think that progress was illuminated earlier in this quarter with a Novo Nordisk FDA meeting as well as a site visit.
At the same time, our team at Scholar Rock has been working diligently with a second fill-finish facility that we chose very carefully that had lines that were validated for our vial configuration, that had a clean inspection history with U.S. and international regulators, that had dozens of commercially approved products at that facility. We just thought they were a great match for us in terms of the urgency to meet the needs of the SMA community. We're also very gratified for that second facility's work to help us accelerate the plans to be in a position here in Q1 to be able to resubmit our BLA with both fill-finish facilities.
I think as Akshay and I noted repeatedly in this call, there's a lot of elegance to having the flexibility and optionality to have these two fill-finish facilities in this BLA resubmission. With alignment of the FDA, there's two independent paths to an approval here for Apitegromab, which will be the world's first and only muscle-targeted treatment for children and adults living with SMA. We're in a good spot with multiple paths to approval and plenty of drug. Either way we get there, we would anticipate at some point in time, you know, both facilities will be approved as part of our file, and we will have wonderful redundancy in our supply chain to support not only the U.S. market but our high ambitions to reach patients in 50 countries around the world.
Thank you for your question.
Thanks.
As a reminder, ladies, and gentlemen, that's star one one to ask the question. Our next question comes from the line of Evan Seigerman with BMO Capital Markets. Your line is open.
Hi there, this is Conor MacKay on for Evan. Thanks for taking our question. Maybe just two quick follow-ups, if we may. The first one, what are the remaining gating items, if any, that still need to be completed at the Catalent Indiana site before approval can occur? Or is this really just a matter of waiting for a reinspection at this point? And then maybe on commercial launch readiness as well. We appreciate some of your earlier comments on inventory, but we're wondering if maybe you could share anything else on how you're thinking about building physician awareness or other pre-promotional activities. Thank you.
Great question. I'll quickly address the first one and then Keith on the second. But I think Akshay laid it out, you know, nicely. We expect an unannounced reinspection in the coming period. The FDA did note on their recent site visit to the Indiana facility that they would be coming back for a reinspection following routine manufacturing activities, which just resumed at the end of last month at that site. So we're expecting an unannounced reinspection in the coming period. We would note that, you know, about three-quarters, by FDA's own dashboard on their website, of re-inspections have some observations associated with those. So we would expect that to happen. That doesn't necessarily preclude the facility from being cleared and reclassified.
That would happen at the last day or the closeout meeting of a reinspection at the Indiana site. Novo would then have 15 days to respond to the Form 483 observations that may or may not be provided. Sometime after that, the FDA would make a decision on clearing and reclassifying the facility. Those are the steps that we would expect at this point. As we've said, quite a bit of progress has been made by Novo and the FDA in Q1. It was a good month from that perspective. Again, in any event, we're very excited to have an established timeline here of up to a six-month review that we would anticipate and hopefully could be faster.
With that, I'll turn it over to Keith because he and his team, while making the most of the extra time that we have had, they are ready to launch now. Keith?
Yeah. Thanks, David. Conor, all I can tell you is that, you know, for all the members of the team, they're seeing this press release this morning, and they're texting in because they're so excited to finally be on the clock. They are ready to launch. We continue our disease state education plans that we have been executing on and building relationships with not just the prescribing physicians, but the entire SMA treatment teams. We've grown our specialty pharmacy network since the September 22nd PDUFA date. We've grown that so that we will be able to serve all patients regardless of what SMA-targeted therapy they're on from the same
Specialty pharmacy that they currently use. We've built out a 10,000-nurse home infusion network. Continued work with our payers. We've been able to expand this beyond national to regional and even Medicare and Medicaid. We've really been testing and retesting our Scholar Rock Supports program so that we can take care of these patients when we finally have approval, and we're able to enroll them in our program and help transition them from a prescription to a patient on treatment. Trust me when I tell you that the team is ready. We will continue to drive hard, but we'll be ready for launch. Thanks.
Yeah, thank you and congrats again.
Thank you. Our next question comes from the line of Kalpit Patel with Wolfe Research. Your line is open.
Hey, Gugan on for Kalpit. Thanks for taking the question, and congrats on the resubmission. In the event of a successful re-inspection, do you anticipate a quicker turnaround than the six-month timeline? And any historical precedent that you could refer investors to in a similar situation for a re-resubmission? Thank you.
Yeah. The resubmission is in. It's been submitted to the FDA. They've validated that we've resubmitted it, and as Akshay noted, we would expect in, you know, upwards to the next 30 days, they would likely apply a Class 2 to this. We wanted to be careful to say up to six months because there are precedents where, you know, once a re-inspection is done and a facility gets reclassified, it can be earlier than that. We certainly want the FDA to do their work and take the time that they need to do their work. That's important. We are continue to be really gratified with the FDA's high level of engagement since our highly collaborative in-person Type A meeting last quarter that Akshay presided over.
We would just be hopeful that in the future, if there is an option to be able to approve this in that six-month window ahead of an action date, that could happen. Of course, we'll leave that up to the agency, and we're just very grateful that the agency continues to be highly engaged and focused on the needs of the SMA community.
Thank you. Our next question comes from the line of Andres Maldonado with H.C. Wainwright. Your line is open.
Thanks for taking my question and sneaking me in here. Just a quick question from the commentary on the payer dynamics here. I guess, how are you thinking about, you know, budget impact discussions where adding Apitegromab may, you know, force trade-offs within rare disease portfolios? I guess the commentary has been you've had productive conversations with payers, but, you know, how should we be thinking about the scenario of how this could potentially slow, I guess, the uptake despite, you know, the strong clinical rationale? Thank you very much.
Yeah, it's a great question. I think Keith and I for a long time have said, like, you know, we expect demand from the physician and patient community to be quite high. We do know that there are a lot of precedents where early on, for a number of reasons, there can be payer headwinds. Again, not a matter of if they'll pay, but when they'll pay. We would see increasingly higher conversion rates that happen faster over time. That we would note a number of different factors, things like miscellaneous J-codes, the fact that this is an every-four-week infusion, the fact that the label may be beyond that of the phase III clinical trial criteria, the establishment of medical policies, and things of that nature.
I think the most important thing you're asking is this is the first-of-its-kind therapy that targets the muscle. For 10 years, we have seen a lot of innovation around motor neuron survival, and I think we in the SMA community are certainly grateful to the three therapies that are out there that can focus on motor neuron survival. As we noted in today's call, the organ that is the principal organ that is clinically affected by this disease is the muscle. For a long time, this has been noted as a very high need of the SMA community. I think what you're saying is, you know, will the payers, you know, be supportive of that. I think at a high level, what Keith and the team is seeing is the payers get it.
They've seen the long-term data of patients that are on an SMN-targeted therapy alone. They see that over time, those patients plateau and revert to the progressing form of SMA, and that in our clinical trial, what we were able to show is those patients move from a loss of motor function to a gain of motor function. We think that that sets up for a very nice foundation of partnership between Scholar Rock, the SMA community, and our payers over time. We're certainly looking forward to that moment in time, Keith, when your team is launching.
Yeah, absolutely. I mean, your point is well taken. It is going to take some time, and that's why we lead to, you know, consistent and steady growth over time because, you know, ultimately with these payers, even with SMN-targeted therapies today, 50% of them are first rejected and have to go through an appeal process. Ultimately, about 87% of the time they're prescribed, that prescription is in fact filled, and we expect that we'll get to a very similar spot with Apitegromab post-launch. Maybe not the first month after launch, but after we get some of those headwinds out of the way that David just referred to. Ultimately, the unmet medical need and the benefit that Apitegromab provides, we believe with the SMA community will win out. Thanks.
Thank you. Ladies, and gentlemen, I'm showing no further questions in the queue. That concludes today's conference call. Thank you for your participation. You may now disconnect.