Scholar Rock Holding Corporation (SRRK) Q2 2026 Earnings Report, Transcript and Summary

Good morning, ladies and gentlemen, and welcome to Scholar Rock's Second Quarter 2026 Conference Call. [Operator Instructions] This call is being recorded on Thursday, August 6, 2026. 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, Board Chair 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 will provide introductory remarks and a business update. Akshay will review our R&D progress. Keith will provide an update on our commercial readiness activities and Vikas will provide a financial update. We will then open the call for questions. 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 safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Any forward-looking statements represent our views only as of 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 good morning. Thanks to everyone for joining our second quarter earnings call. Today, Scholar Rock is operating from a position of strength as we enter a defining period for our company and for the SMA community. Across the business, we are executing with focus, discipline and urgency as we drive towards the U.S. launch of apitegromab this quarter, advance our apitegromab MAA in Europe to approval and prepare for launch in Germany, build momentum across our world-leading anti-myostatin platform and maintain the financial strength to support our ambitions. Most importantly, we are on the threshold of bringing the world's first muscle-targeted therapy to children and adults living with SMA as we advance apitegromab through the final stages of the FDA regulatory process. Let me now provide additional detail on the ongoing review of our apitegromab BLA in the U.S. As a reminder, the sole approvability issue for apitegromab noted in the complete response letter that we received on our priority review PDUFA date last September was related to observations identified at a routine general site inspection of the Catalent Indiana fill/finish facility owned and operated by Novo Nordisk. Since our constructive and collaborative Type A meeting in November, the cadence of activities has reflected the shared understanding between us and the agency of the high unmet need in the SMA community and a shared sense of urgency to bring apitegromab to children and adults with SMA as rapidly as possible. We are grateful for the agency's sustained level of engagement and for our ongoing dialogue, including our March 3 Type C meeting, where we discussed the accelerated progress that we had made at our second fill/finish facility and the agreed-upon data package to facilitate the FDA review of the second fill/finish facility. In alignment with FDA guidance from this discussion, we submitted the apitegromab BLA on March 30 with 2 fill/finish facilities, both Catalent Indiana and our second fill/finish facility. Our BLA was accepted in April with a PDUFA date of September 30. Agency review of our application is progressing well. Importantly, we have significant optionality with 2 independent paths to approval, either through Catalent Indiana or through our second fill/finish facility or both, whichever is determined to be the most rapid. As it relates to Catalent Indiana, the FDA inspection classification following the April 2026 general site inspection is pending. We continue to be very pleased by the progress made at our second fill/finish facility. Importantly, the data package for the FDA's review of the second facility has been submitted and the review is progressing, underscoring our operational excellence at our second site. We now have more vials available from this facility than we do from Catalent Indiana. Notably, vials from both Catalent Indiana and our second facility are now on site at our third-party provider awaiting packaging and labeling upon approval. As we near the final step in the U.S. regulatory process, we are well aware that patients are awaiting the world's first muscle-targeted therapy for this devastating disease. Turning now to our commercial readiness in the U.S. Our team continues to advance our launch preparations across all key functions, ensuring we are prepared to support patients, caregivers and prescribers from day 1. Our team is ready to launch apitegromab at any time prior to and including our September 30 PDUFA date. Keith will discuss our commercial preparations in greater detail shortly. In addition to the U.S., we continue to look forward to serving children and adults living with SMA in Europe. I would now like to provide an update on where we stand in the European regulatory process. The apitegromab MAA includes only Catalent Indiana fill/finish facility and the EMA is awaiting the FDA inspection classification for that facility. In parallel, we are engaging with European regulators with regards to potential inclusion of our second fill/finish facility in the apitegromab application. Importantly, this facility has had recent successful site inspections by the FDA and the EMA. We are grateful for the EMA's continued level of engagement, and we look forward to providing updated guidance on the potential timing of a CHMP opinion upon alignment with the European regulators. Turning to our launch preparations in Europe. We are executing our commercial playbook, building momentum with launch readiness activities and engaging with the SMA community. We are planning for an initial launch in Germany with additional countries and regions to follow as we build out our planned 50-country operating platform. We know it is not a matter of if but when apitegromab will be approved for children and adults with SMA in both the U.S. and Europe, and we continue to work with urgency to reach the 35,000 people with SMA around the world who have received an SMN-targeted therapy. Turning now to our world-leading anti-myostatin pipeline. We continue to make meaningful progress with our key clinical programs. We have robust enrollment in our Phase II OPAL study evaluating apitegromab in infants and toddlers with SMA. We initiated our randomized Phase II FORGE study in patients with FSHD. We are ready to engage with U.S. and European regulators on the development path of our high concentration subcutaneous formulation of apitegromab, which we will do once we have regulatory approvals. And enrollment and dosing is proceeding very well in our Phase I healthy volunteer study for SRK-439, our novel high-potency anti-myostatin antibody. Akshay will discuss these programs in greater detail shortly. Turning now to the balance sheet. We were very pleased to have ended the second quarter of 2026 with $492 million in cash, cash equivalents and marketable securities. This cash balance includes net proceeds of $63 million from our ATM program during the second quarter. Vikas will provide more detail later in the call. In June, we had the opportunity to be with the SMA physician and patient community at the Cure SMA Annual Meeting in Orlando. I was able to sit down with several SMA treating physicians and with a number of patients and their families. And during our time with them, we've heard some very moving stories about the impact apitegromab has had on children and adults who are participating in our ONYX and EAP programs. Our team at Scholar Rock is so inspired by these patients and by their families, and we look forward to ushering in the next phase of innovation for this community. With that, I'll now turn the call over to Akshay for a closer look at our R&D initiatives. Akshay?

Thank you, David, and good morning, everybody. As David shared, we are very pleased that the apitegromab BLA continues to progress through FDA review with 2 independent paths to approval, and we remain on track for a decision by the September 30 PDUFA date. The FDA inspection classification for Catalent Indiana is pending. We had anticipated the classification in late July within 90 days following inspection completion based on the agency's guidelines. We remain engaged with the FDA, and we'll provide updates as appropriate. As it relates to the second fill/finish facility, we're very pleased to report that all necessary data have now been submitted to FDA and the agency's review of those data is progressing well. We're gratified by the agency's continued support since the CRL last September from the constructive and collaborative in-person Type A meeting in November to the early March Type C meeting. Throughout, the agency has appreciated the high unmet need in the SMA community, and we look forward to the final steps in the process. Turning now to Europe. We're pleased with the EMA's review of the apitegromab marketing authorization application and with their continued level of engagement. As we have previously noted, approval in Europe is dependent on FDA clearance of the Catalent Indiana facility, which is currently the sole fill/finish site included in our MAA. The EMA continues to await the FDA's inspection classification for this facility. Additionally, we're engaging with the EMA regarding the process to include our second fill/finish facility in our apitegromab application. Importantly, this facility is in good standing with European regulators. We will provide an update on timing upon alignment with the EMA. Turning to our pipeline. Let me start with the Phase II OPAL trial. We continue to have robust enrollment in the study, which is evaluating apitegromab in infants and toddlers with SMA under the age of 2. As a reminder, this trial is enrolling participants who have been treated with an SMN1-targeted gene therapy or who are receiving ongoing treatment with an SMN2-targeted treatment. This study is important because it is anticipated to expand the impact of apitegromab to the full spectrum of patients, including those treated with Zolgensma. Notably, the rate at which the study is enrolling underscores the significant unmet need and the potential for apitegromab in the youngest of SME patients. Turning now to our next indication for apitegromab, facioscapulohumeral muscular dystrophy or FSHD. FSHD is a rare devastating neuromuscular disease. It is one of the most prevalent inherited muscular dystrophies, and there are no approved therapies to date. We've prioritized FSHD as the next indication for apitegromab for 3 key reasons: first, the significant unmet need since approximately 20% of patients become wheelchair dependent. Second, the compelling preclinical data from the gold standard FLExDUX4 mouse model that provides mechanistic rationale for apitegromab in FSHD. And finally, data from randomized studies in FSHD, which suggests muscle mass can increase and has the capacity to show functional benefit. For example, in studies of either rigorous physical therapy or treatment with anabolic agents, patients with FSHD demonstrated increases in lean mass and muscle function. These data suggest that apitegromab as a monotherapy may have the potential to bring important benefit to FSHD patients. We're very pleased to announce today that we've initiated our Phase II study called FORGE, which is a randomized, double-blind, placebo-controlled trial with a sample size of 60 patients. We're also advancing 2 additional therapeutic programs in our world-leading anti-myostatin pipeline, a high concentration subcutaneous formulation of apitegromab and SRK-439. In our subcutaneous apitegromab program, we showed some very exciting data in January from the Phase I study, which demonstrated that subcutaneous apitegromab appears to have favorable bioavailability and the pharmacodynamic profile comparable to IV administration. Additional development activities are ongoing, and we continue to plan for engagement with U.S. and European regulators later this year following approval of apitegromab. Turning now to SRK-439, our high potency, high-affinity subcutaneously administered myostatin inhibitor. We're very excited about this program and dosing in our Phase I healthy volunteer study is progressing well. We expect to have top-line data from the study later this year. In closing, we're executing with urgency to bring apitegromab to children and adults with SMA, whilst in parallel working to maximize our impact for patients with our world-leading anti-myostatin pipeline across a range of rare, devastating neuromuscular diseases. I'll now turn the call over to Keith to discuss our commercial launch preparations. Keith?

Thanks, Akshay, and good morning, everyone. As David noted, with the potential FDA approval of apitegromab for children and adults with SMA by September 30, our U.S. commercial organization is launch-ready across all key functions, and we are prepared to support patients, caregivers and prescribers from day 1. Given the significant unmet need in SMA, we have moved with urgency to build our commercial operations to ensure that patients who can benefit from apitegromab will have broad and reliable access to apitegromab. In the U.S., despite approximately 78% of children and adults living with SMA receiving an SMN-targeted therapy, 95% of patients continue to experience persistent and progressive muscle atrophy that limits both function and independence. As further evidence of the unmet medical need, data shared with us by Cure SMA show that an estimated 1/3 of people living with SMA in the U.S. have received 2 or more SMN-targeted treatments, either sequentially or in combination. This data again underscores the significant opportunity we have with apitegromab, the world's first muscle-targeted therapy for children and adults with SMA. Since our last earnings call, our U.S. field team continues to broaden their reach, focusing on disease education and awareness around the unmet medical need while also reinforcing a broader understanding of SMA as a disease that consists of both the motor neuron and the muscle, the principal organ impacted by the disease. We are also expanding our reach and frequency across approximately 140 SMA treatment centers, 2,600 prescribing physicians and their multidisciplinary care teams. Through these engagements, our field team is establishing case flows on a center-by-center basis to ensure that upon approval, we are well positioned to support the SMA treatment centers once apitegromab treatment decision has been made. This past quarter, we have also strengthened our Scholar Rock Supports patient services program. The Scholar Rock Supports team is fully trained and prepared to provide comprehensive individualized support to patients and caregivers at launch. Eligible patients and their families will be able to access this program to understand insurance coverage, identify available financial and co-pay assistance and navigate treatment logistics. Turning now to patient engagement. Our connections with the SMA community remains strong. This past June, we had a significant presence at the Cure SMA Annual Meeting in Orlando. Scholar Rock served as a presenting sponsor of the meeting and throughout the week, our teams engaged with health care professionals and members of the SMA patient community. I was very pleased that the Scholar Rock Symposium for health care professionals entitled Expert Perspective on the Evolving Management of Spinal Muscular Atrophy was one of the most attended expert sessions during the meeting. Equally, our patient symposium, Muscle, there's more to the story in SMA was attended by hundreds of SMA patients, caregivers and families. And during this session, we sought their perspective on needs and priorities for people living with SMA. Every interaction we had during this meeting reinforces our determination and further strengthens our commitment to serve patients and families. Turning to U.S. reimbursement. Our market access team continues to advance discussions with national and key regional payers as well as Medicare and Medicaid with the goal of achieving broad reimbursement for eligible patients after approval. Given the significant scope of our efforts and progress we've made in the past several months, we are ready and well positioned to successfully support apitegromab in the U.S. immediately upon FDA approval. Turning now to Europe. We are advancing our launch preparations with a particular focus on Germany as we work with the EMA on the next steps for our application. Our team in Germany is using this additional time to execute the same launch readiness playbook that we have successfully deployed in the U.S. over the last several months. This includes broadening and deepening of relationships with key SMA treatment centers and potential prescribers. In parallel, we are engaging with SMA advocates across Europe, participating in educational programs at various congresses and symposia hosted by patient advocacy organizations. As it relates to reimbursement and patient access, following European Commission approval of apitegromab, we will be prepared to rapidly advance reimbursement submissions in Germany and other key markets. In addition, we are advancing our distributor relationships to extend the commercial reach of apitegromab across multiple additional countries. In closing, we are fully prepared for a successful U.S. launch immediately upon FDA approval, while advancing our launch preparations in Europe and working to establish our 50-country operating platform with the ambition of reaching the estimated 35,000 patients living with SMA worldwide who have received an SMN-targeted therapy. We are ready to usher in the next phase of innovation for children and adults with SMA, one patient, caregiver and family at a time. With that, I'll turn the call over to Vikas for a review of our financial performance. Vikas?

Thank you, Keith. As we have shared previously, our financial objectives for 2026 remain focused on supporting our commercial build to deliver a strong apitegromab launch, funding R&D activities to advance our pipeline and expand our leadership in the myostatin and muscle space and continuing to evaluate opportunities to strengthen our balance sheet in a way that supports long-term shareholder value. Consistent with these priorities, I'd like to briefly review our second quarter financial results. For the second quarter, we reported $108.9 million in operating expenses, which included $19.7 million in noncash stock-based compensation. Excluding stock-based compensation, operating expenses were $89.2 million. As we continue preparing for the anticipated launch of apitegromab, we have strategically increased our commercial investments while keeping our overall operating expenses at the levels generally consistent with the second quarter of 2025. This disciplined approach to capital allocation has enabled us to advance launch readiness while continuing to invest in our key R&D programs and strengthening our global supply chain. Turning to our balance sheet. We ended the second quarter with $492 million in cash, cash equivalents and marketable securities. During the quarter, we further strengthened our cash position with $63 million in net proceeds from our ATM program. Looking ahead, our FDA approval of apitegromab, we will have an option to draw down an additional $150 million from our existing debt facility, and we plan to monetize a priority review voucher to further strengthen our balance sheet. We continue to operate with a disciplined financial plan and our investment priorities remain focused on our apitegromab commercial launch readiness in the U.S. and Europe, strengthening our supply chain to support our expanding pipeline and anticipated global commercial demand for apitegromab over time and advancing our highly innovative clinical programs that Akshay discussed earlier in the call. With that, I'll turn the call back to David. David?

Thanks, Vikas. As we look ahead, Scholar Rock is entering one of the most important chapters in our history from a position of strength. With 55 days until our PDUFA date, we have the team, the financial foundation and the operational readiness to execute with confidence. Across the organization, our teams are prepared, energized and focused on what comes next, bringing forward the world's first muscle-targeted therapy for children and adults with SMA. We are moving into these final 55 days with urgency, discipline and a deep sense of responsibility to the SMA community. We know what is at stake. We know what is possible, and we are ready to deliver. As we close, we are mindful that August is SMA Awareness Month. This is an important opportunity to recognize the strength and resilience of the individuals living with SMA, their families and the advocacy organizations that work tirelessly on their behalf. We are grateful to the patients, caregivers, health care professionals and advocates whose partnership continues to advance awareness, earlier diagnosis and access to care. This month reinforces our commitment to the SMA community and our focus on delivering meaningful innovation that can make a lasting difference for people living with this rare and devastating disease. We look forward to updating you on our continued progress. And with that, we'll now open the line for questions. Operator?

[Operator Instructions] The first question will be coming from the line of Eric Schmidt of Cantor.

Appreciate all the updates. My question is on apitegromab's second fill/finish provider. How confident are you that this facility alone independent of Catalent can support approval by the PDUFA? And can you share any kind of anecdotes from your FDA interactions to support that they're making progress in the review of the package you submitted?

Thanks very much, Eric. Yes, as we noted, really dating back to that March 3 Type C meeting, we were really gratified as we were updating the FDA on the rapid and meaningful progress we were making in our second facility that really together, we agreed that resubmitting our BLA with 2 fill/finish facilities and actually having a plan to enable an apitegromab approval with the fastest path, whether or not at the Catalent Indiana, the second fill-finish facility or both, we were gratified that there was a complete alignment between us and the agency that that was the best approach. As we've noted on the call, the team here at Scholar Rock and the second facility, we're really proud of their efforts to now have more vials of apitegromab ready for launch from this facility than we do from Catalent Indiana. And frankly, than we did from Catalent Indiana at the time of our last PDUFA date, I think, underscores how well we are working together. The review, I think, as both Akshay and I noted, is progressing well. The FDA has all of the data that we agreed upon during our Type C meeting that was going to enable the review and eventual approval from that second facility. So we're pleased with that. And as we're noting, we're corresponding with the FDA on that. I would also note a couple of other facts that I think are important here. That second facility has had successful recent site inspections by FDA and EMA. None of the approvals from this facility in the last 12 months have required a PAI or a PLI. And I think underscoring the performance of that facility during that time, the site underwent multiple routine GMP general site inspections by EMA and FDA. So we feel really good about the position that we are in. And then I might close by saying we contemplated the, well, what if we remove one of the facilities from the application? Could it impact our time line at all? And that was contemplated and discussed between us and the agency. And based upon our discussions with the agency dating back to those March discussions, we do not expect that removing a facility from the application would have any impact on our ongoing review time line at all. So Keith and team are ready to launch at any time between now and up to our September 30 PDUFA date. I'm certainly gratified by Scholar Rock's technical operations and quality team. They have stood up this second fill/finish facility faster than almost any example we can find in the industry. And they now have more apitegromab vials that will be commercially available from this second facility, and they are at our labeling and packaging site awaiting approval. We're super excited. So we'll continue to keep you all updated over these last 55 days, but we feel like we're in a really good position.

And the next question is coming from the line of Kripa Devarakonda of Truist Securities.

Congratulations on all the progress. I have a follow-up question on Eric's question regarding the second site. David, you just mentioned that you can drop one of the sites at any time and it won't delay. But I was wondering if Catalent remains classified as OAI, is there a deadline or a date for administratively withdrawing that site, so it doesn't trigger any delay for your PDUFA?

It's a great question, Kripa. We are in correspondence with the agency. They are -- we're both well aware that the inspection classification is still pending from the April reinspection. As Akshay noted, it's now drifted a bit beyond the 90-day guidance period, but it is only a guidance period from the FDA that they would classify an inspection. And look, we're very direct with one another about when we would reach that step, should we need to reach that step in your example that you provided, let's just say the inspection classification reveals that there's no change to the current OAI classification. We've discussed with the agency, it would be a relatively simple step to notify them that on their signal that we are removing that site and the review would progress with that second facility. So -- and again, with our alignment with the FDA, we would expect no impact to the ongoing time line. So that's where we are. I think Akshay and I are both heartened by the fact that we have ongoing open correspondence with the FDA and the review is progressing well.

Our next question is coming from the line of Tessa Romero of JPMorgan.

So just to double-click here on some of these earlier comments, what are the specific items procedurally from now to September 30 that still need to be kicked off to allow for an approval? I think Akshay used the words final steps. And just to set the record straight, is there any reason to believe that the FDA will not be able to complete this review by September 30?

Yes. I'll just hand it over to Akshay, but just to underscore that last question. There really is no reason to believe that the work that's been done at the second facility and where we believe the FDA is in terms of reviewing the application, which, as you recall, right, our resubmitted BLA really only included the new information from the second facility and the updated safety database. There's no reason to believe the FDA can't get their work done in these next 55 days. Akshay?

Yes. I would just reconfirm that. So starting last November when we had a face-to-face meeting with the FDA with all relevant parties and in March, the FDA guided that Catalent is the lead site in the MAA, but the second site would be welcome because it gives them greater ability to help us get this drug approved in a compliant manner to patients in a high unmet need setting. at the March juncture, we said we're ready to submit a second fill/finish site. They welcome that. We talked through the process of how to get to submission and then the finish line with the PDUFA date, and we are exactly on track with all of that. Their review of the second fill/finish site is progressing well. And as David said, we see no reason why we can't get to the PDUFA date with this drug approved before or at that time. And so it certainly feels on track. And we're very grateful to the agency's guidance and the expeditious manner in which they've been working with us and the true engagement and partnership.

Our next question is coming from the line of Michael Yee of UBS.

This is Madeline on for Michael. Congrats on all the progress and thank you for the updates today. We were just wondering, do you have any color or any feedback from the FDA on that later-than-expected reclassification of the Catalent site, given the decision was sort of expected by the end of July, if you just have any feedback you could pass along.

Yes. The only thing that we would note is, obviously, the inspection report is public as on the 483 observations in that inspection report. We are well aware that Novo Nordisk provided a pretty robust response as they had the option to do within 15 days of that inspection. So there was a lot there for the FDA to review. And I would just note that that guidance period is a guidance period. We're aware that sometimes the FDA does take a bit more time than that 90-day period. And as we noted, we're awaiting that. I'm sure our friends at Novo Nordisk are awaiting that inspection classification. While in parallel, the EMA is awaiting that classification, we're also engaging with our European regulators on the inclusion of our second fill/finish facility. So there really is nothing more to it other than the fact that it does happen. The 90 days are not a statutory requirement. The FDA literally has provided that as guidance. There's probably a lot there that the FDA is reviewing. And we await like everybody else the pending inspection classification. But I'd bring it back to this, both in U.S. and Europe, we're continuing to move forward with this meaningful progress that we've made with our second fill/finish facility, really wanted to make sure that we had a belt and suspenders approach to serving children and adults living with SMA and their families. And we feel like we're in a position of strength as we move forward. And we await like everybody else, that inspection classification.

The next question is coming from the line of Cory Kasimov of Evercore.

I guess I'll shift gears a little bit here and I want to ask about your national and regional payer discussions. And curious how they're framing apitegromab in step edit terms. Are you seeing any payers signal that they'll impose time limits or require a rereview of benefit after a shorter initial authorization?

Thanks, Cory. I'm going to hand that over to Keith for his comments. I would just note as a headline as Keith is prepared to answer that we do believe this robust clinical development program that we've been running for 7-plus years. And again, the fact that we met the highest bar, which was the Hammersmith motor function scale in SMA with a stat sig result from our pivotal SAPPHIRE trial, the robustness of that data sets up very, very well. As you know, and I'll just underscore for everybody listening in. I know you know it quite well, Cory, patients were randomized who are on ongoing SMN targeted therapy to receive either placebo or apitegromab. So they were on these ongoing therapies. And again, to hit that highest bar of the Hammersmith Motor Function Scale in SMA at stat sig with 0.019 p-value, we feel with a very low number of patients, we think sets up very, very well for those national and regional payer discussions. Keith?

Yes. Thanks, David. Look, as I stated in the prepared remarks, the team has been working and really extending not only our reach with these various payers, whether it's the commercial payers or government payers, but also the quality of the meetings by bringing in members from our medical team to discuss the robust clinical data package from our apitegromab studies and ultimately with the goal of making apitegromab available for the broadest possible audience of children and adults living with SMA. So as I've stated before, the real goal with these meetings is to be able to create policies as rapid as possible and policies that we believe will align more with the potential label, the FDA label and less with an inclusion/exclusion criteria from our SAPPHIRE study. With all that being said, if you take a look at the data on treatment for SMA, so I'm talking about the 3 SMN-targeted therapies that are available, almost 100% of them have a prior authorization. So I fully expect that you'll see that apitegromab will have a prior authorization even when we have favorable policies that are constructed.

Next question comes from the line of Amy Li of Jefferies.

Congrats on the progress. I just wanted to put a finer point on the second fill/finish facility. You mentioned that you submitted data that the FDA requested the Type C, which sounds encouraging. But could you give us a sense of what was included in that data package? Are stability runs and release testing for this facility fully complete and you would consider the facility launch ready? And do you expect any additional clearance from the FDA?

Thanks, Amy. Yes, I mean what was inclusive and what we agreed upon was obviously the data that we generated from engineering runs, PPQ runs, the FDA has the full package in hand where we know their review of that is progressing well. We're in correspondence with them on that. Everything is straightforward and perfunctory. And there is, I guess -- just to underscore the question, there is no more testing that is required for the product that has been vialed at that facility at all. And again, as noted, that product is at our third-party labeling and packaging facility awaiting approval to support the launch. And of course, in a belt and suspenders approach, we have vials from Catalent Indiana at the packaging facility as well. So we're in a really good position. We're gratified to have reached an agreement with the FDA in March at the Type C meeting on what was required. I would note that what was required was delivered to the FDA in a very timely fashion because you don't only agree on what is submitted, but when it would be submitted within your framework of your PDUFA date, and we felt like we delivered that in a very, very, very timely fashion. And we're looking forward to these next 55 days to get through the final step and eventually launch apitegromab. So thank you very much for your question.

Our next question is coming from the line of Gary Nachman of Canaccord.

So as you've been preparing for a while now with the commercial team, any other initiatives you need to put in place between now and approval? Or is it just really waiting for the final label? And what's the low-hanging fruit to go after with SMA patients where there could be a fair amount of pent-up demand for apitegromab? And how long do you think it will take to get those patients on board?

Yes, I'll start and Keith will get in. But I think the -- as I mentioned during the call, Gary, we spend a lot of time with the community, patients, their families, the advocacy groups, the health care providers. And our ambition is that any patient living with SMA that can benefit from apitegromab should have access to apitegromab. And so we really do look at this holistically across the 35,000 patients globally that have received an SMN targeted therapy, and we believe that we can really offer meaningful benefits to them. So we do think about it very holistically. Keith can share with you some of the dynamics at launch, but I think our general view is it could vary patient by patient, family by family, physician by physician in terms of their thinking about commencing treatment. Keith?

Yes. Thanks, David. I guess, first of all, what else is there to do? The point I want to make really clear is we are ready if we were to get the call tomorrow. The team is kind of chomping at the bit to really get out there and launch this product. With that being said, there's always additional work that we can do. One of the main things that's taking place that I mentioned is really working with the various centers, so that we can be prepared with these treatment centers on a case-by-case basis to work through the process of enrolling patients into our Scholar Rock Supports program. We cannot begin to do any of this until after we have FDA approval. So we are doing a lot of dry run work here, so that we can have a seamless and flawless execution as we take patients from being prescribed the product to ultimately being able to receive the product. Where do you think some pent-up demand is? I've shared before on previous calls. We do have an early access program. Those will be the first patients that we will be focused on, converting from early access product over to commercial product. That being said, we don't have full line of sight into what insurance coverage these early access patients have. So we're going to have to be going through that process and enrolling them in our program. The next will be our open-label extension patients, our ONYX patients. I want to remind you that they will have to go to a closeout visit of that study before they can even convert over to commercial drug, but we will begin to work them through the process of our Scholar Rock Supports program. So when you ask how long will it take, it will take time, mostly because you're going to see a prior auth with all of our patients that are going to be prescribed apitegromab. The majority of them, you are most likely going to receive a denial for various reasons, whether it's a J-code or a policy not created or for some other reason. That will then send us into an appeal process, which can sometimes take some time. So I think that there will be certain patients that will have coverage that will allow them to go on sooner rather than others. But -- what I've typically seen in other launches is during this first 6 months of launch, while we will be without a J-code, the time -- the average time from prescription to a patient actually being able to get infused is greater than 60 days.

Next question is coming from the line of Marc Frahm of TD Cowen.

Maybe just -- I mean it seems like the medical review is kind of done on both sides of the Atlantic. So maybe you can speak to kind of the labeling discussions? And do you expect a largely identical label? Or do you think there are maybe important differences between the U.S. and European label? And then I'll have a follow-up.

Yes. No, it's an important question. As you know, Akshay and I both addressed that we felt like we were in a really good spot at the end of the last review period with the FDA, and we're going to be picking it up or we picked it up in the BLA right where we last left off and then the comparisons between the 2, Akshay can comment.

Yes. I would just say we are very happy and comfortable with the way the dialogue has gone through the medical review. And obviously, it's not for us to comment on the final label, but we are certainly grateful for the engagement and very constructive conversations. So we look forward to getting this drug approved in U.S. and Europe. And I think we'll be comfortable with how to get it to the right patients.

Yes. And Marc, I think due to the great work by Akshay and the entire team, I think at the end of the day, we ask ourselves, are we going to have an opportunity to serve a meaningful number of patients living with SMA in the U.S. and Europe. And again, eventually, our ambition is to reach patients in 50 countries around the world. And I think that Akshay has put us in a really good -- and the entire team, Jing and the entire team has put us in a really good position to be able to do that. But Akshay is right. until we have final USPIs and SMTCs, I think we'll comment on that when it's the right time. And hopefully, that time is coming in the coming days.

Okay. That's helpful. And then just on the CMC side, EMA clearly seems to be essentially deferring to the FDA on the Catalent Indiana facility. Do you expect them to ultimately act kind of similarly with the second facility? Or is there something about the -- either that facility itself or the flexibility that the FDA has kind of granted you in terms of CMC requirements there that might lead the EMA to kind of be more proactive itself in making a decision?

It's a very thoughtful question, Marc. Akshay?

Yes. I mean, in our prepared remarks, we emphasized that the second fill/finish facility is in very good standing with regulators, and we're delighted that that's in progress with the FDA. Now with respect to the EMA, obviously, any approval for apitegromab that involves the second facility will help. And we are right now heavily engaged with the CHMP to work on the progress of the MA to completion and how we incorporate the second fill/finish facility, if necessary.

Next question is coming from the line of Kalpit Patel of Wolfe Research.

One for Europe. If the FDA maintains the OAI classification for Catalent, can you walk us through your potential time line to get the second fill/finish facility into the MAA and your thoughts on the earliest projected time line for European approval?

Yes. No, thank you very much. And as we noted during the call, while EMA and we await the classification decision from the FDA in parallel, we're having the dialogue. The EMA is very well aware of where we are with the second fill/finish facility. Akshay?

Yes. And as that dialogue is ongoing, I don't want to second guess what the final advice will be vis-a-vis necessity or the mechanism by which we incorporate the second fill/finish facility. But suffice it to say that the engagement and flexibility that both FDA and CHMP have shown us is very gratifying to us. They appreciate the unmet need, and they're working with us. So we will be guided by them. That's an ongoing conversation. And hopefully, soon in due course, we will update every.

I think just as a capper, I think what Akshay and I really gratified, just as we've experienced with the FDA, we're just gratified by the receptivity and the dialogue with EMA. But again, we'll await the specific approach pending the inspection classification and of course, in parallel, our discussions on the second facility.

Our next question is coming from the line of Etzer Darout of Barclays.

This is Luke on for Etzer. For FSHD, on the clinical trial side, I know you guys mentioned that you're looking to focus in slightly less severe patients and you're listing the participation reporting is 1.5 to 3 on a Ricci scale on a 0 to 5 scale. I just want to verify that you're using a modified scale there because I think the Ricci scale is 0 to 10. And could you give some color as to what percentage of the FSHD population falls within that scale range?

Yes. We're using the same Ricci scaling system or scoring system that Roche folks are used. And I think I just want to confirm what you said that we're indeed focusing on patients with the Ricci score of 1.5 to 3 because once you get beyond a score of 3, we know from an FSHD database we have access to, and I don't know whether Roche had it or not, but we know that by MRI, many muscle groups, including the quads, which was the primary endpoint, begin to show significant fat infiltration and fibrosis. So focusing on patients with the higher scores, I think, is a tough ask. And so you need some muscle preserve so that the anti-myostatin can act on it to boost muscle mass and hopefully function too. Also, in terms of functionality, you'll note that their inclusion criteria included 10-meter walk between 4 to 12 seconds. So the upper bound 12, whilst we've stipulated that the upper bound or our 10-meter walk is less than 5. So we're certainly in that mild-to-moderate group of patients whilst they were in the moderate to severe. As to the exact numbers of patients, this is the common muscular dystrophy or there's at least a very significant 5-digit number of patients around the world to help. And I think we're comfortable that we will help many, many patients should this drug ultimately be approved in that space. So I'm not concerned about that. And of course, as we know in all these rare diseases, as soon as the therapeutic appears, the rate of diagnosis improves and the rate of access to therapies improves and patients start getting put on therapy earlier and earlier in the course of their disease. So we're looking forward as we announced today to getting momentum going now in the study. The study is initiated, and we're excited to do this study where we have wonderful mouse data and where we think there's a robust rationale with our drug.

Next question is coming from the line of Basma Radwan of Leerink Partners.

Could you please share your perspective on Regeneron recent updates regarding the Eylea high-dose prefilled syringe? Specifically, management indicated that it plans to add a third plant on the regulatory package to enhance the likelihood of approval. How do you interpret that decision given that the situation is very similar to yours with regard to Catalent's involvement? Do you think it suggests that Catalent problems may be still outstanding? That's it for us.

Well, we know -- first of all, I don't -- I can't really comment on Regeneron other than to say I think we have a very different situation because our second fill/finish facility is different than their second fill/finish facility in this case, from our understanding. So that would just be a bright line. The similarity is that we both have Catalent Indiana in our applications. So I really can't comment on their commentary beyond the fact that I would note that our second fill/finish facility is in good standing with the FDA and EMA. They've had several general site inspections by FDA and EMA in 2025 and 2026. Their last 12 months of approvals for products there, and they have like nearly 3 dozen or more commercially available products from that facility. But in the last 12 months, all approvals from that fill/finish facility, the PLI/PAIs have been waived by the agency. So I would just comment that, yes, as we've noted, the Catalent Indiana inspection classification is still pending. So that could be one thing that what you're referring to means. But they're very different situations in that we have a different second fill/finish facility.

And the last question will be coming from Geoff Meacham of Citigroup.

David, in line with your belts and suspenders comment, does the second facility provide a fully independent approval path? Or are there any elements of the filing that are still dependent on Catalent Indiana? And then second question, maybe for Akshay, what regulatory work will be required to get subcutaneous apitegromab into the next sort of pivotal development? I just wanted to maybe go over that.

Akshay, do you want to take?

Yes. Thanks, Goeff. So on the first question, the second fill/finish facility provides a fully independent path and the details of that were discussed in that March meeting we mentioned in the prepared remarks. So we feel good about that. And I think that sort of speaks to David's belts and suspenders comments. So that's great. And vis-a-vis the subcutaneous apitegromab, just to refresh folks in January, we shared data showing the wonderful sort of PK/PD profile of subcu apitegromab, which makes all this feasible. And we have prepared a briefing document that we will be ready to send very soon after the approval of apitegromab, so that we can engage with regulators and begin that next important phase of development to bring a subcu option for patients. Now that involves a dialogue with the FDA because for different drugs, different paths have been adopted. There's one view of the world that says there can be a PK/PD path matching the PK/PD criteria with IV, the level of myostatin suppression and saturation and so forth. And the other extreme is you need to do more substantive development work. You mentioned pivotal. We are now finalizing the briefing doors to present the path forward that we think is reasonable, but we obviously need to engage with regulators to get their guidance. But as soon as we've done that, we will then provide an update in due course as to the path forward because that will ultimately influence the time line of getting subcu patients.

Thank you. And this does conclude today's program. Thank you so much for joining. You may now disconnect.