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Image source: The Motley Fool. Thursday, Aug. 6, 2026 at 8:00 a.m. ET Vice President of Investor Relations - Laura Ekas Board Chair and Chief Executive Officer - David Hallal President of Research and Development - Akshay Vaishnaw Chief Operating Officer - Robert Keith Woods Chief Financial Officer - Vikas Sinha Operator: 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. Laura Ekas: 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? David Hallal: 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? Akshay Vaishnaw: 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? Robert Keith Woods: 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? Vikas Sinha: 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? David Hallal: 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: [Operator Instructions] The first question will be coming from the line of Eric Schmidt of Cantor. Eric Schmidt: 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? David Hallal: 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 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. Operator: And the next question is coming from the line of Kripa Devarakonda of Truist Securities. Srikripa Devarakonda: 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? David Hallal: 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. Operator: Our next question is coming from the line of Tessa Romero of JPMorgan. Tessa Romero: 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? David Hallal: 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? Akshay Vaishnaw: 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. Operator: Our next question is coming from the line of Michael Yee of UBS. Unknown Analyst: 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. David Hallal: 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 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. Operator: The next question is coming from the line of Cory Kasimov of Evercore. Cory Kasimov: 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? David Hallal: 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? Robert Keith Woods: 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. Operator: Next question comes from the line of Amy Li of Jefferies. Amy Li: 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? David Hallal: 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. Operator: Our next question is coming from the line of Gary Nachman of Canaccord. Gary Nachman: 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? David Hallal: 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? Robert Keith Woods: 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. Operator: Next question is coming from the line of Marc Frahm of TD Cowen. Marc Frahm: 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. David Hallal: 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. Akshay Vaishnaw: 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. David Hallal: 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. Marc Frahm: 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? David Hallal: It's a very thoughtful question, Marc. Akshay? Akshay Vaishnaw: 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'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. Operator: Next question is coming from the line of Kalpit Patel of Wolfe Research. Kalpit Patel: 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? David Hallal: 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? Akshay Vaishnaw: 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. David Hallal: 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. Operator: Our next question is coming from the line of Etzer Darout of Barclays. Unknown Analyst: 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? Akshay Vaishnaw: 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. Operator: Next question is coming from the line of Basma Radwan of Leerink Partners. Basma Radwan Ibrahim: 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. David Hallal: 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. Operator: And the last question will be coming from Geoff Meacham of Citigroup. Geoffrey Meacham: 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. David Hallal: Akshay, do you want to take? Akshay Vaishnaw: 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. Operator: Thank you. And this does conclude today's program. Thank you so much for joining. You may now disconnect. Before you buy stock in Scholar Rock, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the 10 best stocks for investors to buy now… and Scholar Rock wasn’t one of them. The 10 stocks that made the cut are built for long-term growth and could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you’d have $400,209!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you’d have $1,375,393!* That performance is why people listen. With a track record of beating the S&P 500 by 4x, Stock Advisor offers a distinct advantage. Don't miss the latest top 10 list, available with Stock Advisor, and join an investing community built for the long haul. See the 10 stocks » *Stock Advisor returns as of August 13, 2026. This article is a transcript of this conference call produced for The Motley Fool. While we strive for our Foolish Best, there may be errors, omissions, or inaccuracies in this transcript. As with all our articles, The Motley Fool does not assume any responsibility for your use of this content, and we strongly encourage you to do your own research, including listening to the call yourself and reading the company's SEC filings. Please see our Terms and Conditions for additional details, including our Obligatory Capitalized Disclaimers of Liability. The Motley Fool has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy. Scholar Rock (SRRK) Q2 2026 Earnings Call Transcript was originally published by The Motley Fool

Interested in Scholar Rock Holding Corporation? Here are five stocks we like better. FDA decision remains on track for Sept. 30: Scholar Rock’s resubmitted application for apitegromab includes two independent fill-finish facilities, potentially providing alternative approval paths for the SMA treatment. Commercial preparations are advancing: The company says its U.S. launch organization is ready, while it is working with European regulators and planning an initial German launch. Patient access could take time as insurers establish coverage policies. Pipeline and finances support expansion: Scholar Rock started a Phase 2 apitegromab trial in FSHD, continues development of subcutaneous formulations and SRK-439, and ended the quarter with $492 million in cash and marketable securities. 3 Beaten-Down Stocks With Rebound Potential This Earnings Season Scholar Rock (NASDAQ:SRRK) said its resubmitted biologics license application for apitegromab remains on track for a U.S. Food and Drug Administration decision by its Sept. 30 PDUFA date, as the company prepares for a potential launch of the muscle-targeted treatment for spinal muscular atrophy, or SMA. The company’s application includes two fill-finish manufacturing facilities, providing what management described as independent potential paths to approval. The original approvability issue cited in a complete response letter last September concerned observations from a routine inspection at the Catalent Indiana fill-finish facility, which is owned and operated by Novo Nordisk. → Meta’s Earnings Drop Shows Wall Street Wants More Than Ad Growth Scholar Rock resubmitted its application on March 30 after discussions with the FDA, adding a second fill-finish facility. The application was accepted in April. The FDA’s inspection classification from an April 2026 reinspection of the Catalent Indiana facility remains pending. Chairman and Chief Executive Officer David Hallal said the company and FDA agreed during a March Type C meeting that including two fill-finish sites would offer the fastest potential route to approval. The company said it has submitted the data package needed for FDA review of the second facility and that the review is progressing. → 4 Oil and Gas ETF Plays as Prices Stay Sky-High “We have significant optionality with two independent paths to approval, either through Catalent, Indiana or through our second fill-finish facility or both,” Hallal said. Hallal said Scholar Rock now has more apitegromab vials available from the second facility than from Catalent Indiana. Vials from both sites are at a third-party provider awaiting packaging and labeling pending approval. → Sandisk Just Delivered a Blowout Quarter—Here's Why the Stock Is Falling During the question-and-answer session, Hallal said the second facility could support a fully independent approval path. If the FDA were to maintain the Catalent Indiana site’s current official action indicated, or OAI, status, Scholar Rock said it could remove the site from its application after receiving an agency signal, without expecting an effect on the Sept. 30 review timeline. Akshay Vaishnaw, Scholar Rock’s president of research and development, said the company sees no reason the FDA could not complete its review by the PDUFA date. The resubmission included new information relating to the second facility and an updated safety database, according to management. In Europe, Scholar Rock’s marketing authorization application currently lists Catalent Indiana as its sole fill-finish facility. The European Medicines Agency is awaiting the FDA’s inspection classification for that site, according to the company. Scholar Rock is also discussing with European regulators how its second fill-finish facility could be included in the application. Hallal said the facility has recently undergone successful FDA and EMA inspections and is in good standing with European regulators. The company did not provide a timetable for a Committee for Medicinal Products for Human Use opinion, saying it would update investors after further alignment with regulators. The company is preparing for an initial European launch in Germany, followed by other countries as it establishes a planned 50-country operating platform. Management said its ambition is to reach the estimated 35,000 people globally with SMA who have received an SMN-targeted therapy. Chief Operating Officer Keith Woods said the company’s U.S. commercial organization is ready to launch upon FDA approval. Scholar Rock is engaging approximately 140 SMA treatment centers, 2,600 prescribing physicians and multidisciplinary care teams, he said. The company has also trained its Scholar Rock Supports patient-services team to assist eligible patients and families with insurance coverage, financial and copay assistance, and treatment logistics. Woods said the company expects prior authorization requirements for apitegromab, similar to those used for existing SMN-targeted SMA treatments. He said patients in the company’s early-access program would be among the first considered for a transition to commercial product, followed by participants in its open-label extension and ONYX programs. However, Woods noted that access may take time, particularly while the company lacks a J-code and payers establish policies. He said that in comparable launches, the average time between prescription and infusion can exceed 60 days during the first six months. Scholar Rock said enrollment remains robust in its Phase 2 OPAL trial of apitegromab in infants and toddlers with SMA under age two. The study includes participants who have received an SMN1-targeted gene therapy or are receiving an SMN2-targeted treatment. The company also announced initiation of FORGE, a randomized, double-blind, placebo-controlled Phase 2 trial of apitegromab in facioscapulohumeral muscular dystrophy, or FSHD. The trial is planned to enroll 60 patients. Vaishnaw said Scholar Rock selected FSHD because of the absence of approved treatments, the disease’s impact on function, and preclinical and clinical evidence suggesting that gains in muscle mass may translate into functional benefits. The study will focus on mild-to-moderate patients, including those with Ricci scores between 1.5 and 3, he said. Scholar Rock is also advancing a high-concentration subcutaneous formulation of apitegromab. The company plans to engage U.S. and European regulators on its development path after apitegromab is approved. Management said Phase 1 data presented in January showed favorable bioavailability and a pharmacodynamic profile comparable with intravenous administration. For SRK-439, a high-potency subcutaneous anti-myostatin antibody, the company said dosing is progressing in a Phase 1 healthy-volunteer study. Top-line data are expected later this year. Scholar Rock reported second-quarter operating expenses of $108.9 million, including $19.7 million in non-cash stock-based compensation. Excluding stock-based compensation, operating expenses were $89.2 million. The company ended the quarter with $492 million in cash equivalents and marketable securities, including $63 million in net proceeds from its at-the-market equity program during the quarter. Chief Financial Officer Vikas Sinha said Scholar Rock could draw an additional $150 million from its existing debt facility upon FDA approval of apitegromab. The company also plans to monetize a priority review voucher to further strengthen its balance sheet. Management said its 2026 financial priorities include supporting a U.S. and European commercial launch, advancing its clinical pipeline, strengthening its supply chain, and maintaining financial flexibility. Scholar Rock is a clinical-stage biotechnology company focused on the discovery and development of protein therapeutics that selectively target growth factors involved in disease processes. The company's research platform is designed to modulate endogenous growth factor activation and signaling with high specificity, aiming to restore normal biological function across a range of disorders. Scholar Rock's approach is distinguished by its emphasis on engineering antibodies and biologics that interact with growth factor precursors or latent complexes rather than the active form, potentially offering improved safety and efficacy profiles. The company's lead program, SRK-015 (appercept), is an investigational monoclonal antibody targeting the activation of latent myostatin proproteins and is being evaluated for the treatment of spinal muscular atrophy (SMA). This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected]. The article "Scholar Rock Q2 Earnings Call Highlights" was originally published by MarketBeat. View MarketBeat's top stocks for August 2026.

This article first appeared on GuruFocus. Release Date: August 06, 2026 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Scholar Rock Holding Corp (NASDAQ:SRRK) has two independent paths to FDA approval for apitegromab, either through the Catalent, Indiana facility or the second fill-finish facility, providing significant regulatory optionality. The company has more commercially available vials of apitegromab from its second fill-finish facility than from Catalent, Indiana, and these vials are already at a third-party packaging site, ready for launch upon approval. Scholar Rock Holding Corp (NASDAQ:SRRK) ended Q2 2026 with a strong cash position of $492 million, which includes $63 million in net proceeds from its ATM program, and has access to an additional $150 million debt facility and a priority review voucher to monetize. The company's commercial organization is fully launch-ready in the US, with preparations including engagement with 140 SMA treatment centers, 2,600 prescribing physicians, and a trained patient services team, positioning it for an immediate launch upon approval. Scholar Rock Holding Corp (NASDAQ:SRRK) is advancing its pipeline beyond SMA, with robust enrollment in the Phase 2 OPAL study for infants, initiation of the Phase 2 FORGE study in FSHD, and promising progress on a subcutaneous formulation and SRK-439, a high-potency myostatin inhibitor. The FDA inspection classification for the Catalent, Indiana facility remains pending, which is delayed beyond the typical 90-day guidance period and creates uncertainty for the approval timeline. European approval of apitegromab is dependent on FDA clearance of the Catalent, Indiana facility, which is currently the sole fill-finish site in the MAA, potentially delaying the European launch. The company anticipates that most patients prescribed apitegromab will face prior authorization requirements and potential denials, leading to an average time from prescription to infusion of over 60 days during the first six months of launch. Scholar Rock Holding Corp (NASDAQ:SRRK) reported Q2 2026 operating expenses of $108.9 million, including $19.7 million in non-cash stock-based compensation, reflecting increased commercial investments that could pressure future profitability. The company's reliance on a third-party facility (Catalent, Indiana) for regulatory approval in both the US and Europe exposes it to external operational risks, as evidenced by the ongoing CRL-related issues. Warning! GuruFocus has detected 4 Warning Signs with SRRK. Is SRRK fairly valued? Test your thesis with our free DCF calculator. Q: How confident are you that the second fill-finish facility alone, independent of Catalent, can support approval by the PDUFA date, and can you share any anecdotes from FDA interactions to support that they are making progress in the review of the package you submitted? A: David Hillau, Board Chair and CEO, stated that following the March 3rd Type C meeting, there was complete alignment with the FDA on resubmitting the BLA with two fill-finish facilities to enable the fastest path to approval. He highlighted that the company now has more vials of apitegromab ready for launch from the second facility than from Catalent, Indiana. The FDA has all the agreed-upon data, and the review is progressing well. He noted the second facility has had successful recent site inspections by both the FDA and EMA, and that removing a facility from the application would not impact the review timeline. The team is ready to launch at any time up to the September 30th PDUFA date. Q: If Catalent remains classified as OAI, is there a deadline or date for administratively withdrawing that site so it doesn't trigger any delay for your PDUFA? A: David Hillau, Board Chair and CEO, explained that the inspection classification is still pending, slightly beyond the FDA's 90-day guidance period, which is not a statutory requirement. He confirmed that the company has discussed with the agency that if the classification reveals no change to the current OAI status, it would be a relatively simple step to notify the FDA of removing that site, and the review would progress with the second facility with no expected impact to the timeline. Q: What are the specific procedural items from now to September 30th that still need to be ticked off to allow for approval, and is there any reason to believe the FDA will not be able to complete the review by then? A: Akshay Vaishnaw, President of R&D, confirmed there is no reason to believe the FDA cannot complete the review. He detailed that since the November Type A meeting and the March Type C meeting, the FDA guided that Catalent is the lead site but welcomed the second site to help get the drug approved in a compliant manner for a high unmet need. The resubmitted BLA included only new information from the second facility and the updated safety database. The review of the second facility is progressing well, and the company is exactly on track for approval before or at the PDUFA date. Q: Do you have any color or feedback from the FDA on the later-than-expected reclassification of the Catalent site, given the decision was expected by the end of July? A: David Hillau, Board Chair and CEO, noted that the inspection report and 483 observations are public, and Novo Nordisk provided a robust response within the 15-day window, giving the FDA a lot to review. He reiterated that the 90-day period is guidance, not a statutory requirement. While awaiting the classification, the company is moving forward with the second fill-finish facility in both the US and Europe, describing the approach as "belt and suspenders" to serve patients, and feels they are in a position of strength. Q: Regarding national and regional payer discussions, how are they framing apitegromab in step edit terms, or are payers signaling they will impose time limits or require re-review of benefit after a shorter initial authorization? A: Keith Woods, Chief Operating Officer, stated the team is working with commercial and government payers to create policies that align with the potential FDA label and the inclusion/exclusion criteria from the SAPPHIRE study. He noted that almost 100% of the three SMN-targeted therapies have prior authorization, so apitegromab will likely also require prior authorization. The goal is to make apitegromab available to the broadest possible audience, and the team is bringing in medical team members to discuss the robust data package. Q: Could you give us a sense of what was included in the data package for the second fill-finish facility, whether stability runs and release testing are fully complete, and if the facility is considered launch-ready? A: David Hillau, Board Chair and CEO, confirmed that the data package included engineering runs and PPQ runs, which the FDA has in hand. He stated there is no more testing required for the product vials at that facility. The product is already at the third-party labeling and packaging facility awaiting approval, alongside vials from Catalent, putting the company in a strong position for launch. Q: Are there any other initiatives needed between now and approval, and what is the low-hanging fruit to go after with SMA patients where there could be pent-up demand, and how long will it take to get those patients on board? A: Keith Woods, Chief Operating Officer, said the team is ready to launch immediately upon approval. The first patients to convert will be those in the early access program, followed by ONYX open-label extension patients, who must complete a close-out visit before converting. He noted that prior authorizations and potential denials will likely extend the time from prescription to infusion, with an average of greater than 60 days during the first six months of launch without a J-code. Q: Is the medical review done on both sides of the Atlantic, and do you expect a largely identical label between the US and Europe, or are there important differences? A: Akshay Vaishnaw, President of R&D, stated the company is happy with the dialogue through the medical review and is comfortable with how the drug will be positioned. David Hillau, Board Chair and CEO, added that the company will comment on final labels (USPI and SmPC) when it is the right time, but the team is in a good position to serve a meaningful number of patients in Europe and eventually reach patients in 50 countries. Q: If the FDA maintains the OAI classification for Catalent, can you walk us through the potential timeline to get the second fill-finish facility into the MAA and your thoughts on the earliest projected timeline for European approval? A: Akshay Vaishnaw, President of R&D, explained that while the EMA awaits the FDA's classification decision, the company is in parallel dialogue with the EMA about the second facility. He declined to speculate on the final advice but expressed gratitude for the engagement and flexibility shown by both the FDA and CHMP, noting they appreciate the unmet need and are working collaboratively. The company will be guided by regulators and will update guidance upon alignment. Q For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Apitegromab Biologics License Application (BLA) for spinal muscular atrophy (SMA) review by FDA continues to advance with two fill-finish facilities, representing two independent paths to an FDA approval decision by September 30, 2026 Prescription Drug User Fee Act (PDUFA) date FDA review of second fill-finish facility progressing; Data package for FDA review of second fill-finish facility has been submitted; Ample supply available for commercialization upon FDA approval Scholar Rock is prepared for U.S. apitegromab launch immediately upon FDA approval, which may be granted at any time through September 30, 2026 Company engaging with European Medicines Agency (EMA) on next steps for apitegromab Marketing Authorisation Application (MAA); FDA inspection classification of Catalent Indiana LLC (part of Novo Nordisk) is pending Initiated Phase 2 FORGE study evaluating apitegromab in patients with facioscapulohumeral muscular dystrophy (FSHD) Cash, cash equivalents, and marketable securities of $492 million as of June 30, 2026; Includes $63 million in net cash proceeds from Company’s at-the-market (ATM) program Management to host a conference call today at 8:00 a.m. ET CAMBRIDGE, Mass., August 06, 2026--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of patients with rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology, today reported financial results for the second quarter ended June 30, 2026, and provided an update on recent company developments. "We are on the threshold of securing the world’s first ever regulatory approval of a myostatin inhibitor, which will also be the first ever muscle-targeted treatment for children and adults living with SMA," said David L. Hallal, Board Chair and Chief Executive Officer of Scholar Rock. "Backed by a strong balance sheet, our Scholar Rock team is ready to usher in the next phase of innovation for the SMA community in the U.S. immediately upon apitegromab approval." Business Highlights and Upcoming Milestones Apitegromab Apitegromab is an investigational fully human monoclonal antibody designed to inhibit myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle. It is the first and only muscle-targeted therapeutic candidate in SMA to demonstrate a statistically significant and clinically meaningful benefit in a pivotal Phase 3 clinical trial (SAPPHIRE). SMA Program Apitegromab Biologics License Application (BLA) on track for potential FDA approval by September 30, 2026 Prescription Drug User Fee Act (PDUFA) date. The FDA review of the apitegromab BLA is advancing with both the Catalent Indiana fill-finish facility and a second fill-finish facility, representing two independent paths to an FDA approval decision. At the March 2026 Type C meeting, FDA and Scholar Rock agreed to the data package required for FDA review of the second fill-finish facility. That data package has been submitted, and Agency review of the data is progressing well. Ample supply from the second fill-finish facility is available for commercialization upon FDA approval. The FDA inspection classification of Catalent Indiana following an April 2026 general site inspection is pending. U.S. Commercial team prepared to launch apitegromab upon FDA approval. The U.S. Commercial team remains active in the field with SMA prescribers and centers of excellence nationwide, furthering disease education and awareness initiatives. The Company had a significant presence at Cure SMA’s Annual SMA Conference and Annual SMA Research & Clinical Care Meeting, which was held June 23 – 28, 2026 in Orlando, FL. Scholar Rock engaging with European Medicines Agency (EMA) on next steps for apitegromab Marketing Authorisation Application (MAA). The EMA is reviewing the apitegromab MAA, which includes the Catalent Indiana fill-finish facility, and is awaiting the FDA inspection classification of Catalent Indiana. In parallel, Scholar Rock is engaging with the EMA on next steps, including the potential to add the Company’s second fill-finish facility to the MAA. The Company plans to provide updated guidance on timelines for a Committee for Medicinal Products for Human Use (CHMP) opinion upon alignment with the EMA. Continued robust enrollment in Phase 2 OPAL trial. Enrollment and dosing in the Phase 2 OPAL study is ongoing (NCT07047144). The OPAL study is evaluating apitegromab in infants and toddlers with SMA under two years of age who have received an approved SMN1-targeted gene therapy or who are receiving ongoing treatment with an approved SMN2-targeted therapy. Subcutaneous apitegromab progressing. Scholar Rock has developed a high concentration subcutaneous formulation of apitegromab. The Company plans to engage with the FDA and EMA to align on the development path following the regulatory approvals of apitegromab for SMA. Facioscapulohumeral Muscular Dystrophy (FSHD) Program Phase 2 FORGE trial initiated. The Phase 2 randomized, double-blind, placebo-controlled trial, called FORGE, is expected to enroll approximately 60 patients with FSHD who will be randomized 1:1 to receive either apitegromab 10mg/kg IV or placebo every 4 weeks for 52 weeks. The primary endpoint is mean lean muscle volume (LMV) change from baseline at 12 months. Secondary endpoints include safety, pharmacokinetics/pharmacodynamics, and mean LMV change from baseline at 6 months. Several exploratory functional endpoints will also be assessed. SRK-439 SRK-439 is a novel, investigational, subcutaneously administered myostatin inhibitor that binds to pro- and latent myostatin with high affinity and selectivity. Based on preclinical data, SRK-439 has the potential to potently inhibit myostatin and increase muscle mass. Phase 1 study in healthy volunteers ongoing. A Phase 1 study evaluating SRK-439 in healthy volunteers is progressing well with topline data anticipated in late 2026. Second Quarter 2026 Financial Results Scholar Rock reported a net loss of $109.9 million, including stock-based compensation of $19.7 million, for the quarter ended June 30, 2026, compared to a net loss of $110.0 million, including stock-based compensation of $24.4 million, for the quarter ended June 30, 2025. Net loss per common share was $0.84 for the quarter ended June 30, 2026, compared to $0.98 per common share for the quarter ended June 30, 2025. The Company did not record any revenue for the quarters ended June 30, 2026 and 2025. Research and development expense was $58.2 million, including $7.5 million in stock-based compensation, for the quarter ended June 30, 2026, compared to $62.4 million, including $5.8 million in stock-based compensation, for the quarter ended June 30, 2025. General and administrative expense was $50.7 million, including $12.2 million in stock-based compensation, for the quarter ended June 30, 2026, compared to $49.7 million, including $18.6 million in stock-based compensation, for the quarter ended June 30, 2025. As of June 30, 2026, Scholar Rock had cash, cash equivalents, and marketable securities of $492.1 million. This reflects net cash proceeds of $62.8 million from the Company’s at-the-market (ATM) program during the quarter ended June 30, 2026. Conference Call Information Scholar Rock will host a conference call and webcast today, Thursday, August 6, at 8:00 a.m. ET to review its second quarter 2026 financial results and discuss recent business updates. To access the live audio webcast, please go to "Events and Presentations" in the Investors section of the Scholar Rock website at http://investors.scholarrock.com. To participate via telephone, please register in advance here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call. A replay of the webcast will be available on the Company’s website for approximately 90 days. About Scholar Rock Scholar Rock is a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for children and adults with spinal muscular atrophy (SMA) and other rare, severe and debilitating neuromuscular diseases. As a global leader in myostatin biology, a field focused on proteins that regulate muscle mass, the biopharmaceutical company is named for the visual resemblance of a scholar rock to protein structures. Our commitment to unlock fundamentally different treatment approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. Scholar Rock works every day to create new possibilities for patients through its highly innovative anti-myostatin programs, including opportunities in additional rare neuromuscular diseases. Learn more at ScholarRock.com and follow @ScholarRock on X and on LinkedIn. Scholar Rock® is a registered trademark of Scholar Rock, Inc. Availability of Other Information About Scholar Rock Investors and others should note that we communicate with our investors and the public using our company website www.scholarrock.com, including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on X (formerly known as Twitter) and LinkedIn. The information that we post on our website, X, or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended. Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rock’s future expectations, plans and prospects, including without limitation, Scholar Rock’s expectations regarding its growth, strategy, progress and timing of its clinical trials and development programs for apitegromab, including its subcutaneous formulation, SRK-439 and its preclinical programs, and indication selection and development timing, including the timing of any regulatory submissions, decisions and anticipated approvals, the therapeutic potential, clinical benefits and safety of any product candidates, expectations regarding actions by the FDA after its reinspection of the Catalent Indiana facility; the expected timing and outcome of FDA review of the accepted BLA for apitegromab, including the September 30, 2026 PDUFA action date; expectations regarding timing and outcome of EMA review and MAA approval; expectations regarding the availability and timing of commercial supply of apitegromab from Catalent Indiana and a second U.S.-based fill-finish facility, including expected supply from the second fill-finish facility; expectations regarding commercial launch timing, and the achievement of important milestones, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, the potential of its product candidates and proprietary platform. The use of words such as "may," "might," "could," "will," "should," "expect," "plan," "anticipate," "believe," "estimate," "project," "intend," "future," "potential," or "continue," and other similar expressions are intended to identify such forward-looking statements. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, without limitation, whether preclinical and clinical data, including the results from the Phase 3 SAPPHIRE trial and any results from ongoing or future clinical trials, including the Phase 2 OPAL clinical trial, the Phase 2 FORGE trial and the Phase 1 clinical trial of SRK-439, will be sufficient to support regulatory approval or further development; that preclinical and clinical data, including the results from the Phase 2 or Phase 3 clinical trial of apitegromab, data from any ongoing or future trials of apitegromab or data for SRK-439, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidates; whether the FDA will accept the remediations to the Catalent Indiana fill finish facility in response to the FDA observations, whether the updated BLA, including a second fill finish facility, will be sufficient to support regulatory approval, Scholar Rock’s ability to manage expenses or provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline; information provided or decisions made by regulatory authorities; competition from third parties that are developing products for similar uses; Scholar Rock’s ability to obtain, maintain and protect its intellectual property; and Scholar Rock’s dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock’s views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law. View source version on businesswire.com: https://www.businesswire.com/news/home/20260806667041/en/ Contacts Investors Laura Ekas, [email protected] Media Jeff Smith682-401-8428Molly MacLeod, [email protected]

CAMBRIDGE, Mass., July 16, 2026--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK) today announced that it will report second quarter 2026 financial results on Thursday, August 6, 2026, before the financial markets open. The Company will host a conference call and webcast with Scholar Rock management at 8:00 a.m. ET. To access the live audio webcast, please go to "Events and Presentations" in the Investors section of the Scholar Rock website at http://investors.scholarrock.com. To participate via telephone, please register in advance here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call. An archived replay of the webcast will be available on the Company’s website for approximately 90 days. About Scholar RockScholar Rock is a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for children and adults with spinal muscular atrophy (SMA) and other rare, severe, and debilitating neuromuscular diseases. As a global leader in myostatin biology, a field focused on proteins that regulate muscle mass, the biopharmaceutical company is named for the visual resemblance of a scholar rock to protein structures. Our commitment to unlock fundamentally different treatment approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. Scholar Rock works every day to create new possibilities for patients through its highly innovative anti-myostatin program, including opportunities in additional rare neuromuscular diseases. Learn more at ScholarRock.com and follow @ScholarRock on X and on LinkedIn. Scholar Rock® is a registered trademark of Scholar Rock, Inc. Availability of Other Information About Scholar RockInvestors and others should note that we communicate with our investors and the public using our company website www.scholarrock.com, including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on X (formerly known as Twitter) and LinkedIn. The information that we post on our website, X or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended. View source version on businesswire.com: https://www.businesswire.com/news/home/20260716882246/en/ Contacts Scholar Rock Contacts Investors Laura Ekas, [email protected] 917-439-0374Media Jeff Smith682-401-8428Molly MacLeod, [email protected]

Interested in Scholar Rock Holding Corporation? Here are five stocks we like better. FDA accepted Scholar Rock’s resubmitted BLA for apitegromab in spinal muscular atrophy and set a PDUFA date of Sept. 30, 2026. The filing includes two fill-finish facilities, giving the company two potential paths to approval. The company is preparing for launch ahead of approval and said commercial supply from the second manufacturing site should be available in early third quarter. Management also said approval could come before the PDUFA date, and European review is progressing in parallel. Scholar Rock ended Q1 2026 with $480 million in cash and marketable securities. The company is prioritizing launch readiness and R&D while also pursuing additional financing options, including a potential debt draw and monetization of a priority review voucher. 3 Beaten-Down Stocks With Rebound Potential This Earnings Season Scholar Rock (NASDAQ:SRRK) said the U.S. Food and Drug Administration has accepted for review its resubmitted Biologics License Application for apitegromab, the company’s investigational treatment for children and adults with spinal muscular atrophy, and set a PDUFA action date of Sept. 30, 2026. Chairman and Chief Executive Officer David Hallal said the accepted filing includes two fill-finish facilities: Catalent’s Indiana site and a second U.S.-based facility. He said that gives the company “two independent paths to apitegromab approval.” → Insider Sales: Top AST SpaceMobile Insider Cuts Postion Over 30% The update follows a complete response letter issued last September, which Scholar Rock said was tied solely to observations from a routine general site inspection of the Catalent, Indiana fill-finish facility, owned and operated by Novo Nordisk. Hallal said the FDA has since completed an unannounced re-inspection of that facility, and that under FDA guidelines, the agency has up to 90 days to classify the site. Hallal said Scholar Rock resubmitted the BLA in late March “in complete alignment with the FDA” to include both manufacturing sites. The company said the apitegromab drug product required for FDA review and potential approval from the second facility has been filed, and it expects to have commercial supply from that facility available in early third quarter, ahead of the Sept. 30 action date. → Light Speed Returns: Corning Cashes In on NVIDIA Growth Scholar Rock executives repeatedly said the company is preparing to launch apitegromab immediately if approved, including ahead of the formal PDUFA date. In response to an analyst question, Hallal said Class II resubmissions related to manufacturing issues can sometimes receive action before the deadline. “We just know that we need to be prepared because many times Class 2 resubmissions and action can be taken by the agency well ahead of that PDUFA date,” Hallal said. → Years in the Making, AMD’s Upside Movement Has Just Begun Akshay Vaishnaw, president of R&D, said the company anticipates approval of apitegromab for children and adults with SMA “by the end of the Q3,” potentially supported by either or both fill-finish facilities. Vaishnaw also discussed the European regulatory process, saying the company’s Marketing Authorization Application is progressing through EMA review. He said Scholar Rock and the EMA had been scheduled for an oral explanation meeting, but the meeting was no longer needed after the two sides aligned in advance. The company continues to expect a CHMP opinion near mid-year. Vaishnaw noted that approval in Europe also requires FDA clearance of the Catalent Indiana facility. Hallal said European regulators have been kept informed about the FDA process and have been “very flexible” on timing. Chief Operating Officer Keith Woods said Scholar Rock’s U.S. commercial team is preparing for launch “immediately upon approval,” which could occur any time through Sept. 30. He said muscle strength and motor function remain major unmet needs nearly a decade after the introduction of SMN-targeted therapies, citing company-referenced data that 95% of patients continue to experience persistent and progressive muscle atrophy. Woods said data shared by Cure SMA indicated that about one-third of people living with SMA in the U.S. have received two or more SMN-targeted treatments, either sequentially or in combination. He said the company views that as evidence of remaining unmet need and an opportunity for apitegromab as a muscle-targeted therapy. The company said its U.S. field team has reached approximately 140 SMA treatment centers, 2,600 prescribing physicians and multidisciplinary care teams. Woods said Scholar Rock is preparing its patient services program, Scholar Rock Supports, to provide individualized support to patients, caregivers and providers. During the question-and-answer session, Woods said the company has expanded site-of-care planning, including access through partners to more than 10,000 home infusion nurses across the U.S. He also said Scholar Rock has expanded its specialty pharmacy network so patients do not have to use multiple specialty pharmacies for different SMA treatments. In Europe, Woods said Scholar Rock has established its regional headquarters in Switzerland and has local leadership in place in Germany, where it expects to launch following EMA approval. He said the company has hired medical and commercial field teams in Germany and is enrolling patients in a compassionate use program. Beyond the pending SMA application, Vaishnaw said Scholar Rock is continuing to enroll and dose patients in the Phase 2 OPAL trial, which is evaluating apitegromab in infants and toddlers under age 2. The study includes participants treated with SMN1-targeted gene therapy or receiving ongoing SMN2-targeted therapy. The company is also preparing to initiate a randomized, double-blind, placebo-controlled Phase 2 trial called FORGE in facioscapulohumeral muscular dystrophy, or FSHD. Vaishnaw said the study is expected to begin enrollment soon and will include 60 patients. He described FSHD as a rare neuromuscular disease with more than 30,000 diagnosed patients in the U.S. and Europe and no approved therapies. Vaishnaw said Scholar Rock prioritized FSHD based on unmet need, preclinical data from the FLExDUX4 mouse model and prior randomized studies suggesting that increases in muscle mass may be associated with functional benefit in FSHD. Scholar Rock is also advancing a subcutaneous formulation of apitegromab. Vaishnaw said Phase 1 data presented in January showed favorable bioavailability and a pharmacodynamic profile comparable to intravenous administration. The company plans to engage U.S. and European regulators later this year following apitegromab approval. The company also discussed SRK-439, described in prepared remarks as a novel high-potency anti-myostatin antibody currently in Phase 1. Vaishnaw said dosing in a healthy volunteer study is progressing and that top-line data are expected later this year. Chief Financial Officer Vikas Sinha said Scholar Rock ended the first quarter of 2026 with $480 million in cash equivalents and marketable securities. The total includes a $100 million drawdown from the company’s existing debt facility in March and $98 million in net cash proceeds from its at-the-market equity program during the quarter. Sinha said first-quarter operating expenses were $102 million, and that operating expenses excluding stock-based compensation were $84 million. He said Scholar Rock’s 2026 financial priorities remain focused on supporting commercial launch readiness, funding R&D programs and evaluating opportunities to strengthen the balance sheet. Upon FDA approval of apitegromab, Sinha said Scholar Rock will have the option to draw an additional $150 million from its existing debt facility. The company also plans to monetize a priority review voucher to further strengthen its balance sheet. Hallal closed the call by saying Scholar Rock is focused on bringing what it describes as the first muscle-targeted therapy to children and adults living with SMA while building a broader neuromuscular disease pipeline. Scholar Rock is a clinical-stage biotechnology company focused on the discovery and development of protein therapeutics that selectively target growth factors involved in disease processes. The company's research platform is designed to modulate endogenous growth factor activation and signaling with high specificity, aiming to restore normal biological function across a range of disorders. Scholar Rock's approach is distinguished by its emphasis on engineering antibodies and biologics that interact with growth factor precursors or latent complexes rather than the active form, potentially offering improved safety and efficacy profiles. The company's lead program, SRK-015 (appercept), is an investigational monoclonal antibody targeting the activation of latent myostatin proproteins and is being evaluated for the treatment of spinal muscular atrophy (SMA). The article "Scholar Rock Q1 Earnings Call Highlights" was originally published by MarketBeat. View MarketBeat's top stocks for May 2026.

Image source: The Motley Fool. Thursday, May 7, 2026 at 8:00 a.m. ET Chief Executive Officer — David Hallal President — Akshay Vaishnaw Chief Commercial Officer — Robert Keith Woods Chief Financial Officer — Vikas Sinha Need a quote from a Motley Fool analyst? Email [email protected] David Hallal: Thank you, Laura, and good morning. Thanks to everyone for joining our first quarter earnings call. Scholar Rock is positioned for a pivotal year ahead. To that end, today, I am very pleased to announce that the FDA has accepted for review our biologics license application for apitegromab for the treatment of children and adults living with SMA. The agency has assigned a PDUFA action date of September 30. Importantly, the accepted BLA includes 2 fill-finish facilities, Catalent Indiana and a second U.S.-based facility, providing Scholar Rock with 2 independent paths to apitegromab approval. As a reminder, the sole approvability issue for apitegromab noted in the complete response letter last September was related to observations identified during a routine general site inspection of the Catalent Indiana fill-finish facility, which is owned and operated by Novo Nordisk. Since our in-person Type A meeting with the FDA in Q4, we have continued to work constructively and collaboratively with the agency, and we have made steady and rapid progress. During the first quarter, we made meaningful advancements at Catalent, Indiana and our second fill-finish facility. And with our ongoing open communication with the agency, we resubmitted our apitegromab BLA in late March in complete alignment with the FDA to include both facilities. This approach underscores the shared understanding between the FDA and Scholar Rock of the unmet need in the SMA community and the shared urgency to bring apitegromab to children and adults in the U.S. as quickly as possible. I would like to now provide an update on the status of each of these 2 sites. As it relates to Catalent Indiana, we are pleased that following acceptance of our BLA, the FDA completed an unannounced reinspection of the facility. This timing was in line with our expectations as the FDA had noted following multiple engagements with Novo in Q1 that they would conduct an unannounced inspection following routine manufacturing activities, which resumed in late February. We are pleased that the inspection was completed in early Q2 and in accordance with FDA guidelines, the agency has up to 90 days to classify the facility. As it relates to the second fill-finish facility, we continue to be pleased with our ongoing meaningful progress. Importantly, the entirety of the apitegromab drug product required for FDA review and potential approval has been filed. From a commercial supply standpoint, we are well positioned as we expect to have ample commercial apitegromab available from the second facility in early Q3, well ahead of the September PDUFA date. We remain committed to the SMA community, and we are grateful that significant progress continues to be made at a rapid pace. Our U.S. commercial team continues to advance the critical activities and capabilities required to deliver a seamless launch and support patients from day 1. Importantly, the team stands ready to launch apitegromab immediately upon approval at any time prior to, and including the September 30 PDUFA date. In addition to the U.S., we continue to look forward to serving children and adults with SMA in Europe. The review of our MAA is progressing very well, and we expect a CHMP opinion near midyear. We are building momentum with launch readiness activities, and we continue to anticipate a launch in the second half of the year, beginning with Germany. We know it is not a matter of if, but when apitegromab will be approved for children and adults with SMA, and Keith will discuss the continued progress we are making with commercial preparations and our disease awareness initiatives shortly. We continue to advance our world-leading anti-myostatin pipeline with enrollment in our Phase II OPAL study evaluating apitegromab in infants and toddlers with SMA, the anticipated initiation of our randomized Phase II study in patients with FSHD and progress with subcutaneous apitegromab and a novel high potency anti-myostatin antibody, SRK-439 currently in Phase I. Akshay will discuss these programs in greater detail shortly. Turning now to the balance sheet. We are pleased to have ended the first quarter of 2026 with $480 million in cash, cash equivalents and marketable securities. This cash balance includes the drawdown of an additional $100 million from our debt facility, which we took in March. Our cash balance also reflects net cash proceeds of $98 million from our ATM program during the quarter. Vikas will provide more details later in the call. We are building on a solid foundation for our company's growth, which we believe will be steady and consistent through the end of this decade and well into the next as we prepare to serve up to 35,000 children and adults living with SMA around the world who have received at least one SMN targeted therapy. Beginning with SMA, we are excited to be shaping the future of treatment for patients living with rare and devastating neuromuscular diseases. And with that, I'll now turn the call over to Akshay. Akshay? Akshay Vaishnaw: Thanks, David, and good morning, everybody. We're very pleased with advancements in our world-leading anti-myostatin pipeline during the first quarter. Turning first to apitegromab for children and adults with SMA. We're delighted to share that the FDA has accepted the apitegromab BLA. As a reminder, the BLA was resubmitted in alignment with the agency to include both Catalent Indiana and a second U.S.-based fill-finish facility. The approach provides Scholar Rock with 2 independent path to apitegromab approval by the PDUFA action date of September 30. 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 and the current acceptance of the BLA. Throughout, the agency has appreciated the high unmet need in the SMA community, and we now look forward to the final steps in the U.S. regulatory process. Reflecting the agency's vigorous efforts, we were pleased most recently with the timing of the FDA's unannounced reinspection of Catalent Indiana. For FDA guidelines, the agency now has up to 90 days to classify the status of the facility. I'd now like to turn to our second fill-finish facility, where we continue to make meaningful progress. As David noted, the apitegromab drug product required for FDA data review and potential approval has been filed, and we expect to have ample commercial apitegromab from the facility in early Q3 ahead of the September PDUFA date. Based on the significant progress at both facilities, we anticipate approval of apitegromab for children and adults with SMA, which could be supported by either or both facilities by the end of the third quarter. Turning now to Europe. Our MAA for apitegromab for the treatment of children and adults with SMA continues to progress well through EMA review. As evidence of the progress, we have planned to be with the EMA recently for an oral explanation meeting. However, because we and the EMA were able to align prior to the scheduled meeting, we mutually agreed that the oral explanation was no longer necessary. As we highlighted previously, approval in Europe also requires FDA clearance for the Catalent Indiana facility. Based on our discussions with EMA, they're aware of the progress at Catalent Indiana and are comfortable with the review time line that accounts for the FDA's classification as site. We continue to be very pleased with how the review is progressing, and we anticipate a CHMP opinion in the middle of the year. Turning to our pipeline. Let me start with the Phase II OPAL trial. We continue to enroll and dose patients in this study, which is evaluating apitegromab in infants and toddlers 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 therapy. 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. In addition, we believe early intervention with apitegromab could support muscle during a critical early development phase, potentially improving motor outcomes in the youngest of patients with SMA. Turning now to our next indication for apitegromab, facioscapulohumeral muscular dystrophy or FSHD. FSHD is a rare, devastating neuromuscular disease with significant unmet need. More than 30,000 patients are diagnosed in the U.S. and Europe alone, and there are no approved therapies. We prioritized FSHD as the next indication for apitegromab for 3 key reasons: First, the significant unmet need; second, the compelling preclinical data from the gold standard FLExDUX4 mouse model that provides mechanistic rationale for apitegromab in FSHD. And finally, as shown on Slide 11, 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 with the progress of activities to support the initiation of our Phase II study called FORGE in the middle of this year. Enrollment will commence soon in this randomized, double-blind, placebo-controlled trial, which has a sample size of 60 patients. We're also advancing 2 additional programs in our world-leading anti-myostatin pipeline, a subcutaneous formulation of apitegromab and SRK-439. In our subcutaneous apitegromab program, we showed some very exciting data from a Phase I study in January, which demonstrated that subcu apitegromab appears to have favorable bioavailability and a pharmacodynamic profile comparable to IV administration. Additional development activities are ongoing, and we continue to plan for engagements 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, which we discovered by leveraging our world-leading expertise. 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 this 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 apitegromab and our world-leading anti-myostatin pipeline across a range of rare devastating neuromuscular diseases. With that, I'll now turn the call over to Keith to discuss our commercial launch preparations. Keith? Robert Keith Woods: Thanks, Akshay, and good morning, everyone. With the BLA accepted by the FDA, our team continues to operate with urgency as we prepare for the launch of apitegromab immediately upon approval, which may be granted at any time through September 30, 2026. Nearly a decade after the introduction of SMN-targeted therapies, muscle strength and motor function remain the top unmet need with 95% of patients continuing to experience persistent and progressive muscle atrophy. That limits 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. Our U.S. customer-facing team continues to make significant progress in the field with disease education, awareness around the unmet medical need and reinforcing a broader understanding of SMA as a disease which consists of both the motor neuron and the muscle, the principal organ impacted by the disease. In the U.S., we have achieved significant reach across the approximately 140 SMA treatment centers, 2,600 prescribing physicians and their multidisciplinary care teams. Through these engagements, our field team is working to establish case flows on a center-by-center basis to ensure we are well positioned to support the SMA treatment centers once a treatment decision is made. This includes preparations to launch our patient services program, Scholar Rock Supports. This program is designed to provide comprehensive and individualized support to patients, caregivers and providers. In the first quarter, we had a meaningful presence at the Muscular Dystrophy Association meeting in March. During this meeting, our team further engaged with health care professionals. As one example, we hosted a very well-attended industry forum called going beyond the motor neuron to the muscle, expanding the focus of SMA care. We also remain highly focused on patients and community activation. We are building on our disease awareness campaign called Life Takes Muscle, and we continue to have numerous in-person patient and patient advocacy group engagements. Turning to U.S. reimbursement. Our market access team is advancing discussions with national and key regional payers as well as Medicare and Medicaid. With this extra time, we've been able to go deeper and broader across the range of payers. We are ready and well positioned for a successful launch of apitegromab in the U.S. immediately upon approval. Scholar Rock is also making significant progress in Europe. We have established our European headquarters in Switzerland. Also in Germany, where we expect to launch apitegromab upon EMA approval, our local leadership is on board. We have hired our medical and commercial field teams, and we are actively enrolling patients in our compassionate use program. We are making meaningful progress with reimbursement planning to enable rapid patient access. In the broader region, we are advancing reimbursement dossiers in multiple countries, strengthening our distributor relationships and building our EMEA infrastructure to support future commercialization. Additionally, we had a significant presence at the SMA Europe meeting in March in Budapest. Among other high-impact activities, we hosted an SMA disease education workshop and a health care professional symposium, where the attendance reflected a high interest in further understanding SMA and the unmet needs in this disease. In closing, we are investing with discipline to build the commercial foundation necessary to support a world-class launch and to achieve our long-term ambition to bring apitegromab to the estimated 35,000 patients living with SMA around the world who have received at least one SMN-targeted therapy. We are ready to usher in the next phase of innovation for children and adults with SMA, one patient, one caregiver and family at a time. With that, I'll turn the call over to Vikas. Vikas? Vikas Sinha: 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. In keeping with these objectives, I'm pleased to provide our first quarter financial results. For the first quarter, we reported $102 million in operating expenses, which included $80 million in noncash stock-based compensation. Excluding stock-based compensation, operating expenses were $84 million. Turning to our balance sheet. We are very pleased to have ended the first quarter with $480 million in cash, cash equivalents and marketable securities. During the quarter, we strengthened our cash position with the drawdown of an additional $100 million from our existing debt facility, which we took in March. We also had net cash proceeds of $98 million from our ATM program during the first quarter. Looking ahead, upon 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 tight financial plan and our prioritized investments remain focused on our apitegromab commercial launch readiness in the U.S. and Europe, strengthening our supply chain to support our expanding pipeline and our anticipated growing global commercial demand for apitegromab over time and advancing our highly innovative clinical programs that Akshay discussed earlier in the call. With that, I will turn the call back to David. David? David Hallal: Thanks, Vikash. Scholar Rock is poised for a transformative year in 2026. Our priorities are clear, and we are executing with focus, discipline and urgency as we seek to deliver the world's first muscle-targeted therapy to children and adults living with SMA, while also laying the foundation to realize our ambition to develop life-transforming therapies for patients with additional rare and severe neuromuscular diseases globally. We are ready now more than ever to usher in the next phase of innovation forth SMA community, and we look forward to updating you on our continued progress. And with that, we'll now open the line for questions. Operator? Operator: [Operator Instructions] And the first question comes from Eric Schmidt with Cantor. Eric Schmidt: Congrats on all the progress. Maybe just a couple of quick questions on apitegromab approval time lines in the U.S. Team, I know it's not your facility, the Catalent facility, but are you aware of any field notes that were provided to Novo following the reinspection? And then I guess I'm also curious about the statement that you reiterated a couple of times now that approval may come at any time. I know that probably reflects the shared understanding and communication you have with the FDA, but just curious about the intent of that statement. David Hallal: Thanks, Eric. I'll take both. And look, we were obviously very pleased today to have announced that the FDA accepted the BLA with 2 fill-finish facilities. And to be clear, it was a Class II resubmission with a PDUFA action date of September 30, which is sort of per protocol for manufacturing-related issues. So we anticipated that. And of course, as a reminder, we submitted that BLA in complete alignment with the FDA ahead of the reinspection of Catalent Indiana commencing. Look, like since that in-person Type A meeting that we had back in November, all the way through the Type C meeting that we had in early March, we have just been really pleased with the high level of engagement from the agency and sort of the consistent pace and progress across this period of time. So look, what I would note about the reinspection is we were pleased with the timing -- we think the FDA has done their job. We believe that Novo has done their job. And per FDA guidelines, it's really now a 90-day period of time for the FDA headquarters to do their work and make a determination on the classification of the facility. And I think, again, underscoring sort of the 2 paths to approval. I am gratified that our team has made massive amounts of progress with our second fill-finish facility. As noted today, all of the drug that is required for the FDA's review in this BLA at that second facility has been filed. And that product would be available in early Q3. So what you kind of see here, Eric, when we talk about we have to be ready at any time prior to and including September 30, is that let's just do a little bit of math together. The FDA is now in a 90-day period of time to determine classification of Catalent Indiana. We have product that's going to be available in early Q3 from the second fill finish. That sort of brings you to something that is well advanced from the September 30 PDUFA date. And so we just know that we need to be prepared because many times, Class II resubmissions and action can be taken by the agency well ahead of that PDUFA date. And that's really what we mean about it any time prior to. We'll continue to work with the FDA collaboratively, and we continue to be really excited with their level of engagement, again, as I noted from our Type A meeting right through this moment today. And we'll keep you guys apprised on that progress. Operator: [Operator Instructions] And the next question will come from Mani Foroohar with Leerink Partners. Lili Nsongo: This is Lili Nsongo on for Mani. Congratulations on the progress. So now that the reinspection has occurred for the Catalent facility, how much risk -- or maybe I should say, how much confidence do you have in a successful non-experimentated classification for the facility? And how should we think about the capacity split between the 2 facilities in, say, the first year of launch? David Hallal: I didn't get the second part of that question, Lili. On the first part, like as I noted to Eric, we feel like through this process, really since the sole approvability issue with our initial file was the general site inspection that the FDA had at Catalent Indiana. We know that Novo has been working really hard on that site with their initial remediation plan and then subsequently, their follow-up remediation with the FDA. And with a lot of engagement in Q1 with the FDA, as we previously noted, they had an early Q1 meeting that was then followed by a site visit and then subsequently in early Q2, the reinspection. So I think we just need to respect that the FDA has really worked diligently, which we think is a rapid time line given the situation at Catalent Indiana to reinspect that facility. Based on their work, Novo has done their work, and now we want to be respectful of the time that the FDA will now take to make a classification decision. I think importantly, what Akshay and I were noting today is that we have a lot of drug vials from both facilities. And I think if any one of those 2 were to be the basis of the approval, each is going to have plenty of product to launch with. So I think that's great news. I think one thing that maybe isn't lost on us is when you take a 90-day time line for up to a 90-day time line for the FDA to reclassify the Catalent Indiana facility. And then you think about an early Q3 timing of having product available commercially from the second fill-finish, there's definitely an opportunity also that our file could be approved with both fill-finish facilities. And I think that, that was one of the things that Akshay and I wanted to communicate as well. So a lot of optionality here, a lot of good news for patients, a lot of good news for the SMA community. I am really grateful to our internal team at Scholar Rock for doing something pretty remarkable here with our second fill-finish facility, but also grateful to the FDA and Novo for the continued progress at Catalent Indiana, and we will keep you guys apprised at the updates across the board on our application. Lili Nsongo: Great. The second part of my question was about commercial supply capacity split between the 2 facilities, which you also answered. So thank you. Operator: And the next question will come from Tess Romero with JPMorgan. Tessa Romero: I actually wanted to ask a commercial question this morning. Now that Itvisma is fully approved for ages older than 2 years old, how are you thinking about apitegromab being able to be used in combination with that therapy if and when you are approved? David Hallal: Yes. Thank you, Tess. Keith can address how we're thinking about that opportunity. As you noted today, really important information from Cure SMA -- in general, we are prepared to launch apitegromab at any time between now and up to September 30. And I think the incredible work that Akshay has done with our team and engaging the FDA there's going to be a very significant opportunity to serve patients with SMA. So Keith, do you want to comment on really more than anything else, the dynamics in the marketplace and your preparations for launch? Robert Keith Woods: Sure. Thanks for the question, Tess. I guess what I'd say, first of all, is we believe that regardless of the therapy, but any type of a therapy that an SMA patient can potentially benefit from an SMN-targeted therapy, we're agnostic as to which one the treating physician choose because we think that they go hand-in-hand along with our muscle-targeted therapy with apitegromab. Now specifically with Itvisma, in our SAPPHIRE study, we did not study patients that were previously on Zolgensma. As Akshay has noted several times, we are studying them in our OPAL study. And we also shared with you that we do have post Zolgensma patients in our EAP program. So there's some experience out there with it. But as far as being able to utilize apitegromab with it, I think it's going to depend upon the label and where the policies come out with the payers. Operator: And our next question will come from Cory Kasimov with Evercore. Cory Kasimov: I wanted to ask you about the ongoing CHMP review. Coming out of the recent oral explanation, have the questions there have been largely similar to what the FDA has inquired about during its review and now just really boils down to CMC? Or are there other nonmanufacturing items that EU regulators are still trying to get their arms around? David Hallal: Thanks, Cory. Akshay? Akshay Vaishnaw: Yes. Thanks. So obviously, we don't get into the back and forth of regulatory reviews, FDA or EMA. The one thing I can say is that, that oral explanation that was scheduled led to a very good dialogue in advance of the meeting, and we were very happy with the pre-meeting alignment, which led to mutual agreement that there was no need for the meeting. And in fact, as a result, we obviously look forward to continued progress with the review and ultimately to launching the drug in Europe for children and adults with SMA. As to the remaining time line, I commented in the formal remarks that the Catalent Indiana facility continues to support the application, and we look forward to a decision around midyear. But I think overall, the progress has been excellent. David Hallal: And then just tagging on, Cory, just to tag on to Akshay, and Akshay has mentioned this multiple times. We've been having very good open dialogue with the European regulators about what's been happening here with the FDA and Catalent Indiana. So it's been very collaborative, like everything going on here has been a topic of discussion in Europe, and they've really been very flexible in working with us on timing. Operator: And the next question will come from Michael Yee with UBS. Michael Yee: Two questions, really quick. One is a follow-up, just in terms of the fill-finish facility, the second one. Can you remind me -- previously, I recall there was different stability testing and things that had to be completed, but it sounds like this site had sort of been pulled very much forward and was filed earlier, which was fantastic. And so it's the understanding that either of these sites can support approval by September 30, and that's why there's definitely increased confidence and there's not necessarily such a reliance on the Indiana site. And so I have that correct. And the second question is regarding a potential approval and indications. I know previously, there has been some discussion around the broadness of the label, type 1 versus type 2 in different age groups, given the primary endpoint was on a certain age group definition. Can you just remind us about your confidence around general broadness of the label and how we should think about that? David Hallal: Michael, great questions. I'll start on the 2 fill-finish facilities and then Akshay will take up the label. So yes, I mean, I guess at the end of the day, we have an enormous amount of confidence in our BLA as the headline news of what's changed from late last year to this year is really the fact that we have 2 fill-finish facilities in our BLA. One of those, we expect reclassification within a 90-day window from the closeout of the inspection. And subsequently, in that second fill-finish facility, as you aptly noted, we have made massive amounts of progress in accelerating that where all of the drug that is required for the FDA's review and approval has been filed and that drug would be available commercially in early Q3. So when you kind of take that 90-day window, the up to 90-day window per FDA guidelines for the Catalent Indiana facility, when you look at that window of commercial apitegromab being available in early Q3 from the second facility, we are very confident in this window that we're talking about within Q3 and up to the September 30 PDUFA. And I think that what I'm most gratified about is we try to live here at Scholar Rock by a deep commitment to the patients and families that are impacted by SMA. And I'm grateful to the team that we took it upon ourselves to say, okay, let's do better this time than we did last time. Let's not rely on a single fill-finish facility. Let's have multiple paths to get to that point where we can deliver the first ever targeted therapy to patients who are living with this disease and the families that are impacted by this disease, and I think we've been able to do that. And tying -- dovetailing nicely into that is the question that you had on the label and our opportunity to serve a meaningful percentage of the community that is impacted by this disease. And I'll turn it over to Akshay to comment on that. Akshay? Akshay Vaishnaw: Yes. Thanks. Mike, vis-a-vis the label, of course, it's premature to comment on the exact nature of the label before the regulatory deliberations are finalized here in Europe. What I would say is that, generally speaking, the regulators have taken a very important approach to the labels for SMA products. They look at the enrollment criteria of the pivotal studies, which exactly is the population. They look at the portability of the mechanism across the spectrum of disease, and they look at the unmet need. And so I feel like they've been very good with those principles to serve the community. We've been working with them. As you know, when we got the CRL, the draft label was completed. The one outstanding issue was the manufacturing issue. And both in U.S. and Europe, all I can say is we've had constructive regulatory dialogue throughout the period last year and this year, and we look forward to launching this product for children and adults with SMA. David Hallal: And Michael, Akshay and I would just note that as we previously have disclosed that where we were towards the tail end of our last BLA review, we were pleased. And that's where we picked up this new application is exactly where we were at the tail end of the last one on the label, and we look forward to continuing to work with regulators to bring us to the point of approval and delivering apitegromab to the community. Operator: And the next question is going to come from Tazeen Ahmad with Bank of America. Wesley Yon: This is Wesley on for Tazeen. Congrats to the team on all the progress really. I had a question on sort of the game plan going forward now that you have a PDUFA date in hand. So are there any sort of new types of discussions you can have with payers or other like commercial bodies now that apitegromab is officially under review? And is there any sort of new, I guess, strategies or ways that Keith and the commercial team are sort of laying out the groundwork for potential approval? Or is it just kind of just chugging along and doing what's been done already? David Hallal: Well, Wesley, as I think you guys all know Keith very well. You would imagine as disappointed as we were to not launch late last year, we had to look at the opportunity that we had to prepare ourselves to be even better to serve the SMA community. That was our obligation. One such piece of that under Akshay and under Lisa Wyman and team was to make sure that this application was even stronger than the last one, and that's inclusive of now the 2 fill-finish facilities and 2 independent paths to approval. The other obligation that we made is to be better from a commercial perspective. How do you use that time to make sure that you can meet the moment for the SMA community. And I think your question is a good one now with the September 30 PDUFA, but yet being ready for an approval at any time. And with that, I'll hand it over to Keith to talk about the things that he has been doing and what this means for him and the team. Keith? Robert Keith Woods: Yes. Thanks, David. And Wesley, thanks for the question. What I can tell you is that joining the company 4 months prior to the PDUFA date, we were scrambling for that PDUFA date. We would have been able to launch successfully, but we have really been able to take advantage of the additional time that we have. Some specific examples that I've shared in the past, first of all, with payers, we are able to meet with the payers and with our medical team to really discuss apitegromab and the data. So those discussions are ongoing. We've just been able to take them to a much broader range of payers and really deepen the discussions that we have with them specifically around this. Additionally, we built out how our site of care plans will be. I've shared with you before that we now, through our partners, have over 10,000 home infusion nurses available around the U.S. that would be able to provide apitegromab to patients shall they choose to go through home infusion. We've expanded our specialty pharmacy network so that no patient has to go to multiple specialty pharmacies to get their different meds that they may be on for SMA treatment, whether it's their SMN targeted therapy or that apitegromab. And just we continue to really move forward with patient engagement activities. And that's through our program to really have patients demand better treatment for themselves with light takes muscle. And so I can tell you this. I want you to know that the team has been working very hard all the way through this delay, but we are clearly ready to launch now. So whatever that time frame that will be between now and September 30, I want you to know that the team will be ready to be out there the next day, and we will have supply in the channel very rapidly after approval. Operator: And the next question will be coming from Marc Frahm with TD Cowen. Marc Frahm: A lot has been asked already on the PDUFA and apitegromab itself. Maybe just looking at the subcu version. I mean you mentioned you have that data in hand. And once you get the approval for the IV formulation, you'll look to meet with the FDA to discuss it. Just what are the kind of key issues you think you need answers from the FDA on? And kind of what are the range of time lines for when you think you might be able to kind of launch that product depending upon the outcome of those discussions? David Hallal: Thanks, Marc. Akshay? Akshay Vaishnaw: Yes. Thanks, Marc. So I would say there are no issues as such. These things are a matter of just alignment with regulators as to what the optimum path forward to bring another innovation to SMA patients and in this case, it would be subcutaneous apitegromab. The Phase I data were excellent, showing a very good bioavailability and pharmacodynamic overlap between 2 routes of administration. And what we have to do now is to share those data following the approval and align on the path forward in terms of any further development that we needed. So that could be PK/PD data and consideration of any additional safety or efficacy. However, from a safety perspective, obviously, the exposure is maximized with IV apitegromab. And so with the very large database we have in hand already from the studies we've done, we feel very good about safety via additional routes of administration. And so we just want to get on and have those conversations and finalize the path. Once we've done that, obviously, we'll guide you on the time line, premature to speak to that in advance of those conversations. Marc Frahm: Okay. And if I can squeeze in also just on the FORGE trial. Just can you kind of walk through what's different about that trial or maybe the supporting data that apitegromab has been able to generate relative to the efforts that Roche had in FSHD and which ultimately, as of a few weeks ago, they disclosed did not lead to moving into pivotal development. David Hallal: Yes. 3 or 4 points here. Number one, we're obviously very proud of the innovations that have occurred at Scholar Rock with our leading anti-myostatin pipeline. It still remains apitegromab, the only validated anti-myostatin antibody make it through Phase III and delivered the kind of risk and benefit profile that we saw in the Phase III with the SAPPHIRE study in SMA. Whilst we await that approval, obviously, many others are interested in this target. Roche and Chugai are world-leading company. It was sad to see that antibody drop out. We've never really seen any Phase I data or the FLExDUX4 mouse model data from the Chugai-Roche antibody. So we don't quite know the nature of those data, and we await to see how strong they were. We know our data apart from the positive Phase III study, of course, we have very nice data in the FLExDUX4 mouse model with an anti-myostatin approach showing increase in muscle mass and talk and additional function. We know that there are, within FSHD normal fibers that can be boosted by means of an anti-myostatin approach. We know other clinical trials in FSHD that have shown increase in muscle mass and function. So we're very encouraged by our data and our diligence. And finally, we believe the Phase II design is different from the Roche study, specifically the inclusion/exclusion criteria and the severity of the disease that we're enrolling relative to what they enroll, which appears to be quite advanced. And based on our diligence with the experts, we decided because of input from them to go towards the milder end in terms of the Roche scores with patients with established disease where we felt we could still show benefit. And so we remain confident with our validated asset going into that Phase II study and look forward to kicking off very soon. Operator: And the next question will come from Geoff Meacham with Citigroup. Geoffrey Meacham: I had another commercial kind of reimbursement question. Just given the range of options in SMA today, how are you guys thinking about incentivizing switches or maybe deploying a more novel outcomes-based pricing strategy just to help the early stages of the launch? And are the strategies different when you look to the EU and the early launch in Germany versus the U.S. launch? David Hallal: Thanks, Geoff. I think as Keith noted, a cornerstone of our sort of campaign thus far around the disease itself has been an acknowledgment, and we see that the community gets it, that this disease is -- the hallmark is not only the motor neuron, but the resulting muscle atrophy. And so all of this innovation over the last 10 years has been on motor neuron survival and motor neuron health. And this has been needed innovation for the community. And yet, as Keith noted, nearly all patients are wanting their muscle atrophy to be addressed. And this will be the first and only muscle-targeted therapy that's approved. So we don't necessarily really think about switches, Keith, right? We really think about no matter what you choose to do for motor neuron health, we applaud. And we're going to deliver something that addresses the organ that is the principal organ affected by this disease is the muscle. And that's what's been left behind over these 10 years of innovation that we're finally able to address. And putting that into practice, I know, Keith, has been the cornerstone of what you guys have been talking about with the community, and I'll let you take it from here. Robert Keith Woods: Yes. No, Geoff, we're really not going to be focused on any type of switches because what we've shared before is that in our own market research with treating physicians, we know that 3/4 of them have already said that they believe dual modality is the future standard of care for treatment in SMA. So that's directly targeting the motor neuron and directly targeting the muscle -- so we believe that, that will be how this is viewed. And then additionally, from a payer point of view, we did share the data that Cure SMA shared with us in the prepared remarks with roughly 1/3 of patients already receiving more than one SMN targeted therapy. It just continues to drive home the unmet medical need that exists with these SMA patients. But as David just referenced, the principal organ that's impacted in this disease is the muscle, and we look forward to bringing forward the world's first muscle-targeted therapy. Operator: And the next question is going to come from Amy Li with Jefferies Company. Amy Li: David, congrats on all the progress. Just wanted to get a sense of the next steps and time lines for the Catalent site. Based on feedback from the FDA after the reinspection and the Novo closeout meeting, do you expect a Form 483 related to reinspection? And does the speed of your BLA filing acceptance, which was around 30 days compared to the standard 60 days, indicate any FDA urgency or prioritization? And then finally, on the second manufacturing side, I just wanted to clarify, are you maintaining it primarily as a hedge against Catalent? Or is there a potential for approval of both sites? David Hallal: Yes. Thanks, Amy. I'll take that last point first. As we noted when Catalent Indiana was acquired by Novo, we knew that Novo was acquiring that facility really for its own internal purposes, and they would have this transition phase into moving "customers" out because they're not a CDMO. That's not their business model. And so all along, we've recognized that we would want to have and would require to have an additional or more than one fill-finish facilities that are outside of Catalent. So all of that, right, was part of our plan even prior to the Form 483 observations that the FDA had in their general site inspection last year. So I think it's important to note that we see this second fill-finish facility is absolutely vital for all of our global demand. Now we also see Catalent is important. We have drug vial there. We would anticipate that they would be part of our supply chain. And in due time, they're going to phase -- we would phase them out if they're going to be phasing us out. So more than anything else, we see them both as being important. And yet we do think having 2 independent paths to an approval under this BLA is a very significant enhancement to our BLA in 2026 versus the one that we had last year in 2025. And we also think timing is really good. You note the FDA's urgency and how they've been working expeditiously with us. We do think that was really anchored by a very constructive in-person Type A meeting in Q4 that Akshay led with our team down there, and we are just grateful that the FDA has continued to show a sense of urgency and understanding the needs of the community. So more than anything else, we see a world in which apitegromab gets approved with one or the other or both, and we think that, that's a wonderful spot to be in. We'll let the FDA do their work on the review of the second fill-finish facility and the data that has been generated by us on that second fill-finish facility with drugs becoming available in early Q3. And we'll also let the FDA do their work expeditiously and thoroughly on their inspection as well as the inspectors concluded that reinspection recently. So we're excited for what the future brings and more than anything else, I think you guys can see these time lines of the 2 facilities have really come pretty much together. And I think that's a key takeaway to recognize. Operator: And the next question will come from Gary Nachman with Canaccord. Gary Nachman: My congrats as well on all the progress. So David, just to follow-on the last point you were making there. If everything ends up being fine with Catalent with the classification, are you still considering pulling the second fill-finish facility from the BLA to simplify it for the FDA? Or you'll just keep it in there regardless to have that better supply chain, even if it would potentially delay the approval and push it out a little bit? And then just a follow-up. Someone asked before on pricing, but just, I guess, to ask it a little differently. Is there a strategy that would make more sense of launching first in Germany or in the U.S.? Or regardless, it would just be one global price and you're not anticipating any MFN issues. So pricing isn't really a consideration in terms of how you'll stagger the launches? David Hallal: Yes. These are great -- really great questions, Gary. I'll just make one comment and then hand it over to Akshay. When Akshay and I hosted a call, late in Q1 on the resubmission of our BLA, we did actually talk about the alignment that we've had with the FDA, the dialogue that we had with the FDA throughout Q1 about the submission with both fill-finish plants and the optionality that, that really provided us. And so Akshay, do you want to comment on that? And like if there is a meaningful difference in time line, the flexibility that we may or may not have here? Akshay Vaishnaw: Yes. I mean just repeating what you said, I think this has been so important to all the progress that's occurred that there's been very constructive collaborative approach between us and the FDA and indeed with the EMA throughout this whole period. And based on that, we submitted both facilities in the BLA with the full support and alignment. And the most straightforward thing is Catlin Indiana is reclassified, is in compliance, and we can start getting drug out of the pending approval. And the second facility would be withdrawn from the BLA. However, given all the constructive approach that's occurred with the FDA, we'll be guided by them. And in the long run, we clearly want redundancy in the supply chain. And so we look forward to bringing on an additional finish sites. So I think all the options are open for us and the really great position we're in now to serve patients is that by September 30, we're going to be approved by one or the other facility. But in the long run, of course, we'll have established in supply chain. David Hallal: Yes. So Gary, let's just say the FDA has up to 90 days, but they make a decision faster than that. and they still need to review some information on the second fill-finish. As Akshay had even described about a month ago, we would certainly have that flexibility of then just moving that second fill-finish to an sBLA, which was always an option that we had considered as well. So lots of flexibility and optionality there, and it was a very good question. On sequencing and pricing, Keith? Robert Keith Woods: Yes. So first of all, Gary, as I mentioned in the prepared remarks, the team is ready to launch here in the U.S., but also the team is built in Germany. And so if you think about -- is there a preference for one before the other? No. We want to get this across the finish line, both in the U.S. and in Europe. You mentioned how does this overall affect pricing. We go out with our list price here in the U.S. We go out with our list price in Germany and in Europe. Remember, Germany is the only place that we can proactively promote right after EMA approval. And so you're promoting and selling at your list price while you go through the AMNOG process and you go through the reimbursement and establishing that price. So it really wouldn't have an impact. And the bottom line is we're going to be prepared to serve patients in whichever market comes first, and there shouldn't be a substantial impact to our ability to price, negotiate in an overall impact on most favorite nations because we won't be at a point right away that we would even trip the cause of most favorite nations. Operator: And our next question will come from Kripa Devarakonda with Truist. Alexander Xenakis: This is Alex on for Kripa. Congrats on the great news today. We have one about the Roche discontinuation of Emugrobart in FSHD. I wanted to know have you seen any uptick in investigator interest in working with apitegromab for your FSHD trial. David Hallal: Yes. Thanks for that, Alex. So the whole neuromuscular space is very excited about the apitegromab program after in SMA. You're right, the intensity of interest increases. Obviously, everyone is looking forward to the approval in SMA. But the neurology world looks with anticipation towards what a validated anti-myostatin approach like apitegromab can do not just in FSHD, but in a range of diseases. And so we're looking forward to the start of the study, which will be very soon now, Phase II study in FSHD and with additional indications to follow where we'll study this drug. But you're absolutely right. There is plenty of interest and very constructive input as we think about triaging through these indications. Operator: Thank you. This does conclude the question-and-answer session and also concludes today's conference call. 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FDA accepted apitegromab Biologics License Application (BLA) for treatment of children and adults with spinal muscular atrophy (SMA) with September 30, 2026 Prescription Drug User Fee Act (PDUFA) action date Accepted apitegromab BLA includes two fill-finish facilities, Catalent Indiana LLC (part of Novo Nordisk), and a second U.S.-based facility FDA has completed reinspection of Catalent Indiana; classification of facility expected within 90 days following reinspection, in accordance with FDA guidelines Second fill-finish facility on track to have commercial apitegromab supply in early Q3 2026 Scholar Rock is prepared for U.S. apitegromab launch immediately upon FDA approval, which may be granted at any time through September 30, 2026 Cash, cash equivalents, and marketable securities of $480 million as of March 31, 2026; includes an additional $100 million in debt and $98 million in net cash proceeds from the Company’s at-the-market (ATM) program Management to host a conference call today at 8:00 a.m. ET CAMBRIDGE, Mass., May 07, 2026--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of children and adults with spinal muscular atrophy (SMA) and additional rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology to advance musculoskeletal health, today reported financial results for the first quarter ended March 31, 2026, and provided an update on recent company developments. "With the FDA’s acceptance of our apitegromab BLA, we have achieved another critical milestone as we work with urgency to deliver on our mission to bring the world’s first muscle-targeted treatment to the SMA community," said David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock. "We are grateful for the FDA’s continued high level of engagement, and we are pleased that important progress continues to be made at both of our fill-finish facilities. Our U.S. commercial team stands ready to launch apitegromab on or at any time prior to the September 30th PDUFA date." Mr. Hallal continued, "Our balance sheet is strong, our clinical-stage pipeline continues to advance, and we are poised, now more than ever, to usher in the next phase of innovation for patients with SMA." Business Highlights and Upcoming Milestones Apitegromab Apitegromab is an investigational fully human monoclonal antibody designed to inhibit myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle. It is the first and only muscle-targeted therapeutic candidate in SMA to demonstrate a statistically significant and clinically meaningful benefit in a pivotal Phase 3 clinical trial (SAPPHIRE). SMA Program Apitegromab BLA accepted by FDA with September 30, 2026 PDUFA action date. The accepted BLA includes two fill-finish facilities, Catalent Indiana and a second U.S.-based fill-finish facility. FDA completed reinspection of Catalent Indiana. Following the FDA’s acceptance of the apitegromab BLA, the Agency completed reinspection of Catalent Indiana. In accordance with FDA guidelines, classification of the facility is anticipated within 90 days following reinspection. Significant progress continues at second fill-finish facility. Apitegromab commercial supply from this facility is expected to be available early in the third quarter of 2026. Preparations ongoing for U.S. commercial launch. The Commercial team continues to expand its reach and engagement with key stakeholders, including a significant presence at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, which was held March 8 – 11, 2026 in Orlando, FL and at the upcoming 2026 Annual SMA Conference, which is being held June 25 – 28, 2026 in Orlando, FL. The U.S. commercial team is prepared to launch apitegromab immediately upon FDA approval. European Medicines Agency (EMA) regulatory review ongoing. A Committee for Medicinal Products for Human Use (CHMP) opinion for the apitegromab Marketing Authorisation Application (MAA) is anticipated near mid-2026. The Scholar Rock team in Europe continues to engage with key stakeholders on SMA disease awareness and education initiatives, including at the 5th International Scientific Congress on SMA, which was held March 11 – 14, 2026 in Budapest, Hungary. The Company is planning for an apitegromab launch in Europe in the second half of 2026, beginning with Germany. Enrollment progressing in Phase 2 OPAL clinical trial. Patients continue to be enrolled and dosed in the Phase 2 OPAL study (NCT07047144). The trial is evaluating apitegromab in infants and toddlers with SMA under two years of age who have received an approved SMN1-targeted gene therapy or who are receiving ongoing treatment with an approved SMN2-targeted therapy. Subcutaneous apitegromab development continues to progress. Scholar Rock is advancing a subcutaneous formulation of apitegromab intended to provide optionality for patients as a small volume, self- or caregiver-administered anti-myostatin antibody suitable for an autoinjector. A Phase 1 study in healthy volunteers has been completed. Further development activities are ongoing, including anticipated FDA and EMA regulatory engagements following apitegromab approvals. FSHD Program Phase 2 FORGE trial on track for initiation in mid-2026. Scholar Rock is developing apitegromab for the treatment of people with facioscapulohumeral muscular dystrophy (FSHD). FSHD is a rare, progressive neuromuscular disease characterized by muscle atrophy and functional decline, affecting approximately 30,000 individuals across the U.S. and Europe. Initiation of a Phase 2 randomized, double-blind, placebo-controlled trial, called FORGE, is expected in mid-2026. SRK-439 SRK-439 is a novel, investigational, subcutaneously administered myostatin inhibitor that binds to pro- and latent myostatin with high affinity and selectivity (i.e., no GDF11 or Activin A binding). Based on preclinical data, SRK-439 has the potential to potently inhibit myostatin and increase muscle mass. Phase 1 healthy volunteer study ongoing. A Phase 1 study evaluating SRK-439 in healthy volunteers is underway, with topline data expected in the second half of 2026. First Quarter 2026 Financial Results Scholar Rock reported a net loss of $105.5 million, including stock-based compensation of $18.2 million, for the quarter ended March 31, 2026, compared to a net loss of $74.7 million, including stock-based compensation of $13.4 million, for the quarter ended March 31, 2025. Net loss per common share was $0.83 for the quarter ended March 31, 2026, compared to $0.67 per common share for the quarter ended March 31, 2025. The Company did not record any revenue for the quarters ended March 31, 2026 and 2025. Research and development expense was $51.8 million, including $6.5 million in stock-based compensation, for the quarter ended March 31, 2026, compared to $48.7 million, including $4.0 million in stock-based compensation, for the quarter ended March 31, 2025. General and administrative expense was $50.2 million, including $11.7 million in stock-based compensation, for the quarter ended March 31, 2026, compared to $28.4 million, including $9.4 million in stock-based compensation, for the quarter ended March 31, 2025. As of March 31, 2026, Scholar Rock had cash, cash equivalents, and marketable securities of $479.9 million. This reflects a drawdown of $100.0 million from the Company’s debt facility and net cash proceeds of $98.0 million from the Company’s at-the-market (ATM) program. Conference Call Information Scholar Rock will host a conference call and webcast today, Thursday, May 7, at 8:00 a.m. ET to review its first quarter 2026 financial results and discuss recent business updates. To access the live audio webcast, please go to "Events and Presentations" in the Investors section of the Scholar Rock website at http://investors.scholarrock.com. To participate via telephone, please register in advance here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call. A replay of the webcast will be available on the Company’s website for approximately 90 days. About Scholar Rock Scholar Rock is a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for children and adults with spinal muscular atrophy (SMA) and other rare, severe and debilitating neuromuscular diseases. As a global leader in myostatin biology, a field focused on proteins that regulate muscle mass, the biopharmaceutical company is named for the visual resemblance of a scholar rock to protein structures. Our commitment to unlock fundamentally different treatment approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. Scholar Rock works every day to create new possibilities for patients through its highly innovative anti-myostatin program, including opportunities in additional rare neuromuscular diseases. Learn more at ScholarRock.com and follow @ScholarRock on X and on LinkedIn. Scholar Rock® is a registered trademark of Scholar Rock, Inc. Availability of Other Information About Scholar Rock Investors and others should note that we communicate with our investors and the public using our company website www.scholarrock.com, including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on X (formerly known as Twitter) and LinkedIn. The information that we post on our website or on X or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended. Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rock’s future expectations, plans and prospects, including without limitation, Scholar Rock’s expectations regarding its growth, strategy, progress and timing of its clinical trials and development programs for apitegromab, including its subcutaneous formulation, SRK-439 and its preclinical programs, and indication selection and development timing, including the timing of any regulatory submissions, decisions and anticipated approvals, the therapeutic potential, clinical benefits and safety of any product candidates, its ability to address the observations identified in the complete response letter, expectations regarding actions by the FDA after its reinspection of the Catalent Indiana facility; the expected timing and outcome of FDA review of the accepted BLA for apitegromab, including the September 30, 2026 PDUFA action date; expectations regarding the availability and timing of commercial supply of apitegromab from Catalent Indiana and a second U.S.-based fill-finish facility, including expected supply from the second fill-finish facility; expectations regarding commercial launch timing, and the achievement of important milestones, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, the potential of its product candidates and proprietary platform. The use of words such as "may," "might," "could," "will," "should," "expect," "plan," "anticipate," "believe," "estimate," "project," "intend," "future," "potential," or "continue," and other similar expressions are intended to identify such forward-looking statements. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, without limitation, whether preclinical and clinical data, including the results from the Phase 3 SAPPHIRE trial and any results from ongoing or future clinical trials, including the Phase 2 OPAL clinical trial, the Phase 2 FORGE trial and the Phase 1 clinical trial of SRK-439, will be sufficient to support regulatory approval or further development; that preclinical and clinical data, including the results from the Phase 2 or Phase 3 clinical trial of apitegromab, data from any ongoing or future trials of apitegromab or data for SRK-439, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidates; whether the FDA will accept the remediations to the Catalent Indiana fill finish facility in response to the FDA Observations, whether the updated BLA will be sufficient to support regulatory approval, Scholar Rock’s ability to manage expenses or provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline; information provided or decisions made by regulatory authorities; competition from third parties that are developing products for similar uses; Scholar Rock’s ability to obtain, maintain and protect its intellectual property; and Scholar Rock’s dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock’s views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law. View source version on businesswire.com: https://www.businesswire.com/news/home/20260507365694/en/ Contacts Investor Contact Laura Ekas, Ph.D. [email protected] 917-439-0374 Media Contact Molly MacLeod, Ph.D. [email protected] 802-579-5995

CAMBRIDGE, Mass., April 16, 2026--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK) today announced that it will report first quarter 2026 financial results on Thursday, May 7, 2026, before the financial markets open. The Company will host a conference call and webcast with Scholar Rock management at 8:00 a.m. ET. To access the live audio webcast, please go to "Events and Presentations" in the Investors section of the Scholar Rock website at http://investors.scholarrock.com. To participate via telephone, please register in advance here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call. An archived replay of the webcast will be available on the Company’s website for approximately 90 days. About Scholar Rock Scholar Rock is a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for children and adults with spinal muscular atrophy (SMA) and other rare, severe, and debilitating neuromuscular diseases. As a global leader in myostatin biology, a field focused on proteins that regulate muscle mass, the biopharmaceutical company is named for the visual resemblance of a scholar rock to protein structures. Our commitment to unlock fundamentally different treatment approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. Scholar Rock works every day to create new possibilities for patients through its highly innovative anti-myostatin program, including opportunities in additional rare neuromuscular diseases. Learn more at ScholarRock.com and follow @ScholarRock on X and on LinkedIn. Scholar Rock® is a registered trademark of Scholar Rock, Inc. Availability of Other Information About Scholar Rock Investors and others should note that we communicate with our investors and the public using our company website www.scholarrock.com, including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on X (formerly known as Twitter) and LinkedIn. The information that we post on our website or on X (formerly known as Twitter) or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended. View source version on businesswire.com: https://www.businesswire.com/news/home/20260416746634/en/ Contacts Scholar Rock Contacts Investors Laura Ekas, Ph.D. [email protected] 917-439-0374 Media Molly MacLeod, Ph.D. [email protected] 802-579-5995

Apitegromab Biologics License Application (BLA) resubmission and U.S. launch, following FDA approval, are anticipated in 2026 for the treatment of children and adults with spinal muscular atrophy (SMA) FDA completed constructive meeting with Catalent Indiana, LLC (part of Novo Nordisk), with discussion of remediation progress and no additional corrective actions requested by FDA Scholar Rock plans to resubmit BLA upon successful FDA reinspection of Catalent Indiana Apitegromab Marketing Authorisation Application (MAA) review ongoing, with EMA decision anticipated in mid-2026; European launch expected in H2 2026, starting with Germany Secured new debt facility, providing up to $550 million in non-dilutive capital to support commercialization of apitegromab and strategic advancement of key pipeline programs Cash, cash equivalents, and marketable securities of $367.6 million as of December 31, 2025 Management to host conference call today at 8:00 a.m. ET CAMBRIDGE, Mass., March 03, 2026--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to dramatically improving the lives of children and adults with spinal muscular atrophy (SMA) and additional rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology to advance musculoskeletal health, today reported financial results for the fourth quarter and full year ended December 31, 2025, and provided an update on recent company developments. "Our highest priority is to serve children and adults living with SMA by bringing apitegromab through the regulatory review process as quickly as possible," said David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock. "To that end, we are encouraged by the FDA’s continued engagement and shared sense of urgency as Novo Nordisk works expeditiously to remediate its Catalent Indiana facility. We are ready to resubmit our apitegromab BLA following successful reinspection of the site by the FDA." Mr. Hallal continued, "As we prepare to usher in the next phase of innovation for patients with SMA, we continue to strengthen our financial position while aggressively advancing our pipeline and expect 2026 to be a transformative year for Scholar Rock." Business Highlights and Upcoming Milestones Apitegromab Apitegromab is an investigational fully human monoclonal antibody designed to inhibit myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle. It is the first and only muscle-targeted therapeutic candidate in spinal muscular atrophy (SMA) to demonstrate a statistically significant and clinically meaningful benefit in a pivotal Phase 3 clinical trial (SAPPHIRE). SMA Program BLA resubmission and U.S. launch, following approval, expected in 2026. A meeting between FDA and Catalent Indiana occurred early in the first quarter of 2026. The meeting was constructive and included a discussion of Novo Nordisk’s progress remediating the Catalent Indiana facility. No additional corrective actions were requested by FDA. Scholar Rock plans to resubmit the apitegromab BLA following a successful reinspection of the site. U.S. commercial team preparing for launch. The commercial team is expanding its reach and deepening relationships with key stakeholders, including SMA treatment centers and payers. The team’s focus includes educating on the importance of addressing the full motor unit, which consists of the motor neuron and the muscle. European Medicines Agency (EMA) regulatory review ongoing. A decision by EMA on the apitegromab Marketing Authorisation Application (MAA) is expected in mid-2026. The European team continues to engage with key stakeholders on SMA disease awareness and education initiatives. The Company is planning for an apitegromab launch in Europe in the second half of 2026, beginning with Germany. Advancing key activities at second fill-finish facility. Technology transfer continues at a second U.S.-based fill-finish facility to strengthen supply continuity and support future commercial demand. Engineering runs are underway with additional manufacturing runs planned through the second quarter of 2026. Scholar Rock expects to submit a supplemental BLA (sBLA) for this fill-finish facility later in 2026. Phase 2 OPAL clinical trial ongoing. Enrollment and patient dosing continue in the Phase 2 OPAL study (NCT07047144). The trial is designed to evaluate apitegromab in infants and toddlers with SMA under two years of age who have received an approved SMN1-targeted gene therapy or who are receiving ongoing treatment with an approved SMN2-targeted therapy. Development activities for subcutaneous apitegromab progressing. Scholar Rock is advancing a subcutaneous formulation of apitegromab intended to provide optionality for patients as a small volume, self- or caregiver-administered anti-myostatin antibody suitable for an autoinjector. A Phase 1 study in healthy volunteers has been completed, and further development activities are ongoing, including planned FDA and EMA regulatory engagements. FSHD Program Phase 2 FORGE trial on track for initiation in mid-2026. Scholar Rock is developing apitegromab for the treatment of people with facioscapulohumeral muscular dystrophy (FSHD). FSHD is a rare, progressive neuromuscular disease characterized by muscle atrophy and functional decline, affecting approximately 30,000 individuals across the U.S. and Europe. The IND application is cleared, and the Company continues to anticipate the initiation of a Phase 2 randomized, double-blind, placebo-controlled trial, called FORGE, in mid-2026. SRK-439 SRK-439 is a novel, investigational, subcutaneously administered myostatin inhibitor that binds to pro- and latent myostatin with high affinity and selectivity (i.e., no GDF11 or Activin A binding). Based on preclinical data, SRK-439 has the potential to potently inhibit myostatin and increase muscle mass. Dosing continues in Phase 1 healthy volunteer study. A Phase 1 study evaluating SRK-439 in healthy volunteers is underway, with topline data expected in the second half of 2026. Corporate Update Secured new debt facility for up to $550 million in non-dilutive capital from funds managed by Blue Owl Capital (NYSE: OWL). This debt facility is expected to support commercialization of apitegromab and strategic advancement of key pipeline programs. The debt facility matures in February 2032, and consists of the following: $100 million, which became available at closing and was used to retire Scholar Rock’s prior debt facility with Oxford Finance; An additional $100 million to be drawn down in the first quarter of 2026; Up to $150 million available upon FDA approval of apitegromab; and An option for additional incremental facilities of up to $200 million at the mutual consent of Scholar Rock and Blue Owl Capital. Fourth Quarter and Full Year 2025 Financial Results Scholar Rock reported a net loss of $91.0 million, including stock-based compensation of $19.4 million, for the quarter ended December 31, 2025, compared to a net loss of $66.5 million, including stock-based compensation of $9.5 million, for the quarter ended December 31, 2024. Net loss per common share was $0.88 for the quarter ended December 31, 2025, compared to $0.61 per common share for the quarter ended December 31, 2024. For the full year ended December 31, 2025, Scholar Rock reported a net loss of $377.9 million, including stock-based compensation of $75.6 million, compared to a net loss of $246.3 million for the year ended December 31, 2024, including stock-based compensation of $36.6 million. Net loss per common share was $3.29 for the full year ended December 31, 2025, compared to $2.47 per common share for the full year ended December 31, 2024. The Company did not record any revenue for the quarters ended December 31, 2025 and 2024, or for the full years ended December 31, 2025 and 2024. Research and development expense was $46.9 million, including $5.3 million in stock-based compensation, for the quarter ended December 31, 2025, compared to $50.4 million, including $4.0 million in stock-based compensation, for the quarter ended December 31, 2024. For the full year ended December 31, 2025, research and development expense was $208.4 million, including $20.7 million in stock-based compensation, compared to $184.5 million, including $16.0 million in stock-based compensation, for the full year ended December 31, 2024. General and administrative expense was $45.0 million, including $14.1 million in stock-based compensation, for the quarter ended December 31, 2025, compared to $19.0 million, including $5.5 million in stock-based compensation, for the quarter ended December 31, 2024. For the full year ended December 31, 2025, general and administrative expense was $176.2 million, including $54.9 million in stock-based compensation, compared to $67.5 million, including $20.6 million in stock-based compensation, for the full year ended December 31, 2024. As of December 31, 2025, Scholar Rock had cash, cash equivalents, and marketable securities of $367.6 million. This reflects $60.4 million from the exercise of outstanding warrants for the quarter ended December 31, 2025. Conference Call Information Scholar Rock will host a conference call and webcast today, Tuesday, March 3, at 8:00 a.m. ET to review its fourth quarter and full year 2025 financial results and discuss recent business updates. To access the live audio webcast, please go to "Events and Presentations" in the Investors section of the Scholar Rock website at http://investors.scholarrock.com. To participate via telephone, please register in advance here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call. A replay of the webcast will be available on the Company’s website for approximately 90 days. About Scholar Rock Scholar Rock is a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for children and adults with spinal muscular atrophy (SMA) and other rare, severe and debilitating neuromuscular diseases. As a global leader in myostatin biology, a field focused on proteins that regulate muscle mass, the biopharmaceutical company is named for the visual resemblance of a scholar rock to protein structures. Our commitment to unlock fundamentally different treatment approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. Scholar Rock works every day to create new possibilities for patients through its highly innovative anti-myostatin program, including opportunities in additional rare neuromuscular diseases. Learn more at ScholarRock.com and follow @ScholarRock on X and on LinkedIn. Scholar Rock® is a registered trademark of Scholar Rock, Inc. Availability of Other Information About Scholar Rock Investors and others should note that we communicate with our investors and the public using our company website www.scholarrock.com, including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on X (formerly known as Twitter) and LinkedIn. The information that we post on our website or on X (formerly known as Twitter) or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended. Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rock’s future expectations, plans and prospects, including without limitation, Scholar Rock’s expectations regarding its growth, strategy, progress and timing of its clinical trials for apitegromab, SRK-439 and its preclinical programs, and indication selection and development timing, including the timing of any regulatory submissions and anticipated approvals, the therapeutic potential, clinical benefits and safety of any product candidates, its ability to address the observations identified in the complete response letter, expectations regarding resubmission and timing of its BLA for apitegromab upon the successful FDA reinspection of Catalent Indiana, expectations regarding commercial launch timing in the U.S. and in Europe, expectations regarding a new fill finish facility and the achievement of important milestones, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, the potential of its product candidates and proprietary platform. The use of words such as "may," "might," "could," "will," "should," "expect," "plan," "anticipate," "believe," "estimate," "project," "intend," "future," "potential," or "continue," and other similar expressions are intended to identify such forward-looking statements. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, without limitation, whether preclinical and clinical data, including the results from the Phase 3 SAPPHIRE trial, will be sufficient to support regulatory approval, that preclinical and clinical data, including the results from the Phase 2 or Phase 3 clinical trial of apitegromab, or the preclinical data for SRK-439, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidates; whether the FDA will accept the remediations to the Novo Nordisk Bloomington Indiana fill finish facility in response to the FDA Observations, whether Scholar Rock will be able to resubmit its BLA in a timely manner and whether the updated BLA will be sufficient to support regulatory approval, Scholar Rock’s ability to manage expenses or provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline; information provided or decisions made by regulatory authorities; competition from third parties that are developing products for similar uses; Scholar Rock’s ability to obtain, maintain and protect its intellectual property; and Scholar Rock’s dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Annual Report on Form 10-K for the year ended December 31, 2025, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock’s views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law. View source version on businesswire.com: https://www.businesswire.com/news/home/20260303854888/en/ Contacts Investor Contact Laura Ekas, Ph.D. [email protected] 917-439-0374 Media Contact Molly MacLeod, Ph.D. [email protected] 802-579-5995