SRRK

To ask a question during the session, you would need to press star one one on your telephone, and you will then hear an automated message advising your hand is raised. And to withdraw your question, you may press star one one again. This call is being recorded on Thursday 7th May, 2026, i will now turn this call to Laura Ekus, Please go ahead.

Good morning. I am Laura Ekus, Vice President of Investor Relations at Scholar Rock. With me today are David Hallal, Chairman and Chief Executive Officer, Akshay Vaishnaw, President of R&D, Keith Woods, Chief Operating Officer, and Vikas Sinha, Chief Financial Officer. During today's call, David 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 the safe harbor provisions under the Private Securities Litigation Reform Act of 1995.

Any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any future date. I encourage you to go to the Investors and Media section of our website for our most up-to-date SEC statements and filings. With that, I'd like to turn the call over to David. David?

Thank you, Laura, and good morning. Thanks to everyone for joining our Q1 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 thirtieth.

Importantly, the accepted BLA includes two fill-finish facilities, Catalent, Indiana, and a second U.S.-based facility, providing Scholar Rock with two 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 Q1, we made meaningful advancements at Catalent, Indiana and our second fill finish facility. 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 two sites.

As it relates to Catalent, Indiana, we are pleased that following acceptance of our BLA, the FDA completed an unannounced re-inspection 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 one.

Importantly, the team stands ready to launch apitegromab immediately upon approval at any time prior to and including the September thirtieth 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 mid-year.

We are building momentum with launch readiness activities. 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. 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 were pleased to have ended the Q1 of 2026 with $480 million in 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. With that, I'll now turn the call over to Akshay. Akshay?

Thanks, David. Good morning, everybody. We're very pleased with advancements in our world-leading anti-myostatin pipeline during the Q1. 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 two independent paths to apitegromab approval by the PDUFA action date of September 30th.

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 higher 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 re-inspection of Catalent Indiana. Per 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 Q3.

Turning now to Europe, our MAA for apitegromab for treatment of children and adults with SMA continues to progress well through EMA review. Evidence of the progress, we have planned to be with the EMA recently for an oral explanation meeting. 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. We highlighted previously, approval in Europe also requires FDA clearance of the Catalent Indiana facility.

Based on our discussions with the EMA, they're aware of the progress at Catalent Indiana and are comfortable with the review timeline that accounts for the FDA's classification of the site. We continue to be very pleased with how the review is progressing, and we anticipate a CHMP opinion near 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 prioritize 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.

Finally, as shown on slide 11, data from randomized studies in FSHD which suggest 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 two 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-429, 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 and devastating neuromuscular diseases. With that, I'll now turn the call over to Keith to discuss our commercial launch preparations. Keith?

Thanks, Akshay. 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.

Further evidence of the unmet medical need, data shared with us by Cure SMA show that an estimated one-third of people living with SMA in the U.S. have received two 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 Q1, we had a meaningful presence at the Muscular Dystrophy Association meeting in March. During this meeting, our team further engaged with healthcare 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, 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 healthcare 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 one family at a time. With that, I'll turn the call over to Vikas. Vikas?

Thank you, Keith. As we have shared previously, our financial objectives for 2026 remain focused on supporting our commercial build to deliver a strong apitegromab launch, funding R&D activities to advance our pipeline and expand our leadership in the myostatin and muscle space, and continuing to evaluate opportunities to strengthen our balance sheet in a way that supports long-term shareholder value. In keeping with these objectives, I'm pleased to provide our Q1 financial results.

For the Q1, we reported $102 million in operating expenses, which included $80 million in non-cash 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 Q1 with $480 million in 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 Q1. 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?

Thanks, Vikas. 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 for the SMA community, and we look forward to updating you on our continued progress. With that, we'll now open the line for questions. Operator?

Thank you. As a reminder, to ask a question, please press star one one on your telephone and wait for your name to be announced. To withdraw your question, please press star one one again. The first question comes from Eric Schmidt with Cantor Fitzgerald. Your line's open.

Thank you. Congrats on all the progress. Maybe just a couple quick questions on apitegromab approval timelines 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 re-inspection? I guess I'm also curious about the statement that you reiterated a couple 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. Thank you.

Oh, thanks, Eric. I'll take both. Look, we were obviously very pleased today to have announced that the FDA accepted the BLA with 2 fill finish facilities. To be clear, it was a Class II resubmission with a PDUFA action date of September 30th, which is, you know, sort of per protocol for manufacturing-related issues. We anticipated that. Of course, as a reminder, you know, we submitted that BLA in complete alignment with the FDA.

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 the sort of the consistent pace and progress across this, you know, this period of time.

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. 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.

I think, again, underscoring sort of the two 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.

What you kind of see here, Eric, when we talk about, you know, we have to be ready at any time prior to and including September 30th, is that let's just do a little bit of math together. The FDA is now in a 90-day period of time to, you know, 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 30th PDUFA date. 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. That's really what we mean about at any time prior to. We'll continue to work with the FDA collaboratively, and we continue to be, you know, really excited, you know, with their level of engagement, again, as I noted from our Type A meeting right through this moment today. We'll keep you guys apprised on that progress.

Extremely helpful. Thank you.

Thank you. We do ask if you can please limit to one question, and the next question will come from Mani Foroohar with Leerink Partners. Your line is open.

Hi. Good morning. This is Lili Nsongo for Manny. Thank you for taking the question, and congratulations on the progress. 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-MTR indicated classification for the facility? How do we think about the capacity split between the two facility inside the first year launch?

I didn't get the second part of that question, Lili. On the first part, like as I noted, to Eric, you know, we feel like through this process, really since the sole approvability issue, with with our initial file was the general site inspection, that the FDA had at Catalent, Indiana.

We know that Novo's been working, you know, really hard on that site, with their initial remediation plan and then subsequently their follow-up, remediation, with the FDA. 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, and then subsequently in early Q2, the reinspection.

I think we just need to respect that the FDA has really worked diligently, which we think is a rapid timeline given the situation at Catalent, Indiana, to reinspect that facility. They've done their work, Novo's 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 two were to be the basis of the approval, Keith is gonna have plenty of product to launch with. I think that's great news.

I think one thing that maybe isn't lost on us is when you take a 90-day timeline for, you know, by up to a 90-day timeline for the FDA to reclassify the Catalent, Indiana facility, 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. I think that that was one of the things that Akshay and I wanted to communicate as well. 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 with the FDA and Novo for the continued progress at Catalent, Indiana. We will keep you guys apprised at the updates across the board on our application.

Great. Thank you. The second part of my question was about commercial supply capacity split between the two facilities, which you also answered. Thank you.

Thank you, Lily.

Thank you. The next question will come from Tess Romero with JPMorgan. Your line's open.

Hey, guys. Good morning. Thanks so much for taking our question. I actually wanted to ask a commercial question this morning. Now that Evrysdi is fully approved for ages older than two years old, how are you thinking about apitegromab being able to be used in combination with that therapy if and when you are approved? Thank you.

Yeah. Thank you, Tess. You know, Keith can, you know, address how we're thinking about that opportunity as he noted today, you know, really important information from Cure SMA. In general, we are prepared to launch apitegromab at any time between, you know, now and up to September 30th.

I think the incredible work that Akshay has done, with, you know, with our team in engaging the FDA, there's going to be a very significant opportunity to serve patients with SMA. Keith, do you wanna comment on, you know, really more than anything else, the dynamics in the marketplace and your preparations for launch?

Sure. Thanks for the question, Tess. I guess what I'd say first of all is, you know, we believe that regardless of the therapy, but any type of a therapy that an SMA patient can potentially benefit from in 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 Evrysdi, you know, in our SAPPHIRE study, we did not study patients that were previously on ZOLGENSMA.

As Akshay's noted several times, we are studying them in our OPAL study, and we've also shared with you that we do have post-ZOLGENSMA patients in our EAP program. There's some experience out there with it.

As far as being able to utilize apitegromab with it, I think it's gonna depend upon the label and where the policies come out with the payers.

Thank you.

Thank you.

Thank you. Our next question will come from Cory Kasimov with Evercore. Your line's open.

Hey, good morning, guys. Thank you for taking the question. I wanted to ask you about the ongoing CHMP review. Coming out of the recent oral explanation, have the questions there 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 non-manufacturing items the EU regulators are still trying to get their arms around? Thank you.

Thanks, Corey. Akshay?

Yeah, thanks. 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.

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. You know, as to the remaining timeline, I commented in the formal remarks that, the Catalent Indiana facility continues to support, the application, and we look forward to the decision, around mid-year. I think overall the progress has been excellent.

And, and then just tagging on,

Great. Thank you.

Corey, just to tag on to Akshay, and Akshay's 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. It's been very collaborative. Like, everything going on here has, you know, been a topic of discussion in Europe, and they're really been very flexible in working with us on timing.

It's very helpful. Appreciate it.

Thank you. The next question will come from Michael Yee with UBS. Your line is open.

Hey, guys. Thank you. Good morning. 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 testings 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.

Is the understanding that either of these sites can support approval by September 30th, and that's why there's definitely increased confidence, there's not necessarily such a reliance on the Indiana site, I have that correct? 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 and 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? Thank you.

Michael, great questions. I'll start on the, you know, the two fill finish facilities, and then Akshay will take up the label. Yeah, I mean, I guess at the end of the day, you know, we have an enormous amount of confidence in our BLA as the You know, the headline news of what's changed from late last year to this year is really the fact that we have two fill finish facilities in our BLA. One of those we expect reclassification within a 90-day window from the closeout of the inspection.

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.

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 finish facility, we are very confident in this window that we're talking about within Q3 and up to the September 30th PDUFA.

I think that, you know, what I'm most gratified about is we try to live here at Scholar Rock by a deep commitment to the patients and families, you know, that are impacted by SMA. 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 build/finish facility. Let's have multiple paths to get to that point where we can deliver the first-ever muscle-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.

Tying, you know, 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?

Yeah, thanks. Michael Yee, you know, vis-à-vis the label, of course, it's premature to comment on the exact nature of the label before the regulatory deliberations are finalized here and 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 looked at the enrollment criteria of the pivotal studies, which exactly is the population.

They looked at the plausibility of the mechanism across the spectrum of disease, and they looked at the unmet need. I feel like, you know, 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.

Both in the U.S. and Europe, all I can say is we've had constructive regulatory dialogues throughout the period last year, this year, and we look forward to launching this product for children and adults with SMA.

Michael, you know, Akshay, and I would just note that, as we previously had disclosed that, you know, where we were, you know, toward the tail end of our last, you know, BLA review, we were pleased. 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.

Thank you.

Thank you. The next question is going to come from Tazeen Ahmad with Bank of America. Your line's open.

Hi, good morning. This is Wesley on for Tazeen. Congrats to the team on all the progress. I had a question on sort of the game plan going forward now that you have a PDUFA date in hand. Are there any sort of new types of discussions you can have with payers or other, like, commercial bodies now that apitegromab's officially under review? Is there any sort of, you know, new, I guess, strategies or ways that, you know, Keith and the commercial team are sort of laying out the groundwork for potential approval? Or is it just kinda chugging along and doing what's been done already? Thank you.

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 two build/finish facilities and two independent paths to approval.

The other obligation that we made is to be better, you know, from a commercial perspective. How do you use that time to make sure that you can meet the moment for the SMA community?

I think your question is a good one now with the September 30th PDUFA, but yet being ready for an approval at any time. 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?

Yeah. Thanks, David. Wesley, thanks for the question. What I can tell you is that, you know, joining the company 4 months prior to the PDUFA date, we were scrambling for that PDUFA date. We would've been able to launch successfully. We have really been able to take advantage of the additional time that we had. Some specific examples that I've shared in the past, you know, 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.

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, you know, 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. Just we continue to really move forward with patient engagement activities. That's through our program to really have patients demand better treatment for themselves with Life Takes Muscle.

I can tell you this, I want you to know that the team has been working very hard all the way through this delay. We are clearly ready to launch now. Whatever that timeframe that will be between now and September 30th, 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. Thanks.

Got it. Thank you.

Thank you. The next question will be coming from Marc Frahm with TD Cowen. Thanks.

Hey, thanks for taking my questions. A lot's been asked already on the PDUFA and apitegromab itself. Maybe just looking at the sub-Q version. I mean, you mentioned, you know, you have that data in hand, and once you get the approval for the IV formulation, you know, 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? Kind of what are the range of timelines for when you think you might be able to kind of launch that product, depending upon the outcome of those discussions?

Thanks, Marc. Akshay?

Yeah, thanks, Marc. You know, I mean, 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 they have very good bioavailability and pharmacodynamic overlap between the two routes of administration.

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. 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. 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. 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 timelines. Premature to speak to that in advance of those conversations. Thanks.

Okay. Thank you. If I can squeeze in also just on FORGE trial, can you kind of walk through what's different about that trial or maybe the supporting data that apitegromab's been able to generate relative to, you know, the effort that Roche had in FSHD and, you know, which ultimately, as of a few weeks ago, you know, they disclosed did not lead to moving into pivotal development?

Yeah. You know, three or four 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 that's made it through phase II and delivered the kind of risk and benefit profile that we saw in the phase III with the SAPPHIRE study in SMA.

Now, whilst we await that approval, obviously many others are interested in this target. Roche and Chugai are, you know, 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. 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 torque 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 with muscle mass and function.

We're very encouraged by our data and our diligence. Finally, vis-a-vis the phase II design, it 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 enrolled, which appeared to be quite advanced.

Based on our diligence with the experts, we decided, because of input from them, to go towards the milder end in terms of the Ricci scores with patients with established disease where we felt we could still show benefit. You know, we remain confident with our validated asset, going into that phase II study and look forward to kicking off very soon.

Great. Thank you, and congrats on the progress.

Thanks.

Thank you. The next question will come from Geoff Meacham with Citigroup. Your line's open.

Hey, guys. thanks for the question. Morning. I had another commercial kind of reimbursement question. Just given the range of options in SMA, you know, today, how are you guys thinking about incentivizing switches or maybe de-deploying a more novel outcome space pricing strategy just to help, you know, the early stages of the launch? Are the strategies different when you look to the EU and the early launch in Germany versus the U.S. launch? Thank you.

Thanks, Geoff. I think as Keith noted, a cornerstone of our sort of campaign thus far around the disease itself has been, you know, an acknowledgment. We see that the community gets it, that this disease is not only the motor neuron, but the resulting muscle atrophy. You know, all of this innovation over the last 10 years has been on motor neuron survival and motor neuron health.

This has been needed innovation for the community. Yet, as Keith noted, nearly all patients are wanting their muscle atrophy to be addressed. This will be the first and only muscle-targeted therapy that's approved. 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 gonna deliver something that addresses the organ that is, you know, the principal organ affected by this disease is the muscle. That's what's been left behind over these 10 years of innovation that we're finally able to address. Putting that into practice, I know, Keith, that's been the cornerstone of what you guys have been talking about with the community, and I'll let you take it from here.

Yeah. Yeah. No, Geoff, we're really not going to be focused on any type of switches because, you know, what we shared before is that, you know, in our own market research with treating physicians, we know that Q3 of them have already said that they believe dual modality is the future standard of care for treatment in SMA. That's directly targeting the motor neuron and directly targeting the muscle. We believe that that will be how this is viewed.

Additionally, you know, from a payer point of view, we did share the data that Cure SMA shared with us in the prepared remarks. With roughly one-third of patients already receiving, you know, more than one SMN-targeted therapy, it just continues to drive home the unmet medical need that exists with these SMA patients.

As David just referenced, you know, 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.

Thank you. The next question is gonna come from Amy Lee with Jefferies. Your line is open.

Awesome. Thanks so much for taking our question and big congrats on all the progress. Just wanted to get a sense of the next steps and timelines for the Catalent site. Based on feedback from the FDA after the reinspection and the Novo close-out meeting, do you expect a Form 483 related to reinspection? 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?

Finally, on the second manufacturing site, just wanted to clarify, are you maintaining it primarily as a hedge against Catalent, or is there a potential for approval of both sites? Thanks so much.

Yeah. 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, quote-unquote, "customers out" because they're not, they're not a CDMO.

That's not their business model. 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 Indiana. 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.

I think that I think it's important to note that we see this second fill finish facility as absolutely vital for all of our global demand. We also see Catalent Indiana as important. We have drug files there. We would anticipate that they would be part of our supply chain. In due time, they're gonna phase, you know, we would phase them out as they're gonna be phasing us out.

More than anything else, we see them both as being important, and yet we do think having two 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. We also think timing is really good.

You know, you note the FDA's urgency, and how they've been working expeditiously with us. We do think that was really, you know, anchored by a very constructive, in-person Type A meeting in Q4 that Akshay led with our team down there. We are just grateful that the FDA has continued to show, you know, a sense of urgency understanding the needs of the community. More than anything else, we see a world in which upadacitabine gets approved with one or the other or both.

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.

We'll also let the FDA do their work expeditiously and thoroughly on their inspection as well, as the inspectors concluded that re-inspection recently. We're excited for what the future brings. More than anything else, I think you guys can see these timelines of the two facilities have really come, you know, pretty much together. I think that that's a key takeaway to recognize.

Excellent. Thanks so much.

Thank you. The next question will come from Gary Nachman with Canaccord Genuity. Your line's open.

thanks, and my congrats as well on all the progress. 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? 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?

Yeah.

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? You know, pricing isn't really a consideration in terms of how you'll stagger the launches. Thanks.

Yeah. 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. Akshay, do you wanna comment on that? On like if the, if there is a meaningful difference in timeline, the flexibility that we may or may not have here?

Yeah, 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. Based on that, we submitted with both facilities in the BLA with their full support, guidance and alignment.

You know, the most straightforward thing is Catalent Indiana is reclassified, is in compliance, and we can start getting drug out of there pending approval. 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.

In the long run, clearly you want redundancy in the supply chain, we look forward to bringing on an additional fill finish site. I think all the options are open for us. The really great position we're in now to serve patients is that by September 30th, we're gonna be approved by one or the other facility. In the long run, of course, we'll have redundancy in the supply chain.

Yeah. 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, you know, 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. Lots of flexibility and optionality there, and it was a very good question. On sequencing and pricing, Keith?

Yeah. First of all, Gary, as I mentioned in the prepared remarks, the team is ready to launch here in the U.S., also the team is built in Germany. If you think about, you know, is there a preference for one before the other? No. We wanna 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.

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. It really wouldn't have an impact. The bottom line is we're gonna be prepared to serve patients in whichever market comes first. There shouldn't be a substantial impact to our ability to price negotiate and an overall impact on most favored nations because we won't be at a point right away that we would even trip the clause of most favored nations.

Okay. That's very helpful. Thank you.

Thank you. Our next question will come from Srikripa Devarakonda with Truist. Your line's open.

Hi, this is Alex calling for Srikripa. Congrats on the great news today. We had one about Roche discontinuation of emugrobart in FSHD. Wanted to know if you've seen any uptick in investigator interest working apitegromab for your FSHD trial. Thanks.

Yeah, thanks for that, Alex. You know, the whole neuromuscular space is very excited about the apitegromab program after our positive data in SMA. You're right. The intensity of interest has increases. Obviously, everyone's looking forward to the approval in SMA. 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.

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. You're, you're absolutely right. There is plenty of interest and very constructive input as we think about triaging through these indications.

Thanks. Appreciate it.

Thank you. This does conclude the question and answer session and also concludes today's conference call. Thank you for your participation, and you may now disconnect.

Ladies and gentlemen, thank you for standing by. Welcome to the Scholar Rock Holding Corporation Fourth Quarter 2025 Financial Results and Business Update Call. At this time, all participants are in a listen-only mode. After the speakers' presentation, there will be a question-and-answer session. You would then hear an automated message advising your hand is raised, and to withdraw your question, please press 11 again. Please be advised that today's conference is being recorded. I would like now to turn the conference over to Scholar Rock Holding Corporation. Please go ahead.

Good morning. I am Laura Ekas, Vice President of Investor Relations at Scholar Rock Holding Corporation. With me today are David Hallal, Chairman and Chief Executive Officer; Akshay Vaishnaw, President of R&D; R. 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 would like to remind you that during this call, we will be making various statements about Scholar Rock Holding Corporation's expectations, plans, and prospects that constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Any forward-looking statements 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 & Media section of our website for our most up-to-date SEC statements and filings. With that, I would like to turn the call over to David. David?

Thank you, Laura, and good morning. Thanks to everyone for joining our fourth quarter and full year 2025 earnings call. Scholar Rock Holding Corporation 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. Our highest priority is to bring upitigramab to the SMA community as quickly as possible. We remain relentless on behalf of patients, and we are grateful that important progress continues to be made at a steady and rapid pace. Let me briefly summarize the key events that have occurred since our constructive and collaborative in-person Type A meeting in November. First, a week following our Type A meeting, the FDA issued a warning letter to Catalent in Indiana. Next, Novo Nordisk rapidly responded to the FDA by mid-December. Then following Novo's response, FDA reached out prior to the holidays to schedule an early Q1 meeting. That meeting has since taken place, and importantly, at that meeting, the FDA had no additional requests to Novo's remediation plan. And most recently, following the meeting with Novo, we were encouraged that the FDA sent a field team to Catalent, Indiana. At the conclusion of the visit, the FDA once again did not have any additional requests to Novo's remediation plan and stated to Novo that it intends to conduct a site reinspection following routine manufacturing activities, which has since resumed in late February. The cadence of activity since our Type A meeting reflects the shared understanding between us, the FDA, and Novo of the high unmet need in the SMA community and a shared sense of urgency to bring up ipilimumab to children and adults living with SMA as rapidly as possible. We are pleased with FDA's continued level of engagement, and we expect this momentum to continue. Our team is prepared to resubmit the ipilimumab BLA following a successful FDA reinspection of the Catalent, Indiana facility. We are reaffirming our guidance of BLA resubmission and U.S. launch following approval in 2026. Also, I am pleased that progress with a second fill-finish facility is moving quickly to build redundancy into our supply chain. Engineering runs at the facility are now underway, with additional manufacturing runs to follow. We anticipate filing a supplemental BLA for the second filer later this year. As we advance the regulatory process for upitigromab toward approval for patients with SMA in the U.S., our MAA review continues in Europe, and we expect a decision from the European Medicines Agency in mid-2026. With anticipated regulatory approvals in the U.S. and Europe this year, I would like to now turn to our Scholar Rock Holding Corporation commercial launch preparations. In the U.S., our team is deployed in the field and is educating potential prescribers and payers on the unmet need in SMA and the importance of targeting muscle, the principal organ affected in SMA, while also broadening and deepening relationships with the community. In Europe, we are building momentum with launch readiness activities and engaging with the SMA community. We continue to plan for a launch in the second half of the year beginning with Germany. Keith will discuss substantial progress we are making with commercial preparations and our disease awareness initiatives shortly. We know it is not a matter of if but when epitigramab will be approved for children and adults with SMA. We are emboldened by the commitment we have made to the more than 35,000 patients globally living with SMA who have received an SMN-targeted therapy. We are working expeditiously to deliver on our ambition that globally, any patient with SMA who can benefit from abitigromab should have access to opitigromab. This is indeed what we know well and what we do well, and we are confident in the significant opportunity that we have to serve patients with SMA. We are ready now more than ever to usher in the next era of innovation for the SMA community. I would like to now turn to the progress we are making in advancing our world-leading anti-myostatin pipeline. Enrollment and dosing continued in our Phase 2 OVAL study evaluating ipilimumab in infants and toddlers with SMA. Our IND for upitigromab in FSHD is cleared, and we are on track to initiate a robust, randomized, placebo-controlled Phase 2 study later this year. With regards to our subQ formulation of epitromab, we shared the promising results of a Phase 1 study comparing subQ and IV epitogromab in January. We expect to share our clinical and regulatory strategy for the program later this year. And finally, we continue to enroll and dose participants in our Phase 1 study for our highly innovative SRK four thirty nine myostatin inhibitor. We expect to have top-line data from this study in the second half of this year. Turning now to our balance sheet. We were pleased to have added we we are pleased to have ended 2025 with $368,000,000 in cash and cash equivalents. This includes $60,400,000 from the exercise of warrants that were set to expire on December 31. We continue to strengthen our financial position to drive our commercial and R&D priorities. And this morning, we are pleased to announce that we have secured a new debt facility for up to $550,000,000, which Vikas will discuss later in the call. 2026 will be a transformative year for Scholar Rock Holding Corporation. We are ready to resubmit our BLA for epitigramab at any moment. Our U.S. commercial team is working with urgency to prepare the market for the launch of the world's first and only muscle-targeted therapy for children and adults living with SMA. Beyond the U.S., the build-out of our 50-country operating platform is underway in Europe, with other regions and countries to follow. And our highly innovative world-leading anti-myostatin pipeline with epitogromab and SRK-four thirty nine is progressing with strong momentum. The opportunity ahead of us to serve patients with SMA and additional rare and severe neuromuscular diseases is significant. We remain steadfast in our strategy, confident in the determination of our team, and energized by the transformative potential of upitikramap and our broader pipeline. The road ahead is one of purpose, progress, and extraordinary possibility. I will now turn the call over to Akshay for an R&D update. Akshay?

Thank you, David, and good morning, everybody. As David noted, we remain focused on our apritamab BLA registration to bring this important therapy to children and adults with SMA as rapidly as possible. Since being joined by Cure SMA and Novo at our in-person five-day meeting with FDA leadership in November, I have been pleased by the ongoing level of engagement and progress made on the patients. We expect this momentum to continue, and our team is prepared to resubmit the ipilimumab BLA following a successful FDA reinspection of the Kaplan, Indiana facility. I would now like to provide an update on the status of our second drill finish facility, which will strengthen supply continuity and support future commercial demand. As we shared late last year, we are working with a world-class U.S.-based manufacturing facility that has a proven track record of successful FDA and EMA site inspections. Importantly, engineering runs are now underway with additional manufacturing runs planned in Q2, and we continue to expect to submit a supplemental PA BLA with this facility later in 2026. Outside of the U.S., our ipilimumab MAA is progressing through the review process with the EMA, and we continue to anticipate the decision in the middle of this year. Turning to our pipeline, let me start with the Phase 2 OVAL trial evaluating ipilimumab in infants and toddlers under the age two. This trial is enrolling participants who have been treated with an SMN1-targeted gene therapy or who are receiving ongoing treatment with an SNN2-targeted therapy. The study is important for two reasons in particular. First, it is anticipated to expand the impact of the ipilimumab to the full spectrum of patients currently being treated for SMA, as this is the first time we are evaluating the use of opicumab in the organza-treated patients in a clinical trial setting. Second, we believe early intervention with upivimab could support muscle during the critical early development phase, complementing SMN target therapy that aims to preserve motor neuron. By promoting muscle growth on both motor neurons and muscle, muscles are still maturing, apivolumab has a unique opportunity to improve motor outcomes in the youngest patients with SMA. To ensure that no patients are left behind, we continue to enroll patients in this study and dosing long ago. Turning now to our next indication for ipilimumab, parsioscapular humeral muscular dystrophy, or FSHD. FSHD is a rare, devastating neuromuscular disease with significant unmet need. More than thirty thousand patients are diagnosed in the U.S. with Europe alone, and there are no approved therapies. FSHD is caused by dysregulation of DUX4, a protein that can cause muscle damage when inappropriately expressed. Symptoms usually begin in adolescence or early adulthood, with muscle weakness in the face and upper body, but FSHD can impact any muscle in the body. An estimated 20% of patients will become wheelchair dependent. We are prioritizing FSHD as the next indication for ipilimumab for three key reasons. First, there is significant unmet need in this population for a safe and effective therapy. Second, we have preclinical data from the gold-standard FlexFlow four mouse model that provides mechanistic rationale for a cogumab in FSHD. Using this mouse model showed that mystatin inhibition can produce robust increase in muscle mass, significant improvements in muscle force, and consistent gains in endurance after 28 days. Third, there are randomized studies in FSHD that suggest muscle mass can increase in hypercapacities 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 muscle function. These data suggest that the oprimumab as a monotherapy may have the potential bring important benefit to FSHD patients. The FSHD IND is clear, and our next step is to conduct a robust, randomized, double-blind, placebo-controlled Phase 2 study that is expected to enroll 60 patients. The study, or FORGE, is on track to initiate in the middle of this year. We also continue to advance two additional programs in our world-leading anti inflammatory pipeline, a subQ formulation of pipigimod, and s r p four twenty nine. In our s r pipilimumab program, we showed some very exciting data from a Phase 1 study earlier this year. In that study, healthy volunteers received ipilimumab v epiva 100 or 800 mg subQ or 800 mg IV. The data demonstrated that 800 mg subQ resulted in an overlap pharmacodynamic profile with 800 IV. Accordingly, sub qpigramat appears to have favorable bioavailability with the pharmacodynamic profile comparable to IV administration. Additional development activities with subcu efiblimab are underway. We are planning engagements with U.S. and European regulators. Turning after SRP four through nine, we discovered by leveraging our work work leading expertise in targeting mystatin. Four three nine is a subcutaneously administered mystatin inhibitor binding to both pro and latent mystatin with high affinity and selectivity. We recently presented data demonstrating that four twenty nine is 10 times more potent than epinephrine. We have shown in nonhuman primate that four three nine changes in whole body lean map at doses as low as 0.3 mg/kg. We are very excited about this program, and dosing in our Phase 1 healthy volunteer study is well underway. We expect to have top-line data on the study in the second half of this year. In closing, we are executing with focused urgency and bring upivimab to children and adults with SMA whilst in parallel investing with discipline to advance our world-leading anti mastectomy pipeline. The strength of our data and the sustained momentum of our programs underpins our confidence that we can shape the future of treatment for patients living with rare neuromuscular diseases. I will now turn the call over to Keith to discuss our commercial launch preparations. Keith?

Thanks, Akshay, and good morning, everyone. As David noted, our team continues to operate with urgency as we prepare for the launch of opitigramab. Our commercial organization remains focused and disciplined, advancing the critical capabilities required to deliver a seamless launch and support patients from day one. Nearly a decade after the introduction of SMN-targeted therapies, the market continues to grow and now represents nearly $5,000,000,000 in global annual sales. However, while SMN-targeted therapies have brought much needed innovation, muscle strength and motor function remain the top unmet need, with 95% of patients continuing to experience persistent and progressive muscle weakness that limits function and independence. Additionally, three-quarters of neurologists believe multiple modalities are necessary to optimally treat patients with SMA. This data underscores the significant opportunity we have with epitigramab, the world's first muscle-targeted therapy. To this end, our U.S. customer-facing team is active in the field, focused on disease education programs that reinforce a broader understanding of SMA as a disease of the motor unit consisting of both the motor neuron and the muscle, which is the principal organ impacted by the disease. We continue to engage across approximately 140 SMA treatment centers, 2,600 prescribing physicians, and their multidisciplinary care teams throughout the U.S., and our SMA disease education efforts remain a core component of our work in the field. In parallel, we are strengthening and advancing the key elements of our commercial capabilities to ensure launch readiness. We have expanded our specialty pharmacy network to enhance SMA patient and caregiver convenience. SMA patients currently receiving an SMN-targeted therapy from a specialty pharmacy will be able to access epitogromab through that same specialty pharmacy. In addition, through our patient access partners, we have established a home infusion network of more than 10,000 affiliated nurses nationwide. We are also working to ensure we mitigate reimbursement and access bottlenecks. This includes preparations to launch our patient services program, which we have named Scholar Rock Supports. This program is designed to provide comprehensive and individualized support to patients, caregivers, and providers. In addition, we remain focused on patient engagement and community activation. In January, we launched the next phase of our disease awareness campaign, called Life Takes Muscle, aligned with our objective to deepen community awareness of the importance of targeting muscle. And finally, we continue to engage with payers, advancing discussions with national and key regional payers as well as Medicare and Medicaid. At U.S. approval and launch, I look forward to discussing our comprehensive SMA Patient Access Support Program in more detail. While we make substantial progress in preparing for the launch in the U.S., we are also advancing launch readiness across key European markets in anticipation of a mid-2026 EMA decision. In Germany, we have established local leadership, initiated our compassionate use program, and are progressing reimbursement planning to enable rapid access following approval. Across the broader region, we are advancing reimbursement dossiers in multiple countries, strengthening our distributor relationship, and we are building out our EMEA infrastructure to support future commercialization. In closing, we have invested thoughtfully to build the commercial foundation necessary to support a world-class launch, and we believe epitogromab is well positioned to play a central role in the next era of SMA care. Our team is prepared to move quickly upon approval and to deliver on our commitment to the SMA community, one patient, one caregiver, and one family at a time. With that, I will turn the call over to Vikas. Vikas?

Thank you, Keith. Our financial objectives for 2026 remain consistent. We are focused on supporting our commercial build to deliver a strong epididymumab launch, funding R&D activity 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 am pleased to provide our fourth quarter and full year financial results. For the fourth quarter, we reported $91,900,000 in operating expenses, which included $19,400,000 in non-cash stock-based compensation. Excluding stock-based compensation, operating expenses were $72,500,000. For the year ended 2025, we reported $384,600,000 in operating expenses, which included $75,600,000 in non-cash stock-based compensation. Excluding stock-based compensation, operating expenses were $309,000,000 for the year ended 2025. Turning to our balance sheet, we ended 2025 with $368,000,000 in cash and cash equivalents. During the fourth quarter, we strengthened our cash position, adding $60,400,000 from the exercise of a warrant that was set to expire on December 31. We continue to spend on our balance sheet and are pleased to announce today that we secured a new debt facility for up to $550,000,000 with Blue Oak Capital. This debt facility consists of four elements. First, upon closing, $100,000,000 was immediately available to us, which we have used to repay our prior $100,000,000 debt facility with Oxford Finance. Second, an additional $100,000,000 is available to us this quarter, which we expect to draw down by March 31. Then, following FDA approval of apecigumab, we have the option to draw up to $150,000,000 in additional capital. And lastly, we have an option for an additional incremental facility of up to $200,000,000 at the mutual consent of Scholar Rock Holding Corporation and Blue Oak. With that, the facility provides us with additional flexibility as we transition towards a global commercial space company while investing in our pipeline. In addition to the $150,000,000 available from the debt facility upon FDA approval of apritamab, we will look to monetize a priority review voucher to further strengthen our balance. Looking ahead, we continue to operate with a tight financial plan. Our prioritized investments remain focused on our abalizumab commercial launch readiness in the U.S. and Europe, strengthening our supply chain to support the pipeline and commercial demand for our digital map, 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?

Thanks, Vikas. In closing, we remain focused on bringing ofitigramab, the world's first and only muscle-targeted treatment to improve motor function, to children and adults living with SMA as rapidly as possible. We are encouraged by the progress that has been made and by the continued momentum across our regulatory, clinical, and commercial priorities. With a strong foundation, clear strategic priorities, and a world-class team, we are well positioned to make 2026 a transformative year for Scholar Rock Holding Corporation as we continue to work with urgency on behalf of children and adults living with SMA. We look forward to updating you on our continued progress throughout the year, and with that, we will now open the line for questions.

Thank you. Star 11 on your telephone and wait for your name to be announced. To withdraw your question, please press 11 again. We ask you please limit to one question. Our first question is going to come from Eric Thomas Schmidt with Cantor. Your line is open.

Thanks for a very comprehensive update. David, just to put a pin in it, is Novo now ready for reinspection, open for reinspection? And then assuming the reinspection does go, quote, well, what would trigger your resubmission? What do you need to see from that reinspection to be able to push the button on the refiling? Thank you.

Thanks, Eric. So you know, we are gratified really since our Type A meeting in November with the shared sense of urgency and high priority that both FDA and Novo has has has made the remediation of the Catalent Indiana facility, and you got a sense from the call just the drumbeat of progress week after week, month after month. We like the high engagement we continue to see. And given the constructive meeting in early Q1 and then the following sites that that really the gating item now just is a reinspection follows these routine manufacturing activities as Novo moves into full-scale production. As far as you know, our trigger we would look for, obviously, a successful reinspection as you noted, and we are assuming that given the progress that has been made. And that would then trigger. We are at the ready to submit our BLA submission very, very quickly. But it really would be with you know, some level of confidence that it was a successful rate.

And our next question will come from Tazeen Ahmad with Bank of America. Your line is open.

Hi, guys. Good morning. Thanks for taking my question. Not to belabor the point on timing here, but I know you are confident about the ability of Novo to resolve the issue. But in the event that you do have to revert to your backup facility, you have guided to a supplemental filing in the second half of the year. What would happen to timelines if that needed to be the primary filing?

Thanks, Tazeen, very much. As I noted on the call, we are we are gratified in the rapid and steady progress that has been made, you know, between FDA and Novo. And we do think of and the importance of ipilimumab for the SMA community is key driver in this. Not the sole driver, but a key driver in this. I would say that we are pleased with how rapidly we are moving forward with an additional filer, and our assumption is whether or not it were to be a supplemental BLA, which is our plan, or whether or not we had to fall back. We have always looked at that as an back. Important effort on our part no matter what because we cannot control everything in this process. And we do not really believe that that timing would be altered tremendously in terms of if it were not an SBLA. So we thought about it. It is our plan that it will be an SBLA. That is the level of insight, information, and confidence that we have. But, nonetheless, we would be prepared to pivot should need be, on behalf of children and adults living with SMA.

Thank you. And our next question comes from Tessa Thomas Romero with JPMorgan. Your line is open.

Hey, guys. Thanks so much for taking the question this morning. So first one is, can you elaborate on what it meant that the FDA sent a field team? What was the purpose of that? And is that routine? And then the second one, just to loop back on sort of better understanding the next procedural steps post the reinspection and what the timelines could be there, will you get verbal communication or is written documentation what you will see similar to a normal inspection? Thanks.

Yeah. Thanks, Tessa. It is a good question because certainly nothing has been completely ordinary about this process. And I do think what has created some level of extraordinary behavior with kind of a constant drumbeat of progress I think it was really set off by that in-person Type A meeting that we held with FDA and where there really was, with CURE SMA in attendance, with Novo in attendance, there was a shared, you know, sense of urgency to bring opitogramab to patients. And so while I cannot really comment on, you know, what was the overall sort of objective, we do think what it shows is, you know, for just weeks after a really constructive meeting with Novo in early Q1, where there were no new requests by the FDA of Novo into their remediation plan, we think it just continues to show high priority by the FDA to send a field team out to interact with the site and to indicate that, you know, after routine manufacturing activities, which have since recommenced at the facility, they would be in line for a reinspection. So overall, we just feel good about the drumbeat of progress here, and we are quite pleased, and we would expect, given this, you know, sort of rapid and steady pace that we have seen over these last three months, that anything else that follows, the timing of a reinspection, the timing of resubmission, that review, you know, hopefully, it continues to follow sort of this commitment that has been made to rapidly progress the epitogromab file so that we can deliver this drug to children and adults living with SMA. And we will certainly keep you apprised on that progress. Thank you.

Thank you. And our next question comes from Mani Foroohar with Leerink. Your line is open.

Hey, guys. You have Ryan on for Mani. Thanks for taking our question, congrats on the update. Maybe just one sticking with the review. Kind of based off your latest conversations with the FDA, I am curious what your expectations are for a turnaround time following BLA submission to eventual approval. Are there any details still need to be worked out, label, etcetera, with regulators? And then maybe just as a second one on the pipeline, can you talk about the strategy for April? Is this something that you plan to keep in house, look for broader strategic options? Is it best suited in rare neuromuscular diseases, or potential broader application? Thanks.

Thanks, Thanks, Brian. Regarding the timing, again, just to remind, you know, everybody tuning in today, in our CRL that we received last year, the sole approvability issue was the state of compliance at the Catalent, Indiana facility. So we are certainly very focused on working with FDA and Novo on that. As I noted earlier in the call, we would and we are planning, and we are ready to rapidly resubmit our BLA following successful reinspection. And, again, we would just point to without really being able to comment on timing, we would just kind of point to, you know, the evidence of the progress over these last three months and how attentive the FDA has been to remediating this facility and how focused Novo has been to really working with urgency as well, and we will keep you apprised on that timing. Regarding the pipeline at $4.39 auction, Yeah. $4.39, obviously, is a very important, exciting drug. It is a high potency antimyostatin antibody. Appears to us at least in the preclinical work to be about tenfold more potent. So could be a very low volume, small volume, infrequent administration type drug. So I think that creates very interesting and exciting possibilities in the neuromuscular space for us that at least at the current time, we think this is a scholar of the bioreactor, and we have no intentions of harm right there. But we will share further development plans after we get the top-line Phase 1 data rate.

Thank you.

And our next question is going to come from Kripa Devarakonda with Truist. Your line is open.

Hey, guys. Thank you so much for taking my question. Time lines wise, not to be over the point, you expect you continue to expect inspection, BLA resubmission, U.S. launch, everything to happen in 2026. For the launch to be in 2026, can it still happen with the Class 2 submission? Our due diligence suggests this is most likely going to be a Class 2 submission. And in any of your recent conversations with the FDA, was there any hint or for a potential CNPV for epitogromat? Thank you.

I did not get the last part of that, Kripa. Could you say any indication of commissioners—

Commissioner’s priority voucher.

Oh. The national priority voucher. The these are all very good questions, Kripa. And as you might imagine, we have thought about it all. Right? And we, with all of the information that we have and the progress that is made, we were pleased and confident to reaffirm the guidance that we provided today of a 2026 BLA resubmission and U.S. launch upon approval. We would certainly point to sort of this steady FDA prioritization and progress with Novo, you know, over these past weeks and months, and it remains, you know, very steady. And I think, like, we have thought about Class 1 versus Class 2, and what we have seen actually in our own sort of analysis of this, even when Class 2s are sort of granted, oftentimes, the decision is taken up before that six-month timeline. And, again, I am just reminding you that the sole approvability, you know, issue for us has been the status of the Catalan Indiana facility. And, you know, we are pretty we are planning for the resubmission to be happening once we have indication that it was a successful reinspection. So we will keep you apprised at that, but we certainly are, you know, very, very comfortable with the guidance that we have provided. And then regarding, like, the commissioners, sort of, I would just say that we are just staying in close communication with the FDA on all of our different initiatives and just keeping in the forefront the very high priority that exists with the SMA community in the United States to gain access to the world's first and only muscle-targeted treatment. And we look forward to continuing to keep you guys apprised on our regulatory progress there with FDA.

Great. Thank you so much.

Thank you. And our next question will come from Michael Yee with UBS. Your line is open.

Hey, guys. Good morning. I am not going to ask a submission question. Can you talk a little bit about the expectations for the label as it relates to either ambulatory or nonambulatory and with no issues regarding age subgrouping, given that you had what sounds like a very successful review process and only CMC was the outstanding part? How should we think about a broad label? And then a follow-up, assuming approval, for Vakaast, can you just remind us, given that your drug is a weight-based drug, how to think about the comparable pricing relative to other drugs and if models should reflect anything philosophically as it relates to the differences in how the drugs are administered? Thank you.

Thanks, Michael. Akshay, on the label and then, you know, Keith on the weight-based element of the drug and price action.

Yeah. Michael, you know, we were gratified by all the progress made during the original cycle. He had gone to a very advanced stage with the draft label, and the FDA had really worked hard to get to that. So with the cabin issue being the only outstanding issue, we have to split that it. Relatively straightforward to get aligned with the FDA on the final label after a BLA resubmission. Now all of that being said, the details ultimately, that is up to the FDA. But we know from the conversation leading up to the September date that kind of the guiding principles are what—excuse me—what the FDA has shown before in the SMA space, the trial design that supports the approval, that is important. Now if you note that the totality of that package, we have experienced with both nonambulatory and abulatory. We have experience with children two years and older. They have experience in patients on this decline and this in medicine. And so I think that these are important guiding factors. James also previously tended to look at the full applicability or not of the therapy hypothesis and the next of action of the drug to try and maximize getting these drugs in the terrible disease as many patients as possible. Now those are the kind of guiding principle. I think we have to waive the ultimate BLA resubmission and see where we end up. But we have been pleased so far with how straightforward we can get this approach.

Yeah. And then on price, you know, I guess, first of all, it is not really appropriate for us to comment on specifics at this stage. But I do promise you when we have approval and we have our launch call, we will get very specific about the pricing. But, Mike, as you mentioned, because it is weight-based dosing, you are going to see a range. So it is not going to just be one set price for all. But, look, when we think about pricing of lopidogrelimab, we think about three key factors. And it is the rarity and the severity of SMA, it is the progressive nature of the disease, and, you know, in combination with SMN-targeted therapies, our data from both TOPAZ and SAPPHIRE have just demonstrated compelling clinical benefits. So we will get into all of the specifics on pricing on the launch call.

Thank you.

Thank you. And our next question is going to come from Amy Lee with Jefferies.

Hi. Thanks so much for taking our question. So looking ahead to launch, what commercial analogs would you point us to as we think about the initial uptake and launch trajectory? And then maybe another one on subcu api. Do you think approval will require a full clinical study in SMA, a smaller bridging study, or primarily human factor studies? And if you could give us a timeline to market, that would be awesome.

Thanks very much, Amy, and yeah, what I would say is that, you know, for sure, we have been pleased in our engagement with the patient community, the caregiver community, as well as, as Keith noted, neurologists’ appreciation that not only addressing the motor neuron component of the disease, but for the first time, to really be able to address directly the muscle component of the disease, which is a principal organ that is clinically impacted and affected by this disease. We sense that there is a lot of interest in accessing the drug. And that in and of itself could support, like, a very nice uptake at launch. I think what Keith and I have looked at, though, is this is essentially a Q4 week infusion. It will have a miscellaneous J code for some period of time. We know that there are payers, for example, Medicaid, that could be a little sluggish at launch. We recognize payers in and of themselves it is not a matter of if they reimburse, but sometimes it takes time to reimburse. And so we believe robust demand, but we think that will be met with initially some access speed bumps that could impact our launch curve. But overall, the long term that we see for opiticlimab in the U.S. and beyond we feel like is quite significant for us, and we are really looking forward to the eventual approval and then Keith and team launching a pitogram to the SMA community. With respect to your question on subcu and clinical regulatory strategy, I will hand that over to Akshay.

Yeah. Thanks, David. So for subQ berivimab, what we have is very interesting and supportive data that the subQ route is viable, shows excellent bioavailability, and a pharmacodynamic profile. Now we know a lot about afliberumab in terms of PK/PD from our prior work, clearly IV administration. Obviously want to leverage that by saying, you know, this is a drug that is well characterized and studied by different administration. But if we can mimic the appropriate PK/PD, then there is no reason why it cannot be equally safe and effective. Now those are all discussions that we need to have with the FDA. The initial approval of the drug, of course, is very important. But subsequent to that, hope to get aligned with regulators on that approach. So, ultimately, we cannot guide the timelines today, but we are hoping you have progressive regulators. Formulate our final time with them. Discuss the path forward.

Great. Thank you.

Thank you. And our next question will come from Jeff Meacham with Citigroup. Your line is open.

Good morning, guys. This is Jarway on for Jeff. Maybe I was thinking about the second fill finish facility. If you guys were to switch over to that one, would it completely derisk the supply chain from a U.S. and EU launch perspective? And then on the launch, what specific leading indicators of payer and physician readiness are you guys tracking? Maybe if you guys can give some color on that, it would be helpful. Thanks.

Absolutely. I will start with the second vial, and then, Keith, you might need clarification on the second. Yes. Can you repeat the second question, please?

Yeah. Sure. What specific leading indicators are you guys paying attention to to indicate, you know, payer and physician readiness that you are tracking?

Great. So second fill finish. We are really pleased with the progress we have been making. As I mentioned, you know, tech transfer commenced in Q4. Engineering runs are underway, and there are additional manufacturing runs to follow here in the very near term. So we are working urgently. Again, our assumption is this is going to be our second filer. We are going to submit an FBLA. Should we rely on this facility solely, we are confident that we would be derisking as well our U.S. and EU commercial opportunities. So we wanted to be very thoughtful in selecting the right second partner for fill finish, and we are gratified that we have done that. And, also, as I noted, really pleased with the progress that is being made at a very rapid pace. Keith?

Yeah. So first of all, when it comes to the payers, you know, we have been really pleased with the access that our team has been able to get. As I stated in the prepared remarks, to not just the big national payers, but also now regional payers and even some Medicare and Medicaid. While we have had more time, we have been able to have in-depth discussions with them, and our medical team has been able to go through the SAPPHIRE clinical data with them. The bottom line is, just as we have research, just as what has been shared in a lot of the Cure SMA data in some of our own markets, you know, neurologists and patients, they want more, and they need more. And that is why we understand three-quarters of these physicians already believe in multiple modalities to treat this—to treat SMA.

Thank you. And our next question will come from Salvator Caruso with TD Cowen.

Hi. This is Salvator Caruso on behalf of Marc Alan Frahm at TD. Thank you for taking my question. Just one quick question that kind of crossed some Ts and dotted some Is. Regarding the status of the MAA review, will that market also be served by the Novo Catalent Indiana facility? And if so, has the EMA taken any action in response to the FDA inspection findings?

I will start, and then I can hand it over to Akshay. There is a mutual recognition between both FDA and EMA. And so this steady and rapid progress we are making with FDA actually serves us very well for the current MAA review with regulators. And so it is very important that we continue to make this progress forward. As I noted, the continued remediation and eventual, you know, successful reinspection will really support our EMA decision near midyear. And then as I noted, if for some reason we were to rely on the second filer, that would also be very important. But for now, we are very excited with the rapid and steady progress that has been made. Akshay, anything—

Yeah. You covered it, David. I think the other piece that we can close touch with with doing the policy with the—so that is really what it is that is important, and we all await those additional this action by which will obviously not take approvals.

Thank you.

You. The next question will come from Etzer Darout with Barclays. Your line is open.

Great. Thanks for taking the question. Just a couple for me. Has the FDA requested or could they request additional safety data that could extend review of epitogromab? And then on FHSD, just wondered would you be looking at any functional endpoints in the Phase 2 study that you are planning? And could this be a more appropriate indication for SRK nine longer term? Thank you.

Thanks, Etzer. Yeah. It is a great comment, and we can remind you that the BLA resubmission will be a fairly rapid and small resubmission, but there would be an update to sort of our safety database, which was called out in our response letter from the FDA. Akshay can comment on that for FSHD, and then talk about any sort of functional outcome measures.

Okay. Yeah. So we are in line with the FDA. And the budget meeting was useful in many regards, including that and both the which aspect of the safe take place needs to be updated. So that is all agreed to, and so we are ready and prepared with this BLA resubmission. So I do not see any brain issues there, but it is a good question, and, obviously, we should always provide the FDA with a latest safety understanding about which we will do. With respect to the FORGE Phase 2 study in FSHD, the primary endpoint will focus on increasing lead muscle volume measure very sensitive with both imaging techniques. But we will have home state environment treatment, which is a validated approach in FSHD, to understand the functional impact of any potential change in muscle mass. And we look forward, obviously, to those data too.

Thank you. Thank you. And our next question will come from Evan Seigerman with BMO Capital Markets. Your line is open.

Hi. Malcolm Hoffman on for Evan. Thanks for taking our here. Thinking about the financials of the business. I know you mentioned the new debt facility secured with approvals U.S. and Europe coming this year. I just wanted to ask, how are you thinking about expectations for time to profitability, whether you anticipate any additional need for financing ahead of that profitability hinge point. Thanks.

Thanks, Malcolm. Vikas?

Yep. Hi, Malcolm. You know, we have not given forward-looking guidance at all here, but, you know, we will follow most likely the normal rare disease kind of revenue trajectory, which leads you into very similar levels of profitability time frames of two to three years from launch. But, you know, it also depends on how our pipeline progresses during that time, and we will weigh into profitability versus investing into the future. But overall, looking at a fundamental principle of creating long-term shareholder value.

Thanks, Vikas. Thanks, Malcolm.

And our next question comes from Allison Bratzel with Piper Sandler. Your line is open.

Hey, good morning, guys. Thanks for taking the question. Just drilling down on some of the prior discussion around review timing. I know you have talked a lot about FDA's sense of urgency on ipilimumab. I guess, is there good precedent for FDA spending less than six months to review a Class 2 resubmission, and can you just clarify, does your guidance for commercial launch in '26 assume a Class 2 resubmission and the full six-month review? And then separately, just on OPAL, could you talk to what you are seeing on enrollment trends there and just, you know, what that tells you about the underlying awareness of opitigimod in the SMA community. Thanks.

Thanks, Allison. Maybe I will just, you know, point out one example on the Class 2 not taking the full time, and I think it is important that we have been mentioned occasionally here during this current journey, with Regeneron. In a CRL in 2023 at the same facility, Regeneron did have a resubmission. I believe it was a Class 2 resubmission, and yet it was approved within, you know, essentially a sort of a 60-day window. And so but we have more examples than that. I just point to that. It is a little bit relevant given the fact that it was CRL, and it was the same facility. And I think it had to do with some assessment of the facility post an inspection. So I would just point your attention to that.

Yeah. So yeah. Following up on that is what is my treatment for. The—the enrollment's going very well. I mean, I think the first thing say actually is people who get enrollment that very, like, knowledge and appreciation for a muscle-based approach in the patient community and the prescriber community. And Keith has spoken about fact is startlingly high and patients, families, and physicians are waiting the approval of this drug. And consistent with that, the stroke throughout the entire patient. They see the, you know, the possibilities age range and disease severity range. As a community and we have verified by the very nice progress we have had. I am not going to share details today, but, yes, we are seeing a good clip of enrollment and, yeah, as we get later into the year, we will clarify, you know, if the sort of comes into sight. But exactly when we have data and so forth. But are fairly consistent with knowledge of the drug in its potential. It is very good in.

And, Allison, I would just add, as Akshay noted in the prepared remarks, we have a deep commitment to the SMA community, and I am really, really pleased that we are making sure no patients are left behind by opening up this under two study. So we are super excited to be doing this work in the youngest of patients with SMA.

And the next question will come from Kalpit Patel with Wolfe Research. Your line is open.

Hey. This is Dugan on for Kalpit. Previous myostatin inhibitors and FSH increased muscle mass without meaningful functional improvement. You give some color on how eptigramab aims to address this historical hurdle and what clinically meaning functional improvement might be in the planned Phase 2?

Sure. Yeah. So I think you are pointing to either drug that did not have a very clear and well validated mechanism of action and potency safety profile. The earlier generations of adenosinemia have a potency, the selectivity. Of drug that has been in our opinion. More importantly, another one is the another point you raised is the Exelon example. I just suspect. Exelon did a study in FSHD, and they reject low fee in one isolated muscle. Now one cannot expect that to result in global, you know, functional improvement. But we do know separately that globally applied strategies like intense physical therapy, or anabolic agents that increase muscle mass, such as those—mastectomy—or rather growth hormone and testosterone and other similar agents, that those kinds of patients clearly show an increase in muscle mass and also increase in functional capacity. So we incorporated contact myometric testing into the Phase 2 to evaluate changing muscle function. The primary approach, or the primary endpoint, obviously, is to document change in the muscle volume. But we look forward to getting those data, and that is a validated approach to that patient. And we will share the data.

Thanks, Akshay.

Thank you. I am showing no further questions at this time. This will conclude today's conference call. Thank you so much for participating, and you may now disconnect.

Good morning, ladies and gentlemen, and welcome to Scholar Rock's Third Quarter 2025 Conference Call. [Operator Instructions] This call is being recorded on Friday, November 14, 2025. I would now like to turn the conference over to Scholar Rock. Please go ahead.

Good morning. I'm Laura Ekas, Vice President of Investor Relations at Scholar Rock. With me today are David Hallal, Chairman and Chief Executive Officer; Akshay Vaishnaw, President of R&D; Keith Woods, Chief Operating Officer; and Vikas Sinha, Chief Financial Officer. For those of you participating via conference call, the accompanying slides can be accessed in the Events section on the Investors page of our website. During today's call is outlined on Slide 2, 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 the 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 & Media section of our website for our most up-to-date SEC statements and filings. With that, I'd like to turn the call over to David. David?

Thank you, Laura, and good morning. Thanks to everyone for joining our third quarter earnings call today. In April, when I was appointed CEO after 8 years in the Board Chairman role, and on the same day, we brought in Akshay, Keith and Vikas, we were confident that Scholar Rock was positioned to be the next great global biotech powerhouse. We based this on several factors. First, our conviction that the global opportunity with apitegromab in SMA alone offers the potential for many years of sustainable growth that will power our company through the end of this decade and into the next. Second, as world leaders in myostatin biology, our ability to deliver transformative therapies to patients suffering with additional rare, severe and debilitating neuromuscular disorders. And third, leveraging our innovative platform to advance our novel subcutaneously administered myostatin inhibitor, SRK-439. When we joined Scholar Rock, the most significant milestone ahead was the September 22 PDUFA date for apitegromab in SMA, which had been granted priority review. Our BLA was supported by robust data demonstrating apitegromab's efficacy and safety for children and adults living with SMA. Based upon our 188-patient prospective randomized, double-blind, placebo-controlled multinational Phase III trial. This trial showed a statistically significant and clinically meaningful benefit in motor function as measured by the Gold Standard Hammersmith Motor Function Scale for SMA. While we were disappointed to receive a complete response letter on September 22, we were pleased that the strength of our Phase III data was reflected in the FDA's review of our BLA and that the sole approvability issue referenced in the CRL was the status of our third-party fill/finish facility in Bloomington, Indiana, which is owned by Novo Nordisk. We know that it is not a matter of if but when apitegromab will be approved in the U.S. for patients living with SMA. We are emboldened by the commitment we have made to the more than 35,000 patients globally living with SMA who have received an SMN-targeted therapy. We are working expeditiously to deliver on our ambition that globally any patient with SMA who can benefit from apitegromab should have access to apitegromab. And now more than ever, we are confident in the significant opportunity that we have ahead of us to serve the SMA community as we work with the termination to bring this important medicine to children and adults with SMA. This is indeed what we know well and what we do well. I would like to now provide a regulatory update on apitegromab. We had our Type A meeting with the FDA on Wednesday. We are grateful to the agency for their full participation, particularly in the context of a government shutdown. The meeting was in person and included the relevant leaders and decision-makers from the agency, including the neurology division and the Office of Compliance. Our team was joined by Kenneth Hobby, President of Cure SMA and representatives from Novo Nordisk. We were encouraged by the meeting. It was constructive and collaborative. It was clear that there is a shared understanding of the high unmet need for the SMA community and a shared sense of urgency to bring apitegromab to children and adults with this disease. Novo Nordisk detailed the progress they have made in implementing their remediation plan at the Bloomington facility and affirmed that they expect the facility to be ready for reinspection by the end of this year. We discussed the path forward and await the final minutes of the meeting. We will continue to work closely with the FDA and anticipate resubmitting the BLA and U.S. launch following approval of apitegromab for children and adults with SMA in 2026. I'd like to now turn to adding redundancy to our supply chain. When Novo Nordisk purchased the Bloomington site in December of 2024, they plan to internalize the plant for their own products. In light of that, Scholar Rock implemented a plan to add an additional U.S.-based fill/finish facility. Now with the OAI classification, Scholar Rock has accelerated our timelines for an additional vialer. We have selected a world-class commercial facility that has a proven track record and has successfully completed recent site inspections, including with the FDA and EMA. As you know, one of the bottlenecks to rapidly adding a new vialer is securing commercial capacity. This can be a lengthy process. Importantly, we have secured commercial capacity commencing in the first quarter of 2026, and tech transfer is now underway. We anticipate submitting an sBLA for this facility later in 2026. In summary, we will continue to work with urgency to bring this important medicine to the SMA community. We look forward to providing clarity on resubmission timelines as soon as we are able. In addition to the large opportunity we have to serve children and adults with SMA, we continue to strategically advance our pipeline. This includes the Phase II OPAL study progressing apitegromab in a second rare debilitating neuromuscular disorder as well as advancing SRK-439 into the clinic. Akshay will provide additional detail on these activities shortly. Importantly, to reach our ambitions, I am pleased to opportunistically strengthened our balance sheet during the third quarter, and we continue to operate with a tight financial plan. which Vikas will discuss later in the call. This plan is aligned to thoughtful strategic investments to drive long-term value creation. We remain confident in the strength of our strategy, the grid of our team and the transformative potential of apitegromab and our pipeline. The regulatory challenges we face today are temporary, but the opportunities ahead to serve patients are extraordinary. With that, I'll turn the call over to Akshay to provide more detailed update on our R&D progress. Akshay?

Thank you, David, and good morning, everybody. As David noted, we continue to work with urgency to bring apitegromab to children and adults with SMA as quickly as possible. SMA is a rare severe neuromuscular disease resulting in irreversible loss of motor neuron and progressive muscle wasting that diminishes the independence of both children and adults. Apitegromab has the potential to reverse the trajectory of SMA from a loss of motor function to a gain of motor function as demonstrated in the Phase III SAPPHIRE study, underscoring the importance of the potential benefit of this therapeutic. I'd now like to turn to Wednesday's Type A meeting. I was pleased to lead our team at that meeting in Bethesda. As David said, the meeting was in person and included the relevant leaders and decision-makers from the agency, including the neurology division and the Office of Compliance. Our team was joined by Kenneth Hobby, President of Cure SMA and representatives from Novo Nordisk. The meeting was constructive and collaborative. We reviewed the comprehensive data from apitegromab development program, including the Phase II TOPAZ study, which demonstrated that delayed treatment results in suboptimal motor function outcomes. These data underscore the impact of delayed treatment and the urgency to make apitegromab available to the SMA community. At the Type A meeting, it was clear that the CRL we received on September 22 was based solely on the need of the Bloomington facility to be in compliance with CGMP or Current Good Manufacturing Practice regulations. During the meeting, Novo Nordisk, detailed the progress they have made in implementing a robust remediation plan at the Bloomington facility. Novo Nordisk also shared with the FDA that it expects the facility to be ready for the inspection by the end of the year. We remain in close coordination with Novo Nordisk as we await the minutes from the Type A meeting. After Novo's completion of remediation of the Bloomington facility and a site reinspection by the FDA, we anticipate recommission of the BLA and U.S. launch following approval of apitegromab in 2026. As part of our long-term growth plans to serve patients around the world with apitegromab we're also accelerating timelines to bring a second fill/finish facility online. This process requires rigorous validation and regulatory approval to ensure the same quality, safety and efficacy of the drug product. Importantly, we have secured commercial capacity commencing in the first quarter of 2026 and anticipate submitting an sBLA for the second facility later in 2026. Outside of the U.S., we continue to expect a decision from the EMA on our apitegromab Marketing Authorization Application, or MAA, near the middle of next year. Further to our commitment to a broad SMA community, we announced today that we've initiated dosing in our Phase II OPAL trial evaluating apitegromab in infants and toddlers under the age of 2. The trial is enrolling participants who have been treated with an SMN1 targeted gene therapy or who are receiving treatment with an approved SMN2 targeted therapy. It is designed to investigate 2 different doses of apitegromab for a duration of 48 weeks and will assess PK/PD, efficacy and safety. In the OPAL study, early intervention with apitegromab could support muscle during the critical early development phase, complementing SMN targeted therapies that aim to preserve motor neurons. By promoting muscle growth when motor neurons and muscles are still forming, apitegromab has a unique opportunity to improve motor outcomes in young children with SMA. Beyond SMA, we're on track to initiate clinical development activities for apitegromab in a second neuromuscular disorder by year-end. We plan to provide additional information on the disease and the clinical development strategy in early 2026. And finally, we continue to advance our world-leading Anti-myostatin Platform beyond apitegromab. The FDA has cleared the IND for SRK-439, and we're on track to initiate a Phase I study in healthy volunteers before the end of this year. This program is built on the validated approach that delivered apitegromab. Specifically, 439 was designed to be an innovative, subcutaneously administered myostatin inhibitor binding to both pro and latent myostatin with high affinity and selectivity. Based on preclinical data, 439 has the potential to potently inhibit myostatin and increase muscle mass. We expect to have data from the SAD portion of the Phase I study in 2026. In summary, our focus remains on bringing apitegromab to patients and investing with financial discipline to deliver on the promise of our broader pipeline. The strength of our data and the momentum across our programs gives us confidence in the impact we can deliver. Now at this point, I'll turn the call over to Keith to discuss our commercial launch strategy and planning. Keith?

Thanks, Akshay. The SMA community is demanding more. Even with currently available treatments, they need a treatment that directly addresses progressive muscle wasting. Apitegromab demonstrated that ability in our Phase III SAPPHIRE study, and we will be ready to deliver apitegromab to the SMA community upon approval. This is not a matter of if, but when. Our understanding for the demand of apitegromab and our confidence in its potential to address the unmet need for children and adults with SMA continues to strengthen. As we look at SMA globally, nearly a decade following the launch of the first SMN-targeted therapy, the demand for treatment continues to grow. After the first 3 quarters of 2025, annual revenue for current SMA treatments are trending to approximately $5 billion globally with the continued growth of SMN targeted therapies, the need for the world's first muscle-targeted therapy is greater than it has ever been before. Our small, lean and highly experienced U.S. customer-facing team is active in the field and we are using this additional time to enhance our engagement activities and to strengthen our performance against key prelaunch readiness metrics. As a reminder, we are just under 4 months in to our pre-commercial field deployment, whereas most biotech companies typically benefit from a longer runway prior to approval. Nationwide, there are approximately 140 SMA treatment centers and more than 2,600 SMA prescribing physicians. With this additional time, we are working to both broaden and deepen our engagement with these potential prescribing physicians. However, an SMA patient is not just treated by one of these physicians but by a broader cross-specialty SMA treatment team. This team can include physical therapy, pulmonology, orthopedics and more. This additional time is enabling us to better understand the patient journey and the roles of the SMA treatment team in each of these 140 treatment centers and how they influence patient care. Additionally, our market access team is expanding their focus beyond that of national payers to also include top regional payers. This builds on our ambition that any patient with SMA who can benefit from apitegromab should have access to apitegromab. Furthermore, our unwavering commitment to the SMA patient community continues by a partnership at a local and national events and to educate on the importance of targeting muscle. We are deepening our collaboration with the advocacy groups, and we are also building lasting relationships, 1 patient, 1 caregiver, 1 family at a time. In Europe, our efforts continue to drive SMA education and awareness, laying the groundwork to ensure we reach patients efficiently across key markets. Our opportunity to serve patients around the world in SMA is significant. There are an estimated 35,000 people with SMA who have received an SMN-targeted therapy and who could be eligible for treatment with apitegromab. We are making strategic disciplined investments in our launch infrastructure, and we will be ready to execute rapidly once apitegromab is approved. In short, we are ready the strategy is clear. The team is in place and our commitment to the SMA community has never been stronger. Now I will turn the call over to Vikas. Vikas?

Thank you, Keith. Our overarching objectives are to fund our R&D activities to expand our leadership in the myostatin and muscle space to support a strong commercial launch and to extend our runway to meet our eventual timelines for apitegromab approval. In line with these objectives, I'm pleased to provide our third quarter financial results and to discuss our approach to managing our cash runway and investment prioritization moving forward. Turning first to our third quarter results. We ended the third quarter with $369.6 million in cash and cash equivalents. For the quarter, we reported $103 million in operating expenses which includes $18.3 million in noncash stock-based compensation. Excluding stock-based compensation, operating expenses were $85.3 million, which reflects ongoing investments in infrastructure to support apitegromab regulatory approval, commercial readiness and our clinical pipeline. During the third quarter, we strengthened our balance sheet, adding $141.7 million. This cash came from 2 sources. First, we executed our ATM and sold approximately 2.8 million shares, which resulted in net proceeds of $91.7 million. And second, we drew down $50 million from our existing debt facility. As we await apitegromab approval, we continue to operate with a tight financial plan focused on thoughtful capital allocation to advance our clinical pipeline and strategic investments to support commercial readiness. Accordingly, we have adjusted our go-forward operating plan. We have deferred investments across a number of areas, including new hiring, launch expenses that are gated to approval, certain R&D activities, including a third indication for apitegromab and other discretionary spend. Now I'll turn to the 6 prioritized investments we are making. The acceleration of a second fill/finish facility for apitegromab, SMA commercial launch readiness, ONYX apitegromab extension study, the Phase II OPAL study, the second indication for apitegromab and the commencement of SRK-439's clinical development. Turning to our balance sheet. Our current cash balance is $369.6 million, which we expect to be augmented by approximately $60 million in cash from the exercise of outstanding common warrants by year-end. With this, we expect our cash to be sufficient to fund operations into 2027. This cash runway has conservative assumptions and does not reflect any upside from potential sale of apitegromab or a priority review voucher. To further strengthen our balance sheet, we intend to expand our credit facility while preserving our non-dilutive financing options. We will provide further clarity on this as well as our anticipated operating expenses for 2026 during our fourth quarter earnings call. Scholar Rock continues to operate from a position of financial strength with a disciplined approach to capital allocation and a clear focus on supporting our strategic priorities. With that, I'll turn the call back to David. David?

Thanks, Vikas. In closing, Scholar Rock remains focused on near-term execution while building with financial discipline for the future. Our conviction in apitegromab and in our broader strategy is stronger than ever, and we are moving with urgency and purpose to deliver meaningful impact for patients. Our priorities are clear: execute with urgency to bring apitegromab, the world's first and only muscle targeted treatment that improves motor function to children and adults living with SMA as rapidly as possible. Advance apitegromab development activities in the second rare debilitating neuromuscular disease, and that will be followed by additional indications where we can have a transformative impact for patients. We want to progress SRK-439 into the clinic and continue to invest in our future with discipline to support these high-value initiatives. Before I close, I want to share my sincere appreciation for Cure SMA and the SMA patient community. Over these past weeks, I have had the opportunity to meet with many individuals and families living with SMA and the words of support that have been shared with us and with me directly have been tremendously meaningful as we work harder, better and faster to bring this impactful medicine to those who can benefit. With that, we'll now open the line for questions. Operator?

[Operator Instructions] Our first question comes from the line of Mani Foroohar from Leerink Partners.

Congrats on the progress through what's been obviously a choppy period for everyone in the government. I think a couple of quick questions. I know I'm violating the one question rule. One, in terms of thinking about further financing opportunities to top up the tank as necessary, how do you think about debt versus royalty/equity? Like how do you think about relative cost of capital? And what's the most appropriate use once you get to a launch. And then another commercial question. In the early days of launch, it is probable that you will be transitioning from one facility to another. To what extent does that introduce any operational risk going from products from one facility to another? And how can that be addressed ahead of time by you guys now?

Thanks, Mani. Why don't we take the first question first with the cost on the financing options and then I'll come back on the redundancy of supply chain with fill/finish. Vikas?

Yes. Thank you, David. Mani, our first objective here is to bridge the financing until the approval. And the first path to go from the lowest cost of capital is to take additional -- extend our loan facility a little bit more. We are in discussions with that. That will be our first opportunity. Royalty probably comes next. And if it goes too long, and then we have to take a little bit of equity, that will be the last and the most expensive one, which we are trying to avoid at any cost and trying to get it as more non-dilutive first. Does that answer your question?

Yes. So a follow-up. That would imply that, relatively speaking, we should expect you guys to wind down/use ATM much less going forward? Like how does that fit into the strategy?

Yes. Obviously, our first objective is to work with the loan facility and expand upon that. And ATMs are put in place just to take some small augmentation of the capital at an opportunistic view. And we did take it down in the last quarter. Because we are only $50 million loan facility available. We're expanding that loan facility as we discussed. And as soon as we have the new facility in place, will share it with all of you.

And then, Mani, regarding the second vialer, a couple of bottlenecks I've shared with you and others in the past. One of the big ones I highlighted in the call was obviously finding a commercial line that is available, that is -- got the ideal configuration for our vial and our team under the leadership of Lisa Wyman, our Chief Tech and Quality Officer just did an extraordinary job in accelerating our second vialer progress to secure commercial capacity in Q1 and commenced tech transfer in lightning speed since that middle of October timeline when the OAI hit, we thought that, that was really important. Now to get there and to get there quickly, you want to change as little as possible in your second vialer as in your primary vialer, whether or not it's vial configuration, analytical testing, like -- so that actually helps you with speed as well. And those are the things that we'll be focused on. And then the impact in the marketplace really should be almost seamless whether or not we are distributing our apitegromab from the Bloomington facility or the new second vialer, it should really be quite seamless operationally to the marketplace, and we would expect it to be that way.

Our next question comes from the line of Eric Schmidt from Cantor.

Congrats on the progress as well. David and team, for those of us who've kind of been reading the Gory play by play around the Catalent facility in Bloomington, and know some of the prior history and all the past issues. How do you kind of provide confidence that this remediation effort is on good footing and that the inspection will prove positive? And then maybe secondarily, do you expect that to be a Class I or Class II acceptance for the resubmission?

Thanks, Eric. And no doubt, there is a history in the facility. We think the history is really anchored around the quality system, the quality culture and the facility. I think importantly, it largely links back to ownership that did not include sort of the steady hand of Novo Nordisk and their commitment to quality and compliance. And so one of the things that I've been saying often, and certainly, the gory details are gory, right? We got the observations, and we notified you all of those observations back on August 6, our last earnings call, which feels like a lifetime away now. And then we've kind of been riding through the CRL and the OAI. But what we have had a front row seat to is the collaboration with Novo, the commitment from the top of the organization, the changes in the staff that they are making. The integration of the Novo quality system into that facility and then the substantial progress that they've been making on a robust remediation plan, which as they noted to the FDA on Wednesday, they feel like there -- the facility is going to be reinspection ready by the end of this year. We don't think that Novo takes that lightly. We think that they are going through a series of internal exercises to make sure that they are reinspection ready. We would imagine that they'll continue to communicate with the FDA and gather feedback on what might be missing from their remediation plan that they would then need to tweak before any reinspection would take place, but we are surely been pleased with the seriousness and the urgency that from the top of that organization right through that facility, they are taking the remediation plan. Regarding Class I or Class II, I'll turn that over to Akshay for his thoughts as he was presiding over our team in person in Bethesda on Wednesday, and the team just did a fantastic job. Akshay?

Thanks, David. Eric, I just want to reiterate that it was a very constructive and collaborative meeting. And I think the agency, as you might expect, shares, the need for urgency as we all work together to try and get apitegromab to patients. So it's not for us obviously, to second guess and say, will it be Class I or Class II. But we were very heartened by the comments they made and the approach they committed to, to help effect the [indiscernible] to patients. So we need to work with Novo to get their work done. Let's wish them the best get the site reinspected, resubmit the BLA. And I'm confident the agency is going to act with urgency and commitment to this community of patients, which they've always shown when it comes to SMA.

Our next question comes from the line of Tess Romero from JPMorgan.

So to be clear, the BLA that you plan to submit in 2026 for apitegromab will include Catalent as your primary fill/finish and you plan to file the sBLA for the additional fill/finish facility later in 2026 following the potential approval of the BLA. Why is that the right path versus using an additional fill/finish only? And then a follow-up is just on the EMA review. How is that going with respect to manufacturing-related items.

Thanks, Tess. Yes. I mean I think given where we are, given the tone and tenor of the meeting that Akshay presided over this week and again, the progress that Novo has been making which really enabled them to communicate to the FDA that they are on track to be reinspection ready by the end of this year. We just think that, that is the absolute right path for us. We would expect that our BLA would be resubmitted with Catalent as our primary filler. And we would expect a second filler to be added to our file, which was frankly always going to be our plan anyway given the fact that Novo wants that facility for internal purposes. And so that is the path that we are following, obviously, everything that we are doing to accelerate our second vialer is a great insurance policy for us no matter what would happen and I was really gratified by our team's efforts over the course of just the past month with the major progress that they have made to secure commercial capacity at a second vialer and already have tech transfer underway, and of course, we'll be expecting the commencement of our commercial capacity to be leveraged beginning in Q1 of 2026. Regarding the EMA, I'd love to have Akshay comment on that. Obviously, quality and compliance is important to all regulators, including them.

Indeed. And just to review the status of the MAA, the question and answers that go back and forth have proceeded well. So the review continues in exactly the timeframe you'd expect. And as we guided on the formal comments, we expect a decision by the middle of next year. Now vis-a-vis the Catalent manufacturing status and the EMA, there is a mutual recognition procedure. And so there is an interdependency. And I think we obviously agree with that. Though I would point out that everything we're doing for the 2026 resubmission, we, Novo and all our collaborators for the 2026 resubmission of our BLA here in the U.S. launch following approval by the FDA is in line with supporting our MAA. So there's not much more I can say right now, and obviously, we'll keep you updated. But let's stay, on track and as we proceed with the BLA, we hope that supports the EU approval as well.

Our next question comes from the line of Tazeen Ahmad from Bank of America.

Thanks for the detailed update. So can I ask when is the latest that you can have this reinspection for Catalent to be completed and given the green light in order to meet your expectations for a 2026 launch? And then just to play the scenarios for a second. And for whatever reason, the second inspection for Catalent doesn't resolve all issues. How quickly could you pivot to make any application with your second fill/finish? And how would that impact your timelines for 2026. Like would you be able to switch that sBLA filing to a BLA filing and keep the timelines the same as you just mentioned?

Yes. Thanks, Tazeen. It's again, a good question, getting back to what -- or targeting back to Eric's point, there's a history at the facility. It was under prior ownership. They've had a few difficult inspections that have led to Form 483s and in this case, some repeat observations. So I understand and we understand that everybody could share some level of concern and/or skepticism that just getting a reinspection is not the objective. It's a successful reinspection. And we share with Novo Nordisk that, that is the objective. And to really put their own team through not only the remediation plan, but rigorous exercises to be reinspection ready, and we know that they are doing that. Related to your point about what if it doesn't resolve all issues, I think there's 2 ways to look at this Tazeen. Are there still observations in the facility and -- but yet do those observations warrant or not sort of a reclassification of the facility because that's really what we're playing for, a reclassification from OAI to either VAI or NAI. And for that, certainly, that is what the objective is. I think regarding your timeline, I think a reinspection could technically go pretty well into 2026, and we would still be within a frame of our guidance of resubmitting our BLA and then the U.S. launch upon approval. We're obviously pleased that the tone and tenor of our Type A meeting led by Akshay with the FDA with all the key decision makers, all the key groups, I think was constructive. It was collaborative. And there really was a shared understanding of the unmet need and a shared understanding that urgency is necessary to serve a very important patient population. And so we're hoping that all of the steps that would be required that gets us to a reinspection would be done in an expeditious way within the regulatory framework that exists and that Novo will do their part. We don't think that they take lightly indicating to the FDA that they will be reinspection ready by the end of this year. We don't think that, that's a low bar. We think they're holding themselves given the commitment to quality and compliance in the culture of Novo Nordisk, we think they say that with a pretty high hurdle in mind. But back to your question about should like a media right hit that facility. In other words, should the inspection not go well. Then what role would the second vialer play? Well, everything that we've done to accelerate that second vialer would be obviously extremely important for us in terms of should we need to pivot, and it's not an addition of this vial on an sBLA, but it's actually our primary resubmission strategy. There are a number of ways that the FDA, and we expect they've done this in the past. And given the shared urgency would understand some level of potential pathways to expedite adding a second vialer as your first vialer in the form of a BLA. And everything that we're doing to expedite this process, we think, will aid us in case the impact of the inspection is not what we all expect it to be, which is a successful reinspection. And as we continue to work with that second vialer, we can provide further guidance to you as we progress from tech transfer, which is now underway directly into the filling lines that we will be executing in Q1 and Q2, and we'll provide those updates over time.

Our next question comes from the line of Kripa Devarakonda from Truist Securities.

Congratulations on all the progress. Thanks for all the details. So in terms of timelines for resubmission of BLA, I feel like we're all asking the same question, but is the plan to wait for the reinspection and for the OAI to be resolved before you submit the BLA and I understand that Novo has said that they're going to be inspection ready by year-end, but could Novo request an inspection? And finally, would you be able to address whether Novo hired any outside consultant to help with this process?

Yes. Maybe related to the timelines on resubmission. I think that Akshay can comment on our thinking and recognizing that with the collaboration with the FDA and the shared urgency, it's a little dynamic. We don't have our Type A meeting minutes yet, but Akshay can share at least our go-forward plan with respect to that.

Yes. I mean I think base case, Chris, it's safe to say that the reinspection would have to resubmit after that. But as David said, it is a dynamic situation, and we'll do everything possible to resubmit in a fashion to expedite the approval of this drug, which patients need so badly. And we were heartened by the degree of support from the agency during our Type A meeting. So a lot is going to happen in the coming weeks and early part of next year, and we look forward to resubmitting this BLA.

But, for sure, Kripa, given that, again, it's reiterated our sole and primary issue is the classification of this facility and their state of compliance. I think it's a safe assumption that we'd like to see that clear. And be ready to go immediately with a resubmission. That's sort of our go-forward plan at this point.

And I think it's worth adding, David, that resubmission is really compared to the initial BLA resubmission. It's a very different asset. It's a much more contained effort around just the safety update and the CMC aspects of the file. So it's very -- we're ready to file that resubmission at very short notice.

Kripa, related to the -- could Novo request a reinspection. I think in a way, they're signaling that they're ready. We would imagine that Novo and the agency still has some wood to chop. Just how do we feel about the remediation plan? Is there anything left before reinspection needs to be done? I would expect that to be happening, okay? That would be an expectation. But in a way, they put themselves on notice with the FDA that they stand ready to be reinspected toward the end of this year. And we think that, that is really, really important. But as you know, this reinspection will not be announced. It would be like your typical unannounced inspection. And so that you can put yourself on notice and communicate with the Office of Compliance that you're reinspection ready. As they notified at our Type A meeting at the end of this year, but we would expect the FDA to -- when they do reinspect the facility, it would likely be an unannounced inspection. And then your final question about like third parties, I think Novo is really looking very broadly, and they have been working with outside experts and helping them through all of these things, including the remediation plan and the progress with the remediation plan. And we have been pleased with the level of quality and urgency that they are applying to this remediation plan, and we're thankful to them for that.

Our next question comes from the line of Etzer Darout from Barclays.

Just because investors have sort of been circling this September 2026 date in terms of sort of timing for a potential approval, David, maybe if you could help us understand what could -- maybe the FDA minutes unveiled to you on the type of resubmission that you have to make, maybe their timelines around the decision once you have filed -- refiled the BLA?

Yes. Thanks, Etzer. It's a great question. And again, something that I think Akshay wanted to have some robust conversations with the agency on at our Type A meeting. We're obviously not -- we don't have meeting minutes in hand, and we certainly want to allow the agency to do their work. But Akshay can comment on how we're thinking about the resubmission timing and again, whether or not it would be Class I or Class II.

Yes, as far as the minutes are concerned, I think it's always good to get the minutes in hand and reconfirm the impressions that we're conveying to you this morning that they're documented in the minutes of the progress that Novo has made of commitment that everyone is showing to the agency of the matter, Novo's comment about being ready for reinspection and everything we've discussed so far. So we await those minutes, and we're confident that they'll reflect what we're conveying this morning. Now as far as the resubmission is concerned, we just discussed that with the last question. And of course, we'll resubmit as soon as the inspection is done or earlier, if possible. But we'll be guided by the agency through all of that. And we'll also be guided on the review timelines. Now the minutes we get won't spell out the nature of the review timelines for the resubmission. That's not their practice. They await resubmission of the BLA before that's done. But one thing I can tell you is that David emphasized the tone and tenor of the meeting that there was support to act with urgency to get on all parties, including the agency to get this drug to patients as soon as possible.

Our next question comes from the line of Marc Frahm from TD Cowen.

And all the detailed disclosures around this meeting. Maybe in that light, just as you move forward and Novo hopefully is, in fact, in position to be reinspected. Just what do you expect to be able to disclose and kind of on what timeline, particularly given that it isn't even your facility directly, but it is a partner. Will you be able to disclose right when it gets inspected not until maybe some 483s are received? Just what are the disclosure plans there?

Thanks, Marc. I think our disclosure plans will really kind of look in the mirror and focus on us and the things that are material to us that we think are important this year. Obviously, the reinspection timeline when it happens, the outcome of it is really important. So I think we want to be open to sharing the important information with you. Obviously, the way this would work is an inspection takes a week to a couple of weeks. There's generally a closeout meeting. At that closeout meeting there's generally some kind of preliminary assessment when a Form 483, as we've noted in the past, I think, again, it's hard to believe when we first disclosed these Form 483 observations was only last quarter because it feels like for me, it's been a long time. But as you know, a Form 483 usually travels 75% of the time with any inspection. But of course, we wouldn't expect a Form 483 to result in an OAI most of the times. And I think that's what startled all of us. But I think that all along Novo has been approaching this very aggressively. So I think we'll just maintain as we have open lines of communication with you all when we have important information to share, we'll certainly do that. And I think what we've done in the past is even if it wasn't something for us, what we did learn of, let's just say, the classification of the facility as we did just last month in October. We tried to get out in front of that and disclose that and have some dialogue with you all on what that meant. And we'll continue to make a commitment to do the same here as we continue on this journey to an eventual resubmission and U.S. launch upon approval.

Okay. That's helpful. And then maybe just on the idea of waiting for the reinspection to kind of happen in the reclassification before filing. But also in your prior answer is, you noted this would be kind of like an unannounced reinspection once they've communicated that they really are in position to be ready. But kind of a forcing mechanism to that at some level, it could be a submission of a BLA from anyone using this facility. So maybe is there some value of maybe filing ahead to kind of try to force the timeline on the inspection? Or is your expectation that there are just so many other products flowing through this facility that that's kind of going to happen on its own without you guys being the forcing?

Well, Marc, a couple of the things you said are really important. One is that we've heard this too, right? I mean the thing that creates urgency are pending applications. And right now, we don't have a pending application. We have a pending resubmission. At the same time, I would note that we were generally pleased with how constructive and collaborative, the in-person Type A meeting was and that there was this shared understanding of the unmet need and shared urgency. So while we're not on file, I would say a lot of the things, and you're absolutely right on your last point, that there are other pending applications at that site, and that can serve us well. We do think our Type A meeting serves as a really good central point of highlighting while we're not on file, there is real urgency here for a community that is desperately wanting to benefit from the world's first and only muscle-directed therapy. And so we have to continue to work with the agency, be collaborative with them find everyone's right footing on what the right thing to do is, and that is really our go-forward plan. And we think we've built sort of a foundation and framework with the agency frankly, all the way through the initial priority review period up till September 22 and even through Wednesday, a really strong foundation for collaboration for us to work together to resolve this issue.

Yes. Thanks, David. And I just want to reiterate the importance of that collaborative approach. And you mentioned forcing function, so to speak, by resubmitting ahead of the reinspection. I don't know that that's wise. We want to be working closely with the FDA and be guided by them now. as we said, it's a dynamic situation. And if they invite us or they support any kind of resubmission in a particular timeframe in and around the reinspection, we will, of course, we are ready and we can resubmit very efficiently. But this is not about a forcing function. We have to collaborate with the FDA.

Our next question comes from the line of Evan Seigerman from BMO Capital Markets.

With the delays for apitegromab, can you talk more about what your sales or market research team's efforts are to identify patients ahead of the launch? You had mentioned efforts to work with centers of excellence to understand the patient journey better. But do you feel you are developing a more robust number of patients which you could target for therapy following approval, potentially leading to a little bit of a faster uptake than people were probably initially expecting?

Yes. It's a great question. Keith was highlighting it earlier in the call, and I'll turn it over to him for further comments on launch prep.

Yes. Thanks for the question. I guess I'd say first of all, when the September 22 date occurred, that was after 2 months of the team being able to spend time out in the field. And now with the extended time, we're getting to not just visit with the physicians, but we're also getting to meet the SMA treatment teams and getting that full feel. And what is that additionally, not just the SMA treatment teams, we're spending more time with patient advocacy events and getting to speak with patients and their families. And I can tell you what we're hearing is that there's a clear understanding of the unmet medical need and the approach of attacking this disease from a dual modality no longer just the motor neuron but also directly targeting the muscle, and this is being well accepted as we get the opportunity to meet with more people in the community. You add to the fact of the safety profile that apitegromab has demonstrated. And quite frankly, all of our studies, not just our SMA studies, but if you take a look at EMBRAZE, that was also all adults all treated with 10-milligram per kilogram and exceptional safety results. I guess I would end with the fact that at the end of the day, we're offering the world's first muscle-targeted therapy, and in the event, if you have a choice to either be in a situation of having experienced muscle loss or the potential for muscle gain, why wouldn't you want to use apitegromab.

Thank you. This concludes today's conference call. Thank you for participating. You may now disconnect.