Scholar Rock Holding Corporation

Good morning, ladies and gentlemen, and welcome to Scholar Rock's first quarter 2026 conference call. At this time, all lines are in a listen-only mode. Following the presentation, we will conduct a question and answer session. To ask a question during the session, you would need to press star 11 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 11 again. This call is being recorded on Thursday, May 7th, 2026. I would now like to turn the conference over to Scholarock. Please go ahead.

Good morning. I am Laura Ikus, Vice President of Investor Relations at Scholarock. With me today are David Hillel, Chairman and Chief Executive Officer, Akshay Vashna, President of R&D, Keith Woods, Chief Operating Officer, and Vikas Binha, 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.

Thank you, Laura, and good morning. Thanks to everyone for joining our first quarter earnings call. Scholarock 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 epidegromab for the treatment of children and adults living with SMA. The agency has assigned a PDUFA action date of September 30th. Importantly, the accepted BLA includes two fill-finish facilities, Catalan, Indiana, and a second U.S.-based facility, providing Scholarock with two independent paths to a PitagorMap approval. As a reminder, The sole approvability issue for epidechromab noted in the complete response letter last September was related to observations identified during a routine general site inspection of the Catalan Indiana fill finish facility, which is owned and operated by Novo Nordisk. Since our in-person type A meeting with the FDA in Q4, We have continued to work constructively and collaboratively with the agency, and we have made steady and rapid progress. During the first quarter, we made meaningful advancements at Catalan Indiana and our second fill finish facility. And with our ongoing open communication with the agency, we resubmitted our Epidogram App VLA 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 a Pinnacle Map to children and adults in the US 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 cattle in Indiana, we are pleased that Following acceptance of our BLA, the FDA completed an unannounced reinspection of the facility. This timing was in line with our expectations, as the FDA had noted, following multiple engagements with Novo and 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 epitigromab drug product required for FDA review and potential approval has been vial. From a commercial supply standpoint, we are well positioned as we expect to have ample commercial epidegromab available from the second facility in early Q3, well ahead of the September PDUPA 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 critical activities and capabilities required to deliver a seamless launch and support patients from day one. Importantly, the team stands ready to launch Epidicromab immediately upon approval at any time prior to and including the September 30th Paducah 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 CHMT opinion near mid-year. We are building momentum with launch readiness activities, and we continue to anticipate a launch in the second half of the year, beginning with Germany. We know it is not a matter of if but when. Epidermab will be approved for children and adults with SMA, and he 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 oval study, evaluating epitigromab in infants and toddlers with SMA, the anticipated initiation of our randomized Phase II study in patients with FSHD, and progress with subcutaneous epitigromab and a novel high-potency anti-myostatin antibody, SRK439, currently in Phase I. Ache will discuss these programs in greater detail shortly. Turning now to the balance sheet, we are pleased to have ended the first quarter of 2026 with $480 million in cash, cash equivalents, and marketable securities. This cash balance includes the drawdown of an additional $100 million from our debt facility, which we took in March. Our cash balance also reflects net cash proceeds of $98 million from our ATM program during the quarter. Because we'll provide more details later in the call. We are building on a solid foundation for our company's growth, which we believe will be steady and consistent through the end of this decade and well into the next as we prepare to serve up to 35,000 children and adults living with SMA around the world who have received at least one SMN-targeted therapy. Beginning with SMA, we are excited to be shaping the future of treatment for patients living with rare and devastating neuromuscular diseases. And with that, I'll now turn the call over to Akshay. Akshay?

Thanks, David, and good morning, everybody. We're very pleased with advancements in our world-leading anti-myostatin pipeline during the first quarter. Turning first to EpidogramMap for children and adults with SMA, we're delighted to share that the FDA has accepted the EpidogramMap BLA. As a reminder, the BLA was resubmitted in alignment with the agency to include both Kaplan, Indiana and a second U.S.-based fill-finish facility. The approach provides ScholarOp with two independent paths to EpidogramMap approval by the Purdue for 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 high unmet need in the SMA community, and we now look forward to the final steps in the U.S. regulatory process. Reflecting the agency's vigorous efforts We were pleased most recently with the timing of the FDA's unannounced re-inspection of Kaplan, Indiana. For FDA guidelines, the agency now has up to 90 days to classify the status of the facility. I'd now like to turn to our second full finished facility, where we continue to make meaningful progress. As David noted, the Epidogramab drug product required for FDA data review and potential approval has been vile. and we expect to have ample commercial epigramab from the facility in early Q3 ahead of the September PDUFA Day. Based on the significant progress at both facilities, we anticipate approval of epigramab for children and adults with SMA, which could be supported by either or both facilities by the end of the third quarter. Turning now to Europe, our MAA for epigramab for treatment of children and adults with SMA continues to progress well through EMA review. As evidence of the progress, we had planned to be with the EMA recently for an oral explanation meeting. However, because we and the EMA were able to align prior to the scheduled meeting, we mutually agreed that the oral explanation was no longer necessary. As we highlighted previously, approval in Europe also requires FDA clearance to the Kaplan Indiana facility. Based on our discussions with EMA, They're aware of the progress at Catlin, 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 epigromab in infants and toddlers under the age of two. As a reminder, this trial is enrolling participants who have been treated with an FMN1-targeted gene therapy or who are receiving ongoing treatment with an FMN2-targeted therapy. This study is important because it is anticipated to expand the impact of epirgumab to the full spectrum of patients, including those treated with dolgensma. In addition, we believe early intervention with epirgumab 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 epidermal MAP, fascioscapular humeral muscular dystrophy, or FSHD. FSHD is a rare, devastating neuromuscular disease with significant unmet needs. More than 30,000 patients are diagnosed in the U.S. and Europe alone, and there are no approved therapies. we prioritized FSHD as the next indication for epigrammat for three key reasons. First, the significant unmet need. Second, the compelling preclinical data from the gold standard FlexDux4 mouse model that provides mechanistic rationale for epigrammat in FSHD. And finally, as shown on slide 11, data from randomized studies in FSHD, which suggests muscle mass can increase and has the capacity to show functional benefits. 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 epigramab 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 two 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 antimicrobial pipeline, a subcutaneous formulation of epitogramab and SRK439. In our subcutaneous epitogramab program, we showed some very exciting data from a Phase I study in January, which demonstrated that subcutaneous epitogramab 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 epinephrine MAP. Turning now to FRP 4-9, our high-potency, high-affinity subcutaneously administered myosin inhibitor, which we discovered by leveraging our world leading expertise. We're very excited about this program and dosing in our phase one 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 epidurumab to children and adults with SMA whilst in parallel working to maximize our impact for patients with epidurumab and our world leading antimicrobial pipeline across a range of rare, devastating neuromuscular diseases. With that, I'll now turn it over to Keith to discuss our commercial launch preparations. Keith.

Thanks, Akshay, and good morning, everyone. With the BLA accepted by the FDA, our team continues to operate with urgency as we prepare for the launch of epitogrammed immediately upon approval, which may be granted at any time through September 30th, 2026. Nearly a decade after the introduction of SMN targeted therapies muscle strength and motor function remain the top unmet need, with 95% of patients continuing to experience persistent and progressive muscle atrophy. That limits function and independence. As further evidence of the unmet medical need, data shared with us by CURE SMA show that an estimated 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 epigramet, the world's first muscle-targeted therapy. Our US 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 US, we have achieved significant reach across the approximately 140 SMA treatment centers, 2,600 prescribing physicians, and their multidisciplinary care teams. Through these engagements, our field team is working to establish case flows on a center-by-center basis to ensure we are well-positioned to support the SMA treatment centers once a treatment decision is made. This includes preparations to launch our patient services program, Scholar Rock Supports. This program is designed to provide comprehensive and individualized support to patients, caregivers, and providers. In the first quarter, we had a meaningful presence at the Muscular Dystrophy Association meeting in March. During this meeting, our team further engaged with 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, and we continue to have numerous in-person patient and patient advocacy group engagements. Turning to U.S. reimbursement, our market access team is advancing discussions with national and key regional payers, as well as Medicare and Medicaid. With this extra time, we've been able to go deeper and broader across the range of payers. We are ready and well positioned for a successful launch of Epitagromab 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 Epitagromab 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 epidegromab 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 apodigomab launch, funding R&D activities to advance our pipeline and expand our leadership in the myostatin and muscle states. and continuing to evaluate opportunities to send in our balance sheet in a way that supports long-term shareholder value. In keeping with these objectives, I'm pleased to provide our first quarter financial results. For the first quarter, we reported 102 million in operating expenses, which included 80 million in non-cash stock-based compensation. Excluding stock-based compensation operating expenses worth $84 million. Turning to our balance sheet, we are really pleased to have ended the first quarter with $480 million in cash, cash equivalent, 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 receipts of $98 million from our ATM program during the first quarter. Looking ahead, upon FDA approval of Epidromab, we will have an option to draw down an additional $150 million from our existing debt facility, and we plan to monetize a private 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 Apidigomap 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 Apidigomap over time, and advancing our highly innovative clinical programs that Akshay discussed earlier in the course. With that, I will turn the call back to David. David?

Thanks, Vikas. Scholarock 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. And with that, we'll now open the line for questions. Operator?

Thank you. As a reminder to ask a question, please press star 11 on your telephone and wait for your name to be announced. And to withdraw your question, please press star 1-1 again. And the first question comes from Eric Schmidt with Candler. Your line's open.

Thank you, and congrats on all the progress. Maybe just a couple quick questions on epitagromab approval timelines in the U.S. Team, I know it's not your facility, the Catalan facility, but are you aware of any field notes that were provided to NOVO following the reinspection and then I guess I'm also curious about the statement that you reiterated a couple 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.

Thanks, Eric. I'll take both. Look, we were obviously very pleased today to have announced that the FDA accepted the VLA with two fill finish facilities. And to be clear, it was a class two resubmission with a PDUPA action date of September 30th, which is sort of per protocol for manufacturing-related issues. So we anticipated that. And of course, as a reminder, we submitted that VLA in complete alignment with the FDA ahead of the reinspection of cattle in Indiana commencing. Look, 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 consistent pace and progress across this period of time. What I would note about the re-inspection is we were pleased with the timing. We think the FDA has done their job. We believe that Novo has done their job. And per FDA guidelines, it's really now a 90-day period of time for the FDA headquarters to do their work and make a determination on the classification of the facility. And I think, again, underscoring sort of the 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 VLA at that second facility has been vial. And that product would be available in early Q3. So 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 determine classification of cattle in Indiana. We have product that's going to be available in early Q3 from the second till finish. That sort of brings you to something that is well-advanced from the September 30th CEDUFA date. And so we just know that we need to be prepared because many times Class II resubmissions and action can be taken by the agency well ahead of that to do today. And 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. And we'll keep you guys apprised on that progress.

Extremely helpful. Thank you.

Thank you. And we do ask if you can please limit to one question. And the next question will come from Manny. Foroja with Lyrinc Partners. Your line is open.

Hi, good morning. This is Lyrinc. Thank you for taking the question and congratulations on the progress. So now that the inspection has occurred for the Catalan facility, how much risk or maybe I should say how much confidence do you have in a successful non-action-indicated classification for the facility? And how should we think about the capacity slip between the two facilities in, say, the first year of launch?

I didn't get the second part of that question, Lily. 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 Catalan, Indiana. We know that NOVA has been working really hard on that site with their initial remediation plan and then subsequently their follow-up remediation with the FDA. And with a lot of engagement in Q1 with the FDA, as we previously noted, they had an early Q1 meeting that was then followed by a site visit and then subsequently in early Q2, the re-inspection. So I think we just need to respect that the FDA has really worked diligently, which we think is a rapid timeline given the situation of cattle in Indiana to re-inspect that facility. They've done their work, NOVA'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 going to have plenty of product to launch with. So I think that's great news. I think one thing that maybe isn't lost on us is when you take a 90-day timeline for, you know, up to a 90-day timeline for the FDA to reclassify the Catalan Indiana facility, and then you think about an early Q3 timing of having product available commercially from the second fill finish, there's definitely an opportunity also that our file could be approved with both fill finish facilities. And I think that that was one of the things that Akshay and I wanted to communicate as well. So a lot of optionality here, a lot of good news for patients, a lot of good news for the SMA community. I am really grateful to our internal team at Scholarock 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 Catalin, Indiana. And 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. So, thank you. Thank you, Lily.

Thank you. And the next question will come from Tess Romero with JP Morgan. 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 it Visma is fully approved for ages older than two years old, how are you thinking about a pedigree map being able to be used in combination with that therapy if and when you are approved? Thank you.

Yeah, thank you. What test I 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 a Pitigromab at any time between, you know, now and up to September 30th. And I think the incredible work that Akshay has done, you know, with our team in engaging the FDA, there's going to be a very significant opportunity to serve patients with SMA. So, Keith, do you want to comment on, 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 chooses because we think that they go hand-in-hand along with our muscle-targeted therapy with epitagromab. Now, specifically with Epifizumab, you know, in our SAFIRE study, we did not study patients that were previously on Zolgensma. As Akshay has noted several times, we are studying them in our OPAL study, and we've also shared with you that we do have post-Zolgensma So there's some experience out there with it. But as far as being able to utilize a PIDIGR map with it, I think it's going to depend upon the label and where the policies come out with the payers.

Thank you. Thank you. And our next question will come from Corey 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 that EU regulators are still trying to get their arms around? Thank you.

Thanks, Corey. Asha?

Yeah, thanks. So, obviously, we don't get into the back and forth of regulatory reviews, FDA or EMA. The one thing I can say is that that oral explanation that was scheduled led to a very good dialogue in advance of the meeting, and we were very happy with the pre-meeting alignment, which led to mutual agreement that there was no need for the meeting. And in fact, as a result, we obviously look forward to continued progress with the review and ultimately to launching the drug in Europe for children and adults with SMAs. As to the remaining timeline, I commented in the formal remarks that the Catalan Indiana facility continues to support the application, and we look forward to the decision around mid-year. But I think overall, the progress has been excellent.

And then, Corey, just to tag on to Akshay, and Akshay has mentioned this multiple times, we've been having very good open dialogue with the European regulators about what's been happening here with the FDA and cattle in Indiana. So it's been very collaborative. Like, everything going on here is, you know, you know, been a topic of discussion in Europe, and they've really been very flexible in working with us on timing.

It's very helpful. Appreciate it.

Thank you. And 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. And so, is the understanding that either of these sites can support approval by September 30th, and that's why there's definitely increased confidence and there's not necessarily such a reliance on the Indiana site, and so I have that correct? And the second question is regarding a potential approval and indications. I know previously there has been some discussion around the broadness of the label, type 1 versus type 2, 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?

Michael, great questions. I'll start on the two fill finish facilities and then Akshay will take up the label. So yeah, I mean, I guess at the end of the day, we have an enormous amount of confidence in our BLA as the headline news of what's changed from late last year to this year is really the fact that we have 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 vial and that drug would be available commercially in early Q3. So when you kind of take that 90-day window, the up to 90-day window per FDA guidelines for the Catalan Indiana facility, when you look at that window of commercial epithelial map 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. And I think that, you know, what I'm most gratified about is we try to live here at Scholarock by a deep commitment to the patients and families, you know, that are impacted by essence. And I'm grateful to the team that we took it upon ourselves to say, okay, let's do better this time than we did last time. Let's not rely on a single bill 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. And 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. Mike, you know, vis-a-vis the label, of course, it's premature to comment on the exact nature of the label before the regulatory deliberations are finalized here in Europe. What I would say is that generally speaking, the regulators have taken a very important approach to the labels for SMA products. They looked at the enrollment criteria of the pivotal studies, which exactly was the population. They looked at the plausibility of the mechanism across the spectrum of disease. And they looked at the unmet need. And so I feel like they've been very good with those principles to serve the community. We've been working with them. As you know, when we got the CRL, The draft label was completed the one outstanding issue was the manufacturing issue And both the US and Europe all I can say we've had constructive Regulatory dialogue throughout the period last year and we look forward to launching this product for children and adults without funding and Michael, you know Akshay and I would just note that as we previously had disclosed that you know where we were in

you know, toward the tail end of our last, you know, BLA review, we were pleased. And that's where we picked up this new application. It's 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 a PITIGOR map to the community.

Thank you.

Thank you. And 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, really. I had a question on sort of the game plan going forward now that you have a PDUFA date in hand. So are there any sort of new types of discussions you can have with payers or other like commercial bodies now that the PDUFA map is officially under review? And 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 initial approval? Or is it just kind of just 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 fill finish facilities and two independent paths to approval. The other obligation that we made is to be better from a commercial perspective. How do you use that time to make sure that you can meet the moment for the SMA community? And I think your question is a good one now with the September 30th to-do book, but yet being ready for an approval at any time. 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. And, Wesley, thanks for the question. What I can tell you is that, you know, joining the company four months prior to the PDUFA date, we were scrambling for that PDUFA date. We would have been able to launch successfully, but we have really been able to take advantage of the additional time that we have. Some specific examples that I've shared in the past, you know, first of all, with payers. We are able to meet with the payers and with our medical team to really discuss a Pettigrew map and the data. So those discussions are ongoing. We've just been able to take them to a much broader range of payers and really deepen the discussions that we have with them specifically around this. Additionally, we built out, 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 a PITIGRAMAP 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 of Pitogram app. And just we continue to really move forward with patient engagement activities. And that's through our program to really have patients demand better treatment for themselves when life takes muscle. And so I can tell you this. I want you to know that the team has been working very hard all the way through this delay, but we are clearly ready to launch now. So whatever that 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.

Thank you.

Thank you. And the next question will be coming from Mark Fromm with TD Cohen.

Thanks for taking my questions. A lot's been asked already on the PDUFA and the epigram itself. Maybe just looking at the sub-Q version, you mentioned you have that data in hand and once you get the approval for the IV formulation, you'll look to meet with the FDA to discuss it. What are the key issues you think you need answers from the FDA on and kind of what are the range of timelines for when you think you might be able to kind of launch that product depending on the outcome of those discussions?

Thanks, Mark. Asher?

Yeah, thanks, Mark. So, you know, the I would say there are no issues as such. These things are a matter of just alignment with regulators as to what's the optimum path forward to bring another innovation to SMA patients, and in this case it would be subcutaneous a bit of a math. The phase one data were excellent, showing very good bioavailability and pharmacodynamic overlap between two recent administrations. And what we have to do now is to share those data following the approval and align on the path forward in terms of any further development that we needed. So that could be PKPD data and consideration of any additional safety or efficacy. However, from a safety perspective, obviously the exposure is maximized with IV equipment map and so with the very large database we have in hand. Already from the studies we've done, we feel very good about safety via additional risk administration. And so we just want to get on and have those conversations and timelines apart. 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, just can you kind of walk through what's different about that trial or maybe the supporting data that Epidogromab's been able to generate relative to 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 Stover Rock with our leading antimicrobial satin pipeline. It still remains, to a good amount, the only validated antimicrobial satin antibody that's made it through phase two to deliver the kind of risk and benefit profile that we saw in the phase three with the satire study in SMA. Now, whilst we await that approval, obviously, many others are interested in this target. Roche and Shugai are a world-leading company. It was sad to see that antibody drop out. We've never really seen any Phase I data or the FlexDuxMAS model data from the Shugai-Roche antibody. So we don't quite know the nature of those data, and we await to see how strong they were. We know our data. Apart from the positive phase three study, of course, we have very nice data in the Flexbox 4Mouse model with an antimicrobial approach, an increase in muscle mass and torque, and an additional function We know that there are within FSHC normal fibers that can be boosted by means of an anti-dynastatin approach. We know other clinical trials in FSHC that have shown increased muscle mass and function. So we're very encouraged by our data and our diligence. And finally, we believe that they to 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 appears to be quite advanced. And based on our diligence with the experts, we decided, because of input from them, to milder end in terms of the RICI scores with patients with established disease where we thought we could still show benefit. And so, you know, we remain confident with our validated asset going into that phase two study and look forward to kicking off very soon.

Great. Thank you. And congrats on all the progress.

Thank you. Thank you. And the next question will come from Jeff 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 today, how are you guys thinking about incentivizing switches or maybe deploying a more novel outcomes-based pricing strategy just to help the early stages of the launch? And are the strategies different when you look to the EU and the early launch in Germany versus the US launch? Thank you.

Thanks, Jeff. As Keith noted, a cornerstone of our sort of campaign thus far around the disease itself has been an acknowledgement, and we see that the community gets it, that this disease is the hallmark is not only the motor neuron, but the resulting muscle atrophy. And so all of this innovation over the last 10 years has been on motor neuron survival and motor neuron health. And this has been needed innovation for the community. And yet, as Keith noted, nearly all patients are wanting their muscle atrophy to be addressed. And this will be the first and only muscle targeted therapy that's approved. So we don't necessarily really think about switches Keith, right? We really think about no matter what you choose to do for motor neuron health, we applaud and we're going to deliver something that addresses the organ that is, you know, the you know, the principal organ affected by this disease is the muscle. And that's what's been left behind over these 10 years of innovation that we're finally able to address. And putting that into practice, I know, Keith, that's been the cornerstone of what you guys have been talking about with the community, and I'll let you take it from there.

Yeah, no, Jeff, we're really not going to be focused on any type of switches because what we shared before is that in our own market research with treating physicians, we know that three-quarters of them have already said that they believe dual modality is the future standard of care for treatment in SMA. So that's directly targeting the motor neuron and directly targeting the muscle. So we believe that that will be how this is viewed. And then additionally, 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 needs that exist with these SMA patients. But 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. And the next question is going to come from Amy Lee with Jefferson Company. Your line is open.

Awesome. Thanks so much for taking your question, and big congrats on all the progress. Just wanted to get a sense of the next steps and timelines for the Catalan site based on feedback from the FDA after the inspection and the Novo club closeout meeting. Do you expect a form for 83 related to inspection and does the speed of your filing acceptance, which was around 30 days compared to the standard 60 days indicate any FDA urgency or prioritization and then finally. On the second manufacturing site, just wanted to clarify, are you maintaining it primarily as a hedge against Catalan, 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 Catalan Indiana was acquired by Novo, We knew that, you know, NOVA was acquiring that facility really for its own internal purposes, and they would have this transition phase into, you know, moving quote-unquote customers out because they're not a CDMO. That's not their business model. And so, you know, all along we recognized that, you know, that we would want to have and would require to have customers. an additional or more than one uh fill finish facilities that are outside of canada so all of that right was part of our plan you know even prior to um you know the the form 483 observations that the fda had in their general site inspection last year so i think that i think it's important to note that we see this second fill finish facility is absolutely vital for all of our global demand now We also see cattle in Indiana as important. We have drug vials there. We would anticipate that they would be part of our supply chain, and in due time, we would phase them out because they're going to be phasing us out. So more than anything else, we see them both as being important, and yet we do think having 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. And 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, and we are just grateful that the FDA has continued to show, you know, a sense of urgency understanding the needs of the community. So more than anything else, we see a world in which epitomimab gets approved with one or the other or both. And we think that that's a wonderful spot to be in. We'll let the FDA do their work on the review of the second fill finish facility and the data that has been generated by us on that second fill finish facility with drugs becoming available in early Q3. And we'll also let the FDA do their work expeditiously and thoroughly on their inspection as well as the inspectors concluded that re-inspection recently. So we're excited for what the future brings and More than anything else, I think you guys can see these timelines of the two facilities that really come pretty much together. And I think that that's a key takeaway to recognize.

Excellent. Thanks so much.

Thank you. And the next question will come from Gary Natchman with Canada Corridor Lines Open.

Thanks, and my congrats as well on all the progress. So, David, just to follow on the last point you were making there, if everything ends up being fine with Catalan, with the classification, are you still considering pulling the second fill finish facility from the BLA to simplify it for the FDA, or you'll just keep it in there regardless to have that better supply chain, even if it would potentially delay the approval and push it out a little bit? And then just a follow-up. Someone asked before on pricing, but just I guess to ask it a little differently, is there a strategy that would make more sense of launching first in Germany or in the U.S. or regardless, it would just be one global price and you're not anticipating any MFN issues? So, you know, pricing isn't really a consideration in terms of how you'll stagger the launches.

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 VLA, we did actually talk about the alignment that we've had with the FDA, the dialogue that we've had with the FDA throughout Q1 about the submission with both they'll finish plants and the optionality that that really provided us. And so, Akshay, do you want to comment on that? Like, 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. And based on that, we submitted with both facilities in the BLA with their full support, guidance, and alignment. And, you know, the most straightforward thing is Catlin, Indiana, is reclassified, is in compliance, and we can start getting drug out of there, pending approval, and the second facility would be withdrawn from the BLA. However, given all the constructive approach that's occurred with the FDA, we'll be guided by them, and in the long run, we clearly want redundancy in the supply chain, and so we look forward to an additional fill finish site. So I think all the options are open for us, and the really great position we're in now to serve patients is that by September 30th, we're going to be approved by one or the other facility, but in the long run, of course, we'll have redundancy in the supply chain.

Yeah, so Gary, let's just say the FDA has up to 90 days, but they make a decision faster than that, and they still need to review some information on the second fill finish. As Akshay had even described about a month ago, we would certainly have that flexibility of then just moving that second fill finish to an SBLM, which was always an option that we had considered as well. So lots of flexibility and optionality there, and it was a very good question. On sequencing and pricing, Keith?

Yeah, so first of all, Gary, as I mentioned in the prepared remarks, the team is ready to launch here in the U.S., but also the team is built in Germany. And so if you think about, you know, is there a preference for one before the other? No. We want to get this across the finish line both in the U.S. and in Europe. You mentioned how does this overall affect pricing. We go out with our list price here in the U.S. We go out with our list price in Germany and in Europe. Remember, Germany is the only place that we can proactively promote right after EMA approval. And so you're promoting and selling at your list price while you go through the Amnod process and you go through the reimbursement and establishing that price. So it really wouldn't have an impact and the bottom line is we're going to be prepared to serve patients in whichever market comes first and there shouldn't be a substantial impact to our ability to price negotiate and an overall impact on most favorite nations because we won't be at a point right away that we would even trip the cause of most favorite nations.

Okay, that's very helpful. Thank you.

Thank you. And our next question will come from CRIPA, Deborah Conda with Truist. Your line's open.

Hi, this is Alex Long for CRIPA. Congrats on the great news today. We had one about Roche discontinuation of Mugrabart and FSHD. Wanted to know if you've seen any uptake in investigator interest working with Ballarat for your FSHD trials.

Yeah, thanks for that, Alex. So, you know, the whole neuromuscular space is very excited about the Epidermic MAP program after our positive data in SMA. You're right, the intensity of interest has increased. Obviously, everyone's looking forward to the approval in SMA, but the neurology world looks with anticipation towards what a validated antimicrobial approach like Epidermic MAP can do, not just in FSHD, but in a range of diseases. And so, We're looking forward to the start of this study, which will be very soon now, Phase II study in FSHD, and with additional indications to follow where we'll study this drug. But you're absolutely right. There is plenty of interest and very constructive input as we think about triaging through these indications. Thanks.

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.