Scholar Rock Holding Corporation

Ladies and gentlemen, thank you for standing by and welcome to ScholarRock's first quarter financial results and business update call. At this time, all participants are in the listen-only mode. Following the presentation, we will conduct a question and answer session. To ask a question during this session, you will need to press star 11 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 11 again. Please be advised that today's conference is being recorded. I would like now to turn the conference over to ScholarRock. Please go ahead.

Good morning. I'm Rushlee Knopfinger, Vice President of Corporate Affairs and Investor Relations at ScholarRock. With me today are David Halal, Chief Executive Officer, Akshay Vesnau, 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 by going to the events section of the Investors page on our website. During today's call, as outlined on slide 2, David will provide introductory remarks and provide a general business update. Akshay will review our clinical and regulatory progress. Keith will provide an update on our commercial readiness activities, and Vikas will provide commentary on our company's financials and a summary of our 2025 priorities. And then we will 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 ScholarRock'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 -to-date FTC statements and filings. With that, I'd like to turn the call over to David. David?

Thanks, Rashmi, and good morning. Thanks to everyone for joining us on today's call. 2025 is off to a strong start at ScholarRock. In the first quarter, we made significant progress against our top priority of bringing a pitigrmap to patients and families living with and suffering from SMA in the U.S., Europe, and around the world. We were very pleased with the positive Phase III Sapphire trial, where we showed a statistically significant and clinically meaningful improvement in motor function as measured by the primary endpoint, the gold standard Hammersmith scale. And in Q1, to underscore the body of evidence for a pitigrmap for patients with SMA, we were gratified that our BLA was granted priority review by the FDA with a September 22nd to do for date. The team at ScholarRock is working urgently to prepare for our U.S. commercial launch, which will be the first country in a series of launches over the coming years. I am confident under Keith's leadership, we will assemble and deploy an experienced, talented, and patient-centric team committed to the SMA community. Along with Keith, Akshay, and Vikas, we now have the responsibility to bolster ScholarRock's capabilities as we advance our mission to deliver a pitigrmap to patients with SMA. This is what we know well and what we do well. We join ScholarRock at a time of great strength and opportunity as we scale for the next phase of growth to a commercial-stage, fully integrated global biopharmaceutical company. Globally, nearly 35,000 patients with SMA have received SMN-targeted therapies, and at ScholarRock, we are working with urgency to prepare for our global launch, which we anticipate will commence in the U.S. in Q3. We are also beginning to set the table to serve patients with SMA in Europe, Asia Pacific, and Latin America. Our ambition at ScholarRock is that any patient with SMA that can benefit from a pitigrmap should have access to a pitigrmap. I am confident that the global opportunity with a pitigrmap and 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. While we are very focused on our large opportunity to serve patients with SMA, we are continuing to evaluate expanding the study of a pitigrmap into other rare, severe, and debilitating neuromuscular disorders. Akshay will touch upon this shortly. Now, I'd like to turn to our exploratory cardiometabolic program, the EMBRACE -of-Concept Study. Our effort here is to understand the role we may play in the treatment of obesity. We are the world leaders in myostatin biology and know well that GLP-1s have brought needed innovation to patients with obesity and cardiometabolic disorders. While the benefits outweigh the risks, muscle wasting and lean mass loss is something that the medical community is trying to address. The EMBRACE study is our initial effort in the cardiometabolic space and will provide insights that will guide us moving forward. We remain on track to share top-line results in June. Our achievements in 2025 to date underpin the opportunity before us to bring the potentially life-transforming benefits of a pitigrmap to patients and families with SMA. We are focused on delivering for patients, with Akshay leading efforts in collaborating with U.S. and EU regulators, Keith applying a similar approach from Vivgar and MG to the commercial opportunity for a pitigrmap, and Vikas focused on disciplined capital allocation to fuel near and long-term growth. With that, I'll turn the call over to Akshay to provide a more detailed update on our R&D progress. Akshay?

Thanks, David. I'm delighted to be here after six years on the Board of Scholar Rock. As a physician scientist, I could not be more excited to have seen the concept of a pitigrmap, a highly innovative anti-myostatin antibody, go from bench to bedside with our remarkable SAFIRE data. SAFIRE showed that a pitigrmap has the potential to reverse the progression of SMA from a loss of motor function to a gain of motor function. Specifically, the study demonstrated a statistically significant improvement in Hammersmith as patients on placebo worsened. Importantly, patients treated with pitigrmap had an approximately threefold higher chance of a three-point or greater increase in Hammersmith versus those on placebo. Along with the very encouraging safety profile, the SAFIRE data suggests that a pitigrmap has great potential to provide clinically significant benefit to patients with SMA, despite the use of background SMF-target therapies. Additionally, at the Muscular Dystrophy Association Conference in Dallas in March, the detailed data from the SAFIRE trial were presented for the first time. These data demonstrated that treatment with a pitigrmap achieved clinically meaningful and consistent benefit in motor function across pre-specified SMA patient subgroups, including the type of SMN target therapy, age, age of initiation of SMN therapy, and geography. Efficacy was also supported by additional analyses of outcome measures of motor functions, such as the revised upper limb module and the World Health Organization Motor Development Index. As David mentioned, our BLA was accepted under priority review by the FDA, and the MA was validated by the EMA in Europe. Turning first to the FDA, we were gratified that our BLA for a pitigrmap was granted priority review by the agency with a September 22nd PDUFA date. We feel the potential clinical benefits of a pitigrmap, as demonstrated by a Phase III trial, are underscored by the FDA's priority review designation. By definition, a priority review designation by the agency conveys the capacity of a pitigrmap to potentially impact unmet need in SMA by either being a treatment for a serious or high-threatening condition, or provide a significant improvement in safety or effectiveness over existing treatments. I'm excited that our team continues to work collaboratively with regulators and that we remain on track. With the success of SAFIRE, we're just beginning to tap into the broader potential of our truly innovative Myosmatin platform. There's much more we can do with the promise of the pitigrmap and our platform by delivering advanced and severe neuromuscular diseases, including the muscular dystrophies such as DMD and FSHD. Additionally, we're advancing SRK439, a highly innovative and potent sub-Q anti-myostatin antibody to the clinic. Based on preclinical data, SRK439 has the potential to inhibit myostatin and increase muscle mass and create options for our pipeline. We remain on track to file the IMD application for SRK439 to support the first in-human study in Q3. Now, earlier David mentioned the potential role ScholarOpt can play in addressing lean mass in cardiometabolic diseases. As we all appreciate, whilst GLP-1s offer quantitative benefits in terms of weight loss, much more needs to be done from a qualitative perspective regarding preserving lean mass. Notably, a quarter to a third of the weight loss with GLP-1s is due to the loss of muscle. Looking forward, it will be important to preserve muscle from the viewpoint of the associated metabolic benefits and a healthier approach to weight loss. Our EMBRACE study is our ongoing Phase II trial to investigate preliminarily the potential of further developing our highly selective anti-myostatin approaches in patients with obesity with the goal of reducing the loss of lean mass. We look forward to the upcoming readout of our initial data from EMBRACE in June 2025. In summary, we remain focused and on track to deliver on our key priorities this year. We will, one, drive the US approval of Epidgramab in Q3 2025 and advance the Eden Court approval in 2026. Two, initiate a study of Epidgramab for infants and toddlers with SMA under the age of two starting in Q3. Three, file an IND for SRK-429 in the third quarter. And finally, complete our clinical development plans for Epidgramab in additional neuromuscular indications. With that, I'll turn it over to Keith to provide a commercial update. Keith.

Thanks, Akshay, and good morning, everyone. I'd first like to thank our colleagues from Research and Development for their focus and commitment over the years to make the September 22nd PDUFA date for Epidgramab a reality. SMA is a disease impacted by both motor neuron degeneration and muscle atrophy. Today's therapies only address one piece of the puzzle, the motor neurons. Currently, there are no approved muscle targeted therapeutics to treat muscle atrophy. With Epidgramab, we have the opportunity to usher in a new era for the treatment of patients with SMA. This is a progressive and devastating disease that leads to the loss of mobility, limited activities of daily living, and a lack of independence. Despite the advances made in treating SMA with SMN targeted therapies over the last decade, the contemporaneous natural history data shows that the majority of patients still experience progressive muscle degeneration over time. The bottom line, nearly all patients and families living with SMA are demanding a transformative new therapy. This is supported by a Cure SMA survey published last month where 90% of patients identified that new SMA treatment options improving muscle strength is an important need. Our market research and interactions with healthcare professionals tell us that 80% of patients with SMA should start as early as possible in treating patients living with SMA. Today, there are approximately 10,000 patients with SMA in the United States, and roughly two-thirds of them have received an SMN targeted therapy. For these patients, as Akshay shared with our SAFIRE data, Epidgramab showed the potential to reverse the progression of SMA from a loss of motor function to a gain of motor function. Furthermore, globally, there are approximately 35,000 individuals that have already received an SMN targeted therapy. Neurologists recognize that in the future, a treatment approach of dual modalities to target the motor neuron and the muscle will be necessary to treat SMA. At ScholarRock, we have the opportunity to make a meaningful difference for both children and adults living with SMA, first starting in the U.S., then Europe, Asia Pacific, and Latin America. We are currently building on the momentum that has already been established for a successful U.S. launch. Last weekend, several of us at ScholarRock had the opportunity to meet with patients and their families at the Cure SMA Walk and Roll in Boston. We continue our stakeholder engagement and SMA disease education. Life Takes Muscle is the first muscle-focused SMA disease awareness initiative. Our fully staffed U.S. Market Access team is currently meeting with key U.S. commercial and federal payers. Our process of hiring and onboarding our customer-facing team of roughly 50 sales, reimbursement, and patient support personnel is well underway. We expect to be fully staffed by mid-2025, well ahead of our potential launch in late September. Finally, we believe that Epidigromab has the potential to be a -in-class, -in-class therapeutic to establish a new standard of care in SMA. Now I will turn the call over to Vikas. Vikas?

Thank you, Keith, and good morning, everyone. I'm pleased to provide a business update and provide insights into how we are thinking about resource allocation in the future. The opportunity with Epidigromab in SMA alone offers the potential for many years of sustainable growth and will enable strategic, thoughtful investment in our pipeline to develop new indications and new therapies for an increasing number of patients. These pipeline investments will be aligned to our commercial success. We ended the quarter with $364.4 million. During the quarter, we continued to increase our investments in commercial readiness and inventory buildout. As we look ahead, we are prioritizing the commercial launch and ongoing clinical programs. We have an additional $100 million under our debt facility that we can draw down this year to support the upcoming launch, bringing our anticipated runway into 2027. We are working on building a tighter financial plan, and we'll share more details over the next few quarters. As we move forward, I will focus first on driving strong performance with financial discipline, next, investing in capital-efficient commercial buildout, and thoughtful capital allocation to advance our pipeline. With that, I will turn it back to David.

Thanks, Vikas. In closing, we are acutely focused on the key priorities that will enable us to build and scale Scholarock into the next global biotech powerhouse. First, regulatory approvals and the upcoming U.S. launch of Epidigromab for patients with SMA, followed by a series of With that, we'll now open the line for questions. Operator?

Thank you. As a reminder to ask a question, please press star 1-1 on your telephone and wait for your name to be announced. To withdraw your question, please press star 1-1 again. The first question comes from Allison Bretzel with Piper Sandler. Your line is now open.

Hey, good morning, team, and thanks for taking the questions. Just two from me. You know, ahead of Epidigromab launch, could you just frame for us how your discussions with U.S. payers have gone? You know, how receptive are they to coverage of combination therapy for both an SMN targeting and a muscle targeting therapy and SMA patients? And can you also describe that feedback for -U.S. payers and governments as well? And then second, could you just characterize interactions with FDA on the Epidigromab review? Obviously, there's a lot of concern about the state of the agency right now, so just want to know if you have anything to report on there. Thank you.

Thanks, Ali. So why don't I just touch on pricing, then Keith will comment more specifically, and then Akshay will take on the regulatory update question. You know, one of the things that, you know, Keith and I have spent a long time, you know, sort of thinking about and sort of doing as it relates to in general, you know, really what we're talking about is access for a potentially transformative therapy for a small population of patients. And, you know, we would sort of expect, you know, as we think about establishing access plans, that we consider sort of pricing for our therapies that would be reflective of the rarity of the disease, the severity of the disease, and the value that our therapy would provide the patients. In this specific case, patients with SMA. And I think one of the other things that, you know, Keith and I think a lot about is also given the rarity of disease, we would expect that the budget impact, you know, to any one single payer in the US, or even globally, would be very limited for the high value proposition as it relates to sort of the engagement with payers, you know, largely in the US. I'll turn it over to Keith for some more commentary. Thanks,

David. So, Ali, what we know in speaking with payers is that, you know, with SMM targeted therapies alone, patients can still have progressive motor function loss. And at the MBA in Dallas in March, we heard that a proportion of patients are already receiving more than one SMM therapy. So SMM targeted therapy. So right now, it's not uncommon that you'll see a payer pay for more than one of these therapies. As you know, with our data, when we go in addition to an SMM targeted therapy, we see improvement consistent across all age groups of 2 to 21. And so with that in mind, these patients need a better therapeutic and our early discussions with payers have proven to be positive and we'll continue those discussions. As for Europe, we plan to, as you know, we filed and we're going through the regulatory process right now. We will be preparing to go into reimbursement discussions. Let's remember that we are going to sequence through Europe and we will start with Germany in 2026. And then we will roll out over time how we will sequence through the remainder of the countries in Europe. Thanks for the question. Maybe a closer look

at the regular. Sure. Thanks, David. So thanks for the question on FTA interactions. Look, it's a time of evolution and change at the FDA. We all recognize that. But what I'm delighted by is that the pace and the collaborative conversations we've had with the FDA are all going as per routine. And we continue to guide that we're focused on working with the FDA and delivering on that September 22nd to do for date. So really, we're seeing no issues in specific to a program. I've been the SMF approval and we're also delighted that FDA Commissioner Martin McCurry actually made a point that with all the changes, one of the main areas of focus continues to be rare disease and what patients need for rare disease. So it's all systems go here and we continue to work

productively with regulators here and in Europe.

And our next question comes from Michael Yee with Jeffreys. Your line is open.

Hey, guys. Good morning. Maybe just two questions on obesity coming up in June. Given that it's towards the end of the second quarter, but also that there's two different endpoints. I think 24 and 32 weeks. Are you thinking about providing more data to be a more complete package of information to help people out? In terms of longer term follow up data on some of the metabolic parameters that could come out in June. Have you thought about that? And then just holistically, the prior CEO Jay has mentioned a 20 to 40% reduction of muscle loss would be great. But I think people are still sort of trying to grasp what you think a good result is and what is exciting. Can you give some color on that? Thanks.

Yeah, great questions, Mike. Yeah, as actually, and I noted, this is our. First effort at really understanding what role we may be able to play in this space. We know that there is a need that the medical community is trying to address and. I'll turn it over to actually for sort of a closer look at what one might expect in terms of, you know, as you were saying, the 24 weekend point, but then the eight week sort of withdrawal of the therapies as well. Yeah,

thanks Mike. So, you know, the core of it will be providing the 24 week data. That's the main study will certainly provide any necessary follow up information that we have that helps us understand and helps everybody else understand the potential of an anti my starting approach in the obesity space. You know, we're excited to test this hypothesis preliminarily. It's an important one. I think we all appreciate that loss of muscle is almost certainly not a good thing as people lose weight, especially when it's a quarter. The third of the weight loss is due to muscle. And so the primary focus will be the 24 week data changing lean math, the safety and helping us understand the path forward. So I'll leave it at that, but it's not so long to wait now to get the data.

And the next question comes from test Romero with JP Morgan. Your line is open.

Hey, David and team. Thanks so much for taking our questions this morning. So in the US, you noted collaboration with regulators and that you remain on track here. Can you confirm or not? If you have completed your mid cycle meeting? And if so, can you comment on any high level discussions you have had around labeling? And at this time, what has the agency said about the need or lack thereof for an ad com? Thank you.

Thanks, Tess. As actually, and I both noted in the call today, we remain on track and maybe for a closer look on just kind of where we are. Actually can can provide a little bit, little bit more detail. Yeah,

I mean, there's not much more to provide other than the conversations have been extremely constructive and right on track, I would say. After the detail with when and how the various interactions are, I don't really think this is the time or space, but I do want to reassure everybody that our ability today to guide on the call that we're heading to that September 22. A producer date is based on a series of very constructive conversations, so I'll leave it at that and more news as we get it.

And the next question will come from David Nearing Carton with Wed Bush. Your line is open.

Hey, thanks for taking the question. I just had one on the additional potential indicate indications for a particular map. As I recall, there's a lot of exploration and preclinical models, at least of DMV and related disorders. Is there any particular. You know, new development or, you know, new treatments that you think are particularly attractive to. You'll bring a program forward into one of the other musculature neuromuscular indications. You know, just just kind of help us out with thinking about the expansion plans.

Thanks. Thanks very much, David. Yeah,

thanks, David. So, look, I think with a bit of a map and a really unique and potent anti-myocytopenia platform, we have an incredible opportunity to not just help in FMA across the spectrum disease there, but well beyond that into other neuromuscular disorders, which many of which are very severe and life threatening. As you said, we've certainly spoken about and done a lot of good work on DMV and FSHD models to very important muscular dystrophies. We've shared those data. People are very excited about them. And the fact that we validated a pit of a map as having the ability to put on the degree of muscle that it can in such a serious disease. And I think that the FMA obviously bodes well in these other neuromuscular disorders. There are other indications beyond DMV and FSHD. ALS is one that we can talk about as well. And we've got some thinking there and there will be more beyond that. But I think at the current time, we're very encouraged by the model data both from DMV and FSHD. We're working through details of exactly how to begin to study in those disorders and we'll share more details around that later in the year. But there's no question that an agent that is as strong as a pit of a map really has to be investigating

additional indications.

And our next question comes from Gary Natchman with Raymond James. Your line is open.

Thanks and good morning. So if you get the FMA approval on the PDUFA, how quickly will you be able to launch? Will everything be completely in place already on the sales and marketing side, including with access and patient support programs? And how much commercial supply will you have at launch just in terms of meeting the demand? And then also, if the proof of concept data in obesity are positive, just talk about the likely next steps with 439 after you file the IND. You know, what type of Phase 1 study will you run and how long before it can potentially move into a Phase 2? Thanks.

Yeah, thank you. And as Keith noted on the call, he's working with urgency here by mid-year to have the team in place for more commentary on like readiness, you know, to serve patients that approval. Keith? Yeah,

Gary, thanks for the question. I mean, I think the first thing that I want to call out is that the team here has already been at work and has already been building, preparing for this launch for some time. We are fully staffed when it comes to our marketing team. And as I mentioned, our market access team, we have also, we're fully staffed in our leadership team for our patient support programs. We will, as I mentioned in the prepared remarks, we will have the entire commercial team fully staffed by mid-2025. So we should have a couple of months prior to launch. In regard to how soon will we launch after September 22nd date, we're going to be prepared to be out there the next day. I know that the supply chain team is working so that we can have product available to patients as soon as possible. And then I think the last thing that you had asked was in regard to supply. And I want to assure you that we have ample supply to be able to have a very successful launch.

Thanks, Keith. And regarding like, sort of the, the EMBRACE study, the PUPA concept, and I think, Gary, as you noted, how do we think about 439? As Akshay mentioned, we're moving forward with an anticipated IMD for SRK 439 and Q3 no matter what. We see this as just another indication for us that we are the world leaders in myostatin biology, and we're very excited to move this into first in human studies. The beautiful thing about SRK 439 is we have optionality. It can support our ambition in SMA and other additional rare, severe, and debilitating neuromuscular disorders, or depending upon the sort of results that we see with EMBRACE, depending upon our thoughts about further study there and how we might approach it, 439, as I think was previously noted by our team, could have some optionality for us in a different space, that being cardiometabolic disorders and obesity. So, we'll be guided by the data. We absolutely will sort of focus on what we see in this first exploratory readout in the EMBRACE study, and then we'll provide you all here in the coming month or two further plans for SRK 439.

Great. Thank you.

And our next question comes from Kripa Devrakanda with Truist. Your line is open.

Good morning, Kim. Thank you so much for taking our question today. Given that we're getting close to the PdUFA day, just a follow-up question from one of the prior analysts, would you be able to provide any kind of metrics or guidance once the drug is approved or any color on how you see the early demand? And not sure how much you expect the recent executive order on drug pricing to have an impact on orphan disease area, but in light of that executive order, any comments on how you think about pricing in the US versus ex-US? I know it's early to give us the exact numbers, but just qualitatively.

Kripa, two great questions. Why don't I take sort of the president's executive order first, and then Keith can comment a little bit about launch dynamics and kinetics. As you know, and I think you just noted it in your question, it's very early to really comment on the president's executive order on MFN pricing. However, it really does not change in any way our plans to commercialize a Pidigromab not only in the US, but in Europe, Asia Pacific, and Latin America. We do believe should any elements of the executive order be implemented, be implementable, it would obviously be better to have not yet established pricing for a therapy versus, let's just say, products that are already out there with established pricing. In all countries, we would expect that the price of a Pidigromab will be reflective of the rarity of SMA, the severity of SMA, despite the use of all sort of SMN targeted therapies, as we noted, still a loss of motor function, despite the use of those over time. And then, of course, the strong value proposition that a Pidigromab may be able to provide the patients and families with SMA. And then, as I also noted a bit earlier, we think that given the rarity of the disease, a Pidigromab would not really impact, you know, tremendously any real budget of any magnitude of any single payer, let's just say in the US or in any country around the world. So, we think no matter what happens with the president's executive order for MFN, we think we're going to be in a position of strength when we think about the launch of a Pidigromab globally for the 35,000 patients in the world that have received an SMN targeted therapy. For a closer look at sort of your first part of your question on launch dynamics and kinetics, I'll ask Keith to comment.

Yeah, Kripa, thank you for the question. First of all, in regard to guidance, we are not going to be providing guidance at this time. I can give you some aspects that from working in rare disease for now, two decades. One thing that I'm very excited about the SMA marketplace is that, you know, 100% of newborn screening in the US, and as we noted in the prepared statements that, you know, we know of the two thirds of SMA patients of the 10,000 in the US that have already had an SMN targeted therapy. These are good aspects to begin in a launch. Also, the fact that we have the centers of excellence with the, you know, it's highly concentrated because of the Cure SMA model. All of these things give me optimism. Now think about this. These centers of excellence are also going to be many at your academic hospitals. So, we will be going through formulary processes and such, and some of them are quite timely. They're not just set based on our launch date, but when the academic centers and other centers are going through it. So, you know, we expect that between that as well as applying for a J code that we would receive sometime, you know, six plus months later that, you know, we'll prepare for a consistent and steady launch.

Thank you so much.

Thanks, Kripa. The next question comes from Mark from with TV Cohen. Your line is open.

Hi, thanks for taking my question. Maybe just following up a little bit on Kripa's question before, just on launch trajectory. As you mentioned, the SMA population now, you know, has 100% newborn screening in the US. You know, they've gotten very concentrated into these centers of excellence to receive SMN therapy. Like, those would seem to be tailwinds to the launch relative to prior SMA launches, but then, you know, while there's certainly unmet need, maybe it's not quite as dramatic as it was before the original SMN correctors launched, you know, which would maybe be a headwind. Do you think those net out and this, you know, we should look at prior SMA launches as a good proxy for the trajectory that a pitigroup might have? And I guess to the extent that you don't think those are good proxies, is there another launches out there in the rare disease space that you think maybe are more analogous to what we should expect with a pitigroup? Yeah,

it's a great question. Mark. I think, first of all, one of the things that, you know, our team is always focused on as opposed to, you know, looking at other sort of proxies or other launches. We just kind of. Look in the mirror and try to compete with ourselves and think about the best way that we can serve patients immediately and then over time. And so you're right, there's probably some headwinds. There's probably some tailwinds, but I think, you know, a couple things that I would just underscore. SMN targeted therapies in and of themselves with longer term data that's been presented absent of Scholarock have shown that there can be a return to the progressive motor function loss of the disease. Despite what happens earlier when treatment is initiated and then we ourselves showed in the Sapphire study. Obviously, at the time in which we were capturing patients and enrolling them in the study, there was overall loss of motor function. Rather than what we demonstrated with our primary endpoint, the gold standard Hammersmith scales again, a motor function. So we do think that there's a lot of urgency. Still for patients, and I think he even commented on how that was underscored at the last month's presentation by your estimate of what patients and families were looking for. At the same time, we really want to play the long game. You know, independent of sort of launch trajectory, we want to build a team and a model to serve patients immediately and then over time for sort of a steady consistent growth of our business by serving patients in a very meaningful way. I don't know if Keith would like to add a little bit more onto that as well. Keith.

Yeah, I mean, you bring up the point of this, the launch compared to other estimate targeted therapies when there was nothing else available. Okay. So they truly had no other treatment. So I think this instance to urgency to be able to get to that product was great. As we take a look at the information, you know, we find that patients that are on estimate targeted therapies, most of them see their doctor on average two times per year. So it's not like September 22nd happens and they're have an appointment the next week. So I just want to guide you on that. I agree with your statement that, you know, the fact that we know where these patients are as an is an advantage. However, there are going to be some headwinds as David mentioned. Thanks.

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

Hi, Mack Mothman on for Evan. Thank you for taking our question. Recently, we had noted some stronger rebound and spend rather the sales to start 2025 and we're hoping to get your updated thoughts on long term changes that may occur in the SMA market. Specifically with a pit of gromad being used in conjunction with spin rise. Are you internally expecting any longer term growth from spin rise that may benefit a pit of gromad upon launch?

No, it's a great question. I think, you know, one of the things that we would note is that. As actually shared earlier in the. Sapphire study really in a pre specified way, depending upon which estimate targeted therapy. You know, a patient were received, you know, received, you know, we feel like we can help all patients independent of whether or not they received spin rise or or plan. We'll continue to sort of assess the nearly five billion dollar annual market across the three different estimate targeted therapies, whether or not it's the highly innovative gene therapy. So, or the other highly innovative estimate targeted therapies like spin, Raza and risk of glam and we would just expect to follow those dynamics, but recognize with the sapphire data. And then, as actually noted earlier in today's call, the upcoming under to study the Opal study where we would expect more experience for a pit of gromad for patients who have received so, you know, we think we're going to be in a position of strength. To really help and provide potential transformational benefits for all patients that have received targeted therapies over time. Thank you for your question. Appreciate it.

Our next question comes from Andre's Maldonado with H.C. Wainwright. Your line is open.

Hi, everyone. Thanks for taking my question. Just a quick one for me. Given the progress towards U.S. launch and your ongoing readiness in Europe, I guess, how are you evaluating the potential to secure commercial partner for Europe? And in this context, obviously, you want to fire and also lenders across the pipeline. But how would you prioritize the additional indications for pit of gromad in this in this situation? Thank you.

Yeah, thank you. I think we noted this just a few weeks ago on our April 28th call. Given the experience that, you know, this collective team has had, you know, between Alexia and Elm Island, our genetics, we feel like there is nobody better suited to serve patients globally than us directly. So it is not a priority of ours to think about a partner outside of the U.S. We feel like this is something that we know well and in fact do well and will continue to run a playbook that not only do we know the playbook, we feel like we wrote that playbook. As it relates to other indications, as Akshay noted, will be data driven. The team's been doing some wonderful work in looking pre-clinically at additional rare, severe and debilitating neuromuscular diseases. And Akshay will be providing further guidance on that toward the end of this year and into next. Thank you.

And the next question comes from Dennis Kennedy with LifeSci Capital. Your line is open.

Thanks for taking the question. As we look towards the EMBREAD data in June, can you just help frame expectations for the terzepatide monotherapy arm? Specifically, how much of the lean mass loss that we'd expect at one year with terzepatide do you think typically occurs by week 24? Related to that, do you think eight weeks of follow-up post-treatment is sufficient time to demonstrate a weight regain in patients in the terzepatide monotherapy arm?

Yeah, Dennis, these are great questions. And in fact, I think within your questions, you sort of highlight some of the limitations for us in sort of a 24-week endpoint and then eight weeks following. But nonetheless, as Akshay and I have noted, we feel like it's important to understand for a first time if there is a role that we can play. Akshay? Yeah, thanks.

So, Dennis, I mean, terzepatide consistently has shown loss of lean mass as have the other GLP-1. That mass is symmetrical to the overall loss in body mass. And so you can expect that at week 24, you know, about 25 to 30 percent of the weight loss that will have occurred in the terzepatide alone arm will be due to loss of muscle. We're projecting that based on historical data. There's no reason to believe that it'll be any different in the context of this study. Now, will that be the maximum amount of lean mass loss at week 24? I don't think so, because in the pivotal studies, you know, terzepatide was studied for much longer. And overall, the impact on muscle mass is even greater, obviously, as we continue to see patients lose more weight. But within the context of the embrace, further to a third of weight loss at week 24, lean mass loss, we expect. Your other point about is eight weeks of follow-up sufficient? Well, look, you know, this study was an exploratory study to understand the potential of a pedigal map in a pure, clean, safe, potent anti-myostatin approach in the context of obesity. I don't think the eight week follow-up answers the whole question, but you can anticipate that there'll be some weight regain during that period. And the trajectory of that would be quite important to monitor between the two

arms. Great. Thank you. Thanks, Jeff.

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Thank you, Operator. Thanks for everybody joining

the call today, and we'll look forward to continuing to update you and keeping you apprised at our progress as an organization. Thank you.

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