Welcome, everyone, to the 43rd Annual JPMorgan Healthcare Conference. My name is Tessa Romero, and I am one of the senior biotech analysts here at JPMorgan. Our next presenting company is Scholar Rock, and presenting on behalf of the company, we have President and CEO Jay Backstrom. Jay, over to you. Although I don't think the mic is on.
Can you adjust the mic? The volume?
All right. Very good. All right. Well, hey, thank you, Tess. Let me get that here. Giving you a quick, accelerated view of my presentation. All right, so thank you, Tess. Good morning. It's great to be here today to share our excitement for 2025 and beyond. Over here. So I will be making, as a reminder, I'll be making forward-looking statements. So please review the details on this slide, as well as our quarterly and annual filings, that provide a more detailed discussion of the risks inherent in our business. Scholar Rock is a mission-driven company, and I feel privileged to lead a team of talented and dedicated professionals, all focused on creating new possibilities for those living with serious diseases, starting with spinal muscular atrophy.
2024 was an incredible year for Scholar Rock, and we are running into 2025 with tremendous momentum, working with a sense of urgency as we prepare to commercialize apitegromab in SMA, expand on the development of apitegromab, building a pipeline and a product, and advance our anti-myostatin programs in cardiometabolic indications. Building on a successful SAPPHIRE phase 3 trial with transformative benefits for patients receiving apitegromab over standard of care alone, we are finalizing the regulatory applications and preparing for the launch of apitegromab in SMA, where we see a potential $2 billion-plus global opportunity. Work is underway to initiate earlier treatment for those under two years of age with SMA with our OPAL study and to expand the development of apitegromab in other rare, devastating neuromuscular disorders where muscle weakness and atrophy are hallmarks of the disease.
Building on the progress of their cardiometabolic programs, we are advancing toward two significant milestones for the top-line results for EMBRACE, our phase II proof-of-concept study in obesity expected in Q2, and the submission of the IND for SRK-439, our novel anti-myostatin program designed specifically for cardiometabolic disorders in the third quarter. Our success in SMA is directly linked to the scientific insights that was the basis for forming Scholar Rock, our deep understanding of how to selectively target the very complex biology of the TGF-β superfamily of growth factors. Results from SAPPHIRE validate our approach, where we succeeded where others have failed. By hitting the right target, the precursor forms of myostatin at the right time before activation to the mature growth factor, apitegromab is the first and only anti-myostatin to demonstrate functional improvement with our favorable safety profile.
We've applied our scientific insights and world-class protein engineering to build a robust pipeline of highly innovative products across high-value therapeutic areas. I'll focus today's discussion on our industry-leading anti-myostatin programs in neuromuscular and cardiometabolic indications. Now, turning to our number one priority to commercialize apitegromab and transform the treatment of SMA. SMA is a progressive neuromuscular disorder, and despite the remarkable innovation and incredible improvements achieved with the current standard of care treatments, all of which address the SMN protein deficiency, muscle weakness remains a core unmet need. Those living with SMA need and want more, more gain in function, more muscle strength, and importantly, they want to be able to maintain and stabilize their current level of activity. The long-term data for nusinersen treated patients from the CHERISH, SHINE studies illustrates the progressive nature of SMA.
As shown in the graph, after the early gains in the Hammersmith scores, there's a plateauing of effect with no further improvement, followed by a progressive loss of about one point per year. A loss of one to two points can be the difference between maintaining a measure of independence, such as operating a motorized wheelchair. The cumulative effect of progressive loss can have a profound impact on function and independence. We designed SAPPHIRE, a randomized controlled study with apitegromab plus standard of care, either nusinersen or risdiplam, compared to placebo plus standard of care, to see if we could improve and maintain function and address the ongoing unmet need in SMA. We were thrilled to announce that SAPPHIRE met its primary endpoint, the only muscle-targeted therapy with clinical success in SMA.
SAPPHIRE delivered gains of 1.8-point improvement for apitegromab compared to placebo, as measured by the gold standard, SMA-specific Hammersmith Functional Motor Scale at Week 52. This clinically meaningful benefit was statistically significant with a p-value of 0.0192. Importantly, apitegromab demonstrated consistency of effect across age groups 2 through 21 in a broad SMA population. In addition, 30% of patients who were already receiving standard of care achieved an additional three-point or greater improvement in their Hammersmith scores. With respect to safety and tolerability, SAPPHIRE confirmed apitegromab's favorable safety profile, which was consistent with the safety profile seen in over four years of treatment in SMA based on our phase II TOPAZ study and consistent with our highly selective approach to blocking myostatin. The results from SAPPHIRE highlight the potential for apitegromab to transform the standard of care in SMA.
As shown in the graph on the right, displaying motor function over time from SAPPHIRE, those treated with apitegromab gained motor function, while those receiving placebo lost function despite being on standard of care treatment, with the loss in function consistent with the one-point loss per year seen in the long-term follow-up of the nusinersen treated patients. These results demonstrate the potential for apitegromab to alter their trajectory of the course of the disease. The transformative therapeutic potential of targeting the muscle with apitegromab plus standard of care across a broad range of patients, coupled with the exceptional long-term safety and tolerability, validates the approach of targeting both the muscle and the SMN protein deficiency in SMA, setting the stage for a shift in the treatment paradigm to include apitegromab or highly selective muscle-targeted therapy as part of the treatment regimen for this neuromuscular disorder.
We have more to do. We are expanding our reach in SMA and are on track to initiate OPAL, our SMA study in those under two years of age, later this year. OPAL will provide the opportunity to treat even the youngest in need and will also expand our experience in the setting of gene therapy. Time is muscle, and we believe to give children born with SMA the best chance to achieve as much motor function as possible, starting treatment early with apitegromab could optimize outcomes. Now, turning to our commercial preparation, we are making excellent progress and are well-positioned for a highly successful commercial launch. We have the right medicine in the right market, and we're executing the right plan. SMA has benefited from three SMN-targeted therapies, and the market has grown globally to $4.5 billion.
We are highly leveraged by the current three standard of care medicines, as we know the patients, the physicians, the payers, and the infrastructure. There also is alignment across all SMA stakeholders that more is still needed, specifically to maintain or improve motor function by the addition of a muscle-targeted therapy to the treatment paradigm. Considering we are not competing with existing standard of care therapies, and we are the first and only muscle-targeted therapy that has demonstrated transformative therapeutic potential, we believe SMA alone can be a $2 billion global opportunity for us. And the elements that make SMA, excuse me. The elements that make SMA the right market opportunity are optimal for highly successful commercial launch.
Unlike many other rare disease markets where there is a need to improve diagnosis and educate patients to identify patients, SMA patients are already identified and treated with treatment highly concentrated among 250 expert U.S. physicians. Again, there's clear recognition of the unmet need, and SMA is an incredibly organized and motivated patient caregiver and patient community who are looking for additional treatment, and finally, payers are familiar with SMA, and there's established value for improving motor function. Payers also recognize the continued need and are covering sequential treatment. All of these dynamics point to an optimal opportunity for a highly successful launch to usher in a new treatment paradigm in SMA.
To carry out our commercial plan, we have an experienced team in place and have added leaders in marketing, medical affairs, market access, commercial operations, and sales who have successfully launched multiple rare disease medicines, including Spinraza and Evrysdi in SMA. We continue to recruit top talent, as Scholar Rock is now a magnet for attracting outstanding, experienced professionals who want to be part of the next wave of innovation in the treatment of SMA. We are making excellent progress in our launch preparations, and in fact, we've been preparing for the commercial launch for apitegromab and SMA over the past several years through our active engagement with the SMA community. Last year, we launched the first muscle-focused disease education campaign, Life Takes Muscle. We've established deep relationships with patient advocacy organizations in both the U.S.
And Europe, and our medical affairs team have been engaging with QSMA-identified treatment centers. And finally, we are making great progress in our important aspects of commercial preparation. We've secured commercial supply and are partnering with distribution partners who are highly experienced in rare disease and home infusion. We recently hired a deeply experienced payer account team who engaged with U.S. commercial and federal payers to help streamline access for launch. We look forward to building the customer-facing team for our U.S. launch in 2025, and we expect a very efficient footprint of around 50 FTEs. We recognize SMA is a global disease, and our goal is to bring apitegromab to all patients who can benefit. We are progressing with our global expansion, have established our EU entity, and continue to engage with EU healthcare professionals as we finalize the preparation for the marketing authorization. And there is more.
SMA represents only the beginning for apitegromab, and plans are underway to expand the clinical development in adjacent neuromuscular conditions where muscle weakness is a hallmark of the disease. The innovation that continues to emerge across our industry, coupled with success of apitegromab and SMA, provides the opportunity to impact more patients and for apitegromab to potentially become a part of the treatment regimens in these additional rare and devastating neuromuscular conditions. We are charting a course for a multi-billion-dollar neuromuscular franchise, starting with apitegromab and SMA, with expansion to other adjacent devastating neuromuscular disorders, and with more innovation on the way. Now, turning to our cardiometabolic programs, it has been absolutely extraordinary to see the impact of the innovation from our industry, as demonstrated by how the approved GLP-1 receptor agonists have transformed the management of weight loss.
Obesity is a top global public health issue, and the potential public health impact of these highly effective therapies is incredible. With an estimated 40 million people projected to receive treatment with GLP-1 receptor agonists by 2029, the market is anticipated to generate over $100 billion in sales. With the extraordinary amount of weight loss seen with the current GLP-1 receptor therapies, a substantial amount of lean muscle also accompanies the weight loss. Given the role that muscle plays in metabolism, increasing basal metabolic rate, improving insulin sensitivity, as well as maintaining strength, mobility, and overall health, we believe that our highly selective approach to blocking myostatin, when added to the GLP-1 receptor agonists, can enhance their profile by preventing the loss of lean muscle mass without introducing additional toxicities, leading to healthy and sustainable weight management.
We've taken a parallel path with our highly selective anti-myostatin portfolio by conducting EMBRACE, a proof of concept study with apitegromab, and at the same time advancing SRK-439, our highly selective novel anti-myostatin designed specifically for cardiometabolic indications. This strategy has resulted in two important milestones in 2025: the readout of EMBRACE study next quarter and the filing for the IND for SRK-439 Q3. Starting with SRK-439, our research team has generated a robust set of non-clinical studies using the industry standard diet-induced obesity mouse models to study SRK-439 in combination with several GLP-1 receptor agonists and have generated data demonstrating that SRK-439 can preserve lean mass with further reduction in fat mass, improve metabolic parameters, attenuate the fat mass regain after stopping the GLP-1 receptor agonist, and demonstrated greater potency in a direct comparative study with an anti-ActRII antibody.
Overall, the data suggest a best-in-class profile for 439, which has been featured at key scientific conferences in 2024. In addition, the research team recently conducted a study with SRK-439 in combination with tirzepatide, a combination highly relevant for our EMBRACE study. As shown in the figure where the X-axis has changed from baseline and lean mass, we saw the expected decrease in lean mass with tirzepatide alone, the orange bar, but when used in combination, SRK-439 protected from the tirzepatide-induced muscle loss in a dose-dependent manner, starting at the lowest dose studied. There was also dose-dependent enhancement of fat mass loss, improving overall body composition. And to add to our potential best-in-class profile for SRK-439, our highly selective approach to blocking myostatin is designed to optimize benefit-risk as well. The figure illustrates why it is so important to selectively target myostatin and only myostatin.
Given the complex biology and the shared receptors, strategies that target the receptor or activin directly, or other less selective approaches such as ligand traps, can result in unwanted toxicities, an outcome that does not occur when blocking only myostatin. Now, turning to the EMBRACE study, EMBRACE is a randomized double-blind placebo-controlled study in overweight or obese participants comparing apitegromab plus a GLP-1 receptor agonist, either tirzepatide or semaglutide, to placebo plus a GLP-1 receptor agonist. The primary endpoint is a change from baseline in lean mass, as assessed by DEXA scans measured at the end of week 24. Key secondary endpoints include weight loss and safety, with exploratory endpoints including cardiometabolic profile, body composition, and physical function. We also included an additional 32-week exploratory endpoint measured eight weeks after stopping therapy.
The study rapidly enrolled 102 participants, and although the study was designed to include either semaglutide or tirzepatide, due to the speed of recruitment and the timing of semaglutide supply, all subjects in the EMBRACE study received tirzepatide as their GLP-1 receptor agonist. The EMBRACE study aims to demonstrate that treatment with apitegromab can lead to preservation of lean mass when compared with tirzepatide alone, safety and tolerability, can we avoid adding any additional toxicities to the tirzepatide profile, and to explore functional and metabolic measures for inclusion into the SRK-439 program. EMBRACE completed enrollment in September of 2024, and as I stated, we are on track to report top line next quarter. And finally, to put the EMBRACE into context, this figure is from the tirzepatide SURMOUNT-1 study and displays the % change in body weight over time.
The area shaded in green represents the first 24 weeks of the study, the time period corresponding to the efficacy assessment in EMBRACE. As can be seen, accompanying the impressive weight loss during this period is a loss of about 11% lean mass. We are excited about the potential for a highly selective approach to blocking myostatin to address this problem, and we look forward to reporting out the 24-week data next quarter with subsequent release of the 32-week endpoint and other exploratory endpoints at subsequent scientific medical meetings. So in closing, we are well on our way to delivering on our mission to bring transformative medicines to patients and leveraging our R&D success to build a multi-billion-dollar company.
We have taken our scientific insights and selectively targeted myostatin and advanced apitegromab from discovery through clinical development, demonstrating apitegromab to be the first and only muscle-targeted therapy to show motor function improvement above standard of care therapy. We have demonstrated our ability to deliver on time or ahead of schedule for all of our goals we set in 2024, and our preparation for commercial launch is no exception. As we drive for a world-class launch of apitegromab, we continue to expand into other neuromuscular indications as well as advance our cardiometabolic programs. So to close, 2025 will be a transformative year for Scholar Rock. We look forward to reporting on our progress toward achieving our 2025 key milestones as we drive to commercialize and expand the development of apitegromab and advance our cardiometabolic programs. Truly a very, very exciting time and very exciting year for Scholar Rock. Thank you.
For the Q&A, I'd now invite Ted Myles, our Chief Operating Officer and CFO, to join me.
Great. Thank you, Jay. Welcome, Ted. I thought I would start the conversation with kind of a, I think, what is a kind of a key question now that you've had these data for, I don't know, around about three months now. What is the feedback that you're getting from physicians and institutions post the top-line SAPPHIRE results and how they will specifically use the product if and when it's approved?
Yeah, so far, not surprising given the fact that this is a new therapy. There's been enormous enthusiasm and excitement around having an additional therapy to treat in SMA. As patients are already identified, our engagement right now is clinicians are already beginning to think about how they would incorporate it into practice.
Again, I think from the data that we shared, the additive functional improvement, the preservation and maintaining of the function, those are clear areas of need that these clinicians face every day as they take care of children and young adults and adults with this condition. So very much looking forward to having the opportunity to incorporate it into practice.
The October press release and presentation was just a top line. Can you talk a little bit more about what you're going to present to us with the fuller results? I think those were planned for early 2025 and where we could see such results.
So these data deserve being represented in a medical conference and scientific discussion, and so we absolutely have plans to share more details around the SAPPHIRE study in conferences coming up this year.
And of course, we're also interested in getting information out and published, etc. So there's a lot of work going on for that. The typical conferences that we have been participating in over time have been the MDA Conference and QSMA. So we can begin to look and we'll give more detail as we get closer to the conferences announcing acceptance of abstracts to share more detail of when and where. But we're really very excited to get these data out in a very fulsome way.
Can you talk a little bit about what exactly you believe the indication statement is going to look like in your label and what are you proposing the regulators and what are the pushes and pulls there to maybe a more broad label versus maybe a little bit something that's a little bit more restrictive?
Yeah, so to start, I think when it comes down to the final label and indication, obviously that's a subject for FDA review and discussion. In typical fashion, as we think about putting a regulatory application together, one takes a look at what's the prior precedent for indication statements from other approved therapies. I think it's very, very interesting and insightful to look at the labeled indications for the existing treatment. The way I believe FDA has approached SMA, they see it as a single disease. As a result, they have a fairly structured approach to their labeled indication, which is for the treatment of pediatric and adults with SMA, considering they see it as a single disease. If you look at the current labels, there's no granularity around SMA type or ambulatory status, etc.
It's for the treatment of SMA in large part because they see it as a single disease. At least that's our understanding. So with that, I think we would think about going forward with our labeled indication, building off of that foundation of that prior precedent labeled indication structure with the nuance that we've studied children two and above. And so there may be a qualifier for two and above in that pediatric statement. Again, to amplify what I said in the prepared presentation, we're keen to open up that under two study to fully explore all the opportunities for even the youngest with SMA, but kind of going in standard label convention for the treatment of. And at this juncture, we'll see as we go through the regulatory review process, but I think we're on solid ground with that going forward.
Okay. And I love a patient funnel question. So if we think about U.S. and Europe here, right, in terms of the patient pool that would likely be an addressable patient at the time of launch, can you walk us through those numbers? And what I'm really interested in too is in patients that are age over two, what portion of those patients are currently on risdiplam or nusinersen?
Yeah, so maybe I'll start and then Ted can kind of amplify a little bit. But what I've shared is that the current market is $4.5 billion across the three therapies. And so the starting point as we think about introducing apitegromab is all of those untreated patients. So we're already starting with an established patient pool that are receiving therapy. And from our data, we believe that we can actually enhance their function, sustain their function, and maintain it over time. So that would be the starting point.
As you think about within the age group, the majority of patients receiving current therapy probably fall within the 2 to 21 group. I think adults in SMA have been a bit disadvantaged by the timing of the absence of newborn screening and the initiation of therapy. That's a group that I think is coming back more and more into treatment as we see additional innovation, but the starting point is the bulk of the patients on treatment fall within that age group, and Ted, do you want to amplify?
Sure, sure, so about 25,000 patients, U.S. and Europe combined. Two-thirds in the U.S., two-thirds of those patients are already on treatment. So that's first, and as Jay mentioned, within the treatment paradigm currently, the 2 to 21 group is the most treated, over 80%, so that's certainly where we'll go first and where we see a lot of receptivity.
But as Jay said, the older patients really need help as well. Yeah. Okay. How do you get this launch right? Well, planning, execution, top talent. I think what's really key, having a great drug. We think we've got that. We've been planning for success for more than 18 months. We were living in a world really of pre-data and post-data. The plans were ready, ready to go, well thought. As soon as we had data and then capital, we hit go and started executing. So things like commercial supply, the distribution channel, expanding the MSL force so we could engage really, really effectively with all the top SMA centers. Deep long-term investment in the relationships with Cure SMA, U.S. and Europe, and the other advocacy groups and the broader patient community. One example of the execution, we put data out on the 7th. We financed a couple of days later.
The next Monday, we kicked off budget and planning meeting, and our VP of sales was in the seat on the payroll. So that's just one example of how thoughtful we've been about mapping out the plan and then executing the plan. We think we've got an incredible opportunity here. If you were to walk the halls of Scholar Rock, you'd feel the energy, and it's very patient-centric energy to make sure that we get this launch absolutely right, and patients are waiting, so we're ready to serve.
Yeah, and to go back to some of the points that I was raising, I think the fact that the community is looking for this therapy, they've been aware of this coming, the fact that patients are identified already on treatment, I think those parameters really set the stage for us to really be highly successful.
It really does start with having the right medicine and the absolute need. To Ted's point, operationally, we are just running. I kind of made that point in my prepared remarks. We've been very, very successful with execution since I've been leading the company. Not to say that didn't occur before. We have delivered, delivered, delivered, met our goals, established the timelines, meet them, try to beat them. We're doing that. I started again the talk because we're a mission-driven company, because there is passion and commitment across everybody in our organization. As we're bringing it on to be part of launching a new medicine to change the treatment paradigm in SMA, it's just palpable within the company.
I think all of those things really set the stage for us to run to really maximize the chance that we can really make a difference.
Okay. And with this all in mind, what is the right way to think about the ramp? Because I feel like next JPMorgan, you may be approved, right? Or you should be approved, so.
Well, [crosstalk] let's start the shaking up.
Exactly. So how are you? Maybe it's a little bit of a premature question, but based on what you know and what your expectations are around the label, how are you thinking about that?
It is a premature question, but we can point to the, look, we talked about top line, the way we see the global opportunity, right? $2 billion plus. We should tip our hats to the groups that launched the SMN correctors, right?
They really set the market for us, did great things for patients, and the way we think about it, they really paved the road. We know where the patients are. We know how to get to them. Payer access has been established for these drugs, but it's important. This is a progressive disease. It's a neuromuscular disease. The neuro piece is very well taken care of. The muscular piece needs a lot of help, so that's where we're going to go. In terms of the guidance on the ramp, that's a conversation for a different day.
I believe you did double your peak sales potential. I think previously you were quoting around $1 billion plus, and now it looks like $1 billion. Yeah. [crosstalk] Yeah. $1 billion. No, I said $1 billion plus, and then today you were quoting $2 billion plus. So can you talk to us a little bit about what prompted the decision?
Yeah, so let me start with that, and then you can add to that. I think what we tried to share as we described it is that if you think about the $4.5 billion current market opportunity for these existing therapies, right, with the majority of those patients already on treatment. And when we were describing it was a billion-dollar-plus opportunity, we said that even before we had data readout and before we fully understood sort of how competitive the landscape was going to be with us going forward. I think now, as I was trying to lay out earlier, there's a treatment paradigm shift. There's no other muscle-targeted therapies coming into view, at least in the near term.
I think that really positions us to really be able to take full advantage of the data that we've generated and the need. We've updated that to really take that into consideration. Again, those are proposed peak global opportunity revenues, right?
Not necessarily. The $1 billion was put out in May, right? Here we are in January. A lot of good has happened for us to progress the opportunity since then. We thought it was prudent to update.
Okay. You talked a little bit about the OPAL study. How confident are you that this study is poised to succeed?
Very. I think from the need that we have to study under two, it's really driven principally by understanding how to guide dosing for those small infant and under two-age children.
From the classic typing of development and pediatric development, you establish your dose and PK/PD relationship in the SAPPHIRE trial. Then for OPAL, the primary goal really is to understand PK/PD effects to guide dosing. Those parameters are something we anticipate being able to meet to just guiding and running the program. We'll certainly build in some additional assessments of efficacy built along that as well. But the principal goal is for PK/PD and guiding dosing.
Okay. Maybe we'll segue here and talk a little bit about your efforts in obesity. You talked a little bit about the EMBRACE study and the design in your prepared remarks, Jay.
But are you able to get into any more specifics around your powering assumptions, what you're specifically looking for in terms of from a clinical benefit perspective that you think would be exciting for patients and for the company?
So as I laid out the trial design and deliberately put in the graph on the tirzepatide weight loss, right? I want to kind of imagine that. We designed the study as a proof of concept, so really was more directional. And we're actually looking to see whether or not we can change the slope of that curve, which is downward on the lean mass, and change that and basically have white space between what we can do in preserving lean mass from what's expected. And that's to guide further thinking around our program in 439. So we have 102 patients enrolled. That's a very nice number.
That'll give us really good insight into how to really discern the change in lean mass from baseline across the groups. And we're nearing the opportunity where I can just show you the data rather than describing the powering assumptions for it. So that's definitely coming soon. But to be clear, we give a lot of thought about the ability, the timing of when we could do that endpoint. We think 24 weeks was adequate. 100 patients on definitely should show a difference we can believe, given what we're seeing from our non-clinical data. And excited to see. I think it's very interesting. It's a very interesting time, I think, with the GLP-1 receptor agonist. I do think over time, lean mass is going to really be relevant. It's absolutely relevant, I think, for a certain subgroup of patients for sure.
I think the more that we see and the longer treatment is going on, I think this is going to become even more clearly recognized. And we're at the front end of that, which is exciting.
And assuming the study comes up with positive data here, can you talk to us a little bit about how soon you're going to be able to bring SRK-439 into the clinic? What are the gating factors at this point between now and 3Q? Are you pretty much ready or?
Yes. I mean, that's why I said our approach in parallel was to get EMBRACE out so we can demonstrate that our very selective approach to targeting lean mass will work or targeting lean mass down to preserve lean mass. But we've been doing the IND enabling work for 439 over the course of this time. So we're now finishing up.
Typically, those have tox studies you need a certain amount of time on. Those are on track, and we're scheduled to submit the IND in the third quarter, which will be exciting because I think we can, in that opening IND, and even think about we have very rapid onset of action with myostatin. I think we have an opportunity. If you think about our SAPPHIRE data, we saw an effect on function within that first functional measure, which was at eight weeks. So we turned the cellular machine around pretty quickly, and so we can even demonstrate in the IND opening studies our ability to affect lean mass. So we're excited about it, but we're on track for Q3.
Okay. How does BD configure into your key priorities for 2025?
I think you've talked about commercializing yourself in the U.S. and Europe, but how about all the other countries where there are SMA patients? Is that something that's more of a 2026 priority for the company?
Well, as I mentioned in our remarks, I think our goal is to really have an apitegromab available to all patients with SMA regardless of region, right? So that's definitely a stated goal for us. If you think about what we've said previously and what we're going to reinforce is that we feel like we're highly leveraged or positioned to be very successful in the U.S. We think that we can commercialize on our own in the U.S., I mean, in Europe with a classic, very thoughtful approach to key markets and kind of build around that.
From the rest of world global reach, certainly we'll need an opportunity to have others who have been there and can help us with that. So that work is kind of underway as we set the stage. But first and foremost, it's regulatory approval, gear up for U.S. launch, build out Europe, and in parallel, develop the opportunity to really have this as a global product. Because the global sales that we're describing are really I mean, the sales we're describing are global, so we're really driving to do that.
Okay. Maybe final question for me. Can you talk a little bit about the cost structure here, how we should be thinking about the OpEx line as you move into commercializing yourself?
Sure. So OpEx is going to go up, right? We are scaling up to become a commercial company.
We're not forgetting about the research and the development investments. We're continuing to move the pipeline forward as well. Obviously, SAPPHIRE was an expensive proposition, right? That has run its course, and that's coming down. We're continuing to invest in a long-term study. We rolled a lot of the SAPPHIRE patients, virtually all the SAPPHIRE patients, 98% into ONYX, right? We think it's very important to continue to support those patients. ONYX also includes the more than 90% of TOPAZ patients that have continued on therapy for over four years, many approaching five years. While the phase three is over, we're continuing to invest in the development and then also investing in research. Jay talked about expanding apitegromab's reach. Those are small investments to start, right, to explore where else apitegromab can help patients and other rare neuromuscular diseases.
Obviously, commercializing is an expensive proposition. We had a very successful financing in Q4 of last year, thanks to, we had help from a pretty terrific bank. That extended our runway into Q4 of 2026, and that gives us a really very solid foundation to have a successful launch. All right.