Scholar Rock Q1 2025 Earnings Call Transcript

Quartr
Provided by Quartr
May 14, 2025

Key Takeaways

  • Phase 3 SAPPHIRE trial delivered a statistically significant and clinically meaningful motor function improvement in SMA, and the FDA granted the BLA priority review with a September 22 PDUFA date.
  • ScholarRock is preparing for a US commercial launch of ipilimumab in Q3 2025, building a ~50-person sales and patient support team and engaging payers ahead of launch.
  • The company closed Q1 with $364.4 million in cash and an additional $100 million debt facility available, providing runway into 2027 to support the upcoming launch and pipeline investments.
  • EMBRACE proof-of-concept study readout in June 2025 will evaluate upitigramab’s potential to preserve lean mass in obesity patients treated with GLP-1 therapies, guiding future cardiometabolic efforts.
  • ScholarRock plans to file an IND for subcutaneous anti-myostatin antibody SRK439 in Q3 2025 and is exploring additional neuromuscular indications beyond SMA, including DMD and FSHD.
AI Generated. May Contain Errors.
Earnings Conference Call
Scholar Rock Q1 2025
00:00 / 00:00

Transcript Sections

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Operator

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 a listen only mode. Following the presentation, we will conduct a question and answer session. To ask a question during the session, you will need to press 11 on your telephone. You would then hear an automated message advising your hand is raised.

Operator

To withdraw your question, please press 11 again. Please be advised that today's conference is being recorded. I would like now to turn the conference over to Skyler Rock. Please go ahead.

Rushmie Nofsinger
Rushmie Nofsinger
Vice President of Corporate Affairs & Investor Relations at Scholar Rock Holding

Good morning. I'm Rashmi Nopsinger, Vice President of Corporate Affairs and Investor Relations at Scholar Rock. With me today are David Halal, Chief Executive Officer Akshay Vesnau, President of R and 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 two, David will provide introductory remarks and provide a general business update.

Rushmie Nofsinger
Rushmie Nofsinger
Vice President of Corporate Affairs & Investor Relations at Scholar Rock Holding

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.

Rushmie Nofsinger
Rushmie Nofsinger
Vice President of Corporate Affairs & Investor Relations at Scholar Rock Holding

I encourage you to go to the Investors and Media section of our website for our most up to date SEC statements and filings. With that, I'd like to turn the call over to David. David?

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

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 up tinigromab to patients and families living with and suffering from SMA in The US, Europe, and around the world. We were very pleased with the positive phase three 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.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

And in Q1, to underscore the body of evidence for ipilimumab for patients with SMA, we were gratified that our BLA was granted priority review by the FDA with a September 22 PDUFA date. The team at ScholarRock is working urgently to prepare for our US 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 Scolorox capabilities as we advance our mission to deliver ipilimumab to patients with SMA. This is what we know well and what we do well.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

We joined ScholarROC 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 thirty five thousand 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 US 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 ipilimumab should have access to ipilimumab. I am confident that the global opportunity with ipilimumab 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.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

While we are very focused on our large opportunity to serve patients with SMA, we are continuing to evaluate expanding the study of upitigramab 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 EMBRAZE proof 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 ones have brought needed innovation to patients with obesity and cardiometabolic disorders.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

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 upitigromab to patients and families with SMA. We are focused on delivering for patients, with Akshay leading efforts in collaborating with US and EU regulators.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

Key, applying a similar approach from VIBGAR and MG to the commercial opportunity for ipilimumab. And VICAS, 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 and D progress. Akshay?

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

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 ipilogrelimab, our highly innovative anti mice satin antibody, go from bench to bedside with our remarkable SAFIRE data. SAFIRE showed that ipilimumab 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 whilst patients on placebo worsened.

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

Importantly, patients treated with ipilimumab 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 SAFI data suggests that epitogromab has great potential to provide clinically significant benefit to patients with SMA despite the use of background SMA targeted 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 ipilimumab achieved clinically meaningful and consistent benefit in motor function across prespecified 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.

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

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 ipilimumab was granted priority review by the agency with a September 22 PDUFA date. We feel the potential clinical benefits of ipilimumab as demonstrated by a phase three trial are underscored by the FDA's priority review designation. By definition, a priority review designation by the agency conveys the capacity of epitigimod to potentially impact unmet need in SMA by either being a treatment for a serious or life 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.

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

With the success of Saphyr, we're just beginning to tap into the broader potential of our truly innovative myosatin platform. There's much more we can do with the promise of upitamab and our platform by delivering advances in 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 creates options for our pipeline. We remain on track to file the IND application for SRK439 to support the first in human study in Q3.

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

Now earlier, David mentioned the potential role Scholar Ops can play in addressing lean mass in cardiometabolic diseases. As we all appreciate, whilst GLP-one's 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-one is due to the loss of muscle. Looking forward, it will be important to preserve muscle from the viewpoint of the associated metabolic benefit and a healthier approach to weight loss. Our EMBRACE study is our ongoing Phase two trial to investigate preliminarily the potential of further developing our highly selective antimicrobial action approaches in patients with obesity with the goal of reducing the loss of lean mass.

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

We look forward to the upcoming readout of our initial data from IMbraCE 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 upilumab in Q3 twenty twenty five and advance The EU Court approval in 2026. '2, initiate a study of ipilimumab for infants and toddlers with SMA under the age of two starting in Q3. '3, file an IND for SRK-four 29 in the third quarter.

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

And finally, our clinical development plans for ipilimumab in additional neuromuscular indications. With that, I'll turn it over to Keith to provide a commercial update. Keith?

Keith Woods
Keith Woods
COO at Scholar Rock Holding

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 22 PDUFA date for epitigramab 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.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

With epitogromab, 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 ninety percent of patients identified that new SMA treatment options improving muscle strength is an important need.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

Our market research and interactions with healthcare professionals tell us that eighty percent of treating neurologists agree that preserving muscle should start as early as possible in treating patients living with SMA. Today, there are approximately ten thousand 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, epitogromab 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 thirty five thousand 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.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

At ScholarRock, we have the opportunity to make a meaningful difference for both children and adults living with SMA, first starting in The US, 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 Scholar Rock had the opportunity to meet with patients and their families at the Cure SMA Walk and Roll in Boston.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

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.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

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-twenty twenty five, well ahead of our potential launch in late September. Finally, we believe that epitigramab has the potential to be a first in class, best in class therapeutic to establish a new standard of care in SMA.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

Now, I will turn the call over to Vikas. Vikas?

Vikas Sinha
Vikas Sinha
CFO at Scholar Rock Holding

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 apetigramab 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 $364,400,000 During the quarter, we continued to increase our investments in commercial readiness and inventory build out.

Vikas Sinha
Vikas Sinha
CFO at Scholar Rock Holding

As we look ahead, we are prioritizing the commercial launch and ongoing clinical programs. We have an additional $100,000,000 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 build out and thoughtful capital allocation to advance our pipeline. With that, I will turn it back to David.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

Thanks, Vikas. In closing, we are acutely focused on the key priorities that will enable us to build and scale ScholarRock into the next global biotech powerhouse. First, regulatory approvals and the upcoming US launch of epitogromab for patients with SMA, followed by a series of country launches in the coming years. Next, develop upitigromab for additional rare, severe and debilitating neuromuscular diseases. And finally, phase our capital allocation and investments thoughtfully to support our high value commercial and development initiatives.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

On behalf of every member of the ScholarRock team, we are deeply aware of our responsibility to patients and their families, and will work with urgency to ensure that no patient with SMA is left behind. With that, we'll now open the line for questions. Operator?

Operator

The first question comes from Allison Breztsel with Piper Sandler. Your line is now open.

Allison Bratzel
Allison Bratzel
Senior Research Analyst at Piper Sandler Companies

Hey, good morning team, and thanks for taking the questions. Just two from me. Know, ahead of etitigimod launch, could you just frame for us how your discussions with U. S. Payers have gone?

Allison Bratzel
Allison Bratzel
Senior Research Analyst at Piper Sandler Companies

You know, how receptive are they to coverage of combination therapy for both an SMN targeting and a muscle targeting therapy in SMA patients? And can you also describe that feedback for ex U. S. Payers and governments as well? And then second, could you just characterize interactions with FDA on the upadacumab review?

Allison Bratzel
Allison Bratzel
Senior Research Analyst at Piper Sandler Companies

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.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

Thanks, Allie. 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 sort of thinking about and sort of doing as it relates to in general, really what we're talking about is access for a potentially transformative therapy for a small population of patients. And we would sort of expect, as we think about establishing access plans that we consider sort of pricing for our therapies that would be reflective of rarity of the disease, the severity of the disease and the value that therapy would provide to patients in this specific case patients with SMA. And I think one of the other things that Keith and I think a lot about is also given the rarity of disease.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

We would expect that the budget impact to any one single payer in The US, or even globally would be very limited for the high value proposition As it relates to the engagement with payers, largely in The US, I'll turn it over to Keith for some more commentary.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

Thanks, David. So Ali, what we know in speaking with payers is that with SMN targeted therapies alone, patients can still have progressive motor function loss. And at the MDA in Dallas in March, we heard that a proportion of patients are already receiving more than one SMN therapy, so SMN 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 SMN targeted therapy, we see improvement consistent across all age groups of two to twenty one.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

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.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

Maybe a closer look at the regulatory.

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

Sure, thanks David. So thanks, Alex, for the question on FDA 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 22 PDUFA date.

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

So really, we're seeing no issues in to ipilimumab in the SMA approval. And we're also delighted that FDA Commissioner Martin MacKerry actually made a point that with all the changes, one of the main areas of focus continues to be rare disease and delivering on 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.

Operator

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

Michael Yee
Michael Yee
Managing Director at Jefferies Financial Group

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 twenty four and thirty two 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?

Michael Yee
Michael Yee
Managing Director at Jefferies Financial Group

And then just holistically, the prior CEO Jay has mentioned, like 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.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

Yeah, great questions, Mike. As Akshay 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 Akshay for sort of a closer look at what one might expect in terms of, as you were saying, the twenty four week endpoint, but then the eight week withdrawal of the therapies as well. Akshay?

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

Yeah, thanks, Mike. So the core of it, we'll be providing the twenty four week data. That's the main study. We'll certainly provide any necessary follow-up information that we have that helps us understand and helps everybody else understand the potential of an anti myostatin approach in the obesity space. We're excited to test this hypothesis preliminarily.

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

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 to a third of the weight loss is due to muscle. And so the primary focus will be the twenty four week data change in lean mass, the safety and helping us understand the path forward. So I'll leave it at that, but it's not so long to wait until we get the data.

Operator

And the next question comes from Tess Romero with JPMorgan. Your line is open.

Tessa Romero
Tessa Romero
Equity Analyst at J.P. Morgan

Hey David and team, thanks so much for taking our questions this morning. So in The U. S, 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?

Tessa Romero
Tessa Romero
Equity Analyst at J.P. Morgan

And at this time, what has the agency said about the need or lack thereof for an AdCom? Thank you.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

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

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

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. As to the detail of when and how the various interactions occur, 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 PDUFA date is based on a series of very constructive conversations. So I'll leave it at that and more news as we get it.

Operator

And the next question will come from David Nierengarten with Wedbush. Your line is open.

David Nierengarten
Managing Director - Equity Research at Wedbush Securities

Hey, thanks for taking the question. I just had one on the additional potential indications for pidegroom. As I recall, there's a lot of exploration and preclinical models, at least of DMD and related disorders. Is there any particular new development or new treatments that you think are particularly attractive to bring up anagram app forward into one of the other neuromuscular indications. Just this kind of help us out with thinking about the expansion plans. Thanks.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

Thanks very much. David. Akshay.

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

Yeah. Thanks, David. So, look, I think with ipilimumab and a really unique and potent anti mystatin platform, we have an incredible opportunity to not just help in SMA across the spectrum of disease there, but well beyond that into other neuromuscular disorders, 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 DMD and FSHD models, two very important muscular dystrophies. We've shared those data.

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

People are very excited about them. The fact that we've epitogimab as having the ability to put on the degree of muscle that it can in such a serious disease SMA obviously bodes well in these other neuromuscular disorders. There are other indications beyond DMD and FSHD. ALS is one that we can talk about as well and we've got some thinking there. And there'll be more beyond that.

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

But I think at the current time, we're very encouraged by the model data both from DMD 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 ipilimumab clearly has to be investigating additional indications.

Operator

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

Gary Nachman
Gary Nachman
Managing Director - Equity Research at Raymond James Financial

Thanks and good morning. So, you get the SMA 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? How much commercial supply will you have at launch just in terms of meeting the demand? Then also if the proof of concept data in obesity are positive, just talk about the likely next steps with four thirty nine after you file the IND.

Gary Nachman
Gary Nachman
Managing Director - Equity Research at Raymond James Financial

What type of Phase I study will you run and how long before it can potentially move into a phase two? Thanks.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

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 to serve patients at approval. Keith?

Keith Woods
Keith Woods
COO at Scholar Rock Holding

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-twenty twenty five, so we should have a couple of months prior to launch.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

In regard to how soon will we launch after September 22 date, we're gonna 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.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

Thanks, Keith. And regarding like sort of the EMBRAICE study, the proof of concept, and I think Gary, as you noted, how do we think about April? As Akshay mentioned, we're moving forward with an anticipated IND for SRK439 in 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 SRK439 is we have optionality.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

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, April, 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 focus on what we see in this first exploratory readout in the EMGRAVE study, and then we'll provide you all here in the coming month or two, further plans for SRK-four thirty nine.

Gary Nachman
Gary Nachman
Managing Director - Equity Research at Raymond James Financial

All right, great. Thank you.

Operator

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

Srikripa Devarakonda
Srikripa Devarakonda
Analyst at Truist

Good morning team. Thank you so much for taking our question today. Given that we're sort of close getting close to the PDUFA date, 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.

Srikripa Devarakonda
Srikripa Devarakonda
Analyst at Truist

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 exact numbers, but qualitatively.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

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've 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 ipilimumab, 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.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

In all countries, we would expect that the price of epitigromab 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 ipilimumab may be able to provide to patients and families with SMA. And then as I also noted a bit earlier, we think that given the rarity of the disease, ipilimumab would not really impact 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 be in a position of strength when we think about the launch of ipilimumab globally for the thirty five thousand patients in the world that have received an SMN targeted therapy. For a closer look at your first part of your question on launch dynamics and kinetics, I'll ask Keith to comment.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

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, but one thing that I'm very excited about the SMA marketplace is that one hundred percent of newborn screening in The US, and as we noted in the prepared statements that we know of the two thirds of SMA patients of the ten thousand 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, it's highly concentrated because of the Cure SMA model.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

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 we expect that between that as well as applying for a J code that we would receive sometime six plus months later, that we'll prepare for a consistent and steady launch.

Srikripa Devarakonda
Srikripa Devarakonda
Analyst at Truist

Great, thank you so much.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

Thanks, Kripa.

Operator

The next question comes from Mark Fromm with TD Cowen. Your line is open.

Marc Frahm
Marc Frahm
Biotechnology Equity Research Analyst at Cowen

Thanks for taking my questions. Maybe just following up a little bit on Kripa's question before, just on launch trajectory. As you mentioned, the SMA population now has one hundred percent newborn screening in The U. S. They've gotten very concentrated into these centers of excellence to receive SMN therapy.

Marc Frahm
Marc Frahm
Biotechnology Equity Research Analyst at Cowen

Those would seem to be kind of tailwinds to the launch relative to prior SMA launches. But then while there's certainly unmet need, maybe it's not quite as dramatic as it was before the original estimate correctors launched, which would maybe be a headwind. I mean, you think those net out and this we should look at prior SMA launches as a good proxy for the trajectory that epitogrel might have? And I guess to the extent that you don't think those are good proxies, is there another other launches out there in the rare disease space that you think maybe are more analogous to what we should expect with epitogromat?

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

It's a great question, Mark. I think first of all, one of the things that our team is always focused on as opposed to 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 a couple of things that I would just underscore. SMN targeted therapies in and of themselves with longer term data that's been presented absent of scholar rock 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.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

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 scale, a gain of motor function. So we do think that there's a lot of urgency still for patients. And I think Keith even commented on how that was underscored at the last month's presentation by Cure SMA of what patients and families were looking for. At the same time, we really want to play the long game.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

Independent of sort of launch trajectory, we want to build a team and a model to serve patients immediately, and then over time for 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?

Keith Woods
Keith Woods
COO at Scholar Rock Holding

Yeah, I mean, you bring up the point of this, the launch compared to other SMN targeted therapies when there was nothing else available. Okay, so they truly had no other treatment. So I think this sense to urgency to be able to get to that product was great. As we take a look at the information, we find that patients that are on SMN targeted therapies, most of them see their doctor on average two times per year. So it's not like September 22 happens, and they have an appointment the next week.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

So I just wanna guide you on that. I agree with your statement that the fact that we know where these patients are is an advantage. However, there are gonna be some headwinds, as David mentioned. Thanks.

Operator

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

Malcolm Hoffman
Malcolm Hoffman
Senior BioPharma Equity Research Associate at BMO Capital Markets

Mark Moffman on for Evan. Thank you for taking our question. Recently, we had noted some stronger rebound in SPINRAZA 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 eptinezumab being used in conjunction with SPINRAZA? Are you internally expecting any longer term growth from SPINRAZA that may benefit eptinezumab upon launch? I appreciate it.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

No, it's a great question. I think, you know, one of the things that we would note is that as Akshay shared earlier in the SAPPHIRE study, really in a pre specified way, depending upon which SMN targeted therapy, you know, a patient were received, you know, we feel like we can help all patients independent of whether or not they received SPINRAZA or risk to plan. We'll continue to sort of assess the nearly $5,000,000,000 annual market across the three different SMN targeted therapies, whether or not it's the highly innovative gene therapies or GEMMA or the other highly innovative SMN targeted therapies like SPINRAZA and Risdiplam. And we would just expect to follow those dynamics, but recognize with the SAPPHIRE data. And then as Akshay noted earlier in today's call, the upcoming UNDER-two study, the OPAL study, where we would expect more experience for opitigramab for patients who have received Zolgensma, 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 SMN targeted therapies over time. Thank you for your question.

Malcolm Hoffman
Malcolm Hoffman
Senior BioPharma Equity Research Associate at BMO Capital Markets

Appreciate it. Thanks.

Operator

Our next question comes from Andres Maldonado with H. C. Wainwright. Your line is open.

Andres Maldonado
Vice President at H.C. Wainwright & Co., LLC

Hi, everyone. Thanks for taking my question. Just a quick one for me. Given the progress towards U. S.

Andres Maldonado
Vice President at H.C. Wainwright & Co., LLC

Launch and your ongoing readiness in Europe, I guess how are you evaluating the potential to secure a commercial partner for Europe? In this context, obviously you want to fire on all cylinders across the pipeline, but how would you prioritize the additional indications for pitimote or bromine in situation? Thank you.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

Yeah, thank you. I think we noted this just a few weeks ago on our April 28 call, given the experience that, you know, this collective team has had, you know, between Alexion Alnylam argenx. 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 US. Feel like this is something that we know well, and in fact, do well, and we'll continue to run a playbook that not only do we know the playbook, we feel like we wrote that playbook.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

As it relates to other indications, as Akshay noted, we'll be data driven. The team's been doing some wonderful work in looking preclinically 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.

Operator

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

Dennis Kennedy
VP - Research Analyst at LifeSci Capital, LLC

Thanks for taking the question. As we look towards the EMBRIGED data in June, could you just help frame expectations for the tirzepatide monotherapy arm? Specifically, how much of the lean mass loss that we'd expect at one year with tirzepatide do you think typically occurs by week twenty four? And 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 tears appetite monotherapy arm?

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

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, you know, twenty four week endpoint and then eight weeks following. But but nonetheless, as actually, 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?

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

Yeah, thanks. So, I mean, tirzepatide consistently has shown loss of lean math as of the other GLP-one. That mass is symmetrical to the overall loss in body mass. And so you can expect that at week twenty four, you know, 25 to 30% of the weight loss that will have occurred in the tirzepatide alone arm will be due to loss of muscle. We're projecting that based on historical data.

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

There's no reason to believe that it would be any different in the context of this study. Now, will that be the maximal amount of lean mass loss at week twenty four? I don't think so because in the pivotal studies, nertezepatide was studied for much longer and overall the impact from muscle mass is even greater. Obviously, using patients lose more weight. But within the context of embrace order to a third of weight loss at week twenty four would be mean mass loss we expect.

Akshay Vaishnaw
Akshay Vaishnaw
President, R&D at Scholar Rock Holding

Your other point about is eight weeks of follow-up sufficient? Well, you know, this study was an exploratory study to understand the potential of epitigamab in a pure, clean, safe potent anti myosapic 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.

Dennis Kennedy
VP - Research Analyst at LifeSci Capital, LLC

Great. Thank you.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

Thanks, Dennis.

Operator

At this time, I am showing no further questions in the queue.

David Hallal
David Hallal
CEO & Chairman at Scholar Rock Holding

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.

Operator

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

Executives
    • Rushmie Nofsinger
      Rushmie Nofsinger
      Vice President of Corporate Affairs & Investor Relations
    • David Hallal
      David Hallal
      CEO & Chairman
    • Akshay Vaishnaw
      Akshay Vaishnaw
      President, R&D
    • Keith Woods
      Keith Woods
      COO
    • Vikas Sinha
      Vikas Sinha
      CFO
Analysts
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