Scholar Rock Q2 2025 Earnings Call Transcript

Quartr
Provided by Quartr
August 6, 2025

Key Takeaways

  • Positive Sentiment: The FDA has accepted ScholarRock’s BLA for upitigramab under priority review, and a constructive late-cycle meeting suggests the agency aims to complete its review by the September 22 PDUFA date.
  • Neutral Sentiment: During standard GxP inspections, two CDMO sites received observations; ScholarRock and its partners are submitting comprehensive responses and continue to collaborate with the FDA to address any potential reinspection.
  • Positive Sentiment: The MAA for upitigramab has been validated by the EMA, positioning ScholarRock for a potential EU approval near mid-2026, with Germany targeted as the first launch country.
  • Positive Sentiment: The Phase II EMBRAZE proof-of-concept study in obesity met its primary endpoint, showing upitigramab added over 54% lean mass preservation on tirzepatide versus control and maintained an encouraging safety profile.
  • Positive Sentiment: The US commercial launch is on track, with a fully onboarded field team and positive payer interactions—80% of neurologists agree early muscle-targeted therapy is needed in SMA.
AI Generated. May Contain Errors.
Earnings Conference Call
Scholar Rock Q2 2025
00:00 / 00:00

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Operator

Good morning, ladies and gentlemen, and welcome to the ScholarRock's Second Quarter twenty twenty five Business Update Conference Call. This call is being recorded on Wednesday, 08/06/2025. I would like to turn the conference over to Scholar Rock. Please go ahead.

Rushmie Nofsinger
Rushmie Nofsinger
VP - Corporate Affairs & IR at Scholar Rock Holding

Good morning. I'm Rashmi Nafsinger, Vice President of Corporate Affairs and Investor Relations at ScholarRock. With me today are David Hillal, Chairman and Chief Executive Officer Akshay Vashnau, President of R and D Heath Woods, Chief Operating Officer 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
VP - Corporate Affairs & IR at Scholar Rock Holding

Akshay will review our clinical and regulatory progress. Keith will provide an update on our readiness activities for upitigramab and Vikas will provide commentary on our financials. 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 Sellar Rock's expectations, plans and prospects that constitute forward looking statements for the purposes of the Safe Harbor provisions under the Private Securities Litigation Reform Act of 1995. Any forward looking statements represent our views only as of today and should not be relied upon as representing our views as of any future date.

Rushmie Nofsinger
Rushmie Nofsinger
VP - Corporate Affairs & IR 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, Director & Chairman at Scholar Rock Holding

Thanks, Rashmi, and good morning. Thanks to everyone for joining our Q2 update call today. This is an exciting time of great strength and opportunity at ScholarRock. We are scaling for the next phase of growth as a commercial stage fully integrated global biopharmaceutical company. Our three core priorities drive our vision of becoming a global biotech powerhouse.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

First, epitigramab regulatory approvals and following those approvals, The U. S. Launch of epitigramab for children and adults with SMA, followed by a series of country launches in the coming years, starting in Europe with Germany next year. Second, expand upitigramab into additional rare, severe and debilitating neuromuscular diseases. And third, disciplined capital allocation to support our high value commercial and development initiatives.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

With respect to our ongoing regulatory processes, we are working collaboratively with the FDA and European Medicines Agency. We are also urgently preparing for our U. S. Commercial launch as our BLA has been accepted under priority review with a target action date of September 22. As you are aware, GXP inspections are part of the standard FDA review process, including those relating to preapproval inspections, clinical trial site inspections, and manufacturing site inspections.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

These inspections often result in observations requiring responses within the review cycle. The FDA has conducted a full set of inspections and as part of this process noted observations at two of our CDMOs. On Friday, August 1, another pharmaceutical company disclosed observations as part of an FDA general site inspection at their filler, Catalent, Indiana, which was recently acquired by Novo Nordisk. Our fillfinish for upitigramab is conducted at the same site. The general site inspection of the facility was not specific to upitigramab.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

Novo Nordisk submitted a robust and comprehensive response earlier this week to the observations noted by the FDA. For the other CDMO, observations were received at the conclusion of a pre licensing inspection and a comprehensive response will be submitted within the next week. We continue to work collaboratively with the agency. Importantly, the FDA recently completed our late cycle meeting following both site inspections. We are encouraged by the dialogue with the agency at the late cycle meeting where the FDA indicated that they are working towards completing the review of our BLA by our September 22 PDUFA date.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

Earlier this year, we also filed our MAA for upitigramab in the EU, and we continue to work with the European Medicines Agency and expect a potential approval near mid-twenty twenty six. We are planning for Germany to be our first European country launch with an ambition to reach patients with SMA across all of Europe, followed by additional countries in Asia Pacific and Latin America over time. The global opportunity with ipilimumab and SMA alone offers the potential for many years of sustainable growth through the end of this decade and into the next. Along with Keith, Akshay and Vikas and the other leaders at the company, we have bolstered Scolorox capabilities as we advance our mission to deliver ipilimumab to children and adults with SMA. This is indeed what we know well and what we do well.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

In addition to the large opportunity to serve patients with SMA, we will continue to expand our pipeline by planning additional opportunities for epitigramab for children and adults suffering with additional rare, severe and debilitating neuromuscular disorders, which Akshay will discuss in more detail shortly. Importantly, to grow ScholarRock, we are taking a thoughtful, deliberate approach to capital allocation by staging our investments along with our commercial progress in serving the SMA community. Q2 has been exceptionally productive. In the quarter, when Keith joined our team as our Chief Operating Officer, he brought a proven track record of building and leading teams to deliver highly successful rare disease global launches in the neuromuscular therapeutic area, including most recently with Vibgart. Under Keith's leadership, we have assembled an exceptionally experienced, talented and patient centric field team committed to the SMA community.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

Impressively, over just a few months, the team is on board, trained and deployed and are ready to deliver ipilimumab to the SMA community pending approval in September. Despite currently available treatments that have been approved over the past ten years, we are acutely aware that muscle strength and motor function are among the top unmet needs in SMA, which we believe can be addressed with the potential approval of epitigramab, the world's first and only muscle targeted therapy to deliver statistically significant and clinically meaningful improvements in motor function in a pivotal Phase III trial. I would like to now turn briefly to the readout of the positive Phase II EMBRAZE proof of concept study in Q2. The goal of EMBRAZE was to understand the role of targeting myostatin in the treatment of patients with obesity. We are pleased that the EMBRAZE study met the primary endpoint with patients on tirzepatide.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

Epitigramab increased lean mass preservation by greater than 54% compared to tirzepatide alone with a p value equal to 0.001 with an encouraging safety profile. We are very pleased that the highly selective anti myostatin approach from our innovative platform continues to deliver. While we remain focused on advancing epitigramab in clinical development for additional rare, severe and debilitating neuromuscular disorders, EMBRAZE raises the exciting possibility to partner our potent and selective approach to targeting myostatin. As we communicated at EMGRADE's data readout, in addition to SRK-four 39, we have a number of earlier stage research assets, both anti myostatin antibodies and the fusion of those with GLPs, which we think have the potential to be meaningful therapeutic candidates in the future. As we look forward, we remain very focused on the nearly thirty five thousand patients with SMA globally that have received SMN targeted therapies.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

While we anticipate the global launch will commence in The U. S. In Q3, pending regulatory approvals, we are also making preparations to serve children and adults with SMA in Europe, Asia Pacific and Latin America. Our ambition at ScholarRock is that globally, any patient with SMA who can benefit from upitigramab should have access to upitigramab. At this point, 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, and good morning, everyone. SMA is a rare severe neuromuscular disease resulting in irreversible loss of motor neurons and progressive muscle wasting that causes continuous motor function decline throughout life diminishing the independence of both children and adults. There's been progress over the last decade with new therapies for SMA. However, despite the chronic use of these SMN correctors, SMA remains a devastating disease for children, adults and their families living with the disorder. Patients experience muscle wasting that impacts all aspects of mobility: basic activities of daily living like eating, washing, dressing and the ability to live independently.

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

The motor unit has two key components: the motor neuron itself and muscle. SMN target therapies are aimed at preventing motor neuron loss, but muscle, the principal organ effect in SMA, has not been directly addressed. Given that motor function depends not only on neuronal signaling but also on muscle responsiveness, approaches that target muscle from the start are urgently needed. Our SAFIRE trial showed that in those receiving chronic ongoing SMN targeted therapies, apitigramab 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 motor function as measured by the gold standard Hammersmith Motor Function Scale, while 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. In addition, there was a consistent 1.8 improvement in Hammersmith across all ages in our Phase III trial. Along with the encouraging safety profile, the SAFIRE data suggests that epitigramab has a great potential to provide clinically significant benefit to patients with SMA despite the chronic use of SMN targeted therapies. As David mentioned, our BLA was accepted under priority review which recognizes the potential of ipilimumab to be a treatment for a serious or life threatening condition or to provide a significant improvement in safety or effectiveness over existing treatments. I'm excited that our team continues to work collaboratively with regulators towards the September 22 PDUFA date.

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

With respect to Europe, the MAA was validated by EMA and we look forward to further review and the approval of ipilimumab in Europe in 2026. Turning to the next slide, I'm pleased to report that we're on track to initiate the Phase two OPAL trial in children under the age of two. The study is evaluating two different doses over the course of forty eight weeks and will assess PK, PD, efficacy, and safety. Enrollment criteria are broad and infants and toddlers may be enrolled with any approved SMN targeted therapy, including gene therapy. Early intervention with dupitigumab in the OPAL study could support muscle during a critical early developmental phase, complementing SMN target therapies that aim to preserve motor neurons.

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

By promoting muscle growth, when both motor neurons and muscles are still forming, apitigramab has a unique opportunity to improve motor outcomes in babies and toddlers with SMA. Turning to our plans for the study of apitigramab in additional rare and severe neuromuscular disease, at the outset let me state that the potential to prevent muscle loss and enhance muscle growth by blocking myostatin holds great potential across a wide array of diseases. Here you see just two examples of that potential in mouse models of the severe debilitating disorders Duchenne muscular dystrophy or DMD and fascioscapulohumeral dystrophy or FSHD. In the DMD model on the left, the inclusion of an anti myascitin antibody in mice receiving an exon skipping oligo is associated with a dramatic increase in the level of dystrophin and a resulting increase in muscle force. FSHD is a neuromuscular disease resulting in patchy changes with affected and unaffected motor fibers.

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

In the FSHD model on the right, treatment with an anti myosatin antibody results in hypertrophy of unaffected motor units and once again an impressive gain in motor function. We continue our evaluations of an antimycitin approach in DMD and FSHD as well as in other models of disease and will initiate the study of vapigumab in an additional neuromuscular indication by the 2025. In June, we were gratified to announce the exciting results from our IMbraCE proof of concept study. David has covered those findings, including that the study met the primary endpoint with an encouraging safety profile. These findings are important as preservation of lean mass during GLP-one mediated weight loss has the potential to enhance both cardiometabolic and musculoskeletal health.

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

Next, we continue to advance our world leading anti mastectomy platform beyond ipilimumab. Further to our commitment to neuromuscular disease, SRK439 builds on the validated approach that delivered ipilimumab. There's great potential for SRK-four thirty nine to be an infrequent, potent, subcutaneous anti mastatin antibody, and we look forward to exploring its potential as another innovative scholar rock therapy for patients suffering with severe neuromuscular disorders. We remain on track to file the IND application for SRK439 to support the first in human study later this year. I'd like to conclude by reiterating our key priorities: Number one, drive The US approval of ipilimumab in Q3 twenty twenty five and advance The EU toward approval in 2026.

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

Number two, initiate a study of ipilimumab for infants and toddlers with SMA under the age of two in Q3 twenty twenty five. Number three, initiate clinical development of ipilimumab in at least one additional neuromuscular indication by the 2025. And finally, file an IND for SRP-four 39 in the 2025. With that, I'll turn the call over to Keith to provide a commercial update. Keith?

Keith Woods
Keith Woods
COO at Scholar Rock Holding

Thanks, Akshay, and good morning, everyone. With epinegrimab advancing through the regulatory processes in The U. S. And Europe, we are preparing to usher in a new era for the treatment of children and adults with SMA. This is a progressive and devastating disease that leads to loss of mobility, limited activities of daily living, and a lack of independence.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

If muscle is left untreated, it can result in irreversible atrophy. We are excited about epitogimab's potential as the first and only muscle targeted treatment to show clinically meaningful and statistically significant motor function improvements in children and adults living with SMA. The SMA community is calling for a treatment to address progressive muscle degeneration and motor function loss. To underscore this, a 2025 Cure SMA survey showed that ninety percent of patients report that their greatest unmet need is to gain muscle strength. Our market research and interactions with health care 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.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

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. We are on track with the preparation of the global launch of ipilimumab starting with The US. Based on our September 22 PDUFA date, we will execute our commercial launch of ipilimumab immediately following our FDA approval. In Europe, our MAA is under review by the European Medicines Agency, and we are preparing to launch upon approval in 2026. I'm excited to announce that as of today, under the leadership of our US General Manager and Chief Brand Officer, Rebecca McLeod, our US customer facing team is fully onboard, trained, and now deployed in the field.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

Our team is currently engaging with the SMA treatment centers, key opinion leaders, and both public and private payers. We are impressed by the talent, the extensive rare disease experience, and the passion to serve patients that our new team members bring. The team is comprised of many professionals that have substantial experience in serving the SMA community or the broader neuromuscular disease community, and we are united by a commitment to serving these patients. Today in The US, there are approximately ten thousand patients living with SMA, and roughly two thirds of them have received an SMN targeted therapy. These investments we are making position us to reach these patients with epitogrelimab and will provide a blueprint for our global rollout.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

Globally, there are approximately thirty five patients with SMA that have already received an SMN targeted therapy, and we have an enormous opportunity to make a meaningful difference with epitogromab with the potential to reverse progression of SMA from a loss of motor function to a gain of motor function. The entire ScholarRock team is ready to serve the SMA community, and we will move with a sense of urgency to deliver epitigramab to them following approval. 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 our q two business update and provide insights into how we are thinking about resource allocation in the future. The opportunity with Apidigromab 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 $295,000,000 During the quarter, we continued to increase our investment in infrastructure to support commercial readiness and our supply of Epidigromab to support the launch.

Vikas Sinha
Vikas Sinha
CFO at Scholar Rock Holding

As we look ahead, we are prioritizing the commercial launch and our ongoing clinical programs. We have an additional 50,000,000 under our debt facility that we can draw down this year and we also anticipate receiving approximately 16,000,000 from exercise of outstanding common warrants by year end, bringing our anticipated runway into 2027. Additionally, 50,000,000 is available under our debt facility post approval to support the upcoming launch. We also anticipate monetizing our priority review voucher following approval. We continue to operate with a tight financial plan and we'll share more details over the next few quarters.

Vikas Sinha
Vikas Sinha
CFO at Scholar Rock Holding

As a reminder, we will continue to focus 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?

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

Thanks, Vikas. In closing, we are committed to successfully executing on these key priorities, which position us to transform ScholarRock into a premier global biotech leader. First, epitigramab regulatory approvals and following those approvals, The U. S. Launch of epitigramab for children and adults with SMA, followed by a series of country launches in the coming years, starting in Europe with Germany next year second, expand ipitigramab into additional rare, severe and debilitating neuromuscular diseases and finally, disciplined capital allocation to support our high value commercial and development initiatives.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

On behalf of every member of the ScholarRock team, I want to emphasize our unwavering commitment to more than 35,000 patients and their families. Our mission is to move with urgency to ensure that no patient with SMA is left behind. With that, we'll now open the line for questions. Operator?

Operator

Thank you. We will now begin the question and answer session. And your first question comes from the line of Eric Schmidt with Cantor. Your line is open.

Eric Schmidt
Biotechnology Analyst at Cantor Fitzgerald

Well, thanks, and and good morning, ScholarRock team. Appreciate the transparency around your CDMO issues. Can you tell us a little bit more about the specific observation at those two sites? Whether you expect that a reinspection is going to be required prior to eptegromab approval? And whether the two facilities right now are currently able to release other pharmaceutical products? Thank you.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

Yeah. Thanks very much, Eric, and, it's great to be working with you. First, I think as you know, Eric, GMP inspections are super standard as part of the FDA review process. And in fact, in 2024, based on FDA's inspection data dashboard, actually more than 70% of drug quality assurance inspections resulted in observations noted by the FDA. So as I noted in the call, we you know, look, we're disappointed, but we weren't surprised to learn that two of our sites received observations.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

I think the benefit for us is those observations were presented well within our review cycle that allow for a response and obviously a green light. It's always very difficult to sort of read the tea leaves and try to understand what the FDA with those observations may want to do, would they want a reinspection or not. At this moment in time, the very constructive late cycle meeting that again was held after these site inspections and after these observations were provided, again the tone and tenor and collaborative nature of that late cycle meeting certainly indicated to us that the agency is very much working toward completing their review of our BLA by the September 22 PDUFA date. I would also just note that we're obviously working on multiple levels with our CDMO partners. We're a skilled team.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

We're an experienced team. We understand, how best, to work through situations like this, which we are prepared to do.

Eric Schmidt
Biotechnology Analyst at Cantor Fitzgerald

And and, David, do you know whether the two facilities are releasing any product currently?

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

Very good question. The two facilities, continue to operate. Of course, the one that, given the disclosure on August common practice in the industry nor our common practice to name facilities. But in the case of Catalent, Indiana, now owned and operated by Novo Nordisk, certainly, as you know, Eric, that's an important filler across the board for a number of very important products across the industry, and we know they continue to be very active. And the other facility continues to manufacture product as well.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

I think I would also note, and I think this is probably important for our entire audience, our supply chain has produced the ipilimumab drug supply that has now accumulated over six hundred patient years of experience through multiple years of clinical trials, including our pivotal trial, the most recent embrace trial. And I think importantly, I'd like to note, that our launch supply and beyond is manufactured, wild, and ready to go.

Eric Schmidt
Biotechnology Analyst at Cantor Fitzgerald

Thank you very much.

Operator

Your next question comes from the line of Allison Breitzel with Piper Sandler. Your line is open.

Allison Bratzel
Allison Bratzel
Senior Research Analyst at Piper Sandler Companies

Hey, good morning team, and thanks for taking the questions. First, could you just further characterize any interactions with FDA on the epitigramab review outside of the observations at CDMOs you just discussed? Any feedback on your overall confidence in receiving a broad label would be particularly helpful. And then I think you talked about expansion of payer outreach. So could you just frame for us how your discussions with U.

Allison Bratzel
Allison Bratzel
Senior Research Analyst at Piper Sandler Companies

S. Payers are going and just overall receptiveness to coverage of epitogromab on top of SMN targeting therapy? Thank you.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

Yes. No, thanks, Alison. And yes, as we noted, we were very encouraged by our late cycle meeting that we recently had. And Akshay can provide a little bit more context on that. And then as I noted, and Keith did as well, he's just recently deployed a sensational team, and he can comment on some of those payer interactions.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

Related to the label and the interactions with the FDA, Akshay?

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

Yeah. Thanks, David. We had an exceptionally constructive late cycle meeting recently with the FDA. All the different disciplines that needed to be there were there. We talked through the different aspects of the BLA.

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

I think, we were very pleased with the feedback we had. And so, you know, we're working with them, as David said in his prepared statement, towards September 22, and look forward to the rest of the BLA review being completed. And, we we appear to be doing just that right now.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

Yeah, Allison. And as far as the payers, as you as we've noted before, we have a fully staffed payer team. Our outreach has been progressing to be able to be ready for our September 22 PDUFA date. The meetings have been going positive as we've been going through some of the epitogromab data and overall response. But importantly, the durability of response is something that payers have found quite impressive.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

Lastly, to your other question about paying for another therapy for SMA, I would say that you already have better than twenty percent of patients that are receiving more than one SMA therapy that is being paid for by payers at this time. So although the question, has come up, the unmet medical need, is is there, and, therefore, all I can say is the discussions are going positive.

Allison Bratzel
Allison Bratzel
Senior Research Analyst at Piper Sandler Companies

Thank you.

Operator

Your next question comes from the line of David Nierengarten with Wedbush Securities. Your line is open.

David Nierengarten
Managing Director - Equity Research at Wedbush Securities

Hey, thanks for taking my questions. I just had one on the labeling. Have you had labeling discussions with FDA and able to discuss possible labels with payers? Or is that to be discussed with the PDUFA date or as we approach the PDUFA date? And then the second one is if there is unfortunately a delay in the PDUFA, does that possibly put the PRB at risk if there are different changes in the budget that starts, you know, on October 1 of the fiscal year? Thanks.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

Yeah. Maybe I'll take that, second, or last question, David, first. There would be no change in PRV. And, I think, you've all come to know us, this group of management team. We're a we think about everything.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

We're a measured twice, cut once, type of crowd. And, you know, we would think through any scenarios even before, as we mentioned. We weren't totally surprised by observations only because three quarters of the time they happened. And so we would have been, thinking through all of this, and there'd be no no impact, on the PRB, at all, which is, important and very good news. Regarding just the constructive nature of the dialogue and and and the label, I'll have Akshay comment.

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

Yeah. So with respect to that topic, again, we had a very good late cycle meeting. And, as you can expect at the late cycle meeting, all the different aspects of the BLA leading to a potential approval are discussed, including labeling and the timings around who can expect what. We've had those types of interactions. I can't get into more details on that right now, but we were reassured that everyone is working towards September 22.

David Nierengarten
Managing Director - Equity Research at Wedbush Securities

Great. Thank you.

Operator

Next question comes from the line of Tess Romero with JPMorgan. Your line is open.

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

Hi. Good morning, team. So just double clicking back on some of the earlier comments you've made, what is the right way to think about the next step following these observations given proximity to your goal date? And how likely do you think that the outcome of the review will be positive in light of these findings and remain on time? And then my second question is, what does an ideal label look like for epitogranib?

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

And at this point, what do you believe the indication statement should be? Thanks.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

Thank you, Tess. Great questions. So just kind of letting you into our kitchen a little bit. What you might expect, us to be doing is working very closely, with our CDMO partners. So in other words, like, being well aware of the observations, having input on the responses to those observations, And then having levels of transparency both with the CDMOs and and and their communication with the agency, as well as our own continued dialogue, with the agency, through the final stages of our review process.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

And so I would just say that, we have substantial talent, internally. We would, always take a no stone left unturned approach to external experts to help us and help our CDMO partners, think through, you know, the very best way to respond to observations. As I noted, we're confident that Novo submitted, and we actually have access to those those responses that they made earlier this week. And the same will hold true for the responses that are going in within the week related to the other CDMO. So we'll have a great visibility into this and then multiple lines of communication through our CDMO partners as well as the FDA directly.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

Related to the IVEA label, I'd turn over to Akshay and Keith that one of the things we've worked very hard on by taking a no shortcut approach to the development program is the robustness of our Phase II and then Phase III trial. And so obviously when we think about children and adults with SMA, we think about aligning with our partners at Cure SMA really for the best interest of the community of broad label is quite important so that all patients who can benefit from epitigramab will have access to epitigramab. For more comments on the label, I'll hand it over to Akshay.

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

Yep, thanks David. So with respect to that topic, Taphy itself studied individuals two years and older to the age of 21. The primary endpoint was for the group that was two to twelve year old. We obviously hit that primary endpoint. We relayed the data to again today.

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

The important thing is that the data in the thirteen to twenty one were also entirely consistent with what we saw in the two to twelve group. And so I think we can expect that those on the two that were not studied may not be in the label, but certainly above two, we would like to see those in the label given the very exciting findings from Sapphire, all the way up into adulthood. And, you know, if a patient in Sapphire is 21 years old and is experiencing benefit, they're not gonna suddenly stop getting benefit when they're 22. So I think, you know, from a logic perspective, two years and older is a way to think about the group that that that would be eligible. But, again, we cannot give any final information on that until the FDA has finished their deliberations.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

But Yeah.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

And and one last thing I'd I'd comment on in in our, you know, very strong partnership with Cure SMA. They've actually advocated, you know, with the FDA for many years, the importance of sometimes a narrower clinical trial, but a broader label. Recognizing the biology of the disease isn't necessarily different as patients get older. We were gratified that our most recent study in adults, over 100 adults showed the biology of myostatin again held up. That of course was with the EMBRAZE trial.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

But it has been common practice that there can be a slightly narrower patient population in a pivotal trial, but then in acceptance of a broader label. And that's what we're working toward. The ideal label, as Akshay said, would be the patients at two years of age and older would have access to the therapy.

Operator

Thank you. And our next question comes from the line of Mark Pham with TD Cowen. Your line is open.

Marc Frahm
Marc Frahm
Biotechnology Equity Research Analyst at Cowen

Thanks for taking my questions. Maybe first to kind of push a little bit on the pricing discussion that you're having with payers. Just I think we are encouraged that twenty plus percent of patients are getting clinical combination therapy today, you know, even without phase three data supporting that. But maybe the the ask on the payers is a little bit different with that, right, because most of that is one time gene therapy costs that may have already occurred and then on an annual budget basis just one therapy. Just can you talk through how they're kind of thinking through the budget impact of maybe two SMA type pricing drugs in individual patients happening on a concurrent and ongoing basis?

Marc Frahm
Marc Frahm
Biotechnology Equity Research Analyst at Cowen

And then maybe from the R and D side, starting a new indication later this year, should we view that as like one pilot outside of SMA? Or is that just the first of many and we should expect as we go into twenty twenty six additional indications to continue to be added to the pipeline.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

Mark, thanks. Very thoughtful questions. On the pricing side, I would note, of course, as I have in the past, that we'll consider a number of things when establishing the price. Certainly the rarity of SMA, and you talk about budget impact as an individual patient, we obviously think about just how rare SMA is. The severity of the disease despite the use of over the past ten years of SMN targeted therapies, where they have shown in some of their long term data, and we then again underscored in our phase three trial, that patients eventually move to a progressive state of motor function loss.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

And how severe SMA is despite the use of best known standard of care. And then of course, the compelling clinical benefits that we can provide. And some of those were underscored by Akshay earlier. And so we'll think about those things, we'll think about the budget impact. And and one of the things we also have a lot of experience with is looking at even sort of pricing quarters for rare disease therapies, monotherapies in different settings and sort of thinking about sort of where the starting point is today.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

We definitely do not think the error has been in terms of payers willingness to support both physician interest as well as patient interest. And accessing, and Keith will comment in a moment, on the importance of dual modality therapy for this devastating disorder. Keith?

Keith Woods
Keith Woods
COO at Scholar Rock Holding

Yeah, thanks David. Mark, one of the things is, you mentioned that this is using two agents is just post post Zolgensma. And I just wanna call out that we actually have met with patients and physicians that have tried both Risdiplam and SPINRAZA simultaneous. So they're paying for two current agents at the same time. So it's not just post gene therapy.

Keith Woods
Keith Woods
COO at Scholar Rock Holding

The bottom line is that over time, these patients on these SMN targeted therapies, many of them plateau and then they begin to decline and payers are realizing that there is a great deal of unmet medical need that's left. If I think about the fact, as David mentioned, treating with dual modality and targeting the muscle directly as well as the motor neuron, it gives you an opportunity it gives you an opportunity to potentially not only increase your responses we've shown from the Sapphire data, but then also have a durability of response over time. And so that is a meaningful impact, that is valuable to payers.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

And then Mark, regarding the other sort of the pipeline and a product strategy that Akshay is so skillfully leading, I'll have him comment.

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

Yeah, thanks Mark. So we see enormous opportunity for antagonizing mystat in a whole array of neuromuscular disorders. And so, yes, there'll be many opportunities that you'll see us studying. We'll begin with one by the end of the year, but I think it will add to that in in quick succession over time. So much more to come on that.

Marc Frahm
Marc Frahm
Biotechnology Equity Research Analyst at Cowen

Yep. Thank you.

Operator

And your next question comes from the line of Martin Auster with Raymond James. Your line is open.

Thomas Deal
Thomas Deal
VP - Biotech Equity Research at Raymond James

Hi. Hey. Good morning. This is Thomas on for Marty. Thanks for taking our question.

Thomas Deal
Thomas Deal
VP - Biotech Equity Research at Raymond James

Maybe, just one on the OPAL trial here. Anything you can say about expected enrollment time lines there? And maybe just frame for us how to think about the significance of that expansion opportunity if we envision an initial label for ipiligramab in patients two years and older. Thank you.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

Yeah, thank you very much. And consistent with our ambition and mission of company that no patient would ever be left behind, including, you know, babies and toddlers. This is an important study for us, and as Akshay noted. So I'll have him comment a little bit further on the study and expected timelines.

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

Yeah.

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

Thanks. So, you know, with respect to to timelines, you know, we are going to start enrollment in q three, as as I said on the prepared remarks. My usual practice is let's get that going. And then in the coming quarter or '2, as we see momentum build, we'll be able to guide further on the exact completion date of the study. And, you know, with respect to zero to two year old children with SMA and the label, we didn't study under the age of two in the SAFIRE study. We've initiated this study to understand the impact of the drug in that population safety, PKPD efficacy.

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

And as David said, no child should be left behind with this disease, and we think, pitogrelimab can have an impact across the entire age range here. I think in terms of trying to do good with this drug and helping as many kids as possible, one thing I would note is that the prevalent population is much, much larger than the incident population. And so, you know, if the zero to two are the new children being born with SMA, that's dwarfed by the thirty five thousand or greater who currently have SMA or on treatments that we believe could be helped by a drug like upadivimab. So there's a lot for us to do once we get OPAL done and start helping under two, and we'll begin with two years at older.

Thomas Deal
Thomas Deal
VP - Biotech Equity Research at Raymond James

Thank you.

Operator

And our next question comes from the line of Srikripa Devarakonda with Truist Securities. Your line is open.

Kripa Devarakonda
Kripa Devarakonda
VP - Biotechnology Equity Research at Truist Securities

Hey, guys. Thank you so much for taking my question. I just want to go back to the CDMO site inspections. Now if there is a need for another inspection, do you have a sense of timelines as to when you will get to know that by? And, you know, given the CRLs that we've seen with, one of the peers involved, what are typical timelines for potential resolution of these issues? Thank you.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

Yeah, thanks, Kripa. Again, it's hard to predict what the FDA will do. I mean, think you all do this for a living, as do we. And the best that we can do, like you, is assess what has happened from time to time in the industry and what could be some of the different scenarios. But I can tell you that from all of our experience and that of our expert advisers that we are working with along with our CDMOs, the very best thing to do initially is have a robust response to those observations.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

And that is what we've been spending a lot of time on. The robustness of those responses obviously, can have an impact on whether or not any reinspection would be required. And so that's where our effort is being placed. And then again, I think, as I noted, we would be gathering information both through our CDMO partners, but then also directly with the review division. And we'll keep you apprised at that.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

But right now, given the fact that following these site inspections and following the presentation of those observations, we held our late cycle meeting. The FDA completed that late cycle meeting. The dialogue, the tone, content of the discussion was very encouraging with a commitment to move forward toward our PDUFA date under priority review of September 22, and we will certainly keep you and all of our stakeholders apprised on that progress.

Kripa Devarakonda
Kripa Devarakonda
VP - Biotechnology Equity Research at Truist Securities

Great. Thank you so much.

Operator

And our next question comes from the line of Evan Seigerman with BMO Capital Markets. Your line is open.

Evan Seigerman
Evan Seigerman
MD & Senior Research Analyst at BMO Capital Markets

Hi. Two questions from me. Can you provide more color or characterize the interactions from your late cycle review meetings? Did they at all talk about just the difference in efficacy we saw between the 20 the twenty milligram and the ten milligram doses? And then also, Vikas, one for you.

Evan Seigerman
Evan Seigerman
MD & Senior Research Analyst at BMO Capital Markets

As we think about kind of modeling the asset post launch, how should we think about the cadence in the back half of this year and into next year? Thank you, guys.

David Hallal
David Hallal
CEO, Director & Chairman at Scholar Rock Holding

Yes, Evan, great question. Regarding the combined dose analysis, I'll have Akshay comment on that, which obviously, as you know, there's been extensive clinical work done with the 10 and the 20. Most recently, Embrace was 10. Akshay?

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

Yeah. So the late cycle interaction was indeed very constructive and positive. So we have discussed the file fully, including the combined dose analyses with all of you before, and we've had very constructive discussions with regulators around that as well. As you know, we hit the primary endpoint. The important thing is both ten and twenty have an almost identical pharmacodynamic effect.

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

And so it's unsurprising that both showed a positive outcome with efficacy, and combining the two groups increased statistical power, to be able to see the difference versus placebo. And, obviously, we saw gain in most function with ipilimumab versus a decline. And our expectation is that given that the the regulatory practice always do approve the lowest dose that's efficacious, and given that both have equivalent pharmacodynamic impact, we would request that ten milligram per kilogram is the approved dose. I'll leave it there.

Vikas Sinha
Vikas Sinha
CFO at Scholar Rock Holding

Yvanda, on the financial side, we have still to do the 2026 budget planning session. But as as I mentioned in my call, we're gonna be very thoughtful about how we go into next year. Some of the some of the expenses in the clinical side is gonna drop out next year and then we'll replace it with the prioritization as as Akshay was laying out in his section with moving four thirty nine and some of the pipeline forward. So that that's, you know, other indications forward. So that's our key priority and keeping our overall spending in in check.

Vikas Sinha
Vikas Sinha
CFO at Scholar Rock Holding

We do expect it to be in similar lines like what we spend this year.

Operator

Thank you, ladies and gentlemen. This concludes the q and a session, and and concludes today's call. Thank you all for joining. You may now

Executives
    • Rushmie Nofsinger
      Rushmie Nofsinger
      VP - Corporate Affairs & IR
    • David Hallal
      David Hallal
      CEO, Director & Chairman
    • Akshay Vaishnaw
      Akshay Vaishnaw
      President, R&D
    • Keith Woods
      Keith Woods
      COO
    • Vikas Sinha
      Vikas Sinha
      CFO
Analysts
    • Eric Schmidt
      Biotechnology Analyst at Cantor Fitzgerald
    • Allison Bratzel
      Senior Research Analyst at Piper Sandler Companies
    • David Nierengarten
      Managing Director - Equity Research at Wedbush Securities
    • Tessa Romero
      Equity Analyst - Biotechnology at J.P. Morgan
    • Marc Frahm
      Biotechnology Equity Research Analyst at Cowen
    • Thomas Deal
      VP - Biotech Equity Research at Raymond James
    • Kripa Devarakonda
      VP - Biotechnology Equity Research at Truist Securities
    • Evan Seigerman
      MD & Senior Research Analyst at BMO Capital Markets
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