Scholar Rock (SRRK) Q1 2026 Earnings Transcript

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DATE

Thursday, May 7, 2026 at 8:00 a.m. ET

CALL PARTICIPANTS

• Chief Executive Officer — David Hallal
• President — Akshay Vaishnaw
• Chief Commercial Officer — Robert Keith Woods
• Chief Financial Officer — Vikas Sinha

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TAKEAWAYS

• BLA Acceptance -- The FDA accepted the biologics license application for apitegromab, setting a Prescription Drug User Fee Act (PDUFA) action date of September 30.
• Manufacturing Redundancy -- The accepted BLA includes two independent fill-finish facilities: Catalent Indiana and a second U.S.-based site, offering two separate approval paths for apitegromab.
• Catalent Indiana Inspection -- The FDA completed an unannounced reinspection of the Catalent Indiana site, initiating a 90-day window to classify the facility's status.
• Commercial Supply Readiness -- Scholar Rock (NASDAQ:SRRK) expects ample commercial apitegromab from the second fill-finish facility in early Q3, before the PDUFA date.
• Cash Position -- The company ended the quarter with $480 million in cash, cash equivalents, and marketable securities, including a $100 million debt drawdown and $98 million in proceeds from its at-the-market (ATM) program.
• Operating Expenses -- Total operating expenses were $102 million, including $80 million in non-cash stock-based compensation (excluding which, expenses were $84 million).
• Commercial Launch Preparation -- The U.S. commercial team is ready to launch apitegromab immediately upon approval, with key field, reimbursement, and patient support infrastructure in place.
• Europe Regulatory Progress -- The European Medicines Agency (EMA) review for apitegromab is advancing, with a Committee for Medicinal Products for Human Use (CHMP) opinion expected midyear and launch activities targeting Germany first.
• Pipeline Developments -- Enrollment continues in the Phase II OPAL trial for infants and toddlers with SMA, while the Phase II FORGE trial in FSHD is set to initiate soon with a sample size of 60 patients.
• Subcutaneous and Novel Agents -- Scholar Rock reported Phase I data for subcutaneous apitegromab showing favorable bioavailability versus IV, and top-line Phase I data for SRK-439 are expected later this year.

SUMMARY

The FDA's acceptance of apitegromab's BLA with two fill-finish sites provides Scholar Rock with independent regulatory approval pathways and commercial supply assurance. The successful completion of Catalent Indiana’s reinspection and the early Q3 commercial readiness of the second facility position the company for a launch ahead of the September 30 PDUFA date. EMA review is progressing in alignment with FDA timelines, potentially enabling a near-simultaneous launch in Europe. The company possesses significant cash reserves to support its global commercialization plans and pipeline initiatives.

• Scholar Rock’s commercial and medical field teams have engaged over 140 U.S. SMA treatment centers and 2,600 prescribing physicians, preparing individualized support programs for launch.
• The EMA waived the need for an oral explanation meeting after pre-meeting alignment, indicating constructive regulatory engagement.
• Scholar Rock has built significant EMEA infrastructure, including a European headquarters in Switzerland and on-the-ground launch teams in Germany, with reimbursement processes under way across multiple EU countries.
• Patients enrolled in the OPAL trial include those previously treated with SMN1- or SMN2-targeted therapies, addressing broader SMA treatment populations such as post-Zolgensma patients.
• The company highlighted that about one-third of the U.S. SMA population has received two or more SMN-targeted therapies sequentially or in combination, based on data shared by Cure SMA.
• Upon FDA approval, Scholar Rock can access an additional $150 million from its debt facility and plans to monetize a priority review voucher, which management states will further strengthen the balance sheet.
• The company affirmed ongoing discussions with national, regional, Medicare, and Medicaid payers in the U.S, facilitated by more time ahead of approval.
• Subcutaneous apitegromab demonstrated Phase I pharmacodynamic similarity to IV dosing, and post-marketing discussions with regulators are planned to define development and approval pathways for this formulation.

INDUSTRY GLOSSARY

• BLA (Biologics License Application): A regulatory submission for approval to market a biologic product in the U.S.
• PDUFA (Prescription Drug User Fee Act): U.S. legislation that establishes FDA action dates for reviewing new drug applications.
• SMA (Spinal Muscular Atrophy): A genetic neuromuscular condition targeted by apitegromab.
• FSHD (Facioscapulohumeral Muscular Dystrophy): A rare muscle disease studied in the FORGE Phase II trial.
• CHMP (Committee for Medicinal Products for Human Use): The EMA committee responsible for scientific evaluation of medicines for use in the EU.
• EMA (European Medicines Agency): The regulatory agency for evaluation and supervision of medicines in the EU.
• CRL (Complete Response Letter): An FDA communication outlining issues precluding approval of a submitted application.
• ATM Program (At-the-Market Program): A method for a company to raise capital by selling shares directly into the market over time.

Full Conference Call Transcript

David Hallal: Thank you, Laura, and good morning. Thanks to everyone for joining our first quarter earnings call. Scholar Rock is positioned for a pivotal year ahead. To that end, today, I am very pleased to announce that the FDA has accepted for review our biologics license application for apitegromab for the treatment of children and adults living with SMA. The agency has assigned a PDUFA action date of September 30. Importantly, the accepted BLA includes 2 fill-finish facilities, Catalent Indiana and a second U.S.-based facility, providing Scholar Rock with 2 independent paths to apitegromab approval.

As a reminder, the sole approvability issue for apitegromab noted in the complete response letter last September was related to observations identified during a routine general site inspection of the Catalent Indiana fill-finish facility, which is owned and operated by Novo Nordisk. Since our in-person Type A meeting with the FDA in Q4, we have continued to work constructively and collaboratively with the agency, and we have made steady and rapid progress. During the first quarter, we made meaningful advancements at Catalent, Indiana and our second fill-finish facility. And with our ongoing open communication with the agency, we resubmitted our apitegromab BLA in late March in complete alignment with the FDA to include both facilities.

This approach underscores the shared understanding between the FDA and Scholar Rock of the unmet need in the SMA community and the shared urgency to bring apitegromab to children and adults in the U.S. as quickly as possible. I would like to now provide an update on the status of each of these 2 sites. As it relates to Catalent Indiana, we are pleased that following acceptance of our BLA, the FDA completed an unannounced reinspection of the facility. This timing was in line with our expectations as the FDA had noted following multiple engagements with Novo in Q1 that they would conduct an unannounced inspection following routine manufacturing activities, which resumed in late February.

We are pleased that the inspection was completed in early Q2 and in accordance with FDA guidelines, the agency has up to 90 days to classify the facility. As it relates to the second fill-finish facility, we continue to be pleased with our ongoing meaningful progress. Importantly, the entirety of the apitegromab drug product required for FDA review and potential approval has been filed. From a commercial supply standpoint, we are well positioned as we expect to have ample commercial apitegromab available from the second facility in early Q3, well ahead of the September PDUFA date. We remain committed to the SMA community, and we are grateful that significant progress continues to be made at a rapid pace.

Our U.S. commercial team continues to advance the critical activities and capabilities required to deliver a seamless launch and support patients from day 1. Importantly, the team stands ready to launch apitegromab immediately upon approval at any time prior to, and including the September 30 PDUFA date. In addition to the U.S., we continue to look forward to serving children and adults with SMA in Europe. The review of our MAA is progressing very well, and we expect a CHMP opinion near midyear. We are building momentum with launch readiness activities, and we continue to anticipate a launch in the second half of the year, beginning with Germany.

We know it is not a matter of if, but when apitegromab will be approved for children and adults with SMA, and Keith will discuss the continued progress we are making with commercial preparations and our disease awareness initiatives shortly. We continue to advance our world-leading anti-myostatin pipeline with enrollment in our Phase II OPAL study evaluating apitegromab in infants and toddlers with SMA, the anticipated initiation of our randomized Phase II study in patients with FSHD and progress with subcutaneous apitegromab and a novel high potency anti-myostatin antibody, SRK-439 currently in Phase I. Akshay will discuss these programs in greater detail shortly. Turning now to the balance sheet.

We are pleased to have ended the first quarter of 2026 with $480 million in cash, cash equivalents and marketable securities. This cash balance includes the drawdown of an additional $100 million from our debt facility, which we took in March. Our cash balance also reflects net cash proceeds of $98 million from our ATM program during the quarter. Vikas will provide more details later in the call.

We are building on a solid foundation for our company's growth, which we believe will be steady and consistent through the end of this decade and well into the next as we prepare to serve up to 35,000 children and adults living with SMA around the world who have received at least one SMN targeted therapy. Beginning with SMA, we are excited to be shaping the future of treatment for patients living with rare and devastating neuromuscular diseases. And with that, I'll now turn the call over to Akshay. Akshay?

Akshay Vaishnaw: Thanks, David, and good morning, everybody. We're very pleased with advancements in our world-leading anti-myostatin pipeline during the first quarter. Turning first to apitegromab for children and adults with SMA. We're delighted to share that the FDA has accepted the apitegromab BLA. As a reminder, the BLA was resubmitted in alignment with the agency to include both Catalent Indiana and a second U.S.-based fill-finish facility. The approach provides Scholar Rock with 2 independent path to apitegromab approval by the PDUFA action date of September 30.

We're gratified by the agency's continued support since the CRL last September from the constructive and collaborative in-person Type A meeting in November to the early March Type C meeting and the current acceptance of the BLA. Throughout, the agency has appreciated the high unmet need in the SMA community, and we now look forward to the final steps in the U.S. regulatory process. Reflecting the agency's vigorous efforts, we were pleased most recently with the timing of the FDA's unannounced reinspection of Catalent Indiana. For FDA guidelines, the agency now has up to 90 days to classify the status of the facility. I'd now like to turn to our second fill-finish facility, where we continue to make meaningful progress.

As David noted, the apitegromab drug product required for FDA data review and potential approval has been filed, and we expect to have ample commercial apitegromab from the facility in early Q3 ahead of the September PDUFA date. Based on the significant progress at both facilities, we anticipate approval of apitegromab for children and adults with SMA, which could be supported by either or both facilities by the end of the third quarter. Turning now to Europe. Our MAA for apitegromab for the treatment of children and adults with SMA continues to progress well through EMA review. As evidence of the progress, we have planned to be with the EMA recently for an oral explanation meeting.

However, because we and the EMA were able to align prior to the scheduled meeting, we mutually agreed that the oral explanation was no longer necessary. As we highlighted previously, approval in Europe also requires FDA clearance for the Catalent Indiana facility. Based on our discussions with EMA, they're aware of the progress at Catalent Indiana and are comfortable with the review time line that accounts for the FDA's classification as site. We continue to be very pleased with how the review is progressing, and we anticipate a CHMP opinion in the middle of the year. Turning to our pipeline. Let me start with the Phase II OPAL trial.

We continue to enroll and dose patients in this study, which is evaluating apitegromab in infants and toddlers under the age of 2. As a reminder, this trial is enrolling participants who have been treated with an SMN1-targeted gene therapy, or who are receiving ongoing treatment with an SMN2-targeted therapy. This study is important because it is anticipated to expand the impact of apitegromab to the full spectrum of patients, including those treated with Zolgensma. In addition, we believe early intervention with apitegromab could support muscle during a critical early development phase, potentially improving motor outcomes in the youngest of patients with SMA. Turning now to our next indication for apitegromab, facioscapulohumeral muscular dystrophy or FSHD.

FSHD is a rare, devastating neuromuscular disease with significant unmet need. More than 30,000 patients are diagnosed in the U.S. and Europe alone, and there are no approved therapies. We prioritized FSHD as the next indication for apitegromab for 3 key reasons: First, the significant unmet need; second, the compelling preclinical data from the gold standard FLExDUX4 mouse model that provides mechanistic rationale for apitegromab in FSHD. And finally, as shown on Slide 11, data from randomized studies in FSHD, which suggests muscle mass can increase and has the capacity to show functional benefit. For example, in studies of either rigorous physical therapy or treatment with anabolic agents, patients with FSHD demonstrated increases in lean mass and muscle function.

These data suggest that apitegromab as a monotherapy may have the potential to bring important benefit to FSHD patients. We're very pleased with the progress of activities to support the initiation of our Phase II study called FORGE in the middle of this year. Enrollment will commence soon in this randomized, double-blind, placebo-controlled trial, which has a sample size of 60 patients. We're also advancing 2 additional programs in our world-leading anti-myostatin pipeline, a subcutaneous formulation of apitegromab and SRK-439. In our subcutaneous apitegromab program, we showed some very exciting data from a Phase I study in January, which demonstrated that subcu apitegromab appears to have favorable bioavailability and a pharmacodynamic profile comparable to IV administration.

Additional development activities are ongoing, and we continue to plan for engagements with U.S. and European regulators later this year following approval of apitegromab. Turning now to SRK-439, our high potency, high affinity subcutaneously administered myostatin inhibitor, which we discovered by leveraging our world-leading expertise. We're very excited about this program and dosing in our Phase I healthy volunteer study is progressing well. We expect to have top line data from this study later this year. In closing, we're executing with urgency to bring apitegromab to children and adults with SMA, whilst in parallel working to maximize our impact for patients with apitegromab and our world-leading anti-myostatin pipeline across a range of rare devastating neuromuscular diseases.

With that, I'll now turn the call over to Keith to discuss our commercial launch preparations. Keith?

Robert Keith Woods: Thanks, Akshay, and good morning, everyone. With the BLA accepted by the FDA, our team continues to operate with urgency as we prepare for the launch of apitegromab immediately upon approval, which may be granted at any time through September 30, 2026. Nearly a decade after the introduction of SMN-targeted therapies, muscle strength and motor function remain the top unmet need with 95% of patients continuing to experience persistent and progressive muscle atrophy. That limits function and independence. As further evidence of the unmet medical need, data shared with us by Cure SMA show that an estimated 1/3 of people living with SMA in the U.S. have received 2 or more SMN-targeted treatments, either sequentially or in combination.

This data again underscores the significant opportunity we have with apitegromab, the world's first muscle-targeted therapy. Our U.S. customer-facing team continues to make significant progress in the field with disease education, awareness around the unmet medical need and reinforcing a broader understanding of SMA as a disease which consists of both the motor neuron and the muscle, the principal organ impacted by the disease. In the U.S., we have achieved significant reach across the approximately 140 SMA treatment centers, 2,600 prescribing physicians and their multidisciplinary care teams.

Through these engagements, our field team is working to establish case flows on a center-by-center basis to ensure we are well positioned to support the SMA treatment centers once a treatment decision is made. This includes preparations to launch our patient services program, Scholar Rock Supports. This program is designed to provide comprehensive and individualized support to patients, caregivers and providers. In the first quarter, we had a meaningful presence at the Muscular Dystrophy Association meeting in March. During this meeting, our team further engaged with health care professionals. As one example, we hosted a very well-attended industry forum called going beyond the motor neuron to the muscle, expanding the focus of SMA care.

We also remain highly focused on patients and community activation. We are building on our disease awareness campaign called Life Takes Muscle, and we continue to have numerous in-person patient and patient advocacy group engagements. Turning to U.S. reimbursement. Our market access team is advancing discussions with national and key regional payers as well as Medicare and Medicaid. With this extra time, we've been able to go deeper and broader across the range of payers. We are ready and well positioned for a successful launch of apitegromab in the U.S. immediately upon approval. Scholar Rock is also making significant progress in Europe. We have established our European headquarters in Switzerland.

Also in Germany, where we expect to launch apitegromab upon EMA approval, our local leadership is on board. We have hired our medical and commercial field teams, and we are actively enrolling patients in our compassionate use program. We are making meaningful progress with reimbursement planning to enable rapid patient access. In the broader region, we are advancing reimbursement dossiers in multiple countries, strengthening our distributor relationships and building our EMEA infrastructure to support future commercialization. Additionally, we had a significant presence at the SMA Europe meeting in March in Budapest.

Among other high-impact activities, we hosted an SMA disease education workshop and a health care professional symposium, where the attendance reflected a high interest in further understanding SMA and the unmet needs in this disease. In closing, we are investing with discipline to build the commercial foundation necessary to support a world-class launch and to achieve our long-term ambition to bring apitegromab to the estimated 35,000 patients living with SMA around the world who have received at least one SMN-targeted therapy. We are ready to usher in the next phase of innovation for children and adults with SMA, one patient, one caregiver and family at a time. With that, I'll turn the call over to Vikas. Vikas?

Vikas Sinha: Thank you, Keith. As we have shared previously, our financial objectives for 2026 remain focused on supporting our commercial build to deliver a strong apitegromab launch, funding R&D activities to advance our pipeline and expand our leadership in the myostatin and muscle space and continuing to evaluate opportunities to strengthen our balance sheet in a way that supports long-term shareholder value. In keeping with these objectives, I'm pleased to provide our first quarter financial results. For the first quarter, we reported $102 million in operating expenses, which included $80 million in noncash stock-based compensation. Excluding stock-based compensation, operating expenses were $84 million. Turning to our balance sheet.

We are very pleased to have ended the first quarter with $480 million in cash, cash equivalents and marketable securities. During the quarter, we strengthened our cash position with the drawdown of an additional $100 million from our existing debt facility, which we took in March. We also had net cash proceeds of $98 million from our ATM program during the first quarter. Looking ahead, upon FDA approval of apitegromab, we will have an option to draw down an additional $150 million from our existing debt facility, and we plan to monetize a priority review voucher to further strengthen our balance sheet.

We continue to operate with a tight financial plan and our prioritized investments remain focused on our apitegromab commercial launch readiness in the U.S. and Europe, strengthening our supply chain to support our expanding pipeline and our anticipated growing global commercial demand for apitegromab over time and advancing our highly innovative clinical programs that Akshay discussed earlier in the call. With that, I will turn the call back to David. David?

David Hallal: Thanks, Vikash. Scholar Rock is poised for a transformative year in 2026. Our priorities are clear, and we are executing with focus, discipline and urgency as we seek to deliver the world's first muscle-targeted therapy to children and adults living with SMA, while also laying the foundation to realize our ambition to develop life-transforming therapies for patients with additional rare and severe neuromuscular diseases globally. We are ready now more than ever to usher in the next phase of innovation forth SMA community, and we look forward to updating you on our continued progress. And with that, we'll now open the line for questions. Operator?

Operator: [Operator Instructions] And the first question comes from Eric Schmidt with Cantor.

Eric Schmidt: Congrats on all the progress. Maybe just a couple of quick questions on apitegromab approval time lines in the U.S. Team, I know it's not your facility, the Catalent facility, but are you aware of any field notes that were provided to Novo following the reinspection? And then I guess I'm also curious about the statement that you reiterated a couple of times now that approval may come at any time. I know that probably reflects the shared understanding and communication you have with the FDA, but just curious about the intent of that statement.

David Hallal: Thanks, Eric. I'll take both. And look, we were obviously very pleased today to have announced that the FDA accepted the BLA with 2 fill-finish facilities. And to be clear, it was a Class II resubmission with a PDUFA action date of September 30, which is sort of per protocol for manufacturing-related issues. So we anticipated that. And of course, as a reminder, we submitted that BLA in complete alignment with the FDA ahead of the reinspection of Catalent Indiana commencing.

Look, like since that in-person Type A meeting that we had back in November, all the way through the Type C meeting that we had in early March, we have just been really pleased with the high level of engagement from the agency and sort of the consistent pace and progress across this period of time. So look, what I would note about the reinspection is we were pleased with the timing -- we think the FDA has done their job. We believe that Novo has done their job. And per FDA guidelines, it's really now a 90-day period of time for the FDA headquarters to do their work and make a determination on the classification of the facility.

And I think, again, underscoring sort of the 2 paths to approval. I am gratified that our team has made massive amounts of progress with our second fill-finish facility. As noted today, all of the drug that is required for the FDA's review in this BLA at that second facility has been filed. And that product would be available in early Q3. So what you kind of see here, Eric, when we talk about we have to be ready at any time prior to and including September 30, is that let's just do a little bit of math together. The FDA is now in a 90-day period of time to determine classification of Catalent Indiana.

We have product that's going to be available in early Q3 from the second fill finish. That sort of brings you to something that is well advanced from the September 30 PDUFA date. And so we just know that we need to be prepared because many times, Class II resubmissions and action can be taken by the agency well ahead of that PDUFA date. And that's really what we mean about it any time prior to. We'll continue to work with the FDA collaboratively, and we continue to be really excited with their level of engagement, again, as I noted from our Type A meeting right through this moment today. And we'll keep you guys apprised on that progress.

Operator: [Operator Instructions] And the next question will come from Mani Foroohar with Leerink Partners.

Lili Nsongo: This is Lili Nsongo on for Mani. Congratulations on the progress. So now that the reinspection has occurred for the Catalent facility, how much risk -- or maybe I should say, how much confidence do you have in a successful non-experimentated classification for the facility? And how should we think about the capacity split between the 2 facilities in, say, the first year of launch?

David Hallal: I didn't get the second part of that question, Lili. On the first part, like as I noted to Eric, we feel like through this process, really since the sole approvability issue with our initial file was the general site inspection that the FDA had at Catalent Indiana. We know that Novo has been working really hard on that site with their initial remediation plan and then subsequently, their follow-up remediation with the FDA. And with a lot of engagement in Q1 with the FDA, as we previously noted, they had an early Q1 meeting that was then followed by a site visit and then subsequently in early Q2, the reinspection.

So I think we just need to respect that the FDA has really worked diligently, which we think is a rapid time line given the situation at Catalent Indiana to reinspect that facility. Based on their work, Novo has done their work, and now we want to be respectful of the time that the FDA will now take to make a classification decision. I think importantly, what Akshay and I were noting today is that we have a lot of drug vials from both facilities. And I think if any one of those 2 were to be the basis of the approval, each is going to have plenty of product to launch with. So I think that's great news.

I think one thing that maybe isn't lost on us is when you take a 90-day time line for up to a 90-day time line for the FDA to reclassify the Catalent Indiana facility. And then you think about an early Q3 timing of having product available commercially from the second fill-finish, there's definitely an opportunity also that our file could be approved with both fill-finish facilities. And I think that, that was one of the things that Akshay and I wanted to communicate as well. So a lot of optionality here, a lot of good news for patients, a lot of good news for the SMA community.

I am really grateful to our internal team at Scholar Rock for doing something pretty remarkable here with our second fill-finish facility, but also grateful to the FDA and Novo for the continued progress at Catalent Indiana, and we will keep you guys apprised at the updates across the board on our application.

Lili Nsongo: Great. The second part of my question was about commercial supply capacity split between the 2 facilities, which you also answered. So thank you.

Operator: And the next question will come from Tess Romero with JPMorgan.

Tessa Romero: I actually wanted to ask a commercial question this morning. Now that Itvisma is fully approved for ages older than 2 years old, how are you thinking about apitegromab being able to be used in combination with that therapy if and when you are approved?

David Hallal: Yes. Thank you, Tess. Keith can address how we're thinking about that opportunity. As you noted today, really important information from Cure SMA -- in general, we are prepared to launch apitegromab at any time between now and up to September 30. And I think the incredible work that Akshay has done with our team and engaging the FDA there's going to be a very significant opportunity to serve patients with SMA. So Keith, do you want to comment on really more than anything else, the dynamics in the marketplace and your preparations for launch?

Robert Keith Woods: Sure. Thanks for the question, Tess. I guess what I'd say, first of all, is we believe that regardless of the therapy, but any type of a therapy that an SMA patient can potentially benefit from an SMN-targeted therapy, we're agnostic as to which one the treating physician choose because we think that they go hand-in-hand along with our muscle-targeted therapy with apitegromab. Now specifically with Itvisma, in our SAPPHIRE study, we did not study patients that were previously on Zolgensma. As Akshay has noted several times, we are studying them in our OPAL study. And we also shared with you that we do have post Zolgensma patients in our EAP program. So there's some experience out there with it.

But as far as being able to utilize apitegromab with it, I think it's going to depend upon the label and where the policies come out with the payers.

Operator: And our next question will come from Cory Kasimov with Evercore.

Cory Kasimov: I wanted to ask you about the ongoing CHMP review. Coming out of the recent oral explanation, have the questions there have been largely similar to what the FDA has inquired about during its review and now just really boils down to CMC? Or are there other nonmanufacturing items that EU regulators are still trying to get their arms around?

David Hallal: Thanks, Cory. Akshay?

Akshay Vaishnaw: Yes. Thanks. So obviously, we don't get into the back and forth of regulatory reviews, FDA or EMA. The one thing I can say is that, that oral explanation that was scheduled led to a very good dialogue in advance of the meeting, and we were very happy with the pre-meeting alignment, which led to mutual agreement that there was no need for the meeting. And in fact, as a result, we obviously look forward to continued progress with the review and ultimately to launching the drug in Europe for children and adults with SMA.

As to the remaining time line, I commented in the formal remarks that the Catalent Indiana facility continues to support the application, and we look forward to a decision around midyear. But I think overall, the progress has been excellent.

David Hallal: And then just tagging on, Cory, just to tag on to Akshay, and Akshay has mentioned this multiple times. We've been having very good open dialogue with the European regulators about what's been happening here with the FDA and Catalent Indiana. So it's been very collaborative, like everything going on here has been a topic of discussion in Europe, and they've really been very flexible in working with us on timing.

Operator: And the next question will come from Michael Yee with UBS.

Michael Yee: Two questions, really quick. One is a follow-up, just in terms of the fill-finish facility, the second one. Can you remind me -- previously, I recall there was different stability testing and things that had to be completed, but it sounds like this site had sort of been pulled very much forward and was filed earlier, which was fantastic. And so it's the understanding that either of these sites can support approval by September 30, and that's why there's definitely increased confidence and there's not necessarily such a reliance on the Indiana site. And so I have that correct. And the second question is regarding a potential approval and indications.

I know previously, there has been some discussion around the broadness of the label, type 1 versus type 2 in different age groups, given the primary endpoint was on a certain age group definition. Can you just remind us about your confidence around general broadness of the label and how we should think about that?

David Hallal: Michael, great questions. I'll start on the 2 fill-finish facilities and then Akshay will take up the label. So yes, I mean, I guess at the end of the day, we have an enormous amount of confidence in our BLA as the headline news of what's changed from late last year to this year is really the fact that we have 2 fill-finish facilities in our BLA. One of those, we expect reclassification within a 90-day window from the closeout of the inspection.

And subsequently, in that second fill-finish facility, as you aptly noted, we have made massive amounts of progress in accelerating that where all of the drug that is required for the FDA's review and approval has been filed and that drug would be available commercially in early Q3. So when you kind of take that 90-day window, the up to 90-day window per FDA guidelines for the Catalent Indiana facility, when you look at that window of commercial apitegromab being available in early Q3 from the second facility, we are very confident in this window that we're talking about within Q3 and up to the September 30 PDUFA.

And I think that what I'm most gratified about is we try to live here at Scholar Rock by a deep commitment to the patients and families that are impacted by SMA. And I'm grateful to the team that we took it upon ourselves to say, okay, let's do better this time than we did last time. Let's not rely on a single fill-finish facility. Let's have multiple paths to get to that point where we can deliver the first ever targeted therapy to patients who are living with this disease and the families that are impacted by this disease, and I think we've been able to do that.

And tying -- dovetailing nicely into that is the question that you had on the label and our opportunity to serve a meaningful percentage of the community that is impacted by this disease. And I'll turn it over to Akshay to comment on that. Akshay?

Akshay Vaishnaw: Yes. Thanks. Mike, vis-a-vis the label, of course, it's premature to comment on the exact nature of the label before the regulatory deliberations are finalized here in Europe. What I would say is that, generally speaking, the regulators have taken a very important approach to the labels for SMA products. They look at the enrollment criteria of the pivotal studies, which exactly is the population. They look at the portability of the mechanism across the spectrum of disease, and they look at the unmet need. And so I feel like they've been very good with those principles to serve the community. We've been working with them. As you know, when we got the CRL, the draft label was completed.

The one outstanding issue was the manufacturing issue. And both in U.S. and Europe, all I can say is we've had constructive regulatory dialogue throughout the period last year and this year, and we look forward to launching this product for children and adults with SMA.

David Hallal: And Michael, Akshay and I would just note that as we previously have disclosed that where we were towards the tail end of our last BLA review, we were pleased. And that's where we picked up this new application is exactly where we were at the tail end of the last one on the label, and we look forward to continuing to work with regulators to bring us to the point of approval and delivering apitegromab to the community.

Operator: And the next question is going to come from Tazeen Ahmad with Bank of America.

Wesley Yon: This is Wesley on for Tazeen. Congrats to the team on all the progress really. I had a question on sort of the game plan going forward now that you have a PDUFA date in hand. So are there any sort of new types of discussions you can have with payers or other like commercial bodies now that apitegromab is officially under review? And is there any sort of new, I guess, strategies or ways that Keith and the commercial team are sort of laying out the groundwork for potential approval? Or is it just kind of just chugging along and doing what's been done already?

David Hallal: Well, Wesley, as I think you guys all know Keith very well. You would imagine as disappointed as we were to not launch late last year, we had to look at the opportunity that we had to prepare ourselves to be even better to serve the SMA community. That was our obligation. One such piece of that under Akshay and under Lisa Wyman and team was to make sure that this application was even stronger than the last one, and that's inclusive of now the 2 fill-finish facilities and 2 independent paths to approval. The other obligation that we made is to be better from a commercial perspective.

How do you use that time to make sure that you can meet the moment for the SMA community. And I think your question is a good one now with the September 30 PDUFA, but yet being ready for an approval at any time. And with that, I'll hand it over to Keith to talk about the things that he has been doing and what this means for him and the team. Keith?

Robert Keith Woods: Yes. Thanks, David. And Wesley, thanks for the question. What I can tell you is that joining the company 4 months prior to the PDUFA date, we were scrambling for that PDUFA date. We would have been able to launch successfully, but we have really been able to take advantage of the additional time that we have. Some specific examples that I've shared in the past, first of all, with payers, we are able to meet with the payers and with our medical team to really discuss apitegromab and the data. So those discussions are ongoing.

We've just been able to take them to a much broader range of payers and really deepen the discussions that we have with them specifically around this. Additionally, we built out how our site of care plans will be. I've shared with you before that we now, through our partners, have over 10,000 home infusion nurses available around the U.S. that would be able to provide apitegromab to patients shall they choose to go through home infusion. We've expanded our specialty pharmacy network so that no patient has to go to multiple specialty pharmacies to get their different meds that they may be on for SMA treatment, whether it's their SMN targeted therapy or that apitegromab.

And just we continue to really move forward with patient engagement activities. And that's through our program to really have patients demand better treatment for themselves with light takes muscle. And so I can tell you this. I want you to know that the team has been working very hard all the way through this delay, but we are clearly ready to launch now. So whatever that time frame that will be between now and September 30, I want you to know that the team will be ready to be out there the next day, and we will have supply in the channel very rapidly after approval.

Operator: And the next question will be coming from Marc Frahm with TD Cowen.

Marc Frahm: A lot has been asked already on the PDUFA and apitegromab itself. Maybe just looking at the subcu version. I mean you mentioned you have that data in hand. And once you get the approval for the IV formulation, you'll look to meet with the FDA to discuss it. Just what are the kind of key issues you think you need answers from the FDA on? And kind of what are the range of time lines for when you think you might be able to kind of launch that product depending upon the outcome of those discussions?

David Hallal: Thanks, Marc. Akshay?

Akshay Vaishnaw: Yes. Thanks, Marc. So I would say there are no issues as such. These things are a matter of just alignment with regulators as to what the optimum path forward to bring another innovation to SMA patients and in this case, it would be subcutaneous apitegromab. The Phase I data were excellent, showing a very good bioavailability and pharmacodynamic overlap between 2 routes of administration. And what we have to do now is to share those data following the approval and align on the path forward in terms of any further development that we needed. So that could be PK/PD data and consideration of any additional safety or efficacy.

However, from a safety perspective, obviously, the exposure is maximized with IV apitegromab. And so with the very large database we have in hand already from the studies we've done, we feel very good about safety via additional routes of administration. And so we just want to get on and have those conversations and finalize the path. Once we've done that, obviously, we'll guide you on the time line, premature to speak to that in advance of those conversations.

Marc Frahm: Okay. And if I can squeeze in also just on the FORGE trial. Just can you kind of walk through what's different about that trial or maybe the supporting data that apitegromab has been able to generate relative to the efforts that Roche had in FSHD and which ultimately, as of a few weeks ago, they disclosed did not lead to moving into pivotal development.

David Hallal: Yes. 3 or 4 points here. Number one, we're obviously very proud of the innovations that have occurred at Scholar Rock with our leading anti-myostatin pipeline. It still remains apitegromab, the only validated anti-myostatin antibody make it through Phase III and delivered the kind of risk and benefit profile that we saw in the Phase III with the SAPPHIRE study in SMA. Whilst we await that approval, obviously, many others are interested in this target. Roche and Chugai are world-leading company. It was sad to see that antibody drop out. We've never really seen any Phase I data or the FLExDUX4 mouse model data from the Chugai-Roche antibody.

So we don't quite know the nature of those data, and we await to see how strong they were. We know our data apart from the positive Phase III study, of course, we have very nice data in the FLExDUX4 mouse model with an anti-myostatin approach showing increase in muscle mass and talk and additional function. We know that there are, within FSHD normal fibers that can be boosted by means of an anti-myostatin approach. We know other clinical trials in FSHD that have shown increase in muscle mass and function. So we're very encouraged by our data and our diligence.

And finally, we believe the Phase II design is different from the Roche study, specifically the inclusion/exclusion criteria and the severity of the disease that we're enrolling relative to what they enroll, which appears to be quite advanced. And based on our diligence with the experts, we decided because of input from them to go towards the milder end in terms of the Roche scores with patients with established disease where we felt we could still show benefit. And so we remain confident with our validated asset going into that Phase II study and look forward to kicking off very soon.

Operator: And the next question will come from Geoff Meacham with Citigroup.

Geoffrey Meacham: I had another commercial kind of reimbursement question. Just given the range of options in SMA today, how are you guys thinking about incentivizing switches or maybe deploying a more novel outcomes-based pricing strategy just to help the early stages of the launch? And are the strategies different when you look to the EU and the early launch in Germany versus the U.S. launch?

David Hallal: Thanks, Geoff. I think as Keith noted, a cornerstone of our sort of campaign thus far around the disease itself has been an acknowledgment, and we see that the community gets it, that this disease is -- the hallmark is not only the motor neuron, but the resulting muscle atrophy. And so all of this innovation over the last 10 years has been on motor neuron survival and motor neuron health. And this has been needed innovation for the community. And yet, as Keith noted, nearly all patients are wanting their muscle atrophy to be addressed. And this will be the first and only muscle-targeted therapy that's approved. So we don't necessarily really think about switches, Keith, right?

We really think about no matter what you choose to do for motor neuron health, we applaud. And we're going to deliver something that addresses the organ that is the principal organ affected by this disease is the muscle. And that's what's been left behind over these 10 years of innovation that we're finally able to address. And putting that into practice, I know, Keith, has been the cornerstone of what you guys have been talking about with the community, and I'll let you take it from here.

Robert Keith Woods: Yes. No, Geoff, we're really not going to be focused on any type of switches because what we've shared before is that in our own market research with treating physicians, we know that 3/4 of them have already said that they believe dual modality is the future standard of care for treatment in SMA. So that's directly targeting the motor neuron and directly targeting the muscle -- so we believe that, that will be how this is viewed. And then additionally, from a payer point of view, we did share the data that Cure SMA shared with us in the prepared remarks with roughly 1/3 of patients already receiving more than one SMN targeted therapy.

It just continues to drive home the unmet medical need that exists with these SMA patients. But as David just referenced, the principal organ that's impacted in this disease is the muscle, and we look forward to bringing forward the world's first muscle-targeted therapy.

Operator: And the next question is going to come from Amy Li with Jefferies Company.

Amy Li: David, congrats on all the progress. Just wanted to get a sense of the next steps and time lines for the Catalent site. Based on feedback from the FDA after the reinspection and the Novo closeout meeting, do you expect a Form 483 related to reinspection? And does the speed of your BLA filing acceptance, which was around 30 days compared to the standard 60 days, indicate any FDA urgency or prioritization? And then finally, on the second manufacturing side, I just wanted to clarify, are you maintaining it primarily as a hedge against Catalent? Or is there a potential for approval of both sites?

David Hallal: Yes. Thanks, Amy. I'll take that last point first. As we noted when Catalent Indiana was acquired by Novo, we knew that Novo was acquiring that facility really for its own internal purposes, and they would have this transition phase into moving "customers" out because they're not a CDMO. That's not their business model. And so all along, we've recognized that we would want to have and would require to have an additional or more than one fill-finish facilities that are outside of Catalent. So all of that, right, was part of our plan even prior to the Form 483 observations that the FDA had in their general site inspection last year.

So I think it's important to note that we see this second fill-finish facility is absolutely vital for all of our global demand. Now we also see Catalent is important. We have drug vial there. We would anticipate that they would be part of our supply chain. And in due time, they're going to phase -- we would phase them out if they're going to be phasing us out. So more than anything else, we see them both as being important. And yet we do think having 2 independent paths to an approval under this BLA is a very significant enhancement to our BLA in 2026 versus the one that we had last year in 2025.

And we also think timing is really good. You note the FDA's urgency and how they've been working expeditiously with us. We do think that was really anchored by a very constructive in-person Type A meeting in Q4 that Akshay led with our team down there, and we are just grateful that the FDA has continued to show a sense of urgency and understanding the needs of the community. So more than anything else, we see a world in which apitegromab gets approved with one or the other or both, and we think that, that's a wonderful spot to be in.

We'll let the FDA do their work on the review of the second fill-finish facility and the data that has been generated by us on that second fill-finish facility with drugs becoming available in early Q3. And we'll also let the FDA do their work expeditiously and thoroughly on their inspection as well as the inspectors concluded that reinspection recently. So we're excited for what the future brings and more than anything else, I think you guys can see these time lines of the 2 facilities have really come pretty much together. And I think that's a key takeaway to recognize.

Operator: And the next question will come from Gary Nachman with Canaccord.

Gary Nachman: My congrats as well on all the progress. So David, just to follow-on the last point you were making there. If everything ends up being fine with Catalent with the classification, are you still considering pulling the second fill-finish facility from the BLA to simplify it for the FDA? Or you'll just keep it in there regardless to have that better supply chain, even if it would potentially delay the approval and push it out a little bit? And then just a follow-up. Someone asked before on pricing, but just, I guess, to ask it a little differently. Is there a strategy that would make more sense of launching first in Germany or in the U.S.?

Or regardless, it would just be one global price and you're not anticipating any MFN issues. So pricing isn't really a consideration in terms of how you'll stagger the launches?

David Hallal: Yes. These are great -- really great questions, Gary. I'll just make one comment and then hand it over to Akshay. When Akshay and I hosted a call, late in Q1 on the resubmission of our BLA, we did actually talk about the alignment that we've had with the FDA, the dialogue that we had with the FDA throughout Q1 about the submission with both fill-finish plants and the optionality that, that really provided us. And so Akshay, do you want to comment on that? And like if there is a meaningful difference in time line, the flexibility that we may or may not have here?

Akshay Vaishnaw: Yes. I mean just repeating what you said, I think this has been so important to all the progress that's occurred that there's been very constructive collaborative approach between us and the FDA and indeed with the EMA throughout this whole period. And based on that, we submitted both facilities in the BLA with the full support and alignment. And the most straightforward thing is Catlin Indiana is reclassified, is in compliance, and we can start getting drug out of the pending approval. And the second facility would be withdrawn from the BLA. However, given all the constructive approach that's occurred with the FDA, we'll be guided by them.

And in the long run, we clearly want redundancy in the supply chain. And so we look forward to bringing on an additional finish sites. So I think all the options are open for us and the really great position we're in now to serve patients is that by September 30, we're going to be approved by one or the other facility. But in the long run, of course, we'll have established in supply chain.

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

Robert Keith Woods: Yes. So first of all, Gary, as I mentioned in the prepared remarks, the team is ready to launch here in the U.S., but also the team is built in Germany. And so if you think about -- is there a preference for one before the other? No. We want to get this across the finish line, both in the U.S. and in Europe. You mentioned how does this overall affect pricing. We go out with our list price here in the U.S. We go out with our list price in Germany and in Europe. Remember, Germany is the only place that we can proactively promote right after EMA approval.

And so you're promoting and selling at your list price while you go through the AMNOG process and you go through the reimbursement and establishing that price. So it really wouldn't have an impact. And the bottom line is we're going to be prepared to serve patients in whichever market comes first, and there shouldn't be a substantial impact to our ability to price, negotiate in an overall impact on most favorite nations because we won't be at a point right away that we would even trip the cause of most favorite nations.

Operator: And our next question will come from Kripa Devarakonda with Truist.

Alexander Xenakis: This is Alex on for Kripa. Congrats on the great news today. We have one about the Roche discontinuation of Emugrobart in FSHD. I wanted to know have you seen any uptick in investigator interest in working with apitegromab for your FSHD trial.

David Hallal: Yes. Thanks for that, Alex. So the whole neuromuscular space is very excited about the apitegromab program after in SMA. You're right, the intensity of interest increases. Obviously, everyone is looking forward to the approval in SMA. But the neurology world looks with anticipation towards what a validated anti-myostatin approach like apitegromab can do not just in FSHD, but in a range of diseases. And so we're looking forward to the start of the study, which will be very soon now, Phase II study in FSHD and with additional indications to follow where we'll study this drug. But you're absolutely right. There is plenty of interest and very constructive input as we think about triaging through these indications.

Operator: Thank you. This does conclude the question-and-answer session and also concludes today's conference call. Thank you for your participation, and you may now disconnect.

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