Scholar Rock Holding Reports Q2 2026 Results: Full Earnings Call Transcript

Scholar Rock Holding (NASDAQ: SRRK ) released second-quarter financial results and hosted an earnings call on Thursday. Read the complete transcript below. This transcript is brought to you APIs. For real-time access to our entire catalog, please visit for a consultation.

S. launch of apitegromab by September 30th, with two fill-finish facilities ready for approval. The company ended Q2 2026 with $492 million in cash and has increased commercial investments while maintaining consistent operating expenses. Scholar Rock is advancing its anti-myostatin pipeline, including the Phase 2 OPAL trial for SMA and the FORGE study for FSHD.

FDA and EMA interactions are positive, with significant progress on the second fill-finish facility, which could independently support apitegromab approval. S. and preparing for the European market, focusing initially on Germany and planning for a 50-country operating platform. , Vice President, Investor Relations Good morning.

I'm Laura Ekas, Vice President of Investor Relations at Scholar Rock Holding. With me today are David Hallal, Board Chair and Chief Executive Officer, Akshay, President of R&D, Keith Woods, Chief Operating Officer, and Vikas Sinha, Chief Financial Officer. During today's call, David will provide introductory remarks and a business update. Akshay will review our R&D progress, Keith will provide an update on our commercial readiness activities, and Vikas will provide a financial update.

We will then 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 Scholar Rock Holding's expectations, plans, and prospects that constitute forward-looking statements for the purposes of safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any future date. I encourage you to go to the Investors and Media section of our website for our most up-to-date SEC statements and filings.

With that, I'd like to turn the call over to David. David Hallal, Chairman and Chief Executive Officer Thank you, Laura, and good morning. Thanks to everyone for joining our second quarter earnings call today. Scholar Rock Holding is operating from a position of strength as we enter a defining period for our company and for the SMA community across the business.

S. launch of apitegromab this quarter, advance our apitegromab MAA in Europe to approval and prepare for launch in Germany, build momentum across our world-leading anti-myostatin platform, and maintain the financial strength to support our ambitions. Most importantly, we are on the threshold of bringing the world's first muscle-targeted therapy to children and adults living with SMA as we advance apitegromab through the final stages of the FDA regulatory process. S.

As a reminder, the sole approvability issue for apitegromab noted in the complete response letter that we received on our priority review PDUFA date last September was related to observations identified at a routine general site inspection of the Catalent, Indiana fill-finish facility, owned and operated by Novo Nordisk. Since our constructive and collaborative Type A meeting in November, the cadence of activities has reflected the shared understanding between us and the agency of the high unmet need in the SMA community and a shared sense of urgency to bring apitegromab to children and adults with SMA as rapidly as possible.

We are grateful for the agency's sustained level of engagement and for our ongoing dialogue, including our March 3rd Type C meeting where we discussed the accelerated progress that we had made at our second fill-finish facility and the agreed-upon data package to facilitate the FDA review of the second fill-finish facility, in alignment with FDA guidance. From this discussion, we submitted the apitegromab BLA on March 30th with two fill-finish facilities, both Catalent, Indiana and our second fill-finish facility. Our BLA was accepted in April with a PDUFA date of September 30th. Agency review of our application is progressing well.

Importantly, we have significant optionality with two independent paths to approval—either through Catalent, Indiana or through our second fill-finish facility, or both—whichever is determined to be the most rapid. As it relates to Catalent, Indiana, the FDA inspection classification following the April 2026 general site inspection is pending. We continue to be very pleased by the progress made at our second fill-finish facility. Importantly, the data package for the FDA's review of the second facility has been submitted and the review is progressing.

Underscoring our operational excellence at our second site, we now have more vials available from this facility than we do from Catalent, Indiana. Notably, vials from both Catalent, Indiana and our second facility are now on site at our third-party provider, awaiting packaging and labeling upon approval. S. regulatory process, we are well aware that patients are awaiting the world's first muscle-targeted therapy for this devastating disease.

, our team continues to advance our launch preparations across all key functions, ensuring we are prepared to support patients, caregivers, and prescribers from day one. Our team is ready to launch apitegromab at any time prior to and including our September 30th PDUFA date. Keith will discuss our commercial preparations in greater detail shortly. , we continue to look forward to serving children and adults living with SMA in Europe.

I would now like to provide an update on where we stand in the European regulatory process. The apitegromab MAA includes only the Catalent, Indiana fill-finish facility, and the EMA is awaiting the FDA inspection classification for that facility. In parallel, we are engaging with European regulators with regards to potential inclusion of our second fill-finish facility in the apitegromab application. Importantly, this facility has had recent successful site inspections by the FDA and the EMA.

We are grateful for the EMA's continued level of engagement and we look forward to providing updated guidance on the potential timing of a CHMP opinion upon alignment with the European regulators. Turning to our launch preparations in Europe, we are executing our commercial playbook, building momentum with launch readiness activities, and engaging with the SMA community. We are planning for an initial launch in Germany, with additional countries and regions to follow as we build out our planned 50-country operating platform. S.

and Europe, and we continue to work with urgency to reach the 35,000 people with SMA around the world who have received an SMN-targeted therapy. Turning now to our world-leading anti-myostatin pipeline, we continue to make meaningful progress with our key clinical programs. We have robust enrollment in our Phase 2 OPAL study evaluating apitegromab in infants and toddlers with SMA. We initiated our randomized Phase 2 FORGE study in patients with FSHD.

S. and European regulators on the development path of our high-concentration subcutaneous formulation of apitegromab, which we will do once we have regulatory approvals, and enrollment and dosing is proceeding very well in our Phase 1 healthy volunteer study for SRK-439, our novel high-potency anti-myostatin antibody. Akshay will discuss these programs in greater detail shortly. Turning now to the balance sheet, we were very pleased to have ended the second quarter of 2026 with $492 million in cash, cash equivalents, and marketable securities.

This cash balance includes net proceeds of $63 million from our ATM program during the second quarter. Vikas will provide more detail later in the call. In June we had the opportunity to be with the SMA physician and patient community at the Cure SMA Annual Meeting in Orlando. I was able to sit down with several SMA-treating physicians and with a number of patients and their families, and during our time with them we heard some very moving stories about the impact apitegromab has had on children and adults who are participating in our OLE and EAP programs.

Our team at Scholar Rock Holding is so inspired by these patients and by their families, and we look forward to ushering in the next phase of innovation for this community. With that, I'll now turn the call over to Akshay for a closer look at our R&D initiatives. Akshay Thank you, David, and good morning, everybody. As David shared, we're very pleased that the apitegromab BLA continues to progress through FDA review with two independent paths to approval, and we remain on track for a decision by the September 30 PDUFA date.

The FDA inspection classification for the Catalent Indiana facility is pending. We had anticipated the classification in late July, within 90 days following inspection completion based on the Agency's guidelines. We remain engaged with the FDA and will provide updates as appropriate. As it relates to the second fill-finish facility, we're very pleased to report that all necessary data have now been submitted to FDA and the Agency's review of those data is progressing well.

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 throughout. The Agency has appreciated the high unmet need in the SMA community, and we look forward to the final steps in the process. Turning now to Europe, we're pleased with the EMA's review of the apitegromab Marketing Authorization Application and with their continued level of engagement. As we have previously noted, approval in Europe is dependent on FDA clearance of the Catalent Indiana facility, which is currently the sole fill-finish site included in our MAA.

The EMA continues to await the FDA's inspection classification for this facility. Additionally, we're engaging with the EMA regarding the process to include our second fill-finish facility in our apitegromab application. Importantly, this facility is in good standing with European regulators. We will provide an update on timing upon alignment with the EMA.

Turning to our pipeline, let me start with the Phase 2 OPAL trial. We continue to have robust enrollment in the study, which is evaluating apitegromab in infants and toddlers with SMA under the age of two. 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 treatment. 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.

Notably, the rate at which the study is enrolling underscores the significant unmet need and the potential for apitegromab in the youngest of SMA patients. Turning now to our next indication for apitegromab, facioscapulohumeral muscular dystrophy, or FSHD. FSHD is a rare, devastating neuromuscular disease. It is one of the most prevalent inherited muscular dystrophies and there are no approved therapies.

To date, we've prioritized FSHD as the next indication for apitegromab for three key reasons. First, the significant unmet need, since approximately 20% of patients become wheelchair dependent. Second, the compelling preclinical data from the gold standard FLExDUX4 mouse model that provides mechanistic rationale for apitegromab in FSHD. And finally, 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 to announce today that we've initiated our Phase 2 study called FORGE, which is a randomized, double-blind, placebo-controlled trial with a sample size of 60 patients. We're also advancing two additional therapeutic programs in our world-leading anti-myostatin pipeline: a high-concentration subcutaneous formulation of apitegromab and SRK-439.

In our subcutaneous apitegromab program, we showed some very exciting data in January from a Phase 1 study which demonstrated that subcutaneous 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 engagement with US and European regulators later this year following approval of apitegromab. Turning now to SRK-439, our high-potency, high-affinity, subcutaneously administered myostatin inhibitor, we're very excited about this program and dosing in our Phase 1 healthy volunteer study is progressing well.

We expect to have topline data from the 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 our world-leading anti-myostatin pipeline across a range of rare, devastating neuromuscular diseases. I'll now turn the call over to Keith to discuss our commercial launch preparations. Keith Woods, Chief Operating Officer Thanks, Akshay, and good morning, everyone.

As David noted, with the potential FDA approval of apitegromab for children and adults with SMA by September 30th, our US commercial organization is launch-ready across all key functions, and we are prepared to support patients, caregivers, and prescribers from day one. S. Despite approximately 78% of children and adults living with SMA receiving an SMN-targeted therapy, 95% of patients continue to experience persistent and progressive muscle atrophy that limits both function and independence. S.

have received two 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 for children and adults with SMA. S. field team continues to broaden their reach, focusing on disease education and awareness around the unmet medical need while also reinforcing a broader understanding of SMA as a disease that consists of both the motor neuron and the muscle, the principal organ impacted by the disease.

We are also expanding our reach and frequency across approximately 140 SMA treatment centers, 2,600 prescribing physicians, and their multidisciplinary care teams. Through these engagements, our field team is establishing case flows on a center-by-center basis to ensure that upon approval, we are well positioned to support the SMA treatment centers once an apitegromab treatment decision has been made. This past quarter, we have also strengthened our Scholar Rock Holding Supports Patient Services program. The Scholar Rock Holding Support team is fully trained and prepared to provide comprehensive, individualized support to patients and caregivers.

At launch, eligible patients and their families will be able to access this program to understand insurance coverage, identify available financial and copay assistance, and navigate treatment logistics. Turning now to patient engagement, our connections with the SMA community remain strong. This past June, we had a significant presence at the Cure SMA Annual Meeting in Orlando. Scholar Rock Holding served as a presenting sponsor of the meeting, and throughout the week our teams engaged with healthcare professionals and members of the SMA patient community.

I was very pleased that the Scholar Rock Holding Symposium for Healthcare Professionals entitled Expert Perspective on the Evolving Management of Spinal Muscular Atrophy was one of the most attended expert sessions during the meeting. Equally, our patient symposium Muscle: There's More to the Story in SMA was attended by hundreds of SMA patients, caregivers, and families, and during this session we sought their perspective on needs and priorities for people living with SMA. Every interaction we had during this meeting reinforces our determination and further strengthens our commitment to serve patients and families. S.

reimbursement, our Market Access team continues to advance discussions with national and key regional payers as well as Medicare and Medicaid with the goal of achieving broad reimbursement for eligible patients after approval. S. immediately upon FDA approval. Turning now to Europe, we are advancing our launch preparations with a particular focus on Germany as we work with the EMA on the next steps for our application.