Scholar Rock Holding Corporation Common Stock 2026 Q2 Earnings Call

I'm Laura Ekus, Vice President of Investor Relations at Scholar Rock. With me today are David Hallal, Board Chair and Chief Executive Officer, Akshay Vaishnaw, 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. 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'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? Thank you, Laura. Good morning.

Thanks to everyone for joining our second quarter earnings call. Today, Scholar Rock is operating from a position of strength as we enter a defining period for our company and for the SMA community. Across the business, we are executing with focus, discipline, and urgency as we drive towards the U.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. 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. Let me now provide additional detail on the ongoing review of our apitegromab BLA in the U.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. As we near the final step in the U.S. regulatory process, we are well aware that patients are awaiting the world's first muscle-targeted therapy for this devastating disease.

Turning now to our commercial readiness in the U.S., 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. In addition to the U.S., 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 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. 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. We know it is not a matter of if, but when apitegromab will be approved for children and adults with SMA in both the U.S. and Europe. 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 II OPAL study evaluating apitegromab in infants and toddlers with SMA. We initiated our randomized phase II FORGE study in patients with FSHD. We are ready to engage with U.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. Enrollment and dosing is proceeding very well in our phase I 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 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. 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 ONYX and EAP programs. Our team at Scholar Rock is so inspired by these patients and by their families. 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. We remain on track for a decision by the September 30th PDUFA date. The FDA inspection classification for Catalent, Indiana 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. 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.