Scholar Rock Holding Corporation

Good morning, ladies and gentlemen, and welcome to Scholar Rock's third quarter 2025 conference call. At this time, all lines are in listen-only mode. Following the presentation, we will conduct a question and answer session. To ask a question during the session, you will need to press star 11 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 11 again. This call is being recorded on Friday, November 14th, 2025. I would now like to turn the conference over to Scholar Rock. Please go ahead.

Good morning. I'm Laura Ikas, Vice President of Investor Relations at Scholar Rock. With me today are David Hillel, Chairman and Chief Executive Officer, Akshay Vashna, President of R&D, Keith Wood, Chief Operating Officer, and Vika Finha, Chief Financial Officer. For those of you participating via conference call, the accompanying slides can be accessed in the Events section on the Investors page of our website. During today's call, as outlined on slide two, 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 VCAS 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 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 is representing our views as of any future date. I encourage you to go to the Investors in 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, and good morning. Thanks to everyone for joining our third quarter earnings call today. In April, when I was appointed CEO, after eight years in the board chairman role. And on the same day, we brought in Akshay, Keith, and Vikas. We were confident that Scholarock was positioned to be the next great global biotech powerhouse. We based this on several factors. First, our conviction that the global opportunity with Epidigromab and SMA alone offers the potential for many years of sustainable growth that will power our company through the end of this decade and into the next. Second, as world leaders in myostatin biology, our ability to deliver transformative therapies to patients suffering with additional rare, severe, and debilitating neuromuscular disorders. And third, leveraging our innovative platform to advance our novel subcutaneously administered myostatin inhibitor, SRK439. When we joined Scholarock, the most significant milestone ahead was the September 22nd PDUFA date for epitigromab and SMA, which had been granted priority review. Our BLA was supported by robust data demonstrating epitigromab's efficacy and safety for children and adults living with SMA. Based upon our 188 patient prospective randomized double-blind placebo-controlled multinational phase three trial. This trial showed a statistically significant and clinically meaningful benefit in motor function as measured by the gold standard Hammersmith motor function scale for SMA. While we were disappointed to receive a complete response letter on September 22nd, we were pleased that the strength of our phase three data was reflected in the FDA's review of our BLA and that the sole approvability issue referenced in the CRL was the status of our third-party, still-finished facility in Bloomington, Indiana, which is owned by Novo Nordisk. We know that it is not a matter of if, but when a Pitogramab will be approved in the U.S. for patients living with SMA. We are emboldened by the commitment we have made to the more than 35,000 patients globally living with SMA who have received an SMN-targeted therapy. We are working expeditiously to deliver on our ambition that globally, any patient with SMA who can benefit from Opidigromab should have access to Opidigromab. And now more than ever, we are confident in the significant opportunity that we have ahead of us to serve the SMA community as we work with determination to bring this important medicine to children and adults with SMA. This is indeed what we know well and what we do well. I would like to now provide a regulatory update on Epidicromab. We had our Type A meeting with the FDA on Wednesday. We are grateful to the agency for their full participation, particularly in the context of a government shutdown. The meeting was in person and included the relevant leaders and decision makers from the agency, including the neurology division and the Office of Compliance. Our team was joined by Kenneth Hobby, president of CURE SMA, and representatives from Novo Nordisk. We were encouraged by the meeting. It was constructive and collaborative. It was clear that there is a shared understanding of the high unmet need for the SMA community and a shared sense of urgency to bring a Pitigremab to children and adults with this disease. Novo Nordisk detailed the progress they have made in implementing their remediation plan at the Bloomington facility and affirmed that they expect the facility to be ready for re-inspection by the end of this year. We discuss the path forward and await the final minutes of the meeting. We will continue to work closely with the FDA and anticipate resubmitting the BLA and U.S. launch following approval of epidegromab for children and adults with SMA in 2026. I'd like to now turn to adding redundancy to our supply chain. When Novo Nordisk purchased the Bloomington site in December of 2024, they planned to internalize the plant for their own products. In light of that, Scholarock implemented a plan to add an additional U.S.-based fill finish facility. Now with the OAI classification, Scholarock has accelerated our timelines for an additional biler. We have selected a world-class commercial facility that has a proven track record and has successfully completed recent site inspections, including with the FDA and EMA. As you know, one of the bottlenecks to rapidly adding a new biler is securing commercial capacity. This can be a lengthy process. Importantly, we have secured commercial capacity commencing in the first quarter of 2026, and tech transfer is now underway. We anticipate submitting an SBLA for this facility later in 2026. In summary, we will continue to work with urgency to bring this important medicine to the SMA community. We look forward to providing clarity on resubmission timelines as soon as we are able. In addition to the large opportunity we have to serve children and adults with SMA, we continue to strategically advance our pipeline. This includes the Phase II OPAL study, progressing epitogromab and a second rare debilitating neuromuscular disorder, as well as advancing SRK439 into the clinic. Akshay will provide additional detail on these activities shortly. Importantly, to reach our ambitions, I am pleased we opportunistically strengthened our balance sheet during the third quarter, and we continue to operate with a tight financial plan, which Vikas will discuss later in the call. This plan is aligned to thoughtful, strategic investments to drive long-term value creation. We remain confident in the strength of our strategy, the grit of our team, and the transformative potential of epidegromab and our pipeline. The regulatory challenges we face today are temporary, but the opportunities ahead to serve patients are extraordinary. With that, I'll turn the call over to Akshay to provide more detailed update on our R&D progress. Akshay?