Scholar Rock Holding Corporation

Ladies and gentlemen, thank you for standing by. Welcome to the Scholar Rock fourth quarter 2025 financial results and business update call. At this time, all participants are on a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you would need to press star 11 on your telephone. You will then hear an automated message advising your hand is raised. And to withdraw your question, please press star 11 again. Please be advised that today's conference is being recorded. I would like now to turn the conference over to Scholar Rock. Please go ahead.

Good morning. I'm Laura Ekus, Vice President of Investor Relations at Scholar Rock. With me today are David Halal, Chairman and Chief Executive Officer, Akshay Vashna, President of R&D, Keith Woods, Chief Operating Officer, and Vikas Binha, 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'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. 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 fourth quarter and full year 2025 earnings call. Scholarock 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. Our highest priority is to bring Opidigromab to the SMA community as quickly as possible. We remain relentless on behalf of patients, and we are grateful that important progress continues to be made at a steady and rapid pace. Let me briefly summarize the key events that have occurred since our constructive and collaborative in-person Type A meeting in November. First, a week following our Type A meeting, the FDA issued a warning letter to Catalyn, Indiana. Next, Novo Nordisk rapidly responded to the FDA by mid-December. Then, following Novo's response, FDA reached out prior to the holidays to schedule an early Q1 meeting. That meeting has since taken place, and importantly, at that meeting, the FDA had no additional requests to Novo's remediation plan. And most recently, following the meeting with Novo, we were encouraged that the FDA sent a field team to Catalan, Indiana. At the conclusion of the visit, the FDA once again did not have any additional requests to Novo's remediation plan and stated to Novo that it intends to conduct a site reinspection following routine manufacturing activities which has since resumed in late February. The cadence of activity since our Type A meeting reflects the shared understanding between us, the FDA, and Novo of the high unmet need in the SMA community and a shared sense of urgency to bring epitomab to children and adults living with SMA as rapidly as possible. We are pleased with FDA's continued level of engagement, and we expect this momentum to continue. Our team is prepared to resubmit the epigramab BLA following a successful FDA re-inspection of the Catalan Indiana facility. We are reaffirming our guidance of BLA resubmission and U.S. launch following approval in 2026. Also, I am pleased that progress with a second fill finish facility is moving quickly. to build redundancy into our supply chain. Engineering runs at the facility are now underway, with additional manufacturing runs to follow. We anticipate filing a supplemental VLA for the second filer later this year. As we advance the regulatory process for Opitigromab toward approval for patients with SMA in the U.S., Our MAA review continues in Europe, and we expect a decision from the European Medicines Agency in mid-2026. With anticipated regulatory approvals in the U.S. and Europe this year, I would like to now turn to our Scholarock commercial launch preparations. In the U.S., our team is deployed in the field and is educating potential prescribers and payers on the unmet need in SMA and the importance of targeting muscle, the principal organ affected in SMA, while also broadening and deepening relationships with the community. In Europe, we are building momentum with launch readiness activities and engaging with the SMA community. We continue to plan for a launch in the second half of the year, beginning with Germany. Keith will discuss the substantial progress we are making with commercial preparation and our disease awareness initiatives shortly. We know it is not a matter of if, but when, epidegromab will be approved for children and adults 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 Opidicromab should have access to Opidicromab. This is indeed what we know well and what we do well, and we are confident in the significant opportunity that we have to serve patients with SMA. We are ready now more than ever to usher in the next era of innovation for the SMA community. I would like to now turn to the progress we are making in advancing our world-leading anti-myostatin pipeline. Enrollment and dosing continue in our Phase II OPAL study, Evaluating Epidigromab in Infants and Toddlers with SMA. Our IMD for epitigromab and FSHD is cleared, and we are on track to initiate a robust randomized placebo-controlled Phase II study later this year. With regards to our subcutaneous formulation of epitigromab, we share the promising results of a Phase I study comparing sub-Q and IV epitigromab in January. We expect to share our clinical and regulatory strategy for the program later this year. And finally, we continue to enroll in dose participants in our phase one study for our highly innovative SRK439 myostatin inhibitor. We expect to have top line data from this study in the second half of this year. Akshay will discuss these programs in greater detail shortly. Turning now to our balance sheet, we were pleased to have added, we are pleased to have ended 2025 with $368 million in cash and cash equivalents. This includes $60.4 million from the exercise of warrants that were set to expire on December 31st. We continue to strengthen our financial position to drive our commercial and R&D priorities and this morning We are pleased to announce that we have secured a new debt facility for up to $550 million, which Vikas will discuss later in the call. 2026 will be a transformative year for Scholarock. We are ready to resubmit our BLA for epitagromab at any moment. Our U.S. commercial team is working with urgency to prepare the market for the launch of the world's first and only muscle-targeted therapy for children and adults living with SMA. Beyond the U.S., the build-out of our 50-country operating platform is underway in Europe, with other regions and countries to follow. And our highly innovative, world-leading anti-myostatin pipeline with epitagromab and SRK439 is progressing with strong momentum. The opportunity ahead of us to serve patients with SMA and additional rare and severe neuromuscular diseases is significant. We remain steadfast in our strategy, confident in the determination of our team, and energized by the transformative potential of Apidigromab and our broader pipeline. The road ahead is one of purpose, progress, and extraordinary possibility. And with that, I'll now turn the call over to Akshay for an R&D update. Akshay?