Scholar Rock Holding Corporation

Ladies and gentlemen, thank you for standing by and welcome to Scholar Rock's first quarter financial results and business update call. At this time, all participants are in a 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 1-1 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 1-1 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 Rashi Knopfinger, Vice President of Corporate Affairs and Investor Relations at Scholar Rock. With me today are David Halal, Chief Executive Officer, Akshay Vasnau, President of RMD, Keith Woods, Chief Operating Officer, and Vikas Sinha, Chief Financial Officer. For those of you participating via conference call, the accompanying slides can be accessed by going to the events section of the investors page on our website. During today's call, as outlined on slide two, David will provide introductory remarks and provide a general business update. Akshay will review our clinical and regulatory progress. Keith will provide an update on our commercial readiness activities. And Vikas will provide commentary on our company financials and a summary of our 2025 priorities. And then we will 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 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?

Thanks, Rashmi, and good morning. Thanks to everyone for joining us on today's call. 2025 is off to a strong start at Scholarock. In the first quarter, we made significant progress against our top priority of bringing epinegrumab to patients and families living with and suffering from SMA in the US, Europe, and around the world. We were very pleased with the positive phase three SAPPHIRE trial, where we showed a statistically significant and clinically meaningful improvement in motor function as measured by the primary endpoint, the gold standard Hammersmith scale. And in Q1, to underscore the body of evidence for epitigromab for patients with SMA, we were gratified that our BLA was granted priority review by the FDA with a September 22nd PDUPA date. The team at Scholarock is working urgently to prepare for our U.S. commercial launch, which will be the first country in a series of launches over the coming years. I am confident under Keith's leadership we will assemble and deploy an experienced, talented, and patient-centric team committed to the SMA community. Along with Keith, Akshay, and Vikas, we now have the responsibility to bolster Scholaroc's capabilities as we advance our mission to deliver epidegromab to patients with SMA. This is what we know well and what we do well. We join Scholaroc at a time of great strength and opportunity as we scale for the next phase of growth to a commercial stage, fully integrated global biopharmaceutical company. Globally, nearly 35,000 patients with SMA have received SMN-targeted therapies, and at Scholarock, we are working with urgency to prepare for our global launch, which we anticipate will commence in the U.S. in Q3. We are also beginning to set the table to serve patients with SMA in Europe, Asia Pacific, and Latin America. Our ambition at Scholarock is that any patient with SMA that can benefit from epidegromab should have access to epidegromab. I am confident that the global opportunity with epidegromab 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. While we are very focused on our large opportunity to serve patients with SMA, we are continuing to evaluate expanding the study of epitigromab into other rare, severe, and debilitating neuromuscular disorders. Ache will touch upon this shortly. Now I'd like to turn to our exploratory cardiometabolic program, the EMBRAZE proof of concept study. Our effort here is to understand the role we may play in the treatment of obesity. We are the world leaders in myostatin biology and know well that GLP-1s have brought needed innovation to patients with obesity and cardiometabolic disorders. While the benefits outweigh the risks, muscle wasting and lean mass loss is something that the medical community is trying to address. The EMBRACE study is our initial effort in the cardiometabolic space and will provide insights that will guide us moving forward. We remain on track to share top-line results in June. Our achievements in 2025 to date underpin the opportunity before us to bring the potentially life-transforming benefits of epidegromab to patients and families with SMA. We are focused on delivering for patients with Akshay leading efforts in collaborating with U.S. and EU regulators. Key, applying a similar approach from Vivgar and MG to the commercial opportunity for epidechromab. And because, focused on disciplined capital allocation to fuel near and long-term growth. With that, I'll turn the call over to Akshay to provide a more detailed update on our R&D progress. Akshay?