Scholar Rock Holding Corporation

Good morning, ladies and gentlemen, and welcome to this Caller Rocks Second Quarter 2025 Business Update Conference Call. At this time, all lines are in a listen-only mode. Following the presentation, we will conduct a question-and-answer session. If you would like to ask a question during this time, simply press star, followed by the number 1 on your telephone keypad. If you would like to withdraw your question, press star 1 again. This call is being recorded on Wednesday, August 6, 2025. I would like to turn the conference over to Scholar Rock. Please go ahead.

Good morning. I'm Rashmi Nofsinger, Vice President of Corporate Affairs and Investor Relations at Scholar Rock. With me today are David Hillal, Chairman and Chief Executive Officer, Akshay Vashnau, President of R&D, 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 for Opidigrimab, and Vikas will provide commentary on our financials. 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 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 our Q2 update call today. This is an exciting time of great strength and opportunity at Scholarock. We are scaling for the next phase of growth as a commercial stage, fully integrated global biopharmaceutical company. Our three core priorities drive our vision of becoming a global biotech powerhouse. First, epitogrammab regulatory approvals, and following those approvals, the U.S. launch of epitogrammab for children and adults with SMA, followed by a series of country launches in the coming years, starting in Europe with Germany next year. Second, expand epidigromab into additional rare, severe, and debilitating neuromuscular diseases. And third, disciplined capital allocation to support our high-value commercial and development initiatives. With respect to our ongoing regulatory processes, We are working collaboratively with the FDA and European Medicines Agency. We are also urgently preparing for our U.S. commercial launch as our BLA has been accepted under priority review with a target action date of September 22nd. As you are aware, GXP inspections are part of the standard FDA review process, including those relating to preapproval inspections, clinical trial site inspections, and manufacturing site inspections. These inspections often result in observations requiring responses within the review cycle. The FDA has conducted a full set of inspections and, as part of this process, noted observations at two of our CDMOs. On Friday, August 1, another pharmaceutical company disclosed observations as part of an FDA general site inspection at their filler, Catalan, Indiana, which was recently acquired by Novo Nordisk. Our fill finish for Opidigromab is conducted at the same site. The general site inspection of the facility was not specific to Opidigromab. Novo Nordisk submitted a robust and comprehensive response earlier this week to the observations noted by the FDA. For the other CDMO observations were received at the conclusion of a pre-licensing inspection and a comprehensive response will be submitted within the next week. We continue to work collaboratively with the agency. Importantly, the FDA recently completed our late cycle meeting following both site inspections. We are encouraged by the dialogue with the agency at the late cycle meeting where the FDA indicated that they are working towards completing the review of our BLA by our September 22nd PDUFA date. Earlier this year, we also filed our MAA for epidigremab in the EU and we continue to work with the European Medicines Agency and expect a potential approval near mid-2026. We are planning for Germany to be our first European country launch with an ambition to reach patients with SMA across all of Europe, followed by additional countries in Asia Pacific and Latin America over time. The global opportunity with Epidigromab and SMA alone offers the potential for many years of sustainable growth through the end of this decade and into the next. Along with Keith, Akshay, and Vikas, and the other leaders at the company, we have bolstered Scholaroc's capabilities as we advance our mission to deliver epitogromab to children and adults with SMA. This is, indeed, what we know well and what we do well. In addition to the large opportunity to serve patients with SMA, we will continue to expand our pipeline by planning additional opportunities for epitogromab for children and adults suffering with additional rare, severe, and debilitating neuromuscular disorders, which Akshay will discuss in more detail shortly. Importantly, to grow Scholarock, we are taking a thoughtful, deliberate approach to capital allocation by staging our investments along with our commercial progress in serving the SMA community. Q2 has been exceptionally productive. In the quarter, When Keith joined our team as our chief operating officer, he brought a proven track record of building and leading teams to deliver highly successful rare disease global launches in the neuromuscular therapeutic area, including most recently with VivGuard. Under Keith's leadership, we have assembled an exceptionally experienced, talented, and patient-centric field team committed to the SMA community. Impressively, over just a few months, the team is on board, trained, and deployed, and are ready to deliver apidigromab to the SMA community pending approval in September. Despite currently available treatments that have been approved over the past 10 years, we are acutely aware that muscle strength and motor function are among the top unmet needs in SMA, which we believe can be addressed with the potential approval of epitogramab, the world's first and only muscle targeted therapy to deliver statistically significant and clinically meaningful improvements in motor function in a pivotal phase three trial. I would like to now turn briefly to the readout of the positive phase two EMBRAZE proof of concept study in Q2. The goal of EMBRAZE was to understand the role of targeting myostatin in the treatment of patients with obesity. We are pleased that the EMBRACE study met the primary endpoint with patients on terzepatide. Epidigromab increased lean mass preservation by greater than 54% compared to terzepatide alone with a p-value equal to 0.001 with an encouraging safety profile. We are very pleased that the highly selective anti-myostatin approach from our innovative platform continues to deliver. While we remain focused on advancing epitagromab in clinical development for additional rare, severe, and debilitating neuromuscular disorders, EMBRAZE raises the exciting possibility to partner our potent and selective approach to targeting myostatin. As we communicated at EMBRAZE data readout, in addition to SRK439, we have a number of earlier stage research assets. both anti-myostatin antibodies and the fusion of those with GLPs, which we think have the potential to be meaningful therapeutic candidates in the future. As we look forward, we remain very focused on the nearly 35,000 patients with SMA globally that have received SMN targeted therapies. While we anticipate the global launch will commence in the U.S. in Q3, pending regulatory approvals, we are also making preparations to serve children and adults with SMA in Europe, Asia Pacific, and Latin America. Our ambition at Scholarock is that globally, any patient with SMA who can benefit from epitogramab should have access to epitogramab. At this point, I'll turn the call over to Akshay to provide a more detailed update on our R&D progress. Akshay?