Summary of Scholar Rock Holding Conference Call Company Overview - **Company**: Scholar Rock Holding (NasdaqGS:SRRK) - **Event**: Jefferies London Healthcare Conference - **Date**: November 18, 2025 Key Points Industry and Product Focus - Scholar Rock is focused on developing therapies for spinal muscular atrophy (SMA) using their lead monoclonal antibody, ipilimumab, which targets myostatin [2][3] Regulatory Challenges - A positive Phase III study for ipilimumab led to a Biologics License Application (BLA) submission in January 2025, but a Complete Response Letter (CRL) was received on September 22, 2025, due to compliance issues at the GMP manufacturing facility [3][4] - The compliance issues were related to the Novo Catalent facility in Bloomington, Indiana, which received an Official Action Indicated (OAI) notice [4][10] - The FDA and Scholar Rock had a Type A meeting to discuss the path forward, with Novo presenting a robust remediation plan [4][10][12] Remediation and Inspection Timeline - Novo reported that approximately 90% of the remediation work has been completed, focusing on quality management systems [10][11][16] - The FDA is expected to communicate their impressions of the remediation progress soon, with a reinspection planned for early 2026 [12][21] - If the reinspection is successful, Scholar Rock anticipates resubmitting the BLA promptly, with hopes for approval in 2026 [13][30] Market Potential and Launch Strategy - There are over 11,000 SMA patients in the U.S., with two-thirds currently on SMN-targeted therapies [45] - Scholar Rock expects initial patients to be those already on SMN therapies, as ipilimumab is anticipated to be an add-on therapy [46][47] - The company plans to launch ipilimumab as the first monthly IV infusion for SMA, which may change the treatment paradigm [49] Pricing and Market Expansion - The market opportunity in SMA is significant, with Scholar Rock planning to price their product in the U.S. first before expanding to Europe, Japan, and South America [52][54] - The company is aware of the challenges in negotiating prices outside the U.S. but sees the necessity of entering these markets [53] Financial Position and Runway - Scholar Rock ended Q3 2025 with approximately $369.6 million in cash and raised an additional $91.7 million through an ATM and $50 million in loans [58] - The cash runway extends into 2027, assuming no revenue from ipilimumab and not accounting for potential monetization of a Priority Review Voucher (PRV) [58][59] Future Developments - Scholar Rock is working on a NextGen product targeting myostatin, with Phase I data expected next year [55] - There is significant interest in their anti-mystatin portfolio from companies in the obesity space, indicating potential for future collaborations [57] Additional Insights - The meeting highlighted the urgency and collaborative nature of the discussions with the FDA, emphasizing the importance of addressing the unmet need in SMA [23][25] - The company is committed to maintaining a high-quality manufacturing process and is optimistic about overcoming current challenges [18][19]