Scholar Rock (NasdaqGS:SRRK) FY Conference Transcript

Scholar Rock (NasdaqGS:SRRK) FY Conference Summary Company Overview - Company: Scholar Rock - Event: 44th Annual J.P. Morgan Healthcare Conference - Date: January 12, 2026 - Key Speaker: David Hallal, Chairman and CEO Core Industry and Company Insights - Scholar Rock is positioned as a leader in myostatin biology, focusing on treatments for rare neuromuscular disorders, particularly Spinal Muscular Atrophy (SMA) [2][3] - The company has developed Apitegromab, the first myostatin inhibitor to successfully complete a pivotal phase III study, demonstrating clinically meaningful benefits for SMA patients [3][10] - Apitegromab is expected to receive U.S. and European approvals in 2026, with Germany being the initial launch country in Europe [3][5] Key Developments and Milestones - Scholar Rock plans to initiate dosing for a new indication of Apitegromab for Facioscapulohumeral Muscular Dystrophy (FSHD) later in 2026 [4][19] - The company has a strong cash position of approximately $365 million, providing a runway into 2027 to support commercial and R&D initiatives [25] - The phase III trial data showed that about one-third of patients receiving Apitegromab alongside SMN-targeted therapies had significant improvements in motor function, compared to only 12.5% in the placebo group [12] Market Opportunity - The global market for SMN-targeted therapies is projected to reach $5 billion in 2025, with Apitegromab representing a $2 billion-plus opportunity specifically for SMA [18][27] - The company aims to build a 50-country operating platform to serve SMA patients globally, with a focus on the U.S., Europe, and significant markets in Asia-Pacific and Latin America [6][7] Regulatory and Commercial Strategy - Scholar Rock is preparing for a BLA resubmission and U.S. launch following a complete response letter from the FDA, which was related to site inspection issues rather than clinical efficacy [13][15] - The company is actively engaging with national and regional payers to educate them on the unmet needs in SMA and the potential benefits of Apitegromab [16][17] - The U.S. commercial team is focused on disease education and building relationships with the SMA community to ensure successful market entry [16] Future Directions - Scholar Rock is committed to expanding the reach of Apitegromab to ensure no SMA patient is left behind, including those under two years of age [19][26] - The company is also exploring additional neuromuscular diseases for which Apitegromab may provide therapeutic benefits [22][23] - Ongoing development of SRK-439, a more potent myostatin inhibitor, is also a priority, with plans to share data from healthy volunteer studies in late 2026 [24] Financial Outlook - The company has maintained financial discipline while investing in high-value programs, with a focus on preparing for the commercial launch of Apitegromab [25][41] - There is an expectation of strong demand for Apitegromab upon approval, with plans for non-dilutive financing options to support operational needs [41] Conclusion - Scholar Rock is poised for a transformative year in 2026, with significant advancements in the treatment of SMA and a robust pipeline aimed at addressing various neuromuscular disorders [26]