Summary of Scholar Rock Holding FY Conference Call (December 04, 2025) Company Overview - **Company**: Scholar Rock Holding (NasdaqGS:SRRK) - **Focus**: Development of therapies for spinal muscular atrophy (SMA) and other neuromuscular disorders Key Points Industry and Company Developments - **Significant Transformation**: 2025 marked a year of transformation for Scholar Rock, particularly following the positive readout of the Sapphire study in October 2024, which was pivotal for the company's future activities [4][5] - **BLA Submission**: The company filed its Biologics License Application (BLA) with the FDA in January 2025 based on the Sapphire study results [4] - **FDA Priority Review**: In April 2025, the FDA accepted the BLA under priority review due to the high unmet need in SMA, marking the first muscle-targeted therapeutic for the condition [5] Leadership Changes - **CEO Transition**: Jay Backstrom stepped down as CEO in April 2025, with David Hallal taking over. A new executive team was assembled to focus on scaling and growth [6][7] Clinical Trials and Efficacy - **Sapphire Study Results**: The phase 3 trial demonstrated that patients receiving apitegromab showed a gain in motor function compared to those on placebo, achieving a statistically significant p-value of 0.019 [12] - **Response Rates**: Approximately one-third of patients on apitegromab achieved a three-point improvement on the Hammersmith Motor Function Scale, compared to 12% on placebo, indicating a significant therapeutic benefit [13] Regulatory Outlook - **Complete Response Letter (CRL)**: The company received a CRL in September 2025 due to compliance issues at the fill/finish facility, which is now owned by Novo Nordisk. The drug substance facility was not a concern [9][18] - **Type A Meeting with FDA**: A constructive Type A meeting was held in November 2025, where the FDA indicated a willingness to expedite reinspection once Novo Nordisk is ready [20] Future Plans - **Resubmission Timeline**: Scholar Rock expects to resubmit the BLA and launch in the U.S. in 2026, pending resolution of the facility issues [20] - **Second Indication**: The company plans to announce a second indication for apitegromab in a rare neuromuscular disorder in 2026 [9] Market and Payer Strategy - **Payer Discussions**: The company is confident in securing coverage for apitegromab, emphasizing its ability to stabilize and improve motor function in SMA patients compared to existing therapies [33] - **Market Opportunity**: There are approximately 7,000 patients in the U.S. who have received SMN-targeted therapies, with a global patient population of around 35,000, indicating a significant market opportunity for apitegromab [35] Community Engagement - **Building Relationships**: The company is focusing on strengthening relationships with the SMA community, including healthcare providers and patients, to enhance disease awareness and education [32] Additional Insights - **Operational Challenges**: The delay in the approval process has allowed the company to better prepare for launch, including establishing relationships with payers and refining its reimbursement processes [30] - **Importance of Muscle Health**: The company emphasizes that optimal outcomes for SMA patients require addressing both motor neuron survival and muscle health, which apitegromab aims to achieve [31]