Sarepta Therapeutics (SRPT) Update / Briefing Transcript

Summary of Sarepta Therapeutics (SRPT) Conference Call - June 16, 2025 Company Overview - Company: Sarepta Therapeutics (SRPT) - Focus: Development of gene therapies for Duchenne muscular dystrophy (DMD), specifically the treatment Alevitus Key Points Industry Context - Duchenne Muscular Dystrophy (DMD): A severe genetic disorder characterized by progressive muscle degeneration and weakness - Gene Therapy: AAV-mediated gene therapy is used for treatment, with known risks including liver injury Safety Update on Alevitus - Incident Reported: A non-ambulatory patient treated with Alevitus passed away due to acute liver failure (ALF) [3] - Patient Demographics: Over 900 patients have been treated with Alevitus over seven years, with a focus on non-ambulatory individuals [3] - Safety Measures: Following the incident, Sarepta has paused the non-ambulatory study and suspended commercial shipping of Alevitus for non-ambulatory patients until safety protocols are amended [5][16] Proposed Safety Enhancements - Immunosuppression Regimen: Introduction of sirolimus to mitigate liver injury risks associated with AAV-mediated gene therapy [4][8] - Preclinical Data: Studies indicate sirolimus can significantly reduce elevated liver biomarkers and liver injury risk [9][11] - Regimen Impact: Sirolimus does not negatively affect Alevitus expression in key tissues [12] Regulatory and Operational Considerations - FDA Engagement: Sarepta is seeking feedback from the FDA regarding the proposed immunosuppression protocol [5][33] - Revenue Guidance: The company has suspended revenue guidance due to the uncertainty surrounding the non-ambulatory patient treatment timeline [16][52] Patient and Market Implications - Patient Trust: The company aims to rebuild trust within the patient community through transparent communication about risks and benefits [70] - Market Position: Despite the tragic events, the company believes there are no immediate next-gen gene therapies available that could replace Alevitus [29] Future Directions - Clinical Trials: Plans to amend protocols for ongoing studies and potentially expand immunosuppression strategies to other patient populations [66][98] - Financial Discipline: The company will assess its cost structure to maintain financial stability while prioritizing patient safety [75][89] Additional Insights - Ambulatory vs. Non-Ambulatory Patients: The safety signal for ALF has only been observed in non-ambulatory patients, with no similar signals in ambulatory patients [38][61] - Dosing Strategy: Weight-based dosing is consistent across patients, with no correlation found between dosing and liver injury risk [42][92] Conclusion Sarepta Therapeutics is taking significant steps to enhance the safety profile of Alevitus following serious adverse events. The company is focused on implementing a robust immunosuppression strategy while maintaining open communication with regulatory bodies and the patient community. Future updates will be provided as the situation evolves and further data becomes available.