Sarepta reports completion of confirmatory trial commitment for PMO therapies

Sarepta (SRPT) announced the completion of the confirmatory trial commitment for its ultra-rare disease PMO therapies AMONDYS 45 and VYONDYS 53: While the ESSENCE study did not achieve statistical significance on its primary endpoint, results indicate positive and encouraging trends favoring therapy at 96 weeks. Sarepta reports that the study was impacted by the COVID-19 pandemic and, when COVID-impacted data is excluded, meaningful treatment effect is seen on the primary endpoint ESSENCE supported favorable safety profile of AMONDYS 45 and VYONDYS 53. The company intends to schedule a meeting with FDA to discuss path to a traditional approval based on the positive risk-benefits of the therapies from the results of the ESSENCE study and significant positive multi-year real-world evidence.