Overview
- Sarepta reported that the nine-year ESSENCE study of Amondys 45 (casimersen) and Vyondys 53 (golodirsen) failed its primary endpoint despite numerical trends favoring treatment.
- The randomized trial enrolled 225 boys aged 6 to 13 and assessed function over 96 weeks, including a four-stair climb where gains did not reach statistical significance.
- Shares fell roughly 36% in premarket trading on Tuesday, and Reuters noted the stock is down about 80% for the year.
- The company cited COVID-19 disruptions to participation and data collection and pointed to post-hoc analyses suggesting slower disease progression, as analysts warned scrutiny from regulators, payers and physicians will intensify.
- The setback follows July’s brief market withdrawal of Elevidys after three deaths from acute liver failure, even as Sarepta posted stronger-than-expected Q3 revenue of $399.4 million.