Santhera's puldysa flunks late-stage Duchenne study, pulls Euro application

• Santhera Pharmaceuticals (OTCPK:SPHDF -28.8%) has stopped a Phase 3 trial of Duchenne muscular dystrophy (DMD) drug Puldysa (idebenone) after data from an interim analysis concluded the study was unlikely to meet its primary endpoint.
• The setback prompted the company to withdraw the European marketing authorization application and end the global development program for Puldysa.
• The interim analysis was based on the primary endpoint of the study, the change of forced vital capacity % predicted (FVC%p) from baseline to 18 months of treatment. The outcome revealed that the probability of reaching the primary endpoint at the end of the study is too small to merit the continuation of the study.
• Last year, Santhera refiled for European Medicines Agency approval of idebenone. The application followed an earlier, drawn-out effort to get idebenone to market in DMD that ended with an EMA rejection in 2017 and a failed appeal early the following year.
• Now the company will be focused on vamorolone, nonhormonal steroid modulator in DMD and lonodelestat in lung diseases such as cystic fibrosis. Vamorolone is in a Phase 3 trial with top-line data in Q2 of 2021. Lonodelestat is in Phase 1b in cystic fibrosis.
• Santhera will continue preclinical work on its gene therapy project.
• https://seekingalpha.com/news/3620187-santheras-puldysa-flunks-late-stage-dmd-study-withdraws-european-approval-application