Santhera Says It Will Discontinue Development of DMD Drug Puldysa and Restructure

Rare Daily Staff

Santhera Pharmaceuticals said it will discontinue development of Puldysa in patients with the neuromuscular condition Duchenne muscular dystrophy after an interim analysis of its phase 3 study concluded that the study was unlikely to meet its primary endpoint.

Santhera said it will discontinue the study of Puldysa in DMD patients in respiratory decline and who receive concomitant glucocorticoid treatment, withdraw the European marketing authorization application and end the global development program for Puldysa.

The company intends to initiate a restructuring plan for the business with a focus on retaining key functions for bringing DMD drug candidate vamorolone to patients and execute on its other pipeline programs.

Based on the now completed interim analysis, which tested for efficacy, the independent data safety monitoring board recommended the study be discontinued due to futility. The interim analysis was based on the primary endpoint of the study, the change of forced vital capacity percent predicted from baseline to 18 months of treatment. The outcome revealed that the probability of reaching the primary endpoint at the end of the study was too small to merit the continuation of the study. There were no safety concerns noted by the DSMB.

Participants who are enrolled in the study will discontinue study medication and complete the study’s follow-up evaluations.

“While this is obviously not the outcome we expected, all our efforts in DMD will now be focused on progressing the promising drug candidate vamorolone which we recently licensed from ReveraGen to its next inflection point, the readout of 6-month topline data from the pivotal VISION-DMD study planned for the second quarter of 2021,” said Dario Eklund, CEO of Santhera.”

Photo: Dario Eklund, CEO of Santhera