Santhera ends development of idebenone

Santhera Pharmaceuticals (Pratteln, Switzerland) has announced the discontinuation of its Phase III SIDEROS study with idebenone in patients with Duchenne muscular dystrophy (DMD) who are in respiratory decline and receive concomitant glucocorticoid treatment after an independent Data and Safety Monitoring Board (DSMB) said that the study won't probably meet its primary endpoint. 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. There were no safety concerns noted by the DSMB.

Santhera said, it will discontinue the study, withdraw the European marketing authorisation application and end the global development program for idebenone under the brand name 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 pipeline programs lonodelestat for cystic fibrosis and other lung diseases and its discovery-stage gene therapy approach for congenital muscular dystrophy.

Idebenone is a synthetic short-chain benzoquinone and a substrate for the enzyme NAD(P)H:quinone oxidoreductase.

Dario Eklund, Chief Executive Officer of Santhera, commented. “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.”