Santhera to discontinue Phase 3 SIDEROS study and development of Puldysa® in Duchenne Muscular Dystrophy (DMD) and focus on vamorolone.
Original Press Release > Pratteln, Switzerland, October 6, 2020
Santhera Pharmaceuticals announces the discontinuation of its Phase 3 SIDEROS study with Puldysa® (idebenone) in patients with Duchenne muscular dystrophy (DMD) who are in respiratory decline and receive concomitant glucocorticoid treatment. Data from an interim analysis conducted by the independent Data and Safety Monitoring Board (DSMB) concluded that the study was unlikely to meet its primary endpoint. Consequently, Santhera will discontinue the study, withdraw the European marketing authorization application and end the global development program for Puldysa. The Company intends to initiate a restructuring plan to retain key functions for bringing DMD drug candidate vamorolone to patients and execute on its other pipeline programs.
Based on the now completed interim analysis tested for efficacy, the DSMB has recommended the SIDEROS study be discontinued due to futility. The interim analysis was based on the study’s primary endpoint, 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 study’s continuation. There were no safety concerns noted by the DSMB.
Santhera will stop the SIDEROS trial.
Santhera will stop the SIDEROS trial (including extension), and participants who are enrolled in the study will discontinue study medication and complete the study’s follow-up evaluations. Furthermore, following up on the recommendation from the DSMB, Santhera will discuss the impact of ending the SIDEROS study on ongoing expanded access programs with the corresponding regulatory bodies.
Dario Eklund, Chief Executive Officer of Santhera “We would like to thank the patients and the families, as well as investigators and medical professionals, who participated in the SIDEROS study. Without their contributions, we would not be able to advance DMD research. 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.”
In connection with this decision, Santhera intends to start a restructuring process, aligning its operations to focus on progressing vamorolone for DMD, lonodelestat for cystic fibrosis and other lung diseases discovery-stage gene therapy approach for congenital muscular dystrophy.
About SIDEROS
SIDEROS was a clinical trial in DMD, a double-blind randomized placebo-controlled Phase 3 study evaluating the efficacy of idebenone in delaying the loss of respiratory function in patients with DMD. Patients on any stable glucocorticoid treatment scheme and irrespective of the underlying dystrophin mutation or ambulatory status were randomized to receive oral idebenone (900 mg/day three times a day) or placebo for 18 months. The trial’s primary endpoint estimates the treatment difference in FVC%p (forced vital capacity % predicted). Read more about Idebenone.
About Santhera
Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focused on developing and commercializing innovative medicines for rare neuromuscular and pulmonary diseases with a high unmet medical need. Santhera has an exclusive license for all indications worldwide to vamorolone, a first-in-class anti-inflammatory drug candidate with a novel mode of action, currently investigated in a pivotal study in patients with DMD as an alternative to standard corticosteroids. The clinical-stage pipeline also includes lonodelestat (POL6014) to treat cystic fibrosis (CF) and other neutrophilic pulmonary diseases and omigapil and an exploratory gene therapy approach targeting congenital muscular dystrophies. Santhera out-licensed ex-North American rights to its first approved product, Raxone® (idebenone), to treat Leber’s hereditary optic neuropathy (LHON) to Chiesi Group. For further information, please visit www.santhera.com. La Force DMD Blog /worldwide rights of vamorolone for DMD
