Debiopharm International SA, in partnership with Solid Biosciences, recently announced the initiation of pre-clinical animal studies of Alisporivir in the treatment of Duchenne Muscular Dystrophy (DMD).
DMD is a severe form of muscular dystrophy caused by a mutation in the Dystrophin gene, located in the X chromosome, which results in the absence or abnormal production of the dystrophin protein. Since this protein is an essential component of muscle tissue its loss or deficiency leads to muscle degeneration, mainly in the muscle of legs and pelvis, and with progression towards other muscles, including cardiac muscle. As such, DMD-patients begin with impaired walking abilities and progressively loose complete upper body function. As a result, they suffer from cardiac dysfunction, which is DMD-patients’ ultimate cause of death.
This new collaboration will investigate Alisporivir in pre-clinical models of DMD, establishing the proof of concept that may allow Debiopharm to initiate clinical research into this new therapeutic strategy for DMD.
Alisporivir is the first in a new class of drugs called cyclophilin inhibitors whose action is potentially beneficial for multiple conditions, such as Hepatitis C virus (HCV). In fact, in HCV patients Alisporivir exhibited an acceptable safety profile. More recently its action was also demonstrated in muscular diseases, including preclinical models of DMD but also Limb-Girdle and Ullrich-Bethlem myopathy. The new partnership will confirm Alisporivirs’ efficacy in a new animal model mimicking human DMD.
Thierry Mauvernay, Delegate of the Board of Debiopharm Group commented, “We are delighted to start development in muscular dystrophy. We are very pleased to be able to work with Solid Biosciences on this program, combining our deep understanding of Alisporivir with their great expertise and network in DMD and their focus on finding solutions for these young patients suffering from this terrible disease.”
Ilan Ganot, founder and CEO of Solid Biosciences added, “We, at Solid Biosciences, are proud to be partnering with Debiopharm. Alisporivir has exciting efficacy potential in DMD and we look forward to put to work our expertise in this condition to help advance Alisporivir to patients afflicted with DMD. I am particularly grateful to Bob McDonald and Ed Mascioli whose personal and professional commitment to DMD patients is reflected in this relationship.”