Debiopharm International SA (Debiopharm), part of Debiopharm Group, and Solid Biosciences, LLC, a Cambridge based biotech company, announced the start of preclinical studies of Alisporivir, in the rare disease Duchenne muscular dystrophy (DMD).
The objective of the collaboration is to reinforce existing preclinical proof of concept data in additional DMD animal models. This preclinical work, fully funded by the Lausanne-based company, will then lead to Debiopharm initiating the clinical development of this new and promising therapeutic for DMD at its own expense in the near future.
Alisporivir is a non-immunosuppressive cyclophilin inhibitor, with multiple potential indications and has already been tested in more than 1500 patients during clinical development for hepatitis C, demonstrating an acceptable safety profile. Alisporivir is able to prevent mitochondrial-dependent muscular cell death and has shown promising activity in preclinical models of DMD but also Limb-Girdle and Ullrich-Bethlem myopathy. The aim of this collaboration is to confirm the efficacy of the compound in a new model of DMD that more closely resembles the human pathology and to enable the start of clinical development in this indication.
"We are delighted to start development in muscular dystrophy. We are very pleased to be able to work with Solid Biosciences on this programme, 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," said Thierry Mauvernay, delegate of the Board of Debiopharm Group.
"We, at Solid Biosciences, are proud to be partnering with Debiopharm", said Ilan Ganot, founder and CEO of Solid Biosciences.
"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."
DMD is a severe X-linked form of muscular dystrophy that affects approximately 1 out of every 3500 males. It is caused by the absence of dystrophin, a structural protein critical for protecting skeletal and cardiac muscle against contraction-induced injury. Characterized by progressive and pervasive muscular degeneration, patients with DMD lose the ability to walk in the early to mid teens and progress to full loss of upper body function. DMD patients suffer from progressive cardiopulmonary complications, which are the primary cause of mortality, typically in the early to mid 20's.