Debiopharm, Solid Biosciences to explore use of Alisporivir in muscular dystrophy

The companies will commence pre-clinical animal studies in Duchenne Muscular Dystrophy (DMD). The collaboration aims to expand existing preclinical proof of concept data in additional DMD animal models.

Alisporivir is a non-immunosuppressive cyclophilin inhibitor, with several possible indications. It has already been tested in over 1500 patients during clinical development for hepatitis C.

It demonstrated an acceptable safety profile. Alisporivir can avoid mitochondrial-dependent muscular cell death.

Under the collaboration, the companies will confirm the efficacy of the compound in a new model of DMD that resembles the human pathology and to allow the start of clinical development in the indication.

Solid Biosciences founder and CEO Ilan Ganot said: "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."

The absence of dystrophin, a structural protein critical for protecting skeletal and cardiac muscle against contraction-induced injury, causes DMD. It is a severe X-linked form of muscular dystrophy that affects one out of every 3500 males.

Debiopharm said patients with DMD lose the ability to walk in the early to mid teens and progress to full loss of upper body function.